Challenges in Rare Diseases Treatment

There are approximately 7,000 rare diseases, which from a regulatory perspective are defined as those diseases where there are less than 200,000 patients in the US or that affect no more than five in 10,000 of the general population in the EU. "Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases. They are "orphans" because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.

  • Different types of rare diseases
  • Misperceptions about rare diseases
  • Role of Patient Registries
  • Regulatory Approaches and Alternative Methodologies
  • Rising Patient Costs
  • Reimbursement Strategies

Related Conference of Challenges in Rare Diseases Treatment

October 30-November 1, 2017

3rd Annual Congress on Rare Diseases and Orphan Drugs


(10 Plenary Forums - 1 Event)
San Antonio, Texas, USA
March 1-2, 2018

5th International congress on Infectious Diseases

Berlin, Germany
June 11-12, 2018

4th World Congress on Rare Diseases and Orphan Drugs

Dublin, Ireland
July 23-25, 2018

11th World Congress on Infectious Diseases and Therapy

Melbourne, Australia
August 27-28, 2018

9th International Conference on Emerging Infectious Diseases

Zurich, Switzerland
September 17-18, 2018

4th Annual Congress on Infectious Diseases

San Diego, USA
September 27-29, 2018

10thEuro-Global Conference on Infectious Diseases

Rome, Italy
Sep 17-18, 2018

3rd International Conference on Infection Control

San Diego, California, USA

Challenges in Rare Diseases Treatment Conference Speakers

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