Patients

Sponsored by

Track 3
Friday, March 9, 2012

Session 1
Taking Matters into Their Own Hands:
The Role of Patients in Drug Development
9:45AM – 11:00AM

In major world markets, patients have emerged as a significant power in drug development, approval, reimbursement and communication. Patient organizations are playing a critical role in many cases, from co-creation of innovative therapies and delivery systems, to faster approval and wider reimbursement.

For biopharma companies, a successful business model now relies on involving patients and advocacy groups in each stage of development: joint drug development and testing (injectable biologics such as Novo Nordisk’s insulin reached global success due in part to patient-friendly pens); collaboration in speeding approval from regulators, such as the role of patient groups in the fast approval of Novartis’s leukemia drug, Gleevec; understanding of patients’ experience of disease, such as Sanofi Genzyme’s close links with families of patients with Gaucher disease and other orphan diseases.

This panel representing innovating biopharma companies, leading foundations/advocacy groups and key industry experts will explore the role that disease foundations and patient advocacy groups can and should play and discuss current best practices in successful company-patient collaborations throughout the value chain.

Moderator

Chris Garabedian
President and CEO, AVI BioPharma

Panel Speakers

Robert Fisher, M.D., Ph.D.
Scientific Advisory Board Member, The Epilepsy Therapy Project
Professor of Neurology and Director
Stanford Comprehensive Epilepsy Center, Stanford University

Allison Formal
Vice President, Research Business Development
Leukemia & Lymphoma Society

Michael G. Kelly
Chief Scientific Officer
Cure Duchenne

Diane Stephenson, Ph.D.
Associate Director, Coalition Against Major Diseases Critical Path Institute

Session 2
Patient Access to Developmental Drugs
11:30AM – 12:45PM

This session explores proposed regulatory innovation in the area of life-threatening diseases that have unmet medical need. Since the 1962 tightening of evidentiary standards for safety and efficacy, FDA has struggled to balance benefit and risk in a way that helps patients in need of treatment. Our one size fits all approach to drug approval has not allowed individuals to express their own preferences for risk versus potential health improvement, especially in deadly diseases for which no approved treatments are effective and the only chance at survival may lie with new investigational drugs.

The panel session considers the effectiveness of FDAMA’s Accelerated Approval provisions and whether they have produced a net benefit to terminally-ill patients over the last 15 years. In the last 10 years, several initiatives for more meaningful progressive regulatory approval have been argued in Congress and in the Judiciary, including parallel-track and class-level authorization. We will examine these initiatives and discuss new draft legislation proposed by BIO and by Senator Kay Hagan (D-NC). The implications are not just human lives, but also dramatic reduction in regulatory risk for companies considering development in deadly unsolved diseases.

Moderator

Jess Rabourn
Chief Executive Officer, PCUT BioPartners, Inc.

Panel Speakers

Steven K. Galson, M.D., M.P.H.
Vice President, Global Regulatory Affairs, Amgen, Inc.

Julia Jenkins
Director of Public & Government Relations, The EveryLife Foundation for Rare Diseases
Rare Disease Legislative Advocates

Hans Reiser, M.D.
Senior Vice President, Medical Affairs
Gilead Sciences, Inc.

Jaisim Shah
Chief Business Officer, Elevation Pharmaceuticals

Steven Walker, M.S., P.G.
Co-Founder, The Abigail Alliance for Better Access to Developmental Drugs

700 million of anything is hard to ignore. The exponential growth of social media, with nearly 700 million Facebook users, is well documented with increasing numbers of life sciences companies looking at ways to interact with healthcare providers, patients and consumers through social media. The key question, however, is how do you connect the right people with the right message at the right time?

During this panel discussion you will hear about latest social media developments and trends, limitations and major pitfalls. Learn about key innovators and what experiences might be relevant for your company.