[The pharmacist, rare diseases and orphan medicines].
نویسندگان
چکیده
Treatment of rare diseases is often complicated due to the difficulties posed by the research and development of medicines by the pharmacological industry. One of the greatest concerns of this group of patients, which is both scarce and diverse, is access to available medicines. The European Medicine Agency establishes the term orphan medicines and the incentives and bases for their commercialisation. Due to their special characteristics, the majority of orphan medicines require suitable vigilance and control. The hospital pharmacy services are actively involved in their management and supply, as well as in a correct pharmacotherapeutic monitoring of the patients.
منابع مشابه
Rare essentials: drugs for rare diseases as essential medicines.
Since 1977, the WHO Model List of Essential Medicines (EML), published by WHO, has provided advice for Member States that struggle to decide which pharmaceutical technologies should be provided to patients within their public health systems. Originating from outside WHO, an incentive system has been put in place by various governments for the development of medicines for rare diseases ("orphan ...
متن کاملDrugs for exceptionally rare diseases: do they deserve special status for funding?
Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally expensive on a per patient basis. European Union (EU) regulations promote the development of orphan drugs; but ...
متن کاملPW02-027 - CAPS and cost-effectiveness analysis project
Introduction Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally very expensive on a per patient basis. European Union (EU) regulations promote the development of ...
متن کاملNo difference in between-country variability in use of newly approved orphan and non- orphan medicinal products - a pilot study
BACKGROUND Regulators and payers have to strike a balance between the needs of the patient and the optimal allocation of resources. Drugs indicated for rare diseases (orphan medicines) are a special group in this context because of their often high per unit costs. Our objective in this pilot study was to determine, for drugs used in an outpatient setting, how utilisation of centrally authorised...
متن کاملEstimating the budget impact of orphan medicines in Europe: 2010 - 2020
BACKGROUND Orphan drugs are a growing issue of importance to European healthcare policy makers. The success of orphan drug legislation in Europe has resulted in an increasing number of licensed medicines for rare diseases, and many more yet unlicensed products have received orphan drug designation. Increasingly the concerns amongst policy makers relate to issues of patient access and affordabil...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
عنوان ژورنال:
- Anales del sistema sanitario de Navarra
دوره 31 Suppl 2 شماره
صفحات -
تاریخ انتشار 2008