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.

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.

Devices used with regenerative medicine advanced therapies
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled “Evaluation of Devices Used with Regenerative Medicine Advanced Therapies; Draft Guidance for Industry.”

The draft guidance document, when finalized, will provide device manufacturers, applicants, and sponsors engaged in the development of regenerative medicine therapies, with our current thinking regarding evaluation of devices used in the recovery, isolation or delivery of regenerative advanced therapies, which FDA generally refers to as “regenerative medicine advanced therapies” or “RMATs.” Specifically, as required by the 21st Century Cures Act (Cures Act), the draft guidance addresses how FDA intends to simplify and streamline its application of regulatory requirements for combination device and cell or tissue products; what, if any, intended uses or specific attributes would result in a device used with a regenerative therapy product to be classified as a class III device; when a device may be limited to a specific intended use with only one particular type of cell; and application of the least burdensome approach to demonstrate how a device may be used with more than one cell type. More information is available on the Federal Register’s website.

Consultation Deadline: 15th February, 2018

Assessing user fees Under the Biosimilar User Fee Amendments of 2017
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Assessing User Fees Under the Biosimilar User Fee Amendments of 2017.”

This draft guidance concerns FDA’s implementation of the Biosimilar User Fee Amendments of 2017 (BsUFA II) and certain intended changes in policies and procedures surrounding its application. More information is available on the Federal Register’s website.

Consultation Deadline: 16th January, 2018

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

S5(R3) detection of toxicity to reproduction for human pharmaceuticals
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance entitled “S5(R3) Detection of Toxicity to Reproduction for Human Pharmaceuticals.”

The draft guidance was prepared under the auspices of the International Council for Harmonisation (ICH), formerly the International Conference on Harmonisation. The draft guidance replaces the existing guidance entitled “S5(R2) Detection of Toxicity to Reproduction for Human Pharmaceuticals.” The draft guidance is intended to align with other ICH guidances, elaborate on concepts to consider when designing studies, and identify potential circumstances in which a risk assessment can be made based on preliminary studies. It also clarifies the qualification and potential use of alternative assays. More information is available on the Federal Register’s website.

Consultation Deadline: 12th 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.

Pre-submission of facility information related to prioritized generic drug applications
The Food and Drug Administration (FDA or Agency) is announcing the availability of a revised draft guidance for industry entitled “ANDAs: Pre-Submission of Facility Information Related to Prioritized Generic Drug Applications (Pre-Submission Facility Correspondence).”

FDA is revising the draft guidance because, after issuance of the original draft guidance, the Federal Food, Drug, and Cosmetic Act (the FD&C Act) was amended by the FDA Reauthorization Act of 2017, which resulted in changes to the pre-submission of facility information. Pre-submitting facility information enables the Agency to determine whether inspection of a facility is necessary and, if so, to begin inspection planning in advance of an abbreviated new drug application (ANDA) receipt. More information is available on the Federal Register’s website.

Consultation Deadline: 5th January, 2018

Controlled correspondence related to generic drug development
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Controlled Correspondence Related to Generic Drug Development.”

This guidance provides information regarding the process by which generic drug manufacturers and related industry can submit controlled correspondence to FDA requesting information related to generic drug development and the Agency’s process for providing communications related to such correspondence. This guidance also describes the process by which generic drug manufacturers and related industry can submit requests to clarify ambiguities in FDA’ controlled correspondence response and the Agency’s process for responding to those requests. This draft guidance revises the guidance for industry “Controlled Correspondence Related to Generic Drug Development” issued in September 2015. More information is available on the Federal Register’s website.

Consultation Deadline: 2nd January, 2018

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.

Definition of the concept ‘similar medicinal product’
The European Commission has launched a public consultation on a draft regulation amending Regulation (EC) No 847/2000 as regards the definition of the concept ‘similar medicinal product’.

Commission Regulation (EC) No 847/20002 provides a definition of the concept ‘similar medicinal product’, which includes specific cases defining what kind of products are to be regarded as similar for the purposes of the application of Article 8 of Regulation (EC) No 141/2000. That definition should be updated in the light of new scientific and technical knowledge, in particular, due to major developments in the field of biological medicines, and especially advanced therapy medicinal products, and in the light of experience gained with regard to the designation and regulation of orphan medicinal products. More information is available on the European Commission’s website.

