Regulatory News

The latest regulatory news, consultation documents, and adopted guidelines and legislation, mainly from the European Commission, the European Medicines Agency and the Food and Drug Administration.

Measures in the veterinary field to promote 3Rs measures described in the European Pharmacopoeia, applicable to veterinary vaccines
The European Medicines Agency have published: Scientific guideline: Recommendation to marketing authorisation holders, highlighting recent measures in the veterinary field to promote reduction, refinement and replacement (3Rs) measures described in the European Pharmacopoeia – Applicable to veterinary vaccines from 01/01/2017, adopted.

In accordance with the European Convention for the Protection of Vertebrate Animals Used for Experimental and Other Scientific Purposes (Council of Europe), tests performed in animals must be carried out in such a way as to use the minimum number of animals and to cause the least pain, suffering, distress or lasting harm. The European Pharmacopeia (Ph. Eur.) has, over the years, applied these principles by implementing alternative tests and assays that reduce, refine and replace animal use. Therefore, in order to comply with the provisions of Directive 2010/63/EU and Ph.Eur and to secure an undisrupted supply of medicinal products to the European Market, MAHs should take all necessary actions to introduce 3Rs Ph. Eur. methods including submission of variations to marketing authorisations as appropriate. More information is available on the EMA’s website.

Measures in the veterinary field to promote 3Rs measures described in the European Pharmacopoeia, applicable to human vaccines
The European Medicines Agency have published: Scientific guideline: Recommendation to marketing authorisation holders, highlighting recent measures in the veterinary field to promote replacement, reduction, and refinement (3Rs) measures described in the European Pharmacopoeia – Applicable to human vaccines from 01/01/2018, adopted.

In accordance with the European Convention for the Protection of Vertebrate Animals Used for Experimental and Other Scientific Purposes (Council of Europe), tests performed in animals must be carried out in such a way as to use the minimum number of animals and to cause the least pain, suffering, distress or lasting harm. The European Pharmacopeia (Ph.Eur.) has, over the years, applied these principles by implementing alternative tests and assays that replace, reduce and refine animal use. Therefore, in order to comply with the provisions of Directive 2010/63/EU and Ph.Eur and to secure an undisrupted supply of medicinal products to the European Market, MAHs should take all necessary actions to introduce 3Rs Ph. Eur. methods including submission of variations to marketing authorisations as appropriate. More information is available on the EMA’s website.

Developing drugs for treatment of Duchenne muscular dystrophy and related dystrophinopathies
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment.”

This guidance addresses FDA’s current thinking regarding clinical development programs and trial designs for drugs to support an indication for the treatment of one or more dystrophinopathies: Duchenne muscular dystrophy (DMD) and related dystrophinopathies including Becker muscular dystrophy (BMD), DMD-associated dilated cardiomyopathy (DCM), and symptomatic carrier states in females. More information is available on the FDA’s website.

Developing drugs for treatment of early Alzheimer’s disease
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Early Alzheimer’s Disease: Developing Drugs for Treatment.”

This guidance is intended to assist sponsors in the clinical development of drugs for the treatment of the stages of sporadic Alzheimer’s disease (AD) that occur before the onset of overt dementia. This guidance revises the draft guidance for industry entitled “Alzheimer’s Disease: Developing Drugs for the Treatment of Early Stage Disease” issued February 8, 2013. More information is available on the Federal Register’s website.

Consultation Deadline: 17th May, 2018

Developing drugs for treatment of amyotrophic lateral sclerosis
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment.”

The purpose of this guidance is to assist sponsors in the clinical development of drugs for the treatment of amyotrophic lateral sclerosis (ALS). Specifically, it addresses FDA’s current thinking regarding the clinical development program and clinical trial designs for drugs to support an indication for the treatment of ALS. This guidance addresses the clinical development of drugs intended to treat the main neuromuscular aspects of ALS (i.e., muscle weakness and its direct consequences, including shortened survival). More information is available on the Federal Register’s website.

Consultation Deadline: 17th April, 2018

Drugs for treatment of partial onset seizures
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Drugs for Treatment of Partial Onset Seizures: Full Extrapolation of Efficacy from Adults to Pediatric Patients 4 Years of Age and Older.”

The draft guidance provides recommendations to sponsors on the clinical development of drugs for the treatment of partial onset seizures (POS) in pediatric patients. Specifically, it addresses FDA’s thinking regarding clinical development programs that can support extrapolation of evidence of effectiveness in treatment of POS in adults to pediatric patients 4 years of age and older. More information is available on the Federal Register’s website.

Consultation Deadline: 17th April, 2018

Regulatory classification of pharmaceutical co-crystals
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Regulatory Classification of Pharmaceutical Co-Crystals.”

This guidance provides applicants planning to submit new drug applications (NDAs) and abbreviated new drug applications (ANDAs) with information on the appropriate regulatory classification of pharmaceutical co-crystal solid-state forms. This guidance also provides information about the data that applicants should submit to support the appropriate classification of a co-crystal as well as the regulatory implications of the classification. More information is available on the FDA’s website.

European Commission Safety Features Q&A document updated
The European Commission has published an updated version, Version 9, of the Questions and Answers document on the Safety Features for Medicinal Products for Human Use.

Version 9 of the document, dated February 2018, supersedes Version 8. Q&As 1.19, 2.17, 2.18, 2.19, 2.20, 2.21, 3.5, 4.4, 5.6, 5.7, 6.5, 6.6, 7.16, 7.17, 7.18, 7.19, 8.5, 8.6, 8.7, 11.2 and 12.1 were added to the document, while Q&As 2.3, 2.10, 5.5 and 7.4 were revised. Q&A 9.2 was renumbered 11.1 following addition of section 11. More information is available on the Commission’s website.

Expert Panel on Health publishes three opinions to help guide policy makers
The independent Expert Panel on Effective ways of Investing in Health has adopted three Opinions related to access to healthcare, innovative payment models for high-cost innovative medicines and performance of primary care.

The Opinion on innovative payment models for high-cost innovative medicines analyses how national pricing and reimbursement authorities could improve patients’ access to innovative medicines and foster innovation that matters whilst ensuring that health systems are financially sustainable. The Opinion on benchmarking access to healthcare in the EU responds to the request for quantitative and qualitative benchmarks to assess progress in reducing unmet need for healthcare. The Opinion on tools and methodologies for assessing the performance of primary care explores how to measure the performance of primary care. More information is available on the Commission’s website.

Developing drugs and biologics for BCG-unresponsive nonmuscle invasive bladder cancer
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “BCG-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biologics for Treatment.”

The purpose of this guidance is to assist sponsors in the development of drugs, including biologics, for the treatment of patients who have bacillus Calmette-Guérin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC). This guidance is intended for pharmaceutical sponsors, the academic community, and the public and provides a framework, based on current Food and Drug Administration (FDA) thinking, to facilitate the development of drugs to treat this patient population. This guidance discusses pathological diagnosis and staging, risk stratification, and trial design, including assessment of appropriate clinical endpoints. More information is available on the FDA’s website.

Clinical investigation of medicinal products in the treatment or prevention of diabetes mellitus
The European Medicines Agency have published: Scientific guideline: Draft guideline on clinical investigation of medicinal products in the treatment or prevention of diabetes mellitus – Revision 2, draft: consultation open.

This guideline intends to address the EU regulatory position on the main topics of the clinical development of new medicinal products in the treatment or delay in onset / prevention of diabetes. The current revision refers mainly to an update of the safety section with respect to cardiovascular safety, but also updated guidance concerning e.g. treatment effects on diabetes complications, requirements for first line indications, high strength insulin preparations, definitions of hypoglycaemia and development of oral treatments for patients with type 1 diabetes. In addition, some editorial changes have been implemented. More information is available on the EMA’s website.

Consultation Deadline: 15th August, 2018

Microbiology data for systemic antibacterial drugs
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Microbiology Data for Systemic Antibacterial Drugs — Development, Analysis, and Presentation.”

The purpose of this guidance is to assist sponsors in the development, analysis, and presentation of microbiology data during antibacterial drug development. Specifically, this guidance addresses the Food and Drug Administration’s (FDA’s) current thinking regarding the overall microbiology development program needed to support clinical development and approval of antibacterial drugs administered systemically as well as microbiology information collected after approval. This guidance replaces the guidance for industry Microbiology Data for Systemic Antibacterial Drugs — Development, Analysis, and Presentation issued in August 2016. More information is available on the FDA’s website.

Safety and efficacy follow-up and risk management of advanced therapy medicinal products
The European Medicines Agency have published: Scientific guideline: Draft guideline on safety and efficacy follow-up and risk management of advanced therapy medicinal products – Rev. 1, draft: consultation open.

The aim of this guideline is to provide the guidance for the Safety and Efficacy (S&E) follow-up and risk management for advanced therapy medicinal products (ATMPs) according to Article 14(4) of Regulation (EC) No 1394/2007. This regulation requires the European Medicines Agency (EMA) to develop a detailed guideline relating to the post-authorisation follow-up of efficacy and adverse reactions, and risk management for these products. This is the 1st revision of the original ATMP guideline on safety and efficacy follow-up and risk management; the guideline has been revised to take into consideration the experience gained with the authorisation of these products and to define their risks and their risk minimisations measures. In addition, guidance on methodology in order to design post-authorisation S&E follow-up studies is provided. More information is available on the EMA’s website.

Consultation Deadline: 30th April, 2018

Evaluation of advanced therapy medicinal products
The European Medicines Agency have published: Regulatory and procedural guideline: Procedural advice on the evaluation of advanced therapy medicinal product in accordance with Article 8 of Regulation (EC) No 1394/2007, adopted.

