The economic impact of compassionate use of medicines

Abstract Background Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing access to medicines before marketing approval to patients not recruited in trials. The economic impact of clinical trials has previously been investigated. No evidence on the net economi...

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Autores principales:	Claudio Jommi, Federico Pantellini, Lisa Stagi, Maria Verykiou, Marianna Cavazza
Formato:	article
Lenguaje:	EN
Publicado:	BMC 2021
Materias:	Compassionate Use Medicines Economic Impact Italy Public aspects of medicine RA1-1270
Acceso en línea:	https://doaj.org/article/f41042770fd5499b9a7e061ca48ac62e
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spelling	oai:doaj.org-article:f41042770fd5499b9a7e061ca48ac62e2021-12-05T12:06:46ZThe economic impact of compassionate use of medicines10.1186/s12913-021-07255-w1472-6963https://doaj.org/article/f41042770fd5499b9a7e061ca48ac62e2021-12-01T00:00:00Zhttps://doi.org/10.1186/s12913-021-07255-whttps://doaj.org/toc/1472-6963Abstract Background Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing access to medicines before marketing approval to patients not recruited in trials. The economic impact of clinical trials has previously been investigated. No evidence on the net economic benefit of CUP exists. This research aims to address this information gap by estimating the economic consequences of 11 CUP in Italy conducted between March 2015 and December 2020 from the perspective of public health care system in Italy (National Health Service). Eight programs concern cancer treatments, two refer to spinal muscular atrophy, and one is indicated for multiple sclerosis. Methods Since CUP medicines are covered by the industry, the net economic benefit includes: (i) avoided costs of the Standard of Care (SoC) the patients would have received had they not joined the CUP, (ii) costs not covered by the pharmaceutical industry sponsor, but instead sustained by payers, such as those associated with adverse events (only severe side effects resulting in hospitalisation and attributable to CUP medicines), and (iii) costs for combination therapies and diagnostic procedures not used with the SoC. The SoC costing relied on publicly available data. Information on adverse events and diagnostic procedures was retrieved from the CUP and monetized using the relevant fee for episode or service. One CUP was excluded since a SoC was not identified. Results 2,713 patients were treated in the 11 CUP where a SoC was identified. The SoC mean cost per patient ranged from €11,415 to €20,299. The total cost of the SoC ranged between €31.0 and €55.1 million. The mean cost per patient covered by hospitals hosting CUP was equal to €1,646, with a total cost of €4.5 million. The net economic benefit ranged €26.5 million - €50.6 million. Conclusions Despite research limitations, this paper illustrates for the first time the net economic impact of CUP from a public payer perspective. It is important to integrate these estimates with the prospective effects of CUP implementation, i.e., the economic value of the comparative benefit profile of medicines used in CUP versus the SoC, including effects from a societal perspective.Claudio JommiFederico PantelliniLisa StagiMaria VerykiouMarianna CavazzaBMCarticleCompassionate UseMedicinesEconomic ImpactItalyPublic aspects of medicineRA1-1270ENBMC Health Services Research, Vol 21, Iss 1, Pp 1-13 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Compassionate Use Medicines Economic Impact Italy Public aspects of medicine RA1-1270
spellingShingle	Compassionate Use Medicines Economic Impact Italy Public aspects of medicine RA1-1270 Claudio Jommi Federico Pantellini Lisa Stagi Maria Verykiou Marianna Cavazza The economic impact of compassionate use of medicines
description	Abstract Background Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing access to medicines before marketing approval to patients not recruited in trials. The economic impact of clinical trials has previously been investigated. No evidence on the net economic benefit of CUP exists. This research aims to address this information gap by estimating the economic consequences of 11 CUP in Italy conducted between March 2015 and December 2020 from the perspective of public health care system in Italy (National Health Service). Eight programs concern cancer treatments, two refer to spinal muscular atrophy, and one is indicated for multiple sclerosis. Methods Since CUP medicines are covered by the industry, the net economic benefit includes: (i) avoided costs of the Standard of Care (SoC) the patients would have received had they not joined the CUP, (ii) costs not covered by the pharmaceutical industry sponsor, but instead sustained by payers, such as those associated with adverse events (only severe side effects resulting in hospitalisation and attributable to CUP medicines), and (iii) costs for combination therapies and diagnostic procedures not used with the SoC. The SoC costing relied on publicly available data. Information on adverse events and diagnostic procedures was retrieved from the CUP and monetized using the relevant fee for episode or service. One CUP was excluded since a SoC was not identified. Results 2,713 patients were treated in the 11 CUP where a SoC was identified. The SoC mean cost per patient ranged from €11,415 to €20,299. The total cost of the SoC ranged between €31.0 and €55.1 million. The mean cost per patient covered by hospitals hosting CUP was equal to €1,646, with a total cost of €4.5 million. The net economic benefit ranged €26.5 million - €50.6 million. Conclusions Despite research limitations, this paper illustrates for the first time the net economic impact of CUP from a public payer perspective. It is important to integrate these estimates with the prospective effects of CUP implementation, i.e., the economic value of the comparative benefit profile of medicines used in CUP versus the SoC, including effects from a societal perspective.
format	article
author	Claudio Jommi Federico Pantellini Lisa Stagi Maria Verykiou Marianna Cavazza
author_facet	Claudio Jommi Federico Pantellini Lisa Stagi Maria Verykiou Marianna Cavazza
author_sort	Claudio Jommi
title	The economic impact of compassionate use of medicines
title_short	The economic impact of compassionate use of medicines
title_full	The economic impact of compassionate use of medicines
title_fullStr	The economic impact of compassionate use of medicines
title_full_unstemmed	The economic impact of compassionate use of medicines
title_sort	economic impact of compassionate use of medicines
publisher	BMC
publishDate	2021
url	https://doaj.org/article/f41042770fd5499b9a7e061ca48ac62e
work_keys_str_mv	AT claudiojommi theeconomicimpactofcompassionateuseofmedicines AT federicopantellini theeconomicimpactofcompassionateuseofmedicines AT lisastagi theeconomicimpactofcompassionateuseofmedicines AT mariaverykiou theeconomicimpactofcompassionateuseofmedicines AT mariannacavazza theeconomicimpactofcompassionateuseofmedicines AT claudiojommi economicimpactofcompassionateuseofmedicines AT federicopantellini economicimpactofcompassionateuseofmedicines AT lisastagi economicimpactofcompassionateuseofmedicines AT mariaverykiou economicimpactofcompassionateuseofmedicines AT mariannacavazza economicimpactofcompassionateuseofmedicines
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The economic impact of compassionate use of medicines

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