治疗性孤儿,非标签,儿科药物开发:为未成年人提供合理的药物治疗
Klaus Rose1, Jane M Grant-Kels2, Pasquale Striano3
1Klausrose Consulting, Pediatric Drug Developent & More, Riehen, Switzerland.
Expert opinion on pharmacotherapy
|November 11, 2024
概括
旨在改善儿童药物的儿科药物开发 (PDD) 立法,主要用于为未成年人创建药物标签. 研究往往夸大了对儿童广泛药理动力学和安全性测试的需要.
科学领域:
- 药理学和治疗学 药理学和治疗学
- 儿科医学 儿科医学
- 监管科学 监管科学
背景情况:
- "治疗孤儿"的概念出现在20世纪60年代,导致了儿童药物开发 (PDD) 的全球立法.
- 从历史上看,药物开发主要集中在成年人身上,对儿科人口的考虑有限.
- 制药开发的进步已经将重点转移到专门的药物应用,包括针对未成年人的药物.
研究的目的:
- 批判性地评估儿科药物开发 (PDD) 立法的真正目标和结果.
- 检查儿童群体中药理动力学和安全性研究的必要性和范围.
- 分析儿童药物研究的监管框架内的潜在利益冲突.
主要方法:
- 审查历史立法框架及其对儿科药物研究的影响.
- 对在未成年人身上进行广泛的药理动力学和安全性测试的科学理由和理由的分析.
- 审查监管机构的行动,例如欧洲药物署自2007年以来扩大PDD的行动.
主要成果:
- PDD立法的主要结果是为未成年人创建药物标签,而不是开发从根本上新的儿科药物.
- 在新生儿学之外,在儿科人群中进行广泛的临床,药理动力学和安全性研究的理由往往是夸张的.
- 监管扩展和研究人员的资金需求可能会导致过度强调儿科药物研究,造成利益冲突.
结论:
- 儿科药物开发 (PDD) 已经发展成为一个监管过程,专注于儿童治疗的标签,而不是创新.
- 需要重新评估未成年人药理动力学和安全性研究的范围和必要性,将其与成人要求区分开来.
- 研究人员和监管机构之间的潜在利益冲突需要在小儿药物管理方面仔细考虑.
相关概念视频
01:02Prescription, Nonprescription and Orphan Drugs
697
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...
697
01:21Factors Affecting Drug Response: Overview
1.9K
When it comes to infants and young children, they are typically administered smaller doses of medication in comparison to adults. This is primarily because their organ functions still need to fully develop, meaning their bodies are not as efficient at metabolizing or eliminating drugs. Additionally, their blood-brain barrier is more permeable than in adults. As a result, high concentrations of drugs can easily penetrate the central nervous system (CNS), potentially leading to neurological...
1.9K
01:26Drug Administration and Therapy Phases: Overview
409
Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
409
01:28Preclinical Development: Overview
4.2K
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...
4.2K
01:26Drug Discovery: Overview
7.5K
Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
7.5K
01:25Drug Regulation
1.3K
Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
1.3K