If you are very ill or even dying, and existing cures aren't working—or no cure yet exists—chances are excellent that an experimental drug sounds more hopeful than scary to you. For many people, experimental drugs are really their only shot at living, even if they're long shots. This is the uneasy foundation upon which the partnership between drug companies and very sick people is built.
Why not join a clinical trial that's required by the FDA? Well, for one thing, clinical trials aren't possible for everyone. Almost all of them are conducted in cities in university hospitals, which means they're tough to access if you're from a rural area and/or too poor, too employed, or too sick to travel. Clinical trials are also off the table if you have other health issues of almost any kind, including mental health issues.
The Compassionate Use Exception
What's left is the compassionate use exception, also called expanded access and pre-approval access. This is an exception that allows patients to try experimental drugs outside clinical trials while they are still in development. However, it's not easy getting the compassionate use exception, in part because many doctors don't know how it works, and in part because drug companies can just say no.
In practice, the patient and doctor must select the drug in development they want to try. Next, the doctor requests the drug from the company developing it on behalf of the patient. If the company says yes, the doctor fills out a form for the FDA, who then reviews the proposed treatment plan. The last step is permission from whichever institution the doctor works in, because the drug is not approved for use.
If the company says no, no one, including the FDA and the courts, can force them to give you the drug. But if they do, social media or friendly journalists could be your saving grace; no one wants to be that company.
After the doctor gives the FDA the form, it gets reviewed—in emergency cases the review takes less than 24 hours, and in other cases, it's still only four days. According to the GAO, more than 99 percent of such requests are approved, although the FDA may make changes to the dose or schedule.
The last hurdle, institutional approval, is a mixed bag. The investigational review boards (IRBs) of research institutions and universities decide the ethics of these situations, but private hospitals don't typically have IRBs, which can add time to this step. How long this step takes varies significantly, as do outcomes.
A need for reform
The compassionate use exception isn't perfect. However, it's really not meant to be a frequently used option. The clinical trials system, on the other hand, is a mandatory process for all drugs that get to market. If this process was more open and access to clinical trials was granted to more people, the compassionate use exception might not be as important. Reform should include people with other health issues as well—after all, in the real world applications any drug faces, other health issues will present.
For now, an informed patient can take their shot at compassionate use. If you have to, good luck. In the meantime, the rest of us can advocate for improved access to clinical trials, and a reformed regulatory industry.