SMi Group's Orphan Drugs and Rare Diseases 2019 conference takes place in London this October

1. SMi Presents the 9th Annual Conference on…
Holiday Inn Kensington Forum, London, UK
CONFERENCE: 15TH - 16TH
WORKSHOP: 17TH
OCT
2019
Orphan Drugs
& Rare Diseases
Evaluating the Key Challenges to Advance
Commercialisation & Patient Access
PLUS AN INTERACTIVE HALF DAY POST-CONFERENCE WORKSHOP
THURSDAY 17TH OCTOBER 2019, HOLIDAY INN KENSINGTON FORUM, LONDON, UK
www.orphandrugs.co.uk
Register online or fax your registration to +44 (0) 870 9090 712
or call +44 (0) 870 9090 711
ACADEMIC & GROUP DISCOUNTS AVAILABLE
A deep dive into strategies for accelerating
patient access of orphan drugs
Workshop Leaders: Solange Corriol-Rohou, Senior Director Regulatory Affairs & Policy Europe, AstraZeneca
Cecile Ollivier, Chief Operating Officer, Aparito
08.30 - 12.30
SMi Pharma
@SMiPharm
#SMiOrphanDrugs
HIGHLIGHTS FOR 2019:
• HEAR about the success of integrated approaches to
develop a robust early access and evidence generation
plan with Bioconal Emas
• DISCUSS the intervention of pharmaceutical companies
and patient advocates in developing life long rare
disease treatments with Takeda
• EXPLORE the current development of strategies aimed
to improve patient access to orphan drugs with Genetic
Alliance UK
• GAIN INSIGHT into the various challenges facing orphan
drug development with Minoyx Therapeutics
CO-CHAIRS FOR 2019:
Rick Thompson,
CEO, Findacure
Mike Page, Executive Director Global Regulatory
Affairs Portfolio Products, Alexion Pharmaceuticals
FEATURED 2019 SPEAKERS INCLUDE:
Katrin Radl, Senior Manager Evidence Generation
& Patient Access, Takeda
Sheela Upadhyaya,
HST Associate Director, NICE
Donatello Crocetta, VP Global Head Rare
Immunology Franchise, Takeda
Martina Garau, Associate Director,
Office of Health Economics
Jayne Spink,
CEO, Genetic Alliance UK
BOOK BY 31ST MAY AND SAVE £400
BOOK BY 28TH JUNE AND SAVE £300
BOOK BY 30TH AUGUST AND SAVE £200

2. Orphan Drugs & Rare Diseases
Day One | Tuesday 15th October 2019 www.orphandrugs.co.uk
Register online at www.orphandrugs.co.uk
SPONSORSHIP AND EXHIBITION OPPORTUNITIES
SMi offer sponsorship, exhibition, advertising and branding packages, uniquely tailored to complement your company’s marketing strategy. Prime networking
opportunities exist to entertain, enhance and expand your client base within the context of an independent discussion specific to your industry. Should you wish to join
the increasing number of companies benefiting from sponsoring our conferences please call: Alia Malick on +44 (0) 20 7827 6168 or email: amalick@smi-online.co.uk
08.30 Registration & Coffee
09.00 Chair’s Opening Remarks
Mike Page, Executive Director Global Regulatory Affairs Portfolio
Products, Alexion Pharmaceuticals
MARKET ACCESS AND COMMERCIALISATION
OPENING ADDRESS
09.10 Early access for orphan drugs
• Approaches to develop a robust Early Access Program and
associated evidence generation plan considering multiple
stakeholder’s viewpoints
• Insights and considerations for the implementation of Early
Access Programs (EAPs), including the regulatory landscape,
evidence generation plans and the perspective of multiple
stakeholders
Tom Watson, Executive VP Early Access Programs, Bionical Emas
SPOTLIGHT PRESENTATION
09.50 Exploring different possible methods to assess
real value of orphan drugs
• Discussing the success of NICE’s cost effective
approach in putting new orphan drugs on the market
• Overview of new appraisal techniques, including HST
(Highly Specialised Technology)
• Challenges faced by orphan drug product manufacturers
• Introduction of the budget impact threshold developed for
assessment of ultra-orphan drugs
Sheela Upadhyaya, HST Associate Director,
NICE (National Institute for Healthcare and Excellence)
10.30 Morning Coffee
11.00 Drug repurposing – a lifeline for rare disease patients
• Exploring the commercial potential for drug repurposing and
