Challenges in Rare Diseases
Making a pharmaceutical care development program for the treatment of a uncommon disease can, however, prove to be a monumental task. Poor understanding of the characteristic history of the proposed indication due to few observational studies considering disease progression, heterogeneous patient populations with variable phenotypes and clinical courses, geographic dispersion of patients and investigators, regulatory uncertainties, and lack of prior clinical studies to establish a format for study execution, can all prove challenging. While individually rare, orphan diseases are really collectively common, with an estimated 350 million sufferers around the world.
- Patient attrition
- Interface between clinical development and the regulatory process
- Less knowledge on pathophysiology of disease
- Lack of awareness
- Diagnosis
- Developing and using Orphan medicinal products
Related Conference of Challenges in Rare Diseases
February 27-28, 2019
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| Radisson Hotel Narita | Tokyo, Japan
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Challenges in Rare Diseases Conference Speakers
Recommended Sessions
- Clinical Research on Rare Diseases
- Living with a Rare Diseases
- Rare Disease
- Rare Pediatric Diseases
- Treatment for Rare Diseases
- Challenges in Rare Diseases
- Future of Rare Diseases Research
- Infectious Diseases
- Mystery Diagnosis of Rare Diseases
- Neglected Tropical Diseases
- Neurology Rare Diseases
- Orphan Drug
- Plant Pathology
- Rare Cancer Diseases
- Rare Diseases in Aging
- Rare Diseases in Brain
- Rare Diseases in Endocrine System
- Rare Diseases in Eye and Ear
- Rare Gastrointestinal Diseases
- Rare Haematology Diseases
- Rare Immune System Diseases
- Rare Oral Diseases
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