The Food and Drug Administration (FDA) describes patient-reported outcomes (PROs) as “measures of a patient’s health status as reported directly from the patient without added interpretation by a healthcare worker or anyone else, such as a pain scale.”
When collected, these measures can help inform clinical practice, including decision-making about treatments for rare diseases.
While several efforts are underway to encourage the use of PROs in clinical research and patient treatment, actual application of patient-reported outcome data remains inconsistent. Connecting with patients through digital tools already familiar to them and applying PRO-related tools thoughtfully can improve data collection, offering better options for data use.
The Value of Patient-Reported Outcome Measures in Rare Diseases and Specialty Treatments
Patient-reported outcomes provide an important source of information about symptom progression and treatment efficacy. PROs “are both indicators of clinical practice and endpoints of clinical trials without the interpretation of patients’ responses,” write Mio Sakuma and Takeshi Morimoto in a 2023 article in the International Journal for Quality in Health Care.
While several conditions have clear biomarkers for diagnosis, they lack biomarkers to quantify disease progression. Rheumatoid arthritis, for example, relies on testing for inflammatory factors, but a patient’s blood test results do not correlate precisely with the intensity or progression of symptoms.
Even when biomarkers for disease progression exist, they may not translate into clear information on the patient’s quality of life. Patients experiencing severe side effects from a treatment for cancer, for instance, may report worsening personal outcomes even as testing indicates that the treatment is targeting the cancer effectively.
PROs also play a key role in the development of orphan drugs for rare diseases and in identifying new uses for existing orphan drugs. “Within [the] clinical trials framework, PRO may provide evidence to support medicines approval, labeling and marketing claims,” write Maria Manuel Teixeira and fellow researchers in a review of PROs for regulatory approval of oncology medications in the EU between 2017 and 2020.
Engaging Patients in Self-Reported Data Generation
Technology provides ways to connect with patients and collect patient-reported data. Smartphone and internet use are common, write Richard Wike and fellow Pew researchers, and they continue to grow. For example:
- 85 percent of U.S. residents currently own smartphones, while an additional 11 percent own a mobile phone that is not a smartphone.
- 93 percent of U.S. residents report using the internet to access information online or to send and receive email. Of these, 85 percent say they’re online at least once a day.
- 72 percent of Americans say they use social media sites to connect with others and gather information.
While social media and shopping remain among the most common uses of web-based technology for Americans, many use their phones and associated apps for other purposes as well. Tracking health-related information has become a more common use of technology in recent years, manifested in the form of habit tracking apps, pedometers, and smart watches that track factors like heart rate, writes Rajiv Leventhal at Insider Intelligence:
- 63.4 percent of U.S. adults report using healthcare platforms, apps, and other tools to track health-related information in the past year.
- 50 percent of health app users use their app at least once per day.
- Health-related app use has increased 6 percent since late 2018.
Technology offers a way to connect with patients. Yet constant connection to the web also generates a sense of overwhelm, especially for those who use their smartphones or check social media regularly. This sense of overload can be exacerbated by poorly-designed methods of collecting PROs.
“Respondent burden needs to be carefully considered and addressed to avoid high rates of missing data and poor reporting of PRO results,” write Olalekan Lee Aiyegbusi and fellow researchers in a 2022 article in Nature Communications. Failing to consider PRO collection methods from the patient’s perspective can compromise the quality of collected data, which in turn can compromise regulatory decision-making and clinical care.
Effective collection and management of patient-reported outcome data can also lead to more effective results for patients. For example, some oncology medications include PRO data in their drug labeling. However, both the appearance of PRO data in oncology drug labeling and the presentation of this data are inconsistent, write David Cella and fellow researchers in Frontiers in Pharmacology.
By managing information more consistently, drug manufacturers, distributors and others involved in the creation of rare disease treatments can communicate more effectively with patients.
Managing Patient-Reported Outcome Data Effectively
Patient-reported outcome data (PROMs) offer great promise for capturing clinical quality measures in many rare disease treatments. Still, they remain under-utilized. While the healthcare industry has generated many tools for collecting PROs in recent years, less than one percent of clinicians currently use PROMs in their clinical workflows, Jeanette Y. Ziegenfuss and fellow researchers write in a 2022 article in the American Journal of Medical Quality.
“Routine collection of PROMs is difficult to achieve across an entire healthcare organization,” note David A. Snowdon and fellow researchers in a 2023 BMC Health Services Research article. To implement PROMs more effectively, organizations should start with a clear understanding of what information is to be collected and for what purposes.
First steps in managing patient-reported outcomes include distinguishing among patient-reported outcomes (PROs) and related concepts, including patient-reported outcome measures (PROMs) and patient-reported outcome-based performance measures (PRO-PMs). The Centers for Medicare and Medicaid Services (CMS) distinguish among these three concepts as follows:
- PROs include reports that come directly from patients. They include information on function and health-related quality of life, symptoms and their effect on the patient, and health-related behaviors like diet, exercise and smoking.
- PROMs are tools that collect PRO information. They may include surveys, digital platforms, apps and similar tools.
- PRO-PMs collect patient-reported outcomes gathered by PROMs and use it to generate actionable insights. They include status assessments and inventories of patient function. PRO-PMs combine patient-reported outcome information with clinician observations and measurements to allow clinicians to compare patient outcomes over time. Some tools also allow researchers to compare patients to one another.
Using PROs effectively requires more than simply connecting with patients to gather their feedback. Clinicians and others in the healthcare field must apply thoughtful expertise in selecting PROMs, evaluating PRO-PMs, and interpreting information gathered from patients, writes Stephen C. Weber in a 2023 article in RF Quarterly.
Weber advocates the use of p-values as a starting point rather than an ending point for effective PROMs use, and suggests incorporating nuance in PRO interpretations. Thoughtful PROMs use can reduce question fatigue among patients, focus studies on clinically significant information, and tailor PRO-PM results more closely to patients’ definitions of success.
Choosing the right PROMs can help clinicians and other participants in healthcare focus on meaningful patient-reported outcomes and build better tools for assessing and comparing outcomes among patient groups. The right digital platform, for instance, will not only connect patients to providers and researchers but also guide clinicians in making more considered choices about the information requested and its contextualization in patient care.
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