Pivotal Clinical Program Design Addressing the Challenging Market Access Needs for a Rare Disease Therapy

A top 10 pharma company was developing a potentially disease-modifying therapy for a rare disease with high unmet needs and no established standard of care. It needed an efficient but robust pivotal program that could enable first-to-market leadership. Key challenges included low disease awareness, disease heterogeneity, no established endpoints, and more. The pivotal trial design had to balance payer needs with clinical and regulatory feasibility, physician acceptance, and patient/caregiver needs.

Our team began by identifying clinical program options with the client’s core team. We collected input on program options from key client functions (commercial, medical, clinical, access, and regulatory) at the global and country levels. We also conducted primary research to gather input on prioritized clinical program options from external stakeholders (payers, KOLs, regulators, and patient advocacy groups). Next, we Identified and assessed trade-offs, highlighting the impact of different program options on reimbursement, access, pricing, and commercial outcomes. In addition, we identified evidence gaps and initiatives (e.g., evidence generation, RWE, managed entry) to address gaps and achieve better outcomes. To complete the project, we finalized the pivotal program design by bringing together the client’s cross-functional team and ensuring alignment on trade-offs and key decisions.

The client received a pivotal program design that was internally aligned and externally validated. It was robust and included all key components, including the core program design (e.g., study population, comparator, endpoints), target product profile, plans for evidence generation, a risk overview, and risk mitigation strategies for each function.