Mapi presentation

How to capture the patients and caregivers perspectives in rare diseases?

In Advocacy, Orphan Drug Congress, Orphan Drugs Live by Karina KusovaLeave a Comment

How to capture the patients and caregivers perspectives in rare diseases?

Why are rare diseases different? Well, because they are rare! Epidemiology and natural history are not well known due to the small population the disease effects. Rare Diseases can go undiagnosed for a significant period of time, often with debilitating or life-threatening symptoms.  2 out of 3 rare diseases are pediatric diseases, which along with the debilitating symptoms can result in self-reporting being an issue, or impossible.

The Rare Disease industry is paving the way for patient-focused outcome measurement in clinical trials. Examples of this in practice include Ruxolitinib (Jakafi) for the treatment of myelofibrosis, Icatibant (Firazyr) for treatment of acute attacks of hereditary and Dalfampridine (Ampyra) for the treatment of walking in patients with Multiple Sclerosis (MS).

Benoit Arnould, Senior Director, Patient-Centered Outcomes, MAPI Group joined us at the World Orphan Drug Congress USA 2016 to detail why PROs in rare disease research and the challenges for patient-centered outcomes research in rare diseases, which include:

  • Generic measures inappropriate
  • Functional limitations to Self-Reporting
  • Conceptual model too rich and complex
  • Issues for testing quantitative scores

Download his presentation here >>

Join us at the World Orphan Drug Congress USA 2017 to hear more about PROs >>

World Orphan Drug Congress USA 2017

 

Leave a Comment