This document describes the procedure for the evaluation of marketing authorisation applications for ATMPs. This procedure is put in place to establish timely and effective interactions between the EMA and the different committees (CAT, CHMP and the Pharmacovigilance Risk Assessment Committee (PRAC)) in conjunction with the Applicant during the centralised evaluation of an ATMP. The CAT is also responsible for post-authorisation activities of ATMPs. Though not described in this document, the same principles as outlined for the MAA evaluation procedure applies to postauthorisation activities (e.g. variations, renewals, etc.) according to the established timetables for the relevant procedure. More information is available on the EMA’s website.

Qualified infectious disease product designation questions and answers
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Qualified Infectious Disease Product Designation Questions and Answers.”

The Food and Drug Administration Safety and Innovation Act (FDASIA) creates incentives for the development of antibacterial and antifungal drug products that treat serious or life-threatening infections. The purpose of this draft guidance is to provide a resource for information on FDA’s policies and procedures related to the designation of a qualified infectious disease product (QIDP). More information is available on the Federal Register’s website.

Consultation Deadline: 2nd April, 2018

Commission report on Member State penalties for the falsification of medicines
The European Commission has published a Report from the Commission to the European Parliament and the Council on the Member States’ transposition of Article 118a of Directive 2001/83/EC of the European Parliament and the Council of 6 November 2001 on the Community code relating to medicinal products for human use as amended by Directive 2011/62/EU of the European Parliament and of the Council of 8 June 2011.

The Falsified Medicines Directive (2011/62/EU) (FMD) requires all EU countries to put in place proportionate, effective and dissuasive penalties for those involved in the production and circulation of falsified medicines. The report on how this requirement has been met shows a wide variation in penalties across the EU. More information is available on the Commission’s website.

Marker residue depletion studies to establish product withdrawal periods in aquatic species
The European Medicines Agency have published: Scientific guideline: Draft VICH GL57 on studies to evaluate the metabolism and residue kinetics of veterinary drugs in food-producing species: marker residue depletion studies to establish product withdrawal periods in aquatic species, draft: consultation open.

This guidance is one of a series developed to facilitate the mutual acceptance of residue chemistry data for veterinary drugs used in food-producing animals by national/regional regulators. This guidance was prepared after consideration of the current national/regional requirements and recommendations for evaluating veterinary drug residues in the VICH regions. More information is available on the EMA’s website.

Consultation Deadline: 15th June, 2018

Safety and residue data requirements for pharmaceutical veterinary medicinal products
The European Medicines Agency have published: Scientific guideline: Concept paper for the revision of the guideline on safety and residue data requirements for pharmaceutical veterinary medicinal products intended for minor use or minor species (MUMS)/limited market, draft: consultation open.

In order to stimulate the development of veterinary medicines intended for minor uses or minor species (MUMS)/limited market the CVMP has developed guidelines on data requirements for MUMS/limited market veterinary medicinal products for quality, safety and efficacy for pharmaceuticals, as well as a guideline for immunologicals. The guideline that focuses on safety requirements for pharmaceuticals includes a section on extrapolation of maximum residue limits (MRLs) from major to minor species. More information is available on the EMA’s website.

Consultation Deadline: 28th February, 2018

Developing fixed-dose combination drugs for the treatment of hypertension
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Hypertension: Developing Fixed-Dose Combination Drugs for Treatment.”

The purpose of this guidance is to assist sponsors in the clinical development of fixed-dose combination drugs for the treatment of hypertension. The guidance focuses on development of two-drug combinations of previously approved drugs. More information is available on the Federal Register’s website.

Consultation Deadline: 27th March, 2018

SmPC and package leaflet for technetium (99mTc) macrosalb
The European Medicines Agency have published: Scientific guideline: Draft guideline on core summary of products characteristics (SmPC) and package leaflet for technetium (99mTc) macrosalb, draft: consultation open.

This guideline describes the information to be included in the summary of products characteristics (SmPC) and package leaflet for technetium (99mTc) macrosalb. More information is available on the EMA’s website.

Consultation Deadline: 31st March, 2018

Reduction, refinement and replacement (3Rs) measures in the veterinary vaccines field described in the European Pharmacopoeia
The European Medicines Agency have published: Scientific guideline: Recommendation to marketing authorisation holders, highlighting recent measures in the veterinary field to promote reduction, refinement and replacement (3Rs) measures described in the European Pharmacopoeia – Applicable to veterinary vaccines from 01/01/2017, adopted.

In accordance with the European Convention for the Protection of Vertebrate Animals Used for Experimental and Other Scientific Purposes (Council of Europe), tests performed in animals must be carried out in such a way as to use the minimum number of animals and to cause the least pain, suffering, distress or lasting harm. The European Pharmacopeia (Ph. Eur.) has, over the years, applied these principles by implementing alternative tests and assays that reduce, refine and replace animal use. The European Pharmacopoeia has introduced a number of measures via new or revised texts in the veterinary field to promote 3Rs. These requirements are published in the 9th edition and are in force from 01/01/2017. More information is available on the EMA’s website.

Transfer of quality control methods validated in collaborative trials with a view to implementing 3Rs
The European Medicines Agency have published: Scientific guideline: Guidance for individual laboratories for transfer of quality control methods validated in collaborative trials with a view to implementing 3Rs, adopted.

In accordance with Directive 2010/63/EU, the principle of the 3Rs (Replacement, Reduction and Refinement) needs to be considered when selecting approaches for validating quality control tests in laboratories for regulatory testing of human and veterinary medicinal products. Collaborative studies between laboratories may be carried out to introduce new 3Rs methods for regulatory purposes where animal tests have been traditionally used. This guidance aims to facilitate transfer and acceptance of the new methods validated in such trials with a view to implementing 3Rs for testing in a product specific context in laboratories originally involved in the collaborative trial or in new laboratories. More information is available on the EMA’s website.

Reduction, refinement and replacement (3Rs) measures in the human vaccines field described in the European Pharmacopoeia
The European Medicines Agency have published: Scientific guideline: Recommendation to marketing authorisation holders, highlighting recent measures in the veterinary field to promote replacement, reduction, and refinement (3Rs) measures described in the European Pharmacopoeia – Applicable to human vaccines from 01/01/2018, adopted.

In accordance with the European Convention for the Protection of Vertebrate Animals Used for Experimental and Other Scientific Purposes (Council of Europe), tests performed in animals must be carried out in such a way as to use the minimum number of animals and to cause the least pain, suffering, distress or lasting harm. The European Pharmacopeia (Ph.Eur.) has, over the years, applied these principles by implementing alternative tests and assays that replace, reduce and refine animal use. The European Pharmacopoeia has introduced a number of measures via new or revised texts in the human field to promote 3Rs. These requirements are published in Supplement 9.3 and are in force from 01/01/2018. More information is available on the EMA’s website.

Chemistry of active substances for veterinary medicinal products
The European Medicines Agency have published: Scientific guideline: Guideline on the chemistry of active substances for veterinary medicinal products, adopted.

Guideline concerning the application of Directive 2001/82/EC with a view to the granting of a marketing authorisation for a veterinary medicinal product. This guideline replaces the ‘Note for guidance on chemistry of new active substances’ (EMEA/CVMP/541/03/Final) and ‘Chemistry of active substances’ (3AQ5a). It has been revised to cover new and existing active substances in one guideline. More information is available on the EMA’s website.

Date for coming into effect: 19th July, 2018

Public warning and notification of recalls under 21 CFR Part 7
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance entitled “Public Warning and Notification of Recalls Under 21 CFR Part 7, Subpart C; Draft Guidance for Industry and FDA Staff.”

The draft guidance, when finalized, establishes official guidance for industry and FDA staff regarding the use, content, and circumstances for issuance of public warnings and public notification of voluntary recalls under Federal regulations. The intent of the draft guidance is to increase and expedite the appropriate and accurate use of public warnings and public notification, to increase public health protection by better informing the public about violative products being recalled. The draft guidance clarifies and supplements existing policy for industry and FDA staff regarding the use of public warnings and public notification. More information is available on the Federal Register’s website.

Consultation Deadline: 20th March, 2018

Material threat medical countermeasure priority review vouchers
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Material Threat Medical Countermeasure Priority Review Vouchers.”

There is stakeholder interest in FDA’s implementation of the provision of the 21st Century Cures Act (Cures Act) that adds a new section to the Federal Food, Drug, and Cosmetic Act (FD&C Act) on priority review vouchers for material threat medical countermeasure applications. This new section of the FD&C Act makes provisions for awarding priority review vouchers for use with applications to sponsors of material threat medical countermeasure applications that meet the criteria specified by the FD&C Act. This draft guidance explains to internal and external stakeholders how FDA intends to implement the provisions of the new section of the FD&C Act. More information is available on the Federal Register’s website.

Consultation Deadline: 20th March, 2018

Highlights of prescribing information for human prescription drug and biological products
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Product Title and Initial U.S. Approval in the Highlights of Prescribing Information for Human Prescription Drug and Biological Products—Content and Format.”

The labeling regulations for human drug and biological products require that the Highlights of Prescribing Information (Highlights) contain a product title and the year of initial U.S. approval. This draft guidance provides recommendations on the content and format of the product title and initial U.S. approval to bring greater consistency to the presentation of these required elements and to help ensure these elements provide clear and useful information to the health care provider. More information is available on the Federal Register’s website.

Consultation Deadline: 20th March, 2018

Mixing, diluting, or repackaging biological products outside the scope of an approved BLA
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Mixing, Diluting, or Repackaging Biological Products Outside the Scope of an Approved Biologics License Application.”

This guidance sets forth FDA’s policy regarding the mixing, diluting, and repackaging of certain types of biological products that have been licensed under section 351 of the Public Health Service Act (PHS Act) when such activities are not within the scope of the product’s approved biologics license application (BLA) as described in the approved labeling for the product. This guidance describes the conditions under which FDA does not intend to take action for violations of section 351 of the PHS Act and section 502(f)(1), section 582, and, where specified, section 501(a)(2)(B) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 352(f)(1), 21 U.S.C. 360eee-1, and 21 U.S.C. 351(a)(2)(B), respectively), when a state-licensed pharmacy, a federal facility, or an outsourcing facility dilutes, mixes, or repackages certain biological products outside the scope of an approved BLA. More information is available on the FDA’s website.

