'Quality in, quality out', a stepwise approach to EBM for rare diseases promoted by MEN1.

ABSTRACT: Rare diseases pose specific challenges in the field of medical research to provide physicians with evidence based guidelines derived from studies with sufficient quality. An example of these rare diseases is multiple endocrine neoplasia type 1 (MEN1), which is an autosomal dominant endocrine tumor syndrome with an estimated occurrence rate of 2-3 per 100.000. For this complex disease, characterized by multiple endocrine tumors, it proves difficult to perform both adequate and feasible studies. The opinion of patients themselves is of utmost importance to identify the gaps in the evidence based medicine regarding clinical care. In the search for scientific answers to clinical research questions, the aim for best available evidence is obvious. Observational studies within patient cohorts, al

SUBMITTER: van Beek DJ 

PROVIDER: S-EPMC6215791 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature