Stakeholders Call for Regulatory Harmonization, Clarity in FDA’s Rare Disease Research Network
Stakeholders in the FDA’s proposed Rare Diseases Cures Accelerator are asking that the new rare disease clinical trials network focus on ways to improve recruiting, support high-quality data collection and enhance regulatory clarity for rare disease studies.
The FDA received more than 60 comments in response to its launch of the Accelerator, a research network designed by the agency to establish standardized platforms to characterize rare diseases, incorporate the patient’s perspective in outcome assessments and build clinical trial readiness for rare disease trials.
Weighing in on the governance structure of the network, NIH’s National Center for Advancing Translational Science (NCATS) suggested using its well-established Rare Diseases Clinical Research Network (RDRN) as a model. Operating since 2003, RDRN is a network of rare disease research consortia that focuses on clinical trial readiness, data sharing and patient involvement.
NCATS also recommended the network have a multi-level oversight structure, including a program director, program officers, grants specialists, data oversight boards, external advisers and leadership teams representing a broad selection of diseases, disciplines, patient populations and geographic areas.
According to a comment made by the National Organization for Rare Diseases (NORD), the network should also focus on increasing collaboration across organizations and improving the speed at which rare disease trials are successfully executed. The NORD argues that this may ultimately help break down silos currently pervasive in rare disease research. The NORD also noted that the FDA should first work on implementing the Accelerator and its infrastructure on a national scale prior to building it out globally.
The Pharmaceutical Research and Manufacturers of America (PhRMA) also weighed in, agreeing with others’ comments about recruitment and data collection issues. PhRMA’s senior director for science and regulatory advocacy Maria Apostolaros, added that “consistent and robust funding will be needed to attract representative experts to the team and maintain operational delivery.”