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.

Risk assessment requirements to control elemental impurities in veterinary medicinal products
The European Medicines Agency have published: Scientific guideline: Draft guideline on implementation of risk assessment requirements to control elemental impurities in veterinary medicinal products, draft: consultation open.

In order to allow time for regulators to elaborate guidance on the appropriate approach for a risk assessment for a veterinary medicinal product, the CVMP has adopted the following measured approach to the implementation of the monograph to existing veterinary products. The phased-in implementation of the risk assessment of elemental impurities in veterinary medicinal products is to be in accordance with the decision tree indicated in this document. More information is available on the EMA’s website.

Consultation Deadline: 16th November, 2017

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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Post-Complete Response Letter Meetings Between FDA and ANDA Applicants Under GDUFA.”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

Nonclincal study recommendations for microdose radiopharmaceutical diagnostic drugs
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Microdose Radiopharmaceutical Diagnostic Drugs: Nonclinical Study Recommendations.”

This draft guidance is intended to assist developers of microdose radiopharmaceutical diagnostic drugs on the nonclinical studies recommended to support human clinical trials and marketing authorization. The draft guidance discusses how to refine nonclinical study recommendations for this class of drug given its unique characteristics. This draft guidance is intended to provide recommendations for a pathway to full drug development (marketing authorization) for microdose radiopharmaceutical diagnostic drugs. More information is available on the Federal Register’s website.

Consultation Deadline: 13th November, 2017

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

Identifying trading partners under the Drug Supply Chain Security Act
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Identifying Trading Partners Under the Drug Supply Chain Security Act” (draft trading partner guidance).

FDA is issuing this guidance to assist industry and State and local governments in understanding how to categorize the entities in the drug supply chain in accordance with the Drug Supply Chain Security Act (DSCSA). This guidance explains how to determine when certain statutory requirements will apply to entities that may be considered trading partners in the drug supply chain. FDA is also soliciting public input specific to the activities of “private-label distributors” of drug products and whether those activities fall within the definitions under DSCSA of the various trading partners. More information is available on the Federal Register’s website.

Consultation Deadline: 23rd October, 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

Non-clinical evaluation of radiopharmaceuticals
The European Medicines Agency have published: Scientific guideline: Concept paper on the development of guidance on the non-clinical evaluation of radiopharmaceuticals – First version, draft: consultation open.

This concept paper is intended to seek external stakeholder views on potential guidance development related to overarching considerations for non-clinical data in support of clinical development and approval of radiopharmaceuticals. This guidance is intended to complement currently available guidelines (such as ICH M3(R2), ICH S6(R1), ICH S9 or the EMA Guideline on strategies to identify and mitigate risks for first-in-human clinical trials with investigational medicinal products) and includes the different intended uses of radiopharmaceuticals including radiodiagnostics as well as radiotherapeutics. The paper will focus on opportunities to targeted non-clinical programs according to specific development settings and product types and is not intended to duplicate guidance related to dosimetry. More information is available on the EMA’s website.

Consultation Deadline: 31st October, 2017

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.

Predictive biomarker-based assay development in the context of drug development and lifecycle
The European Medicines Agency have published: Scientific guideline: Concept paper on predictive biomarker-based assay development in the context of drug development and lifecycle, draft: consultation open.

The proposed concept paper is intended to be developed into a guideline which will replace the reflection paper on co-development of pharmacogenomic biomarkers and assays in the context of drug development. The guideline will provide recommendations on the interface between predictive biomarker-based assays including companion diagnostic and the development and lifecycle of a medicine. More information is available on the EMA’s website.

Consultation Deadline: 15th November, 2017

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

Use of aminoglycosides in animals in the European Union
The European Medicines Agency have published: Scientific guideline: Draft reflection paper on use of aminoglycosides in animals in the European Union: development of resistance and impact on human and animal health, draft: consultation open.

