The Alliance for Regenerative Medicine Provides Recommendations to EMA, European Commission to Foster Development of ATMP Sector in Europe

BRUSSELS, Belgium and WASHINGTON, DC – March 14, 2018

The Alliance for Regenerative Medicine (ARM), an international advocacy organization representing the cell and gene therapy and broader regenerative medicine and advanced therapies sector, today published its recommendations for fostering development of and patient access to advanced therapy medicinal products (ATMPs) in Europe. These proposals support and complement the European Medicines Agency’s (EMA) and the European Commission’s (EC) Action Plan on ATMPs, issued October 2017.

ARM’s recommendations, available here, elaborate on the organizations’ original proposed initiatives, emphasizing areas of priority, and address various aspects not covered in the EC/EMA Action Plan, with specifics for next steps.

“ARM greatly values the work of the EMA and EC to try to improve the regulatory environment for ATMPs in Europe,” said Jacqueline Barry, Ph.D., Chief Clinical Officer of Cell and Gene Therapy Catapult. “ARM and its members hope to provide further input and clarification as to what is necessary to support the continued innovation this sector brings and to ensure these important products reach patients in need.”

ARM’s recommendations include the following, grouped into four main areas of focus:

  • Research and Development: a pragmatic and flexible approach regarding the licensing requirements for ATMPs, including the use of innovative manufacturing models, the implementation of a “Master File/Cell History File” system; addressing the complexities and delays for clinical trials with ATMPs as Genetically Modified Organisms (GMOs); reaching regulatory convergences of cell, tissue and blood requirements across the European Union; and the adoption of sector-wide standards for product development, manufacturing, and testing.
  • Regulatory Processes: adapting current regulatory processes to reflect the unique characteristics of ATMPs, including making ATMP certification available to non-SMEs or obtaining GMP certification in parallel to clinical trial authorisations; facilitating increased interactions between ATMP developers and regulatory agencies during development; and providing increased guidance on the structure of clinical trial and market access applications for AMTPs.
  • Hospital Exemption (HE) Pathway: ensuring the responsible use of the Hospital Exemption to enable patients to receive an ATMP under controlled conditions in cases where no authorized medicinal product is available for an indication with a high unmet medical need, while protecting patients from unnecessary risks and maintaining the integrity of traditional legal channels for market access. More information can be found in the ARM position paper published in February 2017, available here.
  • Funding, Investment, and Market Access: supporting initiatives that will increase the risk capital available in Europe to promote funding of and investment in ATMPs; fostering the debate with HTA agencies, pricing and reimbursement authorities and policy makers to remove the potential barriers to the adoption of ATMPs by patients in need, with particular emphasis on the need to consider the potential long-term value of ATMPs, to streamline post-approval commitments and real world evidence data collection and to evaluate appropriate and alternative reimbursement models and risk-sharing frameworks adapted to curative treatments.

The EMA’s and EC’s Action Plan on ATMPs is available here.

About the Alliance for Regenerative Medicine:
The Alliance for Regenerative Medicine (ARM) is a global, multi-stakeholder organization that promotes innovation, growth, and delivery of transformative treatments or cures for patients suffering from chronic, debilitating, and often life-threatening diseases, many of which are rare diseases. ARM convenes all stakeholders with an interest in regenerative and advanced therapies to provide a unified voice for our 290+ member organizations, including companies – especially small- to medium-sized enterprises (SMEs); academic/research institutions; non-profit organizations; patient advocacy organizations, and other members of the global advanced therapies community. The organization’s aim is to connect all parts of the innovation lifecycle to address current unmet medical needs of patients, particularly through supporting commercialization objectives via legislative and policy frameworks that enable next generation therapies to reach those who need them. To learn more about ARM, visit