World Orphan Drug Congress Day 1 Tuesday 29th November 2011

last modified: 22 November '11

9am

Chairman's opening remarks

› Andrea Rappagliosi,

VP European Government Affairs & Head of Brussels Office, GSK; Chair, EFPIA HTA Task Force, GSK

Enhancing the global efforts towards orphan drug provision

9.10am

Keynote address: Collaborative innovation: the international ambitions of the IRDiRC

Is the progress made in rare disease treatments only the tip of the iceberg?
Why progress in orphan drugs is being fostered through international collaboration?
What are the challenges and objectives of this global initiative?
How can all orphan drug stakeholders contribute towards fulfilling the patient need?

› Sharon Terry,

CEO, Genetic Alliance

9.40am

Building the strategy to diagnose all and treat 200 rare diseases

Why have these specific objectives been established by the IRDiRC?
What are the scientific and policy frameworks in place to reach the targets set out for 2020?
How will international resources be structured towards reaching the common objectives?
How can research and development in all rare diseases be encouraged but not duplicated?

› Catherine Berens,

Scientific Officer Personalised Medicine Unit, EU Commission

10.10am

Creating a patient-orientated environment through the National Rare Disease Plans

Why do public health strategies on rare diseases need to be harmonised in Europe?
The long term priority is to create a uniform and normalised access to healthcare
How are member states ensuring rare disease sufferers have access to high quality care?
How will member states prioritise best practice when tackling rare diseases?

› Segolene Ayme,

Director of Research, INSERM, EU Commission

10.40am

Morning Refreshments

Pricing, reimbursement and achieving market access

11.10am

Playing devils advocate: blowing the pricing debate wide open

Do rare diseases demand equal reimbursement compared with more common diseases?
Are companies within their rights to demand the highest return for their products?
Who is to blame when the projected market for orphan drugs exceeds expectations?
What can all stakeholders do to remedy orphan drug access?

› Richard Edlin,

Lecturer on Health Economics, University of Leeds

11.40am

Developing new HTA methods to evaluate rare disease treatments

Why traditional methods for the HTA of conventional drugs are not appropriate for orphan drugs?
An introduction to the AGNSS process and its promises for rare diseases treatments
What types of evidence can be generated to serve the needs of HTA and demonstrate the value of orphan drugs?
How can patients and industry collaborate and contribute to HTA decision making?
An overview of European initiatives e.g. CAVOD, EUNetHTA to enhance access for rare diseases’ patients

› Francis Pang,

Senior Market Access and Public Affairs Director, Shire HGT

12.10pm

The decision-making process for ultra-orphan drug availability in England

Why have a national rare disease decision-making process?
How have and how can all stakeholders contribute to its development?
What have been the lessons learned during pilot?
What are the early lessons from implementing the AGNSS process?

› Josie Godfrey,

Head of Policy and Coordination, National Specialised Commissioning Team

12.35pm

Panel discussion: A frank evaluation of the value of an orphan drug treatment

Are orphan drugs pricing themselves out of reach and do they warrant a premium price tag?
Can the value be good enough for approval but not for reimbursement?
Should there be a specific value framework for rare disease dugs?
Sustainability - what if healthcare can no longer foot the orphan drug bill?
Would imposing pricing regulations improve market access the short term or reduce investment incentives in the long term?
How can an agreeable balance of revenue and patient access be reached?

› Segolene Ayme,

Director of Research, INSERM, EU Commission

› Pierrick Rollet,

VP Global Market Access, Advocacy, Communications, GSK

› Richard West,

Committee Secretary, Behcet's Society

1.15pm

Networking Lunch

2.15pm

Strategies for ensuring reimbursement and improving patient access

How do you compile compelling data for approval when patient populations are small?
How to identify and overcome hurdles to achieve rapid market access per member state?
Obtaining substantial clinical data to ensure a successful market introduction
How can post-marketing approval studies confirm efficacy and guarantee reimbursement?
Will orphan drug development shift to require evidence based data to ensure efficacy?

› Carlo Incerti,

SVP Global Medical Affairs, Genzyme

2.45pm

Early access to orphan drugs – the rationale, challenges and options available

Why consider early access?
What are the challenges for early access?
Introduction to the mechanisms, regulatory environment and options for early access.
Current trends in early access
Insight into the potential benefits and risks of early access

› Mark Corbett,

Global Strategic Planning Director, Clinigen

3.05pm

The ultimate beneficiary or the forgotten endpoint: a patient organisation perspective

Ataxia UK – entering into the world of orphan drug treatments, what are the issues?
How do patients view the premium prices of orphan drugs?
How can patient organisations assist in raising disease awareness at a national level?
Can patient organisations influence healthcare authorities to provide full reimbursement?

› Sue Millman,

CEO/General Secretary, Ataxia UK/euro-Ataxia

3.35pm

Speed Networking

4.05pm

Afternoon Refreshments

Expanding orphan drug indications

4.35pm

Expanding product indications through increased scientific understanding

How can rare disease research act as a portal to treat additional diseases?
What are the possibilities as the science of disease pathways become better known?
How can you identify the correct orphan indication during early stage development?
What are the commercial and patient benefits to drug repurposing?

› Peter Myrenfors,

Director of Medical Affairs, Swedish Orphan Biovitrum

5.05pm

Open floor interactive Q&A session

Ethics of repurposing an existing drug towards an orphan drug indication
What are the benefits and drawbacks of developing drugs via repurposing?
What are the ethical implications when pricing a repositioned orphan drug?
How were the clinical primary endpoints established?

5.20pm

Addressing global health issues: the inverse of orphan drug development

High or low prevalence –Does it makes sense to address neglected diseases?
Is approval in Europe or the United States the path for new medicines for low income countries?
What can be learnt from research into neglected diseases?
What are the social and commercial benefits of investment into neglected diseases?

› Jerry Zeldis,

CMO, Celgene

5.50pm