At its September 2020 meeting, PCORI’s Board of Governors approved the release of proposed Principles for the Consideration of the Full Range of Outcomes Data for public comment. This is one component in PCORI’s plan to implement its new statutory mandate to consider the full range of outcomes data in PCORI-funded research. Comments submitted online during a 60-day public comment period, from September 15 through November 13, 2020, are posted below.

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Comments Submitted Online

Submitted

9/15/20 14:17

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

While further discussion is in the following paragraph's, recommend adding the word "meaningfully" before the word "engaged" in the first sentence of the 2nd paragraph to add emphasis. Further definition of what constitutes "meaningful engagement with patients" with examples should be added as a third paragraph to ensure clear understanding by all stakeholders what PCORI expects and how/why it is important to the research.

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

No comment

Principle 3: The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.

No Comment

Principle 4: Beyond the collection of burden and economic impact data, PCORI may support the conduct of certain types of economic analyses as part of a funded research study, to enhance the relevance and value of this information to health care decision-makers.

This phrase is confusing " prohibitions against research findings that include practice guidelines". Does this mean a prohibition of using practice guidelines, recommending changes to guidelines, comparing guidelines, understanding evidence are often used to inform guideline decisions.

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

Advantages: -provide relevant data to help patient advocacy organizations, policy and coverage decision makers to recommend/promote less costly/burdensome treatments -allow end-users to develop and use models based on unique local/regional/ethnicity/cultural characteristics to customize population-based decisions -more suitable for common conditions where heterogeneity of treatment effects can be clearly delineated, such as in large pragmatic trials where recruitment of diverse patient population is assured Disadvantages: -may erode individualized patient-centered decision making in favor of economics if the financial impact is large -may erode doctor-patient relationships and their satisfaction unless there is robust prior engagement in planning, executing and dissemination of the research

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

None of the analyses described are useful by itself. It may be meaningful based on the specific intervention(s) under investigation. Decision models which included study evidence and a base-case, and which allows end-users to modify inputs (including costs) based on local/regional/individual characteristics will be meaningful.

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

Overall, the draft is excellent. It is clear and examples are good. I agree with expanding collection of full range of outcome data. Costs can be tallied within the study to be used in the analyses for the base-case as part of the study report(s). However, as costs varies widely and change over time, publication(s) must include disclaimers to that effect. I would like to see models being developed using study results that are available to skilled organizations upon request to conduct customized analysis. It should only be done in large pragmatic studies where HTE are taken into consideration and decision models be provided to help clinicians/patients to make individualized patient-centered decisions.

Name and Organization

Matt Cheung, Ph.D., R.Ph., University of the Pacific School of Pharmacy

Community

Patient Advocate


Submitted

9/15/20 16:27

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

I think it's great that PCORI is aiming for cost/economic impact, since such considerations are so crucial to patients and families and because economics confers huge disparities in health and its outcomes.

Name and Organization

Harold Lehmann/Johns Hopkins

Community

Researcher,


Submitted

9/25/20 11:12

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

I would submit that included in this is an analysis of expedience of treatment start. We have noted that getting approval for proton therapy has taken significantly longer and with more staff and physician time to get approval. This has resulted in referring physicians to specifically ask that patients not get protons unless crucial.

Name and Organization

anonymous

Community

Provider,


Submitted

9/29/20 13:46

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

Dear PCORI: The Caronavirus Pandemic has forced everyone to accept a broader range of outcomes to data. Social and cultural components must be appreciated. Social distance has limited interpersonal relationships and contingent outcome data. Even religious activities are limited. Outpatient treatments limit doctor-patient relationships Best! Louis Zrebiec,Ph.D Double Minor in Three Major Disciplines [email protected] [email protected]

Name and Organization

Retired

Community

Consumer,Researcher,


Submitted

9/30/20 11:28

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

I am absolutely thrilled to see that PCORI is considering changing its position on including costs as an outcome. There is little more patient-centered than this, particularly the way that cost outcomes are being proposed. The direct costs to patients (in terms of insurance, co-pays, time away from work for both the patient and the caregiver, etc.) as well as the indirect costs (e.g., costs to hospitals that may result in hospital closures in rural areas; differences in costs by insurance type) are critical to patients, too. I think looking at changes in potential earned income may be important, too, particularly for caregivers... I was set to start a new career when I had my premature baby (Expected starting income $75k/year) but had to go part-time instead to take care of him and get him to all of his doctors/therapy appointments (current p/t income $25k/year). I applaud PCORI for expanding outcomes to include costs, both direct and indirect!

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

I agree with this whole-heartedly, as long as these outcomes are looked at in terms of their ultimate impact on the PATIENT (see comments above). Costs to providers, hospitals, insurance, etc. all indirectly impact patient costs and care and should absolutely be measured and evaluated. But keeping the ultimate focus on the PATIENT is key, and the ultimate value that PCORI brings to the table.

Principle 3: The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.

Agree!

Principle 4: Beyond the collection of burden and economic impact data, PCORI may support the conduct of certain types of economic analyses as part of a funded research study, to enhance the relevance and value of this information to health care decision-makers.

Whole-heartedly agree. Excluding cost from the evaluation of intervention effectiveness is a key missing piece from current PCORI research.

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

The benefits are overwhelming. My only concern, as stated above, is that it may shift the focus away from the PATIENT. All outcomes--including economic--need to continue to be tied to how it benefits the patient, either directly or indirectly.

Name and Organization

Anna Johnson

Community

Caregiver,Researcher,Stakeholder,


Submitted

9/30/20 11:30

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

I would suggest a stronger stance on cost-effectiveness and would not like to consider it out-of-scope

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

I really like everything as is, with the addition of the cost-effectiveness comment above

Name and Organization

Karen K. Giuliano, PhD, RN, FAAN, MBA

Community

Researcher,


Submitted

9/30/20 11:31

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

I appreciate the term "economic impact" more than the term "cost". The "economic impact" includes, but is not limited to "cost". Economic impact may also include "opportunity cost". I believe PCORI and patient-centered outcomes research should have latitude to explore and integrate this element into research and informed decisions. Therefore economic impact should include both treatment cost and opportunity costs embedded in (a) treatment option(s)

Name and Organization

Matthew F. Hudson

Community

Patient,


Submitted

10/3/20 16:33

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

1 How to equate the different burden using a economic scale? 2 For research that certain group of trial participant has a well defined economic burden, would funds be provided in the project funding?

