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Communication Strategies. Case Studies. Applied Knowledge.
Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
Chapter 1 Orphan Drug Development in the US
Chapter 2 Orphan Medicine Development in the EU
Chapter 3 Orphan Medicine Development in the UK
Chapter 4 Orphan Drug Development in Japan
Chapter 5 Orphan Drug Development in Australia
Chapter 6 Orphan Drug Development in Canada
Chapter 7 Rare Disease/Orphan Drug Development in the Rest of the World
RAPS latest global regulatory reference guide, Orphan Drug Development for Rare Diseases, covers every aspect of orphan drug development. Topics include history, incentives, benefits, patient engagement, orphan drug designation, marketing authorization, and market access. Real-world case studies and examples are presented.
Orphan Drug Development for Rare Diseases explores challenges facing the developer and provides strategies to overcome them. The strategies involve adopting the applicable regulatory guidance, involving patients, and reviewing precedent and case studies. Each chapter emphasizes the critical role that patients and patient advocacy groups play in each stage of orphan drug development. A number of diverse rare disease organizations are described.
The authors address how a company can reduce its financial burden in the drug development process by taking advantage of grants, involving academia, and working with patients early in the process. Orphan Drug Development for Rare Diseases presents the regulatory tools, resources, and practices for developing orphan drugs, and how to apply them.
This all-inclusive resource spans 30 countries, including ICH regions (US, EU, UK, Japan, Australia, Canada), plus 24 other countries. There are no other publicly available regulatory resources that cover every aspect of the orphan drug development process.