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Early phase drugs and biologicals clinical trials on worldwide leading causes of death: a descriptive analysis

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Abstract

Purpose

To describe the global effort targeting the major causes of mortality in terms of “open” early phase clinical trials with drugs and biologicals.

Methods

Sixteen of the 20 leading causes of death were chosen; 9 of these were also amongst the top 10 causes of death in low-income countries. Studies were identified from the ClinicalTrials.gov database and included phase 1 and/or 2 “interventional” “open” trials, i.e. those recruiting or about to start recruitment. Trials were considered in terms of sponsorship [industry, universities and other organisations (UNO), and US federal agencies (NIH included)], genders and age groups included, and whether they were conducted with drugs and/or biologicals. The search was performed in March 2010.

Results

A total of 2,298 (824 phase 1; 1,474 phase 2) trials were retrieved. Of these, 67% were on trachea, bronchus, and lung cancers (25%); diabetes mellitus (15%); colon and rectum cancers (14%); and HIV/AIDS (12%). In contrast, only 4% were trials on diarrhoeal disease, nephrosis and nephritis, liver cirrhosis, and prematurity and low birth weight. UNO were the first source of funding. Fifty-two percent of phase 1 non-cancer trials were on healthy volunteers. Twenty-nine percent of all trials were co-funded. There were 4.6 times as many drug trials as those with biologicals. Only 7% were conducted with a combination of drugs and biologicals, the majority (78%) on cancers. Discrimination in terms of gender or age group was not observed.

Conclusions

Four of the 16 diseases considered represented 2/3 of early phase trials. Cancers were a top priority for all sponsors. Increasing attention should be given to conditions with current and projected global high mortality rates that had few “open” early phase trials.

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Abbreviations

CTG:

ClinicalTrials.gov

FDA:

U.S. Food and Drug Administration

FED:

U.S. National Institutes of Health and other U.S. federal agencies

HIV/AIDS:

Human immunodeficiency virus/acquired immune deficiency syndrome

ICMJE:

International Committee of Medical Journal Editors

INY:

Industry

UNO:

University/organisation: all other sponsors including community-based organisations

WHO:

World Health Organization

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Competing interests

The author declares having no conflict of interests.

Financial support

This study required no financial support.

Author information

Authors and Affiliations

  1. Department of Preventive Medicine, Public Health and Medical Immunology and Microbiology, School of Health Sciences, Rey Juan Carlos University, Alcorcón, Madrid, Spain

    Rafael Dal-Ré

  2. Departamento de Medicina Preventiva y Salud Pública e Inmunología y Microbiología Médicas, Facultad de Ciencias de la Salud, Universidad Rey Juan Carlos, Avda Atenas s/n, 28922, Alcorcón, Madrid, España

    Rafael Dal-Ré

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Correspondence to Rafael Dal-Ré.

Appendix: ClinicalTrials.gov definitions

Appendix: ClinicalTrials.gov definitions

Recruitment status

Also known as enrollment status. […] Full list of all possible recruitment status values:

  • “Open” recruitment status values: Recruiting, Not yet recruiting, or Available for expanded access.

  • Closed recruitment status values: Active, Not recruiting, Completed, Terminated, Suspended, Withdrawn, Enrolling by invitation, Temporarily not available for expanded access, No longer available for expanded access, or Approved for marketing. […]

Study type

There are three types of studies available in ClinicalTrials.gov:

  • “Interventional”: Studies where individuals are assigned to receive specific interventions. Participants may receive diagnostic, therapeutic or other types of interventions. Assignment of the intervention may or may not be random. Individuals are tracked and biomedical and/or health outcomes are assessed.

  • Observational: Studies without an intervention. […]

  • Expanded access: Records describing the procedure for obtaining an experimental drug or device for patients who are failing on currently available treatments for their condition and also are unable to participate in ongoing clinical trials. […]

Conditions

Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov, conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc. […] Examples: lupus, heart attack, leukemia, risk factors for breast cancer

Interventions

Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied. Interventions can also include less intrusive possibilities such as surveys, education, and interviews. […] Examples: clofibrate, cyclosporine, vitamin e

Funded by

Studies are supported by one or more organizations called sponsors. Support includes contributions of facilities, expertise, and/or financial resources. All sponsors in the database have been assigned one of the following organization types:

  • “National Institutes of Health” (NIH)

  • “ Other U.S. Federal Agency”: Food and Drug Administration (FDA), Centers for Disease Control and Prevention (CDC), Department of Veterans Affairs (VA), etc.

  • “ Industry”: the pharmaceutical companies

  • “University/Organization”: all others, including community-based organizations […]

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Dal-Ré, R. Early phase drugs and biologicals clinical trials on worldwide leading causes of death: a descriptive analysis. Eur J Clin Pharmacol 67, 563–571 (2011). https://doi.org/10.1007/s00228-011-1036-1

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  • DOI: https://doi.org/10.1007/s00228-011-1036-1

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