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.