2014 Program

Frank McCormick, Ph.D., F.R.S., D.Sc. (Hon)

Frank McCormick, Ph.D., F.R.S., D.Sc. (Hon) is the Director of the University of California, San Francisco (UCSF) Helen Diller Family Comprehensive Cancer Center, a multidisciplinary research and clinical care organization that is one of the largest cancer centers in the Western United States, and he is Associate Dean of the UCSF School of Medicine. A native of Cambridge, England, Dr. McCormick received his B.Sc. in biochemistry from the University of Birmingham (1972) and his Ph.D. in biochemistry from the University of Cambridge (1975). Postdoctoral fellowships were held in the United States at the State University of New York at Stony Brook and in London at the Imperial Cancer Research Fund. He has been a Fellow of the Royal Society since 1996. Prior to joining the UCSF faculty, Dr. McCormick pursued cancer-related work with several Bay Area biotechnology firms, including positions with Cetus Corporation (Director of Molecular Biology, 1981-90; Vice President of Research, 1990-91) and Chiron Corporation, where he was Vice President of Research from 1991-92. In 1992 he founded Onyx Pharmaceuticals and served as its Chief Scientific Officer until 1996. His current research interests center on the fundamental differences between normal cells and cancer cells that can allow the development of novel therapeutic strategies. In addition to his positions as Director of the UCSF Helen Diller Family Comprehensive Cancer Center, and Associate Dean of the UCSF School of Medicine, he holds the E. Dixon Heise Distinguished Professorship in Oncology and the David A. Wood Distinguished Professorship of Tumor Biology and Cancer Research in UCSF's Department of Microbiology and Immunology. More recently, he has taken a leadership role at the Frederick National Laboratory for Cancer Research, overseeing an NCI supported national effort to develop therapies against Ras-driven cancers. These cancers include most pancreatic cancers, and many colorectal and lung cancers, and are amongst the most difficult cancers to treat.

Thomas Fleming, Ph.D.

Thomas Fleming, Ph.D. is Professor and Chairman in the Department of Biostatistics and Professor of the Department of Statistics at the University of Washington School of Public Health and Community Medicine. He is also a full member in the Division of Public Health Sciences at the Fred Hutchinson Cancer Research Center. In 1987 he was elected a fellow of the American Statistical Association and in 1988 he was the recipient of the Spiegelman Award from the American Public Health Association in recognition of outstanding contributions to public health research, especially in the areas of oncology and HIV infection. In 2002, he was the recipient of the FDA Commissioner's Special Citation Award for extraordinary contributions to the Agency. Dr. Fleming has been involved in AIDS research efforts for nearly 20 years as an advisor to the National Institute of Allergy and Infectious Diseases (NIAID), as a collaborator in NIAID's multi-center multi-institution AIDS cooperative groups, as a consultant to industry, as an advisor to the FDA, as an educator, and as a researcher of new statistical methodology. Dr. Fleming was involved in the development and coordination of NIAID's national clinical trials program for the prevention and treatment of HIV infection and AIDS. Dr. Fleming also served as an advisor to the FDA for 18 years and has been a voting member of numerous advisory committees for the Center for Drug Evaluation Research and the Center for Biologics Evaluation Research. Since 1987, Dr. Fleming has been a member of the DAIDS Data Monitoring Committee that oversees trials conducted by ACTG and CPCRA, and has served as the chair or a member of data monitoring committees for over 100 industry- and government-sponsored clinical trials. Dr. Fleming also served as the Co-PI of the HIVNET Statistical Center and chair of the HIVNET and HPTN Study Monitoring Committees. In the HPTN, Dr. Fleming served as statistician to the perinatal working group, and as lead statistician on five perinatal studies, three of which are completed, including the pivotal HIVNET 012 trial.

John Powers III, M.D.