Compounded drug products that are essentially copies of a commercially available drug product
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Compounded Drug Products That Are Essentially Copies of a Commercially Available Drug Product Under Section 503A of the Federal Food, Drug, and Cosmetic Act .”

To qualify for exemptions under section 503A of the Federal Food, Drug, and Cosmetic Act (FD&C Act), a drug product must be compounded by a licensed pharmacist or physician who does not compound regularly or in inordinate amounts any drug products that are essentially copies of a commercially available drug product, among other conditions. This guidance sets forth FDA’s policies regarding this provision of section 503A, including the terms commercially available, essentially a copy of a commercially available drug product, and regularly or in inordinate amounts. More information is available on the FDA’s website.

Compounded drug products that are essentially copies of approved drug products
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Compounded Drug Products That Are Essentially Copies of Approved Drug Products Under Section 503B of the Federal Food, Drug, and Cosmetic Act.”

For a drug product compounded by an outsourcing facility to qualify for the exemptions under section 503B of the Federal Food, Drug, and Cosmetic Act (FD&C Act), it must not be “essentially a copy of one or more approved drug products,” and must meet the other conditions in section 503B. This guidance sets forth FDA’s policies concerning the essentially a copy provision of section 503B. More information is available on the FDA’s website.

Clinical investigation of medicinal products for the treatment of rheumatoid arthritis
The European Medicines Agency have published: Scientific guideline: Guideline on clinical investigation of medicinal products for the treatment of rheumatoid arthritis, adopted.

The scope of this guideline is to provide a European common position on pertinent issues relating to the clinical evaluation of medicinal products (synthetic as well as biological DMARDs) for the treatment of RA classified according to international criteria, e.g. ACR-EULAR 2010. Intra-articular products are beyond the scope of this guideline. This document gives guidance on the performance of studies involving drug treatment for RA only. Separate guidance is available for other rheumatic diseases such as osteoarthritis, juvenile idiopathic arthritis, ankylosing spondylitis and psoriatic arthritis, in view of their different pathogenesis and natural histories. More information is available on the EMA’s website.

Date for coming into effect: 1st July, 2018

Good ANDA submission practices
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Good ANDA Submission Practices.”

This guidance is intended to assist applicants preparing to submit to FDA abbreviated new drug applications (ANDAs). This draft guidance highlights common, recurring deficiencies that may lead to a delay in the approval of an ANDA. It also makes recommendations to applicants on how to avoid these deficiencies with the goal of minimizing the number of review cycles necessary for approval. More information is available on the Federal Register’s website.

Consultation Deadline: 5th March, 2018

Labeling for combined hormonal contraceptives
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Labeling for Combined Hormonal Contraceptives.”

This draft guidance provides recommendations on information that should be included in the prescribing information for combined hormonal contraceptives (CHCs), which contain estrogen and progestin. CHC products include combined oral contraceptives (COCs), as well as non-oral products such as transdermal systems and vaginal rings. Many of the labeling recommendations in this draft guidance represent class labeling that should be included in all CHC prescribing information. The draft guidance reflects many of the modifications to prescribing information mandated by the physician labeling rule (PLR) and the pregnancy and lactation labeling rule (PLLR). General advice is provided where modifications to the prescribing information for specific products are needed. More information is available on the Federal Register’s website.

Consultation Deadline: 5th March, 2018

Effectiveness for drugs intended to treat male hypogonadotropic hypogonadism
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Establishing Effectiveness for Drugs Intended to Treat Male Hypogonadotropic Hypogonadism Attributed to Non-Structural Disorders.”

This draft guidance provides key design considerations, including recommendations for patient enrollment criteria and efficacy endpoints, for clinical trials to establish effectiveness for drugs intended to treat male hypogonadotropic hypogonadism associated with obesity and other conditions that do not cause intrinsic damage to the hypothalamus or pituitary gland. This draft guidance is consistent with recommendations FDA received at the December 2014 advisory committee meeting on the appropriate indicated population for testosterone replacement therapy, and the December 2016 advisory committee meeting on hypogonadotropic hypogonadism. More information is available on the Federal Register’s website.

Consultation Deadline: 5th March, 2018

Formal meetings between the FDA and sponsors or applicants of PDUFA products
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products.”

This draft guidance provides recommendations to industry on formal meetings between FDA and sponsors or applicants relating to the development and review of drug or biological products (hereafter referred to as products). The previous guidance for industry “Formal Meetings Between the FDA and Sponsors or Applicants” published May 19, 2009, and the draft guidance for industry “Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products” published March 11, 2015, have been withdrawn. More information is available on the Federal Register’s website.

Consultation Deadline: 29th March, 2018

Best practices for communication between IND sponsors and FDA during drug development
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry and review staff entitled “Best Practices for Communication Between IND Sponsors and FDA During Drug Development.”

The purpose of this guidance is to describe best practices and procedures for timely, transparent, and effective communications between investigational new drug application (IND) sponsors and FDA at critical junctures in drug development, which may facilitate earlier availability of safe and effective drugs to the American public. This guidance applies to communications between IND sponsors and FDA during the IND phase of drug development, including biosimilar biological product development (BPD). More information is available on the FDA’s website.

Manufacturing of sterile medicinal products
The European Commission has launched a targeted stakeholders consultation on the revision of annex 1, on manufacturing of sterile medicinal products, of the Eudralex volume 4.

Annex 1 was first published in 1971, since then it has undergone a number of targeted updates but, until now it has not undergone a full review. This revision is intended to add clarity, introduce the principles of Quality Risk Management to allow for the inclusion of new technologies and innovative processes and to change the structure to a more logical flow. More information is available on the European Commission’s website.

Consultation Deadline: 20th March, 2018

ICH guideline Q12 on technical and regulatory considerations for pharmaceutical product lifecycle management
The European Medicines Agency have published: Scientific guideline: Draft ICH guideline Q12 on technical and regulatory considerations for pharmaceutical product lifecycle management – Step 2b – First version, draft: consultation open.

This new guideline is proposed to provide guidance on a framework to facilitate the management of post-approval chemistry, manufacturing and controls (CMC) changes in a more predictable and efficient manner across the product lifecycle. This guideline aims to promote innovation and continual improvement, and strengthen quality assurance and reliable supply of product, including proactive planning of supply chain adjustments. The guideline strives to promote, for regulators (assessors and inspectors), an improved understanding of the Applicants’ pharmaceutical quality systems (PQSs) for management of post-approval CMC changes. This new guideline is intended to complement the existing ICH Q8 to Q11 guidelines. More information is available on the EMA’s website.

Consultation Deadline: 18th December, 2018

ICH guideline E17 on general principles for planning and design of multi-regional clinical trials
The European Medicines Agency have published: Scientific guideline: ICH guideline E17 on general principles for planning and design of multi-regional clinical trials – Step 5 – First version, adopted.

The purpose of this document is to outline general principles for the planning and design of multiregional clinical trials with the aim of increasing their acceptability in global regulatory submissions. The document addresses some strategic programme issues as well as aspects specific to the planning and design of confirmatory MRCTs and should be used together with other ICH efficacy guidelines, including E2, E3, E4, E5, E6, E8, E9, E10 and E18. More information is available on the EMA’s website.

Date for coming into effect: 14th June, 2018

Chemistry, manufacturing, and controls changes to an approved application
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled “Chemistry, Manufacturing, and Controls Changes to an Approved Application: Certain Biological Products; Draft Guidance for Industry.”

The draft guidance is intended to assist applicants and manufacturers of certain licensed biological products in determining which reporting category is appropriate for a change in chemistry, manufacturing, and controls (CMC) information to an approved biologics license application (BLA). The draft guidance provides applicants and manufacturers general and administrative information on reporting and evaluating changes and recommendations for reporting categories based on a tiered-reporting system for specific changes. The draft guidance, when finalized, is intended to supersede the document entitled “Guidance for Industry: Changes to an Approved Application: Biological Products” dated July 1997 (July 1997 guidance). More information is available on the Federal Register’s website.

Consultation Deadline: 22nd March, 2018

Drug products labeled as homeopathic
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for FDA staff and industry entitled “Drug Products Labeled as Homeopathic.”

This draft guidance describes how FDA intends to prioritize enforcement and regulatory action with regard to drug products, including biological products, labeled as homeopathic and marketed in the United States without the required FDA approval. More information is available on the Federal Register’s website.

Consultation Deadline: 20th March, 2018

Drug products that contain nanomaterials
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Drug Products, Including Biological Products, that Contain Nanomaterials.”

This draft guidance has been developed to provide industry with the Agency’s current thinking for the development of human drug products, including those that are biological products, that contain nanomaterials. The draft guidance also includes recommendations for applicants and sponsors of investigational, premarket, and postmarket submissions for these products. More information is available on the Federal Register’s website.

Consultation Deadline: 19th March, 2018

Waiver of in vivo bioavailability and bioequivalence studies for immediate-release solid oral dosage forms
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Waiver of In Vivo Bioavailability and Bioequivalence Studies for Immediate-Release Solid Oral Dosage Forms Based on a Biopharmaceutics Classification System.”

This guidance provides recommendations for sponsors of investigational new drug applications (INDs), and applicants who submit new drug applications (NDAs), abbreviated new drug applications (ANDAs), and supplements to these applications for immediate-release (IR) solid oral dosage forms, and who wish to request a waiver of an in vivo bioavailability (BA) and/or bioequivalence (BE) study requirement. These recommendations are intended to apply to waivers requested during the IND period and the NDA stage or for ANDAs, i.e., (1) subsequent in vivo BA or BE studies of formulations after the initial establishment of the in vivo BA of IR solid oral dosage forms during the IND period, and (2) in vivo BE studies of IR solid oral dosage forms in NDAs, ANDAs, and supplements to these applications. More information is available on the FDA’s website.