In April 2013, the European Commission (EC) requested advice from the European Medicines Agency (EMA) on the impact of the use of antibiotics in animals on public and animal health and measures to manage the possible risk to humans. The advice was provided by the Antimicrobial Advice ad hoc Expert Group (AMEG). As part of the advice, the AMEG provided a categorisation of antimicrobials according to their risk for public health. This CVMP/AWP reflection paper considers a recommendation from the AMEG for further risk profiling to be undertaken for the aminoglycosides (AGs) to enable them to be placed within the AMEG’s categorisation. More information is available on the EMA’s website.

Consultation Deadline: 31st October, 2017

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.

Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells
The European Medicines Agency have published: Scientific guideline: Concept paper on the revision of the guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells – Superceding document, draft: consultation open.

This Concept Paper proposes a revision of the Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells (EMA/CAT/GTWP/671639/2008) that came into effect in 2012. The guideline covers all cases of genetically modified cells intended for use in humans, independent of whether the genetic modification has been carried out for clinical indication (i.e. gene therapy medicinal products), for manufacturing purposes or any other reason. The genetically modified cells can be of human origin (autologous or allogeneic) or animal origin (xenogeneic cells), either primary or established cell lines. Genetically modified cells of bacterial origin are excluded from the scope of this guideline. The revised guideline referred to in this concept paper will replace guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells (EMA/CAT/GTWP/671639/2008). More information is available on the EMA’s website.

Consultation Deadline: 31st October, 2017

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.

Bioequivalence studies for veterinary medicinal products
The European Medicines Agency have published: Scientific guideline: Draft revised guideline on the conduct of bioequivalence studies for veterinary medicinal products, draft: consultation open.

It is the objective of this guideline to specify requirements for the design, conduct, and evaluation of bioequivalence studies for pharmaceutical forms with systemic action. In addition, guidance is given on how in vitro data in specific cases may be used to allow bridging of safety and efficacy data. More information is available on the EMA’s website.

Consultation Deadline: 31st October, 2017

Immunological veterinary medicinal products intended for minor use or minor species (MUMS)/limited market
The European Medicines Agency have published: Scientific guideline: Guideline on data requirements for immunological veterinary medicinal products intended for minor use or minor species (MUMS)/limited market – Revision 3, adopted.

In order to stimulate the development of new veterinary medicines intended for minor uses or minor species (MUMS)/limited market the CVMP developed a guideline on data requirements for MUMS/limited market for immunological veterinary medicinal products (IVMPs). This MUMS guideline has now been reviewed and revised with the aim of updating the acceptable data requirements in light of experience gained and clarifying, where appropriate, the applicability of these data requirements. More information is available on the EMA’s website.

Date for coming into effect: 1st November, 2017

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

Qualified Person Declaration
The Heads of Medicines Agencies have published: CMDh Questions and Answers, QP Declaration, Revision 2.

The CMDh agreed an update of the Q&A document on QP declaration. A new Q&A has been added to clarify the need for QP declaration(s) in support of certain changes to a Marketing Authorisation, to confirm that the active substance is manufactured in accordance with the detailed guidelines on good manufacturing practice for starting materials. More information is available on the CMDh website.

Implementation of the Falsified Medicines Directive
The Heads of Medicines Agencies have published: CMDh clarifications on questions received on the implementation of the Falsified Medicines Directive.

Following the receipt of several queries on the implementation of the Falsified Medicines Directive over the last year, the CMDh has agreed to publish, for transparency reasons, the feedback given to those queries. More information is available on the CMDh website.

European Commission report on the summary of product characteristics and the package leaflets
The European Commission has published a report to the European Parliament and the Council on the summary of product characteristics and the package leaflets.

This report was prepared pursuant to Article 59(4) of Directive 2001/83/EC according to which the Commission shall present to the European Parliament and the Council an assessment report on current shortcomings in the summary of product characteristics and the package leaflet and how they could be improved in order to better meet the needs of patients and healthcare professionals. More information is available on the Commission’s website.

