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Bulletin of the World Health Organization
Article . 2006 . Peer-reviewed
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Rare essentials drugs for rare diseases as essential medicines

Authors: Stolk, P.; Willemen, M.J.C.; Leufkens, H.G.M.;

Rare essentials drugs for rare diseases as essential medicines

Abstract

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 drugs"). With progress in pharmaceutical research (e.g. drugs targeted for narrower indications), these medicines will feature more often on future public health agendas. However, when current definitions for selecting essential medicines are applied strictly, orphan drugs cannot be part of the WHO Essential Medicines Programme, creating the risk that WHO may lose touch with this field. In our opinion WHO should explicitly include orphan drugs in its policy sphere by composing a complementary Orphan Medicines Model List as an addition to the EML. This complementary list of "rare essentials" could aid policy-makers and patients in, for example, emerging countries to improve access to these drugs and stimulate relevant policies. Furthermore, inconsistencies in the current EML with regard to medicines for rare diseases can be resolved. In this paper we propose selection criteria for an Orphan Medicines Model List that could form a departure point for future work towards an extensive WHO Orphan Medicines Programme.

Country
Netherlands
Related Organizations
Keywords

Ziekenhuisstructuur en organisatie van de gezondheidszorg, Orphan Drug Production, Epidemiology, Health Priorities, Cost-Benefit Analysis, Farmacie(FARM), Biomedische technologie en medicijnen, World Health Organization, Risk Assessment, Health Services Accessibility, Organizational Policy, Resource Allocation, Rare Diseases, SDG 3 - Good Health and Well-being, Social Justice, Humans, Public Health, Public aspects of medicine, RA1-1270, Drugs, Essential, Policy Making, Public Health Administration, Decision Making, Organizational, Needs Assessment

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    popularity
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    Top 10%
    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
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    impulse
    This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
    Top 10%
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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
88
Top 10%
Top 1%
Top 10%
Green
Published in a Diamond OA journal