John Powers III, M.D. is a physician/investigator on faculty as an Associate Clinical Professor of Medicine at the George Washington University School of Medicine. Prior to his current position Dr. Powers was the Lead Medical Officer for Antimicrobial Drug Development and Resistance Initiatives at the US Food and Drug Administration. Dr. Powers was co-chair for the US Federal Inter-Agency Task Force on Antimicrobial Resistance. Prior to joining the FDA, Dr. Powers was assistant professor in the Division of Infectious Diseases at the University of Maryland School of Medicine and he still is on the faculty there. Dr. Powers also actively cares for patients weekly in clinic and attends on the infectious diseases inpatient service. He has been an investigator on over 50 clinical trials. He has particular expertise in the design, conduct and analysis of clinical trials and has published on various aspects of clinical trial design. He has won several teaching awards and was nominated for the 2008 Distinguished Clinical Teacher Award by the National Institutes of Health Fellows Committee. He is a recipient of the 2010 NIH Director’s Award. Dr. Powers received his bachelor’s degree and graduated magna cum laude from the University of Pennsylvania. He received his medical degree and residency training from Temple University School of Medicine, where he also served as Chief Resident. He completed his infectious diseases training at the University of Virginia School of Medicine.

Lisa McShane, Ph.D.

Lisa McShane, Ph.D. holds a doctorate degree in statistics from Cornell University and is a Fellow of the American Statistical Association. She is a member of the Biometric Research Branch at the U.S. National Cancer Institute where she advises the NCI Cancer Diagnosis and Cancer Therapy Evaluation Programs on statistical matters relating to development and use of tumor markers for prognosis, therapy selection, and disease-monitoring. Her research interests include biomarker-based clinical trial design, analysis of high-dimensional genomic data, multiple comparisons methods, surrogate endpoints, measurement error adjustment methods, laboratory quality control, and biomarker assay analytical performance assessment. She co-led the efforts to develop “Reporting guidelines for tumor marker prognostic studies (REMARK)” and “Criteria for the use of omics-based predictors in clinical trials”. She is co-author of more than 100 publications in statistical and biomedical journals and a co-author of the book “Statistical Design and Analysis of DNA Microarray Investigations.”

Ethan Basch, M.D.

Ethan Basch, M.D. is a practicing oncologist and Director of the Cancer Outcomes Research Program at the University of North Carolina in Chapel Hill. His research focuses on patient-reported outcomes in clinical trials, comparative effectiveness, and quality of care assessments. He is as a member of the Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI), a member of the Board of Scientific Advisors of the National Cancer Institute, chairs the Health Outcomes Committee of the Alliance for Clinical Trials in Oncology, and serves on the Board of Directors of the International Society for Quality of Life Research. Currently, he leads the National Cancer Institute's PRO-CTCAE initiative to develop a standardized patient-centered approach to safety reporting in clinical trials. The overall goal of Dr. Basch’s work is to improve our understanding of, and the quality of, patients’ experiences with illness and care.

Ari Gnanasakthy, MSc, MBA

Ari Gnanasakthy, MSc, MBA is an Executive Director at Novartis Pharmaceuticals, and heads the Patient Reported Outcomes Center of Excellence. He has been in the pharmaceutical industry for almost 25 years. Within Novartis he has been in various functions including Biostatistics, Health Economics, Pricing, and Outcomes Research. After receiving his bachelor degree in Mathematics, Statistics and Computing from University of Greenwich, UK, Mr. Gnanasakthy was at Rothamsted Experimental Station (UK) where he was responsible for the statistical analysis of a survey data of agricultural soil in England and Wales, and then at the Milk Marketing Board (UK) where he was part of a team that was responsible for modeling lactation curves of dairy cows. He also has a Master’s degree in Applied Statistics from Sheffield Hallam University (UK) and an MBA from The University of Kingston (UK). In his current role he drives PRO strategies as well as acts as an internal consultant when brand teams assess the potential of PRO assessments in compounds in development. Mr. Gnanasakthy’s extensive experience in the field of statistics and outcome research has resulted in numerous abstracts and almost 40 publications. Throughout his career he has developed and validated over a dozen patient reported outcomes instruments and currently serves as a reviewer for many professional journals including Value in Health.

Paul Kluetz, M.D.

