Clinical Trials

Gaining Early Access to New Medications

Prior to their approval, new drugs being tested for their safety and effectiveness are called either “investigational” or “experimental” medications. In some cases, patients and their doctors may want to gain access to one or more of these types of medications before they’re officially approved by the Food and Drug Administration (FDA).

For example, a patient with a life-threatening disease who cannot be successfully treated with an existing FDA-approved drug may seek treatment with an experimental drug. Or, a patient experiencing severe side effects from an FDA-approved medication may be interested in switching to an investigational drug.

In circumstances such as these, patients can seek access to investigational drugs through one of two routes: clinical trials or the FDA’s expanded access program.

Clinical trials

Clinical trials are controlled studies of investigational drugs, and they’re a necessary part of every drug’s approval process. The main goal of clinical trials is to validate a drug’s safety and effectiveness, but they also provide information about a variety of other factors, including the drug’s associated side effects, correct dosages, contraindications (conditions or other drugs with which the investigational drug should not be mixed), and more. The results of clinical trials help the FDA decide whether to approve drugs and release them for public use.

Without patient participants, clinical trials would not be possible. And patients who participate are granted a number of benefits, perhaps most importantly, early access to potentially revolutionary new medications. By enrolling in a clinical trial, a patient gains access to a new medication not yet available to the public. He or she also plays a big role in advancing medical research and enjoys the very best standard of care.

Expanded access program

Because clinical trials often have very specific criteria that patients must meet in order to enroll (such as age and a lack of other health problems), not everyone is eligible to participate. To gain access to medications that haven’t yet been approved for public use, these patients may be eligible for the FDA’s expanded access program, which is sometimes called the “compassionate use” program.

Before a patient can gain access to an investigational medication through the expanded access program, all of the following factors must be established:

  • The patient’s disease or disorder may benefit from treatment with the drug.
  • The drug will not expose the patient to any unreasonable risks.
  • The patient has no other satisfactory treatment options (e.g., an FDA-approved drug or an existing clinical trial).
  • The manufacturer is willing to make the drug available for expanded access use.

If you’re interested in looking into clinical trials or the expanded access program, start by talking to your doctor or another member of your health care team to see if either might be a reasonable option for you.


Quick Access Programs

The FDA has a variety of programs that help speed the development, approval and availability of drugs that improve the current standard of care, treat serious or life-threatening diseases, and/or address an unmet medical need.

Priority Review Program: If a drug shows the potential to significantly improve the safety or effectiveness of the current standard of care, it may qualify for the FDA’s Priority Review Program. The FDA is required to review applications on drugs in the priority review program within six months, compared to the 10 months it has to review applications under the standard review program. Priority review does not affect the length of the clinical trial period, but it can shorten the amount of time it takes a drug to reach the market.

Accelerated Approval Program: While the standard review program requires absolute knowledge of a drug’s clinical benefit, the Accelerated Approval Program only requires early evidence that points toward the likelihood of the drug’s clinical benefit (called a surrogate or intermediate endpoint). For example, rather than waiting to see if a new cancer drug actually extends survival (clinical benefit), the FDA can approve it for that use based on evidence that the drug shrinks tumors (intermediate endpoint), since that effect is considered reasonably likely to lengthen survival. Using surrogate and intermediate endpoints can save a significant amount of time in the approval process for a qualifying drug.

Fast Track Program: The goal of this program is to get important new drugs to patients more quickly than the standard, priority and accelerated review programs allow. To be designated as “fast track,” a drug must show promise in treating a serious or life-threatening condition and have the potential to address an unmet medical need. Under the fast track program, drug companies can submit their approval applications in pieces, rather than waiting for all of the information to become available (called a rolling review). The FDA receives approximately 100 to 130 Fast Track applications per year and approves approximately 80 percent of them.

Breakthrough Therapy Program: This program is the newest of the quick access programs, and it targets drugs that not only treat a serious or life-threatening medical condition but also that, in early clinical trials, show a substantial improvement and/or clear advantage over currently available treatments. If a drug qualifies for the Breakthrough Therapy Program, the FDA will help guide the drug manufacturer through the most efficient drug development process, thereby helping the drug reach the market faster.



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