Matthew Gordon- Vice President of Real-World Evidence Strategy, Parexel
Over the past few years, the number of regulatory submissions that include real-world evidence (RWE) - insights derived from data collected outside of traditional clinical trials - has increased by more than 50%. This growth can be largely attributed to increased access to real-world data (RWD), from sources such as electronic medical records (EMR) and medical insurance claims as well as the influx of technology sources that enable RWD collection, such as wearables and sensors. Additionally, the role of the patient has increased too, from providing critical insights on quality of life to providing direct access to serving as a direct link to their healthcare data via patient-mediated sharing of healthcare data.
With the increased volume of real-world data comes increased application across the product development lifecycle. From incorporating an external control arm in a rare disease trial to evaluating the post-approval safety of a newly approved product or supporting payer reimbursement decisions, biopharmaceutical companies are increasingly using RWE to bolster their clinical understanding and more effectively and efficiently achieving product approval and reimbursement. However, access doesn’t always lead to appropriate application. To use this trove of insights effectively, sponsors must ask the right questions of potential clinical research organization (CRO) partners to ensure RWE solutions are designed appropriately and delivered with excellence from the start.
Real-World Evidence Has Many Applications That Require the Right Partner to Realize
Real-world evidence has become an umbrella term for a broad range of objectives across the product development lifecycle. A major development in the use of RWE started in the early 2000s when observational, non-interventional studies became a recognized approach to examine safety and respond to requirements from regulators. In many ways, this represented a natural extension of observational research, looking to patients to understand the performance and real-world safety effects of treatments, spawning new advances and acceptance of real-world evidence by multiple stakeholders today.
This shift created an environment where the RWE being collected was increasingly valued and accepted by regulators. It also enabled stakeholders across the healthcare system to embrace RWE through their respective lenses, supporting payer reimbursement decisions, post-approval safety, identifying patients to recruit into trials and helping sponsors understand what pre-clinical programs to invest in.
But in order to best realize these applications, sponsors must work with the right partners – ones that continually evaluate the structures, completeness and quality of the data – to find an optimal approach to meet their objectives.
It is Important for Sponsors to Understand Where and How a CRO is Obtaining its Data
When data is used in RWE studies, it is often not collected with the intent that it will be used in this way, ultimately causing gaps in the information companies are seeking. While some of this is starting to be rectified with our increasingly connected ecosystem of healthcare data, the challenge for companies to fill in the gaps that more traditional studies support remains. It is important for sponsors to evaluate the data structure, completeness and quality of the clinical research organization’s data to understand if the available data can answer the questions at hand.
The data landscape is becoming increasing complex, with some clinical research organizations having their own data and others leveraging external data sources. Curation from a variety of sources can help ensure that data collected is best able to answer the scientific question for which it is being analyzed. This structure is critical as no single dataset can answer all of a company’s questions, and a single dataset may not even be able to answer a single, specific question. Similarly, the application of artificial intelligence and machine learning is only as effective as the data it is applied to. Simply put, clinical research organizations cannot turn data into correct answers if the data does not exist or if it is misinterpreted.
By personalizing approaches for each sponsor, CROs can also eliminate blind spots. For example, at Parexel we have established specific teams dedicated to assessing and understanding clinical data, digital services, and appropriate statistical methodologies, allowing our team to engage the right expertise based on the type and objective use of the data. This allows us to customize study design based on the specific goals of each sponsor.
Further, it can be hard to answer certain questions because data is not collected in a way that is consistent with what is required to answer the sponsor’s specific question. Sponsors and other stakeholders should be clear on where the data is coming from, what data is being used as they conduct the study, and how the data that could support a decision was obtained.
Data Privacy is Critical to Ensure Patients are Protected
As CROs develop RWE studies with sponsors, considerations also must be made related to patient data and privacy, understanding the context in which the data has been collected and applying appropriate data analytic methodologies. While there are some protections in place in current systems, security around personal data must be consistently maintained, especially as patients demonstrate increasing awareness of how they can and should protect their personal health information.
Growing adoption of tokenization is enabling researchers to link data collected through traditional methods with real-world datasets, a key step toward protecting patient privacy while supporting more comprehensive study designs. The use of tokenization spans therapeutic areas and considers the goals of the clinical study, how CROs think about adding exogenous data that wouldn't typically be collected and the use cases that are of most interest to the sponsor that data can be used to solve. The ability to pull in this additional data while still ensuring patient privacy, allows for larger studies that expand the potential capabilities of real-world evidence.
While privacy guardrails can require additional time and cost, these challenges are necessary to ensure patient privacy and continued progress in our use of real-world evidence.
Establishing the Right Partner for an Optimal Approach
The use of RWE continues to grow, especially as traditional approaches to clinical research encounter more administrative hurdles and competition for sites and patients increase. And yet, there is no data panacea that answers every research question. For companies exploring a CRO partner, the right considerations must be taken to establish strong partnerships and customized strategies that deliver the insights required to address challenges and keep them on the path to success.
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