title: Expert Delphi survey on research and development into drugs for neglected diseases.
abstract: BACKGROUND
  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. 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.
  
  
  METHODS
  An 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.
  
  
  RESULTS
  In 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.
  
  
  CONCLUSIONS
  While 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.
authors.affiliation: Dept. of Epidemiology & International Public Health, School of Public Health, Bielefeld University, P,O, Box 10 01 31, 33501 Bielefeld, Germany.
authors.forename: Angela
authors.initials: A
authors.lastname: Fehr
authors.forename: Petra
authors.initials: P
authors.lastname: Thürmann
authors.forename: Oliver
authors.initials: O
authors.lastname: Razum
mesh_terms: D035843:Biomedical Research / Q000458:organization & administration*; D003697:Delphi Technique; D006801:Humans; D007391:International Cooperation; D020407:Internet; D058069:Neglected Diseases / Q000188:drug therapy*; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence
references.citation: Nature. 2007 Sep 13;449(7159):174-5
references.pmid: 17851514
references.citation: Lancet. 2002 Jun 22;359(9324):2188-94
references.pmid: 12090998
references.citation: Trop Med Int Health. 1999 Jun;4(6):412-20
references.pmid: 10444316
references.citation: Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2007 Dec;50(12):1477-83
references.pmid: 18026888
references.citation: PLoS Med. 2005 Feb;2(2):e14
references.pmid: 15736991
references.citation: Health Aff (Millwood). 2009 Nov-Dec;28(6):1750-9
references.pmid: 19887416
references.citation: Nat Rev Drug Discov. 2002 Oct;1(10):821-5
references.pmid: 12360259
references.citation: Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2008 May;51(5):519-28
references.pmid: 18425694
references.citation: Bull World Health Organ. 2006 May;84(5):414-6
references.pmid: 16710555
references.citation: Expert Rev Anti Infect Ther. 2005 Dec;3(6):841-3
references.pmid: 16307495
references.citation: Int J Health Plann Manage. 2009 Jan-Mar;24(1):27-42
references.pmid: 18435430
references.citation: Trop Med Int Health. 2006 Sep;11(9):1335-8
references.pmid: 16930253
references.citation: Lancet. 2004 Jul 24-30;364(9431):380-3
references.pmid: 15276399
references.citation: Nature. 2007 Sep 13;449(7159):166-9
references.pmid: 17851512
references.citation: PLoS Med. 2004 Oct;1(1):e6
references.pmid: 15526054
references.citation: PLoS Biol. 2004 Feb;2(2):E52
references.pmid: 14966544
references.citation: Wkly Epidemiol Rec. 2011 Mar 25;86(13):113-20
references.pmid: 21438440
references.citation: PLoS Med. 2009 Feb 3;6(2):e30
references.pmid: 19192946
references.citation: Trop Med Int Health. 2001 Nov;6(11):945-51
references.pmid: 11703850