How Influential Are Patient/Professional Group Submissions on Reimbursement Decisions for European Medicines Agency Orphan Drugs?

In an attempt to better understand the patient experience, health technology assessment (HTA) agencies have increasingly encouraged patient/professional group involvement in the HTA process, particularly for orphan diseases, which, by definition, are hindered by small sample sizes and limited clinical trials. However, are patient/professional group submissions associated with positive reimbursement decisions for orphan drugs? Furthermore, do the submissions actually influence reimbursement decisions?

Results indicate that patient/professional group submissions are not directly associated with positive reimbursement decisions within orphan drug HTAs. However, these findings reveal that there is a lack of transparent data regarding patient/professional group submissions. 

See our analysis and conclusions in the full research brief.


Original research presented at ISPOR 2015 in Philadelphia.

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