How Prosensa is overcoming the challenges of #raredisease research and #orphandrug development

In Uncategorized by Matt Turner

stethoscopeThe very nature of rare diseases creates clinical challenges for orphan drug developers, therefore immediately following the pre-clinical phase of research; the upmost must be done to achieve the clinical added value (CAVOD) for your orphan product.

So how can minimal yet robust clinical data lead to market approval quickly and if you decide to, how should partnerships be formed in the development stage to enhance success?

In this video, Hans Schikan, CEO, Prosensa outlines the major hurdles facing all orphan drug developers and provides insight into how Prosensa are overcoming these challenges with their lead compound to treat Duchenne Muscular Dystrophy currently in phase III development.

1. How to logistically embrace the rarity of the disease itself

2. Science and animal models

3. How to implement correct manufacturing considerations

4. How to progress successfully through clinical development

5. Functional outcome parameters

6. What the regulatory pathway demands when filing for market authorisation

7. Achieving sustainability with a relevant pricing and reimbursement strategy


Since you are here, we have some other blog posts that may be of interest to you…

Try our latest blog on how David Meeker, CEO, Genzyme created the critical elements of orphan drug success

More blogs are to follow soon but if you want to shape the 2012 agenda, we welcome your comments below.