REAL WORLD EVIDENCE IN CLINICAL TRIALS: SHE DRIVES ME CRAZY

This week’s song that is floating in my head is Fine Young Cannibals’ She Drives Me Crazy. And let me tell you, thinking about using #RWE in #clinicaltrials for regulatory decision-making drives me crazy. If I could actually figure out how to get this song to play while you’re reading this…

I can’t stop… the way I feel. The things RWE does, don’t seem real (think song lyrics). There has been an influx of meetings, guidances, webinars, and articles about the use of #realworldevidence in #regulatorydecisionmaking. The amount of traffic on both pros and cons can drive you crazy.  But, there is a clear and distinct interest to pivot from the randomized, placebo-controlled trials that are considered the gold standard for the last 60 years. #RCT is considered “level 1” evidence generation. There is a strict protocol of visits, procedures, and measurements; there are standardized definitions of outcomes and events; and there are deliberate actions taken to minimize bias (such as the randomization process and the act of blinding treatment). However, RCTs are also considered high-cost, time-consuming, and not necessarily generalizable to real-world practice.

People say… I’m obsessed (think song lyrics) with changing the clinical trial paradigm. But we’re creating new ways to think about clinical trials, drug/device lifecycles, regulatory approvals, and the like. As I’ve pointed out in my previous posts on this topic (https://www.dhirubhai.net/feed/update/urn:li:activity:6617049612438028288/ or https://www.dhirubhai.net/pulse/you-spin-me-right-round-baby-rwe-regulatory-flora-sandra-siami/), RWE can be generated from a variety of sources, whether medical/pharmacy claim, existing registries or other databases, electronic medical/health records #EMR #EHR, integrated lab results, healthcare utilization, wearables or other digital solutions, or prospective data collection on patient reported outcomes #PROs.

However, as with any novel methodology, there are a lot of kinks that need to be worked out, no matter how sexy the idea. The quality of this type of data always arises when having discussions. I can tell you in my own instance, if anyone ever uses my data from #EHR it will show I’m on medications that I’m not on (I don’t take any meds) and it shows my ethnicity is Peruvian, which I’m not. I’ve tried to fix these errors through my providers, but these persistently seem to carry over. This example may not seem like a big deal, but I don’t really utilize the healthcare system much. Just imagine the types of errors that might be prevalent in those patients who do utilize the healthcare system regularly… I was at one of many FDA meetings discussing the use of RWD for regulatory decision. I’ll never forget one of the large pharma executives bravely and somewhat sheepishly standing up and telling the FDA how much he wanted to use RWD but that his clinical trials, regulatory, and quality teams were resistant. Of course, after he made that statement, many in the audience vigorous nodded their heads in the affirmative. The struggle is real.

This waiting ‘round’s killing me (think song lyrics). In this on-demand era, it is sometimes challenging to wait. We want to Go, Go, Go! So let’s do this. Let’s use #realworlddata as “synthetic” or “external” controls and perform single arm trials of the test arm and historical or even prospective propensity-matched patients as controls thru existing datasets or claims. This would replace the placebo-control or standard-therapy control arm or could be used where this is no control available and the treatment difference is large. Let’s use #RWD to capture clinical outcomes that would otherwise supplement the traditional prospective data collection. Let’s perform more pragmatic clinical trials that including a broader patient population and capture the patient-centricity we are yearning. I think we’ve made great strides in using RWD to generate RWE in trials in oncology and rare/orphan disease, especially those that are single-arm trials or where there are no comparators.

RWE drives me crazy… like no one else. It drives me crazy and I can’t help myself (think song lyrics). Let’s continue this conversation.

(If this song is not completely stuck in your head, here is the “official” You Tube video in order to get it stuck in your head: https://www.youtube.com/watch?v=UtvmTu4zAMg You’re welcome!)