Consultation Deadline: 27th November, 2017

Clinical investigation of medicinal products for the treatment and prophylaxis of venous thromboembolic disease
The European Medicines Agency have published: Scientific guideline: Concept paper on the need for a paediatric addendum of the guideline on clinical investigation of medicinal products for the treatment and prophylaxis of venous thromboembolic disease, draft: consultation open.

A number of new European Medicines Agancy guidelines related to clinical investigation of medicinal products for the treatment and prophylaxis of venous thromboembolism (VTE) are already available [1,2,3], but recommendations are applicable only to adults. In contrast to adults, VTE in children is a rare event, but represents a significant management dilemma that requires therapeutic intervention. A paediatric addendum to the guidelines on clinical investigation of medicinal products for the treatment and prophylaxis of VTE is considered necessary to discuss and make methodological recommendations adapted to children. More information is available on the EMA’s website.

Consultation Deadline: 31st January, 2018

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

Clinical investigation of recombinant and 4 human plasma-derived factor VIII products
The European Medicines Agency have published: Scientific guideline: Draft guideline on clinical investigation of recombinant and 4 human plasma-derived factor VIII products, draft: consultation open.

This guideline describes the information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor VIII products is made for use in treatment and prevention of bleeding in patients with haemophilia A. The guidance covers clinical investigations to be conducted pre- and post-marketing authorisation. Guidance is also provided for authorised products where a significant change in the manufacturing process has been made. More information is available on the EMA’s website.

Consultation Deadline: 31st January, 2018

Core SmPC for human plasma derived and recombinant coagulation factor VIII products
The European Medicines Agency have published: Scientific guideline: Draft guideline on core SmPC for human plasma derived and recombinant coagulation factor VIII products, draft: consultation open.

This guideline describes the information to be included in the Summary of Product Characteristics (SmPC) for human plasma derived and recombinant coagulation factor VIII products, which are indicated for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency). In case of an indication claim in von Willebrand’s disease, see also core SmPC for von Willebrand factor products (CPMP/BPWG/278/02). More information is available on the EMA’s website.

Consultation Deadline: 31st January, 2018

Acceptance review for de novo classification requests
The Food and Drug Administration (FDA or Agency) is announcing the availability of the draft guidance entitled “Acceptance Review for De Novo Classification Requests.”

The purpose of this draft guidance is to explain the procedures and criteria FDA intends to use in assessing whether a request for an evaluation of automatic class III designation (De Novo classification request or De Novo request) meets a minimum threshold of acceptability and should be accepted for substantive review. This draft guidance discusses De Novo acceptance review policies and procedures, “Refuse to Accept” principles, and the elements of the De Novo Acceptance Checklist and the Recommended Content Checklist and is being issued to be responsive to an explicit deliverable identified in the Medical Device User Fee Amendments of 2017 (MDUFA IV). More information is available on the Federal Register’s website.

Consultation Deadline: 29th December, 2017

User fees under the Generic Drug User Fee Amendments of 2017
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Assessing User Fees Under the Generic Drug User Fee Amendments of 2017.”

This draft guidance provides stakeholders information regarding the implementation of the Generic Drug User Fee Amendments of 2017 (GDUFA II) and policies and procedures surrounding its application. More information is available on the Federal Register’s website.

Consultation Deadline: 29th December, 2017

Review and revision of European Union herbal monographs and European Union list entries
The European Medicines Agency have published: Scientific guideline: Draft procedure for the review and revision of European Union herbal monographs and European Union list entries, draft: consultation open.

The purpose of this procedure is to enable a consistent and proportionate process in reviewing, and revising all European Union herbal monographs and European Union list entries adopted by the HMPC. The aim of this document is to describe how to identify the criteria/reasons to trigger the revision of European Union herbal monographs and European Union list entries and the associated procedure and timelines for both the review and the revision. Revision 1 pertained to clarify that minor changes of wording without safety implications should not trigger a revision of an European Union list entry. Revision 2 pertains to streamline the review and revision of European Union herbal monographs and list entries. In particular, the revision aimed for improved clarity and transparancy by covering detailed guidance on the review process, including a new review template (i.e. Annex 1). In addition, the procedure for unscheduled review, i.e. review for specific reason in the Reflection paper on the reasons and timelines for revision of final European Union herbal monographs and European Union list entries (EMA/HMPC/326440/2007), has been included in the revision. More information is available on the EMA’s website.

