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FDA Eager for Precision Medicine Under 21st Century Cures Act

The 21st Century Cures Act will give the FDA the tools, funding, and big data it needs to foster innovative, safe, and effective precision medicine therapies for patients.

- The FDA is ready and willing to get started on the precision medicine and drug innovation opportunities created by the 21st Century Cures Act, FDA Commissioner Robert M. Califf, MD, said in a blog post.

FDA Commissioner Robert Califf discusses 21st Century Cures Act and precision medicine

The legislation, signed into law by President Obama on December 13, reduces some administrative requirements for bringing new therapies to market pouring billions of dollars in research funding into the precision medicine community, both of which will help the FDA advance treatment options for cancer and rare diseases, Califf said.

“With Cures, great progress has been made towards our shared goal of advancing regulatory science so that we can continue to speed the discovery, development, and delivery of medical products to prevent and cure disease and improve health while sustaining the evidence framework that enables assurance to the public of the safety and effectiveness of medical products,” he wrote.

The wide-ranging package addresses many areas of the medical research landscape, including patient engagement in the drug development process, the design and execution of clinical trials, and the expansion of expedited pathways for testing and marketing new therapies.

“This new law rightly recognizes that patients should play an essential role in the development of drugs and devices to diagnose and treat their disease, since patients are in a unique position to provide essential insights about what it is like to live with and fight their disease,” said Califf.

The Act will require the FDA to develop industry guidelines about how to collect and use patient experience data during the research and development process, and also directs the FDA to include a statement of how patient experience data was used to inform drug approvals.

Patients may also have more opportunities to participate in streamlined clinical trials as the FDA seeks to make it easier for researchers to delve into rare diseases by sharing data more efficiently.

Modernizing the clinical trial process “has been an important FDA priority for decades, but exciting new approaches are now available, and we need to develop a common understanding of which designs should be used for which clinical issues,” Califf asserted.

“In cancer, for example, we’re already weighing the use of common control trials, which share a control arm, involve multiple different drugs for the same indication, and may even involve different companies.”

This strategy would reduce the number of patients required for research purposes, which could result in a shorter journey from trial design to drug approval. 

The FDA is looking to leverage the 21st Century Cures Act to expedite processes across many of its oversight areas, including applications for new medical devices.

“For devices, this past year was the first full year of operation for FDA’s expedited access pathway (EAP) program, which helps speed the development and availability of certain medical devices that demonstrate the potential to address unmet medical needs for life-threatening or irreversibly-debilitating diseases or conditions,” wrote Califf. “So far, we have granted 24 devices access to this program. Cures builds on EAP by creating the breakthrough device pathway.”

The law will also establish the Limited Population pathway, which will provide guidance and structure for the development of antibiotic and antifungal medications, both of which present unique patient safety challenges.

While the number and strength of drug-resistant “superbugs” is increasing, more antibiotics are not necessarily the answer.  Instead, pharmaceutical developers and providers must use powerful antibiotic and antifungal drugs more judiciously in order to prevent bacteria from evolving the ability to protect themselves from the medications.

The Limited Population pathway program will ensure that providers understand which antibiotics should be reserved for smaller patient populations with the most serious infections while encouraging the development of targeted antibiotics for these groups.

“The statement ‘Limited Population’ will appear prominently next to the drug’s name in labeling, which will provide notice to healthcare providers that the drug is indicated for use in a limited and specific population of patients,” the Commissioner explained.

“The limited population statement, additional labeling statements describing the data, and FDA review of promotional materials, will help assure these drugs are used narrowly to treat these serious and life-threatening infections while additional evidence is generated to assess safety and effectiveness for broader use.”

Califf also expressed excitement about funding for other areas of precision medicine study, including regenerative medicine and data-driven diagnostics.

The Cures Act provides the FDA and the National Institutes of Health (NIH) with funding to develop broad data sharing networks for research purposes, improve the use of real-world evidence in the development process, and retain more research scientists to continue making process towards truly personalized care.

Big data analytics skills, seamless interoperability, and data governance will be critical competencies to develop in order to connect the varied segments of the national research community.

“We are only at the early stage in building a national evidence generation system based on registries, claims data, and electronic health records that will be a rich source of post-market data and an avenue for conducting more efficient research,” Califf acknowledged, but praised the Cures Act for providing the tools to make continued progress.

“We are excited about the major advances in NIH funding, and welcome the increasing focus on rigorous translational science and data sharing reflected in the bill,” he said.

“FDA now stands ready to work with Congress, our sister federal agencies and the medical products ecosystem to implement these important provisions as we continue to work on behalf of all Americans to protect and promote public health and promote innovation in this exciting time.”


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