- The US Food and Drug Administration (FDA) has, for the first time, qualified image-based biomarkers of bone mineral density (BMD) as a surrogate endpoint for bone fractures in clinical trials for osteoporosis medicines
- The qualification is the result of the large-scale global research collaboration co-led by experts from the University of Sheffield in partnership with Harvard Medical School, University of California, San Francisco
- Osteoporosis is a chronic condition that weakens bones and increases the risk of fractures, particularly of the hip and spine. These fractures can have devastating consequences, including chronic pain, disability, loss of independence and increased mortality
Millions of osteoporosis patients worldwide are set to benefit from a major international breakthrough which will transform how new treatments for the debilitating disease are developed.
The US Food and Drug Administration (FDA) has, for the first time, qualified image-based biomarkers of bone mineral density (BMD) as a surrogate endpoint for bone fractures in clinical trials for osteoporosis medicines. This landmark regulatory decision is expected to significantly shorten clinical trials and speed up patient access to new therapies.
The qualification is the result of the large-scale global research collaboration co-led by experts from the University of Sheffield in partnership with Harvard Medical School, University of California, San Francisco.
The University of Sheffield’s internationally recognised bone research expertise was instrumental in generating the robust evidence required for regulatory approval.
Osteoporosis is a chronic condition that weakens bones and increases the risk of fractures, particularly of the hip and spine. These fractures can have devastating consequences, including chronic pain, disability, loss of independence and increased mortality. The condition is estimated to affect up to 500 million people worldwide, yet it remains widely underdiagnosed and undertreated.
Traditionally, osteoporosis drug trials have relied on fracture outcomes, requiring very large patient populations and many years of follow-up. By confirming that changes in bone mineral density reliably predict fracture risk, the FDA’s decision allows future trials to use BMD as a surrogate endpoint. This will enable studies to be smaller, faster and more affordable, accelerating the development of innovative treatments.
The SABRE Project analysed data from 52 randomised clinical trials involving more than 160,000 patients, making it one of the most comprehensive datasets ever assembled in osteoporosis research. The work was delivered through extensive public–private collaboration coordinated by the Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium, with statistical guidance from the FDA under the 21st Century Cures Act.
Professor Richard Eastell, Professor of Bone Metabolism at the University of Sheffield, said: “This FDA qualification is a major advance for osteoporosis research and a powerful example of how collaborative science can deliver real-world impact. By helping to establish bone mineral density as a validated surrogate for fracture risk, this work will accelerate the development of new treatments and ultimately help prevent fractures that can profoundly affect patients’ lives.”
Experts say the decision is expected to stimulate renewed investment in osteoporosis drug development at a time when many patients lack effective treatment options. Faster access to new therapies could reduce fracture rates, improve quality of life for ageing populations and ease pressure on health and care systems worldwide.
The FDA’s decision was announced by the American Society for Bone and Mineral Research (ASBMR), marking a significant milestone for bone health research and reinforcing the University of Sheffield’s position as a global leader in musculoskeletal science.
