Case Study: Indication expansion assessment and evidence requirements for a novel agent in alpha-thalassemia
Project Objective
The client was preparing for an indication expansion of Product X, which was already indicated in other blood conditions, into alpha-thalassemia, an inherited blood disorder
The client wanted to understand the commercial opportunity and risks of indication expansion from an access and reimbursement perspective in Italy, Greece, China, Thailand, and Turkey, which have a high prevalence of disease
The aim was to inform the commercial strategy for indication expansion
Windrose Approach
Windrose conducted secondary research and leveraged the clients’ internal resources to develop preliminary hypotheses for the pricing and market access (P&MA) potential of Product X
This informed comprehensive hypotheses related to Product X’s value requirements as a standalone indication and as an indication expansion, as well as the payer evaluation process and P&MA impact of the indication expansion
Next, we sought feedback from market affiliates to gain an understanding of Product X’s current access status and value expectations to inform launch scenarios for expansion
Importantly, we identified profile attributes and market system characteristics that might have presented challenges to broad reimbursement, as well as potential mitigations to test in research
Finally, we conducted 20, 60-minute in-depth interviews with payers and KOLs to test hypotheses across various launch scenarios
Impact
We provided feedback on stakeholders’ perceptions and understanding of the treatment landscape, with focus on the relevance and expected evolution of categorization of alpha-thalassemia patients
We assessed payer value perceptions of Product X and the potential evidence gaps that exist for P&MA decision-making
Next, we outlined the expected reimbursement population and pricing strategy, highlighting key market-specific insights (e.g., eligibility for the simple renewal pathway in China, claw back vs. confidential discount in Greece)
Finally, we provided market-specific recommendations to shape payer understanding of alpha-thalassemia, bolster the evidence package to prepare for launch, and tailor launch planning to maximize the commercial opportunity of Product X