Summary of product characteristics for anthelmintics
The European Medicines Agency have published: Scientific guideline: Concept paper for the revision of the guideline on the summary of product characteristics for anthelmintics, draft: consultation open.

The current guideline on the Summary of Product Characteristics (SPC) for anthelmintics (EMEA/CVMP/EWP/170208/2005) recommends standard warnings aimed at delaying the development of anthelmintic resistance. The scope of the guideline is currently limited to products for sheep, goats, cattle and horses, and proposes standard text for sections 4.4 and 4.9 of the SPC. The scope of the guideline should be extended to host species other than ruminants and horses, e.g. to pigs and companion animals. In those additional species, resistance emergence is reported in some helminths in given geographical areas. More information is available on the EMA’s website.

Consultation Deadline: 31st March, 2018

ICH Guideline S3A: Note for guidance on toxicokinetics: the assessment of systemic exposure in toxicity studies
The European Medicines Agency have published: Scientific guideline: ICH Guideline S3A: Note for guidance on toxicokinetics: the assessment of systemic exposure in toxicity studies – Questions and answers – Step 5 – First version, adopted.

The S3A guideline was successfully implemented in 1994. However, in recent years, analytical method sensitivity has improved, allowing microsampling techniques to be widely used in toxicokinetic (TK) assessment. This Q&A document focuses on points to consider before incorporating the microsampling method in TK studies, acknowledges its benefits, and some limitations, for assessment of TKs in main study animals and its overall important contribution to the 3Rs benefits (Replacement, Reduction and Refinement) by reducing or eliminating the need for TK satellite animals. More information is available on the EMA’s website.

Systemic antibacterial and antifungal drugs
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Systemic Antibacterial and Antifungal Drugs: Susceptibility Test Interpretive Criteria Labeling for NDAs and ANDAs.”

This guidance provides recommendations on fulfilling the new labeling requirements for susceptibility test interpretive criteria for prescription systemic antibacterial and antifungal drugs as established by section 3044 of the 21st Century Cures Act (Cures Act) (Public Law 114-255). It describes FDA’s recommendations on (1) how to make these labeling changes for antimicrobial drugs with approved new drug applications (NDAs) or abbreviated new drug applications (ANDAs), (2) language to remove from the labeling of such drugs, and (3) labeling language to use for all antimicrobial drugs to reference the FDA’s Susceptibility Test Interpretive Criteria web page. More information is available on the FDA’s website.

Monoclonal antibodies for veterinary use
The European Medicines Agency have published: Scientific guideline: Questions and answers on monoclonal antibodies for veterinary use, adopted.

The range of clinical indications with potential for treatment with mAbs is very wide. Currently, in human medicine, a number are authorised for use as anti-cancer medicines and in medicines against diseases affecting the immune system, such as rheumatoid arthritis. To date, the CVMP and its Scientific Advice Working Party (SAWP-V) have addressed a limited number of scientific advice requests concerning mAb products. This activity indicates that a number of mAbs for use as veterinary medicinal products are in development. Indeed, in February 2017, the CVMP recommended the granting of a marketing authorisation for Cytopoint (lokivetmab), the first monoclonal antibody in a veterinary medicine in the EU, intended for the treatment of dogs with atopic dermatitis. Following a review of the scientific information relating to mAbs, a number of areas (in the form of questions) were identified that would benefit from consideration by relevant experts and the elaboration of specific guidance in the form of questions and answers (Q&A). information is available on the EMA’s website.

QRD guidance on the use of approved pictograms on the packaging of veterinary medicinal products
The European Medicines Agency have published: Regulatory and procedural guideline: Quality review of documents (QRD) guidance on the use of approved pictograms on the packaging of veterinary medicinal products authorised via the centralised (CP), mutual recognition (MRP) and decentralised procedures (DCP), adopted.

The need for a harmonised, approved ‘catalogue’ of pictograms was identified and this draft guidance was developed as a collaboration between the QRD (vet) group, the CMDv, CVMP and industry stakeholders. The use of pictograms from Annex 1 of this guidance will apply to the national authorisation procedures, including MRP and DCP and to the centralised procedure (EMA). The aim of the guidance is to outline the approval requirements and processes where pictograms from Annex 1 are used on the product literature. More information is available on the EMA’s website.

Product name placement, size, and prominence in promotional labeling and advertisements
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Product Name Placement, Size, and Prominence in Promotional Labeling and Advertisements.”

This guidance clarifies the requirements for product name placement, size, prominence, and frequency in promotional labeling and advertisements for prescription drugs. The disclosure of the product name in promotional labeling and advertisements is important for proper identification and to ensure safe and effective use. This guidance also articulates the circumstances under which FDA intends to refrain from taking enforcement action regarding these requirements. More information is available on the FDA’s website.

Drug development in pediatric rare diseases
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Pediatric Rare Diseases—A Collaborative Approach for Drug Development Using Gaucher Disease as a Model.”

This draft guidance focuses on drug development for pediatric patients with Gaucher disease. In particular, it proposes for consideration a novel approach to improve the efficiency of drug development in pediatric rare diseases using Gaucher disease as an example. The emergence of concomitant trials for multiple investigational drug products for the treatment of rare diseases can pose significant challenges to effective drug development, because there are limited numbers of patients for any given rare condition worldwide. This approach discusses the feasibility of the development of multiple drug products in a time-efficient manner while minimizing the number of patients necessary to be treated with placebo. More information is available on the Federal Register’s website.

Consultation Deadline: 5th February, 2018

Juvenile animal studies and impact on anti-cancer medicine development and use in children
The European Medicines Agency have published: Scientific guideline: Results of juvenile animal studies (JAS) and impact on anti-cancer medicine development and use in children, adopted.

This project was aimed to assess the impact and the utility of JAS on paediatric medicinal product development, and the example of anti-cancer medicines was chosen as they are expected to show toxicity that most likely affects developing tissues and organs, in both humans and animals. More information is available on the EMA’s website.

Preparation of EU herbal monographs for well-established and traditional herbal medicinal products
The European Medicines Agency have published: Scientific guideline: Guideline on the assessment of clinical safety and efficacy in the preparation of EU herbal monographs for well-established and traditional herbal medicinal products – Revision 1, adopted.

This guideline describes the legal background and recommendations for the assessment of data that are used to prepare European Union herbal monographs (formerly called Community herbal monographs) on herbal medicinal products and the European Union list of herbal substances, preparations and combinations thereof for use in traditional herbal medicinal products. The areas of herbal medicinal products with well-established medicinal use and traditional herbal medicinal products
are addressed.

Revision 1 pertains to an update of the document to current standards taking into account advances over the last 10 years and established practice and legal interpretations. Developments and details in the assessment methodology have so far been mainly reflected in template revisions (such as Assessment report template), but also other documents such as the public statement EMA/HMPC/473587/2011. In addition to the alignment with other documents the revision aimed for improved clarity and transparency by shortening some sections or providing more detail on some particular aspects of the assessment process, e.g. as regards specific population groups.

More information is available on the EMA’s website.

Chemical and pharmaceutical quality documentation concerning investigational medicinal products in clinical trials
The European Medicines Agency have published: Scientific guideline: Guideline on the requirements for the chemical and pharmaceutical quality documentation concerning investigational medicinal products in clinical trials – Revision 1, adopted.

This guideline addresses the documentation on the chemical and pharmaceutical quality of IMPs and Auxiliary Medicinal Products containing chemically defined drug substances, synthetic peptides, synthetic oligonucleotides, herbal substances, herbal preparations and chemically defined radioactive/radio-labelled substances to be submitted to the competent authority for approval prior to beginning a clinical trial in humans. It includes the requirements for IMPs and Auxiliary Medicinal Products to be tested in phase I, phase II, phase III and phase IV studies as well as the requirements for modified and unmodified comparator products and IMPs to be tested in generic bioequivalence studies.

The guideline is to be seen in connection with Regulation (EU) No. 536/2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC, which came into force on June 20, 2014. Since clinical trials will often be designed as multi -centre studies, potentially involving different Member States, it is the aim of this guideline to define harmonised requirements for the documentation to be submitted throughout the European Union. This guideline replaces the “Guideline on the requirements to the chemical and pharmaceutical quality documentation concerning investigational medicinal products in clinical trials” (CHMP/QWP/185401/2004 final).

More information is available on the EMA’s website.

Date for coming into effect: 20th March, 2018

GMP specific to advanced therapy medicinal products
The European Commission has published a set of guidelines on good manufacturing practice (GMP) specific to advanced therapy medicinal products (ATMPs).

The new guidelines adapt the European Union GMP requirements to the specific characteristics of ATMPs and address the novel and complex manufacturing scenarios utilised for these products. The guidelines foster a risk based approach to manufacture and testing of such products. The guidelines ensure that these novel medicinal products are consistently produced and controlled according to high quality standards, for the benefit and the safety of patients. This initiative is part of the joint action plan launched by the Directorate General for Health and Food Safety (DG SANTE)External link icon and the European Medicines Agency (EMA) in October 2017 to foster the development of ATMPs. More information is available on the Commission’s website.

European Commission Safety Features Q&A document updated
The European Commission has published an updated version, Version 8, of the Questions and Answers document on the Safety Features for Medicinal Products for Human Use.

Version 8 of the document, dated November 2017, supersedes Version 7. Q&As 1.17, 1.18, 7.13, 7.14, 7.15, 8.4 and 10.1 were added were added to the document, while Q&As 1.2, 2.12 and 3.3 were revised. More information is available on the Commission’s website.

Pharmaceuticals in the environment
The European Commission has launched a public consultation on pharmaceuticals in the environment.

