First Opinion is STAT’s platform for interesting, illuminating, and maybe even provocative articles about the life sciences writ large, written by biotech insiders, health care workers, researchers, and others.
To encourage robust, good-faith discussion about issues raised in First Opinion essays, STAT publishes selected Letters to the Editor received in response to them. You can submit a Letter to the Editor here, or find the submission form at the end of any First Opinion essay.
“Curing rare childhood diseases will falter unless Congress steps up,” by Joe and Courtney Dion
My heart breaks for the Dion family who desperately deserve hope for a cure to the ultra-rare disease that afflicts their children, Peter and Maggie. Sadly, they are not alone: 95% of the 10,000 or so rare diseases noted by the medical community still lack an FDA-approved treatment.
I worry that the FDA is not currently optimized to review new advancements in drugs that could give hope to such small patient populations. That’s because its experts on trial design, regulations, genetics, pharmacology, and specific rare diseases are scattered throughout the agency. As a result, none of the existing divisions is adequately able to understand how complex diseases like limb-girdle muscular dystrophy 2C affect different patients, the nuances and unique challenges of clinical trials for rare diseases — especially among children — and how new drug candidates may meaningfully improve outcomes.
To reorient the FDA’s focus and expertise, it should establish a Center of Excellence for Rare Diseases (sometimes called an Intercenter Institute for Rare Diseases). Doing so would bring resources under one roof and one mission, ensuring that the FDA does not duplicate efforts or apply different standards to multiple review processes. This realignment would help the FDA streamline inefficiencies in the current process and incentivize the development of new drugs, whose developers will be more confident that their treatment candidates can be evaluated appropriately. The FDA’s Oncology Center of Excellence is already giving hope to millions of Americans with cancer, and there is no reason to think a similar organization for rare diseases wouldn’t lead to long-lasting positive impacts for patients. Plus, the idea of an Center of Excellence for Rare Diseases at FDA has widespread support among patient groups and bipartisan lawmakers.
As a patient advocate, I’ve met thousands of families like the Dions. And I want the same for all of them: a chance to live and live well. It’s time for the FDA to create a Center of Excellence for Rare Diseases.
— Terry Wilcox, co-founder and chief mission officer of Patients Rising
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I can’t agree more. We need to take action and impress upon Congress how important the voucher program is. My 11-year-old daughter Sophie also has a rare muscular dystrophy — limb-girdle muscular dystrophy R1 — and desperately awaits a treatment or cure before she loses her mobility.
— Jessica Fabre
“The J&J lawsuit should be a wakeup call to the PBM industry,” by Jake Frenz
The J&J lawsuit has the advantage of being novel, but it will be very difficult to prove negligence. For one thing, the claim of negligence uses cost plus drugs (CPD) for comparative pricing when CPD really only covers generics — and only a small fraction of those.
“In 2023, across our [CVS Caremark] entire member population of approximately 90 million Americans, three brand-name drugs (Humira, Ozempic, and Stelara) cost more than every single generic drug combined.” So wrote David Joyner, CEO of CVS Caremark.
PBMs are a necessary evil because they’re the only actor with negotiating leverage against drug manufacturers for both generic and branded drugs. Even CPD isn’t always the cheapest.
— Dan Munro
“Empathy should guide responses to reported vaccine injuries,” by Kizzmekia S. Corbett-Helaire
Thanks for the support. I had vaccine-induced encephalitis that has destroyed my career and my life and was almost fatal (by my own hand). How do I know it was the vaccine? Because I got it twice with each dose. I have been dismissed by the medical community, government, and others. Use my name and contact as you wish to help in efforts to finally push the government and public health care community to come to terms with the devastation to me and so many others. I am real and this problem is by no means rare.
— Patrick Slater, Austin Ear Clinic
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While this essay is a beginning to examining the failure of the medical community to investigate the harms resulting from the Covid-19 vaccine, there must be far more done to remedy the abysmal failure of the CDC and NIH to examine the Vaccine Adverse Event Reporting System (VAERS) data. Why are the CDC and NIH reluctant to conduct double-blind studies on their protocols and policy recommendations?
The public has lost faith in these institutions because they have abandoned science to pursue other objectives. In contrast, many of the European medical agencies have pursued investigations to learn from their mistakes during the Covid-19 pandemic but not the CDC and NIH. Why?
— Joseph Merritt