Real-World Evidence

Early discussions about comparative effectiveness research (CER) in the United States focused on defining “comparative” and determining the implications for research. Health care stakeholders were talking about the broad array of what should be compared, such as drugs, surgery, devices, physical therapy, primary care doctors versus specialists, how care is delivered, and what the relevant comparisons are among the varied technologies and services. Today, the focus has shifted from looking simply at what should be compared to how comparative studies should be designed to answer the practical questions about “effectiveness,” particularly in real world settings. The question now becomes does a treatment, service, or method of delivering care work when applied in real world, clinical practice environments?

To answer that question, health care stakeholders need to think beyond traditional randomized controlled trials (RCTs). One way to consider is to make use of real-world databases that record the processes of care patients receive, such as electronic medical record (EMR) and medical claims databases. We also might work with patient registries that focus on patients with a specific disease or who receive a particular treatment or device and record the medical outcome for those patients; or these registries could be based on a specific population so that researchers can see what happens within that population.



Because more of these public databases are becoming available, we need to ensure that there are sound ways to use the information to address effectiveness questions. Analyses of these data are already underway, and as results become available, we will have to pay better attention to them and any limitations inherent in those results. For example, studies that use these data cannot randomize patients to treatments or services to be studied, and thus the bias of confounding, and other potential biases, need to be considered so that differences observed in results are due to the treatments used and not other factors that were not a part of or controlled for in the study.

Another way of looking at the real world is via practical clinical trials that relax some of the constraints of a normal trial and better reflect the real world conditions of clinical practice. Participants in routine RCTs usually must meet very precise clinical criteria, and thus the information gained from the trial may not be “generalized” to a broader group of patients. Typical RCTs also define the frequency of visits and tests to be performed, and compliance with treatments is closely monitored and controlled. These characteristics are not typical of usual care patients receive and thus the real benefits over usual care are more difficult to assess. In practical trials, patients may be randomized to treatments under study, but subsequent care may be determined by the doctor based on his or her clinical judgment. Practical trials often include a comparison group receiving usual care rather than a placebo comparison.

Blog Post

CER Tweets of the Week: The State of Accountable Care, JAMA on PCORI’s Perspective, Real-World Impact of CER (October 13-17)

Health care stakeholders tweeted about accountable care, patient-centered outcomes research (PCOR) and the real-world impact of comparative...
Blog Post

CER Tweets of the Week: Health Affairs on the High Value of Specialty Medications, Pediatric CER Guidelines, Patient Heterogeneity (October 6-10)

This week, comparative effectiveness research (CER) stakeholders tweeted articles about specialty medications, CER guidelines and individual...
Blog Post

CER Tweets of the Week: Reports on Health Care Quality, Fellowships, and PCORI Turns 4

A report on health care quality from the U.S. Department of Health and Human Services, postdoctoral fellowships and the Patient-Centered Outcomes...
Blog Post

CER Tweets of the Week: Patient Engagement a Common Theme Among CER Stakeholders (September 22-26)

Patient engagement was a hot topic among comparative effectiveness research (CER) stakeholders on Twitter this week. @SUMedicine bioethicist David...
Press Release

NPC Study Outlines When & How Health Decision-Makers Should Consider Individual Patient Differences

Washington, DC (July 29, 2014)—New research led by the National Pharmaceutical Council (NPC) can assist health care stakeholders to understand when...
Press Release

NPC Annual Stakeholder Survey: Comparative Effectiveness Research Is Important, But Impact on Health Care Decision-Making Is Still on the Horizon

(Washington, DC, May 19, 2014)—A new survey of health care stakeholders reveals continued optimism for the use of comparative effectiveness research...
Press Release

CER Collaborative Improving Health Outcomes Through New Comparative Evidence Tools

(Washington, DC, March 24, 2014)—Three peer-reviewed articles published in Value in Health offer important guidance to aid formulary decision-makers...
Press Release

NPC, UMD Study: Factors That Cause Different Treatment Responses Are Not Well Understood or Studied

Washington, DC, November 4, 2013—Although researchers and health care providers widely recognize that individuals with stage IV prostate cancer react...
Commentary and Testimony Page

The State of CER and the Environment for Health Care Decision Making

This month, NPC released its third annual survey on stakeholders’ views on comparative effectiveness research (CER), “The State of Comparative...
Commentary and Testimony Page

Keeping Our Eye on the CER Ball

Until the cherry blossoms begin to bloom later next month, most of the water cooler conversations in Washington will focus on the federal budget....
Commentary and Testimony Page

Leveling the Playing Field? Could a Landmark Decision Alter the CER Communications Landscape?

A growing chorus of voices is publicly sharing data on the effectiveness of treatment options, but one viewpoint has long been shut out of key areas...
Commentary and Testimony Page

Living Up to Its Name? The Role of Patient-Centered Research

Anyone who has ever received bad news from his or her health care provider or held a loved one’s hand when a diagnosis was delivered can understand...
YouTube Videos

Real-World Impact of Comparative Effectiveness Research Findings on Clinical Practice

National Pharmaceutical Council Chief Science Officer Robert W. Dubois, MD, PhD, discusses a peer-reviewed study suggesting that changes are needed...
YouTube Videos

Using Real-World Evidence to Increase Health Care Access, Improve Quality & Reduce Costs

Mitchell Sugarman, vice president of health economics, policy and payment at Medtronic Cardiac and Vascular Group, explains that health care decision...
YouTube Videos

CER and the Environment for Health Care Decision-Making

The National Pharmaceutical Council’s (NPC) fourth annual survey of health care stakeholders is continuing to shed light on the current environment...
YouTube Videos

Webinar: CER Collaborative: Tools You Can Use

Collaborative members hosted a webinar explaining how to use the tools they developed to create greater uniformity and transparency when evaluating...
Blog Post

CER Tweets of the Week: The State of Accountable Care, JAMA on PCORI’s Perspective, Real-World Impact of CER (October 13-17)

Health care stakeholders tweeted about accountable care, patient-centered outcomes research (PCOR) and the real-world impact of comparative...
Blog Post

CER Tweets of the Week: Health Affairs on the High Value of Specialty Medications, Pediatric CER Guidelines, Patient Heterogeneity (October 6-10)

This week, comparative effectiveness research (CER) stakeholders tweeted articles about specialty medications, CER guidelines and individual...
Blog Post

CER Tweets of the Week: Reports on Health Care Quality, Fellowships, and PCORI Turns 4

A report on health care quality from the U.S. Department of Health and Human Services, postdoctoral fellowships and the Patient-Centered Outcomes...
Blog Post

CER Tweets of the Week: Patient Engagement a Common Theme Among CER Stakeholders (September 22-26)

Patient engagement was a hot topic among comparative effectiveness research (CER) stakeholders on Twitter this week. @SUMedicine bioethicist David...

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