OBJECTIVES: To determine how impactful patient-reported outcome (PRO) data from clinical trial programs are on market access decision making in oncology and other disease areas in decentralized markets.
METHODS: A review of regulatory, health technology assessment (HTA), and third-party websites and published literature was the basis for six qualitative one-on-one interviews conducted with payer decision-makers (payers) in Brazil (1), Italy (1), Spain (1), and the United States (3) in 2014.
RESULTS: Reviews conducted of HTA content and reimbursement decisions indicate that HTA bodies have varying levels of familiarity and confidence in PRO data. All six payers indicated that it is worthwhile to collect PRO data in clinical trials for oncology, particularly in phase 3 and postmarketing studies. The payer in Spain was aware of a specific example where PRO data were crucial to decision-making for oncology. However, all six payers indicated that PRO data will increase in importance over the next 5 to 10 years and could be a key differentiator for new therapies. Payers did not differentiate the importance of PRO data by cancer type. All six payers indicated that the quality of the PRO evidence is paramount to consideration. In addition, they felt that PRO data currently have the most impact at the local level; one US payer noted there is significant opportunity to use PRO data to justify preferential product use and could be incorporated into hospital contracting.
CONCLUSIONS: There are minimal requirements or guidelines specifically addressing whether and how health care decision-makers use PRO evidence; therefore, inclusion of PRO data in payer decision-making is currently determined on a case-by-case basis. There is growing recognition that the patient’s perspective is important in market access in decentralized markets.
