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

Abstract

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.

Hopes and concerns regarding the implementation of expanded access protocols in amyotrophic lateral sclerosis

“…This information needs to be openly shared with the candidate patient during the consenting process. It is comforting to learn that most medications in the EAP program described by Yerton et al were well tolerated and that no treatment‐related serious adverse events were reported 4 …”

“…This process of establishing and maintaining an EAP resembles that of a clinical trial. It is hardly surprising that running an expanded access program required over $600 per participant each month, despite waiver of fees by the authors' IRB, and not including the costs of the investigational product or of the time of the investigators 4 …”

“…It is hardly surprising that running an expanded access program required over $600 per participant each month, despite waiver of fees by the authors' IRB, and not including the costs of the investigational product or of the time of the investigators. 4 Once the IND application is filed, obtaining FDA approval for an EAP is not difficult. More than 99% of such requests were allowed to proceed.…”

“…It is comforting to learn that most medications in the EAP program described by Yerton et al were well tolerated and that no treatment-related serious adverse events were reported. 4 Notwithstanding the above issues, EAPs hold promise for ALS patients and their families by giving them access to more treatment alternatives. This also provides ALS patients with hope, which is essential in the fight against this devastating and progressive illness.…”

“…Patients in such situations often seek treatment options beyond FDA‐approved drugs. In this issue of Muscle and Nerve , Yerton et al report the experience of launching and maintaining multiple expanded access protocols (EAPs) in ALS since 2018 from a single tertiary ALS referral center in the United States, highlighting both the feasibility and challenges of developing and maintaining such programs 4 . This is a timely and valuable report as the interest in expanded access among ALS patients and clinicians has substantially risen following the congressional approval of the Accelerating Access to Critical Therapies for ALS (ACT for ALS) Act in December of 2021.…”