FDA-AACR Workshop: To Test or Not to Test – That is the Question: DPD Deficiency and Weighing Potential Harms - 01/16/2025

---

Background

The U.S. Food and Drug Administration (FDA) Oncology Center of Excellence and the American Association for Cancer Research (AACR) are collaborating on a one-day hybrid public workshop to explore the considerations for dihydropyrimidine dehydrogenase (DPD) deficiency testing for patients receiving fluoropyrimidine drugs as part of their cancer treatment regimen. 

Fluoropyrimidine drugs, including fluorouracil (5-FU) and capecitabine, are frequently used to treat various solid tumors and are important as part of the standard of care regimen in both the early and advanced cancer treatment settings. The DPD enzyme plays a vital role in the breakdown of these drugs into non-toxic products, and individuals with certain genetic variants in the DPYD gene may have complete or partial DPD deficiency, which can lead to an increased risk of severe adverse reactions. While tests for DPD deficiency are available, none have received FDA marketing authorization. In 2022, the FDA product labeling for capecitabine was updated to include revised information on DPD deficiency, and 5-FU product labeling was similarly updated in 2024. In conjunction with the 2022 labeling revisions for capecitabine, FDA also issued its response to a Citizen Petition requesting labeling revisions recommending pre-treatment testing be considered to identify patients with DPD deficiency. 

The purpose of this workshop is to provide a multidisciplinary forum to examine the existing information and evidence surrounding DPD deficiency testing, hear about the current clinical approaches to DPD deficiency testing from practicing oncology healthcare providers, and discuss future directions for advancing the field. 

Meeting Goals

• To discuss the current DPD deficiency testing landscape, including history, and testing over the years
• To understand current clinical approaches to DPD deficiency testing of oncology healthcare practitioners 
• To examine potential approaches for advancing the field and moving forward

Meeting Information

This is a hybrid public event that will take place at the Bethesda Marriott located on 5151 Pooks Hill Road in Bethesda, MD 20814

Registration

Visit AACR Website for additional information

Workshop Materials

• Agenda here
• Biographies here

Contacts

Rukiya Umoja, PharmD, RPh
Associate Director, Regulatory Science and Policy
Office of Science Policy and Government Affairs
American Association for Cancer Research
1401 H Street, NW, Suite 740 | Washington DC 20005
215-309-4350
Rukiya.umoja@aacr.org | www.AACR.org

Brad Davidson, PhD
Analyst, Regulatory Science and Policy
Office of Science Policy and Government Affairs
American Association for Cancer Research
1401 H Street, NW, Suite 740 | Washington DC 20005
Brad.davidson@aacr.org | www.AACR.org

Angela James, MSN, RN
Senior Health Scientist
Oncology Center of Excellence (OCE)
Office of the Commissioner (OC)
U.S. Food and Drug Administration
10903 New Hampshire Ave | Silver Spring, MD 20993
202-0317-0312
Angela.james@fda.hhs.gov