Real world evidence (RWE) is changing the overall data landscape and it has potential to advance the evaluation of real world performance (comparative effectiveness) of healthcare technologies by providing a greater quantity and quality of evidence. However, many are concerned that non-randomized RWE may be substituted for RCT data and thus increase uncertainty about effectiveness. This presentation sets out the opportunities and challenges for use of RWE by payers and HTA bodies to evaluate health care technologies.

Current uses, opportunities and challenges were identified via a literature review and interviews with nine experts. Interim results were discussed at the 2017 ICER Policy Summit, which brought together leaders from payer and life sciences organizations, to develop specific and actionable recommendations for the use of RWE in drug coverage and policy decision-making.

RWE is utilized for multiple purposes in the US and globally, including: aiding design of drug development pathways; supporting regulatory approval decisions; monitoring safety; and informing HTA assessments and payer coverage decisions. Some stakeholders see great value in RWE and want to make greater use of these data sources, including for: drug effectiveness evaluations (including supplementing network meta-analyses); innovative study designs (including pragmatic trials); real time patient monitoring; and adaptive pathways or coverage with evidence development. However, others see numerous challenges, many of which are related to the quality and reliability of RWE sources. Acceptance of an expanded future role for RWE is not universal, and payers and developers must work together to find mutually beneficial strategies for progressing the development and use of RWE.

Specific and actionable recommendations will be presented which highlight the role that each stakeholder group can play in overcoming the challenges and realizing the potential for RWE.