Evaluating promising investigational medical countermeasures: Recommendations in the absence of guidelines

Nahid Bhadelia, Lauren Sauer, Theodore J. Cieslak, Richard T. Davey, Susan McLellan, Timothy M. Uyeki, Mark G. Kortepeter

Research output: Contribution to journalArticle

Abstract

Emerging and re-emerging infectious diseases pose growing global public health threats. However, research on and development of medical countermeasures (MCMs) for such pathogens is limited by the sporadic and unpredictable nature of outbreaks, lack of financial incentive for pharmaceutical companies to develop interventions for many of the diseases, lack of clinical research capacity in areas where these diseases are endemic, and the ethical dilemmas related to conducting scientific research in humanitarian emergencies. Hence, clinicians providing care for patients with emerging diseases are often faced with making clinical decisions about the safety and effectiveness of experimental MCMs, based on limited or no human safety, preclinical, or even earlier product research or historical data, for compassionate use. Such decisions can have immense impact on current and subsequent patients, the public health response, and success of future clinical trials. We highlight these dilemmas and underscore the need to proactively set up procedures that allow early and ethical deployment of MCMs as part of clinical trials. When clinical trials remain difficult to deploy, we present several suggestions of how compassionate use of off-label and unlicensed MCMs can be made more informed and ethical. We highlight several collaborations seeking to address these gaps in data and procedures to inform future clinical and public health decision making.

Original languageEnglish (US)
Pages (from-to)46-53
Number of pages8
JournalHealth Security
Volume17
Issue number1
DOIs
StatePublished - Jan 1 2019

Fingerprint

public health
Public health
Compassionate Use Trials
Guidelines
Public Health
Clinical Trials
Disease
Research
safety
infectious disease
Emerging Communicable Diseases
Safety
Endemic Diseases
lack
incentive
drug
Pathogens
pathogen
decision making
pharmaceutical

Keywords

  • Drug development
  • Ebola
  • Emerging infectious diseases
  • Ethics
  • Medical countermeasures
  • Outbreaks

ASJC Scopus subject areas

  • Health(social science)
  • Emergency Medicine
  • Safety Research
  • Public Health, Environmental and Occupational Health
  • Management, Monitoring, Policy and Law
  • Health, Toxicology and Mutagenesis

Cite this

Bhadelia, N., Sauer, L., Cieslak, T. J., Davey, R. T., McLellan, S., Uyeki, T. M., & Kortepeter, M. G. (2019). Evaluating promising investigational medical countermeasures: Recommendations in the absence of guidelines. Health Security, 17(1), 46-53. https://doi.org/10.1089/hs.2018.0092

Evaluating promising investigational medical countermeasures : Recommendations in the absence of guidelines. / Bhadelia, Nahid; Sauer, Lauren; Cieslak, Theodore J.; Davey, Richard T.; McLellan, Susan; Uyeki, Timothy M.; Kortepeter, Mark G.

In: Health Security, Vol. 17, No. 1, 01.01.2019, p. 46-53.

Research output: Contribution to journalArticle

Bhadelia, Nahid ; Sauer, Lauren ; Cieslak, Theodore J. ; Davey, Richard T. ; McLellan, Susan ; Uyeki, Timothy M. ; Kortepeter, Mark G. / Evaluating promising investigational medical countermeasures : Recommendations in the absence of guidelines. In: Health Security. 2019 ; Vol. 17, No. 1. pp. 46-53.
@article{8637b029fe0145ae9f19aa02396493a0,
title = "Evaluating promising investigational medical countermeasures: Recommendations in the absence of guidelines",
abstract = "Emerging and re-emerging infectious diseases pose growing global public health threats. However, research on and development of medical countermeasures (MCMs) for such pathogens is limited by the sporadic and unpredictable nature of outbreaks, lack of financial incentive for pharmaceutical companies to develop interventions for many of the diseases, lack of clinical research capacity in areas where these diseases are endemic, and the ethical dilemmas related to conducting scientific research in humanitarian emergencies. Hence, clinicians providing care for patients with emerging diseases are often faced with making clinical decisions about the safety and effectiveness of experimental MCMs, based on limited or no human safety, preclinical, or even earlier product research or historical data, for compassionate use. Such decisions can have immense impact on current and subsequent patients, the public health response, and success of future clinical trials. We highlight these dilemmas and underscore the need to proactively set up procedures that allow early and ethical deployment of MCMs as part of clinical trials. When clinical trials remain difficult to deploy, we present several suggestions of how compassionate use of off-label and unlicensed MCMs can be made more informed and ethical. We highlight several collaborations seeking to address these gaps in data and procedures to inform future clinical and public health decision making.",
keywords = "Drug development, Ebola, Emerging infectious diseases, Ethics, Medical countermeasures, Outbreaks",
author = "Nahid Bhadelia and Lauren Sauer and Cieslak, {Theodore J.} and Davey, {Richard T.} and Susan McLellan and Uyeki, {Timothy M.} and Kortepeter, {Mark G.}",
year = "2019",
month = "1",
day = "1",
doi = "10.1089/hs.2018.0092",
language = "English (US)",
volume = "17",
pages = "46--53",
journal = "Health security",
issn = "2326-5094",
publisher = "Mary Ann Liebert Inc.",
number = "1",

