Alliance for Regenerative Medicine Responds to EU Pharmaceutical Roadmap

Leading international advocacy group calls for effective regulation, clinical trial support, and value-based reimbursement that benefits patient access to ATMPs

The Alliance for Regenerative Medicine (ARM), the leading international advocacy organisation dedicated to realising the promise of regenerative medicines and advanced therapies, today published its response to the EU Pharmaceutical Strategy Roadmap which is designed to improve and accelerate patients’ access to safe and affordable medicines and to support innovation in the EU pharmaceutical industry. The full response can be found on the European Commission website.

While ARM welcomes the Roadmap’s recognition of the wider industry’s contribution to the EU economy and its focus on ensuring patients’ access to safe, state-of-the-art therapies, ARM wants to ensure a continued pathway for the EU to benefit from innovative and life-changing advanced therapy medicinal products (ATMPs). In support of that goal, ARM recommends the Pharmaceutical Strategy include the following priorities:

• Ensuring that regulatory procedures are robust, transparent, evidence-based and harmonised in Europe by:
• limiting the Hospital Exemption, and other regulatory carve outs, that threaten patient safety
• Streamlining regulatory requirements for clinical trials with ATMPs across Europe by:
• ensuring smooth implementation of the upcoming clinical trial regulation
• reconsidering the scope and implementation of the genetically modified organism (GMO) legislation of medicinal products, to reduce burdensome requirements for gene therapy clinical trials
• Ensuring patients’ timely access to ATMPs by:
• launching new initiatives to build Real World Evidence (RWE) infrastructure and disease registries at the pan-European level
• coordinating ATMP clinical assessment at the EU level, while adapting Health Technology Assessment (HTA), pricing and reimbursement processes to capture and value the long-term benefits and risks of ATMPs
• facilitating cross-border treatment with ATMPs
• supporting the adoption of innovative pricing and payment models for ATMPs to ensure continued patient access to innovative therapies while preserving the sustainability of health systems in the EU

Janet Lambert, CEO of ARM, commented: “Europe is a leader in ATMP innovation and adoption. It is critical that the EU Pharmaceutical Strategy furthers the ability of patients to benefit from these transformative therapies and supports vigorous growth in the ATMP sector to drive further advances. We strongly encourage the EU to streamline regulation where it can do so safely, harmonise regulations across Member States and address risks to patients stemming from inappropriate Hospital Exemptions.

“The Roadmap must also ensure health technology assessments are appropriately structured to reflect the long-term benefits of ATMPs and the increasing role of real-world evidence in establishing value-based reimbursement that drives sustainable innovation.”

ARM looks forward to further consultation on the EU Pharmaceutical Strategy.  The international advocacy organisation will be actively engaged in the process of ensuring patients are safe and have access to life-changing therapies whilst innovators continue to bring forward the next scientific advancements and transformative therapies.

Press inquiries

For more information or for media requests, please contact Kaitlyn (Donaldson) Dupont from the Alliance for Regenerative Medicine at kdonaldson@alliancerm.org or Consilium Strategic Communications at ARM@consilium-comms.com.

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 350+ 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.