2011 Ultra Orphan Conference Wrap Up

Addressing the Needs of the Ultra Orphan Patient Populations by Creating and Measuring Value: Strategies for Delivering Outcomes and Value for Patients, Physicians, Payers and Manufacturers

September 19 – 21, 2011; St. Louis, MO

The primary key to a successful drug launch is creating value. The world needs therapies that create value through lower costs and higher quality. Orphan drugs can be very effective, but are almost always so expensive that overlooking their intrinsic value is easy to do. Therefore, defining, delivering, and measuring value post-launch becomes a critical component to any orphan drug strategy. How value is defined depends on who is asked.

What is the value of therapy and support programs to patients, payers, physicians, manufacturers, to the healthcare system, and society -- and why should anyone care?

Over 120 industry attendees gathered in St. Louis September 19 – 21 at Centric Health Resources’ 4th annual Ultra Orphan conference focused on defining, creating, and measuring value. The theme was consistent throughout all of the speakers, who presented opinions, observations, research, challenges, and solutions all around the topic of economic and clinical value in orphan markets. The attendees were challenged with taking away at least one idea that their teams can implement now. Here are a few ideas that were shared by several conference attendees:

  • “Quantifying economic impact of a disability, measured before and after initiation of treatment, measured by frequency of injury, consumption of pain drugs, and days missed from work/school.”
  • “Develop a positive dossier that addresses value to the patient and not financial component such as reduced opioid usage, fewer pain medications used, depression, days missed from work.”
  • “Work more closely with advocacy to understand true value to patient and caregiver as opposed to standard outcomes measures. Incorporate the outcomes into a patient registry that would track outcomes/value over time in combination with other factors affecting care.”
  • “Use specialized nurses to administer and report long term outcomes and compliance issues, measured by adherence over time and quality of life improvement.”

Please enjoy the following summaries and video clips from our fourth annual conference focused on strategies for delivering outcomes and value for patients, physicians, payers, and manufacturers.

Day 1

The Politics of Drug Pricing

Michael McCaughan, Editor in Chief, Prevision Policy, LLC

Michael McCaughan

Back by popular demand for the fourth year in a row, Michael McCaughan of Provision Policy started our value quest at the societal level, stating that “orphan drugs are a public policy concept.” This is not a market. Society has already assigned a value by passing the orphan drug legislation. However, he advised, manufacturers should avoid the mistake of thinking that if everyone else is selling orphan drugs for $200,000/year, “so can I.” If there is not a provable, measureable value, as defined by each stakeholder, society may not buy it.

  • Key themes in recent pricing controversies in the U.S.: What drives the policy and the politics?
  • What makes orphan drugs special—and what doesn’t
  • Potential policy solutions and implications for the Ultra Orphan sector

Watch Video


Economic and Clinical Value: Table Stakes for Future Success

Rita Numerof, PhD., President, Numerof & Associates, Inc. (NAI)

Rita Numerof

Dr. Numerof has been a pioneer in the area of economic and clinical value, anticipating the impact of changing global payer attitudes on the fundamental business model of the healthcare industry. She cautions that when all four characteristics of an industry in transition occur, including 1) regulatory shifts, 2) technological change, 3) change in market expectations, and 4) changes in the competitive landscape, the implications for companies, in this case pharma, are significant. In this environment, isolated changes made internally with the goal of addressing these shifts are not enough; it requires new capabilities across the business in order to survive and thrive.

  • How is value defined by patients, physicians, and payers?
  • The manufacturer’s opportunity
  • Making the case for lifetime value

Watch Video


Panel: Payer, Patient, Physician, Manufacturer

Moderator: Jill E. Sackman, D.V.M., Ph.D., Senior Consultant, Numerof & Associates, Inc.
Maria Lopes, M.D., M.S., Chief Medical Officer, AMCHealth/Geisinger Health System
Erin Hugger, Ph.D., Worldwide Business Development, GSK Rare Diseases
Molly Stuart, CEO, International Pemphigus & Pemphigoid Foundation
John Doux, M.D., Board Certified Dermatologist

Panel: Payer, Patient, Physician, Manufacturer

As a follow up to Dr. Numerof’s introduction to economic and clinical value, our panel members offered their viewpoints of what “value” means to their constituent group. Dr. Jill Sackman moderated the panel, beginning with opening remarks from each panel member. Key takeaways include:

