Legislative Outlook – What’s Ahead for Comparative Effectiveness Research?

July 24, 2019

Public Briefing

A decade following the authorization of the Patient-Centered Outcomes Research Institute (PCORI), the significance and potential impact of comparative effectiveness research (CER) remains at the forefront of the health policy conversation. CER has offered an approach to evaluate the outcomes of different health care methods and identify which treatments matter most to patients. Comparative effectiveness research also has the potential to inform broader health care policy conversations on value, costs, and delivery system reform. This webinar outlined the various PCORI reauthorization bills and amendments that have been discussed by House and Senate committees to date. Our panel of experts explored some of the critical questions facing congressional staff and discuss potential next steps in the legislative process.    


Tanisha Carino, Ph.D., Executive Director, FasterCures, Milken Institute
Gail Wilensky, Ph.D., Senior Fellow, Project HOPE
Anand Parekh, M.D., MPH, Chief Medical Advisor, Bipartisan Policy Center
Sarah J. Dash, MPH, President and CEO, Alliance for Health Policy

This event was made possible by a Patient-Centered Outcomes Research Institute Eugene Washington PCORI Engagement Award.


9:30 a.m. – 9:35 a.m.          Welcome and Introductions

  • Sarah J. Dash, MPH
    President and Chief Executive Officer, Alliance for Health Policy

9:35 a.m. – 10:30 a.m.        Moderated Discussion with Audience Q&A

  • Tanisha Carino, Ph.D.
    Executive Director, FasterCures, Milken Institute
  • Anand Parekh, M.D., MPH
    Chief Medical Advisor, Bipartisan Policy Center
  • Sara van Geertruyden, J.D.
    Executive Director, Partnership to Improve Patient Care
  • Gail R. Wilensky, Ph.D.
    Senior Fellow, Project HOPE

Presentation: Legislative Outlook – What’s Ahead for Comparative Effectiveness Research?

Event Resources

Key Resources (listed chronologically, beginning with the most recent)

“Patients Need a Health System That Helps Them Make Informed Decisions.” Carino, T., Boutin, M. The Hill. July 19, 2019. Available at http://allh.us/pU4V.

“Highlights of PCORI-Funded Research Results.” Patient-Centered Outcomes Research Institute. February 2019. Available at http://allh.us/XqhR

“Comparative Effectiveness Research: Activities Funded by the Patient-Centered Outcomes Research Trust Fund.” United States Government Accountability Office. March 23, 2018. Available at http://allh.us/43CG.

“The ICER Value Framework: Integrating Cost Effectiveness and Affordability in the Assessment of Health Care Value.” Pearson, S. Value in Health. March 2018. Available at http://allh.us/dTQV.

“The Policies and Politics of Creating a Comparative Clinical Effectiveness Research Center.” Wilensky, G. Health Affairs. July/August 2009. Available at http://allh.us/qBVy.


Additional Resources (listed chronologically, beginning with the most recent)

“Supporting Patient-Centered Research.” Finkel, T. AcademyHealth (blog). June 18, 2019. Available at http://allh.us/w46U.

“Commentary: The Patient-Centered Outcomes Research Institute Has Lived up to Its Name.” Gingrey, J. and Coelho, T. Modern Healthcare. June 3, 2019. Available at http://allh.us/wRNJ.

“Patient Engagement in Research: Early Findings from the Patient-Centered Outcomes Research Institute.” Forsythe, L., Carman, K. Szydlowski, V., et al. Health Affairs. March 2019. Available at http://allh.us/GXAC.

“A Policymaker’s Perspective on Comparative Effectiveness Research: History and Prospects.” Allen, T. Health Affairs Blog. October 13, 2018. Available at http://allh.us/KWea.

“Building Data Capacity for Patient-Centered Research in HHS.” Office of the Assistant Secretary for Planning and Evaluation. March 23, 2018. Available at http://allh.us/w3Uc.

“Choosing Wisely Campaigns: A Work in Progress.” Levinson, W., Born, K., Wolfson, D. JAMA. May 15, 2018. Available at http://allh.us/Bmdh.

“Policy Strategies for Aligning Price and Value for Brand-Name Pharmaceuticals.” Pearson, S. Nichols, L, Chandra, A. Health Affairs. March 15, 2018. Available at http://allh.us/wERQ.

“Exploring Patient-Centered Comparative Effectiveness Research.” Martin, H. Bipartisan Policy Center (blog). June 2, 2017. Available at http://allh.us/brfc.

“Improving the Relevance and Consistency of Outcomes in Comparative Effectiveness Research.” Tunis, S., Clarke, M., Gorst, S., et al. Journal of Comparative Effectiveness Research. March 2016. Available at http://allh.us/UNnV.

“Supporting Better Physician Decisions at the Point of Care: What Payers and Purchasers Can Do.” Contreary, K., Rich, E., Collins, A., et al. Mathematica Policy Research. February 10, 2016. Available at http://allh.us/U98D.

“Developing Evidence That Is Fit for Purpose: A Framework for Payer and Research Dialogue.” Sabharwal, R., Graff, J., Holve, E., et al. The American Journal of Managed Care. September 17, 2015. Available at http://allh.us/pNAJ.

“Clinical Comparative Effectiveness Research through the Lens of Healthcare Decisionmakers.” Price-Haywood, E. The Ochsner Journal. Summer 2015. Available at http://allh.us/c3ky.

“Comparative Effectiveness and Cost-Effectiveness Analyses Frequently Agree on Value.” Glick, H., McElligott, S., Perfetto, E., et al. Health Affairs. May 2015. Available at http://allh.us/yDKr.

“The PCORI Perspective on Patient-Centered Outcomes Research.” Frank, L., Basch, E., Selby, J., et al. JAMA. October 15, 2014. Available at http://allh.us/MenN.

“A Systematic Review of Stakeholder Engagement in Comparative Effectiveness and Patient-Centered Outcomes Research.” Concannon, T., Fuster, M., Saunders, T., et al. Journal of General Internal Medicine. December 2014. Available at http://allh.us/vREm.

“Stakeholder Participation in Comparative Effectiveness Research: Defining a Framework for Effective Engagement.” Deverka, P., Lavallee, D., Tunis, S., et al. Journal of Comparative Effectiveness Research. March 2012. Available at http://allh.us/RmdW.

