First Opinion is STAT’s platform for interesting, illuminating, and 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.
the story
“‘Institutional neutrality’ is the way to go for universities,” by Westyn Branch-Elliman and Shira Doron
the response
I read with interest the recent piece by my fellow academic physicians on the topic of ‘institutional neutrality.’ I disagree with the opinion of the authors and will use the words of Elie Wiesel to summarize why: “We must take sides. Neutrality helps the oppressor, never the victim.”
— Regina LaRocque, M.D., M.P.H., Division of Infectious Diseases, Massachusetts General Hospital, Harvard Medical School
the story
“Trump’s dangerous Covid-19 revisionism,” by Rick A. Bright
the response
Rick Bright is spot on, highlighting the many failures of leadership and objective decision making by Donald Trump and others high up in his administration who reported to him. Dr. Bright did not mention another important fact when talking about perhaps the only success of the Trump White House vis-a-vis Covid vaccine development. And that is that the rapid development of the mRNA vaccines was based on decades of basic science that prepared the way and work on a prototype coronavirus vaccine based on the Middle East respiratory syndrome (MERS) virus related to both SARS and Covid but with limited human-to-human transmission. This is why the National Institutes of Health Vaccine Research Center was able to begin planning to develop a Covid vaccine within hours of publication of the virus’s sequence on Jan. 10, 2020. They had the critical part of the virus mapped, synthesized, and inserted into the critical protective lipid envelope that Moderna, a strategic research partner with NIH for several years already, two weeks later. Because of this the critical Phase 1 safety and immunogenicity human trials were actually begun a month later. Astounding and even better than warp speed, because we had long made the investments, developed the science base, and had learned how to make a vaccine well in advance of the need that suddenly presented itself in December 2019 and early 2020.
— Gerald Keusch, Boston University School of Medicine
the story
“Traditional randomized trials don’t work for ultra-rare diseases like Barth syndrome,” by Emil D. Kakkis
the response
As we stand on the threshold of a revolution in therapeutics driven by AI and never-before-imagined scientific advancements, it will be critical to ensure that rare disease patients are not left behind. Of the roughly 10,000 rare diseases discovered so far, 95% of them have no FDA-approved treatment or cure, which means that more than 30 million Americans suffering from a rare disease — and their loved ones — are desperately clinging to the hope that there will be a breakthrough for them. With this goal in mind, rare disease advocates from around the country are gathering in Washington this month for a discussion that will help shape the FDA’s new Rare Disease Innovation Hub. In the past, patients have been disappointed by promising new treatments hitting a snag in the process as a result of confusing and contradictory feedback sometimes given by different parts of the FDA. I am optimistic that the hub will help drive the drug discovery, development, and approval processes forward — and commend the FDA for recognizing the desperate need for a stand-alone entity focusing on rare diseases. The FDA has signaled the Rare Disease Innovation Hub will be set up to help provide advice early in the development process, which is critical because clarity and standardized guidance from experts who understand small trial design and other niche technical and clinical areas will help drug companies anticipate future challenges and work to resolve them before applying.
However, many drugs designed for rare diseases are already working their way through the FDA’s review process without the benefit of such guidance. As Dr. Kakkis rightly asserts in his essay, “Barth syndrome should not be short-changed now by virtue of not having the benefit of the hub.” In the interest of fairness, the makers of all rare disease drug candidates should be given a chance to work with the hub and to appeal directly to experts there should they hit an unforeseen snag with their application. Moreover, there should be one decisionmaker at the hub who will have the final say about issues raised during the process. All of this will help prevent government red tape and associated delays and ensure that any potentially conflicting guidance provided by different parts of the FDA’s fragmented infrastructure can be ironed out.
— Terry Wilcox, Patients Rising