The role of patients and caregivers in health preference research (HPR) and patient-reported outcomes (PROs) research is critical for fostering patient-centered care. HPR involves recognizing and integrating the preferences of patients and caregivers into healthcare decision-making, using techniques to measure these preferences quantitatively. This ensures that healthcare services align with the needs and desires of patients, leading to more personalized and effective care. [1,2]

PROs are defined by the NIH as direct feedback from patients on their feelings and capabilities while managing chronic diseases or conditions. PROs are increasingly collected in both clinical research and practice, offering insights directly from patients about their symptoms, daily functioning, and overall quality of life, beyond what clinical measures can capture. This patient perspective is crucial for understanding the comprehensive impact of diseases and treatments. PROs assess various health-related concepts, particularly in quality and performance measurement, including health-related quality of life, functional status, symptoms and symptom burden, health behaviors, and patient healthcare experiences.[3-6]

Patients play a vital role in defining research priorities, participating actively in research processes, and providing valuable preferences data and PROs. Their first-hand experience with diseases and treatments guides researchers to focus on the most relevant and impactful areas of study. Patient advisory boards and focus groups ensure that research agendas reflect the true needs and preferences of the patient population. In participatory research models, patients collaborate with researchers in designing studies, developing survey instruments, and interpreting results, ensuring that the research is grounded in real-world experiences.[3-6]

Caregivers are equally important in health care and research, providing crucial support for patients, especially those unable to fully participate due to age, cognitive impairment, or severe illness. They help complete PRO measures, offer proxy reports based on their observations, and manage personal electronic health records. Caregiver experiences inform the development of care models that are more responsive to the needs of both patients and caregivers, helping to identify gaps in care and areas where support services can be improved.[7-9]

Involving patients and caregivers in health preference and PRO research significantly enhances the relevance and validity of findings by ensuring that studies reflect real-world experiences and preferences. This inclusion improves research quality, participant recruitment, and the design of patient-facing materials, while ensuring that study results are effectively communicated to patient communities. Proper planning for patient and caregiver involvement, addressing compensation, roles, training, and support, is essential for optimizing their contributions throughout the research process. This approach not only improves research quality but also empowers patients and caregivers by giving them a voice in the research process, ultimately leading to a more engaged and informed healthcare experience.[4,8-10]

While involving patients and caregivers in HPR and PRO research offers significant benefits, it also presents several persistent challenges. Securing a demographically diverse patient population can be challenging, often hindered by insufficient logistical, financial, or educational resources needed to effectively incentivize and engage patients. Sustaining patient participation over time is another hurdle, exacerbated by the risk of tokenism where a single patient is expected to represent an entire demographic. Moreover, patients frequently express dissatisfaction when their engagement results are not shared with them, which can deter future participation. The availability and readiness of caregivers also impact the quality of care transitions for patients post-hospitalization, underscoring the importance of clearly defining and supporting caregiver roles to enhance interventions during the discharge process.[10,11]

To successfully involve patients and caregivers in health preference and PRO research, meticulous planning and resource allocation are essential to ensure representative participation and address literacy and language barriers. Understanding the specific preferences of diverse subgroups is crucial; for instance, investigating issues like menopausal symptom relief for women requires targeted approaches. Clearly articulating the purpose of involving patients as research partners helps establish their role and fosters trust. Selecting patient partners who have prior research experience and established relationships with researchers, and including multiple patient partners on the study team, enhances the effectiveness of their involvement. Such clarity and preparation can build greater trust and commitment to the research project. Moreover, researchers must balance the need for scientific rigor with the practicalities of effectively integrating patient and caregiver input, ensuring that the research is both robust and reflective of real-world experiences.[10-12]

Patients and caregivers are invaluable partners in health preference and patient-reported outcomes research. Their involvement ensures that healthcare research and decision-making are grounded in the realities of those who are most affected by health conditions and treatments. By continuing to engage patients and caregivers in meaningful ways, we can advance towards a more patient-centered healthcare system that delivers better outcomes and higher satisfaction for all.

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References:

1. Craig BM, Lancsar E, Mühlbacher AC, Brown DS, Ostermann J. Health preference research: an overview. Patient. 2017;10(4):507-510.
2. Van Overbeeke E, Vanbinst I, Jimenez-Moreno AC, Huys I. Patient centricity in patient preference studies: the patient perspective. Front Med. 2020.
3. Smith MY, Janssens R, Jimenez-Moreno AC, et al. Patients as research partners in preference studies: learnings from IMI-PREFER. Res Involv Engagem. 2023;9(1):21.
4. Garvelink MM, Ngangue PA, Adekpedjou R, et al. A synthesis of knowledge about caregiver decision making finds gaps in support for those who care for aging loved ones. Health Aff. 2016;35(4):619-26.
5. Committee on Family Caregiving for Older Adults; Board on Health Care Services; Health and Medicine Division; National Academies of Sciences, Engineering, and Medicine; Schulz R, Eden J, editors. Families caring for an aging America. Washington (DC): National Academies Press (US); 2016 Nov 8. Available from: https://www.ncbi.nlm.nih.gov/books/NBK396398/.
6. Zendjidjian XY, Boyer L. Challenges in measuring outcomes for caregivers of people with mental health problems. Dialogues Clin Neurosci. 2014;16(2):159-69.
7. Patrick K, Kebbe M, Aubin D. A home for patient-oriented research. CMAJ. 2018;190(20)
8. Manafo E, Petermann L, Mason-Lai P, Vandall-Walker V. Patient engagement in Canada: a scoping review of the ‘how’ and ‘what’ of patient engagement in health research. Health Res Policy Syst. 2018;16(1):1-11.
9. Yeh MY, Wu SC, Tung TH. The relation between patient education, patient empowerment and patient satisfaction: a cross-sectional-comparison study. Appl Nurs Res. 2018;39:11-7.
10. Woodward EN, Castillo AI, True G, Willging C, Kirchner JE. Challenges and promising solutions to engaging patients in healthcare implementation in the United States: an environmental scan. BMC Health Serv Res. 2024;24(1):29.
11. Van Schelven F, Boeije H, Mariën V, Rademakers J. Patient and public involvement of young people with a chronic condition in projects in health and social care: a scoping review. Health Expect. 2020;23:789-801.
12. Craig BM, Mitchell SA. Examining the value of menopausal symptom relief among US women. Value Health. 2016;19(2):158-66.