OBJECTIVES: Launching an orphan drug in the U.S. can be challenging. Hurdles may include limited clinical trial data, lack of comparators or established endpoints and the need to clarify which outcomes are relevant to payers. In this study, we designed and implemented a value communications plan (VCP) in support of Product X which called for early, robust and extended engagement with payers.

METHODS: The VCP called for: (1) an expert panel (payers and clinicians) to validate an already-developed evidence generation plan (EGP); (2) following the trial data readout, a U.S. payers’ advisory board to obtain their insights into the clinical data and input into the Product X draft value story (VS); (4) VS testing via payer interviews; (5) completion of a payer toolkit, value pack, dossier in the Academy of Managed Care Pharmacy (AMCP) format and global value dossier (GVD) shortly after the Food and Drug Administration’s approval of Product X.

RESULTS: The expert panel confirmed what the relevant outcomes were from the payer perspective. The advisory board highlighted the importance of robust clinical data and specific rather than general value messages; it also allowed payers to advice which datasets/outcomes would serve the VS best. The VS reflected what resonated to payers due to their early involvement in its draft. Early development of tools and the AMCP-format dossier ensured timely U.S. formulary submissions and affiliate alignment globally. Following the U.S. launch, EU5 payer advisory boards and interviews were critical for the VS, tool and GVD updates, demonstrating the value of sustained payer engagement for the European Medicines Agency submission.

CONCLUSIONS: Early and continued payer engagement is vital. VCP integration with an EGP informed by early scientific advice is important to align the clinical and payer imperatives. Effective payer engagement enhances evidence communication and promotes the advancing of evidence to action.