ARC Developing a Disease-Specific PRO Tool for Patients with ATTR Amyloidosis

ARC is developing a patient-reported outcome (PRO) measurement tool in ATTR amyloidosis to be used to understand and measure the impact of the disease on patients from the patient’s perspective.

What is a Patient-Reported Outcome (PRO) Measure?

A PRO is a standardized set of survey questions administered to patients. PROs focus on aspects of health that cannot be observed by a healthcare provider. Best practice for development of a PRO is to include patient input to design surveys that are easy to understand, relevant, and meaningful to patients. The goal is to measure what matters most to patients, such as how the disease and/or treatment impact their daily life, physical functioning, and mental health status.

PROs can help improve patient lives in a variety of ways. First, PROs help developers of new drugs identify important impacts of a disease from a patient’s perspective. Second, PROs can be used in clinical trials to measure how a patient responds to treatment. These data may support whether a treatment can be approved by regulatory agencies, such as the Food and Drug Administration (FDA), and released to the market by drug companies. Third, PRO data are a consideration in pricing, insurance coverage and reimbursement decisions for specific treatments. Fourth, PROs are used in clinical practices to help doctors and patients make critical healthcare decisions. For example, PRO data may highlight important factors in the treatment decision process, such as the severity of side effects or a treatment’s ability to alleviate specific symptoms.

Some PROs ask a wide range of questions about health and well-being and can be used to collect information regardless of the disease or condition they might have. Others are specific to a certain disease. Disease-specific PROs are useful because they are particularly good at helping detect changes in a patient’s health or quality of life. They also ensure that the PRO adequately assesses the most important aspects of the disease experience.

Why is an ATTR-specific PRO Important?

At this time, there are no PROs that were developed for and with input from patients with ATTR amyloidosis. ATTR amyloidosis is a complex disease and patients’ experiences can widely differ.  A PRO developed specifically for ATTR amyloidosis patients may improve how disease and treatment impacts are measured. If an ATTR-specific PRO is widely used, it will allow for comparison of results across clinical studies and clinic use and the collection of data that will help understand changes in patient health over time.

Are you a patient? You can help advance PRO research by participating in a phone interview. Sign up here.