US Pharm. 2014;39(6):4.  

There is nothing more  heartwrenching than watching someone who has been diagnosed with a terminal illness slowly slip away after all medical treatment and drug-therapy options have been exhausted. This is particularly true when the person is a close friend or relative, and the experience engenders a feeling of overwhelming helplessness.

My brother was diagnosed with an aggressive form of prostate cancer a few years ago. He endured many difficult medical treatments and drug therapies, which extended his life for a short period of time. Sadly, it was too little too late, and he eventually fell prey to cancer’s ravenous and oftentimes unstoppable metastasis, which carried the poisonous cells to his vital organs and brought about his untimely death. I have unfortunately witnessed the same progression of cancer in my parents, other relatives, and close friends. Aside from being diagnosed with a terminal illness, they all had one view in common at the time of their death: the future looked bright for the development of groundbreaking drugs that had shown positive preliminary results in both animal and human clinical trials to treat their forms of cancer. And while my brother was successful in getting into one clinical trial, he was unceremoniously shut out of others.

This scene, regrettably, is being played out by terminally ill patients and their caregivers throughout this great country, despite the fact that the United States has one of the best healthcare systems, and some of the best pharmaceutical manufacturing and research facilities, in the world. For exam ple, recently reported on a family in Vermont where two young brothers have the same threatening disease. One child was fortunate enough to get into a clinical trial for a new drug; the other wasn’t. The condition of the boy in the trial appears to be improving, while his brother is getting worse.

Although some grass-roots efforts are under way on a state-by-state basis to pass legislation allowing “compassionate use” of unapproved drugs that show early promise, the bureaucracy and red tape involved make this an arduous and time-consuming task. Some say that giving a drug that has not been adequately tested by the FDA might offer false hope to a terminally ill patient. Still others are concerned it might create an outlet for selling medications on the black market that weren’t even submitted to the agency for approval.

For its part, the FDA says it is keenly aware of the situation and is not ignoring it. A Wall Street Journal blog posted last month explains that the FDA claims that nearly every request it gets for compassionate use of a drug is approved. According to the blog, FDA data show that “in the fiscal year ended last October, the agency approved 863 requests [for compassionate use of a new drug entity], or 99 percent of all cases reviewed.” The article reveals that the situation is further complicated by the fact that “the [FDA] only provides the pathway. If a company is not willing to entertain a request for compassionate use, then it doesn’t come to [the FDA].” A spokesperson for the agency reports that “most of the time we only know about the applications where companies are willing to make drugs available.” While I approve of the security blanket that drug approvals by the FDA provide to all American citizens, there has to be a more effective means of getting experimental drugs from pharmaceutical companies to terminally ill patients when the risk-benefit ratio tips in favor of the dying patient’s benefit.

To comment on this article, contact .