使用外部数据来根据NICE对瘤药物技术评估的整体生存率推断
Audrey Petitjean1, Huiyu Shang2, Ash Bullement3,4
1Sanofi, Lyon, France.
Journal of medical economics
|May 20, 2025
概括
外部数据,包括现实世界的数据,越来越多地用于瘤单一技术评估 (STA) 中的整体存活率 (OS) 估计. 尼斯一般接受这种外部证据,但强有力的敏感性分析和明确的理由是必不可少的.
科学领域:
- 卫生经济学和结果研究研究.
- 瘤学药物评估 瘤学药物评估
- 卫生技术评估 卫生技术评估
背景情况:
- 国家卫生和护理卓越研究所 (NICE) 单一技术评估 (STA) 评估瘤药物.
- 准确估计整体存活 (OS) 对这些评估至关重要.
- 往往需要外部证据来补充OS外推的试验数据.
研究的目的:
- 评估NICE在瘤学STA中使用外部证据来估计整体存活 (OS).
- 在操作系统推断中识别外部数据的来源,类型和应用.
- 评估NICE证据审查小组 (ERG) 和评估委员会对外部数据的接受程度.
主要方法:
- 2021年1月至2023年3月期间,NICE评估的瘤学STA的系统审查.
- 提取有关操作系统推断方法的数据,使用外部数据,数据源,以及NICE的接受.
- 分析在不同的建模方法中使用外部数据的频率和理由.
主要成果:
- 在82个符合条件的STA中,32个 (39%) 使用了外部数据来进行OS推断.
- 通常使用的外部数据包括试验数据 (44%),现实数据 (47%) 和临床意见 (25%).
- 外部数据为事件后的过渡,治愈假设,长期存活率和代孕分析提供了信息;NICE在50%的病例中接受了外部数据.
结论:
- 外部数据,包括真实世界的数据和临床意见,经常被纳入瘤学STAs用于OS估计.
- 尼斯的ERG和评估委员会通常接受外部数据的使用.
- 敏感性分析和数据和方法的明确理由对于接受瘤学评估中的外部证据至关重要.
更多相关视频
07:42Patient-Derived Tumor Explants As a "Live" Preclinical Platform for Predicting Drug Resistance in Patients
Published on: February 7, 2021
4.8K
08:29Ex Vivo Treatment Response of Primary Tumors and/or Associated Metastases for Preclinical and Clinical Development of Therapeutics
Published on: October 2, 2014
14.6K
相关概念视频
Cancer Survival Analysis
313
Cancer survival analysis focuses on quantifying and interpreting the time from a key starting point, such as diagnosis or the initiation of treatment, to a specific endpoint, such as remission or death. This analysis provides critical insights into treatment effectiveness and factors that influence patient outcomes, helping to shape clinical decisions and guide prognostic evaluations. A cornerstone of oncology research, survival analysis tackles the challenges of skewed, non-normally...
313
Kaplan-Meier Approach
67
The Kaplan-Meier estimator is a non-parametric method used to estimate the survival function from time-to-event data. In medical research, it is frequently employed to measure the proportion of patients surviving for a certain period after treatment. This estimator is fundamental in analyzing time-to-event data, making it indispensable in clinical trials, epidemiological studies, and reliability engineering. By estimating survival probabilities, researchers can evaluate treatment effectiveness,...
67
Actuarial Approach
50
The actuarial approach, a statistical method originally developed for life insurance risk assessment, is widely used to calculate survival rates in clinical and population studies. This method accounts for participants lost to follow-up or those who die from causes unrelated to the study, ensuring a more accurate representation of survival probabilities.
Consider the example of a high-risk surgical procedure with significant early-stage mortality. A two-year clinical study is conducted,...
Consider the example of a high-risk surgical procedure with significant early-stage mortality. A two-year clinical study is conducted,...
50
Combination Therapies and Personalized Medicine
4.8K
Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
4.8K
Comparing the Survival Analysis of Two or More Groups
107
Survival analysis is a cornerstone of medical research, used to evaluate the time until an event of interest occurs, such as death, disease recurrence, or recovery. Unlike standard statistical methods, survival analysis is particularly adept at handling censored data—instances where the event has not occurred for some participants by the end of the study or remains unobserved. To address these unique challenges, specialized techniques like the Kaplan-Meier estimator, log-rank test, and...
107
Targeted Cancer Therapies
7.4K
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
There are several types of targeted therapies against...
7.4K
