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. 2023 Aug 1;18(8):1101-1107.
doi: 10.2215/CJN.0000000000000167. Epub 2023 Apr 13.

Overcoming Barriers to Drug Development in Children with CKD

Mona Khurana  1 , Gunter F Egger  2 , Lynne Yao  1 , Aliza Thompson  3 , Chrissi Pallidis  2 , Stuart L Goldstein  4 , Benjamin L Laskin  5 , Shamir Tuchman  1 , Meaghan A Malley  6 , Katrin Uhlig  7   8
Affiliations

Overcoming Barriers to Drug Development in Children with CKD

Mona Khurana et al. Clin J Am Soc Nephrol. .

Abstract

Like all sick children, children with CKD need access to safe and effective medicines that have been formulated and examined specifically for them. Despite legislation in the United States and the European Union that either mandates or incentivizes programs for children, conducting trials to advance the treatment of children continues to prove to be a challenge for drug developers. This is also the case for drug development in children with CKD, where trials face challenges in recruitment and completion and where there remains a substantial time lag between initial approval of a medicinal product for use in adults and completion of studies that result in the addition of pediatric-specific labeling for the same indication. The Kidney Health Initiative commissioned a workgroup of diverse stakeholders ( https://khi.asn-online.org/projects/project.aspx?ID=61 ), including participants from the Food and Drug Administration and the European Medicines Agency, to think carefully through the challenges in drug development for children with CKD and how to overcome them. This article provides an overview of the regulatory frameworks in the United States and the European Union that govern pediatric drug development, the current landscape of drug development and approval for children with CKD, the challenges in conduct and execution of these drug trials, and the progress that has been made to facilitate drug development for children with CKD.

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Conflict of interest statement

G.F. Egger and C. Pallidis report employment with the European Medicines Agency. S.L. Goldstein reports consultancy for Akebia, Baxter Healthcare, Bayer, Bioporto, Inc., BioProducts Labs, Fresenius, Kaneka, Inc, Leadiant, MediBeacon, Medtronic, NuWellis, Otsuka, Reata, and Renibus; ownership interest in MediBeacon, Inc; research funding from Baxter Healthcare, Bioporto, ExThera, Medtronic, and NuWellis; honoraria from Baxter Healthcare and Fresenius; patents or royalties from Vigilanz; an advisory or leadership role for MediBeacon; and speakers bureau for Baxter Healthcare and Fresenius. B.L. Laskin reports ownership interest in Acorda Therapeutics, AT&T, Charter Communications, Comcast, DHT Holdings, Duke Energy, Electra Battery Materials, Ford Motor, Happiness Development, Johnson Controls, Medtronic, Nu Holdings, TE Connectivity, Verizon, and Warner Brothers Discovery; research funding from Viracor-Eurofins; research sample testing free of charge; and patent: Compositions and Methods for Treatment of HSCT-Associated Thrombotic Microangiopathy (US Patent Number PCT/US2014/055922, 2014). M.A. Malley reports employment with Travere Therapeutics and ownership interest in Travere Therapeutics and Altimmune. A. Thompson and L. Yao report employment with the US Food and Drug Administration. S. Tuchman reports ownership interest in Apple, American Electric Power, Boeing, BP, and Disney. S. Tuchman's spouse reports employment with the Eating Recovery Center. K. Uhlig reports employment with Agios Pharmaceuticals, ownership interest in Agios Pharmaceuticals, and an advisory or leadership role for Kidney Health Initiative Board of Directors. K. Uhlig's spouse reports consultancy for S2N Health. All remaining authors have nothing to disclose.

Figures

Figure 1
Figure 1
Increase of pediatric label changes in the United States and the European Union. (A) Number of pediatric labeling changes that occurred pursuant to PREA, BPCA, and the Pediatric Rule from 1998 to 2021. Figure reprinted from ref. . (B) EMA marketing authorizations of medicines with pediatric indication. After the EU Paediatric Regulation entered into force in 2007, the number of new medicines with a pediatric indication at the time of first marketing authorization plus the number of new pediatric indications added to authorized medicines more than doubled from 30 of 217 approvals (14%) in 2004–2006 to 74 of 295 approvals (25%) in 2014–2016. BPCA, Best Pharmaceuticals for Children Act; EMA, European Medicines Agency; EU, European Union; PREA Pediatric Research Equity Act.
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References

    1. Archdeacon P, Shaffer RN, Winkelmayer WC, Falk RJ, Roy-Chaudhury P. Fostering innovation, advancing patient safety: the kidney health initiative. Clin J Am Soc Nephrol. 2013;8(9):1609–1617. doi:10.2215/CJN.01140113 - DOI - PMC - PubMed
    1. Bhatti S, Sanders C. Paediatric Regulations: US and EU: Similar but Different. European Pharmaceutical Contractor, Spring 2011. Accessed February 20, 2023. http://www.samedanltd.com/magazine/11/issue/149/article/2893
    1. Penkov D, Tomasi P, Eichler I, Murphy D, Yao LP, Temeck J. Pediatric medicine development: an overview and comparison of regulatory processes in the European Union and United States. Ther Innov Regul Sci. 2017;51(3):360–371. doi:10.1177/2168479017696265 - DOI - PMC - PubMed
    1. Bourgeois FT, Kesselheim AS. Promoting pediatric drug research and labeling—outcomes of legislation. N Engl J Med. 2019;381(9):875–881. doi:10.1056/NEJMhle1901265 - DOI - PubMed
    1. Best Pharmaceuticals for Children Act, Pub. L. 107-109. (2002). Reauthorized in 2007 by Pub. L. 110-85 (Food and Drug Administration Amendments Act), and Permanently Reauthorized in 2012 by Pub. L. 112-114 (Food and Drug Administration Safety and Innovation Act).

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