“Comparative Effectiveness Research.” Donnelly, J. Health Affairs. October 8, 2010. Available at http://allh.us/kJNu.

“The Political Fight over Comparative Effectiveness Research.” Igleheart, J. Health Affairs. October 2010. Available at http://allh.us/J8yP.

“How Will Comparative Effectiveness Research Affect the Quality of Health Care?” Docteur, E., Berenson, R. Urban Institute. February 2010. Available at http://allh.us/Ddwu.

“Rethinking Randomized Clinical Trials for Comparative Effectiveness Research: The Need for Transformational Change.” Luce, B., Kramer, J., Tunis S., et al. Annals of Internal Medicine. August 4, 2009. Available at http://allh.us/9nvJ.

“The Policies and Politics of Creating a Comparative Clinical Effectiveness Research Center.” Wilensky, G. Health Affairs. July/August 2009. Available at http://allh.us/qBVy.

“Comparative Effectiveness Research: The Need for a Uniform Standard.” Gottlieb, S., Klasmeier, C. American Enterprise Institute. June 2009. Available at http://allh.us/PyhU.

“Implementing Comparative Effectiveness Research: Priorities, Methods, and Impact.” The Brookings Institution. June 2009. Available at http://allh.us/Kakw.

“Comparative Effectiveness Research and Evidence-Based Health Policy: Experience from Four Countries.” Chalkidou, K., Tunis, S., Lopert, R., et al. The Milbank Quarterly. June 2009. Available at http://allh.us/6Fky.

“Developing a Center for Comparative Effectiveness Information.” Wilensky, G. Health Affairs. November 2006. Available at http://allh.us/JBMt.



Tanisha Carino FasterCures, Milken Institute, Executive Director

tcarino@fastercures.org   202-336-8900

Anand Parekh Bipartisan Policy Center, Chief Medical Advisor

aparekh@bipartisanpolicy.org   202-204-2400

Sara van Geertruyden Partnership to Improve Patient Care, Executive Director

sara@pipcpatients.org   202-688-0226

Gail R. Wilensky Project HOPE, Senior Fellow



Experts and Analysts

Joseph Antos American Enterprise Institute, Wilson H. Taylor Scholar in Health Care and Retirement Policy

JAntos@aei.org   202-862-5983

Eric B. Bass Johns Hopkins Evidence-based Practice Center, Director


Jeannie Baumann Bloomberg Law, Reporter


John Bulger Geisinger Health Plan, Chief Medical Officer

JBulger@ geisinger.edu

Nancy L. Keating


Harvard Medical School, Professor of Health Care Policy in the Department of Health Care Policy

keating@hcp.med.harvard.edu    617-432-3093   

Joey Mattingly University of Maryland School of Pharmacy, Assistant Professor in the Department of Pharmacy Practice and Science

jmattingly@rx.umaryland.edu   410-706-8068

Eugene Rich Mathematica Policy Research, Senior Fellow

erich@mathematica-mpr.com   202-250-3544

Morgan H. Romine Duke-Margolis Center for Health Policy, Policy Fellow in Strategic Engagement

morgan.romine@duke.edu   202-621-2815

Sean Tunis Center for Medical Technology Policy, Founder and Senior Strategic Advisor

Sean.tunis@cmtpnet.org   410-547-2687  



Arlene Bierman Agency for Healthcare Research and Quality, Director, Center for Evidence and Practice Improvement 

arlene.bierman@ahrq.hhs.gov   301-427-1500

Carolyn Clancy Department of Veterans Affairs, Deputy Undersecretary for Health, Discovery, Education and Affiliated Networks

carolyn.clancy@va.gov   202-461-9121

Michael Lauer National Institutes of Health, Deputy Director for Extramural Research

Michael.lauer@nih.gov   301-496-1096

Janet Woodcock


Food and Drug Administration, Director of the Center for Drug Evaluation and Research

Janet.Woodcock@fda.hhs.gov   301-797-3200



Andrew Barnhill GlaxoSmithKline, Director of Federal Policy and HHS Strategy


Alfiee M. Breland-Noble


Georgetown University Medical Center, Project Director of AAKOMA at the Center for Trauma and the Community

ab2892@georgetown.edu   202-687-4812

Jennifer Bright Momentum Health Strategies, President; Innovation and Value Initiative, Executive Director
jennifer@momentumhealthstrategies.com   703-628-0534
Sarah Emond Institute for Clinical and Economic Review, Executive Vice President and Chief Operating Officer

semond@icer-review.org   617-528-4013

James Gelfand The ERISA Industry Committee, Senior Vice President of Health Policy

jgelfand@eric.org   202-627-1922

Greg Gierer America’s Health Insurance Plans, Senior Vice President of Policy

ggierer@ahip.org   202-778-3200

Jennifer Graff National Pharmaceutical Council, Vice President of Comparative
Effectiveness Research
Andrew Hu Patient-Centered Outcomes Research Institute , Director of Public Policy and Government Relations

ahu@pcori.org   202-370-9439

Jennifer Jones Blue Cross Blue Shield Association, Legislative and Regulatory Policy Director


Chip Kahn Federation of American Hospitals, President and Chief Executive Officer

ckahn@fah.org   202-624-1534

R. Shawn Martin American Academy of Family Physicians, Senior Vice President of Advocacy, Practice Advancement and Policy

smartin@aafp.org   202-232-9033  

Amy M. Miller Society for Women’s Health Research, Chief Executive Officer

amiller@swhr.org   202-223-8224

Eleanor Perfetto


National Health Council, Senior Vice President of Strategic Initiatives

eperfetto@nhcouncil.org   202-785-3910

Sue Peschin Alliance for Aging Research, President and Chief Executive Officer speschin@agingresearch.org   202-293-2856


(This is an unedited transcript. Please refer to the video for direct quotes and accuracy.)

