Eurordis presentation

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www.eurordis.org

• A 2007 Update on European policy
• on drug development and regulatory affairs
• the Eurordis Perspective on Biosimilars

THE NORD CORPORATE COUNCIL MEETING 21 MAY 2007,
WASHINGTON DC

21 May 2007, Washington, Capilol Hilton
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www.eurordis.org

• The European Organisation
• for Rare Diseases

Yann Le Cam Chief Executive Officer

21 May 2007, Washington, Capilol Hilton
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CONTENT

• Eurordis and its advocacy priorities in 2007
• The newly adopted EU Regulation on Drugs for
  Paediatric Use
• The soon to be adopted EU Regulation on Advanced
  Therapies
• Orphan drug policy in EU a 2007 update
  Eurordis approach
• Biosimilars in Europe Eurordis approach for
  rare diseases

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EURORDIS IN BRIEF

• Founded in 1997
• Patient-driven
• Non governmental - Not for profit
• 286 member organisations in 33 countries
• gt 1.000 rare diseases covered
• 18 staff in Paris and Brussels
• 120 volunteers involved in task forces, steering
  committees, working groups, Board of Directors,
  translation
• gt 400 patient groups participated in Eurordis
  activities in 2006
• Budget 2007 2,3 million
• Members (40), Public (30), Indus (20), Other
  private (10)

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EURORDIS MISSION

• To build a strong pan-European community of
  patient organisations and people living with rare
  diseases
• To be their voice at the European level
• and directly or indirectly to fight against
  the impact of rare diseases on their lives.

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EURORDIS PRESENCE IN EUROPE
286 members in 33 countries (20 EU countries)
Countries with a National Alliance
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ADVOCACY POLICY ACHIEVEMENTS

• Contribution to the adoption of the EU Regulation
  on Orphan Medicinal Products in 1999
• Contribution to the adoption of the EU Regulation
  on Medicinal Products for Paediatric Use in 2006
• Contribution to the promotion and maintenance of
  rare diseases as a EU public health policy
  priority
• Contribution to the promotion and maintenance of
  rare diseases as a priority in the EU Research
  Framework Programmes

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ADVOCACY POLICY DEVELOPMENT

• Front line advocacy for the EU Regulation on
  Advanced Therapy Medicinal Products, possible
  adoption 2007
• Promote rare diseases as a long term public
  health policy
• Commission Communication Recommendations on
  Rare Diseases
• EU High Level Group on Rare Diseases
• National rare disease plan in EU Member states
• Promote policy on centres of expertise and
  European reference network for rare diseases at
  national EU levels

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EURORDIS IS PRESENT IN MANY EUROPEAN INSTITUTIONS
AND PLATFORMS

• European Institutions
• Committee for Orphan Medicinal Products at the
  EMEA (COMP)
• EMEA/CPMP Working Group with Patients
  Organisations
• Rare Disease Task Force at DG Health and Consumer
  Protection
• EU Health Policy Forum at DG Health and Consumer
  Protection
• EU High Level Groups on Health Services and
  Medical Care
• European Platforms
• European Patients Forum (EPF)
• International Alliance of Patients Organizations
  (IAPO)
• European Platform for Patients Organisations,
  Science, and Industry (EPPOSI)
• European Forum for Good Clinical Practice (EFGCP)

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EU PAEDIATRIC REGULATION

• Name EU Regulation on Medicinal Products for
  Paediatric Use
• Status Published December 27, 2006
• Final Agreement of European Parliament Council
  June 1, 2006
• Commission Proposal September 2004
• Launch under EU French Presidency 2000
• Overall, 6 years of policy development,
  legislative process...
• Very much patterned on US Legislation while
  trying to learn from experience gained in the USA
• Initiated by regulators and European Commission,
  supported first by academia and patients, last by
  industry, not by payers
• Front line advocacy by rare diseases groups
  (Eurordis)

