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T-21: Characteristics of Expanded Access Programs Inclusive of Children in the United States

Poster Presenter

Jit Sheth

Postdoctoral Medical Affairs Fellow
Alnylam Pharmaceuticals and Northeastern University
United States

Objectives

The purpose of this review is to identify and describe Expanded Access Programs (EAPs) that have been available for children (birth to 17 years old) in the United States (US). The identified EAPs will be qualitatively assessed to identify trends in types of programs and therapeutic areas.

Method

A database analysis of ClinicalTrials.Gov National Registry was conducted to identify EAPs that included patients from birth to 17 years old. A programmable web scraper was used to extract data from the Study Record Detail of each EAP. Extracted data were qualitatively analyzed.

Results

Since the implementation of ClinicalTrials.gov in September 2000 until January 30, 2019, 78 EAPs have included patients between birth to 17 years old. Of the 78 identified EAPs, 74% (n=57) included chemically synthesized drugs, 23% (n=19) included biological products, 1% (n=1) included a combination of a chemically synthesized drug with a medical device, and 1% (n=1) included medical devices alone. Clinicaltrials.Gov classified the EAPs into three expanded access types: individual patients, intermediate-size population, and treatment investigational new drug (IND)/protocol. Of the EAPs, 45 were exclusive to individual patients, 10 were exclusive to intermediate-size populations, and 14 were exclusive to treatment IND/protocol populations. All other programs identified (n=9) included mixed expanded access types; the majority of these (n=5) combined individual patients and treatment IND/protocol populations. The programs were submitted to ClinicalTrials.Gov in various years spanning from 2005 to 2018. Of all EAPs, 19% were initiated prior to 2017, 29% were initiated in 2017, and 51% were initiated in 2018. Of the interventions that utilized EAPs, 86% (n=67) were indicated for chronic conditions and 14% (n=11) were indicated for acute conditions. Therapeutic focus among the EAPs were varied, but among all interventions, 22% (n=17) were used to treat oncologic conditions, 22% (n=17) were used to treat hematologic conditions, 21% (n=16) were used to treat neurologic conditions, and the other 36% (n=28) were comprised of 12 other conditions.

Conclusion

EAPs are pathways regulated by the Food and Drug Administration (FDA) that allow manufacturers to provide patients access to investigational therapies. EAPs can be supportive for pediatric populations, as children are among those patient populations who are most underrepresented in clinical trials. Our review highlights the recent sharp increase in availability of EAPs inclusive of children. The majority of these EAPs were established to treat individual patients. These types of EAPs require physicians to request access to the interventions directly from the FDA and drug manufacturers. It can be speculated that the growing practice of including children in EAPs may be due to the emergence of targeted novel therapeutics in difficult to treat conditions. It may also be due to an increase in awareness and understanding of EAPs from physicians through the enactment of the Right to Try Act in 2017. This bill acted as an alternative pathway, alongside existing EAP policies, allowing the federal government to increase access to experimental products. This study was limited by the availability of information on ClinicalTrials.gov. Inconsistencies during classification could have been made by the registry or EAP sponsor and could not have been filtered out.