Sarah  Dash:                       Good morning and welcome to our webinar on the Legislative Outlook- What’s Ahead for Comparative Effective Research. I am Sarah Dash, President and CEO of the Alliance for Health Policy, and I will be facilitating today’s discussion. For those who are not familiar with the alliance, we’re a non-partisan organization dedicated to advancing knowledge and understanding of health policy issues. We do not lobby, advocate, or take any policy or political positions ourself, our mission is to educate the health policy community. During today’s webinar, our panelists will explore the future of comparative effectiveness research, or CER, as the reauthorization of the Patient Centered Outcomes Research Institute, or PCORI, one of the largest entities conducting and funding this type of research is discussed in Congress.

Sarah  Dash:                       This webinar was made possible by a PCORI Eugene Washington engagement award. If you are interested in joining the Twitter conversation use the hashtag #AllHealthLive and follow us @AllHealthPolicy. I’m going to briefly orient you to the go to webinar platform and review some technical notes. We’ve taken a screenshot of the attendee interface. You should see something that looks like this on your computer desktop in the upper right corner. You can click the orange arrow to minimize and maximize this menu. When you joined today’s webinar, you were muted and you will be throughout the presentation. Please use the question panel to chat with us about any technical issues you may be experiencing.

Sarah  Dash:                       You may also send in questions that you have for the panelists at any time. We will collect these and address them during the broadcast. You will also find all of the materials that accompany this webinar including a copy of the slides, a resource list, and an experts list on our website, allhealthpolicy.org and a recording of today’s webinar will be available there soon as well.

Sarah  Dash:                       Now I am please to introduce our esteemed panel of experts. Joining us today, we have Dr. Tanisha Carino who is the Executive Director of FasterCures, a Milken Institute Center. Throughout her career Dr. Carino has been at the forefront of collaborative efforts to improve the lives of patients. She most recently led the U.S Policy Function for GlaxoSmithKline after spending more than decade with Avalere Health where she founded the Center on Evidence Based Medicine. Prior to Avalere, Dr. Carino worked in the Medicare program to improve access for it’s beneficiaries and support the development of real world evidence.

Sarah  Dash:                       Next we have Anand Parekh, who is Chief Medical Officer at the Bipartisan Policy Center where he provides clinical and public health expertise across the organization. Prior to joining BPC, he completed a decade of service at the Department of Health and Human Services. He is a board certified internal medicine physician, and an adjunct professor of Health Management and Policy at the University of Michigan School of Public Health.

Sarah  Dash:                       Sara van Geertruyden, is the Executive Director of the Partnership to Improve Patient Care. She is also a partner at Thorn Run Partners, which she joined in January 2011. From 1996 to 2003 Sara worked on Capitol Hill during which time she spent over three years as a legislative assistant overseeing Senate finance committee issues for health and welfare. Throughout her distinguished legal career Sara has represented hospital systems, pharmaceutical companies, healthcare providers, physicians and coalitions.

Sarah  Dash:                       Last we’re pleased that Dr.Gail Wilensky will be joining us. Dr.Wilenksy is an economist and senior fellow at Project Hope, which she joined in 1993. Previously Dr.Wilensky directed the Medicare and Medicaid program and served in the White House as Senior Advisor on health and welfare issues to President George H.W Bush. She was also the first Chair of the Medicare Payment Advisory Commission. Her expertise is on strategies to reform healthcare in the United States with particular emphasis on Medicare, comparative effectiveness research and military healthcare. Dr.Wilensky was testifying on Capitol Hill and I believe will be joining us by phone at some point during this webinar.

Sarah  Dash:                       Finally, I’ll briefly review our agenda for this morning’s conversation. We’re going to cover four main sections, which you see outlined here. First, Sara van Geertruyden is going to walk through some slides to orient everybody to the latest congressional activity around PCORI reauthorization. We will then have a conversation that touches on the remaining reauthorization items for Congress, it’s impact on different stakeholders, as well as the intersection with other Health Policy Conversations. We’ll have time during the final thoughts section to answer any remaining audience questions. Throughout the webinar please do again, submit your questions through the questions panel on your screen, and we’ll weave them through the conversation.

Sarah  Dash:                       So with that, I’d like to now turn it over to Sara to share her overview. Thank you.

Sara van G.:                        Thank you. I guess next slide. So thank you for allowing me to join you. My name is Sara van Geertruyden. I am with the Partnership to Improve Patient Care. Next slide. Next slide. Just to provide a bit of a level set and I will try to not spend too much time on the background and maybe try to spend a bit more time on where we are today, but before PCORI it’s important just to remember that there were several bills funding comparative effectiveness research around 2007. The Partnership to Improve Patient Care is a coalition that was organized by Tony Coelho, who as some of you may remember, is the author of The American’s with Disabilities Act, but he organized this coalition in late 2008 to support these efforts towards a comparative effectiveness research institute. Next slide.

Sara van G.:                        During that time, what ended up first happening was that The American Recovery and Reinvestment Act, which some of us will remember as ARRA, provided $1.1 billion for a major expansion of comparative effectiveness research, and then called on The Institute of Medicine to recommend a list of priority topics to be the initial focus of a new national investment in CER. Some of you may remember that as it was that list on 100 projects that they recommended to move forward. Next slide.

Sara van G.:                        But in order for us to get to PCORI, there were politics that had to overcome from both the right and the left. On the right, a few Republicans warned that there was little to stop the federal government from using comparative effectiveness research results to average out which medications or treatments would achieve similar results for less cost. And when that happens, according to members like Senator Pat Roberts, it won’t be long before Medicare starts rationing care. Next slide.

Sara van G.:                        On the left, the challenge was that a lot of folks on the left wanted to establish this institute within AHRQ, to create a Center for Comparative Effectiveness, which would not necessarily be independent. It would allow for cost comparisons, and they also wanted for the institute to allow for use of cost effectiveness data, and for that data to then be translated into coverage decisions. Next slide.

Sara van G.:                        To marry these two positions from the far right and the far left, PCORI sort of emerged as a compromise and as something that everybody could support. Senator Max Baucus and Senator Kent Conrad banded together to create the Comparative Effectiveness Research Act in 2008, which evolved into the Patient Centered Outcomes Research Act of 2009, and it was passed as part of the Affordable Care Act as a compromise. Next slide.

