Shifting the paradigm for ATMPs: Adapting reimbursement and value frameworks to improve patient access in Europe

A new wave of potentially curative treatments called Advanced Therapy Medicinal Products (ATMPs) has the potential to transform the lives of patients and revolutionise the way we think, manage and resource healthcare.
Speaking about today’s publication of a new report on ATMPs, EFPIA Director General, Nathalie Moll said; “Innovation only matters if it reaches patients. Ensuring that patients get faster, more equitable and sustainable access to innovative medicines in Europe is a goal we share with patients, EU institutions, and national governments. That is why EFPIA has published a new White Paper “Shifting the paradigm for ATMPs: Adapting reimbursement and value frameworks to improve patient access in Europe”. Our hope is that the paper will support collaborative discussions on introducing these new, ground breaking therapies in to health systems across Europe.”
Of the 8,000 medicines in development, 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 the lives of patients and our healthcare systems, providing new therapeutic options for diseases for which there may be limited or no available treatments, and in some instances being potentially curative.
New therapies for spinal muscular atrophy and severe combined immunodeficiency have already been approved, with more in the pipeline targeting solid tumours, neurological disorders, pain and a host of other conditions.
New therapies that revolutionise the care model require a paradigm shift in the way they are assessed, valued and financed. In many cases ATMPs are a one-off, curative treatment replacing a life-time of medical interventions. While the upfront costs are considered high and rooted solely in the medicines budget in year one, the benefits are delivered across the entire health and social care system over the life time of the patient. Working together, current budget impact analyses and payment models have to evolve in order to harness the potential of these ground-breaking advances in medical science and transform the lives of patients. In addition, new development pathways for ATMPs means the evidence package  at launch may not address all the questions posed in traditional health technology assessments. New approaches are needed urgently. 
EFPIA members have come together to propose a concrete list of recommendations that could accelerate ATMP availability and increase patient access to these ground-breaking treatments. Addressing the key challenges identified, EFPIA’s recommendations seek to support payer decision-making and ensure sustainable ATMP access.
Nathalie Moll went on to say “As we welcome this new generation of therapies, we hope that this White Paper will serve as a roadmap, helping identify the necessary steps at EU and Member State level, to ensure the full value of ATMPs are realised so patients can benefit from these transformative treatments. 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 patients.”
This White Paper also aims to contribute to the discussions on the Pharmaceutical Strategy for Europe, where 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 single-use therapies. The industry is fully committed to progressing collaborative approaches that can meet the needs of patients and our healthcare systems.