}

TY - JOUR

T1 - Evaluating promising investigational medical countermeasures

T2 - Recommendations in the absence of guidelines

AU - Bhadelia, Nahid

AU - Sauer, Lauren

AU - Cieslak, Theodore J.

AU - Davey, Richard T.

AU - McLellan, Susan

AU - Uyeki, Timothy M.

AU - Kortepeter, Mark G.

PY - 2019/1/1

Y1 - 2019/1/1

N2 - Emerging and re-emerging infectious diseases pose growing global public health threats. However, research on and development of medical countermeasures (MCMs) for such pathogens is limited by the sporadic and unpredictable nature of outbreaks, lack of financial incentive for pharmaceutical companies to develop interventions for many of the diseases, lack of clinical research capacity in areas where these diseases are endemic, and the ethical dilemmas related to conducting scientific research in humanitarian emergencies. Hence, clinicians providing care for patients with emerging diseases are often faced with making clinical decisions about the safety and effectiveness of experimental MCMs, based on limited or no human safety, preclinical, or even earlier product research or historical data, for compassionate use. Such decisions can have immense impact on current and subsequent patients, the public health response, and success of future clinical trials. We highlight these dilemmas and underscore the need to proactively set up procedures that allow early and ethical deployment of MCMs as part of clinical trials. When clinical trials remain difficult to deploy, we present several suggestions of how compassionate use of off-label and unlicensed MCMs can be made more informed and ethical. We highlight several collaborations seeking to address these gaps in data and procedures to inform future clinical and public health decision making.

AB - Emerging and re-emerging infectious diseases pose growing global public health threats. However, research on and development of medical countermeasures (MCMs) for such pathogens is limited by the sporadic and unpredictable nature of outbreaks, lack of financial incentive for pharmaceutical companies to develop interventions for many of the diseases, lack of clinical research capacity in areas where these diseases are endemic, and the ethical dilemmas related to conducting scientific research in humanitarian emergencies. Hence, clinicians providing care for patients with emerging diseases are often faced with making clinical decisions about the safety and effectiveness of experimental MCMs, based on limited or no human safety, preclinical, or even earlier product research or historical data, for compassionate use. Such decisions can have immense impact on current and subsequent patients, the public health response, and success of future clinical trials. We highlight these dilemmas and underscore the need to proactively set up procedures that allow early and ethical deployment of MCMs as part of clinical trials. When clinical trials remain difficult to deploy, we present several suggestions of how compassionate use of off-label and unlicensed MCMs can be made more informed and ethical. We highlight several collaborations seeking to address these gaps in data and procedures to inform future clinical and public health decision making.

KW - Drug development

KW - Ebola

KW - Emerging infectious diseases

KW - Ethics

KW - Medical countermeasures

KW - Outbreaks

UR - http://www.scopus.com/inward/record.url?scp=85061838583&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85061838583&partnerID=8YFLogxK

U2 - 10.1089/hs.2018.0092

DO - 10.1089/hs.2018.0092

M3 - Article

C2 - 30724616

AN - SCOPUS:85061838583

VL - 17

SP - 46

EP - 53

JO - Health security

JF - Health security

SN - 2326-5094

IS - 1

ER -