Presentation (CTI)

OBJECTIVES: In 2025, major policy reforms significantly reshaped pharmaceutical pricing and market access in the US and EU. The Most Favored Nation (MFN) policy was introduced in the US to align its drug prices with peer OECD countries. The Joint Clinical Assessment (JCA) was introduced in the EU to standardize relative effectiveness assessment between new and existing treatments across member states. Here we provide a global evidence generation strategy incorporating these policies plus the 2022 Inflation Reduction Act (IRA) and recommend steps for pharmaceutical manufacturers to optimize launch.
METHODS: A targeted literature review of policy papers and peer-reviewed publications was conducted to determine the requirements, impact and implications of these policies on evidence needed for launching new treatments in the US and EU.
RESULTS: Three to five years prior to launch, manufacturers should design clinical trials focusing on relative effectiveness and appropriate PICOs, and initiate RWE planning to support IRA price negotiations. One to two years before launch, manufacturers should develop ITCs and SLRs for JCA, map IRA eligibility timelines, and optimize launch sequence. EMA and JCA submissions should then be developed in parallel and US value narratives aligned with CMS negotiation criteria (i.e. clinical differentiation and unmet need). Post-launch, RWE for comparative effectiveness, subpopulation benefits and safety analysis should be initiated. Medicare utilization should be tracked to assess likelihood of IRA negotiation whilst global pricing strategies should be monitored to anticipate further MFN negotiations. JCA outcomes for national HTA submissions should be leveraged. Direct to consumer US channels should be created to reduce costs associated with intermediaries.
CONCLUSIONS: Recent policy changes have substantially increased market access requirements for value demonstration. Manufacturers must proactively integrate and optimize clinical development, evidence generation, and pricing and reimbursement strategies across US and EU markets to overcome these and secure high-value wide-ranging market access.