Abstract
BackgroundIn 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.MethodsWe 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.ResultsDuring 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.ConclusionsEven 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.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-016-0420-4) contains supplementary material, which is available to authorized users.
Highlights
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
Assessing the outcomes of a funding program on independent research on drugs requires answering three main questions: 1) were the studies concluded and their findings published? 2) were the results potentially relevant for clinical practice and regulatory decisions? and 3) given the overall outcomes, is it possible to substantiate the specific role of independent research? Our study suggests that a positive answer can be given to all these issues
In early 2016, an analysis on rare disease trials registered in CTG showed that less than half of the studies (47 %) had results published in scientific journals within 3 years after the conclusion [17]; only 35 % of the studies had the findings reported at the CTG website, despite the requirements of the US Food and Drug Administration to posting results within 1 year after trial completion
Summary
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 [6, 7]. Finance for this program comes through an innovative policy: all international and national pharmaceutical companies operating in Italy are required to contribute 5 % of their yearly expenditure for promotional initiatives to a national fund.
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