2012-Program

Accelerating Anticancer Agent Development and Validation Workshop

Bethesda North Marriott Hotel and Conference Center

May 16-18, 2012


Wednesday, May 16

7:00-8:00 am Registration/Continental Breakfast
8:00-8:30 am

Opening Remarks

  • H. Kim Lyerly, M.D.    
  • Gregory Reaman, M.D.
  • Elizabeth Thompson
  • Gregory Curt, M.D.    
8:30-10:00 am

Session I: Lessons from Successes in Oncology
Moderator:  Sandra Swain, M.D.

  • Success in the approval of oncology agents
    Speaker:  Anthony Murgo, M.D.    
  • Potential strategies to accelerate the process:  Industry perspective
    Speaker:  C. Glenn Begley, M.D., Ph.D.    
  • Pre-competitive cooperation using existing databases to develop knowledge
    Speaker: Gregory Curt, M.D.    
10:00-10:30 am Break
10:30-12:30 pm

Session II: Core Curriculum
Modules are conducted concurrently, and will be repeated on Thursday, May 17. Learners attend one module of their choice on each day (total of two).

  • Module 1: Early IND stage drug development: Clinical and non-clinical considerations
    Moderator: John Leighton, Ph.D.
    Speakers: Haleh Saber, Ph.D.     and Marc Theoret, M.D.    
  • Module 2: Clinical pharmacology expectations for drug development
    Moderator: Nam Atiqur Rahman, Ph.D.
    Speakers: Stacy Shord, Pharm.D.    , Gene Williams, Ph.D.
  • Module 3: Considerations for clinical trial design to support marketing approval
    Moderator: Gregory Reaman, M.D.
    • Clinical trial endpoints, SPA, approval mechanisms
      Speaker: Paul Kluetz, M.D.    
    • Pitfalls in drug development
      Speaker: Tatiana Prowell, M.D.    
12:30-1:30 pm Lunch
1:30-3:00 pm

Session III: Pictures of Success in the Development of Drugs with Companion Diagnostics: Panel Discussion
Moderator: Shakun Malik    

  • Xalkori
    Speakers: Shakun Malik, M.D.; Karen Bijwaard, M.D.
  • Zelboraf
    Speakers: Geoffrey Kim, M.D.; Donna Roscoe, Ph.D.
    Panelists: Robert Becker, Jr., M.D., Ph.D.; Elizabeth Mansfield, Ph.D.; Keith Wilner, Ph.D.;
    Robert Justice, M.D.; Patricia Keegan, M.D.; Richard Pazdur, M.D.
3:00-3:30 pm Break
3:30-5:30 pm

Session IV: Case Studies
Case studies are conducted concurrently, and will be repeated on Friday, May 18. Learners attend one case study of their choice on each day (total of two)

