Identifying health technology assessment evidence gaps and providing practical recommendations for successful market access 

The client is developing a first-in-class investigational antibody therapy to treat severe autoimmune disease. Following success of the pivotal trials, regulatory submissions are in progress in the US and EU, with HTA and reimbursement decisions planned to follow. 

The overall evidence requirements for HTA can be high. Identifying and developing a comprehensive evidence package that meets the critical needs of HTA is key to achieving market access and commercial success. 

• To identify and rank the key gaps between current and planned evidence generation and the requirements of UK HTA agencies (NICE and SMC) for pricing and reimbursement decision-making. 

• To recommend activities to address identified gaps and advise on optimal strategy for value demonstration. 

We conducted an initial review of key materials, including: clinical guidelines, previous HTA decisions, the client’s current evidence package and evidence generation plans, and key evidence for the potential comparator set. 

Based on the materials review, we produced a situation analysis to: 

• • • Establish the current standard of care and treatment pathway 

    • Identify the range of plausible positionings for the new product and the appropriate comparator set for HTA 

    • Align on the current and planned evidence package for HTA submission 

    • Document the key assumptions for submission, including expected results from evidence generation activities in progress

    • Provide a high-level overview of the key issues for HTA. 

Following alignment with the client on the situation analysis, we conducted a detailed HTA gap analysis, providing: 

• • • Comprehensive assessment of the challenges for a positive pricing, reimbursement, and HTA decision 

    • Practical recommendations on how to address these challenges 

    • Clear prioritization of challenges, recommended activities, and mitigation strategies.

• Situation analysis (interim project report) 

• Gap analysis and recommendations (final report) 

The HTA gap analysis, although focused on the UK, provided important insights for the client’s preparations for access across a large number of markets. The extent of analysis and level of scrutiny in UK HTA is very high and the gap analysis served as a comprehensive guide to issues common across HTA agencies that use cost-effectiveness analysis in decision-making. It also highlighted a number of critical areas relevant to HTA agencies that focus on assessment of clinical benefit alone. 

Our work facilitated alignment on realistic prospects for pricing and access, the priorities for evidence generation and optimal product positioning and value proposition. The final deliverable included clear recommendations on new evidence generation and cost-effectiveness model structure.