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PCOR: What is it and Why is it Important to Patients with a Rare Disease?

Nurse sitting next to patient and touching the patient's hand

The Patient-Centered Outcomes Research Institute (PCORI) promotes research that emphasize hypotheses answering critical questions from patients and caregivers. They focus specifically on generating results that can be easily applied to patients and specifically underscore the importance of engaging patients throughout the research process. They believe that patient-engaged research will culminate in findings that will be increasingly utilized in a clinical setting.

What exactly is Patient-Centered Outcomes Research? PCOR investigates commonly used treatment options of a clinical disease in a real-world setting. It fills evidence gaps such as “are there different ways of managing the clinical condition of interest? Is it difficult to choose? Do different doctors recommend different treatment approaches?” Most importantly, patients are involved in research design and conduct, ensuring that the investigation provides solutions to vital patient questions. PCOR answers the question “which of the available options is best and for which patients?” and produces evidence that helps patients, caregivers, and physicians make more informed healthcare decisions.

Patient-Centered Outcomes Research is especially crucial for patients with rare diseases such as ITP. Considering that a cure for ITP has not yet been discovered, there tend to be differences in the way this disorder and its symptoms are treated in a clinical setting. While it’s wonderful that patients have a plethora of therapy options, questions still remain regarding the best method to treat ITP. In this scenario, utilizing the main tenets of PCOR to develop studies that contribute evidence on the best way to manage the disorder will considerably influence patient and caregiver quality of life. Ultimately, PCOR will be invaluable for PDSA and ITP patients, as the development of patient-centered studies will surely yield meaningful information on the best way to manage ITP for each patient.

PDSA is developing an application for funding consideration through PCORI to look at the experiences of ITP patients using second-line therapies on ITP management and quality of Life. We want to learn if there are different groups of ITP patients who may benefit more from receiving one type of second-line therapy first, to reduce the amount of trial and error that occurs when selecting the most appropriate therapy to use when access to available choices is not an issue. Types of second-line therapies include thrombopoietin receptor-agonists, rituximab, and immunosuppressive agents (non-steroid). This would be the first study of its kind to compare these three types of second-line therapies to understand their advantages over each other. If our application is accepted, the study would take place over five years.

We want to hear from you! Patient feedback is paramount for PCOR research, and if it’s important to you, it’s important to us! Please let us know if you think our study would be meaningful for ITP patients by sending your comments to PDSA’s Research Program Manager, Jennifer DiRaimo at jdiraimo@pdsa.org


What Causes ITP?

Title graphic of What Causes ITP video

The specific cause behind why ITP develops is usually unknown and can differ from person to person. Watch this video with Donald Arnold, MD to learn more.

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