This article is based on a commentary in the American Journal of Bioethics.
With the roll out of Obamacare we should pause to appreciate not only the lethal inefficiency of bureaucratic control of such a complicated system as health care, but how unethical and even brutal it can be when people’s lives are determined by the whims of ideologically-charged committees of experts. And there is no more tragic example than that of the regulatory system for approving new and lifesaving medicines. Indeed in terms of bioethics and basic morality this is the Tuskegee syphilis experiment of our time.
There is a continuous stream of cases reported in the media of desperate people trying to access medicines which have demonstrated promise. Yet organizations such as the National Coalition for Cancer Survivorship (NCCS) actively campaign to stop such people accessing the only hope they have to stay alive, all under the false premise that “properly conducted” trials need to be performed. The basic, inescapable form of this argument is that ‘this panel has decided that you have been chosen to be sacrificed so that others can (maybe) live.’ Many brave Senators and Congressmen and women have risked scorn from the established academic-scientific complex for daring to challenge Big Academe’s slavish, pompous, unsophisticated and pseudo-religious-like adherence to ‘evidence-based medicine.’ We should applaud these law-makers, because they actually support a far more convincing and sophisticated understanding of the issue than the NCCS and their ilk.
Over the years, Courts have rejected attempts to frame access to lifesaving medicines as a constitutional right, most notably in the case of the Abigail Alliance. Regardless of whether one agrees with the judicial reasoning of the Courts, a new push needs to be made, based on the lack of benefit of the clinical trial ritual. Courts have generally considered a balance of interests rather than an absolute rejection of constitutional protections of access to life-saving medicines, but as evidence-based medicine is increasingly coming under scrutiny, courts need to update their balancing equation.
Constitutional protections aside, it is essential to develop a more moral, ethical, and efficient system for approving all new potentially lifesaving medicines: a system, I argue, based on free-market principles in which all clinical and preclinical information is evaluated at each step by patients, physicians, researchers, regulators, and third-party-payers.
Who should benefit from new drugs and how this benefit is distributed across different current and future patients is intimately involved with one of the main arguments against a more free-floating approach to drug regulation, as summarized in an analysis of the landmark Abigail Alliance case: “Expanded access may reduce clinical trial enrollment, which in turn will slow the completion of the clinical trials needed for approval. If potential subjects had the choice to either enroll in a clinical trial with a placebo control and a 50 percent chance of obtaining treatment or enter an expanded access program knowing that they would receive access to the unapproved therapy, few would choose to enroll in clinical trials”
As reported in the Lancet, the argument that “Allowing access to treatments outside the clinical trial setting slows the general availability of some drugs by as much as a year by reducing the number of people in trials … When possible, people would choose the route that ensures they will receive the therapy being tested instead of risking the chance they will be assigned to the placebo arm of a trial” was supported by groups such as the National Coalition for Cancer Survivorship when opposing more liberal access to new medicines in the Abigail Alliance case.
The circular nature of this argument can be understood by reference to a classic scene from the cartoon “South Park.” The character Stan’s suitability to join a group of extreme animal liberationists was determined by the otherwise random reaction of a goat. In other words, if you establish a system based on superstitious nonsense, then in order to comply you have to undergo the ritual. In this regard, innocent people are being sacrificed because the likes of NCCP have been indoctrinated to worship the FDA’s goat.
Smith and Pell’s landmark 2003 critique of evidence-based medicine in the British Journal of Medicine (when they satirically demonstrated the lack of appropriate evidence for the efficacy of parachutes) is particularly relevant to this argument. They pondered, whether “we continue our quest for the holy grail of exclusively evidence based interventions and preclude parachute use outside the context of a properly conducted trial. The dependency we have created in our population may make recruitment of the unenlightened masses to such a trial difficult. If so, we feel assured that those who advocate evidence based medicine and criticize use of interventions that lack an evidence base will not hesitate to demonstrate their commitment by volunteering for a double blind, randomized, placebo controlled, crossover trial.”