Risk Management and Drug Safety Summit Agenda

DAY ONE: Thursday, Oct. 1, 2009

8:00 a.m. – 8:45 a.m.

Registration and Continental Breakfast

8:45 a.m. – 9:00 a.m.

Welcome and Introduction by Chairpersons

9:00 a.m. – 10:00 a.m.

Risk Management in Drug Development — A European Perspective
Due to the failure of industry, regulators and politicians, the long-term safety of medicines has failed. Industry has failed to adhere to commitments previously given at the time of licensing, and the regulatory bodies have failed to put suitable systems in place. Politicians become engaged only after problems have arisen. Sir Breckenridge will discuss how European risk management programs differ from REMS in the U.S. and suggest that regulators should consider asking for risk-benefit assessment at regular intervals throughout the life cycle of a medicine rather than just risk assessment. He’ll also discuss using agreed-upon frameworks so that different stakeholders can become involved in the debate.

Attendees will learn:
  • The success — and failures — of industry, regulators and policymakers in managing drug safety
  • The key differences between U.S. and EU regulatory schemes and enforcement
  • What emerging standards could be used to assure all stakeholders are in agreement
  • How to use risk-benefit assessment throughout the product life cycle
Sir Alasdair Breckenridge, Chairman, Medicines and Healthcare products Regulatory Agency, UK

10:00 a.m. – 10:30 a.m.

Networking and Refreshment Break

10:30 a.m. – 11:15 a.m.

Risk Management Plans Post-FDAAA — An FDA Perspective
FDAAA has given the FDA enhanced authorities regarding postmarket safety of drugs. Under FDAAA, the FDA may determine if a REMS is needed to ensure that the benefits of the drug outweigh the risks of the drug prior to approval or postapproval if the FDA becomes aware of new safety information. This presentation will address how the FDA is managing the REMS provisions under FDAAA.

Attendees will learn:
  • How RMPs, RiskMAPs and REMS relate
  • Content and format of REMS
  • Examples of REMS
Jane Axelrad, Associate Director for Policy, CDER, FDA

11:15 a.m. – 12:00 p.m.

CDER’s Efforts to Improve Risk Management and Drug Safety
While we have a new administration and new officials in charge of the FDA, the goals of the agency remain the same: oversight of the entire life cycle of drugs — from premarket drug testing and development through drug approval, postmarket surveillance and risk management.

Attendees will learn:
  • How CDER is using additional resources and enforcement authorities to improve postmarket surveillance and drug safety
  • What changes the agency has made in response to the globalization of the industry and international risk management concerns
  • The FDA’s efforts to strengthen the science that supports drug safety at every stage of the drug life cycle
  • Status report on FDAAA and how its implications are still being felt throughout the drug industry
  • How the FDA’s Sentinel Initiative will improve postmarket surveillance and safety
Janet Woodcock, M.D., Director, CDER, FDA

12:00 p.m. – 1:00 p.m.


1:00 p.m. – 1:45 p.m.  

How Do We Know That REMS Are Effective? A Guide to Evaluating Risk Management Strategies
As the first approved REMS approach their assessment timelines, sponsors are determining whether the minimization interventions are working to maintain the benefit-risk balance or if adjustments need to be made in the REMS. This presentation will provide innovative techniques for evaluating REMS such as stakeholder surveys and analyses of external data sources.

Attendees will learn:
  • Best practices for measuring the risk-benefit balance of REMS
  • Conducting stakeholder surveys that yield useful data
  • What external data sources provide insights into REMS effectiveness
Annette Stemhagen, Dr.P.H., FISPE, Senior Vice President, Safety, Epidemiology, Registries & Risk Management, United BioSource Corporation

1:45 p.m. – 2:30 p.m.

Developing, Implementing and Managing Complex REMS: Tysabri Case Study
Due to the rare risk of progressive multifocal leukoencephalopathy (PML) associated with Tysabri (natalizumab), there is an ongoing risk management program to further evaluate the safety of natalizumab — the Tysabri Outreach: Unified Commitment to Health (TOUCH™) Prescribing Program. The TOUCH Prescribing Program is a mandatory prescribing program in the U.S. into which all patients, physicians and infusion centers must enroll prior to initiating natalizumab treatment.

Attendees will learn:
  • Lessons in developing, implementing and managing a complex REMS
  • An update on the status and evolution of the Tysabri REMS
  • The benefit-risk profile of Tysabri
Carmen Bozic, M.D., Vice President and Global Head of Drug Safety and Risk Management, Biogen Idec

2:30 p.m. – 3:15 p.m.

