“Behind every data point is a patient story - and it’s the responsibility of healthcare researchers and practitioners to listen, measure, and respond.”
As Vice President of Patient-Centered Outcomes Assessment at RTI Health Solutions, Lori McLeod, PhD has built a career around measuring the directly unmeasurable: constructs like fatigue, itching, depression, and, increasingly, treatment burden and experience.
We spoke with McLeod about the science behind patient-reported outcomes, how psychometrics (quantitative psychology) can help make sense of steroid-toxicity, and why the right question is as important as the right answer.
McLeod began her journey in rural North Carolina with a love of math and a double major in statistics and math education. Her early career in educational testing opened a path into psychometrics – with a focus on the science of measuring latent (i.e., unobservable) variables. That passion eventually led her to RTI Health Solutions, where she collaborates with other passionate researchers to help shape outcome strategies in therapeutic areas ranging from dermatology and gastroenterology to neurology and respiratory disease.
“We’ll all be patients someday or love someone who is. This work ensures that what matters to patients isn’t lost in the data.”
Understanding the invisible
The goal of questionnaire development and psychometrics in patient-focused drug development, McLeod explains, is to transform subjective experiences into reliable, validated measures that can stand up to clinical scrutiny. Whether the concept is depression or anxiety (two common side effects of steroid-toxicity), her team develops tools (patient-reported outcome measures: PROMS) that take the form of questionnaires. These PROMS can be used in clinical trials and studies to accurately capture these constructs from the patient perspective.
It’s not as simple as it seems.
"For example, lab values don’t tell you about fatigue. Clinicians might miss mood shifts. Patients are the only ones who can report unobservable outcomes, and we need to ask the right questions to hear them properly.”
McLeod has conducted psychometric evaluations of many existing PROMs and believes the field would benefit from more consistency across global regulatory bodies and payers. Often, studies need to use multiple instruments to capture and report on the same constructs for different decision makers across the drug development lifecycle.
In an attempt to standardize measurement and provide reliable, precise, and flexible instruments, regardless of the specific disease being studied, the NIH developed a series of PRO measures known as the PROMIS (Patient-Reported Outcomes Measurement Information System). These tools are used in clinical research and practice to capture the patient’s perspective on their symptoms, functioning, and well-being, enabling more patient-centered care. More recently, Critical Path Institute’s Rare Disease Clinical Outcome Assessment Consortium was formed to provide precompetitive support aimed at identifying tools and methods for collecting outcomes data for rare conditions.
“I think we've come a really long way with the development of the PROMIS measures. The system is designed to assess a range of constructs, like physical function and fatigue, across therapeutic areas in a construct-specific but disease-agnostic way.
The available item banks facilitate the creation of measures scored using the same scale and enable clinical researchers to more easily compare core sets of outcomes across different studies and in different populations.”
McLeod envisions a future where these measures are used more often to support advancements that inform group- and individual-level decisions.
“If the item banks provide precision across the range of interest, you could get more precision from fewer people for group-level decisions…smaller studies with better outcomes.”
Another promising application for this approach could be to track and measure the outcomes for shared decision-making, where patients and clinicians agree upon the outcomes that matter most for each individual patient.
Steroid-treated patients with chronic conditions that need long-term treatment are prime candidates for such approaches to be introduced, as the side effects of a treatment protocol need to be balanced against the benefits they provide in terms of disease and symptom control.
“You ask patients upfront: What are your goals? What outcomes matter to you? Then, you measure change relative to those goals.
This methodology, while still gaining acceptance, could revolutionize how treatment success is defined - especially in complex conditions with highly variable symptom profiles.”
Elevating the patient voice
In McLeod’s view, the field has made progress but still has far to go. Regulatory guidance emphasizes the importance of patient input, yet inconsistent expectations across global agencies continue to pose challenges. Still, as a member of a research team focused on bringing forward the patient voice, she remains optimistic–especially as use of patient-centered outcomes is becoming more important for decision makers beyond the regulatory hurdles, including health technology assessment and payers.
“We’re using data not just to justify treatment benefits but to understand what ‘better’ looks like for the people living with a condition. That’s a powerful shift.”
For McLeod, the future of outcomes research lies in truly centering the patient - right from the start. She sees growing potential in approaches like goal attainment scaling and adaptive assessments, where patients define what treatment success looks like for them, and researchers measure progress against those individual goals. It’s a shift from one-size-fits-all to what-matters-to-me.
She also points to item banks and adaptive testing as tools that could unlock greater precision with smaller, more tailored studies - especially useful in rare or heterogeneous conditions.
McLeod’s work reminds us that behind every health-related data point is a patient story - and the real science is making sure those patient stories are heard.
Lori McLeod, PhD is Vice President, Patient-Centered Outcomes Assessment at RTI Health Solutions. McLeod is a psychometrician with more than 25 years of measurement experience, including expertise in instrument development and evaluation, as well as experience developing appropriate health outcome strategies and defining meaningful change.
McLeod has published numerous related manuscripts in Quality of Life Research, Value in Health, Pharmacoeconomics, Mayo Clinic Proceedings, and Psychometrika. She has experience in a wide variety of therapeutic areas, including chronic pain, dermatology, oncology, neurology, psychiatry, respiratory, sleep disorders, urology, gastroenterology, and sexual dysfunction. McLeod also serves as adjunct faculty in the Department of Health Policy and Management at the University of North Carolina at Chapel Hill, on the editorial advisory board for Value in Health, and on the Board of Directors for the International Society for Quality of Life Research (ISOQOL).