Pharmaceutical companies may need to submit complementary Real-World Evidence (RWE) generated from Hypothesis Evaluating Treatment Effectiveness studies using RWD (RWD-HETE) to reinforce clinical evidence in Health Technology Assessment (HTA). The study aimed to understand the HTA bodies’ theoretical requirements for RWE, their real uses and acceptance across 7 countries.

Description:

Acceptance of RWE was described across 7 countries (Brazil, Canada, France, Germany, Italy, Spain, United-Kingdom) through readiness grids covering: 1- methodological and theoretical requirements based on HTA guidelines, 2- real uses based on industrials’ experience, 3- acceptance based on case-studies and 4- a targeted literature review to complement the findings.

Some countries (France, Germany, United-Kingdom) have developed methodological guidelines, which differ in terms of study design and data sources. Reported real uses have shown that local data sources are preferred. Submission, assessment and appraisal processes are specific to each HTA body; some HTA agencies allow early meetings for protocol and/or results validation but it is not always used in practice. Most affiliates do not have external stakeholders involved in protocol and results validation, and only some HTA bodies involve key opinion leaders, experts or medical societies in assessment and appraisal (Brazil, Canada, United-Kingdom, in some circumstances). Case-studies highlight that differences in submission, assessment and appraisal impact observed acceptance.

Lessons Learned:

While there are still disparities between HTA agencies on the acceptance of RWE, countries seem to face common challenges to explore the potential for RWD-HETE. There is a need from HTA bodies to: 1- be specific on the formal process to facilitate the collaboration and communication between industrials, external stakeholders and HTA bodies; 2- publish clear and detailed guidelines on RWD-HETE, including RWD methodologies supporting evidence for new types of clinical trials likely to accelerate the availability of innovative medicines.

Stakeholder perspective:

Pharmaceutical industry (7 HTA countries)