Securing patient access to highly innovative products, in particular for orphan drugs, in a sustainable way is a key concern for payers, industry and patients alike. This project was designed to foster open, constructive debate with experts from different stakeholders providing their personal views on how (orphan) drug value should be assessed and appraised.
Main Goal: Develop a conceptual platform to engage stakeholder experts and structure a constructive debate; set-up and facilitate a roundtable discussion and develop a whitepaper based on its results
Main Result: Belgian price and reimbursement landscape analysis focused on orphan drugs; whitepaper describing conceptual framework, roundtable process and discussion results
The Belgian landscape analysis clearly identifies specific challenges for orphan drug price and reimbursement procedures. While stakeholders recognize the specificity of OMPs and this specificity is – to some extent – taken into account in price and reimbursement evaluations, no specific tools for integration of value elements particular to OMPs are routinely used in price and reimbursement evaluations. As such, OMP specific elements of value are most often implicitly taken into account.
Explicit inclusion of patient-specific metrics is not straightforward, both from the evaluator’s and the submitter’s perspective, due to the lack of standardized tools and limited feasibility of a fully quantitative analysis, reinforced by the rarity of OMP conditions. In general, for evaluation of OMP value in its broader context, involvement of external experts is required. There is, however, no clear consensus on the extent or way in which patient and healthcare practitioner expertise could be integrated.
Ensuring continued access to innovative therapies while safeguarding sustainability and maximizing value requires the possibility to engage in open and constructive dialogue on the principles underlying price and reimbursement procedures with different stakeholders. Our approach demonstrates how a structured approach may assist in providing a platform to engage on and provides first insights into the challenges orphan drugs face and their potential solutions from a proof-of concept roundtable discussion.