Successfully Launching Advanced Therapy Medicinal Products in Europe

Advanced Therapy Medicinal Products (ATMPs), including cell and gene therapies, have transformative potential to change lives. For patients with few or no treatment options, ATMPs open up a world of possibility in what would otherwise be a bleak future.

As science evolves, the rare disease patient community will not be the only beneficiary. Today, there are numerous therapies in development designed to target debilitating chronic conditions that affect an increasingly ageing population. Parkinson’s, Alzheimer’s, diabetes and cardiovascular disease could all be afflictions of the past if these treatments come to fruition.

As new technologies emerge, a myriad of social, ethical and economic questions remain around how they can reach patients. Are societies ready to embrace the potential of ATMPs? Are our health systems, designed for conventional medicines, sufficiently adaptable to support access to such treatments? How can we reconcile disparate national regulatory and value frameworks across Europe to facilitate greater acceptance of ATMP evidence and expedite access? Do clinicians and patients understand these new forms of treatment and their key benefits? Assuming they do, how can health systems and society afford them?

APCO Worldwide advises clients on navigating the rapidly evolving and complex global health care environment. We spoke to 12 industry experts to get their perspectives on these questions as discussions on the reforms of the EU pharmaceutical framework and joint Health Technology Assessments (HTAs) are underway. For companies looking to Europe as a future commercial market for ATMPs, we hope these perspectives serve to inform strategic planning on how to navigate the evolving policy environment.

All experts highlighted that preparation for ATMP launches needs to begin as early as possible during the development stages. Early engagement with ecosystem stakeholders (patient organizations, health care professionals, policymakers and implementation bodies) enhances alignment with stakeholders’ expectations and policy agendas. Importantly, the existing perception of ATMPs as ‘high cost and high risk’ needs to be reframed to ‘high opportunity and high value’ for patients, health systems and society as a whole.

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