Expanded Access following Clinical Trial or Open Label Extension (OLE)
Post-Trial Expanded Access - An FDA Statement
Transitioning patients from a clinical trial or Open Label Extension (OLE) study can pose opportunities and reduce burden to pharmaceutical companies.
The term "post-trial" access is the provision of investigational medications to a clinical trial participant after successfully completing an interventional clinical trial, both for Phase 2 or 3. A pharmaceutical company may consider this when the trial has met its objective, of collection of efficacy or safety information needed to support registration. It is often the case that a pivotal clinical trial is double blind, and patients requiring treatment for a prolonged interval beyond the end of the clinical trial are offered an open label extension (OLE) study. An OLE is a GCP-controlled clinical trial and is considered a 'covered' registration study. For a pharmaceutical company that means as many bells and whistles as a clinical trial, just simpler because patients are all receiving open label investigational medicinal product. Sure, is simpler than a clinical trial, but it is also a registration study. Pharmaceutical companies frequently face the dilemma of an OLE going on for several years, wanting to provide continued treatment to patients in need, and not knowing how to transition the program into something simpler that is not a registration trial. The key question to ask is 'do we need the data?' to fulfill a registration requirement. If the answer to that question is 'no', pharmaceutical companies need to consider the alternatives.
One solution is Expanded Access programs (EAPs). EAPs are not registration studies. These allow for the closure of the registration studies, enable timely submission of data, and continued treatment of patients.
Because of confusion and misunderstanding of EAPs, in March 2019 the FDA Commissioner Scott Gottlieb MD, Director of FDA's Center for Drug Evaluation and Research, Janet Woodcock MD, and Director of FDA's Center for Biologics Evaluation and Research, Peter Marks MD, issued a statement "Expanded access - Looking Forward". An excerpt from this statement is useful to put EAPs into perspective as a treatment option for Pharmaceutical companies: "..... 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." Further, ".... 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 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). As another option, the sponsor could authorize a patient's own physician to obtain a single patient EA investigational new drug (IND) application."
The FDA issued clarification around post-trial treatment options to remind pharmaceutical companies about options. This specifically addresses the pharmaceutical company dilema about what to do when ready to close an OLE.
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