Background Italian Ministerial Decree 8 May 2003, amended in 2008, on the therapeutic use of an investigational medicinal product, represents the legislative frame defining a Compassionate Use Program (CUP). Accordingly, an investigational drug can be requested to the manufacturing Company for use outside clinical trials, for a single patient or a group, if alternative treatments are not available. Safety and efficacy data from completed or ongoing phase III trials – in life threatening condition, data from completed phase II – must be available and comparable, and a positive opinion be adopted by the local Ethics Committee (EC).
Purpose The aim of this surveillance was to achieve a picture of the CUPs at Hospital level. The outcome as subsequent Marketing Authorisation (MA) of the involved products was also considered.
Materials and methods The authors analysed data on the CUPs reported in our local Clinical Trial database, managed by a Pharmacist of the EC scientific Secretariat, in the timeframe between July 2006 and July 2011. The following main parameters were considered: active substance, ATC, patients' number, therapeutic indication, MA.
Results More than 66 patients were involved in 27 CUPs that received a positive opinion by EC. Sixteen programs were approved in oncology (59%), 11 in autoimmune/neurological diseases (41%); the higher number of patients (17) in neuroendocrine tumours. CUPs accounted for 18 different active substances, the most representatives being Everolimus (18 patients), Riluzole (14), Rituximab (7), Panitumumab (5), Abiraterone (4), Nilotinib (3). Eleven active substance out of 16 in oncology CUPs obtained a subsequent centralised MA. The cost for patients' monthly treatment with the a.m. products (except abiraterone) can be calculated as 143352 €.
Conclusions CUPs may be therapeutic options for patients in settings where there still are unmet medical needs and can also represent a cost-saving opportunity for the Hospital.
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