Pharmacy Outcomes Research Group, Kaiser Permanente National Pharmacy, Downey, California.
Drug Evaluation, Strategy, and Outcomes, Kaiser Permanente National Pharmacy, Downey, California.
JAMA Intern Med. 2023 Apr 1;183(4):290-297. doi: 10.1001/jamainternmed.2022.6787.
Drug expenditures in the US are higher than in any other country and are projected to continue increasing, so US health systems may benefit from evaluating international regulatory and reimbursement decision-making of new drugs.
To evaluate regulatory decisions and health technology assessments (HTAs) in Australia, Canada, and the UK regarding new drugs approved by the US Food and Drug Administration (FDA) in 2017 through 2020, as well as to estimate the US cost per patient per year for drugs receiving negative recommendations.
In this cross-sectional study, recommendations issued by agencies in Australia, Canada, and the UK were collected for new drugs approved by the FDA in 2017 through 2020. All data were current as of May 31, 2022.
Authorizations and HTAs in selected countries.
All FDA-approved drugs were matched by active ingredient to decision summary reports published by drug regulators and HTA agencies in Australia, Canada, and the UK. Regulatory approval concordance and reasons for negative recommendations were assessed using descriptive statistics. For drugs not recommended by an international agency, the annual US drug cost per patient was estimated from FDA labeling and wholesale acquisition costs.
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. 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.
This cross-sectional study assessed reasons for which drugs recently approved by the FDA were refused marketing authorization or not recommended for public reimbursement in other countries. Drugs with limited international market presence may require close examination by US health care professionals and health systems.
美国的药品支出高于其他任何国家，且预计将继续增加，因此美国卫生系统可能受益于评估新药品在国际监管和报销决策方面的情况。
评估澳大利亚、加拿大和英国在 2017 年至 2020 年期间对美国食品和药物管理局（FDA）批准的新药的监管决策和健康技术评估（HTA），并估计接受负面推荐的药品的每位患者每年的美国费用。
在这项横断面研究中，收集了澳大利亚、加拿大和英国各机构针对 2017 年至 2020 年期间 FDA 批准的新药发布的建议。所有数据均截至 2022 年 5 月 31 日。
选定国家的授权和 HTA。
所有 FDA 批准的药物均通过活性成分与澳大利亚、加拿大和英国的药物监管机构和 HTA 机构发布的决策摘要报告相匹配。使用描述性统计数据评估监管批准的一致性和负面推荐的原因。对于未被国际机构推荐的药物，根据 FDA 标签和批发采购成本估算每位患者每年的美国药物费用。
FDA 在 2017 年至 2020 年期间批准了 206 种新药，其中 162 种（78.6%）在获得美国批准后至少有 1 个其他监管机构在中位数（IQR）12.1（17.7）个月内获得了营销授权。相反，由于对风险效益评估不利，5 种 FDA 批准的药物被国际监管机构拒绝上市许可。由于临床获益不确定或价格过高，澳大利亚、加拿大或英国的 HTA 机构对另外 42 种 FDA 批准的药物给予了负面的报销建议。被国际机构拒绝授权或不建议报销的 47 种药物的中位数（IQR）每位患者每年的美国费用为 115281 美元（166690 美元）。20 种药物用于肿瘤学适应症，36 种药物通过加速监管途径或孤儿药法案获得 FDA 批准。
这项横断面研究评估了最近获得 FDA 批准的药物在其他国家被拒绝上市许可或不建议公共报销的原因。国际市场占有率有限的药物可能需要美国医疗保健专业人员和卫生系统的密切审查。
