oncology protocol design and patient report outcomes:

Purpose Evidence suggests that the patient-reported outcome (PRO) content of cancer trial protocols is frequently inadequate and non-reporting of PRO findings is widespread. This qualitative study examined the factors influencing suboptimal PRO protocol content, implementation, and reporting, and use of PRO data during clinical interactions.

Full Answer

What do we know about Patient-Reported Outcomes?

Patient-reported outcomes, including symptomatic adverse events and physical function, are direct measures of how a patient is feeling and functioning while on therapy and therefore are directly relevant to the measurement and interpretation of tolerability.

When to use Patient-Reported Outcome measures (pro) in medical product development?

Refer to the guidance for industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims for situations where the PRO endpoint will be used as the primary evidence of effectiveness.

Why master protocol trials in Radiation Oncology?

The goal of master protocol trials in radiation oncology is not to slow innovation, but rather to allow more efficient and reliable evaluation and identification of high-value innovations for the most appropriate clinical indications.

How important is patient experience data in oncology drug development?

There has been a substantial shift in oncology drug development in the last decade, with recognition that the measurement and analysis of patient experience data is important complementary information when assessing benefit–risk [ 2, 3 ].

How do you develop a patient-reported outcome?

Strategies for Collecting High-Quality Patient-Reported OutcomesSet Clear Goals. ... Use Validated Questions When Possible. ... Avoid Multiple Interpretations of Questions. ... Keep It Short and Sweet. ... Ensure Patient Understanding. ... Choose the Best Method for the Target Population. ... Protect a Patient's Right to Refuse. ... Conclusion.

What are patient-reported outcomes in clinical trials?

Patient-reported outcomes (PROs) are any report of the patients' perspectives about the impact of disease and treatment on their health status, for example quality of life and symptoms, without the interpretation of a clinician, or anyone else [1, 2].

What are two types of patient-reported outcome measures?

There are two basic types: General health PROMs can be used to survey patients with any condition. They usually focus on general well-being, mental health and/or quality of life. Condition-specific PROMs usually concentrate on the symptoms of a particular disease.

What is patient-reported outcome data?

A patient-reported outcome (PRO) is "an outcome measure based on a report that comes directly from the patient (e.g., study subject) about the status of the patient's health condition without amendment or interpretation of the patient's response by a clinician or anyone else,” according to the National Health Council. ...

Why patient reported outcomes are important?

Patient-reported outcomes provide information on the patient experience and can be the target of therapeutic intervention.

Why use patient reported outcome measures?

The national Patient Reported Outcome Measures (PROMs) programme began in 2009. The purpose of PROMs is to collect information, from patients themselves, about how well the health service is treating them. PROMs allow us to understand the difference that healthcare interventions make to people's quality of life.

What is an example of a patient reported outcome?

Health-Related Quality of Life Numerous generic health status measures, such as the Medical Outcomes Study Short Form SF-36 (and related measures) and the Sickness Impact Profile are classic examples.

What are examples of patient outcomes?

Results. The attributes of patient outcomes include (1) patient functional status (maintained or improved), (2) patient safety (protected or unharmed), and (3) patient satisfaction (patient reporting of comfort and contentment).

What are PREMs and PROMs?

▪ Patient Reported Outcome Measures (PROMs) ▪ Real time patient feedback facilitated by Patient. Reported Experience Measures (PREMs)

What are reported outcomes?

Patient-Reported Outcomes (PROs) are defined by the National Quality Forum as “any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else.” Most healthcare organizations consistently measure things like ...

What is a patient reported outcome instrument?

1.1 PATIENT-REPORTED OUTCOMES (PROS) CMS defines a PRO as any report of the status of a patient's health condition or health behavior that. comes directly from the patient, without interpretation of the patient's response by a clinician or. anyone else.

What are report outcome measures?

Patient-reported outcome measures (PROMs) are used to assess a patient's health status at a particular point in time. PROMs tools can be completed either during an illness or while treating a health condition. In some cases, using pre- and post-event PROMs can help measure the impact of an intervention.

Why is the FDA's core set of clinical outcomes important?

A core set of clinical outcomes that are meaningful to patients and clinicians and sensitive to the intervention and that isolate the effect of the drug has been proposed by FDA as an important starting point to generate data more useful for regulatory decision making across cancer clinical trials.

What is the challenge of epro data collection?

There are operational challenges to gathering PRO data electronically. Backup power sources should be made available to ensure that the data are collected without device error, and patients should be able to easily access technological support services. From a data management perspective, data integrity and protection of patient-level data must be considered in an ePRO data collection modality (e.g., password protection, fingerprint). Data management and security is mandated as part of the 21 CFR Part 11 requirement to mitigate potential contamination of clinical data sets [ 9 ].

What is a pro assessment?

A patient-reported outcome (PRO) assessment is a measurement based on a report about the status of a patient's health condition that comes directly from the patient without amendment or interpretation of the patient's response by a clinician or anyone else [ 1 ]. PRO assessments in cancer clinical trials can be used to complement traditional safety and efficacy data by providing an accurate description of symptoms and their functional impacts experienced by patients undergoing anticancer therapy. There has been a substantial shift in oncology drug development in the last decade, with recognition that the measurement and analysis of patient experience data is important complementary information when assessing benefit–risk [ 2, 3 ]. Successful integration of PRO into cancer clinical trials is not accomplished by any one single drug development stakeholder. As previously described by Basch and colleagues, the Food and Drug Administration (FDA) and industry must collaborate closely to develop rigorous PRO endpoints and find opportunities to communicate the results, including potential inclusion of PRO information into FDA product labeling [ 4 ]. With an increasing amount of PRO being collected as part of cancer clinical trials, there are opportunities for PRO experts including patients, advocates, social scientists, statisticians, clinicians, and regulators to identify best practices to maximize the utility of this rich data source. The 2019 Accelerating Anticancer Agent Development and Validation (AAADV) Workshop assembled a panel to discuss “Patient-Reported Outcomes in Oncology Clinical Trials: Clinical Trial Design and Operational Issues Toward Regulatory Grade Clinical Experience.” This session was a series of perspectives on the collection, interpretation, and analysis of rigorous patient-reported outcome data generated in cancer clinical trials, with an emphasis on the progress that has been made in the last 5 years.