The following is the summary of “Participant Recruitment From Low- and Middle-Income Countries for Pivotal Trials of Drugs Approved by the U.S. Food and Drug Administration” published in the December 2022 issue of Internal medicine by Awan, et al.
People from all over the world, including those living in low and middle-income nations, are brought in for clinical trials that help the FDA approve new medications sold in the United States. Ethical research conduct and generalizability are impacted by the locations where participants are recruited for pivotal studies. This paper aims to detail how patients from low- and middle-income countries (LMICs) are enrolled in pivotal studies of recently licensed medications for cancer, neurological disease, and cardiovascular disease. Cross-sectional study design. ClinicalTrials.gov, FDA data, and published studies were used to identify pivotal trials of novel cancer, cardiovascular, and neurological medications approved between 2012 and 2019. Country hosts and available enrollments within each host country were tallied. The primary measure was the number of people from LMICs who enrolled in the pivotal studies. The percentage of participants from LMICs in the pivotal trials.
This study compiled information from 144 high-impact clinical studies and 66 novel medicines. A random sample of cancer approvals (28 of 85 medications [33%]) matched with their pivotal trials (61 of 210 trials [29%]) was evaluated, along with all cardiovascular (12 drugs, 29 trials) and neurological (26 drugs, 54 trials) approvals. Researchers in the fields of oncology (56%), cardiology (79%), and neurology (56%) all recruited patients from low- and middle-income countries. Seventy-one trials (or 55%) of those conducted across multiple countries lacked information on enrollment by individual nations. Of the studies that reported enrollment by nation, 8% of cancer patients, 36% of heart patients, and 17% of brain patients were recruited from LMICs.
The study had some caveats, namely that it only included cancer medications authorized by the FDA in one of the 3 groups. Not included were pivotal trials that used unapproved medications or pharmaceuticals for off-label uses. The majority of participants in pivotal trials for medications with an FDA green light come from low- and middle-income countries. Publications and FDA documents rarely include information on recruitment by nation.