The Importance of Real World Evidence, and the Future of Trials
August 14, 2023 –
A bit over four years removed from the height of the Covid-19 epidemic, and there’s still conflicting information on the virus as well as the treatment. Certainly, this was a much more unprecedented situation, where in which we needed to react quicker than the normal trial would or could ever allow. Thankfully through the vigilance of the science community, group had been studying the much weaker grandfather of COR-SARs for many years prior to that point. All of this considered, without further research into what Covid has become, and the long term effects the virus and vaccine(s) has had, we’re doomed to repeat our devasting history with infectious diseases – and frankly any mutation for that matter. But do not fear – the FDA is in fact here.
At this point, we all know what RWE and RWD is. If not there’s a google search bar right above you. And hopefully you already know observational studies have been going on in an uncontrolled/controlled environment for at least over half a century(oldest documentation I could find). However, very few know that in 2016, the 21st Century Cures Act (AKA the Cures Act) was signed with the intention of accelerating the approval of drugs and devices that lack proper trialing and evidence if certain criteria are passed. Again, you all have google and can look up the bill if you wish to learn more about those criteria. But I’ll let you know it was aimed at things like the growing opioid epidemic, rare/new diseases and of course preventative medicines and gene therapies are now much more poised to be approved.
Ultimately, this is a major reason why the US was able to get a vaccine together so quickly for COVID-19, and why we’ll be able to do so in the future. “But what does this have to do with Real World Evidence?” Well, it was observational data that was first used to move forward on almost any drug target that’s been accelerated. Real World Data is almost the only (ethical) vehicle for which that congressional act can exist. Having studies and documented outcomes of patients in and after treatment will be vital to challenges such as future viral outbreaks, advancement in current medicine, and complex indications.
Why do we need Real World Evidence?
It sort of seems redundant, doesn’t it? The simple fact is – we do a clinical trial, and then we study what happens some more after that. Seems like you almost should just do one or the other. Well for one, safety and efficacy is the whole point of any of this. We could probably even do with an extra layer of it. The main supplemental advantage to the data after (or before) the trial is the fact that life isn’t controlled like a clinical trial is. There are many factors that can play a role into a body’s acceptance or rejection of a drug. One of the main factors being time. Sick people often don’t have it. But the body can go through some different absorption depending on it’s exposure or prolonged exposure to certain compounds, of course.
The Medical journal “Neurology” published evidence recently from extended observations, stating that people using proton pump inhibitors (PPIs) – which are found in common heartburn medicines – could increase their risk of dementia by more than 33%. This is of course, devastating news. It’s hopefully something that can be fixed in newer and unblinded versions of the trials. That being said, there’s a certain amount of research that needed to be taken over more time. And not only that, many people are involved in order to maintain and properly curate this data.
Now that this was being observed and monitored in such a way, there are likely thousands that require reactive and preventative treatment that we can begin to help immediately. In addition, these studies don’t just need to be done for drug on the market. They can be much more epidemiological. When at risk and often marginalized demographics become exposed or just have a higher genetic disposition to disease – this is when real world evidence moves us forward. Often times in a clinical trial, there isn’t a large focus on genetic disposition due to demographic, or smaller sub-groups with higher risks. It’s a completely separate subject, but there usually isn’t even a large enough sample size to determine this in the trial, due to lack of patient diversity.
I love this industry for the marvels that are made possible through bright minds. I love this people in it for being able to help others. When we think of Pharmaceuticals, we often just think f the Drug being created and prodigious profits. With much ado over mistrust of major entities; we never consider the rigorous research that goes into preventing maladies and future global issues.
The Future of Real World Evidence
AI Alert! AI Alert! For any of the faint of heart people that strongly believe that robots will take over the planet, the rest of this article is not for you. Don’t get me wrong. Robots likely will take over the planet, it’s just not going to happen because you told ChaptGPT to do it.
Now that we’ve got that out of the way, it’s important to understand that again – Real World Evidence and Data isn’t some new thing. Machine Learning and Artificial Intelligence also, is not that new. Both have been a around for a while. And while you may be convinced it’s all apart of a major governmental scheme to hack your brain and turn you into a consumer, the truth of the matter is that these advancements exist because some people go learn for a VERY long time. Some people spend their entire life learning. To these people, we owe a lot of thanks.
So where do we go from here? Well as I’ve insinuated above, the intersection of AI and RWE is here folks. Since the Cures Act almost every major Clinical Research Organization created new groups surrounding the subject to provide continuous integrations to their clients. And in that short amount of time, we’ve already come such a long way.
Groups like IBM have been bullish on large data sets since data existed, so it’s only right they got in on the fun. The major consultancy’s CRO subsidiary has had decades of experience in data collection and deep analysis. Watson Health is one of many off-chutes of their multiplicitous platform, that has incredibly powerful real-time capabilities and built-in predictive analysis capabilities for many basic longitudal and regressive figuring.
IQVIA is probably the most well known. They were one of the first groups to see the fortitude in building out a Real-World Data Platform, and considering they had been a top functioning CRO for some time; IQVIA’s data repository (I’m assuming) is one of the best. Often heralded for it’s rigid regulatory standards and audit properties, IQVIA’s RWE platform has much more exciting AI capabilities. [Securely] Leveraging the insights and data gained and stored from previous trials, the platform provides predictive analysis and outcomes that support and assist with study design. You may not like robots, but to be able to leverage sound mathematics (instead of a human brain) to measure risk and safety within a trial will no doubt lead to better outcomes.
Now that the world knows about AI – practices like these will likely come under some fire. And rightfully so – when it comes to clinical trials, additional checks and balances can almost never be a bad thing. However, we should only scrutinize this for the sake of investing even more. Real World Evidence is not going away. And quite frankly, it’s trial data we can all understand – so we should all be more enthusiastic about it’s advancements. Top pharmaceutical groups have already been using AI for the comparative analysis and adverse event detection. If we could finally harness the power of deep learning to detect issues like that of the PPI subjects [mentioned earlier] we can truly unlock the power of clinical trials and Drug Development.
