Single-Patient Expanded Access: WCG IRB Expands Dedication and Experience in 2020

Last year, WCG IRB received two-and-a-half times the number of single-patient expanded access (EA) requests compared to what it had received in each of the three prior years. Requests for COVID-19 therapies accounted for 67% of the total number of requests. No single-patient EA requests were submitted to WCG IRB which were not approved, and each was overseen seamlessly and fully in compliance with FDA regulations.

Of the requests received for indications other than COVID-19, 65% of the EA applications were for oncology or oncology-related indications. They included multiple applications for patients with breast cancer, prostate cancer, lung cancer, pancreatic adenocarcinoma, cholangiocarcinoma, ovarian cancer, osteosarcoma, and head and neck cancers. Applications were also received for single patients with carcinoid tumor, uterine cancer, acute leukemia and lymphoma, among others. 

The next most frequent condition for which single-patient EA was requested was mercury toxicity, for the provision of an investigational chelating agent, which accounted for 18% of the non-COVID-19 requests. The next most frequent therapeutic area in which we received EA requests was in infections and infectious diseases other than COVID-19, accounting for 9% of non-COVID-19 requests; specific indications included infections with acinetobacter baumannii, mycobacterium, HIV, and toxoplasmosis in pregnancy. The next most frequent indication was neurology, with 5% of non-COVID-19 requests for indications including ALS, epilepsy, Spondylometaphyseal dysplasia sedaghatian-type syndrome (with neurologic symptoms), and Lennox-Gastaut Syndrome. Requests were also received for investigational products to treat idiopathic pulmonary fibrosis, cold agglutinin disease, and hemophilia B among other indications.

In patients with COVID-19, most requests (55%) were for convalescent plasma, which were outside the large expanded access program being run by Mayo Clinic. Twenty-seven percent of requests were for access to remdesivir; which were received either before Gilead established the large-population expanded access program or were for patients who were not eligible for that program. Requests for products based on stem cells or amniotic fluid components accounted for 12% of COVID-19 submissions, and the remainder of the requests were for antibody-based or antiviral agents.

WCG IRB has, for many years, provided the review and oversight of single-patient expanded access requests, under which individuals can obtain investigational products to treat illnesses and conditions for which they have no other treatment options.¹ WCG IRB provides this review in accordance with existing FDA regulations for expanded access, and accepts the FDA form 3926 as the IRB application for review.  Submissions may be emergency requests, in which the investigational product was used in an urgent medical setting, and application is submitted to the IRB within 5 days after product use, or they may be non-emergency use in which the submission is made and approved by the IRB prior to product administration. Responses to the non-emergency applications are generally provided by WCG IRB within 24-48 hours after complete submission. 

WCG IRB remains committed to facilitating the single-patient EA process for patients who are using the FDA pathway to obtain investigational products for serious or life-threatening diseases. More information about this program can be found at https://www.wcgirb.com/services/expanded-access-compassionate-use/, under IRB Review Services.