News - October 18, 2022

Event Recap – TRANSFORM Alliance Charter Launch (13 October 2022)

EUCOPE discusses solutions across the R&D lifecycle to build a European system fit for transformative therapies

On the 13th of October 2022, the European Alliance for Transformative Therapies (TRANSFORM) came together with the MEP Interest Group to launch an MEP Charter in the European Parliament. The charter presents seven (7) main policy recommendations to promote safe and timely access to ATMPs while ensuring Europe remains attractive for investment and promoting healthcare systems sustainability.

Advanced Therapy Medical Products (ATMPs) are complex medicines that offer transformative therapeutic opportunities for both rare and chronic conditions. The past years have marked an increase in the number of cell and gene therapies coming to the market.

Over the coming years, the number of approved ATMPs is expected to continue to grow. This underlines the need to ensure that the legislative, regulatory and access frameworks are sufficiently flexible and capable to respond to these therapies to continue encouraging innovation and patient access.

How is the EU planning to enable access to safe and timely patient access to transformative therapies?

The EU is launching a holistic review of the pharmaceutical legislative environment, including the General Pharmaceutical Legislation and Orphan Medicinal Products Regulation & Paediatric Regulations, including how to incentivize innovation and the role of patients’ data. During the meeting, MEP Ondřej Knotek (RE, Cz) stressed the importance of shaping the policy framework “in which the patient is at the core of recommendations”. We need to create a dynamic, patient-centred innovation ecosystem to address areas of medical need to ensure that regulatory requirements remain appropriate for the development of ATMPs.

While ensuring that patients are included throughout the development pathway of ATMPs, expert groups and EUCOPE also call for enhancing the EU’s global competitiveness. The EU market can be made more attractive for investment, foster more early-stage R&D and shorten timelines for drug approval. EUCOPE and the TRANSFORM alliance call for the introduction of a more flexible regulatory system that includes enabling the use of real-world evidence to address uncertainties for ATMPs. Early and frequent dialogue between developers and regulatory bodies provides developers with the reassurance they need to navigate the European approval process without delay. Representatives from both the European Commission, Stefaan Van der Spiegel (DG SANTE), and the Czech EU Presidency, Tomáš Boráň (Director of the Marketing Authorisation Section at State Institute for Drug Control) outlined the importance of facilitating dialogue between stakeholders Mr Van der Spiegel  suggests, coordination between all stakeholders and decision-makers is key to ensure an appropriate regulatory framework for ATMP”.

On the topic, EUCOPE’s Victor Maertens remarks TRANSFORM has looked and will continue to look at the full lifecycle of cell and gene therapies. We need a regulatory system that is future-proof and tailored to the unique elements ATMPs have and bring.

EUCOPE believes it is essential that the EU remains globally attractive for investment to encourage innovation and access. This requires an agile, flexible, and appropriate regulatory framework for ATMPs that is founded on science and is future-proof. The regulatory environment must be stable, predictable and effective to encourage R&D investment in the ATMP industry. To promote access, Member States must support cross-border patient access, explore innovative payment models and risk-sharing approaches that promote healthcare sustainability, while continuing to encourage innovation.

Europe finds itself at an inflection point and the review of the pharmaceutical environment in Europe will have far-reaching implications for decades to come, for both ATMPs and health technologies more broadly. EUCOPE’s Secretary-General, Dr Alexander Natz, laid out our thoughts on the current reviews here.

As the voice of small and mid-sized pharmaceutical innovators, EUCOPE will continue to call for common sense policies that reflect scientific realities and encourage innovation and investment.

For more information about our work with the TRANSFORM Alliance or to join our Cell & Gene Therapy Working Group, please contact Victor Maertens.