Draft evidence report from the Institute for Clinical and Economic Review estimates the potentially curative rare disease treatment could offset about $1.4m in health system costs over a lifetime.
bluebird bio’s gene therapy for beta thalassemia, Zynteglo (betibeglogene autotemcel), would be cost effective at a price of $2.1m, especially if payments are spread over five years and based on outcomes, the Institute for Clinical and Economic Review (ICER) tentatively concludes in a draft evidence report released 13 April.
“Despite remaining uncertainties, the evidence suggests that beti-cel provides net health benefits to patient with [transfusion dependent thalassemia] and given...
Highlights from Day 3 of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.
Successive annual reductions in European drug prices following the pandemic are affecting the global sales of generic pharmaceutical companies. Additionally, the imposition of US import tariffs may result in generic drug supply becoming unprofitable.
UroGen’s Zusduri was approved despite an advisory committee vote recommending against it in May. It is the first approved drug for a type of recurring bladder cancer.
EFPIA says reforms make the continent less attractive for innovative firms.
Nanoscope’s experimental gene therapy, MCO-010, is designed to target the broader retinitis pigmentosa population, regardless of the underlying gene mutation, with a completed US regulatory submission expected in early 2026.
Pharma company financial guidance emerged largely unscathed from a first quarter punctured by a weakening US dollar and threats of import tariffs on drugs. With other pressures either already evident or on the horizon, guidance revisions may be unavoidable.
Aside from several sizable global out-licensing deals, South Korean bioventures saw a cancellation of other alliances, updated their pipelines with losses and gains and reported clinical trial progress in the second quarter, while domestic IPO sentiment also improved.