Paul Kluetz, M.D. is a board certified medical oncologist and internist who has been a clinical reviewer at the FDA since 2010. Dr. Kluetz attended the University of Pittsburgh School of Medicine and went on to complete internal medicine residency at the University of Maryland and his medical oncology fellowship at the National Cancer Institute (NCI) in Bethesda, M.D. At the NCI, he worked with Dr. Bill Dahut focusing on early phase clinical trials of anti-angiogenic agents in prostate cancer. At the FDA, Dr. Kluetz has focused on genitourinary oncology drug development. He participated in the clinical review of the oral anti-cancer drug abiraterone acetate (Zytiga®) as well as the novel alpha-emitting radiopharmaceutical Radium-223 (Xofigo®), both of which were granted FDA approval to treat metastatic prostate cancer. He has represented the FDA as a speaker at several oncology drug advisory committee meetings on topics including the accelerated approval of oncology drugs and challenges involved in the development of products for non-metastatic castration resistant prostate cancer. Dr. Kluetz is engaged with the academic community both through peer-reviewed publication and as a speaker at various workshops and international meetings. He has developed a particular interest in several regulatory topics including defining clinical benefit in oncology trials, the use of expedited programs such as accelerated approval, and the opportunities and challenges associated with patient reported outcomes data. Dr. Kluetz remains clinically active, treating prostate cancer patients at the National Institutes of Health and practicing inpatient medicine at Georgetown University Hospital.

Stephen Coons, Ph.D.

Stephen Coons, Ph.D. is Executive Director of the Patient-Reported Outcome (PRO) Consortium at the Critical Path Institute (C-Path). C-Path, an independent, non-profit organization, established the PRO Consortium in cooperation with the U.S. Food and Drug Administration and the pharmaceutical industry for the purpose of developing, evaluating, and qualifying PRO measures for use in clinical trials. Dr. Coons joined C-Path after a 23-year career in academia and retains the title of emeritus professor at the University of Arizona. His last academic role was professor in the College of Pharmacy and the Mel & Enid Zuckerman College of Public Health at the University of Arizona in Tucson. In addition, he served as co-director of the Arizona Cancer Center’s Behavioral Measurements Shared Service. After receiving a BS degree in pharmacy from the University of Connecticut, he completed a hospital pharmacy residency at the VA Medical Center in Phoenix and earned an MS degree in pharmacy, an MEd degree in higher education, and a Ph.D. in pharmacy (administrative and behavioral sciences) at the University of Arizona. His post-doctoral training in health outcomes research was completed at the University of California, San Diego (UCSD). Previous academic appointments have been in the colleges of pharmacy, medicine, and allied health professions at the University of Kentucky, and in the Division of Health Care Sciences at the UCSD School of Medicine. Dr. Coons is a fellow in the American Association of Pharmaceutical Scientists. For the past two decades, the primary focus of his research has been the measurement of patient-reported outcomes.

Richard Schilsky, M.D.

Richard Schilsky, M.D. is currently the Chief Medical Officer of the American Society of Clinical Oncology. He earned his M.D. at the University of Chicago Pritzker School of Medicine in 1975. Following a residency in Internal Medicine at the University of Texas Southwestern Medical Center and Parkland Memorial Hospital, he received training in Medical Oncology and Clinical Pharmacology at the National Cancer Institute from 1977 to 1981. He then served as Assistant Professor of Medicine at the University of Missouri-Columbia School of Medicine from 1981-1984 when he returned to the University of Chicago. At the University of Chicago, Dr. Schilsky rose to the rank of Professor of Medicine (tenured) and served as Director of the University of Chicago Cancer Research Center (1991-99), as Associate Dean for Clinical Research (1999-2007) and as Chief of the Section of Hematology-Oncology (2009-2012). From 1995-2010, Dr. Schilsky also served as Chairman of the Cancer and Leukemia Group B, an NCI-sponsored national cancer clinical trials group. An international expert in gastrointestinal malignancies and cancer pharmacology, he has served on a number of peer review and advisory committees for the NCI including as a member and chair of the NCI Board of Scientific Advisors and as a member of the Clinical and Translational Research Advisory Committee. Dr. Schilsky also served as a member and chair of the Oncologic Drugs Advisory Committee of the Food and Drug Administration. Dr. Schilsky has served as a member of the Board of Directors of ASCO and of the Conquer Cancer Foundation of ASCO and as ASCO President 2008-2009.

Kald Abdallah, M.D., Ph.D.

Kald Abdallah M.D., Ph.D. is the Head of North America Medical Affairs (NAMA) for Immunology at Sanofi, and has worked in the pharmaceutical industry as well as in academic research for the last 13 years. His medical training is in Nephrology and Allergy/ Clinical Immunology. He has more than 10 years of medical practice experience. Dr. Abdallah has extensive medical and research training with an M.D. and a Ph.D. degree from Sao Paulo University and a research fellowship from Harvard Medical School. He is a member of the Project Data Sphere initiative (www.projectdatasphere.org) team and leader of the onboarding process team.

