Emerging Opportunities To Streamline Cancer Drug Development
Meeting Information: December 9, 2016 | 8:30 a.m. - 4 p.m. | Arlington, VA 22202
A new era of rapid scientific advancement in cancer research has led to the development of safe, effective, and potentially life-saving drugs for many patients who, until recently, had few treatment options. As our understanding of molecular drivers of cancer has expanded, so have opportunities to develop better and more targeted drugs. However, there is much potential for making the established methods used to evaluate these innovative therapies more efficient, potentially contributing to more affordable drugs for patients. The time is now for all constituencies involved in research and development to re-assess current processes and consider new practices to streamline drug development. The goal: ensure timely access to cost-effective drugs for all cancer patients while stimulating innovation.
In this second workshop in a series, the President’s Cancer Panel will convene leaders and stakeholders across multiple sectors to identify key actions that could transform, and potentially reduce costs of, drug development. Questions to be considered include the following:
- What factors contribute to the clinical development costs for a given drug?
- Could actions be taken to make cancer drug development more efficient, patient-centric, and less costly?
- What key regulatory and legal factors affect development processes for all drugs?
- Which clinical trial models and methods could most efficiently identify safe and effective drugs? Could more meaningful clinical trial outcomes make trials more efficient?
- What regulatory and scientific issues are important for appropriately evaluating and approving combination therapies?
This workshop will be co-chaired by Gary Gilliland, MD, PhD, President and Director, Fred Hutchinson Cancer Research Center. Dr. Gilliland is a national leader in cancer genetics, precision medicine, and drug development. Presentations and moderated discussions among participants will inform the Panel’s recommendations in a formal report to the President of the United States after the conclusion of series workshops.
Follow the Panel on Twitter at @PresCancerPanel for updates during the workshop. Join the conversation using #CancerRxValue.
Meetings of the President’s Cancer Panel are open to the public.
Agenda
| Time | Agenda Item |
|---|---|
| 8:30 a.m. | Registration |
| 9:00 a.m. | Welcome and Introductions |
| 9:10 a.m |
Session 1, Part 1: Bringing Drugs that Add Value to Market Faster Presentation: Richard Pazdur, MD, Acting Director, Oncology Center of Excellence, U.S. Food and Drug Administration (15 min) Moderated Discussion (40 min) |
| 10:10 a.m. | Break |
| 10:25 a.m. | Session 1, Part 2: Bringing Drugs that Add Value to Market Faster |
| 11:25 a.m. |
Session 2, Part 1: Accelerating Throughput and Learning from Clinical Trials Presentation: Lisa LaVange, PhD, Director, Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration (10 min) |
| 11:40 a.m. | Public Comment |
| 11:45 a.m. | Lunch |
| 12:45 p.m. |
Session 2, Part 2: Accelerating Throughput and Learning from Clinical Trials Presentation: James Zwiebel, MD, Chief, Investigational Drug Branch, Cancer Therapy Evaluation Program, National Cancer Institute (10 min) Moderated Discussion (60 min) |
| 1:55 p.m. | Break |
| 2:10 p.m. |
Session 3: Evaluating and Approving Combination Therapies Presentation: Gary Gilliland, MD, PhD, President and Director, Fred Hutchinson Cancer Research Center (10 min) Presentation: Roy Baynes, MD, PhD, Senior Vice President and Head, Global Clinical Development, and Chief Medical Officer, Merck Research Laboratories (10 min) Moderated Discussion (60 min) |
| 3:30 p.m. | Conclusions and Cross-Cutting Recommendations |
| 3:45 p.m. | Public Comment |
| 3:55 p.m. | Wrap-Up and Next Steps |
| 4:00pm | Adjourn |
Participants
| Name | Title & Affiliations |
|---|---|
| Margaret Anderson, MSc |
Executive Director |
| Roy Baynes, MD, PhD |
Senior Vice President and Head |
| Donald A. Berry, PhD | Professor Department of Biostatistics The University of Texas MD Anderson Cancer Center |
| Danielle Carnival, PhD |
Chief of Staff and Senior Policy Director |
| Jason V. Cristofaro, JD, PhD |
Intellectual Property Program Manager
|
| Kathleen A. Denis, PhD |
Associate Vice President
|
| Ann M. Geiger, PhD, MPH | Chief Healthcare Assessment Research Branch Healthcare Delivery Research Program Division of Cancer Control and Population Sciences National Cancer Institute @NCI_AMGeiger |
| Gary Gilliland, MD, PhD | President and Director Fred Hutchinson Cancer Research Center |
| Hill Harper, JD | Member President’s Cancer Panel Author, Actor, and Philanthropist @hillharper |
