In a recent edition of ‘Trends’ entitled ‘Real-World Evidence Revolution’, Paul Simms (Chairman of eyeforpharma) articulated a thought I have had running through my head for a while now:
“… the health economy is changing fast, moving from measuring inputs (e.g. number of patients seen, pills sold ect.) to outputs (most importantly, patient outcomes).”
I get the feeling we have an idea of what Real-World Evidence (RWE) is and can see what it can offer, but forget why we need it, and why we need it now. This quote is ultimately the manifestation of the driving force of this need.
What it seems to come down to is a focus less on the efficacy of a given compound on the biology and more onto its effectiveness in the real world. To put it bluntly, worrying slightly less about how a chemical reacts with a biological system, and more about how it will effect the patient as a person (i.e. actual outcomes and changes to Quality of Life ect.). This is because effectiveness will, in the end, govern the success of a given treatment more than the efficacy of a compound.
Over the years, a number of treatments have underperformed relative to their pre-launch clinical trials. Usually because they didn’t take account of a key factor that became apparent once the treatment was released into the real world. In the case of Acomplia/Rimonaboant, suitably large study populations were not utilised to truly assess the safety of the medication, or Exubera/’Inhalable Insulin’, where patient preference under real-world conditions led to an unsound premise, that Diabetes patients were scared on needles. Ultimately, it is effectiveness, not efficacy that will translate into the products commercial impact.
This is why we need RWE.
The funny thing is, this isn’t a new problem. The problem is acting on this knowledge that I am guessing most of the readers of the article already know.
A lack of action, comes mainly from the shackles of legacy. To get regulatory approval, everyone in the industry automatically jumps to a Randomise Control Trial (RCT) as the answer. And yes, an RCT is the gold standard for proving efficacy, which is very important. However, as many of us know, once you know your treatment does benefit the patient (i.e post Phase II) your considerations need to start stretching beyond just the therapeutic effect, but also the effect in the real world. In short, this is the point to consider the pragmatic study designs and start initiating broader RWE studies.
So next time you are planning a study, or sitting around a boardroom table deciding on the development pathway for you treatment, yes think about the RCTs and studies needed to get regulatory approval, but also consider what are the other questions you really want to answer before you launch your treatment. Think about these questions first, then decide on how you want to answer them.