Regulatory news - April 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)

Brexit to trigger redistribution of centrally authorised products

In Europe, the centralised procedure allows a Marketing Authorisation Holder (MAH) to market a medicine across all EU Member States including Iceland, Liechtenstein and Norway on the basis of a single marketing authorisation application to the European Medicines Agency (EMA). Today, the great majority of new, innovative medicines pass through this procedure; ATMPs must be authorised in this way.

During the procedure, a rapporteur and a co-rappoteur from the National Competent Authority (NCA) (i.ethe MHRA for the United Kingdom) are appointed to coordinate the scientific assessment within the scope of the relevant committee.

Following the UK’s withdrawal from the European Union, the MHRA will no longer be able to engage in centralised procedure”.Consquently, the UK’s centrally authorised product (CAP) portfolio (over 370 procedures) will be transferred to new rapporteurs and co-rapporteurs from the EU27 Member States(MS) plus Iceland and Norway.

The redistribution of the UK CAP portfolio comprises UK rapporteurs and co-rapporteurs from Committee for Medicinal Products for Human Use (CHMP), Pharmacovigilance Risk Assessment Committee (PRAC) and Committee for Advanced Therapies (CAT).

According to the EMA, the methodology for the reallocation of medicines takes into account “both the diverse expertise in the European medicines regulatory network and the workload associated with each medicine”. This methodology is intended to allow Member States to participate in EMA activities according to their individual capacity and be easy to implement.

The new (co)-rapporteurships will be communicated to the relevant MAH before the end of April and are expected to take responsibility for the re-allocated products after 30 March 2019.

Click here for more information.

Upcoming workshop on pediatric medicine - 7–8 June 2018

The European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) will hold its tenth annual workshop on 7–8 June 2018 at the European Medicines Agency (EMA).

This year the annual workshop will discuss:

  • short perspectives of the various stakeholders involved in paediatric research (patient/young people advisory groups, research networks, learned societies, national paediatric associations/societies, ethics committees, regulators, HTA bodies and industry);
  • how to avoid duplication of trials, how to improve communication, how to divide work between groups and on the challenges of clinical trials in (ultra) rare diseases.

The registration is open until 28 May 2018.

Click here for more information.

Medicines and Healthcare Products Regulatory Agency (MHRA)

Publication of the MHRA Corporate plan 2018-2023

The MHRA has released its Corporate Plan for the next five years. This plan outlines the Agency’s longer-term objectives and defines a strategy for achieving themwith consideration to “ensure a smooth EU exit under any scenario.”

The new plan has retained the same five areas of improvement identified in the 2013-2018 plan, and the MHRA have subsequently turned these into objectives for 2018 through 2023:

  1. Public health and partnerships: protect public health and promote patient safety by ensuring the safety, efficacy and quality of medicines and health care products in the UK and internationally
  2. Enhancing innovation: support and enhance innovation and accelerate routes to market to benefit public health and be a magnet for life sciences
  3. Proactive, robust surveillance: deliver robust proactive surveillance for medicines and medical devices to achieve measurable public health benefit
  4. Secure global supply chains: ensure the safe production and supply of medicines and medical devices through
  5. Organisational excellence/efficiency: be an exemplar organisational excellence and efficiency

The MHRA plans to improve the use of real-world evidence data to deliver robust proactive surveillance for medicines and medical devices. Notably, as part of its objective to enhancing innovation, the MHRA outlines:

“support the development of Advanced Therapies Medicinal Products (ATMPs) and novel biologicals by providing useful standards and all-stage expert scientific advice”

The MHRA recognises “the growing importance of global standards” as products develop for global markets. As such, reinforcement of international partnerships and development of new standards for ATMP are part of MHRA’s goal.

Click here to access to the MHRA corporate plan in full.

UK aims to adopt EU Clinical Trial Regulation

The new clinical trials regulation (CTR) aims to streamline the clinical trial application process through a harmonised assessment procedure and single portal for all EU clinical trials as well as simplified reporting procedures. The CTR is is expected to comeinto force sometime during the potential Brexit transition period lasting until March 2020 although the final implementatio date will depend on the availability of the European IT portal currently in development.

In the statement to the Hosue of Lords, Baroness Goldie emphasised the UK’s interest in being included in the CTR should this be feasible stating:

If the CTR comes into force during the implementation period, it will apply to the UK. If this opportunity does not come to pass, the Government will seek to bring into UK law all relevant parts of the EU regulation that are within the UK’s control.

Click here to view the transcript from Baroness Goldie.

European Directorate for the Quality of Medicines (EDQM)

Ph. Eur. Commission reactivates its Gene Therapy Products (GTP) Working Party

The Ph. Eur. Commission decided recently to reactivate its dedicated GTP Working Party. Its main task will be:

  • to revise the general chapter “5.14 Gene transfer medicinal products for human use”,
  • to participate in the revision of chapter 2.6.35 “Quantification and characterisation of residual host cell DNA”,
  • to assess any other needs for revising general chapters or elaborating new Ph. Eur. texts related to gene therapy in consideration of the latest developments in the field.

Experts from both Ph. Eur. and non Ph. Eur. member states are welcome to participate in this work. Registration is open up to 4 May 2018.

Click here for more information.


New Guideline on Investigational In Vitro Diagnostics (IVD) in Oncology Trials

This new guideline describes an optional streamlined submission process for determining whether use of an investigational in vitro diagnostic (IVD) in a clinical trial for an oncology therapeutic is considered significant risk (SR), nonsignificant risk (NSR), or exempt.

The sponsor should submit all information about the oncology codevelopment programme (including information about the investigational IVD) to the appropriate center (Center for Biologics Evaluation and Research, CBER or Center for Drug Evaluation and Research, CDER). The guideline details how the information on the IVD should be presented in the Investigational New Drug application (IND) submission. The CBER or CDER will then work in close collaboration with the Center for Devices and Radiological Health (CDRH) to determine the risk.

This is a step toward our goal of having a common filing for a drug and diagnostic system where the drug is co-developed with a diagnostic test,” FDA Commissioner Scott Gottlieb said

Click here to access to the guideline.

Public consultations




Consultation period



Postmarketing Safety Reporting for Combination Products

21 Mar to 19 Jun 2018

Draft guidance



Consultation period



Technical and Regulatory Considerations for Pharmaceutical Product Lifecycle Management

16 Nov 2017 to 18 Dec 2018

Draft guidance