Case Study: Ex-US Orphan Indication Expansion & Launch Sequence Development
Project Objective
Our client was evaluating an orphan indication for Product X, which is currently indicated for another orphan indication in Europe, Japan, and Latin America
While the indication expansion represents a revenue opportunity, increasing the eligible population size compounded by a higher dose may result in a price renegotiation and increased budget impact concerns
Therefore, the client had 3 main objectives: 1) identify P&MA implications of indication expansion and risk mitigation, 2) assess evidence required to support market access and 3) determine the optimal launch sequence
Windrose Approach
We conducted secondary research on both indications, and then performed an analog analysis to provide insight into past payer reactions to indication expansion
The analogs and secondary research informed comprehensive hypotheses related to Product X’s value requirements, payer review process, the P&MA impact of a new indication and a higher dose on payer decision making
Finally, we conducted 22 in-depth interviews with stakeholders from France, Germany, Spain, UK, Taiwan and Brazil
Impact
Windrose provided the following:
Stakeholder perceptions of Product X in the new indication, including value, price and market access expectations and evidence requirements to support access and shape the clinical trial design
Perceptions of Product X’s indication extension, analysis of dosing scenarios, and expected payer management, including requirements to mitigate population size uncertainty
Qualitative launch sequence analysis, including international reference pricing (IRP) risk mitigation strategies
Clear and practical global recommendations as well as market-specific insights which the client used to inform their indication expansion strategy for Product X
Post launch indication, Estimated IRP impact, Windrose Consulting Group
Post launch indication, Illustrative Pricing, Windrose Consulting Group