A Blueprint To Advance Patient-Centered Core Impact Sets


Editor’s Note

This article is part of a Health Affairs Forefront short series, “Enhancing Value By Evaluating Health Care Services.” The series discusses ways to extend the use of tools for clinical and economic evaluation beyond medical technologies to the services and procedures that account for the bulk of health care spending; the goal is to create a more robust evidence base for the effectiveness and value of health care services. The articles in the series were completed with support for the authors from the Research Consortium for Health Care Value Assessment, a partnership between Altarum and VBID Health, through a grant from the Pharmaceutical Research and Manufacturers of America (PhRMA). PhRMA extended complete independence to Altarum to select researchers and specific topics. Health Affairs retained review and editing rights. Readers interested in more details on the topics discussed below can check out Perfetto, E.M., Oehrlein, E.M., Love, T.R. et al. Patient-Centered Core Impact Sets: What They are and Why We Need Them. Patient (2022).

All parts of the health ecosystem should be accountable for making care affordable and of high value to patients. Numerous measures and endpoints are used to study and monitor health conditions and interventions. The concepts captured by the multitude of measures and endpoints used in the health ecosystem vary across studies and often are misaligned with what patients report matters to them. For example, rheumatoid arthritis patients report that pain and fatigue are their biggest concerns and impact functional ability. Yet, traditional clinical measures for monitoring these patients include an array of biomarkers that are often not correlated with the outcomes that matter to the patients. For other diseases, loss of a job or career due to disease or career responsibilities, and resulting economic impacts, are what is reported as important.

The current environment, characterized by a haphazard approach to deciding what to measure for which diseases, results in confusion, missed opportunities, inefficiencies, and frustrations. Patients and clinicians lack data relevant for decision making; health technology assessors hear from patients about what matters but find little data they can use; and systematic reviewers identify few studies using the same measures and endpoints. The key question is: What should be measured and monitored?

Stakeholders assessing the impacts of diseases and treatments on patients could benefit from having a resource to guide them on what patients report as most important. Thus, the National Health Council (NHC) is offering a solution: patient-centered core impact sets (PC-CIS), a patient-prioritized list of the impacts a disease and its treatments have on patients, carers, and families. We intentionally use the broad term “impacts” to include direct health impacts and all other disease- and treatment-related implications (for example, caregiver stresses, financial burden, and so forth). Disease-specific PC-CIS are intended to guide stakeholders toward capturing aligned data concepts, especially those that matter most to patients. Such a resource could harmonize approaches, providing more uniform targets, and optimize data-collection efforts for research and care. Unfortunately, PC-CIS do not currently exist.

Thus, the NHC is spearheading a multistakeholder effort to create a PC-CIS blueprint and toolkit, a “how-to” manual that can be used to develop a PC-CIS for a specific disease, related diseases, or population(s). PC-CIS development begins by engaging patients, carers, and families; thus, we envision and emphasize patient-community leadership. Our goal is a clear, sound PC-CIS-development pathway for patient groups and their partners (for example, consortia with researchers, government, industry, and so forth). This article describes our approach to PC-CIS-blueprint development (slated for mid-2022 completion) and accomplishments, work products, and learnings to date.

The PC-CIS Vision

Our PC-CIS efforts are guided by a multistakeholder Advisory Committee established at the inception of the initiative. This group of experts co-creates and provides input on, guidance for, and review of work products. As an initial step, a conceptual framework of PC-CIS was developed in the first few months of the project, as depicted in exhibit 1, that captures the NHC’s foundational principles The effort begins with patient/family engagement to understand what is important to patients/families; a patient-leadership role (for example, governance) is expected, and the patient voice should not be able to be overruled by other stakeholders’ views. In addition, disease and treatment impacts on a patient’s life extend beyond direct health impacts (for example, clinical outcomes). Broader impacts must be captured as well.

Exhibit 1: PC-CIS conceptual framework

Source: Authors’ analysis.

The framework depicts the wide range of possible life impacts a patient (and carer/family) might experience related to disease or treatment, narrowed to a core set through patient engagement and prioritization. The process must consider issues such as equity and representativeness, and social determinants of health. Patients from whom insights are collected must be representative of those with the disease. Impacts prioritized by patients and families feed into a prioritization process that incorporates inputs from other stakeholders and an environmental scan. The process protects patient input from being outvoted. The resulting PC-CIS is a set of prioritized impact concepts—the things patients and other stakeholders tell us should be studied and monitored in research and care and should serve as a foundation for decision making.

