On 16 December 2016, the European Medicines Agency (“EMA”), along with the European Biopharmaceutical Enterprises (“EBE”) organised the fifth annual regulatory conference concerning the optimisation of the development of advanced therapy medicinal products (“ATMPs”) to meet patient needs (“the ATMP conference”).

Background

The aim of the ATMP conference was to discuss initiatives to improve advanced therapy medicinal products access to patients. The EMA further presented an update concerning specific requirements for gene therapy medicinal products (“GTMP”). More generally, the conference gathered stakeholder feedback on standards for development and commercialisation of ATMPs.

The ATMP conference was attended by public and private stakeholders, including patients, academia, regulators, investors and industry.

The EMA action plan

On 27 May 2016, the EMA held a multi-stakeholder expert meeting concerning development of ATMPs. At this meeting, proposals and solutions were discussed by stakeholders and EMA in four key areas:

  • facilitating research and development;
  • optimising regulatory processes for ATMPs;
  • moving from hospital exemption to marketing authorisation;
  • improving funding, investment and patient access.

At the ATMP conference, the EMA announced its intention to issue an action plan in January 2017 which will outline concrete steps to address the above four key concerns in relation to ATMPs (“the action plan”).

The action plan was presented at the ATMP conference by the EMA Head of Specialised Scientific Disciplines Department, Ana Hidalgo-Simon, in a presentation concerning “Initiatives to improve ATMPs access to patients”.

As stated in the presentation, this action plan is intended to provide regulatory and scientific support for ATMPs, while prioritising on solutions that can be implemented quickly. In the action plan, priority will be given to suggestions that can be commenced in 2017 or beyond, that are feasible, and that are within the EMA’s jurisdiction.

Update on the Gene Therapy Products Guidance

At the ATMP conference, the representative of the UK Medicines & Healthcare products Regulatory Agency (“MHRA”) at the EMA’s Committee for Advanced Therapies (“CAT”), Christiane Niederlaender, provided an update on the EMA revised guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products (“the GTMP guideline”).

In this update, clarification was provided regarding the scope of the revised GTMP guideline. The EMA has structured the guideline in accordance with the format of the electronic common technical document (“eCTD”). Its main aim is to ensure that correct data is included by applicants in their EU marketing authorisation applications. The EMA’s mandate only allows the EMA to harmonise the GTMP guideline with EU requirements for marketing authorisations. The GTMP guideline may not be further harmonised with international requirements despite requests from stakeholders to this effect.

In addition, the latest amendments introduced by the EMA to the GTMP guideline were presented. These changes include clarifications of the use of non-clinical data from animal and homologous models and additional information on methods for long-term follow-up and dose finding in clinical development of GTMPs.

The revised GTMP guideline is expected to be finalised in the first half of 2017. Once finalised, it will replace the 2001 Note for guidance on the quality, preclinical and clinical aspects of gene therapy medicinal products.

See our previous blog post “EMA launches public consultation on draft Guideline concerning the development of GTMPs” for more details on the contents of the revised guideline.

For further information concerning the ATMP conference visit : http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/events/2016/10/event_detail_001334.jsp&mid=WC0b01ac058004d5c3