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Is the FDA swapping ‘right to try’ with ‘let them die’?
The U.S. Food and Drug Administration recently dealt a crushing blow to families affected by deadly childhood diseases by denying promising treatments. All caring Americans should greet this decision with disgust. Not only is the decision disgraceful, but it’s also politically hypocritical.Bureaucrats should not let political agendas or indecision get in the way of potentially lifesaving decisions when parents of terminally ill children are more than willing to take the risk.Earlier this month, Democrats claimed that forcing able-bodied people to work and shortening enrollment periods for the “Un-Affordable” Care Act would kill people — a prime example of “Chicken Little” hyperbole without facts.Politically driven agendasWhat is factual, however, is that one of their own could actually be responsible for real deaths. The FDA, under the leadership of Dr. Vinay Prasad — a Bernie Sanders supporter — just denied two lifesaving treatments for children with rare diseases, despite these treatments having passed through the Trump administration's approval process.Dr. Prasad, a self-identified "lifelong progressive Democrat," is the FDA’s chief medical and scientific officer and the director of the Center for Biologics Evaluation and Research. With an extensive research background, particularly in oncology, he is clearly qualified for his position. He has even rejected efforts from pharmaceutical companies to push COVID-19 vaccines for anyone over the age of 12, citing sound "patient freedom" science.But he needs to go — and not just because socialists shouldn’t be in the Trump administration (or any U.S. government position). His ideology denies kids who are on the verge of dying the opportunity to live.Children don’t have time to waitEarlier this month, Prasad rejected two potential treatments for devastating childhood diseases: Duchenne muscular dystrophy and Sanfilippo syndrome.The life expectancy for a Duchenne patient is 22 years. In the process, it destroys a child’s muscles and causes neurological deficiencies.Sanfilippo syndrome, often called “childhood Alzheimer’s," is fatal. Few children live beyond their early teens, accompanied with cognitive, functional, and muscle decline.Take Sadie, for example, a little girl who is currently battling Sanfilippo syndrome. Her website, "Saving Sadie Rae," tells her story. She is an adorable, perky little fairy princess, adored by her family (and her Instagram fans) and is in the process of dying from Sanfilippo.Her parents are loving, dedicated caregivers, and her mother is on a mission to encourage the FDA to reverse its decision that, to parents like Sadie’s, is a blinking red light that says, “We don’t care about your kid.”Although the FDA has delayed its final decision until at least next year, Sadie’s parents, along with others, are painfully aware: Their kids don’t have time to wait.Duchenne and Sanfilippo parents are just regular people. They aren’t doctors or lobbyists. They're ordinary people facing an unimaginable situation, and they’re desperate for hope. They want the FDA to move forward with promising treatments.Bureaucratic backlog killsIn a recent conversation I had with former FDA Associate Commissioner Peter Pitts, who works closely with the Duchenne muscular dystrophy community, he expressed frustration with the agency.In recent years, the FDA has thankfully begun to listen more carefully to the parents of children with orphan diseases, meaning that they are rare but devastating. This does not throw regulatory science out the window, but gives a voice to parents willing to accept higher risks for what the FDA might view as tertiary benefits of new therapeutics — because the alternative and end point is early death.Pitts continued:The question of "what is the risk tolerance" of this community has been made abundantly clear by the community itself — parents are willing to accept higher risks to potentially provide less suffering and longer life for their children. That begs the question whether Dr. Prasad’s well-known dislike of the pharmaceutical industry — often in lockstep with Democrat talking points — has trumped the wishes of the disease community — and with callous disregard for the quality of life for children suffering with Duchenne or Sanfilippo syndrome. That’s almost too awful to comprehend.Although the developer of the Sanfilippo treatment claims it has “robust” confirmation of efficacy, FDA bureaucrats fussed about its manufacturing procedures, which the company says are unrelated to the quality of the gene therapy.Is this genuine concern or just an excuse?RELATED: HHS surmounts obstacles set by Democrat-appointed judges, gives thousands of bureaucrats the boot Photo by Trigga via iStock/Getty ImagesSimilarly, when rejecting the Duchenne cell therapy, the FDA insisted that the biotech company provide more "substantial evidence of effectiveness." But the real evidence that matters is that children and young adults are suffering and dying without treatment.Technology, bioengineering, and gene therapy are blessings of hope for every family and patient suffering from orphan diseases.Give kids their one chanceBureaucrats should not let political agendas or indecision get in the way of potentially lifesaving decisions when parents of terminally ill children are more than willing to accept the high risk under President Trump’s “right to try” initiative or any other FDA-approved protocols. They pray these treatments will help their children, but they fully understand that they might also only benefit future children with these diseases.That level of selflessness should be considered by the FDA when making these decisions.Parents of children with fatal orphan diseases have written the FDA an unequivocal permission slip. Perhaps under new leadership at the Medical and Scientific Office, it will finally take this hall pass and run with it — on behalf of children who can’t.
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