UK HTA Submission for a Rare Lysosomal Storage Condition

Aim

Our health economics, HTA and Literature Reviews and Synthesis teams collaborated on this project with the aim to deliver a comprehensive HTA package, including development of both NICE and SMC submission dossiers, as well as conducting strategy workshops, systematic literature reviews, model support and clinical validation, followed by expert post-submission support and a mock evaluation committee meeting to maximise the likelihood of positive recommendations

Approach

Patient-centred value narrative

In light of the client’s strong relationship with patient groups, we conducted a workshop to support the client with developing an action plan to ensure impactful patient involvement in the HTA process. This involved developing a step-by-step roadmap detailing how patient involvement is invited in the NICE process. By prioritising patient involvement, the submission was able to capture key aspects of the unmet needs experienced by patients and how the product was able to address these.

Strategic input

We led a strategy workshop ahead of dossier development to align on key challenges and opportunities, before setting up strategic checkpoints at key points in the submission process to ensure the narrative aligned with latest data availability and internal discussions. Strategic input was provided throughout the project, including recommendations for evidence generation opportunities.

Maximising opportunities in the STA route

We developed a robust narrative and strategic solutions to mitigate the uncertainty associated with the rarity of the disease, despite NICE evaluation via the STA route.

Impact

• Unprecedented and first-ever time that draft NICE technology appraisal guidance were published before the MHRA marketing authorisation was granted
• Fastest delivery of positive NICE technology appraisal guidance, with only one evaluation committee meeting