Initiating Rare Disease Drug Discovery Projects

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.

Highlights

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?