Orphan Drugs: Getting Arms around Rare DiseasesDuygu Koyuncu Irmak*
Department of Clinical Operations, INC Research, Turkey
- *Corresponding Author:
- Duygu Koyuncu Irmak
Associate Director of Clinical Operations, INC Research
Nida Kule Is Merkezi Goztepe Merdivenkoy Mah
Bora Sokak No: 1 Kat: 14 D: 51-52, Goztepe
Kadikoy 34732 Istanbul, Turkey
E-mail: [email protected]
Received date: March 17, 2017; Accepted date: April 11, 2017; Published date: April 18, 2017
Citation: Irmak DK (2017) Orphan Drugs: Getting Arms around Rare Diseases. J Comm Pub Health Nurs 3:167. doi:10.4172/2471-9846.1000167
Copyright: © 2017 Irmak DK. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution and reproduction in any medium, provided the original author and source are credited.
Finding ways to bring new therapies for rare diseases to patients in a timely manner, effectively and affordably is an important public health challenge. The key concern for decision makers in the health authorities for all medicinal products including Orphan Drugs is that the treatment demonstrates efficacy through “substantial evidence” from adequate, well-planned, well-controlled clinical trials
A successful clinical development programs in rare diseases starts with a tailored approach to ensure the right methodology is employed for the target rare disease therapy. The research methodology needs to be evaluated specifically for each rare disease and the target therapy in the light of all available scientific knowledge by all experts acting in all stakeholders.