Perry Nisen, M.D. Ph.D.

Perry Nisen, M.D. Ph.D. is the Senior Vice President, Science and Innovation at GlaxoSmithKline. He previously held leadership roles as interim Chief Medical Officer, Senior Vice President and Head of Oncology Research and Development, and of Clinical Pharmacology and Discovery Medicine. In those roles he has been involved in the successful discovery and development of multiple marketed products. He is a member of key governance, scientific advisory and investment Boards at GlaxoSmithKline. Perry’s leadership helped establish an end-to-end research and development center in China. He is a champion for data sharing efforts. Perry holds a BS from Stanford University and M.D. and Ph.D. from the Albert Einstein College of Medicine. Formerly, he was the Lowe Foundation Professor of Neuro-oncology at the University of Texas Southwestern School of Medicine, then Vice President, Cancer Research at Abbott Laboratories.

Patrick Archdeacon, M.D.

Patrick Archdeacon, M.D. is a medical officer in the Office of Medical Policy in the Center for Drug Evaluation and Research (CDER) at FDA. His work includes involvement in the Clinical Trials Transformation Initiative (a public-private partnership that identifies practices to increase the quality and efficiency of clinical trials) and the Kidney Health Initiative (a public-private partnership created to advance scientific understanding of the kidney health and patient safety implications of new and existing medical products and to foster development of therapies for diseases that affect the kidney). He also serves as the clinical lead for the Office of Medical Policy team supporting the development of the Sentinel Initiative – a system that will draw on existing automated healthcare data from multiple sources to actively monitor the safety of medical products continuously and in real time. Dr. Archdeacon originally joined FDA in 2008 as a medical officer in the Division of Special Pathogens and Transplant Products in the Office of New Drugs prior to transferring to the Office of Medical Policy in 2011. He attended medical school at Columbia University’s School of Physicians and Surgeons. Before joining FDA, he completed his training in internal medicine at the New York Presbyterian Hospital and in nephrology and transplant nephrology at the University of North Carolina.

Regulatory Considerations in the Development of Genetically Engineered Cellular Products

An in-depth discussion of regulatory, clinical and manufacturing considerations in the development of cell and gene based immunotherapy products. Panelists will discuss considerations ranging from the objectives of early-phase trials to chemistry, manufacturing and controls (CMC) issues. The discussion will cover considerations such as endpoints assessment, defining benefit, feasibility in manufacturing, investigator training, goals of dose escalation and optimization, understanding/managing toxicity and long-term follow-up and assessments.

Biomarker Facilitated Drug Development : The Role of Circulating Tumor Cells and Tumor Cell-free DNA

This session will discuss advances surrounding Circulating Tumor Cells (CTCs) and Circulating Cell-Free Tumor DNA (ctDNA). Speakers on the cutting edge of scientific and clinical research across varied tumor subtypes will review current applications and discuss breakthroughs in using these markers for detection, prognosis, prediction and monitoring. A special focus will be placed on what the advancement of technologies to detect CTCs and ctDNA means to patients with early or late stage disease and what the future will bring in terms of incorporating CTCs and ctDNA into clinical practice. The expert panel will also weigh in on the potential of these markers to expedite the drug approval process and discuss how practically that vision may become reality.

Improved Strategies for Selecting Recommended Phase 2 Dosing: Moving Beyond Maximum Tolerated Dose

Balancing the benefits and risks of cancer therapies is critical in order to provide longer survival while maintaining or improving quality of life. A key to achieving this balance is identifying the dose at which efficacy is maximized and toxicity is minimized; a dose that is too high can render an otherwise effective drug intolerable, while a dose that is too low can result in the expected therapeutic benefit of a drug not being achieved. The need to develop new drugs quickly often takes precedence over the need to find the “right” dose. In the current oncology paradigm the objective of early phase trials is to determine the highest tolerable dose, based on the assumption that higher doses will provide greater efficacy. During the small-group discussion we will identify where the dose often goes wrong in clinical development. Ideas for expanding assessments in early trials to obtain necessary information to guide dosing in future clinical trials will be discussed. Drugs that have successfully used a biologically effective dose paradigm will be explored as examples.

