The Alliance for Regenerative Medicine (ARM) welcoming the opportunity to provide feedback on the roadmap to the EC pharmaceutical strategy has sent the following response.

ARM is the leading international advocacy organisation dedicated to realizing the promise of advanced therapy medicinal products (ATMPs) and represents 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. ATMPs, including cell therapies, gene therapies and tissue engineered products, constitute a new category of innovative products whose full potential is beginning to emerge.

The ATMP sector is creating transformative, durable treatments and potential cures for some of humankind’s most devastating diseases – many currently untreatable via conventional treatments – through the use of ground-breaking scientific discoveries and technologies.

The Covid-19 crisis revealed the importance of investing in healthcare, particularly in areas of unmet needs. ATMPs can play an important role to meet these needs and represent an opportunity for Europe to take a leading role in embracing this new field of innovation.

To ensure patients across Europe have access to safe and effective ATMPs, ARM believes the following priorities should be addressed in the EC pharma strategy:

(1) Ensuring that regulatory procedures are robust, transparent, evidence-based and harmonized in Europe

• limiting hospital exemption and other regulatory carve outs that threaten patient safety

(2) Streamlining regulatory requirements for clinical trials with ATMPs across the different Member States,

• ensuring smooth implementation of the upcoming clinical trial regulation • reconsidering the scope and implementation of the GMO legislation to medicinal products.

(3) Ensuring patients’ timely access to ATMPs by

• launching new initiative to build RWE infrastructure and disease registries at pan-European level.

• coordinating ATMP clinical assessment at EU level, while adapting the HTA evaluation, pricing and reimbursement processes to capture and value the long-term benefits and risks of ATMPs,

• facilitating cross-border treatment with ATMPs, and

• 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.

More background information on the above priorities is provided in the attached document. ARM is committed to engage and bring the ATMP sector’s perspective in an inclusive and solution-driven dialogue with the Commission, Member States and all interested parties to facilitate the development and access to innovative treatment for the patients’ benefit.