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In this third installment of our Real World Data series, Jill Sackman speaks to Dr. Dell Kingsford Smith, Vice President, Global Pharmaceutical Pricing for Janssen about identification, implementation and potential pitfalls along the way.

By Jill Sackman on Nov 16, 2012

Among the participants at the Real World Data and Health Outcomes event in Boston on November 29 is Dr. Dell Kingsford Smith, Vice President, Global Pharmaceutical Pricing for Janssen.  Jill Sackman of Numerof & Associates, Inc. spoke with her about why she feels this issue is so critical.

“How did real world data first come onto your radar screen?”

That is an interesting question!  I think real world data has always been there in terms of post-marketing surveillance and safety monitoring.  Initially the impetus was from HTA markets outside of the U.S. regarding coverage with evidence development and ex post effectiveness reviews.  In the U.S., it was, in a small way, present before Health Reform, and in particular payer organizations focused on clinical and economic evidence.  The real impetus in the U.S. has come from healthcare reform.  There was always a focus on health outcomes studies, but the move toward more studies with active comparators and broader patient populations in effectiveness research has been much more significant after reform. 

“How did the organization become involved in thinking about real world data and comparative effectiveness research?  What was the reaction?” 

Initially, there were a variety of views.  There were some who believed it was an opportunity to show the value of our medicines in the real world, while others saw it as a threat.  The threat would be in terms of what the potential comparative effectiveness outcomes might be and the significant additional cost of producing the data. 

“How was the decision made that real world data was an on-going opportunity and not a fad?”

For those within the company who saw the increasing focus on real world data as an opportunity, there was a belief that as a medical innovator developing differentiated medicines for diseases with high clinical need, then real world data would provide a validation of these superior benefits.  This position has become more compelling over time.

“How did real world data gain broader acceptance?”

There was a committed senior leadership focus on real world data.  We had a senior team focused on it in 2010, and they reviewed the range of real world data from social media to EMR to registries, and then made recommendations.  In terms of market access and medical affairs, we instituted processes whereby real world data considerations were embedded into product development.  These processes were instituted at the end of 2010 and implemented in 2011. 

“What were some of the challenges or surprises you found?” 

The biggest challenges were around education internally, and around aligning goals and objectives, particularly for the product development teams. 

The first challenge is that the planning to collect real world data has to take place prior to launch.  It's not necessarily an intuitive process when you've historically been an organization geared around randomized, controlled clinical trial data for marketing approval and you now need to think about different modalities for getting and evaluating data.  Real world data is the 4th dimension -- expanding the evidence universe in ways that randomized clinical trials never could.  There needs to be recognition that this type of data has significant value at different points in the product lifecycle. 

The second challenge is the capabilities required to implement.  For a start this means different types of collaboration and partnerships externally.  You need to collaborate on data and the data may not be wholly owned by the company.  Then here is understanding of the different skills for data collection and analytics.  This provides a real challenge for people who are biostatistically focused to eliminate bias in the clinical trial setting to move into the evolving methodologies of real world data. 

We also need to have a funding and resource prioritization framework.  Every company will have to have a different approach to funding and prioritization based on their structure. It's not one size fits all.  The key isn't where funding comes from, just that real world data is appropriately and specifically funded.  . 

“How will real world data be implemented?”

For Janssen the main players have been market access and medical affairs.  We will always have loose collaboration across our regions and operating companies, but the strategy is global.  We think globally and implement locally.  And if you reflect on the nature of real world data, there is really no way to escape that.  What you need in the U.S. is usually different than what you need in Europe or Asia or Latin America.  Then the challenge is how you fund and resource it.  Companies need to think about their portfolio, their structure and culture and then build an implementation framework accordingly. 

“What’s on the horizon for you?”

In the spectrum of real world data research and in the fullness of time, you will be able to obtain new insights to feed back into R&D.  This could mean products, diagnostics, or services.  Ultimately, this will take us to new technology and solutions for patients, and new consensus in our body of therapeutic and preventive knowledge.  If you compare where we are now to the emergence three decades ago of what were now well-established RCT methodologies, we will eventually get to a research culture where we're as confident in real world data outcomes as we are in RCTs. But it's not going to happen tomorrow- or even in a year or two.  It will take time to build a body of knowledge and accepted operating procedures.  It's challenging and at the same time very exciting. 

“Do you have any tips or warnings for readers?”

Companies need to have champions at a senior level, and they will need to demonstrate the usefulness of real world data quickly in a very concrete way.  They need to have new capabilities and ways of learning from day one. 

“Any last thoughts?”

It's really about thinking about real world data as a fourth dimension, expanding knowledge in ways that our current randomized clinical trials can't.  And it can be a very powerful strategic tool.  Although randomized clinical trials will always be the foundation of evidence at launch, real world data will have the power to truly inform the product lifecycle, taking you to integrated care models in ways that RCTs can't.  This includes thinking about how healthcare is best delivered -- using medicines and procedures in the context of care delivery.  Given the triple aim of health reform (quality, patient experience and cost), real world data is the sort of evidence that can really get us there.