The role of patient-reported outcomes in health technology assessments: global practices and future implications

Patient-reported outcome (PRO) data capture patients' perspectives on their disease and its treatments; however, use of such data in health technology assessments (HTA) and pricing/reimbursement processes is limited. We provide considerations to support the incorporation of PROs in payer decision-making.

A review of guidance documents released by payers until 2024 regarding requirements for using PRO evidence and scientific literature published during 2015-2024 on the trends of PRO use by payers informed the development of a discussion guide. Using this, 15 experts from seven countries were interviewed (one-on-one) to seek their opinions on PRO use in HTAs and identify potential barriers to its adoption.

PRO-related guidance focused on the validity and reliability of PRO instruments, risk for bias, missing data, and economic modeling. Guidance varied between countries with essential details often missing. Expert interviews revealed that PRO use in payer evaluations may depend on established endpoints for indications and varied decision approaches and frameworks. Barriers to PRO use include the lack of capacity or technical expertise, instrument validity concerns, and data accuracy.

Barriers to the use of PRO data for evaluations require further efforts from all relevant stakeholders to promote PRO incorporation in payer decision-making.