  • HALAVEN - Eisai Pharmaceuticals (eribulin mesylate)    
    Eisai and FDA will discuss key issues that arose during the development of eribulin mesylate for the treatment of patients with refractory metastatic breast cancer. Topics will include: overcoming the challenges to discovery and synthesis of a complex molecule originally derived from a sea sponge, advantages and potential pitfalls of the "treatment of physician's choice" comparator, and overall survival (OS) versus progression-free survival (PFS) as a primary endpoint.
    Eisai Team FDA Team
    • Tushar Kokate, Ph.D.
    • Alton Kremer, M.D.
    • Michael Lewis, Ph.D.
    • Annmarie Petraglia, Ph.D.
    • Larisa Reyderman, Ph.D.
    • Martha Donoghue, M.D.
    • Steven Lemery, M.D.
    • Anne Pilaro, Ph.D.
    • Stacy Shord, Ph.D.
    • Weishi Yuan, Ph.D.
  • JAKAFI - Incyte Corporation (ruxolitinib phosphate)    
    This case study will present various issues in the drug development program for Jakafi. Topics will include drug class; unique clinical pharmacology aspects; pivotal trial designs to evaluate the effect of Jakafi on myelofibrosis; the role of the phase 1/2 trial of Jakafi in setting the stage for the phase 3 trials; the use of the SPA mechanism; use of novel endpoints (spleen size reduction and Patient Reported Outcomes); and successful method of PRO data collection
    Incyte Team FDA Team
    • Ronald Falcone, Ph.D.
    • Victor Sandor, M.D.
    • Lothar Tremmel, Ph.D.
    • Swamy Yeleswaram, Ph.D.
    • Kris Vaddi, Ph.D.
    • Neil Wummer
    • Wei Chen, Ph.D.
    • Albert Deisseroth, M.D.
    • Kyung Lee, Ph.D.
    • Satjit Brar, Ph.D.
  • XALKORI - Pfizer Pharmaceuticals (crizotinib)    
    Pfizer and the FDA will focus on the development of Xalkori for the treatment of patients with ALK-positive advanced non-small cell lung cancer. The session will focus on the communications and co-ordination amongst Center for Drug Evaluation and Research (CDER), Center for Devices and Radiological Health (CDRH), Pfizer and Abbott that led to the development and then approval of the drug in tandem with a companion in vitro diagnostic test.
    Pfizer Team FDA Team
    • Erling Donnelly, Ph.D.
    • Diane Matsumoto, Ph.D.
    • Hakan Sakul, Ph.D.
    • Patrick Schnell, M.D.
    • Paulina Selaru, M.S.P.H.
    • Weiwei Tan, PhD
    • Keith Wilner, PhD
    • Karen Bijwaard, M.D.
    • Brenda Gehrke, Ph.D.
    • Shakun Malik, M.D.
    • Pengfei Song, Ph.D.
    • Shenghui Tang, Ph.D.
  • ZELBORAF - Roche Pharmaceuticals (vemurafenib)    
    In this case study FDA and Roche will discuss key issues that arose during the development of vemurafenib for the treatment of patients with unresectable or metastatic melanoma with BRAFV600E mutation. Topics will include: development of a companion diagnostic, evaluation of drug efficacy in a "test-negative" population, management strategies of unexpected toxicities, and what to do when things go right.
    Roche Team FDA Team
    • Angelique Braen, PhD, DABT
    • Linda Burdette, PhD
    • Suzanne Cheng, PhD
    • Lesley Farrington
    • Joseph Grippo, PhD
    • Andrew Joe, MD
    • Matthew Klimek, PharmD
    • Betty Nelson
    • Keith Nolop, MD
    • Fei Su, PhD
    • Robeena Aziz, Ph.D.
    • Jeanne Fourie Zirkelbach, Ph.D.
    • Geoffrey Kim, M.D.
    • William McGuinn, Ph.D.
    • Donna Roscoe, Ph.D.
    • Qiang Xu, Ph.D.
  • ZYTIGA - Janssen Biotech ( abiraterone acetate)    
    Janssen Biotech and FDA will discuss key opportunities and challenges encountered during the development of Zytiga (abiraterone acetate) which culminated in its approval. It is indicated in combination with prednisone for the treatment of men with castration-resistant prostate cancer who have received prior chemotherapy with docetaxel. Topics will include: early phase development, FDA-sponsor interaction, SPA agreements, food effect studies, and safety monitoring of pivotal trials.
    Janssen Team FDA Team
    • Milin Acharya, Ph.D.
    • Robert Charnas, Ph.D.
    • Thian Kheoh, Ph.D.
    • Michael Meyers, M.D.
    • Arturo Molina, M.D., M.S.
    • Johan Monbaliu, Ph.D.
    • Kelly Johnson Reid, M.S.
    • Robeena Aziz, Ph.D.
    • Paul Kluetz, M.D.
    • Yangmin Ning, M.D.
    • Elimika Pfuma, Ph.D.
    • Haleh Saber, Ph.D.
    • Howard Soule, Ph.D.
    • Lijun Zhang, Ph.D.
5:30-8:00 pm

Reception/Dinner/Dinner Panel
“Financing Oncology Drug Development: The Changing Landscape”
Panelists: Josh Bilenker, M.D.   ; Anthony Fiorino, M.D., Ph.D.   ; Donald Light, M.S., Ph.D.   

   

Thursday, May 17

7:30-8:00 am Continental Breakfast
8:00-9:30 am

Session V: Monitoring Success
Moderator: Gregory Curt, M.D.

  • Limiting patient access to new agents based on emerging safety and efficacy profiles
    Speaker: Scott Patterson, M.D., Ph.D.   
  • Access and expanded access
    Speaker: Gerard Kennealey, M.D.   
  • Drug safety and pharmacovigilance in the setting of oncology drugs
    Speaker: Patrick Schnell, M.D.   
9:30-10:00 am Break
10:00 am -12:00 pm

Session VI: Core Curriculum
A repeat of Wednesday's modules. Learners should choose one module from the following list that is different from Wednesday's choice.

