An ALS therapy is being tested. So is the FDA

A high-profile, experimental therapy for ALS is teeing up a crucial test of the FDA’s ability to withstand political pressure. The drug, from Amylyx Pharmaceuticals, has been the subject of an intense campaign by ALS patients and advocacy organizations, which assert that the FDA has stubbornly blocked access to therapies that might add a glimmer of hope to an otherwise deadly diagnosis. Now the FDA’s expert advisory committee is set to make a recommendation next week on the drug, for which initial results of a small Phase 2/3 trial show a modest benefit. Should the larger datasets show otherwise, the FDA may be in a bind as the agency’s expert advisory committee meets for the first time since the agency approved the controversial Alzheimer’s drug Aduhelm over the advice of its experts. STAT’s Nicholas Florko has more in STAT+.

Only half of clinical trial results for kids are reported

It’s been a long time since people had to make the case for testing drugs or other interventions in children, rather than just treating them as small adults. And the complexity of securing consent from parents is a well-understood obstacle. A new study in Pediatrics points out more challenges in pediatric clinical trials: completing them, reporting their results, and publishing them. Looking at more than 13,000 trials over more than 10 years, the researchers found that 11% were never finished, most often because they couldn’t recruit enough participants. Only half of completed trials reported or published their results within three years, a share that was higher for government-funded and academic trials than industry trials. The authors raise concerns about companies’ bias toward releasing only positive findings and urge all parties to do better at pushing pediatric medical research forward. Read more from STAT's Ed Silverman.

Polygenic embryo testing targets common diseases and prompts questions

Testing embryos conceived via IVF before implantation is already fraught with ethical questions, as in potentially choosing sex or other traits. The current process alerts prospective parents to genes known to cause such rare disorders as cystic fibrosis or Huntington’s disease. New research in Nature Medicine from Silicon Valley companies MyOme, Spring Fertility, and Natera promises to reveal risk for 12 common diseases, including cancer, heart disease, and type 2 diabetes. The researchers derived polygenic risk scores from 10 parents and 110 five-day-old embryos and then compared those predictions to predictions generated from 10 children’s tissue samples. The accuracy rate between predictions hit 99%, they report. There are many caveats: Genomic databases are overwhelmingly European, access to such tests will be uneven, and common diseases have social and environmental determinants that genetics can’t capture — and that society must address, an editorial says.

Closer look: The biggest PBMs gain leverage in drug price negotiations

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PBMs — short for pharmacy benefit managers, the behemoth intermediaries that negotiate drug prices, process claims, and create networks of pharmacies — sit at the center of a complicated web for how Americans pay for drugs, and their rising gross revenues reflect a number of broader trends: Manufacturers’ list prices are going up, new drugs have ever-higher price tags, and more people with insurance are filling more prescriptions. Those numbers also include pharmacies’ fees and the drug rebates that PBMs receive before they are paid out to insurers and employers. PBMs’ profit margins now hover around 4-5% of gross revenues, according to the filings, which equates to tens of billions of dollars in profit annually. STAT’s Bob Herman explores in STAT+ how the PBM industry has only grown despite its motto of lowering drug costs.

Adding aspirin to help prevent blood clots didn't help Covid patients in the ICU, study says

Building on hard-won knowledge from early in the pandemic, a new clinical trial looking at severely ill ICU patients adds to evidence on how to prevent blood clots. When the immune system responds to infection with inflammation, platelets go into overdrive, leading to clots in blood vessels and lungs — a complication of severe Covid first spotted in early 2020. Adding antiplatelet (aspirin) therapy to anticoagulation (heparin) therapy has been tried, but recent studies have found little benefit and some harm, in the form of bleeding. A new study in JAMA that randomly assigned critically ill patients already on an anticoagulant to also receive aspirin, another antiplatelet drug, or nothing extra found no improvement from the added antiplatelet drug. “The accumulated data should provide physicians with the rare confidence to do less rather than more,” a linked editorial notes.

GI infections dropped early in the pandemic. Could it be all the hand-washing?

Gastrointestinal infections in England fell by a little over half in the first seven months of the pandemic compared to the same months before 2020, a new study in BMJ Open says, prompting speculation about the role of public health measures in the striking change. Researchers tracked patients’ calls to health clinics and visits to doctors for vomiting and diarrhea, as well as lab tests for microbial illnesses, noting that pathogens mostly spread by person-to-person contact, such as norovirus and Shigella, had the biggest drop. They also queried Google Trends, where searches for “food poisoning,” “gastroenteritis,” and “sickness bug” dipped while “hand-washing” and “disinfection” climbed. Many reasons could explain this, including people not getting care during lockdown, but the researchers couldn’t rule out better hand hygiene, less social contact, social distancing, more surface cleaning, and closed restaurants and other businesses.

What to read around the web today

St. Jude fights donors’ families in court for share of estates. ProPublica
Plagiarism scandal puts renowned concussions doctor under scrutiny. New York Times
What makes a biotech a biotech? STAT+
Scientists watch a memory form in a living brain. Wired
Teva and Allergan reach $107 million deal with Rhode Island over opioid crisis. STAT+