On Monday, the Food and Drug Administration proposed expanding access to experimental treatments for patients with life-threatening diseases. Pharmaceutical companies and government agencies conduct thousands of studies every year to test drugs for commercial use. How can you get your hands on these new drugs?
First, get sick. To qualify for a clinical trial that determines whether a drug is safe and effective, you generally need to have the ailment in question. (Early phases of a drug study will ask for some healthy subjects.) Most patients find trials through their personal physicians, although you don't need your doctor's permission to enroll. (Find listings of clinical trials here.) You will need to meet the study's eligibility requirements, which specify age, gender, medical history, and stage of the disease. Studies often take eligible patients on a first-come, first-served basis. (If you don't get in, there's a chance the FDA will approve early access to the drugs under "compassionate use" provisions.)
Even if you enroll in a trial, you're not guaranteed a chance to try the new drug. In one phase of a clinical trial, subjects are randomly divided into two or three groups, only one of which actually receives the experimental treatment. The others serve as control groups and receive either previously approved treatments or placebos.
Either way, you won't have to pay for the treatment you receive. In trials for non-FDA-approved drugs—as opposed to studies looking at new uses for approved drugs—the sponsors must cover the cost. (There's a chance this will change—the FDA's proposed plan includes guidelines for when companies can charge patients a fee.) There may be hidden costs: If a study requires that a patient take another drug simultaneously with the experimental medication, the patient's insurance may have to cover it. Patients may also have to foot the bill for travel to the nearest trial site—these can be community hospitals, private doctors' offices, or specialized medical centers. Sometimes, pharmaceutical companies will help out with transportation expenses, either through direct payments or donations to third-party patient-support groups.
Your experimental drugs may not work at all—roughly 90 percent of drugs that begin human testing never make it to the market. They could even cause you harm. Before treatment can begin, you need to sign an "informed consent" document, which spells out potential side effects and makes sure you understand the risks. By signing it, you confirm that you're a volunteer and that no one is coercing you into taking the drugs. It also protects the study's sponsor from lawsuits. Well, not always: Four British trial patients received $25,000 in compensation payments from a biotech company this year after each suffered multiple organ failure and one of them had to have his fingers and toes amputated. They're currently suing Parexel, the company that conducted the trial.
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The Explainer thanks Maria Hardin of the National Organization for Rare Disorders.