Daily Politics from the New Statesman Rare diseases: from lived experience to lasting treatments | Sponsored
Mar 1, 2026
Terry Pirovoulakis, a parent who led fundraising to develop a gene therapy for his son Michael with SPG50. Sam Barrell, CEO of LifeArc, who funds and shapes rare-disease translation. They discuss long diagnostic journeys, gaps in data and regulation, parent-led innovation, and calls for joined-up policy and a national rare-disease champion.
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Rare Disease Burden Matches Cancer But Lacks Treatments
- Rare diseases affect about 3.5 million people in the UK, roughly the same number as cancer patients, but 95% lack an approved treatment.
- Around 80% of rare diseases are genetic and 70% present in childhood, highlighting missed opportunities for early intervention.
Systemic Barriers Slow Rare Disease Progress
- Major systemic gaps include slow diagnosis, fragmented data, limited investment, and complex regulatory pathways that are ill-suited to small patient populations.
- These barriers make trial design, evidence generation, and reimbursement especially hard for ultra-rare conditions.
Scale Newborn Genomic Screening Now
- Expand genomic sequencing early, ideally at newborn screening, because about 80% of rare diseases are genetic and early detection enables earlier interventions.
- Sam and Terry pointed to pilot newborn genomic programs and Spain's one-draw sequencing as models to emulate.