FDA, academia, and industry join forces to shepherd new therapies to patients

Imagine an artificial hand that can be controlled by a person’s thoughts and transmit the sensation of touch back into a person’s brain. This type of prosthetic hand already exists, but many of the amputees who could benefit from these new devices can’t access them.

The gap is due, in part, to the fact that the U.S. Food and Drug Administration (FDA) has yet to approve these artificial limbs for all amputees. Instead, these new prosthetics are restricted to a handful of patients each year, on an experimental basis. But an innovative collaboration among the FDA, Stanford University, and the UCSF Schools of Pharmacy and Medicine aims to bring transformative new medical therapies like these to all patients more quickly and to consider patients’ preferences in the FDA’s approval of new devices.

Researchers with the UCSF-Stanford Center of Excellence in Regulatory Science and Innovation (UCSF-Stanford CERSI) work with the FDA to provide the science that can help speed the FDA’s decision-making process. On January 12, CERSI researchers gathered at the UCSF Mission Bay campus with representatives from industry and the FDA, as part of CERSI’s first Innovations in Regulatory Science Summit, to brainstorm better ways to collaborate.

Whether patients know it or not, the FDA plays a huge role in their treatments and in their lives. The agency is responsible for the regulation of over 20,000 prescription drug products and 6,500 medical device product categories. In an era of unprecedented biomedical innovation, the FDA review and approval backlog is long, and the agency is welcoming new collaborations with academia and industry to address the issue.

CERSI

The Innovations in Regulatory Science conference welcomed attendees from UCSF-Stanford CERSI, industry, and the FDA.

UCSF-Stanford CERSI is one of four CERSIs nationwide, and the only one located on the West Coast. It was founded in 2014 to catalyze interactions in one of the most important hotspots for biotechnology development in the world—the San Francisco Bay Area. The Bay Area is considered the birthplace of the modern biotechnology industry, and it’s home to four top-tier research universities and nearly 300 biotechnology companies supporting over 300,000 full-time jobs.

“There are many new medical technologies waiting to be tested and approved, from digital health products to stem cell therapies,” said Kathy Giacomini, PhD, co-director of UCSF-Stanford CERSI and a faculty member in the Department of Bioengineering and Therapeutic Sciences, a joint department of the UCSF Schools of Pharmacy and Medicine.

UCSF-Stanford CERSI researchers are currently engaged in nearly 30 regulatory science projects. One project aims to improve the labeling of pediatric medications to reduce errors in drug dosing. Another project seeks to streamline the secure entry of health data for simultaneous use in a patient’s electronic health record and in a clinical trial.

UCSF’s Mission Bay Conference Center was down to standing room-only for the Innovations in Regulatory Science Summit. After introductory remarks from UCSF Chancellor Sam Hawgood, MBBS, and Stanford President Marc Tessier-Lavigne, PhD, talks moved to how to accelerate the required safety trials for medical products, how to address the new role of artificial intelligence in health, and why gene therapies are currently a better fit for rare, versus common, diseases.

FDA

Peter Marks, MD, PhD, is the director of the Center for Biologics Evaluation and Research at the U.S. Food and Drug Administration.

“If the FDA is going to continue its leadership in the science-based regulation of products, we need to be interacting with academic experts and continuing to work together to advance the applied scientific research necessary to understand these products,” said Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research (CBER) at the FDA.

Marks appeared on a panel discussing the new types of clinical trials that are being accepted by the FDA in support of product approvals. “As innovators, academic scientists have a unique perspective to contribute to [our regulatory] process and help bring safe and effective treatments to patients as quickly as possible,” he said.

For the attendees from startups and corporations, navigating regulatory science could mean saving millions or even billions of dollars. But for patients, it could mean that treatments and cures arrive sooner.

“We’re showcasing what regulatory science really is, what is needed for these products to be approved,” said Giacomini. “We are working with the FDA to bring new treatments to patients, but it’s time to bring the third player into the picture, and that’s industry itself.”