The authors, from the National Health Council, the EveryLife Foundation for Rare Diseases, and the Innovation and Value Initiative, unveil a project to develop a blueprint for creating patient-centered core impact sets.
“Patient centricity”—engaging with patients as partners, rather than doing on behalf of patients—is foundational for our 3 organizations.*
As evidenced by the birth of the Patient-Centered Outcomes Research Institute and the FDA's Patient-Focused Drug Development initiative, we have seen progress in advancing patient-centric outcome measurement as part of research and medical product development. But, many stakeholders agree, patient centricity needs to be better integrated into all health decisions. As we turn our attention as a nation to how we will pay for high-value health care, we need to get this right.
Engaging with patients as partners has demonstrated that measuring only traditional clinical outcomes is not enough to develop an evidence base that supports all aspects of patient-centered decision-making. Accordingly, we have seen increased attention on outcomes such as feeling, function, preferences, and quality of life, captured using patient-reported outcome measures.
But, in our experience, patients often report impacts from their disease or treatment that go beyond health outcomes, such as stresses on their finances or ability to work, or effects on their caregivers and families. If we are going to achieve patient centricity in health decisions, we must change our thinking and terminology, from a lexicon geared primarily toward traditional clinical outcomes to broader patient-relevant impacts.
Our organizations have joined together on a project to develop a blueprint for creating patient-centered core impact sets (PC-CISs), which include, but are broader than, traditional health outcomes and move to capture the broad range of impacts a disease and its treatment have on a patient’s daily life. A number of groups have laid the groundwork by developing core outcome sets. We want to build on and expand that work to include the full range of patient-centered outcomes and other impacts under this new umbrella term.
We believe that a multitude of stakeholders must be involved in this endeavor: patients, caregivers and patient groups, researchers, clinicians, payers, and government bodies, among others. The resulting core sets must be open source and freely available; no one body or organization can “own” them.
The charge we have laid out for ourselves is immense. We will tackle it incrementally and start by convening stakeholders to produce a common blueprint for standardizing approaches to, and methods for developing, PC-CISs. The objective is to ensure sound, patient-centered methods are followed and impact sets are aligned.
Moreover, a blueprint will allow contributors and users to have confidence a PC-CIS is a sound and useful tool for a range of decision-making uses. The blueprint will need supporting tools and resources, such as a common taxonomy to capture the range of impacts patients report as important. This can be part of incremental steps to follow, as well as inform a research agenda to help fill gaps identified along the way.
The ongoing pandemic has taught us that even the most daunting challenges can be met through dedicated focus on methods and science, on the needs and experiences of patients, and on collaborative exchange of data and learning through action. Applying this approach to understanding and capturing PC-CISs will help all of us move toward decision-making that is both informed by and to the highest benefit of patients.
*About the contributors
Eleanor M. Perfetto, PhD, MS, is executive vice president of Strategic Initiatives of the National Health Council
Annie Kennedy is chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases
Jennifer Bright MPA, is executive director of the Innovation and Value Initiative