There’s now a faster, more effective way to test new treatments for osteoporosis thanks in part to a decade-long effort led by Mary Bouxsein, PhD, a senior investigator in the Center for Advanced Orthopedic Studies at BIDMC, and Professor of Orthopedic Surgery at Harvard Medical School.

For the first time, the United States Food and Drug Administration (FDA) has approved bone mineral density, measured via non-invasive imaging, as a qualified endpoint in clinical trials for osteoporosis drugs. This means investigators can now use increases in bone density to demonstrate that a treatment is working, rather than waiting years to track whether patients experience fewer fractures.

“Having a valid imaging endpoint will allow smaller trials,” said Dr. Bouxsein. “It allows investigators to obtain results sooner, reducing both the cost and time of clinical trials. Ultimately, the goal is getting novel medications to patients sooner.”

The previous regulations requiring fractures as the key endpoint meant that studies had to include a large number of research participants and took a long time, Bouxsein explained.

The FDA ruling stems from the Study to Advance BMD as a Regulatory Endpoint (SABRE) initiative, an international collaboration co-led by Dr. Bouxsein and colleagues Dennis Black, PhD, of the University of California, San Francisco, and Richard Eastell, MD, of the University of Sheffield. Conducted under the umbrella of the Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium and the American Society for Bone and Mineral Research (ASBMR), the SABRE Project analyzed data from 52 clinical trials involving more than 160,000 participants, one of the largest efforts of its kind in osteoporosis research. Bouxsein first sketched the idea in 2008 and formally began the SABRE effort with Dr. Black in 2013.

Osteoporosis is common, as one in two women and one in four men over age 50 will suffer a fracture due to osteoporosis. Importantly, these fractures are associated with significant disability and increased mortality. Yet fewer than 30% of patients who experience an osteoporosis-related fracture are treated for the underlying disease within six months of their fracture. Dr. Bouxsein points out that while nearly all heart attack patients leave the hospital on medication to manage their cardiovascular disease, most people who sustain a fracture never receive follow-up care for their bone health.

“Underdiagnosis is also a major issue,” Dr. Bouxsein noted. “Fewer than half of women receive the bone density scans they should when they turn 65, to get a baseline measure of their bone health.”

The new ruling, she said, may help change that equation, by making it easier and more cost-effective to develop new medications and by attracting fresh investment to a field long seen as stagnant. Even ahead of the FDA’s decision, the osteoporosis space had already begun to see renewed attention from biopharma and venture capital groups anticipating the shift.

While current osteoporosis therapies are effective, they are not as widely adopted as they could be due to patient fears about very rare side effects and other inconveniences that can make compliance difficult. There’s now a real opportunity, Dr. Bouxsein explained, to develop drugs that are just as effective but more convenient for patients, potentially including oral medications that could replace injectables and help more people adhere to treatment.

“We’re excited. This is really paradigm shifting for our field,” Bouxsein said. “This work could not have been done without the phenomenal data sharing by pharmaceutical companies, highlighting the value of public-private partnerships. We hope that our effort provides a model for surrogate endpoint development in other therapeutic areas.”