Parachutes and Pills: How Observational Data Can Save Lives
Would you jump out of a plane if there were only 50/50 odds that your backpack contained a parachute? Probably not. Yet that is how a controlled study on the safety and efficiency of parachutes would work. You and several dozen others would be randomly sorted into two groups, handed either a parachute as part of the test group or an empty backpack as part of the control group, then asked to jump.
If such a study sounds absurd, it is probably good that one has never been conducted. Such a controlled, evidence-based test, that subjected half the participants to severe injury or death due to their placement in the control group, would be wildly unethical.
So why do we willingly accept real costs to human life when considering the approval of new medical technologies for terminally ill patients?
All new drugs, treatments, and devices must pass a series of onerous tests mandated by the Food and Drug Administration (FDA). These tests include multiple clinical studies testing for safety and efficacy that can add years of delays and millions of dollars to the costs of bringing the new product to market.
These delays have real-world consequences. Individuals with terminal illnesses or debilitating diseases are often forced to wait with potentially life-saving innovations just out of arm’s reach. The limited number of openings in clinical trials means that for every patient selected, many more are denied. Even if a patient is fortunate enough to make it into a clinical trial, they may still be placed into the control group. Instead of a potentially life-saving medicine, patients in this group are given a placebo. They are handed an empty backpack.
This was the case with Thomas McLaughlin and Brandon Ryan - two cousins who developed the same form of cancer. When both were accepted into a clinical trial for a new drug, only McLaughlin received the treatment. While McLaughlin’s condition improved, Ryan lost his fight.
In extreme circumstances, shouldn’t individuals be trusted to make their own decisions? Many states believe so and have enacted Right to Try laws permitting terminally ill patients to access non-FDA approved drugs outside of clinical studies. Currently, 33 states have such laws, but they alone are insufficient to address the moral shortcomings of the wider regulatory system.
Scott Gottlieb’s nomination to head the FDA brings an opportunity to reshape the kinds of regulations holding back medical innovation. Gottlieb has spoken favorably of reforms to “fast-track” certain drugs and treatments.
The FDA could adopt a federal Right to Try standard giving patients in all states the same access to potentially life-saving treatments.
Next, the FDA can allow for greater use of observational data alongside evidence-based approaches. By observing how parachutes work in the real world, for example, we can present a clear case that they are safe to use. This kind of observational data can be utilized in situations where evidence-based testing results in harm to individuals. In fact, The 21st Century Cures Act already requires the FDA to consider observational data when considering new uses for drugs, devices, or treatments already approved. Expanding the use of observational data would reduce both the costs and time of bringing new products to the market, and the ethical challenges presented by clinical trials.
Expanding observational data would also allow the United States to expedite approval for drugs already in use in the European Union. Utilizing observational data on the effects of drugs that have been approved for a decade or more in other countries would eliminate the need to slowly and expensively re-test all those same drugs here.
With new treatments increasingly tailored to meet the needs of individual patients, large-scale clinical testing will increasingly become infeasible. By changing the regulatory mindset, the FDA can bring about a wave of new life-saving drugs and other medical innovations, all while lowering costs. For those who believe that evidence-based methodologies are the only way, here is your backpack. Welcome to the control group.
Rick Barton is a policy analyst and research fellow at the Charles Koch Institute in Arlington, Virginia.