Rare Diseases and Orphan Drugs: Preparing for Commercialization – Timing, Challenges and Solutions

In Orphan Drug Congress, Orphan Drugs Live, Whitepapers by Freya Smale

Rare Diseases and Orphan Drugs: Preparing for Commercialization – Timing, Challenges and Solutions provided by MarketVision Research

Rare diseases represent a large, complex and heterogeneous group of conditions with a few common similarities affecting a small proportion of individuals in a population. In Europe a rare disease is defined as a disease affecting 1 in 2,000 people, whereas in the United States it is a disease affecting 1 in 200,000. The statistics are well-known and often cited. There are 6,000 to 8,000 identified known rare diseases globally affecting an estimated 350 million people.

Orphan drugs represent a significant potential that has drawn substantial attention and investment from venture capital start-ups and, increasingly, Big Pharma. Although it presents a new opportunity, it also presents unique clinical, regulatory and commercial challenges. Unlike other mainstream pharmaceutical products that are developed to address larger disease populations with treatment in the primary and secondary care setting, making orphan drugs available for treating rare disease reveals a completely different set of challenges that are not easily addressed in current pharmaceutical setup and distribution models.

Download the whitepaper on Rare Diseases and Orphan Drugs: Preparing for Commercialiation- Timing, Challenges and Solutions for more on:

  • Patient Centricity
  • Unmet Needs
  • Commercial Questions
  • Disease Recognition
  • Disease Diagnosis
  • Patient Management
  • Long-term Follow-up and Monitoring
  • Clinical Development
  • Potential Market Research Solutions

Whitepaper provided by: David Moak and Joel Schindler Ph.D., MarketVision Research

MarketVision Research are sponsoring the World Orphan Drug Congress USA 2016. Join them by registering today >

World Orphan Drug Congress USA 2016