Cindy Geoghegan

Cindy Geoghegan has represented the patient voice on task forces for the National Cancer Institute, the Johns Hopkins Kimmel Cancer Center, and the American Association for Cancer Research (AACR), American Association for Clinical Oncology (ASCO), Stand Up to Cancer and other research agencies. She has been recognized for her efforts in breast cancer advocacy by the AACR, the U.S. Health Care Finance Administration (now CMS), Yoplait, Kellogg’s, and BMW, and has also been recognized with a Komen Lifetime Volunteer Award. Ms. Geoghegan was formerly CEO of Y-ME National Breast Cancer Organization, where she returned the organization to its unique mission of peer-to-peer support, and drastically reduced expenses to enhance Y-ME’s 24/7 hotline and network of 150 breast cancer survivors. She was previously executive advisor of scientific community relations for Susan G. Komen for the Cure®, where she worked closely with Komen’s chief scientific advisor and scientific advisory board on grants strategy, scientific programs, education materials and public policy initiatives. As a founder of Komen’s Maryland Affiliate, she created a community grants program, led education efforts and worked on statewide legislative policy efforts. When she relocated she was elected president of Komen’s Greater New York City Affiliate, and received Komen’s “Affiliate of the Year” distinction and became the largest revenue producer in the 120-plus-affiliate network. She continues her work as a patient advocate and is currently a Principal of Patient and Partners LLC.

Immune Checkpoint Blockade and Cancer: Therapeutic Advances and Future Strategies

The ability to harness the power of the immune system in the fight against cancer holds great appeal for researchers, clinicians and patients, alike. One such approach is immune checkpoint blockade which aims to engage the immune system to recognize tumors as foreign. This strategy was validated clinically with the 2011 FDA approval of the anti-CTLA4 antibody, ipilimumab, for the treatment of unresectable or metastatic melanoma. Other checkpoint blockade inhibitors are in various stages of clinical development including anti-PD1/PDL1 antibodies which show encouraging data in clinical studies for a variety of cancers, including lung cancer and melanoma. This session will discuss both the current status, challenges and future strategies to exploit checkpoint blockade to improve cancer outcomes.

Expanding the Preclinical Evidence for Early Phase Studies of New Targeted Agents in Children: Strategies for Prioritization and Regulatory Considerations

Recent regulations in both the US and European Union have mandated that plans for pediatric clinical trials occur earlier in the drug development process. Preclinical testing is critically important in oncology drug development and particularly so for targeted agents in pediatrics. Information gained from adult preclinical studies often cannot be extrapolated to pediatric tumors given different histologies and varying importance of the same molecular pathways. Limited patient populations also require thoughtful planning and clinical trial design for testing these new agents. This session will focus on the platforms available for pediatric preclinical testing of molecularly targeted agents, examples of agents taken into the clinic using these methods, and discussion of the limitations of challenges with these models. We will also discuss the prioritization of the many available targeted agents to be taken into pediatric clinical trials in oncology and unique trial designs that are used.