The main background document draws attention to the risk posed by some pharmaceuticals to the environment, for example to fish populations, and to the possibility that human health might be affected via the environment. The document presents 30 possible policy options for achieving more environmentally sustainable use of pharmaceuticals, important among other things to help the EU achieve the UN Sustainable Development Goals, in particular Goal 6 (“Clean Water and Sanitation”), as well as objectives in EU legislation such as the “good status” objective in the Water Framework Directive. The consultation results will be used to identify a shorter list of policy options for possible follow-up in proposals for measures, subject to impact assessment as appropriate. More information is available on the European Commission’s website.

Consultation Deadline: 21st February, 2018

Conduct of pharmacokinetic studies in target animal species
The European Medicines Agency have published: Scientific guideline: Draft guideline on conduct of pharmacokinetic studies in target animal species – Revision 1, draft: consultation open.

The objectives of this guidance are to specify the pharmacokinetic factors to be investigated, acknowledging that this will depend on the active substance and its use, and to provide recommendations for the conduct of pharmacokinetic studies for the purpose of supporting the clinical part of the dossier for a veterinary pharmaceutical product. In addition, general guidance is given on pharmacokinetic-pharmacodynamic modelling and population pharmacokinetics, should applicants opt to pursue these approaches. More information is available on the EMA’s website.

Consultation Deadline: 31st May, 2018

Clinical development of medicinal products for the treatment of Autism Spectrum Disorder
The European Medicines Agency have published: Scientific guideline: Guideline on the clinical development of medicinal products for the treatment of Autism Spectrum Disorder (ASD), adopted.

Autism Spectrum Disorder (ASD) is amongst the most common and varied disorders in paediatric psychiatry. It impacts significantly on social, occupational and other important areas of functioning. It is a lifelong condition and although various therapies and interventions are available few are supported by scientific studies. Pharmacotherapies approved to date for the management of ASD have been nonspecific for the condition (e.g. atypical antipsychotics for the control of behavioural disturbances) and do not target the core symptoms. This document is intended to provide guidance on the evaluation of new products in ASD; it should be read in conjunction with other EMA and ICH guidelines, which may apply to similar conditions and patient populations. More information is available on the EMA’s website.

Date for coming into effect: 1st June, 2018

Evaluation of anticancer medicinal products in man
The European Medicines Agency have published: Scientific guideline: Guideline on the evaluation of anticancer medicinal products in man, adopted.

The guideline on anticancer medicinal products adopted in 1996, and revised in 2001 and 2003, focused on conventional cytotoxic compounds. In 2005, a major revision was undertaken, aiming at covering non-cytotoxic compounds, to expand on the sections on exploratory trials and to provide more guidance with respect to methodological issues. Later, there followed an appendix on methodological issues related to the use of PFS and in early 2010 an appendix on haematological malignancies followed. The main guideline was subsequently updated in line with these appendices, e.g. with regard to confirmatory studies based on aims of therapy and relative toxicity, while the section on condition specific guidance was expanded and placed in a separate Appendix 4. Since then a new Appendix 2 has been adopted, concerned with patient reported outcomes and health-related quality of life. The purpose of the 5th revision of the main guideline is to address current changes in the therapeutic landscape that affect the requirements with regard to collection and reporting of safety data in order to inform the benefit-risk evaluation, including a need for more differentiated and detailed safety data presentation. More information is available on the EMA’s website.

Date for coming into effect: 1st April, 2018

Evaluating the abuse deterrence of generic solid oral opioid drug products
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “General Principles for Evaluating the Abuse Deterrence of Generic Solid Oral Opioid Drug Products.”

This guidance is intended to assist a person who plans to develop and submit an abbreviated new drug application (ANDA) (hereinafter potential ANDA applicant) to seek approval of a generic version of a solid oral opioid drug product that references an opioid drug product with abuse-deterrent properties described in its labeling. The guidance recommends studies, including comparative in vitro and pharmacokinetic (PK) studies, that the potential ANDA applicant should conduct and submit to FDA in an ANDA to demonstrate that a generic solid oral opioid drug product is no less abuse deterrent than its reference listed drug (RLD) with respect to all potential routes of abuse. More information is available on the FDA’s website.

Comments received on ‘Draft Guideline on manufacture of the finished dosage form’
The European Medicines Agency have published: Scientific guideline: Overview of comments received on ‘Draft Guideline on manufacture of the finished dosage form’ (EMA/CHMP/QWP/245074) – Revision 1.

More information is available on the EMA’s website.

Improving the product information for EU medicines
The European Medicines Agency (EMA) has published an action plan to improve the product information (PI) for EU medicines, an information package for patients and healthcare professionals that accompanies every single medicine authorised in the EU and explains how it should be used and prescribed.

One of the key areas of this plan is to explore how electronic or digital means can be used to improve accessibility to medicines’ information by patients and healthcare professionals. EMA together with the European Commission will organise a multi-stakeholder workshop in the third quarter of 2018 to develop key principles for the use of electronic formats. To ensure a comprehensive overview of all ongoing EU initiatives at the workshop, EMA will start a mapping exercise involving all stakeholders (patients and consumers, healthcare professionals, pharmaceutical industry and national competent authorities). To facilitate this mapping exercise, stakeholders are invited to send an overview of initiatives on electronic/digital formats for the product information that they are aware of or working on. More information is available on the EMA’s website.

Consultation Deadline: 28th February, 2018

Evaluating drug effects on the ability to operate a motor vehicle
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Evaluating Drug Effects on the Ability to Operate a Motor Vehicle.”

The purpose of this guidance is to assist pharmaceutical sponsors in the evaluation of the effects of psychoactive drugs on the ability to operate a motor vehicle. More information is available on the FDA’s website.

Developing drugs for treatment and prevention of recurrent herpes labialis
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Recurrent Herpes Labialis: Developing Drugs for Treatment and Prevention.”

The purpose of this guidance is to assist sponsors in the development of drugs for the treatment and prevention of recurrent herpes labialis (RHL). Specifically, this guidance addresses the Food and Drug Administration’s (FDA’s) current thinking regarding the overall development program and clinical trial designs to support the development of drug products with an antiviral mechanism of action used to prevent and/or treat RHL caused by either herpes simplex virus type 1 or 2 (HSV-1 or HSV-2) in immunocompetent subjects. More information is available on the FDA’s website.

Recommended statement for OTC aspirin-containing drug products labeled with cardiovascular related imagery
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Recommended Statement for Over-the-Counter Aspirin-Containing Drug Products Labeled With Cardiovascular Related Imagery.”

This guidance is intended to promote the safe use of OTC aspirin drug products by encouraging drug manufacturers, packagers, and labelers marketing aspirin drug products with cardiovascular related imagery to include a statement that reminds consumers to talk to their doctor or other healthcare provider before using aspirin for the professional indication of secondary prevention of cardiovascular events. More information is available on the FDA’s website.

Developing direct-acting antiviral drugs for treatment of chronic hepatitis C virus
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Chronic Hepatitis C Virus Infection: Developing Direct-Acting Antiviral Drugs for Treatment.”

The purpose of this guidance is to assist sponsors in the clinical development of direct-acting antiviral (DAA) drugs for the treatment of chronic hepatitis C (CHC) from the preinvestigational new drug application (pre-IND) stage through the new drug application (NDA) and postmarketing stages. More information is available on the FDA’s website.

Formal dispute resolution for sponsor appeals
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry and review staff entitled “Formal Dispute Resolution: Sponsor Appeals Above the Division Level.”

This guidance provides recommendations for industry and review staff on the procedures in the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) for resolving scientific and/or medical disputes between CDER or CBER and sponsors that cannot be resolved at the division level. This guidance describes the formal dispute resolution (FDR) procedures for sponsors that wish to appeal a scientific and/or medical issue to the office or center level and provides a structured process for resolving disputes. More information is available on the FDA’s website.

New analytical methods/technologies in the quality control of herbal medicinal products
The European Medicines Agency have published: Scientific guideline: Concept paper on the development of a reflection paper on new analytical methods/technologies in the quality control of herbal medicinal products, draft: consultation open.

Quality control is a prerequisite to assure safe and effective use of (traditional) herbal medicinal products, which are complex mixtures of numerous phytochemical constituents. For the majority of herbal substances, herbal preparations and (traditional) herbal medicinal products the active constituents are not known or are only partly understood. Requirements on quality control are based on Directive 2001/82/EC and Directive 2001/83/EC and specified in detail in Annex I (Directive 2003/63/EC) and relevant guidelines published by EMA (see references). Binding quality standards are also described in the relevant monographs of the European Pharmacopeia. There is an established set of methods (such as HPLC, TLC, GC, etc.) which is currently used in quality control of (traditional) herbal medicinal products (HMPs). In total, the system established cannot exactly measure all features or constituents in parallel, but is an appropriate and generally applied convention. When considering introduction of new methodology, this will usually be complementary to the existing methodology. Although some new techniques offer the option to create a type of “holistic” fingerprint, it is necessary to consider if such approaches offer added value in terms of quality control. More information is available on the EMA’s website.

Consultation Deadline: 30th April, 2018

Clinical investigation of medicinal products for the treatment of axial spondyloarthritis
The European Medicines Agency have published: Scientific guideline: Guideline on the clinical investigation of medicinal products for the treatment of axial spondyloarthritis – Revision 1, adopted.

This document is a revision of the Guideline on clinical investigation of medicinal products for the treatment of ankylosing spondylitis (CPMP/EWP/4891/03) which came into effect in May 2009. It should be considered as general guidance on the development of medicinal products for the treatment of axial spondyloarthritis and should be read in conjunction with other European and ICH guidelines which may apply to this disease area and patient population. The current revision has taken into account that clinical practice has evolved since publication of the previous guideline and acknowledges that patients with axial spondyloarthritis (axial SpA) who do not fulfill the modified New York (mNY) criteria of ankylosing spondylitis (AS) can present with disease activity and functional impairment similar to those observed in patients with AS. These patients, captured under the term non-radiographic axial SpA, are considered in this revised CHMP guideline. The new guideline also reviews relevant treatment goals, new outcome measures for the treatment as well as the design of confirmatory trials in the light of the currently available treatment options. More information is available on the EMA’s website.

