EM2 Economic IMPACT of Compassionate Use of Medicines

Abstract BACKGROUND: Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing earlier access to medicines to patients not recruited in trials. While the economic impact of clinical trials has been already investigated, no evidence on the net economic benefit of CUP exists. This research aims to fill the information gap by estimating the economic consequences of 11 CUP in Italy conducted between May 2015 and December 2020 from the perspective of health care payers. Eight programs concern cancer treatments, two refer to drugs for spinal muscular atrophy, and one is indicated for multiple sclerosis.METHODS: The net economic benefit includes the avoided costs from the Standard of Care (SoC) the patients would have received if they had not joined the CUP, and costs not covered by the pharmaceutical industry but instead sustained by payers, such as those associated to adverse events (only severe sides effects resulting in hospitalisation and attributed to CUP medicines), and 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: 2,712 patients were treated in the 11 CUP, where SoC was identified. The SoC mean cost per patient ranges 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 from €26.5 million to €50.6 million (€17.8 million - €42.0 million for cancer treatments). CONCLUSIONS: Despite research limitations, this paper illustrates for the first time the net economic impact of CUP in oncology patients from a 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.

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Economic impact of compassionate use of medicines.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.e18840 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. e18840-e18840

Author(s):

Claudio Jommi ◽

Federico Pantellini ◽

Lisa Stagi ◽

Marianna Cavazza

Keyword(s):

Economic Impact ◽

Economic Value ◽

Indirect Costs ◽

Standard Of Care ◽

Diagnostic Procedures ◽

Cancer Drugs ◽

Compassionate Use ◽

Net Benefit ◽

Total Cost ◽

Mean Cost

e18840 Background: The economic impact of clinical trials in the perspective of trial sites has been already investigated. Instead, there is no evidence on the economic net benefit of compassionate use programs for medicines (CUP). This research aims to fill the information gap, investigating the economic consequences of 8 CUP in Italy carried out from May 2015 to December 2020 in the hospitals’ perspective. These programs concern five cancer medicines (alectinib, atezolizumab, cobimetinib, polatuzumab vedotin, trastuzumab emtansine TDM-1), for a total of 8 programs. Methods: Economic net benefit includes avoided costs for standard of care (SoC) the patient would have received if he/she has not joined the CUP and costs not covered by the pharmaceutical industry and sustained by the hospital hosting CUP. The latter include costs of adverse event (only severe sides effects generating hospitalisation and ascribed to medicines used in CUP), combination therapies and diagnostic procedures not covered by the sponsor. SoC costing relied on publicly available estimation. Adverse events and diagnostic procedures were retrieved from the CUP and monetized using the relevant fee for episode. Results: 1635 cancer patients were treated in eight CUPs. The SoC mean cost per patient ranges from €13355 to €28098 for all cancer drugs. The total cost of the SoC ranges from €21.8 - €45.9 million. The mean cost per patient covered by hospitals hosting CUP was equal to €2732 for cancer drugs, with a total cost of €4.5 million. The net economic benefit ranges from €17.4 million to €41.5 million for cancer treatments. Conclusions: Despite its limitations this paper illustrates for the first time the net economic impact of CUP in oncology patients in the perspective of payers. Additional evaluations are ongoing to better understand the overall effects of CUP implementation, i.e. the economic value of the comparative benefit profile of medicines used in CUP versus the SoC, including potential effects on indirect costs.

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The economic impact of compassionate use of medicines

BMC Health Services Research ◽

10.1186/s12913-021-07255-w ◽

2021 ◽

Vol 21 (1) ◽

Author(s):

Claudio Jommi ◽

Federico Pantellini ◽

Lisa Stagi ◽

Maria Verykiou ◽

Marianna Cavazza

Keyword(s):

Adverse Events ◽

Economic Impact ◽

Economic Benefit ◽

Muscular Atrophy ◽

Diagnostic Procedures ◽

Public Health Care ◽

Marketing Approval ◽

Compassionate Use ◽

Total Cost ◽

Mean Cost

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.

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Siblings and Discordant Eligibility for Gene Therapy Research: Considering Parental Requests for Non-Trial "Compassionate Use”

Clinical Ethics ◽

10.1177/1477750920983571 ◽

2021 ◽

pp. 147775092098357

Author(s):

Jamie Webb ◽

Lesha D Shah ◽

Alison Bateman-House

Keyword(s):

Decision Making ◽

Gene Therapy ◽

Economic Impact ◽

Best Interests ◽

Decision Making Process ◽

Compassionate Use ◽

Gene Therapy Trial ◽

The Family ◽

Access Request ◽

Therapy Trial

Deciding whether to grant an expanded access request for a child whose sibling is enrolled in a gene therapy trial involves a number of complex factors: considering the best interests of the child, the psychosocial and economic impact on the family, and the concerns and obligations of researchers. Despite the challenges in coming to a substantively fair outcome in cases of discordant eligibility, creating a procedurally fair decision-making process to adjudicate requests is essential.

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VP99 Economic Impact Of rpFVIII In The Management Of Acquired Hemophilia A

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462317003610 ◽

2017 ◽

Vol 33 (S1) ◽

pp. 194-194

Author(s):

Valentina Lorenzoni ◽

Isotta Triulzi ◽

Giuseppe Turchetti

Keyword(s):

Economic Impact ◽

Hemophilia A ◽

Recombinant Dna ◽

National Health System ◽

Unit Price ◽

Compassionate Use ◽

Acquired Hemophilia A ◽

Acquired Hemophilia ◽

Bleeding Episodes ◽

The Impact

INTRODUCTION:Acquired hemophilia A (AHA) is a rare coagulation disease characterized by frequent bleeding episodes treated with plasma-derived products and bypassing agents as rFVIIa and aPCC. Similar to the previous plasma-derived porcine FVIII and without its side effects, pFVIII (Obizur®) is a porcine recombinant factor VIII produced with the recombinant DNA technique. The study analyzes the economic impact of pFVIII compared to the other available therapies in order to manage the bleeding episodes in AHA patients.METHODS:To assess the impact of the introduction of pFVIII in the market-mix of products for the management of AHA a budget impact analysis was conducted from the perspective of the Italian National Health System (INHS) and considering a three-year time horizon. Consumption of products, products’ wastage, needs for additional treatment in case of failure of first line therapy, laboratory tests, hospitalization and drug wastage were considered for cost estimation. Model inputs were derived from literature, preliminary experience with the use of pFVIII for compassionate use, and from the updating of previous evidence by data collected among a panel of clinical experts. Univariate sensitivity analysis was performed to explore overall uncertainties in input parameters.RESULTS:The management of a bleeding episode considering conventional treatment is EUR8,229,621 per year, with an overall cost over three years equal to EUR24,688,864. The introduction of pFVIII leads to an overall costs saving ranging from EUR2,253,938 and EUR1,196,985 when the treatment duration is varied between 5 and 6.5 days, according to data from compassionate use or literature, respectively.CONCLUSIONS:The model outlined a significant reduction of all the components of direct costs for the INHS when Obizur® is introduced into the market with an ex-factory unit price equal to EUR2.32/IU.

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