Lastly, the framework depicts the many possible uses for PC-CIS. While each use will require fit-for-purpose measures, all should be aligned with the core set. PC-CIS can inform research by providing a starting point for setting objectives and selecting endpoints. It can inform care by guiding such activities as quality-measure development and clinical-decision support systems into alignment with patient views and needs.

Environmental Scan

An environmental scan was conducted to identify if PC-CIS exist and locate resources currently available to support PC-CIS development. The scan was guided by Advisory Committee input and conducted in spring 2021. It included review of the peer-reviewed and gray literature and an internet search for publications and websites. While we did not identify any efforts that directly align with the PC-CIS vision, we identified numerous related initiatives and resources that have the potential to support PC-CIS development.

The resources most closely resembling the vision for PC-CIS are efforts to produce core outcome sets (COS). These initiatives provide a bevy of methods, experiences, and processes that can be leveraged for PC-CIS development. For example, through the COMET Initiative and additional efforts such as their PoPPIE working group, minimum standards have been identified for COS design that could be adopted.

Past and current COS efforts are a valuable and integral foundation to PC-CIS development. However, having been initiated for more targeted audiences, they do not meet all the patient-centered characteristics of PC-CIS. We could not identify any led by patients, but we did identify several with established patient roles. Some did not engage patients at inception but do include engagement in more recent years. COS initiatives do not typically begin outcome identification with patient input but rely on a literature search. This means the “universe” of potential outcomes may not reflect diverse patient priorities. But since they are so closely related, part of our blueprint work will be to answer the questions: Can an existing COS be transformed into PC-CIS and by what methods?

Building The Blueprint

The next stage is building the blueprint and an accompanying resources toolbox to support the work. The first step was to hold brainstorming sessions with the Advisory Committee to co-create a draft table of contents with four major components: an introduction and background section to provide the rationale for PC-CIS, describing need and uses; a methods section outlining methods and approaches for creating PC-CIS; a resource toolbox providing useful supporting materials for getting the work done; and a description of pilot studies and use cases.

Our work plan to turn the draft table of contents into the full blueprint includes the phased kickoff of four multistakeholder work groups addressing each of the four sections of the table of contents. The work groups will comprise members of the Advisory Committee, NHC members, and other invited experts. The Resources Workgroup, charged with developing the resource toolbox, kicked off its work in September 2021. Its role is to assess and select from among resources identified in the environmental scan those relevant and useful to PC-CIS development. The Background and Methods Workgroups began their work in fall 2021 and align with the Resource Workgroup’s efforts. The Pilot Workgroup begins its work later in 2022 when the methods draft is complete.

From Impact Concepts, To Measures, To Endpoints In Context

It is envisioned that a disease-specific PC-CIS will serve as a standard resource stakeholders will turn to, to help them understand patent experiences, and to guide them in selecting measurement targets for research, care provision, and other uses, as depicted in exhibit 1. A PC-CIS will be layered in the information it provides. Insights and experiences from patients will inform the impact concepts most important to patients, for example, pain, function, family stresses, and financial toxicity. But concepts are not measures. The measures needed to capture the concepts may exist or need to be developed. PC-CIS can also list available measures demonstrated as sound in specific contexts of use.

However, PC-CIS also will include core impacts for which measures do not yet exist. This does not mean researchers are forced to collect data without a fit-for-purpose measure. It instead means the concept has a place in the PC-CIS as a reminder to the field that aligned measure development should be part of the research agenda for the disease. In the interest of transparency, any study or program should clearly state that an impact important to patients was not included, state the reason(s) why, and what the omission of that impact might mean for interpretation and use of findings.

