Listening to Patients: Developing Patient-Reported Outcome Measures to Improve Patient Care
Historically, a patient’s subjective report of how they feel has not been viewed as a reliable form of evidence when it comes to FDA approval of a drug, device, or medical procedure. Such reports were considered “soft” data, while quantifiable data such as blood cell counts, cholesterol levels, or death itself, were considered the kinds of “hard” data on which regulatory approval decisions needed to be based.
In the past decade, however, leading clinicians, scientists, and regulators have embraced the importance of patient-reported outcomes in evaluating treatment efficacy and outcomes. Not only are PROMs seen as necessary and valuable in evaluating efficacy in drug and device trials, but they are also recognized as irreplaceable in many clinical research areas because they provide data that cannot be captured any other way. In short, these measures are increasingly indispensable.
PROMs are especially relevant when objective measures of treatment benefits are lacking or unreliable. Do patients feel any better, for example, after taking treatment X vs. treatment Y, and how closely does the patient’s experience match the objective benefits of treatment? For an increasing number of common conditions, it’s the patient’s degree of bother, discomfort, or distress that is the main target of treatment, and this aspect is best assessed via a scientifically constructed and validated PROM. Frequently, PROMs are also used for diagnostic screening. A short questionnaire can be used to identify patients at risk for a condition, or those who may need further assessment and alternative treatments.
HealthCore has extensive experience developing and validating PROMs for a variety of stakeholders. In collaboration with clinical research partners, and with funding from the National Institutes of Health, HealthCore is developing a novel PROM instrument for assessing underactive Bladder (UAB), a potentially common urologic syndrome in men and women which can lead to incontinence, leakage, and other highly bothersome difficulties with urination.
This new patient-reported diagnostic aid, or symptom screening tool, will help physicians to identify patients with UAB symptoms who may need treatment and assess potential benefits of treatment for underactive bladder. The design and construction of this symptom screening tool is based on three stages of qualitative interviews and focus groups with carefully selected patients in collaboration with our partner clinical sites across the country. The iterative process of screener development and validation is depicted above.
HealthCore has developed similar PROM screening instruments for understanding symp¬toms of hypogonadism in men, as well as symptoms of vulvovaginal atrophy (or genitourinary syndrome of menopause) in women. HealthCore has also developed a novel PROM instrument for assessing subjective aspects of cognitive disturbance in schizophrenia.
Meticulously analyzed qualitative interviews and focus groups with patients serve as the cornerstone for identifying and measuring subjective aspects of these common and highly distressing conditions. With an outstanding team of qualitative researchers, HealthCore is well positioned to create the kind of thorough, validated PROM instruments required by the next generation of researchers, leading to more efficient and effective care for patients.