Forecasting Global Value Assessments for Rare-Disease Drug Launch

The client faced difficult questions about how payers would react to its product, considering the endpoints and patients included in its clinical trials. The client’s ultra-rare target indication—which is largely uncharacterized, is typically identified via differential diagnosis, lacks a diagnosis code, has unknown prevalence, and affects a heterogeneous patient population—posed significant challenges in anticipating payer perception and reimbursement. The client sought our expertise in anticipating payers’ value assessments of its product, mitigating any risks to adequate reimbursement, forecasting consequences for revenue, and synthesizing business intelligence takeaways for future clinical trial design and pre-launch planning.

First, we conducted a structured literature review of academic articles and health technology assessments (HTAs) on the indication, assessing its epidemiology, disease burden, standard of care, clinical guidelines and recommendations, unmet needs, and health economic indicators such as quality-adjusted life years. We also reviewed the client’s clinical trial protocols and data to identify gaps in evidence generation.

Second, we assessed the coverage and reimbursement landscape for a set of analogue products in US and international markets. For each analogue, we analyzed how clinical development programs translated into product labels and payer reimbursements.

Third, using information gathered during previous steps, we convened two advisory boards, one on the US market and another on international markets. These advisory boards included physicians, payers, pharmacy benefit managers, patient advocates, HTA experts, and other stakeholders. They assessed the client’s target product profile; provided guidance on pricing, reimbursement, and access strategy; evaluated coding barriers and opportunities; and identified clinical and real-world evidence programs that could be incorporated into the client’s commercialization strategy for each market.

Our work has enabled the client to anticipate payer and HTA value assessments of its product across a range of complex international markets. Using insights and recommendations from this project, the client can better articulate the product’s value proposition and has identified reimbursement and access challenges and refined its pricing strategy. It has also deepened its general understanding of how regulators, payers, and HTA bodies assess different clinical trial endpoints, which will enable more refined future clinical trial designs that maximize the value propositions and reimbursement potential for its products.