Documents

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.

RWE4Decisions held trans-national multi-stakeholder workshops to discuss CED plans for two fictitious highly innovative treatments for rare disorders. The nature of the fictitious treatments was contrasting as one treatment was life-long and the other once-in-a-lifetime. Each rare disease had no existing disease modifying treatments, and the new treatments had a high price and major uncertainties in the evidence base available to HTA/Payers. Pros and cons of real-world data sources that might resolve the decision-relevant uncertainties were considered. Challenges in accessing the data arising due to the rarity of the condition, alignment of post marketing data collection requirements, publication of detailed data collection plans and data governance of data provided by highly specialised centres were discussed. Potential actions that could be taken by individual stakeholders or collaborative initiatives were agreed.

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.

Authored by Professor Lieven Annemans, University of Ghent. This paper develops a technical but pragmatic tool and methodology that allows the uncertainties in evidence for a specialised treatment for a rare disease to be made explicit, to be prioritized and to be addressed in an adequate and timely way. Additionally, the paper provides guidance on the potential of real-world evidence (i.e. data collected outside the context of randomised controlled clinical trials; RWE) to help address such uncertainties.