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Pediatric Drugs, Accelerated Approval, and Prospects for Reform

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References

  1. U.S. Food and Drug Administration. Accelerated approval. 2023. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/accelerated-approval. Accessed 10 July 2023.

  2. U.S. Food and Drug Administration CDER Accelerated Approval Program Database. 2023. https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approval-program. Accessed 10 July 2023.

  3. Sachs RE, Donohue JM, Dusetzina SB. Accelerated approval—taking the FDA’s concerns seriously. N Engl J Med. 2022;387(3):199–201. https://doi.org/10.1056/NEJMp2204487.

    Article  PubMed  Google Scholar 

  4. U.S. Food and Drug Administration. FDA grants accelerated approval to first drug for Duchenne muscular dystrophy. FDA; FDA. 2020. https://www.fda.gov/news-events/press-announcements/fda-grants-accelerated-approval-first-drug-duchenne-muscular-dystrophy. Accessed 10 July 2023.

  5. U.S. Food and Drug Administration. FDA Commissioner and Chief Scientist announce decision to withdraw approval of Makena. 2023. https://www.fda.gov/news-events/press-announcements/fda-commissioner-and-chief-scientist-announce-decision-withdraw-approval-makena. Accessed 10 July 2023.

  6. Lim KRQ, Maruyama R, Yokota T. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Dev Ther. 2017;11:533–45. https://doi.org/10.2147/DDDT.S97635.

    Article  CAS  Google Scholar 

  7. Sarepta Therapeutics, Inc. 2023. https://investorrelations.sarepta.com/sec-filings. Accessed 10 July 2023.

  8. Drug Companies Are Minting Billions on Unproven Treatments With FDA Shortcut. Bloomberg.com. 2023. https://www.bloomberg.com/news/articles/2023-05-14/fda-fast-tracked-drugs-make-companies-billions-on-unproven-claims. Accessed 7 Sept 2023.

  9. Sarepta Therapeutics, Inc. A randomized, double-blind, dose finding and comparison study of the safety and efficacy of a high dose of eteplirsen, preceded by an open-label dose escalation, in patients with Duchenne muscular dystrophy with deletion mutations amenable to exon 51 skipping (Clinical Trial Registration No. NCT03992430). clinicaltrials.gov. 2023. https://clinicaltrials.gov/ct2/show/NCT03992430. Accessed 10 July 2023.

  10. Ballreich J, Socal M, Bennett CL, Schoen MW, Trujillo A, Xuan A, Anderson G. Medicare spending on drugs with accelerated approval. Ann Intern Med. 2022;175(7):938–44. https://doi.org/10.7326/M21-4442.

    Article  PubMed  Google Scholar 

  11. Centers for Medicare & Medicaid Services. 2023. https://www.cms.gov/newsroom/press-releases/hhs-secretary-responds-presidents-executive-order-drug-prices. Retrieved 6 June 2023.

  12. Reardon S. ‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work? Nature. 2023. https://doi.org/10.1038/d41586-023-01799-z.

    Article  PubMed  PubMed Central  Google Scholar 

  13. Teemkin E, Trin J, King, Spaling LLLP. FDA’S accelerated approval pathway: a rare disease perspective. https://rarediseases.org/?s=accelerated+approval. Retrieved 8 Sept 2023.

  14. Cooley LP. FDORA changes to the FDA accelerated approval program. 2023. https://www.cooley.com/news/insight/2023/2023-01-31-fdora-changes-to-the-fda-accelerated-approval-program#:~:text=Enacted%20on%20December%2029%2C%202022,authority%20in%20several%20key%20areas. Accessed 7 Sept 2023.

  15. Lupkin, S. FDA has new leverage over companies looking for a quicker drug approval. NPR. 2023. https://www.npr.org/sections/health-shots/2023/03/03/1160702899/fda-enforcement-drug-approval-manufacturer-promises. Accessed 7 Sept 2023.

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Funding

This work was supported in part by Arnold Ventures. The funder had no role in formulation of the research question, study design, study conduct, interpretation, manuscript development, or the decision to submit the manuscript for publication.

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  1. Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, 624 N. Broadway, Baltimore, MD, 21205, USA

    Jeromie Ballreich, Hyung-Seok Kim & Mariana Socal

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  1. Jeromie Ballreich
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  2. Hyung-Seok Kim
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  3. Mariana Socal
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Correspondence to Jeromie Ballreich.

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Ballreich, J., Kim, HS. & Socal, M. Pediatric Drugs, Accelerated Approval, and Prospects for Reform. Pediatr Drugs 26, 5–8 (2024). https://doi.org/10.1007/s40272-023-00597-4

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