A large multinational pharmaceutical player is developing a new therapy for sleep disorders including narcolepsy. The overarching goal of the work was to understand the key drivers of value for the new treatment across major global markets, the evidence required to support these value drivers, and any adjustments required to support a targeted, reimbursable product profile. The overall workstream included multiple, related projects including economic modeling, evidence assessment, and early scientific advice.
Economic model and evidence gap assessment
- Development of economic model to assess potential economically justifiable price and drivers of value
- Assessment of missing data required to fully populate the economic model and the impact of these data on the model results
- Separately, assessed the clinical trial program and proposed RWE plans to identify gaps in the evidence base
- Collectively, derived a clear understanding of the drivers of value for the new narcolepsy treatment and mapped these drivers to the existing TPP; also identified additional evidence needs to fully justify these drivers of value
Early HTA advice
Managed HTA Scientific Advice process for NICE (England) and CADTH (Canada):
- Development of questions to ask HTAs, resulting briefing book and agreed company positions on each question
- Multiple workshops with client to fully align on questions & company position
- Mock advice meetings to ensure readiness for discussion and response to questions
- Meeting materials and facilitation of client participation in advice meeting
Insights gained from scientific advice provided external validation of key value drivers and evidence needs.
Based upon the outcomes from the economic model and evidence gap assessments we were able to identify key drivers of value and match these to the existing target product profile and based upon the outcomes of the HTA advice, we were able to identify the key requirements from an HTA perspective to accept the drivers of value. We developed guidance and recommendations for the client to prioritize gaps in evidence and value communication to ensure that their existing target product profile was adjusted to reflect a reimbursable target. Our recommendations were accepted moving into the further development stages.