Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review
Abstract
1. Introduction
2. Materials and Methods
2.1. Protocol, Registration, and Reporting
2.2. Eligibility Criteria, Information Sources, and Search Strategy
2.3. Study Selection, Data Extraction, and Data Synthesis
2.4. Quality Assessment
2.5. Statistical Analyses
3. Results
3.1. The Literature Search and Screening Results
3.2. Quality Assessment Results
3.3. Overall Characteristics of Studies Included in the Review
3.4. Cost-Effectiveness Profiles of the Overall Studies
3.5. Cost-Effectiveness Profiles of ATMPs for Rare Blood Cancer and Hemophilia B Treatment
3.6. Cost-Effectiveness Profiles of ATMPs for Spinal Muscular Atrophy Type I Treatment
3.7. Cost-Effectiveness Profiles of ATMPs for Retinal Diseases with RP E65-Mutation Treatment
4. Discussion
5. Conclusions
Supplementary Materials
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Conflicts of Interest
References
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Study Characteristics | Inclusion Criteria | Exclusion Criteria |
---|---|---|
Study type | Research articles, articles in the press, evaluation studies, evidence-based studies, comparative studies, economic evaluations, assessments, communications, and original investigations | Books, book reviews, commentaries, conference proceedings, rationale and/or design, corrections/retraction, dissertation/thesis, editorials, literature reviews, evidence reviews, scoping reviews, letters, periodicals, conference abstracts, study protocols, consensus, notes, systematic reviews, conference papers, erratum, short surveys, conference reviews, chapters, reports, case reports, case studies, review articles, clinical trials, viewpoint articles, rapid communication, gray literature |
Publication stage | Published articles | Preprints; under publication articles |
Publication availability | Full open-access articles | Non-open access articles; partial open access articles |
Language | English | All other languages |
Setting | All | Not applied |
Time frame | All | Not applied |
Patient (P) | Patients diagnosed with a rare disease eligible for treatment with ATMPs | Patients without a rare disease, or not eligible for ATMP treatment |
Intervention (I) | Subjects treated with any ATMPs authorized by the EMA with an orphan designation. (withdrawn or not-renowned marketing authorizations ATMPs were not considered eligible) (Supplementary Material: Table S2) | Patients treated with withdrawn, non-renewed, or non-orphan-designated ATMPs |
Comparator (C) | Standard of care (SoC), best supportive care (BSC), and other therapies | Studies with inappropriate or unreported comparators |
Outcomes (O) | Cost-effectiveness profile, cost and quality-adjusted life years gained, incremental cost-effectiveness ratio (ICER), and incremental cost-utility ratio (ICUR) | Studies not reporting relevant economic outcomes |
Study design (S) | Economic evaluation, cost-effectiveness analysis, and cost-utility analysis | Non-economic study types |
International Nonproprietary Name | Therapeutic Indications * | ICER Mean | Incremental QALY Mean (SD) | References |
---|---|---|---|---|
Axicabtagene ciloleucel | Diffuse large B-cell lymphoma (DLBCL) (87%) Folicular lymphoma (FL) (13%) | EUR 59,183/QALY | 2.43 (1.33) | [19,21,22,23,24,25,26,27,28,29,30,31,32,33,34,35,36,38,39,40,41] |
Tisagenlecleucel | Acute lymphoblastic leukaemia (ALL) (60%) Diffuse large B-cell lymphoma (DLBCL) (40%) | EUR 55,013/QALY | 5.64 (2.9) | [23,30,43,44,45,46,47,48,49,61,63,64,65,67,69,70,71,72,78,79] |
Onasemnogene abeparvovec | Spinal muscular atrophy (SMA) | EUR 89,566/QALY | 13.23 (9.50) | [62,68,77] |
Voretigene neparvovec | Biallelic RPE65-mediated inherited retinal disease | EUR 115,572/QALY | 5.98 (1.02) | [59,60,73] |
Brexucabtagene autoleucel | Mantle cell lymphoma (MCL) (80%) Acute lymphoblastic leukemia (ALL) (20%) | EUR 53,774/QALY | 4.40 (1.48) | [50,51,52,53,54] |
Ciltacabtagene autoleucel | Multiple myeloma (MM) | EUR 58,511/QALY | 3.07 (0.18) | [55,56,57] |
Idecabtagene vicleucel | Multiple myeloma (MM) | EUR 138,877/QALY | 1.59 (0.58) | [55,56,57,74] |
Lisocabtagene maraleucel | Diffuse large B-cell lymphoma (DLBCL) | EUR 48,881/QALY | 1.88 (0.21) | [30,75] |
Etranacogene dezaparvovec | Hemophilia B | EUR −1,937,936/QALY | 0.60 | [42] |
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© 2025 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
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Serino, M.; Krstin, M.; Mucherino, S.; Menditto, E.; Orlando, V. Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review. Healthcare 2025, 13, 1894. https://doi.org/10.3390/healthcare13151894
Serino M, Krstin M, Mucherino S, Menditto E, Orlando V. Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review. Healthcare. 2025; 13(15):1894. https://doi.org/10.3390/healthcare13151894
Chicago/Turabian StyleSerino, Marianna, Milana Krstin, Sara Mucherino, Enrica Menditto, and Valentina Orlando. 2025. "Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review" Healthcare 13, no. 15: 1894. https://doi.org/10.3390/healthcare13151894
APA StyleSerino, M., Krstin, M., Mucherino, S., Menditto, E., & Orlando, V. (2025). Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review. Healthcare, 13(15), 1894. https://doi.org/10.3390/healthcare13151894