Objectives: In 2000 the Orphan Drug Act was designed to supported drug development for rare disease in Europe. Now thirteen years after the Act was passed, orphan drug development has immensely increased and health care systems are challenged to find more appropriate assessment mechanism for the reimbursement of orphan drugs. Public awareness has lately been raised by major discussions about the discontinuation of reimbursement for several orphan drugs. By conducting qualitative interviews this article aims to identify the view of different stakeholders about recent and future changes within the reimbursement assessment and its evidence requirements for orphan drugs. Methods: Twenty semi-structured interviews were conducted with relevant stakeholders from the orphan drug community. Interviewees were scientific experts, reimbursement agencies, industry and patient organizations from five European countries. The interviews were analyzed with the framework analysis technique. Results: All twenty stakeholders have reported about recent or future changes in their national reimbursement practice for orphan drugs. The most emerging theme focused around more scrutiny in the reimbursement assessment of orphan drugs. Conclusions: All twenty stakeholders gave recommendations about a stronger European cooperation for the value assessment of orphan drugs, with eleven stakeholders suggesting a European reimbursement system for orphan drugs.
Krueger, L. J., Tamminga, J. J., Wijnen, B., Hiligsmann, M., & Evers, S. M. (2014). AN EUROPEAN OVERVIEW OF THE FUTURE CHANGES IN EVIDENCE REQUIREMENTS FOR THE REIMBURSEMENT OF ORPHAN DRUGS-A STAKEHOLDER ANALYSIS. Value in Health, 17(3), A236. https://doi.org/10.1016/j.jval.2014.03.1377