How patient-centered is your research?
April 2025
What makes research patient-centered is a topic of debate among researchers and the healthcare ecosystem.
At Carelon Research, patient-centered research:
- Is guided and informed by patient preferences, experiences, beliefs, and needs.
- Incorporates direct input from the patient on health outcomes and lived experience.
- Prioritizes research questions, comparators, and outcomes that matter to patients.
- Acknowledges the patient as a key stakeholder and end user of research.
- Pursues strategies to disseminate published findings back to patients in lay language.
What makes research patient-centered is a topic of debate among researchers and the healthcare ecosystem. While there is broad agreement on the value of doing patient-centered research, the best approach to achieve patient-centered research is less clear. As Carelon Research continues to expand and diversify its patient-centered research capabilities, we reflect on the numerous insights presented by researchers at the 2023 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) conference. We also consider how the perspectives and research presented align with the patient-centered research we collaboratively develop with our partners and clients.
Patient-centered research is not based on a singular approach
During the 2023 ISPOR panel discussion “Using Clinical Outcome Assessments (COAs) in Your Research Study Does Not Necessarily Make It Patient-Centric,” Eleanor Perfetto, Ph.D. M.S., professor at University of Maryland School of Pharmacy and former executive vice president of the National Health Council, defined patient-centered as, “any process, program, or decision focused on patients in which patients play an active role as meaningfully engaged participants, and the central focus is on optimizing use of patient-provided information. [It’s] doing things with — not for or to — patients.” This definition is a reminder that bringing the voice of the patient into research and intentionally using the information to guide decision-making is an essential part of patient-centered research.
Does this mean that only those studies that “actively” include patient advisors as research partners can be patient-centered? Not necessarily. While a bi-directional partnership between researcher and patient advisor(s) is ideal, it is not realistic or feasible for every research project. Defining patient-centeredness as an all-or-nothing active engagement is unnecessarily limiting. Instead, patient-centered strategies can be placed on a continuum (PCORI and PLOSone ) bookended by a minimum standard for deriving patient insights to inform research and an extensive longitudinal partnership with patient-led decision-making (see graphic below).
Method of engagement in patient-centered research
|
|
|
Consult |
Collaborate |
Lead |
|
|---|---|---|---|---|---|
|
Frequency |
Intermittent |
One-time |
One-time |
Regular |
Regular |
|
Type of Communication |
Unidirectional |
Unidirectional |
Unidirectional |
Bidirectional |
Bidirectional |
|
Decision-making authority |
Unaware of decisions |
Unaware of decisions |
Unaware of decisions |
Advise on decisions |
Lead decisions |
|
Method |
Website Email blasts Static webcasts |
Person provides information as a subject and a measuable data point |
Focus groups Interviews Panels Crowdsourcing Patient advocacy consult |
Co-I Advisory committee |
PI or Co-PI |
This figure demonstrates a more inclusive vision for patient-centered research that acknowledges the pragmatic need for cost-effective methods that can be applied in real-world data settings. Leveraging secondary sources of patient experience data such as published qualitative studies, patient journey maps, and social media content can efficiently inform the design of a retrospective cohort on a limited budget (Oehrlein et al 2022 ). A prospective study, however, with a longer time frame and a larger budget may benefit from the investment of a bidirectional patient partnership to inform the development of study materials and guide the selection of recruitment and retention strategies. The patient-centered approach selected should be matched to the research question, study design, stage of research, existing patient insights publicly available, budget, and study timeline. Carelon Research operates under this more inclusive definition of patient-centered research maintaining the intention to use patient-provided information to guide research decisions while also acknowledging the need for flexibility in the types of methods utilized for a range of real-world research initiatives.
Patient-centered research is more than patient-reported outcomes
Some researchers measure patient-reported outcomes (PROs) as key study endpoints in an effort to make their research more patient-centered. PROs, which are directly reported by the patient, can expand our understanding of the patient experience. In fact, collecting PROs to measure outcomes patients care about is considered a cornerstone of patient-centered research methodologies methodologies (PC-3 PCORI Methodology Standards ). Surprisingly, there has been little growth in the proportion of industry-sponsored clinical oncology trials measuring PROs in the past 10 years (between 14–18%) despite a doubling in the number of clinical trials conducted and FDA’s encouragement to engage patients in drug development. These results were presented in a poster at ISPOR 2023 by Margaret Mordin, M.S., Vice President of Market Access and Outcome Strategy at RTI Health Solutions, titled “Trends in the Inclusion of Patient-Reported Outcomes in Industry-Sponsored Interventional Oncology Clinical Trials: Analysis of ClinicalTrials.gov (2013-2022).” At face value, these trends suggest the research community has made little progress in understanding patient preferences and experiences with oncology interventions.
As described above, other strategies may be included as part of a broader patient-centered approach including:
- The engagement of patient advisors in the conceptualization or design of the study.
- The utilization of secondary sources about patient experience to prioritize comparators and outcomes.
- The use of qualitative methods (focus groups or interviews) to inform conduct, interpretation, or dissemination of results.
Implementing any one of these methods would make the research patient-centered even in the absence of a PRO instrument.
On the other hand, not all clinical trials that include PROs are patient-centered. Dr. Perfetto stated ideal state of patient-centeredness is the intersection of patient-centered outcomes (outcomes important to patients) and patient-reported outcomes (outcomes reported directly from patients). A direct-to-patient method is not enough to claim patient-centeredness. PROs created or selected without patient input may not be measuring or prioritizing what matters to patients. If patients are intended to be the end users of a product, treatment, or program, researchers should validate assumptions about what is important to patients with patient experience data or patients themselves.
The Carelon Research definition of patient-centered research aspires to be true to the principle of “doing with — not for or to — patients.” It acknowledges the need to have direct input from patients — from primary or secondary sources — and the intention to use the information to guide and prioritize research or programmatic decisions in cases where the patient is the end-user. While this approach does take more time and financial resources, we believe the effort will result in research that is more relevant, feasible, and trustworthy.
For more information about our patient-centered research capabilities including patient-centered research design, patient engagement services, approaches to deriving patient insights and strategies for developing patient-facing materials, visit our Patient-Centered Research page.
