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The Cost of Biotech Innovation: Exploring Research and Development Costs of Cell and Gene Therapies

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Abstract

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.

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References

  1. Bak A, Friis KP, Wu Y. Translating cell and gene biopharmaceutical products for health and market impact. Product scaling from clinical to marketplace: lessons learned and future outlook. J Pharm Sci. 2019;108(10):3169–75. https://doi.org/10.1016/j.xphs.2019.05.027.

    Article  CAS  PubMed  Google Scholar 

  2. Citeline Informa. A total of 2602 trials were listed, of which 985 in Ph I, 683 in Ph II, 557 in Ph I/II, 140 in Ph III, 68 in Ph II/III, 129 as Ph IV and 20 as N/A or other. The following therapeutic classes were included in the analysis, with trial status as “open”: “Gene therapy; Gene delivery vector; Cellular therapy, chimaeric antigen receptor; Cellular therapy, tumor-infiltrating lymphocyte; Cellular therapy, T cell receptor; Cellular therapy, stem cell; Cellular therapy, other”; www.Informa.com. Accessed 8 Oct 2022.

  3. ASCGT Clinical Trials Finder. A total of 2556 trials were listed, of which 2268 were “open”, 271 “not yet recruiting” and 17 “active, not recruiting”. https://asgct.careboxhealth.com/. Accessed 8 Oct 2022.

  4. FDA. LUXTURNA (voretigene neparvovec-rzyl). https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna. Accessed 8 Oct 2022.

  5. FDA. ZOLGENSMA (onasemnogene abeparvovec). https://www.fda.gov/vaccines-blood-biologics/zolgensma . Accessed 8 Oct 2022.

  6. FDA. KYMRIAH (Tisagenlecleucel). https://www.fda.gov/biologicsbloodvaccines/cellulargenetherapyproducts/approvedproducts/ucm573706.htm. Accessed 8 Oct 2022.

  7. FDA. YESCARTA. FDA approves axicabtagene ciloleucel for large B-cell lymphoma. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/yescarta-axicabtagene-ciloleucel. Accessed 8 Oct 2022.

  8. Ned Pagliarulo, Cell, gene therapy company funding reaches new heights, despite setbacks; BiopharmaDive; https://www.biopharmadive.com/news/cell-gene-therapy-record-funding-investment-ipo/605261/. Accessed 8 Oct 2022.

  9. DiMasi JA, Hansen RW, Grabowski HG, Lasagna L. Cost of innovation in the pharmaceutical industry. J Health Econ. 1991;10(2):107–42. https://doi.org/10.1016/0167-6296(91)90001-4. (PMID: 10113009).

    Article  CAS  PubMed  Google Scholar 

  10. DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: new estimates of drug development costs. J Health Econ. 2003;22(2):151–85. https://doi.org/10.1016/S0167-6296(02)00126-1.

    Article  PubMed  Google Scholar 

  11. DiMasi JA, Grabowski HG, Vernon J. R&D costs and returns by therapeutic category. Drug Inf J. 2004;38(3):211–23. https://doi.org/10.1177/009286150403800301.

    Article  Google Scholar 

  12. DiMasi JA, Grabowski HG. Economics of new oncology drug development. J Clin Oncol. 2007;25(2):209–16. https://doi.org/10.1200/JCO.2006.09.0803. (PMID: 17210942).

    Article  PubMed  Google Scholar 

  13. Vernon JA, Golec JH, Dimasi JA. Drug development costs when financial risk is measured using the Fama-French three-factor model. Health Econ. 2010;19(8):1002–5. https://doi.org/10.1002/hec.1538. (PMID: 19655335).

    Article  PubMed  Google Scholar 

  14. Mestre-Ferrandiz J, Sussex J, Towse A. The R&D cost of a new medicine. Office of Health Economics (OHE) research; 2012. (ISBN 978-1-899040-19-3).

    Google Scholar 

  15. DiMasi JA, Grabowski HG, Hansen RW. Innovation in the pharmaceutical industry: new estimates of R&D costs. J Health Econ. 2016;47:20–33. https://doi.org/10.1016/j.jhealeco.2016.01.012.

    Article  PubMed  Google Scholar 

  16. Prasad V, Mailankody S. Research and development spending to bring a single cancer drug to market and revenues after approval. JAMA Intern Med. 2017;177(11):1569–75. https://doi.org/10.1001/jamainternmed.2017.3601.

    Article  PubMed  PubMed Central  Google Scholar 

  17. Wouters OJ, McKee M, Luyten J. Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018. JAMA. 2020;323(9):844–853. https://doi.org/10.1001/jama.2020.1166

  18. Jayasundara K, Hollis A, Krahn M, Mamdani M, Hoch JS, Grootendorst P. Estimating the clinical cost of drug development for orphan versus non-orphan drugs. Orphanet J Rare Dis. 2019;14(1):12. https://doi.org/10.1186/s13023-018-0990-4. (PMID:30630499;PMCID:PMC6327525).

    Article  PubMed  PubMed Central  Google Scholar 

  19. U.S. Securities and Exchange commission (SEC); https://www.sec.gov/reports. Accessed 8 Oct 2022.

  20. FDA website, Approved Cellular and Gene Therapy Products; https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products. Accessed 8 Oct 2022.

  21. FDA website, FDA CDER 21st Century Review Process document. https://www.fda.gov/media/78941/download#:~:text=The%20timelines%20for%20NMEs%20and,of%20submission%20of%20the%20application. Accessed 15 Oct 2022.

