RWE4Decisions

Advances in the digitization of health systems and expedited regulatory approvals of innovative treatments have led to increased potential for the use of real-world data (RWD) to generate real-world evidence (RWE) to complement evidence from clinical trials. However, health technology assessment (HTA) bodies and payers have concerns about the ability to generate RWE of sufficient quality to be pivotal evidence of relative treatment effectiveness. Consequently, there is a growing need for HTA bodies and payers to develop guidance for the industry and other stakeholders about the use of RWD/RWE to support access, reimbursement, and pricing. We therefore sought to (i) understand barriers to the use of RWD/RWE by HTA bodies and payers; (ii) review potential solutions in the form of published guidance; and (iii) review findings with selected HTA/payer bodies. Four themes considered key to shaping the generation of robust RWE for HTA bodies and payers were identified as: (i) data (availability, governance, and quality); (ii) methodology (design and analytics); (iii) trust (transparency and reproducibility); and (iv) policy and partnerships. A range of guidance documents were found from trusted sources that could address these themes. These were discussed with HTA experts. This commentary summarizes the potential guidance solutions available to help resolve issues faced by HTA decision-makers in the adoption of RWD/RWE. It shows that there is alignment among stakeholders about the areas that need improvement in the development of RWE and that the key priority to move forward is better collaboration to make data usable for multiple purposes.

This paper goes beyond strategic intent to consider actions that regulators, HTA bodies, Payers, clinicians and patients could take to improve the use of real-world data to inform their decision. Case studies of recent Payer/HTA assessments about highly innovative technologies showed that there was a lack of clarity about the Payer/HTA questions that could be answered by RWD and how the quality of real-world evidence (RWE) could be assessed. For each stakeholder group, recommended actions to support the generation, analysis and interpretation of RWD to inform decision-making were developed.

The paper discusses the usefulness of RWD throughout the lifecycle of innovative medicine, and the current issues in the collection, interpretation and implementation of RWD. In addition good practice principles and necessary actions to improve the use of RWD are proposed.

This paper outlines an overview of the principles of “value based pricing“ and reimbursement, followed by a discussion of the current uncertainty regarding clinical and financial outcomes at time of market access of innovations. A proposal is presented for outcomes based agreements that deal with this uncertainty and its causes.