The Rare Disease Cures Accelerator- Data and Analytics Platform: Value for Drug Development in Neuromuscular Diseases


Real World Data - Disease registries, natural history, post marketing surveillance

Poster Number: 157


Jane Larkindale, Dphil, Vanessa Boulanger, Pamela Gavin, Richard Liwski, Klaus Romero, Michelle Campbell


1. , 2. NORD, 3. NORD, 4. Critical Path Instititute, 5. Critical Path Institute, 6. FDA, CDER

The Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP) is an integrated database and analytics hub designed to help build tools to accelerate drug development across rare diseases. It is being developed by the Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD) through a collaborative grant from FDA. The RDCA-DAP promotes sharing of patient-level data and encourages standardization of collection of new data, as well as aiding in analysis. As clinical trials have been performed in many neuromuscular diseases (NMDs) and there are high quality natural history studies, NMDs are ideal for integration into this platform, in terms of both needs of current drug development and existence of high-quality data that can inform future studies. C-Path’s work in Duchenne Muscular Dystrophy and Friedreich’s Ataxia integrated over 1,000 patients’ data exemplifying this opportunity. NORD’s network of patient groups and natural history studies, including for Spinal Muscular Atrophy with respiratory distress type 1 and Guillain-Barré syndrome, adds an important opportunity to incorporate an untapped source of data.

Despite many drug targets and technologies, NMD drug development is frequently slowed by the low numbers of patients and the lack of comprehensive quantitative characterization of diseases. This means that design of clinical trials that can reliably evaluate the efficacy and safety of a potential therapy is challenging. Developing a clear understanding of how each disease progresses, as measured by defined outcome measures and/or biomarkers, will allow development of clinical trial protocols that efficiently determine if a new therapeutic is effective or not. This would accelerate clinical development, make it less expensive, and encourage new companies to develop rare disease drugs. The RDCA-DAP launched in September 2019, and several pilots are underway to demonstrate the utility of the platform.