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Office of Patient-Centered Outcomes Research
Using patient-focused approaches to enhance our understanding of the patient experience
Information for Patients
Learn about why patient-centered outcomes matter.
Spotlight
Dr. Mendoza presents at International Society for Quality of Life Research annual meeting
Office of Patient-Centered Outcomes Research
The goal of the Office of Patient-Centered Outcomes Research (OPCORe) is to integrate the voice of the patient, and in particular, the use of patient-centered outcomes into early-phase clinical trials.
What are clinical outcomes assessments?
Patient-centered outcomes assessment starts with an understanding of the impact of a disease and its treatment on patients, and what patients value from a treatment perspective. It measures things that are important to patients, such as how a treatment will affect symptoms and their day-to-day functioning.
Such patient-focused approaches use clinical outcomes assessments (COAs), in which information is gathered on the impact of treatment on the person and can markedly enhance the understanding of the patient experience in cancer clinical trials.
COAs can broadly be classified into four distinct groups based on how data is collected:
- PROs – patient-reported outcomes
- ClinROs - information gleaned from clinical observation
- ObsROS - other observer reported outcomes (ObsROs),
- PerfOs - and data from performance on a task or test (PerfOs)
In cancer-related clinical trials, regulatory agencies and advocacy groups increasingly encourage using PROs to describe the clinical benefit of a therapeutic regimen.
This includes using information on disease-associated symptoms or functions to help determine treatment efficacy and understand treatment-associated side effects. These measures, which collect information directly from the patient, without interpretation by somebody else, improve researchers’ understanding of the patient perspective on the treatment’s impact.
Why are patient-centered outcomes important?
Regulators, clinicians and patients need meaningful information about a treatment’s benefits and risks in order to make important treatment decisions.
Additionally, these measures also provide valuable insights about the impact of the disease and its treatment that help improve patient care even when the treatment does not turn out to have sufficient activity against the cancer.
Why are patient-centered outcomes important in early-phase trials?
There has been a shift in the clinical trial landscape towards early-phase studies, where data from well-designed and pivotal phase I/II trials increasingly leads to accelerated drug approval.
Early-phase clinical trials primarily investigate toxicity and tolerability. Standard toxicity assessments provide only limited or inadequate information. Patient-focused approaches using COAs to gather information about the impact of treatment on the person, not only the tumor, would markedly enhance the understanding of the patient experience in these trials.
Standardizing and incorporating PROs into earlier phase trials would allow them to be optimized, improve study design and allow more rigorous and systematic assessment of PROs in later-phase trials.
These more systematic PRO designs may then increase the chance of a PRO being incorporated into a product’s label. That could ultimately empower patients, caregivers, and healthcare providers to make more informed treatment decisions leading to better outcomes and better quality of life for patients.
For Researchers
We look forward to collaborating with researchers at different institutions.
For Patients and Advocates
Learn about how we are adding the voice of the patient into early-phase cancer clinical trials.
For Trainees and Trainers
Our postbac fellows receive strong mentoring as they work with our multidisciplinary research teams. Learn more about opportunities to incorporate PROs into clinical research training.
News & Events
International Society for Quality of Life Research held their annual meeting on October 18-21, 2023 in Calgary, Canada
10/24/2023 - Dr. Mendoza presented the favorable patient-reported symptoms associated with the use of carboplatin and bevacizumab in the treatment of recurrent low-grade and anaplastic supratentorial, infratentorial and spinal cord ependymoma at the annual meeting of the International Society for Quality of Life Research held in Calgary, Canada on October 18-21, 2023. Patients who received the treatment in this multi-center Phase II trial reported improvement in cognitive and neurological symptoms.
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Dr. Tito Mendoza attended the Quality of Life Roundtable hosted by the National Brain Tumor Society
09/26/2023 – As a stakeholder, Dr. Tito Mendoza attended the Quality of Life Roundtable hosted by the National Brain Tumor Society. More than 60 pediatric & young adult patients, caregivers, and stakeholders across the brain tumor community attended the roundtable to examine the unique needs, priorities, and challenges faced by patients with brain tumors and their loved ones.
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OPCORe Hosted a Multi-Stakeholder Working Meeting to Discuss Core Symptomatic Toxicities in Clinical Trials
09/14/2023 – Since the publication by Reeve et al in 2014 there has been an explosion of new classes of anticancer drugs with a wide range of unique toxicities. An objective approach to item selection from a library is needed and could start with an updated, brief “core symptomatic AE” set. Criteria for the selection of core symptom items may depend on the actionability, prevalence, and severity of the chosen symptoms. Patient preference is another important consideration that Lee Jones, a patient advocate, shared with the group. The group is currently working on several manuscripts that will inform selection of core symptomatic toxicities in early and late-phase interventional cancer trials.
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Interprofessional Communications Curriculum, July 17-18, 2023
07/25/2023 - OPCORe staff participated in an Interprofessional Communication Curriculum on July 17 & 18. The course focuses on communication training in oncology and is based on the eight domains of the National Consensus Project Guidelines for Quality Palliative Care.
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07/13/2023 - OPCORe is looking forward to having Dr. Betty Ferrell from the City of Hope & her Interprofessional Communication Curriculum team to NIH for a 2-day workshop this month. The workshop will provide communication training & education for clinical care providers working with cancer patients.
OPCORe Postbacs Presented at the NIH Postbac Poster Day
04/21/2023 - OPCORe postbacs, Ciara Locke and Sefanit Berhanu, presented their posters at the 2023 NIH Postbac Poster Day.