Accelerating Anticancer Agent Development and Validation Workshop
2014 Program

Wednesday, May 7

7:00 - 8:00am Registration/Continental Breakfast
8:00 - 8:30am Opening Remarks
8:30 - 9:30am Keynote Address: The Development of Therapies Against Ras-driven Cancers
Speaker: Frank McCormick, Ph.D., F.R.S., D.Sc. (Hon) (University of California, San Francisco)
9:30 - 10:00am Break
10:00am - 12:00pm FDA Core Curriculum #1
Modules are conducted concurrently, and will be repeated on Thursday and Friday. Learners will have the opportunity to attend all three modules.
  • Module 1: Bridging the Gap Between Drug Discovery and Clinical Trials
  • Speakers: Todd Palmby, PhD and Marc Theoret, MD
  • Module 2: Clinical Pharmacology: Finding the Right Dose for All Patients
  • Speakers: Julie Bullock, PharmD and Sarah Schreiber, PharmD
  • Module 3: From Activity to Meaningful Clinical Benefit: Considerations for Clinical Development Programs to Support U.S. Marketing Approval
  • Speakers: Paul Kluetz, MD and Tatiana Prowell, MD
12:00 - 1:00pm Lunch
1:00 - 2:30 pm Plenary Session: Key Issues in the Design, Conduct and Analysis of Oncology Trials
Moderator: Thomas Fleming, PhD
  • Challenges in the Use of Biomarkers as Endpoints: Direct and Indirect Measures of Patient Benefit
  • Speaker: John Powers, M.D.; George Washington University
  • Challenges in the Development and Validation of Biomarker-based Tests for Personalized Therapeutic Decision-making in Oncology
  • Speaker: Lisa McShane, Ph.D.; National Cancer Institute
  • Confirmatory vs. Exploratory Analyses in Clinical Trials
  • Speaker: Thomas Fleming, Ph.D.; University of Washington
2:30 - 3:00pm Break
3:00 - 5:00pm Case Studies #1
Case studies are conducted concurrently, and will be repeated on Thursday and Friday. Learners will have the opportunity to attend all three case studies.
  • Gazyva® (obinutuzamab), Genentech
  • Indicated for the treatment of patients with previously untreated chronic lymphocytic leukemia, in combination with Leukerin® (chlorambucil)
  • Gilotrif® (afatinib), Boehringer Ingelheim Pharmaceuticals
  • A kinase inhibitor indicated for the first-line treatment of patients with metastatic non-small cell lung cancer whose tumors have epidermal growth factor receptor exon 19 deletions or exon 21 (L858R) substitution mutations
  • Perjeta® (pertuzamab), Genentech
  • For use in combination with trastuzumab and docetaxel for the neoadjuvant treatment of patients with HER2-positive, locally advanced, inflammatory, or early stage breast cancer
  • Sylvant® (siltuximab), Janssen Biotech, Inc.
  • Indicated for treatment of patients with multicenter Castleman’s disease (MCD) who are human immunodeficiency virus (HIV-) - negative and human herpes virus - 8 (HHV-8) - negative.
5:00 - 6:30pm Welcome Reception

Thursday, May 8

7:00 - 8:00am Fellows and New Investigators Breakfast (advance sign-up required)
An opportunity to discuss career development opportunities in drug development and regulatory sciences with leaders in the academic, corporate and government sectors. Open to fellows and investigators with five years or less of experience in the drug development field.
7:30 - 8:00am Continental Breakfast
8:00 - 9:30am Plenary Session: Patient Reported Outcomes and Patient-focused Drug Development
Moderator: Ethan Basch, MD
  • Defining Patient Benefit
  • Speaker: Ethan Basch, M.D.; University of North Carolina, Chapel Hill
  • Successes and Failures of Patient-reported Outcomes: Industry Perspective
  • Speaker: Ari Gnanasakthy, MSc, MBA; Novartis Pharmaceuticals
  • Current Thinking on the Inclusion of Patient Perspectives
  • Speaker: Paul Kluetz, M.D.; U.S. Food and Drug Administration
  • The PRO Consortium of the Critical Path Institute
  • Speaker: Stephen Coons, Ph.D.; Critical Path Institute
  • Patient Perspective
  • Speaker: Cindy Geoghegan, Patient and Partners LLC
9:30 - 10:00am Break
10:00am - 12:00pm FDA Core Curriculum #2
  • Module 1: Bridging the Gap Between Drug Discovery and Clinical Trials
  • Speakers: Todd Palmby, PhD and Marc Theoret, MD
  • Module 2: Clinical Pharmacology: Finding the Right Dose for All Patients
  • Speakers: Julie Bullock, PharmD and Sarah Schreiber, PharmD
  • Module 3: From Activity to Meaningful Clinical Benefit: Considerations for Clinical Development Programs to Support U.S. Marketing Approval
  • Speakers: Paul Kluetz, MD and Tatiana Prowell, MD
12:00 - 1:00pm Lunch
1:00 - 3:00pm Case Studies #2
  • Gazyva® (obinutuzamab), Genentech
  • Gilotrif® (afatinib), Boehringer Ingelheim Pharmaceuticals
  • Perjeta® (pertuzamab), Genentech
  • Sylvant® (siltuximab), Janssen Biotech, Inc.
3:00 - 3:30pm Break
3:30 - 5:00pm In-depth Discussion Groups
Discussion Groups are conducted concurrently. Learners may attend one from the following list. Discussion groups will be closed to sign-up when attendance capacity has been reached.
  • Immune Checkpoint Blockade and Cancer: Therapeutic Advances and Future Strategies
  • Moderator: Louise Perkins, MS, PhD; Melanoma Research Alliance
    Speakers: Renzo Canetta, MD; Bristol-Myers Squibb
    Charles Drake, MD; Johns Hopkins University
  • Regulatory Considerations in the Development of Genetically Engineered Cellular Products
  • Moderator: Mark Velleca, MD, PhD; Leukemia and Lymphoma Society
    Speakers: Bambi Grilley, RPh; Baylor College of Medicine
    Ke Liu, MD; U.S. Food and Drug Administration
    Isabelle Rivière, PhD; Memorial Sloan-Kettering Cancer Center
    Daniel Takefman, PhD; U.S. Food and Drug Administration
  • Master Protocols: Logistical Considerations for Companion Diagnostics and Targeted Combination Therapy
  • Moderator: Marc Theoret, MD; U.S. Food and Drug Administration
    Speakers: Gideon Blumenthal, MD; U.S. Food and Drug Administration
    Patricia Cortazar, MD; U.S. Food and Drug Administration
    Patricia LoRusso, DO; Karmanos Cancer Institute
    Shakun Malik, MD; National Cancer Institute
  • Expanding the Preclinical Evidence for Early Phase Studies of New Targeted Agents in Children: Strategies for Prioritization and Regulatory Considerations
  • Moderator: Meredith Chuk, MD; U.S. Food and Drug Administration
    Speakers: Peter Houghton, PhD; Ohio State University and Nationwide Children’s Hospital
    Gregory Reaman, MD; U.S. Food and Drug Administration
    Malcolm Smith, MD, PhD; National Cancer Institute
    Brenda Weigel, MD; University of Minnesota
  • Improved Strategies for Selecting Recommended Phase 2 Dosing: Moving Beyond Maximum Tolerated Dose
  • Moderator: Julie Bullock, PharmD; U.S. Food and Drug Administration
  • Biomarker Facilitated Drug Development : The Role of Circulating Tumor Cells and Tumor Cell-free DNA
  • Moderator: Julia Beaver, MD; U.S. Food and Drug Administration
    Speakers: Daniel Danila, MD; Memorial Sloan-Kettering Cancer Center
    Sandy Finestone, Susan G. Komen
    Matthias Holdhoff, MD; Johns Hopkins University
    Minetta Liu, MD; Mayo Clinic
    Ben Ho Park, MD, PhD; Johns Hopkins University
5:00 - 7:00pm Networking Reception
6:00pm Advocates Dinner (advance sign-up required)
An overview of the workshop proceedings with emphasis on the role of the advocate in cancer drug development. Open to patient advocates affiliated with advocacy organizations or foundations.

