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Positioning for a novel, ultra-orphan specialty drug: KOL and payer deep dive
Client Problems
- A privately-held, development stage pharmaceutical company wanted to understand the prevalence and unmet needs of an ultra-orphan disease and how a new drug might be priced and positioned to optimize uptake
- More tactically, the team wanted to know how to identify these ultra-rare patients for clinical trials and commercialization
What We Did
- Primary research: Qualitative in-depth phone-Internet interviews with commercial payers and KOL specialists (geneticists and pediatric endos)
- Secondary research: Benchmarking analysis of ultra-orphan analogs for pricing, patient identification schemes and positioning
Our Results And Insight
- Uncovered the treatment flow -- diagnosis and treatment starts with a small group of specialists and expands into the community
- Uncovered a common set of pricing benchmarks by mapping the price-prevalence relationship in ultra-orphans
- Prioritized the key factors driving the payer coverage decision, several of which were unique to the client’s situation
- Recommended a set of targeted, cost-effective pre-commercial tactics focusing on 3 key areas
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Service Areas Details
Market Research (KOL, Payor, Benchmarking)
Therapeutic Areas Details
Gene Therapy & Orphan Disease (Orphan)
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