Posted on December 22, 2022

By Krystal Redman (KR), DrPH, MHA, (they/she), Executive Director

Patient-Reported Outcomes – They May Be Beneficial But…

Patient-reported outcomes (PROs) are self-reported assessments provided directly by patients. According to Chen et al. (Chapter 13 – Incorporating Patient-Reported Outcomes Into Early-Phase Trials (editors:  Kummar and Takimoto) Novel Designs of Early Phase Trials for Cancer Therapeutics, 2018), “PROs are a type of measurement based on a report that comes directly from the patient about the status of their health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.” PROs can be used for research, to improve clinical and supportive care, and for quality assessment purposes as they provide reliable and valid data.

Dr. Shoshana Rosenberg, ScD, MPH (Weill Cornell Medicine), one of the presenters for the session entitled “What are PROs and evidence for benefit?” proposed that PROs, rather than clinician evaluations, have been demonstrated to better capture the patient experience. Additionally, there is growing evidence supporting the routine incorporation of PROs into clinical practice. It was postulated that the use of PRO-based interventions are associated with improved symptom management, enhanced patient-provider communication, and better outcomes, including quality of life and survival, in patients with advanced cancer. 

Among patients in the post-active treatment and survivorship phase of care, PROs are critical in identifying and understanding late and long-term effects of treatment, identifying unmet needs, and determining if survivors are receiving appropriate follow-up care. There is also data in the survivorship setting demonstrating how PROs can predict other outcomes, such as adherence to aromatase inhibitors in breast cancer patients. PROs are used for many purposes including improvement of clinical and supportive care, for research aimed at supporting quality of life, and to capture the  patient’s  experience throughout the cancer care continuum.

Despite the claimed utility of PROs, the questions persist about their benefit. Many studies have leveraged PRO-based interventions to improve communication, improve symptom management, and improve outcomes such as quality of life and survival. Additionally, PROs can be used to inform treatment decision-making and assess the unmet needs of the patient. Typically, PROs are administered when the patient is in the waiting room. Oftentimes patients do not desire to complete an assessment when they have other priorities, concerns, or stressors on their mind. Patient reported outcome measurements (PROM) often do not consider possible disparities and factors such as age, race, culture, religion, gender, sexuality, literacy level, language, mode of delivery (paper vs. tablet vs. link), participant burden (time it takes to complete), etc.

Dr. Rosenberg stated that when selecting PROs, it’s critical to ensure that the information learned is generalizable and that the benefit patients can gain through the incorporation of PROs into cancer care is equitable. Prior to a provider implementing PROs in their practice due to their own beliefs of the possible benefits, they also need to consider the disparities in PRO collection. In addition to the disparities and considerations noted above, non-white patients are less likely to participate in PROs in clinical trials.

We know that non-white people are less likely to participate in clinical trials and much work needs to be done in the health and science community to address this gap. But just like the issues around Black and Brown folx not participating in clinical trials, when implementing PROs, trust must be built. Why would a patient feel comfortable enough to open up and share their experience, including details about how they are feeling or other personal information if they 1) do not trust their provider, and 2) do not believe that anything will be done to support or “fix” their issues?

In summary, PROs have demonstrated significant benefit in both clinical and research settings and randomized trials have shown that PROs can meaningfully improve patient outcomes. Importantly, ongoing pragmatic trials will help to optimize the implementation and utility of PROs across the cancer care continuum — however, this may not be generalizable to other patient populations, in particular communities and patients who exist deep within the margins. 

Just as I started to question why no researcher was naming some of the major considerations with PROs and identifying equitable strategies for implementation, Dr. Oluwadamilola (Lola) Fayanju, MD, MA, MPHS, FACS (Perelman School of Medicine at the University of Pennsylvania) began her discussion on “How to incorporate PROs into clinical care”. BCAction is very familiar with Dr. Fayanju’s work as she joined me in a thought-provoking discussion about the systemic inequities in breast cancer diagnosis and treatment outcomes, and what we can do to address these disparities, during our 2021 Billie Gardner Loulan Memorial Benefit. 

Dr. Fayanju noted that PROs can enhance shared decision-making. And when administered in multilingual, level-appropriate, and culturally aware formats, PROs can be used to address the individual and collective health status of potentially vulnerable groups including children, older adults, those with disabilities, and those from racial/ethnic minority backgrounds, thereby providing an opportunity not only to address the needs of individuals but also to identify potential disparities across groups.

Dr. Fayanju stated that PROs are now recognized as important for care and research: they  help facilitate direct and timely communication from patients, they offer benefits for SDM adherence, they improve quality of life and even survival, and they may reveal previously unknown side effects and challenges. In addition, there is an opportunity to identify vulnerable patients and promote equity BUT also the danger of exacerbating disparities in communication and outcomes. 

Dr Fayanju stated that we need multifaceted strategies, modalities, and settings to optimize patient engagement. “For a lot of our patients, cancer is just one more burden in a life that is already so burdensome.”

She challenged us to think about how disparities can influence PROs. For instance, it’s very hard for patients to share information around being unhoused, family dynamics, etc. without feeling like there will not be anything done to help or support them. It is also  a known fact that patients seldom get a chance to have enough face-to-face time with their provider, which would allow for relationship and trust building. Oftentimes, patients feel rushed. 

Patty Spears, BS (University of NC) spoke from the perspective of a patient advocate and quoted the statement by Sandra Finestone,PROs consider not only WHAT IS THE MATTER with the patient, but also WHAT MATTERS to the patient.”

Ultimately, the best decision is made when everyone is fully informed of the actual benefits and harms that are important to patients! These are things patients should consider before deciding if they should engage in PROMs or not, and what possible benefit it might have for them. BUT if there isn’t trust, there isn’t a relationship, and if culturally humble strategies are not met, how would the patient know if anything could be done or offered to support them if they were to disclose possible hardships in their health or living conditions? 

Also, rather than a long and detailed PRO tool that the patient has to complete online or in the office, how about asking just one question: what is impacting your quality of life right now? The answer to this can change over time and this should be a free-text response not multiple choice, in order to allow patients the opportunity to express what truly matters to them.

Overall, PROs can have many benefits, but more research needs to be done, focusing on diverse patient populations, and clinicians need to have a robust implementation strategy that considers the needs and identities of their patient base. The creation and development of any PROs needs to not only take into account patient groups but also involve patients in the creation of them. Trust and rapport needs to exist in order for all patient groups to feel comfortable engaging. To lessen any potential burdens for engagement, clinicians should consider a few short and to-the-point questions, such as “what is most important to you today?”, “What is impacting your quality of life today?”, and then once they have their answer, do something about it.