Thursday, November 5, 2009
Hosted by
7:45am Breakfast Presentation
REMS Turn Two Years Old: “Terrible Two’s” or REMS 2.0?
Gary Slatko, M.D., MBA, Chief Medical Officer, ParagonRx
In September 2007, Congress enacted the FDA Amendment Act granting FDA sweeping new authorities, including the ability to require Risk Mitigation and Evaluation Strategies (REMS) of any product with risks that cannot be adequately addressed by labeling alone. In this session, participants will review both benchmarks from the first two years of REMS and the lessons learned that may help determine whether their company will experience 2010 as a continuation of hard-to-manage REMS or as the year a more predictable REMS 2.0 emerged.
8:45 Organizer’s Welcome and Chairperson’s Opening Remarks
Micah Lieberman, Executive Director, Conferences, Pharmaceutical Strategy Series, CHI
John Ferguson, M.D., Ph.D., Vice President & Global Head, Pharmacovigilance & Medical Safety, Novartis Vaccines & Diagnostics
9:00 Proactive Safety: Life Cycle Risk Management from Cradle to REMS and Beyond
Gary Bloomgren, M.D., MBA, Executive Director, Global Safety, Amylin Pharmaceuticals
Current standards in drug safety require a continuum of risk management efforts to better optimize the understanding of product benefit-risk from the time a product enters clinical development throughout its marketed life. This presentation will principally focus on the developmental RMP and the REMS and how a life-cycle approach to product risk management play a role in timely benefit/risk assessment and product approval. Examples will be shared of practical experiences in this space.
9:30 Proactive Pharmacovigilance: Risk Management during Pre-Market Clinical Development
Elizabeth Garrard, Pharm.D, R.Ph, Chief Safety Officer, Drug Safety Alliance, Inc.
Proactive safety surveillance or “Pharmacovigilance” during pre-market clinical development can be very effective for the identification of safety signals. The role of Pharmacovigilance has traditionally been focused on detection and evaluation of signals for new adverse drug reactions (ADRs) in the post-market phase of the product life-cycle in order to secure early detection and to introduce measures to manage risks. However, there is need to proactively identify and incorporate newer approaches for managing safety information in the clinical trial setting and devising early phase safety mitigation methodologies to address emerging product risks.
Effectively identifying safety signals during clinical development vs. after the product is introduced to market
Implementing the elements of an organized and focused adverse event management plan
Pragmatic approaches to the development of a pharmacovigilance and/or risk management plan to contain and mitigate risk identified during clinical development
10:00 Networking Refreshment Break
10:45 Active Drug Safety Surveillance: A Tool to Improve Public Health
Yola Moride, Ph.D., Associate Professor of Pharmacy, Université de Montréal
Ensuring that drugs have an acceptable safety profile and are used safely is a major public health priority. A shared goal amongst regulators, industry and the public is to assess strategies to increase the speed and predictive value of generating and evaluating safety signals, and to identify next steps to improve the US, Canadian and European systems for identifying and evaluating potential safety signals. With the increasing availability of electronic health data, opportunities have emerged to more accurately characterize and confirm potential safety issues. The gain for public health from a highly coordinated network of population-based databases for active surveillance is great and within reach, although operational questions remain. A collaborative network must create a working definition of a safety signal, screening algorithms, and criteria and strategies to confirm or refute a signal once identified through screening.
11:15 Optimizing and Evaluating Product Safety: Leveraging Patient Registries and Observational Studies
Peggy Schrammel, Vice President, Registries and Post Approval Development, United BioSource Corporation (UBC)
Observational research designs have an established role in evaluating the long-term safety of a product among a large and diverse patient population. Still, special attention must be taken to ensure that observational research designs focus on specific issues of interest and do not turn into data dumping exercises with seemingly limitless scope. Study streamlining, from protocol development through data collection, is essential to focused research and cost containment. Finally, demonstrating the value of observational research beyond those involved in product safety can be a daunting but doable task. This session will highlight:
Real world examples of how observational research supported the long-term safety profile of several drugs and devices;
Relevant issues of study design and streamlined study execution; and,
How observational research can support both product safety and market access goals.
