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Expanded Access and Right To Try Requests: The Community Oncologist's Experience
Marjorie E Zettler1, Yolaine Jeune-Smith1, Bruce A Feinberg1
1Cardinal Health Specialty Solutions, Cardinal Health, Dublin, OH.
Purpose:
For patients with cancer who have exhausted approved treatment options and for whom appropriate clinical trials are not available, access to investigational drugs through the US Food and Drug Administration's Expanded Access (EA) program has been an alternative since the program's inception more than 30 years ago. In 2018, federal Right To Try legislation was passed in the United States, creating a second pathway-one that bypasses the US Food and Drug Administration-to obtain unapproved drugs outside of clinical trials. The use of the two programs by community medical oncologists and hematologist-oncologists has not been studied.
Methods:
Between October 2019 and February 2020, community oncologists-hematologists from across the United States completed web-based surveys about EA and Right To Try pathways for accessing unapproved drugs for their patients. Physicians were asked about their utilization of, and perceptions of, the two programs.
Results:
Of the 238 physicians who completed the survey, 46% indicated that they had attempted to gain access to an investigational drug for a patient using the EA program, whereas 14% reported attempting to use Right To Try pathway to obtain an unapproved drug for a patient. Eighty-nine percent of those who tried to use the EA program reported success in obtaining the investigational drug versus 73% of those who attempted to use the Right To Try pathway.
Conclusion:
Our survey found that most community oncologists-hematologists were aware of both the EA and Right To Try pathways, but there is room for improvement in understanding and utilization of the programs.
Insights
Most oncologists know about Expanded Access (EA) and Right To Try pathways for investigational cancer drugs, but understanding and use could be better. EA was more frequently used and successful than Right To Try.
Area of Science:
- Oncology
- Drug Development
- Health Policy
Background:
- Expanded Access (EA) has provided an alternative pathway for patients with cancer to access investigational drugs for over 30 years when standard treatments and clinical trials are unavailable.
- The 2018 federal Right To Try legislation introduced a second, FDA-bypassing pathway for obtaining unapproved drugs outside of clinical trials.
- The utilization and perceptions of these two distinct pathways by community oncologists and hematologist-oncologists remained unstudied.
Purpose of the Study:
- To investigate the awareness, utilization, and perceptions of the Expanded Access (EA) program and the Right To Try legislation among community medical oncologists and hematologist-oncologists in the United States.
- To compare the reported success rates of using EA versus Right To Try for accessing investigational drugs for patients with cancer.
Main Methods:
- A web-based survey was administered to community oncologists-hematologists across the United States between October 2019 and February 2020.
- The survey collected data on physicians' awareness, attempts to use, and perceived effectiveness of both the EA and Right To Try pathways for obtaining unapproved drugs for their patients.
Main Results:
- Out of 238 surveyed physicians, 46% had attempted to use the EA program, while 14% had attempted to use the Right To Try pathway.
- Success rates for obtaining investigational drugs were higher for the EA program (89%) compared to the Right To Try pathway (73%).
Conclusions:
- Community oncologists-hematologists demonstrate general awareness of both EA and Right To Try pathways.
- There is a noted disparity in the utilization and success rates between the EA program and the Right To Try legislation, indicating a need for improved understanding and application of these access programs.
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