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PCORI February 2014

AAMC Patient Centered Research Newsletter

As I See It - Who says groundwork isn't glamorous?

The Patient Centered Outcomes Research Institute marked its third anniversary with a piece in last week's New England Journal, PCORI at 3 Years: Progress, Lessons and Plans. The publication coincides with major funding announcements representing a significant increase in funds for comparative effectiveness research (CER) through targeted mechanisms and large pragmatic studies. PCORI reports that nearly two-thirds of its roughly $500 million in funding commitments to date have supported CER.

In the Spring 2014 funding cycle, up to $122 million more will be used to support direct comparisons between two or more alternatives for the prevention, diagnosis, treatment or management of disease, and an additional $35 million is dedicated specifically to comparing obesity treatment options and transitional care services to reduce preventable hospital readmissions. These targeted mechanisms place an explicit focus on relevance of the findings to low-income, minority and rural underserved populations, a sign of PCORI's commitment to driving inclusive clinical research.

Ann Bonham., Ph.D.
Chief Scientific Officer, AAMC

Who says groundwork isn't glamorous?

CER has come a long way in three short years, considering how politically charged it became prior to the passage of the Affordable Care Act. This is due in no small part to the investments PCORI has made in engaging a broad swath of stakeholders at every stage. Establishing a climate for research where the norm is the meaningful inclusion of patients, providers, payers and other end users in the creation, evaluation and implementation of research questions and results is a major feat.

Our history is replete with examples of important research results published in major journals sitting idle for too long before being embraced or disseminated. Including key stakeholders upfront will go a long way to alleviating this implementation gap.

To its credit, PCORI has introduced rigorous evidence-based methodologies for engaging these stakeholders, including standards for demonstrating inclusiveness. While perhaps not glamorous, these processes are as essential to realizing the promise of CER and implementation research as those technologies enabling the establishment of pluripotent stem cells, next generation genetic sequencing, and large scale data analysis are to fulfilling the promise of basic science.

PCORI has also invested in a new collaborative data-management infrastructure. The National Patient-Centered Clinical Research Network will enable researchers to access and combine large data sets across multiple networks to evaluate treatment strategies and care processes to improve health outcomes.

Infrastructure for big data compatibility and brokered agreements for data sharing may seem arcane, but these are precisely the building blocks necessary to maximize the utility of CER investments. Without this groundwork, PCORI's potential long term impact would be limited by the underlying issues which currently constrain the translation and implementation of research results.

Momentum is spreading

PCORI investments in the meaningful inclusion of all stakeholders in CER as well as the more recent investment in a data infrastructure to allow for large-scale research are gaining momentum through key partnerships. PCORI has joined with the National Institute on Aging to employ its inclusive research methods to the comparison of multiple interventions against usual care for preventing injurious falls in the elderly. And it is working with the Agency for Healthcare Research and Quality to mine registry-based cohorts to compare the relative effectiveness of less invasive treatment options in women with symptomatic uterine fibroids.

Other agencies, including the FDA, are moving to weigh patient input more substantially; witness, for example, the FDA's Patient Preference Initiative to incorporate patient-reported preferences in the medical device regulatory process. Patient advocacy organizations like Genetic Alliance are rapidly advancing the privacy controls and other protections necessary to induce more patients to contribute self-reported data to registries for research purposes.

PCORI's building block approach over the past 3 years has been central to creating this momentum. The recent announcement of the Eugene Washington PCORI Engagement Awards focused on encouraging more active engagement of stakeholders in the research process is another positive step forward.

All told, the past 3 years represent a promising start for PCORI. The rapid growth of our AAMC Research on Care Community, a learning collaborative of nearly 250 researchers and clinical providers dedicated to clinical effectiveness and implementation science to improve quality, safety and equity of care for their institutions' patient populations signals a strong interest among the academic medical community for continued engagement in this area of patient- and population outcomes research championed by PCORI. So far, it seems to me that PCORI is on the right track.

Of Related Interest

LOIs are due 3/7/2014 for several PCORI funding announcements including large simple clinical trials

PCORI 4th Quarter progress report

A Summary of PCORI's commitment to CER research from Dr. Joe Selby

Other Links and Resources

Patient perspective on a clinical trial enrollment experience

Early Benefit Assessment of New Drugs in Germany - Results from 2011 to 2012

Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012

A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials

Interpreting Treatment Effects From Clinical Trials in the Context of Real-World Risk Information

Silencing Many Hospital Alarms Leads To Better Health Care - NPR

Where Have All The Inpatients Gone?

PCORI Approves $5 Million to Fund Methods Research with NIH PROMIS (Patient Reported Outcomes Measurement Information System)

National Trends in Patient Safety for Four Common Conditions, 2005-2011

Patient Centered Medical Home impact on cost and quality review

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