Clinical Benefit Evaluation in Oncology
Summary
The assessment of clinical benefit in oncology aims to determine whether new treatments deliver meaningful improvements in patient outcomes, such as survival, quality of life and symptom control. Traditional measures include overall survival, which captures the time from treatment initiation to death, and disease-specific endpoints such as progression-free survival or tumour response rates. Regulatory authorities and health technology assessment bodies increasingly scrutinise the validity of surrogate endpoints—intermediate measures that may predict longer-term outcomes—requiring robust evidence of correlation with final patient-centred endpoints. Economic considerations, including cost-effectiveness and budget impact, also play a key role in shaping access and reimbursement decisions worldwide. Recent years have witnessed efforts to harmonise methodological standards for endpoint validation, integrate patient-reported outcomes into trial design and refine value frameworks that balance clinical efficacy with safety profiles and affordability. This multifaceted approach seeks to ensure that novel therapies offer genuine advances over existing options and justify their adoption across diverse health-care settings.
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Clinical Benefit Evaluation in Oncology publication trend
The graph below shows the total number of articles in clinical benefit evaluation in oncology across all publications each year (not limited to Nature Index journals).
Technical terms
Overall survival (OS): The length of time from initiation of treatment until death from any cause, widely regarded as a definitive endpoint in cancer trials.
Progression-free survival (PFS): The period during which a patient’s disease does not worsen, used as a surrogate endpoint to assess treatment efficacy more rapidly than overall survival.
Surrogate endpoint: An intermediate clinical or biological marker intended to substitute for a direct measure of clinical benefit, requiring validation against final patient-centred outcomes.
Health technology assessment (HTA): A systematic evaluation of the properties, effects and impacts of health technologies, incorporating clinical, economic and social dimensions to inform policy and reimbursement decisions.
References
- Surrogate endpoints in oncology: when are they acceptable for regulatory and clinical decisions, and are they currently overused?. BMC Medicine (2017).
- Access to cancer medicines deemed essential by oncologists in 82 countries: an international, cross-sectional survey. The Lancet Oncology (2021).
- Progression-free survival as a surrogate for overall survival in oncology trials: a methodological systematic review. British Journal of Cancer (2020).
- FDA Acceptance of Surrogate End Points for Cancer Drug Approval: 1992-2019. JAMA Internal Medicine (2020).
- Surrogate Endpoints in Health Technology Assessment: An International Review of Methodological Guidelines. PharmacoEconomics (2020).
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