Risk Management Approaches for Class II Opioid Analgesic Drugs
Opioid analgesics have been used with success in pain management for decades but have proven to carry significant risks as well. What are these risks? How can they be managed? Can we preserve patient benefit while protecting public health? This presentation will outline considerations and challenges for the pharmaceutical industry in managing the risks associated with Class II opioid analgesics and the regulatory landscape.

Attendees will learn:
  • The latest from the FDA regarding the Class II opioid REMS requirements
  • Implications from the FDA’s recent public meetings — what’s on the horizon?
  • Finding common ground among industry, regulators, physicians and patients
Juergen Schmider, M.D., Ph.D., Corporate Safety Officer and Vice President Global Pharmacovigilance & Epidemiology, Cephalon

3:15 p.m. – 3:45 p.m.

Networking and Refreshment Break

3:45 p.m. – 4:30 p.m. 

The FDA in the Age of Obama
Discussions of FDA reform are often held without understanding the agency in the proper context. What does “reform” mean? And how will an agency of 11,000 career professionals change (or will it) under a new administration and a new commissioner?

Attendees will learn:
  • How the agency is thinking about constituent communications
  • What “transparency” means and how the FDA will use the concept to change the way it does business
  • Whether “comparative effectiveness” will become the third leg of the drug approval process
Peter Pitts, President, Center for Medicine in the Public Interest

4:30 p.m. – 5:30 p.m.

Managing Benefits and Risks Throughout the Product Life Cycle — Roche’s REMS and NEOS Experiences
The recent emphasis put on RMP/REMS is a formidable incentive to manage in a much more robust way the benefit-risk perspective of medicines whether in development or approved and in use. REMS' consequences for creating and marketing new medicines are important and affect the organizational structure, human resources, key cross-functional processes, methods for running clinical studies as well as analyzing, reporting and governance. This presentation will detail Roche’s significant experience in RMP/REMS programs and a company re-engineering program — New Era of Safety (NEOS).

Attendees will learn:
  • Important gaps and how to address them — understanding the benefit-risk decisionmaking by prescribers and patients
  • Adaptative design for RMP/REMS based on their outcome
  • Best practices for developing transparency with safety governance committees
Philippe van der Auwera, M.D., Ph.D., Global Head of Safety Risk Management and European Qualified Person for Pharmacovigilance, F. Hoffmann-La Roche AG

5:30 p.m. – 7:00 p.m.    

Adjournment and Networking Reception

DAY TWO: Friday, Oct. 2, 2009

8:00 a.m. – 8:45 a.m.   

Continental Breakfast

8:45 a.m. – 9:00 a.m.

Welcome and Introduction by Chairpersons

9:00 a.m. – 10:00 a.m.

Panel Discussion: Developing REMS Early in the Life Cycle — Can Patient Understanding of Risks Be Determined During Clinical Trials?
It has been suggested that REMS should be considered very early in the process — during clinical trials — so it can be tested for effectiveness. For investigational products, companies have been notified that a REMS will be required in complete response letters. That adds three to nine months to the product review cycle. Could valuable time and money be saved by communicating with the FDA during end-of-Phase II meetings and asking if a “practice” REMS can be tested in Phase III? This panel presentation will address if — or when — REMs should be introduced and how. Bring your thoughts and share them with the experts and your colleagues.

Gerald Faich, M.D., M.P.H., FISPE, Senior Vice President, Safety, Epidemiology, Registries & Risk Management, United BioSource Corporation

Juergen Schmider, M.D., Ph.D., Corporate Safety Officer and Vice President, Global Pharmacovigilance & Epidemiology, Cephalon
Sir Alasdair Breckenridge, Chairman, Medicines and Healthcare Products Regulatory Agency, UK
Philippe van der Auwera, M.D., Ph.D., Global Head of Safety Risk Management and European Qualified Person for Pharmacovigilance, F. Hoffmann-La Roche AG

10:00 a.m. – 11:00 a.m.

Panel Discussion: Achieving Predictability and Transparency in REMS
The industry is comfortable with certain aspects of REMS — medication guides and appropriate labeling. But more work is needed, including validating the elements of safe use for tools involving restricted distribution programs to manage patients receiving the drugs, plus detecting safety signals and deciding when to stop administering drugs. While it’s an ongoing science, having predictability and transparency in the system of when tools are actually validated would help in the development of REMS systems as the industry moves forward. Panelists will describe the latest successes — and challenges — to improve predictability and form consensus on validation standards.

Brian Harvey, M.D., Ph.D., Vice President, Regulatory Policy, Sanofi-Aventis U.S.

Peter Pitts, President, Center for Medicines in the Public Interest
Meredith Manning, Partner, Hogan & Hartson
Florence Houn, M.D., Vice President, Regulatory Policy & Strategy, Celgene Corporation
Mark Ammann, Pharm.D., Vice President, Regulatory Affairs, United BioSource Corporation