Sara van G.:                        These are some of the key patient protections that are in the PCORI statute that as we move forward with reauthorization, folks seem very focused on protecting. First, it defines comparative effectiveness research as comparative clinical effectiveness research, adding that word clinical in there. Precluded PCORI’s research as the sole source of a Medicare coverage decision. The idea behind that being that we didn’t want to use one study to drive a coverage decision. They should always be looking at the breadth of studies in that area. Banned the use of quality adjusted life year measures by PCORI and in Medicare. For those of you who’ve never heard of the QALY, that stands for Quality Adjusted Life Year, and it is a metric that attributes a lower value to a year in the life of a person with a disability, or is perceived as also discriminating against seniors who have fewer life years left, so it’s a very controversial measure to be using.

Sara van G.:                        It also made PCORI an independent institute. It gave two seats to patients, one to consumers on the Board of Governors. Focused on patient needs, outcomes and preferences, and there’s a significant amount of language in the statute around subpopulations and the need for studies to be focused on subpopulations. It also provided support for patient and consumer representatives to be engaged and authority for advisory panels. Next slide.

Sara van G.:                        Over the last nine, 10 years, the real goal here too has been keeping distance from repeal efforts. Because PCORI was passed as part of the Affordable Care Act, there has always been a risk that PCORI could potentially be lumped into something related to the Affordable Care Act repeal and replace debate. In 2010, after PCORI was passed we spent all that time since then making sure that both sides of the aisle understand PCORI, sort of see what the value of PCORI, to try to mitigate against that. And we’ve been successful so far. Next slide.

Sara van G.:                        Here’s where we are today. PCORI is up for reauthorization. It’s a very exciting time. Their current authorization ends at the end of this fiscal year. We are expecting at this point for PCORI to be part of a health extenders package, including other programs that also require reauthorization. Senators Cassidy from Louisiana, Capito from West Virginia, Warner from Virginia, Van Hollen from Maryland are leading reauthorization efforts in the Senate. The wonderful thing about this is that it’s bipartisan. We are expecting a discussion draft from those offices in the very near future. We haven’t seen it yet, but we’re all waiting with bated breath. On July 17th, Congresswoman Degette led a committee markup in the house Energy and Commerce Committee of the Community Health Investment Modernization and Excellence Act that included a three year reauthorization of PCORI. As many of you may know, folks are really pushing for ten. I think that’s as much as they could get out of that mark. Then earlier on June 26th, the [inaudible 00:11:53] Committee passed the Protecting Access to Information for Effective and Necessary Treatment or Patient Act of 2019. That was a seven year reauthorization which is better, and it did include just some minor changes to PCORI’s national research priorities to include substance abuse, mental health, and maternal morbidity and mortality. Next slide.

Sara van G.:                        So leading up to this year and knowing that it was going to be a really big year, Tony Coelho and Dr.Phillip Gingrey got together and advocated for creation of this Friends of PCORI reauthorization coalition. The idea being that we wanted to include as many voices and as many organizations in this discussion as possible to make sure that PCORI was reauthorized with as little controversy as possible. We have convened a bipartisan large growing group of patient/family caregiver, provider, employers, payers, industry and research stakeholders that have an interest in supporting reauthorization of PCORI in 2019. We have I think at this point, and it’s growing so it could be more, but we have about 172 member organizations. Some of you may have seen that there was a letter signed by nearly 170 organizations to both House and Senate leaders supporting PCORI re auth. The main issues that were included in that letter was strong support for the funding mechanism to be 10 years. So, we’re still pushing for 10 years as it was the first time.

Sara van G.:                        And just pushing for PCORI to stay true to it’s mission of patient centered research and maintaining its mandate to comparative clinical effectiveness research. There are resources online if you want to go to reauthorizePCORI.org. And please, always feel free to reach out to any of us as well, because we’re happy to help with advocacy efforts. Next slide.

Sara van G.:                        This last slide just highlights some of the key issues that we are expecting to come up. Keep in mind that while the House made minimal changes, the Senate is expected to make some changes as part of their discussion draft, which is why we expect them to proceed in that sort of process to get feedback. The key issues that we’re expecting to have to debate are, representation on the Board of Governors. Right now the Board of Governors has 21 members that represent all the various stakeholder groups. Some from the payer community would like to have more representation on the Board of Governors. The length of reauthorization. So while we’re all out there advocating for 10 years and I think we’ll have a larger discussion about why that’s so important on this panel, but the length of reauthorization is going to be a big issue. Then in terms of comparative clinical effectiveness research versus cost effectiveness analysis.

Sara van G.:                        One of the main issues that was debated 10 years ago was whether PCORI would use that cost for quality to do a cost effectiveness analysis and I think we hope to not have to re-litigate that again. Then prioritization of high cost treatments. I think there are some who would think that PCORI should be doing more of that. Then dissemination and use of evidence. I think too, this next phase of PCORI what’s really going to be important is how this information gets translated and used at the point of care. I will turn it back over to the group for discussion.

Sarah  Dash:                       Great thank you Sara for that overview. We’re now going to turn to discussion and I’m going to ask Anand, you were in the secretary’s office at HHS I believe around the time that, that initial investment in CER was made. Tell us a bit about any kind of perspective you have from the formation of PCORI and lessons learned from what’s happened so far that you think apply to the current conversation.

Anand Parekh:                  Sure Sarah, and great to be with everyone on the phone today. Again, my name is Anand Parekh from the Bipartisan Policy Center. In a previous life, as Sara mentioned, when I was serving as Deputy Assistant Secretary of Health at HHS back in 2009, the Recovery Act as Sara van Geertruyden just mentioned, appropriated $1.1 billion and devoted it to comparative effectiveness research, and I was part of a group, HRQ got some money and IH got some money, but the office of the Secretary also received some money and then made some critical decisions about where those dollars should be spent and what the focus ought to be. I think my recollection back from that time, a lot of discussions were focused on what are the priority topics? Who are the priority populations? How do we make sure that we set up an infrastructure, a data infrastructure for CER? I was back then one of the early proponents that, even though we were just getting started, that we need to think of semination and translation from day one.

Anand Parekh:                  I think what that initial $1.1 billion [inaudible 00:17:31] back at the Recovery Act was jump start our thinking at the department about this. I think it also provided some foundational work for what would then after the passage of the Affordable Care Act a year later, the creation of PCORI, what would then become the function of PCORI. I think just looking back that was instructive for the department. I think it was instrumental for the department and even thought PCORI was of course an independent institute when it was formed in 2010, I think some of the work, particularly some of the research findings were helpful that PCORI as it started getting going.

