Medications approved in the U.S. but not in other countries should be examined closely by health professionals and health systems before use, according to researchers who analyzed new drug approvals from 2017 through 2020. “More than one-fifth of the new drugs approved by the FDA in 2017 through 2020 were either refused marketing authorization or not recommended for reimbursement in Australia, Canada, or the UK as of May 2022 due to unfavorable benefit-to-risk profiles, uncertain clinical benefit, or unacceptably high price,” the authors conclude. “Ongoing review of international regulatory authorization refusals and formulary recommendations can support safe and cost-effective clinical decision-making by US health systems and payers.”
The U.S. is unique among the large democratic states in its lack of “public health technology assessment (HTA) strategies in response to increasing drug prices to determine the value of new treatments by conducting comparative clinical and economic analyses,” write the authors. A cross-sectional study of recommendations issued by the drug authorities in the U.S., Australia, Canada, and the U.K. from 2017 through 2020 looked at decisions and HTAs. Reasons for negative recommendations were analyzed, and costs were assessed for drugs available only in the U.S.
“The FDA approved 206 new drugs in 2017 through 2020, of which 162 (78.6%) were granted marketing authorization by at least 1 other regulatory agency at a median (IQR) delay of 12.1 (17.7) months following US approval,” the authors write. “Conversely, 5 FDA-approved drugs were refused marketing authorization by an international regulatory agency due to unfavorable benefit-to-risk assessments. An additional 42 FDA-approved drugs received negative reimbursement recommendations from HTA agencies in Australia, Canada, or the UK due to uncertainty of clinical benefits or unacceptably high prices. The median (IQR) US cost of the 47 drugs refused authorization or not recommended for reimbursement by an international agency was $115,281 ($166,690) per patient per year. Twenty drugs were for oncology indications, and 36 were approved by the FDA through expedited regulatory pathways or the Orphan Drug Act.”