BACKGROUND: Health authorities and payers increasingly recognize the importance of patient perspectives and patient-reported outcomes (PROs) in health care decision making. However, given the broad variety of PRO endpoints included in clinical programs and variations in the timing of PRO data collection and country-specific needs, the role of PRO data in reimbursement decisions requires characterization. Payer perspectives on patient-reported outcomes in health care decision making: oncology examples
OBJECTIVE: To determine the impact of PRO data on market access and reimbursement decisions for oncology products in multiple markets and to assess the impact of PRO data collected after clinical progression on payer decision making.
METHODS: A three-part assessment (targeted literature review, qualitative one-on-one interviews, and online survey) was undertaken. Published literature was identified through searches in PubMed/MEDLINE and Embase. In addition, a target search was conducted of health technology assessment (HTA) agency websites in the United States (US), the United Kingdom (UK), France, and Germany. Qualitative one-on-one interviews were conducted with 16 payers from the RTI Health Solutions global advisory panel in 14 markets (Australia, Brazil, France, Germany, Italy, South Korea, Netherlands, Poland, Spain, Sweden, Taiwan, Turkey, the UK, the US [n=3]). Of the 200 payers and payer advisors from the global advisory panel invited to participate in the online survey, 20 respondents (China, France, Germany, Spain [n=2], Taiwan, the UK, and the US [n=13]) completed the survey, and 6 respondents (Australia, South Korea, and the US [n=4]) partially completed the survey.
RESULTS: Reviews of the literature and publicly available HTAs and reimbursement decisions suggested that HTA bodies and payers have varying experience with and confidence in PRO data. Payers participating in the survey indicated that PRO data may be especially influential in oncology compared with other therapeutic areas. Payers surveyed offered little differentiation by cancer type in the importance of PRO data but felt it most important to collect PRO data in phase 3 and postmarketing studies. Payers surveyed also anticipated an increasing significance for PRO data over the next 5 to 10 years. Characteristics of PRO data that maximize influence on payer decision making were reported to be (1) quality, well-controlled, and transparent PRO evidence; (2) psychometric validation of the PRO measure in targeted populations; and (3) publication in peer-reviewed journals. In markets with decentralized health care decision making, PRO data currently have more influence at the local level. Inclusion of PRO data in cancer treatment guidelines is key for centralized markets. Payers surveyed generally considered collecting PRO data postprogression to be useful. Of the 16 interviewees, 11 indicated that it is worthwhile to collect PRO data postprogression and that positive PRO data may support continued therapy at the physician’s discretion upon regulatory approval, even in progressive disease.
CONCLUSIONS: PRO data may help to differentiate treatments, particularly after clinical progression in oncology. Payers worldwide recognize high-quality PRO data as a key component of their decision making process and anticipate the growing importance of PRO data in the future.
