儿童癌症药物的全球格局和开发管道:综合分析与仪表板生成
Celeste Cleveland1, Ambinintsoa H Ralaidovy2, Chrys Jallo3
1Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ, USA; Department of Child Health, University of Arizona College of Medicine, Phoenix, AZ, USA.
The Lancet. Child & adolescent health
|June 28, 2025
概括
尽管取得了进展,但儿童癌症药物的可获得性仍然有限. 这项研究分析了全球儿科瘤药物开发,揭示了儿童批准药物和配方中的重大差距,突出了公平治疗的障碍.
科学领域:
- 儿科瘤学 儿科瘤学
- 药物开发 药物开发
- 全球健康 全球健康
背景情况:
- 儿童癌症的生存率有所改善,但获得必需药物仍然是一个挑战.
- 在全球范围内,儿童癌症药物的可用性和开发存在重大差异.
研究的目的:
- 分析正在使用或开发的儿童癌症药物的全球格局和管道.
- 识别儿科瘤学药物获取和开发中的障碍.
主要方法:
- 搜索了国际临床试验注册平台 (ICTRP) 的儿科瘤学试验 (2007-2022).
- 提取了关于药物机制,开发阶段,配方,储存和监管状态的数据.
- 单独分析细胞疗法.
主要成果:
- 确定了440种独特的儿童癌症药物,55%是向疗法或免疫疗法.
- 只有45%的口服药物具有儿科友好的配方; 37%需要冷藏.
- 低百分比的药物被EMA (8%) 和FDA (19%) 批准用于儿科,但在批准方面存在很大的滞后.
结论:
- 全球儿科瘤学药物环境揭示了儿童获得有效治疗的关键障碍.
- 公开可用的数据应该指导药物开发,配方,监管批准和能力建设的优先级.
相关概念视频
Cancer Therapies
8.0K
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...
8.0K
Combination Therapies and Personalized Medicine
5.1K
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...
5.1K
Preclinical Development: Overview
4.9K
Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
4.9K
Prescription, Nonprescription and Orphan Drugs
857
Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
857
Targeted Cancer Therapies
7.8K
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...
7.8K
Treatment Resistant Cancers
3.4K
Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
3.4K


