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

Giuseppe Traversa, Lucia Masiero, Luciano Sagliocca and Francesco Trotta. Orphanet Journal of Rare Diseases 2016 11:36 DOI: 10.1186/s13023-016-0420-4

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Our data suggest that adequately powered randomized trials can represent the gold standard also for rare diseases. There are also important implications for clinical practice, as can be expected by projects characterized by clinical endpoints and extensive follow-up. It is unlikely that many of these studies may have been conducted in a for profit perspective, given the absence of commercial interest.
We documented that, even though it takes time to set up and conduct a funding program for independent research, the overall 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.

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