Scott Gottlieb is an extremely creative and knowledgeable commissioner of the Food and Drug Administration (FDA), but he will need to marshal all his talents to update an archaic regulatory regime that faces unprecedented challenges.
On May 30, President Trump signed the “Right to Try Act,” allowing terminally ill patients access to investigational drugs once they have been cleared as safe, but before they are proven efficacious for any disease. In short, Congress said, Why wait for lengthy FDA clinical trials to prove a drug is effective when people are dying?
One of the legislation’s authors, Sen. Ron Johnson (R-WI), bluntly stated that the intent of the law is to “weaken” the FDA. This is an understatement.
The next question to be asked is, “Why should only terminally ill patients have access to investigational drugs?” If a patient has a chronic illness that will shorten his or her life, and currently available medicines are not working, why not also allow that patient access to safe experimental drugs?
The Right to Try Act is revolutionary in its populist-libertarian premise that FDA efficacy trials are superfluous. Once a drug is proven safe, throw it into the marketplace and let patients and their physicians decide if it works.
The act is not the only emerging challenge to the FDA’s authority; the information technology revolution has also weakened its aura.
Decades ago, data generated in an FDA clinical trial was the “gold standard” of information about a drug. Doctors and health insurance companies had difficulty questioning clinical trial data because it was generally the only reliable data that existed, and drug company sales reps were forbidden from sharing any other information with doctors, as they still are.
With the advent of “big data,” however, studies can be conducted on the real world performance of a drug in millions of patients by crunching data in their medical records. These studies might point to valuable information such as effective uses of a drug that was not discovered in clinical trials.
Any pharmaceutical sales executive worth their salt will tell you that the balance of power has shifted away from pharmaceutical marketers who used to have a monopoly with clinical trial data to health plans and pharmacy benefit managers who can employ big data to assess a drug’s value.
These trends will make the FDA’s continued resistance to the information revolution increasingly difficult.
For example, suppose researchers at MIT using millions of de-identified medical records, discovered that a certain drug that had been approved by the FDA to treat pain was also wildly effective in preventing heart attacks. Now, suppose these researchers published the results in a peer-reviewed journal. Wouldn’t this information be important for physicians to know?
Yet, because the study is not an FDA trial, if a pharmaceutical sales representative were to share the study with a physician, that drug company would be vulnerable to a multi-billion dollar Department of Justice lawsuit — even if the study were extremely reliable.
Courts are beginning to catch up to the obsolescence of this regulatory regime. Rulings at the District Court and even Appellate Court level have asserted that discussions between physicians and biopharmaceutical sales representatives, even about unapproved uses of drugs, enjoy First Amendment protections.
When, for example, the Second Circuit Court of Appeals overturned the criminal conviction of a pharmaceutical sales representative on First Amendment grounds, the DOJ chose not to appeal the ruling to the Supreme Court, maybe sensing where the legal winds are blowing.
Even the FDA was taking baby steps towards acknowledging these trends when they recently revised rules to allow pharmaceutical companies to share certain data not derived from clinical trials with health insurers. As with Right to Try, the premise of this decision raises the question: if this information might be valuable to health plans, why wouldn’t it also be valuable to physicians?
As with any revolution, however, one must worry about excesses. Congress cannot allow a “wild west” culture where baseless claims can be made to doctors about the effectiveness of drugs.
On the other hand, in our information-driven culture, the FDA cannot succeed in keeping credible information bottled up in various regulatory silos or, as with Right to Try, the silos will be hauled down. The information revolution is here, and the FDA, like every other institution, either needs to ride the wave by adjusting to it or risk being swallowed by it.
William Smith, Ph.D., is Visiting Fellow in Life Sciences at the Pioneer Institute in Boston.
The views and opinions expressed in this commentary are those of the author and do not reflect the official position of The Daily Caller.