Regulatory Round-up - October 2022

EUROPE

European Directorate for the Quality of Medicines (EDQM)

Council of Europe takes steps to support self-sufficiency in tissues and cells for human application and facilitate sharing of knowledge

The Committee of Ministers of the Council of Europe has adopted a recommendation encouraging member states to harmonise the collection of data on the availability and use of tissues and cells according to a predefined set of parameters and definitions. At present, only a fragmented and incomplete picture of tissue and cell activity is available, at both national and European levels. Current efforts to collect relevant activity data are undermined by the lack of consensus and clarity on the data needed for different purposes, harmonisation of the terminology used, and a legal mandate to collect this type of data. The result is that data collection fails to achieve the desired goals but is a huge burden for the establishments entrusted with the task.

This text aims to support self-sufficiency in tissues and cells for human application and to facilitate data sharing across borders, with the ultimate goal of ensuring rational, fair, timely and equitable access to safe tissues and cells for human application.

The text of the recommendation is available on the Council of Europe Committee of Ministers’ website.

European Medicines Agency (EMA)

New EU data quality framework aims to align stakeholders

On the 10^th of October 2022, the joint Big Data Steering Group, created by the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA), has published the Data Quality Framework for EU medicines regulation. The purpose of this framework is to put stakeholders on the same page regarding data quality.

The draft Data Quality Framework for EU medicine regulation is now open for public consultation until the 18^th of November 2022.

First therapy to treat transplant patients with post-transplant lymphoproliferative disease

At its October 2022 meeting, EMA’s human medicines committee (CHMP) has recommended a marketing authorisation in the European Union (EU) for Ebvallo (tabelecleucel) for the treatment of adult and pediatric patients who have a serious complication following solid organ transplantation (SOT) or bone marrow transplantation (hematopoietic cell transplant - HCT) called EBV+ PTLD. Please find more details here.

Guideline on core SmPC, Labelling and Package Leaflet for advanced therapy medicinal products (ATMPs) containing genetically modified cells.

The guideline was first published on the 26^th of October 2022. It defines the information to be included in the summary of products characteristics (SmPC), labelling and package leaflet for advanced therapy medicinal products (ATMPs) containing genetically modified cells. This applies to allogeneic or autologous, including viral vector modified and genome edited cells.

Consultation on Good Practice Guide for the use of the Metadata Catalogue of Real-World Data Sources

EMA has published a good practice guide for the use of the metadata catalogue of real-world data sources for consultation. The catalogue is intended to facilitate the discovery of data sources to generate evidence for regulatory purposes and to support the assessment of study protocols.

In the draft guide, EMA and the Heads of Medicines Agencies describe how assessments of sources should consider quality in relation to both the reliability of the primary data and the relevance of the data source. EMA has provided a use case to describe a six-step process for identifying suitable data sources.

The draft is open for comment until 16 November 2022. EMA is aiming to release the catalogue late next year.

UNITED KINGDOM

Medicines and Healthcare products Regulatory Agency (MHRA)

Increasing the efficiency of your clinical trial authorisation (CTA) application and reducing the need for subsequent substantial amendments

In this Medicines and Healthcare products Regulatory Agency (MHRA) blog, information is provided on how to facilitate the review of initial CTA applications in order to gain increased chances of approval. Please find details here.

Unique opportunity for healthcare professionals to influence future MHRA safety communications and safety reporting systems

On the 13^th of October 2022, a 14-week public consultation was launched to enable healthcare professionals across the UK to have their say on how they wish to receive vital safety information, how they’d like to be engaged, and to feedback on the Yellow Card safety reporting system. Views expressed during the consultation will support healthcare professionals to deliver the best care to patients.

Please find detail about the consultation, including how to take part here.

NHS England

Accelerating genomic medicine in the NHS

On the 12th of October 2022, NHS England published the first ever NHS genomics strategy, signaling the next big step in healthcare in the NHS and the journey to realize the potential of genomics for patients and communities.

The published document sets out priorities for delivering on the NHS vision and ambition over the next 5 years and how the system will need to work together to improve outcomes for patients and support the continuing scientific and technological advances in genomics.

USA

Food and Drug Administration (FDA)

Human Gene Therapy for Neurodegenerative Diseases Guidance for Industry

The guidance, issued in October 2022 by the Center for Biologics Evaluation and Research, provides recommendations to sponsors developing human gene therapy (GT) products for neurodegenerative diseases affecting adult and pediatric patients.

This guidance finalises the draft guidance of the same title dated January 2021.

Comparability Protocols for Post approval Changes to the Chemistry, Manufacturing, and Controls Information in an NDA, ANDA, or BLA

On the 14^th of October 2022, the final guidance was published in the Federal Register.

This guidance is to assist original applicants and holders of approved new drug applications (NDAs), abbreviated new drug applications (ANDAs), and biologics license applications (BLAs) with implementing a chemistry, manufacturing, and controls (CMC) post approval change using a comparability protocol (CP).

A CP is a comprehensive, prospectively written plan for assessing the effect of proposed post approval CMC change(s) on the identity, strength, quality, purity, and potency of a drug product, including a biological product (i.e., product), as these factors may relate to the safety or effectiveness of the product (i.e., product quality).

OTAT Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls

On the 7^th of December 2022, the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) is hosting a virtual town hall to answer stakeholder questions related to cell therapy chemistry, manufacturing, and controls (CMC), including tissue-engineered medical products regulated by OTAT. This event is part of a series to answer questions from stakeholders about a variety of topics on which OTAT has regulatory oversight.

Please find details of this webinar here and the recording of the first event held on the 29^th of September 2022 here.

INTERNATIONAL

International Conference on Harmonisation (ICH)

ICH Guideline Q5A(R2) on viral safety evaluation of biotechnology products derived from cell lines of human or animal origin

The ICH Guideline Q5A(R2), which is currently under revision considers testing and evaluation of the viral safety of biotechnology products derived from characterised cell lines of human or animal origin. It outlines data that should be submitted in the marketing application/registration package.

ICH M11 draft Guideline reaches Step 2 of the ICH process

The ICH M11 draft Guideline on Clinical electronic Structured Harmonised Protocol (CeSHarP), Technical Specification and Template reached Step 2 of the ICH process on 27 September 2022.

This new guideline is proposed to provide a comprehensive clinical protocol organisation with a standardised content including a template presenting the formatting and structure of the protocol and a technical specification presenting the technical attributes that enable the interoperable electronic exchange of protocol content.

ARM and NIIMBL Release Project A-Cell to Bring Quality by Design Principles to Cell-Based Therapy Manufacturing

The Alliance for Regenerative Medicine (ARM) and the National Institute for Innovation in Manufacturing BioPharmaceuticals (NIIMBL) has published Project A-Cell, a multistakeholder collaboration to incorporate Quality by Design (QbD) principles into cell-based therapy CMC programs as well as organized a six-part live webinar series highlighting specific A-Cell chapters which will culminate in a workshop in June of 2023. Please find more information on these events here.

Public consultations

European medicines agency (EMA)

Medicines and Healthcare products Regulatory Agency (MHRA)

Food and Drug Administration (FDA)

International Conference on Harmonisation (ICH)

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