BACKGROUND Proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) are a novel class of medications for patients with familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease requiring additional lipid lowering beyond dietary measures and statin use. Because of the drugs' high cost, rates of prescription approval by payers may be low. We aimed to identify payer...

Background There is an active debate about the role that endpoints other than overall survival (OS) should play in the drug approval process. Yet the term 'surrogate endpoint' implies that OS is the only critical metric for regulatory approval of cancer treatments. We systematically analyzed the relationship between U.S. Food and Drug Administration (FDA) approval and publication of OS evidence...

Since the establishment of a specific approval pathway for similar biological medicinal products, so-called “biosimilars,” several biosimilars have been licensed and become available in the European Union (EU). However, despite a stringent approval process, acceptance of biosimilars in the medical community continues to be low. This appears to be especially true for therapeutic indications, for...

The perception that agencies are out of control arises from the fact that in being called on to make fundamental value judgments they have moved outside their accustomed sphere of activity, outside their expertise, and outside the established system of controls. This perturbation ofthe regulatory process will not be corrected until the agencies are relieved of the necessity of makingjudgments t...

In their cautious review, Shah and Shah [1] emphasized differences in regulations of personalized medicine (PM) among the three major authorities, the US Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. Specific points regarding the differences, however, were not raised for the drugs they selected for dis...

BACKGROUND To save costs, the Australian Government recently deferred approval of seven new medicines recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) for up to 7 months. OBJECTIVES The aim of this research is to examine the timelines of PBAC applications following approval by the Therapeutic Goods Administration (TGA), allowing the recent Cabinet delays to be considered i...

This study analyzes the innovativeness, clinical evidence, and efficacy of new drug indications with US Food Drug Administration (FDA) approval for breast, ovarian, endometrial, cervical cancer. We identified 26 drugs FDA in 55 gynecological cancer from 2000 to 2021. For each indication, we collected drug, trial characteristics labels. Hazard ratios (HRs) overall survival (OS), progression-free...