Consultation Deadline: 27th January, 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.

In vitro and clinical drug interaction studies
The Food and Drug Administration (FDA or Agency) is announcing the availability of two draft guidances for industry entitled “In Vitro Metabolism- and Transporter-Mediated Drug-Drug Interaction Studies” (in vitro DDI guidance) and “Clinical Drug Interaction Studies—Study Design, Data Analysis, and Clinical Implications” (clinical DDI guidance).

These two draft guidances will update and replace the revised draft guidance for industry entitled “Drug Interaction Studies—Study Design, Data Analysis, Implications for Dosing, and Labeling Recommendations” issued February 21, 2012 (2012 draft guidance). These draft guidances are intended to assist drug developers in the planning and evaluation of drug-drug interaction (DDI) potential during drug development. In particular, the in vitro DDI guidance focuses on in vitro experimental approaches for evaluating metabolizing enzyme- and transporter-based drug interaction potential and how to extrapolate in vitro data to decide on the need for clinical DDI studies. The clinical DDI guidance focuses on clinical studies that evaluate the potential for DDIs, which alter a drug’s pharmacokinetics by modulating the effects of drug metabolizing enzymes and transporters, and advises sponsors on the timing and design of the clinical studies, interpretation of the results, and options for managing DDIs in patients. Together, these two draft guidances describe a systematic, risk-based approach to the assessment of DDIs. More information is available on the Federal Register’s website.

Consultation Deadline: 23rd January, 2018

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.

Post-complete response letter meetings between FDA and ANDA applicants Under GDUFA
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Post-Complete Response Letter Meetings Between FDA and ANDA Applicants Under GDUFA.”

This guidance is intended to clarify the criteria for granting post-complete response letter (CRL) meeting requests and the scope of discussions for granted meeting requests. This guidance provides procedures that will promote well-managed post-CRL meetings and help ensure that such meetings are scheduled and conducted in accordance with the time frames set forth in the GDUFA Reauthorization Performance Goals and Program Enhancements Fiscal Years 2018-2022 (GDUFA II Goals or Commitment Letter). More information is available on the Federal Register’s website.

Consultation Deadline: 15th December, 2017

Supplementary protection certificates (SPCs) and patent research exemptions
The European Commission has launched a public consultation on supplementary protection certificates (SPCs) and patent research exemptions.

The Single Market Strategy, adopted in October 2015, announced that the Commission will ‘consult, consider and propose further measures, as appropriate, to improve the patent system in Europe, notably for pharmaceutical and other industries whose products are subject to regulated market authorisations’. In particular, the Strategy aimed to explore a recalibration of certain aspects of patent and supplementary protection certificate (SPC) protection, and announced that this recalibration could comprise the following 3 elements: the creation of a European SPC title, an update of the scope of EU patent research exemptions, and the introduction of an SPC manufacturing waiver. With this consultation the Commission seeks the views of stakeholders on the SPC and patent research exemption of SPCs. More information is available on the European Commission’s website.

Consultation Deadline: 4th January, 2018

Use of extrapolation in the development of medicines for paediatrics
The European Medicines Agency have published: Scientific guideline: Reflection paper on the use of extrapolation in the development of medicines for paediatrics, draft: consultation open.

This reflection paper aims to provide guidance to applicants and assessors on the main regulatory requirements that are expected to be met for the evaluation of extrapolation approaches in development of medicines for children. However, indicating preferences for the use of particular quantitative methods to address specific objectives of paediatric development is not within the scope of this document. The principles outlined should encourage further exploration of potentially suitable methods for specific situations, and choice of strategies should be justified. More information is available on the EMA’s website.

Consultation Deadline: 14th January, 2018

Determining whether to submit an ANDA or a 505(b)(2) application
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Determining Whether to Submit an ANDA or a 505(b)(2) Application.”

