Evaluating innovation. Part 2: Development in neurosurgery

Schnurman, Zane; Kondziolka, Douglas

doi:10.3171/2015.1.jns142664

Cited by 12 publications

(20 citation statements)

References 23 publications

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“…Compared with the prior study of development, 27 this is on the more rapid spectrum and lower range of articles, but is expected and agrees with previous findings given FDA approval 2 years after the first publication and significant RCT utilization.…”

Section: Off-label Innovationsupporting

confidence: 89%

“…27 Of those procedures, the mean number of initial studies prior to PSA was 13.7 (range 4-25) and the mean number of years to PSA was 9.1 (range 5-13). 27 Neither characteristic followed a normal distribution, but instead clustered into multiple groups depending on 2 key factors: use of RCTs and FDA approval.…”

Section: Acceptance Of Off-label Therapiesmentioning

confidence: 99%

“…27 This makes sense because FDA approval serves as a trustworthy endorsement of efficacy, and investigators may accept the procedure and move on to refinement. Similarly, studying a procedure with RCTs resulted in fewer total studies prior to acceptance.…”

Section: Acceptance Of Off-label Therapiesmentioning

confidence: 99%

“…Similarly, studying a procedure with RCTs resulted in fewer total studies prior to acceptance. 27 This is also sensible, as higher-quality evidence may lead to more confident belief in efficacy and acceptance, even if fewer total patients were investigated. * Percentage values are relative to the total number of initial studies for the given procedure.…”

Section: Acceptance Of Off-label Therapiesmentioning

confidence: 99%

“…This was seen in a previous study of vagus nerve stimulation for treatment-resistant depression. 27 After the largest RCT to date reported limited efficacy, the community appeared to disregard the result and continued refinement until the Center for Medicare and Medicaid Services issued a National Coverage Decision rejecting payment 2 years later, which resulted in refinement cessation. 27 On the other hand, the patterns seen with rhBMP-2 indicate that the response to reports of morbidity may be more robust.…”

Section: The Effect Of Technology-related Morbiditymentioning

confidence: 99%

See 4 more Smart Citations

Off-label innovation: characterization through a case study of rhBMP-2 for spinal fusion

Schnurman¹,

Smith²,

Kondziolka³

2016

SPI

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OBJECTIVE Off-label therapies are widely used in clinical practice by spinal surgeons. Some patients and practitioners have advocated for increased regulation of their use, and payers have increasingly questioned reimbursment for off-label therapies. In this study, the authors applied a model that quantifies publication data to analyze the developmental process from initial on-label use to off-label innovation, using as an example recombinant human bone morphogenetic protein 2 (rhBMP-2) because of its wide off-label use. METHODS As a case study of off-label innovation, the developmental patterns of rhBMP-2 from FDA-approved use for anterior lumbar interbody fusion to several of its off-label uses, including posterolateral lumbar fusion, anterior cervical discectomy and fusion, and posterior lumbar interbody fusion/transforaminal lumbar interbody fusion, were evaluated using the “progressive scholarly acceptance” (PSA) model. In this model, PSA is used as an end point indicating acceptance of a therapy or procedure by the relevant scientific community and is reached when the total number of peer-reviewed studies devoted to refinement or improvement of a therapy surpasses the total number assessing initial efficacy. Report characteristics, including the number of patients studied and study design, were assessed in addition to the time to and pattern of community acceptance, and results compared with previous developmental study findings. Disclosures and reported conflicts of interest for all articles were reviewed, and these data were also used in the analysis. RESULTS Publication data indicated that the acceptance of rhBMP-2 off-label therapies occurred more rapidly and with less evidence than previously studied on-label therapies. Additionally, the community appeared to respond more robustly (by rapidly changing publication patterns) to reports of adverse events than to new questions of efficacy. CONCLUSIONS The development of off-label therapies, including the influence of investigative methods, regulation, and changing perspectives, can be characterized on the basis of publication patterns. The approach and findings in this report could inform future off-label development of therapies and procedures as well as attempts to regulate off-label use.