The Value Of Gene Therapy In Spinal Muscular Atrophy

Situation:

• In the devastating childhood disease, spinal muscular atrophy (SMA), current treatments offered modest symptomatic benefit in a minority of sufferers
• Although the client was developing a potentially curative gene therapy, they needed to translate the clinical benefit into economic terms to engage payers and health technology organizations

By working with the client to develop a cost-effectiveness and budget impact model for a gene therapy in SMA that overcame unique challenges of modeling an early childhood neuromuscular genetic disorder they were able to change the prevailing mindset on how SMA progression is characterized.