| Clifford A. Hudis, MD, FACP |
Chief Executive Officer |
| Suleika Jaouad |
New York Times Columnist |
| Aaron Kesselheim, MD, JD |
Associate Professor of Medicine |
| Lisa LaVange, PhD |
Director |
| Lori M. Minasian, MD |
Deputy Director |
| Shelley Fuld Nasso, MPP |
Chief Executive Officer |
| Richard Pazdur, MD |
Acting Director |
| Vinay K. Prasad, MD, MPH |
Assistant Professor |
| Barbara K. Rimer, DrPH |
Chair |
| Mace Rothenberg, MD |
Chief Development Officer |
| Abby B. Sandler, PhD |
Executive Secretary |
| George A. Scangos, PhD |
Chief Executive Officer |
| Greg Simon, JD |
Executive Director |
| Philip J. Stella, MD |
Medical Director |
| Owen Witte, MD | Member President’s Cancer Panel University Professor of Microbiology, Immunology, and Molecular Genetics Director Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research University of California, Los Angeles |
| James Zwiebel, MD |
Chief |
Summary
Today, we live in an exciting era in which advances in the understanding of the molecular basis of cancer and the role of the immune system are creating opportunities for more effective, potentially curative, treatments. Some breakthrough therapies already are improving outcomes for many patients. However, innovative treatment strategies pose new challenges for research and development, and the resulting drugs often come at substantial cost to patients, their families, and the healthcare system. The President’s Cancer Panel is focusing on these issues in its 2016-2017 series of meetings, Ensuring Patients’ Access to High-Value Cancer Drugs.
On December 9, 2016, the Panel convened its second workshop in the series in Arlington, Virginia, bringing together leaders and stakeholders in cancer research and drug development—patients and patient advocates, oncologists, statisticians, and intellectual property specialists, as well as representatives from the biopharmaceutical industry, academic research institutions, the U.S. Food and Drug Administration (FDA), and other government agencies. Key scientific, regulatory, and clinical trial factors that influence cancer drug development were considered, and participants discussed actions that could be taken to optimize drug development processes, accelerate learning from clinical trials, and ensure timely market entry of innovative and highly effective drugs.
The Panel heard concerns about lack of coordination and duplication of effort in cancer drug development. Several participants suggested that sharing and more effective use of data would facilitate more efficient drug development. Clinical trial results—including both positive and negative outcomes—should be made public, and electronic health record data should be used to evaluate the benefits of drugs in clinical practice and identify opportunities for improvement (this issue is addressed in the 2016 Panel report Improving Cancer-Related Outcomes with Connected Health). Knowledge of the underlying biology of cancer and mechanisms of drug action also could be increased if academic researchers had earlier access to investigational agents and were able to evaluate drug combinations in model systems.
Participants also noted opportunities to make cancer clinical trials more effective and efficient through innovative trial designs, including adaptive and fluid approaches that allow researchers to build on early results and allocate resources to investigation of the most promising interventions. The importance of continuing to identify and test potentially synergistic combination therapies that may help avoid or overcome drug resistance was emphasized. The Panel was gratified to hear about ongoing work by FDA, academic research centers, and the biopharmaceutical industry to accelerate progress through innovative trial design and collaboration.
In the midst of excitement about progress in drug discovery and concerns about problems in our systems, we must not lose sight of the ultimate goal of creating high-value drugs for patients—drugs that offer potential for cure, significantly longer survival, and/or increased quality of life when used in real-world settings. Use of patient-reported outcomes is helping researchers and healthcare providers assess patients’ needs, preferences, and responses to treatment. Clinical trials should incorporate questions important to patients, and financial and logistical barriers to trial participation should be eliminated whenever possible.
Information about future workshops in this series will be posted to the President’s Cancer Panel website as it becomes available. Workshop findings and recommendations will be presented in the Panel’s 2017 Report to the President of the United States.