Hypertension indication: drug labeling for cardiovascular outcome claims
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Hypertension Indication: Drug Labeling for Cardiovascular Outcome Claims.”

This guidance is intended to assist applicants in developing labeling for cardiovascular outcome claims for drugs that are indicated to treat hypertension. With few exceptions, current labeling for antihypertensive drugs includes only the information that these drugs are indicated to reduce blood pressure; the labeling does not include information on the clinical benefits related to cardiovascular outcomes expected from such blood pressure reduction. However, blood pressure control is well established as beneficial in preventing serious cardiovascular events, and inadequate treatment of hypertension is acknowledged as a significant public health problem. The Food and Drug Administration (FDA) believes that the appropriate use of these drugs can be encouraged by making the connection between lower blood pressure and improved cardiovascular outcomes more explicit in labeling. This guidance recommends standard labeling for antihypertensive drugs except where differences in labeling are supported by clinical data. More information is available on the FDA’s website.

Implementation strategy of ICH Q3D guideline
The European Medicines Agency have published: Scientific guideline: Implementation strategy of ICH Q3D guideline, adopted.

The purpose of this document is to address specific considerations to enable the practical implementation of ICH Q3D Guideline for Elemental Impurities in the European Union. It is intended to provide guidance for Applicants/MAHs, drug product, drug substance and excipient manufacturers, as well as regulators. In addition to new applications, it will also apply to variations to existing authorised medicinal products. More information is available on the EMA’s website.

Scientific data for use in HMPC assessment work
The European Medicines Agency have published: Regulatory and procedural guideline: Procedure for calls for scientific data for use in HMPC assessment work, adopted.

In accordance with Directive 2001/83/EC as regards traditional herbal medicinal products through Directive 2004/24/EC, it is the task of the Committee on Herbal Medicinal Products (HMPC) to establish a list of herbal substances, preparations and combinations thereof for use in traditional herbal medicinal products (Article 16f). Furthermore, the HMPC shall establish European Union herbal monographs for herbal medicinal products (Article 16h). The HMPC has also undertaken to ensure that monographs and list entries remain up-to-date (scientific state of the art) by assessing regularly the need for their revision. The legislation does not specify how scientific data relating to the assessment work for the monographs and list entries should be identified and compiled. The HMPC discussed a number of possibilities and agreed on a practice subject to 3 conditions. More information is available on the EMA’s website.

Updated EU-US agreement on mutual recognition of inspections of medicine manufacturers
The European Commission has published the full text of an agreement adopted between the European Commission and the United States Food and Drug Administration to mutually recognise inspections of premises where medicines are produced.

With this update of the 1998 Agreement on good manufacturing practices, EU and US regulatory authorities will be able to rely on each other’s information as regards facilities in the EU or US that manufacture medicines and active pharmaceutical ingredients for the European and American markets. The agreement will not affect the process of approving medicines, as it focuses only on inspections of manufacturing sites. The enhanced cooperation with US regulatory authorities will improve the EU’s ability to identify and address problems at factories before they become a public health risk. It will also reduce the administrative burdens and costs facing pharmaceutical manufacturers, including smaller producers. More information is available on the Commission’s website.

Triggers for inspections of bioequivalence trials
The European Medicines Agency have published: Regulatory and procedural guideline: Guidance on triggers for inspections of bioequivalence trials: quick scan, adopted.

This document represents a non-exhaustive overview of issues, which are taken into account during the assessment phase. Identification of other triggers not mentioned in this document is possible. This list is to be considered a quick scan. The topics listed in this document are intended to assist the assessor in deciding on whether to consult or to seek input from their GCP inspectorate on the need for a GCP inspection and on the best way forward. More information is available on the EMA’s website.

Traditional herbal medicinal products and simplified registrations for homeopathic medicinal products
The European Medicines Agency have published: Regulatory and procedural guideline: Traditional herbal medicinal products and simplified registrations for homeopathic medicinal products: pharmacovigilance requirements and EudraVigilance access – Note for clarification.

