Emerging Commercialization Strategies for Addressing the Needs of Ultra-Orphan Patients
Accelerate Product Launch and Profitability by Building Regulatory Requirements into Distribution
August 24 – 26, 2009, St. Louis, MO
Event Summary
Over 70 industry professionals attended Centric Health Resources’ 2nd 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 also included ample opportunity for networking, including a welcome reception the evening prior to the conference, 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
Michael McCaughan, Editor-In-Chief, F-D-C Reports, Inc., Elsevier Business Intelligence, gave an eye-opening overview of the new FDA, with its tougher enforcement tools; the new reality of the marketplace, where the traditional sponsor-centric world is ending and tighter ties to the patient are crucial for commercial success; and how the new regulatory model favors the smaller, more nimble “biotech” organization, one that can grasp and implement a culture of risk management. Download Presentation | Watch Video
William Sullivan, Specialty Pharmacy Solutions, LLC, led an audience discussion focused on barriers caused by REMS and how prepared, or not, the industry is to deal with those barriers. Issues identified include potential drug access limitations, the impact on discretionary physician off-label use for patients with limited therapy options (most orphan drug populations), privacy issues, the status of manufacturers’ capability to support programs with detailed patient tracking and the ability of organizations to implement the culture change needed to embrace the new world of REMS. Download Presentation
David Galardi, Pharm.D, Apogenics, examined the “nuts and bolts” of REMS, including history, resources, definitions, and techniques for meeting REMS obligations and maintaining commerce. He stated that many orphan drugs are best offered in limited or close distribution models, where access can be maintained, payer relationships preserved, patients can be monitored, physicians and their practices can benefit from few touch points, and REMS can be fulfilled with pristine data collection. Download Presentation | Watch Video
Jayson Slotnik, Attorney at Law, Hogan & Hartson, LLP, and Diane Edquist Dorman, Vice President, Public Policy, National Organization for Rare Disorders (NORD), tackled the subject of the new Administration and the impact on orphan drug development.
Jayson presented a health reform status update including the latest from each chamber and the administration on the topics of follow-on biologics (FOB), comparative effectiveness, insurance reform, and changes to the Medicaid rebate program 340B. Patients stand to benefit from annual & lifetime caps, comparative effectiveness research, guaranteed issue & renewal, catastrophic coverage, pre-existing conditions, genetic non-discrimination, and phase-out of the “donut hole.” Download Presentation | Watch Video
Diane shared that NORD is advocating for FOB legislation that will result in fair exclusivity, an unambiguous, transparent regulatory pathway, FDA flexibility and authority to determine what data it needs, timely resolution to patent disputes, and prohibition of frivolous lawsuits that delay competition. Download Presentation | Watch Video
Dennis Matheis, President, Anthem Blue Cross and Blue Shield of Missouri, provided an overview of current reimbursement trends and demonstrated how payers are approaching pay for performance, value based benefit design (VBBD) and comparative effectiveness. Implications for manufacturers include:
- Continued focus on cost
- P4P will continue to grow with focus on outcomes
- Value Branded Benefit Design will expand with increased impact
- Cost, outcomes (value) will determine success of newly developed pharmaceutical agents.
Download Presentation | Watch Video
Dovetailing from Dennis’ overview of reimbursement trends, payer access and contracting for orphan drugs was next on the agenda. The audience listened to a panel discussion by leading consultants in the industry who have experience working with companies on managed care strategies for orphan drugs. Jim Long, Principal and Co-founder, BioSolutia, moderated the discussion with Dean Erhardt, Principal, D2 Pharma Consulting, and David Galardi of Apogenics sitting in on the panel.
The final topic on Day 1 was a panel discussion focused on the power of health management outcomes.
Dr. Warwick Charlton, M.D., MBA, Chief Medical Officer, Centric Health Resources, Inc., explained how a well-run health management program can result in a positive ROI for sponsors in higher compliance and higher patient loyalty rates. Download Presentation
Two examples of such outcomes were presented:
- Dr. Robert Sandhaus – Medical Director, AlphaNet, explained that the ADMAP program for patients with Alpha-1 Antitrypsin Deficiency was designed to supplement medical care by helping patients and families better understand their disease and treatment. The results of the program, measured over a two-year period, were recently published in the Journal of COPD, February, 2009, 6:1-10. Download Presentation
- Dr. David Sirois, DMD, PhD, Chairman of the Board of Directors, The International Pemphigus & Pemphigoid Foundation (IPPF), provided outcomes to date from a program that launched in May, 2008 to provide peer health coaching and health management to patients with pemphigus and pemphigoid. During the past year, 47% active patients improved disease control while enrolled, and of the 95 active patients, 57 were taking prednisone, and 39 (68%) reduced prednisone use while enrolled. Download Presentation
John Walsh, Chairman of the Board of Centric Health Resources; President, CEO, and co-founder, The Alpha-1 Foundation, explained that there are ways to work with consumer groups that benefits patients, benefits industry, and strengthens service and brand. Mr. Walsh provided guidelines for industry and consumer groups:
- Be ethical
- Understand mutual benefits and challenges
- Keep boundaries clear
- Manage expectations
Download Presentation
Day 2
While Day 1 focused on trends and issues related to the development and launch of orphan drugs, including REMS, comparative effectiveness, and healthcare reform, Day 2 was focused on solutions for dealing with some of these challenges.
Craig Kephart, President & CEO, Centric Health Resources, Inc., presented the benefits of integrating REMS requirements with distribution. Companies that are willing to put the right structure in place to monitor patients, collect data, and measure outcomes should find that REMS can work to their advantage. Companies should look for opportunity to identify/capture efficiencies and/or capture channel margin to offset costs. Download Presentation | Watch Video
Wendy White, Founder & President, Siren Interactive, presented the popular subject of social networking and how to use it to reach patients.There is a conversation going on, and you have an opportunity to influence the dialogue. The key success factor is to provide “shareworthy” content. How do you create shareworthy content? She advises:
- Listen and Learn
- Orientate Around Your Patient’s Journey
- Educate -- Don’t Sell
- Measure and Analyze
Download Presentation | Watch Video
Doug Paul, Vice President and Partner, MME Consultants, presented an enlightening lecture titled, “Including Distribution and Patient Services in Ultra-Orphan Pricing: The intersection of the forgotten P’s of the marketing mix.” What is the Missing P? Patients! He encouraged the audience to leave a lot, not all, of your “pharma” thinking at the door. Welcome to the community, you are no longer in a therapeutic market.
Doug presented the Extended Product Model, which demonstrates that yes, we actually have to differentiate! Differentiate around services, clinical materials, patient support, reimbursement assistance, training, delivery, product features, and guarantees, to name a few. Download Presentation | Watch Video