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Real world evidence has become a topic of much discussion and debate in 2012, and over the coming weeks we are going to take an in-depth look at the promise, and advantages, shown by exciting new frontier in clinical data...

By Craig Sharp on Oct 24, 2012

Over the course of the next four weeks, eyeforpharma will be looking in detail at Real World Evidence. Where it came from, how it can be used and how big pharma companies are already integrating it into their drug development processes. For those of you who are new to this topic, this interview with provide a broad overview of RWE and it's uses to the industry, next week, we will begin a series of detailed industry interviews- providing you with a close look at how big pharma are currently utilising RWE.

This week features a Q&A with Jill Sackman of Numerof & Associates Inc. Jill E. Sackman (D.V.M., Ph.D.) is a Senior Consultant for Numerof & Associates, Inc. and is an internationally recognized surgeon, medical researcher and author with more than 25 years experience in surgery, and the pharmaceutical and medical device industries.

Craig: "Why would you say so much importance is given to Real World Data and how would you say it first appeared on everyone’s radar? Was it a slow build-up? Was it sudden?"

Jill: That’s a great question. We really see late-phase and health economic outcome research as a part of a growing risk management strategy by manufacturers.  So it has been appearing in correlation with the increasing challenges from public and private payers.

As the economic challenges have increased, and the demand from payers for more robust or comparative evidence has increased, so has the need for RWE.  So in an increasingly economically-strained world, this has become important and that’s why we’ve seen it raise its head in the last couple of years, it’s highly relevant.

Now from a historical perspective, it’s been understood for a long time that randomised clinical studies fail to capture how products will perform once they’re released into the broader population.  I think people have recognised that and if you scan the literature, it goes back many, many years.  It’s not a new concept that our RCTs provide a great core of robust data in understanding how products work, but then fail to really address how they perform more broadly.

So to answer your question, RWE and the need for more longitudinal observational studies - and that’s what they address - is very different from RCTs.  That concept has been around for a very long time and it probably comes more out of the social sciences versus the bench sciences.  It hasn’t been a push from a pharmaceutical perspective until recently because of the economic challenges and the push payers are giving.

"Do you think our concept of Real World Data is something that’s been brought across from other industries? Advertising and consumer marketing for example?"

    

I think that’s a really interesting question. Procter and Gamble comes to mind, and I can't think of a company that does a better job in consumer marketing.  They understand the whole lifecycle, the consumer market and what drives purchasing decisions.

And another company that’s got a lot of press in the US is Target; they can predict, at certain ages, if you're going to move or send your kids off to college or have a baby based on purchasing habits. They’re able to really profile the consumer. I think that is something Pharma has not done historically and in many ways direct to consumer products are more sophisticated in understanding the behavioural lifestyle changes that drive the decision making process.  And the opportunity for Pharma to use that? I think it’s huge.

"What would you say was the industry’s initial reaction in the advent of RWE? How well do you think the industry has been able to integrate this concept?"

I think the initial reaction has been mixed. I think conceptually people are on board because it makes logical sense; I think that a lot of multi-national pharmaceutical companies want to think the US isn’t going to change any time in the near future so let’s just continue as we’ve done.  For firms working in Europe or Asia or Latin America, they think “Well we’ve done this, we’ve really done this all along.” Getting organisations, particularly clinical or health economic groups, to think strategically about data from a product lifecycle perspective... it’s a new concept for many, regardless of what market they play in.

If you were to, on the flip side, say what does it look like on the device or diagnostics side?  I think those organisations are several years or more behind Pharma.  They haven’t been generating clinical data much at all, unless they’ve been in a high risk device category (PMA).  Establishing registries or longitudinal observational data to look at the value or effectiveness of the product in market is really new.

"Where do you believe the true value of RWE lies for Pharma?"

Randomised clinical trials, RCTs, will always be seen as the standard for establishing the causation between a therapy and outcome in a narrow population.

Yet, even when you have data from RCTs that demonstrates benefit for a product, it’s optimal role in clinical practice is still unanswered. And physicians, practitioners, clinicians, as well as payers are asking these questions!  Great clinical study in a really controlled population, but how is it going to really work in my practice?  How is it going to work across the variety of different genetic and socioeconomic backgrounds of my patients?