Sarah  Dash:                       Great thanks. Tanisha I’d like to turn to you. You now lead FasterCures and you’ve been working on patient centered efforts for a long time. Tell our audience a bit about why do you think PCORI is important, and what do you think patients are looking for in this next round of reauthorization?

Tanisha Carino:                 Thanks Sarah. I’d like to thank the Alliance for the invitation to be here today and Sarah thank you for such a great set up. As Sarah mentioned FasterCures has been around for over 25 years, and we’ve had a unique mission that transcends any one disease area or treatment. And that mission has really been to identify and disrupt the bottlenecks that slow progress of medical advances reaching patients. A lot of the work that we’re known for has been in creating efficiencies and enhancing the network of disease organizations that are just getting on their feet, and have turned to us for counsel to connect with their peers and for resources.

Tanisha Carino:                 For those of you who may have missed it, this past Friday Marc Boutin, who’s the CEO of the National Health Council issued an op-ed in the Hill, and the punchline is that PCORI authorization should be a priority, and with the 10 years that we’ve seen in an investment is only the beginning of what really needs to happen to have a system that really is patient centered. When PCORI was first established, and one of the things that we’ll talk about is that there seemed to be this dichotomy between what is patient centered research, and what is comparative effectiveness research? Over the last decade what we’ve seen is really the convergence of the two, in that this idea of patient centered research has been infused across the entirety of healthcare. Whether it’s the FDA and it’s efforts around patient engagement in medical product development. Even values, that’s been organization [inaudible 00:20:09] who arguably are doing more and more to connect directly with patients and their consumers to better to understand what is really relevant to them, to keep them healthy.

Tanisha Carino:                 For us, one of the most critical aspects of PCORI is its relationship apt to the patient community. If you look at GuideStar their over 18,000 organizations listed in GuideStar that are focused on disease research, and although Research America said they’re only 3% of the total pie for research, everyone on this call could probably agree that they are critical and powerful [inaudible 00:20:44]. Over the last year FasterCures has done a survey of patient organizations to really better understand what they do in terms of investing in research, and how expansive that investment has been. As all of us can agree to, patient organizations play an incredibly unique role in a level of trust they have with their communities and their ability to be responsive. They fit in the middle of the ecosystems and are able to aggregate data across institutions that may not typically share data. They’re able to identify what type of data isn’t being collected by the traditional data collectors and researchers, and they’re increasingly investing in data resources and infrastructure to help [inaudible 00:21:29] investments that other researchers, as well as medical product developers could have in pursuing treatments in their area.

Tanisha Carino:                 One of the things I would reflect on in terms of the importance of the time frame for reauthorization is this idea [inaudible 00:21:43]. That PCORI plays a role in developing infrastructure and data, and that’s not a three year endeavor. PCORI’s already started in us, and in PCORI’s work, they funded 20 patient powered research networks that really were intended to pilot different approaches to building direct capacity around the patient organization. You can look at the work of Global Health and Living Foundation and the COPD Foundation, Epilepsy, to better understand the investment that they made with that funding to create data assets that could be mobile apps and are very unique to the needs and the issues they have. I do think that PCORI plays this role and increasingly becomes important for us to have patients as equal partners for all sorts of actors in the system, and PCORI really is one of the few independent funding sources that these groups can turn to.

Sarah  Dash:                       Great thank you so much Tanisha. I believe that Gail Wilensky has joined us.

Gail W.:                                I have

Sarah  Dash:                       Hi how are you? Thank you.

Gail W.:                                My challenge is getting in.

Sarah  Dash:                       No problem. We are talking Gail, about sort of the foundation of PCORI and you were one of the people who really jump started the conversation more than 10 years ago about the need for an entity to look at comparative effectiveness research. I wonder if you could just briefly give us your thoughts on lessons learned from the founding of PCORI as they relate to current conversations.

Gail W.:                                Thank you. I want to explain why as somebody who doesn’t come out of a comparative effectiveness research, or a biomedical research background got so focused on comparative effectiveness research. The reason was an acknowledgment that in the United States, if we’re going to be able to effectively slow healthcare spending and improve the value for what we get, we have to find a way that will work for us. And to me that meant getting better information about what works when for whom under what circumstances, and then making sure we had the right financial incentives in place for these to be used and adapted by patients, insurers, their physicians, etc.

Gail W.:                                That other countries use other much more directed ways frequently, global widgets, etc., but that, that is not going to be a stable solution in the United States. My normal focus is on the financial incentives that need to be placed, and of course I still worry about that, but it became clear to me that the basic information that is what works when for whom under what circumstances, and not just limited to pharmaceuticals as they frequently are in other countries, but to include devices and surgical procedures as well is the critical foundational knowledge to make all of that work. That kind of basic knowledge is about as close to a public good as I as an economist, public finance economist at that, can think of. That is once it’s there you don’t want to have people having to reinvest in order to get the same information. You don’t want to block them out, but it takes both time and resources to generate that information, and it needs to be done in a really credible way. That meant public funding at least in part, but also being done in a way that would be above suspicion.

Gail W.:                                To me a PCORI which is a quasi-public, quasi-private, that uses monies from fees and from government sources and for the most part farms out the actual research to the usual academic research areas to be done, really does embody what I think we would need in the U.S to have credible research collected. If the research is regarded as tainted or biased toward the payer, it will be very hard to have it adopted, particularly if it’s controversial in any way, and frequently these comparative effectiveness research findings have the potential at least to be controversial. From someone who liked the old ways we did things without too much information. It is why PCORI and what it stands for is so key as a component to learning how to spend smarter and spend better.

Sarah  Dash:                       Thank you so much Gail for that overview. I want to ask because we’ve all talked a bit about the research priorities. Sara van Geertruyden you mentioned that originally there was an ION study around the research priorities, that there are some perhaps tweaks coming in the legislation, so let’s talk about that. What research has PCORI done and how has it been used? Do you want to start us off Sara, and then I’m going to go to Anand.

