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Drug Development, Trial Design, and Endpoints in Oncology: Adapting to Rapidly Changing Science.
G M Blumenthal1, K B Goldberg1, R Pazdur1
1Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Clinical Pharmacology and Therapeutics
|January 12, 2017
Summary
Advancements in understanding cancer genetics and immunology have spurred targeted and immunotherapies. Drug development now uses seamless trial designs and expedited FDA programs to bring innovative cancer treatments to patients faster.
Area of Science:
- Oncology
- Cancer Genetics
- Cancer Immunology
- Drug Development
Background:
- Enhanced understanding of genetic and immunologic factors driving cancer.
- Progress in developing targeted therapies and immunotherapies for cancer treatment.
Purpose of the Study:
- To describe the evolution of cancer drug development models.
- To highlight the role of seamless drug development and expedited regulatory pathways.
Main Methods:
- Review of traditional versus modern drug development paradigms.
- Analysis of expedited programs by the US Food and Drug Administration (FDA).
Main Results:
- The traditional linear drug development model is evolving.
- Seamless drug development incorporates expansion cohorts in early trials.
- FDA expedited programs accelerate availability of transformative therapies.
Conclusions:
- Modern oncology drug development is more adaptive and patient-centric.
- Seamless designs and expedited approvals facilitate earlier patient access to novel cancer treatments.
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