2012 Ultra Orphan Conference Wrap Up

Innovative Approaches for Addressing the Needs of Ultra Orphan Patient Populations: Real Solutions for Today’s Challenges

September 17 - 19, 2012
Chase Park Plaza Hotel St. Louis, MO

Each year we have selected a key theme based on trends and activities of interest to the orphan market. Since 2009 when big pharma made a decision to add more focus to orphan therapies, there has been a lot of discussion around understanding the marketplace and identifying challenges and differences compared to larger markets. We know what the problems are. Let’s talk about solutions.

Centric asks the question: What will be the solutions we talk about 5 years from now?

The “orphan” market is here and it’s moving fast … we are on the doorstep of true personalized medicine. Centric is proud to bring together those of us in the industry to exchange ideas in order to continue to bring life-saving therapies to the patients who desperately need them.

Over 120 industry attendees gathered in St. Louis September 17 – 19 to participate in the 5th anniversary of Centric Health Resources’ “Innovative Approaches for Addressing the Needs of Ultra Orphan Patient Populations” educational series.

Please enjoy the following summaries and video clips from our fifth annual conference focused on real solutions for today’s challenges in the ultra orphan space.

Day 1

Public Policy and Orphan Drugs: Preparing for an Era of Austerity

  • The future of health care reform after the Supreme Court: How the changing climates will (and won’t) affect orphan drugs
  • The new FDA law and the Lake Wobegone Effect: If every drug is above average, will Orphan Drugs still be special?
  • Big Deficits vs. Small Patient Populations: New challenges to premium pricing

Michael McCaughan, President, Prevision Policy, LLC

Watch Video | Download Provision Policy’s User Guide to PDUFA V

Michael McCaughan

Different approaches for different companies – is there value in each?

Thomas Collet, CEO, Phrixus Pharmaceuticals
Alvin Shih, COO, Rare Disease Research Unit, Pfizer, Inc.
Kent Rogers, Vice President, Managed Markets, Acorda Therapeutics
Moderated by: Chris Garabedian, CEO, Sarepta Therapeutics (formerly AVI BioPharma)

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Thomas Collet, Alvin Shih, Kent Rogers, and Chris Garabedian

The Orphan Drug Conundrum – Is it an investment bubble or will patients, and society, truly be better off in the long run?

  • Is the model broken? If so, how did it get that way?
  • What are the worst case and the best case scenarios and how do we avoid the worst?
  • What will have to change to avoid the worst case and is it even possible?
  • How do we not mess this up?

Andrew Curtis, Head of Specialty Care Business Unit External Opportunities & Alliance Management, Pfizer Inc.
Bill Frezza, Fellow, Competitive Enterprise Institute, Boston, MA
John Walsh, President & CEO, Alpha-1 Foundation
Michael McCaughan, President, Prevision Policy, LLC
Matthew Patterson, Entrepreneur in Residence, OrbiMed Advisors
Moderated by: Catherine Angell Sohn, Pharm.D, CLP, President, Sohn Health Strategies

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Andrew Curtis, Bill Frezza, John Walsh, Michael McCaughan, Matthew Patterson, and Catherine Angell Sohn

Problem: Finding patients to support orphan drug development

  • Development efforts can be aided by an understanding of the patient populations, its makeup and rapid deployment
  • Beyond epidemiologic data, increase the awareness within certain populations that are prone to specific genetic conditions, and increase the rate of screening
  • Learn how a fledging patient organization focused its efforts to attract biopharmaceutical development to their disease, and helped support and speed those development efforts.

Solution: There is no single approach, but there are common threads that can help

Daniel Darvish, MD, HIBM Research Group/ARM
Moderated by: Patti Engel, President and CEO, Engage Health

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Patti Engel and Daniel Darvish, MD

Problem: There is a perception that manufacturers can’t talk to patients.

  • What are the regulatory hurdles (pre-market promo, HIPAA)
  • The reward is greater than the risk
  • Solutions on how companies have addressed, balancing risks with safeguards

Solution: Understanding the real guidelines and opportunities available to reach patients through advocacy, patient education, distribution and social media.

Melissa Hogan, Writer, Speaker, Advocate, SavingCase
Barbara Wuebbels, Associate Director for Global Patient Advocacy Program, BioMarin
Mark Krueger, Mark Krueger & Associates, Inc.
Alan Gilstrap, Director, Global Advocacy Development-Cardiovascular, Genzyme
Moderated by: Wendy White, President & CEO, Siren Interactive

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Melissa Hogan, Barbara Wuebbels, Mark Krueger, Alan Gilstrap, and Wendy White

Problem: The review process for rare disease trial design is prohibitive

  • Biomarkers are critical to the acceleration of drug development
  • COPD biomarker qualification consortium and impact on rare lung diseases
  • The value of creating a liaison with the FDA and NIH

Solution: COPD Foundation Biomarker Qualification Consortium (CBQC)

John Walsh, President & CEO, Alpha-1 Foundation

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John Walsh

Problem: Post market surveillance and REMS requirements can be onerous

  • Involving patients can streamline the process & improve understanding
  • Changing the role of the physicians
  • Using patient consent as viral marketing to physicians

Solution: Patient registries done right can ease the pain and deliver value

Kyle Brown, President & CEO, PatientCrossroads

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Kyle Brown

Day 2

The myth of the homogenous orphan market and the reality of disparate futures

  • Review previous, current, and future orphan products: Rituxan... orphan product just like Kalydeco?
  • Examine orphan HTA assessments and foreseeable changes
  • Assess challenges in reimbursing the impending set of “one and done” therapies – not a homogenous group either

Doug Paul, Partner, MME, LLC

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Doug Paul

Conflict-free supply chain models to meet the needs of individual patients

  • What is the cost of conflict and complexity?
  • The value is in creating health, not in moving products
  • The transformation from B2B to B2C

Penny Bemus, VP of Business Development, Dohmen

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Penny Bemus

Genetic Attack on Cancer Shows Promise

  • Patient testimonial
  • Whole genome sequencing-the promise and the challenges

Lukas Wartman, M.D., Instructor of Medicine, Division of Oncology, Washington University School of Medicine

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Lukas Wartman, M.D.

State and Future of the Industry Address – Personalized Medicine

  • The success of personalized medicine depends on its ability to demonstrate value to the healthcare system
  • Understanding the usefulness of personal genetic risk and pharmacogenomic information in health management and clinical decision-making
  • What does this mean for the future of the health care marketplace and “ultra orphan” initiatives?

Greg Simon, Visionary Strategist & Innovative Thinker, formerly SVP Worldwide Policy at Pfizer, Inc.

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Greg Simon

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