successes of rare disease patient groups in helping to drive these
projects to the clinic
• Current developments in the field of social investment to fund
generic drug repurposing and its potential for ultra-rare diseases
• Discussing how to encourage a research interest in rare and
ultrarare diseases, potentially leading to the development of
new treatments
Rick Thompson, CEO, Findacure
11.40 Collaborations in rare disease research and commercialisation
• Discussing how rare disease research and commercialisation
differs due to the variety of cases
• More interlinks needed between patients, clinicians, scientists,
authorities and industry professionals both nationally and
internationally
• Addressing importance of close collaborations with patients
when mapping out the patient’s journey
• Highlighting benefits of understanding rare disease
• Intervention of pharmaceutical companies to develop
appropriate lifelong treatments
Katrin Radl, Senior Manager Evidence Generation & Patient
Access, Takeda
12.20 Networking Lunch
ORPHAN DRUG POLICY – REGULATION AND LEGISLATION
13.20 Overview of current orphan drug regulation
• Discussion on current state regulation of orphan drugs, and
the level of scientific rigour needed for orphan drug approval
(accepting small patient group studies and short studies)
• Assessing the importance of clinical evidence during the launch
of an orphan drug, taking into account the difficultly in planning
and executing clinical trials for rare/ultra-rare diseases
• Synopsis of the evidence requirements before and after the
registration and open access to a new drug
Mike Page, Executive Director Global Regulatory Affairs Portfolio
Products, Alexion Pharmaceuticals
14.00 MoCA early dialogue between payers and manufacturers:
An Opportunity to Improve Decision Making for Orphan Drugs
in Europe?
• What is the MoCa process and its aim? Which parties are
involved and what are the applications to date?
• What is the Transparent Value Framework, how is it used within
MoCA, and how can it be improved?
• What are the advantages and disadvantages of participating in
MoCa from different perspectives?
• Final reflections on pan-European assessment for orphan drugs
Martina Garau, Associate Director, Office of Health Economics
14.40 Afternoon Tea
15.10 Technology and orphan drug development
• Experiences and perspectives on the various challenges in the
development of orphan drugs and the available facilitating and
accelerating toolbox
• Analysing the challenges associated with the data sharing and
analysis for rare disease research
• Changes in market prospects for rare diseases following the
improvement in efficiency of drug development
Maria Pascual, Chief Regulatory Officer, Minoryx
INTERNATIONAL CASE STUDY
15.50 Overview of rare diseases in South Africa
• Current work surrounding the implementation of orphan drug
policy in South Africa – no policy present but moving towards
universal healthcare
• Importance of including patients’ best interests during the
legislation of orphan drugs as it helps increase the speed of the
approval process
• How to provide a better rare disease space – ensuring
pharmaceutical companies and governing bodies are not just
taking the cost-effective approach but also ensuring minimum
patient withdraw
Kelly Du Plessis, CEO & Lead Patient Advocate, Rare Diseases
South Africa
16.30 Chairman’s Closing Remarks and Close of Day One
OFFICIAL MEDIA PARTNER SUPPORTED BY

3. Orphan Drugs & Rare Diseases
www.orphandrugs.co.uk Day Two | Wednesday 16th October 2019
Alternatively fax your registration to +44 (0)870 9090 712 or call +44 (0)870 9090 711
MARKETING PARTNERSHIP OPPORTUNITIES
SMi Group is offering companies the opportunity to partner on our dedicated events in order to help raise your company profile, add value,
create awareness of your products/services to our key audience within the pharmaceutical industry. Interested in partnering?
