When it comes to chronic diseases like arthritis, heart disease, and cancer, “getting better” is unfortunately not always the same as “feeling better.” Doctors have clear, biological measures that determine whether a treatment is successful in battling an illness—tracking tumor size, detecting the presence of cancerous cells, or even just measuring cholesterol. But determining how a patient is feeling? That’s a different story.
There are many reasons why this isn’t so cut and dried. One is that these treatments often bring with them side effects which, at best, cause mild discomfort and, at worst, can be even more troublesome than the symptoms of the disease. Take chemotherapy for example—while it saves the lives of many cancer patients, it can also cause debilitating pain, nausea, and fatigue. When these effects become worse than the symptoms of the cancer itself, patients may become inclined to stop treatment altogether.
For other chronic conditions, like arthritis, for which pain is the primary symptom, the right treatment is one that best alleviates pain while bringing minimal side effects. But different treatments can be more effective at lessening pain for some patients than for others, and tolerance thresholds vary widely from individual to individual.
So how do physicians know how well a pain medication is working, or how debilitating side effects have become? Our patients have to tell us. Formally, we call these self-assessments “patient-reported outcomes” (PROs), and they are crucial to both individual patient care and clinical studies that evaluate new therapies. While blood tests and CAT scans can tell us if a treatment is working physically, they can’t tell us if a patient's pain has lessened enough to resume normal activity, if the illness has affected his or her social relationships—basically, the degree to which disease symptoms or side effects have impacted the patient's daily experiences. Answers to these questions help us determine if a course of treatment is not only curing disease, but also improving the patient’s quality of life.
Unfortunately, there are almost as many tools to record, measure and evaluate PROs as there are symptoms, making it difficult to compare these outcomes from patient to patient and across populations. Tracking symptoms across populations is crucial, because it helps us determine what treatments are most effective for large groups of people and what symptoms or side effects need to be addressed by further research.
Take the chemotherapy example—in the past, its most commonly reported side effects were pain and nausea. Physicians and researchers recognized this, and developed ways to manage these symptoms. We’ve learned from PROs that the most common side effects are now fatigue and trouble falling/staying asleep. With this knowledge, researchers can focus on determining what causes these symptoms, and finding ways to alleviate them.
This is why my research group, along with a network of collaborators and support from the National Institutes of Health, set out to develop a standard set of computerized questionnaires to help physicians gather PROs for many different illnesses. These questionnaires and their defined sets of answers are designed so that patients can more clearly communicate their symptoms to their physicians in a common language. For example, rather than just asking how often and to what degree a patient experiences pain, based on their own personal criteria, the questions seek out a range of emotional and physical responses—how often the pain makes you cry, if it prohibits you from working for two hours at a time, or limits your ability to walk up the stairs, for example.
The final output provides researchers with a clearer and more consistent picture of individual experience. And, because patients are asked the same questions with the same standard set of quantifiable responses, researchers can easily and reliably compare pain and other symptoms levels from patient to patient and across large groups. This will generate a valuable pool of data for evaluating treatments, allowing patient experience to be accounted for in the same evidence-based way as factors like tumor size, cholesterol levels, and blood cell counts.
This project, known as the Patient-Reported Outcomes Measurement Information System (PROMIS), began in 2004, and we’ve since released PRO questionnaires for general use in clinical practice and research. We have also produced more targeted assessments for emphysema, cancer, depression, back pain, heart failure, multiple sclerosis, arthritis, and several other conditions. We are now working to further validate the questionnaires we’ve produced, as well as develop new ones for diseases such as cerebral palsy, stroke, gastrointestinal conditions and fibromyalgia, as well as general rehabilitation.
It is our hope that, through widespread use of the PROMIS system and related works, we can elevate the patient experience to the same level of review as clinical tests by measuring these experiences in a standard, straightforward, and scientifically rigorous way. This will contribute to more evidenced-based healthcare for our nation, and better quality of life for our patients. After all, when it comes to treating illness, “How are you feeling?” might be the most important question.