Regulatory news - November 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)

Public consultation on use of patient disease registries for regulatory purposes

The cross-committee task force on registries published this month a discussion paper on methodological and operational considerations in the use of patient disease registries for regulatory purposes. Gene therapy products are designed to achieve therapeutic effect through permanent or long-acting changes in the human body. As a result of long term exposure, patients may be at increased risk of undesirable and unpredictable outcomes which can potentially occur years after administration and appropriate long-term follow-up may be required. Patient registries can be an alternative to fulfil this requirement. This paper discusses methodological and operational aspects of patient disease registries. It focuses on important principles from a regulatory perspective and makes proposals on what might be considered good registry practice to support the collection of high quality registry data. This paper also discusses important methodological aspects of studies performed with registry data to increase regulators’ confidence in their results.

Interested parties can send their comments and suggestions by 30 June 2019. EMA will consider the responses and finalise the document in consultation with the relevant EMA committees by the end of 2019.

Click here to view the discussion paper

Workshop on how to better support medicine developers in the generation and preparation of quality data packages for PRIME and Breakthrough Therapy applications

The EMA hosted a workshop on 26 November 2018 on support to quality development in early access approaches. The aim is to discuss between Regulators (notably EMA, FDA) and Industry technical quality challenges and scientific and regulatory approaches that could be used to facilitate development and preparation of robust CMC data packages, enabling timely access to medicines whilst providing assurance that patient safety and product quality are not compromised. Since the launch of EMA’s PRIME scheme in 2016, and FDA’s Breakthrough Therapy designation programme in 2012, both programmes have succeeded in driving innovation but experience gained with these programmes has shown that applicants who are part of the scheme often face challenges in meeting the data requirements on quality and manufacturing development.

These general discussions will be further elaborated through a number of specific industry case studies (covering chemical molecules, biologicals and ATMPs) and a discussion of experiences to date from early access approaches.

The conclusions from the workshop will be captured in a report, which will be published. The development of further follow-up guidance may be considered.

Click here for further information

EMA updates guidance on publication of clinical data for medicinal products for human use

The EMA released a new guidance document summarizing the major and minor changes made to Policy 0070, publication of clinical data for medicinal products for human use.

There is now more clarification on the publication of withdrawn applications. For withdrawn applications, where there is a confirmed re-submission date or resubmission has already taken place, a delay in the publication of the application can be requested.

The guidance also provides more clarification on the publication of clinical studies where a study’s main period/phase of a clinical study is ongoing at the time of publication.

Click here for the guidance document.

EMA updates guidance on good pharmacovigilance practices (GVP)

The EMA published legislation for pharmacovigilance in July 2012. To support the implementation of this legislation, the EMA has published a set of guidelines focusing on Modules on pharmacovigilance processes and Product- or Population-Specific Considerations.

The guidance will supersede the 2007 Guideline on the Conduct of Pharmacovigilance for Medicines Used in the Pediatric Population.

Due to the number of changes in the scientific and regulatory environment, this has had an impact on the conduct of pharmacovigilance in the pediatric population. A public consultation was launched in August 2018 for new Product- or Population-Specific Considerations, in particular, the paediatric population. This was taken into account for Chapter P IV of the guidline. The guideline provides pediatric-focused recommendations for several pharmacovigilance processes including risk management plans, reporting age information, signal management, periodic safety update reports (PSURs) and safety communication.

Click here for guidance document.


Brexit Withdrawal Draft Agreement

A Draft Agreement on the withdrawal of the United Kingdom of Great Britain and Northern Ireland from the European Union and the European Atomic Energy Community (the Draft Withdrawal Agreement, 14 November 2018), detailing the arrangements for the UK to leave the EU has been agreed by the UK government and the EU. The Withdrawal Agreement sets out UK/EU obligations during a transition period from 29 March 2019 until at least the end of December 2020 and includes detail on people, goods on the market, medicines regulation, intellectual property, state aid rules, the Irish border and access to EU databases.

However, the draft withdrawal agreement has to be passed by UK Parliament before it can come into effect. Parliament will vote on the bill on the 11th of December,

Click here to view the Draft Withdrawal Agreement


ANSM implements Fast Track clinical trial authorization program

France’s medicines regulator, the ANSM, has set up a fast track programme for patients to access innovative treatments quicker. Clinical trial applications will expedited without compromising patient safety with processing times not exceeding 25 or 40 days depending on if it’s a new or known substance. The current regulations have processing times of 60 days.

The ANSM said. “This new system aims to shorten clinical trial authorization application processing times, prepare the ANSM for greater responsiveness in view of upcoming European regulations on clinical trials—coming into effect no later than 2020—and improve the quality and safety of the clinical trials proposed in submitted applications.”

Click here for further information


Food and Drug Administration (FDA)

FDA to follow EMA with move from London to Amsterdam

The FDA has decided to move their London offices to Amsterdam alongside the EMA to continue to facilitate collaboration.

Click here to view the article

FDA Facilitates the Use of Surrogate Endpoints in Drug Development

In order to enhance the use of surrogate endpoints (SEs) in drug development and facilitate the development of new and innovative products, the FDA has made additional resources available for drug development progammes intenting to use SEs.

Resources include a Surrogate Endpoint Table, summarizing SEs that the FDA has accepted or could accept as primary efficacy endpoints in drug development programmes.

Click here for more information

Public consultations



Consultation period


Evaluation of the legislation on medicines for children and rare diseases (medicines for special populations)

12 Oct 2018 to 4 Jan 2019

Public consultation





Consultation period


Technical and Regulatory Considerations for Pharmaceutical Product Lifecycle Management

16 Nov 2017 to 18 Dec 2018

Draft guidance