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

Although unanswered questions remain, scores of observational studies and s everal small randomized clinical trials (RCTs) indicate that lung volume re duction surgery (LVRS) offers safe and effective palliation for a relativel y well defined subset of patients with advanced emphysema. Nonetheless, Med icare and other insurers stopped reimbursement for the procedure. Subsequen tly, two multicenter RCTs on LVRS, the National Emphysema Treatment Trial ( NETT) and the Overholt-BlueCross Emphysema Surgery Trial (OBEST), were laun ched 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 ben efit 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 stud ies. In such a setting, the observational approach is more appropriate. Ind eed, 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, be nefits for certain patients may be demonstrated and randomization could inv olve unacceptable health hazards. Patients from this population should be o ffered the choice between participating in RCTs and having the operation ou tside the study. Imposition of financial restrictions that bars access to a therapy with known benefit is a questionable practice. (C) 2001 by The Soc iety of Thoracic Surgeons.