Focusing the spotlight on the real Scott GottliebIn recent weeks, the media has been focusing on Dr. Scott Gottlieb, Deputy Commissioner at the Food and Drug Administration. Unfortunately, they've been focusing on everything but his track record, medical background and vision. They have tried to characterize him as the pharmaceutical industry's puppet.
Not just the one the media invented
Robert Goldberg, Ph.D.
Medical Progress Today
September 15, 2005
In reality, he represents the sort of change that special interests and the media both hate and fail to understand.
The public can thank Scott Gottlieb for advancing the science of personalized medicine at the Food and Drug Administration. In his year long stint as Senior Adviser to then-FDA Commissioner Mark McClellan, Dr. Gottlieb was one of the principal authors of an FDA mission statement setting forth a goal of using new scientific tools to tailor drugs to our genetic differences. Consistent with that vision, Gottlieb helped reorganize the FDA's Office of Cancer Drugs around faster approval of medicines based on such insights. And he was McClellan's point person in getting reluctant drug reviewers to approve Fabrazyme, an orphan drug that controls the overproduction of fat cells that in turn destroy the vital organs of infants and children until they die. Gottlieb designed an analysis that would predict risk and benefit without additional clinical trials.
Gottlieb followed Dr. McClellan to the Centers for Medicare and Medicaid Services, where he spent another year trying to speed up the approval of new drugs and devices and encourage their developers to come up with meaningful evidence that such products improved and lengthened lives. Before then, Medicare simply paid for new products after a two-year wait without getting a commitment from companies to provide such information. Going forward, they will have to.
After he left Medicare, Dr. Gottlieb went to the American Enterprise Institute where he has concentrated on developing a 21st century approach to making drugs safer. This approach includes real time analysis of electronic patient records to discover possible safety problems and additional benefits, and using that information to continually refine and improve patient care. Throughout his brilliant career, Gottlieb was an emergency room physician where he made life or death decisions in a way that was more immediate than many FDA staffers. When not writing for AEI or tending to patients, he published a newsletter on biotechnology, a combination of observations on the FDA and biotech stock recommendations.
In short, Dr. Gottlieb is committed to making medicines safer and more effective by making them more personalized.
Indeed, in his absence, the FDA's progress towards personalized medicine and technologically enhanced post-market drug evaluation has languished. The FDA, under attack from the media and Congressional critics for approving Vioxx, has been in steady retreat and are nervous about approving any new medicine that entails any risk. These critics have already attained the policy changes they have sought: the FDA is more cautious, more likely to delay drug approval to obtain more but meaningless safety studies, and less likely or able to implement approaches to drug evaluation that are more personalized.
Dr. Gottlieb's return to the FDA therefore represents the sort of medical progress that special interest groups and media oppose. Gottlieb wants to expedite the approval of life saving drugs. They do not. He wants a transparent system of drug monitoring that will provide patients and physicians instant information on the best and safest use of medicines for them. They want trial attorneys to decide who gets what medicines.
Attacking Gottlieb is making it less likely the FDA will approve drugs targeted for diabetics that do poorly on other medicines. And it ensures that he and the agency will spend less time on science-based reforms. Patients will pay for the politically motivated attack on Deputy Commissioner Gottlieb with their suffering and indeed, their lives.
For instance, over 20 million Americans suffer from diabetes. It is one of the fastest growing diseases in our country and around the world, and a leading cause of stroke and kidney failure. Yet, fully 20 percent of those with diabetes don't respond to all the medicines on the market. As David Altshuler, director of the Medical and Population Genetics program at MIT's Whitehead Institute and an endocrinologist at Massachusetts General Hospital, observes: "The treatments we currently use to treat most patients are grossly ineffective. In type 2 diabetes, many people don't respond."
Most drugs don't work for a large group of diabetics because neither pills or shots successfully deliver insulin in ways that mimic the body's natural production. Studies show that for many people, an inhaled form of insulin will likely provide effective treatment where injections have failed. Like all drugs, there are side effects for a small percentage of people. But with proper and proactive follow-up, including studies comparing drug response to genetic variations, doctors can tailor diabetes drugs to the patients who will benefit most from particular drugs. As the new deputy commissioner of the FDA, Dr. Gottlieb takes these problems personally and knows what the agency needs to do to get more personalized diabetes medicines to patients.
Robert Goldberg, Ph.D. is director of the Center for Medical Progress at the Manhattan Institute for Policy Research.