Conference Coverage

Pediatric indications appear in one-third of orphan drug approvals



– Pediatric indications appeared in approximately 36% of orphan drug approvals between 2000 and 2017, according to data from the Food and Drug Administration.

“Given the impact of rare disease on children, it is of particular interest to better understand where new drug approvals and advances are occurring, through the lens of pediatrics,” said Kathleen L. Miller, Ph.D., and Michael Lanthier of the Food and Drug Administration in Silver Spring, Md. They presented their findings in a poster at the NORD Rare Summit, held by the National Organization for Rare Disorders.

Overall, 31% of 314 orphan drug approvals by the FDA between 2000 and 2017 had a pediatric and adult indication, 5% had pediatric-only indications, and 64% had adult-only indications.

For the 112 pediatric indication approvals, 14% were for inherited blood disorders, 14% for inherited metabolic disorders, 13% for rare cancers, 10% for antidotes and medical countermeasures, 9% for infectious diseases, 8% for auto-inflammatory diseases, 7% for neurologic disorders, and 25% for other conditions.

Approximately 63% of the total orphan drug approvals during the study period were for rare cancers or genetic disorders (138 approvals and 59 approvals, respectively). Although 90% of the rare cancer approvals were for adults only, 84% of genetic disorder drug approvals had pediatric indications, Dr. Miller and Mr. Lanthier noted.

When analyzed by submission type, pediatric indications were included in 52% of new orphan formulations, 35% of orphan secondary indication approvals, and 32% of new molecular entity approvals.

Although more research is needed, the results suggest that “pediatric indications represent a sizable proportion of orphan drug approvals in both a range of therapeutic areas and in a range of approval types,” the investigators concluded.

The researchers are employed by the FDA, which sponsored the study. They had no financial conflicts to disclose.

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