The prospect of stem cell injections as a noninvasive treatment to improve patient outcomes is an appealing concept, one that has created significant buzz in recent years, attracting the attention of the national media and scientific community. The relatively novel treatment is predicated on the idea that it may be possible for stem cells to differentiate into cartilage cells and repair a previous areas of injury while also inducing an anti-inflammatory effect. However, there are currently no FDA-approved indications for therapeutic mesenchymal stem cell (MSC) injections. The use of MSC injections as regenerative therapy continues to grow as an area of acute interest for many clinicians and patients, despite a lack of high-quality evidence suggesting clinical efficacy.
Because the intervention is not FDA-approved, no major insurers will currently pay for the cost of the procedure. Patients who express interest in stem cell therapies are left with little alternative but to pay substantial out-of-pocket costs to receive these treatments within or outside of participation in clinical trials. Additionally, patients seeking MSC injection therapy may turn to alternative providers who are untrained in orthopaedic interventions and are misleading patients with unproven promises of efficacy. Alexander R. Vaccaro, MD, PhD, MBA, FAAOS, president of Rothman Orthopaedic Institute, and his team have taken these factors into consideration in developing a study protocol to responsibly address these issues and meaningfully add to the clinical body of evidence.
Clinicians at the Rothman Orthopaedic Institute developed a study protocol approved by the institutional review board which allows MSC injection therapy for a limited number of musculoskeletal conditions while collecting data on patients undergoing such treatment. Within this protocol, a subset of physicians who have an interest in the intervention and have undergone appropriate training have been authorized to offer bone marrow retrieval, MSC preparation, and MSC injection to interested and eligible patients.
Researchers reviewed the published literature to identify patient populations and conditions most likely to benefit from injection of MSCs. Both patient outcomes and the larger body of published evidence will be closely monitored and reevaluated, but, at study onset, the patients meeting the following criteria are eligible for inclusion: early hip osteoarthritis (Tonnis grade I, II, or III), avascular necrosis of the femoral head (Ficat grade I or II), early arthritis of the knee (Kellgren-Lawrence grade I, II, or III), degenerative disc disease, facet joint arthritis, or spinal cord injury. Patients are informed of the known risks; if consent is given, they are prospectively enrolled into the study and internal study registry.
Upon enrollment, patients undergo financial counseling to review all costs associated with the procedure. The total out-of-pocket expenses are estimated at $3,000. As part of the study design, Rothman Orthopaedic Institute attempts to minimize costs for patients, offering the intervention for one-half to one-third of the typical cost in the same geographic region, with significantly lower overhead going to the institution and treating clinician.
There are several reasons for this financial decision. The most salient, however, may be in consideration of the patient. “If it doesn’t work, this intervention is just a way of making money,” says Dr. Vaccaro. The organization made a conscious decision to not use an unproven intervention to simply generate revenue at the cost of the patient. In addition to this consideration, the typical high cost of the intervention can also create a financial barrier for inclusion, limiting the patient population to those with the means to pay for the intervention out of pocket.
A portion of the administration costs collected will automatically be allocated to fund future research. Another portion of the funds will be allocated to financially incentivize patients to report long-term outcomes, which improves the robustness and completeness of the data in the registry. Patients receive a $50 gift card per visit to complete patient-reported outcome (PRO) questionnaires at the six-, 12-, 26-, and 52-week marks. Because long-term PROs can be notoriously difficult to collect, it is proposed that this financial incentive will serve as a sustainable model for registry growth.
The creation and maintenance of an internal PRO study registry that tracks patients’ long-term outcomes post-
procedure are integral to the approach and long-term
success of this program. With enough data, researchers will be able to identify factors within patient populations and musculoskeletal conditions that are more likely to respond favorably to the intervention. It is anticipated that a large number of patients will be entered into the prospective database, allowing participating physicians to conduct peer-reviewed research and frequently publish findings.
By mitigating patients’ out-of-pocket costs, tracking patient progress with an internal registry, and incentivizing PROs, the Rothman Orthopaedic Institute seeks to responsibly contribute to the larger knowledge base. Striking a balance between responsibly providing patients with cutting-edge technologies and practicing evidence-based medicine will likely always be a challenge. However, some of the strategies employed in this study may serve as a blueprint to appropriately walk that line.
Ryan Pezold, MA, is a director within the AAOS Department of Clinical Quality and Value. He can be reached at email@example.com.