Rare disease advocates also argue that the Medicare’s government drug price negotiation orphan drug exemption – which only applies when a drug has one orphan designation – will harm other government efforts aimed at more efficient drug development.
The limited orphan drug exemption in the Inflation Reduction Act’s Medicare price negotiation program could shift more drug use, particularly for rare diseases, into the off-label space and make it more challenging to ensure insurance reimbursement for those products, rare disease advocates worry.
The US Centers for Medicare and Medicaid Services has said that the IRA allows only orphan drugs with one rare...
Chiesi’s orphan drug Raxone has secured an English funding recommendation for the treatment of Leber's hereditary optic neuropathy, a decade after the orphan drug was approved for marketing. Chiesi told the Pink Sheet about its “long, and often challenging” road to reimbursement.
Biogen has U-turned on its original decision not to file Qalsody for marketing approval in the UK for treating certain patients with amyotrophic lateral sclerosis. The move appears to have been triggered by a change in the reimbursement pathway agreed for the product.
Just a week and a half into his tenure at the agency, the new CDER Director George Tidmarsh was also tapped to serve as acting head of CBER. Prasad’s swift ouster at CBER may point to a softening in cell and gene therapy regulation but could also portend even more scrutiny on vaccines.
The FDA recommended removal of a voluntary hold on shipments of the gene therapy to younger patients after finding that the death of an 8-year-old boy in Brazil was unrelated to Elevidys. Discussions on safety of DMD treatment for non-ambulatory patients is ongoing.
