The FDA and Doctors: Why We Need to Pay Attention
I am often surprised that any doctors and medical students miss the mark on what FDA stands for. Certainly, the confusion is understandable—“Federal Drug” versus “Food and Drug”—but each time I catch the mistaken reference, I am reminded how minimalized this federal institution is among the medical community.
Of course, the FDA does regulate drugs, but on a larger scale, its regulatory powers touch nearly 25% of the US economy by way of over-the-counter and prescription drugs, biologics, medical devices, cosmetics and tobacco products, and foods—all goods that can be important elements in health, illness, and medicine.
It seems, that despite many hours of classroom and bedside instruction in our medical schools, few of us hear more than a cursory lecture on FDA as part of our pharmacology course, and occasional comments about FDA from our attending physicians.
Considering the important regulatory role the FDA has over agents and products so important to medicine and health, how can doctors know so little about its history, reach, and policy-making process?
One reason is that the FDA has no formal jurisdiction for the practice of medicine.
As we know, our licenses to practice come from our respective states, and the states, not the federal government, regulate the practice of medicine. Actually the FDA’s jurisdiction and powers reside in its authority over which agents are allowed to be entered into interstate commerce, that is, to be marketed, not in controlling the practice of medicine. Additionally, even the Congress and federal courts have repeatedly confirmed that doctors may use and apply any means in the practice of medicine, requiring only that doctors have a reasonable basis to believe that they will be likely to help patients. Of course, we must be able to obtain drugs and devices in order use them to help our patients, and in controlling what is allowed to be marketed FDA does have a significant impact on the practice of medicine. This power, which is a legally vested power, and how it is exercised, is fundamentally a legal rather than a scientific and medical matter. And it is in this sense that FDA is much more an interest of the legal rather than the medical world.
Upon realizing the broad regulatory power of the FDA we all might suppose it comprises one of the largest and most costly components of the government. In fact, the FDA’s approximately 15 000 employees and $4.5 billion annual budget (2013) are such that it is only through voluntary compliance by industry that a coherent system is attained. Can you imagine that among the FDA’s many duties it inspects 16 000 drug production plants annually in the United States and abroad to ensure drug purity and safety? Even this effort allows inspection of plants only every 3 to 4 years.
I believe few doctors realize that it is because of the FDA regulations that the United States is 1 of only 2 countries that allow direct-to-consumer drug advertising. The FDA totally controls all drug, device, and biologic label contents and ultimately advertising. When we prescribe and instruct patients to use agents in ways not on the label, our usage is considered to be “off-label” but is sometimes called “unapproved.” Many physicians are uncomfortable with such use.
FDA must approve drug names before the drugs are allowed to be marketed. We read about FDA advisory committees and even know people who have served on them. However, it is not widely realized that most drugs are approved with such committee input, while over 90% of devices are approved with no human study. Neither doctors nor the public are very much aware of how limited the database is for most new devices, especially compared to the wealth of information available for new drugs.
Most doctors today would be surprised to know that until 1962 the law did not require the FDA to ensure that drugs would work, that is, be effective. Only safety was required. Although doctors generally understand that human drug trials, divided into phases 1, 2, and 3, are a very important part of the requirement to prove to the FDA that a potential drug is both safe and effective, it might be surprising to many that these clinical trials are the most costly part of developing a drug.
Clearly, science and medicine are key to the success of the FDA’s public health mission. I believe FDA will be better served when more doctors are tuned into its goals, policies and actions. And while most doctors will not participate directly with the FDA, most will participate indirectly through their specialty and other medical societies. Importantly, knowledge of the FDA and its policies and procedures can serve individual doctors in many ways: making us more comfortable using agents off-label, improving our ability to evaluate the value and safety of devices and drugs, increasing our knowledge about the regulation of supplements and nutriceuticals, and improving our ability to help patients who want unapproved agents.
To my mind, these and other insights offered by knowing more about the FDA, allow us to be more thoughtful and perceptive physicians. Additionally, we may be personally enriched by being better connected to the public debate on the many societal issues related to matters such as tobacco regulation and the level of data needed for approvals, versus the inhibitory effect high costs have on development. It is with these thoughts in mind and believing that physicians like to have a formal method by which to approach a new body of knowledge that I wrote The FDA for Doctors.*
William H. Eaglstein, MD, is chairman emeritus in the department of dermatology and cutaneous surgery at University of Miami’s Miller School of Medicine in Miami, FL and the author of The FDA for Doctors (Springer 2014).
*Written from a physician’s perspective for physicians, The FDA for Doctors (Springer 2014) provides an in-depth look at one of the most formidable regulatory institutions in America, putting into the spotlight, its societal role, policies, goals, and challenges. For a preview of the book online, visit http://www.springer.com.