I think where RWE’s true value is going to lie is in defining how products will perform in clinical practice and a clinical setting in the broader patient population -- really, real world data for real world settings.   We need that data from a patient-outcomes perspective and payers are increasingly pushing for that information.

     "You mentioned RCTs being the “gold standard”, which of course they are. But do you see RWE becoming a complimentary accompaniment to RCTs as an industry default?"

Absolutely Craig, I think we’re talking about the right data for the right stage in the product’s lifecycle.  You obviously can't be collecting longitudinal observational data when you’re in clinical development.  And you need to be looking at causation, outcomes and safety in a narrow population.

But once you start to get into the broader population post-launch, I think there’s going to be an increase in the requirement by payers and providers as well as regulatory agencies, for evidence that shows “what does this look like after a period of time, in this kind of population more broadly?”

"So you’re saying it won’t just become the industry norm, it will also become a regulatory requirement?"

Oh, absolutely.  I think you’re going to start seeing regulatory agencies requiring the collection of this data.  Really, this is what the FDA and the European Commission are already doing by requiring post-market safety surveillance or clinical follow up.  Certainly the FDA is in the process of defining what more structured post-market safety surveillance is going to look like, and that to me is the code word for RWE or RWD.

"Do you see social media platforms playing a larger role in providing RWE going forward?"

I think it could be a really interesting source of data for pharmaceutical or device companies.

The focus groups or advocacy groups, particularly in the orphan or ultra-orphan space, they do a lot of this data collection through these small groups, because patients want to talk to other patients and many of them about side effects or any challenges they have with their disease or medication and I think that would be a fabulous source for mining data for a company.

And then of course social media, the ability to use social media as you’re conducting more structured longitudinal observational studies, the opportunity there is also significant.

"Can you clarify which kinds of data are needed at which point in the product lifecycle?"

For regulatory approval, I think manufacturers are still going to need randomised clinical studies to demonstrate safety and efficacy against established standards.  I mean, this has to start somewhere; you can't put a product out into a broader population without knowing how it will perform, at least in a defined population, right?

The utilisation of longitudinal studies in real clinical practice after products have been launched should continue as new products enter the market place. It’s not a static environment, and if a competitive product comes on the market, companies should be thinking about how they are differentiated and what clinical studies would support a differentiated positioning.

Here’s an example. About a year ago the FDA commissioner, Dr Hamburg, revoked the agency’s approval of breast cancer’s indication for Avastin, after they felt there was evidence that the drug really was not safe and effective for the indication.  They were starting to see side effects that they felt were more significant than any clinical benefit.  So you’re going to start seeing agencies wanting to see some of this real world evidence data to maintain approval status.  It’s certainly going to be important for payers and I think you’re going to increasingly see providers asking for this data.

"You mentioned Target earlier on, how retail stores can detect whether or not you’re pregnant based on consumer habits, purchases? Do you think there’s scope for pharma to utilise similar measurements for, say, adherence?"

Oh absolutely! Absolutely, because when you start to get into the psychology and sociology and all the issues around why people don’t take medications or why people don’t lose weight or why people don’t manage their health better, you do have to understand those issues.  I’m completely on board with that thinking.

"And finally Jill I’ll ask you to take a look into your crystal ball for me, how soon do you think RWE to really take hold? How long until it becomes the norm?"

It will depend upon the economic pressures put on the market and it’s probably going to be high cost therapeutic areas that will get hit first. Ones that are really expensive, where the payers are putting a lot of pressure on - oncology’s probably one. Where there is economic pressure, companies will start to invest more in doing this.  And it’s likely to be iterative, it isn’t going to happen in big waves and as we described, it’s going to be complementary to RCTs.  It’s going to be just increasing your bag of tricks, your portfolio of things you can do to really support your product.

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Next Wednesday we will be continuing our Real World Series with the first of our industry insights, featuring Bruce Taylor, Director of Government Strategy and Relations at Roche .

Jill Sackman will be hosting a panel alongside Johnson & Johnson, Roche and Medtronic on Real World Data at our Boston conference next month. For more information check out the agenda or download the brochure at our website.