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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

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Biogen’s Aduhelm rollout goes from bad to worse

pharmaphorum

The company’s chief executive Michel Vounatsos said he was “obviously disappointed” with the result, which he maintained resulted mainly from a lack of clarity on reimbursement which has delayed patient access. Vounatsos also insisted that Aduhelm’s price tag has not been a factor discouraging treatment with the drug.

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STAT+: Pharmalittle: Some employers cut insurance coverage for weight loss drugs; U.S. urges ADHD pill makers to boost production

STAT

 The reimbursement cuts, along with restrictions that other employers are implementing, mark the financial downside of the drugs’ medical triumph. Besides the $26,500 annual price tag, treatment could cost U.S. The outlays are straining the finances of some plans, including those funded by employers. The

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PharmaShots Weekly Snapshots (January 30 - February 03, 2023)

PharmaShots

4D Molecular Therapeutics Receives the US FDA’s IND Clearance of 4D-150 for the Treatment of Diabetic Macular Edema Date: Feb 03, 2023 | Tags: 4D Molecular Therapeutics, 4D-150, Diabetic Macular Edema, Regulatory, US, FDA, IND AstraZeneca and Amgen Receive the US FDA’s Approval of Tezspire (tezepelumab) for the Treatment of Severe Asthma (..)

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Risk-sharing agreements are growing at a rate of 24%

Pharmaceutical Technology

This is not the first treatment to come with a high price tag. Partly in response to such drugs, risk-sharing agreements (RSAs) have increased in popularity and aim to mitigate the risks of including high-cost drugs on reimbursement lists and healthcare plans.

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Drug-resistant epilepsy drug Ontozry backed by NICE

pharmaphorum

. “The FAD is a significant milestone for cenobamate and takes us closer towards reimbursement in England and Wales,” commented Stuart Mulheron, general manager UK & Ireland, Angelini Pharma.

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2021 Highlights of Chronic Care Medicine

PharmD Live

Fee-for-service reimbursement falls as quality performance pay increases. A 3.75% cut in the 2022 Medicare conversion factor, which calculates reimbursement for procedures under fee-for-service, is mandated under a budget neutrality provision and comes after a pay bump from Congress that expires in 2022. See it here.

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