ISSUE: To allow for early authorisation and aim for early patient access, marketing authorisation applications for ATMPs are often granted whilst the evidence is still immature. Assessing these medicines has therefore posed challenges for regulators, Health Technology Assessment (HTA) decision-makers and healthcare payers. To collect additional information and mitigate the financial risk of reimbursing these therapies under large uncertainties, outcome-based reimbursement and delayed-payment models are frequently debated as promising strategies to create win-win-win situations form a patient, industry and healthcare payer perspective.

OVERVIEW: The main underlying uncertainties in the evidence supporting ATPMS from a regulatory and HTA perspective will be presented (15 min) and how the type of uncertainty and level of expected added benefit can be used to determine which kind of managed entry agreement is most suitable (15 min). The panellist with an patient, industry, healthcare payer and HTA perspective (30 min) will discuss how early dialogues could support mutual understanding of the most pressing underlying uncertainties and how horizon scanning can play a valuable part in informing and preparing stakeholders throughout the life cycle of ATMPs. Moreover, the discussion will go into what role the new EU HTA regulation could take in supporting uncertainty management and payment and reimbursement decision-making and provide perspective on implementing outcome-based reimbursement models in the future .

Audience participation will include discussion on how clinical uncertainties early in the life cycle of ATMPs have a rippling down effect to later stages in the payment and reimbursement decision-making, what guidance is needed to ease reimbursement decision-making and how the current and future policy landscapes support the ability to create win-win-win situations for patients, manufactures and healthcare payers for the upcoming ATMPs. This interactive and informative panel will be valuable to regulators, HTA agencies, healthcare payers, policymakers, researchers and industry analysts interested in payment and reimbursement strategies.

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