Daniel K. Inouye College of Pharmacy, University of Hawai'i at Hilo.
The Queen's Medical Center, Honolulu, Hawai'i.
JAMA Netw Open. 2024 Oct 1;7(10):e2442204. doi: 10.1001/jamanetworkopen.2024.42204.
Having diverse participants in clinical trials ensures new drug products work well across different demographic groups, making health care safer and more effective for everyone. Information on the extent of Native Hawaiian and Pacific Islander participation in clinical trials is limited.
To examine representation of Native Hawaiian and Pacific Islanders in clinical trials leading to the first US Food and Drug Administration (FDA) approvals for the 10 drug products with the top worldwide sales forecasts in 2024.
DESIGN, SETTING, AND PARTICIPANTS: Cross-sectional secondary analysis of existing data from clinical trials that took place from 2006 to 2021 in the US. All clinical trials that were included in the FDA first approval application for the 10 drug products were evaluated in this study. Data were analyzed from February to August 2024.
Participation in a clinical drug trial.
Comparison of the proportion of Native Hawaiian and Pacific Islander participation in clinical trials for the 10 drug products with top sales forecasts in 2024 to the Native Hawaiian and Pacific Islander population proportion.
In this cross-sectional study of 139 062 individuals, Native Hawaiian and Pacific Islander participation in clinical trials for the 10 drug products with top sales forecasts was either unknown or low. For 6 of the 10 drug products (60%), the number of Native Hawaiian and Pacific Islander participants was not documented. All trials that reported Native Hawaiian and Pacific Islander participation had fewer Native Hawaiian and Pacific Islander participants than would be expected based on their US population proportion, with 2 of the differences being statistically significant. Of the trials that disaggregated Native Hawaiian and Pacific Islander participants from other racial groups, the number of Native Hawaiian and Pacific Islander participants was 8 for risankizumab-rzaa (0.38% of participants vs 0.49% of the population; percentage point difference, -0.11%; 95% CI, -0.37% to -0.15%), 7 for bictegravir/emtricitabine/tenofovir alafenamide (0.38% of participants vs 0.49% of the population; percentage point difference, -0.10%; 95% CI, -0.39% to 0.18%), 27 for 4vHPV/9vHPV (0.15% of participants vs 0.46% of the population; percentage point difference, -0.31%; 95% CI, -0.37% to -0.26%), and 90 for BNT162B2 COVID-19 vaccine (0.20% of participants vs 0.52% of the population; percentage point difference, -0.32; 95% CI, -0.36% to -0.27%).
In this cross-sectional study, limited documentation and participation of Native Hawaiian and Pacific Islander individuals in clinical trials for drug products with top sales forecasts was found. This is especially concerning because Native Hawaiian and Pacific Islander individuals have a higher risk than other racial groups for type 2 diabetes, cancer, and several other conditions the products examined in this study treat. Given the importance of enrolling Native Hawaiian and Pacific Islander participants in clinical trials, sites should be established in key geographic regions, such as Hawai'i, and postmarket studies should be conducted within Native Hawaiian and Pacific Islander populations.
临床试验中参与者的多样性确保了新药物产品在不同人群中都能有效发挥作用，从而使每个人的医疗保健更加安全有效。关于夏威夷原住民和太平洋岛民参与临床试验的程度的信息有限。
检查在导致美国食品和药物管理局（FDA）首次批准的 2024 年全球销售额最高的 10 种药物产品的临床试验中，夏威夷原住民和太平洋岛民的代表性。
设计、地点和参与者：对 2006 年至 2021 年期间在美国进行的临床试验的现有数据进行的横断面二次分析。本研究评估了这 10 种药物产品的 FDA 首次批准申请中包含的所有临床试验。数据分析于 2024 年 2 月至 8 月进行。
参与药物临床试验。
将 2024 年销售预测最高的 10 种药物产品中夏威夷原住民和太平洋岛民参与临床试验的比例与夏威夷原住民和太平洋岛民的人口比例进行比较。
在这项针对 139062 人的横断面研究中，10 种销售预测最高的药物产品中夏威夷原住民和太平洋岛民参与临床试验的情况要么未知，要么很低。对于这 10 种药物产品中的 6 种（60%），没有记录夏威夷原住民和太平洋岛民参与者的数量。所有报告夏威夷原住民和太平洋岛民参与的试验中，夏威夷原住民和太平洋岛民参与者的数量都低于根据其美国人口比例预期的数量，其中 2 项差异具有统计学意义。在将夏威夷原住民和太平洋岛民参与者与其他种族群体分开的试验中，risankizumab-rzaa 的夏威夷原住民和太平洋岛民参与者人数为 8 人（参与者的 0.38%，而人口的 0.49%；百分点差异，-0.11%；95%CI，-0.37%至-0.15%），bictegravir/emtricitabine/tenofovir alafenamide 为 7 人（参与者的 0.38%，而人口的 0.49%；百分点差异，-0.10%；95%CI，-0.39%至 0.18%），4vHPV/9vHPV 为 27 人（参与者的 0.15%，而人口的 0.46%；百分点差异，-0.31%；95%CI，-0.37%至-0.26%），BNT162B2 COVID-19 疫苗为 90 人（参与者的 0.20%，而人口的 0.52%；百分点差异，-0.32%；95%CI，-0.36%至-0.27%）。
在这项横断面研究中，发现销售预测最高的药物产品临床试验中，夏威夷原住民和太平洋岛民个人的文件记录和参与有限。这尤其令人担忧，因为与其他种族群体相比，夏威夷原住民和太平洋岛民患 2 型糖尿病、癌症和本研究中检查的其他几种疾病的风险更高。鉴于在临床试验中招募夏威夷原住民和太平洋岛民参与者的重要性，应在关键地理位置（如夏威夷）建立站点，并应在夏威夷原住民和太平洋岛民群体中进行上市后研究。
