Current and Future Trends in P&R Decisions of Oncology and Onco-Hematology Drugs in Spain: A Retrospective and Forum Discussion Study

OBJECTIVES: The pricing & reimbursement (P&R) decision for a new drug in Spain is based on the criteria set in the Law on Guarantees and Rational Use of Medicinal Products (Royal Decree 1/2015) and the Therapeutic Positioning Report (TPR). Despite a clear framework, the weight of the different factors used is not transparent. This study aims to understand the main factors influencing P&R decisions in the first negotiated indication of oncology and onco-hematology drugs in Spain as well as future trends. The factors explored are related to the clinical trial design, efficacy results, orphan drug designation, perceived added clinical value, and time-to-market.

METHODS: Based on the Law on Guarantees and the information contained in the TPR, a series of hypotheses on the relationship between several variables and the reimbursed price in Spain have been developed and then statistically analyzed using Wilcoxon or Kruskal-Wallis tests.

RESULTS: A sample of 32 drugs was analyzed for their first indication. There were statistically significant differences or trends between variables such as orphan drug designation, the line of treatment, the perceived added clinical value, and the reimbursed price. However, no relationship was observed in the clinical trial design or the clinical trial efficacy results.

CONCLUSIONS: Findings of the analysis indicate the added clinical value as a key criterion in decision-making process. However, there is no clear relationship between the weight of the other factors when predicting P&R results, although there are some trends between certain variables and the reimbursed price. The P&R process should be based on a more structured, transparent, and predictable methodology that articulates the priorities set within the Royal Decree. It would be interesting to perform the analyses with a larger sample, but these results can serve as a starting point to promote the transparency of the evaluation process in Spain.