Is the Era of Tree to Choose' Medicine Upon Us?

• Author: Hemphill, Thomas A.
• Position: Free to Choose Medicine, 3rd ed. - Book review

Free to Choose Medicine, 3rd ed.

By Bartley J. Madden

83 pp.; Heartland Institute, 2018

Over a decade ago, Bart Madden unveiled the genesis of his "Free to Choose Medicine" concept in the pages of Regulation (see "Breaking the FDA Monopoly," Summer 2004). He developed those ideas in the monograph Free to Choose Medicine, the third edition of which was released this April. Just a few weeks later, President Trump signed "Right to Try" legislation giving terminally ill Americans greater access to investigational drug treatments that have undergone Food and Drug Administration Phase I safety and early efficacy testing but have not completed the full FDA testing regimen and are not yet available to the public.

Madden's arguments support policies like "Right to Try," but there is much more to what he proposes than simply giving terminal patients access to experimental drugs. In this review, I sketch out his proposal and offer some practical suggestions for increasing the possibility that it will one day become law.

Need for Free to Choose Medicine / On the first page of the first chapter, Madden states the purpose of his Free to Choose Medicine concept:

This book makes the case that we need to be free to make informed decisions about whether to use not-yet-approved therapeutic drugs--that is, new drugs that have successfully passed initial safety trials, generated preliminary efficacy data, and may offer us the opportunity to improve our health or even save our life. He believes that the result of the 1962 federal legislation expanding the FDA's monopoly authority to test drugs and judge whether they are "safe and effective" has been ever-increasing prescription drug prices. He argues this is because of the agency's continuing demand for more expensive and time-consuming clinical trials to minimize the likelihood of injury and death (and adverse publicity for the FDA) and expand understanding of the experimental drugs' effects. More importantly, he argues powerfully that these lengthy clinical trials come at a steep cost in the unreported "invisible graveyard" of patients with serious or immediately life-threatening conditions who die while the drug undergoes the extensive testing necessary to be brought to the U.S. market.

He gives four reasons why there has not been a growing grassroots movement demanding reform of the FDA in order to make experimental drugs more readily available. First, it's easier to observe (and the media report on) adverse side effects (including death) from approved drugs as well as new information from extensive trials. People assume that unapproved drugs would be even riskier, which results in political pressure for even stricter regulation. Second, the FDA and its supporters sincerely believe they possess the moral high ground; pharmaceutical companies that would be inclined to question FDA policies are, unsurprisingly, fearful of antagonizing the regulators whose decisions can mean the difference between company failure and success. Third, most Americans are unaware of what economists call the "opportunity costs" of not being free to make an informed choice about the best drug treatment for ourselves. Fourth, only a small percentage of people at any one time realize they are experiencing pain, suffering, and the prospect of death because of denied access to new drugs.

The drugs-to-patient system / Madden applies a systems theory approach in his analysis, one that elevates the goal of providing more drugs to more patients and that focuses on eliminating questionable constraints on those drugs. He believes his systems approach "produces genuine insights that reveal a path to true reform."

The existing FDA drug approval system consists of two primary stages: the pre-clinical stage and the clinical trials stage. Historically, for every 5,000 substances that drug companies initially investigate, about 250 enter the formal preclinical research and testing stage where there is basic evaluation for patient safety. This stage typically takes three to six years to complete. Only about 10 of those substances exhibit enough promise to induce a pharmaceutical company to file an Investigational New Drug (IND) Application with the FDA to move on to Phase I testing where they are safety-tested on healthy volunteers. Roughly eight of those substances then enter Phase II safety and efficacy clinical trials where they are tested on volunteers who have the condition the substance is intended to treat. Only about three of those substances move on to Phase III efficacy clinical trials, involving larger testing groups. Finally, only about one drug out of those original 5,000...