Contact Simi Sapal, Marketing Manager on +44 (0) 20 7827 6162 or email: ssapal@smi-online.co.uk
08.30 Registration & Coffee
09.00 Chair’s Opening Remarks
Rick Thompson, CEO, Findacure
PATIENT ADVOCACY AND ENGAGEMENT
OPENING ADDRESS
09.10 Improving patient access to orphan medicinal products
• Current developments of approaches to help educate
orphan drug manufacturers and stake holders
• Highlighting the benefits of the collaboration of patient
panels to provide a more targeted approach to orphan
drug development
• Exploring the influence of patient groups in providing early
access of orphan treatments for rare disease cases where
there’s no alternative
• Importance of investing in rare disease spaces to provide
future opportunities
Jayne Spink, CEO, Genetic Alliance UK
09.50 Building an educational digital community in rare diseases
- a PAC first Mentality
• Overview and philosophy for developing and commercializing
products in rare disease
• Why changing our mentality from taking to giving ultimately
benefits the whole rare ecosystem
• Sharing ways to help connect rare disease communities by
addressing unmet needs
• Real world examples of effective and ineffective communication
and its overall impact on orphan drug commercialization and
marketing
Dan Donovan, CEO and Co-founder, Rare Life Solutions
10.30 Morning Coffee
SPOTLIGHT PRESENTATION
11.00 Charting the path to diagnosis for patients with
rare diseases
• Discussing the importance of partnership between
pharmaceutical, digital enterprises and patient associations
• Present the Global Commission to end the diagnostic odyssey
of children with Rare diseases project
• Discussing the importance of the adoption of disruptive
technology and advancement of diagnostic solutions in rare
disease space
Donatello Crocetta, Vice President Global Head Rare
Immunology Franchise, Takeda
11.40 Patient Panel Discussion: perspective on patient
engagement
• Highlighting issues associated with engagement,
including lack of reimbursement for their time
Moderator: David Rose, Head of Business Development,
Rare Revolution Magazine
12.20 Networking Lunch
CHANNELS OF THE ADVANCEMENTS OF ORPHAN DRUGS
13.20 CHIESI CASE STUDY: Holuclar
• Introduction of stem cell-based therapies into the rare disease
space
• Case study – The first EMA stem cell based approved therapy
for treating LSCD (Limbal stem cell deficiency): Holoclar,
what has been done until now and where are we
• Peculiarity of the product: the Holoclar Medical Service
Sara Carlot, Medical Advisor Rare Disease Unit Head,
Chiesi Farmaceutici
14.00 Talent recruitment in rare diseases
• Summary of successes in providing guidance to local
employable markets and providing recruitment solutions
• Overview of the variations within the orphan drug sector when
recruiting for different positions
• Recognising the impact of varying talent recruitment methods
within rare diseases
• Key considerations when implementing a hiring strategy talent
Steve Smith, Head of Rare Disease and Cell & Gene Therapy,
Next Phase Recruitment
“Great conference, very informative and
insightful. Touched on many different and equally
important aspects needed to advance patience
access and innovation.
”Healx, previous attendee 2018
14.20 Afternoon Tea
14.50 Biology and biomarker-powered drug discovery and pipeline
management
• Overview of Braincure’s discovery engine – a unique and
biology-powered in silico approach to accelerate preclinical
drug discovery
• Case study on the molecular framework of BDE, how it
does not require data-training associated with AI
• Pipeline of translational opportunities for digital technology
and low cost/high success precision medicine
Krysztof Potempa, CEO, BRAINCURES
15.30 How publicly-available data and AI can help prioritise rare
disease and orphan drug clinical projects
• What we’ve learnt – the challenges of using publicly-available
data
• The opportunities and challenges of AI in Pharma
• Particular challenges of predict rare disease outcomes
Eddie Pease, Chief Technology Officer, PharmaForesight
16.10 Chair’s Closing Remarks and Close of Day Two
SUPPORTED BY

4. HALF-DAY POST-CONFERENCE WORKSHOP
Thursday 17th October 2019
8.30 - 12.30
Holiday Inn Kensington Forum, London, UK
A deep dive into strategies
for accelerating patient access
of orphan drugs
Workshop Leaders: Solange Corriol-Rohou, Senior Director
Regulatory Affairs & Policy Europe, AstraZeneca
Cecile Ollivier, Chief Operating Officer, Aparito
Overview of the workshop:
Together with Dr Cecile Ollivier, COO of APARITO, who as
a former EU regulator drove the Paediatric Extrapolation
Initiative, we will address the main issues, opportunities
and barriers, that are usually met when developing a
product for patients with rare diseases. Development
strategies, early interactions with patients, regulators and
HTA representatives while considering the global picture,
will be addressed with the support of appropriate experts
and through case examples.
Why should you attend:
An opportunity to interact with specialists, to share learnings
through interactive sessions, identify solutions and stay abreast
of new methodologies, e.g. use of RWD or of wearables, and
processes.
