Why miltefosine-a life-saving drug for leishmaniasis-is unavailable to people who need it the most

Hdl Handle:
http://hdl.handle.net/10144/619157
Title:
Why miltefosine-a life-saving drug for leishmaniasis-is unavailable to people who need it the most
Authors:
Sunyoto, T; Potet, J; Boelaert, M
Journal:
BMJ Global Health
Abstract:
Miltefosine, the only oral drug approved for the treatment of leishmaniasis-a parasitic disease transmitted by sandflies-is considered as a success story of research and development (R&D) by a public-private partnership (PPP). It epitomises the multiple market failures faced by a neglected disease drug: patients with low ability to pay, neglect by authorities and uncertain market size. Originally developed as an anticancer agent in the 1990s, the drug was registered in India in 2002 to treat the fatal visceral leishmaniasis. At the time, miltefosine was considered a breakthrough in the treatment, making it feasible to eliminate a regional disease. Today, access to miltefosine remains far from secure. The initial PPP agreement which includes access to the public sector is not enforced. The reality on the ground has been challenging: shortages due to inefficient supply chains, and use of a substandard product which led to a high number of treatment failures and deaths. Miltefosine received orphan drug status in the USA; when it was registered there in 2014, a priority review voucher (PRV) was awarded. The PRV, meant to facilitate drug development for neglected disease, was subsequently sold to another company for US$125 million without, to date, any apparent impact on drug access. At the heart of these concerns are questions on how to protect societal benefit of a drug developed with public investment, while clinicians worldwide struggle with its lack of affordability, limited availability and sustainability of access. This article analyses the reasons behind the postregistration access failure of miltefosine and provides the lessons learnt.
Publisher:
BMJ Publishing Group
Issue Date:
3-May-2018
URI:
http://hdl.handle.net/10144/619157
DOI:
10.1136/bmjgh-2018-000709
PubMed ID:
29736277
Submitted date:
2018-05-18
Language:
en
ISSN:
2059-7908
Appears in Collections:
Leishmaniasis/Kala Azar

Full metadata record

DC FieldValue Language
dc.contributor.authorSunyoto, Ten
dc.contributor.authorPotet, Jen
dc.contributor.authorBoelaert, Men
dc.date.accessioned2018-05-22T14:51:13Z-
dc.date.available2018-05-22T14:51:13Z-
dc.date.issued2018-05-03-
dc.date.submitted2018-05-18-
dc.identifier.citationWhy miltefosine-a life-saving drug for leishmaniasis-is unavailable to people who need it the most. 2018, 3 (3):e000709 BMJ Glob Healthen
dc.identifier.issn2059-7908-
dc.identifier.pmid29736277-
dc.identifier.doi10.1136/bmjgh-2018-000709-
dc.identifier.urihttp://hdl.handle.net/10144/619157-
dc.description.abstractMiltefosine, the only oral drug approved for the treatment of leishmaniasis-a parasitic disease transmitted by sandflies-is considered as a success story of research and development (R&D) by a public-private partnership (PPP). It epitomises the multiple market failures faced by a neglected disease drug: patients with low ability to pay, neglect by authorities and uncertain market size. Originally developed as an anticancer agent in the 1990s, the drug was registered in India in 2002 to treat the fatal visceral leishmaniasis. At the time, miltefosine was considered a breakthrough in the treatment, making it feasible to eliminate a regional disease. Today, access to miltefosine remains far from secure. The initial PPP agreement which includes access to the public sector is not enforced. The reality on the ground has been challenging: shortages due to inefficient supply chains, and use of a substandard product which led to a high number of treatment failures and deaths. Miltefosine received orphan drug status in the USA; when it was registered there in 2014, a priority review voucher (PRV) was awarded. The PRV, meant to facilitate drug development for neglected disease, was subsequently sold to another company for US$125 million without, to date, any apparent impact on drug access. At the heart of these concerns are questions on how to protect societal benefit of a drug developed with public investment, while clinicians worldwide struggle with its lack of affordability, limited availability and sustainability of access. This article analyses the reasons behind the postregistration access failure of miltefosine and provides the lessons learnt.en
dc.language.isoenen
dc.publisherBMJ Publishing Groupen
dc.rightsArchived with thanks to BMJ Global Healthen
dc.titleWhy miltefosine-a life-saving drug for leishmaniasis-is unavailable to people who need it the mosten
dc.identifier.journalBMJ Global Healthen

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