This guidance is intended to serve as a foundational guidance to assist applicants in determining which one of the abbreviated approval pathways under the Federal Food, Drug, and Cosmetic Act (the FD&C Act) is appropriate for the submission of a marketing application to FDA. More information is available on the Federal Register’s website.

Consultation Deadline: 12th December, 2017

Assessing user fees under the Prescription Drug User Fee Amendments of 2017
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Assessing User Fees Under the Prescription Drug User Fee Amendments of 2017.”

This draft guidance concerns FDA’s implementation of the Prescription Drug User Fee Amendments of 2017 and certain proposed changes in policies and procedures surrounding its application. More information is available on the Federal Register’s website.

Consultation Deadline: 12th December, 2017

Format and content of a REMS document
The Food and Drug Administration (FDA or Agency) is announcing the availability of a revised draft guidance for industry entitled “Format and Content of a REMS Document.”

A Risk Evaluation and Mitigation Strategy (REMS) document, which is part of a REMS that is required by FDA, establishes the goals and requirements of the REMS. This revised draft guidance describes a new recommended format for a REMS document. The new format was developed based on extensive stakeholder feedback. This guidance revises and supersedes the draft guidance entitled “Format and Content of Proposed Risk Evaluation and Mitigation Strategies (REMS), REMS Assessments, and Proposed REMS Modifications,” that was published by FDA on October 1, 2009. More information is available on the Federal Register’s website.

Consultation Deadline: 11th December, 2017

Requests for reconsideration at the division level Under GDUFA
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Requests for Reconsideration at the Division Level Under GDUFA.”

This guidance provides recommendations for industry on the procedures for resolving scientific and/or regulatory issues or matters between FDA and applicants of abbreviated new drug applications (ANDAs) that wish to pursue a request for reconsideration within the review discipline at the division level or original signatory authority. This guidance also provides information for applicants to consider before pursuing a request for reconsideration, procedures for submitting a request for reconsideration, and the Agency’s process for responding to those requests. More information is available on the Federal Register’s website.

Consultation Deadline: 11th December, 2017

Developing antiviral drugs for prophylaxis and treatment of RSV infection
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Respiratory Syncytial Virus Infection: Developing Antiviral Drugs for Prophylaxis and Treatment.”

The purpose of this draft guidance is to assist sponsors in all phases of antiviral drug development for prophylaxis and treatment of disease caused by respiratory syncytial virus (RSV) infection. More information is available on the Federal Register’s website.

Consultation Deadline: 11th December, 2017

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.

ANDAs for certain highly purified synthetic peptide drug products that refer to listed drugs of rDNA origin
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “ANDAs for Certain Highly Purified Synthetic Peptide Drug Products That Refer to Listed Drugs of rDNA Origin.”

The Federal Food, Drug, and Cosmetic Act (FD&C Act) permits any person to submit to the FDA an abbreviated new drug application (ANDA) to seek approval to market a generic version of a previously approved drug product. This draft guidance is intended to assist potential applicants in determining when an application for a synthetic peptide drug product (specifically glucagon, liraglutide, nesiritide, teriparatide, and teduglutide) that refers to a previously approved peptide drug product of recombinant deoxyribonucleic acid (rDNA) origin should be submitted as an ANDA rather than as new drug application (NDA). More information is available on the Federal Register’s website.

Consultation Deadline: 4th December, 2017

ANDA submissions — amendments to Abbreviated New Drug Applications Under GDUFA
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “ANDA Submissions—Amendments to Abbreviated New Drug Applications Under GDUFA.”

This draft guidance is intended to explain to applicants how the review goals established as part of the Generic Drug User Fee Amendments Reauthorization of 2017 (GDUFA II) apply to amendments to abbreviated new drug applications (ANDAs) and prior approval supplements (PASs) to FDA under the Federal Food, Drug, and Cosmetic Act (FD&C Act). This draft guidance describes amendment classifications and categories and explains how amendment submissions may affect an application’s review goal dates. The draft guidance also describes how FDA will review amendments submitted to ANDAs and PASs received prior to October 1, 2017, the effective date to implement the GDUFA II review goals. More information is available on the Federal Register’s website.

