Background The compassionate use of drugs in Italy is a way of using drugs available in foreign countries but not in Italy. They have the authorization for the same therapeutic purpose but are not available on the market, however they are in clinical trials (CTs) or have been tested in phase III or – if the patient is in a critical condition – have been successful in phase II CTs.
As regards evaluating the efficacy of the treatment, a basic aspect of the CT is to evaluate the safety of the drugs used. Great attention is focused on this theme with sponsored CTs; in fact by law (decree 211/2003) the subjects liable for pharmacovigilance are expressly listed and stress is put on reporting adverse drug reactions (ADRs). Regarding the use of compassionate drugs, there are no laws regulating the reporting of ADRs.
Purpose To find out how any reports of possible ADRs emerging from compassionate studies are managed in ordinary clinical practise.
Materials and Methods For this purpose the database used in our organisation was essential: all of our centre’s CTs are listed there. The number of CTs classified by type (for profit, not-for-profit, compassionate) was extracted, focusing on the compassionate use CTs in particular. Then the number of patients treated was drawn from it as well as the pathologies and the ADRs pointed out during meetings with the physicians.
Results From this analysis it appeared that in our centre 197 studies (st) are active: 147 are for profit, 27 not-for-profit and 23 compassionate. Among these last ones, 8 are active in the sarcoma department, 5 in haematology, 3 in paediatrics, 6 in medical oncology and 1 in urology. The pathologies being examined are: GIST 4 active sts, 6 patients (pts), Hodgkin’s lymphoma 1 st (2 pts), T-cell lymphoma 1 st (1 pt), myeloma 2 sts (1 and 15 pts), thyroid carcinoma 1 st (1pt), bone metastases 1 st (4 pts), melanoma 2 sts (1 and 30 pts), villonodular tenosynovitis 1 st (4 pts), prostatic adenocarcinoma 1 st (4 pts), breast cancer 2 sts (3 and 2 pts), leiomyosarcoma 1 st (2 pts), myxoid liposarcoma 1 st (6 pts), brain stem glioma 1 st (8 pts), NET 1 st (17 pts), acoustic neuroma 1 st (1 pt), idiopathic myelofibrosis 1 st (1 pt), LLC 1 st (1 pt). Although the CTs for compassionate use are fewer than other kinds of trials and require a very low number of treated pts – also because it is a matter of a named patient use and in particular conditions – this does not justify not reporting ADRs. As such drugs are used for critical pts and often for non-approved uses, it seems useful to focus on this aspect, as it allows to more and better investigations on the side of the safety of the drugs.
Conclusions The results obtained underline the necessity of better awareness of the problem. As far as our centre is concerned, the results led us to hold meetings with the physicians and to plan interventions in order to make them aware of the problem and in order to start a process of pharmacovigilance with compassionate-use drugs.
No conflict of interest.
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