Date for coming into effect: 1st May, 2018

Statistical principles for clinical trials
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance entitled “E9(R1) Statistical Principles for Clinical Trials: Addendum: Estimands and Sensitivity Analysis in Clinical Trials.”

The draft guidance was prepared under the auspices of the International Council for Harmonisation (ICH), formerly the International Conference on Harmonisation. The draft guidance clarifies, updates, and extends the earlier “E9 Statistical Principles for Clinical Trials” in two main areas. Concerning estimands, it provides a framework for discussion of how the aims of a trial relate to the proposed statistical analysis. Concerning sensitivity analysis, it discusses how to use additional analyses to address concerns about the validity of assumptions underlying the main analysis. The draft guidance is intended to better align the choice of statistical methods with questions of regulatory importance and to improve the reliability of decisions about and representations of the effects of medical products. More information is available on the Federal Register’s website.

Consultation Deadline: 30th April, 2018

Prophylaxis or treatment of respiratory syncytial virus (RSV) disease
The European Medicines Agency have published: Scientific guideline: Draft guideline on the clinical evaluation of medicinal products indicated for the prophylaxis or treatment of respiratory syncytial virus (RSV) disease, draft: consultation open.

The guideline covers the clinical development of vaccines and monoclonal antibodies for the prevention of RSV disease and direct acting antiviral agents for the treatment of RSV disease. The focus is on the assessment of safety and efficacy in populations most likely to develop RSV lower respiratory tract infection and severe RSV disease, including (newborn) infants and toddlers, older children predisposed to develop severe RSV disease and the elderly. The guideline also addresses vaccination of pregnant women with the aim of preventing RSV disease in their infants.
The draft guideline proposes some considerations on nonclinical investigations of efficacy and risk of vaccine-associated enhanced disease to support clinical trials with preventive or therapeutic products directed at RSV. More information is available on the EMA’s website.

Consultation Deadline: 30th April, 2018

State of children’s medicines in the EU
The European Commission has presented a report to the European Parliament and the Council, on progress made in children’s medicines since the Paediatric Regulation came into force 10 years ago.

This report is an essential intermediate step in the debate on a joint vision about the future parameters for paediatric and orphan medicines. Before proposing any amendments, the Commission will evaluate – in consultation with stakeholders and experts, how the combined effects of the Orphan and Paediatric Regulation can support medicine development in subpopulations of particular need, e.g. children with cancer. Results of this reflection will be presented by 2019 to allow the next Commission to take informed decision about possible policy options. More information is available on the Commission’s website.

Quality documentation concerning biological investigational medicinal products in clinical trials
The European Medicines Agency have published: Scientific guideline: Guideline on the requirements for quality documentation concerning biological investigational medicinal products in clinical trials – Revision 1, adopted.

This guideline addresses the specific documentation requirements on the biological, chemical and pharmaceutical quality of IMPs containing biological / biotechnology derived substances. Moreover, this guideline lists, as regards documentation on the biological, chemical and pharmaceutical quality of the IMP, examples of modifications which are typically considered as ‘substantial’. More information is available on the EMA’s website.

Evaluation of new active substance status of chemical substances – veterinary
The European Medicines Agency have published: Scientific guideline: Reflection paper on the chemical structure and properties criteria to be considered for the evaluation of new active substance (NAS) status of chemical substances – Veterinary, adopted.

This reflection paper is intended to reflect the current experience of the Quality Working Party (QWP), of the Committee for Medicinal Products for Veterinary Use (CVMP) and the Co-ordination Group for Mutual Recognition and Decentralised Procedures-Veterinary (CMDv) concerning the definition of a New Active Substance (NAS) in the context of preparation of dossiers and submissions of applications for Marketing Authorisation (MAA) in the Centralised Procedure (CP), the Mutual Recognition Procedure (MRP)/Decentralised Procedure (DCP) and purely national procedures for chemical medicinal products for veterinary use under Article 12(3) of Directive 2001/82/EC as amended. The paper describes the chemical structure and properties criteria to be taken into account by the competent authorities to qualify a chemical active substance as NAS, as well as the required elements to be submitted by applicants to substantiate their claims. More information is available on the EMA’s website.

Excipients in the labelling and package leaflet of medicinal products for human use
The European Medicines Agency (EMA) and the European Commission have updated the annex to the European Commission guideline on excipients in the labelling and package leaflet of medicinal products for human use.

The updated annex contains all excipients that must be declared in a medicine’s labelling and package leaflet and their agreed safety warnings. The main aim of this update is to take into account safety concerns which are not currently addressed in the existing annex to the guideline. More information is available on the EMA’s website.

ICH guideline E18 on genomic sampling and management of genomic data
The European Medicines Agency have published: Scientific guideline: ICH guideline E18 on genomic sampling and management of genomic data – First version, adopted.

The scope of this guideline pertains to genomic sampling and management of genomic data obtained from interventional and non-interventional clinical studies. Genomic research can be conducted during or after a clinical study. More information is available on the EMA’s website.

Date for coming into effect: 28th February, 2018

ICH E11(R1) guideline on clinical investigation of medicinal products in the pediatric population
The European Medicines Agency have published: Scientific guideline: ICH E11(R1) guideline on clinical investigation of medicinal products in the pediatric population – Revision 1 (addendum), adopted.

The purpose of this addendum is to complement and provide clarification and current regulatory perspective on topics in pediatric drug development. More information is available on the EMA’s website.

Date for coming into effect: 28th February, 2018

Classification of veterinary medicinal products indicated for minor use minor species (MUMS) / limited market
The European Medicines Agency have published: Scientific guideline: Guidance on the classification of veterinary medicinal products indicated for minor use minor species (MUMS) / limited market – Revision 1, adopted.

This guideline is based on the Revised Policy for classification and incentives for veterinary medicinal products indicated for minor use minor species (MUMS)/limited market (EMA/308411/2014).
This revision (Rev.1) is an administrative update to ensure that the document corresponds with the revised guidelines on data requirements for veterinary medicinal products intended for MUMS/limited market that were adopted by CVMP in 2017. The opportunity has been taken to simplify the request process for applicants and to introduce a small number of textual changes aiming to improve the clarity of the document without changing the meaning or intention. More information is available on the EMA’s website.

Companies requesting classification as minor uses minor species (MUMS) / limited markets
The European Medicines Agency have published: Regulatory and procedural guideline: Q&A – European Medicines Agency guidance for companies requesting classification as minor uses minor species (MUMS) / limited markets, adopted.

This guidance document addresses a number of questions that applicants requesting classification of products/indications for minor use or minor species (MUMS)/ limited market may have. More information is available on the EMA’s website.

Completeness assessments for Type II API DMFs under GDUFA
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Completeness Assessments for Type II API DMFs Under GDUFA Guidance for Industry.”

This guidance is intended for holders of Type II active pharmaceutical ingredient (API) drug master files (DMFs) that are or will be referenced in an abbreviated new drug application (ANDA), an amendment to an ANDA, a prior approval supplement (PAS) to an ANDA, or an amendment to a PAS (generic drug submissions). More information is available on the FDA’s website.

ANDA submissions – prior approval supplements under GDUFA
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “ANDA Submissions – Prior Approval Supplements Under GDUFA.”

This guidance is intended to assist applicants preparing to submit to FDA prior approval supplements (PASs) and amendments to PASs for abbreviated new drug applications (ANDAs) under section 505(j) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 355(j)). More information is available on the FDA’s website.

Advancement of emerging technology applications for pharmaceutical innovation and modernization
The Food and Drug Administration (FDA or Agency) is announcing the availability of a guidance for industry entitled “Advancement of Emerging Technology Applications for Pharmaceutical Innovation and Modernization.”

This guidance finalizes the draft guidance issued December 23, 2015, which provides recommendations to pharmaceutical companies interested in participating in a program involving the submission of emerging manufacturing technology. The program is open to companies that intend to include the technology as part of a regulatory submission including an investigational new drug application (IND), original or supplemental new drug application (NDA), abbreviated new drug application (ANDA) or biologic license application (BLA), or application-associated Drug Master File (DMF) reviewed by the Center for Drug Evaluation and Research (CDER), and where that technology meets other criteria described in this guidance. More information is available on the Federal Register’s website.

Expedited programs for serious conditions – drugs and biologics
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Expedited Programs for Serious Conditions – Drugs and Biologics.”

The following four FDA programs are intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of a serious or lifethreatening condition: fast track designation, breakthrough therapy designation, accelerated approval, and priority review designation (see section IV for an overview of the programs). The purpose of this guidance for industry is to provide a single resource for information on FDA’s policies and procedures for these four programs as well as threshold criteria generally applicable to concluding that a drug is a candidate for these expedited development and review programs. More information is available on the FDA’s website.

Clinical investigation of new medicinal products for the treatment of acute coronary syndrome
The European Medicines Agency have published: Scientific guideline: Guideline on clinical investigation of new medicinal products for the treatment of acute coronary syndrome – First version, adopted.

This guideline replaces ‘Points to consider on the clinical investigation of new medicinal products for the treatment of acute coronary syndrome (ACS) without persistent ST segment elevation’ (CPMP/EWP/570/98). More information is available on the EMA’s website.

Date for coming into effect: 1st March, 2018

Clinical investigation of medicinal products for the treatment of chronic heart failure
The European Medicines Agency have published: Scientific guideline: Guideline on clinical investigation of medicinal products for the treatment of chronic heart failure – Revision 2, adopted.

This guideline replaces the Note for Guidance on clinical investigation of medicinal products for the treatment of cardiac failure (CPMP/EWP/235/95, Rev 1). More information is available on the EMA’s website.

