Breaking the FDA Monopoly on Drug Access

Published July 1, 2007

The following is an excerpt from the booklet More Choices, Better Health: Free to Choose Experimental Drugs, by Bartley J. Madden–the second in a several-part series. The complete booklet is also available online at

If you were facing a life-threatening illness, would you want the freedom to try an experimental drug? Would you be willing to take responsibility, including the risk of adverse side effects, for your decision to use drugs not yet approved by the Food and Drug Administration (FDA)?

What would your answer be if the health problem were non-life-threatening–macular degeneration, severe arthritis, or another debilitating condition? Answers vary, depending on an individual’s evaluation of the risk and scope of adverse effects versus potential health improvement.

The problem is that in today’s regulatory environment, your tradeoff evaluation doesn’t matter. FDA does not allow the use of not-yet-approved drugs, except in clinical trials and certain highly restricted circumstances.

The Abigail Alliance for Better Access to Developmental Drugs has been instrumental in promoting Senate Bill 1956 (the ACCESS Act). It addresses seriously ill patients’ right to access promising drugs before completion of the full gamut of FDA clinical trials.

ACCESS does not fundamentally change the FDA process; that is not its purpose. The goal of ACCESS is admirable–yet achieving that goal is partially dependent on FDA cooperation in formulating and administering implementation rules.

Regulatory Creep

Avoiding the entanglements of FDA rules is not easy. As Henry Miller, a medical doctor and former FDA regulator, noted in his book To America’s Health: A Proposal to Reform the Food and Drug Administration (2000), “What many fail to realize is that a regulatory statute, even if it is not amended, is not static. When the statute is first enacted, its implementation is generally narrow and limited to the specific requirements of the law, and its impact, therefore, is often modest. As time goes on, however, each successive generation of administers tends to redefine the scope of jurisdiction and add new requirements.

“Seldom does the scope narrow; almost never do requirements disappear. Regulation begins to take on a life of its own,” Miller wrote. “And as regulators interpret statutes ever more broadly and comprehensively, they become, in effect, a special-interest group with a vested interest in expanded responsibilities, budgets, and empires.”

Miller continued, “In the absence of effective, conscientious congressional oversight, what develops is an increasingly burdensome and inefficient regulatory system. Nowhere can this be seen more clearly than in the evolution of premarket licensing mechanisms for drugs.

“The current system of oversight of pharmaceutical development includes no mechanism for public accountability … [and] premarket approval severely limits individual freedom of choice. Personal autonomy is subjugated to government controls. Citizens are precluded from obtaining products they wish to purchase and have no recourse other than to await government approval,” Miller noted.

Drug Monopoly

One might well be concerned about how FDA would formulate rules to implement legislation designed to reduce its regulatory power. Nevertheless, passage of the ACCESS Act would be a genuine step forward in helping some patients with life-threatening illnesses, while chipping away at FDA’s absolute control of access to unapproved drugs.

To allow individuals to express preferences for risk would undermine FDA’s monopoly on drug access. FDA contends it needs total control in order to benefit society–future patients–by applying rigorous statistical evaluations to its extensive clinical trials data. According to FDA, patient/doctor freedom to use not-yet-approved drugs would interfere with clinical trial enrollment.

Finally, there is an FDA assumption, unspoken, that patients and their doctors are incapable of making decisions about experimental drugs.

But there is more involved than merely segmenting consumers into risk-takers and risk-avoiders. The critical point was made by economist Friedrich Hayek: “Nobody can communicate to another all that he knows because much of the information he can make use of, he himself will elicit only in the process of making plans for action. As he will not merely make use of given knowledge, he discovers what he needs to know in order to make appropriate actions.”

As Vernon Smith put it in a 2005 article in Review of Austrian Economics, “People don’t know what they will do until they face particular circumstances, and then they start to come up with solutions.”

Changing Values

Many who have not experienced the heavy personal cost associated with the current FDA process are unlikely to demand freedom of choice. Hearing media reports of recalls of FDA-approved drugs such as Vioxx, they are most likely to support additional FDA testing if asked in a survey.

But put those individuals in a different context. If they became afflicted with ALS (amyotrophic lateral sclerosis, commonly referred to as Lou Gehrig’s disease), they would be faced with a deterioration of muscular function and death within three to five years. They and their family members would experience an order-of-magnitude shift in their attitudes and need to gain knowledge about ALS in general and, in particular, about ongoing prospects for not-yet-FDA-approved drug treatments for ALS.

Their responses to a survey about FDA’s current practices and an expansion of its power almost surely would be different.

As for changing times, we can almost certainly expect accelerating medical innovations in the future.

Optimal System

Now consider an environment in which consumers have up-to-date and easily understood information via the Internet about the ongoing safety and effectiveness of experimental drugs. We would expect to observe more drugs in early-stage clinical trials with effectiveness that makes obsolete existing FDA-approved drugs.

Wouldn’t this environment motivate more consumers to want patient/doctor control of the decision to use experimental drugs, rather than an FDA monopoly on access?

Where is the present level of FDA regulation of new drugs compared to the optimum level? No one really knows. Let’s think about how a system could work that is designed to reveal the optimum level.

Bartley J. Madden ([email protected]) is an independent researcher in Naperville, Illinois.

For more information …

S. 1956, The ACCESS Act,