NHS Sickle Cell & Thalassaemia Screening Programme

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United Kingdom
Thalassaemia Society
1976 – Present Day
Running a Successful Society
To empower those affected by or working
with thalassaemia and to be their definitive
source of information, education and
research
Mission Statement
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1. To encourage promote and support all areas of prevention and to
allow accurate, informed choice.
2. To create and maintain the highest standard of care for
thalassaemics.
3. To encourage, promote and support all thalassaemics in socially
integrating and achieving their full potential.
4. To encourage, promote and support research in the treatment and
care of thalassaemia.
5. To resource the UKTS to fulfil, sustain and grow it activities in the
above areas.
6. To work towards a cure for Thalassaemia.
The Aims of a Society
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Organizational Structure
of the UKTS
The Management Committee
PRESIDENT
SECRETARY
TREASURER
ASSISTANT
TREASURER
VICE
PRESIDENT
BOARD
MEMBER
BOARD
MEMBER
BOARD
MEMBER
Management Committee Structure
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Research and Education
Over the past 35 years the UKTS has donated more than £2 million
pounds to research. Although all of our projects are important we above
all try to fund those that stand out head and shoulders above the rest, and
that can help change the lives of thalassaemics all over the UK and world.
 £5,000 contribution to the development of the Syringe Driver
(pump) in 1976.
 Investing in the development of the oral chelator, Deferiprone (L1),
funded and supported by the UKTS to the tune of £ 750,000.
 £27,500 for Growth and Development Research.
 Research into prevalence, transmission and treatment of Hepatitis C
(£54,000 in 1990)
 Support for Bone Disease research and treatment developments.
 Funding for thalassaemia patients’ register to 2002.
Research Funding
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 Development and set up of the first ever Pre Natal
Diagnosis screening, Dr M. Petrou, UCLH, £30,000 in
1979.
 Development of The National Standards For The Clinical
Care Of Children And Adults With Beta Thalassaemia in
2005 and 2008.
 Funding research into prognostic value of MRI imaging
£33,600.
 Creation and distribution of individual Patient Held
Record.
 Targeted workshops, doctors and patients conferences
every year, (£20,000-30,000).
 Assessment of novel beta globin transcription units within
retroviral vectors for gene therapy (£60,000 in 2000-3)
Research Funding cont.
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 Pre-implantation genetic diagnosis for thalassaemia £64,940
Dr M. Petrou, UCLH, 2003.
 £70,000 – development of an in utero approach for
thalassaemia gene therapy , Dr M. Antoniou & Dr S.
Waddington, KCL, 2008.
 £35,000 – development & characterisation of zinc finger
nucleases specific for the human beta globin gene, Dr A. Porter
& Prof I. Roberts ICL, 2009.
 Commissioned research to study psychosocial issues and
impacts affecting the transition from childhood to adulthood of
adolescents with Beta-Thalassaemia, Dr. S. Dyson, de Montfort
University, 2010
Research Funding cont.
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• £ 289,478 Asian Awareness Campaign in 1997/2000 to
increase awareness of the condition amongst the UK Asian
Communities and improve their INFORMED CHOICE
available for prevention to the individuals and couples in
these communities who are planning a family.
 £ 20,000 on education of at risk communities, through ethnic
radio, television, magazines, newspapers and website (per
year)
 £ 20,000 Website and publications.
 £ 20,000 – £ 30,000 Targeted workshops and patients
conference (per year)
 £ 16,000 for the Patient Held Record
Education Funding
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 £ 3,000-£ 5,000 Annual support grants for promising doctors
to attend TIF’s e-MSc course on Haemoglobinopathies at UCL.
 Development of 3 DVDs for increasing awareness about
screening, and treatment in the at risk community and the
general population.
 Development and implementation of a 3 year North England
Awareness Project in association with the NHS SC&T
Screening Programme for outreach and community work
carried out by the UKTS focusing on communities affected by
Thalassaemia in the north of England.
Education Funding cont.
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NHS Sickle Cell & Thalassaemia Screening Programme
The NHS Sickle Cell & Thalassaemia Screening Programme aims to
establish high quality newborn screening programmes for sickle cell
disorders and antenatal screening programmes for sickle cell and
thalassaemia. Members of Steering Committee and all subcommittees.
