Actions for stakeholders to develop better real-world evidence for HTA bodies/payers decision making.

Objective: In 2020, RWE4Decisions, a multi-stakeholder initiative commissioned by the Belgian payer, published stakeholder actions to support the generation, analysis, and interpretation of real-world evidence (RWE) to inform the decision making of health technology assessment (HTA) bodies/payers for highly innovative medicines in the European Union (EU). Since 2020, changes in the decision-making environment and advancements in RWE have created an impetus to update stakeholder actions for the EU and Canada.

Methods: RWE4Decisions' experts led focus groups with individual stakeholder groups (HTA bodies/payers, pharmaceutical industry, clinicians, patients, registry holders, and data analytical experts). Each focus group crafted new actions for their stakeholder, then the actions were discussed and revised in a multi-stakeholder meeting, a public webinar, and a public consultation. Themes across actions and meetings were identified.

Results: Detailed new actions for each stakeholder group are presented. Key themes identified are the need to address interorganizational fragmentation regarding secondary data use and methodologies to build robust RWE. HTA bodies/payers need to develop a common vision about the potential use of RWE. The role of the whole clinical team as primary data collectors is critical. Opportunities for scientific advice across the life cycle of a medicine are essential, and the implementation of RWE guidance related to HTA is paramount. Progress requires specific, operational actions and a collective effort by a variety of stakeholders.

Conclusions: Carrying out these actions will facilitate the development of methodological best practices for generating RWE to inform HTA of highly innovative medicines and build trust between stakeholders in the use of RWE.