PUB - Publikationen an der Universität Bielefeld

Background Rare diseases and neglected diseases are characterized by deficits in drug research and development (R&D) activities owing to market failures. Rare diseases do not offer a lucrative market because of the very small numbers of patients affected; neglected diseases, in contrast, are highly prevalent, but in poor and marginalized populations in developing countries. Public health policy responded to the R&D deficit for rare diseases with the adoption of orphan drug acts, i.e. regulatory instruments which contain financial and non-financial incentives for the pharmaceutical industry to encourage R&D into treatments for rare diseases. Similar legislation for neglected diseases does not exist, even though neglected diseases were part of the initial concepts which formed the basis for orphan drug acts. The debate about applying orphan drug acts to neglected diseases is ongoing in the scientific community. At the same time, a draft for an international medical R&D treaty has been developed, which proposes to restructure funding for medical research and development globally, and to heighten the role and financial obligations of the public sector, especially for neglected diseases. Objectives The prime objective of this research project was to gather stakeholders’ opinions on the desirability and the feasibility of implementing a regulatory instrument to promote R&D into drugs for neglected diseases. Orphan drug regulations, the draft Medical Research and Development Treaty and their R&D-promoting mechanisms served as frames of reference. A secondary objective of this project was to explore the acceptance and the feasibility of the method of the Policy Delphi for our research question. Methods An international online-Delphi survey was conducted with stakeholders of different backgrounds and professional affiliations. Their opinions were compiled and analyzed on causes for the treatment deficit for neglected diseases, on a possible definition of neglected diseases, on desirable and feasible measures to promote neglected disease R&D, and on the desirability and feasibility of a regulatory instrument to foster R&D for neglected diseases. Results 117 (first round) and 56 (second round) stakeholders participated in the survey. In both rounds of survey, the majority of the respondents (88.4% first round, 86.8% second round) advocated the development of a regulatory instrument to promote R&D for neglected diseases. Most respondents (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 with skepticism, key provisions of 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 R&D Treaty. Conclusions While not all features of orphan drug regulations and of the draft Medical Research and Development Treaty 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.