Plain Language Summary

The article investigates how real-world evidence (RWE) is being used in health technology assessment reassessments by 6 major HTA agencies. These agencies include those from Canada, England, France, Germany, The Netherlands, and Australia. The study highlights that RWE is increasingly utilized for reassessing health technologies, especially in cases involving cancer treatments and rare diseases, to resolve uncertainties that were present at the time of initial product launch.

The research analyzed 40 health technology assessment reassessments conducted between 2018 and 2023. It found that more than half of these reassessments incorporated RWE, showing its growing role in the evaluation process. RWE was particularly prevalent in reassessments concerning orphan drugs, which are medicines used to treat rare conditions. This indicates that RWE is valuable in circumstances where traditional clinical trials are challenging or infeasible.

RWE helps address clinical uncertainties, such as those related to the effectiveness and safety of treatments. However, the study found that no new RWE comparative effectiveness studies were conducted for these reassessments. Instead, RWE was often used alongside updated clinical trial data to support decision making. Despite its potential, RWE's use in health technology assessment reassessments is not yet optimized. Agencies and sponsors need to work together to align on evidence needs and improve study feasibility, ensuring that RWE can effectively contribute to reassessments.

For patients, this means that RWE can potentially enhance the evaluation of treatments they rely on, ensuring therapies are safe and effective. Healthcare decision makers should consider adopting a lifecycle approach to assessments, which involves evaluating treatments at multiple points to capture their evolving impact on health systems. Researchers are encouraged to focus on improving the quality and application of RWE, as its validity remains a concern due to possible bias and data quality issues.

To optimize the use of RWE, HTA agencies should allow sufficient time for collecting real-world data, which typically matures over several years. Agencies should also issue detailed guidance on generating and using RWE, including specifying preferred data types for different types of uncertainties. Through better collaboration and clearer guidelines, agencies can ensure that RWE not only complements clinical trial data but plays a crucial role in health technology reassessments.

^{Note: This content was created with assistance from artificial intelligence (AI) and has been reviewed and edited by ISPOR staff. For more information or for inquiries on ISPOR’s AI policy, click here or contact us at info@ispor.org.}