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Finding Common Ground on New FDA Regulatory Approach for Regenerative Cell Therapy

By Janet M. Marchibroda, Timothy Swope,

Thursday, June 9, 2016

As Congress continues its efforts to advance medical innovation, we have a tremendous opportunity to resolve differences and find common ground on ways to advance cell therapies—the next generation of groundbreaking treatments that show promise for patients in the areas of cardiology, neurology, oncology, ophthalmology, and orthopedics, while preserving the Food and Drug Administration (FDA) gold standard for safety and efficacy.

Despite the benefits of these treatments, few cell therapies are accessible to patients given the current regulatory framework which largely treats cells—even from one’s own body—as drugs, requiring up to $1 billion and 12 years of development time before a therapy can get to market. Both Europe and Japan have updated their regulatory processes to enable patient access to safe cell therapies. A significant amount of policy activity is now underway in the United States.

Last December, the Bipartisan Policy Center released a set of recommendations in its report, Advancing Regenerative Cellular Therapy: Medical Innovation for Healthier Americans, which proposed a new regulatory pathway tailored to the unique attributes of cell therapy. It was developed with the guidance of scientific and academic experts in the field. That same recommendation was also included in BPC’s July 2015 report, Advancing Medical Innovation for a Healthier America, which was co-chaired by former Senate Majority Leader Bill Frist, MD and former Rep. Bart Gordon.

Since the publication of that report, BPC has received considerable positive feedback and interest from stakeholders and policymakers, some of whom suggested that building upon and leveraging existing FDA expedited review programs for qualifying therapies would gain more widespread support than developing a new conditional approval pathway for cell therapy. In response to this feedback, BPC reconvened its expert panel again in April to explore and consider alternative approaches. We concluded that extending the existing FDA accelerated pathway to expressly include regenerative therapeutic products could serve as a considerable step forward in modernizing the U.S. regulatory approach and enabling patients in need to have access to such therapies, once determined to be safe and effective.

However, certain provisions would be required to support the unique and distinct attributes of human cells, tissues, or related products within FDA’s accelerated pathway program. As suggested in current FDA guidance, the treatment of a serious condition should be able to include a chronic, persistent, or recurring condition that affects day-to-day functioning without taking into account the availability or lack of alternative treatments.1 This is important given the unique nature of cell therapies. For example, injection of mesenchymal stem cells into an arthritic knee can reduce inflammation and reconstruct cartilage to improve function, yet device implants exist as alternative treatments to this therapy.

We also suggest that FDA include a written description of its rationale for decision-making to provide guidance to the field. All other requirements for accelerated pathway would remain the same, including use of surrogate or intermediate clinical endpoints and other aspects of the traditional development process including investigational new drug applications, controlled phased clinical research, and Phase IV trials. FDA would also continue to maintain full discretion in providing approval for only those therapies it deems safe and effective.

BPC has a long history of working with those that have differing viewpoints to find common ground on pragmatic, politically viable policy solutions that can make it over the finish line. We recognize compromise is needed to support action on a new regulatory approach on cell therapy. We call upon all interested parties to engage in respectful dialogue and reasoned debate on the best path forward. We all know that the status quo is not working for cell therapies. Let’s work together to change it.

A flexible regulatory approach that preserves the gold standard for safety and efficacy and also takes into account the unique aspects of cell therapies is needed to support patient access to these treatments that show great promise for those with Alzheimer’s disease, Parkinson’s disease, heart disease, diabetes, cancer, and stroke. Patients can’t wait. And we can’t let them down.

Read BPC’s Fact Sheet: Advancing a New Pathway for Regenerative Cell Therapy


Existing definitions of “serious conditions” (from FDA’s Guidance-Expedited Programs for Serious Conditions: Drugs and Biologics, or 21 CFR 312.300(b)(1)) already incorporate the concept of substantial impact on day-to-day functioning. “Available therapy” or existence of alternative treatments has also been defined in the context of unmet medical need to allow a new treatment if it provides efficacy comparable to those of available therapies while reducing risk and documenting benefit.

KEYWORDS: 114TH CONGRESS, 21ST CENTURY CURES, FOOD AND DRUG ADMINISTRATION