Around 30 million EU citizens suffer from one of more than 6000 identified rare diseases. Despite a recent surge in the development of orphan drugs, most rare diseases lack approved pharmaceutical treatment options. Although progress in genomics facilitates tailored treatments, clinical testing is hampered by limited population size and appropriate statistical techniques. As innovative perspectives are needed PersiST will improve statistical design and analysis techniques in small clinical trials used for treatment evaluation in rare diseases.

PersiST is strongly linked to the current major EU research initiatives for rare diseases (EJP-RD, ERN, IMI-c4c), and builds on three successfully completed EU research projects focusing on small populations: Asterix, IDeAl and InSPiRe. PersisSTs 15 early stage researchers (ESRs) will develop and apply innovative design and analysis methodologies around the topics “outcome”, “modelling” and “uncertainty” for clinical trials in rare diseases. Progress will be achieved through a multidisciplinary, cross-sector training environment. The supervisory team for each ESR will include a broad range of perspectives including medicine, regulatory and methodological. ESRs will do part of their project at pharma companies and attend cross-skill drug development meetings.

A comprehensive training program will foster employability of researchers in various sectors. All ESRs will be involved in designing and analysing real trials and obtain the complimentary skills to be effective in multidisciplinary collaborations. Success will be guaranteed through an established supervisory team and quality control implemented as a generic part of project management. Thus PersiST directly supports the Rare Disease European joint strategy by bringing innovative methodology to implementation in rare disease clinical trials, by strengthening and expanding collaborative networks, and by establishing a group of excellently trained researchers.