CAR-T therapies progressed to market at an unprecedented pace. Due to their accelerated FDA approvals, long-term data on how patients fare post-treatment isn’t readily available. As the number of patients commercially treated with CAR-T therapy grows, long-term outcomes data has the potential to influence the entire industry of personalized cell and gene therapies.
As with most therapies regulated by the FDA, both currently approved CAR-T therapies require the tracking and reporting of long-term patient outcomes, to truly measure the efficacy of these treatments throughout patients’ lives, post-therapeutic intervention.
The absence of long-term outcomes data for CAR-T therapies can largely be attributed to the diseases that they treat: rare, severe and advanced, with smaller patient populations. Because of this, recruiting for large controlled clinical trials has been a challenge. As the approved therapies continue to treat patients, the long-term data will be a valuable source of information on the effectiveness and safety.
But, tracking an ever-expanding group of patients for decades can be a demanding task for cell and gene therapy developers that may not have the infrastructure in place to monitor patients for up to 15 years. Outsourcing tracking to experts, therefore, can minimize room for error, as well as time and resource burden on companies focusing on bringing therapies to market.
In May, our research partner the CIBMTR (Center for International Blood and Marrow Transplant Research®) announced a collaboration with Kite, a Gilead company, to collect and analyze the long-term outcomes of patients treated with Kite’s Yescarta CAR-T therapy. With more than 45 years of collecting health outcomes data worldwide, tracking patient outcomes post-therapeutic intervention is no new task for the CIBMTR.
As the selected administrator of the SCTOD (Stem Cell Therapeutic Outcomes Database) contract for the HRSA-sponsored C.W. Bill Young Cell Transplantation Program, the CIBMTR collects, stores and analyzes all U.S. stem cell transplant outcomes in the secure database. Our global research collaboration has the infrastructure in place to track patient outcomes at any time points required by regulatory authorities, and has successfully done so for more than 475,000 patients, post-hematopoietic stem cell transplant.
“It’s a thrilling time for CAR-T and other adoptive cell transfer therapies, and we’re only at the beginning of tapping their potential to treat cancer,” said Mary Horowitz, M.D., Chief Scientific Director of the CIBMTR. “Long-term studies of patients treated with these groundbreaking therapies will provide important insights into how the drugs work and which patients are best positioned to benefit from them.”
Regulators are also interested in retrospective data stored in outcomes databases. In 2017 the FDA issued guidance on how the agency intends to use evidence in medical device approvals and post-market review, among other regulatory decisions.
As a growing number of pharmaceutical companies look to enter the cell and gene therapy space, such databases can ensure that innovative therapies are safe and effective, while also providing the retrospective data to expedite approvals, and ultimately, the delivery to patients who need them most.
Learn more about our outcomes data collection and analysis capabilities.