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11:45 Break-Out Discussion Groups and Morning Session Wrap-Up:
Concurrent break-out discussion groups are interactive, guided discussions hosted by a facilitator or set of co-facilitators to discuss some of the more poignant questions facing the industry. Delegates will join a table of interest to them and become an active part of the discussion at hand. It is an informal yet informative format that allows attendees to learn from each other and make some new contacts. To get the most out of this interactive session and format please come prepared to: share examples from your work, vet some ideas with your peers, be a part of group interrogation and problem solving, and, most importantly, participate in active idea sharing.
Table #1:
Current Challenges and Operational Strategies in Execution of Dynamic Registries and Observational Studies
Sean D. Kennedy, MPH, Director, Registry and Observational Studies, Covance Periapproval Services
• What are your most challenging operational obstacles in managing your long - term registry or observational study?
• What are your most effective practices to keep patients and sites actively engaged in your long-term program?
• How are registry and observational studies going to evolve in the future?
Table #2:
Challenges and Opportunities for Outsourcing and Off-shoring Post Approval-Drug Safety
Kamala Rai, M.D., Head-Clinical Development, International Clinical Research Operations, Novartis India Limited
Jill Robinson, R.Ph., M.B.A., Vice President, Global Safety Surveillance & Epidemiology, Wyeth
• What are the key leverage points for effective outsouring of post approval drug safety,
• In a highly regulated environment what are the points to consider when outsourcing or offshoring post approval drug safety
• What could be considered as an optimum and what are the challenges in maintaining status-quo
• What activities of post approval safety is "safe" to outsource?
• How could lean procedures and methods be developed to ensure effective outsourcing?
• How to develop a consistent win-win situation while outsourcing and offshoring post approval drug safety?
Table #3:
Engineering the future of REMS
Mary Mease, R.Ph., M.P.H., Senior Director, Global Medical Affairs, Epidemiology and Outcomes Research, Quintiles (former FDA Science Policy Analyst, CDER)
• Should industry embrace a universal REMS platform?
• Is there added value in having a neutral party oversee the REMS implementation?
• Is storing REMS data in a single location useful? Are there concerns for doing this or reasons why this shouldn’t be done?
• Is there as an opportunity for industry to build bridges with its stakeholders (prescribers and pharmacists) by streamlining the REMS process?
Table #4:
How to Evaluate the Effectiveness of Your Risk Minimization Strategies
Yola Moride, Ph.D., FISPE, Associate Professor of Pharmacy, Université de Montréal; President, International Society for Pharmacoepidemiology (ISPE)
Table #5:
Strategies for Safety Signal Detection
Elizabeth Garrard, PharmD, Chief Safety Officer, Drug Safety Alliance, Inc.
• What are the reasons and available methods and technological tools necessary for integration of pre and post market safety data?
• What are the elements of a robust signal management system?
• What has prompted the recent evolution of signal detection technology?
• What do signal detection technology tools need to bring for the future?
• In-stream vs retrospective signal detection, what are the advantages of being proactive?
Table #6:
Conditional Licensing: What extra real-world data might be required?
John Parkinson, Director, GPRD, Medicines & Healthcare Products Regulatory Agency (MHRA)
• Would a sample based registry be an option- at what level of sample?
• What type of data would you need to collect?
• How long might you need to monitor the registry?
Table #7:
Ensuring DMC Independence
Lukas Makris, Ph.D., Senior VP & Chief Statistician, United BioSource Corporation
• Selecting the Committee
• Assessing and Mitigating Conflicts of Interest
• Determining DMC member compensation
• Identifying the mandate of the DMC and scope of responsibilities
• Best practices in meeting procedures to maintain independence
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12:30pm Networking Luncheon
Pharmacovigilance across the Lifecycle: Integrating Safety into Organizational Culture and Operations
Uwe Maennl, M.D., Ph.D., MBA, Vice President, Global Head Pharmacovigilance, Quintiles
Everybody seems to agree that the concept of integrated lifecycle safety and risk management is where we as an industry are heading. However, turning this concept into reality is where the challenges exist. Through case study examples this presentation will offer attendees practical approaches to bring their organizations further into this effective and necessary 21st Century safety paradigm.
