Balancing drug-test benefits

? Authoritative sources from a well-known pharmaceutical company divulged to us the truth about compassionate use of experimental drugs. This is a heart-rending issue involving untold numbers of critically ill people who are desperate for a cure.

The perceived culprit is the Food and Drug Administration (FDA), but this is not justified by the facts because the FDA has given a virtual green light for drug companies to disseminate most experimental drugs to critically or terminally ill patients.

On the other hand, a recent article in the Washington Post blamed the situation on the system, claiming that the patient pool for testing would dry up in the face of widespread compassionate distribution because potential test volunteers would have to acknowledge that they might be receiving placebos rather than the actual drug. This type of testing, it is believed, is necessary for FDA approval. Not so, says our source.

Tests that involve both the actual drug and placebos are called double-blind tests. The idea is to determine whether the drug actually provides a benefit, but this could not be accomplished, so the theory goes, if the patient knows whether he or she is receiving the real thing. But our source tells us that no reputable drug company would knowingly give a placebo to a critically ill patient.

Instead, the drug companies conduct comparative tests in which a new drug is matched up with a drug already on the market that is known to be beneficial. And here is where the problem lies. Patients do not want old drugs any more than they want placebos. They want the latest drugs, and they tend to rush to the newest, albeit experimental, drug even when there is no evidence that it will be better than what is already on the market. This can result in lower sales of existing drugs without a commensurate benefit to the public.

One critically ill cancer patient who is close to us obtained an experimental drug that proved to be exceedingly beneficial, but it caused a severe rash, so she discontinued it for a couple of weeks. When she asked for the pills again, she was turned down because she had broken the test protocol. Here is a case of someone who was allowed to have the drug for compassionate purposes because she did not meet the test guidelines, but then was denied the drug for failing to abide by the test requirements.

This particular case exemplifies the problem. The drug company wants to be compassionate, but it also wants FDA approval. Further, it wants there to be a lucrative market once it receives that approval, which it cannot do if patients are already rushing to the next experimental, yet-to-be-FDA-approved drug.

Meanwhile, the patient simply wants to survive.

What is the solution? If the FDA completely steps away, every snake oil company in the country will be free to peddle false-hope elixirs to desperate people. Yet, if legitimate drug companies open the floodgates to every new experimental drug, patients may give up drugs that are benefiting them for drugs that will not, with the result that deaths will increase while pharmaceutical profits decrease. And it is profits that drive the research that creates the miracle drugs. But the alternative of denying drugs to desperate people is equally unacceptable.

The solution can be achieved through an expedited system. Under the Prescription Drug User Fee Act, priority approval can be obtained in six months, and compassionate use can be authorized during clinical trials. But compassionate use should be allowed after minimal, limited testing that could be performed in one to three months. In this way, only those drugs that show promise would be released for distribution, and then only to critically ill patients.