John Hood

Congratulations to Karim Mikhail on his appointment as Director of CBER. I read his post carefully. One line belongs at the front of every review: “The scientific record tells you what a therapy does. It cannot tell you what waiting for the treatment costs.” I have sat with families whose disease has no approved treatment. For them a review clock is far more than a metric. It is time they may not have. He is right to put that sentence first. It decides how the rest of the job gets done. Every regulatory decision carries two risks: the risk of approving a product, and the risk of not approving it. Both are real. Both land on real people. Only one of them makes headlines. It is easy to say no when you fear tomorrow’s story about a side effect. We will never see the headline about the child who died, the patient who went blind, or the parent who ran out of time because a promising therapy never reached them. For rare and fatal diseases with no other hope, that silence is not neutrality. It is a cost. This is not an argument for lowering the bar. The FDA must still keep products off the market that carry real risk and offer no meaningful benefit. Inspections matter. Data integrity matters. Informed consent matters. Post-approval commitments and rigorous safety monitoring are how you give desperate patients a chance without abandoning the scientific method. Rigor and urgency are not opposites. Rigor is what makes speed worth anything. Speed without rigor is recklessness. Rigor that ignores the patient running out of time is a quieter failure — easier to defend in a memo, and just as final for the family. Where traditional gold-standard trials cannot be done — too few patients, too rapid a decline, a first-in-class mechanism that will never fill a conventional control arm — the honest answer is not “come back in ten years.” Use the best evidence that can actually be generated. Put the uncertainty in plain language. Require the monitoring that protects the next patient. Then trust American physicians and American patients to decide. The FDA’s job is to make sure the science is sound and the information is complete. Then let patients and doctors do what is best for them. The FDA’s only client is the American patient. Full stop. That is why Advisory Committees matter. Open them. Staff them with people who actually treat the disease. Disclose conflicts out loud, under penalty of perjury, before another word is spoken. Give complex science more than a truncated day. Let patients speak. A modest functional gain that looks unimpressive on a slide can be the difference between a life lived and a life lost. I have enormous respect for the career scientists inside CBER (I have gotten to know many including at other FDA centers) The work on both sides of the desk is harder than the other side believes. Leadership’s job is to give them a north star that is patient-first, America-first, and innovation-first at the same time — and then to have their backs when they apply it honestly. “Frontier science only matters when it reaches patients.” Congratulations to Karim. The families who do not have ten years need CBER to get this right. Weigh both risks. Tell the truth about uncertainty. Protect people from empty products. Do not protect process at the expense of the only person the agency exists to serve.