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NEW REQUIREMENTS

• Marketing autorisation application must include
• Data on the use of medicines in children
• Resulting from an agreed Paediatric Investigation
  Plan
• Possible waivers of this requirement for
  medicines unlikely to benefit children
• Possible deferrals of the timing of the
  requirement
• To ensure medecines are tested only when it is
  safe to do so
• To prevent the requirement to delay the
  autorisation for adult

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NEW REWARDS

• 6-months extension of patent protection
  (supplementary protection certificate) for all
  the product line if studies are performed
  whatever the outcomes
• Paediatric Use Marketing Autorisation (PUMA) a
  new type of Marketing Autorisation allowing for
  10 years of data protection on new innovation
  (new studies) on off-patent products
• For orphan medicines, a 2-years extension of
  market exclusivity
• gt 10 2 years of market exclusivity on adult
  paediatric indications on the orphan medicinal
  products studied in children
• gt a very strong new incentive for compliance to
  requirement

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SUPPORTING MEASURES

• New Paediatric Committee (PDCO) at the EMEA
  including Member states representatives, CHMP
  members, experts (pediatricians, methodologists,
  etc)and patient representatives
• Free Scientific Advice for the industry, provided
  by the EMEA
• EU network of investigators and trial centres to
  conduct the research and development required (EU
  funding)
• EU inventory of the therapeutic needs of children
  to focus research, development and authorisation
  of medicines (ongoing)
• Publicly available database of paediatric studies
• Commitment to EU funding into studies on
  off-patent medicines for children (the so-called
  M.I.C.E. Programme)
• gt Real new opportunities in Europe for companies
  specialised in orphan drugs development

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NEXT STEPS

• EMEA website (available)
• Regulation
• Frequently asked questions (very well presented)
• Some existing Guidelines
• Enter into force 26 January 2007 gt requirement
  will apply for all MAA for products submitted
  from 26 July 2008
• Implementing rules by European Commission and
  related Guidelines by EMEA to be published
  (ready)
• Creation of the Paediatric Drug Committee and
  appointment of members (July 2007)
• EMEA staff some recuited (3), more ongoing
  recruitments
• Publication of the inventory of needs (expected
  2007 based on work by the Paediatric Experts
  Group at EMEA
• EU Budget appropriation for M.I.C.E.Programme??

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EU ADVANCED THERAPY REGULATION

• Name EU regulation on Advanced Therapy Medicinal
  Products
• Status ongoing legislative process
• Commission Proposal November 2005
• European Parliament rapporteur MEP Miroslav
  Mikolasik (Slovaquia, EPP) key shadow role of
  MEP Peter Liese
• European Parliament Hearing May 2006 a turning
  point
• Major debates on ethics and embryonnic stem cells
• Adoption by European Parliament at first reading
  in Plenary session (vote 403 yes vs 264 no)
  against the opinion of the rapporteur on April
  25!
• Process started in 2003
• Four years of advocacy Eurordis leading patient
  voice on EU Advanced Therapy Regulation and
  crucial on ESC research
• More details on Eurordis website with many links
  to official texts, press releases, position
  papers, statements

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WHAT IS GOING TO CHANGE?

• Marketing authorisation for advanced therapy
  products will be EU centralised procedure
• Creation of the Committee for Advanced Therapies
  (CAT) within the European Medicines Agency (EMEA)
  to provide advice in the process the CAT will
  be in charge of developing criteria and
  guidelines for the evaluation of these products
• Stronger pharmacovigilance long term provision
  of technical and risk management requirements to
  ensure quality and safety.
• Simplified and stronger procedure
• More competent experts on evaluation committee
• Adapted general guidelines and personnalised
  advice to sponsors
• International competitiveness of EU

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NEXT?

