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CNS Orphan Diseases- the alternative route in CNS
9 April 2013
CNS Orphan Diseases- the alternative route in CNS

The challenges of drug development in CNS is well known, the recent failures of the large phase III trials in Alzheimer's disease illustrates the problem. There is however an alternative route in CNS which can prove to be more accesible  to small and medium sized companies, and that is CNS Orphan diseases. Diseases such as Progressive supraneuclear Palsy (PSP), Fronto Temporal Dementia (FTD) or Huntington's disease can be initial substitutes for the likes of Alzheimer's disease or Parkinson's disease in which to test the effectiveness of a drug.

This masterclass will look at the challenges and potential advantages of developing drugs in CNS Orphan diseases, compared to larger CNS indications and also looks at the support that is available to drug developers in orphan diseases. It will look at case studies of drug companies who have followed this route.

Masterclass agenda

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8:30

Registration & Coffee

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9:00

Welcome & Introductions

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9:10

Overview of orphan diseases- regulatory and other support available

  • EU orphan disease support
  • Definition of orphan disease, process for obtaining orphan drug designation
  • Regulatory support
  • Protocol and outline of process for scientific advice
  • Approval under special circumstances, conditional approval, review of regulations and discussion
  • Marketing exclusivity, fee reductions, financial support summary
  • US orphan disease support
  • Definition of orphan disease, process for obtaining orphan drug designation
  • Special protocol assessment
  • FDA support
  • Fast track review
  • Marketing exclusivity and fee waiver     
  • Running a US and EU orphan disease strategy at the same time - discussion
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    10:10

    Challenges of CNS orphan diseases compared to the larger CNS indications

  • Clinical trial design issues in CNS orphan diseases
  • Rating scales, primary end point selection
  • Data on decline, making the case for clinical benefit
  • Use of patient registries
  • Patient recruitment issues and strategies in orphan diseases and large CNS indications
  • Discussion on challenges in large CNS indications
  • Size of clinical trial
  • Cost of clinical trial
  • Competitive landscape
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    11:00

    Morning Coffee

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    11:20

    Huntington’s disease Case study of drug development in CNS orphan diseases

  • Background and prevalence
  • Symptoms and progression, targets for drug treatment
  • Clinical trial design challenges
  • Pharma industry approach looking at Lundbeck’s tetrabenazine
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    12:30

    Lunch

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    13:30

    Progressive Supranuclear Palsy – case study

  • Background and prevalence
  • Symptoms and progression, targets for drug treatment
  • Clinical trial design challenges
  • Pharma industry approach looking at Allon Therapeutics Davunetide
  • Discussion
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    14:30

    Frontotemporal Dementia – case study

  • Background and prevalence
  • Symptoms and progression, targets for drug treatment
  • Clinical trial design challenges
  • Pharma industry approach looking at Allon Therapeutics Davunetide
  • Discussion
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    15:30

    Afternoon Tea

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    16:00

    Amyotrophic Lateral Sclerosis (ALS) – case study

  • Background and prevalence
  • Symptoms and progression, targets for drug treatment
  • Clinical trial design challenges
  • Pharma industry approach looking at Sanofi Aventis riluzole
  • Discussion
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    17:00

    Questions and close of Masterclass

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