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Initiating Rare Disease Drug Discovery Projects
Date:9/19/2012

e and in identifying potential patients for clinical studies.Many opportunities remain to develop treatments for untreated rare diseases. However, ultra rare diseases with high infant mortality present major challenges.Can more than one drug be a commercial success for treating rare indications?What are the best ways of identifying patients for recruitment into clinical trials?Why it is necessary to consider logistic issues in planning clinical studies?What are the requirements for a clinical candidate to treat a rare disease?What factors determine whether a small-molecule or biologic strategy is most suitable?

 

About the author

•Disclaimer

EXECUTIVE SUMMARY

•Choosing rare diseases to target

•Preclinical development

Introduction

•Key points regarding rare disease drug discovery

Choosing rare diseases to target

•Summary

•Introduction

•Key issues

•Commercial potential

- Prevalence

- Geographic distribution

- Disease understanding

- Available experts

- Current treatments

- What is similar?

•Conclusion

Preclinical development

•Summary

•Introduction

•Screen or repurpose?

- Repurposed

- Directed approaches

- Taking advantage of orphan drug status

•Other issues

- Small molecule or biological?

- Biological test models

- Requirements of a clinical candidate

•Conclusions

Appendix

•Scope

•Methodology

•Glossary/abbreviations

•Bibliography/references

 

FIGURES

•Figure: Schematic relationship between rare disease prevalence and commercial returns

•Figure: Identifying suitable patients for clinical studies

•Figure: The influence of prevalence on research st
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