The purpose of this document is to clarify pharmacovigilance requirements for traditional herbal medicinal products registered further to a simplified registration procedure (traditional use-registration) on the basis of Article 16a of Directive 2001/83/EC1 and homeopathic medicinal products registered further to the special, simplified registration procedure under Article 14(1) of Directive 2001/83/EC and the process for obtaining access to adverse reaction reports in EudraVigilance for traditional use herbal medicinal products. More information is available on the EMA’s website.

EMA Adaptive Pathways Workshop
The European Medicines Agency have published a report on the Adaptive Pathways Workshop – a meeting with stakeholders that was held at EMA on Thursday 8 December 2016.

The European Medicines Agency held this workshop in collaboration with the European Commission to gather the views and proposals from stakeholders on the adaptive pathways approach, in light of the practical experience gained during the pilot project EMA ran between March 2014 and August 2016, and to plan the next steps in the exploration of this concept. Adaptive pathways is a scientific concept of medicines development and data generation intended for medicines that address patients’ unmet medical needs. EMA will publish the presentations, video recording and a workshop report on this page. More information is available on the EMA’s website.

Replication competent endogenous retrovirus RD114 in starting materials and final products of feline and canine vaccines
The European Medicines Agency have published: Regulatory and procedural guideline: CVMP Risk Management Strategy – Managing the risk of the potential presence of replication competent endogenous retrovirus RD114 in starting materials and final products of feline and canine vaccines.

The Committee for Veterinary medicinal products (CVMP) established an ad hoc expert group (AHEG) in 2015 to assist in the development of a risk management strategy for the potential presence of replication competent RD114 in feline and canine vaccines. The AHEG was requested to reflect on any proposed risk mitigation measures and perform an impact assessment on the effect of these measures upon the availability of feline and canine vaccines. The risk management strategy has been elaborated in the light of newly available regulatory guidance and takes account of the most recent scientific data provided by manufacturers of canine and feline vaccines and in context with published literature. More information is available on the EMA’s website.

Oral explanations at CVMP
The European Medicines Agency have published: Regulatory and procedural guideline: Guidance to applicants / marketing authorisation holders on oral explanations at CVMP.

This document is intended to provide practical guidance to companies invited to give oral explanations to the Committee for Medicinal Products for Veterinary Use (CVMP); however, the same principles will usually apply for oral explanations at CVMP Working Parties and other CVMP Advisory Group meetings (irrespective of the type of application / procedure under discussion). More information is available on the EMA’s website.

Supplementary protection certificates and patent research exemptions for sectors whose products are subject to regulated market authorisations
The European Commission has published a roadmap for the Inception Impact Assessment “Optimising the Internal Market’s industrial property legal framework relating to supplementary protection certificates (SPC) and patent research exemptions for sectors whose products are subject to regulated market authorisations.”

More information is available on the Commission’s website.

Section 5.1 of the summary of product characteristics on pharmacodynamic properties for pharmaceutical products
The European Medicines Agency have published: Regulatory and procedural guideline: Question and answer on the information contained within section 5.1 of the summary of product characteristics on pharmacodynamic properties for pharmaceutical products.

This question and answer was developed to aid the writing or update of section 5.1 of the summary of product characteristics (SPC). These principles were agreed by the Committee for Medicinal Products for Veterinary Use (CVMP) and the Veterinary Coordination Group for MRP/DCP (CMDv) at their respective meetings in January 2017. More information is available on the EMA’s website.

Requirements for transactions with first responders
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Requirements for Transactions with First Responders under Section 582 of the Federal Food, Drug, and Cosmetic Act — Compliance Policy.”

This guidance addresses the compliance of dispensers that engage in transactions with first responders and the compliance of first responders with the provisions in section 582 of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 360eee-1) related to the exchange of product tracing information (i.e., transaction information, transaction history, and transaction statements) and verification. This guidance also addresses the compliance of trading partners that engage in transactions with first responders with the requirement for conducting business only with authorized trading partners. More information is available on the FDA’s website.

