One of the most important components of the experimental-drug development process is the compassionate program, says Jerome Groopman, chief of experimental medicine at Beth Israel Deaconess Medical Center. Also called expanded access, the compassionate program allows patients to receive drugs that have not yet been approved by the Food and Drug Administration.

Compassionate programs were originally a response to AIDS activists, but now include other life-threatening diseases. But because the FDA has no formal regulations covering such programs, and because they are not part of the regular drug approval process, patients who could benefit from new treatments die before being able to obtain them.

• The drugs involved are usually in Phase III of the drug approval process -- controlled clinical trials using select subsets of patients -- or are awaiting approval by the FDA.

• The patients requesting compassionate access may include those who are too sick to participate in the controlled studies.

• For each compassionate access program, companies make a proposal to the FDA -- which is usually approved if it fulfills minimum requirements for scientific and ethical merit, and targets a population with the disease that was included in previous trials.

• At their own expense, drug companies provide drugs free to patients, and analyze and present data on their treatment to the FDA.

• However, since FDA drug approval is uncertain, the drug is often not in full production, quantities are limited and patients may be selected by lottery.

Furthermore, since patients given compassionate access are likely to be sicker and suffer toxic side-effects, drug companies risk having their products tarred with a bad reputation.

Compassionate access could be expanded, says Groopman, if such programs were made part of the regular drug approval, and if companies were given tax-write-offs for the expense or patents with extended lives.

Source: Jerome Groopman (Harvard Medical School), "Merciful Medicine," New Republic, April 26-May 3, 1999.