• Possible adoption by EU Council of Ministers on
  31 May 2007
• If not gt new text by the Commission, new
  rapporteur, second reading at European
  Parliament, second Council gt gt 1 year
• If yes gt overall a very short timeline 4 years
• Creation of Committee Advanced Therapy and
  implementation not before 2008
• Overall, Europe offers today
• An opportunity for US based companies working on
  advanced therapies for rare diseases to develop
  innovative products addressing unmet medical
  needs in a favourable environment
• A clear political and stakeholders support for
  research and therapy development on all new
  advanced therapies including ESC
• A robust new regulatory framework to develop
  these advanced therapies

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EU POLICY ON ORPHAN DRUG UPDATE 2007

• Status
• 450 orphan drug designated
• 37 authorised as of May 2007
• COMP 3rd mandate 2006-2009
• Chair Pf Kerstin Westermark (Sweden), Co-Chair
  Birthe Holme (Eurordis)
• COMP Working Group Interested Parties, Chair
  Yann Le Cam
• Consolidation
• Commission report on EU Orphan Regulation
  sucessfully passed
• Very positive and a success in consolidating the
  regulation
• Guideline on Article 8.2 for the review of orphan
  status at 5 years
• Economic visibility on maintenance of orphan
  status
• Orphan status is assessed (a) initialy when
  applying for ODD (b) systematicaly and based on
  same criteria when applying for MA no big issue,
  low burden for MAH, a safety net for regulators
  ( c) at 5 years on very exceptional occasion if
  triggered by a Member state and if MAH fails to
  demonstrate it still fulfill the criteria,
  flexibity in applying the policy

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EURORDIS APPROACH ADDING VALUE TO EU ORPHAN
STATUS (done)

• Lower fees
• Fee waivers (for orphan status)
• Fee reductions (for protocola assistance and
  scientific advice)
• Fee reductions for Small and Medium Size
  Enterprises for MAA, post-MA..
• Market exclusivity
• Additional incentive 10 2 years if paediatric
  studies performed
• Securing the incentive Review at 5 year severely
  minimized
• Guideline on clinical trial in small population
  (February 2007) to promote design and statistical
  methods requiring less patients, less time, less
  resources, lower failure rate in development

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EURORDIS APPROACH ADDING VALUE TO EU ORPHAN
STATUS (ongoing)

• Significant benefit
• Toward really significant benefit translating
  into real clinical benefits for patientsand
  favouring the first company inputting more
  burden on the second and following ones
• Guideline under development based on clearer
  concept experience
• US/EU
• Toward common format of orphan applications at
  FDA and EMEA
• Allowing parallel designation, lower work on
  sponsors, speed up process to get designation on
  both side, promote orphans with the right message

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EURORDIS APPROACH ADDING VALUE TO EU ORPHAN
STATUS (to do)

• Clinical Research Grants for Orphan Designated
  Drugs
• Possibly starting 2009
• Phase 1 2 of designated orphan drugs
• Specific call for proposal by DG Research at
  European Commission (FP7)
• Availability to patients Price
• Common policy criteria for health technology
  assessment between MSs
• Common policy criteria for pricing
  reimbursement between MSs
• EU centralised procedure on a volunteer based
• EU centralised procedure created by European
  Commission
• Tax credit by Member states
• Commission Communication Recommendations on
  Rare Diseases
• National Rare Disease Plan in EU Member states

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BIO SIMILARS IN EUROPE

• Revised EU Pharmaceutical Legislation adopted in
  2004
• Comprehensive regulatory framework for the
  evaluation, authorisation and monitoring of
  biosimilar products
• These specific provisions being part of the
  pharmaceutical regulatory framework lay down how
  the biological similarity of the substance to an
  innovative medicine can be scientifically
  established, as a precondition for the
  authorization to market the product in the EU.
• Unlike generics, where chemical identity has to
  be proven, biological similarity is assessed
  following the performance of additional trials,
  set up on case by case base
• Bottom line Comparability study needed to
  generate evidence substantiating the similar
  nature in term of quality, safety and efficacy
  between the similar biological product and the
  reference one

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BIO SIMILARS IN EUROPE

• Scientific guidelines on the various quality,
  pre-clinical and clinical aspects that are
  specific to biosimilars are being developed by
  the European Medicines Agency (EMEA)
• Main guideline Guideline on Similar Biological
  Medicinal Products
• EMEA Draft Guideline (CHMP) published November
  2004
• Public Consultation deadline February 2005
• Adoption (CHMP) October 2005
• Coming into effect November 2005
• Guideline does not apply to gene and cell therapy
  (future Reg.)
• 1st EU marketing autorisation for similar
  biological medicinal product Omnitrope
  (somatropin, Sandoz) comparable to Genotropin
  (Pfizer) April 2006

