Patient-Reported Outcomes

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Patient-reported outcomes (PROs), or measures elicited directly from patients, can provide insights into patients’ well-being that cannot be captured by laboratory values. PROs, which can include measures of physical symptoms, emotional health, and treatment preferences, have been shown to enhance shared decision-making between physicians and patients, enhance workflow efficiency when used regularly, and allow for more nuanced predictions of disease trajectory (13). The US Food and Drug Administration and the Standard Protocol Items: Recommendations for Interventional Trials PRO extension have each emphasized the need to include PROs as clinical trial endpoints, and kidney patients have prioritized that PROs be included in their overall treatment plans (46). As a result, PROs have continued to gain attention and interest in the nephrology community.

In this piece, Devika Nair, MD, a postdoctoral clinical research fellow in nephrology at Vanderbilt University, whose own research interests involve PROs, interviews Debbie Gibson, MD, professor of medicine in pediatric nephrology, and Noelle Carlozzi, PhD, associate professor of medicine in physical medicine and rehabilitation, both at the University of Michigan’s Institute for Healthcare Policy and Innovation. She also interviews Derek Forfang, a kidney transplant patient and passionate patient advocate, who is a member of the National Kidney Foundation’s Kidney Advocacy Committee, Public Policy Committee, and a member of the Kidney Patient Advisory Council.

Drs. Gibson and Carlozzi discuss the limitations in and opportunities for incorporating PROs into clinical care and research, and Mr. Forfang provides his perspective as a patient and research partner.

  • DN = Devika Nair; DG = Debbie Gibson; NC = Noelle Carlozzi; DK = Derek Forfang

DN: What are some limitations of current PROs and challenges to incorporating them into care?

DG and NC: Several of the PROs we are currently using are relatively new, and as such we are still collecting data about their clinical utility (both strengths and weaknesses). These data are integral to using PROs effectively in a clinical setting. PROs also need to be used in conjunction with other reports to provide a full clinical picture. For example, the intersection between patient report, clinician report, and biomarker data is, in and of itself, a very important piece of information. The congruence and discrepancies can provide a rich clinical picture and present new challenges. Ultimately, PROs give patients a voice to highlight the different aspects of care that are important to them.

DG: Relatively few disease-specific PROs have been developed for individual kidney diseases. The use of generic PROs may be reasonable but they will benefit from a dedicated effort to ensure that concepts important to patients are included in the PRO and that the response range is appropriate for these patients.

DN: What are some ways PROs can be better incorporated? What can healthcare professionals do with the information obtained from PROs?

NC: Ideally, PROs could be incorporated into a patient visit, and things could be flagged that the clinician might want to address in the clinic visit, either because it indicates that something has changed for the patient or because there is an elevation that might suggest a clinically significant problem. In this manner, appropriate referrals might be made. Information from PROs may also raise patient-specific concerns that might otherwise not be addressed during a clinical care visit.

DG: In practice today, many health systems are collecting PROs. Some use the electronic health system to gather the responses and plot longitudinal results to facilitate the use of PROs in the clinical setting. PROs are being collected in dialysis facilities as well. This information can be incorporated into the routine clinical data review to inform patient interactions and management decisions. Currently, many practices and health systems do not have a comprehensive approach to PRO assessment and tracking. Although progress is being made, we encourage healthcare professionals to take the time to review the results and include this review in patient interactions.

DN: What are some future areas that researchers and clinicians need to focus on with regard to PROs? What about the need to move beyond health-related quality of life?

NC: Health-related quality of life (HRQOL) is the impact of a disease on an individual’s physical, mental, and social health and well-being. Several different symptoms are commonly included under this umbrella. There are certainly aspects of HRQOL that we don’t currently capture (meaning that measures are not currently available to assess all of the different factors that relate to HRQOL). In particular, although we have several measures that are generic (meaning they can be used to assess symptoms and problems across multiple health conditions), we frequently lack disease-specific measures—or measures that capture the different aspects of a disease that are more unique. In many ways, there is still a lot of work to be done in terms of HRQOL and capturing the concerns that are most relevant to our patients.

DG: We have many opportunities. Consider the need to generate evidence to support intervention(s) for specific PRO findings, generate evidence to facilitate interpretation of the PRO results as absolute values or change in scores from one assessment to the next, generate PROs fit for adults and children, and generate observer-reported outcomes (ObsROs) for parents or family members to report on signs of disease when the patient is unable to do so. This is an area rich in opportunities, and I believe our healthcare delivery will benefit greatly as this area of assessment matures.

