How Do Patients Get Access to Investigational Drugs?

Investigational drugs can provide an avenue for patients to try new oncology drugs prior to the treatments completing the FDA approval process. FDA approval is the last step in the drug development process, but once a drug has proven itself to meet a certain safety profile; it’s ready to be tested for safety and effectiveness in human patients.

Typically, patients who wish to gain access to investigational drugs do so through participation in an FDA-approved clinical trial. Physicians sometimes suggest this to patients who may not be responding well to their initial treatment regimen. In other instances, family members inquire about such opportunities. In either case, permission must be obtained from both the investigational drug’s sponsor company and the FDA before access can be granted for any patient.

Investigational drugs can provide hope for cancer patients. Many states, including Michigan, Arizona, Louisiana and Colorado, have passed or are considering “Right-to-try” legislation. Right-to-try legislation affords terminally ill patients to ability to try investigational new drugs that have made it through the first round of FDA approval without necessarily having to join a clinical trial.

In most cases, however, the patient must have the blessing of their physician before taking advantage of right-to-try. Physicians must directly request permission from the investigational drug’s sponsor company to use the unapproved drug on their patient.

Most examples of right-to-try legislation are based on the FDA’s “Compassionate Use Exemptions” for investigational drugs. Patients with the most to gain and the least to lose from trying investigational new drugs are typically the best candidates for compassionate use exemptions.

For patients, families and physicians seeking more information about investigational new drugs, the National Cancer Institute (NCI) maintains a database — the PDQ database — which tracks the status of ongoing studies. NCI's Cancer Trials website, along with information specialists at NCI's Cancer Information Service at 1-800-4-CANCER (1-800-422-6237), are available to provide more information about these opportunities.

TD2, an excellent drug development service specializing in getting the best and most innovative oncology treatments to patients in need, can also provide information.