Date for coming into effect: 1st March, 2018

European Commission adopts acts on Good Manufacturing Practices for medicines
The European Commission has adopted two legal acts aimed at improving patient safety in the EU through good manufacturing practices (GMP) that ensure the highest quality of medicines for human use.

The first act is an implementing directive that sets out principles and guidelines of GMP in medicines where the manufacture or import is subject to a manufacturing authorization, as per Article 40 of the Community code Directive (2001/83/EC).

The second act is a delegated regulation that sets out GMP for investigational medicinal products, as required by the Clinical Trials Regulation (536/2014/EU), and detailed arrangements for inspections. This legal act ensures the highest quality of medicinal products used in clinical trials and prepares the smooth entry into force of this Regulation.

The principles and guidelines for GMP set out in these acts take into account recent updates to the well-established EU rules on the safety of medicines. Whether a medicinal product is already on the market, or still undergoing a clinical trial, the newly adopted acts aim to ensure the highest level of quality for medicines for the benefit of the patients as well as consistency between the GMP requirements for both types of medicinal products.

More information is available on the Commission’s website.

Multi-strain dossiers for inactivated vaccines against avian influenza, bluetongue and foot-and-mouth disease
The European Medicines Agency have published: Scientific guideline: Draft guideline on data requirements for multi-strain dossiers for inactivated vaccines against avian influenza (AI), bluetongue (BT) and foot-and-mouth disease (FMD) – Rev.1, draft: consultation open.

This guideline was first published in 2010 based on general scientific and regulatory principles in advance of much practical experience from assessing applications through European authorisation procedures. This revision was prepared following a review by CVMP of issues raised by stakeholders based on their experience of operating the guideline. Only minor changes were considered necessary to the guideline itself and an accompanying ‘Question and Answer’ document was produced to address the topics raised by stakeholders that are not specifically addressed within the revised guideline. More information is available on the EMA’s website.

Consultation Deadline: 31st March, 2018

Risk evaluation and mitigation strategies document using structured product labeling
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Providing Regulatory Submissions in Electronic Format—Content of the Risk Evaluation and Mitigation Strategies Document Using Structured Product Labeling.”

This draft guidance is being issued in accordance with the Food and Drug Administration Safety and Innovation Act (FDASIA), which amended the Federal Food, Drug, and Cosmetic Act (the FD&C Act) to require that certain submissions under the FD&C Act and the Public Health Service Act (PHS Act) be submitted in electronic format, beginning no earlier than 24 months after issuance of final guidance on electronic format for submissions. The draft guidance describes how FDA plans to implement the requirements for the electronic submission of Risk Evaluation and Mitigation Strategies (REMS) documents in certain submissions under new drug applications, abbreviated new drug applications, and biologics license applications, beginning no earlier than 24 months after issuance of the final guidance. More information is available on the Federal Register’s website.

Consultation Deadline: 5th March, 2018

Medicinal products intended exclusively for markets outside the Community under Article 58 of Regulation (EC) No 726/2004
The European Medicines Agency have published: Regulatory and procedural guideline: European Medicines Agency procedural advice on medicinal products intended exclusively for markets outside the Community under Article 58 of Regulation (EC) No 726/2004 in the context of cooperation with the World Health Organization.

This document addresses a number of questions that users of Article 58 of Regulation (EC) No 726/2004 may have. It provides an overview of the EMA’s position on issues that are typically addressed in pre-submission meetings.
More information is available on the EMA’s website.

Estimands and sensitivity analysis in clinical trials
The European Medicines Agency have published: Scientific guideline: Draft ICH E9 (R1) addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials, step 2b – Revision 1, draft: consultation open.

This addendum presents a structured framework to link trial objectives to a suitable trial design and tools for estimation and hypothesis testing. This framework introduces the concept of an estimand, translating the trial objective into a precise definition of the treatment effect that is to be estimated. It aims to facilitate the dialogue between disciplines involved in clinical trial planning, conduct, analysis and interpretation, as well as between sponsor and regulator, regarding the treatment effects of interest that a clinical trial should address. More information is available on the EMA’s website.

Consultation Deadline: 28th February, 2018

Detection of toxicity to reproduction for human pharmaceuticals
The European Medicines Agency have published: Scientific guideline: Draft ICH S5 (R3) guideline on reproductive toxicology: detection of toxicity to reproduction for human pharmaceuticals, step 2b – Revision 3, draft: consultation open.

The purpose of this guideline is to provide key considerations for developing a testing strategy to identify hazard and characterize reproductive risk for human pharmaceuticals. The guidance informs on the use of existing data and identifies potential study designs to supplement available data to identify, assess, and convey risk. More information is available on the EMA’s website.

Consultation Deadline: 28th February, 2018

Dissolution specification for generic solid oral immediate release products with systemic action
The European Medicines Agency have published: Scientific guideline: Reflection paper on the dissolution specification for generic solid oral immediate release products with systemic action – First version.

This paper discusses the suitability of the dissolution method and the specifications for in vitro dissolution of orally administered generic drug products with immediate release characteristics. Where applicable, this reflection paper should be read in connection with the principles of relevant guidelines listed as references. More information is available on the EMA’s website.

Comments on dissolution specification for generic solid oral immediate release products with systemic action
The European Medicines Agency have published: Scientific guideline: Overview of comments received on ‘Draft reflection paper on the dissolution specification for generic solid oral immediate release products with systemic action’ – First version.

This document lists the comments submitted by interested parties (organisations or individuals) on the draft document as released for consultation, together with the responses of the EMA. More information is available on the EMA’s website.

Antibacterial therapies for patients with an unmet medical need for the treatment of serious bacterial diseases
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Antibacterial Therapies for Patients With an Unmet Medical Need for the Treatment of Serious Bacterial Diseases.”

This guidance is intended to assist sponsors in the clinical development of new antibacterial drugs. Specifically, the guidance explains the FDA’s current thinking about possible streamlined development programs and clinical trial designs for antibacterial drugs to treat serious bacterial diseases in patients with an unmet medical need, including patients who have a serious bacterial disease for which effective antibacterial drugs are limited or lacking. More information is available on the FDA’s website.

Data elements for transmission of individual case safety reports
The European Medicines Agency have published: Scientific guideline: International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use topic E 2 B (R3): Questions and answers: Data elements for transmission of individual case safety reports – Step 5, adopted.

This Q&A document provides clarifications for the harmonized interpretation of the E2B(R3) IG package and should be reviewed in conjunction with the IG package. This will facilitate the implementation of the electronic transmission of Individual Case Safety Reports (ICSRs) in the ICH regions. The sections of this Q&A document corresponds to the organization of the E2B(R3) IG. More information is available on the EMA’s website.

Allogenic stem cell-based products for veterinary use
The European Medicines Agency have published: Scientific guideline: Questions and Answers on allogenic stem cell-based products for veterinary use: Specific questions on extraneous agents, adopted.

Freedom from extraneous agents is a crucial aspect of quality evaluation of stem cell-based preparations. Therefore, appropriate acceptance criteria for starting and raw materials of human or animal origin need to be established. Following a review of the scientific information relating to stem cells, a number of areas have been identified that would benefit from further consideration by relevant experts and, where appropriate, the elaboration of specific guidance in the form of question and answer document (Q&A). Three specific questions for further consideration have been identified relating to freedom from extraneous agents aspects. More information is available on the EMA’s website.

Promotion of pharmacovigilance reporting
The European Medicines Agency have published: Scientific guideline: Reflection paper on promotion of pharmacovigilance reporting, adopted.

Experience to date within the regulatory network suggests that there may be an issue relating to under-reporting of adverse events associated with the use of veterinary medicinal products (VMPs) particularly with regard to use in food producing animals. This document will provide an overview of the different tools used by national competent authorities (NCAs) and the European Medicines Agency (EMA or the ‘Agency’) to date to promote pharmacovigilance (PhV) reporting. In addition, further activities that may be beneficial in increasing PhV reporting in general and particularly with regard to food producing animals will be examined. More information is available on the EMA’s website.

Generic Drug User Fee Amendments of 2012
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Generic Drug User Fee Amendments of 2012: Questions and Answers Related to Self-Identification of Facilities, Review of Generic Drug Submissions, and Inspections and Compliance.”

This guidance provides answers to questions arising from the implementation of the Generic Drug User Fee Amendments of 2012 (GDUFA) (Public Law 112-144, Title III), commonly referred to as GDUFA. The questions and answers (Q&A) format is intended to promote transparency and facilitate compliance by the generic drug industry. The first draft of this document was issued pursuant to 21 CFR 10.115 in August 2012 and a revised draft was issued in September 2013. FDA is issuing the final guidance as two separate guidances. This guidance includes questions and answers related to self-identification, review of generic drug submissions, and inspections and compliance. This final guidance clarifies some of the questions and answers included in the previous versions based on FDA’s experience in implementing GDUFA. More information is available on the FDA’s website.

Involvement of young patients/consumers within EMA activities
The European Medicines Agency have published: Regulatory and procedural guideline: Principles on the involvement of young patients/consumers within EMA activities, adopted.

The purpose of this document is to establish the principles for the involvement of young patients, consumers, and their carers, in the activities of the Agency’s scientific committees and working parties in a consistent and efficient manner, whenever such involvement is appropriate in the interest of the ongoing scientific assessment within a particular (scientific) committee. More information is available on the EMA’s website.

NORs, PARs, DSp and normal variability of process parameters
The European Medicines Agency have published: Scientific guideline: Questions and answers: improving the understanding of normal operating range (NOR), proven acceptable range (PAR), design space (DSp) and normal variability of process parameters.

This question and answer document is aimed at improving the understanding of NORs, PARs, DSp and normal variability of process parameters. More information is available on the EMA’s website.