Consultation Deadline: 4th December, 2017

Formal meetings Between FDA and ANDA applicants of complex products Under GDUFA
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Formal Meetings Between FDA and ANDA Applicants of Complex Products Under GDUFA.”

This draft guidance describes an enhanced pathway for discussions between FDA and a prospective applicant preparing to submit (or an applicant that has submitted) to FDA an abbreviated new drug application (ANDA) for a complex product. Specifically, this draft guidance provides information on requesting and conducting product development meetings, pre-submission meetings, and mid-review-cycle meetings with FDA. This draft guidance will assist applicants in generating and submitting a meeting request and the associated meeting package to FDA for complex products to be submitted under the Federal Food, Drug, and Cosmetic Act (FD&C Act) and as contemplated in the commitments made by FDA in connection with the reauthorization of the Generic Drug User Fee Amendments for Fiscal Years (FYs) 2018-2022 (GDUFA II). More information is available on the Federal Register’s website.

Consultation Deadline: 4th December, 2017

ANDA submissions — refuse-to-receive standards
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “ANDA Submissions—Refuse-to-Receive Standards: Questions and Answers.”

This draft guidance is intended to assist applicants preparing to submit abbreviated new drug applications (ANDAs) and certain prior approval supplements (PASs) to ANDAs. This guidance provides answers to questions we have received from applicants regarding the guidance for industry, “ANDA Submissions—Refuse-to-Receive Standards” (RTR Standards guidance). The questions and answers address general issues about the organization of an ANDA, filing decisions made by FDA, the review of and deficiencies related to Drug Master Files (DMFs), product quality, and bioequivalence (BE) and clinical reviews, and are intended to clarify the deficiencies that may cause FDA to refuse to receive (RTR) an ANDA. More information is available on the Federal Register’s website.

Consultation Deadline: 4th December, 2017

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.

Oncology pharmaceuticals: reproductive toxicity testing and labeling recommendations
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Oncology Pharmaceuticals: Reproductive Toxicity Testing and Labeling Recommendations.”

The purpose of this guidance is to assist sponsors in reproductive toxicity assessments (mainly of embryo-fetal development) for oncology pharmaceuticals and to provide recommendations for product labeling on duration of contraception following cessation of therapy to minimize potential risk to a developing embryo/fetus. The guidance also clarifies FDA’s current thinking on when nonclinical studies for reproductive toxicology assessment may not be needed (e.g., for pharmaceuticals intended for use in postmenopausal women only). The intended outcome of this guidance is to provide for more consistent labeling for oncology pharmaceuticals and to reduce the conduct of nonclinical studies that are not informative on product use. More information is available on the Federal Register’s website.

Consultation Deadline: 28th November, 2017

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.

Statistical approaches to evaluate analytical similarity
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Statistical Approaches to Evaluate Analytical Similarity.”

This draft guidance, when finalized, will provide advice on the evaluation of analytical similarity to sponsors interested in developing biosimilar products. Specifically, this draft guidance describes the type of information a sponsor of a proposed biosimilar product should obtain about the structural/physicochemical and functional attributes of the reference product, how that information is used in the development of an analytical similarity assessment plan for the proposed biosimilar, and the statistical approaches recommended for evaluating analytical similarity. More information is available on the Federal Register’s website.

Consultation Deadline: 21st November, 2017

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

Applications for marketing authorisation/registration of well-established and traditional herbal medicinal products
The European Medicines Agency have published: Scientific guideline: Draft guideline on non-clinical documentation in applications for marketing authorisation/registration of well-established and traditional herbal medicinal products – Revision 1, draft: consultation open.

This guideline is intended to give advice for preparing and assessing applications for marketing authorisation of well-established herbal medicinal products and for the registration of traditional herbal medicinal products. It should be read in conjunction with the general requirements set out by Directive 2001/83/EC1, in particular its Annex I, and general methodological requirements published by the EMA. More information is available on the EMA’s website.

Consultation Deadline: 30th November, 2017

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.

Manufacture of the finished dosage form
The European Medicines Agency have published: Scientific guideline: Guideline on manufacture of the finished dosage form, adopted.

