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To a national plan strategy for Rare Diseases in Belgium

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To a national plan ( strategy ) for Rare Diseases. in Belgium. Herwig Jansen ... Ibuprofen is used to treat Patent ductus arteriosus in neonates (RD) ... – PowerPoint PPT presentation

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Title: To a national plan strategy for Rare Diseases in Belgium


1
To a national plan ( strategy ) for Rare
Diseases in Belgium
  • Herwig Jansen

Rue Juliette Wytsmanstraat 14 1050 Brussels
Belgium T 32 2 642 51 11 F 32 2 642 54 10
email info_at_iph.fgov.be http//www.iph.fgov.be
2
Definition Rare Disease (RD)
  • Disease prevalence lt 5 /10.000 (EU)
  • Depends of
  • population (Leprosy is a rare disease in
    Belgium, but common in central Africa)
  • region (Thalassemia, is rare in Northern Europe,
    but frequent in the Mediterranean region)
  • - time (AIDS was an extremely rare disease, later
    it was rare, and now it is a more and more
    frequent disease in certain populations)

Other definitions USA lt 1/200.000 WHO lt 6.5
10 / 10.000 JAPAN lt 1/50.000
Lists of RD ? NIH list (n6000)
3
Definition of Orphan Drug (OD)
  • Medicine to treat a rare disease

Ibuprofen is used to treat Patent ductus
arteriosus in neonates (RD) Viagra is used for
the treatment of Pulmonary Arterial Hypertension
(RD)
Not always clear
Real Orphan Drugs and drugs with Orphan Drug
Status for some conditions
4
Because of their rarity and due to the lack of
sufficient scientific and medical knowledge, many
patients are not diagnosed. Their diseases
remain unidentified
Prader-Willi syndrome
Orphan drugs have no priority for
commercialization ( no profit )
Rare diseases are serious chronic diseases, and
are often life-threatening. Patients without a
diagnosis and with inappropriate care are the
ones who suffer the most.
Pompe disease
5
Paradox of rarity
Paradox of rarity the diseases are rare, rare
diseases patients are many. It is therefore not
unusual to have a rare disease.
EU (27 countries) 28 million people have a
rare disease combined population of BE and L
and NL (6 of EU population)
6
Rare Disease vs Genetic Disease
  • All genetic diseases are rare
  • Most Rare Disease are genetic but not all
  • The concept of genetic diseases is the one used
    for Rare diseases.
  • NL erfocentrum / VSOP
  • UK rare disease condition treated by an
    expensive,
  • non affordable drug

Progeria (Hutchinson-Gilford syndrome )
7
Birth of the concept of RD
  • USA
  • 1983 Orphan drug regulation
  • 1993 Office of Rare diseases
  • EUROPEAN CONTINENT
  • 1985 Denmark Rare Disorders Denmark
  • 1989 Sweden Ägrenska (unikt nationellt
    kompetenscentrum)
  • 1995 France Office of Orphan drugs
  • 1996 Orphanet
  • 1997 Eurordis
  • 1998 Italy Priority in Public Health

8
European actions
History
  • 1999-2003 Community action programme on RD
  • 2003-2007 RD a priority in the EU Public Health
    Prog.
  • 2008-2013 RD a priority in the EU Health Prog.
  • 2008-1013 White Paper  Together for Health a
    strategic approach for the EU 
  • FP6 (Orphan Platform ERA-net) and FP7
  • Orphan Medicinal Product Regulation (1999)

9
European actions
Open process of development
  • Involvement of stakeholders
  • EU Task Force on Rare Diseases (2004-2009)
  • (Herwig Jansen WIV-ISP is a member) COUNCIL
    RECOMENDATION
  • Public consultation
  • Launched in Lisbon (Nov 2007)
  • 584 responses from 15 Member States (MS)
  • Broad support for a coherent framework policy
  • Impact assessment
  • Improved efficiency for patients, health systems
    and health authorities

10
European actions
Priorities
  • Improving recognition and visibility on rare
    diseases
  • The EuorordisCare2 Survey (2004) ? 25 of
    patients had to wait 5-30 yrs from early symptoms
    to confirmatory diagnosis
  • Before confirmatory diagnosis, 40 first received
    an erroneous diagnosis
  • 25 of patients had to travel to a different
    region, 2 to a different country for diagnosis
  • The EU will contribute to the ongoing process of
    revision of the ICD (Including RD)
  • Commission will establish an inventory of RD to
    be periodically updated
  • Commission will continue to support RD
    information networks through the Health program
    and the FP7
  • Supporting policies on rare diseases in the MS
  • Developing European cooperation, coordination and
    regulation for RD

11
European actions
Priorities
  • Improving recognition and visibility on rare
    diseases
  • Supporting policies on rare diseases in the MS
  • The Member states are invited to establish
    national or regional action plans for RD by
    implementation of the actions suggested in the
    Communication and Recommendation (due date 2011)
  • The commission will provide European guidelines
    for the elaboration of these action plans
    (EUROPLAN)
  • Developing European cooperation, coordination and
    regulation for RD

