Alliance for Regenerative Medicine Calls for Multi-Stakeholder Pan-European Initiative to Fast-Track Real World Evidence in Support of Patient Access to Advanced Therapies

The Alliance for Regenerative Medicine (ARM), the leading international advocacy organisation dedicated to realising the promise of regenerative medicines and advanced therapies, has today called for pan-European action to fast-track the use of Real World Evidence (RWE) for advanced therapy medicinal products (ATMPs),  ensuring patients have access to these transformative therapies.

ARM’s newly published position paper provides clear recommendations for the European Commission to include as part of the EU Pharmaceutical Strategy, which is designed to improve and accelerate patients’ access to transformative therapies that are safe and affordable medicines and to support innovation in the EU pharmaceutical industry. ARM’s full response to the EU Pharmaceutical Strategy Roadmap can be found on the European Commission website.

ARM calls on the European Commission to fast-track the use of RWE for ATMPs, by convening a multi-stakeholder forum, including subject matter experts from ARM, manufacturers and patient advocacy groups, to set up a European infrastructure for regular RWE use. ARM believes this collaborative approach will benefit regulators, HTA bodies, payors, manufacturers, healthcare professionals and, most importantly, patients.

This group should be focused on the following priorities:

• Adopting common standards and data requirements 
• Creating an implementation roadmap for healthcare centres across Europe, ensuring cross-border consistency of RWE usage and interoperability through registries and other collection and analysis vehicles
• Complying with legal requirements including GDPR

Investment in pan-European RWE infrastructure for ATMPs will be particularly effective if clinical assessment were to be coordinated at the EU level (joint clinical assessment), while adapting national Health Technology Assessment (HTA), pricing and reimbursement processes to capture and value the long-term benefits of ATMPs. An integrated RWE infrastructure, collecting evidence of ATMP use and effectiveness across Europe, will also facilitate cross-border treatments, which are of vital importance in cases when only a limited number of specialised centers across Europe can administer the treatment.

A significant number of ATMPs are expected to obtain European marketing authorization in the years to come, resulting in an increasingly urgent need to address inefficiencies coming from a fragmented, inconsistent and often unusable RWE ecosystem in Europe.  ARM advocates for the creation of pan-European infrastructure and processes to develop standards for the collection, analysis and use of RWE for ATMPs and for this infrastructure to be fully integrated in the future establishment of a European Health Data Space.

Paige Bischoff, SVP Public Affairs at ARM, commented: “ARM strongly believe the EU must take a central, co-ordinating role to ensure that RWE use for ATMPs is fast-tracked to properly meet the needs of all stakeholders and enhance access to innovation.  RWE is integral to
collecting evidence of ATMP use and effectiveness.  Solely relying on traditional clinical trials, without benefit of the proper gathering, management and use of Real World Evidence will likely create delays in guaranteeing patient access to transformative regenerative medicines and advanced therapies -- a delay that patients in need cannot afford. As the voice of the ATMP sector globally, ARM looks forward to being part of an inclusive and solution-driven dialogue with the European Commission and other relevant stakeholders in shaping the path forward for the fast and effective use of RWE for ATMPs.”

The COVID-19 pandemic has highlighted even further the importance and urgency of having a common RWE infrastructure across Europe, in order to ensure critical data is available in times of crisis, when the access to healthcare centres and the conduct of traditional clinical trials are hampered. 

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organisation dedicated to realising the promise of advanced therapy medicinal products (ATMPs). ARM promotes legislative, regulatory and reimbursement initiatives in Europe and internationally to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing ATMPs. In its 11-year history, ARM has become the voice of the sector, representing the interests of 360+ members worldwide and 70+ members across 15 European countries, including small and large companies, academic research institutions, major medical centres and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.