Module232025

12/05/2025

Analysis and Outcome under US Orphan Drug Act (2012–2018) of Orphan Products which Included Combined Adult and Paediatric Trials Simultaneous Drug Approvals for both Populations

In the analysis, a total of 45 drug and biological products were approved from 2012 to 2018 under the ODA, utilizing a total of 102 combined paediatric and adult trials. Of this group of OD trials, 54% (55/102) were for biological products and 46% (47/102) were for drug products

Drug Products: When evaluating the drug approvals by the number of drug products, hematology 20% (4/20) was the most common therapeutic area All of drug products were approved simultaneously for paediatric use for the population studied along with the adult drug approval

Biological Products: Most orphan approvals for biological products were hematology studies 44% (24/55) as the major therapeutic area, follow by oncology 18% (10/55) All biological products received approval for adults and the paediatric patients included in the combined trials at the time

Combined Pediatric and Adult Trials Submitted to the US Food and Drug Administration 2012–2018 I. Tanaudommongkon et al., Clin Pharmacol Ther. 2020 November ; 108(5): 1018–1025

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M Dehlinger-Kremer, 14 May 2025

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Combined Adult/Adolescent Trials So Far

A review of clinicaltrials.gov in February 2021* revealed that

Adolescents are eligible to enroll alongside adults in 23% (43/185) of phase 3 interventional trials involving conditions such as asthma, atopic dermatitis, hypertension, inflammatory bowel disease, or SARS-CoV-2 infection.

In certain indications, the majority of phase 3 trials are enrolling adolescents with adults 62% (13/21) for asthma and 52% (11/21) for atopic dermatitis .

*G. Noel et al., Inclusion of Adolescents in Adult Clinical Trials: Report of the Institute for Advanced Clinical Trials for Children’s Pediatric Innovation Research Forum: Therapeutic Innovation & Regulatory Science (Apr. 2021) 55:773–778

The Organisation for Professionals in Regulatory Affairs

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M Dehlinger-Kremer, 14 May 2025

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