Survey Points to Challenges and Promise of Expanded Access Treatment
(Thursday, March 16, 2023) Over the last 3 decades, expanded access or emergency use treatments have become quite common and clinical experience from expanded use of investigational products has even been used to provide additional safety data to the FDA and enrich the real-world evidence (RWE), particularly for rare diseases. But use of expanded access protocols also raises concerns about the protection of patients, best practices for the physicians who ask for these, and the companies that provide patients with investigational products as treatments. A survey of oncologists conducted last year showed that expanded access (EA) is particularly common in cancer patients. Oncologists interviewed for the survey indicated their comfort with using investigational products on their patients, particularly the patients who had either exhausted the use of or were unable to be treated with available FDA-approved treatments. Oncologists consider EA “as an unexceptional means to obtain promising products”. The EA to an investigational drug is quite different from a patient participating in a clinical trial. Unlike a controlled clinical trial, the patient is guaranteed treatment with the investigational product in EA and expect benefits even if there are clearly told that the risks and benefits are unknown. Irrespective of whether a patient benefits from the investigational product, EA certainly provides important RWE for the risk of the product and may also yield useful benefit info. In an opinion article published this week in the Journal of American Medical Association, the authors content, and we agree, that the companies owing the investigational product being requested for EA should assure the following three steps: First, the expanded access program should be available to all eligible patients similarly. Companies do that by posting their policies for EA on their websites and have transparent review practices for EA requests. Second, EA should be done with IRB and FDA review. FDA has a greater than 99% record of approving emergency use INDs. Involving IRBs and FDA in the approval of the EA would yield independent assessment of the eligibility of a patient and the physician for the EA. Third, the clinicians and patients getting a product under EA should be encouraged to collect maximum possible data on the use of the product, akin to that collected in the clinical trials. Since this data would be useful for the manufacturer, they might need to provide resources to the clinicians for the additional documentation. Expanded access programs could be valuable to all parties involved, if done properly. AUTHOR
Dr. Mukesh Kumar Founder & CEO, FDAMap Email: mkumar@fdamap.com Linkedin: Mukesh Kumar, PhD, RAC Instagram: mukeshkumarrac Twitter: @FDA_MAP Youtube: MukeshKumarFDAMap |
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