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

If the study result could not be implemented due to the extra cost of care. For example, for a study of healthcare of homeless population, extra cost of social care would probably help in the health of homeless population. The funds for the extra social care is probably not be available from the healthcare provider system.

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

How are cost of labor from different region of the country be incorporated? For example extra labor cost in caring for elderly patients can vary a lot for different region of the country.

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

It would be interesting to have a economic value equivalent for different burden for the patients especially for CER that constitute 2 very different intervention with 2 very different burden.

Name and Organization

Ting Pun PCORI ambassador

Community

Caregiver,


Submitted

10/7/20 19:40

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

Re: proposed “Principles for the Consideration of the Full Range of Outcomes Data” The Society of General Internal Medicine (SGIM) is a member-based internal medical association of more than 3,300 of the world’s leading academic general internists dedicated to improving the access to care for all populations, eliminating healthcare disparities and enhancing medical education. SGIM's mission is to cultivate innovative educators, researchers, and clinicians in academic general internal medicine, leading the way to better health for everyone. The members of the Society advance the practice of medicine through their commitment to providing comprehensive, coordinated and cost-effective care to adults, educating the next generation of outstanding physicians, and conducting cutting-edge research to improve quality of care and clinical outcomes of all patients. SGIM supports the proposed principles, specifically the addition of economic analyses to clinical studies. For many patients the costs and economic consequences of their medical care are important considerations as they work with their physicians to choose treatments and evaluate the likely outcomes of those choices. Including these elements in PCORI-sponsored studies will allow the portfolio to be more patient-centered over time by providing patients and other stakeholders with rigorous analyses of these highly relevant outcomes. We understand that the original statute supporting PCORI continues to restrict support for cost-effectiveness analyses or the use of quality-adjusted-life-years as outcomes, and that these proposed principles would not allow for economic outcomes to be the primary outcomes for PCORI-supported research. Nevertheless, the principles as developed will improve what is already a high-quality portfolio. We would suggest that PCORI add economic elements to the current methods portfolio, seeking to support studies of the most accurate ways to assess cost, economic burden, or economic benefit in a patient-centered manner. These new studies would be consistent with PCORI’s revised mandate and could also yield a better understanding of the types of economic analyses which PCORI will support in the future. The Society of General Internal Medicine, which advocated for PCORI during its initial ten-year authorization and, again, in its recent reauthorization, is pleased to support these principles and pledges to continue to work with the Board and staff to advance patient-centered research in the years ahead.

Name and Organization

Dr. Jean Kutner, President, Society of General Internal Medicine

Community

Provider,


Submitted

10/14/20 13:27

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

Patient burden: Data on re-admission rates is also important to consider in this principle, as patients who continually are re-admitted to the hospital are at greater risk. Including patients in the research is critical, it is imperative that researches find creative ways to truly engage residents, having them participate in one thing, but they must have a voice at the table so their opinions must have a higher weight.

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

Readmission rates should also be an indicator in this principle. An important data element to evaluate in stakeholder outcome is patient engagement, - how are the providers able to engage each patient in their own care, so that the patient is better prepared to continue their care and thereby reducing cost of care.

Principle 3: The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.

Since patients should be involved from the onset and to develop the research question, adding a certain % of patient participation in the process would ensure more meaningful participation.

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

There is high value in these requirement as it provides a more holistic approach to looking at the outcomes and ensures others are considered. It is not only the financial outcomes for stakeholders, but more importantly is looking at the patient and system outcomes. This data should be evaluated in all studies.

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

Utilization should consider hospital days but also readmission rates - # of days to readmission

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

The document provides lots of very important data, this is my first time doing these reviews and feedback and found the document difficult to read and difficult to understand the role of a reviewer. Seems to be written for clinical/research staff and not for patient or advocates.

Name and Organization

Margarita Holguin

Community

Patient,


Submitted

10/15/20 13:28

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

All of the principles use the term "may" implying that capturing data on burden and economic impact are optional. Is the intent to make this optional or required? There are many advantages to making it optional, but making it required may pose a problem for researchers who already have a very wide array of stakeholders involved in a study. For example, a PCORI project that requires collaboration across several disciplines may be maxed out in terms of cost and complexities, and adding a required burden and economic impact assessment would be problematic.

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

Great idea to allow limited assessment of economic impact in PCORI studies. Not a good idea to require this in every study.

Name and Organization

Eve Carlson, Ph.D., National Center for PTSD, Stanford University School of Medicine

Community

Researcher,


Submitted

11/10/20 16:04

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

Regarding "a requirement for all PCORI-funded research to capture..." - While I definitely appreciate the sentiment, I do not support ALL researchers attempting to capture this type of data. Researchers with no experience evaluating costs should not be required to add a cost analysis on top of their existing methods if they are not trained or able to do so. Those who explicitly aim to capture cost information should demonstrate expertise to do so appropriately. When is the capture of this data not helpful? - When the data sources and costing methods are so biased that it doesn't actually help advance what we know about economic impact. For example, many researchers estimate drug costs using "list price" or "wholesale acquisition costs" (because it is easily accessible) - however this type of data source for a drug price is systematically biased by overestimating the true cost of the drug on the health system.

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

In terms of types of analyses - I recommend PCORI specifically consider multiple types of quantitative "cost-of-illness" methods using both real-world data as well as economic modeling (e.g. microsimulation, Markov process, cost-minimization analysis). Further, I think PCORI would benefit from seeking health economics experts to help establish best practices for cost evaluation in PCORI-funded studies. Additionally, PCORI should consider qualitative methods where researchers use in-depth interviews, focus groups, and Delphi panels to capture broader themes of types of burden and economic impact data. This type of work can be very informative to understand all the types of costs experienced from the patient or other stakeholder perspective. For example, we worked directly with Hepatitis C patients to better understand the way patients actually prioritized variables (including costs) when making the decision to be treated (Mattingly et al. The Patient. 2019;12:631-8.). Finally, the use of systematic review methods for clinical and economic burdens of disease can be very helpful as well and can be done relatively cheaply (Here are two open-access examples we've done in Lyme disease and HCV in the past year: Mattingly et al. Parasites & Vectors. 2020;13:341; Mattingly et al. Pharmacoeconomics. 2020;38:927-939).

