I joined Worldwide in March 2018 and, together with my team, expanded Worldwide’s ability to support sponsors’ medical affairs, health economics, and evolving real-world evidence departments.
While preparing for the 2018 ISPOR meeting, I put together six recommendations on evaluating the “real world” of real-world evidence (RWE).
Connect with Jeff at ISPOR (or at a time convenient for you) to learn more about observational and health economics outcomes research, patient registries, and other real-world evidence topics.
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- Real-world evidence isn’t much without real-world data.
Evidence is just a word unless there are data to support one’s assertions; frankly, evidence doesn’t really exist without real-world data. On the other hand, data does not necessarily equate to evidence until one has analyzed what is trying to be proven; who is the target of evidence; and, importantly, what level of evidence is necessary in achieving persuasion, which is directly related to #2.
- Real-world evidence isn’t much without real-world data.
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- Not all real-world evidence is created equal.
Building on that theme, I regularly encourage our clients to work backward from what they want to achieve with their real-world evidence study. Doing this allows them to see the value and potential return on investment of generating data. Moreover, the limitations of RWE can also be considered, which brings me to #3.
- Not all real-world evidence is created equal.
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- Evidence does not equal persuasion.
Even with irrefutable supporting data, evidence must be appropriately conveyed in the right context, using the right terminology, and reflecting the values of the person or entity one is seeking to inform and influence. And this is not at all incompatible with science; quite the contrary, it’s an acknowledgement that even the best science needs a compelling, contextual story.
- Evidence does not equal persuasion.
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- Real-world evidence is not a panacea.
I certainly can’t see it replacing the randomized controlled trial (RCT) as the gold standard for drug approval. And, while even the FDA recognizes that evidence from RCTs is of limited value for day-to-day medical decision-making, we still need controlled experimentation for determining product effectiveness. However, when one considers the burden and treatment of a disease and a product’s development and commercialization life cycle, then RWE can play a vital role.
- Real-world evidence is not a panacea.
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- The real world is ‘real’ messy.
OK, so you’ve established your goal for your RWE study and the appropriate level of evidence, and now it comes down to obtaining the real-world data (RWD). Assuming the RWD are not a finger-snap away via a dive into an existing database, processes and expectations for capturing data in the real world are dramatically different from those appropriate for RCTs, every step of the way. This is an issue with both operational and organizational implications. We’ll get there one day, but even then, there will be important limitations (see #6).
- The real world is ‘real’ messy.
- You can’t engage with a database.
If you’re looking to develop RWE, that presupposes you’re looking to use the evidence to convince somebody (prescriber, patient, payer, or the FDA) of something. If, for example, you’re looking to in actual practice settings, the process used to compile RWD — an observational study, for example — is also, in part, the mechanism that can be leveraged to convey the findings.
Drop me a line if you’d like to connect at ISPOR or have immediate questions.
Connect with Jeff at ISPOR (or at a time convenient for you) to learn more about observational and health economics outcomes research, patient registries, and other real-world evidence topics.