Sara van G.:                        Sure. I think one of the wonderful things about PCORI, and if you look at the statute, the way that they actually defined comparative clinical effectiveness research and the areas for PCORI to prioritize it’s really broad. I think that, that was done purposefully by Congress to make sure that they weren’t just researching drug A versus drug B, but also getting to what Gail talked about, looking at devices, looking at treatment pathways, looking at delivery systems. The goal being how do we use this information to improve how we deliver healthcare? Now I do think that at this stage there will be a debate in Congress about whether to increase PCORI’s prioritization of things that are high cost, which I think is a debate that folks are willing to have, but I think getting down to it, PCORI has done some impact studies that really are showing that if the system… If there was uptake of those findings. For instance one of the studies is children with oral antibiotics versus IV and the study showed that oral antibiotics work just as well. There are several studies, and I think PCORI should be doing more of this, but there are several studies that show how much we could be both improving and lowering cost in our health systems, by improving quality of care, by using those studies as the outcomes from those studies.

Sara van G.:                        We’re excited and I think it really boils down to trust, and it’s because PCORI has patients engaged in the prioritization process, the agenda setting process and then within each project there is patient engagement, and that leads to trust in the findings so that hopefully in these next 10 years we can really advance dissemination and use of that information at the point of care. But trust is a really big part of it, and it requires that PCORI be focusing on things that matter to patients when they’re making healthcare decisions.

Sarah  Dash:                       Thanks Sara. Anand, talk a bit about the research prioritization process and maybe what’s the timeframe for some of this research?

Anand Parekh:                  Sure. I want to just echo everything Sara just said. I think her points were so critical. I think it’s fair to say that over the last 10 years, it’s really been in the last few years where all of the work and the labor is now resulting in results that can be disseminated and translated, so I think many of us have been waiting for a time when some of these results can be communicated to stakeholders and as Sara mentioned the example of kids and oral versus IV antibiotics, there are additional studies just like that. Another one focuses on a simple decision making tool for people who go to the ER with chest pain, but who are at low risk for a serious heart problem. PCORI funded research found that with shared decision making between a patient as well as a provider, using that tool cuts hospital admission rates, keeps people at home, saves billions of dollars over five years. It’s just an example of patient centered research that is now coming into fruition, is being disseminated, translated and the opportunities for adoption are immense.

Anand Parekh:                  I think from a timeframe perspective Sarah, I think we’re going to increasingly see more and more research, the transparent way be disseminated, I think adoption by the healthcare sector with patients and at the forefront I think is going to be critical. There are issues as you all have mentioned that Congress will look at. Should PCORI invest more in behavioral health? Should it focus more on high cost illnesses? Should it focus more on head-to-head trials? I think those are important issues. I would probably say that I think PCORI’s already thinking about this, and has over the last several years, if you look at their track record, have started to move in this direction. I think everyone is really on the same page. There are issues related to reauthorization which I’m sure we’ll get to, but I think the most important point is PCORI needs to be appreciated in a bipartisan way, and reauthorized and supported in a bipartisan way.

Sarah  Dash:                       Great thanks. Let’s get to this question. The issue around high cost treatments have come up a couple of times. There are some stakeholders who argue that we’re really not receiving enough ROI if you will on the studies that PCORI has funded to date. Others who argue that perhaps there hasn’t been enough time to derive valuable insights from these studies. Is there a balance between these two ideas and what does it mean to say invest in higher cost treatments. Tanisha do you have thoughts on that?

Gail W.:                                I just want to make one clarification please, before we proceed with this part. And that is it ought to be termed high cost/high volume. You need to be careful. This is certainly true in the Medicare program, but you need to be careful that you also pick up some of the medium class but very high volume procedures or interventions because it really is that combination that designates where you want to make sure you’ve invested money in trying to find the best strategies for particular subsets of the population, or provider types. Just wanted to have that clarification thrown in.

Sarah  Dash:                       I appreciate that clarification, thank you. Tanisha?

Tanisha Carino:                 I think Gail makes a great point, because it’s not just high unit cost, it’s high volume, but it’s also where there is real uncertainty for what the patients and the physician community want to do. Or even the payer community by in large. I also would just encourage… The idea of dissemination and impact of the research, it doesn’t just begin with how you communicate it, and to Sara’s point, it also begins with the ability for PCORI to work collaboratively with the patient organizations, with other audiences that it believes it needs to have strong relationships with. It disseminates the work, but you’re really just nailing down what are the top of my issues that are coming up? And the last thing I’ll say is, a lot of it is really about timing. We don’t have even uncertainty about questions. A lot of times we have uncertainty for things that are new, things that haven’t even come to market. This is increasingly where we’re hearing, a lot of questions as to what’s the science behind what we see coming through the various exciting pipelines across the industry? I do think that in addition to this idea of what you study, it’s really the relationship you have to distill that down and the timing of that research, which really speaks to the kind of research you can fund for it to have relevance and impact.

Sarah  Dash:                       Great thank you. Let’s talk for a minute just about just the funding source just as a point of perhaps education and conversation. Right now PCORI is funded through as Gail said a mix of public funds, Medicare, general revenue, as well as a tax on insurance plans. Is there any discussion right now about that funding source? About changing it? And what does that conversation look like? Sara do you want to start us off?

Sara van G.:                        Yeah I’ll start thinking about my answer. I do think that from the payer perspective there have been some discussions. I would also though note that at least as far as I know from the organizations that are part of [inaudible 00:36:07] reauthorization, most stakeholders support the current funding stream. It will be debated, and it will be a hot topic, but I think the ideas around changing the funding streams are coming more from the payers and I don’t think that it’s really coming from anyone else.

Gail W.:                                I mean you could make an argument that this is a public function, but you can also say that by hitting the payers it will be passed on, that’s the expectation. I think in this case it is the reality, which in a messy way means that basically a combination of those who are insured and those who in any way make use of the healthcare system, which is most of us at some point, plus through the public sector will end up paying. I don’t think it is worth the fight of trying to change the funding source and trying to explain that it’s hard to track who ends up actually paying when you have this particular mix. It’s worked over the first 10 years and I guess that’s good enough. I wouldn’t want to take that on as a fight.

Sarah  Dash:                       Okay, let me ask Gail, there’s actually an audience question that is for you and of course others would love to hear from as well. The question is kind of getting to hear of one of the controversies, which is what do you see as implications for not adding cost analysis as part of PCORI’s mission? Can you say a little more about that?

