OBJECTIVES: To understand how PRO data from clinical trials are utilized in market access decision making in oncology and other disease areas in markets with centralized healthcare systems.
METHODS: PubMed/MEDLINE, Embase, ISPOR databases, and regulatory and HTA websites for the EMA, the UK, France, and Germany were searched to identify PRO data included in regulatory and HTA submissions of four oncology drugs: bevacizumab, pemetrexed, sunitinib, and crizotinib. One-on-one interviews were conducted with 10 payers from different countries with centralized healthcare systems in 2014. An online assessment was conducted (December 8, 2014, to March 4, 2015) with 5 completed surveys (China, France, Germany, Taiwan, the UK) and 2 partially completed surveys (Australia and South Korea) by payers from the RTI Health Solutions Global Payer Advisory Panel.
RESULTS: Reviews of HTA and reimbursement decisions indicate that HTA bodies have varying levels of familiarity and confidence in PRO data. All 10 payers indicated that it is worthwhile to collect PRO data in clinical trials for oncology, particularly in phase 3 and postmarketing studies. Payers speculated that PRO data will increase in importance over the next 5-10 years and could be a key differentiator for new therapies. Payers offered little differentiation in the importance of PRO data by cancer type. Payers indicated that the quality of the PRO evidence is paramount. Adoption of PRO data by national and international cancer guidelines is key for centralized markets. Literature review findings corroborated with payer interviews indicated that inclusion of PRO data for crizotinib was crucial to achieving a price premium in Germany, with additional benefit based on PRO data on symptoms and quality of life.
CONCLUSIONS: There is growing recognition that the patient perspective is important in market access decision making in centralized markets and PRO data can be a key differentiator among therapeutic options.
