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The Readout Damian Garde

Submit questions here by Friday, Nov. 1, for a Q&A with Dr. David Liu, a researcher at the Broad and MIT, and one of the inventors of a new type of gene editing using CRISPR. STAT Plus subscribers will be able to read the full Q&A on Nov. 12. 

The CF Foundation has $500 million to bridge a medical gap

Last week’s FDA approval for Vertex Pharmaceuticals means that roughly 90% of Americans with cystic fibrosis have an available treatment. Finding therapies for the remaining 10% is going to take some ingenuity, and one of the most lavishly funded nonprofits is taking it on.

As STAT’s Andrew Joseph reports, the Cystic Fibrosis Foundation has promised $500 million to fund research and development of treatments that expand the pool of patients getting a benefit. The organization, which made more than $3 billion by selling its royalty rights to Vertex’s drugs, will dole out the money over six years, earmarking the majority of funds for clinical-stage ideas.

That will mean supporting efforts to treat those who don’t benefit from Vertex’s treatments but also investing in potential cures that rely on gene therapy, genome editing, and other newfangled technologies. 

Read more.

The FDA is expanding a controversial tool

Remember when someone accidentally reported a clinical trial patient’s symptoms to a public FDA adverse event tracking system and sent Sarepta’s shares crashing this summer?

The FDA may soon require companies to submit adverse event reports to that system on purpose — though they still won’t be intended for the public’s eyes.

The agency announced yesterday that it would like to start accepting serious, drug-related side effect reports from clinical trials to its Adverse Event Reporting System, otherwise known as FAERS.

Companies already have to report serious side effects that happen during trials to the agency, but today those reports are sent as e-mail attachments or (gasp) on paper. Requiring companies to report side effect data to FAERS instead will allow the agency to better compare safety reports from before and after a drug’s approval, the agency says.

However, that requirement won’t take effect for years. The FDA’s guidance is still just a draft — and even after it's finalized, companies will have two years of voluntary reporting to adjust to the new system.

What’s going to happen to AMAG Pharmaceuticals?

Yesterday, AMAG Pharmaceuticals sat through a daylong FDA panel to determine whether Makena, a drug meant to prevent premature birth, should stay on the market. And the result, while undeniably negative, still leaves plenty of questions for the company.

The panelists resoundingly voted no when asked whether Makena had demonstrated a benefit over placebo, but the question over whether it should be pulled from shelves was more complicated. Nine of the 16 panelists recommended the FDA revoke the drug’s approval, while the rest recommended the Makena stay on the market with the condition that AMAG run another trial that confirms a benefit.

The agency’s final decision could be informed by factors well outside of AMAG’s control. As Cowen analyst Ken Cacciatore has pointed out, despite the controversy over Makena’s effects, there remains a strong demand from clinicians and patients. And if AMAG’s product is yanked off the market, the only available version of the drug would come from compounding pharmacies, which are spottily regulated.

Falsified drug data is still a big problem

Despite the drug lobby’s claims that overseas manufacturers largely comply with U.S. regulations, a new analysis suggests that problems with data integrity are widespread and more alarming than simple errors.

Writing in STAT, Katherine Eban and Sony Salzman detail how their review of thousands of FDA documents revealed persistent violations with manufacturers in China and India. In India, about 25% of the plants inspected committed some sort of data violation, while in China, that figure hovers just above 32%. For U.S. plants, the number is 15%.

To distinguish negligent practices from egregious ones, the researchers isolated key words in FDA’s inspection reports, such as falsification, destruction, and backdating. By that metric, 55% of inspections in India and 65% in China found deceptive practices.

Read more.

More reads

  • Taking stock of Alzheimer’s research: Experts offer takes on Biogen, the NIH, and the amyloid hypothesis. (STAT Plus)
  • Pfizer smashes expectations with tafamidis launch. (EP Vantage)
  • Amgen third-quarter revenue falls 3%, biosimilar sales rise. (Reuters)
  • The ‘cancer growing in cancer medicine’: pharma money paid to doctors. (STAT)

Thanks for reading! Until tomorrow,

Wednesday, October 30, 2019


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