title: 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.
authors.affiliation: Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, 3508 TB Utrecht, Netherlands.
authors.forename: Pieter
authors.initials: P
authors.lastname: Stolk
authors.forename: Marjolein J C
authors.initials: MJ
authors.lastname: Willemen
authors.forename: Hubert G M
authors.initials: HG
authors.lastname: Leufkens
mesh_terms: D003362:Cost-Benefit Analysis; D003659:Decision Making, Organizational; D018954:Drugs, Essential / Q000191:economics / Q000600:supply & distribution; D006292:Health Priorities; D006297:Health Services Accessibility / Q000191:economics; D006801:Humans; D020380:Needs Assessment; D016134:Organizational Policy; D009965:Orphan Drug Production; D011050:Policy Making; D011635:Public Health Administration; D035583:Rare Diseases / Q000188:drug therapy* / Q000191:economics; D040841:Resource Allocation; D018570:Risk Assessment; D012935:Social Justice; D014944:World Health Organization
chemical_list: D018954:Drugs, Essential