2010 Ultra Orphan Conference Wrap Up
Emerging Commercialization Strategies for Addressing the Needs of Ultra-Orphan Patients
Maximize Product Launch and Extend Product Life Cycle: One Patient at a Time!
September 27 – 29, 2010; St. Louis, MO
Over 90 industry professionals attended Centric Health Resources’ 3nd Annual Ultra Orphan Drug Conference. Representatives from manufacturers, consulting firms, specialty pharmacies, venture capital firms, and patient advocacy organizations enjoyed a wide range of topics, knowledgeable speakers, interactive discussions. Thank you to all who attended and participated in the event.
In addition to two days of engaging content, the event kicked-off with a pre-conference workshop and ample opportunity for networking at the welcome reception after the workshop, and a trip to see a Cardinals baseball game after the close of Day 1.
If you were unable to attend, here’s what you missed:
Day 1
Welcome and Opening Remarks
Craig Kephart, President & CEO, Centric Health Resources, Inc.
Big or Small: Drug Development Post-Health Care Reform
Michael McCaughan, Editor in Chief, Prevision Policy, LLC
- What Health Care Reform Does (and Doesn’t) Change About Drug Development
- How FDA Regulation Is Evolving at the Same Time
- Where We Are Headed Next: Big, Diversified Pharma Giants and Small, Patient-Focused Innovators
21st Century Medicine: The Promise and the Problems
Dr. Andy von Eschenbach, Senior Director for Strategic Initiatives, Center for Health Transformation; Former Commissioner for the Food and Drug Administration; and Former Director of the National Cancer Institute, National Institutes of Health
- Identify the radical change in medicine brought about by progress in science and technology
- Discuss the implications for the healthcare delivery system when medicine is personalized, predictive, preemptive and participatory
- Develop the structural and functional changes required across the spectrum of discovery–development–delivery to bring the fruits of R&D to patients
Panel: We all share a common goal of getting drugs to market faster, cheaper, and safer.
Jayson Slotnik, Attorney, Foley Hoag, LLP, Washington, D.C.
Peter Saltonstall, President & CEO, National Organization for Rare Disorders
Michael McCaughan, Editor-in-Chief, Prevision Policy, LLC
Moderated by Wayne Oliver, Vice President and Georgia Project leader, The Center for Health Transformation
- How healthcare reform helps or hurts this effort
- Where is reform going from here, and
- What should companies be doing now to be prepared
The Effect of REMS on Product Commercialization
David A. Galardi, Pharm.D., Commercialization, Apogenics Inc.
- The View from the Top
- Commercialization Changes to Risk, Reimbursement, Distribution and Access
- The Fallacy of REMS Standardization
Patient Panel
Patients and caregivers from the Huntington’s Disease, Duchenne MD, and Alpha-1 Antitrypsin Deficiency communities Moderated by Diane Edquist Dorman, Vice President, Public Policy, National Organization for Rare Disorders
- Hear their journey to diagnosis and the impact of living with a rare disorder
- Types of support that are meaningful
Patient Centered Health Management/REMS 2.0
- Pemphigus Vulgaris and related bullous disorders: Outcomes of a model for improving Ultra-Orphan illness through an individualized Health Management Program.
David Sirois, DMD, PhD, President of the Board of Directors, International Pemphigus & Pemphigoid Foundation (IPPF)
- New models for delivering health management
Doug Carlson, VP Health Management & Quality, Centric Health Resources, Inc.
Selling in a Competitive Ultra-Orphan Disease Market
Bill Zabel, Vice President, U.S. Sales, Talecris Biotherapeutics
- Leveraging distribution to maximize outcomes
- Using patient advocates to raise disease awareness
- Managed care strategies to gain buy-in and support
New Complexities in Pricing Orphan and Ultra-Orphan Drugs
Doug Paul, Partner, MME LLC
- Orphan drugs and their prices are less rare
- Changes in ultra-orphan markets
- Increased competition
- Big pharma enters
- When can lower prices increase volume?
- Downward price migration is no longer a myth
- Where are your patients?
Day 2
Manufacturer Viewpoints and Initiatives
Roger Garceau, M.D., FAAP, Senior Vice President, R&D and Chief Medical Officer, NPS Pharmaceuticals, Inc.
- Why and how investing in the orphan drug marketplace makes sense
- NPS’ strategies for success
- Predictions on how innovation will take place in the rare disease market going forward
Pfizer, Inc.
Ed Mascioli, M.D., Vice President, BioTherapeutics R&D, Orphan and Genetic Diseases, Pfizer, Inc.
- Pfizer's past experience in discovering, developing, and commercializing therapies for rare diseases
- How Pfizer is restructuring to position itself to compete in this marketplace
- The Orphan and Genetic Diseases Research Unit's goals and objectives
Getting the Product to the Patient – Who is taking care of your customer?
Craig Kephart, President & CEO, Centric Health Resources, Inc.
- Articulate your patient strategy
- Aligning incentives with channel partners
- Where to start; tools to help
Reimbursement Implications and Strategies for Distribution Model Options
Dean Erhardt, Principal, D2Pharma Consulting, LLC
- Reimbursement care updates and trends related to orphan drugs
- Reimbursement strategies for exclusive, limited, and open distribution models
- Comparative effectiveness – how and when will it impact orphan drugs?
News & Events
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