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BIO SIMILARS IN EUROPE

• EMEA Biosimilar Medicinal Working Party
• Created March 2005
• 8 experts
• Chair Pekka Kurki
• Mandate
• Review update guidelines
• Advise CHMP SAWP
• International cooperation with other regulatory
  authorities
• Liaison with interested parties (including
  hearing of industrie PAG)
• WorkPlan 2007

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EURORDIS APPROACH TO BIO SIMILARS

• Not a A priority but a C priority for
  Eurordis no position paper of Eurordis on the
  topic, but develop knowledge base, vigilance,
  follow up, work through other partners
• Priority given to EU Paediatric Regulation, EU
  Advanced Therapy Regulation, consolidation of EU
  Orphan Regulation adding value to orphan status
• Regulating biosimilars is a broad issue impacting
  all biotechnology derived products for frequent
  and rare diseases
• Some specific issues recognised as relevant to
  small populations, therapies for rare diseases
  and orphan drugs
• Biosimilars are less costly
• Biosimilars are as safe efficient
• Eurordis relies on European Patient Forum (EPF)
  and on International Alliance of Patient
  Orgnaisations (IAPO) to address biosimilars
  policy and to represent Eurordis (with our
  support)

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EURORDIS APPROACH TO BIO SIMILARS

• Biosimilars are as safe efficient? Yes, but
• If properly studied gt comparability study
• Why a rare disease patients would like to move
  from her/his therapy to a clinical trial to
  assess the same quality, safety and efficacy?
• How long is it going to take to recruit the
  number of patients needed for a comparability
  study in a therapeutic area where there is
  already a treatment available in a small
  population?
• What will be the level of confidence of
  prescribing doctors and of patients and families
  when the drug is approved?
• Biosimilars are less costly? Yes, maybe
• With clinical trials in such conditions for an
  orphan drug it still has to be demonstrated that
  the cost of teh new drug will really be lower
  than the reference orphan drug

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EURORDIS APPROACH TO BIO SIMILARS

• So, what comes first for Eurordis?
• Safety of products for patients and comfort comes
  first, no compromise
• EMEA Guidelines on Biosimilar product is really
  ok for products intended for rare diseases and we
  dont have any concerns yet
• No hurry EU Orphan regulation started 2000, 10
  years exclusivity 2 years for most products gt
  2012 vs Policy on biosimilars 2004 Guidelines
  2005 which will be modified based on experience
  gained on all biological products other than
  orphanwe have the time to observe analyse
  based on real world experience before making
  recommentions
• No undermining of the orphan drug dynamic in EU
  and sustain economic visibility for orphan drug
  marketing holder
• Price is an issue Economic burden for society
  is an issue Real access for patient is an issue
  gt these issues are real but biosimilars will
  not solve them and tehy will have to be addressed
  from somewhere else

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EUROPEAN CONFERENCE ON RARE DISEASES LISBON 2007
Join us!

• When 27-28 November 2007
• Where Lisbon, Portugal
• Expected number of participants 500
• Number of speakers 40
• 2 full days around key themes
• Overview of rare diseases policies at EU and MSs
  level Commission Communication
• New policy on Rare Disease Centres of Expertise
• New European networks of services to patients
• Session on research policies EU strategy
• Session on orphan drugs in EU
• 5 languages English (main), French, German,
  Spanish, Portuguese

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www.eurordis.org

Yann Le Cam Chief Executive Officer ylecam_at_eurordi
s.org
Fabrizia Bignami Therapeutic Dev.
Officer fbignami_at_eurordis.org
François Houÿez Health Policy Officer fhouyez_at_euro
rdis.org
Flaminia Macchia European Public Affairs
Officer fmacchia_at_eurordis.org
Anja Helm Manager of relations with
POs ahelm_at_eurordis.org
Jerome Parisse-Brassens Communications
Officer jparisse_at_eurordis.org