Initiating Rare Disease Drug Discovery
In comparison with major diseases, the targeting of rare diseases poses many different challenges,
necessitating consideration of bespoke R&D strategies for successful drug discovery efforts to be
successful. This report examines the issue that need to be considered in the earliest stages of embarking
on a rare disease project.
Features and benefits
Understand why rare diseases make attractive targets for launching new drug discovery projects.
Identify the key challenges that are presented by low patient prevalences.
Assess how the research strategy chosen can be influenced by the disease prevalence.
Understand the importance of considering issues of similarity versus other orphan drugs.
Compare the scenarios in which repurposing existing drugs offers advantages over the
development of novel drugs, and vice versa.
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Published: August 2012
No. of Pages: 40
Price: Single User License – US$2875 Corporate User License – US$10781
While disease prevalence has an impact on the commercial viability of a new treatment, returns are
achievable even for ultra rare conditions. Rare diseases can provide sufficient opportunities for
commercialization of multiple therapeutic agents. This is the case even for some ultra rare conditions
such as Gaucher disease and Fabry disease.
Both clinical experts and patient groups provide valuable resources in understanding a rare disease 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.
Your key questions answered
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?
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