Rare essentials: drugs for rare diseases as essential medicines


Rare essentials: drugs for rare diseases as essential medicines


Stolk P; Willemen Marjolein JC; Leufkens HGM


Bulletin Of The World Health Organization




Humans; decision making; Health Services Accessibility; Needs Assessment; Risk Assessment; Cost-Benefit Analysis; Organizational Policy; Public Health Administration; Policy Making; Rare Diseases; Organizational; Resource Allocation; Health Priorities; Drugs; World Health Organization; Essential; Orphan Drug Production; Social Justice


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.


Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).


Journal Article

Citation List Month



Stolk P; Willemen Marjolein JC; Leufkens HGM, “Rare essentials: drugs for rare diseases as essential medicines,” Pediatric Palliative Care Library, accessed April 14, 2024, https://pedpalascnetlibrary.omeka.net/items/show/13265.