Patient-Reported Outcomes Including Health-Related Quality of Life in Cancer Clinical Trials

Dear Colleagues,

The assessment of Health-Related Quality of Life (HLQoL) and Patient-Reported Outcomes (PROs) is increasingly recognized as an essential component of a high-quality clinical trial. Regulatory bodies, including the European Medicines Agency (EMA) and the Food and Drug Administration (FDA), are increasingly recommending PRO assessment in clinical research. These recommendations will have an increasing impact on clinical researchers, all health care industries, and patients and their caregivers.

Publishing PRO outcomes can be challenging for many reasons, including space limitations in clinical journals, in addition to challenges in the collection of the full PRO data set.  Given increased HRQL and PRO research, and the wider application of these outcomes, there is a need for more publication outlets for scientifically sound PRO research. We offer an opportunity to submit and discuss the successes and challenges in collecting, analyzing and interpreting PRO outcomes in cancer clinical trials.

HLQoL and PRO are sometimes used interchangeably and so we will start with definitions.

Health-related quality of life is the value assigned to the duration of life as modified by the impairments, functional states, perceptions, and social opportunities that are influenced by disease, injury, treatment, or policy. HRQoL cannot be measured with a single question but it operates in the realm of measuring treatment impact and taking into account various factors.

The United States Food and Drug Administration introduced the term patient-reported outcomes (PROs). A key component of the FDA definition is that the measure conveys information reported by the patient that is not filtered by an observer or clinician. In the United Kingdom the term, patient-reported outcome measures (PROMs) is widely used. Some authors use the terms HRQL, health status, PROs, and PROMs interchangeably. HRQoL is one component of a PRO. 

A working group subsequently known as the PRO Harmonization Group was a working group established by the FDA. This task force identified several types of measures that fall under the PRO umbrella, including HRQOL, functional status, symptom status, overall well-being, satisfaction with care, and treatment adherence.

Quality of Life research has a long and successful history of publishing research on methods and applications of health-related quality of life (HRQL) and patient-reported outcomes (PROs).

Ongoing challenges in producing high-quality PROs have been reported and these include ethical issues, for example, a lack of reporting of PROs even if they are collected, a lack of clarity around the PRO-specific research question, details on the methodology of PRO collection including the use of electronics, and discussion regarding the management of the PRO in real time, ensuring that the participants understand why the PRO questions are being asked and how the information will be used. The standardization of PRO in the methodology of clinical trials will address challenges of incomplete data collection and the lack of HCP responses to data being collected.

We are pleased to invite you to contribute to this increasingly important field of clinical research.

The aims of this Special Issue encompass high-quality research in Patient-Reported Outcomes (PROs) including Health-Related Quality of Life (HRQoL) and their use in cancer clinical trials. This collection will allow researchers to focus on the appropriate selection, reporting, and interpretation of PROs in cancer clinical trials, as well as the use of technological advances in the integration of PROs in cancer clinical trials. 

A number of guidelines have been developed to improve the quality of PRO reporting in clinical trials including the CONSORT PRO guidelines, the SPIRIT PRO Protocol Guidance Checklist, the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), and the PRO Ethics Guidelines, and these will be used to guide the review of submissions.

The scope of this Special Issue includes original clinical trials with clear adherence to PRO guidelines, critical reviews of the use of PROs in clinical trials, and methodological papers related to the following thematic areas:

1. Measurement of Patient-Reported Outcomes (PROs).
• The appropriate development, validation, and adaptation of PRO measures in cancer clinical trials.

• • • Attention to how current PROs, especially those tools that concentrate on PRO-CTCAE, are adapted to new and novel treatments such as immunotherapies which may have prolonged toxicities and an effect on QoL.

• The application of PROs, including HRQoL tools, in different cancer patient populations.
2. Enhancing patient engagement through PRO collection.
3. Ethical considerations in using patient-reported data in clinical trials.
4. Technological Innovations in PROs and HRQoL.

• • The use of digital tools for collecting PRO data.
  • Big data and machine learning applications for analyzing PRO and HRQoL data.

5. Longitudinal and cohort studies that explore the relationship between PROs and cancer outcomes.

We look forward to receiving your contributions.

Dr. Caroline Hamm
Guest Editor

Manuscript Submission Information

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• patient-reported outcomes
• guidelines
• health-related quality of life
• clinical trials
• ethical considerations
• prognosis