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 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.

The role of clinical trials in the sustainability of the Italian national health service cancer drug expenditure

“…In many cases, compassionate use represents an early access tool to bridge the gap between approval and reimbursement timelines. Jommi et al in 2021 estimated that avoided drug costs reported in compassionate use studies measuring only drug costs ranged from €1,746 to €49,301 per patient 21. Our analysis confirms that the guarantee of the free supply of drugs by the pharmaceutical company for compassionate use represents an important economic opportunity.…”

International Country-Level Trends, Factors, and Disparities in Compassionate Use Access to Unlicensed Products for Patients With Serious Medical Conditions

“…These additional costs may affect CU activity, especially in countries where health care costs are paid out of pocket. 47 Overall, there is a need for the active involvement and collaboration of several stakeholders, including regulators, industry, physicians, and patients, for the CU access process to better address patients’ needs and to specifically support countries in building up their CU access capabilities.…”

“…Although the provision of therapeutic products is generally free of charge in CU, ancillary costs may have to be borne by the patient, health care professionals, or third-party payers, depending on the local circumstances. These additional costs may affect CU activity, especially in countries where health care costs are paid out of pocket . Overall, there is a need for the active involvement and collaboration of several stakeholders, including regulators, industry, physicians, and patients, for the CU access process to better address patients’ needs and to specifically support countries in building up their CU access capabilities.…”

Value generated by the multiplier effect of investments by the pharmaceutical industry in Italy: proposals to promote competitiveness and attractiveness

“…A questi vantaggi, si associano i benefici dei programmi di uso compassionevole (CUP, quantificati tra € 26,5 mln ed € 50,6 mln netti nei soli 11 programmi condotti in Italia tra il 2015 e il 2020) ( 9 ) e quelli non economici della ricerca clinica, tra cui l’accesso precoce per i pazienti alle terapie innovative, con conseguente miglioramento degli esiti clinici e della qualità di vita dei pazienti e dei caregiver in generale ( 10 ).…”