Drugs

CDER’s Cavazzoni Spearheading Rare Disease Program

David McFarland

— 3 min read
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CDER director Patrizia Cavazzoni is personally spearheading CDER’s new rare disease efforts under its recently announced Accelerating Rare disease Cures (ARC) program, which is designed to speed and increase the development of effective and safe treatment options addressing the unmet needs of patients with rare diseases. Delivering a Center update at the Food and Drug Law Institute’s (FDLI) annual meeting 6/15 in Washington, DC, Canazzoni said she is leading the program directly with a number of senior CDER officials to put a lot of focus on these activities, and to make sure that any barriers that emerge are addressed in a timely fashion.

 

Cavazzoni noted that about half of all drug approvals since 2015 have been for rare diseases, and since the Orphan Drug Act passed in 1983 the agency has approved over 600 of them. Currently, there are about 7,000 rare diseases, and the vast majority do not have approved treatments.

 

An online announcement last month says developing drugs for rare diseases and conditions can be complex due to:

  • the challenges in using well-established trial designs;
  • the complexity of endpoint selection if there is a limited understanding of the disease’s natural history; and
  • the difficulty in performing and interpreting rare disease clinical trials with small patient populations.

In its first year, CDER says, ARC will focus on strengthening internal and external partnerships and will engage with external experts to help identify solutions for the challenges in rare disease drug development. “The basis for this program is really to turbocharge the work that we have been doing in CDER and across FDA,” Cavazzoni said. CDER will create an umbrella and framework that will “allow us to create the connective tissue across all the activities and provide additional resources to certain activities, and develop new teams that might address gap areas that we have seen, such as translational sciences,” she said.

 

Another area that Cavazzoni says she is excited about is the modernization of clinical trials through decentralized trials. She said it is one of the few bright spots brought on by the Covid-19 pandemic and decentralized trials will continue post-pandemic. “We have seen an uptick on the utilization of decentralized clinical trials for obvious reasons during the lockdowns, social distancing, and overwhelmed hospitals.” The clinical trial community rapidly embraced decentralized clinical trial tools with digital health technologies (e.g., sensors and software applications) helping to facilitate the conduct of decentralized clinical trials. “Going forward, we are working on guidance on for decentralized clinical trials because we see applications beyond the pandemic,” she said. “Obviously, we’ll certainly have some challenges, and we'll have to work with sponsors to make sure that there is clarity about what to do or not to do, but we certainly see this as one of the good outcomes of the pandemic and … the experience has led the world to take the plunge into decentralized clinical trials, and remote and centralized monitoring.”

 

Earlier this year, FDA published a draft guidance, Digital Health Technologies for Remote Data Acquisition in Clinical Investigations, with recommendations for sponsors, investigators, and other interested parties on the use of digital health technologies for remote data acquisition from participants in clinical investigations evaluating medical products. The document defines a digital health technology as a system that uses computing platforms, connectivity, software, and/or sensors for healthcare and related uses.The guidance outlines recommendations intended to facilitate the use of digital health technologies in a clinical investigation as appropriate for the evaluation of medical products.

 

Additionally, Cavazzone highlighted CDER’s planned work to modernize post-market safety “and we are starting by defining a roadmap for what safety post market safety will be in three years.” She said the elements being looked at include the regulatory framework, and leveraging the use of existing and emerging technologies, such as robotics and artificial intelligence and how those could be applied to adverse event reporting and processing. Cavazzoni said CDER is reorganizing postmarketing surveillance along the lines of the guiding principles used in the modernization of the new drug review program, which was anchored on cross-discipline work and developing integrated assessments of the application. Safety reviewers will use a standardized assessment, “instead of just throwing memos at each other,” Cavazzoni said. She also said CDER has begun setting up multidisciplinary drug safety teams, which will work together to identify new signals, validate the potential risks, and then determine how to mitigate them. [Editor’s note: click here for more details on modernizing post market safety.] 

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