Expanded access protocol (EAP) program for access to investigational products for amyotrophic lateral sclerosis (ALS)

• Published in: Muscle & nerve 2023-06, Vol.67 (6), p.456-463
• Main Authors: Yerton, Megan, Winter, Allison, Gelevski, Dario, Addy, Grace, Kostov, Anthony, Lieberman, Cassandra, Weber, Harli, Doyle, Michael, Kane, Geli, Cohen, Caroline, Parikh, Neil, Burke, Katherine M., Rohrer, Margot, Stirrat, Taylor, Bruno, Margaret, Hochman, Alison, Luppino, Sarah, Scalia, Jennifer, D'Agostino, Derek, Sinani, Ervin, Yu, Hong, Drake, Kristin, Hagar, Jennifer, Sherman, Alexander V., Babu, Suma, Berry, James D., Cudkowicz, Merit E., Paganoni, Sabrina
• Format: Article
• Language: English
• Subjects: Amyotrophic lateral sclerosis; Amyotrophic Lateral Sclerosis - drug therapy; Biomarkers; Clinical trials; expanded access; FDA approval; Humans; investigational product; IP (Internet Protocol); motor neuron disease; Time Factors; United States; United States Food and Drug Administration
• ISSN: 0148-639X; 1097-4598
• DOI: 10.1002/mus.27819

Introduction/Aims Expanded access protocols (EAPs) are a Food and Drug Administration (FDA)‐regulated pathway for granting access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. There is limited information about the use of EAPs in amyotrophic lateral sclerosis (ALS); the aim of this report is to share the design, operational features, and costs of an EAP program for ALS. Methods The program was launched in 2018 at a single center. In alignment with FDA guidance, protocols were designed as individual (single participant) or intermediate size. Inclusion criteria were broad (e.g., no restrictions due to long disease duration or low vital capacity). Safety information was collected in all EAPs. Selected biomarkers were collected in nine of the EAPs. Results From July 2018 through February 2022, 17 EAPs were submitted for FDA and institutional review board (IRB) approval. The mean time from submission to approval from the FDA and IRB were 24 days and 37 days, respectively. A total of 164 participants were enrolled and, of these, 77 participants were still receiving IP as of February 2022. The mean duration of participation in an EAP was 12.6 mo. No drug‐related serious adverse events were reported from any of the EAPs. Average site cost was $613.47 per participant per month, not including IP costs. Conclusion EAPs provide a framework through which access to IP can be safely provided to people with ALS who do not qualify for clinical trials. Site resources are needed to launch and maintain these programs. See Editorial on pages 433–435 in this issue.