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Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet

Rare diseases (RD) pose serious challenges in terms of both diagnosis and treatment. Legislation was passed in the US (1983) and in EU (2000) aimed to reverse the previous neglect of RD, by providing incentives for development of “orphan drugs” (OD) for their management. Here we analyse the current...

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Autores principales: Luzzatto, Lucio, Makani, Julie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8784510/
https://www.ncbi.nlm.nih.gov/pubmed/35082665
http://dx.doi.org/10.3389/fphar.2021.770640
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author Luzzatto, Lucio
Makani, Julie
author_facet Luzzatto, Lucio
Makani, Julie
author_sort Luzzatto, Lucio
collection PubMed
description Rare diseases (RD) pose serious challenges in terms of both diagnosis and treatment. Legislation was passed in the US (1983) and in EU (2000) aimed to reverse the previous neglect of RD, by providing incentives for development of “orphan drugs” (OD) for their management. Here we analyse the current situation in Africa with respect to (1) sickle cell disease (SCD), that qualifies as rare in the US and in EU, but is not at all rare in African countries (frequencies up to 1–2%); (2) paroxysmal nocturnal haemoglobinuria (PNH), that is ultra-rare in Africa as everywhere else (estimated <10 per million). SCD can be cured by bone marrow transplantation and recently by gene therapy, but very few African patients have access to these expensive procedures; on the other hand, the disease-ameliorating agent hydroxyurea is not expensive, but still the majority of patients in Africa are not receiving it. For PNH, currently most patients In high income countries are treated with a highly effective OD that costs about $400,000 per year per patient: this is not available in Africa. Thus, the impact of OD legislation has been practically nil in this continent. As members of the medical profession and of the human family, we must aim to remove barriers that are essentially financial: especially since countries with rich economies share a history of having exploited African countries. We call on the Global Fund to supply hydroxyurea for all SCD patients; and we call on companies who produce ODs to donate, for every patient who receives an expensive OD in a high income country, enough of the same drug, at a symbolic price, to treat one patient in Africa.
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spelling pubmed-87845102022-01-25 Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet Luzzatto, Lucio Makani, Julie Front Pharmacol Pharmacology Rare diseases (RD) pose serious challenges in terms of both diagnosis and treatment. Legislation was passed in the US (1983) and in EU (2000) aimed to reverse the previous neglect of RD, by providing incentives for development of “orphan drugs” (OD) for their management. Here we analyse the current situation in Africa with respect to (1) sickle cell disease (SCD), that qualifies as rare in the US and in EU, but is not at all rare in African countries (frequencies up to 1–2%); (2) paroxysmal nocturnal haemoglobinuria (PNH), that is ultra-rare in Africa as everywhere else (estimated <10 per million). SCD can be cured by bone marrow transplantation and recently by gene therapy, but very few African patients have access to these expensive procedures; on the other hand, the disease-ameliorating agent hydroxyurea is not expensive, but still the majority of patients in Africa are not receiving it. For PNH, currently most patients In high income countries are treated with a highly effective OD that costs about $400,000 per year per patient: this is not available in Africa. Thus, the impact of OD legislation has been practically nil in this continent. As members of the medical profession and of the human family, we must aim to remove barriers that are essentially financial: especially since countries with rich economies share a history of having exploited African countries. We call on the Global Fund to supply hydroxyurea for all SCD patients; and we call on companies who produce ODs to donate, for every patient who receives an expensive OD in a high income country, enough of the same drug, at a symbolic price, to treat one patient in Africa. Frontiers Media S.A. 2022-01-10 /pmc/articles/PMC8784510/ /pubmed/35082665 http://dx.doi.org/10.3389/fphar.2021.770640 Text en Copyright © 2022 Luzzatto and Makani. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Pharmacology
Luzzatto, Lucio
Makani, Julie
Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet
title Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet
title_full Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet
title_fullStr Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet
title_full_unstemmed Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet
title_short Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet
title_sort treating rare diseases in africa: the drugs exist but the need is unmet
topic Pharmacology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8784510/
https://www.ncbi.nlm.nih.gov/pubmed/35082665
http://dx.doi.org/10.3389/fphar.2021.770640
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