title: Italian program for independent research on drugs: 10 year follow-up of funded studies in the area of rare diseases.
abstract: BACKGROUND
  In 2005 the Italian Medicines Agency (AIFA) started a program on independent research on drugs, with the aim to promote clinical research in areas of limited commercial interest. For 3 years (2005-2007) an area of the program was reserved to studies in the field of rare diseases. There is a concern that public funding of research may be wasted. We investigated the outcome of the program.
  
  
  METHODS
  We conducted a cohort study on the projects that were funded by the AIFA in the area of rare diseases. The outcomes were the proportion of published studies, time to publication, impact factor of the publishing journals and relevance for clinical practice. We retrieved published articles through a literature search in peer reviewed biomedical journals indexed by Pubmed. We used the Kaplan-Meier method to estimate the cumulative probability of publication by time from project starting to publication.
  
  
  RESULTS
  During the period 2005-2007, 62 projects were funded in the area of rare diseases. Most of the studies (n 39; 63%) had a randomized design and in 22 (35%) the control group received an active treatment. For 39 studies (63%) we retrieved a publication in a peer reviewed journal. The median time to publication was 74 months and, at the maximum period of follow up (109 months), the cumulative probability of publication reached 77%. The median impact factor was 5.4 (range 1.4-52.4). Considering the clinical relevance, more than 30% of the published articles presented conclusive findings; an additional 10% of the studies reached potential breakthrough findings.
  
  
  CONCLUSIONS
  Even though it takes time to set up and conduct a funding program for independent research on drugs, the results are highly rewarding. Independent funding is crucial in supporting studies aimed at answering questions that are relevant for clinical practice despite the lack of sufficient commercial interest.
authors.affiliation: Pharmacoepidemiology Unit, National Centre for Epidemiology, National Institute of Health, Viale Regina Elena 299, 00161, Rome, Italy. giuseppe.traversa@iss.it.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Traversa
authors.affiliation: Italian National Transplant Center, National Institute of Health, Viale Regina Elena 299, 00161, Rome, Italy.
authors.forename: Lucia
authors.initials: L
authors.lastname: Masiero
authors.affiliation: ARSan Agenzia Regionale Sanitaria Regione Campania, Centro Direzionale di Napoli, Isola F9, 80143, Naples, Italy.
authors.forename: Luciano
authors.initials: L
authors.lastname: Sagliocca
authors.affiliation: Department of Epidemiology, Lazio Regional Health Service, Via Cristoforo Colombo 112, 00147, Rome, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Trotta
mesh_terms: D015706:Bibliometrics; D035843:Biomedical Research; D002986:Clinical Trials as Topic; D015331:Cohort Studies; D006801:Humans; D007558:Italy; D035583:Rare Diseases; D012189:Retrospective Studies
keywords: Bibliometrics; Clinical trials; Cohort studies; Independent research; Rare diseases