Roosevelt Institute | Cornell University

Miracle Compounds: Ethical Issues in the Drug Approval Process

By Frank SunPublished April 6, 2014

Clinical trials in the FDA drug approval process has misled patients, for the sake of scientific precision, into believing they will be the next ones to try the new "wonder" compound. Can this be justified by the lives saved in the long run? \r\n\r\n

 By Frank Sun, 04/06/2014           
             The drug approval process in the United States is an extremely lengthy and selective one — only 1 out of about 10,000 compounds are approved over a span of 16 or more years. This leaves many patients with serious, even fatal conditions awaiting treatments that have yet to hit the market. Two types of errors can occur in the Food and Drug Administration's (FDA) drug approval process. The lengthy process can result in Type II errors, in which the FDA fails to approve a safe, effective drug in time, leading to the deaths of many patients who otherwise could have been cured of their diseases and survived. However, Type II errors are often overlooked because Type I errors occur when innovation is prioritized over safety and an approved drug is found to be extremely harmful in post-marketing studies. A significant example of this was Merck's Vioxx, which was eventually recalled due to its severe cardiovascular side effects. Thus, safety has continuously been prioritized over innovation since side effects can be fatal and pharmaceutical firms face large penalties for their mistakes.

            The approval process is designed with safety and precision in mind. Clinical trials are run in three phases, with Phase II being significant in determining whether a drug is effective in comparison to a placebo or a control drug already on the market. Herein lies the issue with the randomized, controlled trials. Compounds combatting cancers such as melanoma have been involved in extremely controversial ethical debates with regards to their testing and approval. PLX4032 is a miracle compound that, in trials, has been shown to reduce tumor growths in 81% of patients for an average of eight months.  Compare this to chemotherapy and radiation, which only worked in 15% of patients for an average of two months. However, because researchers need to know whether the effects of PLX4032 are significant, real, and safe, they must split their patient test group in half, so half of the patients receive general chemotherapy that has been known to be fairly ineffective. In study comparisons, patients receiving the PLX4032 compound have shown extremely positive results with very significant reductions in tumors while the control group receiving chemotherapy suffer through more tumors and an increased likelihood of death.

            The question remains: Is it fair to use a random generator to determine who gets the wonder drug and who gets placed in the control group? Especially with conditions like cancer, life and treatments become extremely precious and many patients are exploited for research because they are refused access to the new compounds. Policymakers and bioethicists are trying to find a compromise to help save more lives without compromising FDA research. This involves allowing patients to enter the clinical trials of PLX4032's competitors, which pressures the FDA to approve drugs more quickly, or simply to allow compassionate access and making the drug available to a select group of extremely ill patients who are willing to risk the side effects. Policymakers and the FDA are afraid that tactics being used to change the approval process would result in an increased rate of Type I errors (which are highly publicized) since research precision will be compromised due to a lack of research subjects and stable results.

            The FDA has decided to make exceptions for certain compounds being tested and breakthrough drugs are entering the market faster than ever. However, many drugs are like PLX4032 and are still in the process of being approved for patients suffering from severe illnesses like cancer and AIDS. Until then, Type II errors will continue to persist despite the fact that many of these drugs have been proven safe and their processes are solely being prolonged for the sake of precise research. Not all patients will be lucky enough to be placed on a new wonder compound, and those who do not will continue to suffer. Our healthcare system and scientific community are left asking critical questions: Is it ultimately better to save the lives of patients sooner while risking future consequences, or is it more ethically sound to allow more patients to die for the sake of the greater good in the future?