Understanding the new and evolving paradigm of integrated lifecycle safety and risk management; clarify the impact of this holistic view and approach
Breaking down the organization silos; how to gain buy-in from and develop risk communication strategies with internal stakeholders
Developing a system that evolves with and follows a product; operational challenges that come with the integrated approach
1:55 Chairperson’s Opening Remarks
Cherif Benattia, M.D., former Vice President, Pharmacovigilance & Public Health, Vertex Pharmaceuticals, Inc.; CEO, Patient Safety 1st, APhaRC
2:00 Designing and Analyzing a Randomized Control Trial for Detecting and Understanding Safety
Ralph B. D’Agostino, Sr., Ph.D., Professor of Mathematics, Statistics, and Public Health, Boston University
With the plethora of new and old approved drugs the issues of safety for both short term and long term chronic use, have been of critical concern. The standard methods of studying drug safety via registries and spontaneous reporting have come under heavy criticism. The use of randomized control trials (RCTs) have been suggested in a number of settings. We review some of the issues in designing and analyzing these RCTs. Challenges are discussed and experiences from some recent trials are used for illustrations.
Understand the issues in attempting to design an RCT for safety
Discuss end points, retention issues, missing data problems and analyses
2:30 Clinical Trials : Real World : Benefit : Risk – The Data Box
John Parkinson, Director, GPRD, Medicines & Healthcare Products Regulatory Agency (MHRA)
Traditionally clinical trial data and real world data have been kept far apart, even though there is a continuum of activities through Phase 3, licensing and everyday use. Equally if risk minimization is an obvious aim so benefit maximization should be an ideal. These four, clinical trials, real world, benefit and risk are the 4 sides of the data box. Boxing the data is the key to post marketing activities.
3:00 Networking Refreshment Break
3:45 Co-Presentation: Designing an Epidemiologic Research Strategy to Support Risk Management
Nicolle Gatto, Ph.D., Senior Director, Oncology BU Group Head, Epidemiology, Safety and Risk Management, Pfizer, Inc.
Jamie Geier, Ph.D., Associate Director, Epidemiology, Safety and Risk Management, Pfizer, Inc.
A proactive epidemiologic research strategy (ERS) is necessary to effectively manage postapproval risk and to evaluate real-world drug safety. Epidemiology provides a critical contribution to risk management through: Quantifying and assessing identified, potential and theoretical risks related to drug safety throughout the product lifecycle; specific epidemiology sections as required for all new EMEA and FDA filings of Risk Management Plans (RMPs) and Pediatric Investigation Plans (PIPs); designing pre- and post-approval epidemiology studies and ensuring regulatory compliance; and; evaluating education or behavioral risk management interventions. This talk will discuss:
The optimal time for developing an ERS
The elements of an effective ERS and steps for development of each
How to identify scenarios in which post-approval epidemiology studies are necessary
How to plan ahead for post-approval study conduct
Case-studies of effective epidemiology research strategies
4:15 Learn How to Maximize Your Product’s True Value through Registry and Observational Study Data to Ensure Product Safety Awareness for your Consumers
Michael Hill, Project Manager of Registry and Observational Studies, Covance Periapproval Services
In the 21st Century the importance and need for Registry and Observational Study data is more critical. Not only to meet the increased number of surveillance and post marketing requirements required by regulatory agencies, but to also ensure that necessary safety data are collected in unmonitored populations. This presentation will demonstrate the effective use of these types of programs in gathering safety data and ensuring consumer knowledge and awareness about safety concerns. Case studies will be presented for ongoing programs that show demonstrable value of these strategies.
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4:45 Break-Out Discussion Groups and Afternoon Session Wrap-Up:
Concurrent break-out discussion groups are interactive, guided discussions hosted by a facilitator or set of co-facilitators to discuss some of the more poignant questions facing the industry. Delegates will join a table of interest to them and become an active part of the discussion at hand. It is an informal yet informative format that allows attendees to learn from each other and make some new contacts. To get the most out of this interactive session and format please come prepared to: share examples from your work, vet some ideas with your peers, be a part of group interrogation and problem solving, and, most importantly, participate in active idea sharing.
Table #8:
Understanding and Utilizing the Many Emerging and Technology-Enabled Methods to Identify, Elucidate and Assess the Basic Biology of Adverse Events
Jack A. Reynolds, former-Senior Vice President of Research and Development and Worldwide Head of Safety Sciences and Comparative Medicine, Pfizer; Chairman, Drug Safety Executive Council (DSEC)
• How can knowledge of the biology or mechanism of an adverse event impact risk mitigation planning and pharmacovigilance?
