Patient Access to Unapproved Drugs: An Ethical Debate

Jul 5, 2011
Stetson Thacker

Developmental Drugs - Epsos.de

The history of patient access to unapproved drugs and an overview of the medical ethics involved in the debate over patient access to investigational drugs.

At the age of 17, Rhett Davis was diagnosed with a rare blood disorder that is termed paroxysmal nocturnal hemoglobinuria or PNH. However, he did not begin to get the blood clots that would eventually decimate his liver and cause his kidneys to fail until he was in his thirties. Fortunately for Davis, he was able to access an investigational new drug (IND) called eculizumab, which dramatically improved his condition. Davis’s path to achieve this IND was extremely fortuitous because he qualified for “compassionate use” of eculizumab under the IND regulation and eculizumab happened to be an experimental drug that was far enough along in its extended approval process to be legally given to a patient under the “compassionate use” IND regulations.

Not all patients are as fortunate as Davis. A young woman named Abigail Burroughs died as a result of a rapidly growing tumor. While she was dying, Abigail and her father Frank attempt to gain access to Erbitux and Iressa, which were two experimental drugs that were being tested on tumors in the colon and lung. Abigail did not qualify to be admitted to the clinical drug trials because her tumor was in her head and neck despite that both IND treated the underlying cause of Abigail’s tumor. Abigail soon died. Erbitux and Iressa were later approved by the Food and Drug Administration. Her father co-founded an organization called Abigail Alliance for Better Access to Developmental Drugs.

Abigail Alliance

Abigail Alliance sued the (FDA) in district court; however, the FDA’s motion to dismiss was granted. On appeal, a panel of three judges overruled the district court’s decision and decided that “on a physician recommendation mentally competent terminally ill patients with no alternatives have a constitutional right to obtain potentially life-saving drugs that are eligible for post-phase 1 testing.” This was a major victory for Abigail Alliance and all terminally ill patients desperately awaiting their miracle drug.

Ethical Debate

The ethical debate concerning patient access to unapproved drugs still ensues. Those who advocate for the access assert that the patients who desire the drug are in no position to lose anything by trying the experimental treatment. They also criticize the FDA’s restrictive policies and procedures for drug approval, which involved passing three clinical trials before becoming eligible for approval and entering the market.

On the other hand, many assert that the FDA’s regulations are protecting people from dangerous drugs that could cause more harm than good, even to patients who are terminally ill. They also argue that granting patients’ access to IND will dramatically reduce the number of patients who enroll in clinical trials, which will cause drug development to stagnate. Furthermore, granting access to INDs circumvents the authority of the FDA to regulate the pharmaceutical companies and may allow pharmaceutical companies to market false hope to these patients. This is especially concerning given that numerous developmental drugs that enter clinical trials never make it to the market because they are too dangerous or ineffective. Furthermore, physicians that are willing to prescribe post-phase 1 INDs are still liable for any harm that comes to the patient. The drug companies whose IND is used is also liable for any harm coming to the patient from the IND.

Thus, the debate about the ethics of allowing or restricting patient access to investigational drugs is a typical aporia. However, there are some issues that can be settled. For instance, patients should not have access to cosmetic IND or INDs that do not treat serious or life-threatening conditions. Moreover, when a current alternative is available the IND should not be sought for by the patient. In addition, since legal access has been granted to patients after phase 1 of clinical trials, doctors prescribing, and companies supplying, these IND should be protected from liability because there is little to no financial conflict of interest for them to provide the drug inappropriately and they will be reluctant to provide the treatment to the terminally ill patient if they are not protected from liability.