Advanced Therapy Medicinal Products (ATMPs) are at the forefront of global scientific innovation in healthcare. These pioneering treatments, which include gene therapies, somatic cell therapies and tissue engineered products, have the potential to transform patients’ lives, providing new therapeutic options for diseases for which there may be limited or no available treatments, and in some instances being potentially curative. In the context of the recently published Pharmaceutical Strategy for Europe, the European Commission recognises ATMPs as a generational milestone and acknowledges the need for new pricing and reimbursement frameworks that help address the shift from payment over time for chronic treatments to upfront costs for these often one-time therapies. With multiple approved products and numerous more in the pipeline, the timing is now to collectively build a futureproof innovation model for ATMPs.

This White Paper identifies key challenges ahead and proposes a set of recommendations to start collectively building that future-proof innovation model.

ATMPs fundamentally change the value assessment framework, being usually associated with a single upfront payment, despite benefits extending many years into the future and spreading to societal areas beyond savings in the healthcare system. Moreover, the current evidence package of ATMPs at launch may not address all questions posed in traditional Health Technology Assessments (HTA), demonstrating cost-effectiveness can be challenging and the holistic value of ATMPs is not being consistently recognised. This new generation of ground-breaking treatments calls for a two-pronged paradigm shift, enabling the advent of a new wave of innovation and delivering better health outcomes for patients. Such a change requires a collaborative, multi-stakeholder environment.

EFPIA members have come together to propose a concrete list of recommendations that could increase patient access and accelerate the availability of innovative, potentially curative ATMPs. They address the challenges identified, focusing on two pillars:

.- Promoting patient access to ATMPs while supporting payer decision-making
 .-Ensuring sustainable ATMP access for healthcare systems and patients

A broader, holistic value assessment is anticipated to enable timely patient access to ATMPs. Collaboration between industry, the European Medicines Agency (EMA) and Health HTA agencies is
needed to harmonise evidence requirements and facilitate greater acceptance of ATMP evidence, including indirect treatment comparisons, surrogate endpoints, and real-world evidence (RWE).

Innovative payment models can help address payers’ remaining evidentiary questions while helping to spread the upfront cost of ATMPs, securing timely patient access and alleviating the financial burden. A longer distribution of costs over time and the restructuring of existing budget silos could also tackle sustainability concerns. Horizon scanning is another key element of the ATMPs access toolbox, as it allows to inform financial commitments ahead of landmark launches and prevent delays in access and uptake.

As we welcome this new generation of therapies, we hope that this White Paper will serve as a roadmap towards identifying the steps that are necessary at EU and Member State level, to ensure the full value of ATMPs is realised and patients can benefit from unprecedented opportunities. A paradigm shift is never an easy task and we are convinced that collectively, we can rise to the occasion and deliver on the shared commitment of our industry and the healthcare systems for patient