Mind blown at DIA's Real World Evidence Conference Last Week

Real World Evidence (RWE): Wow, I did not truly understand the implications of this industry buzzword that’s been flitting around my peripheral vision the last couple of years. Sitting at DIA’s 2nd Annual RWE conference last week it hit me that we are experiencing a quiet revolution in progress.

In the Digital Age absolutely everything a human being does every minute of the day is touched by technology, and therefore can now be measured, quantified and reported. This is so significant relative to human history I am starting to think society should reset the clock to equate Y2K with Year zero, which would make this year 19 D.A. (Hey future historians- you heard it here first!!). The industry is just starting to realize the implications on drug development.

Some of the best and brightest from industry and various regulators from the US, Japan, Canada and Brazil were in attendance, brainstorming how these data- entirely non-existent when all drug development rules, laws, guidances and processes were created- will be harvested to add to the understanding, use, development and approval of medicines. 

A sampling of this delicious brain candy:

• When a sponsor develops a drug, the sponsor has one boss- the regulator. Everything pre-approval is done to “please” that boss- the only questions that matter and get answered are the boss’ questions. In contrast, when the drug gets approved, the sponsor now has three completely different “bosses”- the payers, providers, and patients. RWE is the language that can translate priorities among all bosses. As of 2019, we haven’t found the Rosetta Stone yet but we are going to eventually.
• Payers own massive amounts of claims data and are comparing against huge EHR databases and clinical trial data. In an era where information is power, I found myself a little uncomfortable, bordering on outright frightened, watching the spectacular savvy with which Anthem and Optum presented their findings. 
• The giant brains at MIT are looking to innovate drug development and they’re betting big on RWE:  check out LEAPS. Looking forward to great things coming out of there! 
• For all the work, time and money that goes into clinical data, they amount to a mouse next to the elephant of now-collectable RWE. This has the power to completely transform drug development, long term. How? No one knows exactly, but massive impact is inevitable. 
• Inclusion/exclusion criteria don’t exist in the Real World, so RWE can take us far beyond clinical data, into sub groups, endpoints and comparators that are unimaginable for a clinical trial. To think that we as the drug industry, who think we know our drugs so well because we work so incredibly hard at clinical trials, know only a fraction of what we could know about how the drugs we make affect the patients that consume them. Wow!
• The key to RWE is methodology: how shape the vast pool of unstructured data into comparable objects, in a way that is peer-reviewable, replicate-able, and with a level of quality that all “bosses” have confidence in. (The elusive Rosetta Stone!)
• If such methods can be developed and widely adopted, RWE could potentially answer questions about drug development faster and less expensively than clinical trials- in certain instances. Unanimous agreement that RWE will never replace clinical trial data in all instances.
• China is ahead of the US! Further along with guidances and approvals involving RWE. Ouch!
• Sponsors are cautiously embracing RWE, but if it takes 5+ years to convince FDA to accept RWE for, say, a label extension, very few sponsors are going to make that investment because the opportunity cost is too high. 

For more check out these great references:  Great read on FDA's Framework for RWE, as addressed in the 21st Century Cures Act of 2106:  https://www.fda.gov/media/120060/download. Also, FDA’s draft RWE guidance: https://www.fda.gov/media/124795/download