FDA Not Planning New RM Approval Pathway: Report

The latest Tufts Center for the Study of Drug Development Impact Report reviews the burgeoning regenerative medicine (RM) market worldwide and the differing regulatory approaches taken in the U.S., Europe, and Japan. The report (subscription required) says that FDA does not plan to establish a separate regulatory pathway for RM and will instead rely on current expediting mechanisms such as the Breakthrough Therapy Designation.

Europe’s Advanced Therapy Medicinal Products scheme is helping to create a viable pathway to market for cell and tissue therapies, it says, while Japan installed a RM regulatory scheme in 2014 that Tufts says could cut the trajectory for approvals from 7-10 years to three years.

The report says that RM therapies have a high potential to address a broad range of unmet medical needs and a strong product pipeline is paving the way for a global market that could reach $68 billion by 2020. Cell therapy products account for 76% of the 915 RM products in development in the U.S., Europe, and Japan, with gene therapy products accounting for the balance.

In the U.S., the researchers say, musculoskeletal-related conditions, led by osteoarthritis in people age 60 and older, account for $850 billion in annual direct and indirect healthcare costs.

A report chart lists 17 RM cell therapy approvals to date in the U.S., two in Europe, and four in Japan. One gene therapy product has been approved in both the U.S. and Europe, and another in Europe alone. And one tissue engineering product has been approved in Europe.

“RM development has been shaped by region-specific events,” Tufts says. “Although the U.S. Congress in 1995 banned federal funding for research on embryos and development of new stem cell lines, the country’s dominance of classical tissue engineering of bone, skin, and cartilage spawned the current cell therapy field. Japanese researchers created induced pluripotent stem cells, and became leaders in the field. Early setbacks in gene therapy caused research in this area to slow for several years in the U.S., opening the door for researchers in Britain, Italy, and France to make a number of advances. Despite divergent product development trajectories, the three regions share common challenges: limited funding due to uncertain commercial pathways; uncertain prospects for reimbursement and medical practice update; complexities in manufacturing and scale-up; and regulatory frameworks designed for conventional small-molecule drugs unsuited to new RM therapeutic paradigms.”