Gail W.:                                Sure. As you may have guessed, I have thought long and hard about this and also spoken and written about it. The expensive time consuming hard part of comparative effectiveness research is getting the basic information about what works where, for whom, under what circumstances. The costing out what that means in dollars is a relatively quick and not costly, although there are some challenging issues like what discount rate do you use? That is what interest rate do you use, if it’s something that as a benefit or a cost over time, that usually will swamp a lot of the answer. It is basically the quick not expensive part once you have the basic comparative effectiveness research. I think as a politically expedient matter, and as a practical matter, there is not a problem in having the public and the private payers doing their own cost analysis. Once they have good reliable, unbiased, believable numbers, information about the comparative effectiveness research. Yes you do at some point want to bring in the cost information, but you can do that, CMS can do that for Medicare. The state’s could do that for Medicaid. The private payers can do it when they make their medical coverage decisions if they have that underlying comparative effectiveness research information.

Gail W.:                                I would say that it needs to be done, it just doesn’t need to be done by government because it is both quick and not a costly calculation once you’ve got the basic information. I don’t think that is worth the kind of fight that would result when you have members of Congress or members of the public getting very upset with the thought of who’s calculating the [inaudible 00:40:20] and whether, or not those are quality adjusted life years and disability adjusted life years for those listeners that don’t spend their lifetimes worrying about this.

Gail W.:                                You can calculate that once you have the base information. So I don’t think that’s where you want to keep your attention, and if it allows us to go forward and gives the base information for other payers to use, public or private, that’s all that counts.

Anand Parekh:                  If I can add to Gail’s comment. I agree with her. I think the conversation about cost needs to be shifted from the rationing concerns of a decade ago, to a conversation focused on transparency. The need for more transparency in healthcare is the bipartisan goal. Transparency with both quality and cost information, patients and consumers desire both. I think what has happened is the debates from a decade ago really led to a lingering, chilling effect. I think amongst both the research community and PCORI about how to address cost, but if you look at the statute there’s nothing that says that PCORI can’t think about cost when it identifies research priorities. There’s nothing in the statute that says it can’t ask researchers to provide cost information on intervention, which is different from doing cost effectiveness research. There’s nothing in the statute that says HHS can’t use the findings in its coverage payment reimbursement determinations. It only says the research can’t be the sole basis for determination. I just think there has been an extreme caution that’s been taken not to touch cost because of the conversations from a decade ago and I think honestly that’s hampered the ability for all the stakeholders who address this issue in an ideal way.

Anand Parekh:                  I agree with Gail, I don’t think we need to bring into the discussion during reauthorization the cost effectiveness analysis piece into this. I think there is enough in the statute that we can begin to address cost as a society, as we should, but I think that chilling effect needs to be removed and I think people need to think about this more from a transparency perspective.

Gail W.:                                And people of course, people on Congress would have to grant CMS Medicare the right to set reimbursement using comparative effectiveness research if it were even to be considered as appropriate, and not necessarily for coverage. I think that ought to be safety and efficacy, as it is now. I do think it is frustrating that Medicare, which has severe funding challenges in its next decade future, can’t look at comparative effectiveness research and set reimbursements according to those findings, but it doesn’t have a statutory authority to do so. It’s a separate issue and I think it’s something that has potential, again not as a coverage issue, but as a reimbursement issue on the grounds of why should you pay more if it doesn’t do more? But that has been a hard sell as well.

Sarah  Dash:                       I want to ask just one more along these lines and Tanisha, you work with many, many of those 18,000 patient groups in the country. Is there the sense that if you refocus the conversation around cost to listening to what matters to patients, that you actually get more effective higher quality care that meets their goals and perhaps that is a way to, I think as Gail said, spend smarter because you’re spending on the patient priorities. Can you say just a bit more about that?

Tanisha Carino:                 Yeah. No, I think that it’s undeniable that affordability and cost are at the top of every families mind as it relates to healthcare, but when we dig into what it is they actually mean, they’re typically talking about their out-of-pocket cost, or their non-medical costs. Like the time it’s going to take to get to their doctors appointment, their travel cost. These costs that have a huge impact on how patients feel confident and ready to actually pursue their care. Cost definitely comes into play, but I think that as you get closer to what patients and their families really care about, it’s really these out-of-pocket costs. And sometimes they talk about if they do pursue a certain treatment, the cost that could be avoided.

Sarah  Dash:                       Great thanks. We have about 12 minutes left in today’s webinar. It’s going really fast. I want to ask just again, I think we touched on it a bit, but it’s one of the biggest conversations that’s happening right now is around the length of PCORI reauthorization. We have a bill for a three year, a bill for seven year reauthorization, and of course the original was for 10 years, so can we chat for a few minutes about the practical implications of three versus seven versus 10? Are we ourselves up to another similar conversation in whatever length of time it is? Anand do you want to start us off?

Anand Parekh:                  Sure I can just make three quick points. One is I think of course the longer the length of reauthorization it sends a message about our values and what we prioritize as a nation, as well as our health policy priorities. The longer the reauthorization, is a signal, it’s a marker that this is important. The second is just a statement that I do need to make that we never have these conversations when Congress comes to discussing NIH’s nearly $40 billion annual budget focused on basic translational traditional clinical research, but patient centered outcomes research is simply an extension of that continuing medical research and there needs to be a similar appreciation. I would argue that if Congress doesn’t invest in areas such as CER or patient centered outcomes research, it undercuts the much larger $40 billion annual investments NIH is making, because they inevitably delay the delivery of the right care to the right patient at the right time. I think just the fact that we’re having these discussions, should it be three years, seven years, 10 years, when we don’t have it for our broader medical research enterprise, I think we need to move past it.

Anand Parekh:                  I think the last point I’ll just make from a researcher perspective. If you’re a young academic researcher out there and you’re looking for funding sources, if there’s a source that is not stable, or is short term or might only be there for one year or three years versus a source of funding that you know is going to be there for 10 years or longer, you’re always going to opt for the latter, so it’s really important for the actual researcher community doing the work that they see that there is a commitment to this type of research, because that’s where they’re going to ground their career. That’s the career decision they’re going to make in terms of what grants to apply for, what to focus on. So I think looking at it from the researcher perspective as well as many other perspectives, but particularly from a researcher perspective, the longer the reauthorization the better.

