The EMA has recently published the outcome of a multi-stakeholder meeting with experts and regulators on Advanced Therapy Medicinal Products (ATMP) held at the Agency on Friday 27 May 2016. The workshop was organised within the context of current challenges faced by developers and aims to explore ways for stimulating innovation in Europe, facilitating European research and development and accelerating patients’ access to ATMPs.
The workshop was attended by academics and researchers, representatives from patients and healthcare professionals organisations, small and large pharmaceutical companies, the investment community, health technology assessment bodies, national competent authorities and the European Commission (EC).
During the workshop stakeholders described issues they are facing and came up with ideas and suggestions how to tackle these issues. In relation to research and development the main proposals were:
- Take more pragmatic approach with licensing requirements for ATMPs
- Improve implementation of GMO requirements
- Harmonise cell, tissue, and blood requirements across EU
- Increase incentives and regulatory support
- Provide more ATMP specific guidance (e.g. on comparability), workshops and training
- Promote novel development tools (organoids, extrapolation, modelling/simulation, biomarkers).
In relation to current issues with the regulatory process it was suggested to
- Streamline EMA internal regulatory processes for ATMPs
- Promote use of early access tools (PRIME, adaptive pathways, ITF, scientific advice, certification and HTA parallel advice)
- Publish overview of national requirements (for GMOs and tissue, cell and blood products) and move towards greater uniformity
- Develop guidelines on investigational medicinal products and comparability
- Adapt concept of orphan similarity to ATMPs.
The main discussion point around the hospital exemption was the different implementation of the exemption clause in Member States. Thus, stakeholders, inter alia, called for
- Implement hospital exemption more uniformly across Member States
- Make details of hospital exemption products in each Member State publicly available
- Collect clinical data and experience systematically
- Provide support with manufacturing and pharmacovigilance activities
- Consider restricting hospital exemption to areas of high unmet medical need where no ATMP is licensed.
In relation to funding, investment and market access stakeholders made the following proposals:
- Raise awareness of financial incentives
- Provide vehicle for SMEs and academia to seek early parallel EMA/HTA advice
For HTAs and payers:
- Engage earlier in development process
- Provide platform for informal dialogue
- Prioritise funding based on realistic patient benefit
- Deploy funding more effectively, with multi-stakeholder input and monitoring
The EMA has stated that the European Commission, national competent authorities and the Agency have already started discussing the feasibility of these proposals but have not taken any decisions yet.