Dear Health Care Provider Letters
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry and FDA staff entitled “Dear Health Care Provider Letters: Improving Communication of Important Safety Information.”

DHCP letters are correspondence ― often in the form of a mass mailing from the manufacturer or distributor of a human drug or biologic or from FDA ― intended to alert physicians and other health care providers about important new or updated information regarding a human drug or biologic. This guidance provides recommendations on (1) when to issue a DHCP letter, (2) the types of information to include in a DHCP letter, (3) how to organize that information so that it is communicated effectively to health care providers, and (4) formatting techniques to make the information more accessible. More information is available on the FDA’s website.

European Surveillance of Veterinary Antimicrobial Consumption
The European Medicines Agency have published: Regulatory and procedural guideline: European Surveillance of Veterinary Antimicrobial Consumption (ESVAC) Vision and Strategy 2016-2020, adopted.

This document seeks to define the vision and strategy for the ESVAC activity for the period 2016-2020. Once finalised, this strategy will be used as the basis for the successor to the current ESVAC project plan 2013-2016 (EMA/42322/2013). The EU Medicines Agencies Network Strategy to 2020 (EMA/MB/151414/2015) includes support for ESVAC as an important element within Theme 2, Objective 4 ‘Focus on key public and animal health priorities including antimicrobial resistance’. Activities within the Network Strategy, including ESVAC, will be delivered through inclusion within the multi-annual work plans of both the Agency and the Heads of Medicines Agencies (HMA). More information is available on the EMA’s website.

Support for applications on Article 58
The European Medicines Agency have published: Regulatory and procedural guideline: Support for applications on Article 58.

Applicants have the opportunity for certain medicinal products intended exclusively for markets outside the European Union to apply for a scientific opinion from the European Medicines Agency (EMA), in cooperation with the World Health Organization (WHO). To this effect, the Agency supports the medicine development and registration processes from an early stage and offers regulatory mechanisms to help candidates for Article 58 applications as early as possible. Companies developing such medicinal products are invited to liaise with the EMA for their products to make full use of these opportunities. More information is available on the EMA’s website.

SmPC and Package Leaflet for nanocolloidal technetium (99mTc) albumin
The European Medicines Agency have published: Scientific guideline: Guideline on core SmPC and Package Leaflet for nanocolloidal technetium (99mTc) albumin – First version, adopted.

This guideline describes the information to be included in the Summary of Products Characteristics (SmPC) and Package Leaflet for nanocolloidal technetium (99mTc) albumin. More information is available on the EMA’s website.

Documents obtained or resulting from pharmacovigilance inspections
The European Medicines Agency have published: Regulatory and procedural guideline: Union guidance on record keeping and archiving of documents obtained or resulting from pharmacovigilance inspections, adopted.

The scope of this document is to provide high level principles for the record keeping and archiving of documents in relation to EU pharmacovigilance (PhV) inspections of marketing authorisation holders (MAHs) of medicinal products for human use carried out by the competent authorities of Member States of the European Union. More information is available on the EMA’s website.

Post-orphan medicinal product designation procedures
The European Medicines Agency have published: Regulatory and procedural guideline: Post-orphan medicinal product designation procedures: guidance for sponsors.

This guideline covers the information and procedures applicable to orphan designated products. More information is available on the EMA’s website.

Article 31 non-pharmacovigilance referrals
The European Medicines Agency have published: Regulatory and procedural guideline: Questions and answers on Article 31 non-pharmacovigilance referrals.

This guidance document addresses a number of questions which stakeholders, in particular the marketing authorisation holders (MAHs)/applicants, may have on an Article 31 non-pharmacovigilance referral procedure. It provides an overview of the European Medicines Agency’s (the Agency) practical and operational aspects with regards to the handling of Article 31 non-pharmacovigilance referral procedures. More information is available on the EMA’s website.

