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Positioning for a novel, ultra-orphan specialty drug: KOL and payer deep dive

Service Areas
Therapeutic Areas

Gene Therapy & Orphan Disease

Client Type

Pre-clinical client

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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