European Policies for Rare Disease Patients: Workshop

Improving Access to Orphan Drugs in Europe

Mark Rothera

Vice President and General Manager

Europe, Middle East & Africa

Shire Human Genetic Therapies (HGT)

European Health Forum Gastein

6 October 2011

18.30 – 20.00

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SHIRE’S COMMITMENT TO RARE DISEASE PATIENTS

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To be as brave as the people we help

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We enable people with life-altering conditions to lead better lives

$Market capitalisation: $18.5 billion*

~5,000 employees globally

Top 37 biopharmaceutical

company worldwide1

2010 Revenuesof $3.5B2

1 Pharm Exec 50, 2 2010 Sales*As of September, 2011

NORD 2010 Partners in Progress Award

Specialty

Pharmaceuticals (SP)ADHD, Gastrointestinal (GI),

Renal, Hematology

Advanced BioHealing(ABH)

Regenerative Medicine

Human GeneticTherapies (HGT)

Orphan Diseases

Facility of the Year awards

Bringing Value to Patients, Healthcare Systems and Society

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ORPHAN DRUG PRICING AND REIMBURSEMENT ISSUES

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2. Can we afford it? (budget impact)

Available BudgetBudgetImpact

1. Is it worth it? (cost-effectiveness)

Clinical effect of

comparator

Added clinical effect

Cost of drug X V

R

D Is the additional clinical

effect worth paying for ?

V: Cost of drug X

R: Cost of comparator

D: Additional cost of drug X

Conventional Health Technology Assessment (HTA)

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Current HTA Methods Can Result in Restricted and Inequitable Access to Orphan Drugs for Patients

Conventional HTA scope too narrow to demonstrate the full potential of orphan drugs

Rarity means weight of evidence is not the same as for conventional diseases

� Small, heterogeneous populations

� Short duration of follow-up of studies

� Limited scientific understanding/ consensus on clinical endpoints

� Limited hard clinical outcomes such as survival

� Limited natural history data

� Lack of consensus/data on comparators

HTA does not sufficiently recognise rarity, seriousness of the condition, availability of alternative therapies, innovation nor burden on patient and society

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Shire Supports a Distinct HTA Methodology the Evaluation of Orphan Drugs

Example: New Advisory Group for National Specialised Services (AGNSS) Process, in England

•Shire was selected as the partner of choice for the AGNSS process for very rare diseases, to ensure:

• HTA procedures are flexible enough

• A broader set of considerations are used

• Cost-effectiveness is a minor consideration

• Views of patients and caregivers included

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To be as brave as the people we help

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Clinical Added Value of Orphan Drugs (CAVOD)

� June 2009: Council Recommendation on RD adopted

“Sharing Member States′ assessment reports on the therapeutic or

clinical added value of orphan drugs at Community level where the

relevant knowledge and expertise is gathered, in order to minimise delays

in access to orphan drugs for rare disease patients”

• CAVOD Mechanism is a work in progress:

• Common assessment report • Opportunity to reduce patient accessdelays

IMPORTANCE OF EARLY ACCESS PROGRAMMES FOR RARE DISEASE PATIENTS

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Early Access Programmes are Critical for Orphan Drugs- More Harmonisation is Needed

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Country Early Access Access

Germany No Easy

Austria UC/NP Slow

Belgium UC/NP Slow++

Denmark UC/NP Complex

Finland UC/NP Complex

France TUA Rapid

Spain UC/NP Classic

Greece UC/NP Classic

Ireland UC/NP Classic

Italy TUA Classic

Luxemburg UC/NP Classic

The Netherlands UC/NP Classic

Portugal Depends on case Depends on case

The UK UC/NP Slow

Sweden UC/NP Easy

Source: EMA, London. UC = Compassionate Use, NP = Nominative Base of patients, TUA = Temporary Use Authorisation

To be as brave as the people we help

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Early Access Programmes enabled Hunter patients to receive ERT 6-12 months faster than

normal.

0

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12000

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20000

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Cumulative vials shipped: July 2006-Dec 2007

Actual Total cumulative pre & post launch vials sold Actual Pre-approval cumulative vials sold

Jul 06

Aug 06

Sep 06

Oct 06

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Case Study: Launch of Elaprase in Hunter syndrome Mucopolysaccharidosis II (MPS II)

0%

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Jun-06

Jul-06

Aug-06

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Jan-07

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Jun-07

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To be as brave as the people we help

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Sales data only for W. Europe

Case Study: Launch of Elaprase in Hunter syndrome Mucopolysaccharidosis II (MPS II)

Less than One Year after EC Approval, 66% of Diagnosed Hunter Patients had Access to

Elaprase Treatment in Western Europe

Elaprase Patients as % of Diagnosed Patients

% of Diagnosed Pool treated

with Elaprase

EC

Ap

pro

val

LOOKING TO THE FUTURE OF RARE DISEASE POLICIES IN EUROPE

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EU OMP Regulation and the Importance of National Plans

Council Recommendation on Rare Diseases, June 2009

�Adopt national plan by the end of 2013

�Ensure integration of multi-level initiatives

�Define priority actions

�EUROPLAN framework

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EU orphan drug regulation: 12 years on

�>750 orphan designated products

�As of 2011, over 65 orphan drugs approved

�Uniformity of advice

�Centralised procedure for Marketing Authorisation

�Industry incentivised to develop orphan drugs

Looking to the Future

� Shire is committed to continued investment and development of innovative treatments for rare diseases

� Essential to have a stable and favourable policy and regulatory framework to incentivise development and patient access to orphan drugs

� Shire is committed to shaping and supporting European actions related to orphan drugs as well as at the member state level

� Shire recognises the purpose of HTA and is leading the way in the development of methods and processes which fully reflect and capture the value of orphan drugs for patients, payers, physicians, and policymakers

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Thank You

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