Zynteglo May Herald “New Dawn” of Many Seven-Figure Gene Therapies

August 05, 2022

An OptumRx drug pipeline report highlights Zynteglo, a curative gene therapy treatment for beta-thalassemia, and Skysona, a gene therapy for a cerebral adrenoleukodystrophy. FDA approval decision are imminent for both. Price tags of $2 million are expected.

The CMS Spend on Accelerated Approval Drugs. Is It Time To Tap the Brakes?

May 27, 2022

Two studies published this week documented the billions that Medicare and Medicaid spend on drugs that have been granted accelerated approval by the FDA based on surrogate end points. A study reported today in JAMA Health Forum found that only 6 of the 22 confirmatory trials used clinical outcomes.

KFF Drug Cost Panelists Discuss Rebates, Compulsory Licensing and COVID-19 Vaccine Development

May 23, 2022

Kirsten Axelsen, Richard Frank and Rachel Sachs agreed that the rapid development of the COVID-19 vaccines was a government-business success story. There was less to celebrate as the Kaiser Family Foundation panelists also unpacked the legal issues and economic consequences of drug rebates, international reference pricing, high deductible health coverage and compulsory licensing.

Patterns in New Formulation Approvals Suggest “Evergreening” by Drugmakers, Researchers Report

May 23, 2022

Findings in JAMA Health Forum show that new formulations were more likely for blockbuster drugs and drugs that received accelerated approval. Proxy measures of clinical usefulness and other elements of therapeutic value were not associated with new formulations.