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Infection

, Volume 47, Issue 1, pp 3–5 | Cite as

Neglected tropical diseases in Europe: rare diseases and orphan drugs?

  • Guido CalleriEmail author
  • Andrea Angheben
  • Marco Albonico
Editorial
  • 354 Downloads

Abstract

Neglected tropical diseases are becoming more and more frequent in Europe due to the increasing immigration from endemic areas. Nonetheless specific treatments are scarcely available in many European countries, since they are neither officially licensed nor marketed. Only a few referral health centres can afford to access drugs for NTDs due to complex bureaucracy and high cost, importing or providing them via the WHO. Health professionals and institutions in this domain should solicit other stakeholders (such as NGOs, the civil society, scientific societies) to sensitize health authorities to improve access to treatment for such debilitating diseases.

Keywords

Neglected tropical diseases Orphan drugs Access to care Migrants Parasitic 

Manuscript

Neglected tropical diseases (NTDs) are a group of diverse communicable diseases which are highly prevalent in tropical countries, causing a significant burden of morbidity, mortality, suffering and economic loss [1]. Moreover, their burden is particularly significant in low-income countries and in marginalized populations living in poverty, in close contact with vectors and animal reservoires of infectious diseases, and with limited access to safe water, sanitation and health care. Neglected tropical diseases mainly affect neglected populations.

They contrast to the so-called “big three” (malaria, tuberculosis and HIV infection), which drain most attention, interest, research and financing worldwide. This is one of the reasons why NTDs are called neglected.

The NTDs list defined by the World Health Organization (WHO) includes: Buruli ulcer, Chagas disease, dengue and chikungunya, dracunculiasis, echinococcosis, foodborne trematodiases, human African trypanosomiasis, leishmaniasis, leprosy, lymphatic filariasis, mycetoma, chromoblastomycosis and other deep mycoses, onchocerciasis, rabies, ectoparasites, schistosomiasis, soil-transmitted helminthiases, snakebite envenoming, taeniasis/cysticercosis, trachoma and yaws [2].

Some of these diseases are potentially fatal (e.g., strongyloidiasis, African trypanosomiasis, dengue), and some of them cause severe disability (e.g., schistosomiasis, filariasis, Chagas disease).

The migration from low-income countries to Europe, as well as international travels, are making these conditions more and more frequent nowadays even out of endemic areas [3, 4], but the European health systems are far to be prepared to face these increasing challenges.

Most NTDs are parasitic and deserve-specific treatment. Being categorized under “rare diseases” in Europe (although they affect more than 1 billion people in endemic countries), the status of their specific treatment is often referred to as orphan drugs, which allows them gaining profit incentives by the law in Europe [5]. Moreover, none of these drugs used to treat NTDs is listed under the orphan drugs in Italy (Orphanet) [6] nor the national drugs formulary contains the most part of them. This situation does happen also in other European countries, mostly because NTDs are rare; drugs are often old and cheap, so that their market has a limited appeal to the pharma industry and trade. Therefore, their access in Europe is often difficult, despite extensive donations are improving availability in endemic countries worldwide [7].

Nitazoxanide, suramin, eflornithine, melarsoprol, benznidazole, triclabendazole, miltefosine, diethylcarbamazine, praziquantel, thiabendazole, primaquine, meglumine antimoniate, and ivermectin, are just examples of medications for NTDs which, in spite of being included in the WHO essential medicines list [8] and not being replaceable by other ones, are not marketed in Italy. Most of them are not even licensed by the Italian drug regulatory agency (Agenzia Italiana del Farmaco—AIFA) [9] and for some of them the importation and distribution by public health centres and infectious diseases services are unfeasible. To sensitize the Ministry of Health and AIFA on the problem of low access to drugs for NTDs, following the meeting “Ivermectin days” held in Verona in November 2017, a network of partners was set up including representatives of the civil society, NGOs with focus on migrants’ health, the Italian Society of Tropical Medicine and Global Health, and other Institutions dealing with NTDs. The poor accessibility of NTDs drugs is similar in Spain (Agenzia Española de Medicamentos y Productos Sanitarios—AEMPS) [10] and slightly better in Germany (Bundesinstitut für Arzneimittel und Medizinprodukte—BfArM) [11] (Table 1), and possibly in other European countries. Unregistered drugs must be searched abroad, and their provision requests complicated bureaucracy and a significant waste of time, making in fact them available only for selected referral centres. Physicians must prescribe these drugs under their own responsibility, and the costs, which are increased by the import process, often burden the patients. Many of them cannot afford to buy the drugs, and thus simply do not get treated.

Table 1

Orphan drug licensing in Italy, Spain and Germany

Drug

Main target conditions

License

AIFA Italy

License

AEMPS Spain

License

BfArM Germany

Benznidazole

Chagas disease

No

No

No

Diethylcarbamazine

Lymphatic filariasis

No

No

No

Eflornithine i.v.

African trypanosomiasis

Withdrawn

No

No

Ivermectin tabs

Strongyloidiasis, onchocerciasis

No

No

Yes (for scabies only)

Meglumine antimoniate

Leishmaniasis

Withdrawn

Yes

No

Melarsoprol

African trypanosomiasis

No

No

No

Miltefosine

Leishmaniasis

No

No

Yes

Nitazoxanide

Giardiasis amoebiasis, cryptosporidiosis

No

No

No

Praziquantel

Schistosomiasis, cestodes, trematodes

No

No

Yes

Primaquine

P. vivax/P. ovale malaria

Withdrawn

No

No

Quinacrine*

Refractory giardiasis

No

No

No

Suramin

African trypanosomiasis

No

No

Yes

Thiabendazole

Strongyloidiasis, ancylostomiasis

Withdrawn

Withdrawn

No

Triclabendazole

Fascioliasis

No

No

No

*Not in the WHO essential medicines list

As far as population movements from low-income countries to Europe are not due to arrest—they are actually on the rise—health authorities should face the problem and get organized to allow these conditions be properly treated. Public registries of stocking should be made available to public hospitals and outpatient clinics of National Health Systems, and drugs should be either imported or locally produced. Accessibility to “orphan” drugs for NTDs, which are becoming increasingly more common in Europe, should be improved, providing simple and rapid registration routes and reducing costs, to protect both the patients and the community.

Notes

Acknowledgements

The authors wish to thank Dr. Giacoma Cinnirella, Dr. Gerardo Rojo-Marcos and Dr. Thomas Zoller for providing the information on drug licensing in Italy, Spain and Germany.

Compliance with Ethical Standards

Conflict of interest

On behalf of all authors, the corresponding author states that there is no conflict of interest.

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Copyright information

© Springer-Verlag GmbH Germany, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Travel Medicine UnitAmedeo di Savoia Hospital, ASL Città di TorinoTorinoItaly
  2. 2.Centre for Tropical DiseasesIRCCS Sacro Cuore Don Calabria HospitalNegrar-VeronaItaly
  3. 3.Department of Life Sciences and Systems BiologyUniversity of TorinoTorinoItaly

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