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EuropaBio Information Day

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Rare Diseases. 46% of all Bio Pharmaceuticals authorised by FDA between 1995 ... Rare Disease therapy development has taught us that the way forwards is to work ... – PowerPoint PPT presentation

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Title: EuropaBio Information Day


1
EuropaBio Information Day
  • The Invisible Revolution
  • Looking back, looking forward

2
Michael Griffith
  • Chief Executive
  • Fighting Blindness
  • Chairman
  • European Platform for Patients Organisations,
    Science and Industry

3
Learning from Rare Diseases the Potential for
Change
  • May 30th 2006

4
Michael Griffith
  • Chief Executive, Fighting Blindness
  • Chairman, EPPOSI
  • Chairman, Debra Ireland
  • Chairman, IPPOSI
  • Chairman, MRCG
  • Chairman, C4C
  • Management Committee, Retina Intl.
  • Executive Committee, Debra International

5

6
What can we learn from Rare Diseases
  • 46 of all Bio Pharmaceuticals authorised by FDA
    between 1995 and 2000 were for rare diseases
  • Reduced a little but over the past 5 years 30
    of newly authorised Biologics have been Orphan
    Products
  • Marlene Haffner, Director of Office Orphan
    Products Development, FDA

7
  • Similar Position in Europe

8
Status of Orphan Applications April 2006
9
Status of Orphan Applications
10
Biotech Products in Orphan Applications
11
Future
  • Expect this trend to continue and increase
    common diseases breaking down into sub groups
    (personalized medicines)
  • Many drugs authorized for rare diseases have
    become blockbusters e.g.Epogen/Amgen and
    Procrit/Ortho Biotech - 5b pa 2001)
  • Many people now express doubt about big Pharma
    blockbuster search approach

12
Whats Different about Rare Diseases?
  • Logical approach to therapy development
  • Basic research to establish cause of disease
  • Development of method to address that cause
    (intelligent drug design)

13
Economics
  • Latest cost of drug development 2 billion
  • Escalating at incredible speed
  • Unsustainable in longer term

14
Economics (2)
  • Now feel intelligent drug design approach will
    become the norm, even for big Pharma
  • Economics are very challenging
  • What can we learn from rare diseases?

15
Large Multinational Biotech Companies
  • Many Biotech companies which started in Rare
    Diseases have become huge multinationals
  • e.g.
  • Amgen
  • Genentech
  • Genzyme

16
Learning from Rare Diseases
  • Orphan Medicinal Products Regulation / Orphan
    Drugs Act
  • Committee for Orphan Medicinal Products
  • Several examples of Patient Groups working
    closely with Industry e.g. Pompe Disease
    associations working with Pharming/Genzyme
    (Myosyme)

17
Learning from Rare Diseases (2)
  • Patients supported basic research, clinical
    research discussions with regulators and now
    reimbursers.
  • No reason why this type of approach cannot exist
    for common diseases
  • Developments in this regard already e.g. Proposed
    Patient Committees at EMEA for Paediatric
    Medicines Advanced Medicines

18
Patient Revolution
  • Rare Disease therapy development has taught us
    that the way forwards is to work with Patient
    Groups
  • Patient revolution is happening (recent book by
    Simon Roosendaal)

19
Patient Revolution (2)
  • Appointment of 2 Patient Representatives to main
    Board of EMEA
  • Patient representation must be by patient groups,
    not by research groups studying patients or by
    insurers or sick funds

20
Patient Revolution (3)
  • Patients and industry must work together to
    ensure equitable and timely access to beneficial
    innovation
  • Involvement of patients in consensus building in
    difficult societal issues such as the value of
    innovation, reimbursement, health care budgeting,
    access etc, is essential

21
Funding Patient Groups
  • All this must be recognised and underpinned by
    funding from EU as patient groups are working on
    shoestring budgets
  • Such funding as they get often comes from
    industry

22
Winston Churchill
  • Some regard private enterprise as if it were a
    predatory tiger to be shot. Others look upon it a
    s a cow that can be milked. Only a handful see it
    for what it really is the strong horse that
    pulls the whole cart

23
EU Funding
  • The EU Commission seems to oscillate between the
    tiger and the cow.
  • It should recognise that at best industry can
    only part fund patient organisations
  • Face its responsibility to fund these groups.

24
Conclusion
  • We can learn better ways to develop treatments
    from seeing what has happened over the last 25
    years in Rare Diseases.
  • Essential to work with patient groups
  • Essential for EU to fund these groups.
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