OBJECTIVES: To compare evidence synthesis strategies for EU’s Joint Clinical Assessment (JCA) versus US CMS's Inflation Reduction Act (IRA) Drug Pricing Negotiation (DPN) process.
METHODS: We compared guidelines for JCA vs IRA in terms of products covered, approval/marketing stages, endpoints, and types of evidence.
RESULTS: There are substantial differences between evidence synthesis requirement for the JCA vs IRA. The JCA guidance applies to new oncology medicines, advanced therapies (i.e., cell and gene therapies), and medical technologies for 2025; it will apply to therapies for rare diseases starting January 2028 and all new medicines starting January 2030. The IRA DPN applies to products approved or licensed in the US for 7 (small-molecules) or 11 years (biologics). The JCA guidance refers to evidence of clinical benefits of given treatment versus existing treatments while the DPN guidance requires evidence enabling comparison of clinical as well as other benefits (e.g., caregiver perspectives, productivity) for given treatment versus its primary comparators. Submissions for both the DPN and the JCA require careful consideration of comparators and relevant outcomes (e.g., region-specific treatment practices [JCA], improvements in clinical endpoints [JCA and DPN], as well as changes in symptoms or PROs, changes in productivity and QoL, and caregiver perspectives [DPN]) for a given product and indication. This evidence will be collected via systematic literature reviews and reported via both qualitative evidence synthesis and quantitative methods such as indirect treatment comparison (ITC); for the JCA, these data must be provided within a structured timeline and are subject to updates.
CONCLUSIONS: While differences exist between evidence synthesis requirements for the DPN and the JCA with respect to designing and conducting quantitative and qualitative evidence synthesis, for both submissions, there is the potential to gain efficiencies, including through the use of AI-assisted platforms like Nested Knowledge or DistillerSR to assist in literature reviews and evidence synthesis.
