Dr. Aaron S. Kesselheim, who served on the F.D.A. advisory panel that thought-about the Alzheimer’s drug and resigned from the committee after it was accredited, stated Dr. Califf’s background operating medical trials can be beneficial for main the company.
“I don’t suppose it’s essentially disqualifying,” stated Dr. Kesselheim, a professor of drugs at Harvard Medical Faculty and Brigham and Ladies’s Hospital. “I believe the truth that he labored for such a very long time in medical trials demonstrates that he has experience in understanding what goes into a very good medical trial. Hopefully, he can convey that into his function as an F.D.A. commissioner.”
However Dr. Kesselheim objected to Dr. Califf’s efforts, when he was the commissioner, to permit drug corporations to promote off-label makes use of for his or her merchandise, noting that sufferers could be endangered by medicine which might be prescribed for makes use of that the F.D.A. has not accredited. “That to me is a purple flag,” Dr. Kesselheim stated. “Hopefully, he’s moved previous that as an thought, as a result of it will be a horrible thought.”
Throughout his first tenure on the company, Dr. Califf centered on modernizing the gathering and use of digital well being knowledge to reply questions on medicine and medical units that might not be gleaned from medical trials.
He additionally sought to extend the usage of “real-world proof” — case research and particular person affected person experiences — to tell regulatory selections. Some public well being specialists worry that counting on knowledge exterior of randomized medical trials endangers sufferers; Dr. Califf believes there’s room for each approaches.
His willingness to contemplate such knowledge brought about controversy in 2016, when the Meals and Drug Administration overruled its specialists to approve a brand new drug for remedy of a uncommon, deadly muscle illness, regardless of lack of proof that it labored.
The drug, eteplirsen, was given conditional approval amid fierce lobbying by younger muscular dystrophy sufferers, their dad and mom and the drugmaker, Sarepta Therapeutics. Dr. Ellis F. Unger, who labored beneath Dr. Woodcock on the time, known as the drug “primarily a scientifically elegant placebo.”