Navigating the Reimbursement Conundrum in Drug Repurposing: Balancing Access, Pricing, and Innovation

Drug repurposing holds enormous potential but suffers from many well-documented challenges that span issues with data access, collaboration, R&D, business case, legal and regulatory ¹. Within the regulatory challenge, there has been a lot of talk about difficulties with navigating regulatory routes with a repurposed drug as a non marketing authorization holder, and indeed that remains challenging in the case of shelved drugs and where becoming a marketing authorization holder is not an option ². But what comes after marketing authorization is not commonly considered. We know examples of repurposed drugs that had steep price increases and became unaffordable for patients and health systems (e.g. colchicine ³, mexiletine ⁴). On the other hand, we know that there may not always be a viable business case for repurposing a drug because the drug would not pass the reimbursement stage at a profitable price. The fragmentation of reimbursement processes, with each jurisdiction having their own set of rules and reimbursement criteria, adds an additional challenge. This talk is about the tension between preventing evergreening and incentivizing innovative drug repurposing, how this affects pricing and reimbursement policies in major markets, and what policy changes could be made in the context of drug repurposing. It will start with an overview of the regulatory and reimbursement ecosystem drug repurposers operate in, including the proposed EU pharmaceutical legislation, and EU regulation on Health Technology Assessment with the requirement for joint clinical assessments. Incentives for innovating and main regulatory and reimbursement challenges are discussed. The societal impact of different pricing mechanisms is presented and illustrated in an example HTA process with value-based pricing as the guiding principle. Finally, ideas will be put forward to address the reimbursement conundrum.