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

Overall, I applaud PCORI's initial draft for these principles and I am excited to continue supporting the mission of the organization through my work as an advisor and researcher. This is a major step in the evolution of PCORI and it will really help patients be brought into the broader discussion of "Value" in health care.

Name and Organization

Joey Mattingly, PharmD, MBA, PhD; Associate Professor; University of Maryland School of Pharmacy

Community

Researcher,


Submitted

11/13/20 0:15

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

Perhaps, "patients and their families" rather than "patients" alone to acknowledge that some burdens are felt as much on families as patients themselves. I am thinking specifically of issues/topics related to children or adults for whom family members are primary caregivers.

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

This is a nice explanation of the principle. No comments.

Principle 3: The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.

What if patient stakeholders identify cost as a relevant outcome for a specific study? Is that sufficient enough information to include cost as an outcome? Is PCORI considering guidance to research teams on "the feasibility of capturing" burden and economic impact data?

Principle 4: Beyond the collection of burden and economic impact data, PCORI may support the conduct of certain types of economic analyses as part of a funded research study, to enhance the relevance and value of this information to health care decision-makers.

As appropriate, can research teams propose subpopulation analyses of economic impacts as well? To what extent is PCORI considering the contribution/association/confounding of health equity considerations when considering economic analyses?

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

Potential disadvantage: How to account for systemic health equity challenges experienced by different groups of patients? Would an economic impact analysis effectively be interpreted the same for one population in one part of the country as it would for another population in another part of the country? Potential disadvantage: As noted, data collection limitations can be a disadvantage. Research teams may find themselves having to impute information on burden from biased or incomplete literature, casting potential doubt over findings. (I'd encourage PCORI to consider as stringent a stance as possible to ensure study findings are not misinterpreted while also being realistic about the likelihood of data limitations so as not to deny the opportunity to study economic impacts in a widespread way. It's a fine line to tread.) Advantage: Economic impacts/burdens are relevant to all PCORI-relevant stakeholders given that many health care decisions are made based, in part, by economics/costs. Types of studies: (1) When the options being studied have similar economic costs (dollars), a study of other burdens will not overwhelm other stakeholder-relevant outcomes. (2) When the collection of economic impact or burden data is not overly complex or has more than minor limitations. (3) When there are considerable ethical concerns relevant to one or more of the treatment options (particularly withholding treatment) such that a study of economic impact or burdens leads to a potentially controversial tipping of scales. (In this case, I guess, it does contribute to overall importance, so the opposite would be true...no ethical concerns.) (4) When the primary stakeholders that benefit from an economic analysis are patients themselves.

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

Costs relevant to the intersection of school and health. For example, missed days of school among children, young adults, etc. due to illness. As appropriate and measurable, other metrics relevant to schooling. Perhaps, challenging to quantify in "dollars" but burdensome nonetheless. Quantifiable in dollars perhaps via missed work by parents or guardians. Some studies might also include a time component relevant to burden. Thinking of time required to travel versus, perhaps, not traveling and utilizing telemedicine. Would be interested in broad consideration of potential costs and benefits to public payers as these costs are ultimately societal costs (via taxpayer dollars).

Name and Organization

Anonymous

Community

Caregiver,


Submitted

11/13/20 11:19

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

Employers, health plans and other sponsors make decisions that directly impact patients and patient-outcomes. If patients need certain information to make better choices, that information should also be accessible to those stakeholders (e.g. employers and other plan sponsors) who make critical healthcare decisions in how to provide plan coverage for patients and advocacy and support to guide patient decision-making. Although the idea of “consumerism” is to create an empowered individual who can make their own healthcare decision, in reality patients and consumers rely heavily on the support and coverage provided by employers. The complexity and diversity in healthcare makes this support essential for patients to navigate the issues they are presented with. When looking at patient out of pocket costs, there is a need to address the economic and environmental issues regarding low-wage workers – as a subset of a population. Their needs many times are overlooked and research in this area would greatly support employer healthcare planning. This premise also does not acknowledge the broader economic impact on employers and employees related to total cost of care, not limited to the cost-sharing. Although patients have out-of-pocket expenses at the point of care, employers and their employees generally share in the total cost of care burden through premiums.

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

There needs to be an understanding by the research community of an inherent difference between the health plan payer and the employer plan sponsor. Employers are the actual purchasers or financiers of healthcare. Their motivations are distinct from health plans in their desire to have healthy and effective workforce while mitigating the cost burden of their second biggest expense as an organization. It is important to note that employers also offer various programs or services not implemented through the health plan that impact patient health and wellbeing. These programs often represent the cutting edge of innovation that needs to be studied and leveraged more broadly. “Employer burden” should not only be evaluated by absence and or productivity issues – to do so, limits the value of research that can be conducted to support employers across a broad array of issues and concerns that ultimately impact patient-centered outcomes. The concept of employee productivity and absence will vary by job function and industry. Employers also consider factors such as employee experience and the impact on overall wellbeing and the ability to perform effectively at work. It is also important to research how programs and interventions impact or impacted by: o Psycho-social, mental health issues for a segment(s) of the workforce o Social determinants of health that impact the workforce o Heath equity and disparity issues that impact delivery of care or implementation of programs o Access to care issues, especially those related to the low-wage worker

Principle 3: The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.

I will repeat part of what was shared in the previous principle however focus it more on the need for relevant data as it is important to be sure to include appropriate data sources - or "inputs" - so that a broader picture of what that data is informing can be realized. Data on the following areas would be critical to developing that picture and include: o Psycho-social, mental health issues for a segment(s) of the workforce o Social determinants of health that impact the workforce o Heath equity and disparity issues that impact delivery of care or implementation of programs o Access to care issues, especially those related to the low-wage worker

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

The advantage is that it makes the researcher look at how the research study could influence cost decisions and whether it is practical to implement a particular program or intervention. A main disadvantage is that a requirement such as this is that it make cost and economic impact the main driver to consider any research - and that would limit the importance of other factors that weigh in on the value equation that include a quality component.

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

additional analysis on what constitutes "value" - this brings in a quality of care and care delivery, focus that would be very helpful to employers specifically in making decisions in their healthcare planning.

