Must Know Tips to achieve successful clinical development of an orphan drug

rare disease, orphan drugs, clinical development, aegerion pharmaceuticals, regulatory approvalOrphan Drug development to treat rare diseases offers a chance for the pharmaceutical industry to think and act differently during clinical design and development. So given the reputation of the World Orphan Drug Congress and the pioneering debates that have been staged every year since 2010, I thought it would be beneficial for the orphan drug industry to learn some of the "top tips" that all developers must put in place if they are to successfully gain market authorisation for their orphan product.

To create a bit of context, having spoken with Dr Massimo Boriero, Vice President of Europe at Aegerion Pharmaceuticals, it can be summarised that the major challenges affecting the rate of clinical development for an orphan product are the "limited number of patients and the difficulties [of complying] with all the regulatory requirements". Nonetheless, great opportunities lie in the form of "protocol assistance with the European Medicines Agency" in order to speed up the clinical development.

Dr. Boriero – who will address the question of whether orphan drug development is a pioneer to forge an enhanced clinical development strategy – listed his top 3 must know insider tips to achieve successful clinical development:

  • Define the target product profile together with clinical, regulatory and marketing
  • Include HTA elements in your clinical development plan
  • Strong expertise of each functional team member – there is no place for mistake

As a leaving comment, I posed the question asking what are the foreseeable game changers set to influence the regulatory procedure and pathway to the market for an orphan product?

  • Patient's input and early HTA assessment
  • Compassionate Use and Named Patient Programs

You can read more from the confirmed speakers of the 2012 World Orphan Drug Congress in the run up to the congress by following out Speaker Interview Series:

Current posts include Ron Ellis, CEO, Endocyte provide his perspectives on the "Challenges of partnering in the rare disease industry"

Or learn what the VP Business Development at CSL Behring, Jeannie Joughin has to say on whether HTA & payment decisions are impeding access to rare disease therapies.

For more updates on pharma, biotech and R&D, follow us on Twitter: @biopharmaevents or join our LinkedIn group: BioPharma- Networking for Pharmaceuticals, Biotech and R&D

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