Uses For PC-CIS

PC-CIS can support a variety of uses (see exhibit 1) as well as the Patient-Centered Outcomes Research Institute’s (PCORI) expanded mandate. PCORI reauthorization legislation, signed into law in 2019, directs PCORI to fund studies “to capture data on the potential burdens and economic impacts of medical treatments, items, and services.” Direct medical costs, especially out-of-pocket costs; nonmedical costs to patients and families, and effects on future costs; and work loss and absenteeism are all impacts of diseases and treatments that could be included in a PC-CIS. PCORI’s Principles for the Consideration of the Full Range of Outcomes Data in PCORI-Funded Research are intended to expand the types of outcomes and impacts on patients’ lives captured in research, including burdens and economic impacts related to disease and treatment. Understanding these impacts is essential to informing the decisions patients make with carers and care teams, as well as those made by other stakeholders such as payers and employers. In short, PC-CIS is a potential mechanism by which PCORI could achieve the goals of its guiding principles.

A few examples illustrate how PC-CIS could contribute to many potential downstream uses. One is in the area of value assessment. Value assessors need two elements for patient-centered work: the impacts important to patients and the data that capture those impacts for inclusion in models and value frameworks. A criticism of value assessment over the past few years is that it does not fully capture the patient voice. Value assessors responded by instituting mechanisms to engage patients and seeking patient-centered data.

However, a significant barrier value assessors still encounter is lack of data on the impacts patients care about. Thus, PC-CIS can provide value assessors direction on the patient-centered impacts that must be included and, over time, the data needed. This also ties to PCORI’s Principle No. 3. While PCORI still cannot support cost-effectiveness analyses, it can focus on patient and family burden, especially economic burden. PCORI’s new efforts can identify data targets and support data collection on patient-centered impacts that will find their way into downstream value and health technology assessments.

PC-CIS could markedly change the paradigm in use and analyses of real-world evidence (RWE). LEAPS (Learning Ecosystems Accelerator for Patient-centered, Sustainable innovation) is working to modernize how RWE is planned, collected, produced, and used across learning health systems. A recent publication on LEAPS’ ongoing pilot in rheumatoid arthritis describes a challenging barrier: lack of consistent patient-important impacts embedded in upstream data-collection efforts. While meaningfully partnering with patients and other stakeholders added tremendous value to the project’s data analysis, it could not overcome immense data gaps. The team spent time developing and using proxy measures for patient outcomes because the claims and electronic medical record data had no clear indicators of how patients function or feel. The existence of a PC-CIS could be the Rosetta Stone across multiple health care silos from basic research to continuous improvement to RWE and back again.

Moving Forward

We are energized by the PC-CIS initiative and have our course set to accomplish a great deal this coming year. A thorough blueprint will provide assurance a PC-CIS is a sound and useful tool for a range of decision-making uses and will inform a research agenda to fill gaps identified along the way. With a multistakeholder approach, focused on the needs and experiences of patients, and a collaborative exchange of data and learnings, capturing PC-CIS will help all of us move toward research and care informed by and benefiting patients.

Authors’ Note

We would like to acknowledge the guidance of our PC-CIS Advisory Committee. We also want to acknowledge the generous support of our PC-CIS founding sponsors, Innovation and Value Initiative and Everylife Foundation for Rare Diseases, and additional sponsors, Edwards Lifesciences, Johnson & Johnson, Novartis, PhRMA, and Takeda. The development of this manuscript was funded by a grant from Altarum. Eleanor Perfetto and Elisabeth Oehrlein were staff members at the National Health Council (NHC) at the time this article was prepared. Silke C. Schoch is currently a staff member at the NHC. The NHC is a not-for-profit, membership organization supported through membership dues and sponsorships. The complete list of members and sponsors is located on the NHC website at https://nationalhealthcouncil.org. Perfetto is a part-time faculty member at the University of Maryland Baltimore School of Pharmacy and has received funding from the Food and Drug Administration, Excerpta Medica, PhRMA Foundation, PhRMA, Merck, Innovation and Value Initiative, Gilead, Novartis, and Pfizer.

Elisabeth M. Oehrlein is the founder of Applied Patient Experience, LLC.

Annie Kennedy works for the EveryLife Foundation for Rare Diseases, which is a sponsor of the NHC PC-CIS initiative. Jennifer Bright is the CEO of the Innovation and Value Initiative (IVI), which is a sponsor of the NHC PC-CIS initiative. T, Rose Love is a PhD candidate and graduate research assistant at the University of Maryland Baltimore School of Pharmacy. She has received consulting fees from the NHC and received remuneration for Voice of the Patient activities as a Voice of the Patient Volunteer with Nephcure.

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