Executive Summary

PDS Symposium IX on rare cancer registries was a virtual meeting held on Oct. 7, 2020.

For the first time in our annual symposia series, we are met online. As a result, more of you were able to engage which will help advance this research area that is ripe for fresh attention and poised for new initiatives and progress.

Rare cancer registries hold potential to solve some of the most difficult challenges to advancing treatment of these rare cancers, which account for more than 20% of the cancers reported worldwide.

The symposium brought together multiple stakeholders representing all sides of this challenge: Practice in the clinic, Pharmaceutical research, Policy and regulatory science, and Patient advocacy to brainstorm the operational dynamics between the groups.

We were honored and excited to welcome keynote speaker Dr. David Fajgenbaum, the groundbreaking physician-scientist, disease hunter, speaker, and author of the national bestselling memoir, Chasing My Cure: A Doctor’s Race to Turn Hope Into Action.  He has also developed and led an innovative approach to research through the Castleman Disease Collaborative Network (CDCN).

We expect registries can assist in four key areas:

  • Refine clinical decision making and more effectively manage patient therapy.
  • Assist pharma in designing comparator arms efficiently, thus accelerating the drug development process.
  • Inform regulators who must decide whether to approve new medicines and how to use them in rare cancers.
  • Provide relief for the suffering patients with better treatment strategies.


  • Bill Louv, PhD, President; Project Data Sphere
  • Julia Beaver, MD, Chief of Medical Oncology, Oncology Center of Excellence; US Food and Drug Administration

Keynote: Rare Cancer Registries – Impact on Clinical Practice, Drug Development and Regulators

  • David Fajgenbaum, MD, MBA, MSc, FCPP, Assistant Professor of Medicine, Translational Medicine & Human Genetics; University of Pennsylvania

Panel 1: How Do Rare Cancer Registries Impact Clinical Decision Making?

Moderator: Dave Reese, MD, Executive Vice President of Research & Development; Amgen, Inc.

  • David Miller, MD, Medical Oncologist; Massachusetts General Hospital
  • Gregory Reaman, MD, Associate Director, Pediatric Oncology, Oncology Center of Excellence; US Food and Drug Administration
  • Ting Yu, MD, MS, Medical Director; EMD Serono, a business of Merck KGaA, Darmstadt Germany

Panel 2: How Do Rare Cancer Registries Advance Drugs in Development?

Moderator: Zhen Su, MD, MBA, Senior Vice President, Head of US & Global Oncology Franchise; EMD Serono, a business of Merck KGaA, Darmstadt Germany

  • Martha Donoghue, MD, Pediatric Solid Tumors Scientific Liaison; US Food and Drug Administration
  • Gregory Friberg, MD, Vice President Global Development, Therapeutic Area Head Heme/Onc & Bone; Amgen, Inc.
  • Michael Wong, MD, PhD, FRCPC, Professor, Department of Melanoma Medical Oncology, Division of Cancer Medicine; The University of Texas MD Anderson Cancer Center

Fireside Chat: Generating Clinical Evidence for Biomarker-Driven Indications

  • Sean Khozin, MD, MPH, Vice President, Global Head of Data Strategy; Janssen Research & Development, LLC, to interview Josh Bilenker, MD, Chief Executive Officer; Loxo Oncology, Inc.

Call to Action & Next Steps

  • Andy Crighton, MD, Chief Executive Officer; CEO Roundtable on Cancer and Project Data Sphere

Full Event Replay