  • Module 1: Early IND stage drug development: Clinical and non-clinical considerations
    Moderator: John Leighton, Ph.D.
    Speakers: Haleh Saber, Ph.D.    and Marc Theoret, M.D.   
  • Module 2: Clinical pharmacology expectations for drug development
    Moderator: Nam Atiqur Rahman, Ph.D.
    Speakers: Stacy Shord, Pharm.D.   , Gene Williams, Ph.D.
  • Module 3: Considerations for clinical trial design to support marketing approval
    Moderator: Gregory Reaman, M.D.
    • Clinical trial endpoints, SPA, approval mechanisms
      Speakers: Paul Kleutz   , M.D., Tatiana Prowell, M.D.
    • Pitfalls in drug development
      Speaker: Tatiana Prowell, M.D.   
12:00-1:00 pm Lunch
1:00-2:30 pm

Session VII: Critical Issues for Pivotal Trials
Moderator:  Jeffrey Abrams, M.D.

  • Assuring the applicability of foreign clinical trials to U.S. patients
    Speaker: Jeffrey Humphrey, M.D.   
  • Accrual of under-represented minorities
    Speaker: Worta McCaskill-Stevens, M.D., M.S.    
  • The engagement of U.S. NCI-supported cooperative groups: Update on structure/reorganization
    Speaker: Meg Mooney, M.D., M.B.A.   
2:30-3:00 pm Break
3:00-5:00 pm

Session VIII: In-depth Discussion Groups

  • Developing active immunotherapy agents
    Discussion Leader: Peter Bross, M.D.   
  • The co-development of companion diagnostics
    Discussion Leader: Elizabeth Mansfield, M.D.   
  • Drug development in rare cancers
    Discussion Leaders: Katherine Thornton, M.D.    
    Speakers: Ann Pariser, M.D.   ; Gwynn Ison, M.D.    ;Albert Deisseroth, M.D.   
  • Innovative trial designs and endpoint considerations for pediatric cancer drug development
    Discussion Leader: Frank Balis, M.D.   
  • How patient-reported outcomes are used in oncology drug development
    Discussion Leaders: Elizabeth Thompson, Ethan Basch , M.D.   ;
    Brandon Hayes-Lattin, M.D.; Lori Minasian, M.D.   ; Howard Soule,Ph.D.; Virginia Kwitkowski, M.S., R.N., ACNP-BC
  • Evolving global considerations in cancer drug development
    Discussion Leaders: Toshio Miyata, M.D. (Japan)   ; Iordanis Gravanis, M.D., Ph.D. (EMA), Naoko Takebe, M.D.(NCI), Yuhang Zhao, Ph.D. (China), W. Wictor Jedrzeczak, M.D.   
5:00-6:30 pm Reception
   

Friday, May 18

7:30-8:00 am Continental Breakfast
8:00-10:00 am

Session IX: Case Studies
A repeat of Wednesday's case studies. Learners should choose one case study from the following list that is different from Wednesday's choice.

