Knowledge of Efficacy of Treatments in Lung Cancer Is Not Enough, Their Clinical Effectiveness Should Also Be Known

The benefits established in efficacy trials, usually randomized, controlled trials conducted under highly controlled circumstances with maximized internal validity, can frequently not be demonstrated in clinical practice at the community level. Effectiveness trials are tools to evaluate the applicability of a treatment in a wider setting with maximized external validity, to observe uncommon adverse events, and to identify factors influencing the main outcomes and risks. Important areas in relation to lung cancer treatment that will benefit from effectiveness trials include gefitinib monotherapy and bevacizumab therapy combined with cytotoxic chemotherapy for advanced non-small cell lung cancer. These therapies were found to produce life-threatening nonhematologic toxicity at a high incidence of up to 5%; however, the risk factors for these toxicities have not yet been fully established. Effectiveness trials of adjuvant chemotherapy after surgery with long-term follow-up are also important to obtain reliable information as to secondary malignancy and noncancer-related deaths. Development of an infrastructure for effectiveness trials is crucial because of the necessity to deal with large numbers of patients, sometimes as many as 10,000 patients, from many hospitals. The extensive research time involved and the considerable cost of these trials may be reduced with the use of Internet resources. Effectiveness trials are a fundamental step toward bridging the gap between clinical research and clinical practice and effectively implementing new therapies in clinical practice.