Three Mothers’ Plea to the F.D.A.: Save Our Children

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To the Editor:

Re “Why Can’t More Children Get the Treatment That Saved My Son’s Life?,” by Elizabeth Currid-Halkett (Opinion guest essay, Feb. 24):

Our three children, ages 5 and 7, battle a rare, relentless and ultimately fatal disease called cystinosis. We recently found hope in the initial phase of a gene therapy clinical trial that was shown to be safe and yielded very promising results — a therapy that could one day save our children’s lives. Our biggest fear is that it will not be accessible to them or others in desperate need.

Dr. Currid-Halkett beautifully articulated the fears of parents like us, who find hope in the promise of new therapies but face, as she said, “roadblocks that prevent more families from gaining access to these new treatments,” including “dissent over how flexible regulators should be in interpreting clinical trial results and taking qualitative improvements into account.”

She cites Dr. Peter Marks, the director of the F.D.A.’s Center for Biologics Evaluation and Research, for recently making a courageous call for the approval of Elevidys, a treatment for patients with Duchenne muscular dystrophy. We applaud Dr. Marks’s wisdom and perspective. However, the F.D.A. reviewers’ initial rejection is a cautionary tale of how patient access to lifesaving therapies could be impeded by a narrow interpretation of efficacy.

We implore the F.D.A. to consider what Dr. Currid-Halkett calls “a more flexible view of treatment efficacy without losing focus on safety.” Specifically, this lens should be applied to the way outcomes are measured, including quality of life improvements, to avoid denying lifesaving treatments to all patients in need.

For example, if a 1-year-old who receives a gene therapy treatment is cured of their disease and a 10-year-old who receives the same treatment isn’t cured but is granted a longer, healthier life as a result, should we not make treatment available to both of them?

Every child with a life-threatening disease deserves a fighting chance. We hope the F.D.A. agrees. The lives of our three children depend on it.

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