对先进治疗药物的偏好:了解关于创新医疗干预措施价值的出版文献
N Ferizović1, P Lorgelly2, C S Clarke1
1University College London, UK.
概括
先进治疗药物 (ATMP) 显示出有前途,但面临着补偿挑战. 患者对这些基因和细胞/组织疗法的偏好始终强调临床益处,不确定性,风险,治疗负担和生活质量作为关键价值属性.
科学领域:
- 药物经济学 药物经济学
- 卫生技术评估 卫生技术评估
- 患者报告的结果
背景情况:
- 先进疗法药物 (ATMP) 是具有显著治疗潜力的创新基因和细胞/组织治疗方法.
- 尽管有许诺,但ATMP在决策过程中遇到重大障碍,特别是关于退款的问题.
- 了解患者感知价值对于成功采用ATMP和进入市场至关重要.
研究的目的:
- 系统地审查ATMPs发表的基于偏好的研究.
- 确定影响患者和利益相关者对这些新疗法的偏好的主要价值属性.
- 为进一步研究ATMP的综合价值奠定基础.
主要方法:
- 在多个数据库 (MedLine,Embase等) 进行了全面的文献审查. 在2024年3月3日之前.
- 寻找基因疗法和基于细胞/组织的疗法之间的差异化策略.
- 纳入标准侧重于同行评审的初级研究或使用声明偏好方法的系统性审查.
主要成果:
- 13项研究符合纳入标准 (8项基因疗法,5项细胞/组织疗法).
- 在两个ATMP类别中确定了一致的价值属性.
- 关键属性包括临床益处,不确定性,风险,治疗负担和生活质量.
结论:
- 目前关于ATMP偏好的发表研究是有限的.
- 基因和细胞/组织疗法的价值属性基本一致.
- 临床益处,不确定性,风险,治疗负担和生活质量是ATMP价值的关键决定因素.
更多相关视频
08:29Ex Vivo Treatment Response of Primary Tumors and/or Associated Metastases for Preclinical and Clinical Development of Therapeutics
Published on: October 2, 2014
15.1K
07:40Preparation of Peripheral Blood Mononuclear Cell Pellets and Plasma from a Single Blood Draw at Clinical Trial Sites for Biomarker Analysis
Published on: March 20, 2021
18.2K
相关概念视频
Combination Therapies and Personalized Medicine
5.8K
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...
5.8K
Targeted Cancer Therapies
8.6K
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...
8.6K
Drug Products: Biologics, Biosimilars and Interchangeables
208
Body:Biologics, derived from living sources such as humans, animals, or microorganisms, represent a significant category of pharmaceuticals. These complex molecules, developed through advanced biotechnological methods or purified from natural sources, include essential medical treatments like insulin and growth hormones. The complexity of biologics arises from their large molecular structures and the intricate processes required for their production, making them distinct from conventional...
208
Microorganisms in Medicine and Therapeutics
908
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
908
Clinical Trials: Overview
4.5K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
4.5K
Clinical Trials
10.1K
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
There are four phases in a clinical trial. A phase one...
10.1K
