- 2023 Annual Meeting
- The PCORI Strategic Plan
- Clinical Effectiveness and Decision Science
- Dissemination and Implementation
- Evaluation and Analysis
- Healthcare Delivery and Disparities Research
- PCOR Infrastructure & Innovation
- Our Vision & Mission
- Financial Statements and Reports
- The PCORI Strategic Plan
- Board of Governors
- Methodology Committee
- Authorizing Law
- Evaluating Our Work
- PCORI's Advisory Panels
- Procurement Opportunities
Past Opportunities to Provide Input
- Stakeholder Views on Components of 'Patient-Centered Value' in Health and Health Care (2023)
- PCORI's Proposed Research Agenda (2021-2022)
- Proposed National Priorities for Health (2021)
- Proposed Principles for the Consideration of the Full Range of Outcomes Data in PCORI-Funded Research (2020)
- Proposed New PCORI Methodology Standards (2018)
- Data Access and Data Sharing Policy: Public Comment (2017)
- Proposed New PCORI Methodology Standards (2017)
Comment on the Proposed New and Revised PCORI Methodology Standards (2016)
- 1. Standards for Formulating Research Questions
- 10: Standards for Studies of Diagnostic Tests
- 12. Standards on Research Designs Using Clusters
- 13: General Comments on the Proposed Revisions to the PCORI Methodology Standards
- 2: Standards Associated with Patient-Centeredness
- 3: Standards for Data Integrity and Rigorous Analysis
- 4: Standards for Preventing and Handling Missing Data
- 5: Standards for Heterogeneity of Treatment Effects
- 6: Standards for Data Registries
- 7: Standards for Data Networks as Research-Facilitating Structures
- 8. Standards for Causal Inference Methods
- 9. Standards for Adaptive Trial Designs
- Peer-Review Process Comments (2014)
- Draft Methodology Report Public Comment Period (2012)
- Past Opportunities to Provide Input
It’s been an eventful year for Scott Berns, MD, MPH. In 2019, Berns and his wife, Leslie Gordon, MD, PhD, were keynote speakers at the PCORI Annual Meeting. Then, in November, Berns was elevated to Chair of PCORI’s Advisory Panel on Rare Disease. In observance of Rare Disease Day, on February 29, Berns discusses the advisory panel's activities and goals.
I was thrilled to become the chair of the PCORI Advisory Panel on Rare Disease in November. As we observe Rare Disease Day on February 29, it’s important for me to take account of what the panel has accomplished in the past, and what we look to do moving forward. My intention is to build on the past discussions of the Advisory Panel on Rare Disease with an aim to provide the best advice to PCORI leadership on how to serve the needs of the rare disease community.
|How I Cured My Rare Disease
In a related blog post, PCORI Advisory Panel on Rare Disease member Doug Lindsay shares his remarkable odyssey to overcome a rare health condition.
Dr. Matt Cheung, whom I succeeded as Chair, urged us at our last meeting in the fall to continue to identify cross-cutting research topics that need to be addressed in rare disease—such as sleep disorders and transitions from pediatric to adult care—and to look for ways to tackle the challenges of performing comparative effectiveness research among rare disease populations. And, as always, we’ll continue to identify gaps where research is needed, and advise PCORI on what kinds of rare disease studies could be most beneficial to patients and those who care for them.
In addition to advising PCORI, and in the spirit of the panel’s charter, I think there’s an opportunity for the panel to help galvanize the rare disease field. Personally, I have a two-year opportunity as Chair to connect the dots from my personal and career experiences with the dots at PCORI. As someone who has been personally affected by rare disease and now also works to improve systems of care for those living with sickle cell disease, I am intimately aware of the need for urgent action and increased national momentum (I elaborate more fully on this in a recent article from the National Institute for Children’s Health Quality). I am eager to bring that energy to the panel. I have plans to attend or speak at multiple convenings this spring related to rare disease, including the Chan Zuckerberg Initiative’s Rare as One Network.
But it’s not just me—all of our panelists are connecting dots. Our advisory panel members represent an array of key organizations, research institutions, and families in the rare disease field, and we all have contacts beyond our own organizations and beyond PCORI. We need to leverage those connections, spreading the word about PCORI and bringing back recommendations to PCORI on how to better engage the rare disease research community. In addition, many rare disease advocacy organizations are, by nature, small—both in numbers and in resources. We can raise awareness about and provide access to opportunities through PCORI that these small organizations might not even be aware of.
As I’ve written previously, each rare disease can seem like a grain of sand, but collectively, those grains of sand make up a beach. As we look forward to our panel’s spring meeting, I’m confident that PCORI’s work, and the work of the Advisory Panel on Rare Disease, can bring communities interested in rare disease research together to move forward collectively.
Upcoming Funding Opportunity on Rare Disease Research
PCORI funded the development of PCORnet®, The National Patient-Centered Clinical Research Network, to address an urgent national need—capacity to conduct patient-centered outcomes research faster, more efficiently, and at lower cost, by leveraging the power of health data, reusable research infrastructure, and unique patient partnerships. The vision for PCORnet was not only that it could serve as a resource to expedite all kinds of comparative clinical effectiveness research (CER), but that its scale would allow for improved research on rare disease.
At the March 2020 meeting, PCORI’s Board of Governors will consider a funding opportunity to use PCORnet to conduct research on rare diseases.
PCORI has funded more than 30 patient-centered CER studies to help patients and those who care for them make better-informed decisions about treating rare diseases. For more information, see our fact sheet, Research Topic page, and a list of our rare disease projects.
The views expressed here are those of the author(s) and not necessarily those of PCORI.
What's Happening at PCORI?
The Patient-Centered Outcomes Research Institute sends weekly emails about opportunities to apply for funding, newly funded research studies and engagement projects, results of our funded research, webinars, and other new information posted on our site.