Exploring Alternative Financing Models and Early Access Schemes for Orphan Drugs: a Belgian Case Study

Abstract

Background: Although some countries have made specific adjustments in their healthcare system to accommodate often-expensive orphan drugs, availability of these orphan drugs is still not evident. This study investigates alternative financing models and early access schemes for orphan drugs in the context of the Belgian healthcare system. Methods: Three focus group discussions were held with a panel of eleven experts from the Belgian Drug Reimbursement Committee, the Colleges for Orphan Drugs, the pharmaceutical industry, physicians, ethicists and pharmacists. Retrieved data were pseudonymised, analysed and coded according to the Qualitative Analysis Guide of Leuven.Results: Experts disfavoured an insulated orphan drug fund as well as private insurance, as isolation of a separate budget and a mostly profit-driven mechanism, respectively, would contradict the Belgian fundamental principle of solidarity. Moreover, an insulated fund could potentially reproduce the same budgetary constraints as the general reimbursement system albeit on a smaller scale. As the Special Solidarity Fund is intended for urgent care and exclusively accommodates eligible to the criteria, its design would not allow general financing of orphan drugs. Overall, implementation of an alternative financing model was not endorsed, instead, suggestions to improve the current reimbursement system were preferred; increased collaboration and transparency, robust and quality real-world evidence but also digitalization of data were elements mentioned. Alleviating administrative burden and simplifying the admission process for compassionate use program/medical need program and early treatment reimbursement should be prioritized to facilitate early access. Furthermore, a legal framework for off-label use could stimulate proper adoption. Efforts on collaboration of expertise centres and coordination of orphan drug databases across Europe could foster a robust data network to support orphan drug availability in individual countries. Conclusions: This research reveals that reassessing current financing models and early access schemes by eliminating inadequacies, may be more conducive than establishing alternative systems to increase availability of orphan drugs in Belgium. Other jurisdictions may rely on this information to review their own models of early access and financing and cultivate a more sustainable delivery of orphan drugs.