With an estimated 8,000 rare diseases identifi ed and
worldwide sales expected to exceed $175bn by 2020,
commercialising new orphan medicines remains highly
attractive to the pharmaceutical industry, although
greater regulatory and market access challenges are to be
overcome for a product to be successful.
Developing medicines for rare diseases give rise to unique
challenges, which often require creative solutions and
innovative development strategies to overcome.
This workshop will look at best practices for the development
of orphan medicines, how to avoid common pitfalls and
how to “plan for success”.

Workshop LEADER


Alex Bloom

Director of Regulatory Affairs, Cell Medica Ltd

Dr Bloom acts as Director of Regulatory Affairs at Cell Medica
(UK), a niche biopharmaceutical company specialising in the
development of cell therapies to treat rare diseases. He also
acts as Principal Consultant for Aceso Consulting Ltd, offering
strategic and regulatory support to the biopharmaceutical
industry. In the past ten years, he has worked on multiple
rare disease programs, including the oversight of more
than a dozen orphan drug applications in the EU and US.

Cell Medica Ltd

Cell Medica is committed to improving patients’ lives through
the signifi cant therapeutic potential of cellular immunotherapy.
Our approach is to apply innovative technologies with
the aim of improving the treatment of cancer and
immune reconstitution following hematopoietic stem cell
transplant. We have developed a business platform that
has positioned Cell Medica as a leader in the manufacture,
development and commercialization of cellular products.

Chiesi Farmaceutici (www.chiesi.com) is a researchfocused
international group, headquartered in Parma
(Italy). Chiesi researches, develops and commercializes
innovative pharmaceutical solutions in the respiratory,
specialist medicine, and rare diseases areas. In 2008, Chiesi
launched the spin-off company Holostem, dedicated to
the development of tissue engineered and gene therapy
products. In 2014, the fi rst advanced therapy candidate was
approved by EMA, becoming the fi rst medicinal product
containing stem cells in EU.

Workshop programme

8:30 Registration & Coffee

9:00 Strategy and profiling: laying the foundations for success

• The importance of a target product profi le
• Understanding your regulatory environment
• Knowing your patient population: the value of
natural history studies and patient registries

Alex Bloom

Alex Bloom, Director of Regulatory Affairs, Cell Medica Ltd
View Bio

Diego Ardigo

Diego Ardigo, Project Lead, CHIESI FARMACEUTICI S.p.A.
View Bio

11:00 Morning Coffee

11:30 Development, approval and marketing

• Clinical development in small populations:
opportunities and limitations
• Expedited development pathways and early
access schemes
• Developing a market access strategy and the
value of real world data

Alex Bloom

Alex Bloom, Director of Regulatory Affairs, Cell Medica Ltd
View Bio

Diego Ardigo

Diego Ardigo, Project Lead, CHIESI FARMACEUTICI S.p.A.
View Bio

12:30 End of Workshop


Holiday Inn Kensington Forum

97 Cromwell Road
London SW7 4DN
United Kingdom

Holiday Inn Kensington Forum

Holiday Inn Kensington Forum is perfectly situated in one of London’s most luxurious and beautiful areas within South Kensington. 

The hotel is just 2 minutes walk from Gloucester Road tube station for convenient travel to Hyde Park, London Eye, Tower Bridge plus more of London’s top attractions. There are also easy and direct links to some major transport hubs including Victoria, Kings Cross St Pancras, Paddington and Heathrow. 

This distinctive hotel in south London has so much to offer to make all guests really feel at home. The latest Holiday Inn relaunch is not just about the new look and feel for the hotel but to offer guests more benefits during their stay including a pillow menu for extra comfort during their sleep and a curved shower rail for more spacious feel. 

In addition to our 906 rooms, all business guests can take advantage of our meeting and conference facilities including High Speed Internet Access and unlimited Starbucks coffee at The Academy. Our hotel’s professional event planners are on board to help take the hard work and stress away from planning your next event. 

So whether you in London on business or pleasure, make the Holiday Inn London Kensington Forum your first choice of hotel and book your accommodation for our lowest internet rate guarantee. 



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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