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5 Updates on Patient-Reported Outcomes Assessments


The field of patient-reported outcomes (PROs) has witnessed a slow and steady progress over the past few years. Here are a few recent developments with utilizing and measuring PROs.

In the world of value-based care, patient participation in identifying treatment goals is vital. A significant component of this process are patient-reported outcomes (PROs). The field has witnessed a slow and steady progress over the past few years. Here are a few recent developments with utilizing and measuring PROs:

1. Quality of life results from the ABRAZO trial

Phase 2 results from the ABRAZO trial, conducted in patients with advanced stage breast cancer treated with talazoparib found that their global health status (GHS) and quality of life (QOL) were maintained from baseline.1

The 2 cohorts were divided based on whether patients had received prior platinum-based therapy (C1) or 3 or more platinum-free cytotoxic-based regimens (C2), and PROs were evaluated at day 1 (baseline), every 6 weeks for an initial 24 weeks, and every 12 weeks thereafter until disease progression. The median time to deterioration for C1 and C2 was 2.8 and 5.5 months, respectively. No significant change was noted in the overall change from baseline in GHS/QOL for either cohort. Additionally, patients reported improved breast and arm symptoms. However, patients receiving C1 had overall deterioration in their role function while the C2 group had deterioration in dyspnea symptoms.

2. Measuring PROs in HSCT

A cross-sectional study led by researchers at the University of Colorado’s Blood Cancer and Bone Marrow Transplant Program assessed patient and caregiver stress prior to an allogeneic hematopoietic stem cell transplant (HSCT) because research suggests it has an impact on posttransplant patient outcomes.2

The parameters that were measured in 148 patient—caregiver dyads included patient and caregiver anxiety, depressive symptoms, perceived stress, sleep quality, caregiver burden, and patient QOL. While patient distress increased caregiver distress, a significant association was documented between caregiver distress and patient QOL. The authors conclude that their findings shine light on the importance of providing support to patients and their caregivers during the pretransplant period.

3. MEDCAC view of PROs for CAR T-cell treatment

In August this year, Medicare Evidence Development and Coverage Advisory Committee (MEDCAC), an independent panel of experts who provide advice to CMS on specific clinical topics, held a meeting that saw participation by developers of chimeric antigen receptor (CAR) T-cell treatments, health researchers, and policy makers. The meeting was aimed to assess whether current scientific evidence is sufficient to apply PROs to health outcomes research, in addition to exploring challenges around the validity, reliability, and generalizability of existing PROs.

The meeting saw opposition from Novartis—which manufactures tisagenlecleucel (Kymriah), the first CAR T-cell treatment to be FDA approved—around CMS’ national coverage determination analysis (NCA) for CAR T therapy. The argument was that an NCA would be a barrier to patient access. Additionally, there were concerns that PROs would be burdensome for providers and patients. Researchers who study PROs stressed the value they bring to drug development but agreed that it remains a work in progress.

4. Efficient capture of PROs in an electronic health record

Attempts are being made to passively monitor and document patient health status and deliver customizable reports to clinicians.3

The platform—Capturing and Analyzing Sensor and Self-Report Data for Clinicians and Researchers (COMPASS)—utilized oncologist interviews to identify the parameters that would be vital to support clinical care delivery. It was then evaluated in 3 patients with cancer. The results showed that physicians were interested in monitoring vital statistics, symptoms, and functional status, including physical activity level, weight, fatigue, sleep quality, and anxiety. Patients reported being enthusiastic about their physicians monitoring their health status, especially their symptoms, and that they would use such a device.

5. Challenges with using PROs in drug development

The lack of consistent standards and guidance documents could be the primary reasons for measurement and utilization of PROs in drug development. According to Theresa Mullin, PhD, associate director for strategic initiatives for the Center for Drug Evaluation and Research at the FDA, “stakeholders need clear guidance on methods to use and access to information on what measures have already been used and accepted.”

In her email to The American Journal of Managed Care®, Mullin wrote that the FDA is facilitating a dialogue among stakeholders to identify appropriate measures and standards by accounting for patient perspectives overall, beyond just the PROs.


  1. Hurvitz SA, Quek RGW, Turner NC, et al. Quality of life with talazoparib after platinum or multiple cytotoxic non-platinum regimens in patients with advanced breast cancer and germline BRCA1/2 mutations: patient-reported outcomes from the ABRAZO phase 2 trial. Eur J Cancer. 2018;104:160-168. doi: 10.1016/j.ejca.2018.09.003.
  2. Sannes TS, Simoneau TL, Mikulich-Gilbertson SK, et al. Distress and quality of life in patient and caregiver dyads facing stem cell transplant: identifying overlap and unique contributions [published online October 24, 2018]. Support Care Cancer. 2018. doi: 10.1007/s00520-018-4496-3.
  3. Lucas AR, Bass MB, Rothrock NE, et al. Development of an eHealth system to capture and analyze patient sensor and self-report data: mixed-methods assessment of potential applications to improve cancer care delivery. JMIR Med Inform. 2018;6(4):e46. doi: 10.2196/medinform.9525.
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