OBJECTIVES Real-world evidence (RWE) complements safety and efficacy data generated from randomized controlled trials and reflects real-life use of health technologies in clinical practice. In developed markets, regulators are accelerating access to new interventions, and payers can utilise RWE to manage uncertainty around the value of new products. However, the use of RWE in emerging markets is less clear. We investigated current practices for RWE use and its impact on reimbursement processes for pharmaceutical products.

METHODS Targeted searches of grey literature and websites of health-technology assessment (HTA)/reimbursement agencies in England (National Institute of Health and Care Excellence; NICE), Australia (Pharmaceutical Benefits Advisory Committee; PBAC) and Canada (Canadian Agency for Drugs and Technologies in Health; CADTH) were conducted to identify policies for RWE use in HTA and reimbursement processes. Evidence exemplifying the impact of RWE on reimbursement processes was also extracted.

RESULTS Formal policies for RWE use in reimbursement decision-making were identified in three areas: scoping before assessment (NICE, CADTH), during HTA assessment to address clinical and cost effectiveness and manage uncertainty through conditional reimbursement (NICE, PBAC, CADTH), and after technology assessment to inform reassessments and confirm value (NICE, PBAC, CADTH). Examples, predominantly from developed markets, of the direct impact of RWE on reimbursement processes were identified during reimbursement decision-making - including in pharmacoeconomic analysis, conditional reimbursement and to support the release of full payment - and after reimbursement, to counter formulary exclusion and to provide evidence for disinvestment. Initiatives for RWE generation and use, and challenges and potential solutions for use of RWE in reimbursement processes were also identified.

CONCLUSIONS In developed markets, RWE influences all stages of the reimbursement decision-making process, however policies for RWE use differ between HTA/reimbursement agencies in different countries. A more consistent policy approach could be valuable and could inform development of procedures for RWE use in emerging markets.