Should Terminally Ill Patients Have the Right to Take Partially Tested Drugs?

by VR Sreeraman on Sep 7 2007 8:29 PM

As the United States considers allowing experimental drugs to be given to people at the end of life, two experts in this week’s BMJ debate whether terminally ill patients should have the right to take drugs after initial safety (phase I) trials but before final approval.

Professor Emil Freireich at the University of Texas believes that patients should be allowed to judge the risks for themselves.

Around half a million people will die from cancer related causes in the Unites States this year, and many are given a hopeless prognosis, he writes. For most, the option of participating in clinical trials of new drugs that offer some promise helps them remain optimistic, but most cancer patients are unable to participate in these trials.

So why not offer these drugs to these patients on a compassionate basis, he asks?

He points to several objections including safety, interference with the development of the drug and with the clinical trial process. But he rejects these and argues that patients with advanced cancer and limited life expectancy should have the same privilege as all individuals in a free society – that is, to decide their own benefit: risk ratio.

It is tragic, he says, that regulatory bodies have created a circumstance where people have to live in an aura of hopelessness even though they have the will, the resources, and the ability to expose themselves to the risk of participating in investigational studies and to enjoy the potential for benefit.

The solution is legislation or judicial action to permit expanded access to experimental treatments for patients with limited life expectancy, he concludes.

But Dean Gesme, a medical oncologist in Minneapolis believes that use of drugs after phase I testing and outside clinical trials will damage both individuals and science.

He points out that more than 90% of drugs entering phase I trials are found unacceptable, and, of those approved, most provide incremental improvements rather than lifesaving treatments.

The allure of promising new drugs continues to engender false hope, he says, which may delay approval and erode the clinical trials system by substituting clinical enthusiasm and wishful thinking for evidence based medicine.

And who will bear the costs of open access to these partially tested drugs, he asks? Will government and others who are now seeking to minimise payments for marginally beneficial therapies be willing to pay for unproved drugs outside of formal clinical trials?

While doctors dream of the miracle cure for each of their terminally ill patients, we must accept the duty and responsibility to conform to both principles of evidence based medicine and the precepts of appropriate end of life care, he concludes.