Reporting of Study Participant Demographic Characteristics and Demographic Representation in Premarketing and Postmarketing Studies of Novel Cancer Therapeutics

Varma, Tanvee; Wallach, John R.; Miller, Jennifer E.; Schnabel, Dominic; Skydel, Joshua J.; Zhang, Audrey D; Dinan, Michaela A.; Ross, Joseph S.; Gross, Cary P.

doi:10.1001/jamanetworkopen.2021.7063

Cited by 31 publications

(29 citation statements)

References 48 publications

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“…Second, we need to enhance transparency around who is enrolled in clinical research. While most clinical trials publicly report the numbers of enrolled women, few publicly report the numbers of enrolled patients identifying as racial or ethnic minorities 5. We suggest more rigorously collecting and disclosing data about the racial and ethnic identity of clinical trial participants.…”

Section: Recommendationsmentioning

confidence: 98%

“…Although black, Latinx and Indigenous patients have been disproportionately affected by the COVID-19 pandemic, evidence suggests racial and ethnic minorities may be under-represented in COVID-19 clinical trials 1 2. In fact, in the USA, racial and ethnic minorities are under-represented in clinical trials for most therapeutics and vaccines 3–5. Clinical trial participants are more often male and tend to be whiter, younger and healthier than real-world patients, raising concerns about the safety and effectiveness of novel therapeutics and vaccines for under-represented populations 6–8…”

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confidence: 99%

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Diversity in clinical research: public health and social justice imperatives

et al. 2022

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It is well established that demographic representation in clinical research is important for understanding the safety and effectiveness of novel therapeutics and vaccines in diverse patient populations. In recent years, the National Institutes of Health and Food and Drug Administration have issued guidelines and recommendations for the inclusion of women, older adults, and racial and ethnic minorities in research. However, these guidelines fail to provide an adequate explanation of why racial and ethnic representation in clinical research is important. This article aims to both provide the missing arguments for why adequate representation of racial and ethnic minorities in clinical research is essential and to articulate a number of recommendations for improving diversity going forward.Appropriate racial and ethnic representation and fair inclusion help (1) increase the generalisability of clinical trial results, (2) equitably distribute any benefits of clinical research and (3) enable trust in the research enterprise.

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Section: Recommendationsmentioning

confidence: 98%

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confidence: 99%

Diversity in clinical research: public health and social justice imperatives

et al. 2022

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“…Governments, research sponsors and investigators have obligations to—collectively—promote the health of all. Yet, compared with the patients who actually have the conditions being studied, new medicines and vaccines are often disproportionately tested on populations who are healthier, younger, more likely to identify as white,26 27 and live in urban areas28 and high-income or upper-middle-income countries 1. When the evidence base for new interventions is dominated by data from unrepresentative populations, it may not generalise to patient populations with different disease severities, comorbidities, dietary and nutritional profiles, access to supportive care, age groups and geographies.…”

Section: Fair Subject Selectionmentioning

confidence: 99%

Ethical considerations in international clinical trial site selection

Miller

Millum

2022

BMJ Glob Health

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New medicines and vaccines are predominantly tested in high-income countries. However, as the COVID-19 pandemic highlighted, the populations who can benefit from these interventions are not limited to these wealthier regions. One-third of novel Food and Drug Administration approved drugs, sponsored by large companies, treat infectious diseases like tuberculosis and HIV, which disproportionately affect low-income and middle-income countries (LMICs). The medicines for non-communicable diseases (NCDs) are also relevant to LMIC health needs, as over three-quarters of deaths from NCDs occur in LMICs. There are concerns clinical trial data may not extrapolate across geographical regions, as product effectiveness can vary substantially by region. The pentavalent rotavirus vaccine, for example, had markedly lower efficacy in LMICs. Efficacy variations have also been found for other vaccines and drugs. We argue there are strong ethical arguments for remedying some of this uneven distribution of clinical trial sites by geography and income. Chief among them, is that these disparities can impede equitable access to the benefits of clinical research, such as representation in the evidence base generated to guide prescribing and use of medicines and vaccines. We suggest trial site locations should be made more transparent and for later stage trials their selection should be informed by the global distribution of disease burden targeted by an experimental product. Countries with high prevalence, incidence, severity or infection transmission rates for targeted diseases should have real opportunities to engage in and enrol their populations in trials for novel medicines and vaccines.

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“… 2 However, pivotal trials leading to FDA approval are necessarily limited in the populations they cover. 3 Although attention has been given to limitations in the demographic diversity of the trial population, 4 , 5 less attention has been paid to whether a narrowly defined clinical trial population is then used as the basis for extrapolation of trial findings to a broader population of patients than was represented in those trials. Controversially, the FDA initially approved aducanumab in 2021 for all patients with Alzheimer disease, even though it was studied only in those with mild disease.…”

Section: Introductionmentioning

confidence: 99%

Use of Extrapolation in New Drug Approvals by the US Food and Drug Administration

2022

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Key Points Question How often does the US Food and Drug Administration extrapolate the findings of a drug’s pivotal trial data to its approved indication, in terms of disease severity, subtype, or use of concomitant medication? Findings In this cross-sectional study of 105 new drugs approved from 2015 to 2017, extrapolation beyond pivotal trial participants to other patient populations was noted 23 times in 21 drug approvals. Extrapolation of disease severity was most common, followed by disease subtype and concomitant medication use. Meaning The findings of this study suggest that extrapolation by the US Food and Drug Administration beyond pivotal trial data to final indications is common, highlighting the need for close postapproval monitoring.

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Reporting of Study Participant Demographic Characteristics and Demographic Representation in Premarketing and Postmarketing Studies of Novel Cancer Therapeutics

Abstract: This cross-sectional study investigates demographic data reporting and demographic representation by sex, age, and race/ethnicity in premarketing and postmarketing studies of novel cancer therapeutics.

Cited by 31 publications

References 48 publications

Diversity in clinical research: public health and social justice imperatives

Diversity in clinical research: public health and social justice imperatives

Ethical considerations in international clinical trial site selection

Use of Extrapolation in New Drug Approvals by the US Food and Drug Administration

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