This guideline replaces the note for guidance on the manufacture of the finished dosage form (CPMP/QWP/486/95). The note for guidance has been updated to reflect the requirements as laid down in the current legislation Directive 2001/83/EC, and to follow the format and content of the Common Technical Document (CTD) Module 3 dossier. It also addresses current manufacturing practices in terms of complex supply chains and worldwide manufacture. In addition, the content and principles of the ICH Q8 guideline (ref 1) are also taken into account. More information is available on the EMA’s website.

Date for coming into effect: 14th February, 2018

Clinical development of new agents to treat pulmonary disease due to Mycobacterium tuberculosis
The European Medicines Agency have published: Scientific guideline: Addendum to the guideline on the evaluation of medicinal products indicated for treatment of bacterial infections to address the clinical development of new agents to treat pulmonary disease due to Mycobacterium tuberculosis – Revision 1, adopted.

This revision of the Addendum to the Note for guidance on the evaluation of medicinal products for treatment of bacterial infections to address the clinical development of new agents to treat disease due to Mycobacterium tuberculosis (EMA/CHMP/EWP/14377/2008 Rev. 1) has been produced in response to recent advances and changes in focus in the field. More information is available on the EMA’s website.

Date for coming into effect: 1st February, 2018

Pharmaceutical development of medicines for use in the older population
The European Medicines Agency have published: Scientific guideline: Reflection paper on the pharmaceutical development of medicines for use in the older population – First version, draft: consultation open.

The reflection paper describes aspects that medicines developers may consider when designing medicines for older people, such as selecting appropriate routes of administration and dosage forms, dosing frequency, excipients, container closure systems, devices and technologies, and user instructions in the product information. More information is available on the EMA’s website.

Consultation Deadline: 31st January, 2018

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.

Guideline on Influenza vaccines – Quality module
The European Medicines Agency have published: Scientific guideline: Guideline on Influenza vaccines – Quality module Revision 1, adopted.

The need to update the current guidelines regarding the quality, non-clinical and clinical development of influenza vaccines was recognised in the wake of the 2009-2010 influenza pandemic, as the Agency conducted its “lessons learnt” exercise. More information is available on the EMA’s website.

Date for coming into effect: 1st February, 2018

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.

Assessment and control of DNA reactive (mutagenic) impurities in pharmaceuticals to limit potential carcinogenic risk
The European Medicines Agency have published: Scientific guideline: International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use guideline M7 (R1) on assessment and control of DNA reactive (mutagenic) impurities in pharmaceuticals to limit potential carcinogenic risk – Step 5, adopted.

This guideline emphasizes considerations of both safety and quality risk management in establishing levels of mutagenic impurities that are expected to pose negligible carcinogenic risk. It outlines recommendations for assessment and control of mutagenic impurities that reside or are reasonably expected to reside in final drug substance or product, taking into consideration the intended conditions of human use. More information is available on the EMA’s website.

Date for coming into effect: 1st February, 2018

Off-label use of antimicrobials in veterinary medicine in the European Union
The European Medicines Agency have published: Scientific guideline: Draft reflection paper on off-label use of antimicrobials in veterinary medicine in the European Union, draft: consultation open.

This document intends to define off-label use and provide relevant examples of off-label use of antimicrobials in animals and the underlying reasons for these practices. The circumstances when off-label use is compatible with responsible use of antimicrobials will be explored. The goal is to identify and focus on areas that may cause unacceptable public and animal health risks due to dissemination of antimicrobial resistance. Off-label antimicrobial use in companion animals and food-producing animals will be addressed. More information is available on the EMA’s website.

Consultation Deadline: 31st January, 2018

First-in-human and early clinical trials with investigational medicinal products
The European Medicines Agency have published: Scientific guideline: Guideline on strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products – Revision 1, adopted.

This document addresses non-clinical and clinical issues for consideration prior to the first administration of an investigational medicinal product in humans. It also addresses the design and conduct of early clinical trials, including trials with integrated protocols. Emphasis is put on defining the uncertainty associated with the medicine tested at each step of the development and describing how the potential risks that might arise from this uncertainty will be addressed. The revision was made in cooperation with the EU Clinical Trials Facilitation Group (CTFG). More information is available on the EMA’s website.

Date for coming into effect: 1st February, 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 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