Drugs

Rare Disease Clinical Trial Networks

David McFarland

— 2 min read
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The Biotechnology Innovation Organization (BIO) says it appreciates FDA efforts to address challenges associated with developing therapies for rare diseases that do not yet have treatments but, given the number of identified rare diseases, is concerned and has questions about the potential scope of a single rare disease clinical trial network. Responding to an FDA request for information, BIO says it may be more appropriate to consider the development of more than one rare disease clinical trial network. “It will also be important for FDA to clearly outline the scope and mission of the clinical trial network(s),” the comment letter says.

BIO suggests that FDA consider these immediate and long-term objectives/milestones for the rare disease clinical trial networks: 

  •          establish a steering committee including patients, caregivers, patient organizations, clinicians, drug developers, and other health authorities;
  •          engage stakeholders through a public meeting and comment process;
  •          analyze existing clinical trial network(s) and develop a scope and mission for the rare disease network(s);
  •          develop standards for the clinical trial network(s) including common data elements, mechanisms, and platforms for data/information sharing, structured institutional review board, data collection, and identification of potential third parties to manage and implement the network(s); and
  •          continue to engage stakeholders on lessons learned and opportunities for improving the clinical trial network(s).

In its letter, Pharmaceutical Research and Manufacturers of America (PhRMA) says it believes that global clinical trial networks for rare diseases “may help to facilitate and expedite rare disease drug development and allow for broader patient participation in clinical trials, both domestically and globally.” The trade group recommends that global health authorities such as the European Medicines Agency, Health Canada, and the Pharmaceuticals and Medical Devices Agency should be involved in establishing rare disease global clinical trial networks.

“While there are barriers to overcome for the implementation of rare disease clinical trial networks, the response to the Covid-19 pandemic and the new paradigms being utilized to address those needs can serve as a valuable lesson in the efforts to implement a rare disease clinical trial network,” PhRMA writes. “PhRMA believes FDA should examine the regulatory activities and modernized approaches that have been incorporated during this pandemic and seek to apply them more broadly where it may be beneficial and appropriate for rare disease drug development and regulatory decision-making.”

The National Organization for Rare Disorders says it has been a Rare Disease Cures Accelerator partner since its start and is uniquely positioned to provide to FDA insights on the startup, implementation, and maintenance of clinical trial networks for rare diseases.

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