Patient Reported Outcomes in Clinical Cancer Trials

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What are Patient Reported Outcomes (PROs) and why are they important to patients?
Joseph Steward,  Moore’s Cancer Center, UCSD Health
What are Patient Reported Outcomes and why are they important to patients?

Patient reported outcomes (PROs) can be thought of as any report or measurement of a patient’s health that comes directly from the patient. PROs have become increasingly important in the care of patients with cancer not only to better understand the wide range of symptoms patients experience from their condition, but also to characterize side effects that come from new therapies being developed to better treat and increasingly cure different types of cancer.  Symptoms that come from a cancer therapy are called adverse events, which can include not only physical side effects, but also emotional or any side effect that changes a patient’s quality of life during treatment. PROs are important to patient’s because they allow the patient to directly inform all aspects of their own treatment, based on their perceived health and quality of life during care.

 

Why are PROs getting more attention recently?

The primary reason PROs have become increasingly important in cancer care is because of the explosion of novel therapies being developed to treat all types of cancer. New areas of cancer treatment such as immunotherapy have allowed patients to live for years with their cancer as a more chronic disease, as compared to only months with older forms of treatment. Although these new therapies can lead to lasting benefits in a large number of patients, they can also cause harmful side effects that are hard to predict in other patients. Therefore, it’s highly important to understand all harmful side effects from a new therapy before it can be approved by the United States Food and Drug Administration.

To better increase the focus on patient quality of life during the development of new cancer therapies the US Congress passed the 21st Century Cures Act in 2016. The 21st Century Cures Act aims to advance new drug therapies while increasing the focus on patient well being by requiring the FDA to publish PRO data with a new medication application. This represents a shift to “patient centered” drug development in the United States, making PROs of adverse events and their own quality of life an essential component for a new therapy approval.

 
How are PROs measured in clinical trials, and how are they used to evaluate benefits and harms of new therapies?

PROs are measured in clinical trials via questionnaires the patient completes during the course of their treatment. Patients self-report various health measures being assessed in the clinical trial at specific time points measure health changes during the course of treatment. While paper questionnaires have been used in the past, patients can now commonly complete questionnaires via internet and mobile phone based methods.

The three most common categories of measurement included in patient reported outcome questionnaires focus on clinical issues, including reporting of disease related symptoms, self-reporting of adverse effects from the therapy, and overall physical functioning during treatment. Safety assessment of adverse events are normally measured using a scale from 1 to 5, 5 being most severe and 1 being the least. Measurement of adverse events represents one of the most important patient reported outcome measures because these ultimately determine the tolerability and safety of a new treatment. If the side effects from a new therapy are too severe during a trial it cannot be approved by the FDA for wider use. Measurement of disease related symptoms refers to the measurement symptoms which are known to come from a patient’s cancer versus the therapy. Patient self-reporting of these symptoms is important because they serve as a method to assess the benefit of a new therapy to patients. If patients continue to report disease related symptoms, this could indicate that a new therapy fails to provide the expected health benefit to the patient. Finally, reporting of physical functioning during treatment refers to any change in a patient’s normal daily physical functioning such as mobility during treatment. Measurement of a patient’s perceived physical functioning and how it has changed during treatment is important because it represents an assessment of a patient’s change in their perceived health and quality of life.  Each of these categories of patient self-reporting represents health measures that ultimately allows the FDA to determine if a new therapy will provide clinical and quality of life benefit to patients undergoing treatment. 

 
Patient Self Reporting of Emotional Well Being and Overall Quality of Life in Routine Treatment and Cancer Survivor Care

PRO measures are also increasingly being used to assess patient emotional health and well being after diagnosis and during routine treatment. A diagnosis of cancer commonly causes a marked increase in patient self-reported levels of stress, anxiety and depression. Diagnosis and treatment also cause significant social sources of stress, such as disruption to normal family life and extreme financial difficulty due to increasing costs of healthcare. Although these symptoms are difficult to measure quantitatively, they represent important components of a patient’s overall quality of life and directly influence health outcomes. There’s also been an increased focus on improving care and quality of life for cancer survivors as new therapies decrease mortality rates for many cancer types. Survivors of cancer face lifelong complications from their disease and treatment that must be continually monitored. Similarly, survivors of cancer normally face lifelong emotional and psychological stress from their disease and treatment which harms their overall quality of life. PROs are therefore increasingly being used in routine treatment and survivorship care to understand all ways to improve patient quality of life. 


 

*Joseph Steward works at Moore’s Cancer Center UCSD as part of the Clinical Research staff focusing on genetics and genomics. 

Joseph graduated from UCSD in 2015 with a BS in Biology, and began working in oncology and the application of genomics and clinical care (understanding all the mutations that cause cancers, uses in early detection like liquid biopsies, and matching targeted therapies to patients based off of their oncogene genotypes).  He is passionate about all areas of patient advocacy and education, and is interested in patient support and helping to create educational resources for patients.  Thank you Joseph for volunteering and helping GRACE!

 

 

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