Godman Brian, Malmström Rickard E, Diogene Eduardo, Jayathissa Sisira, McTaggart Stuart, Cars Thomas, Alvarez-Madrazo Samantha, Baumgärtel Christoph, Brzezinska Anna, Bucsics Anna, Campbell Stephen, Eriksson Irene, Finlayson Alexander, Fürst Jurij, Garuoliene Kristina, Gutiérrez-Ibarluzea Iñaki, Hviding Krystyna, Herholz Harald, Joppi Roberta, Kalaba Marija, Laius Ott, Malinowska Kamila, Pedersen Hanne B, Markovic-Pekovic Vanda, Piessnegger Jutta, Selke Gisbert, Sermet Catherine, Spillane Susan, Tomek Dominik, Vončina Luka, Vlahović-Palčevski Vera, Wale Janet, Wladysiuk Magdalena, van Woerkom Menno, Zara Corinne, Gustafsson Lars L
Division of Clinical Pharmacology, Department of Laboratory Medicine, Karolinska Institutet, Karolinska University Hospital Huddinge Stockholm, Sweden ; Medicines Use and Health, Strathclyde Institute of Pharmacy and Biomedical Sciences, University of Strathclyde Glasgow, UK.
Clinical Pharmacology Unit, Department of Medicine, Karolinska Institutet, Karolinska University Hospital Solna Stockholm, Sweden.
Front Pharmacol. 2014 Jun 10;5:109. doi: 10.3389/fphar.2014.00109. eCollection 2014.
There are potential conflicts between authorities and companies to fund new premium priced drugs especially where there are effectiveness, safety and/or budget concerns. Dabigatran, a new oral anticoagulant for the prevention of stroke in patients with non-valvular atrial fibrillation (AF), exemplifies this issue. Whilst new effective treatments are needed, there are issues in the elderly with dabigatran due to variable drug concentrations, no known antidote and dependence on renal elimination. Published studies showed dabigatran to be cost-effective but there are budget concerns given the prevalence of AF. These concerns resulted in extensive activities pre- to post-launch to manage its introduction.
To (i) review authority activities across countries, (ii) use the findings to develop new models to better manage the entry of new drugs, and (iii) review the implications based on post-launch activities.
(i) Descriptive review and appraisal of activities regarding dabigatran, (ii) development of guidance for key stakeholder groups through an iterative process, (iii) refining guidance following post launch studies.
Plethora of activities to manage dabigatran including extensive pre-launch activities, risk sharing arrangements, prescribing restrictions and monitoring of prescribing post launch. Reimbursement has been denied in some countries due to concerns with its budget impact and/or excessive bleeding. Development of a new model and future guidance is proposed to better manage the entry of new drugs, centering on three pillars of pre-, peri-, and post-launch activities. Post-launch activities include increasing use of patient registries to monitor the safety and effectiveness of new drugs in clinical practice.
Models for introducing new drugs are essential to optimize their prescribing especially where concerns. Without such models, new drugs may be withdrawn prematurely and/or struggle for funding.
在为新型高价药物提供资金方面，当局与公司之间可能存在冲突，尤其是在涉及有效性、安全性和/或预算问题时。达比加群是一种用于预防非瓣膜性心房颤动（AF）患者中风的新型口服抗凝剂，它就是这一问题的典型例子。虽然需要新的有效治疗方法，但由于药物浓度可变、没有已知解毒剂且依赖肾脏排泄，老年人使用达比加群存在问题。已发表的研究表明达比加群具有成本效益，但鉴于房颤的患病率，仍存在预算担忧。这些担忧导致在上市前至上市后的广泛活动，以管理其引入。
（i）审查各国当局的活动；（ii）利用研究结果开发新模型，以更好地管理新药的引入；（iii）审查基于上市后活动的影响。
（i）对达比加群相关活动进行描述性审查和评估；（ii）通过迭代过程为关键利益相关者群体制定指南；（iii）在上市后研究后完善指南。
为管理达比加群开展了大量活动，包括广泛的上市前活动、风险分担安排、处方限制以及上市后对处方的监测。由于担心其预算影响和/或过度出血，一些国家拒绝给予报销。建议开发一种新模型和未来指南，以更好地管理新药的引入，重点围绕上市前、上市期间和上市后活动的三个支柱。上市后活动包括增加使用患者登记系统，以监测新药在临床实践中的安全性和有效性。
引入新药的模型对于优化其处方至关重要，尤其是在存在担忧的情况下。没有这样的模型，新药可能会过早撤出市场和/或难以获得资金。
