Maria Sanna -

Involvement of patients is becoming a fundamental principle throughout drug development and during regulatory advice and assessment. Patients have been increasingly involved in European Medicines Agency (EMA) and Food and Drug Administration (FDA) procedures and the value of their input is broadly recognized by industry as well as the regulatory authorities.

Assessment of a clinical outcome can be made through report by a clinician, a patient, a non-clinician observer, a performance-based assessment, or through other methods.

A patient-reported outcome (PRO) is a type of clinical outcome assessment based on a report that comes directly from the patient about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.

Cancer trials typically employ standardized efficacy assessments using overall survival and tumor measures, and safety assessments provided by clinician reporting of adverse events. FDA acknowledges the potential added value of incorporating PRO measurement of symptoms and functional impacts into the benefit/risk assessment in appropriately designed trials.

In June 2021 FDA issued a draft Guidance on Measuring Patient-Reported Outcomes in Cancer Clinical Trials to address how to incorporate assessment of PRO in clinical trials  and the frequency of these PRO assessments.

The non-binding recommendations were developed by the Oncology Center of Excellence (OCE), Center for Drug Evaluation and Research (CDER), and Center for Biologics Evaluation and Research (CBER) at the FDA.

Authors defined a core set of PROs as “including disease symptoms, symptomatic adverse events, and physical function, that may be important contributors to a patient’s health-related quality of life (HRQOL) and that may be sensitive to the effect of the disease and treatment under study has been described.”

RPN has successfully supported companies in clinical program development. Our team is able to provide innovative strategic solutions to optimize clinical development in support of the targeted label indication and, to gain regulatory approval.

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