Google the question “what is real-world evidence.” One of the first citations will be a link to the Food and Drug Administration (FDA) page defining both real-world evidence (RWE) and real-world data (RWD). The FDA asserts that RWE and RWD “are playing an increasing role in health care decisions.” In fact, the FDA uses RWE and RWD to monitor postmarketing safety and adverse events, as well as to assist in making regulatory decisions. Many contend that while RWE and RWD represent the “real world,” there still is a need for controlled clinical trials to first prove the efficacy and safety of a drug. Once efficacy and safety are proven, only then should RWE and RWD be considered. One feature article in this issue uses a case study to theoretically examine the benefits of RWE now and in future efforts in pathways design. The other feature presents a real-world analysis of retrospective data to better understand the feasibility and safety of outpatient administration of a drug.
Tumor lysis syndrome (TLS) is a group of metabolic abnormalities that can occur as a complication of cancer treatments, where a large number of cancer cells die within a short period, releasing their contents into the blood. It is potentially fatal if not caught early. Although consensus exists regarding best practices to manage most aspects of TLS, rasburicase, recently approved by the FDA for the prevention and treatment of TLS in patients being treated for leukemias and lymphomas, is much more expensive than conventional therapy, and it is unclear if it reduces the risk for the development of kidney complications, or even death. There is also a question of whether patients receiving rasburicase should be hospitalized vs being administered in an ambulatory setting. Scott C Howard, MD, MSc, and colleagues conducted a real-world retrospective cohort analysis to assess the baseline characteristics of patients with lymphoma and leukemia receiving rasburicase vs no rasburicase in the outpatient setting for the management of TLS-related hyperuricemia and to evaluate the impact of rasburicase use vs no rasburicase use on return hospital visits.
The majority of drugs being considered for and approved via accelerated FDA programs over the past few years have been cancer drugs. Although the agency has maintained that these accelerated approvals must pass vigorous regulatory muster, evidence suggests that post-approval studies are frequently not completed and, when they are, the results often do not replicate results as initially presented. The process of clinical pathways development in oncology is, at first glance, fairly straightforward: to assess all available published evidence in terms of efficacy, toxicity, and cost. But what happens when evidence is evolving too fast or is incomplete in the case of some drugs that received accelerated approval? Andrew Hertler, MD, FACP, and colleagues explore the current state of pathways development and maintenance, using the rapid advancements in treatment options for chronic lymphocytic leukemia as a case study. The broader implications of these insights are then discussed and collated for pathways developers for application in other disease states with similarly evolving treatment landscapes.
