Presentation (CTI)

OBJECTIVES: Amyotrophic lateral sclerosis (ALS) is a rare, rapidly progressive neurodegenerative disease with profound clinical, economic, and societal consequences. There are limited disease-modifying therapies available, resulting in high unmet need. This study quantified the socioeconomic burden of ALS and estimated the potential commercial and societal value of effective ALS therapies across seven countries (UK, US, Canada, France, Germany, Italy, and Australia), while examining implications for health policy and HTA decision-making.
METHODS: We applied three complementary valuation approaches. First, we monetised the annual socioeconomic burden of ALS by combining quality-adjusted life year (QALY) losses and direct healthcare costs. Second, we estimated commercial value using proxy pricing based on tofersen, scaled to the full ALS population over a 10-year horizon. Third, we adapted a published Markov model to evaluate hypothetical treatment scenarios with varying effectiveness. Estimates were generated from healthcare system and payer perspectives, with qualitative assessment of wider societal costs and spillover effects.
RESULTS: ALS results in a loss of approximately 13 QALYs per person and imposes substantial costs, with annual healthcare spending of $178 million in the UK and $2.5 billion in the US, rising threefold when societal costs are included. A successful ALS treatment could generate $26-$40 billion in cumulative commercial value in the US and $1.7 billion in the UK over 10 years; complete disease eradication could deliver up to $143 billion across countries studied. Conventional HTA modelling showed that only highly effective therapies were consistently cost-effective, revealing structural limitations in current HTA approaches for rare, high-burden diseases.
CONCLUSIONS: ALS represents a major opportunity for health impact and economic value. Yet, current HTA frameworks systematically undervalue innovation in rare, severe, progressive diseases with high unmet need and high background care costs. Policy reforms incorporating broader value elements are essential to incentivise investment and accelerate therapeutic development in ALS.