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A Protocol for Comprehensive Assessment of Bulbar Dysfunction in Amyotrophic Lateral Sclerosis ALS
Published on: February 21, 2011
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Expanded access protocol (EAP) program for access to investigational products for amyotrophic lateral sclerosis
Megan Yerton1, Allison Winter1, Dario Gelevski1
1Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Muscle & Nerve
|March 17, 2023
Summary
Expanded access protocols (EAPs) provide a safe pathway for amyotrophic lateral sclerosis (ALS) patients ineligible for clinical trials to access investigational products (IPs). This single-center program demonstrated feasibility and safety, with no drug-related serious adverse events reported.
Area of Science:
- Neurology
- Clinical Pharmacology
- Regulatory Science
Background:
- Expanded Access Protocols (EAPs) are FDA-regulated pathways for investigational product (IP) access in serious diseases.
- Limited data exists on EAP utilization in amyotrophic lateral sclerosis (ALS).
Purpose of the Study:
- To report the design, operational aspects, and costs of a single-center EAP program for ALS patients.
- To evaluate the feasibility and safety of providing IP access through EAPs in ALS.
Main Methods:
- A single-center EAP program was established in 2018, adhering to FDA guidelines for individual or intermediate-sized protocols.
- Broad inclusion criteria were used, with safety information collected for all participants and biomarkers in selected EAPs.
- Data on protocol submissions, approvals, enrollment, and costs were collected from July 2018 to February 2022.
Main Results:
- 17 EAPs were submitted, with mean FDA and IRB approval times of 24 and 37 days, respectively.
- 164 participants were enrolled, with 77 still receiving IP as of February 2022; mean participation duration was 12.6 months.
- No drug-related serious adverse events were reported. Average site cost was $613.47 per participant per month (excluding IP costs).
Conclusions:
- EAPs offer a safe and feasible framework for providing investigational product access to ALS patients ineligible for clinical trials.
- Successful implementation and maintenance of EAP programs require dedicated site resources.
