Practicing oncologists typically rely on data from clinical trials as a source for making treatment-related decisions. However, new or rare oncology areas are often limited by the clinical trial data available, as agents for some of these indications can be approved on the basis of phase II trials, ie, an inherently smaller patient population. This makes real-world evidence a compelling option. Real-world data are increasingly being used to inform clinical decision-making, and new technologies are being developed to help research scientists extract, manipulate, and analyze data in real time and in real-world settings.
Oncologists in the United Kingdom are setting new standards for the use of creative health-related activities to generate real-world data and make them available for research without conceding patient confidentiality. Real-world evidence can improve patient outcomes, treatment pathways, reimbursement decisions, and clinical development. Let’s take a closer look at how oncologists are using (and gathering) real-world evidence, as well as when the use of real-world data is valuable and appropriate.
Beyond the Trial: Where Real-World Evidence Offers Value
While real-world evidence can’t replace data derived from clinical trials, it can complement or support the information disclosed in trial results. In this context, real-world evidence is especially appropriate for understanding disease progression and survival rates for time periods that extend beyond a trial, or to assess treatment efficacy in different patient subgroups or for niche indications.
Real-world evidence can often determine the value of a treatment, its clinical efficacy, or its cost-effectiveness—especially in the absence of clinical trial data or to further corroborate study findings. Real-world evidence is most widely applied in the appraisal of drugs for rare diseases. While today it’s mostly used as supportive evidence, applications of real-world data can provide useful information to better define standards of care and understand the burden associated with illness.
Real-world evidence can inform drug development by helping to identify unmet treatment needs in the population. Furthermore, access to real-world data is likely to accelerate the drug development process by providing valuable clinical insights into patient selection and budgetary impact, or informing health technology assessments. There’s also a place for real-world evidence in the postmarketing landscape, demonstrating the impact of a new treatment in a real-world context.
Real-World Data: Changing the Cancer Treatment Paradigm
Real-world data are compelling as supportive evidence, and they also offer the potential for a new paradigm in cancer treatment. For one, real-world evidence is important in addressing clinical uncertainties for treatments approved for specific disease-management schemes. Second, personalized care is a growing trend in cancer treatment, and more patients are actively collaborating in their own care regimen.
Real-world evidence could change decision-making practices when it comes to cancer care by addressing the expanding knowledge gaps that make up the vast oncology landscape or by complementing traditional data sources. By providing fuller information on the use and cost of cancer drugs, real-world data make it easier to anticipate the best approach to care as well as the treatment cost for an individual patient.
Real-world evidence can also guide treatment decisions for treatment combinations not supported by clinical trials. Certain forms of cancer yield efficacious results from the use of combined treatment modalities. However, clinical trials can’t keep up with the numerous drug combinations, and real-world data are especially important for discovering and promoting new indications for combination therapies.
Challenges of Real-World Evidence and the Future
The biggest challenge of integrating real-world data into oncology practice is accessing cancer phenotypes linked to genomic data. As more data are captured and processed by various healthcare registries, pooling various data sources is the next trick. With technology in our corner, it will become easier to access real-world data sets securely and while protecting patient privacy; but releasing real-world evidence today is still a delicate process that requires adherence to several ethical guidelines.
To ensure patient confidentiality, there is an alternate approach in the works: using a simulated data set that contains the same indicators and same data structure as the original set, but no actual patient data. The simulated set matches original measures in terms of age distribution, tumor stage, and treatment received, among other factors. While these simulated data tables are currently being validated, they will eventually be freely available.
The role of real-world evidence in clinical decision-making will expand in the future as it supports value-based pricing (a drug’s value will depend on whether the same drug can have clinical value in different tumors or be used in different combinations) and personalized medicine. Current challenges of real-world evidence relate to data quality, data infrastructure and access, and appropriate governance of data; the value of real-world evidence in the future of cancer care will involve the facilitation of clinical trials that generate robust, real-world data.