Undertaking a health economic assessment to understand the reimbursed price potential for an acute respiratory distress syndrome therapy in the UK.
Cell and gene therapies are renowned for their high manufacturing costs which dictate a high target price in order to be commercially viable. To secure reimbursement at a commercially viable price, it is important to ensure that the therapy’s incremental cost to the healthcare system (above current treatment options) is compensated by the incremental benefits it delivers. Where the incremental benefit in clinical terms is impaired, the manufacturing strategy should focus on reducing manufacturing costs so that commercially viable profit margins are not eroded.
Acute respiratory distress syndrome (ARDS) is a serious condition characterised by widespread inflammation in the lungs and represents a major cause of morbidity and mortality in the critical care setting.
ARDS affects approximately:
33k patients in the UK
400k-500k patients in Europe,
the United States and Japan
There are limited interventions and no effective drug treatments for ARDS, making it an area of high unmet clinical need. In pre-clinical studies MultiStem® cell therapy (by Athersys, Inc.) demonstrated the capacity to reduce inflammation, support tissue regeneration and promote homeostasis.
In 2015, Athersys Limited (an affiliate of Athersys, Inc.) and the Cell and Gene Therapy Catapult, were awarded a grant from Innovate UK to support a Phase 2a clinical study evaluating the administration of MultiStem cell therapy to ARDS patients. In parallel to running this trial and as part of this funding the Health Economics and Market Access team at the Cell and Gene Therapy Catapult undertook a health economic assessment to understand MultiStem’s reimbursed price potential in the UK. They reviewed factors relevant for NHS adoption including comparative clinical effectiveness of the novel therapy vs. the standard of care, cost-utility and budget impact considerations.
We identified the key drivers of product value, so that evidence generation activities were steered accordingly.
- We characterised the interrelationship between reimbursed price potential and the
- Target Product Profile (TPP) parameters with an emphasis on:
o Overall survival (OS)
o Length of stay (LOS) in intensive care unit (ICU)
o LOS in hospital post-ICU discharge
- By understanding the interrelationship between product value, reimbursed price potential and profit margins, we identified efficacy and manufacturing cost thresholds for commercial viability which were then used to inform clinical development and manufacturing strategies accordingly.
- By quantifying budget impact and assessing its implications, we helped refine assumptions on therapeutic positioning and forecasts of anticipated volume of uptake.
The Cell and Gene Therapy Health Economics and Market Access team provides health economics, pricing, reimbursement and market access expertise to accelerate and maximize cell and gene therapy uptake across global healthcare markets. We guide new product development and commercialisation by informing data generation activities, optimizing value proposition, developing and implementing global pricing, reimbursement and value communication strategies.