Abstract
Orphan diseases and neglected diseases may both be defined as diseases for which discovering and development of new treatments are assumed not to provide substantial financial return to companies willing to take on the costs. In general, the term “orphan diseases” is used for rare diseases in the industrialized world, meaning that the limited number of patients prevents sufficient return (Editorial 2007; Heemstra et al. 2008; Joppi et al. 2009), whereas the term “neglected diseases” is used for diseases in the developing world, where the poor income of the patients prevents sufficient return (Oprea et al. 2009; Trouiller et al. 2001). In both cases, pharmaceutical companies will refrain from investing in drug development. Initiatives in the European Union and the United States have resulted in registration of drugs for orphan diseases (Heemstra et al. 2008; Joppi et al. 2009). Incentives to develop drugs for neglected diseases are made through public-private partnerships (PPP), which are public-health-driven not-for-profit organizations that drive neglected-disease drug development in conjugation with industry groups. An example is Medicine for Malaria Venture (Moran 2005). In 2005, half the activity in this field was performed in PPP with small companies, whereas the other half was performed in PPP with large companies such as Glaxo SmithKline, Novartis, Astra Zeneca, and Sanofi-Aventis (Moran 2005). PPP has resulted in drugs having a major impact on global health such as ivermectin (Mectizan), which halved the global burden of onchocerciasis between 1990 and 2000, praziquantel (Biltricide), which has helped to control schistosomiasis in Brazil and other endemic countries, and artemether-lumefantrine (Coartem), which has delivered Africa its first safe effective new antimalarial drug for many years (Moran 2005). An unusual program of combination therapy is presently running, in which the pharmaceutical company Merck & Co. donates Mectizan and GlaxoSmithKline donates Albendazole (Zentel) to combat river blindness in exposed areas in Africa (The Mectizan donation program). A successful outcome of this program might eliminate the disease from the infected areas. As an appreciation of the pharmaceutical company Novartis’ involvement in the development of Coartem, the American Food and Drug Agency (FDA) has awarded the company quick access to register new drugs (Anderson 2009).
Original language | English |
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Title of host publication | Bioactive Compounds from Natural Sources : Natural Products as Lead Compounds in Drug Discovery, Second Edition |
Number of pages | 26 |
Publisher | CRC Press |
Publication date | 1 Jan 2011 |
Pages | 525-550 |
ISBN (Print) | 9781439822296 |
ISBN (Electronic) | 9781439870662 |
Publication status | Published - 1 Jan 2011 |