New National Database Could Revolutionize Comparative Effectiveness Research

A little-known government research agency with limited funding is engaging in one of the most ambitious medical data-gathering projects to date. The Patient-Centered Outcomes Research Institute (PCORI) is aiming to integrate patient data from 29 health data networks across the country to provide a diverse, national, ready-made database of information that can be used in comparative effectiveness research (CER) studies without the need to search for and recruit new participants. By mid-2015, PCORI plans to have PCORNet, the National Patient-Centered Clinical Research Network, operational and ready to support CER studies.

PCORI

PCORI was created by section 6301 of the Patient Protection and Affordable Care Act (ACA) (P.L. 111-148). Its purpose is to fund CER studies that aid patients, caregivers, and clinicians in making informed health care decisions. CER essentially helps determine what drugs, devices, and treatment options are the most effective. While patients, caregivers, and clinicians are all looking for effective options, their priorities may differ. Dr. Rainu Kaushal, a researcher at Weill Cornell Medical College, told the Washington Post, “As a provider, I may be interested in how a serum marker changes with a treatment. But patients may be more interested in how it affects how they feel, their ability to exercise and eat.”

But funding such studies is not always easy. In 2013, PCORI approved a plan to dedicate up to $1.03 billion to research funding over the course of two years; in contrast, President Obama’s 2015 budget request included $30.4 billion for the National Institutes of Health (NIH). NIH-scale studies may cost more than $150 million, meaning that PCORI only has the funds to mount two to three such studies each year. Understandably, PCORI began to consider different types of projects that would maximize the contributions they could make to CER.

PCORNet

Cue the idea for a national research database, which has been around for years. PCORNet will integrate data from 29 sources: 11 Clinical Data Research Networks (CDRNs) and 18 Patient-Powered Research Networks (PPRNs). CDRNs are partnerships among health systems that have the ability to collect and use information from multiple data sources, including electronic health records (EHRs). Examples include academic medical centers, community hospitals, health plans, and Veterans Health Administration clinics. PPRNs are patient-formed groups that promote awareness and seek information to improve their health outcomes. Nine of the PPRMs participating in PCORNet focus on common conditions, such as cardiovascular disease; the other nine focus on rare conditions, such as muscular dystrophy and Phelan-McDermid Syndrome. Funding for the 29 sources was announced on December 17, 2013.

The initiative requires patients, clinicians, and health care systems to be actively involved in the use of the data, leading a “shift in clinical research from investigator-driven to patient-centered studies.” During the 18-month development phase, the awardees will conduct pilot projects to test and refine data models. They will also develop policies on data sharing, security, and privacy protection. A Coordinating Center led by the Harvard Pilgrim Health Care Institute and the Duke Clinical Research Institute will provide guidance and management. The team includes experts in data standards, data security and privacy, medical ethics, patient-reported outcomes, project management, and research communications and dissemination.

According to the Washington Post, privacy experts believe that general consent forms that patients sign at the time of treatment should suffice to allow for the use of aggregate data that are collected. PCORI emphasizes that patients will not be identified. Furthermore, organizations have the right to decline research proposals.

Impact

A significant benefit of PCORNet will be its ability to provide data from “real-world” settings gathered in “real-time.” Dr. Francis Collins, Director of the NIH and a member of PCORI’s Board of Governors, refers to the current pace of CER studies as “agonizingly slow,” and notes how much more efficient it would be if researchers could access existing networks that include data for millions of patients who have already agreed to participate in research. “By visiting their regular health care providers, many more patients would have the opportunity to become active participants in medical research with the potential to improve not only their own health, but the health of all Americans.”

If the project is successful, it could have a profound impact on CER in the United States. Maybe by then, a few more people will recognize PCORI’s name.