Challenges in Rare Diseases
There are approximately 7,000 rare diseases. Such rare diseases generally have a genetic basis, frequently affecting patients in childhood, and are often progressive and life threatening in nature. These characteristics can have highly destructive psychological impact on children suffering from these diseases. The major challenge for R&D is to look after the impact of unique characteristics of the rare disease patients on clinical development process and the interface between clinical development and the regulatory process. A fundamental challenge in drug development for rare diseases is that there is relatively little known about the pathophysiology of disease. In addition to these challenges, for most of the rare diseases there are no available therapies.
- Patient attrition
- Interface between clinical development and the regulatory process
- Less knowledge on pathophysiology of disease
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Challenges in Rare Diseases Conference Speakers
Recommended Sessions
- Challenges in Rare Diseases
- Clinical Case Report on Rare Diseases
- Clinical Research on Rare Diseases
- Future of Rare Diseases Research
- Immune System Diseases
- Infectious Disease
- Living with Rare Diseases
- Mystery Diagnosis of Rare Diseases
- Orphan Drugs
- Rare Autoimmune Diseases
- Rare Cancer Diseases
- Rare Dermatological Diseases
- Rare Diseases
- Rare Diseases in Aging
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- Rare Diseases in Endocrine System
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- Rare Genetic Diseases
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- Rare Pediatric Diseases
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