Analysis Shows How Often FDA OKs Drugs Despite Mixed Or Failed Results
The joint Harvard-Yale research found that of 210 new therapies approved from 2018 through 2021, 21 of the drugs didn't meet one or more of their goals, or end points. Those 21 drugs were approved to treat cancer, Alzheimer's disease, and other illnesses, USA Today reported.
USA Today:
1 In 10 New Drugs Don't Achieve Their Main Goals Despite FDA Approval, Study Finds
One in 10 new drugs were cleared by federal drug regulators in recent years based on studies that didn't achieve their main goals, a new study shows. The study by Harvard and Yale researchers found that of 210 new therapies approved by the Food and Drug Administration from 2018 through 2021, 21 drugs were based on studies that had one or more goals, or end points, that weren't achieved. Those 21 drugs were approved to treat cancer, Alzheimer's and other diseases. (Alltucker, 2/13)
More on cancer research —
Stat:
CAR-T Cancer Therapy Reduces Risk Of Multiple Myeloma Relapse
The CAR-T cancer therapy called Abecma reduced the risk of multiple myeloma relapse by half compared to standard treatment, according to results of a Phase 3 clinical trial published Friday. (Feuerstein, 2/10)
Stat:
Elf Bar Tried — And Failed — To Donate To A Major Cancer Group
A popular Chinese vape company says it is donating thousands of dollars to the American Cancer Society in an effort to stop youth vaping, but the cancer organization says it never agreed to the partnership, and it’s ordering the company to stop. (Florko, 2/14)
In other pharmaceutical and biotech news —
Reuters:
Martin Shkreli: I'm Not In Contempt Over Drug Industry Ban
Martin Shkreli on Friday urged a U.S. judge not to hold him in civil contempt for failing to provide federal and state regulators with information to determine whether he is violating a lifetime ban from working in the pharmaceutical industry. In a filing in Manhattan federal court, Shkreli said he has complied with the February 2022 ban "as extensively as possible and in good faith," and has provided the materials sought by the Federal Trade Commission (FTC) and seven states. (Stempel, 2/10)
Axios:
Hyped Weight Loss Drugs Raise Supply, Equity Concerns
Buzz from celebrities and social media influencers around the off-label use of diabetes drugs for weight loss is prompting a spike in prescriptions and concerns about cost and possible shortages. More than 5 million prescriptions were written for Novo Nordisk's Ozempic or Eli Lilly's Mounjaro for weight management in 2022, up from about 230,000 in 2019, per Komodo Health. (Reed, 2/13)
NPR:
Lasers, Robots And Tiny Electrodes Are Transforming Epilepsy Treatment
When Tom's epileptic seizures could no longer be controlled with drugs, he started considering surgery. Tom – who asked that we not use his last name because he worries that employers might be alarmed by his medical history – was hoping doctors could remove the faulty brain tissue that sometimes caused him to convulse and lose consciousness. (Hamilton, 2/14)
Stat:
Could A Swallowable Device Make Diagnosing GI Diseases Easier?
Diagnosing gastrointestinal disorders is an uncomfortable process. It might involve sticking a long tube with a camera attached down a patient’s throat, or inserting a small catheter through a patient’s nostril. A team of researchers from the Massachusetts Institute of Technology, the California Institute of Technology, and New York University, looking to explore more comfortable options, has designed an ingestible device that doctors can monitor as it moves through the GI tract. (Lawrence, 2/13)
Bloomberg:
Biotech Went Through IPO Boom. Now Industry Shakeout Is Underway
The biotech industry is in shake-out mode after the ranks of public drug developers swelled in recent years amid an IPO boom. A scrum for capital among the expanded pool of companies and the make-or-break nature of clinical trials drove a contraction in the number of public biotechs last year, with some scooped up by larger rivals while others shuttered. As the sector stabilizes after last year’s slump, a divergence in fortunes is taking center stage. (Bradham, 2/13)
Stat:
Two Entrepreneurs Try To Upend The Biotech Funding Model
Greg Bowman, a University of Pennsylvania biophysicist, had the kind of idea biotech venture capitalists salivate over. His lab had been working on how to drug “cryptic sites” on proteins that were previously thought unreachable by drug molecules. Machine learning, he believed, would make new types of medicine possible. The idea seemed ready to be spun out of his lab and into a company. (DeAngelis and Herper, 2/14)
Stat:
Scientists Grapple With Ethics Of Stem Cell Research
When biologist Alysson Muotri started tinkering with tiny balls of nerve cells in the lab more than a decade ago, his goal was simply to understand early brain development and neurological disease. He didn’t realize he was stumbling into an ethical minefield. His University of California, San Diego team found that when human stem cells were grown into so-called brain organoids in the lab, these tiny 3D structures produced regular waves of electrical activity that resembled what researchers see in a full-size human brain when they place electrodes along a person’s scalp. (Wosen, 2/13)
KHN:
Your Money Or Your Life: Patient On $50,000-A-Week Cancer Drug Fears Leaving Behind Huge Medical Debt
After several rounds of treatment for a rare eye cancer — weekly drug infusions that could cost nearly $50,000 each — Paul Davis learned Medicare had abruptly stopped paying the bills. That left Davis, a retired physician in Findlay, Ohio, contemplating a horrific choice: risk saddling his family with huge medical debt, if he had to pay those bills from the hospital out-of-pocket, or halt treatments that help keep him alive.
“Is it worth bankrupting my family for me to hang around for a couple of years?” Davis pondered. “I don’t want to make that choice.” (Schulte, 2/14)
