Letter to FDA on the Cost to the Public of the Pediatric Trial Exclusivity Extension

Today, Knowledge Ecology International (KEI) submitted a letter to the Commissioner of the Food and Drug Administration (FDA) regarding the mismatch between the cost to perform pediatric extension trials and the cost to the public of the six month extension of the drug monopoly.

Under Section 505A of the Federal Food, Drug, and Cosmetic Act, the FDA can request pediatric studies on a drug, and with that request, grant a six month extension of its patent and regulatory monopolies. This extension is granted regardless of the outcome of the studies – succeed or fail, the company gets this extended exclusivity period. Additionally, though the FDA must request the studies, companies can and do regularly lobby the FDA to issue a request.

KEI’s letter to the FDA notes that “While the benefits of the US Food and Drug Administration’s pediatric exclusivity program in terms of new safety and efficacy data in pediatric populations cannot be denied, the costs are significant, and in some cases so large as to call into question the appropriateness of using the pediatric extension as an off-budget means to fund the studies.”

In the letter KEI calls on the FDA to coordinate and manage the pediatric study extension with a focus on getting the data it seeks in a more cost effective manner, though assessing the costs of the trials, the costs of the extension to the public, or whether the public interest would be better served through public funding of the studies.

KEI’s letter includes a table which summarizes recent FDA requests for pediatric studies, Medicare and Medicaid outlays, and sizes of enrollment for these studies, illustrating the considerable variances in the potential costs to run the studies, and costs of the continued exclusivities for the public (in the form of high prices).

KEI’s letter to the FDA Commissioner is available here: KEI-Letter-FDA-PED-Extensions-20Sept2023