12
European actions
Priorities
  • Improving recognition and visibility on rare
    diseases
  • Supporting policies on rare diseases in the MS
  • Developing European cooperation, coordination and
    regulation for RD
  • To recommend inclusion of national initiatives in
    the national plans of RD
  • Create repertory of existing centers of Expertise
  • Establish a procedure for designation and
    accreditation methodology of EU reference
    Networks for Rare Diseases
  • Centers of Expertise
  • National centers
  • Regional centers
  • Where patients may be referred
  • Reference networks
  • (of centers of expertise)
  • Characterised by communication
  • Production of guidelines
  • Shared expertise for patients

13
European actions
Council recommendation
  • April 24, 2009 Working party on Public Health
    (All MS delegates) has worked out the Council
    Recommendation on a European action in the field
    of rare diseases ( initially written by the Rare
    disease task force)

Future actions
  • June, 2009 approval of the Council
    Recommendation
  • June Oktober 2009 transformation of the RDTF
    to a Advisory Board of the EU commission
  • End 2011 call for Tender on improving definition
    of rare diseases taking into account incidence
    aspects (0.5 million of budget)
  • End 2013 classification and codification (as
    topic advisory group for WHO)
  • 2010 Orphanet database translation to 22
    languages
  • 2010 - 2014 support to the creation of EU
    networks on information on RD
  • 2010 - 2014 support to the creation of EU
    registries and databases on RD

14
European actions
Post EU regulation
  • 2001 Dutch Steering Committee on Orphan Drugs
  • 2002 Italian regional centres of reference
  • 2003 German support to research networks
  • 2003 Spanish support to research networks
  • 2004 French National Plan
  • 2006 ERA-NET for research programs
  • 2006 Belgian steering group for Rare Diseases and
    Orphan Drugs
  • 2007 Portuguese national plan
  • 2007 Romanian initiative of rare diseases
  • 2007 Italian Registry of Rare Diseases (Istituto
    Superiore di Sanità Roma)
  • 2008 Bulgarian national plan
  • 2008 Orphanet in 38 countries

15
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16
To a national action plan in Belgium
  • No coordinated activity before 2006
  • December 2006 1st National Symposium on Rare
    Diseases
  • January 2008 the Management committee if the
    fund has been officially established as a Fund in
    the King Baudouin Foundation
  • KCE Project evaluation of procedures about
    orphan medicines
  • December 2008 Minister of public health meets
    the fund and promises an extension of facilities
    for Rare Diseases in the existing plan of Chronic
    Diseases
  • February 2009 Hearing Commission Public Health
    Resolution (RD) Avontroodt to the Federal
    Parliament
  • The Belgian Orphanet antenna has been established
  • A HTA project on Orphan Drugs started and should
    be finalized in December.

17
FONDS ZELDZAME ZIEKTEN
18
Funding members Dec 2006
19
Aims of the fund Rare Diseases Orphan Medicins
  • AIMS
  • Collection of epidemiologic data
  • Recognition of the specific needs and access to
    therapies
  • Information for patients, professionals and the
    public
  • Training of professionals including medical
    doctors
  • Screening and testing procedures and facilities,
    including reimbursement
  • Identify Centers of Reference
  • Improve access to orphan drugs
  • Support for those who care for the patients
  • Stimulate research
  • National and International collaboration
    (Europlan)

NATIONAL ACTION PLAN
20
Creation of 4 working groups
  • WG 1 Collection of epidemiological data and How
    to establish a National Register for rare
    diseases WG leader Herwig Jansen (WIV ISP)
  • WG 2
  • (2a) Cost (of the management) for patients,
    linked to the question how far is society
    willing to go, how far solidarity will reach?
  • (2b) Redefinition of rare diseases. Certain
    developments are threatening the principles of
    solidarity WG leaders Claude Sterckx and Marc
    Boogaert
  • - WG 3 Information to patients and their family,
    health professionals and general public
    collecting reliable information, adapted to the
    Belgian situation and taking care of language
    needs. WG leader Klaartje Bruyninckx.
  • WG4 Centres of Reference WG leaders François
    Eyskens and Jean-Jacques Cassiman.

21
ORPHANET Belgium Sci Advisory Board Belgium
  • Members
  • Abramovicz Marc, ULB
  • Bogaert marc, RIZIV
  • Bours Vincent, ULg
  • Cassiman David, KUL
  • Delcroix Marion, KUL
  • Eyskens Francois, UA
  • Jansen Herwig, WIV-ISP
  • Longueville Jacques, UG
  • Schoffski Patrick, KUL
  • Velu Thierry, ULB

Orphanet team Cassiman Jean-Jacques Rens Iris
Swinnen Elfriede Veerle Govers
22
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23
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24
Summary
  • Before 2006 no coordinated action in Belgium
  • Today
  • Coordinated action is becoming reality
  • Resolution to the Belgian Parliament
  • Fund rare diseases and orphan medicines is
    charged to write a Belgian strategy for Rare
    Diseases

25
To a national plan ( strategy ) for Rare
Diseases in Belgium
  • Herwig Jansen

Rue Juliette Wytsmanstraat 14 1050 Brussels
Belgium T 32 2 642 51 11 F 32 2 642 54 10
email info_at_iph.fgov.be http//www.iph.fgov.be
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