Best practices in designing preapproval information engagements for US health care decision makers

As high-cost and innovative therapies continue to enter the market, health care decision makers (HCDMs) are expressing a need for early information on a product’s clinical and economic impacts. Preapproval information exchange (PIE) fulfills these data needs by allowing manufacturers to share drug information with HCDMs prior to US Food and Drug Administration approval. With recent regulatory milestones, such as the Pre-approval Information Exchange Act of 2022, HCDMs look to leverage PIE to forecast budgets and inform reimbursement decisions. However, a lack of stakeholder alignment has challenged the evolving applications of PIE. In addition, manufacturers are still seeking regulatory clarity regarding best practices, and many are developing their own policies for content dissemination. Varying practices have led to heterogeneity of preapproval communications across manufacturers, which may not fully align with HCDM needs and interests. However, recently collected survey data from FormularyDecisions, which focused on HCDM perceptions of both PIE webinars and other formats of PIE (eg, PIE decks and dossiers), indicate that HCDMs have strong preferences regarding the timing and content shared in preapproval engagements. Additionally, product indication and clinical trial information are highly valued, and although desired by HCDMs in other studies, in FormularyDecisions survey data, exact pricing data do not currently appear to be a critical component of PIE. Preapproval communications are expected less than 1 year before anticipated product approval, and PIE webinars, specifically, should prioritize therapeutic areas and products anticipated to have a significant impact on organizational budgets. Although HCDMs prefer nonmanufacturer representatives for PIE webinars and virtual presentations, health outcome liaisons or medical science liaisons are ideal among manufacturer representatives for in-person preapproval engagements. The expectations of HCDMs should be considered as manufacturers establish PIE practices to ensure the exchange of quality and relevant information.