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

The National Alliance of Healthcare Purchaser Coalitions is happy to see the focus of outcomes data be broadened to consider other stakeholders including employers and a look at additional measures to the clinical and patient perspective. As Employers, health plans and other sponsors make decisions that directly impact patients and patient-outcomes it is important to have evidence-based research that will support their critical healthcare decisions in how to provide plan coverage for patients and advocacy and support to guide patient decision-making. The complexity and diversity in healthcare makes this support essential for patients to navigate the issues they are presented with and helps employers provide benefits and services that are credible and effective.

Name and Organization

Margaret Rehayem, National Alliance of Healthcare Purchaser Coalitions

Community

Purchaser,


Submitted

11/13/20 14:18

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

Not Dead Yet is a national disability rights group whose focus includes protecting the right to receive desired life-sustaining care. Not Dead Yet has signed on to the comment submitted by the Partnership to Improve Patient Care, but we would like to submit a short additional comment. A significant concern is the impetus behind the 2019 amendment to the PCORI reauthorization, which included the following ominous language: “Research shall be designed, as appropriate, to take into account and capture the full range of clinical and patient-centered outcomes relevant to, and that meet the needs of, patients, clinicians, purchasers, and policy-makers in making informed health decisions. In addition to the relative health outcomes and clinical effectiveness, clinical and patient-centered outcomes shall include the potential burdens and economic impacts of the utilization of medical treatments, items, and services on different stakeholders and decision-makers respectively.” The PCORI Principles further clarify that “PCORI is charged with considering the full range of outcomes that meet the needs of ‘clinicians, purchasers, and policy- makers in making informed health decisions,’ in addition to those that meet the needs of patients.” While it is comforting that PCORI reaffirms “the limitation that PCORI ‘not develop or employ a dollars-per-quality adjusted life year (or similar measure that discounts the value of a life because of an individual’s disability) as a threshold to establish what type of health care is cost effective or recommended,’” the 2019 amendment muddies the waters at best. The principles are very well word-smithed, but the health insurers and other stakeholders whose highest priority is increasing their economic bottom line are very good at word-smithing as well. The amendment is a loophole, a potentially dangerous loophole, slipped into the PCORI authorizing legislation. PCORI will have to be vigilant to prevent various stakeholders from using PCORI to their economic benefit at the expense of patients. Given the longstanding cost-cutting pressures in the healthcare system, now increased by the COVID crisis, this will be no small task. We hope that you will seek the assistance of a broad array of disability organizations in your efforts.

Name and Organization

Not Dead Yet

Community

Patient,


Submitted

11/13/20 15:13

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

On behalf of the 54 million American adults and 300,000 children in the US with doctor-diagnosed arthritis, the Arthritis Foundation appreciates the opportunity to comment on the Patient-Centered Outcomes Research Institute (PCORI) Proposed Principles for the Consideration of the Full Range of Outcomes Data. We are supportive of the new statutory authority allowing PCORI to consider the full range of clinical and patient-centered outcomes and “the potential burdens and economic impacts of the utilization of medical treatments, items, and services on different stakeholders and decision-makers respectively.” Through its extensive patient engagement portfolio, the Arthritis Foundation has developed a deep set of quantitative and qualitative data showing the direct and indirect economic impact of health care on patients and their treatment. There are increasing levels of attention across the healthcare ecosystem on the need to better address the impact of health coverage on patients, and we believe PCORI is well-positioned to coordinate efforts and fill gaps through its research scope. There are specific current and anticipated health care trends in arthritis in which this could be particularly useful, including the introduction of biosimilars into the marketplace and emerging therapeutic classes within osteoarthritis. We appreciated the ability to attend the two webinars PCORI held to solicit feedback on the principles from a cross-section of stakeholders, and points that we encourage PCORI to consider include: • While cost and economic burden is important to evaluate, we must start with patient needs and desired outcomes. • It is essential that PCORI evaluate priority gaps in current cost data and work to fill existing gaps, not duplicate data that is collected elsewhere. • The importance of understanding the return on investment for patients, healthcare providers, and payers. • The importance of identifying how the work of PCORI will cascade to other efforts in comparative effectiveness and cost effectiveness research. • The opportunity for PCORI to work towards making data around costs to patients standardized and systematically available. • The potential role for PCORI to increase transparency of cost data to patients. • There are significant challenges in collecting data that PCORI will need to overcome, as research participants will be covered by a range of entities with different benefit structures, contractual payment rates, etc. • Employee engagement with the cost data may be an effective resource for patient advocacy groups, rather than at the individual patient level. • The cost of inaction and discontinuation of care is important to evaluate. As PCORI considers the full range of economic impacts, we would like to highlight and expand upon some of the key themes above: Cost of Inaction and Discontinuation of Care. As important as the cost and efficacy of an intervention is, so is the cost of inaction. What are the health impacts when a patient’s care is interrupted or they are unable to begin treatment? Reasons for this are myriad, and include: misdiagnosis, delays in diagnosis, insurance barriers like step therapy and prior authorization, cost and coverage barriers, lack of access to providers, and unforeseen interruptions like those experienced during a pandemic. For chronic diseases like arthritis, interruptions in treatment or a lack of treatment often lead to worsening of disease, which can then lead to additional and often costly interventions like hospitalizations and surgeries. We have many patient examples on the impact of interruptions in treatment leading to infections, surgeries, and even permanent disability. We know this leads to higher overall health costs in both the immediate and long-term, but do not have comprehensive data on the full impact. Further, there is a ripple effect with costs associated with symptoms like pain and fatigue which can be worsened by interruptions in care. These costs include direct health costs, but also indirect costs from lost productivity and disruptions to daily life for both the patient and their caregivers. Filling Data Gaps. There is a lack of comprehensive data around burden and economic impact of health care interventions that PCORI could help fill. These include: • Comprehensive data on the use and impact of insurance protocols like prior authorization and step therapy across payers. From Arthritis Foundation surveys, we know that prior authorization ranks as the top health care challenge for people with arthritis, and that a significant number of people spend more than 5 hours a month managing their health coverage. Coupled with focus groups and interviews, we know that these types of barriers can lead to interruptions in care, which can then lead to worsening of disease and higher overall health costs, as mentioned above. We strongly believe data should be systematically collected on total cost of care and believe PCORI could play a key role here. • Holistic factors that impact patient outcomes, such as wellness programs and behavioral incentives. An example in arthritis might be interventions that lead to optimal mental and physical preparedness for total joint replacement, and access to post-surgical services like physical therapy to maintain the gains of that intervention. These are indictors for better post-surgical outcomes and emphasis on the systematic collection of this type of data for comparative effectiveness research purposes could lead to wider implementation of best practices around an entire episode of care, leading to lower overall health costs. • Data on the impact of delays in diagnosis and treatment. For chronic diseases, timely diagnosis and treatment are critical to overall health outcomes, and this data could help drive provider education and communication, which could then drive increased rates of timely diagnosis. Moving from diagnosis to treatment, many patients fall through the cracks from the time they receive a prescription to filling and taking the prescription. This is another under-studied area of health care and a patient-centered approach to improving care should include identifying the reasons for these gaps and engaging patient groups to better understand these gaps. Standardization of Data Collection. There are significant challenges in data collection currently, including the fact that benefit design varies greatly across payers, and data is not uniformly collected, utilized, and disseminated by stakeholders. We appreciate that PCORI will not directly engage in cost effectiveness analysis of particular products and that work of this nature will continue to be conducted by groups like the Institute for Cost Effectiveness Research (ICER), the Innovation and Value Initiative (IVI), and the Professional Society for Health Economics and Outcomes Research (ISPOR), among others. One of the greatest challenges these organizations face in evaluating therapies is the inconsistent way that data is collected in the real world, which then hinders the ability conduct an accurate cost effectiveness analysis. PCORI can play a meaningful role in changing the culture of Real World Evidence to be more inclusive of the patient experience, thus helping to shift the paradigm of cost effectiveness analysis toward a more patient-centered future. Useability and Relevance. We strongly believe that cost data should be more transparent and available to patients to better inform and empower them in their own treatment decisions. This includes indirect costs that can contribute to their ability to adhere to treatments and engage with their clinicians; and indirect costs such as transportation costs, time off work, and other costs that factor into treatment decisions and adherence. This data should be disseminated back to patients and providers to promote shared decision-making. We encourage PCORI to ensure the findings from its research are transparent, accessible, and presented in a way that will guide patients and providers directly in building treatment plans. Improving Methodology. We appreciate that use of the quality-adjusted life year (QALY) falls outside PCORI’s scope, as we have significant concerns about the QALY as an accepted methodological approach to assessing value. We hope the implementation of these updated principles can help usher in the development of a more patient-centered methodology as a future standard. As we work towards improved methodologies, there is an urgent need for all stakeholders to coordinate efforts and align on a more patient-centered approach. The Arthritis Foundation works closely with organizations like ISPOR, ICER, and IVI, all of whom work primarily in value assessment, and there are many stakeholders - including patients, payers, and employers - who utilize value assessments in different ways, yet there is no central body accountable for moving all of this work forward. There is a particular need to identify best practices around incorporation of Real World Evidence in Comparative Effectiveness Research and coverage decisions, in addition to holistic costs to the patient, which fits in line with PCORI’s principles. Further, many patient and provider groups engage in comparative effectiveness efforts and have registry data or other data that can greatly enhance PCORI’s work. We encourage PCORI to conduct a landscape of these resources and work closely with all stakeholders to ensure we are well coordinated, and not duplicating efforts, working at cross-purposes, or missing key resources. We are collectively challenged by a lack of standardization in health cost data, benefit design, registry data, and other patient outcomes data, in addition to a lack of consensus on the optimal methodology for assessing value and appropriately collecting and including patient experience data. It is a strong priority of the Arthritis Foundation to overcome these barriers, and to participate in working towards a solution, and we look forward to working with PCORI so the important research that PCORI funds can make the most meaningful impact possible on patient care and health outcomes. Thank you again for the opportunity to comment and we look forward to future opportunities to engage and partner with PCORI.

