Inclusion of Infants and Neonates in Pediatric Orphan Product Approvals.

The Orphan Drug Act (ODA) of 1983 was enacted to provide financial incentives to drug sponsors to develop therapies for rare diseases. Although this act increased the number of orphan products approved, there are still a limited number of products available for the pediatric population because orphan drug products are exempt from the Pediatric Research Equity Act. The objectives of this study were (i) to evaluate the pediatric orphan drug studies submitted to the US Food and Drug Administration (FDA) in the period of 2007-2018 and (ii) to examine whether orphan drug products were fully labeled with a pediatric indication in infants and neonates.

Analysis of the Real-Time Oncology Review (RTOR) Pilot Program for Approvals of New Molecular Entities.

The United States Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) established the Real-Time Oncology Review (RTOR) pilot program in 2017 to streamline the review process for oncology drug applications with the applicant and the Agency agreeing upon a piecemeal strategy and timeline for module components. The Prescription Drug User Fee Act (PDUFA) review clock does not officially start until the final component is submitted. Participation requires careful planning of time and resources due to the multiple submissions and interactions with the FDA.

Appropriateness of thrombophilia testing in patients in the acute care setting and an evaluation of the associated costs.

Thrombophilia testing is rarely recommended in acute care settings due to the high likelihood of false-positive and false-negative results. Inappropriately performing these tests in the acute care setting is associated with inaccurate interpretation and an increased economic burden. In this retrospective analysis, the appropriateness of thrombophilia tests ordered for patients in an acute care setting was evaluated in terms of both clinical utility and economic costs.