{"title":"生物技术创新成本：探索细胞和基因治疗的研发成本。","authors":"Marco T Sabatini, Mark Chalmers","doi":"10.1007/s40290-023-00480-0","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Clinical development paradigms for cell and gene therapies appear to be different to those of more conventional treatments: therefore, it is informative to explore this from the perspective of investments required to bring a new cell and/or gene therapy to the market. While there are a number of studies in the literature analyzing clinical-stage R&D costs for novel therapeutics, these are 'modality-agnostic' and thus do not elucidate costs specifically for the emerging class of cell and gene therapies.</p><p><strong>Objectives: </strong>The objective of this study was to understand the research and development (R&D) costs associated with the clinical development of new cell and gene therapy assets METHODS: As part of our analysis of clinical-stage R&D costs for cell and gene therapies, we focused our efforts on cell and gene therapy assets recently approved by the US Food and Drug Administration (FDA) or expected to receive FDA approval by the end of 2024. A total of 25 therapies were identified for the study, 11 of which had sufficient level of detail for our clinical-stage R&D costing study. We calculated the clinical-stage R&D costs to bring a new cell and/or gene therapy to the market following a three-step approach, starting with (1) calculation of the out-of-pocket investment reported in US SEC reports; (2) we adjusted these figures for the risk of failure by applying a clinical trial phase-dependent attrition risk rate; (3) we accounted for the cost of capital of 10.5%.</p><p><strong>Results: </strong>After accounting for R&D attrition rate (i.e., costs of failed programs) and applying a cost of capital at 10.5%, we estimate that the clinical-stage R&D investment required to bring a new cell and/or gene therapy to market is US$1943 M (95% CI US$1395 M, US$2490 M).</p><p><strong>Conclusion: </strong>This knowledge can inform financial planning for biopharma companies looking to enter the space and inform policy makers within the context of the commercialization and pricing of such therapies.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":null,"pages":null},"PeriodicalIF":3.1000,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"1","resultStr":"{\"title\":\"The Cost of Biotech Innovation: Exploring Research and Development Costs of Cell and Gene Therapies.\",\"authors\":\"Marco T Sabatini, Mark Chalmers\",\"doi\":\"10.1007/s40290-023-00480-0\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Clinical development paradigms for cell and gene therapies appear to be different to those of more conventional treatments: therefore, it is informative to explore this from the perspective of investments required to bring a new cell and/or gene therapy to the market. While there are a number of studies in the literature analyzing clinical-stage R&D costs for novel therapeutics, these are 'modality-agnostic' and thus do not elucidate costs specifically for the emerging class of cell and gene therapies.</p><p><strong>Objectives: </strong>The objective of this study was to understand the research and development (R&D) costs associated with the clinical development of new cell and gene therapy assets METHODS: As part of our analysis of clinical-stage R&D costs for cell and gene therapies, we focused our efforts on cell and gene therapy assets recently approved by the US Food and Drug Administration (FDA) or expected to receive FDA approval by the end of 2024. A total of 25 therapies were identified for the study, 11 of which had sufficient level of detail for our clinical-stage R&D costing study. We calculated the clinical-stage R&D costs to bring a new cell and/or gene therapy to the market following a three-step approach, starting with (1) calculation of the out-of-pocket investment reported in US SEC reports; (2) we adjusted these figures for the risk of failure by applying a clinical trial phase-dependent attrition risk rate; (3) we accounted for the cost of capital of 10.5%.</p><p><strong>Results: </strong>After accounting for R&D attrition rate (i.e., costs of failed programs) and applying a cost of capital at 10.5%, we estimate that the clinical-stage R&D investment required to bring a new cell and/or gene therapy to market is US$1943 M (95% CI US$1395 M, US$2490 M).</p><p><strong>Conclusion: </strong>This knowledge can inform financial planning for biopharma companies looking to enter the space and inform policy makers within the context of the commercialization and pricing of such therapies.</p>\",\"PeriodicalId\":19778,\"journal\":{\"name\":\"Pharmaceutical Medicine\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":3.1000,\"publicationDate\":\"2023-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pharmaceutical Medicine\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1007/s40290-023-00480-0\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2023/6/7 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q2\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pharmaceutical Medicine","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1007/s40290-023-00480-0","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2023/6/7 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
The Cost of Biotech Innovation: Exploring Research and Development Costs of Cell and Gene Therapies.
Background: Clinical development paradigms for cell and gene therapies appear to be different to those of more conventional treatments: therefore, it is informative to explore this from the perspective of investments required to bring a new cell and/or gene therapy to the market. While there are a number of studies in the literature analyzing clinical-stage R&D costs for novel therapeutics, these are 'modality-agnostic' and thus do not elucidate costs specifically for the emerging class of cell and gene therapies.
Objectives: The objective of this study was to understand the research and development (R&D) costs associated with the clinical development of new cell and gene therapy assets METHODS: As part of our analysis of clinical-stage R&D costs for cell and gene therapies, we focused our efforts on cell and gene therapy assets recently approved by the US Food and Drug Administration (FDA) or expected to receive FDA approval by the end of 2024. A total of 25 therapies were identified for the study, 11 of which had sufficient level of detail for our clinical-stage R&D costing study. We calculated the clinical-stage R&D costs to bring a new cell and/or gene therapy to the market following a three-step approach, starting with (1) calculation of the out-of-pocket investment reported in US SEC reports; (2) we adjusted these figures for the risk of failure by applying a clinical trial phase-dependent attrition risk rate; (3) we accounted for the cost of capital of 10.5%.
Results: After accounting for R&D attrition rate (i.e., costs of failed programs) and applying a cost of capital at 10.5%, we estimate that the clinical-stage R&D investment required to bring a new cell and/or gene therapy to market is US$1943 M (95% CI US$1395 M, US$2490 M).
Conclusion: This knowledge can inform financial planning for biopharma companies looking to enter the space and inform policy makers within the context of the commercialization and pricing of such therapies.
期刊介绍：
Pharmaceutical Medicine is a specialist discipline concerned with medical aspects of the discovery, development, evaluation, registration, regulation, monitoring, marketing, distribution and pricing of medicines, drug-device and drug-diagnostic combinations. The Journal disseminates information to support the community of professionals working in these highly inter-related functions. Key areas include translational medicine, clinical trial design, pharmacovigilance, clinical toxicology, drug regulation, clinical pharmacology, biostatistics and pharmacoeconomics. The Journal includes:Overviews of contentious or emerging issues.Comprehensive narrative reviews that provide an authoritative source of information on topical issues.Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by PRISMA statement.Original research articles reporting the results of well-designed studies with a strong link to wider areas of clinical research.Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pharmaceutical Medicine may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.
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