Quality of life as an endpoint

Quality of life (QoL) assessment adds useful information to the efficacy and safety data obtained in clinical studies. It contributes to the evaluation of different treatments and identifies patients who might benefit from supportive interventions. QoL data can be used to inform policy and resource allocation, reveal benefits to patients despite objective toxicity, and be of prognostic value. Evaluation of patient’s QoL might help to determine the suitable moment to start specific palliative interventions.

Clinical trial endpoint selection

Commonly used endpoints are based on survival, tumor response, and symptom assessment. Historically, overall survival (OS) has been viewed as the most effective measure as it addresses the biology of the tumor and the natural history of disease. PFS possesses significance because it assesses tumor shrinkage and stabilization of disease.

Phase II trials

Phase II studies provide initial assessment of efficacy in a more homogeneous patient population. Screening out of ineffective drugs is an objective here, as is the identification of promising new agents for future evaluation. The activity of a compound is assessed in a given tumor type, which allows for selection of tumor types for further study. Also, safety (types and incidence of AEs) is further defined in a specific patient population/ disease setting.

Phase I trials

Phase I trials consist of the dose escalation portion and the cohort expansion portion. The dose escalation part is dedicated to assessment of PK, safety and MTD. This segment usually encompasses only very few patients for each of several dose levels. In the expansion phase, a greater number of patients is recruited into one or two selected dose levels with the purpose of decreasing confidence intervals.

Preface – Part II

Due to the innovations in the field of oncological treatment that have profoundly changed our perception of modern cancer therapy, the design of clinical trials had to be modified appropriately with the purpose of allowing for the assessment of the distinct effects of new drugs. This means that long-standing paradigms had to be abandoned, and new principles took their place.

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