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Real world evidence: Not just “big data”

Guest Contributor   |     September 22, 2015   |   SHARE THIS

Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today's challenges and opportunities. The Catalyst welcomes guest contributors including patients, stakeholders, innovators and others to share their perspectives and point of view. Like in our Conversations series, views represented here may not be those of PhRMA, though they are no less key to a healthy dialogue on issues in health care today. 

To continue the dialogue about Real World Evidence and what it can mean for new therapy options for patients, we're pleased to host a guest blog from Tom Hubbard, vice president of policy research, The Network for Excellence in Health Innovation.

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Over the last several years, interest in using data from the “real world” has blossomed into its own, movement for the use of Real World Evidence (RWE). Definitions of what constitutes RWE vary but in general it entails data collection and analysis about the use, benefits and risks of medicines that fall outside the bounds of the classic Randomized Clinical Trial, including use of data that is routinely collected in the daily practice of medicine, and thus reflective of the heterogeneous patients seen in real world practice settings.

Experts from many sectors, from patient advocacy groups to clinical leaders, share a sense that appropriate use of RWE can be a very important, potentially transformative force in health care decision making in the years ahead, including on the approval, adoption and safe and effective use of therapies.For example, it was retrospective analysis of 40 years of use of beta blockers among heart attack patients that created the basis for using beta blockers more broadly to control hypertension – a development that has played a part in the continuing reduction of cardiovascular disease and strokes. In recent months, RWE has yielded the suggestion that metformin, the widely used diabetes medication, may have cancer-fighting properties.  A health care system that relied solely on randomized clinical trials might have taken years to generate such insights, if it generated them at all.

Real World Evidence holds promise as a transformative force but there’s no guarantee it will be used in a manner that leads to improved health care unless key stakeholders share common standards, principles and approaches to the collection, analysis, and replicability of RWE, and regulations allow evidence based on these data to be used and communicated appropriately with all decision makers, including health care professionals to enhance patient care.

This was made clear to us in a high-level expert roundtable that The Network for Excellence in Health Innovation (NEHI) convened late last year.

The roundtable explored a number of the challenges to the use of RWE, such as the challenge of using routinely collected data (data rarely collected for the purpose of clinical study ) that often requires a  great deal of cleaning and formatting – or, a “data janitor” – to make it useful for research.  There are many challenges, including questions of patient consent and patient privacy, issues of transparency and replication. There is also the uneven, asymmetrical regulation of RWE studies. Manufacturer generation and communication of real world studies by companies is often circumscribed by regulation, while for the most part, studies by a rapidly-increasing number of other players (including payers and providers) are not. Finally, RWE is rarely used by the FDA for the assessment of the benefit/risk balance of products, including for the expansion of approved indications.

NEHI’s roundtable also highlighted some of the opportunities to address these issues. These opportunities include pending U.S. Senate action on legislation that parallels the House-passed 21st Century Cures bill; the upcoming user fee reauthorizations; the continued evolution of PCORI’s PCORnet network and the eventual decision on PCORI’s continuation following its statutory sunset date in 2019; the Obama Administration’s Precision Medicine Initiative and its ongoing series of Open Data initiatives at the FDA, NIH and CMS; as well as such industry-sponsored initiatives as the PhRMA-EFPA Principles for Responsible Clinical Trial Data Sharing.     

In a new issue brief released by NEHI today, we outline the issues and opportunities covered in the roundtable and as well as the general state-of-play in RWE. This issue brief can serve as a great starting point for a deeper discussion among shareholders about a shared vision for good use of RWE. A shared vision may lead us more rapidly to better use and more personalized therapies for patients. 

Tom Hubbard is the Vice President for Policy Research at The Network for Excellence in Health Innovation (NEHI). He leads NEHI's projects on comparative effectiveness research, patient medication adherence and prevention and wellness initiatives. Mr. Hubbard leverages his policy and technology experience to examine ways the country's health care system can be transformed, focusing on the promotion of medical innovation and the improvement of quality and efficiency in clinical care.

Guest Contributor

Guest Contributor The Catalyst welcomes guest contributors, including patients, stakeholders, innovators and others, to share their perspectives and point of view on issues facing our industry and the health care system.

Topics: FDA

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