  • Value to patients is defined at the individual level, not on a patient population level
  • Rare disease patients really do lack knowledge about their disorder, therapies, and the healthcare system in general, which creates an opportunity for the manufacturer to provide value through patient education.
  • Value to a clinician includes minimal hassle of getting a patient on a therapy, and overall, by “living up to the promise of the drug.”
  • Payer value includes consideration of the total cost of care and whether or not the drug and the service wrap demonstrate decreased healthcare utilization over time, the demand for data to demonstrate outcomes, and for orphan drugs in particular, the need for data to be provided for the total population since each payer will only have a few patients.
  • Perspectives on variation in the value definition
  • How the value definition may change over time
  • What should companies be doing to demonstrate value to each constituent

Hear additional comments, as well as the manufacturer’s perspective, by viewing the video clips below.

Watch Video (Maria Lopes, M.D., M.S.)
Watch Video (Erin Hugger, Ph.D.)
Watch Video (Molly Stuart)


Reducing the Development Burden for Ultra Orphan Therapies – The Challenges of Personalized Medicine.

Ed Kaye, M.D., Chief Medical Officer, AVI BioPharma

Ed Kaye

By way of a case study describing the science and development process around AVI BioPharma’s lead DMD therapeutic candidate based on the company’s proprietary exon-skipping technology, Dr. Kaye suggested ways to reduce the development process, involve multiple constituent groups, and utilize various sources of funding to drive clinical work forward as efficiently as possible.

  • Personalized genetic medicine – unique regulatory, reimbursement, and development challenges
  • Opportunities – approval for a class of therapy, reducing development costs up front
  • Individualized outcomes and quality of life tools designed to measure the impact of therapies on specific diseases

Setting up and Supporting Your Value

Doug Paul, PharmD, M.S., Partner, MME, LLC

Doug Paul

Doug Paul has presented at every Centric Ultra Orphan Drug Conference since the first event in 2008, helping us to understand the challenges of pricing orphan drugs, key factors to consider, and tips on how and where to start thinking about a pricing strategy. This year Doug focused on how the consumer thinks about price and value. He noted that customers do not recognize value because most marketers do not try to sell to it. Understanding value, then, comes from looking at the Functional, Economic, Lifestyle, and Emotional aspects of each patient (“FELE” your way through the value). Taking the next step is critical, which is the effort of actually supporting that value with relevant services, and measuring and communicating outcomes as a result.

  • Experience in Ultra Orphan ex-U.S. that may be coming
  • Take the good, avoid the bad from that experience
  • Need to start measuring from day one

Watch Video


How to Generate and Measure Value through Primary Market Research

Kenneth Tomaszewski, President, Ph.D., M.S., President, KJT Group
Course Director, Health Economics, University of Rochester

Kenneth J. Tomaszewski

Dr. Tomaszewski shared lessons learned from his extensive health services and market research background, much of which has focused on the pharmaceutical industry, medical device, and cost-effectiveness (outcomes) research. By partnering with key opinion leaders (KOLs) in a particular disorder, valuable insights can be gained about physician and patient attitudes, opinions, propensity to prescribe, and potential compliance challenges, for example. However, Dr. Tomaszewski warned of the dangers of relying solely on KOL input and neglecting to perform thorough market research. This is particularly important in rare diseases where, as attendees learned during the panel discussion and other sessions that value is in the eyes of the individual patient.

  • Which outcomes can be influenced?
    • How this is unique for orphan drugs
  • Constituent model
    • Which outcomes are most important to each group?
    • Research to tailor messaging and marketing
  • Research case studies
    • Orphan disease patients
    • Healthcare professionals who diagnose and treat orphan disease patients

Watch Video


MODDERN Cures Act: Advancing Diagnostics and Capturing Lost Opportunities for Patients

Marc Boutin, Executive Vice President & Chief Operating Officer, National Health Council

Marc Boutin

Marc Boutin is the executive vice president and chief operating officer of the National Health Council, the only organization of its kind that brings together all segments of the health care community to provide a united voice for the more than 133 million people with chronic diseases and disabilities and their family caregivers. Despite increased spending on research since the year 2000, the number of new FDA drug approvals has remained flat. The Modernizing Our Drug & Diagnostics Evaluation and Regulatory Network (MODDERN) Cures Act offers four solutions to reduce barriers that prevent therapies for unmet needs from reaching the patients who need them. Listen and watch as Marc clearly articulates the barriers and the solutions, and learn more about the MODDERN Cures Act and how you can support it.

