First Opinion is STAT’s platform for attention-grabbing, illuminating, and perhaps even provocative articles in regards to the life sciences writ giant, written by biotech insiders, well being care employees, researchers, and others.
To encourage sturdy, good-faith dialogue about points raised in First Opinion essays, STAT publishes chosen Letters to the Editor obtained in response to them. You’ll be able to submit a Letter to the Editor right here, or discover the submission kind on the finish of any First Opinion essay.
“Curing uncommon childhood ailments will falter until Congress steps up,” by Joe and Courtney Dion
My coronary heart breaks for the Dion household who desperately deserve hope for a treatment to the ultra-rare illness that afflicts their youngsters, Peter and Maggie. Sadly, they aren’t alone: 95% of the ten,000 or so uncommon ailments famous by the medical group nonetheless lack an FDA-approved remedy.
I fear that the FDA is just not presently optimized to assessment new developments in medication that would give hope to such small affected person populations. That’s as a result of its specialists on trial design, laws, genetics, pharmacology, and particular uncommon ailments are scattered all through the company. Because of this, not one of the present divisions is sufficiently capable of perceive how complicated ailments like limb-girdle muscular dystrophy 2C have an effect on completely different sufferers, the nuances and distinctive challenges of scientific trials for uncommon ailments — particularly amongst youngsters — and the way new drug candidates could meaningfully enhance outcomes.
To reorient the FDA’s focus and experience, it ought to set up a Heart of Excellence for Uncommon Illnesses (generally known as an Intercenter Institute for Uncommon Illnesses). Doing so would deliver assets underneath one roof and one mission, making certain that the FDA doesn’t duplicate efforts or apply completely different requirements to a number of assessment processes. This realignment would assist the FDA streamline inefficiencies within the present course of and incentivize the event of latest medication, whose builders will likely be extra assured that their remedy candidates could be evaluated appropriately. The FDA’s Oncology Heart of Excellence is already giving hope to thousands and thousands of Individuals with most cancers, and there’s no motive to assume an identical group for uncommon ailments wouldn’t result in long-lasting constructive impacts for sufferers. Plus, the concept of an Heart of Excellence for Uncommon Illnesses at FDA has widespread help amongst affected person teams and bipartisan lawmakers.
As a affected person advocate, I’ve met 1000’s of households just like the Dions. And I would like the identical for all of them: an opportunity to reside and reside nicely. It’s time for the FDA to create a Heart of Excellence for Uncommon Illnesses.
— Terry Wilcox, co-founder and chief mission officer of Sufferers Rising
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I can’t agree extra. We have to take motion and impress upon Congress how essential the voucher program is. My 11-year-old daughter Sophie additionally has a uncommon muscular dystrophy — limb-girdle muscular dystrophy R1 — and desperately awaits a remedy or treatment earlier than she loses her mobility.
— Jessica Fabre
“The J&J lawsuit needs to be a wakeup name to the PBM trade,” by Jake Frenz
The J&J lawsuit has the benefit of being novel, however will probably be very tough to show negligence. For one factor, the declare of negligence makes use of value plus medication (CPD) for comparative pricing when CPD actually solely covers generics — and solely a small fraction of these.
“In 2023, throughout our [CVS Caremark] whole member inhabitants of roughly 90 million Individuals, three brand-name medication (Humira, Ozempic, and Stelara) value greater than each single generic drug mixed.” So wrote David Joyner, CEO of CVS Caremark.
PBMs are a crucial evil as a result of they’re the one actor with negotiating leverage in opposition to drug producers for each generic and branded medication. Even CPD isn’t all the time the most affordable.
— Dan Munro
“Empathy ought to information responses to reported vaccine accidents,” by Kizzmekia S. Corbett-Helaire
Thanks for the help. I had vaccine-induced encephalitis that has destroyed my profession and my life and was nearly deadly (by my very own hand). How do I do know it was the vaccine? As a result of I bought it twice with every dose. I’ve been dismissed by the medical group, authorities, and others. Use my title and call as you want to assist in efforts to lastly push the federal government and public well being care group to return to phrases with the devastation to me and so many others. I’m actual and this downside is on no account uncommon.
— Patrick Slater, Austin Ear Clinic
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Whereas this essay is a starting to analyzing the failure of the medical group to analyze the harms ensuing from the Covid-19 vaccine, there should be way more performed to treatment the abysmal failure of the CDC and NIH to look at the Vaccine Opposed Occasion Reporting System (VAERS) knowledge. Why are the CDC and NIH reluctant to conduct double-blind research on their protocols and coverage suggestions?
The general public has misplaced religion in these establishments as a result of they’ve deserted science to pursue different goals. In distinction, lots of the European medical companies have pursued investigations to study from their errors throughout the Covid-19 pandemic however not the CDC and NIH. Why?
— Joseph Merritt