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CNS Orphan Diseases – the alternative route in CNS
8 April 2014
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 accessible to small and medium sized companies and that is the 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 or Parkinson's disease in which to test the effectiveness of a new 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 and Introductions

Susan McGoldrick

Susan McGoldrick, CEO, QCTR Ltd

Audrey Jamieson

Audrey Jamieson, Business Development Manager, QCTR Ltd

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9:10

Overview of orphan diseases – regulatory and other support available

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9:50

Challenges of CNS orphan diseases compared to larger CNS indications

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10:30

Morning Coffee

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11:00

Case studies of drug development in CNS orphan diseases:

These case studies will look at clinical trial design, endpoint selection and look at real life examples of how companies have approached these challenging indications:
• Huntington’s disease and the tetrabenazine story
•Amyotrophic lateral sclerosis and the
dexpramipexole story
• Progressive surpranuclear palsy and the davunitide
story
• Frontemporal dementia and the memantine story

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12:20

Closing Remarks

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12:30

End of masterclass

VENUE

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Coram Street, London, United Kingdom

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Holiday Inn Bloomsbury

Coram Street
London WC1N 1HT
United Kingdom

Holiday Inn Bloomsbury

Holiday Inn London-Bloomsbury is just 2 minutes' walk from the Tube at Russell Square station, for speedy connections across the capital. We're 10 minutes by bus from Eurostar connections at St Pancras station, and a 45-minute taxi ride from London Heathrow Airport. Check into your stylish Guest room, air-conditioned and connected to wireless Internet.

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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.

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