This presentation was given during the panel portion of the PMED Opening Workshop on Day 1.

1. Precision Medicine in Oncology
Sirisha Mushti
Division of Oncology, CDER, FDA
SAMSI-PMED 2018

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Disclaimer
This presentation reflects the views of the presenter and should not be
construed to represent FDA’s views or policies
www.fda.gov

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Precision Medicine (NCI Definition)
A form of medicine that uses information about a person’s genes,
proteins, and environment to prevent, diagnose, and treat disease. In
cancer, precision medicine uses specific information about a
person’s tumor to help diagnose, plan treatment, find out how well
treatment is working, or make a prognosis.
Example: Targeted therapies to treat specific types of cancer cells,
such as HER2-positive breast cancer cells, or using tumor marker
testing to help diagnose cancer.
FDASA seminar 2017

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Targeted Therapy
• A therapy with a specific measurable molecular target; implies:
– Benefits the target population
– Population defined by a molecular biomarker (generally a
predictive biomarker)
• Precision Medicine
– Optimize Benefit-Risk Ratio
FDA SA Seminar 2017

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Example
Extent of targeted therapies in Lung Cancer
Source: ASCO 2018
What we achieved so far and room for further improvement based
on precision medicine
www.fda.gov

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Biomarkers
Treatment works only in Marker + Larger Treatment Effect in Marker +
Detrimental Treatment Effect in Marker -
www.fda.gov

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Study Designs based on biomarkers

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• Enrichment Designs
– Enrich the study population
– Enrichment by prognostic marker
– Enrichment by predictive marker
• Master Protocols
– Multiple diseases, Multiple treatments, Multiple molecular
markers
– Non-comparative studies, comparative studies (confirmatory
or exploratory)
www.fda.gov