Story 01
Summary
The NCI Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE®) Measurement System was developed to evaluate symptomatic toxicities by self-reporting in adults, adolescents, and children participating in cancer clinical trials.
Current State
Cancer therapies often have substantial side effects, and these adverse effects can limit treatment tolerability. In cancer clinical trials, the standard approach for identifying, grading, and reporting adverse events (AEs) is via clinician report.
Direct reporting of patients’ experiences of symptomatic AEs using patient-reported outcomes (PROs) can complement AE reporting by clinicians, thereby improving the detection and ultimately the management of both short- and long-term treatment-related toxicities. It is also essential to address language-related barriers to cancer clinical trials participation and to ensure that all trial participants have equitable access to self-reporting symptomatic AEs using PROs.
Strategy
Develop approaches that minimize the toxicities from cancer treatment and that detect and ameliorate the short- and long-term effects of treatment.
DCCPS Contribution
Safety and tolerability are fundamental to conclusions about the effectiveness of cancer therapies, including the comparative effectiveness of different treatments being studied. The clinician-reported Common Terminology Criteria for Adverse Events (CTCAE) is the standard AE reporting approach. The patient-reported PRO-CTCAE Measurement System, led by DCCPS, was designed to be used as a companion to the CTCAE to characterize the frequency, severity, interference, and presence/absence of symptomatic toxicities. Examples of symptomatic toxicities included in the CTCAE that can be meaningfully reported by patients include pain, fatigue, nausea, diarrhea, shortness of breath, blurred vision, problems with memory and concentration, and rash. The PRO-CTCAE Item Library includes 124 items representing 78 symptomatic toxicities drawn from the CTCAE. The Pediatric PRO-CTCAE module permits self-reporting by children and adolescents ages 7–17 (Ped-PRO-CTCAE) and includes a version for caregiver-proxy reporting when children are unable to self-report (Ped-PRO-CTCAE [Caregiver]). The PRO-CTCAE Measurement System is publicly available at the PRO-CTCAE website, along with technical information to guide the design and interpretation of trials that include PRO-CTCAE, as well as tools and training materials to support implementation in cancer research.
A multidisciplinary team of scientific leaders in outcomes measurement and AE reporting in cancer clinical trials, drawn from across NCI, supports the adoption of PRO-CTCAE in a variety of cancer research contexts, including observational studies and cancer-directed treatment trials. This team also collaborates with the US Food and Drug Administration, the European Medicines Agency, and other regulatory agencies around the world to scale up the implementation of PRO-CTCAE in regulatory decision-making.
PRO-CTCAE is currently being used by hundreds of investigators in academic settings and in industry-sponsored trials worldwide to characterize symptomatic adverse events in phase 1, 2, and 3 cancer treatment trials and to inform regulatory decision-making. The current evidence base supports descriptive reporting of symptomatic AEs using PRO-CTCAE to complement standard toxicity reporting by clinicians using the CTCAE.
With public-private funding from industry through over 20 cooperative research and development agreements, the PRO-CTCAE Measurement System has been validated in more than 60 languages. This robust set of translations supports the inclusion of culturally and linguistically diverse populations in cancer clinical trials and addresses language-related barriers to the equitable participation of underrepresented groups.
The PRO-CTCAE Measurement System is intended to enhance the precision and reproducibility of AE reporting in cancer clinical trials, complement and extend the information provided by clinician reporting using CTCAE, and represent the patient perspective of the experience of symptomatic AEs and treatment tolerability.
Story 02
Summary
The Exercise and Nutrition Interventions to Improve Cancer Treatment-Related Outcomes (ENICTO) initiative seeks to develop effective behavioral treatments to improve the completion of cancer therapy.
Current State
The concept of “precision oncology” captures the idea that each person’s cancer treatment and tolerance of cancer therapy is unique and that their treatment should reflect their individual characteristics, including response to behavioral interventions that may facilitate completion of cancer therapy.
Strategy
Develop approaches that minimize toxicities from cancer treatment and that detect and ameliorate the short- and long-term effects of treatment, including secondary malignancies in cancer survivors.
DCCPS Contribution
Cancer treatment-related side-effects are common and may impact future physical and emotional outcomes. They may also increase the risk of scaling back or discontinuing effective treatments such as chemotherapy. While leading organizations have formally recommended general physical activity for cancer survivors following treatment, and exercise interventions during cancer treatment are known to improve self-reported physical functioning, fatigue, anxiety, and depression, it is unclear whether these interventions improve important clinical outcomes, such as completion of prescribed chemotherapy. To address this knowledge gap, DCCPS established the Exercise and Nutrition to Improve Cancer Treatment-Related Outcomes (ENICTO) Consortium, comprising four clinical trials involving 10 research sites, a coordinating center, and NCI. The clinical trials plan to enroll over 900 cancer survivors before or during cancer treatment to determine whether exercise interventions, with or without a nutrition component, impact chemotherapy completion, adverse events, patient-reported outcomes, and more.
While each trial independently addresses its specific aims, the coordinating center and consortium structure provides an opportunity to pursue research questions collectively that would not be possible by individual research sites alone. Toward these ends, ENICTO has formed working groups to standardize data collection procedures, create a data repository (publicly available at ENICTO’s conclusion), develop cross-consortium research opportunities, and complete data analytics activities. The ENICTO Consortium has also launched the Exercise Oncology/Oncology Nutrition (EON) Network to engage researchers, trainees, and clinicians outside of ENICTO in the consortium’s work and facilitate networking and collaboration. The EON Network offers quarterly webinars and a newsletter with information on upcoming events, research summaries of interest to the exercise oncology and oncology nutrition fields, and relevant research funding opportunities.
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