Name and Organization

Anna Hyde, Arthritis Foundation

Community

Patient,


Submitted

11/13/20 16:57

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

Thank you for considering patient and caregiver impacts in PCORI-funded studies. Measuring these aspects is becoming critically important. Having patients and caregivers involved in research design will help ensure that studies are measuring the endpoints that are important to them. In the “Potential Burdens and Economic Impacts” call-out, suggest adding time away from work/usual activities, childcare costs, and out of pocket costs for caregivers as well as patients. Although these are listed under patient burden, caregivers may also experience these costs. The “Potential Burdens and Economic Impacts” list includes: “Capture all substituted utilization/costs” but this statement is unclear as written. These examples also focus on economic parameters. Will there be consideration of other endpoints related to functional/social impacts to patients and caregivers as well? Economic endpoints may not be the only ones that matter to patients/caregivers.

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

We agree that other stakeholders should also be taken into consideration when assessing burden and cost outcomes. In addition to burden and economic consequences, we suggest including quality of care delivered to be included as a potential area of assessment in PCORI-funded studies. Sometimes costs and burdens to a healthcare system are increased with initiation of new programs that improve quality of care, and therefore the incremental increases/decreases in care quality should also be considered.

Principle 3: The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.

We agree that the burdens and economic impacts should be appropriate and aligned with the clinical aims of the study. Would PCORI accept and fund studies assessing only burden and economic outcomes without a clinical component?

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

We would suggest that the capture of burden and economic impact not be a hard and fast requirement in all PCORI-funded studies, but rather have these endpoints be a consideration for appropriate studies. The advantages to including these endpoints include capturing data important to patients, caregivers, and other stakeholders in the healthcare process. However, capturing burden/economic impact data may not be feasible or necessary in every research study. The methods used to collect burden/economic impact are often different than those used to collect clinical outcomes and therefore the requirement that all studies must include these endpoints may place unnecessary roadblocks on clinical researchers and/or weaken study design. Similarly, there may be studies of value that focus solely on burden/economic endpoints rather than clinical endpoints, and therefore we would urge PCORI to fund studies that do not have clinical endpoints where appropriate.

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

As stated above, there may be studies of value that focus solely on burden/economic endpoints rather than clinical endpoints and we would argue that these studies also be funded when deemed appropriate. However, understanding potential researcher bias would be a consideration in funding these research projects. Additionally, limiting the analysis to aggregate data would be necessary to comply with privacy laws and regulations.

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

Again, thank you for considering patient, caregiver, and health system impacts in PCORI-funded studies. Our evolving healthcare system should be evaluated not only on clinical endpoints, but on endpoints that matter to different stakeholders to ensure the findings are relevant to multiple stakeholders. PCORI should not require these endpoints be included in every study, but should weigh their inclusion as appropriate.

