Do patients have a right to experimental drugs?
Telling a patient that he or she has a life-threatening-perhaps terminal-disease may be one of a doctor's toughest responsibilities.
If the available treatments don't help your patient, and he asks to get an experimental drug he's heard about, should he be able to have it?
This summer, the US Court of Appeals for the District of Columbia held that terminally ill patients do not have the constitutional right to get experimental drugs unless they're enrolled in a clinical trial or take part in other Food and Drug Administration treatment options.
The Abigail Alliance was founded in 2001 when 21-year-old Abigail Burroughs sought early stage drug treatment for head and neck cancer. One experimental drug her family sought to get was eventually approved-after she died from the cancer.
While any patient death is heart breaking, allowing more patients to get early stage experimental drugs could throw a monkey wrench into the clinical trials process.
I recently spoke with Robert J. Korst, MD, medical director of the Blumenthal Cancer Center at The Valley Hospital in Ridgewood, NJ. Valley Hospital is in the forefront of conducting cardiac and oncology clinical trials. The hospital has several clinical trials ongoing, and Dr. Korst will soon be starting up trials for a specific gene therapy on esophageal cancer.
Korst is keenly sensitive to patient hopes and expectations, and also sees the importance of clinical trials. He feels that giving patients greater access to early stage unproven drugs could subvert the clinical trials process. According to Korst, recruiting patients for randomized clinical trials is a growing challenge. Patients may opt out of participating when they learn that they may not get the drug that they want, he says. Also, today's consumers may be cynical about researchers' motivations.
Beyond that, patients may not want to endure the travel, monitoring, and additional testing that clinical trials may require. And often, patients can't participate in clinical trials because they don't qualify or the appropriate trial is closed.
Yet patients can get experimental drugs in several other ways. Patients with critical illnesses can get unapproved drugs through the FDA's Compassionate Use program, in which a treating physician may request a manufacturer to allow an individual patient to use an experimental drug. The FDA also has an Expanded Access program, which allows thousands of selected patients with a particular condition to get unapproved drugs. Beyond that, experimental drugs that appear to be significantly better than current treatments can be fast-tracked.
Terminal or incurable illnesses are emotionally wrenching-much more for patients, but also for their physicians. Whether or not early stage drugs are appropriate for a patient doesn't make it any easier to face a patient and deny them what they may think is their last wisp of hope.