Regulatory news - March 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)

EMA workshop on paediatric regulation

Following the European Commission’s report covering 10 years of the EU Paediatric Regulation, the EMA held a workshop “toidentify and discuss potential improvements to the implementation of the European Union’s (EU) regulatory framework specific for paediatric medicines”. The regulation ensures that paediatric medicine development is considered as an integral part of the overall development of medicines.

These positive results do however not evenly spread among all therapeutic areas, but concentrate in some, often linked to research priorities in adults rather than children. Whilst the report confirmed an overall positive impact of the regulation, it also highlighted that the development in terms of agreed and completed Paediatric Investigational Plans (PIPs) does not necessarily correspond with the paediatric disease burden, which underlines the fact that paediatric medicine development is often driven by adult development.

For these reasons, the EMA and the European Commission held a workshop on 20th March 2018 to identify and discuss potential improvements to the Paediatric Regulation.

The main objectives were :

  • To learn from experiences and ideas regarding criteria and methodologies that could be used to identify diseases/conditions of unmet paediatric medical needs
  • To exchange ideas on measures to proactively address obstacles to timely completion of paediatric investigation plans (PIPs)
  • To identify operational challenges in relation to paediatric procedures and exchange ideas for process improvements
  • To inform stakeholders about ongoing and future initiatives of international collaboration of regulators for paediatric medicine development
  • To inform stakeholders about planned transparency measures regarding clinical research and new medicines for children

A report of the workshop and presentations from the event will be published on EMA’s website in due course.

Click here to view the EMA press release.

United Kingdom

Medicines and Healthcare products Regulatory Agency (MHRA)

UK guidance on ‘GxP’ data integrity and definitions

The document, developed based on fundamental failures identified by MHRA and international regulatory partners during inspections, provides guidance on the data integrity expectations that should be considered by organisations across all GxP sectors including Good Laboratory Practice (GLP), Good Clinical Practice (GCP), Good Manufacturing Practice (GMP), Good Distribution Practice (GDP) and Good Pharmacovigilance Practice (GPvP).

The guidance has a high degree of alignment with documents published by other regulators such as Pharmaceutical Inspection Co-operation Scheme (PIC/S), World Health Organisation (WHO), Organisation for Economic Co-operation and Development (OECD) and European Medicine Agency (EMA) but clarifies MHRA’s position on data integrity and the minimum expectation to achieve compliance. The guidance promotes notably the use of a risk-based approach to data management that includes data risk, criticality and lifecycle. It provides as well terms definition and interpretations.

Click here to view the guidance.

Prime Minister raises potential for Associate Membership of the of EMA post-Brexit

In support of the biopharmaceutical industry, the British Prime Minister Theresa May explored the potential of the UK remaining part of the European Medicines Agency (EMA) by becoming an associate member.

In a balanced message, the UK Prime Minister said:

Membership of the EMA would mean investment in new innovative medicines continuing in the UK, and it would mean these medicines getting to patients faster as firms prioritize larger markets when they start the lengthy process of seeking authorisations…the UK regulator assesses more new medicines than any other member state. And the EU would continue to access the expertise of the UK's world-leading universities.”

However a draft guideline published by the the European Council in the days following this speech stated:

“the European Union will preserve its autonomy as regards its decision-making, which excludes participation of the UK as a third-country to EU institutions, agencies or bodies

As it stands, comments shared both by the UK and the EU highlight that political negotations continue.

Click here to view the Prime Ministers speech on future UK-EU relations in full.

Click here to view the European Council draft guideline.


Food and Drug Administration (FDA)

FDA develops methods predicting stem cell activity to ensure safe and effective therapies

As of January 2018, no mesenchymal stem-cell based clinical trials have resulted in FDA-approved treatments. The FDA has highlighted in a recent blog post, some interesting research being carried out by it’s Cellular and Tissues Therapy Branch into improving methods for modelling efficacy and safety of stem cells, and in particular predicting the behavior of mesenchymal stem cells (MSCs) that have been stimulated by growth factors.

In a lab-based manufacturing process, MSCs are exposed to an environment very different from the body which might change the way the cells behave after they are put into a patient. The FDA is looking at addressing some of these nonclinical challenges by research into MSC morphology to identify the way changes in the size and shape of stimulated MSCs may predict their future behavior.

According to the FDA, in the short-term this a ‘functionally-relevant morphological profiling’ approach, can improve the ability to measure the extent to which there are similarities or differences in cell preparations and to compare findings with the profile of specific cell types associated with the biological functions being selected. In the long-term however, this knowledge may help to predict whether stimulated stem cells will perform the relevant biological function when administered to patients.

Click here for more information on this research.

FDA discusses upcoming pilot programme on Complex Innovative trial Designs (CID)

One of the objectives of the US Prescription Drug User Fee Act (PDUFA VI) is to facilitate the advancement and use of complex clinical trial designs. These adaptative clinical trial designs allows for prospectively planned modifications to one or more aspects of the design based on accumulating data from subjects in the study.

FDA has announced it will be launching a new pilot programme on Complex Innovative Designs (CID) in clinical trials and has published content from a public workshop covering this topic. As part of the pilot programme, the FDA will select up to two ‘highly innovative’ trial designs per quarter. Developers will have the opportunity to engage closely with the regulator through two meetings. These will be led by the statistical review components within CDER or CBER. The FDA will uses the selected CID as a case study for continuing education/information and any public information will be agreed with the sponsor before release.

A number of points related to this programme still need to be further defined such as the trials which would be eligibile/prioritised.

Click here to view FDA’s slides

FDA discusses Patient-Focused Drug Development (PFDD) guidance

As part of 21st Century Cures Act of 2016 and Prescription Drug User Fee Act (PDUFA VI), the FDA is committed to more effectively incorporating the patients’ voice into drug development. There are several barriers whic currently hinder the collection and incorporation patient experience data into drug development including a lack of a methodological framework for identifying what measures are the most important to patients.

To address this, a Plan for Issuance of Patient‐Focused Drug Development (PFDD) Guidance has been developed by the FDA with the primary goals of:

  • Facilitating and advancing use of systematic approaches to collecting and utilizing robust and meaningful patient and caregiver input to more consistently inform drug development and regulatory decision-making
  • Encouraging identification and use of approaches and best practices to facilitate patient enrollment and minimizing the burden of patient participation in clinical trials
  • Enhancing understanding and appropriate use of methods to capture information on patient preferences and the potential acceptability of tradeoffs between treatment benefit and risk outcomes
  • Identifying the information that is most important to patients related to treatment benefits, risks, and burden, and how to best communicate the information to support their decision making

The public workshop held on 19th March should help FDA for issuance of new guidelines.

Click here to view FDA’s slides

Public consultations



Consultation period



Guideline on safety and efficacy follow up and risk management of Advanced Therapy Medicinal Products

01 Feb to 30 Apr 2018

Draft guideline



Consultation period



Technical and Regulatory Considerations for Pharmaceutical Product Lifecycle Management

16 Nov 2017 to 18 Dec 2018

Draft guidance