RWE: Opportunity, Challenges and Sourcing Simplified

What is RWE?

As a relatively new means of conducting research in the life sciences, RWE, or real-world evidence, is data that comes from “real world settings” such as a clinical practice. Studies driven by real world evidence, or real-world data (RWD), can utilize data from medical records or claims databases,¹ be conducted prospectively in observational studies or adhere to a mixed, hybrid-design approach.² Thus, RWE does not originate from the tightly controlled environment of clinical trials; instead, RWE replicates the same behaviors one would see in a real-world setting or typical clinical practice. In fact, one could say RWE provides essential insights on a range of outcomes that are representative of an everyday clinical setting, while randomized clinical trials provide efficacy and safety and are crucial for product registration.

In the context of drug development, the Phase 1-3 clinical trial phase is where researchers study whether or not a drug is safe, effective and does what it is intended to do – make people better.²

As the clinical trials progress, more patients are added in each phase, more evidence is collected to prove the drug is safe and effective, then the clinical trial data is presented to a regulatory agency and hopefully receives a stamp of approval to be placed on the market. Once this occurs, doctors start prescribing the drug to their patients, which is where the bulk of RWE is produced, as the data on the new drug is recorded in medical and claims records as well as through patient experience surveys. RWE studies are then conducted to answer a variety of questions that were not answered in the clinical trial setting: How do patients actually adhere to the drug regimen? Is the drug prescribed in other indications? What is the real cost of the product to the patient and healthcare system? RWD can also be used to improve future clinical trials by informing researchers of the populations most likely to benefit from product regimen or to identify additional outcomes to assess.

How RWE Works

RWE demonstrates how a product performs in the real world among the general population while taking into consideration factors that could influence the outcome of a disease (e.g. co-morbidities, human behavior, provider treatment patterns). On the other hand, data from clinical trials shows how a product performs under a tightly controlled and closely monitored setting that provides or even favors the best environment for the product to perform successfully and eventually reach the market. RWE studies dictate whether a product is still safe and effective once it does in fact hit the market — a real world setting — and is affected by the aforementioned factors that can influence the natural course of disease and product performance.² While there is much value to be gained from RWE, there are challenges with analyzing this type of data.

If a researcher is not careful to take certain considerations into account when designing a study, confounders are more likely to be introduced and influence the results.³ Within the general population, people sometimes have more than one disease, so they are prescribed different medications that could interact with each other. Smoking, age and weight are all elements that can potentially affect the outcome. However, this scenario is a more accurate representation of the general population and provides more information on outcomes of the product when these factors are introduced.

With all that being said, clinical trials have been and are still considered the gold standard within research, which is why historically RWE had not received much attention in years past.³ Clinical trials have the distinct benefits of effectively randomizing the product among different treatment groups, minimizing confounders and maintaining causality in studies. However, people are also realizing the benefits of RWE in helping to truly understand the most accurate outcomes of their product. And because of this, regulatory agencies are increasingly asking for more data from RWE studies.

Challenges Associated with RWE

Despite the recent interest in RWE, it does not come without challenges. Firstly, an RWE study must include real world data gleaned from hospital records, patient registries, claims databases or collected prospectively from the clinician or patient. This information is traditionally not easily accessible, as most all countries tightly regulate access to medical records and patient data.³ Not to mention, data quality is an issue because medical records are not designed for research; their main purpose is for a clinical practice to treat patients.³ Another challenge is finding the right data to use for a study that showcases the value of a product, which is only compounded by finding the right data source.

Recently, contract research organizations (CROs), consulting groups, the pharmaceutical industry and even the payer organizations have begun expanding into RWE services. Research organizations are increasingly realizing that in order to conduct successful RWE studies they must seek external partnerships and outsource the work to these vendors with RWE experience. Organizations specializing in RWE services can accelerate access to RWD and provide insight into appropriate study design for specific research questions. Yet, it’s not easy finding the right RWE supplier — visibility into which group owns and can access data is murky at best, and if they do have access to the data, what do they have access to? All of these factors may hinder finding the right data. Analyzing the data and proving the value of a product further slows down time to insight, which is not cost-effective.

Sourcing Simplified: The HEOR & RWE Marketplace

Currently, many researchers not using an online marketplace attempt to find the right RWE supplier through word of mouth such as asking friends, colleagues or academic institutes, among others, creating a sort of spider web of connections that can add several months to a study. The new HEOR & RWE Marketplace, a collaboration between two trusted life science brands, HealthEconomics.Com and Scientist.com, addresses the challenges RWE stakeholders face today by connecting research organizations and RWE service providers all on one platform. The Marketplace makes it easier for organizations to find suppliers who have 1) appropriate data access that research organizations require 2) the expertise to design and implement RWE studies 3) expertise in specific therapeutic areas 4) and an understanding of the data they own as well as its origin. All of these benefits, coupled with the fact that it shortens time to find a qualified supplier, drastically reduces timelines and helps drive time to insight.

For existing Scientist.com users looking to incorporate RWE studies into their current pipeline of projects, this new partnership is expanding our already large supplier network. And if you have preferred suppliers, the Marketplace reduces the time to contract with an award-winning compliance tool that follows the same checks and balances of a client’s internal organization. This framework provides procurement and legal teams more control over the proposals, reducing the time to contract. This can be a huge benefit if a client has a regulatory question that needs a quick response so more time can focus on the quality of the research.

We recognize that there is an increasing need for access to HEOR and real-world data to make faster and more informed healthcare decisions and help guide treatment approaches. The HEOR & RWE Marketplace is an online solution that accelerates the sourcing and procurement process for researchers and suppliers of HEOR/ RWE and market access services. By streamlining the process for buyers and sellers, the Marketplace will deliver reduced time to contract, quicker time to insight and faster market access.

References

1. Miksad, R. A., & Abernethy, A. P. (2017). Harnessing the Power of Real-World Evidence (RWE): A Checklist to Ensure Regulatory-Grade Data Quality. Clinical pharmacology and therapeutics, 103 (2), 202-205. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5814721/
2. Sherman, R. E., et al. (2016). Real-world evidence: What is it and what can it tell us? The New England Journal of Medicine. 375 (23): 2293-2297. Available at: http://buster.zibmt.uni-ulm.de/dien/DPV-Wiss-Real-World%20Evidence%20-%20What%20Is%20It%20and%20What%20Can%20It%20Tell%20Us.pdf
3. Hampson, G., et al. (2018). Real world evidence for coverage decisions: Opportunities and challenges. A Report from the 2017 ICER Membership Policy Summit. Institute for Clinical and Economic Review. Available at: https://icer-review.org/wp-content/uploads/2018/03/ICER-Real-World-Evidence- White-Paper-03282018.pdf