Workshop Agenda:
08.30 Registration & Coffee
09.00 Workshop leader introduction
09.10 The regulatory framework: current and future?
09.50 The impact of digital tools
10.30 Morning Coffee & Networking Break
11.00 HTA position and expectations
11.45 Case study examples illustrating success
of wearable devices for rare diseases
12.20 Workshop leader closing remarks
12.30 Close of Workshop
About the workshop Leader:
Solange Corriol-Rohou joined AZ R&D in 2004
and is currently Sr. Director of Regulatory Affairs &
Policy for Europe. She is a pulmonologist and an
immuno-allergist by training. Solange, in her role
as the chair of the Clinical Development Expert Group
at the European Federation of Pharmaceutical Industries
and Associations (EFPIA), has organized jointly with the
European Medicines Agency (EMA) several workshops
(e.g. on Modelling & Simulation in 2011, Dose Finding/
Dose Response in 2014, Paediatric Extrapolation in 2016).
She has also experience working in Innovative Medicines
Initiative projects, e.g. PROactive in COPD or ADAPT
SMART, and within ICH where following the revision of the
ICHE11 paediatric guideline, she is currently keeping the
momentum with the ICHE11A Paediatric Extrapolation
Guideline.
Cécile Ollivier is a senior health engineer with
an MSc in international drug development and
registration. She have extensive experience in
global drug development in paediatric and rare
disease. Prior to joining Aparito, Cécile was a scientific
officer in the Paediatric office and the Science and
Innovation office at the European Medicines Agency
(EMA) for over 10 years. She provided technical and
regulatory expert guidance on the design, conduct and
interpretation of paediatric developments across multiple
therapeutic areas. She has reviewed over 200 paediatric
investigation plans (PIPs) and paediatric scientific advice
(SA) across all phases of the development. Cécile has
been recognised for her work leading and developing
the EMA paediatric extrapolation strategy at EU and
global level, including the EMA/FDA harmonisation for
Gaucher disease and global harmonisation of criteria
for development in paediatric Pulmonary Arterial
Hypertension (PAH) with patients, healthcare professionals,
FDA and Health Canada. She was also an expert in the E11
R(1) working group and the paediatric standing group for
the International Conference of Harmonization (ICH).

5. SMi PHARMACEUTICAL
EVENT PLANNER 2019
JUNE 2019
Prefilled Syringes West Coast
3rd - 4th June 2019, San Diego, USA
Lyophilisation
3rd - 4th June 2019, London, UK
Pharmaceutical Microbiology West Coast
5th - 6th June 2019, San Diego, USA
SEPTEMBER 2019
BioBanking
25th - 26th September 2019, London, UK
Biosimilars
25th - 26th September 2019, London, UK
OCTOBER 2019
Wearable Injectors and Connected Devices
9th - 10th October 2019, London, UK
Cleanroom Technology
9th - 10th October 2019, London, UK
Orphan Drugs & Rare Diseases
15th - 16th October 2019, London, UK
Highly Potent Active Pharmaceutical Ingredients USA
21st - 22nd October 2019, Boston, USA
Lyophilization USA
23rd - 24th October 2019, Boston, USA
NOVEMBER 2019
Respiratory Drug Delivery
13th - 14th November 2019, London, UK
Ophthalmic Drugs
18th - 19th November 2019, London, UK
SPONSORSHIP AND EXHIBITION
OPPORTUNITIES
SMi offer sponsorship, exhibition, advertising
and branding packages, uniquely tailored
to complement your company’s marketing
strategy. Prime networking opportunities exist
to entertain, enhance and expand your client
base within the context of an independent
discussion specific to your industry.
Should you wish to join the increasing
number of companies benefiting from
sponsoring our conferences please call:
Alia Malick on +44 (0) 20 7827 6168
or email: amalick@smi-online.co.uk

6. ORPHAN DRUGS & RARE DISEASES 2019
Conference: Tuesday 15th & Wednesday 16th October 2019, Holiday Inn Kensington Forum, London, UK
Workshop: Thursday 17th October 2019, Holiday Inn Kensington Forum, London, UK
4 WAYS TO REGISTER
ONLINE at www.orphandrugs.co.uk
If you have any further queries please call the Events Team on tel +44 (0) 870 9090 711 or you can email events@smi-online.co.uk
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