Omakase has presented the last POSTER “Drivers of Successful Reimbursement Decisions of Orphan Drugs in Spain” at World Orphan Drug Congress Europe were the results of the logistic regression analysis we have just conducted to assess which clinical and regulatory variables influence the Price and Reimbursement (P&R) decisions for Orphan Drugs (ODs) in Spain. In Spain, the Interministerial Commission on Drug Pricing (CIPM) includes in their P&R resolutions the criteria used to justify such decisions; however, no information is provided on how these criteria are defined and measured.
Regarding the results of the logistic regression analysis, ODs with a positive Therapeutic Positioning Report (TPR) conclusion (p<0.1), not subject to a conditional approval by the European Medicines Agency (EMA) (p<0.5) and approved without the obligation to conduct a post-authorisation safety study (PASS) (p<0.5) were statistically significant, and therefore, would be more likely to obtain P&R approval in Spain. Therefore, this study highlights that traditional evaluation criteria, such as safety and efficacy, would be the main drivers of positive P&R decision for ODs in Spain. A new pathway to assess the additional benefit is needed to interpret the value of ODs.