HUM-MOLGEN events: Late Phase Drug Development World Europe 2010

09.00 Management of differences (or variations) within the EU guidelines for non-interventional studies

Overview
The EU clinical trials directive (2001/20/EC) and the GCP Directive (2005/28/EC), has permitted stability,
conformity and security in conducting interventional studies. Yet, the directive only governs interventional
studies. This interactive workshop will present you with facts and challenges about the issues confronted while setting up an observational study, a chart review or a registry throughout Europe. Attendees will come away from this workshop with practical and usable information to share with their product teams.

The workshop will begin at 09:00 and end at 16:00. Lunch and refreshments will be provided.

Workshop content

The management and conduct of post-authorisation safety-type NIS
Country-Specific Considerations for Conducting

Non-Interventional Studies

Legal and Ethical Issues in Non-Interventional Studies
Criteria to Include in all NIS to Ensure Compliance
Reporting and Publication Obligations: What Do the Authorities Want
Patient Information and Declaration of Content, Patient Assuranc
Data Protectio
New Regulations on Non-Interventional Human Studies Expected
Recommendations for Conducting NIS in Practice
Discussion: key considerations for conducting NIS
Identifying key decision points during NIS development
Establishing a guideline for conduction of NIS

Your workshop leaders

- Hazel Wohlfahrt, Executive Director, EU Clinical Operations, UBC
- Jess Sohal, Executive Director, EU Clinical Operations, UBC

This workshop will be conducted by United BioSource Corporation leaders. UBC, United BioSource Corporation, is a global pharmaceutical services organisation that combines deep scientific knowledge with broad execution expertise across the entire lifecycle continuum. Our focus is on generating real-world data to support the development and commercialisation of medical products for emerging and established life science companies.

Day One Wednesday 1st December 2010

09.00 Opening remarks from the chair

Jeff Trotter, Executive Vice President - Phase IV Development, PharmaNet, United States

INDUSTRY STRATEGIC DRIVERS

09.15 KEYNOTE: Regulatory approval is only one hurdle on the path to market access: challenges and answers to meet multiple stakeholders’ needs in later stage drug development

Successful market access requires careful consideration of multiple stakeholders’ needs & requirements during drug
development, including patients, HCPs, regulators, HTAs,
payers and governments
Demands on demonstration of value and efficacy are not
harmonized and often require national measures in addition to
complex global developments
Some of the challenges and GSK’s approaches will be
discussed

Dr Tony Hoos, Senior Vice President, European Medical Affairs, GlaxoSmithKline, United Kingdom

09.45 Measuring health outcomes for regulatory decision-making: resolving the conflicting needs of clinicians and economists

Guidance issued by agencies including NICE requires the values of the general public are used to measure health benefits but FDA pronouncements on the use of PROs contradicts this
Clinical trials designed to establish evidence of safety and efficacy may not be the appropriate mechanism for testing cost-effectiveness
Decisions driven by social preferences are unlikely to adequately represent “real” benefits to “real” patients
It is time to question the dominant influence of health economists in determining choices in measuring health outcomes

Professor Paul Kind, Professor Centre for Health Economics Alcuin College, University of York, United Kingdom

10.15 Speed networking followed by morning refreshments

POST APPROVAL STUDIES

11.15 Pharmacovigilance and risk management in a post-approval setting

Managing risk effectively in late phase studies

Dirk Teuwen, Vice President and head of Global Clinical Safety & Pharmacovigilance, UCB Group, Belgium

11.45 Patient management in non-interventional studies

Planning and regulations
EC and data protection
Diaries & PROs
Patient follow-up

Andrea Spannheimer, Vice President, i3 Innouvs Late Phase - International, United Kingdom

12.15 CASE STUDY: The future of IIIb/IV studies post Avandia

Post Avandia: IIIb & IV studies will be key to the proper management of the iterative stewardship of comparative benefit risk of marketed products
Review of task force addressing management of local phase IV studies
Lessons learnt: complexity, the need for strengthening processes and transparency

Prof Philippe Van der Auwera, Global Head of Safety Risk Management and EU-QPPV, F.Hoffmann-La Roche Ltd, Switzerland

