Abstract
Purpose
Compassionate use of forthcoming drugs has become an increasing pathway through which patients can take advantage of promising medicines. We aimed to analyse the main features of the requests of compassionate use submitted to the Independent Ethics Committee (IEC) of the University Hospital of Bologna in the period 2010–2015.
Methods
The present analysis concerns the requests of compassionate use received by the IEC in the period 2010–2015. For each requested drug, we paired the date of the first request to our IEC with the date(s) of (a) submission to EMA, (b) CHMP positive opinion, and (c) European marketing authorization (if issued).
Results
In the period 2010–2015, our IEC received compassionate use requests for 610 patients. Most of the requests concerned patients suffering from solid or haematological cancers not responsive to first or second line of treatment. Sixty-five couples of medicine/clinical condition (corresponding to 56 individual medicines) were submitted to our IEC, and 62 of them regarded products following the centralised procedure at the EMA. Twenty-one out of the latter (34%) had already obtained CHMP positive opinion.
Conclusions
Our results indicate that compassionate use of forthcoming medicines represents a not negligible portion of the therapies utilized in hospital care. The observed large resort to medicines still on trial may suggest that doctors are more aware with the potential benefits of the new drugs. However, this trend may also indicate an increasing marketing activity of the pharmaceutical industry, addressing to get the clinicians used to the upcoming medicines.
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Acknowledgments
The opinions expressed herein are those of the authors and do not necessarily reflect the views of the IEC of the University Hospital St. Orsola-Malpighi.
Authors’ contribution
Study conception and planning: N. M., G. C., S. P., and D. M.
Acquisition, analysis, or interpretation of data: N. M., G. C., S. P., M. M.
Drafting of the manuscript: N. M., G. C., S. P..
Critical revision of the manuscript for important intellectual content: N. M., D. M., M. M.
All the authors approved the final version of the manuscript.
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The manuscript does not contain clinical studies or patient data. For this type of study, formal consent is not required. The authors declare that they have no conflict of interest. No founding source.
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Table 1S
Medicines requested for compassionate use in the period 2010–20151 and the diseases as reason of the request2 ordered according to the date of the first request (DOCX 38 kb)
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Montanaro, N., Melis, M., Proni, S. et al. Six-year activity on approval of compassionate use of medicines by the Ethics Committee of the University Hospital of Bologna (Italy): time to update rules and recommendations. Eur J Clin Pharmacol 73, 479–485 (2017). https://doi.org/10.1007/s00228-016-2186-y
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DOI: https://doi.org/10.1007/s00228-016-2186-y