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Patient-Centered Measurement: How Close Are We?


When it comes to medical product discovery, development, and delivery, patients need to be front and center – but how close (or far away) are we from truly having a system that routinely measures what matters to patients?
A panel of industry, regulatory, and patient leaders will discuss what’s next for patient-centered measurement. Panelists will define patient-centered measurement; identify novel strategies for capturing patient-centered outcomes; assess the progress in product development, regulatory approval, and delivery; and address how to advance the science of patient input in a way that fulfills the unmet needs of patients across diseases.

Speakers include:

  • Cynthia Grossman, Director, Science of Patient Input, FasterCures, a center of the Milken Institute 
  • Paul Kluetz, Acting Associate Director of Patient Outcomes, Oncology Center of Excellence, U.S. Food and Drug Administration
  • Catherine Kopil, Director, Research Partnerships, The Michael J. Fox Foundation for Parkinson's Research
  • Ellen Schultz, Senior Researcher, American Institutes for Research
  • Tanisha Carino, Executive Director, FasterCures, a center of the Milken Institute (moderator)


“Despite being around for decades, or maybe because of it, myths circle around the definition and use of patient-reported outcomes (PROs) like the cloud of dust around the Peanuts character Pig-Pen.”

This observation, which originated from a 2017 workshop led by FasterCures, a center of the Milken Institute, set the stage for FasterCures’ March 22, 2018, webinar, “Patience-Centered Measurement: How Close Are We?”

The benefits of PROs include addressing unmet needs, facilitating better decision-making across every aspect of the R&D process, and aligning care priorities within diverse patient populations. However, the path to properly utilizing patient-centered measurements and tracking PROs is just starting to be paved.

FasterCures executive director Tanisha Carino moderated the discussion and tapped into experts across regulatory, research, and patient groups to track progress in integrating patients as partners and putting patient input at the heart of biomedical R&D.

Setting the foundation for measurement

Throughout the last five years, FasterCures has worked to strengthen patient-centered measurement and patient input as critical facets of medical decisions. To begin the process of adopting patient-centricity into the medical R&D system, stakeholders must first define patient-centered measurement.

Ellen Schultz, a senior researcher at American Institutes for Research (AIR), and the AIR team developed a standard definition of this process when she realized only two years ago that one had not yet been created. Schultz stated that patient-centered measurement is “measurement that is driven by the patients’ expressed preferences, needs, and values that inform progress toward better health, better care, and lower cost.”

In order to achieve patient-centered measurement, R&D professionals should adopt an objective and holistic view of patient and family needs across the full spectrum of measurement. Schultz also shared AIR’s five principles of patient-centered measurement and examples of measurement that would fulfill each. Shultz stated that “measurement is patient-centered when it is co-created with patients as equal partners in measure development.” A key staple of co-created measurement is the shared decision-making authority between patients and stakeholders regarding how data are collected, reported, and used.

Implementing patient-centered measurement “in the real world”

Paul Kluetz, acting associate director of patient outcomes in the Oncology Center of Excellence (OCE) at the U.S. Food and Drug Administration, echoed the importance of continuity in measurement. Kluetz expressed three key areas that are already being implemented through the OCE’s Patient-Focused Drug Development initiative to further patient-centricity: “engaging with patients and advocacy groups, fostering research into measurement of the patient experience, and generating a science-based recommendation for regulatory policy.”

This initiative strives to assess the full patient experience, derive what improvements or declines in quality of life are clinically meaningful, and determine how stakeholders can use this information to their benefit. Kluetz highlighted the need for collaboration among patients, caregivers, and industry to advance patient-centered care.

FDA’s Office of Hematology and Oncology Products is currently incorporating PRO measurement through a collaboration with the Division of Research of the Permanente Medical Group. The study will investigate how patient perspectives of physical functioning throughout treatment can complement existing tumor and survival measures.

The value of looking through the “patient lens”

As a foundation established by a patient for patients and the world’s largest nonprofit funder of Parkinson’s disease research, the Michael J. Fox Foundation (MJFF) has a unique and critical patient lens guiding its work.

Catherine Kopil, director of research partnerships at MJFF, demonstrated the value of direct-from-patient data through Fox Insight, an online clinical study gathering a robust sample of people with and without Parkinson’s disease worldwide. Every 90 days, more than 10,000 participants complete virtual study visits, enabling scientific collaboration toward patient-relevant outcomes. “Patients are the real experts. They live with their conditions 24/7, and they have insights to share,” Kopil said.

She explained that with a far-reaching disease such as Parkinson's, a major challenge is developing a representative cohort to make informed decisions. Kopil and MJFF see a future with person-centered therapy, and to realize this future, stakeholders must “detach from the view that clinicians know best” and “leverage a patient-centric model where stakeholder decisions are grounded in patient input.”

The next steps to push patients’ perspectives forward

How do we realize the immense potential that patient-centered measurement and patient-reported outcomes (defined as outcomes reported by patients without filtering or interpretation by others) can offer?

Cynthia Grossman, director of science of patient input at FasterCures, debunked several myths to answer this question.

Specifically, Grossman broke down the idea that PROs are inherently patient-centered, sharing that just because a patient can report on something directly, that doesn’t mean the measure is capturing something of importance to patients.

Grossman stated that physicians and nurses fail to capture significant patient concerns when a “measurement strategy is not aligned with what matters to that patient.” Similarly to Kopil, Grossman expressed the need to individualize treatment on a patient-by-patient basis, as opposed to “plug and play measures without the necessary engagement of the intended patient population.”

Grossman laid out final steps to advance PROs: drive adoption of patient-centered PROs, define the return on investment, and celebrate successes.

Where do we go now?

To close the session, Carino asked each speaker to describe the most significant obstacles to creating a system where PROs are captured by research, reflected in regulatory decision-making, used in payer decisions for incentivizing care, and adopted as a central point in care decision-making between a doctor and patient.  

Kopil shared that figuring out how endpoints translate into practice is a significant gap. Developing an understanding of why traditional outcome measures are not practical in real-world settings, and modifying them or creating new measures that can span the continuum, will move the needle for patient-centered measurement.

Echoing these comments, Kluetz added that a learning health-care system with consistent measurements would be immensely beneficial in post-market monitoring, and would illustrate the value of different therapies. Closing the loop between the information available at the time of approval and additional evidence generated in a post-market setting would be an improvement for all parties involved.

Schultz highlighted the barrier of measurement burden, especially in clinical settings where physician burnout is already a difficult issue. She suggested a method of working backward: if a measure is feasible at the point-of-care, it will very likely be possible in a clinical trial setting.

Grossman’s final comments revolved around the importance of defining terminology. Throughout the session, the speakers often focused their remarks on clarifying the terms and definitions they were applying. However, the same cannot be said for the world of patient-centered measurement. Defining the universe of measures and terminology would go a long way to ensure stakeholders across medical research are starting out on the same page, rather than spending precious time and energy getting there.

Stakeholders in the medical research ecosystem are only beginning to uncover the benefits of PROs and patient-centered measurement. FasterCures is eager to continue pushing forward patient-centered measurement and highlighting innovative approaches that will change the conversation around PROs in the years to come. With the insights shared during the webinar and the current innovative initiatives underway, the stakeholder community is one step closer to improving the care of numerous patient populations.

Related resources:

And register for our next webinar, FDA’s Patient-Focused Drug Development Initiative: What Have We Learned?, on June 7.