The PhenX Toolkit: Standard Measurement Resources for Cancer Outcomes and Survivorship Research

Larissa Nekhlyudov, MD, MPH; Keith Bellizzi, PhD, MPH; Andrew Galligan, MD, MS; Bellinda King-Kallimanis, PhD; Deborah K. Mayer, PhD, RN, AOCN, FAAN; Christine Miaskowski, RN, PhD; Talya Salz, PhD; Catherine McCarty, PhD, MPH; Lisa Cox, MS; Christine Hill, MPA; Tabitha P. Hendershot, BA; Deborah R. Maiese, MPA; Carol M. Hamilton, PhD


J Natl Cancer Inst. 2023;115(4):473-476. 

This brief report introduces the Cancer Outcomes and Survivorship domain in the PhenX Toolkit (consensus measures for Phenotypes and eXposures), which includes 15 validated measurement protocols for cancer survivorship research that were recommended for inclusion in this publicly available resource. Developed with input from the scientific community, the domain provides researchers with well-established measurement protocols for evaluating physical and psychological effects, financial toxicity, and patient experiences with cancer care. The PhenX Toolkit, funded by the National Human Genome Research Institute since 2007, is an online resource that provides high-quality standard measurement protocols for a wide range of research areas (eg, smoking cessation, harm reduction and biomarkers, and social determinants of health). Use of the PhenX Cancer Survivorship Outcomes and Survivorship domain can simplify the selection of measurement protocols, data sharing, and comparisons across studies investigating the cancer survivorship experience.

Widespread use of standard measurement protocols, also known as instruments or measures, can promote the impact of individual studies by allowing for collection and comparison of data to assess population-level disease burden, patient-oriented outcomes, and effectiveness of interventions. The PhenX Toolkit (consensus measures for Phenotypes and eXposures;, is a publicly available catalog of high-quality, well-established measurement protocols (defined as a standard, reproducible approach for collecting data, such as with a questionnaire, physical measurement, or bioassay) to assess phenotypes and exposures in studies with human participants. Funded by the National Human Genome Research Institute of the National Institutes of Health since 2007, the Toolkit contains measurement protocols in 30 research domains covering topics such as smoking cessation, harm reduction and biomarkers, and social determinants of health. The goal of this report is to introduce the scientific community to the newly developed Cancer Outcomes and Survivorship domain, which contains 15 recommended standard protocols applicable for use among the adult cancer survivor population.

The Cancer Outcomes and Survivorship domain was developed using a well-established consensus process to identify and recommend protocols for inclusion in the PhenX Toolkit.[1] First, the PhenX steering committee developed the initial scope for the domain, which guided the selection of experts to form a working group (WG) with expertise in psychometrics, symptom management, cancer health services research, cancer survivorship care delivery, and nursing (Supplementary Table 1, available online). Second, the WG aligned the initial scope with a recently published quality of cancer survivorship framework.[2] The framework was developed in collaboration with the National Cancer Institute and input from a variety of stakeholders, including clinicians, researchers, patients, and payers, using a variety of methods to identify domains and indications for cancer survivorship care quality. Guided by the framework, the WG modified the initial scope for the Cancer Outcomes and Survivorship domain to include physical effects, psychological effects, financial and/or employment, quality of life and/or function, chronic medical conditions, communication and/or decision making, and patient and/or caregiver experience (Table 1).

Third, the WG used established PhenX criteria for selecting protocols,[1] with priority given to those for which standard protocols already existed, are in the public domain, and are low burden to study participants and investigators. Each WG member was assigned to search for protocols within the areas most aligned with their expertise. In addition to searching the broader literature, WG members considered protocols already in the PhenX Toolkit that were pertinent to cancer, recommended by previously assembled expert WGs (Supplementary Table 2, available online). During an in-person meeting in August 2019, each member presented findings on their assigned scope element and recommended specific protocols for the WG to consider.

The WG achieved consensus on 16 protocols that would complement those already in the Toolkit and, specifically, to advance cancer outcomes and survivorship research. To get feedback from the scientific community, the 16 protocols were distributed to more than 2900 registered Toolkit users who were asked whether these protocols should be included in the Cancer Outcomes and Survivorship domain. In addition to providing a yes-or-no response, respondents had the option to provide a free-text response. The feedback was generally positive; the only concern was the lack of protocols focused on children. In response, the WG decided to focus this domain exclusively on adults and recommended that PhenX consider developing a childhood and adolescent cancer domain in the future. The PhenX steering committee approved the WG's final recommended protocols.

The Cancer Outcomes and Survivorship domain contains 15 protocols (Table 1). The WG prioritized well-established protocols, including the Medical Expenditure Panel Survey, the Patient-Reported Outcomes Measurement Information System, and the Consumer Assessment of Healthcare Providers and Systems Cancer Care Survey. The Toolkit provides a data collection worksheet, a data dictionary compatible with the database of Genotypes and Phenotypes and Research Electronic Data Capture and links to Cancer Data Standards Registry and Depository common data elements. Further, for each protocol, psychometric properties (eg, validity and reproducibility) as well as information such as life stage of the study population and mode of administration are included, when available in the source general references. This information permits investigators to determine whether a particular protocol is suitable for their study design. As part of the PhenX process, principal investigators are contacted for permission to include protocols in English and Spanish languages. A list of languages in which a protocol is available is noted in the Toolkit.

The WG identified several areas that would benefit from additional development. Collecting comprehensive cancer type and treatment data, including recurrent disease, is challenging, and no standard methods exist.[3] Further, the WG found limitations in survey instruments assessing surveillance for cancer recurrences and new cancers. Although emerging data collection methods, such as natural language processing and machine learning, are increasingly used to capture cancer history, clinical treatments, and other information from clinical notes, work is needed to understand how to leverage these techniques to benefit large cancer survivorship cohorts in clinical, translational, and other research.

Incorporating standard data collection protocols into new and existing research may facilitate cross-study analyses and evidence synthesis, enhance the scientific impact of individual studies, and promote collaboration among researchers. The Cancer Outcomes and Survivorship domain offers validated protocols across different areas of cancer survivorship research, as suggested by the Cancer Survivorship Quality Framework and supported by others who have conducted similar assessments, accounting for different stakeholders.[4–8] Although PhenX does not dictate which protocols should be used, this newly developed domain offers well-established protocols to assess important outcomes in cancer survivorship care. Investigators are encouraged to review the source and general references to obtain additional information, including psychometric properties, before selecting protocols that meet their research needs. The PhenX Toolkit continues to evolve as a resource that is updated over time.

With 18 million cancer survivors in the United States, and millions more worldwide, a considerable segment of the population stands to benefit from expanded research efforts. Using the PhenX Cancer Outcomes and Survivorship domain can make it easier for the research community to identify protocols for a research study and promote standard measurement of the cancer survivorship experience and health outcomes.