  • HALAVEN - Eisai Pharmaceuticals (eribulin mesylate)    
    Eisai and FDA will discuss key issues that arose during the development of eribulin mesylate for the treatment of patients with refractory metastatic breast cancer. Topics will include: overcoming the challenges to discovery and synthesis of a complex molecule originally derived from a sea sponge, advantages and potential pitfalls of the "treatment of physician's choice" comparator, and overall survival (OS) versus progression-free survival (PFS) as a primary endpoint.
    Eisai Team FDA Team
    • Tushar Kokate, Ph.D.
    • Alton Kremer, M.D.
    • Michael Lewis, Ph.D.
    • Annmarie Petraglia, Ph.D.
    • Larisa Reyderman, Ph.D.
    • Martha Donoghue, M.D.
    • Steven Lemery, M.D.
    • Anne Pilaro, Ph.D.
    • Stacy Shord, Ph.D.
    • Weishi Yuan, Ph.D.
  • JAKAFI - Incyte Corporation (ruxolitinib phosphate)    
    This case study will present various issues in the drug development program for Jakafi. Topics will include drug class; unique clinical pharmacology aspects; pivotal trial designs to evaluate the effect of Jakafi on myelofibrosis; the role of the phase 1/2 trial of Jakafi in setting the stage for the phase 3 trials; the use of the SPA mechanism; use of novel endpoints (spleen size reduction and Patient Reported Outcomes); and successful method of PRO data collection
    Incyte Team FDA Team
    • Ronald Falcone, Ph.D.
    • Victor Sandor, M.D.
    • Lothar Tremmel, Ph.D.
    • Swamy Yeleswaram, Ph.D.
    • Kris Vaddi, Ph.D.
    • Neil Wummer
    • Wei Chen, Ph.D.
    • Albert Deisseroth, M.D.
    • Kyung Lee, Ph.D.
    • Satjit Brar, Ph.D.
  • XALKORI - Pfizer Pharmaceuticals (crizotinib)    
    Pfizer and the FDA will focus on the development of Xalkori for the treatment of patients with ALK-positive advanced non-small cell lung cancer. The session will focus on the communications and co-ordination amongst Center for Drug Evaluation and Research (CDER), Center for Devices and Radiological Health (CDRH), Pfizer and Abbott that led to the development and then approval of the drug in tandem with a companion in vitro diagnostic test.
    Pfizer Team FDA Team
    • Erling Donnelly, Ph.D.
    • Diane Matsumoto, Ph.D.
    • Hakun Sakul, Ph.D.
    • Patrick Schnell, M.D.
    • Paulina Selaru, M.S.P.H.
    • Weiwei Tan, PhD
    • Keith Wilner, PhD
    • Karen Bijwaard, M.D.
    • Brenda Gehrke, Ph.D.
    • Shakun Malik, M.D.
    • Pengfei Song, Ph.D.
    • Shenghui Tang, Ph.D.
  • ZELBORAF - Roche Pharmaceuticals (vemurafenib)    
    In this case study FDA and Roche will discuss key issues that arose during the development of vemurafenib for the treatment of patients with unresectable or metastatic melanoma with BRAFV600E mutation. Topics will include: development of a companion diagnostic, evaluation of drug efficacy in a "test-negative" population, management strategies of unexpected toxicities, and what to do when things go right.
    Roche Team FDA Team
    • Angelique Braen, PhD, DABT
    • Linda Burdette, PhD
    • Suzanne Cheng, PhD
    • Lesley Farrington
    • Joseph Grippo, PhD
    • Andrew Joe, MD
    • Matthew Klimek, PharmD
    • Betty Nelson
    • Keith Nolop, MD
    • Fei Su, PhD
    • Robeena Aziz, Ph.D.
    • Jeanne Fourie Zirkelbach, Ph.D.
    • Geoffrey Kim, M.D.
    • William McGuinn, Ph.D.
    • Donna Roscoe, Ph.D.
    • Qiang Xu, Ph.D.
  • ZYTIGA - Janssen Biotech ( abiraterone acetate)    
    Janssen Biotech and FDA will discuss key opportunities and challenges encountered during the development of Zytiga (abiraterone acetate) which culminated in its approval. It is indicated in combination with prednisone for the treatment of men with castration-resistant prostate cancer who have received prior chemotherapy with docetaxel. Topics will include: early phase development, FDA-sponsor interaction, SPA agreements, food effect studies, and safety monitoring of pivotal trials.
    Janssen Team FDA Team
    • Milin Acharya, Ph.D.
    • Robert Charnas, Ph.D.
    • Thian Kheoh, Ph.D.
    • Michael Meyers, M.D.
    • Arturo Molina, M.D., M.S.
    • Johan Monbaliu, Ph.D.
    • Kelly Johnson Reid, M.S.
    • Robeena Aziz, Ph.D.
    • Paul Kluetz, M.D.
    • Yangmin Ning, M.D.
    • Elimika Pfuma, Ph.D.
    • Haleh Saber, Ph.D.
    • Howard Soule, Ph.D.
    • Lijun Zhang, Ph.D.
10:00-10:30 am Break
10:30 am-12:00 pm

Session X: Surrogate Endpoints to Predict “Clinical Benefit”
Moderator: Thomas Fleming, Ph.D.

  • Challenges in the use of biomarkers as endpoints in phase 3 trials
    Speaker: Thomas Fleming, Ph.D.   
  • FDA Perspective: Pathologic CR in neoadjuvant breast cancer
    Speaker: Patricia Cortazar, M.D.   
  • Changing the landscape of progression-free survival as a primary endpoint
    Speaker: Rajeshwari Sridhara, Ph.D.   
12:00 pm Adjourn