Engaging payers in the rare disease space to prepare the market environment for the entry of a gene therapy

We collaborated with a pharmaceutical company to develop a payer engagement strategy for a novel siRNA-based therapeutic intended for the treatment of a rare liver disease. The pricing and market access team engaged us to assess how the company could interact with payers and payer advisory stakeholders to advance the field prior to launch and to develop a comprehensive plan for their engagement. The asset’s clinical data is highly encouraging, and an extensive program is set to generate evidence to demonstrate its value in payer negotiations. Engagement with national, regional, and local payers will inform strategies to meet payer needs and position the asset for optimal pricing and reimbursement.

Market context and project objectives

The patient journey in the target indication is complex, with diagnosis and management limited to supportive measures. The lack of specific treatment and diagnostic guidelines contributes to knowledge gaps and limited disease awareness. However, the landscape is evolving, with patient-centric initiatives and professional associations launching new activities. The impact of different stakeholders on future payer decision-making is anticipated to be influenced by local features related to the disease management landscape, as well as market access and pricing and reimbursement country profiles. The project aimed to understand the payer's perspective on the space and formulate insights and tactics for local payers and stakeholder engagement.

Our approach and deliverables

Phase 1 focused on assessing the stakeholder landscape through primary research with various payer groups, including national insurances, P&R bodies, KOLs, and pharmacies. The module covered the mapping of the stakeholder landscape, analysis of the budget allocation process as well as perceptions of unmet needs and value expectations of future treatments and data requirements.

Phase 2 was dedicated to developing a base concept for payer engagement, outlining appropriate activities, tactics, and levels of engagement in pre-launch and post-launch settings. We designed and populated a playbook to assist country offices in planning and prioritizing stakeholders and potential initiatives to engage them. We articulate disease- or market-specific considerations influencing potential variation in engagement frameworks.

In Phase 3, we prepared and rolled out a playbook to assist countries with mapping and prioritization. Additionally, we formulate a plan of action moving forward, prepare templates and a list of guiding questions, and develop actionable practical recommendations to engage with payers and payer advising stakeholders