Dataset Information

Italian program for independent research on drugs: 10 year follow-up of funded studies in the area of rare diseases.

ABSTRACT: 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.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.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.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.

SUBMITTER: Traversa G

PROVIDER: S-EPMC4828875 | biostudies-literature | 2016 Apr

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Italian program for independent research on drugs: 10 year follow-up of funded studies in the area of rare diseases.

Traversa Giuseppe G Masiero Lucia L Sagliocca Luciano L Trotta Francesco F

Orphanet journal of rare diseases 20160412

<h4>Background</h4>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.<h4>Methods</h4>We conducted a cohort study on the projects that were funded by the AIFA in ...[more]

PMID: 27068647

Similar Datasets

Project description:BackgroundIn the 2.8 years of the Diabetes Prevention Program (DPP) randomised clinical trial, diabetes incidence in high-risk adults was reduced by 58% with intensive lifestyle intervention and by 31% with metformin, compared with placebo. We investigated the persistence of these effects in the long term.MethodsAll active DPP participants were eligible for continued follow-up. 2766 of 3150 (88%) enrolled for a median additional follow-up of 5.7 years (IQR 5.5-5.8). 910 participants were from the lifestyle, 924 from the metformin, and 932 were from the original placebo groups. On the basis of the benefits from the intensive lifestyle intervention in the DPP, all three groups were offered group-implemented lifestyle intervention. Metformin treatment was continued in the original metformin group (850 mg twice daily as tolerated), with participants unmasked to assignment, and the original lifestyle intervention group was offered additional lifestyle support. The primary outcome was development of diabetes according to American Diabetes Association criteria. Analysis was by intention-to-treat. This study is registered with ClinicalTrials.gov, number NCT00038727.FindingsDuring the 10.0-year (IQR 9.0-10.5) follow-up since randomisation to DPP, the original lifestyle group lost, then partly regained weight. The modest weight loss with metformin was maintained. Diabetes incidence rates during the DPP were 4.8 cases per 100 person-years (95% CI 4.1-5.7) in the intensive lifestyle intervention group, 7.8 (6.8-8.8) in the metformin group, and 11.0 (9.8-12.3) in the placebo group. Diabetes incidence rates in this follow-up study were similar between treatment groups: 5.9 per 100 person-years (5.1-6.8) for lifestyle, 4.9 (4.2-5.7) for metformin, and 5.6 (4.8-6.5) for placebo. Diabetes incidence in the 10 years since DPP randomisation was reduced by 34% (24-42) in the lifestyle group and 18% (7-28) in the metformin group compared with placebo.InterpretationDuring follow-up after DPP, incidences in the former placebo and metformin groups fell to equal those in the former lifestyle group, but the cumulative incidence of diabetes remained lowest in the lifestyle group. Prevention or delay of diabetes with lifestyle intervention or metformin can persist for at least 10 years.FundingNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).

Project description:Sotos syndrome is caused by a gene deletion with an autosomal dominant pattern of inheritance. Cerebral gigantism, hypotonia and joint hyperextensibility are characteristic features of this syndrome. A percentage of these patients develop progressive scoliosis early in life. In the literature, few studies on the evolution of scoliosis in Sotos syndrome have been published. We retrospectively evaluated eight patients diagnosed with Sotos syndrome and scoliosis treated at the Garrahan Children Hospital between 1988 and March 2009. Clinical charts and imaging studies were assessed. Eight patients (19%) presented with scoliosis and seven of them (87.5%) required surgical treatment. The mean follow-up was 9.5 years (range 3-18). Mean age at first consultation was 5.2 years (range 1.1-11.2). Mean Cobb angle for scoliosis at first consultation was 34.3° (range 20°-42°) and the mean Cobb angle for kyphosis was 45.6° (range 30°-90°). Mean age at surgery was 11.2 years (range 3.7-18.10). The surgical procedures performed were instrumented posterior arthrodesis, alone or combined with anterior arthrodesis, instrumented anterior arthrodesis, while one patient is currently in treatment with growing rods. Preoperative mean Cobb angle for scoliosis was 72.3° (range 54°-130°) and for kyphosis was 59.8° (range 30°-108°); postoperative mean Cobb angle for scoliosis was 45.5° (range 6°-90°) and for kyphosis was 40.2° (range 30°-80°). There were three early complications (pleural effusion in two cases and death due to sepsis in one) and two late complications (kyphosis above the instrumentation area and dislodgement of the proximal hooks). Incidence of scoliosis in Sotos syndrome is high and thus close monitoring of patients with Sotos syndrome during growth is important for early detection of this entity. Joint hyperextensibility and hypotonia that are characteristic of the syndrome should be considered at the moment of surgery to avoid short fusions.

Dataset Information

Italian program for independent research on drugs: 10 year follow-up of funded studies in the area of rare diseases.

Publications

Italian program for independent research on drugs: 10 year follow-up of funded studies in the area of rare diseases.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets