This workshop will provide insights into the orphan drug regulatory landscape towards clinical execution and its accompanying challenges, with emphasis on the rare disease patient’s perspective.

It includes insights into the current possibilities and challenges within the regulatory framework plus the necessary requirements to transform the regulatory strategy into feasible clinical development planning.

Furthermore, the crucial role of the patient and the patient advocacy groups in all aspects of Clinical Development Planning and the organisational and practical challenges during execution of the clinical trials will be considered.

• To get insight into the overall process from the regulatory perspective (e.g. orphan drug designation) towards running clinical studies in rare disease patients.
• To successfully transform regulatory strategies into practical clinical development planning and subsequently running successful clinical trials in rare disease indications.
• To understand how to collaborate with patient advocacy groups in the most successful way for your orphan drug development program.
• To learn the importance of potential strategies to lower patient burden and thereby increase patient recruitment and retention in rare disease studies and thus benefit overall study success rate.

Workshop LEADER


Thijs op de Weegh

Business Development Manager, PSR Orphan Experts

PSR Orphan Experts

PSR is the leading expert and full service orphan drug CRO in the rare disease niche. PSR is specialized in running complex orphan drug clinical development programs (Natural History Studies, Phase I – IV studies & Registries) and regulatory projects (e.g. Orphan Drug Designations, Clinical Development Plans, EMA and FDA interactions (Scientific Advice /(pre-)IND/PIP) requiring innovative regulatory and clinical approaches. PSR has performed over 140 Orphan Drug projects to date.

Workshop programme

13:30 Registration

14:00 Opening remarks and introductions

14:10 Regulatory framework & how to successfully transform regulatory strategies into practical clinical development planning

Steve Pinder

Steve Pinder, Director, Envestia

• Brief outline of Orphan Drug regulations
• Common misinterpretations of orphan drug legislations
• Regulatory strategies vs Clinical development strategies 

15:30 Coffee

15:45 The crucial role of patient advocacy groups in a successful orphan drug development program

Nicolas Sireau

Nicolas Sireau, Chairman and CEO, AKU Society

• Public/private funding & crowd funding
• Role of patient advocacy groups in rare disease clinical trials 

16:30 Lowering patient burden & practical / logistical challenges to ensure successful clinical study execution

Christa Verburg

Christa Verburg, Senior Clinical Project Manage, PSR Orphan Experts

• Patient recruitment & retention strategies
• Lowering patient burden
• Logistical / practical challenges

17:15 Closing remarks

17:30 End of workshop


Holiday Inn Regents Park

Carburton Street
London W1W 5EE
United Kingdom

Holiday Inn Regents Park

Choose the well-connected Holiday Inn London-Regent's Park hotel, with a superb central London location and speedy transport links. Holiday Inn London-Regent's Park is in a leafy and cosmopolitan area of central London, a 10-minute walk from bustling Oxford Street. Leave your car in our NCP managed underground car park, and explore London by Tube. Great Portland Street Tube station is 25 metres from the hotel, from where you can reach the City and Canary Wharf in 30 minutes, and London Heathrow Airport in 45 minutes.

Wireless Internet is available throughout the hotel, and you can invite up to 300 people to events at the Academy Conference Centre, with an IT technician and break-out zones. Holiday Inn London-Regent's Park is a 10-minute walk from Santander's offices and businesses in the BT Tower. Stroll 5 minutes to Regent's Park, where you'll find London Zoo and pretty Primrose Hill. We're a 10-minute walk from Bond Street boutiques and 20 minutes from Buckingham Palace and cruises on the River Thames.

Ask our Concierge to plan your day out and book West End theatre tickets. Room Service is available 24 hours at Holiday Inn London-Regent's Park, or dine in the vibrant Junction Restaurant. Our Junction Bar has a menu of light bites, and a hot breakfast buffet is served daily.



speaker image






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.


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.


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, SMI 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.


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@smi-online.co.uk

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