"If I am Not Eligible for a Trial, Can I Still Get Access to Experimental Drugs? "

There are some circumstances that may allow patients access to an experimental drug off of a trial.  Emergency Use IND of an experimental drug may be allowed if a patient does not meet eligibility criteria or a protocol does not exist.  In life-threatening cases where there is no standard acceptable alternative treatment available and when there is not time to submit an investigational new drug (IND) form, or IRB approval, the FDA may authorize a single use shipment of an experimental agent via telephone or fax.   This FDA emergency use provision is an exemption to the prospective FDA and IRB approval process.  Informed consent must still be obtained, and the IRB must be notified following emergency use of a drug.   Such examples would include snake venom antidotes or rare infection antibiotics, or rare life-threatening infectious exposures needing immediate vaccination.  Another example of an emergency use IND would be for a Jehova’s Witness patient who has life threatening bleeding and in need of access to experimental synthetic blood.    In general, cancer would rarely fall under emergency use exemptions, unless a complication from a cancer treatment required emergency access to an experimental drug.

            Off trial access to experimental cancer drugs may sometimes be allowed by four other mechanisms.  Expanded Access Programs (EAP) of study drugs may be offered by the pharmaceutical company as it finishes phase III testing before final FDA approval.  When no satisfactory alternative exists for a life-threatening or debilitating illness (such metastatic cancer), the FDA allows mechanisms to obtain investigational drugs through IRB approved process without compromising human safety.  In these cases, safety, survival and efficacy date may still be collected with an EAP. The largest EAP ever conducted was sponsored by Astra-Zeneca, which supplied over one million lung cancer patients with Iressa for free ($414 million dollars worth of drug) in 2002 before it became FDA approved in 2003.   Cancer EAPs existed in 2003 for myelodyspastic syndrome using 5 Azacytadine, and for mesothelioma using Alimta.   Also in 2003, metastatic colon cancer patients who exhausted all treatments and who are not eligible for a trial, could obtain monoclonal antibody erbitux as an EAP coordinated through the National Organization for Rare Disorders (NORD).

            Another avenue of access to investigational drugs are Treatment INDs or Parallel Tracks.  These protocols are for groups of patients (off of a research trial) and may be granted if IRB approved and preliminary safety trials have been completed and the pharmaceutical company is actively pursuing approval and willing to provide the drug for free.  Treatment INDs were largely the result of AIDS activists who positively influenced the FDA to expand access to new experimental drugs before final approval.   The NCI and FDA established an agreement to distribute investigational agents under protocols (but outside of controlled clinical trials) to oncologists for specific tumor types.  This Group C Treatment IND allows patient access to drugs distributed only by the NIH under NCI protocols.  Because Group C drugs are dispensed without research intent, the FDA generally waives the need for IRB approval.