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How the FDA monitors side effects, how CMS may help pay for sickle cell therapy, a new pain drug's promise

January 31, 2024
Reporter, Morning Rounds Writer
Good morning. Two treatments in the news today have complicated stories: gene therapy for sickle cell disease and a new, non-opioid drug for pain. 

health

Asthma inhaler case prompts questions on how the FDA delivers warnings on drugs

Did you know the asthma drug Singulair comes with a warning about suicidal thoughts as a potental side effect? You may have learned that, as I did, from a recent New York Times story suggesting such a warning falls short, leaving clinicians and patients in the dark and perhaps in danger. STAT's Theresa Gaffney explores what systems are in place at the FDA to monitor adverse reactions after a drug is approved and act in response to them.

It's a balance between safety and access to medicines, Ameet Sarpatwari, assistant director of the Program on Regulation, Therapeutics, and Law at Brigham and Women's Hospital, told her. "I think there are definitely situations for many drugs where you can say, no, we're not really at the right balance, and the FDA should be taking a more aggressive stance in promoting patient safety." Read more about what systems are in place and how they work. 


gene therapy

Pilot would help pay for costly sickle cell treatment

New gene therapies for sickle cell disease come with multimillion-dollar prices that could bankrupt financially strapped state Medicaid programs, despite predictions that the one-time expense would be less than lifetime care costs. Sickle cell disease affects an estimated 100,000 Americans, an estimated 60% of whom rely on Medicaid for health care coverage. Yesterday the federal government revealed a long-awaited "access model" designed to blunt the cost that state Medicaid programs would pay for these curative treatments. 

Here's how it will work: CMS will seek outcomes-based agreements, negotiating pricing and rebates with the drug manufacturers — Vertex Pharmaceuticals and Bluebird Bio — over the next several months on behalf of states that choose to join the program, starting in January 2025. Bluebird has already created a specific outcomes-based agreement for its therapy, which costs $3.1 million. Vertex, which priced its therapy at $2.2 million, declined to discuss its plans. STAT's Ed Silverman explains.


public health

Syphilis cases are rising to rates not seen since 1950Screen Shot 2024-01-30 at 1.19.01 PM

CDC

Syphilis cases keep going up in the U.S., the CDC tells us in a report yesterday that shows an increase of 9% in 2022. But for reasons the agency's scientists can't explain, new gonorrhea cases also fell by 9% for the first time in 10 years — too soon to know if it's a trend. Syphilis is less common than gonorrhea or chlamydia, but it's considered more dangerous. 

Total syphilis cases surpassed 207,000 in 2022, the highest since 1950. It disproportionately affects gay and bisexual men, but numbers are growing among heterosexual men and women, and increasingly affecting newborns. Also yesterday, the Department of Health and Human Services said it would enlist the National Syphilis and Congenital Syphilis Syndemic Federal Task Force to slow the spread, with a focus on those most significantly impacted. The Associated Press has more here.



closer look

'FDA has to be flexible': Janet Woodcock reflects on more than 30 years at the agencyGettyImages-1351289804

Chip Somodevilla/Getty Images

It's no exaggeration to say that when Janet Woodcock leaves the FDA, it's the end of an era. She joined the FDA's biologics division in 1986 but soon moved to the drug side, where she oversaw hundreds of reviews, watched new therapies evolve, and weathered clashes with Congress and patient advocates. She took a look back with STAT's Sarah Owermohle.

What's different now for the agency?

More and more people are questioning authority and even authoritative scientific opinions, and the FDA is a part of that. It's all the regulators, it's all the authorities, it's the people in universities and everything. FDA has to be flexible.

What do you mean by 'flexible?'

It needs to be an agency that can react appropriately to societal trends, to recognize those and to be able to respond to them within its parameters, its authorities, its role. 

How would you answer critics of accelerated approvals?

Some people might say there were some that didn't work out. Well, that's how accelerated approval is set up. Is that wrong? You hold back all of these until you have definitive evidence? Based on the results, you would have a lot of people who wouldn't be alive. Was that worth it to have all that certainty?

Read the full interview.


pharma

New Vertex pain drug stirs optimism and questions

Early yesterday morning, Vertex Pharmaceuticals shared late-stage trial results for a pain medication showing it was safe and effective while offering modest pain relief. But there was a catch, as STAT's Jonathan Wosen reported: The small molecule drug VX-548 didn't do better than a combination of acetaminophen and the opioid hydrocodone, a key secondary endpoint, for patients who took it after a tummy tuck or bunion surgery. And in the bunionectomy study, the opioid comparator beat VX-548 on pain relief.

The results set off a fresh debate about whether the drug would succeed, should it win approval. Pain experts and market observers pointed out that any new non-opioid pain medication would reverse a long history of pharma failure in this field, but wondered if payers might be reluctant to cover the drug for treating moderate to severe acute pain — where the company plans to seek first approval — without clear and consistent evidence the drug works as well as opioids. Jonathan has more.


reproductive health

Babies are arriving sooner, with a bump at 37 weeks
Screen Shot 2024-01-30 at 2.17.36 PM

National Center for Health Statistics

Looking back to 2014 and allowing for pandemic fluctuations from 2020 through 2022, a new National Center for Health Statistics report tracks a shift toward singleton births after shorter gestational ages. While preterm (before 37 weeks) and early term (37 to 38 weeks) birth rates climbed by 12% and 20%, respectively, full-term (40 weeks) and late and post-term birth rates fell, by 6% and 28%, respectively. These trends were similar across maternal age, race, and Hispanic origin. The largest change for a single-week gestation at term was an increase of 42% for births at 37 weeks.

Some history: Preterm birth rates in the U.S. rose by more than one-third from 1981 to 2006, sparking concern and focusing attention on the risks for babies born between 34-36 weeks of gestation, or late preterm. These late preterm births accounted for 70% of all preterm births over that time period, today's report notes.


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What we're reading

  • Philips to stop selling sleep apnea machines in U.S. under tentative agreement with FDA, DOJ, Associated Press
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  • Cour Pharmaceuticals, a biotech taking aim at autoimmune diseases, raises $105 million, STAT
  • What to know about Elon Musk's Neuralink, which put an implant into a human brain, NPR

  • Opinion: What the pope is missing about commercial surrogacy, STAT

Thanks for reading! More tomorrow,


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