Pre-Launch – Positioning and Value

Windrose conducted a pricing, market access and evidence gap assessment across US, EU4 and UK for two novel agents in a muscle weakness disorder

The client is developing two assets with different mechanisms of action (MoAs) for the treatment of a muscle weakness disorder
Chronic therapy currently entails generic immunosuppressants, with branded and biosimilar biologics used selectively, while blood-related products or procedure remain the only options for as-needed treatment of disease exacerbation; however, the landscape is evolving as new agents are expected to precede the launch of the client’s two assets
The client would like pricing and market access insights and recommendations to support commercial and development decisions in a dynamic competitive context and asset co-positioning

Assessed payer considerations for the disease landscape, clinical value perceptions and P&MA potential in the chronic and as-needed exacerbations settings
Identified value drivers and evidence gaps for reimbursement to drive development of risk mitigation strategies within the client cross-functional team
Evaluated all factors for the US, EU4 + UK markets individually, and for both the overall population and different patient subgroups, taking into account a potential loss of orphan drug designation

Generation of payer hypotheses on the clinical, humanistic and economic burden, current treatment options and unmet needs of the muscle weakness disorder
Analysis of the clinical value of emerging treatments by market and the differentiation of client assets against such products by treatment setting
Detailed evaluation of payer value perceptions of the target product profile for different positioning options, including recommendations on the trial design and endpoints that could increase value, and anticipated payer evaluation outcomes
Assessed price potential by positioning option via multiple approaches, including bottom-up, value-based methodologies based on price references and cost offsets, and a top-down analysis leveraging orphan drug analogues
Identified evidence gaps for standalone use and co-positioning; facilitated subsequent discussions within the client cross-functional team on payer uncertainty mitigation strategies through brainstorming workshops