Gail W.:                                I agree and I think three, because of the whole contracting process that gets involved for researchers, in terms of doing their work, would be unfortunate but better than not reauthorizing. Seven would be terrific. 10 is obviously even longer, and has some appeal, but I think if it’s at all possible to get beyond three it would be very helpful for the reasons Anand just indicated.

Tanisha Carino:                 I would agree with everything that’s been said. When you think about three years, to get a contract and to see results on a three year basis, those are very short term research engagements. Now I’d also argue to say that we have a lot of other alternative sources for that type of short term research. There are plenty of private organizations that do very quick comparative effectiveness reviews of systematic evidence and of the evidence that exists and I think that what we’ve all seen we need more of is actual, true, novel insights and evidence generation much less infrastructure that still needs to be built. It’d be very difficult to see how that could be built on a three year basis.

Gail W.:                                Yeah I want to again emphasize that it’s really important that we make investments in surgical procedures and medical interventions. There is far more, not enough comparative, but far more information available about pharmaceuticals and to a lesser extent on devices, especially in basic devices. There is precious little that does effective comparative effectiveness research on procedures that involve medical or surgical interventions in comparison to other ways of intervening, and a lot of money is spent in this area and it’s extremely important to remember this is not just about drugs and devices.

Sara van G.:                        I would just say from an advocacy perspective, a long reauthorization is a priority for Friends of PCORI reauthorization. So all of those organizations and stakeholders who have joined Friends of PCORI reauthorization. For them, this is one of the big issues. They’re pushing for 10, and so seven is certainly better than three, but I think you’re going to see these groups continue to push for 10.

Sarah  Dash:                       What are the arguments for three or seven or less than 10 year that you’ve heard?

Sara van G.:                        The three and the 10 is what has come up on the House side. So you had the three year reauthorization in Energy and Commerce and the seven year out of Ways and Means, those two bills will have to be reconciled. On the Senate side, we’re still waiting to see what’s in the discussion draft. It is unclear to me whether it will be three, seven or 10. Our hope is that it’s at a minimum seven, but we’re still advocating for 10.

Sarah  Dash:                       Okay. We just have only about five minutes left, which is amazing to think. So I want to ask this next question about other reauthorization concerns or implementation ideas. One of the things that was brought up was the representation on the Board of Governors, but as each of you kind of looks forward to the next phase of PCORI, assuming it is reauthorized, what do you think are the most important implementation priorities from the get go? Tanisha can I start with you on this one?

Tanisha Carino:                 Sorry I lost you for just second Sarah, maybe you can start with someone else first, sorry.

Sarah  Dash:                       That’s totally fine. Implementation priorities from day one. Gail do you want to take a stab at that?

Gail W.:                                It is looking at the 10 to 20 largest total cost areas that we are spending money either in the public programs or in general. They will probably be similar except that there will be some orientation toward diseases and interventions of the over 65 population that will be slightly greater in CMS and public programs, but that’s where the heavy use of healthcare comes in. Really look to see the areas in which there is debate about the best way to go forward. Some areas, even if they’re expensive, leave very little debate in the medical world about how to proceed. It is focusing on those areas frequently related to cancer, particularly in terms of some of the very high cost therapeutic interventions that are being developed to try to understand better the likely outcomes. Not that we’re likely to deny them, but we need to know something about their implications and especially it would be helpful in being clear about who should get priority access to some of these very new, expensive biologics and other interventions. We’re not a country that tends to say no in terms of making expensive interventions available. We do have the option if we ever chose to use it to make it some more expensive if there’s little or no likelihood of benefit to someone with a particular medical condition. Especially if there are other interventions available.

Sara van G.:                        This is Sara and I would also add I think from the patient perspective, putting my [inaudible 00:54:54] hat on, one of the priorities that we have always had is that the information from PCORI then be translated into effective decision making tools, decision aids, things that can be used to make that information usable at the point of care. That’s I think something that over the next hopefully 10 years we’d like to see become a big priority is how you take all of this information you know about a particular condition and translate it into tools that patients can then use to get to that right care, right patient, right time. Because different patients will react differently to the same treatment, and the benefit of PCORI’s work is that hopefully we’re getting to the bottom of why people react differently to different ways of being treated so that they can then discern at an earlier stage which one would work better for them.

Sarah  Dash:                       Thank you Sara. Tanisha or Anand do you want to give your final thoughts?

Tanisha Carino:                 I’ll keep mine brief. I think like any investment, investing in infrastructure is critical because it pays dividends down the road. And when you think about where there are other capital sources, we have a real big gap is actually investing in things like patient registries that are run by the disease foundations in which patient communities trust. So that’s where I would encourage PCORI is really helping the patient community build the infrastructure they need to continue to answer the questions and conduct research that is relevant to them down the road.

Anand Parekh:                  This is Anand, I’ll just add by saying I think my mantra’s the same as it was 10 years ago, I think dissemination and translation has to be a key priority. Certainly patients want this, healthcare professionals need this, surely our healthcare system needs this. As we move slowly, every so slowly, as we transform healthcare from a volume based to a value based system I think making sure that we can point patients to interventions that are the most effective for them, not just what’s the newest or what’s the shiniest, but really true innovation and what works for patients is what we all want, so I think disseminating and translating the research findings so that they can be utilized, adopted by all stakeholders is absolutely critical.

Sarah  Dash:                       Great thank you. Well you’ve all given a really important charge for the next phase, and I want to thank you. Unfortunately this is all the time we have on our webinar today, but I want to thank everyone who is listening for joining us, and I encourage you again to take a look at allhealthpolicy.org where we will have materials as well as contact information for people you may want to reach out to for further information. Please take a moment if you will to complete the brief evaluation survey that you will receive immediately after the broadcast ends as well as via email later today. And speaking of patient centered, for those who are in the Washington D.C area, please do save the date for our next in person public event on July 29th, which will focus on what is next for patient experience measurement. And join our list if you haven’t already done so to hear about the next webinar and future health policy educational events. Thank you again to our panelists for joining today’s webinar, and have a wonderful day. Thank you.