National Standards Clinical Care of Patients with Beta
Thalassaemia 2005 (revised in 2008)
Co-authored by a multi-professional group and designed to address
the concerns that clinical services though of a high standard were not
consistent around the country. Have become a leading document for
clinicians of UK and international model.
Leading Member of the UK Dept. of Health Clinical Service
Development Group and The National Haemoglobinopathies
Project
Created to develop the vision for integrated, equitable and effective
haemoglobinopathy lifespan care and to implement it across England.
High Level Participation
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Permanent Member of EURORDIS (European Rare Diseases
Organisation).
UKTS Expert patients contribute their knowledge and expertise in many fields
to help other patients with Rare Conditions in the EU
Permanent Member of EPPOSI.
A collaborative not-for-profit, partnership-based multi-stakeholder think tank
based in Brussels, to develop workable European Models for Chronic
Conditions Management, HTAs, Rare Diseases and foster Innovation.
Founding and long term Board Member of TIF.
UKTS expert patients have been continuously contributing in all areas of TIF
projects, initiative and activities as well as on many delegation missions to
countries around of the world. Taken part in meetings with Government
Ministries, Health Authorities, clinicians, patients and parents and have
assisted many countries in developing better Healthcare and effective Patient
associations.
High Level Participation cont.
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Regular contributor to international EU conferences
on Healthcare Policy, Development, Regulation, Access to
Medications, Health Technology Assessments and their impact.
Advisors to the European Medicines Agency (EMA)
As the EU’s Drug Regulatory Authority we advise their Scientific
Advice W.P. for Drug licensing and approval of new upcoming
medicines relevant to thalassaemia.
Member of UK All Parliamentary Party Group for Sickle
Cell and Thalassaemia regularly meeting and lobbying senior
parliamentarians in both Houses of Lords and Commons to
improve patient services and conditions.
High Level Participation cont.
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Advisors to the National Institute of Clinical Excellence
(NICE)
Advising them on the remit, scope and criteria, of their assessment
for access by patients to all chelators on the NHS. Involved in the
18 month evaluation for chelator efficacy and economic benefit
driving home the importance and benefits to the patients having
free access to all 3 medications.
Founding and Board Member of the National
Haemoglobinopathies Register (NHR) An NHS wide
database of patient events, and outcomes aiming to a monitor
patients with red cell disorders in the UK. It collects data required
by the Department of Health from haemoglobinopathy centres
aiming to optimise and to improve patient care, nationwide.
High Level Participation cont.
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Founding and Board Member of the UK Forum of
Haemoglobinopathies
Member of this professional organisation of doctors working on
HbOs contributing the patient view on the peer review of
children's care services in hospital, future policy implementation,
ensuring UKTS Standards of Care are implemented nationwide.
Board Member of the Genetic Alliance Group
A national charity of patient organisations with a membership of
over 140 charities supporting all those affected by genetic
disorders; aiming to improve the lives of people affected by
genetic conditions by ensuring that high quality services and
information are available to all who need them.
High Level Participation cont.
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All mature societies at some point will need to re-evaluate their long
term strategy.
As treatment, quality of life and life expectancy increases then we
find that a society will need to continue research as thalassaemia
evolves from now as a condition. However we also need to recognise
that there will be a shift towards the following:
 Attentions to the needs of an older thalassaemic population.
 More demand and pressure by the developing world for a cure.
 Effective parliamentary/health service lobbing.
 New challenges arising i.e. Personalised Medicine treatments.
The Future.... is ours to reach it
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At both local and international levels we need not just to
continue the work that we do but to also provide a global
strategy for all society :
 One standard worldwide, a common fund, open mindedness to new
treatments and ideas.
 Genetic Research into curing Thalassaemia
 Optimising Treatment & Protocol Standards
 Effect care delivery that allows patients their full economic activity
and education.
 By looking after ourselves, by questioning, by taking responsibility
for our health and our treatment, by focusing on the positive elements
of thalassaemia we become the informed patient.
 By becoming the informed and expert patient we increase our survival
rate and achieve “normality”.
The Future....is ours to reach it
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THANK YOU
e-mail : [email protected]
website : www.ukts.org
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