First Opinion is STAT’s platform for attention-grabbing, illuminating, and provocative articles concerning the life sciences writ giant, written by biotech insiders, well being care staff, researchers, and others.
To encourage strong, good-faith dialogue about points raised in First Opinion essays, STAT publishes chosen Letters to the Editor acquired in response to them. You’ll be able to submit a Letter to the Editor right here, or discover the submission type on the finish of any First Opinion essay.
the story
“‘Institutional neutrality’ is the way in which to go for universities,” by Westyn Department-Elliman and Shira Doron
the response
I learn with curiosity the latest piece by my fellow educational physicians on the subject of ‘institutional neutrality.’ I disagree with the opinion of the authors and can use the phrases of Elie Wiesel to summarize why: “We should take sides. Neutrality helps the oppressor, by no means the sufferer.”
— Regina LaRocque, M.D., M.P.H., Division of Infectious Ailments, Massachusetts Basic Hospital, Harvard Medical College
the story
“Trump’s harmful Covid-19 revisionism,” by Rick A. Vivid
the response
Rick Vivid is spot on, highlighting the various failures of management and goal determination making by Donald Trump and others excessive up in his administration who reported to him. Dr. Vivid didn’t point out one other necessary reality when speaking about maybe the one success of the Trump White Home vis-a-vis Covid vaccine improvement. And that’s that the speedy improvement of the mRNA vaccines was based mostly on a long time of fundamental science that ready the way in which and work on a prototype coronavirus vaccine based mostly on the Center East respiratory syndrome (MERS) virus associated to each SARS and Covid however with restricted human-to-human transmission. For this reason the Nationwide Institutes of Well being Vaccine Analysis Heart was in a position to start planning to develop a Covid vaccine inside hours of publication of the virus’s sequence on Jan. 10, 2020. They’d the vital a part of the virus mapped, synthesized, and inserted into the vital protecting lipid envelope that Moderna, a strategic analysis companion with NIH for a number of years already, two weeks later. Due to this the vital Part 1 security and immunogenicity human trials had been really begun a month later. Astounding and even higher than warp pace, as a result of we had lengthy made the investments, developed the science base, and had realized the best way to make a vaccine nicely prematurely of the necessity that instantly introduced itself in December 2019 and early 2020.
—Gerald Keusch, Boston College College of Drugs
the story
“Conventional randomized trials don’t work for ultra-rare illnesses like Barth syndrome,” by Emil D. Kakkis
the response
As we stand on the edge of a revolution in therapeutics pushed by AI and never-before-imagined scientific developments, will probably be vital to make sure that uncommon illness sufferers aren’t left behind. Of the roughly 10,000 uncommon illnesses found thus far, 95% of them haven’t any FDA-approved remedy or treatment, which signifies that greater than 30 million Individuals affected by a uncommon illness — and their family members — are desperately clinging to the hope that there will likely be a breakthrough for them. With this objective in thoughts, uncommon illness advocates from across the nation are gathering in Washington this month for a dialogue that may assist form the FDA’s new Uncommon Illness Innovation Hub. Prior to now, sufferers have been upset by promising new therapies hitting a snag within the course of on account of complicated and contradictory suggestions generally given by totally different components of the FDA. I’m optimistic that the hub will assist drive the drug discovery, improvement, and approval processes ahead — and commend the FDA for recognizing the determined want for a stand-alone entity specializing in uncommon illnesses. The FDA has signaled the Uncommon Illness Innovation Hub will likely be set as much as assist present recommendation early within the improvement course of, which is vital as a result of readability and standardized steerage from specialists who perceive small trial design and different area of interest technical and scientific areas will assist drug corporations anticipate future challenges and work to resolve them earlier than making use of.
Nevertheless, many medication designed for uncommon illnesses are already working their manner by means of the FDA’s evaluation course of with out the good thing about such steerage. As Dr. Kakkis rightly asserts in his essay, “Barth syndrome shouldn’t be short-changed now by advantage of not having the good thing about the hub.” Within the curiosity of equity, the makers of all uncommon illness drug candidates must be given an opportunity to work with the hub and to enchantment on to specialists there ought to they hit an unexpected snag with their utility. Furthermore, there must be one decisionmaker on the hub who could have the ultimate say about points raised in the course of the course of. All of this may assist stop authorities pink tape and related delays and be certain that any probably conflicting steerage supplied by totally different components of the FDA’s fragmented infrastructure may be ironed out.
— Terry Wilcox, Sufferers Rising
