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Paying For Gene Therapy: Biggest Cost Challenges Two To Three Years Away?

 
• By Cathy Kelly

Only about one-quarter of the 40 gene therapies projected to launch by the end of 2025 would have potential US patient populations of more than 50,000. But a handful of treatments could target millions of individuals.

Flurry Of Gene Therapies To Reach Market In Next Two Years • Source: Alamy

Upcoming gene therapies for diabetic neuropathy, osteoarthritis and wet age-related macular degeneration are on track for launch in the US within the next three years and have expected patient populations in the multi-millions, which could present a significant challenge for payers, including Medicare and state Medicaid programs.

To date, the individual impact of gene therapies on the market, even with prices that approach and exceed $1m, may seem relatively manageable. Most gene therapies on the market and due to reach it in the next two years target patient populations that are well below the 200,000 statutory threshold for orphan drugs, according to a

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Tougher Approval Standards May Follow Vinay Prasad’s Appointment To Lead US FDA’s CBER

 
• By Sarah Karlin-Smith

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
• By Kate Rawson

Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.

Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
• By Sue Sutter

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
• By Sue Sutter

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

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Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
• By Sue Sutter

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
• By Sue Sutter

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

Ryoncil Chronology: Three Review Cycles, Two CRLs, One Dispute Resolution Request

 
• By Sue Sutter

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.