Name and Organization

Heidi Waters, Otsuka Pharmaceutical Development & Commercialization, Inc.

Community

Researcher,


Submitted

11/13/20 21:36

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

We commend PCORI for expanding its funding scope to include the full range of outcomes important to patients and caregivers. PCORI’s research is crucial to understanding the comparative effectiveness of different care options, and its impact on patient outcomes. To improve access to care it is important to leverage data and learnings from the everyday experience of IBD patients being treated in a variety of settings, including community and academic practices. The ability to further capture data on the burden and economic impact of the studied care options will add information that will be useful to patients and their providers when deciding on a treatment plan. In addition, looking at the full range of outcomes will provide the opportunity to include indirect factors that are taxing on patients and caregivers, which will drive towards research that is more meaningful to patients Further, as the price of care options rise, patients are experiencing increasingly restrictive insurance plan designs that erode patient and provider shared decision making. The patient and caregiver community is concerned that this erosion is resulting in severe and irreversible health consequences for patients that are made even more tragic by the sense that the consequences could have been avoided. PCORI’s proposal to add the full range of outcomes for comparative clinical effectiveness research provides an important opportunity to explore the patient-provider shared decision-making relationship, and how this is impacted by such insurer restrictions. These proposed principles will also allow for the assessment of health policies and their impact on patient health and quality outcomes, their cost to the healthcare system, and whether insurance-mandated care such as prior authorization criteria impacts clinical outcomes. The answers to these questions would provide important data that could be discussed between the key healthcare system stakeholders - patients, providers, and payers - to address access to care issues in a holistic manner. This approach will facilitate the development of clear and efficient care guidelines that incorporate patient-provider shared decision-making.

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

The Foundation agrees with PCORI’s proposal to consider the full range of outcomes on other stakeholders when the outcome impacts patients in the near-term or long-term. The patient-provider shared decision-making relationship is frequently complicated by other health policy decision-makers such as payers. In addition to informing patient and provider discussions on treatment plans, cost data could improve discussions with payers about health insurance formulary design and other policies which impact patient and provider shared decision-making and patient access to care. For payers, inflammatory bowel disease (IBD) is a therapeutic area that not only has treatment classes that drive high cost such as targeted immune modulators (TIMs), including biologic therapies, but also has multiple treatments as choices. Therefore for disease areas like IBD it is important that all stakeholders agree on the outcome measures used to assess value. For example, assessment of the long-term value for money of TIMs based on long-term benefits of these interventions is not always accurate nor reliable due to the refractory nature of the disease. To date, most payers evaluate drugs for their formulary based on the clinical literature, which is often incomplete, particularly when considering step-wise therapy selection in longitudinal or chronic disease processes. Providing more opportunities to fund research to generate real-world data based on the full range of outcomes is critical. In addition, PCORI should focus on development of transparent and validated outcomes that are jointly agreed upon by all stakeholders. Further, in the IBD patient population there is a need to bring more advanced products to the market to provide options to patients who are either non-responsive or become refractory. Market accessibility evaluations of these new treatments would not only look at clinical efficacy and safety data but depend on showing value of the product in the real world. As new high-cost treatments come into the market, capturing the full range of potential outcomes with the addition of these therapeutic options is becoming ever more important to facilitate decision making and optimize care.

Principle 3: The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.

The Foundation agrees with PCORI’s focus on the clinical aims of comparative effectiveness studies and that cost data can add an extra layer of information to inform treatment and policy decisions. As mentioned above, a frequent dichotomy seen in patient care is understanding the importance of patient-provider shared decision making versus payer policies. Payer policies may hamstring the ability of patients and providers to select the treatment they think best for the patient. The perception of the impact of payer-mandated care and the interruption of shared decision-making is that it can lead to severe and irreversible health outcomes. Further, these deleterious clinical outcomes can have far reaching economic impacts that have not been traditionally accounted for. Formally studying these two approaches to care - shared decision making versus payer-mandated care – can provide important data to allow patients, providers, and payers to engage in a dialogue about formulary design. Because restrictive payer formularies are largely driven by cost and business considerations, cost data and in particular the downstream impacts of formulary-based modifications in care, will be an important component of these discussions. As a patient-first organization, the Foundation also agrees that study outcomes should be relevant and important to patients and other stakeholders. From the patient perspective, the conduct of comparative effectiveness research assessing restrictive formulary design mechanisms such as insurance-mandated step therapy, copay accumulators, and high deductible health plans would be of high interest to study.

Principle 4: Beyond the collection of burden and economic impact data, PCORI may support the conduct of certain types of economic analyses as part of a funded research study, to enhance the relevance and value of this information to health care decision-makers.

The Foundation recognizes the statutory limitations placed on PCORI when considering the full range of outcomes. However, there is still much opportunity to be gained from the collection of cost data that PCORI can support. The Foundation, along with several other patient and provider voluntary health organizations, has long been concerned about the impact of formulary design in eroding patient-provider shared decision making. Better understanding the costs associated with intact shared decision-making, and the costs associated with insurance-mandated care, both in the near-term and long-term, could inform important explorations on these two care approaches with significant impact on patient health outcomes. There is also great need to further evaluate the sequence and timing of medications used alone or in combination over the life course of patients with these diseases.

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

Payers have significant influence on patient access to care. PCORI should consider a wide range of cost considerations to understand how care options impact all stakeholders in the nation’s health care system. When possible, an integrated approach should be considered by linking real-world data sources (e.g., clinical, patient-reported, and medical and pharmacy claims data) together to understand the full picture of cost and value. Examples of types of data points these analyses should consider collecting include direct costs to the patient, caregiver or broader family burden, costs to the system, and cost and time related to condition-specific treatment follow-ups such as all doctor visits, lab tests, radiographic testing, urgent and emergent care utilization, procedural costs, and self-reporting and monitoring activities. In addition, indirect costs such as cost of travel and parking and opportunity cost such as lost time from work or leisure is very important to understand for chronic, lifelong diseases such as IBD.

