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Zynteglo Is Cost Effective At $2.1M, ICER Proposes; Validation For Bluebird’s Pivot To US?

 
• By Cathy Kelly

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.

Money
ICER says the Zynteglo price could be cost effective • Source: Alamy

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...

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BIO Notebook: MFN Pricing, Next-Gen Obesity R&D, FDA’s Rare Disease Hub & Reaction To Review Program

 
• By Sarah Karlin-Smith, Mandy Jackson, and Alaric DeArment

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.

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• By Andy Smith

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• By Joseph Haas

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Europe’s Drugmakers Lament EU Pharma Package

 
• By Kevin Grogan

EFPIA says reforms make the continent less attractive for innovative firms.

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BIO China Deal Perspectives: A Dramatic Impact On Industry’s R&D Pipelines

 
• By Mandy Jackson

Biopharma executives and investors at the BIO convention noted the competition emerging from China with high-quality assets that could make strong additions to their portfolios.

Altimmune’s Phase IIb MASH Data Send Share Price Plunging

 
• By Joseph Haas

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Removal Of REMS For CAR-Ts Could Greatly Expand Their Reach

 
• By Alaric DeArment

The FDA eliminated REMS requirements for approved CAR-T cell therapies that have limited their availability, a move that could enable increased use in the community setting.