2016 Medical Gas Container-Closure Rule
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry (small entity compliance guide) entitled “2016 Medical Gas Container-Closure Rule Questions and Answers.”

This guidance is intended to help small businesses better understand and comply with recently issued regulations on current good manufacturing practice (CGMP) and labeling for medical gases. More information is available on the FDA’s website.

Abuse potential of drugs
The Food and Drug Administration (FDA or Agency) is announcing the availability of a guidance for industry entitled “Assessment of Abuse Potential of Drugs.”

This guidance is intended to assist sponsors of investigational new drugs and applicants for approval of a new drug in evaluating whether their new drug product has abuse potential. Specifically, this guidance provides recommendations for assessing the abuse potential of central nervous system (CNS)-active new drugs. Drug products with abuse potential generally contain drug substances that are active within the CNS and produce psychoactive effects such as euphoria and hallucinations. Thus, if a drug substance is CNS-active, the new drug product containing that drug substance will likely need to undergo a thorough assessment of its abuse potential and may be subject to control under the Controlled Substances Act (CSA). This guidance finalizes the draft guidance of the same name issued on January 27, 2010. More information is available on the Federal Register’s website.

Emergency use authorization of medical products
The Food and Drug Administration (FDA or Agency) is announcing the availability of a guidance for industry and other stakeholders entitled “Emergency Use Authorization of Medical Products and Related Authorities.”

The purpose of this guidance is to explain FDA’s current thinking on the authorization of the emergency use of certain medical products under certain sections of the Federal Food, Drug, and Cosmetic Act (the FD&C Act) as amended or added by the Pandemic and All-Hazards Preparedness Reauthorization Act of 2013 (PAHPRA). The provisions in PAHPRA include key legal authorities to sustain and strengthen national preparedness for public health, military, and domestic emergencies involving chemical, biological, radiological, and nuclear (CBRN) agents, including emerging infectious disease threats. More information is available on the Federal Register’s website.

Repackaging of certain human drug products by pharmacies and outsourcing facilities
The Food and Drug Administration (FDA or the Agency) is announcing the availability of a final guidance for industry entitled “Repackaging of Certain Human Drug Products by Pharmacies and Outsourcing Facilities.”

This guidance describes the conditions under which FDA does not intend to take action for violations of certain provisions of the Federal Food, Drug, and Cosmetic Act (the FD&C Act), when a State-licensed pharmacy, a Federal facility, or an outsourcing facility repackages certain human drug products. More information is available on the Federal Register’s website.

Current Good Manufacturing Practice requirements for combination products
The Food and Drug Administration (FDA) is announcing the availability of a final guidance for industry and FDA staff entitled “Current Good Manufacturing Practice Requirements for Combination Products.”

The guidance describes and explains the document on current good manufacturing practice (CGMP) requirements for combination products, which published in the Federal Register of January 22, 2013, and includes general considerations for CGMP compliance as well as analysis of hypothetical scenarios. More information is available on the Federal Register’s website.

Recommended warning for OTC acetaminophen-containing drug products
The Food and Drug Administration (FDA) is announcing the availability of a guidance for industry entitled “Recommended Warning for Over-the-Counter Acetaminophen-Containing Drug Products and Labeling Statements Regarding Serious Skin Reactions.”

This guidance is intended to inform manufacturers, members of the medical and scientific community, and other interested persons that at this time FDA does not intend to take action against the marketing of single- and combination-ingredient, acetaminophen-containing, nonprescription (commonly referred to as over-the-counter (OTC)) drug products bearing a warning as described in the guidance alerting consumers that the use of acetaminophen may cause severe skin reactions. More information is available on the Federal Register’s website.

Electronic drug product reporting for human drug compounding outsourcing facilities
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Electronic Drug Product Reporting for Human Drug Compounding Outsourcing Facilities Under Section 503B of the Federal Food, Drug, and Cosmetic Act.”

