Regulatory news - October 2018

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


European Commission (EC)

EC/EMA publish an action plan on pediatric medicines

Following on from the publication of the 10-year report on the paediatric regulation last year, some challenges have been identified for paediatric medicines. The European Medicines Agency (EMA) and the European Commission (EC) have published this month a joint action plan to support the development of medicines for children in Europe.

The plan takes into account recommendations collected during the workshop held on March 2018. The action plan is structured around five topics areas:

  1. identifying paediatric medical needs;
  2. strengthening cooperation between decision makers;
  3. ensuring timely completion of paediatric investigation plans (PIPs);
  4. improving the handling of PIP applications;
  5. increasing transparency around paediatric medicines.

The action plan should address the challenges identified by the EC/EMA and increase the efficiency of paediatric regulatory processes in the current legal framework and boost the availability of medicines for children. However, the completion of this action plan takes into consideration the Brexit, EMA relocation and business continuity plan. Some actions are on hold and some deadlines are likely to be revisited. The completion of this action is not expected within the initial 2-year timeframe.

Click here to view the joint action plan

EC launches a public consultation on pediatric and orphan legislation

As mentioned on their roadmap, the EC opened a public consultation to evaluate legislations on medicines for special populations: medicines for children and rare diseases.

The purpose of this joined evaluation will be i) to look at the strengths and weakness of these two pieces of legislation, both separately and in combination, and ii) give insight on how the various incentives related to this legislation have been used.

All stakeholders are invited to share their views via this public consultation until 4 January 2019 to help to shape the EU policy on medicines for children and rare diseases.

Click here to access to the EC public consultation

European Medicine Agency (EMA)

CAT provides update on the guideline providing requirements for investigational ATMPS

According to the Committee for Advanced Therapies (CAT) monthly report, the guideline on “requirements for investigational ATMPs” have been discussed. The document should be published by the end of 2018.

Click here to view the CAT monthly report

Workshop with stakeholders on support to quality development in early access approaches (i.e. PRIME, Breakthrough Therapies)

The EMA will host on 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.

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 to view the draft agenda

Report on expert meeting on genome editing technologies used in medicines development

Genome editing technologies are progressing with an unprecedented speed. Medicinal products developed using these novel technologies may potentially ameliorate or cure genetic diseases. However, they also come with complexities and challenges during their development, manufacture, evaluation and ultimately making them available to patients. The EMA held an expert meeting on genome editing on October 2017 and just published the meeting report in which leading academic institutions as well as industry stakeholder with the drug development programs using genome editing discussed current challenges and opportunities and provided case studies of the most advanced development.

Click here to view the EMA’s report


Food and Drug Administration (FDA)

FDA advices sponsors to elaborate “data management plan”

Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) and Medicines and Healthcare products Regulatory Agency UK (MHRA) held a workshop in October on “Data Integrity in Global Clinical Trials – Are We There Yet?”. It provided regulatory perspectives on data management, data blinding to minimize introduction of bias, and the role of audit trails in assessing data integrity in global clinical trials. During this workshop, FDA explained the necessity for sponsors to prepare a “data management plan”, a roadmap to achieve reliable, high-quality and statistically sound data. This plan constitutes an essential living document necessary throughout the lifecycle of a study, it should include how the document will be reviewed, approved and finalised and it links to standard operating procedures (SOPs) in place governing its use and modifications.

Click here to view records of the event

FDA publishes draft guidance on rare diseases: early drug development and the role of pre-IND meetings

The guidance aims to assist sponsors developing rare diseases products in the preparation of pre-IND meeting. A pre-IND meeting is often the first regulatory communication between the sponsor and FDA regarding the development program for an investigational drug. This meeting can be particularly helpful to discuss the specific challenges of rare disease drug development and where regulatory flexibility can be justified. Because of the limitations in patient numbers, it is important to maximise the contribution of each patient in the clinical development program.

The guidance focuses on various considerations including pharmaceutical quality, nonclinical, clinical pharmacology, clinical, and additional considerations on expedited programs, companion diagnostics, orphan product incentives and pediatric studies.

Click here to view the draft guideline

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