ENABLING TECHNOLOGIES IN LATE PHASE DEVELOPMENT

12.45 Using web-based capture in late phase research and patient registries

Working with naive sites / researchers and general practice environments
Enabling patients to use the web for PRO
Patient registries
Use of technology in emerging and global markets
Using standards for efficient regional PMS studies

Mr Scott Dixon, Vice President, Global CRO Partnerships, Phase Forward, United States

13.15 Lunch

14.15 Opening remarks from the chair

Krista Payne M.Ed., Director and Research Scientist, Health Care Data Capture, UBC

14.20 Practical approaches to using EDC in late phase studies

Discuss practical approaches to implementing EDC
Address common challenges in designing post-approval studies
Illustrate best practices via several case studies

Patrick Chassaigne, Director, Late Phase Solutions, Medidata Solutions Worldwide, United States

DEMONSTRATING VALUE IN THE LATE PHASE: REGULATORY AND POLICY SETTING

14.50 Challenges faced in meeting HTA requirements

Requirements for ongoing health outcomes data following regulatory approval, to support health technology appraisal and reimbursement
Research requirements and opportunities across the late phase as HTA bodies request increasing amounts of data and regularly re-review the reimbursement/access status of medicines

Dr Bryan Johnstone, Senior Director, Global Health Outcomes, Eli Lilly and Company, United States

15.20 The Right Evidence, Using the Right Design, and the Right Tools

Multi-national Retrospective Chart Review, Cross-sectional and Prospective Study Designs
Research aims achieved, challenges encountered, and lessons learned

Krista Payne M.Ed., Director and Research Scientist, Health Care Data Capture, UBC

15.50 Afternoon refreshments

16.20 CASE STUDY: Late phase research and health outcomes/health economics

The need: why generating the data? What type of data is needed?
The means: generating the data: type of studies
The solution: using the data to support value proposition
Case studies: challenges and success stories

Mr Jorge Arellano, Director iHE, Evidence Generation, Amgen, United Kingdom

16.50 The inclusion of late phase studies in health technology assessment: a perspective of NICE

Use of evidence based information in the decision making process
NICE recommendations and roll out

Zoe Garrett, Technical Adviser (Centre for Health Technology Evaluation), NICE, United Kingdom

17.20 Regulatory expectations for phase IIIb/IV research

Update: regulatory environment surrounding late phase studies

Speaker to be announced

17.50 Evening drinks reception

Day Two Thursday 2nd December 2010

09.00 Opening remarks from the chair

Hani Zaki, Senior Vice President, Late Phase Services, PRA International, United States

COMMERCIAL AND MARKETPLACE ENVIRONMENT

09.10 Effective planning and management to maximize value in long-term post-marketing programs

Start-up strategies to build momentum, engagement and collaboration
Planning across multiple studies, multiple stakeholders, and multiple objectives
Addressing efficiencies and creating opportunities to maximize ROI

Maria Harrison, Global Head, PRA International, United States

09.40 Observational studies in the late phase

How do we leverage observational studies to satisfy a range of competing needs?
What are the competing design options, and how do we select between them?
Leveraging e-Health records in observational studies: prospectively and retrospectively
Late phase lifecycle management

Andrew Roddam, Head International Epidemiology, Amgen, United Kingdom

10.10 Observational research: areas of consensus and divergence

The results from a recent survey on observational research will be presented
What operational concerns and expectations do sponsors have for observational studies?
What challenges need to be addressed to optimise observational research?

Jeff Trotter, Executive Vice President - Phase IV Development, PharmaNet, United States

10.40 Morning refreshments

11.10 Register-based research: a goldmine for late phase drug development

Increasing demands for health economic documentation
Conditional approvals may be more common
Traditional RCTs have insufficiencies
Health registries may give population-based real-life information
Nordic countries have excellent and valid health registries

Dr Olav Flaten, Medical Director, GlaxOSMithKline, Norway

11.40 Use of registries, claims data bases and EMRs in assessing safety, co morbidities and treatment profiles in oncology patients

Specific database needs related to the evaluation of safety profiles, co morbidities, and treatment patterns
Availability and selection of databases
Examples: European and US databases
Examples: specific types of safety issues and treatment pattern studies

