Conference on the Life Cycle of Orphan Drug Development & Commercialization

Conducting Effective Orphan Drug Research and Clinical Trials to Expedite Orphan Drug Approval

When:    January 16-18, 2013
Where:   Boston, MA

Key Topics

Improve current rare disease research methods used in early stages of orphan drug development with Genzyme
Evaluate where the rare disease research units fit in the portfolio for large and niche pharma companies with Pfizer
Utilize translational medicine to be effective in orphan drug development with Vertex
Collaborate with patient advocacy groups to increase patient recruitment in clinical trials with BioMarin
Review the FDA Safety and Innovation Act and its impact on orphan drug reserach and development with National Organization for Rare Disorders

Key Features

2 Pre-Conference Workshops on January 16, 2013

  1. Pre-Conference Workshop A: Integrating Gene Therapy and Stem-Cell Therapy Treatments in Rare Disease Clinical Trials with ReGenX Biosciences, LLC
  2. Pre-Conference Workshop B: Exploring New Solutions to Reduce the Orphan Drug Funding Gap with Asklepion

Event Focus

The orphan drug and rare disease industry is one of the hottest topics in modern day medicine. With blockbuster drugs coming off patent, there is a great need for pharmaceutical companies to diversify their portfolios by exploring niche markets. The increasing presence of high-profile manufacturers in the orphan drug industry provides these companies with this opportunity.

The Life Cycle of Orphan Drug Development & Commercialization Conference will focus on the current landscape of rare disease and orphan drug development, different forms of structural based drug designs, and specifically designed clinical trials.  Through these clinical trials, and by having connected to the right patients to participate, will increase productivity and expedite orphan drug approval and commercialization of the final product.

By attending this conference, delegates will gain a comprehensive view of the orphan drug and rare disease industry and will have a clearer understanding of the natural histories of rare diseases and how to most effectively treat them. Through maximizing translational research methods to evaluate bio-markers and surrogate markers in the patient’s body, this will assist in the design of orphan drug clinical trials resulting in quickly obtaining results and being able to send the orphan drug in for FDA approval.

Attending This Conference Will Enable You To:

  1. Assess the current rare disease department within the pharmaceutical industry
  2. Analyze current orphan drug developments and treatment methods in clinical trials
  3. Select the optimal patient target market to engage in rare disease clinical trials
  4. Review orphan drug regulations and accelerate orphan drug approval
  5. Address the importance of reimbursement and commercialization in the orphan drug industry

Industry leaders attending this conference will benefit from a dynamic presentation format consisting of workshops, panel discussions, and industry-specific case studies that provide accurate, real-world knowledge. Attendees will experience highly interactive conference sessions, 10-15 minutes of Q&A time after each presentation, 4+ hours of networking, and exclusive online access to materials post-event.

Register here.

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