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Small Cell Lung Cancer External Control Arm

Until recently, first line therapy for extensive stage small cell lung cancer has remained unchanged for over a decade. That translates to thousands of patients who volunteered for dozens of clinical trials that did not lead to a new treatment. Nearly half of those patients were randomized to receive standard-of-care therapy – ostensibly the same treatment they would have received if they had not enrolled on the trial. Open sharing of these clinical trial data sets through the Project Data Sphere® (PDS) platform has allowed us to see that standard-of-care treatment outcomes remained stable as well.

By receiving de-identified, patient-level data from several clinical trials using the same standard-of-care therapy, our analytics team (led by Professor Lorenzo Trippa, Dana-Farber Cancer Institute) combined the control arms from those trials into a larger, aggregated external data set. The method used to construct this External Control Arm will be published in 2020 and shared through the PDS platform.

Now that the External Control Arm has been developed, we will demonstrate applications of this tool in clinical trial interpretation and to benchmark real world data. In collaboration with our colleagues at the U.S. Food and Drug Administration, we will push the limits of the use of this type of data to understand how such external data can be productively used to improve clinical trials.

Gains made in this program can translate into smaller clinical trials with more patients receiving promising new therapies, accelerated trial timelines, and a potential reduction in the overall cost of bringing a new cancer therapy to market.

When the External Control Arm program launched at FDA-PDS Symposium VI in August 2018, there were big expectations but only a little data to support this research program.

Working from a list of studies developed from www.clinicaltrials.gov, stakeholders within the oncology clinical research community were contacted, resulting in data from three additional studies (to date) being provided to the Project Data Sphere platform. Data from these three studies, plus the data already available within the platform, provided the critical data mass to support true progress in the development of the first external control arm.

Armed with a Letter of Support from FDA encouraging organizations to provide SCLC datasets in support of this research program, Project Data Sphere has additionally projected that data from six more studies should become available for this research program in the near future.

Provide Data

Task Force Chair

Jacob Sands
Dana-Farber/Harvard Cancer Center


Brian Alexander, MD, MPH
Chief Executive Officer
Foundation Medicine
Gheorghe Doros, PhD
Professor of Biostatistics
Boston University School of Public Health
Antonio Fojo, MD, PhD
Co-Director, Adrenal Center, Division of Hematology/Oncology
Columbia University Medical Center
Greg Friberg
Vice President Global Development
Geoff Kim, MD
Vice President of Applied Innovation
Michael Krams
Global Head of Quantitative Sciences
Janssen Pharmaceuticals
Karol Pencina, PhD
Instructor in Medicine and Chief Biostatistician
Brigham and Women’s Hospital and Harvard Medical School
Michael Pencina
Vice Dean for Data Science and Information Technology
Duke University Medical Center and Duke Clinical Research Institute
Rifaquat Rahman
Instructor in Radiation Oncology
Brigham and Women’s Hospital
Frank Rockhold
Professor of Biostatistics and Bioinformatics
Duke University Medical Center and Duke Clinical Research Institute
Lorenzo Trippa, PhD
Associate Professor of Biostatistics
Steffen Ventz
Research Scientist
Harvard School of Public Health/Dana-Farber Cancer Institute
Patrick Wen
Professor of Neurology and Director of the Center for Neuro-Oncology
Dana-Farber/Harvard Cancer Center

Program Manager

Jon McDunn, PhD
Project Data Sphere


Dataset Received

Bayer | April 24, 2019

Major Milestone

On January 4, 2019, the FDA’s Oncology Center of Excellence issued a Letter of Support encouraging people to contribute small cell lung cancer data to Project Data Sphere.

Major Milestone

FDA-PDS Symposium VI: Small Cell Lung Cancer External Control Arm: A Paradigm Shift in Drug Evaluation/Registration | August 8, 2018

Dataset Received

Amgen | August 3, 2018

Dataset Received

Alliance for Clinical Trials in Oncology | July 31, 2018

Dataset Received

Alliance for Clinical Trials in Oncology | October 23, 2017

Dataset Received

Eli Lilly | January 30, 2015