Although the most common way for patients to get access to an investigational new drug is through a clinical trial, there are other avenues that physicians and patients can pursue.
Certain investigational new drugs have expanded access protocols. These protocols typically apply to investigational drugs that have been studied extensively and are just waiting on FDA approval for marketing. The FDA process can be slow at times, and expanded access allows a greater number of patients to try the treatment. These treatments normally demonstrate success against certain types of cancers, meaning the evidence is there.
It is up to the investigational new drug application sponsor to apply and make their products available through expanded access. In general, the FDA only approves such an application when there is no other treatment already on the market that has demonstrated similar efficacy.
Special Exemption or Compassionate Exemption
Special exemption, or compassionate exemption, can apply to patients who do not otherwise meet the participation criteria for a clinical trial for an investigational new drug. To qualify for this, a patient’s physician must get in touch with the investigational drug’s sponsor company to provide the medical background and treatment history of the patient. Cases are considered on an individual basis, and the FDA must also approve the application.
In most cases, the sponsor company and FDA must jointly determine that the patient will either experience significantly prolonged survival prospects and an improved quality of life prior to granting compassionate exemption.
When determining whether or not a patient is a good candidate for investigational new drugs, the following questions should be considered:
- Is the patient a good candidate for a clinical trial?
- Has the patient been subjected to the traditional gamut of medications and therapies for their condition?
- Is the active agent in the investigational new drug application related to the disease in question?
- What are the risks of exposing the patient to investigational new drugs?
- Is there sufficient reason to believe that the patient would benefit from the investigational drug in question?
Unfortunately, even if the patient is an appropriate candidate for compassionate exemption on paper, the request can be denied if the drug is either in short supply or high demand.
For patients, families and physicians who want more information about investigational new drugs, the National Cancer Institute (NCI) maintains a database — the PDQ database — to track 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 also available to provide further information.
At TD2, we specialize in getting the best and most innovative oncology treatments to patients in need. Contact us for more information today.