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Do More with Less: Four RWE Approaches that Increase your Return on Investment
Sep 14, 2020
An increasing need for real world evidence (RWE), the associated rising cost of conducting clinical studies, and a demand for faster results, are driving forces for innovation. Providers, patients, and life science companies are finding new and better ways to capture real world data (RWD), and to leverage technology to generate deeper, broader, and more reliable insights in less time and at a lower cost.
However, to generate the most value from RWE, life science companies need to understand which RWE study designs are most appropriate and most fit-for-purpose, and how to ensure they deliver reliable, unbiased results. Discussed below are some of the most innovative, cost- effective solutions to generate robust RWE.
Pragmatic trials
Pragmatic trials evaluate the effectiveness, safety, and/or health-economic benefits of a new or existing treatment in routine care. They are conducted in real world care settings, such as clinics, hospitals, and health systems, and involve diverse, representative populations. They are the design of choice to evaluate effectiveness of treatments in routine care, particularly where there are differences in formulations, and/or frequencies of dosing and blinding or masking of treatment is not desirable.
One of the most appealing features of a pragmatic trial is that it achieves the objectivity of a clinical study by randomizing patients to treatment assignment. As a result, this study design offers a reliable and cost-effective way to demonstrate the real world impact of marketed products without the cost and burden of a traditional, randomized clinical trial (RCT).
In one example, the U.S. Food and Drug Administration (FDA) approved the inclusion of real-world data in product labeling for the antipsychotic INVEGA SUSTENNA based on pragmatic trial results. The study showed that patients who were randomized to an injectable, long-acting form of the drug experienced delayed time-to-treatment-failure compared to the most commonly prescribed daily oral treatments, resulting in significant health cost savings, reduced hospitalizations, and fewer long-term stays1.
Tips for pragmatic trials
- Verify that the clinical questions you ask resonate with clinicians, patients, and regulators.
- Ensure that there is clinical equipoise between the treatment arms.
- Expect to collaborate with multiple sites, academia, and/or delivery networks to achieve the scale and diversity of data needed.
- Consider offering investigator grants and patient incentives to drive interest and encourage engagement for the duration of the study.
External Comparator studies
External comparators captured from RWD help establish comparative context for clinical research. They are particularly valuable for single-arm trials, where control arms are not ethical or feasible due to rare or small patient populations, and/or the progressive nature of the disease. External comparators can also serve alongside single-arm treatment registries to provide context for post-approval safety studies.
In these studies, a cohort of patients are identified from RWD who mimic RCT eligibility criteria to add comparative context to clinical research. Health data may come from electronic medical records (EMR), patient registries, claims, or other sources.
The use of external comparator studies to support approval of single-arm trials has increased significantly in the U.S. In one example, Bavencio received accelerated approval from the FDA and the EMA for the treatment of metastatic Merkel cell carcinoma based on the pivotal results of the JAVELIN Merkel 200 study, which showed patients receiving Bavencio had a 33% response rate, in contrast to two different real world benchmarks showing a 10% response rate in a European registry and 29% from US oncology EMR data2.
Tips for external comparator studies
- Be sure eligibility criteria closely mirrors the RCT to avoid bias in study results.
- These studies are most appropriate when the effect size of the interventional arm is expected to be large compared to the external arm.
- Engage with regulators and other stakeholders prior to initiating the study to align on the best approach.
Innovative Extension studies
Innovative Extension studies are useful to measure longer term effectiveness and/or safety outcomes among patients who have participated in a clinical trial. The goal is to continue to observe patients after the completion of the clinical trial (or several trials) and capture long-term outcomes based on prior or ongoing exposure to the product(s). This approach can be used to simplify long-term treatment of trial patients before a drug is commercially available, monitor for long term patient benefits or risks, and reduce the cost and burden of long-term follow up studies.
Innovative Extension studies often use direct-to-patient data capture strategies to reduce or eliminate the need for physician and site involvement. This direct-to-patient approach can ease the burden on sites and investigators, and make the study less costly as a result of lower site burden.
Tips for extension studies
- Consider consolidating multiple protocols in a single extension study to save costs and simplify delivery of treatment to patients after the trials are complete.
- The primary endpoint of interest may influence the data collection approach. Using patient reported outcomes (PROs), virtual data collection, and secondary data can lower the cost of these studies and reduce burden on providers and/or patients.
- Try to start planning the extension study as early as possible to simplify execution and boost enrollment.
Enriched studies
Enriched studies use existing healthcare data coupled with selective prospective data collection on the same patient for robust, patient-centered insights into real-world experience.
Enriched studies integrate primary and secondary data at the patient level to build a comprehensive patient record. The primary data addresses the specific study objective and may be gathered through electronic case report forms (eCRF), PROs, and/or devices. Then, those insights are integrated with data gathered from electronic medical records (EMRs), claims reports, registries, and other secondary sources.
This is a useful model for meeting post-approval evidence needs because researchers can prospectively collect data that is not readily available from secondary sources, while leveraging available health data where possible. This approach increases study value by gathering clinically rich data on diverse subgroups with less site burden.
Tips for Enriched studies
- The enriched methodology may be appropriate when seeking efficiencies in data collection, reducing physician and/or patient burden, and providing data-driven long-term follow-up.
- Umbrella protocols may be considered to cover country-specific variability in the availability/suitability of secondary data.
- Ethics committee communications must address data linkage and may require additional submissions.
- Technological solutions may be required to enable data extraction and linkage.
A data-driven transformation
These novel study designs and data collection methods promise to transform the pharmaceutical research environment with patient-generated data. Going forward, we expect to see continued evolution of these RWE methods through the use of advanced technology and direct-to-patient data collection strategies. These cost effective solutions allow researchers to generate more comprehensive RWE in a timely manner.
IQVIA can help you analyze and understand the ever-expanding ecosystem of clinically-rich data in the context of your organization’s needs. By combining advanced analytics with unparalleled data and scientific expertise, IQVIA provides customers with innovative real world evidence approaches to meet stakeholder needs throughout the product lifecycle.
To learn more, contact us or listen to the IQVIA webinar, Innovative Approaches to Generate Real World Evidence, to learn more about this topic.
1 Alphs L, Benson C, Cheshire-Kinney K et al. J Clin Psychiatry 2015; 76(5): 554-561.
2 Becker JC, Lorenz E, Ugurel S, et al. Evaluation of real-world treatment outcomes in patients with distant metastatic Merkel cell carcinoma following second-line chemotherapy in Europe. Oncotarget 2017; 8(45): 79731 – 79741.
eBook: Explore Transformative RWD Sources
Discover how you can leverage RWE to accelerate study timelines, shorten time to market, cut research costs, and more. In this interactive eBook, read case examples on how key client questions were answered in various therapeutic areas and glean insights on how you can use RWD to answer challenging research questions.