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case studies

Forecasting global value assessments for rare disease drug launch


Enabling clients to anticipate payer and HTA value assessments across a range of complex international markets.


Emerging biotechnology company

Name of service provided

Pre-launch pricing, market access and value proposition strategy


Key insights and strategic recommendations across the countries of interest (Australia, Canada, EU, Japan, UK, and US)


Our team came together to help an emerging biotechnology company develop a robust multi-national pricing and commercialization strategy for a drug to treat an ultra-rare neurodegenerative disorder. 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 is largely uncharacterized, is typically identified via differential diagnosis, lacks a diagnosis code, has unknown prevalence, and affects a heterogeneous patient population. This posed significant challenges in anticipating payer perception and reimbursement.

The client required 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.


Forecasting Global Value Assessments for Rare Disease Drug Launch



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 analog products in US and international markets. For each analog, we analyzed how clinical development programs translated into product labels and payer reimbursements.

Third, leveraging these insights, 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 our insights and recommendations, the client better articulated the product’s value proposition, and refined its pricing strategy in line with reimbursement and access challenges. 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.

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