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Congratulations to Dr. Amanda King on her Virtual Reality Protocol Manuscript and Interim Analysis Publications
03/23/2023 - Cancer patients experience distress and anxiety when undergoing imaging studies to monitor disease status, yet these symptoms are not always appropriately identified or well-managed. Use of virtual reality (VR) has been shown to improve psychological symptoms in other solid tumor patients, but this innovative interventional strategy has not been explored in patients with central nervous system (CNS) tumors. A manuscript outlining the study protocol of a phase 2 feasibility trial was published in BMC Cancer and an interim analysis of this trial was published in the Journal of Neuro-Oncology and presented at the American Psychosocial Oncology Society in Portland, OR (see picture below). The interim analysis demonstrated that VR use in patients with brain tumor patients was both feasible and acceptable in targeting psychological symptoms prior to neuroimaging evaluations. Trial enrollment will continue to assess for intervention efficacy and the authors plan to expand enrollment to other cancer populations in the future.
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Their poster describing the VR interim analysis was selected as the award-winning poster at the American Psychosocial Oncology Society National Conference (APOS) meeting in Portland, OR.
American Psychosocial Oncology Society Conference Presentation
03/20/2023 - The COVID-19 pandemic and associated mitigation procedures have significantly altered daily life in ways that may disproportionately affect patients with central nervous system (CNS) tumors. The purpose of this study (which was presented at the American Psychosocial Oncology Society in Portland, OR) was to explore differences in symptom burden and interference, mood disturbance, and quality of life in CNS tumor patients during the 1st year of the pandemic, compared to pre-pandemic assessments. The authors found that symptom burden for these patient remained high and was relatively unchanged during the pandemic, but there was an increase in depressive symptoms reported on PROMIS and EQ-5D-3L instruments. Factors that increased risk for clinically significant depression included co-occurring distress, use of psychotropic medications, low tumor grade, and male gender. Dr. Amanda King from the Office of Patient-Centered Outcomes Research & coauthors propose that use of innovative tools and interventions that can be utilized remotely may better identify and target mood disturbance for patients with CNS tumors.
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New Manuscript Published
03/09/2023 - In this phase I trial of combining ado-trastuzumab emtansine (T-DMI) with metronomic temolozomide (TMZ) in women with metastatic HER2+ breast cancer to the brain after previous occurrence and local treatment, patients who responded to treatment showed lower symptom burden, less interference with daily functions and better cognitive function. These outcomes were measured using the brain tumor module of the MD Anderson Symptom Inventory and the NeuroQOL Cognition Function with completion rates of 99% by Cycle 15 and 90% by Cycle 41. Read more
Welcome Our New Trainees!
02/06/2023 - OPCORe welcomes the newest members to our team. Ciara Locke and Sefanit Berhanu, newest members of OPCORe, participated in the day of action activity and made hygiene kits for the Small Things Matter charity organization.
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The link between psychological distress and survival in solid tumor patients: A systematic review
01/11/23 - Past research has shown that solid tumor patients experience high levels of psychological distress at the time of diagnosis, yet little is known about how this symptom may adversely impact clinical outcomes. This systematic review explored the literature linking distress with survival in solid tumor patients and found a weak to moderate relationship, suggesting that this symptom may be a negative prognostic factor. The authors hope this work will provide better understanding of the clinical implications of untreated distress and that it may enable development of targeted interventions for cancer patients. Learn more
Relationship between RANO-PRO Working Group standardized priority constructs and disease progression among malignant glioma patients: A retrospective cohort study
11/14/22 - Recognizing the importance of clinical outcomes assessments (COAs), the Response Assessment in Neuro-Oncology-Patient Reported Outcome (RANO-PRO) Working Group recommended inclusion of core symptoms and functions in clinical care or research for malignant glioma patients. This study evaluated the association of the recommended symptoms (pain, perceived cognition, seizures, aphasia, symptomatic adverse events) and functions (weakness, walking, work, usual activities) with disease progression in these patients. Learn more
Dr. Mendoza speaks at the International Society for Quality of Life Research
10/20/22 - Dr. Mendoza discussed the use of patient-reported outcomes in predicting severe adverse events in cancer patients receiving immunotherapy during the 29th annual conference of the International Society for Quality of Life Research held in Prague, Czech Republic Oct 19-22.
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Patient-reported symptom burden in patients with rare cancers receiving pembrolizumab in a phase II Clinical Trial
10/15/22 - In a prior study, it has been shown that pembrolizumab, an immune checkpoint inhibitor has favorable toxicity profile and antitumor activity in advanced rare cancer patients (e.g., squamous cell carcinoma of the skin, carcinoma of unknown primary, paraganglioma). In this companion study to that early phase trial evaluating pembrolizumab, we focused on how patients feel and function while undergoing treatment. We hope that this information will assist clinicians with their symptom management approach, aid researchers in designing symptom intervention trials and help patients with what to expect during their treatment. Learn more
PRO in Patients With Rare Cancers: Pembrolizumab
10/02/2022 - Congratulations to Dr. Mendoza for his Oncology Tube feature highlighting his work in rare cancers and also OPCORe! Learn more
Team
OPCORe Team
Internal Advisors
External Advisory Board
Contact
Have questions or need additional information?
Please send an email to CCR.OPCORe@mail.nih.gov
Building 82, Room B03A
9030 Old Georgetown Road
Bethesda, MD 20892-1750
E-fax: 301-480-5529