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

Complex, multi-factorial diseases, like Crohn’s Disease and ulcerative colitis, would greatly benefit from PCORI incorporating the full range of outcomes into their funding opportunities. The Foundation continues to be concerned about the high out of pocket costs, the high cost of care, and suboptimal treatment access experienced by this patient community. We support efforts to bring the topic of value into research, and note that insurance mandated mechanisms such as step therapy further reduce the value of advanced drugs and block access to treatments that the Foundation has strived for decades to help bring to market. All stakeholders must come together to improve their processes to bolster patient access. Methodologically gathered real-world data around the economic impact and burden of disease would improve the rigor of methods to assess value, and ultimately the validity and usefulness of such value assessments. The Foundation has established a next generation data platform, IBD Plexus™, to centralize and harmonize real world data to facilitate real world evidence to answer high impact research questions and improve the lives of patients living with Crohn’s disease and ulcerative colitis. The first-of-its kind, national-scale, cloud-based platform integrates clinical, patient-reported, genomic and molecular data from diverse research study cohorts, real world clinical care settings, and patients’ experiences. In addition, the Foundation recently partnered with a large data provider to link IBD Plexus datasets to medical and pharmacy claims to help accelerate efforts around value-based payments and formulary design. We are thrilled to see PCORI’s proposed principles include the incorporation of the full range of clinical and economic outcomes that impact patients into your funding opportunities. We look forward to continued partnerships with PCORI to improve clinical care and patient outcomes.

Name and Organization

Sarah Buchanan, Crohn's & Colitis Foundation

Community

Patient,


Submitted

11/13/20 22:04

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

The American Geriatrics Society (AGS) agrees that PCORI-funded research must consider the full range of outcomes and that PCORI’s ability to examine economic impacts has been a limitation. One way of getting around the limits set in PCORI’s authorizing statue would be to focus attention on “Days at home”, which has gained increased recognition as a valuable patient-centered outcome: Gill TM, Gahbauer EA, Leo-Summers L, Murphy TE, Han L. Days spent at home in the last six months of life among community-living older persons. Am J Med. 2019;132:234-239.

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

The American Geriatrics Society believes that the impact on payers (e.g., costs to Medicare) are important in guiding policy decisions.

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

The American Geriatrics Society believes there are advantages and disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact of data. One advantage of making this a requirement would be that all PCORI-funded research would be useful in guiding policy. The disadvantage is that for some research (e.g., methodology research) economic impact may not be relevant. It may also add to the expense of conducting some studies, which could make it more difficult to complete the research within the limits of funding ceilings for specific types of award. Indicators of “scenarios/types of studies where capture of economic impact will not contribute to overall importance” include methodology grants that are not testing the impact of an intervention and interventional research in which anticipated effects are primarily on quality of life or other outcomes that are expected to be cost neutral.

Given the limits set in PCORI’s authorizing statute, it is clear that data collection is in scope, and that cost-effectiveness analysis is out of scope. Understanding that there are circumstances where additional analysis of data may offer benefit – but that economic analysis should not be a primary goal of PCORI-funded studies – we seek input on the type of analyses that will benefit stakeholders, and the specific uses and advantages of each type of analysis.

The American Geriatrics Society agrees that these analyses are important. For studies including persons with serious illness, we suggest including “days spent at home”. In addition, “days spent on hospice” for those receiving this service. Using costs of interventions can be difficult because of geographic variation of labor costs and perhaps national averages should be used. In addition, costs must consider training and ramp-up. Sometimes, these are divided into start-up and steady state. Furthermore, generalization of costs and economic impact studies are limited because of: 1) the arbitrary nature of how costs are assigned (e.g. wide variability in costs for the same procedure in different locations and the artificially inflated cost of many drugs); 2) difficulty assigning costs to behavioral interventions which can vary by how they are implemented; 3) variability in cost-benefit depending on the payment model. The American Geriatrics Society recommends that impact analyses include both short-term and future impacts depending upon the study population and interventions being studied. For example, an intervention administered to a seriously ill population may have cost savings within a year or two but by 5 years many receiving the intervention will no longer be alive. The American Geriatrics Society also suggests PCORI consider proposing studies that include “universal”, preferably patient reported, outcomes that are appropriate across all conditions being studies and include generalizable measures of utilization such as days in the community which is more inclusive than hospital days.

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

The American Geriatrics Society recommends that PCORI consider patient-identified outcomes (eg, priorities, personal goals) which may be more relevant than traditional disease-based or general quality-of-life measures. From a geriatrics perspective, outcome assessments for older persons should include functional measures, since the functional independence has repeatedly been identified as the top priority. See: Fried TR, Tinetti ME, Iannone L, O'Leary JR, Towle V, Van Ness PH. Health outcome prioritization as a tool for decision making among older persons with multiple chronic conditions. Arch Intern Med. 2011;171(20):1854-6. The use of surrogate outcomes (like Hgb A1c or lipid level) are less preferred.

Name and Organization

Erin Obrusniak, The American Geriatrics Society

Community

Non-profit


Submitted

11/17/20 16:56

General feedback on the Proposed Principles for the Consideration of the Full Range of Outcomes Data

-- First and foremost, many congratulations on PCORI’s reauthorization! I applaud the new statutory mandate to consider the full range of outcomes data in PCORI-funded studies. Cost is a shared concern across stakeholders and its transparency will empower patients and foster best practices. -- The potential economic burden to patients (Principle 1) should be the number one consideration but also be relevant to other stakeholders. This aligns with PCORI’s patient-centric culture. -- Factors such as diversity, geography, social determinants of health, trust, adherence, payer mix, etc., can impact cost to patients. Applicants could assess cost “in the context of” these factors. For example, government versus commercial coverage, differences within plans, high deductibles, and different benefit designs (medical versus pharmacy) have an impact on cost in the context of payer mix and should be collected. Categorizing what I call “cost determinants of health (CDH)” in these contexts may allow a deeper understanding of them for all stakeholders and guide future research. -- Both direct patient out-of-pocket costs and indirect patient/caregiver costs are important to gather. These secondary costs (transportation, lost work hours, child and adult care, etc.) are certainly relevant. Any hidden costs will be critical to capture financial toxicity to patients and caregivers. Not only is collection of the cost of the treatment or intervention needed, cost to the decision team, and ultimately the total cost of care are vital. -- Drug spend is a significant expense for patients, payers, and health systems. The growth of the specialty drug pipeline, orphan drugs, treatments for complex conditions, and maintenance therapies for chronic conditions all contribute to this increasing trend. Other major trends, partly related to an overarching rise in retailization of healthcare, include a shift in reimbursement to value based care as well as a shift in delivery sites of service from inpatient to outpatient, home, and virtual telehealth settings. Depending on the what is being measured, these changes could show cost savings to patients/caregivers, payers, health systems, and other stakeholders versus cost burden. One suggestion is to adopt cost savings language as a part of PCORI’s principles. -- Collection of economic impacts related to utilization of healthcare services provides unique value opportunity for patients, other stakeholders, and future study applicants to view the data collected and potentially inform decisions. The application of this cost data to future efforts may be instrumental in building a more collaborative and patient-centric healthcare system. A central cost data repository for PCORI-funded studies could be established with possible interoperability. -- Establishing a PCORI cost advisory panel as part of the collective effort around patient and multi-stakeholder cost exposure.

