Dr. Scott Gottlieb promises patient safety, proven science to guide FDA decisions

FDA Nominee: Dr. Scott Gottlieb vows to put patients first at FDA

Dr. Scott Gottlieb, a cancer survivor and physician, promised at a confirmation hearing Wednesday that patient safety and proven science will guide the Food and Drug Administration‘s decision-making if he’s confirmed as President Donald Trump’s choice to lead the agency.

“I am a cancer survivor, I was treated for cancer during my last tour at FDA,” Dr. Gottlieb said in his opening statement to the Senate Health, Education, Labor and Pensions Committee, “so I know the importance of what American medicine does — and what the FDA does– for every one of us.”

During his confirmation hearing, Dr. Gottlieb’s extensive experience in nearly every facet of health care was on full display as he laid out his vision for a patient-focused, science-based regulatory agency.

“I’m going to be guided by the science, and the expertise of the career staff, and what’s good for patients,” he told senators. “The American people deserve to trust that the agency is led in an impartial manner — guided only by the science that informs its work — and an abiding faith to the public health.”

Patient advocates say that his impressive performance before the Senate’s health panel demonstrates why Dr. Gottlieb is the best person to lead the agency.

“Scott Gottlieb’s vision for the FDA puts patients first,” said Jonathan Wilcox, co-founder and policy director of Patients Rising. “At Wednesday’s confirmation hearing, he proved that he’s the right leader to bring great, lasting and transformative change at the FDA.”

The False Dichotomy: Speed or Safety

A former deputy commissioner at the FDA, Dr. Gottlieb is receiving broad support from patient advocates, physicians, and health care policy experts, who are hopeful that he can eliminate the bureaucratic red tape that delays treatment innovations from reaching patients.

“Dr. Scott Gottlieb is a medical school professor with bureaucratic know-how and a long, pro-science resume,” Hank Campbell and Alex Berezow of the American Council on Science and Health write at USA Today. “That makes him an excellent choice to head the Food and Drug Administration.”

In recent years, patients living with rare diseases have strongly criticized the FDA’s slow and cumbersome approval process, which, patients say, stifles  hope and blocks patients from accessing potentially life-saving treatments. Advocates for patients living with rare diseases say that the FDA’s regulations of clinical trials should conform to the era of precision medicine.

“Too many new medicines today for too many diseases though, especially rare diseases, are not reaching patients as fast as necessary,” says John Crowley, who is on a quest to find a cure for his two children diagnosed with Pompe disease, a severe and often fatal neuromuscular disorder. “Dr. Gottlieb’s nomination is a great day for science, for medical innovation and above all, for patients and families living with life threatening and disabling conditions.”

In 2012, Gottlieb wrote about the regulatory obstacles for patients living with Hunter-syndrome, a rare disease that affects fewer than 2,000 patients worldwide. The small number of affected patients makes it difficult — in some cases impossible — to meet standards for a traditional clinical trial.

“In order to approve the drug for use, the FDA required the trial to involve 96 patients with Hunter syndrome — some 20% of all Americans afflicted with the disease,” he wrote in a piece for National Affairs.

In responding to tough questioning by senators, Dr. Gottlieb challenged health care leaders to reject what he calls the false dichotomy between speed and safety.

“We should reject a false dichotomy that it all boils down to a choice between safety and speed,” he explained. “We should be reminded always, that we save lives by allowing good things to happen, but we also save lives when we keep bad things from happening.”

FDA’s “Real Appetite for Change”

Dr. Gottlieb’s impressive performance before the key Senate panel comes at a crucial time for an agency embracing health care innovation and patient engagement.

“The FDA is showing a real appetite for change, and the agency is likely to benefit enormously from Gottlieb’s experience in the industry,” writes David Shaywitz, a visiting scientist in the Department of Biomedical Informatics at Harvard and chief medical officer at DNAnexus. “With the right leadership, the FDA — however improbably — might lead us out of our present health-care miasma.”

He points to recent patient engagement programs, such as the Patient-Focused Drug Development Initiative, the Patient Representative Program and openFDA, as evidence of the agency’s changing culture.

At Wednesday’s hearing, senators endorsed that vision for a stronger patient focus at the FDA.

“You know first-hand how medical treatments affect patients and families,” committee chair Sen. Lamar Alexander Jr. said during Wednesday’s confirmation hearing. “My hope is that you will help move the agency forward so that America’s patients benefit from the remarkable discoveries our nation’s researchers are working on.”

For more on Dr. Scott Gottlieb’s background, check out our post, “Why Patients Win with Physician-Scholar-Survivor Scott Gottlieb at FDA.”

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