FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) are pleased to announce their partnership with the Critical Path Institute (C-Path) to initiate planning for a consortium aimed at developing tools appropriate for use in clinical development programs for alpha-1 antitrypsin deficiency (AATD), a rare disease causing progressive destruction of lung tissue leading to premature mortality or need for lung transplant.
C-Path is an independent, nonprofit, public-private partnership that aims to accelerate the pace and reduce the costs of medical product development through the creation of new data standards, measurement standards, and methods standards that aid in the scientific evaluation of the effectiveness and safety of new therapies.
There is currently no cure for AATD and current treatments do not adequately address the needs of patients. No new treatments have been developed for AATD over the last three decades, largely due to the lack of endpoints and other clinical development tools for studying treatments. There’s an urgent need for biomarkers in AATD that would predict long-term outcomes to use as surrogate endpoints for clinical trials of new therapies as well as a need for biomarkers that could be used to support efficient drug development, such as biomarkers for enrollment.
This collaborative pre-consortium will provide an opportunity for industry, regulatory agencies, academia, and the patient community to contribute to facilitating medical product development for the benefit of individuals living with AATD and their families.
FDA values input and feedback from knowledgeable stakeholders and is committed to working with advocacy groups, academics, industry, patients, and other partners to help identify solutions for the challenges in rare disease drug development.