Expanded Access to New Medicines
Expanded Access is the Federally authorized use of un-marketed medicines for the purpose of treatment, prevention, or diagnosis of serious or life-threatening health conditions. Think of Expanded Access programs (EAPs) as special clinical trials for people who can’t get into regular clinical trials.
Jess Rabourn (AX-S Pharma) presents the four minute explainer on Expanded Access programs (EAPs)
The world’s first Expanded Access programs (EAPs) took place in the United States in the 1960s. In 1962 US Congress had required the FDA to tighten its restrictions on the transport and use of any new medicines in clinical development, creating a humanitarian need for exemptions for exploratory use in very ill patients who -for various possible reasons- could not participate in clinical research trials.
The 1960s through 1980s saw dozens of Expanded Access programs in tropical contagions, cancer, and heart disease, including two large programs sponsored by the National Institutes of Health. In 1987, FDA promulgated the first written regulations for Expanded Access programs, which were defined as “treatment use” clinical trials for patients and physicians who could not part in research clinical trials.
Today, regulators in most countries allow some form of Expanded Access to unlicensed medicines – either for truly investigational products, or -in some countries- just products that have been approved in one or more other countries but not yet in their own country.
Classic Expanded Access programs (EAPs) are structured like research trials, but with more-inclusive eligibility criteria. Like research trials, they have a single uniform protocol and centrally monitored safety data collection. An EAP can enroll patients at just one site or at many sites. They can be very large; for example, the Iressa (gefitinib) EAP in non-small-cell lung cancer and the Videx (didanosine) EAP in AIDS enrolled more than 20,000 patients each.
The regulation of Expanded Access is not widely understood in the life science and healthcare industries. Its place in the clinical development of new medicines has been a topic of confusion for many.
Ax-S Pharma was created to eliminate the confusion. Through the industry-leading Expanded Access Summit, our custom workshops, on-demand explainers, and now the Ax-S Thursdays, e-Conference Series on Expanding Access to New Medicines, we provide a definitive hub for information on early access to new medicines.