Friday, May 9

7:30 - 8:00am Continental Breakfast
8:00 - 9:30am Plenary Session: Maximizing Efficiencies in Drug Development through Data Sharing
Moderator: Richard Schilsky, M.D.
  • Project DataSphere
  • Speaker: Kald Abdallah, M.D., Ph.D.; Sanofi US
  • GlaxoSmithKline’s Data Sharing Initiative
  • Speaker: Perry Nisen, M.D., Ph.D.; GlaxoSmithKline
  • Responsible Sharing of Clinical Trial Data
  • Speaker: Barbara Bierer, MD; Harvard University
  • Panel Discussion: Ethical Considerations and Regulatory Concerns Panelist
  • Panelist: Patrick Archdeacon, M.D.; U.S. Food and Drug Administration
9:30 - 10:00am Break
10:00am - 12:00pm FDA Core Curriculum #3
  • Module 1: Bridging the Gap Between Drug Discovery and Clinical Trials
  • Speakers: Todd Palmby, PhD and Marc Theoret, MD
  • Module 2: Clinical Pharmacology: Finding the Right Dose for All Patients
  • Speakers: Julie Bullock, PharmD and Sarah Schreiber, PharmD
  • Module 3: From Activity to Meaningful Clinical Benefit: Considerations for Clinical Development Programs to Support U.S. Marketing Approval
  • Speakers: Paul Kluetz, MD and Tatiana Prowell, MD
12:00 - 12:30pm Box Lunches
12:30 - 2:30pm Case Studies #3
  • Gazyva® (obinutuzamab), Genentech
  • Gilotrif® (afatinib), Boehringer Ingelheim Pharmaceuticals
  • Perjeta® (pertuzamab), Genentech
  • Sylvant® (siltuximab), Janssen Biotech, Inc.
2:30pm Workshop Adjourns

Comments are closed.