FDA seeks input on rare disease clinical trials network

Regulatory NewsRegulatory News | 29 May 2020 |  By 

To further the establishment of a rare disease clinical trials network, the US Food and Drug Administration (FDA) is asking for input from the public and a broad array of stakeholders.
In a press release, the agency noted that it is “looking to provide a more cooperative approach” in supporting the drug development pipeline for rare diseases.
The call for information comes as the FDA is launching a Rare Disease Cures Accelerator. This accelerator will establish common platforms and approaches that can support rare disease characterization and the development of standard core clinical outcome assessments and endpoints pertaining to rare diseases, as well as provide support for conducting clinical trials in rare disease populations.

"For the approximately 7,000 known rare diseases, less than 10% have an FDA-approved treatment available. As drug development for rare diseases can be challenging due to the small number of patients and limited understanding of the variability and progression of the diseases, the FDA is committed to developing bold new approaches to harness the infrastructure of global clinical trial networks,” said Anand Shah, FDA Deputy Commissioner for Medical and Scientific Affairs. 

The accelerator efforts began in 2019 with a $10 million Congressional appropriation. Early initiatives focused on disease characterization and standardizing data measures; the FDA is now turning to the support of clinical trials.
The agency “is interested in understanding what work is currently being done and what work needs to be done to address the third component of its rare disease cure is accelerator,” said the FDA. “Because of the small size of rare disease populations and global occurrence of rare conditions, networks needed to support rare disease drug development would also have global reach and operations.”
Specific areas where FDA is seeking stakeholder input include knowing what the short- and long-term objectives should be for a global clinical trials network, as well as how such a network should be structured.
The right mix of disciplines for staffing, guidance for study protocol design and standard procedures for trial conduct and patient data protection all need to be determined, and FDA is asking for input in these areas. The agency is also asking stakeholders to weigh in on how disease-agnostic and disease-specific centers and services can each best be utilized; additional input is requested on how broad – or disease-specific – networks need to be.
Stakeholders who have experience with successful models of governance for global clinical trial networks are asked to share their experiences, and FDA is asking whether existing networks can complement the new rare diseases clinical network. Specifically, FDA refers to the National Institutes of Health’s Rare Diseases Clinical Research Network and Experimental Therapeutics Clinical Trials Network and the European Medicines Agency’s European Network of Paediatric Research, among others.

Other areas where the FDA is asking stakeholders to weigh in include infrastructure requirements, necessary funding for establishing and sustaining the network, key milestones and timelines for implementation, and challenges and barriers to standing up and sustaining a global rare disease clinical trials network.
Written and electronic input is due to the FDA by 30 July 2020.

This article was updated on 1 June 2020.


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Tags: FDA, rare diseases, US

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