FDA Statement on Post Trial Treatment

Although we often focus on EA as providing access to patients who cannot enter clinical trials, EA can also be considered as a mechanism for those who have participated in a trial in order to allow them to continue receiving a drug that may have provided benefit. At the end of a trial, sponsors may continue to provide treatment to participating patients through an extension study to gather additional rigorous information that's needed to support the subsequent marketing application. Alternatively, if the purpose is primarily to provide the drug to patients who continue to need it, an EA program may be used for either moderately sized populations

(intermediate EA) or large size populations (treatment EA), often when most studies in support of approval have been completed. As another option, a sponsor could authorize a patient's own physician to obtain a single patient EA investigational new drug (IND) application. Once a trial is complete, EA is generally available when clinical trial results show that the drug is effective in the studied population. However, sometimes drugs that have not shown benefit across the overall study population may still be providing benefit for individual patients. In such situations, EA may have a role in allowing patients to have continued access to the drug. We would generally support such efforts. Providing EA to patients who undertook the risks that are inherent to participating in any clinical trial is an acknowledgement of the contribution these patients have made to the overall drug development program. Of course, in situations where an experimental treatment is potentially associated with substantial risk -- and where the therapy has not been demonstrated to be effective in a trial -- continued access must be carefully considered by the physician and the patient, and weighed by the sponsor, especially if further development of the drug is being reconsidered. As mentioned previously, we know there are other considerations. A decision about whether to allow EA is weighed against the potential impact on the development program; the primary goal of any drug development program must be to expeditiously bring the drug to the market, thereby providing broad access to patients who need the drug. Many novel therapies are being developed by small companies that may have only a single product. The supply of investigational product may only be sufficient to support the clinical research trials, and not be adequate to support EA. In addition, certain biologics, such as cell and gene therapies, are particularly complex and expensive to manufacture, so the cost to the company of providing such access may be prohibitive. We want to reassure sponsors that providing a drug under EA very rarely impacts development timelines. We urge sponsors to consider EA in appropriate settings; and especially after patients who are showing promise on an experimental drug complete therapy in a clinical trial setting.