The development of new therapies to treat orphan diseases is particularly perilous, due to limited patient populations, high costs, and difficulty in measuring clinical effectiveness. FDA officials are addressing these issues through the Rare Disease Cures Accelerator (RDCA) initiative in the Center for Drug Evaluation and Research (CDER). The goal is to shift away from a system of fragmentary research methods plagued by poor quality evidence to a more coordinated research approach and methods that can expedite development of drugs to treat some of the 7,000 rare diseases affecting 25,000-30,000 people.
Main strategies involve establishing common platforms and standardized tools and infrastructure to improve the quality of clinical trial data, explained Theresa Mullin, CDER acting director for strategic initiatives, at the “rare summit” sponsored by the National Organization for Rare Disorders (NORD) in Washington, D.C. last week. Mullin described how a more cooperative and collaborative approach can improve the quality of study endpoints, promote trial designs that accelerate learning, reduce mis-steps that cause failures and delays, and generate evidence needed for regulatory approval.
The RDCA provides the infrastructure for a cooperative approach to designing and conducting clinical trials in rare diseases to improve testing, protocols, and patient enrollment in clinical trials, Mullin noted. FDA is collaborating with the Critical Path Institute, NORD, patient groups, industry and other stakeholders on a framework for conducting more standardized rare disease natural history studies to help target treatment and design appropriate clinical trials. This involves examining the underlying causes of a disease, how it changes over time, variables in symptoms and experiences, and how the disease differs across patient populations. Patient perspectives remain key to providing insight into the most important impacts and what constitutes “meaningful change” in symptoms.
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