In an effort to cope with controversies over “compassionate use” of experimental drugs, Johnson & Johnson has appointed America’s best-known bioethicist to head an independent panel to assess requests.
Art Caplan, of New York University School of Medicine, will head a company panel examining cases of desperately-ill patients who believe that a drug which has not been approved by the Food and Drug Administration will help them.
Dr Caplan compares the Johnson & Johnson panel to what the United Network for Organ Sharing (UNOS) has achieved with its allocation process. "We want to establish a model that will create a structured policy of allocation based on equality, need and efficacy to ensure that the utility of our scarce resources are maximized," he said. "If successful, [it] will serve as a model for others in industry and in government to follow."
In recent years, these requests have become more common. Patients and their families have applied pressure via social media to drug companies to give them access to experimental treatments. The drug companies are reluctant because the drugs are in short supply or unproven and possibly harmful. Some states have attempted to short-circuit the clinical trial process for these heart-rending cases by passing “right to try” laws.
It is estimated that the FDA granted about 1800 requests from drug companies to allow compassionate use in the 2014 fiscal year. It approved nearly all of them.
Compassionate use is an attempt to strike a balance between patient autonomy and informed consent. On the one hand, patients are making their own choices after being informed of the risks. On the other hand, they may be too sick or may not have the background to understand the risks. Often the odds of an experimental treatment working are as low as 10%. As a recent article in the New England Journal of Medicine pointed out, “it may well not be in the interest of patients, however sick they may be, to have easier access to products that are ineffective and may actually worsen their clinical status.”
Furthermore, by diverting resources from clinical trials, “compassionate use” could hold up the approval process and delay the moment when other patients can access the drug. Sometimes, as in the recent Ebola epidemic, the allocation of experimental drugs can affect whole populations and a country's foreign policy. Navigating these stormy waters is a job which requires not only medical knowledge but tact and adroit political skills.
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