Experts publicly debate on- and off-label use of BMPs at Medicare coverage meeting

Health and medical professionals comprising the Medicare Evidence Development & Coverage Advisory Committee of the Centers for Medicare & Medicaid Services met September 22 to consider the available evidence about using bone morphogenetic proteins clinically in on- and off-label applications.

The public meeting was convened so participants could discuss and vote on in a public forum the various issues related to use of bone morphogenetic proteins (BMPs) in the clinical setting and determine the applicability of the data to the Medicare population.

CMS officials noted the meeting was unrelated to any potential national coverage decision concerning BMPs.

PMA indications

Several areas were addressed during the 1-day meeting, which included two expert presentations, scheduled public comments, discussions and six questions that panelists voted on. The outcome of the voting showed panel members were most confident in the adequacy of available evidence for using BMPs on-label in lumbar spine fusion for the only FDA Premarket Approval (PMA) indication and their ability in that application to improve at least one outcome related to pain, patient function and adverse events.

Panelists were nearly as confident in the data available for using BMPs on-label for treating acute, open tibial fractures according to the FDA-approved PMA indication, based on results of the voting.

However, the panel, which consisted of individuals involved in orthopedic surgery, pain management, neurosurgery, neuroscience, nursing, health policy and economics, and other areas, found the evidence weakest for using BMPs off-label for uses other than lumbar and cervical spine, based on their votes.

Toward evidence-based use

Meeting chair Clifford Goodman, PhD, of CMS, said, “This meeting gives a pretty clear — and I hope very helpful — signal to researchers, practitioners, patients, innovative companies and others about the kinds of evidence that we hope to be thinking about putting into the pipeline.”

Goodman noted that as the need for bone growth and regeneration in spine fusions and nonunion and fracture treatments increases, “we will have better ways to address this very important clinical problem that are indeed evidence-based, that can inform patient and doctor decisions, can inform third-party payment decisions and can inform further research.”

During the first part of the meeting Julie Glowacki, PhD, professor of orthopedic surgery at Harvard Medical School and an orthopedic researcher at Brigham and Women’s Hospital in Boston, presented background information of how recombinant human bone morphogenetic proteins (rhBMPs) were developed for clinical use, including a discussion of how dosing and carriers affect their use.

Thomas Ratko, PhD, associate director, Blue Cross Blue Shield Association, Technology Evaluation Center, EPC, in Washington D.C., provided the CMS-commissioned technical assessment of BMPs in clinical use, entitled, “Bone Morphogenetic Protein: The State of the Evidence of On-Label and Off-Label Use,” which included an extensive literature review.

Orthopedic, spine groups represented

During the time allotted for scheduled public comments, representatives from various medical organizations discussed their members’ use of BMPs. William G. De Long Jr., MD, spoke on behalf of the American Academy of Orthopaedic Surgeons; Christopher M. Bono, MD, represented the North American Spine Society; and R. Patrick Jacob, MD, FACS, represented the American Association of Neurological Surgeons and Congress of Neurological Surgeons — each giving a 7-minute presentation.

Based on their votes, panelists indicated that the conclusions drawn from the BMP evidence currently available may not applicable to individuals older than 65 years. They were slightly more confident that such conclusions may be reflective of the community-based setting.

“I think one of the evidence gaps or limitations is the lack of generalizability of the data that we heard today to the real world practice in regard to the Medicare population and I think a way to address that evidence gap is to include large observational cohort studies and analyses of administrative claims data in future analyses to determine the true effectiveness of these procedures,” said panelist Kevin J. Bozic, MD, MBA, of the Department of Orthopaedic Surgery and Institute for Health Policy Studies, University of California, San Francisco.

Panelist Andrew Sloan, MD, FACS, a neurosurgeon at University Hospitals of Cleveland, discussed some gaps in understanding the BMPs’ mechanism of action. “I am really struck by how little we really seem to understand biologically” about BMPs, he said in his concluding comments.

In November 2006 CMS held a similar meeting during which a panel, called the Medicare Coverage Advisory Committee at the time, examined the evidence for using fusion to treat lumbar degenerative disc disease.

Reference:
www.cms.gov

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