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What is this event about?



Rare diseases have been largely neglected until very recently. Lack of research and lack of interest from the pharmaceutical industry has meant that very few of the 6000+ rare diseases had any treatment available.

The Regulation (EC) 141/2000 of the European Parliament of 16 December 1999 was introduced to try to change this situation. This legislation introduced a new set of regulations which, if properly understood and followed, can provide significant incentives to companies to develop treatments for rare diseases.

The development of such treatments has many unique challenges and this Masterclass aims to help delegates understand what differentiates orphan drugs from other drugs and to approach the design of development programs for rare diseases.

Our Masterclass will be delivered through a combination of diversely skilled speakers who will give presentations, case studies and encourage interaction and discussion. This is designed to help inspire delegates with an understanding of and a synergistic approach for successful orphan drug development.



Where is the event?

This Masterclass will take place in central London, UK 


Who is leading this event?  

Tony Hall, Chief Medical Officer,PSR Group, The Netherlands


Tony Hall graduated in London with first class honours in physiology & pharmacology from King's College then as a doctor at the Royal Free Hospital. He specialised in Emergency Medicine and joined the pharmaceutical industry in 1994. At Boehringer Ingelheim he was responsible for the strategic development of many clinical programmes and for two global products. He worked at Yamanouchi before starting his own business.

Now as Chief Medical Officer of PSR, Tony has an in-depth knowledge of the orphan drug market, applicable regulations and procedures.  He provides advice and guidance on development plans for orphan drug designation, protocol assistance and clinical programmes. He has built trusted relationships with patient groups to help ensure their voice is heard during PSR’s clinical trial involvement. 




  In association with



PSR is a leading expert in Orphan drug development. We provide the essential ingredients necessary to help our clients through the logistical and regulatory hurdles of clinical study programs for these products.

PSR has established links with physician networks, key opinion leaders, government bodies and patient groups; we can provide regulatory expertise and access to scientific (protocol) advice at the EMEA level, all of which helps to ensure the best design of the clinical program.

PSR has the experience necessary to set up and run complex clinical trials in rare diseases, where access to patients and overcoming the logistical challenges of sparsely distributed patients are the keys to success. Our approach is tailored to enable us to perform efficient and successful orphan drug studies, which often require a small number of sites per country spread over several countries together with novel approaches to ensure protocol compliance.

By attending this event you will be better able to:

Understand the background of orphan drugs and the related legislation.
Learn how to write an orphan drug application for Europe and the US.
Appreciate the key aspects of designing clinical trials for orphan drugs.
Evaluate the importance of patient registries for rare diseases.
Gain insight into the perspective of the rare disease patient.
Observe the effect of reimbursement issues on patient access to orphan drugs.


• Designing clinical trials for orphan indications
• Rare diseases from the patient’s perspective
• Market access and reimbursement for orphan drugs
• Patient advocacy groups
• Guide to writing an orphan drug application (EU&US)
• Variations in patient access between countries
• Predictors of success or failure
• Roles & achievements of EURORDIS


Who would benefit? 

Pharma & large biotech
Medical Directors
Medical Advisers
Heads of clinical research
Regulatory affairs
Market access  







Masterclass programme

8:30 Registration & Coffee

9:00 Welcome & Introduction to Orphan Drugs

9:45 Guide to writing an orphan drug application (EU&US)

10:45 Morning Coffee

11:15 Designing clinical trials for orphan indications

12:15 Registries for rare diseases

13:00 Networking Lunch

14:00 Rare diseases from the patient’s perspective

14:45 Patient advocacy groups

15:30 Afternoon Tea

16:00 Market access and reimbursement for orphan drugs

16:45 Conclusions and end of masterclass


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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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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