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Estimands and the Patient Journey: Addressing the Right Question in Oncology Clinical Trials
- Source :
- JCO precision oncology. 3
- Publication Year :
- 2022
-
Abstract
- The diversity of patient journeys can raise fundamental questions regarding the evaluation of drug effects in clinical trials to inform clinical practice. When defining the treatment effect of interest in a trial, the researcher needs to account for events occurring after treatment initiation, such as the start of a new therapy, before observing the end point. We review the newly introduced estimand framework to structure discussions on the relationship between patient journeys and the treatment effect of interest in oncology trials. In 2017, the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use released a draft addendum to its E9 guideline. The addendum introduces the concept of an estimand to precisely describe the treatment effect of interest. This estimand framework provides a structured approach to discuss how to account for intercurrent events that occur after random assignment and may affect the assessment or interpretation of the treatment effect. The framework is expected to improve coherence between trial objectives, design, analysis, and interpretation, as illustrated by examples in oncology disease settings. The estimand framework was applied to design a trial for a chimeric antigen receptor T-cell therapy. The treatment effect of interest was carefully defined considering the range of patient journeys expected for this particular indication and treatment. The trial design was developed accordingly to assess that treatment effect. All parties involved in the design of clinical trials need to consider possible patient journeys to define appropriate treatment effects and corresponding trial designs and analysis strategies. The estimand framework provides a common language to address the complexity introduced by varied patient journeys.
- Subjects :
- Cancer Research
medicine.medical_specialty
business.industry
media_common.quotation_subject
01 natural sciences
Clinical Practice
Clinical trial
010104 statistics & probability
03 medical and health sciences
0302 clinical medicine
Oncology
030220 oncology & carcinogenesis
medicine
Treatment effect
0101 mathematics
Intensive care medicine
business
Diversity (politics)
media_common
Subjects
Details
- ISSN :
- 24734284
- Volume :
- 3
- Database :
- OpenAIRE
- Journal :
- JCO precision oncology
- Accession number :
- edsair.doi.dedup.....2e3b464cdebbdd06a01f63947c32ec2d