Regulatory news - February 2018

Keep up to date with regulatory news from around the world with the Cell and Gene Therapy Catapult regulatory newsletter.


European Medicines Agency (EMA)

Chimeric Antigen Receptor (CAR) T-cell therapy registries workshop

On the 9th Februray 2018, the EMA hosted a workshop on Chimeric Antigen Receptor (CAR) T-cell therapy registries. The main objectives of the workshop were:

  • To facilitate the long-term follow up of CAR-T cell products in a real world setting and enable the generation of meaningful efficacy and safety data using haemato-oncological registries.
  • To agree on implementable recommendations on core data elements to be collected, patient consent, governance, quality assurance and registry interoperability.
  • To agree on recommendations to optimise collaboration among registry holders, marketing authorisation holders and applicants and regulators.

Further information on the outcomes of this workshop will be published by the EMA in due course.

Click here to access to the agenda.

New report published covering EMA activities regarding 3Rs implementation on animal testing

EMA has published its first biennial report on efforts to Replace, Reduce and Refine (3Rs) the use of animals in regulatory testing of medicinal products. The report summarises the Agency’s actions (carried out by the Joint Committee for Medicinal Products for Human Use (CHMP)/Committee for Medicinal Products for Veterinary Use (CVMP) 3Rs Working Group (J3RsWG)).

The 3Rs principles should be integrated in all aspects of the development, manufacture and testing of medicine.

Whilst the EMA encourages developers to follow these principles whenever possible, it also acknlowedges that the use of animal models is still an important tool during drug development:

Although the ultimate aim is to replace the use of live animals in medicine testing, they continue to be necessary in some areas of medical research to protect human and animal health and the environment, until further scientific advancements enable the development of adequate alternatives.

Since its launch, the J3RsWG has contributed to the development of two guidelines and two reflection papers on 3Rs:

Click here to see the report in full.

EMA initiates building approval process for EMA premises in Amsterdam

On the 28th February, European Medicines Agency’s (EMA) Management Board voted on the revised offer of the Dutch government regarding the Agency’s new permanent premises in the business district Zuidas and endorsed the notification to the EU’s Budgetary Authority of EMA’s intention to move to the new building. The Dutch authorities have committed to building permanent premises to address EMA’s specific needs, and have confirmed to the Board that the permanent building will be ready by 15 November 2019.

Click here for more information.

European Directorate for the Quality of Medicines (EDQM)

Revision to monograph 2.6.27. microbiological examination of cell-based preparations

A draft revision to monograph 2.6.27. microbiological examination of cell-based preparations is currently under public consultation until 30 June 2018. It includes a clarification in suitability. This to avoid confusion between the term ‘validation’ and ‘confirmation of the suitability of the method’ for the automated growth-based method. The critical parameters described specificity (absence of false positive results), sensitivity, reproducibility and robustness are to be verified as part of confirmation of method suitability.

Click here to access to the draft revised monograph.

United Kingdom

Medicines and Healthcare products Regulatory Agency (MHRA)

50th Promising Innovative Medicine (PIM) designation granted

In 2014, MHRA launched the early access to medicines scheme (EAMS). The scheme aims to give patients with life threatening or seriously debilitating conditions access to medicines prior marketing authorisation when there is a clear unmet medical need.

This voluntary scheme is split into two phases:

  1. the promising innovative medicine (PIM) designation;
  2. the early access to medicines scientific opinion.

If a positive scientific opinion is granted, the scheme aims to accelerate patient access. This month, the EAMS scheme reached a milestone 50th PIM designation. 18 positive scientific opinions have been awarded since the scheme was launched.

Click here to see MHRA press release

UK and China sign Memorandum of Understanding on Medicine and Device Regulation

This month, the UK MHRA and the Chinese Food and Drug Administration (CFDA) have signed a memorandum of understanding (MoU) . This MoU aims to foster the exchange of safety information on medicines and medical devices. Dr Ian Hudson, Chief Executive Officer at MHRA recently said in statement:

China is a world leader in the market for raw materials for the pharmaceutical industry and closer collaboration with MHRA will support the promotion of innovation, good practice, and protect UK patients

Click here to see MHRA press release


Food and Drug Administration (FDA)

Public comments received on draft Regenerative Medicine Advanced Therapy guideline

As part of the 21st Century Cures Act, FDA announced a new policy framework for regenerative medicine advanced therapies (RMAT) in November 2017 which has been open for public consultation until 15th February 2018.

The objective of the RMAT designation is to accelerate the development of cell therapies (including gene-modified cells), therapeutic tissue engineering products, human cell and tissue products or any combination product using such therapies or products if it is “intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition” and “preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition”.

Comments received on the draft guideline Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, highlight the overlap between RMAT and breakthrough designation which raises the need for further clarification, notably the level data to obtain an RMAT versus the breakthrough designations.

All comments on this guidance are available to view here.

FDA to Launch New Pilot Program for Orphan Designation Requests

As part of the US Rare Disease Day, FDA announced a new pilot program to make the orphan designation request process more efficient. Despite the successes of the Orphan Drug Act (over the last 35 years, drugs and biologics have been developed and approved for 650 rare disease indications), there are still no treatments for the vast proportion of rare diseases or conditions. In response to that finding, FDA launched the Orphan Drug Designation Modernization Plan in June 2017. Since doing so, the FDA has eliminated the backlog of orphan drug designation requests, implemented a 90-day timetable for processing new designation requests and established an FDA Orphan Products Council to further address scientific and regulatory challenges pertaining to orphan products.

This month, FDA announced several additional actions including:

  • implementing a new orphan drug designation form accompanied by an on-line tutorial. According to FDA, the form should be easier for sponsors to complete designation requests; and make it more efficient for FDA to review.
  • entering into a new Memorandum of Understanding with the National Organization for Rare Disorders to conduct outreach with patient affairs staff to enhance the incorporation of patient experience into regulatory discussions
  • planning a public meeting on 9th May 2018 to help FDA to prepare for the changing landscape of orphan drug development shuch as cancer drugs and biologics that target a tumor’s specific genetic features rather than its location in the body.

Click here for more information

Workshop on use of Complex Innovative Designs (CID) in clinical trials

The FDA has announced it will be conducting a public workshop to discuss complex innovative designs (CID) in clinical trials of drugs and biological products on March 20th 2018.

The purpose of this public meeting is to:

  1. facilitate discussion and information sharing about the use of CID in drug development and regulatory decision making
  2. obtain input from stakeholders about the CID pilot program.

Click here for more information.

FDA adopts ICH Good Clinical Practice (GCP) Addendum

The GCP addendum, implemented since June 2017 in EU, is now available in the US with a guidance entitled “E6(R2) Good Clinical Practice: Integrated Addendum to E6(R1)”. According to the FDA, the revised guidance encourages implementation of improved and more efficient approaches to clinical trial design, conduct, oversight, recording, and reporting, and also updates standards regarding electronic records and essential documents. The guidance is intended to improve clinical trial quality and efficiency, while maintaining human subject protection and reliability of trial results.

Click here to access to the guideline.

Public consultations









Draft guidance on the health institution exemption (HIE) – IVDR and MDR

22 Jan 2018 to 31 Mar 2019

Draft guidance







Technical and Regulatory Considerations for Pharmaceutical Product Lifecycle Management

16 Nov 2017 to 18 Dec 2018

Draft guidance