Over the past decade, progress has been made in planning and conducting clinical trials for rare disease drug development. In 2018, for the first time ever, a majority of new molecular entities approved by the FDA were orphan drugs to treat rare diseases. However, of the approximately 7,000 known rare diseases, less than 10% have an FDA-approved treatment available. Rare disease drug development continues to be challenged by the small numbers of patients and limited understanding of the variability and progression of each disease.
To support innovation and quality in the drug development pipeline for rare diseases, FDA established a “Rare Disease Cures Accelerator”. Through the Rare Disease Cures Accelerator, FDA aims to facilitate a cooperative approach and common standardized platforms to better characterize rare diseases, incorporate the patient’s perspective in clinical outcome assessment measures, and build clinical trial readiness in the pre-competitive space.
In September 2019, FDA launched a series of efforts to begin building capabilities in these areas, including:
- A data analytics platform.
The platform provides an integrated database and analytics hub designed to promote the secure sharing of existing patient-level data and encourage the standardization of new data collection. The aim is to receive and protect data from a variety of sources that can inform rare disease characterization, clinical trial design and other critical questions in rare disease drug development.
This data analytics platform provides a resource through which authorized users (e.g., disease researchers and drug developers) can access patient-level clinical data for a particular rare disease, which may be analyzed to better understand disease progression and the disease heterogeneity across the affected patient population. This in turn can inform trial design, selection of endpoints and other important considerations for drug development, avoiding mistakes that can cause delays or failures in the development of new drugs for rare diseases
The Rare Disease Cures Accelerator - Data Analytics Platform (RDCA-DAP) effort is being led by the Critical Path Institute through a cooperative grant agreement from the FDA. To learn more, please visit the Critical Path Institute website.
- Core sets of patient-focused measures
FDA’s pilot grant program to develop standard core sets of clinical outcome assessments (COAs) and endpoints for specific disease indications is a part of the Agency’s patient-focused drug development efforts.
Four awards under this grant program are for work on measures highly relevant to rare diseases. One award supports work to identify COAs and endpoints for use when developing acute pain therapeutics in infants and young children, primarily those 0-2 years. Another award supports work to develop and validate COAs with applicability across a range of chronic conditions that assess physical function using patient-reported and performance outcomes. Once developed, these core sets will be made publicly available at no cost or minimal cost.
The COA Grant Program is being led by CDER’s Patient-Focused Drug Development Program Staff. To learn more, please visit the CDER Pilot Grant Program: Standard Core Clinical Outcome Assessments (COAs) and their Related Endpoints website.