ReviewBMJ global health2018
Why miltefosine-a life-saving drug for leishmaniasis-is unavailable to people who need it the most.
Review in BMJ global health, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 76 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
76 citing papers in PubMed, 3 syntheses or guidelines pooled it, 152 citations in OpenAlex.
- Progress, impacts and lessons from market shaping in the past decade: a systematic review.Frontiers in public health · 2025Pooled it
- Clinical criteria for Mucosal Leishmaniasis diagnosis in rural South America: A systematic literature review.PLoS neglected tropical diseases · 2022Pooled it
- Safety and efficacy of allylamines in the treatment of cutaneous and mucocutaneous leishmaniasis: A systematic review.PloS one · 2021Pooled it
- Miltefosine and emerging Leishmania (Mundinia) in Southeast Asia: Molecular insights, therapeutic challenges, and future strategic implementation.PLoS neglected tropical diseases · 2026Review
- Priority without progress: the FDA's neglected tropical disease voucher program after 18 years.Health affairs scholar · 2026Article
- Weight-band-based simplification of oral allometric miltefosine dosing in paediatric patients with visceral leishmaniasis.The Journal of antimicrobial chemotherapy · 2026Article
- Insights into drug resistance inADMET & DMPK · 2026Review
- Oral 18-methoxycoronaridine activity in simian and murineFrontiers in pharmacology · 2026Article
- Interventions for Neglected Diseases Caused by Kinetoplastid Parasites: A One Health Approach to Drug Discovery, Development, and Deployment.Pharmaceuticals (Basel, Switzerland) · 2025Review
- Examining Pricing and Availability for Neglected Tropical Disease Therapies in the United States.Open forum infectious diseases · 2025Article
- Mechanism Insight of Cell Death Signaling by Thymol Derivatives on Trypanosomatidae Protozoan Parasites.Antibiotics (Basel, Switzerland) · 2025Article
- Mucocutaneous Leishmaniasis With Oral Involvement: A Case Report.Case reports in dentistry · 2025Article
- Article
- Cutaneous leishmaniasis: epidemiology, treatment access and translational challenges of topical therapies.Frontiers in microbiology · 2025Review
- Effectiveness of Antileishmanial Treatments in Three Endemic Areas of Colombia: A Community-Based Cohort Study.The American journal of tropical medicine and hygiene · 2024Article
- Synthesis and biological evaluation of 4,7,9-trisubstituted benzoxazepines as antileishmanial agents.Bioorganic & medicinal chemistry letters · 2024Article
- 1,2,4-Oxadiazole Derivatives: Physicochemical Properties, Antileishmanial Potential, Docking and Molecular Dynamic Simulations ofMolecules (Basel, Switzerland) · 2024Article
- Combination Therapy and Phytochemical-Loaded Nanosytems for the Treatment of Neglected Tropical Diseases.Pharmaceutics · 2024Review
- Innovating Leishmaniasis Treatment: A Critical Chemist's Review of Inorganic Nanomaterials.ACS infectious diseases · 2024Review
- American Cutaneous Leishmaniasis: Imported cases in Berlin 2000-2023.PLoS neglected tropical diseases · 2024Article
16 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
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.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.