Name and Organization

Anonymous

Community

Stakeholder,


Submitted

11/30/20 15:37

Principle 1: PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.

Many existing survey instruments include items that address the burden of illness on patients’ work ability and work participation. Typical items ask about functional limitations or difficulties performing tasks at work (e.g., concentrating, working carefully, sitting, or standing at work), days missed due to illnesses, and scales that can convert on-the-job impaired performance to percent of effort or wages. When studies use these instruments on representative samples of employed patients, caregivers, and their households, the data can provide employers with valuable insights into how workforce illnesses might impact their own business operations. This could in turn motivate efforts to better understand the prevalence of serious health issues among their own employees and design benefits policies that prioritize needs, as well as expand employers’ ability to better quantify the value of better care and increased investments in workforce health. Stratifying data over time and by business units—or utilizing natural experiment approaches when access to interventions or programs differs randomly—would also permit correlating population health with key business performance indicators (KPIs) to show the potential business value of access to affordable, high quality care. Many of the current patient-reported approaches to health and productivity measurement lack survey items that address patient and family members’ financial burden of illness. For example, surveys that ask about workdays missed due to illness could include follow-up questions about income lost on those days (including the gap between normal wages and replacement wages paid by Workers’ Compensation and short-term disability insurance), about the ability to make up work or lost income, and the downstream ability to afford care relative to other necessities. This information would give employers and policy makers a clearer picture of the income protection provided by employee benefits and paid leave laws and help set priorities for aligning public and private efforts to minimize the financial burden of illness.

Principle 2: PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.

Many studies that focus on the economic value of care quality omit the impact on disability leave outcomes, as well as the impact of lost days on employer business performance. This represents a missed opportunity to demonstrate to employers (as purchasers of labor and payers for health benefits) that high quality treatments have a business value to the extent that they decrease the likelihood of illness episodes that require extended time away from work or shorten the duration of recovery when leave is needed. The inclusion of leave outcomes would also permit a full accounting of the economic burdens borne by patients, caregivers, and their households. Disability insurance policies (including statutory temporary disability insurance in states such as California, Hawaii, New Jersey, New York, and Rhode Island), typically replace about two-thirds of claimants’ wages—meaning that employees lose about one-third of their earnings for the duration of an illness leave. These income losses increase to about half of an employees’ if they require long-term disability leave, which can last for several years until a claimant resumes their work ability or is judged eligible for Social Security Disability Insurance benefits, which is usually a precursor to exit from the labor force. While most sources of disability data leave that could permit cohort analyses of treatment alternatives are proprietary, many large disability insurance carriers and third-party administrators (TPAs) of self-insured employers’ disability benefits contribute data to the nonprofit Integrated Benefits Institute’s data consortium. Summary information on leave outcomes for specific conditions (defined at the ICD-10 level) can serve as industry benchmarks for leave costs and durations, across industries, disability plan design dimensions and demographic categories such age and sex.

Principle 3: The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.

By necessity, the clinical aims of a study will and should dictate the measurement of primary clinical outcomes. From the perspective of patients and caregivers, however, clinical outcomes cannot be separated meaningfully from improvements in their health-related quality of life—which for many will include the ability to participate fully in the labor force. This perspective on the clinical impact to patients and caregivers also provides a link the operational and financial impact to employers. The reverse is also true: outcomes data related to illness-related absences and ability to participate fully in work can provide insights on the clinical value of care, even where clinical outcomes are not collected directly. As described in comments to Principles 1 and 2, both patient-reported and claims data represent approaches to capturing both clinical and economic impacts on patients and family members, and on employers as purchasers of labor and payers for employee benefits. In principle, researchers can incorporate either approach into the design of studies that use treatment and control groups and can collect data representing experiences before and after the observation period. Where leave data cannot be accessed from carriers or TPAs, records of payments to research subjects combined with an administered questionnaire represent an alternative approach.

Principle 4: Beyond the collection of burden and economic impact data, PCORI may support the conduct of certain types of economic analyses as part of a funded research study, to enhance the relevance and value of this information to health care decision-makers.

As described in comments to Principles 1 through 3, collection of patient-reported or claims data on the economic of burden illness provides an approach to assessing the value of high quality care to multiple stakeholders, without the necessity of assessing cost effectiveness or imputing cost-per-quality-adjusted-life-year thresholds. This stems from the assumption that the economic benefits of treatments accrue directly to patients, caregivers, and their households in the form of income protection (that is, by preserving current employees’ ability to work), and indirectly to employers whose output depends on their employees’ availability to work and perform unimpaired by symptoms of illness or the side effects of treatments.

What are the advantages/disadvantages of a requirement for all PCORI-funded research to capture burden and economic impact data? What are reliable indicators of scenarios/ types of studies where the capture of this data will not contribute to overall importance or value of the research study findings?

The advantages of capturing burden and economic impact data can be summarized in the comments to Principles 1 through 4: using patient-reported or claims data to capture the economic burden of illness and the potential benefits of high-quality permit assessments of treatment value in terms of income protection for employees, caregivers, and their households, and productivity enhancement for employers as purchasers of labor and payers of employee benefits. Outcomes data related to illness-related absences and ability to participate fully in work also can provide insights on the clinical value of care, even where clinical outcomes are not collected directly. Aside from requiring some additional preparation to collect and evaluate data, we see no disadvantages. We also envision no scenario where the capture of economic impact data will not contribute to the importance or value of study findings.

Name and Organization

Integrated Benefits Institute (IBI)

Community

Research Organization


 

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