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Ther Innov Regul Sci. 2017 Mar;51(2):146-152. doi: 10.1177/2168479016680253. Epub 2016 Jan 1.
Considerations for Use of Investigational Drugs in Public Health Emergencies.
Kirchoff MC1, Pierson JF1.
Author information
Abstract
The paradigm for the use of investigational drugs in public health emergencies has been recently tested to prevent and treat highly infectious and lethal diseases. Examples include the successful implementation of vaccine and therapeutic clinical trials during the recent Ebola outbreak in West Africa. On the other end of the spectrum was the Emergency Use Authorization (EUA) of peramivir in the treatment of H1N1 influenza virus that did not provide an opportunity to collect data or understand the effectiveness of the EUA program. Between the gold standard of a randomized controlled clinical trial and the problems associated with EUAs are the domain of expanded access protocols that may provide an avenue to make products available while awaiting licensure. This paper will examine the regulatory pathways in the United States (US) for the use of investigational drugs in a public health emergency as well as considerations when making these products available outside the US. Descriptions of the applications of the various approaches will be presented. Regardless of the pathway chosen, public health and clinical research planners need to work together to consider several factors associated with the respective options and maintain a goal of working toward the collection of data to support licensure before faced with future outbreaks. Finally, this paper will consider the lessons learned from public health response in the context of investigational drugs in other diseases where "right to try laws" may pose opportunities, as well as challenges.
KEYWORDS:
Ebola; compassionate use; expanded access protocol; public health emergency; randomized controlled trials
PMID: 28663940 PMCID: PMC5486404 DOI: 10.1177/2168479016680253
Free PMC Article
Considerations for Use of Investigational Drugs in Public Health Emergencies.
Kirchoff MC1, Pierson JF1.
Author information
Abstract
The paradigm for the use of investigational drugs in public health emergencies has been recently tested to prevent and treat highly infectious and lethal diseases. Examples include the successful implementation of vaccine and therapeutic clinical trials during the recent Ebola outbreak in West Africa. On the other end of the spectrum was the Emergency Use Authorization (EUA) of peramivir in the treatment of H1N1 influenza virus that did not provide an opportunity to collect data or understand the effectiveness of the EUA program. Between the gold standard of a randomized controlled clinical trial and the problems associated with EUAs are the domain of expanded access protocols that may provide an avenue to make products available while awaiting licensure. This paper will examine the regulatory pathways in the United States (US) for the use of investigational drugs in a public health emergency as well as considerations when making these products available outside the US. Descriptions of the applications of the various approaches will be presented. Regardless of the pathway chosen, public health and clinical research planners need to work together to consider several factors associated with the respective options and maintain a goal of working toward the collection of data to support licensure before faced with future outbreaks. Finally, this paper will consider the lessons learned from public health response in the context of investigational drugs in other diseases where "right to try laws" may pose opportunities, as well as challenges.
KEYWORDS:
Ebola; compassionate use; expanded access protocol; public health emergency; randomized controlled trials
PMID: 28663940 PMCID: PMC5486404 DOI: 10.1177/2168479016680253
Free PMC Article