Jerzy Tyczynski, Director, Epidemiology, Abbott, United States

12.10 CASE STUDY: Registries, PASS Requirements and REMS: one company’s experience

Follow the course of marketing a drug for a rare and often fatal disease with known liver effects and potential teratogenicity
Authorisation under exceptional circumstances in EU; PASS required; successful outcome
Authorisation under sub-part H in US; drug registry required, RiskMAP and REMS
Registries for new indications; disease instead of drug

Dr Eleanor Segal, Vice President, Medical Safety Officer, Actelion Pharmaceuticals, United States

12.40 PANEL SESSION: The value of EHR as a tool in observational research

How will EHRs change the future of global research studies?
Do studies which collect patient data directly from EHRs offer a cost effective and logistically viable alternative to the traditional methods of data collection?

Mr Gary Coward, Senior Portfolio Director, Parexel International, United States

13.10 Lunch

OPERATIONAL CONSIDERATIONS

14.10 Highly cost effective clinical trials

Randomisation at point of care offers great potential
Real world nature of studies important
Large numbers possible

Dr John Parkinson, Director, GPRD, MHRA, United Kingdom

14.10 Publication planning for late phase research

Changing environment around the publishing of industrysponsored research and the importance of compliance
Importance of early publication planning for late-phase clinical research
How to maximise value from the data generated through
publishing in peer-reviewed journals

John P Gonzalez, Global Skills Lead – Publications, AstraZeneca, United Kingdom

14.40 Comparative effectiveness research in the post approval setting

What is CER?
Designing the right study for the right questions
Minimizing the impact of bias

Dr Amy Sing, Associate Group Director - Post Marketing, Genentech, Inc, United States

15.10 Medical communication in late phase studies

Different nature of communications for phase IIB/IV studies, where the priority is to bring the product closer to clinical practice and therefore the customer is primarily the clinician/ patient and not the regulator
Ethical obligation for transparency, sharing resultsboth
positive and negative: the GSK experience

Dr Emilio Arbe, Senior Director of Clinical Research, Stiefel, a GSK company, United Kingdom

15.40 Afternoon refreshments

16.40 CASE STUDY: Case study in CNS clinical research: integrating policy, health economic and clinical outcomes

Focus on an ongoing schizophrenia comparative effectiveness trial, a study of an antipsychotic medication
Incorporation of economic outcomes and the pragmatic aspects of the study

Dr Joe Hulihan, Vice President, Medical Affairs, CNS, Ortho-McNeil Janssen Scientific Affairs, LLC, United States

17.10 CASE STUDY: Managing safety risk in late phase development in Metabolism

Ralph Schimmer, Global Head Drug Safety Sciences - Metabolism and Cardiovascular, F.Hoffmann-La Roche Ltd, Switzerland

Post conference workshop: Friday 3rd December 2010

09.00 Designing, implementing and managing successful patient registries; delivering expected output

Overview
This workshop will provide the participants with useful insights and recommendations based on own experience from industry and CROs and will focus on;

How to maximize the value of your patient registry
How to avoid some common hurdles and pitfalls

The workshop will start at 9am and will be finished by 4pm and lunch and refreshments will be provided.

Workshop content

The contribution and value of patient registries depending on scope; case examples
Defining the scope of the registry; interactions with stakeholders to assure the registry fulfils their demands
Defining the optimal design and duration of the patient registry depending on desired outcome
How to assure you have the needed criteria defined to efficiently select optimal vendor for data capture
Legal and regulatory considerations
Considerations regarding registry governance, data ownership and the use of data for both regulatory and scientific publications
Implementing a patient registry to optimal operate during long term follow up despite busy clinical routine practice; data quality and completeness
Analyzing registry data, how to avoid some common hurdles

Your workshop leader
- Elizabeth Hernberg-Ståhl, Senior Consultant, Late Phase Solutions Europe AB

Late Phase Solutions Europe AB is an independent consultancy focusing on providing strategic and operational guidance, project management and operations for implementing and managing late phase drug development activities e.g. patient registries. Late Phase Solutions Europe provides you with more than 20 years of international experience from the pharmaceutical industry in medical affairs/late phase activities.