Advocates ask U.S. trade rep to allow countries to sidestep Trikafta patents

Cystic fibrosis patients around the globe are clamoring for access to Trikafta, a potent drug from Vertex Pharmaceuticals that can treat 90% of patients with the inherited respiratory disorder. But it's very expensive: The U.S. list price is nearly $322,000 — which is likely why only 12% of CF patients in nearly 100 countries are receiving the treatment.

Several advocacy groups are now asking U.S. Trade Representative Katherine Tai to allow the governments of Brazil, Ukraine, South Africa, and India to sidestep or revoke patents on Trikafta, should they ask. In a process called compulsory licensing, countries can allow a public agency or generic drug maker to copy a patented medicine without the consent of the brand name company that owns it. Vertex has been blocking generic competitors by pursuing patents in a number of countries, but the process is preventing many patients from receiving life-saving medications.

brackets

Forget basketball, it's time for STAT Madness!

Our annual, bracket-style biomedical research competition kicks off again this week! STAT Madness entrants include some of 2022's most compelling discoveries, and we beseech you, our lovely readers, to vote on the best of the best. The research ranges from xenotransplantation to precision Covid tests to robotic drug delivery capsules. Popular voting begins March 1.

There will be six single-elimination rounds, and the winner of the popular vote will be announced April 4. A handful of the top contenders will be asked to present their research at the 2023 STAT Breakthrough Summit, which will be held May 3-4 in San Francisco.

GOVERNMENT FUNDING

Where are the NIH pediatric research dollars going?

Although the NIH distributed $362 million to fund pediatric clinical trials over three recent years, many of the results were never published. Specifically, of the 354 trials studied in a recent JAMA analysis, there was only a 54% likelihood that results would be published four years after the grants were completed. This absence of trial data underscores how a lack of transparency is causing millions of dollars in research funding to be wasted.

Already, fewer research dollars are granted for clinical trials involving children. "Studies have estimated that as many as 50% of drugs used in children are used in an off-label fashion, largely because industry is less interested in investing in pediatric conditions given the smaller markets and profitability for pediatric products," a co-author of the analysis said. "So pediatric research depends primarily on federal funding and there is a critical need for this funding to help fill in gaps."

living forever

A decision due on Reata's ataxia drug

Friedrich's ataxia is a rare neurodegenerative disorder with little in the way of viable treatments. Reata Pharmaceuticals has developed a controversial drug called omaveloxolone that might alter the course of this deadly disease — but regulators may be torn. On one hand, the clinical data are limited and not particularly convincing. On the other, patients truly are desperate for anything that might slow the progressive damage to the brain. The agency is expected to announce its decision by Tuesday.

A single trial, completed in 2019, showed omaveloxolone improved neurological function compared to placebo. But regulators told Reata that the results were "not exceptionally persuasive." So it's a regulatory coin flip — and the company's stock could either jump dramatically on the news, or plummet. We'll see.

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