Abstract
BackgroundTropical infectious diseases are called neglected, because they are, inter alia, characterized by an R&D deficit. A similar deficit exists for rare (orphan) diseases which neither promise a sufficient return on R&D investment. To encourage the development of treatments for rare diseases, orphan drug acts were created which contain financial and non-financial incentives for the pharmaceutical industry. Similar instruments aimed exclusively at neglected diseases do not yet exist. Proposals for a regulatory approach to promote R&D for neglected diseases include the application of selected orphan drug incentives, or the implementation of a Medical Research and Development Treaty (MRDT) with national funding obligations for medical R&D. We compiled and analyzed experts' opinions on causes for the treatment deficit for neglected diseases and on desirable and feasible measures to promote neglected disease R&D. Hereby, the focus was on mechanisms contained in orphan drug regulations and in the Medical Research and Development Treaty draft (Discussion draft 4, 2005). Lastly, we solicited experts' opinions on the desirability and feasibility of a regulatory instrument to foster R&D for neglected diseases.MethodsAn international online-Delphi survey was conducted with 117 (first round) and 56 (second round) experts of different professional backgrounds and professional affiliations who formulated and ranked causes and solutions related to the treatment deficit for neglected diseases.ResultsIn both rounds of survey, the majority of the participating experts (88.4% first round, 86.8% second round) advocated the development of a regulatory instrument to promote R&D for neglected diseases. Most experts (77.9% first round, 79.3% second round) also considered this to be a feasible option. With the exception of market exclusivity, which was viewed critically, key provisions contained in orphan drug regulations were judged favorably also for neglected diseases. A majority (87.1% first round, 77.2% second round) supported national funding obligations for neglected diseases which are proposed by the Medical Research and Development Treaty draft.ConclusionsWhile not all features of orphan drug regulations and of the MRDT draft received equal support, the view was expressed that a regulatory instrument would be a desirable and feasible measure to promote R&D for neglected diseases.
Highlights
Tropical infectious diseases are called neglected, because they are, inter alia, characterized by an research and development (R&D) deficit
Comparable legislation focusing on neglected diseases does not yet exist, even though tropical infectious diseases had originally been included both in the concept of “drugs of limited commercial value” [20] which formed the basis for the U.S Orphan Drug Act, and in early drafts for the European legislation [21]
The treatment deficit for neglected diseases can neither be attributed to a single cause, nor can it be remedied by a single measure
Summary
Tropical infectious diseases are called neglected, because they are, inter alia, characterized by an R&D deficit. A similar deficit exists for rare (orphan) diseases which neither promise a sufficient return on R&D investment. To encourage the development of treatments for rare diseases, orphan drug acts were created which contain financial and non-financial incentives for the pharmaceutical industry. For other so-called tool-deficient diseases, such tools are still lacking [2,3] With their often chronic and crippling character, neglected diseases cause immense suffering for the individual patient. A similar R&D deficit exists for rare diseases whose small patient populations neither promise adequate returns on investment [16,17,18,19]. Public health policy responded to the treatment deficit for rare diseases with the adoption of orphan drug acts, i.e. regulatory instruments with financial and non-financial incentives for the pharmaceutical industry. Sponsors of designated orphan products benefit from fee waivers, protocol assistance and, under the U.S Orphan Drug Act, from grants and tax credits
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