Rare disease market access strategy

A pre-commercial biopharma needed to know where its lead asset belonged in the treatment sequence once two new therapies arrived and what evidence would lead payers to cover it.

S662-121

New entrant efficacy benchmarked against scenarios

Situation

Market access challenge

Client is a pre-commercial stage biopharma focused on genetic diseases with a unique early-stage platform.
Has built knowledge base on market dynamics for lead indication target. Leadership team wanted additional perspective on market access especially given the recent introduction of two new therapies.
Needed fact base and analysis on several questions as input for a future board meeting.

Project

Drug perception & market analysis

Leveraged secondary research and 20+ expert interviews to assess perception and provide qualitative assessment of program opportunity, including relative positioning with respect to new entrants.
Developed economic model for program opportunity in Japan and China.
Provided recommendation on initial development plan and key unknowns to explore in next 12-18 months.

Blinded profiles tested with 20+ clinical experts

Frameworks used to set line-of-therapy strategy

Outcome

Repositioned development plan

Client revised to strategy to ensure that it was appropriately positioned with respect to new therapies (1st line vs. 2nd line) and incorporated the relevant end-points to make the differentiation and value as clear as possible.

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