title: Orphan drugs assessment in the centralised procedure.
abstract: On the basis of the author's experience as member of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and in order to facilitate the access of new orphan drugs to the patients, some suggestions were given. Among these the following should be taken into account by the regulatory bodies: 1) conditional approval or approval under exceptional circumstances should be granted more frequently; 2) the opinion of international societies for rare diseases should be taken into greater account by the EMA Committees; 3) the guidelines requirements should be interpreted more flexibly; 4) in comparison to the fulfilment of primary and secondary endpoints, the improvement of the quality of life should justify the approval of a new orphan drug; 5) the rigidity of guideline requirements should not prevail over the unmet medical need for severe and lethal rare disorders; 6) the statistical values of clinical data to the limit of significance should not prevail over the opinion of patients' associations and international scientific societies; 7) the current legislation should be amended.
authors: Centro di Biotecnologie Farmaceutiche, Università degli Studi di Roma "Tor Vergata", Rome, Italy. nistico@uniroma2.it
authors: Giuseppe
authors: G
authors: Nisticò
mesh_terms: D017277:Drug Approval / Q000331:legislation & jurisprudence; D004345:Drug Industry; D006801:Humans; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence