EMA Updates on Advanced Therapy Evaluations, Risk Management

The European Medicines Agency (EMA) on Thursday released updated procedural advice on how it evaluates advanced therapy medicinal products (ATMPs) such as cell and gene therapies.

Alongside the updated advice document, EMA also released a new draft guideline on follow-up safety and efficacy requirements and risk management for ATMPs. According to EMA, the updated documents provide needed clarity to developers of ATMPs, who are often small or medium-sized enterprises (SMEs) or academic spinoffs that may need additional assistance navigating regulatory processes.

Both documents are also part of the joint action plan announced by EMA and the European Commission in October to bolster the development of advanced therapies.

Evaluating Advanced Therapies

EMA's procedural advice document for ATMPs was initially developed following discussions among the agency's scientific committees in 2009 and describes the evaluation process and review timetable for ATMPs, as well as the roles and responsibilities of the various rapporteurs and committees involved in the assessments.

EMA says the updated advice document serves to "streamline some procedural aspects, strengthen collaboration between EMA's scientific committees and address specific needs of ATMP developers in the initial marketing authorisations."

Specifically, EMA says it has streamlined the process for adopting the list of questions and outstanding issues generated by its committees and clarified when in-person responses to those questions or issues are needed.

Additionally, the revised procedure allows for longer "clock-stops" during the evaluation of ATMPs to give sponsors more time to respond to questions raised by EMA or its scientific committees.

Follow-Up and Risk Management

EMA also launched a three-month public consultation on a revision to its guidelines on safety and efficacy follow-up and risk management for ATMPs.

By their nature, ATMPs are cutting edge products whose safety and efficacy may not always be fully known at the time of approval. As such EMA requires marketing authorization holders to follow a risk management plan and often requests that they complete additional postauthorization safety and efficacy studies.

EMA says the revised guideline is meant to strengthen and clarify its requirements for post-authorisation activities for ATMPs building on its experience with authorised advanced therapies over the last decade.

Specifically, EMA says the guideline provides new recommendations for detecting and mitigating risks and gives advice on postauthorization study design and methodology.

EMA, Procedural Advice, Follow-Up and Risk Management, Revised Guideline