  • Promoting the development of companion diagnostics of breakthrough treatments
  • Encourage development of breakthrough drugs and biologics.

Watch Video


Value & Outcomes approach at Pfizer

Greg Simon, Senior Vice President, Patient Engagement Group, Pfizer, Inc.

Greg Simon

At the end of a long day of engaging speakers and lively discussion, there was no better way to end the sessions than with the enthusiastic, passionate, and energetic message delivered by Greg Simon of Pfizer. Greg presented a compelling argument that we need to combat the challenges that we face in healthcare with “disruptive innovation” by expanding space and contracting time. He calls for changing business models and new ways of thinking about our markets and the patients that we serve. He identifies the need to truly understand our scarce and surplus resources and to utilize, and treat them, appropriately. And Greg offers some ideas around engaging clinical trial patients early on as they offer their time and themselves to the cause of new drug development.

  • The value of satisfying the unmet need
  • Approaching personalized medicine
  • Focus on the patient experience

Watch Video


Day 2

Translating Value Propositions into the Language and Currency of Payers

Adina Safer, Partner, AccelusHealth Partners, LLC

Adina Safer

After enjoying the Cardinals’ come-from-behind win the night before, and with plenty of coffee on hand, Day 2 of the conference started off with a popular subject: demonstrating value to payers. Adina Safer, whose expertise crosses the areas of pharmacy benefit management, medical benefit management, specialty pharmacy services & specialty distribution as well as Medicare part D coverage dynamics, explained the various aspects of understanding, developing, communicating an economic model to prove value for orphan therapies.

  • How does an orphan drug company best approach managed care
  • What a manufacturer needs to do and show to demonstrate value to managed care
  • How to position your product and what kind of economic models are key to proving value

Watch Video


Value Defined at sanofi-aventis

William L. Daley, M.D., M.P.H, Vice President, Medical Affairs, Aging, Business Development and Licensing, SANOFI US

William L. Daley, M.D., M.P.H

Dr. Daley understands the importance of putting the patient first and including the patient early in the development process. The patient is the roadmap to a drug that truly meets the unmet need. And what is the unmet need? It’s not just the disorder; it also includes how the disease affects the patient, the caregivers, and the family and understanding what and where the defect is. All of these factors will allow researchers and biopharmaceutical companies to develop more effective treatments, education, and valuable patient support services, creating value in the marketplace. Ultimately what we are really striving for is value to the individual. And people’s definition or perception of value changes over time and due to changing circumstances. Measuring this value over time is critical, said Daley, and “if you don’t measure it, you probably don’t care.” He believes that a good indicator of value is patient satisfaction, evidenced by patient loyalty.

  • Sanofi’s experience in discovering, developing, and commercializing therapies for rare diseases
  • Shift focus from cost to value
  • Communicating and achieving value

Watch Video


Building Partner Relationships to Deliver and Measure Outcomes

Craig Kephart, President, Centric Health Resources, Inc.

Craig Kephart

With the high per patient cost of many orphan products, demonstrating value to all constituents is particularly challenging considering the current and future state of the healthcare industry. Increasing regulatory hurdles, the entrance of big pharma, the impact of genomics, and continued margin pressure, all call for a rethinking of the way that drugs are brought to market. Specialty pharmacies are being squeezed by payers, and as margins suffer, so does the level of service for patients. Hear Craig Kephart describe how new patient care and supply chain models can help connect orphan drug manufacturers with the patients that they serve, eliminate conflict, reduce costs and complexity while maximizing health outcomes.

  • Start with the end in mind
  • Value, Outcomes, Experience, Tools
  • Direct model positioning for today’s and future specialty pharmacy market

Watch Video


State of the Industry Address – Pharma/BIO 3.0

James C. Greenwood, President & CEO, Biotechnology Industry Organization (BIO)

James C. Greenwood

Jim Greenwood, President & CEO of BIO, concluded our 2011 conference with a look at the work that the organization has been doing to create the right policy environment for continued investment in biotech research, development, and commercialization. He noted that the Orphan Drug Act is proof that changing incentives gets results. Listen to a summary of BIO’s proposals to Congress that are a combination of small business investor credits to incentivize investment in biotechnology and FDA reforms that add innovation to the FDA’s mission statement.

  • Biotechnology innovation is the best hope of finding treatments and cures for rare diseases
  • We need the right policy environments to unleash the promise of biotech innovation
  • We need an FDA that turns hope into cures

Watch Video


Special thanks to the following organizations that contributed to the success of the conference:


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