Impact of RWE on re-assessment of medicines by HTA agencies

The use of real-world evidence (RWE) is important for access and reimbursement decisions for a number of reasons. Use of RWE may help improve understanding of the patient population who will receive a medicine. It may also improve the understanding of relative effectiveness of medicines by supplementing evidence from conventional randomised controlled trials (RCTs). (for more information, see Real-World Evidence and its Importance in Medicines Development).

RWE may be presented at the initial assessment of a medicine by a health technology assessment (HTA) agency, or it may be collected after authorisation and used in re-assessments.

Haute Autorité de Santé (HAS), the national HTA agency in France, undertook a review as part of IMI GetReal to assess the association between the level of reimbursement granted after a medicine was re-assessed by the agency and the alignment of RWE at the time of re-assessment with the RCTs available previously. It was not possible to conclude with certainty that the reimbursement decisions made at the re-assessment of the medicine were directly related to the (newly available) RWE, as other factors may have contributed to the decision.

For more information about this review, see Comparison of Reimbursement Decisions Before and After Real-World Evidence Review in France.

Managed entry agreements or conditional reimbursement

Real-world evidence may be requested by an Health Technology Assessment (HTA) agency in the context of a managed entry agreement to better understand areas where there was greater uncertainty in the original submission for reimbursement. Managed entry agreements, also referred to as managed access agreements or conditional reimbursement schemes, are arrangements where an HTA agency recommends reimbursement of a technology under certain conditions, such as further data collection (commonly real-world data) to resolve areas of uncertainty, combined with some form of commercial or risk-sharing agreement (such as a price reduction). Such agreements are common in situations where the decision maker considers the technology to be very promising but there is a high level of uncertainty about the relative effectiveness or other aspects of the medicine. The intention is to re-assess the technology after the evidence is collected or generated.

From 2006 to 2012, Zorginstitut Netherlands (ZIN), the national HTA agency in the Netherlands, introduced a managed entry agreement or conditional reimbursement scheme (referred to as conditional financing). A review of the scheme was undertaken as part of the GetReal project, which is summarised in the RWE Navigator page on Implementation of Conditional Reimbursements Schemes: Experience from the Netherlands.

IMI ADAPT-SMART undertook some work on the use of managed entry agreements in the context of adaptive pathways. For more information, see the report of the ADAPT-SMART Workshop on managed entry agreements.