• Are the data or information from preclinical safety or basic science credible, reliable and predictive to the degree it can be used for humans?
• How can the identification and implementation of biomarkers derived from this new knowledge be improved to enable enhanced or more precise signal detection?
• When new knowledge of an adverse event is generated from these methods or there are emerging signals, how can the dynamic changes in the benefit/risk profile of a drug be proactively managed in real time to avoid communication frenzies?
Table #9:
"Flu Vaccination" Case-based Discussion: How do we Study Safety in Special Populations such as Children, Pregnant Women, the Elderly?
Sonia Hernández-Díaz, M.D., DrPH, Associate Professor, Epidemiology, Director, Pharmacoepidemiology Program, Harvard School of Public Health
Cherif Benattia, M.D., former Vice President, Pharmacovigilance & Public Health, Vertex Pharmaceuticals, Inc.; CEO, Patient Safety 1st, APhaRC
• Why do we need to worry about them?
• Why are they special?
• How are we studying them now?
• Future directions. Shall we include them in RCT?
Table #10:
Suicidality: Drug Safety and Risk Communication
Kelly Posner, Ph.D., Principal Investigator, Columbia/FDA Classification Project for Drug Safety Analyses, Director of the Center for Suicide Risk Assessment, Columbia University, Child Psychiatry, New York State Psychiatric Institute/Columbia University
Jamie Geier, Ph.D., Associate Director, Epidemiology, Safety and Risk Management, Pfizer, Inc.
Table #11:
Integrating Drug Safety Knowledge Longitudinally Across a Compound's Lifecycle
Michael Forstner, Ph.D., Drug Safety Risk Management, F. Hoffman-La Roche; former Senior Risk Engineer, Zurich Financial Services
• When do you start to think about risk management?
• How do you ensure that data are not forgotten along the way? How to overcome the silo mentality?
• How to avoid extensive post-approval studies?
Table #12:
REMS and How Do We Avoid the Approvable Letter?
Gary Bloomgren, M.D., MBA, Executive Director, Global Safety, Amylin Pharmaceuticals
With FDAAA, REMS have become a common stumbling block in gaining product approval. This forum will be an open discussion of options and issues to consider in anticipating this topic early and effectively.
• What are your general knowledge/experience with implementation of a REMS plan?
• What could one do to better anticipate the need for a REMS?
• What are the barriers to implementing these proactive efforts?
Table #13:
FDAAA and REMS: Separating Risk Assessment and Risk Minimization
Kelly D. Davis, M.D., Vice President, Epidemiology & Risk Management, United BioSource Corporation
• When is a risk minimization strategy appropriate?
• How do Post-marketing Observational Studies fit in?
• How can each of these approaches be leveraged to address overall safety and regulatory concerns?
Table #14:
Physician & Patient Considerations When Designing & Conducting Phase IV Safety Endpoint Trials
Eric Gemmen, MA, Senior Director, Medical Affairs, Epidemiology & Outcomes Research, Quintiles Late Phase
• How to minimize the research workload for physicians so they can practice medicine while conducting research
• How to manage the trial in order to minimize undue risk to the patient
• Design options in post-approval endpoint trials (placebo-controlled, observational cohort, adaptive, non-inferiority, etc.)
• Circumstances under which it is appropriate to stop a phase IV endpoint trial early
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5:30 Networking Cocktail Reception
6:30 Close of Day Two
Day 1 | Day 3
For questions or suggestions about the meeting, please contact:
Micah Lieberman
Executive Director, Conferences
Pharmaceutical Strategy Series
Cambridge Healthtech Institute (CHI)
T: (+1) 541.482.4709
E: mlieberman@pharmaseries.com
For exhibit, partnering and sponsorship information, please contact:
Arnold Wolfson
Business Development Manager
Pharmaceutical Strategy Series
Cambridge Healthtech Institute (CHI)
T: (+1) 781.972.5431
E: awolfson@healthtech.com
For media and association partnerships, please contact:
James Prudhomme
Marketing Manager
Cambridge Healthtech Institute (CHI)
T: (+1) 781.972.5486
E: jprudhomme@healthtech.com