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Rare Disease Market Access and Orphan Drug Commercialization

Rare Disease Market Access and Orphan Drug Commercialization

Rare Disease Market Access and Orphan Drug Commercialization

Featured Faculty:

David Brumley, MD MBA
Senior Medical Director
Tufts Health Plan
Photo Not Available Karen Fardig, JD
Vice President, Legal
Head Counsel, Gaucher and Fabry
Shire
Usman Iqbal Usman Iqbal
Senior Medical Affairs Leader, Neuroscience, Global Medical Affairs
AstraZeneca
David Lemus
Chief Executive Officer,
Sigma-Tau Pharmaceuticals
Board, PhRMA
Board, BioHealth Innovation
Chairperson,
Proteros biostructures GmbH
Board, MediGene AG
Board, Axela
Stephanie Okey Stephanie Okey, MS
Head, North America, Rare Diseases
Senior Vice President, U.S. General Manager
Genzyme Corporation

Join market access and commercialization executives from pharmaceutical and biotech companies within divisions of rare, genetic, and orphan diseases to gain a renewed understanding of the commercial dynamics of rare disease and orphan drugs so that manufacturers can forecast and commercialize orphan products successfully. This Summit evaluates market forces, their economic impact, and the implications for orphan drug development, pricing, reimbursement, and patient and market access.

The rare disease market is an important field for biotech and pharmaceutical manufacturers. With few competitors in the space for specific molecules and indications, brands have historically maintained significant influence on market and pricing indicators to support the costs associated with the patient population, research, and development. Recent regulatory legislation leading to reduced development timelines and an array of commercial incentives have enhanced the value of orphan drugs and shifted the focus of drug development into this market. While the FDA, NIH, venture capitalists, and patient advocacy groups are supporting more orphan drug development, many payers, PBMs, and health systems are making determinations based on pricing, accessibility, long term costs, alternative therapies, and the ultimate value of the product. Evaluate new regulatory initiatives, support orphan patient groups, and identify new payment models to set the foundation for a sustainable and ethical rare disease market.

Top Reasons to Attend:

  • Assess methods to increase orphan drug speed to market at a lower price to the patient
  • Discuss the impending economic evolution in the rare disease landscape with senior executives
  • Understand factors affecting reimbursement and formulary decisions
  • Benchmark strategies for pricing and cost saving valuation of orphan drugs
  • Discover recent findings on global orphan drug patient access issues

See the Attendee List of the 2014 Summit

Testimonials from attendees of the 2014 Summit:

“Well worth my time – I learned a great deal – unique and innovative forum.” – Gil Litalien, PhD, Executive Director

“The World Congress on Economics and Orphan Drug Commercialization was a wonderful opportunity to interact with senior level industry and government executives to concentrate on solutions to common problems in commercial development.” – Anne Marie Finley, MS, President, Biotech Policy Group, LLC

“This was the most content-consistent meeting with excellent information exchange, consistent in quality delivery. The speakers and presentations were clear and valuable.” – Haya Taitel, Vice President, New Product Commercialization, Kadmon

“It is imperative that our industry stakeholders come together in forums like this conference and address the issues we face. Together we can make a difference.” – Dave Reynolds, Vice President, Praxis Rx Health Services

“The two day summit was very informative about the specifics of commercialization. The speakers were knowledgeable and offered future focuses required in the rare disease specialties.” – Cherylann Gregory, CEO and President, Specialty Pharmacy Nursing Network

“Great discussion addressing the challenges in the orphan drug market.” – Marcel van Kuijck, PhD, Senior Director and Global Head, Rare Disease Registries, Genzyme, a Sanofi Company

“Super!” - Charles Shasky, President, Biotechnomics; Head, Pharmacoeconomic Comparative Effectiveness Research, Aetna


Call for Papers: Submit Abstracts for Presentations

Rare Disease Market Access and Orphan Drug Commercialization is now accepting abstract submissions for 2015! Deadline is June 5th, 2015. Click here for details.


To Inquire about Sponsorship and Executive Networking Opportunities, contact:
Suzanne Carroll
Business Development Manager
781-939-2648
[email protected]

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