相关实验视频

Updated: May 8, 2025

Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients

Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients

Published on: October 15, 2017

四种孤儿药物定价模型的应用:对lumasiran的案例研究

Noa Rosenberg^1,2, Evert Manders^1,2, Sibren van den Berg^1,2

¹Medicine for Society, Platform at Amsterdam University Medical Center - University of Amsterdam, Amsterdam, The Netherlands.

Orphanet journal of rare diseases

|December 24, 2024

概括

需要为像卢马西兰这样的孤儿药物制定新的定价模式. 折扣现金流 (DCF) 模型显示了在为罕见疾病进行报销谈判时设定价格基准的潜力.

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
孤儿药物定价孤儿药物定价

背景情况:

孤儿药物的高价格和不确定的有效性挑战了医疗技术评估.
价格比较的新方法对于偿还谈判至关重要.
这项研究评估了基于成本的价格模型,用于治疗1型原发性高氧沙流症的治疗方法卢马西兰.

研究的目的:

评估四种孤儿药物定价模型的适用性.
通过使用基于成本的要素,建立卢马西兰的价格基准.
为罕见疾病治疗的定价和退款谈判提供信息.

主要方法:

应用了四种定价模型:新型癌症定价模型 (NCP模型),创新药物AIM模型 (AIM模型),折扣现金流模型 (DCF模型) 和实际期权收益率模型 (ROROR模型).
估计的价格范围使用最低和最大的场景,基于来自注册表,文献,证券交易委员会文件和专家意见的数据.
进行了敏感性分析,以确定有影响力的参数.

主要成果:

国家诊断中心模型:€87,000€224,000/患者/年.
根据AIM模型,每年33万欧元至34万欧元/患者.
在DCF模式下:€182,000€748,000/患者/年.
在ROROR模式下:每年81,000欧元到273,000欧元/患者.

结论:

这四种定价模式产生了广泛而异质的价格范围.
由于其全面的参数,DCF模型可能最适合于卢马西兰.
最低DCF价格可以作为谈判的起点;建议增加输入变量的透明度.

关键词:

这就是Lumasiran.孤儿药品孤儿药品是指孤儿药品的产品.定价模型的价格模式一次性高氧化尿症1型

更多相关视频

Bioluminescence-Based Tumor Quantification Method for Monitoring Tumor Progression and Treatment Effects in Mouse Lymphoma Models

Bioluminescence-Based Tumor Quantification Method for Monitoring Tumor Progression and Treatment Effects in Mouse Lymphoma Models

Published on: July 7, 2016

A Murine Ommaya Xenograft Model to Study Direct-Targeted Therapy of Leptomeningeal Disease

A Murine Ommaya Xenograft Model to Study Direct-Targeted Therapy of Leptomeningeal Disease

Published on: January 29, 2021

相关概念视频

Prescription, Nonprescription and Orphan Drugs

Prescription, Nonprescription and Orphan Drugs

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...

One-Compartment Open Model for IV Bolus Administration: Estimation of Elimination Rate Constant, Half-Life and Volume of Distribution

One-Compartment Open Model for IV Bolus Administration: Estimation of Elimination Rate Constant, Half-Life and Volume of Distribution

The one-compartment open model is a simplified approach used in pharmacokinetics to understand the distribution and elimination of a drug administered through an intravenous bolus. This model assumes rapid drug dispersal throughout the body and elimination using a first-order process. Key pharmacokinetic parameters, such as the elimination rate constant (k), half-life (t1/2), and the apparent volume of distribution (Vd), can be estimated from this model. The elimination rate is calculated...