Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis.

Introduction/aims: Expanded access protocols (EAPs) allow individuals ineligible for clinical trials to receive investigational products. EAP data can be collected in parallel to randomized clinical trials (RCTs) and serve as a source of evidence in clinical practice. Here, we present the results of a National Institutes of Health (NIH)-funded EAP for amyotrophic lateral sclerosis (ALS).

Financing drug development via adaptive platform trials.

We propose a new approach to funding disease-specific drug development via a variation of the adaptive platform trial. This trial is designed to test a portfolio of drug candidates in parallel, with the cost of the trial partially covered by investors who receive payments from a royalty fund of the candidates in exchange for investment. Under realistic assumptions for cost, revenue, probability of success, drug sales, and royalty rates, investors may expect a return of 28%, but with a 22% probability of total loss.

Operational Development and Launch of an Adaptive Platform Trial in Amyotrophic Lateral Sclerosis: Processes and Learnings From the First Four Regimens of the HEALEY ALS Platform Trial.

Introduction/aims: Platform trials present several advantages over traditional interventional clinical trials. Here, we provide a detailed description of the operational framework of the HEALEY ALS Platform Trial.

Methods: Platform-level procedures for regulatory oversight, safety, and site management were developed prior to trial launch.

Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.

Introduction/aims: Expanded access (EA) is a Food and Drug Administration-regulated pathway to provide access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. The aim of this report is to share the design and operations of a multicenter, multidrug EA program for amyotrophic lateral sclerosis (ALS) across nine US centers.

Methods: A central coordination center was established to design and conduct the program.

Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis.

Platform trials allow efficient evaluation of multiple interventions for a specific disease. The HEALEY ALS Platform Trial is testing multiple investigational products in parallel and sequentially in persons with amyotrophic lateral sclerosis (ALS) with the goal of rapidly identifying novel treatments to slow disease progression. Platform trials have considerable operational and statistical efficiencies compared with typical randomized controlled trials due to their use of shared infrastructure and shared control data.

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

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.

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development.

Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner.

Adjusted cost analysis of video televisits for the care of people with amyotrophic lateral sclerosis.

Introduction: We previously reported our amyotrophic lateral sclerosis (ALS) video televisit experience. Here we report on video televisit versus in-clinic costs, adjusting for perceived medical usefulness (MU).

Methods: We take the patient-perspective and a focused institutional-perspective.

Developing multidisciplinary clinics for neuromuscular care and research.

Multidisciplinary care is considered the standard of care for both adult and pediatric neuromuscular disorders and has been associated with improved quality of life, resource utilization, and health outcomes. Multidisciplinary care is delivered in multidisciplinary clinics that coordinate care across multiple specialties by reducing travel burden and streamlining care. In addition, the multidisciplinary care setting facilitates the integration of clinical research, patient advocacy, and care innovation (e.