  22. Chi Heem Wong. Kien Wei Siah, Andrew W Lo, Estimation of clinical trial success rates and related parameters. Biostatistics. 2019;20(2):273–86. https://doi.org/10.1093/biostatistics/kxx069.

    Article  PubMed  Google Scholar 

  23. Thomas DW, Burns J, Audette J, Carrol A, Dow-Hygelund C, Hay M. Clinical development success rates, 2006–2015. San Diego: Biomedtracker; 2016.

    Google Scholar 

  24. Hay M, Thomas DW, Craighead JL, Economides C, Rosenthal J. Clinical development success rates for investigational drugs. Nat Biotechnol. 2014;32:40–51.

    Article  CAS  PubMed  Google Scholar 

  25. Clinical Development Success Rates and Contributing Factors 2011–2020 Informa, https://pharmaintelligence.informa.com/~/media/informa-shop-window/pharma/2021/files/reports/2021-clinical-development-success-rates-2011-2020-v17.pdf. Accessed Jan 2023

  26. Brealey, Myers, Allen. Principles of corporate finance. McGraw Hill, 10th edition, Chapter 9 SBN 978-0-07-353073-4, MHID 0-07-353073-5

  27. Damodaran A. Damodaran Online. Retrieved 15th March 2023, from http://www.damodaran.com. Accessed Jan 2023

  28. Federal Drug Administration-Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug (IND) applications . https://www.fda.gov/regulatory-information/search-fda-guidance-documents/chemistry-manufacturing-and-control-cmc-information-human-gene-therapy-investigational-new-drug. Accessed Jan 2023

  29. Food and Drug Administration. News and Events. Remarks by Commissioner Gottlieb to the Alliance for Regenerative Medicine’s Annual Board Meeting. 2018. https://www.fda.gov/NewsEvents/Speeches/ucm608445.htm. Accessed Jan 2023

  30. GlobalData. Cancer T-Cell Therapy: safety and cost concerns. 2016. Retrieved from Research & Development Magazine: https://www.rdmag.com/news/2016/02/cancer-t-cell-therapy-safety-and-cost-concerns. Accessed Jan 2023

  31. Lopes, A, Sinclair, A, Frohlich, B. Cost analysis of cell therapy manufacture: autologous cell therapies, part 2. 2018. Retrieved from BioProcess International: https://bioprocessintl.com/manufacturing/cell-therapies/cost-analysis-of-cell-therapy-manufacturing-autologous-cell-therapies-part-2/. Accessed Jan 2023

  32. Food and Drug Administration. Considerations for the development of chimeric antigen receptor (CAR) T cell products. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-development-chimeric-antigen-receptor-car-t-cell-products. Accessed Jan 2023

  33. Food and Drug Administration. Human gene therapy products incorporating human genome editing. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-products-incorporating-human-genome-editing. Accessed Jan 2023

  34. RIEF-Regenxbio and Avexis Announce Expansion of Relationship For Spinal Muscular Atrophy treatments. U.S. https://www.reuters.com/article/brief-regenxbio-and-avexis-announce-expa-idUSFWN1P30PJ. Accessed 20 Mar 2023.

  35. BioMarin—transformative medicines through genomic and development expertise. (n.d.). BioMarin—transformative Medicines Through Genomic and Development Expertise. https://www.nature.com/articles/d43747-021-00166-z. Accessed 20 Mar 2023.

  36. Bluebird annual report 2021; https://investor.bluebirdbio.com/static-files/2aede37c-6058-4834-bcc1-6c17c8449fcd. Accessed 20 Mar 2023.

  37. American Society of Gene & Cell Therapy (ASGCT). Q3 2022 Quarterly Data Report. https://asgct.org/global/documents/asgct-citeline-q3-2022-report.aspx . Accessed 20 Mar 2023.

  38. Food and Drug Administration. Long Term Follow-Up After Administration of Human Gene Therapy Products; https://www.fda.gov/media/113768/download . Accessed 20 Mar 2023.

  39. Touchot N, Flume M. Early insights from commercialization of gene therapies in Europe. Genes (Basel). 2017;8:78. https://doi.org/10.3390/genes8020078.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  40. Sabatini MT, Xia T, Chalmers M. Pricing and market access challenges in the era of one-time administration cell and gene therapies. Pharmaceut Med. 2022;36(5):265–74. https://doi.org/10.1007/s40290-022-00443-x. (Epub 2022 Aug 22 PMID: 35994198).

    Article  PubMed  Google Scholar 

  41. Hanna E, Remuzat R, Auquier P. Gene therapies development: slow progress and promising prospect. J Mark Access Health Policy. 2017;5:1265293. https://doi.org/10.1080/20016689.2017.1265293.

    Article  PubMed  PubMed Central  Google Scholar 

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Authors and Affiliations

  1. EY, 1 More London Place, London, SE1 2AF, UK

    Marco T. Sabatini

  2. EY, Harcourt Centre, Harcourt Street, Dublin 2, Ireland

    Mark Chalmers

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Correspondence to Marco T. Sabatini.

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Conflicts of Interest

Authors are employees of E&Y and have been involved/exposed to a number of engagements for both public bodies (healthcare providers) and private bodies (pharma/biotech companies).

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Authors’ Contributions

MTS conceived the research, analysed the data, performed the literature search. MTS wrote the manuscript. MTS and MC critically revised the work. All authors have read and approve the final manuscript and agree to be accountable for this work.

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Sabatini, M.T., Chalmers, M. The Cost of Biotech Innovation: Exploring Research and Development Costs of Cell and Gene Therapies. Pharm Med 37, 365–375 (2023). https://doi.org/10.1007/s40290-023-00480-0

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