Short on time? Those are the highlights from our latest Bulletin, or scroll down for the full story.
Amanda Cole, Director
Investment flows to where returns are highest
This seems obvious, yet in healthcare we can feel uneasy talking this way. Still, it underscores a central ambition: to align high returns (for innovators) with high value (for patients), so R&D investment flows to the breakthroughs we most need.
Among our spotlight publications, we’re proud to share the Commercial Case for investing in Amyotrophic lateral sclerosis (ALS), developed in support of the Longitude Prize on ALS. This five-year, £7.5 million challenge aims to spur research into how AI can transform drug discovery, echoing the original 18th-century Longitude Prize, which famously catalysed a solution to determining a ship’s longitude at sea.
We show how a successful ALS treatment — the most common form of motor neurone disease — would generate major returns for healthcare systems and investors alike. Just as the first Longitude Prize revolutionised navigation, we believe AI can reshape drug discovery for ALS, a devastating condition with urgent unmet need.
The innovation ecosystem is complex, with the essential funding and investment coming from diverse sources along the development pathway. In a new Health Affairs Scholar publication, we map that ecosystem. As scrutiny of global drug pricing grows, we must keep examining how it shapes future R&D incentives and investments.
Why ALS innovation represents both societal and commercial value
The commercial case for investing in ALS
Amyotrophic lateral sclerosis (ALS) is a rare, rapidly progressive disease with limited treatment options and delayed diagnosis, creating a major unmet need. The economic burden is high, with millions lost in healthcare costs and quality-adjusted life years per patient, rising further when societal costs are included. Emerging ALS therapies offer the potential to transform patient outcomes and deliver commercial value, as advances in AI, gene therapy, and adaptive trials speed up development.
Do current policy models capture real-world drug investment decisions?
Understanding biopharmaceutical investment decision-making: how does Congressional Budget Office's model compare to investor insights?
The US Congressional Budget Office developed a model to assess how changes in pharmaceutical revenues might affect future innovation. Interviews with investors suggest that this model simplifies how investment decisions are made and may underestimate the impact of policies. The findings highlight the importance of aligning policy models more closely with real-world investment dynamics to improve drug development and innovation.
Drug shortages are on the rise – but what is their impact?
Drug shortages are an increasing concern worldwide, with implications for patients, healthcare systems, and budgets. We proposed a framework to estimate the cost of a shortage and illustrate its application through three case studies: Trastuzumab, Statins, and Saline. These examples highlight how shortages can lead to health losses, price increases, and system-wide disruption.
We're delighted that Adrian Towse was the recipient of the Avedis Donabedian Outcomes Research Lifetime Award at ISPOR Europe this week. In his honour, we've created an interactive HEOR-themed crossword featuring everyday health economics terms which you can play on our website.
