Home
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

clock

8:30

Registration & Coffee

clock

9:00

Welcome & Introductions

clock

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
  • clock

    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
  • clock

    11:00

    Morning Coffee

    clock

    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
  • clock

    12:30

    Lunch

    clock

    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
  • clock

    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
  • clock

    15:30

    Afternoon Tea

    clock

    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
  • clock

    17:00

    Questions and close of Masterclass

    VENUE

    Holiday Inn Kensington High Street

    Wrights Lane, Kensington, London, W8 5SP, United Kingdom

    A number of our clients have been approached by third party organisations offering to book hotel rooms. We would advise that you do not book through them as they are not representing the SMi Group. SMi Group books all hotel rooms directly. If you are approached by a third party organisation then please contact us before making any bookings. If you have already booked a hotel room using a third party organisation, we would highly recommend contacting the hotel you were booked into to ensure a booking has been made for you. We would also advise you to please check the terms and conditions of the booking carefully.
    HOTEL BOOKING FORM

    SAVE TO


    Outlook Calendar  OUTLOOK CALENDAR
    Google Calendar  GOOGLE CALENDAR
    ICal Calendar  ICAL CALENDAR
    Yahoo! Calendar  YAHOO! CALENDAR

    Holiday Inn Kensington High Street

    Wrights Lane
    Kensington
    London
    United Kingdom

    Holiday Inn Kensington High Street

    HOTEL BOOKING FORM

    Title

    SubTitle
    speaker image

    Content


    Title


    Description

    Download

    Title


    Description

    Download

    Title


    Description


    Download


    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

    CPD AND PROFESSIONAL INSTITUTES

    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SAE Media Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

    GLOBAL CPD

    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@saemediagroup.com

    Event Title

    Headline

    Text
    Read More

    I would like to speak at an event

    I would like to attend an event

    Group Booking

    Please complete the below form and a member of SAE Media Group’s booking team will be in contact within 24 hours

    I would like to sponsor/exhibit at an event

    SIGN UP OR LOGIN

    Sign up
    Forgotten Password?

    Contact SAE Media Group

    UK Office
    Opening Hours: 9.00 - 17.30 (local time)
    SAE Media Group , Ground Floor, India House, 45 Curlew Street, London, SE1 2ND, United Kingdom
    Tel: +44 (0) 20 7827 6000 Fax: +44 (0) 20 7827 6001
    Website: http://www.smgconferences.com Email: events@saemediagroup.com
    Registered in England - SMi Group Ltd trading as SAE Media Group




    Forgotten Password

    Please enter the email address you registered with. We will email you a new password.

    Thank you for visiting our event

    If you would like to receive further information about our events, please fill out the information below.

    By ticking above you are consenting to receive information by email from SAE Media Group.
    Full details of our privacy policy can be found here https://www.smgconferences.com/privacy-legals/privacy-policy/.
    Should you wish to update your contact preferences at any time you can contact us at data.privacy@smgconferences.com.
    Should you wish to be removed from any future mailing lists please click on the following link http://www.smgconferences.com/opt-out

    Fill in your details to download the brochure

    By submitting this form you agree to our privacy policy and consent to receiving communications, you may opt out at any time.