US Food and Drug Administration (FDA) Commissioner Scott Gottlieb and Center for Biologics Evaluation Research (CBER) Director Peter Marks on Wednesday warned that the agency will step up its enforcement efforts against companies illegally marketing stem cell therapies.
Gottlieb and Marks also said the agency will look into new ways to “delineate an efficient development path” for low-risk stem cell therapies being developed by firms that have filed investigational new drug applications (INDs) and are engaging in the regulatory process “in a responsible manner.”
In the US, thousands of clinics have flown under the radar for years offering unproven stem cell therapies to patients with little evidence of their safety or efficacy.
Part of the issue is the regulatory ambiguity surrounding exceptions to premarket approval for human cells, tissues and cellular and tissue-based products (HCT/Ps) that are regulated solely under Section 361 of the Public Health Service Act
for homologous use, minimal manipulation or use within the same surgical procedure.
In November 2017, FDA announced a new framework for regulating regenerative medicines
that included a 36-month period of enforcement discretion during which the agency said it would focus its enforcement efforts on treatments that raise the greatest safety concerns. The aim of the framework is to clarify what types of products and treatments are excepted from premarket review and to encourage firms to engage with FDA by filing investigational new drug (IND) applications and eventually seeking approval for products that do not meet the exemptions.
Despite these efforts, and other actions, including lawsuits
to dozens of firms
, many stem cell suppliers and clinics are resisting
the agency’s attempt to more closely regulate stem cell therapies.
Letters and Enforcement
The announcement Wednesday comes after FDA sent a warning letter
to Altamonte Springs, Fl-based firm Cord for Life last week for distributing human umbilical cord blood derived cellular products that do not qualify for any of the exceptions spelled out in 21 CFR 1271 that would exempt them from premarket approval.
In addition to not qualifying for these exemptions, FDA cited Cord for Life for six good manufacturing practice violations, including failing to establish and follow procedures to prevent microbiological contamination and failing to set up an adequate system for monitoring environmental conditions in an aseptic processing area.
FDA also says it has sent another 20 letters to other manufacturers and health care providers that offer unapproved stem cell products informing them of the agency’s compliance and enforcement policy for stem cell products.
“We’ve seen modest progress by the industry in coming into compliance, but more work needs to be done. There’s no room for companies that place patients at risk through products that violate the statutes and our regulations, including by not having in place an IND or biologics license,” Gottlieb and Marks said.
While Gottlieb and Marks said that FDA will continue inspecting
stem cell suppliers and clinics to ensure compliance, they emphasized that FDA “will not shy away from taking further steps when [it] sees bad actors taking advantage of patients.”
, Warning Letter