Case Study: Global Pricing & Market Access Strategy for a Novel Enzyme Replacement Therapy in a Rare Genetic Disorder
Project Objective
A global pharmaceutical company had a Phase II/III enzyme replacement therapy in development for a rare genetic disorder that targeted symptoms not addressed by current standard of care. The new therapy would be used in combination with the standard of care – a product that they also owned
Given inconclusive trial evidence, there was a needed to align on specific challenges and possible innovative strategies in each market
The objective was to develop a robust global pricing and market access strategy plan, including innovative strategies to address data package challenges and portfolio considerations across markets to facilitate decision making
Windrose Approach
We facilitated and moderated discussions with market access affiliate teams for each in-scope market to understand specific access challenges and opportunities
Summaries of market-specific risks and opportunities were provided to facilitate internal alignment on the global pricing and access strategy
We conducted a cross-market hypothesis assessment of payer evidence requirements and expected Health Technology Assessment (HTA) outcome, impact of orphan disease status, innovative access pathways, and potential value-based agreements
In addition, we conducted an evidence gap assessment and determined stakeholder engagement and affiliate empowerment strategies to support the global P&MA strategy
Impact
We developed a comprehensive global P&MA strategy plan to support product launch planning, including portfolio considerations, risk-benefit assessment to facilitate prioritization, and alternative solutions such as patient access schemes and value-based agreements
The P&MA strategy included a robust assessment of available evidence, gaps, and proposed solutions for key concerns as well as a stakeholder engagement strategy at the global and affiliate levels to further optimize access
The client was prepared for market entry, mitigating evidence challenges and optimizing portfolio considerations and value, and the final strategy document was used as a foundation and guide for the development of tailored market-specific access strategies and future affiliate support activities
