Changing incentives to ACCELERATE drug development for paediatric cancer

Teresa de Rojas (1) , Pamela Kearns (2) , Patricia Blanc (3) , Jeffrey Skolnik (4) , Elizabeth Fox (5) , Leona Knox (6) , Raphael Rousseau (7) , François Doz (8) , Nick Bird (6) , Andrew Pearson (9) , Gilles Vassal (10, 11)

1 ACCELERATE
2 University Hospitals Birmingham [Birmingham, Royaume-Uni]
3 Imagine for Margo - Children Without Cancer
4 INOVIO Pharmaceuticals
5 St Jude Children's Research Hospital
6 Solving Kids' Cancer UK
7 LGC - Laboratoire de Génie Chimique
8 Institut Curie [Paris]
9 ENVIRONMENTAL RESEARCH INSTITUTE THE UNIVERSITY OF WAIKATO HAMILTON NZL
10 PMNCO - Prédicteurs moléculaires et nouvelles cibles en oncologie
11 Département de cancérologie de l'enfant et de l'adolescent [Gustave Roussy]

Abstract Background More effective incentives are needed to motivate paediatric oncology drug development, uncoupling it from dependency on adult drug development. Although the current European and North‐American legislations aim to promote drug development for paediatrics and rare diseases, children and adolescents with cancer have not benefited as expected from these initiatives and cancer remains the first cause of death by disease in children older than one. Drug development for childhood cancer remains dependent on adult cancer indications and their potential market. The balance between the investment needed to execute a Paediatric Investigation Plan (PIP) in Europe and an initial Paediatric Study Plan (iPSP) in the US, coupled with the potential financial reward has not been sufficiently attractive to incite the pharmaceutical industry to develop drugs for rare indications such as childhood cancer. Methods We propose changes in the timing and nature of the rewards within the European Paediatric Medicine Regulation (PMR) and Regulation on Orphan Medicinal Products (both currently under review), which would drive earlier initiation of paediatric oncology studies and provide incentives for drug development specifically for childhood indications. Results We suggest modifying the PMR to ensure mechanism‐of‐action driven mandatory PIP and reorganization of incentives to a stepwise and incremental approach. Interim and final deliverables should be defined within a PIP or iPSP, each attracting a reward on completion. A crucial change would be the introduction of the interim deliverable requiring production of paediatric data that inform the go/no‐go decisions on whether to take a drug forward to paediatric efficacy trials. Conclusion Additionally, to address the critical gap in the current framework where there is a complete lack of incentives to promote paediatric‐specific cancer drug development, we propose the introduction of early rewards in the Orphan Regulation, with a variant on the US‐Creating Hope Act and its priority review vouchers.

Mots clés: Drug development; Incentives; Paediatric oncology; Paediatric regulation; Supplementary protection certificate

Domaines: Sciences du Vivant [q-bio]

Teresa de Rojas, Pamela Kearns, Patricia Blanc, Jeffrey Skolnik, Elizabeth Fox, et al.. Changing incentives to ACCELERATE drug development for paediatric cancer. Cancer Medicine, 2023, 12 (7), pp.8825-8837. ⟨10.1002/cam4.5627⟩. ⟨hal-04493098⟩