This guidance explains how facilities that elect to register with FDA as outsourcing facilities are to submit drug product reports, consistent with section 503B of the Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 353b). Section 503B of the FD&C Act provides that a facility that elects to register with FDA as an outsourcing facility must report to FDA certain information about the drugs compounded at that outsourcing facility in the form and manner that FDA may “prescribe by regulation or guidance.” This guidance describes who must report and what information they must provide and explains that drug compounding reports must be submitted in structured product labeling (SPL) format using FDA’s electronic submissions system. More information is available on the FDA’s website.

Prescription requirements for compounding human drug products
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Prescription Requirement Under Section 503A of the Federal Food, Drug, and Cosmetic Act.”

This guidance sets forth the FDA’s policy concerning certain prescription requirements for compounding human drug products for identified individual patients under section 503A of the Federal Food, Drug, and Cosmetic Act (FD&C Act or Act). It addresses compounding after the receipt of a prescription for an identified individual patient, compounding before the receipt of a prescription for an identified individual patient (anticipatory compounding), and compounding for office use (or office stock). More information is available on the FDA’s website.

Botanical drug development
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Botanical Drug Development.”

This guidance describes the Center for Drug Evaluation and Research’s (CDER’s) current thinking on appropriate development plans for botanical drugs to be submitted in new drug applications (NDAs) and specific recommendations on submitting investigational new drug applications (INDs) in support of future NDA submissions for botanical drugs. In addition, this guidance provides general information on the over-the-counter (OTC) drug monograph system for botanical drugs. Although this guidance does not intend to provide recommendations specific to botanical drugs to be marketed under biologics license applications (BLAs), many scientific principles described in this guidance may also apply to these products. More information is available on the FDA’s website.

Demonstration of biosimilarity to a reference product
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Clinical Pharmacology Data to Support a Demonstration of Biosimilarity to a Reference Product.”

This guidance is intended to assist sponsors with the design and use of clinical pharmacology studies to support a decision that a proposed therapeutic biological product is biosimilar to its reference product. This guidance pertains to those products—such as therapeutic biological products—for which pharmacokinetic (PK) and pharmacodynamic (PD) data are needed to support a demonstration of biosimilarity. Specifically, the guidance discusses some of the overarching concepts related to clinical pharmacology testing for biosimilar products, approaches for developing the appropriate clinical pharmacology database, and the utility of modeling and simulation for designing clinical trials. More information is available on the FDA’s website.

Principles of regulatory acceptance of 3Rs
The European Medicines Agency have published: Scientific guideline: Guideline on the principles of regulatory acceptance of 3Rs (replacement, reduction, refinement) testing approaches, adopted.

In accordance with Directive 2010/63/EU, the principle of the 3Rs (Replacement, Reduction and Refinement) needs to be considered when selecting testing approaches to be used for regulatory testing of human and veterinary medicinal products. A general overview is provided on implementation of 3Rs principles in this context. More information is available on the EMA’s website.

ANDA Submissions – Refuse-to-Receive Standards
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “ANDA Submissions – Refuse-to-Receive Standards.”

This guidance is intended to assist applicants preparing to submit to FDA abbreviated new drug applications (ANDAs) and prior approval supplements (PASs) to ANDAs for which the applicant is seeking approval of a new strength of the drug product. The guidance highlights deficiencies that may cause FDA to refuse to receive (RTR) an ANDA. An RTR decision indicates that FDA determined that an ANDA is not substantially complete. A substantially complete ANDA is “an ANDA that on its face is sufficiently complete to permit a substantive review.” More information is available on the FDA’s website.

Safety of residues of veterinary drugs in human food
The European Medicines Agency have published: Scientific guideline: VICH GL54 studies to evaluate the safety of residues of veterinary drugs in human food: general approach to establish an acute reference dose (ARfD), adopted.

The current guideline addresses the nature and types of data that can be useful in determining a toxicological acute reference dose (ARfD) for residues of veterinary drugs, the studies that may generate such data, and how the ARfD may be calculated based on these data. More information is available on the EMA’s website.

Date for coming into effect: 1st November, 2017

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