For more than 30 years, the FDA has supported patients’ access to investigational medical products for treatment, outside of participation in a clinical trial, when appropriate. The FDA remains deeply committed to this effort. Helping to facilitate access to promising medicines for patients with serious or immediately life-threatening diseases or conditions when no comparable or satisfactory alternative therapy options are available is a high priority for the agency.
We cannot know in advance whether a drug obtained through expanded access (EA) will provide a benefit for these patients who have no other FDA-approved treatment options and cannot enroll in a clinical trial. But such access can often represent a patient’s final hope for a potentially effective treatment. For the mother with breast cancer, it may lead to extra time to attend a daughter’s graduation or offer a father with ALS the chance to be at his son’s wedding. Maybe such access can lessen suffering in the last few months of a patient’s life. Or maybe it enables a child with a rare and fatal disease to live many more years. Fostering these opportunities are among our most critical obligations.
Since 2010, drug sponsors and manufacturers have given the opportunity of such access to more than 13,000 patients. The FDA applauds those companies. While pharmaceutical development is ultimately a business, health care is about people’s lives. Companies that innovate to develop effective new treatments show their commitment to the patients their drugs can potentially help by providing such access. The willingness of many sponsors to provide their products to patients at no cost or at the cost of manufacturing is a testament to their action on this public health commitment.
As addressed in the preamble to our 2009 regulations, use of investigational drugs in treatment settings generally involves careful balancing that considers (i) the interests of patients with serious or immediately life-threatening diseases, who need to be able to make decisions about their health care, including using experimental therapies; (ii) the need to protect potentially vulnerable patients from unacceptable risks; and (iii) society’s interest in ensuring that treatments for these diseases are developed and approved for marketing to enable broad access.
But sometimes, even in circumstances where the availability of a medicine through EA would appropriately balance these considerations, such access is not available. This is often true when it comes to the ability of patients to continue to use a promising medicine after the completion of a clinical trial.
We are writing to encourage sponsors to offer EA in such circumstances, when continued access to a promising medicine at the completion of a clinical trial would be appropriate under the EA programs.
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.