OBJECTIVES To evaluate regulatory decisions and health technology assessments (HTA) in Australia, Canada, and England of new drugs approved by the US Food and Drug Administration (FDA) in 2017-2020 and estimate the US treatment cost per drug.

METHODS Information on new drugs approved by the FDA in 2017-2020 was extracted from Drugs@FDA and analyzed against regulatory decisions in Australia, Canada, and England. Reimbursement recommendations were collected from the Australian Pharmaceutical Benefits Advisory Committee (PBAC), the Canadian Agency for Drugs and Technologies in Health (CADTH), and the United Kingdom National Institute for Health and Care Excellence (NICE). For drugs not recommended by an international regulatory or HTA agency, US treatment costs per year were estimated from FDA product labeling and Wholesale Acquisition Cost (WAC) listed in Redbook (IBM Micromedex). Regulatory information, HTA reports, and WAC were current as of July 2021. Descriptive statistics were conducted to assess approval concordance, reasons for negative HTA recommendations, and characteristics of drugs receiving negative assessments.

RESULTS The FDA approved 206 new drugs in 2017-2020, of which 72% were granted marketing authorization by at least one other regulatory agency at a median delay of 10.1 months following US approval. Conversely, 4 drugs (abaloparatide, betrixaban, emapalumab, pexidartinib) were refused marketing authorization due to unfavorable benefit-to-risk assessments. 40% of FDA-approved drugs evaluated by PBAC, CADTH, or NICE received negative reimbursement recommendations due to uncertainty of clinical benefit and/or unacceptably high price. Approximately half of the drugs were for oncology indications and most were approved by the FDA through expedited review pathways and/or granted Orphan Drug designation. The average US treatment cost of new FDA drug approvals receiving negative appraisals from international agencies was $150,817/year.

CONCLUSIONS Review of drug approvals and HTA agency recommendations in other countries can provide evidence to support clinical decision-making of new drugs by US health systems and payers.