What are “Real-world Data”?
“Real-world data” (RWD) refer to data collected in a real-life setting within a heterogeneous population. That is, not in a randomized controlled trial (RCT). In turn, analysis of these multi-source data provides real-world evidence (RWE) supporting the value of a product. This is especially important to confirm its long-term safety and efficacy outside of RCTs.
Why We Need Real-world Data
One needs to consider the inherent limitations of RCTs when interpreting the findings. Of note, selection bias is a limiting factor of most RCTs. Often, they only include patients within a specific age range, with limited comorbidities, and good performance status. This is obviously not representative of the real world. RWD can help identify subpopulations not included in RCTs that may benefit from a product. Further, the treatment adherence is usually higher in RCTs than in the real-world setting. This may negatively affect the treatment outcomes in the real world. However, collecting relevant RWD can help combat issues with suboptimal dosing or poor adherence.
Furthermore, RWD can help assess the associated costs and resource utilization of a new product. This includes costs related to adverse events, which may be underrepresented in RCTs. The product may also show enhanced performance in specific subpopulations compared to its competitors. However, this is difficult to assess in RCTs with homogeneous samples. For example, patients with certain biomarkers or symptoms may benefit more than others from a treatment. Accordingly, RWE is an important tool for working towards personalized medicine. This is another major benefit of RWD.
Other important uses include facilitating patient recruitment for RCTs and longitudinal studies. RWE is important for generating hypotheses and also plays a role in enabling healthcare quality improvements. Finally, it solves the ethical problem of including a placebo group in RCTs.
Importance of Real-world Data for Optimal Payer-positioning
Importantly, RWD are also essential for negotiating payer-positioning. That is, RWE helps with coverage and reimbursement decisions. In other words, RWE helps ensure a drug gets to the market at the right price. Specifically, RWE helps identify the exact risk-to-benefit ratio and demonstrate the economic value of the product. This includes how the product differs from its competitors in clinical or economic value. Thus, collecting RWD helps payers understand the benefit of the product in their target population. In turn, this supports market access decision-making. For example, data of interest may include the disease incidence and burden, unmet needs, and treatment patterns. By involving the payer in designing the analyses, you ensure RWE relevant to their target population. Further, identifying real-life limitations of any competitors promotes successful negotiations with the payers. Again, RWE is vital in this process.
Sources of Real-world Data
Ideally, RWD should come from multiple sources. These include patient registries, databases, surveys, and chart reviews. Further, observational cohort studies, claims data, and population-based surveys represent important tools. However, although using multiple sources ensures representative data, some challenges exist. Especially, inconsistencies may arise due to different study designs or collection methods. For RWE to reach its full potential, we need standardized guidelines regarding data collection and analysis. Until then, designing appropriate RWE studies and analyses is essential. Leveraging the expertise of the relevant stakeholders can greatly facilitate this process.
Role of Real-world Evidence in Improving Drug Access
Even after establishing a product, strategic use of RWD helps maintain or improve drug market access. Focusing on hard endpoints such as mortality or hospitalization rates is one strategy. Emphasizing its value in specific high-risk populations with unmet needs is another. Further, RWE of reduced resource utilization/cost and acceptable long-term safety is essential for payers to favor the drug.
Most questions regarding existing products can be addressed using available data sources, including those mentioned above. However, depending on the specific data required, these sources cannot always be leveraged. Proactively determining the best method for generating RWE can support maintained or improved drug access. Moreover, this also aids in differentiating the product from its competitors. Consequently, if used efficiently and promptly, RWE can help facilitate payer-positioning and improve drug access to patients.
Barriers to Real-World Evidence
Despite its many benefits, there are still barriers to collecting and using RWE. These include uncertain costs to stakeholders and concerns about privacy breaches.
Moreover, as RWE is becoming increasingly important, so is the need to gather data from multiple sources. For example, you need both database analyses and prospective data collection to achieve strong RWE related to the relevant market. Proactively developing a comprehensive strategy for collecting RWE serves multiple purposes. This helps prioritize data sources and identify suitable study designs to address the research question. Furthermore, using a solid strategy and methods supports the product’s value and market access.
Nevertheless, regulations or standardized guidelines defining appropriate data collection, integration, analysis, and use are lacking. Thus, determining the optimal approach to collect and analyze RWD can be challenging. To this end, stakeholder insights must be leveraged prior to, and throughout, the process. Luckily, in today’s increasingly digital world, there are cost- and time-effective means to achieve this.
Advisory boards comprising the relevant stakeholders are essential for leveraging expert insights. In the RWD setting, advisory boards serve multiple purposes. They can help establish appropriate collection or analysis methods. Moreover, they can be used to provide feedback on established protocols. The stakeholders may include payers, physicians, researchers, or patient support groups. However, the advisors are often living in different regions or countries. Hence, getting them to meet in person can be both difficult and expensive. One way to circumvent this is to conduct online advisory boards using virtual collaboration platforms that allow advisors to provide feedback when convenient, saving them the travel time.
On virtual advisory board platforms, advisors can co-develop or modify research protocols using asynchronous annotation tools. Furthermore, several digital advisory boards can be conducted over a specified period, each customized to build on the insights obtained in the previous. If needed, webinar-based or in-person meetings can be organized at critical points of the process. Using a managed, digital system also helps minimize the administrative burden by automating project elements such as email reminders and collation of responses into transcript reports.
The Future of Real-world Data
What is clear about RWE is that this is an exciting research area that will continue to rapidly expand in the near future. The clinical implications of RWE are potentially game-changing. Nonetheless, until clear guidelines exist, there will continue to be controversies surrounding its use. To overcome the current barriers, a lot of work is still needed. However, don’t let this dissuade you. Generation of appropriate RWE supporting your product’s value can greatly facilitate payer negotiations and improve revenue. Thus, the vast potential of RWD to improve drug access far outweighs these temporary obstacles.
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