Drug Development and Approval: Part II

Orphan drugs

Drugs that are used to treat rare diseases affecting less than 200,000 Americans are referred to as orphan drugs. Since only a small number of patients need these drugs, it may not be possible for the drug’s sponsor to recover the drug development costs. Under the Orphan Drug Act, sponsors are given incentives to develop new drugs for the treatment of rare conditions, including several different types of cancer.

Making the drugs available to patients

Drugs that are being studied and have yet to receive FDA approval are called investigational drugs. Access to these drugs is available only to patients who have enrolled for clinical trials. The drug may receive FDA approval after the end of the clinical trial if there is evidence to prove that benefits of the drug outweigh its potential risks. Based on the awareness that patients, after having tried all available treatment options, may be willing to try new treatments with higher risks, the FDA has initiated the following three programs to ensure that patients with serious illnesses such as cancer can have access to investigational drugs.

Special exception/compassionate exemption: Although enrolling in a clinical trial is the best way to access investigational drugs, these trials often have eligibility criteria (strict rules) to determine if they will be appropriate for a patient. In case the trial is considered inappropriate for a patient, a special exception/compassionate exemption can be used to provide that patient with access to the investigational drug. An exception request should be submitted to the FDA jointly by the patient’s doctor and the sponsor of the drug. The FDA reviews every application on a standalone basis.

Treatment INDs: The treatment Investigational New Drug (treatment IND) program run by FDA allows drug sponsors to request that access to an investigational drug be provided to patients who are not considered appropriate for clinical trials or those for whom no other suitable treatment option may be available. There should be sufficient evidence to prove the drug’s effectiveness and also that it does not have unreasonable risks.

Group C drugs: An agreement between the National Cancer Institute (NCI) and the FDA, the Group C program seeks to provide oncologists with investigational drugs that normally are available via participation in an NCI protocol. Usually these drugs are in their phase III trials and there is evidence to prove their effectiveness in relation to a specific type of tumor. Using this route and after obtaining informed consent, patients who may not have participated in clinical trials can still have access to and benefit from a potentially effective treatment.