一次性治疗的基于证据的价值评估的发展:Tisagenlecleucel作为一个案例研究
Theodore Laetsch1, Jie Zhang2, Hongbo Yang3
1Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Applied health economics and health policy
|April 29, 2024
概括
更新的分析表明,tisagenlecleucel是一种仿真抗原受体T细胞 (CAR-T) 疗法,比以前估计的更具成本效益. 新的5年数据显示了改善的结果,支持其在治疗急性淋巴细胞白血病中的价值.
科学领域:
- 在瘤学瘤学.
- 卫生经济学 卫生经济学
- 药物经济学 药物经济学
背景情况:
- 对CAR-T等一次性疗法的经济评估是具有挑战性的,因为长期结果不确定.
- 来自ELIANA试验的最近5年的数据和tisagenlecleucel的现实证据为重新评估之前的成本效益分析 (CEA) 提供了机会.
研究的目的:
- 对儿童/年轻成人复发性或耐火性急性淋巴细胞白血病 (r/r ALL) 中的tisagenlecleucel之前的CEA进行系统文献综述 (SLR).
- 评估来自ELIANA试验和CAR-T制造进步的5年疗效数据对更新的CEA模型的影响.
主要方法:
- 搜索了OVID MEDLINE/Embase和HTA数据库,用于对r/r ALL的tisagenlecleucel进行经济评估.
- 包括公开报告的增量成本效益比率 (ICER) 的研究,提取临床证据数据,模型结构,长期疗效推断和结果.
- 更新了以前的CEA模型,使用来自ELIANA的5年总生存率 (OS) 数据和现实世界的CAR-T输液率.
主要成果:
- 在SLR中包含了15项独特的研究;大多数发现 tisagenlecleucel 具有成本效益.
- 之前的研究低估了与观察到的ELIANA数据相比,3年和5年的OS率.
- 更新的CEA与5年的ELIANAOS和更高的输注率增加了质量调整后的生命年 (QALYs) 和生命年 (LYs),减少了ICER与比较疗法.
结论:
- 对 tisagenlecleucel 的初始成本效益预测可能低估了其经济价值.
- 付款人应随着新数据的出现,考虑灵活的报销政策和对新瘤疗法的持续评估.
更多相关视频
06:08Assessment of Chimeric Antigen Receptor T Cell-Associated Toxicities Using an Acute Lymphoblastic Leukemia Patient-Derived Xenograft Mouse Model
Published on: February 10, 2023
1.3K
06:15Tumor Treating Field Therapy in Combination with Bevacizumab for the Treatment of Recurrent Glioblastoma
Published on: October 27, 2014
27.4K
相关概念视频
Tumor Immunotherapy
520
Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.
520
Combination Therapies and Personalized Medicine
4.9K
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...
4.9K
Targeted Cancer Therapies
7.6K
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...
7.6K
Cancer Therapies
7.6K
Cancer therapies are various modes of treatment, such as surgery, radiation therapy, and chemotherapy that are administered to cancer patients.
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
7.6K
![Dynamic Imaging of Chimeric Antigen Receptor T Cells with [18F]Tetrafluoroborate Positron Emission Tomography/Computed Tomography](/_next/image?url=https%3A%2F%2Fcloudfront.jove.com%2FCDNSource%2Fteasers%2F62334.jpg&w=3840&q=50)