European Union individual case safety report (ICSR) implementation guide
The European Medicines Agency have published: Regulatory and procedural guideline: European Union individual case safety report (ICSR) implementation guide, adopted.

This guidance specifies the technical requirements and the process of transmission of individual case safety reports (ICSRs) and is applicable to all stakeholders, which are exchanging ICSRs electronically within the European Economic Area. More information is available on the EMA’s website.

Selection and justification of starting materials for the manufacture of chemical active substances
The European Medicines Agency have published: Scientific guideline: Reflection paper on the requirements for selection and justification of starting materials for the manufacture of chemical active substances, adopted.

This reflection paper aims to clarify some of the expectations of EU competent authorities arising from the guidance found in ICH Q11 (Development and Manufacture of Drug Substances (Chemical Entities and Biotechnological/Biological Entities) regarding the information to be submitted in marketing authorisation dossiers to justify the selection of starting materials. Whilst ICH Q11 is not generally applicable to veterinary products, the principles outlined in this document should apply equally for active substances destined to treat both humans and animals. More information is available on the EMA’s website.

Development of non-substantially manipulated cell-based ATMPs
The European Medicines Agency have published: Regulatory and procedural guideline: Development of non-substantially manipulated cell-based advanced therapy medicinal products: flexibility introduced via the application of the risk-based approach.

This document aims to illustrate some of the possibilities and limitations of the risk-based approach using the example of an ATMP based on cells that have not been subjected to substantial manipulation and that are not intended for the same essential function: a de-novo development of autologous bone marrow or peripheral blood CD34+ cells for treatment of acute myocardial infarction. More information is available on the EMA’s website.

Gaucher disease: a strategic collaborative approach from the EMA and FDA
The European Medicines Agency have published: Scientific guideline: Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration, adopted.

This Strategic Collaborative Approach document is not a formal guidance; the purpose of this document is to facilitate paediatric drug development particularly in the field of Gaucher disease. The general principles presented should be viewed as suggestions only. More information is available on the EMA’s website.

Criteria to waive target animal batch safety testing for inactivated vaccines for veterinary use
The European Medicines Agency have published: Scientific guideline: VICH GL50: Harmonisation of criteria to waive target animal batch safety testing for inactivated vaccines for veterinary use – Revision 1, adopted.

The objective of this guideline is to provide internationally harmonized recommendations for criteria on data requirements to waive target animal batch safety testing of inactivated veterinary vaccines in regions where it is required. More information is available on the EMA’s website.

Date for coming into effect: 1st May, 2018

Criteria to waive target animal batch safety testing for live vaccines for veterinary use
The European Medicines Agency have published: Scientific guideline: VICH GL55: Harmonization of criteria to waive target animal batch safety testing for live vaccines for veterinary use – First version, adopted.

The objective of this guideline is to provide internationally harmonized recommendations for criteria on data requirements to waive target animal batch safety testing of live veterinary vaccines in regions where it is required. More information is available on the EMA’s website.

Date for coming into effect: 1st May, 2018

Allogenic stem cell-based products for veterinary use
The European Medicines Agency have published: Scientific guideline: Questions and Answers on allogenic stem cell-based products for veterinary use: specific questions on sterility, adopted.

Following a review of the scientific information relating to stem cells, a number of areas have been identified that would benefit from further consideration by relevant experts and, where appropriate, the elaboration of specific guidance in the form of a questions and answers document (Q&A). More information is available on the EMA’s website.

European Commission Safety Features Q&A document updated
The European Commission has published an updated version, Version 7, of the Questions and Answers document on the Safety Features for Medicinal Products for Human Use.

Version 7 of the document, dated June 2017, supersedes Version 5. Q&As 1.15, 1.16, 2.14, 2.15, 2.16, 5.4, 5.5 and 7.12 were added to the document, while Q&As 1.8, 3.4, 4.3 5.3, 7.11 and 8.1 were revised. More information is available on the Commission’s website.

Clinical evaluation of QT/QTc interval prolongation and proarrhythmic potential for non-antiarrhythmic drugs
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final revised guidance for industry entitled “E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs — Questions and Answers (R3).”

This guidance is a revision of the ICH guidance titled E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs – Questions and Answers (November 2008). More information is available on the FDA’s website.

Regulatory guidance for industry to prepare for the UK’s withdrawal from the EU
The European Medicines Agency (EMA) and the European Commission have published guidance to help pharmaceutical companies to prepare for the United Kingdom’s withdrawal from the European Union. The guidance relates to both human and veterinary medicines.

The questions-and–answers document concerns information related to the location of establishment of a company in the context of centralised procedures and certain activities, including the location of orphan designation holders, qualified persons for pharmacovigilance (QPPVs) and companies’ manufacturing and batch release sites. More information is available on the EMA’s website.

EMA 2016 Annual Report
The European Medicines Agency (EMA) has published its 2016 annual report.

The report focuses on the Agency’s key achievements in the areas of medicine evaluation, support to research and development of new and innovative treatments and the safety monitoring of medicines in real life. It also highlights some of the Agency’s main projects, initiatives and achievements in 2016, contains three interviews with stakeholders and EMA representatives on topics of major interest in the area of medicines and health in 2016 , and provides a large amount of data and figures that illustrate the work of EMA and its impact. More information is available on the EMA’s website.

Information guide for healthcare professionals on biosimilar medicines
The European Medicines Agency (EMA) and the European Commission have published an information guide for healthcare professionals on biosimilar medicines.

The objective of the guide is to provide healthcare professionals with reference information on both the science and regulation underpinning the use of biosimilars. More information is available on the EMA’s website.

Procedural advice for users of the centralised procedure for similar biological medicinal product applications
The European Medicines Agency have published: Regulatory and procedural guideline: European Medicines Agency procedural advice for users of the centralised procedure for similar biological medicinal product applications.

This document addresses a number of questions which users of the Centralised Procedure may have. It provides an overview of the EMA position on issues, which are typically addressed during the course of Pre-Submission Meetings. More information is available on the EMA’s website.

Preparation for Brexit by marketing authorisation holders of centrally authorised medicinal products
The European Commission and the European Medicines Agency have issued a “Notice to marketing authorisation holders of centrally authorised medicinal products for human and veterinary use”, reminding marketing authorisation holders of centrally authorised medicines of their legal obligations in preparation for Brexit.

More information is available on the EMA’s website.

Strategic approach to pharmaceuticals in the environment
The European Commission has published the roadmap “Strategic approach to pharmaceuticals in the environment.” The Roadmap aims to inform stakeholders about the Commission’s work in order to allow them to provide feedback and to participate effectively in future consultation activities

More information is available on the Commission’s website.

Publication of clinical data for medicinal products for human use
The European Medicines Agency have published: Regulatory and procedural guideline: External guidance on the implementation of the European Medicines Agency policy on the publication of clinical data for medicinal products for human use – Version 1.2, adopted.

The European Medicines Agency policy on the publication of clinical data for medicinal products for human use (hereafter referred to as ‘Policy 0070’) was developed by the European Medicines Agency (EMA), in accordance with Article 80 of Regulation (EC) No 726/2004. Policy 0070 was adopted by the EMA Management Board on 2nd October 2014 and subsequently published on the EMA website. More information is available on the EMA’s website.

Clinical data publication (CDP)
The European Medicines Agency have published: Regulatory and procedural guideline: Questions and answers (Q&As) on the external guidance of Policy 0070 on clinical data publication (CDP).

This page lists some questions that applicants/Marketing Authorisation Holders (MAHs) may have on the procedure for the publication of clinical data. It provides an overview of the European Medicines Agency’s position on issues that are typically addressed in discussions or meetings with applicants/MAHs. It will be updated regularly to reflect any new guidance updates during the implementation of Policy 0070. New or revised questions are marked with ‘New’ or ‘Rev’ together with the relevant date. More information is available on the EMA’s website.

Collaboration between the European Medicines Agency and academia
The European Medicines Agency have published: Regulatory and procedural guideline: Framework of collaboration between the European Medicines Agency and academia, adopted.

This framework aims to reinforce and further develop the collaboration between the European Medicines Agency (EMA hereafter) and academia by clarifying scope, formalising and structuring interactions in the wider context of the European regulatory system for medicines. More information is available on the EMA’s website.

Statistical methodology for the comparative assessment of quality attributes in drug development
The European Medicines Agency have published: Scientific guideline: Draft reflection paper on statistical methodology for the comparative assessment of quality attributes in drug development, draft: consultation open.

The reflection paper provides current regulatory considerations regarding statistical aspects for the comparative assessment of quality attributes in the settings of pre- and post-manufacturing change, biosimilar development as well as generics development. It raises open issues from a methodological perspective addressing questions related to comparison objectives, sampling strategies, sources of variability, acceptance ranges and statistical analysis approaches to conclude on the similarity of two drug products based on quality attribute data. A main objective of the reflection paper is to establish a framework and a common language to facilitate future discussion among stakeholders and to invite comments in relation to the issues raised. More information is available on the EMA’s website.

Consultation Deadline: 31st March, 2018

Safety features on the packaging of medicinal products for human use
The European Commission has published: Commission Delegated Regulation EU) 2016/161 of 2 October 2015 supplementing Directive 2001/83/EC of the European Parliament and of the Council by laying down detailed rules for the safety features appearing on the packaging of medicinal products for human use.

The delegated act detailing the characteristics of the safety features, how medicine authenticity should be verified, and by whom, was adopted on 2nd October 2015 and published, after scrutiny by the European Parliament and the Council, on 9th February 2016. To facilitate the implementation of the delegated Regulation, the Commission has also prepared a “Questions and Answers” document.

In addition, the regulatory requirements to be followed to notify the EMA of the placing of the unique identifier and/or the anti-tampering device on centrally authorised products are detailed in an implementation plan developed by the EMA and the European Commission and published in the “product information templates” section of the EMA website.

More information is available on the Commission’s website.

Date for coming into effect: 9th February, 2019