DN: Can you tell me a little bit about your role as a patient advocate?

DF: As an ESRD patient who has dealt with diabetes for almost my entire life, my focus is on chronic disease and its impact on my patient population. Having a leadership role in the patient community, I feel my role is to help other patients understand that PRO measurements can affect our care. The Cen- ters for Medicare & Medicaid Services (CMS) Quality Incentive Program and Dialysis Facility Compare programs are two good examples of opportunities for this.

DN: What do you think are some challenges that patients and members of their care team face when responding to PROs?

DF: Transparency is key to having patients better align their goals with their care team. I believe most patients don’t understand measures or even know they exist; I know I didn’t. I thought measures were something the clinic had to worry about, and I thought they had nothing to do with me individually. Having a basic understanding of the difference between clinical measures and a PRO is also important.

DN: In your opinion, what are some future areas for focus in the development of additional PROs?

DF: I’m thinking about the six areas defined by the Meaningful Measures Initiative. One area that stands out is the goal to “Strengthen Person and Family Engagement as Partners in [Their] Care.” This measure is broken down to three areas. The first involves care being personalized and aligned with patient goals. Last year I participated in a CMS Technical Expert Panel on PROs. Two topics prioritized by patients and providers involved patients’ life goals and the need for patients to feel safe in their dialysis facilities. Understanding what is important to patients and creating an individual care plan around their culture, goals, and values can help better activate patients in their own care. If a patient desires to travel, work, see friends more often, or transition to a more palliative approach, each of these should be a topic of discussion when a care plan is developed.

A second area involves a patient’s experience and his or her functional outcomes. We already have some PROs that measure this, such as the In-Center Hemodialysis Consumer Assessment of Healthcare Providers and Systems (ICH-CAHPS) Survey and the Kidney Disease Quality of Life (KDQOL) instrument, but certain things still aren’t captured. I think the ICH-CAHPS could be improved, both by making the survey questions more specific, adding questions around fear or perception of retaliation, and by including patients in the debriefing of the results when facilities receive their scores. The KDQOL instrument doesn’t take into account how patients adapt to their changing functional status. I took the survey recently and scored fairly low because I’ve been in a wheelchair for the past 2 years, but I feel that the quality of my life is very good. I travel, socialize, have interests and hobbies, and the work I do volunteering is extremely rewarding. So, although the wheelchair has been limiting in some ways, I still continue to achieve and do the things that are important in my life. PROs should create a dialogue between the patient and the care team, both to improve quality of care and to improve outcomes that are important to patients.

The third area involves ensuring that end-of-life care is delivered according to a patient’s preferences.

DN: What do you wish clinicians and healthcare providers knew about PROs?

DF: I know PROs can be burdensome, but if the right questions are asked and the answers allow for meaningful feedback to patients and providers, they are worth the time. If I were a clinician I would want to know my patients’ culture, goals, and values, because this could reduce nonadherence, improve shared decision-making, and so on. I would also want my patients to understand my goals for them as a clinician. We need to better align our goals, and PROs are a new way to measure success, but more importantly, they open communication. We as patients can help our clinics achieve success if we are welcome to speak up and get involved. We want to partner with you.

DN: Thanks to everyone for your valuable insights into this important issue.

March 2019 (Vol. 11, Number 3)

Suggested Reading

1. Patient-reported outcomes. The National Quality Forum. July 17, 2018. https://www.qualityforum.org/Projects/n-r/Patient-Reported_Outcomes/Patient-Reported_Outcomes.aspx.

2. Hughes TM, et al. Association of shared decision-making on patient-reported health outcomes and healthcare utilization. Am J Surg 2018; 216:7–12.

3. Lango MN, et al. Baseline health perceptions, dysphagia and survival in head and neck cancer. Cancer 2014; 120:840–847.

4. United States Food and Drug Administration. Guidance for Industry. Patient-reported outcome measures: Use in medical product development to support labeling claims. Federal Register 2009; 74:65132–65133.

5. Calvert M, et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: The SPIRIT-PRO extension. JAMA 2018; 319:483–494.

6. Urquhard-Secord R, et al. Patient and caregiver priorities for outcomes in hemodialysis: An international nominal group technique study. Am J Kidney Dis 2016; 68:444–454.