Limitations of randomized clinical trials for evaluating emerging operations: the case of lung volume reduction surgery

Ann Thorac Surg. 2001 Aug;72(2):649-57. doi: 10.1016/s0003-4975(01)02636-4.

Abstract

Although unanswered questions remain, scores of observational studies and several small randomized clinical trials (RCTs) indicate that lung volume reduction surgery (LVRS) offers safe and effective palliation for a relatively well defined subset of patients with advanced emphysema. Nonetheless, Medicare and other insurers stopped reimbursement for the procedure. Subsequently, two multicenter RCTs on LVRS, the National Emphysema Treatment Trial (NETT) and the Overholt-BlueCross Emphysema Surgery Trial (OBEST), were launched with the stipulation that the procedure would not be paid for outside these trials. Thus access to LVRS has been denied to patients who could benefit but do not wish to participate in an RCT. Emerging operations, unlike new drugs or devices, pass through evolutionary changes and frequently fail to produce data that meet the scientific rigor required by randomized studies. In such a setting, the observational approach is more appropriate. Indeed, almost all operations in the present surgical armamentarium have been evaluated and have evolved through observational studies without the use of RCTs. By the time new operations are standardized and qualify for RCTs, benefits for certain patients may be demonstrated and randomization could involve unacceptable health hazards. Patients from this population should be offered the choice between participating in RCTs and having the operation outside the study. Imposition of financial restrictions that bars access to a therapy with known benefit is a questionable practice.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Bias
  • Cost Control / legislation & jurisprudence
  • Humans
  • Pneumonectomy / economics*
  • Pulmonary Emphysema / economics*
  • Pulmonary Emphysema / surgery
  • Randomized Controlled Trials as Topic / statistics & numerical data*
  • Reimbursement Mechanisms / economics*
  • Treatment Outcome
  • United States