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WWW.MATRIX-NI.ORG
MATRIX REPORT : VOL 2
10.2008
LIFE & HEALTH SCIENCES
HORIZON PANEL REPORT
PROSPERITY AND HEALTH DELIVERED BY SCIENCE
PREPARED FOR MATRIX BY

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MATRIX LIFE & HEALTH
PANEL MEMBERS SCIENCES HORIZON
PANEL MEMBERS
Alan Blair Bernie Hannigan (Co Chair)
Albert Sherrard Albert Sherrard (Co Chair)
Bryan Keating Alan Stitt (QUB)
Clare Passmore Bert Rima (QUB)
Colin Elliott John Lamont (Randox)
Damien McDonnell (Chair) Michael Neely (HSC R&D Office)
Ed Vernon David Brownlee (HSC Innovations)
Frank Bryan Neville McClenaghan (UU & Diabetica Ltd)
Gerry McCormac Peter Donnelly (BioBusiness NI)
Bernie Hannigan Stephen Barr (Almac)
Jim McLaughlin Tony Bjourson (UU)
Werner Dubitzky (UU)

3. PAGE 03
PROSPERITY AND HEALTH
DELIVERED BY SCIENCE
NORTHERN IRELAND’S LIFE & HEALTH The whole sector employs around 4,000 Over the past 18 months our intensive work
SCIENCES COMMUNITY IS READY people, many in genuinely sustainable, with colleagues from academia, industry and
TO MEET THE SECTOR’S GLOBAL high-value jobs. Around 30 companies in the healthcare has led to many insights and a
CHALLENGES. region have a clear focus on research and real understanding of how best to move this
development (R&D) - some very intensively sector forward. For us, this was a pleasure
The Life & Health Sciences Horizon Panel so - and in 2005 they spent some £33 and a privilege.
was formed in 2007 to recommend actions million on the area, directly employing 600
that will accelerate the development of R&D staff in the process. It is no secret that We would like to thank all of those who
our vibrant life and health sciences sector, in recent years major opportunities have participated in the compilation of this report
boosting economic benefits for the region, resulted from rising investment in R&D. and to acknowledge the work of Phil Towers
and improving the quality of health and and Diana Iacob of PricewaterhouseCoopers
wellbeing products and services available. Given our region’s small size in the who assisted at all stages in the work of the
context of this vast global industry, we Life & Health Sciences Horizon Panel.
With the trend towards individuals taking accept that we cannot excel at everything.
increased responsibility for their own health However, this report puts forward strategic
and vitality increasing amid the spectre of recommendations that identify the key R&D
new or re-emerging infectious diseases and capabilities and future market opportunities
the need for new treatments, this sector can to exploit for maximum economic return for
be hugely important. the decades ahead.
Our work highlights the strength and depth By implementing these recommendations we
of the sector and advances made to date. can ensure not just our continued success
Comprising around 60 companies with a but look forward to the time when Northern
combined turnover of some £400 million, Ireland will feature in the world rankings of
the industry is growing. These businesses significant participants in the huge global
create wealth through products as diverse healthcare and pharmaceuticals market. This
as medical devices and diagnostics, would provide great economic benefits and
pharmaceuticals, bio-pharmaceuticals and position us to meet the challenges that our Bernie Hannigan & Albert Sherrard
medical disposals, as well as biotechnology- healthcare systems are facing. Joint Chairs - Life & Health Sciences
based services and clinical trials. Horizon Panel

4. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
LIFE & HEALTH SCIENCES
HORIZON PANEL REPORT

5. PAGE 05
EXECUTIVE SUMMARY
THE ECONOMIC IMPORTANCE
OF THE HEALTH TECHNOLOGIES
& LIFE SCIENCES SECTOR
Importance to the Northern Ireland (that includes NIBEC and the Centre emergence of new diseases due to
economy: high value-add, export oriented for Advanced Cardiovascular Research), increased global travel and climate
sector Biomedical Sciences Research Institute, Centre changes; growing antibiotic resistance in
for Cancer Research and Cell Biology, the some existing diseases.
Over the past five years, Northern Ireland Northern Ireland Clinical Cancer Centre and the • Focus on prevention and pay-for
industry, academia and government have made McClay Research Centre for Pharmaceutical performance. In a bid to control spiralling
significant commitment to the development of Sciences. costs, a growing number of governments,
life sciences capabilities. According to Invest in both developed and developing countries,
NI data, in excess of $160 million has been In addition to its intrinsic clinical practice are trying to shift the focus from the
budgeted for infrastructure enhancement, expertise, the clinical sector is placing an treatment of disease to its prevention. At
collaborative research, new product increasing focus on clinical research and the same time, industry will be expected to
development and staff development initiatives. development (also known as translational prove to healthcare payers that its products
This illustrates the local stakeholders’ medicine) and commercialisation of innovation really work and provide value for money.
determination to maximise the opportunities arising from its activities. Its strengths lie in the • Increasing role of Information
deriving from a long history of technological seven Recognised Research Groups and the Technology (IT). This is anticipated to
innovation, backed by a highly educated, young Northern Ireland Clinical Research Network become a key enabler to more effective
workforce and an internationally recognised that includes HSC Innovations. storage and retrieval of patient records,
research base. improved tracking of medical research and
A growing global market therapy outcomes, and better remote care.
The business sector counts approximately 60 At global level, the sector is set to benefit from • Pharma industry crisis. The
companies, offering a very diverse range of rising demand for effective medicines as the pharmaceutical industry is undergoing a
products, services and capabilities. Data from population ages, new medical needs emerge period of change as it seeks to increase the
Invest NI and DETI suggests that the industry, and the disease burden of the developing world pace of innovation in face of its collapsing
as a whole, has a combined turnover of around increasingly resembles that of the developed blockbuster model and financial pressure
£310m and employs approximately 4,000 world. By 2020, the global pharmaceuticals from healthcare funders. Biotechnology and
people. This tends to be a high value-add market is projected to be worth some $1.3 gene therapy are anticipated to yield more
sector and export-oriented, with around 80% trillion. A number of key trends are anticipated new products than the traditional molecular
of sales generated from external markets. to shape-up the future of this industry: chemistry-based R&D activity.
• Globalised market place. Collaborative
The academic sector offers a strong science • Changes in disease patterns put product development across boundaries
base, both in life sciences and related sectors, more pressure on healthcare costs. and companies is anticipated to become
through its two world class Universities and Increase in chronic diseases as a result more pervasive. India and China are fast
the six regional Colleges of Further & Higher of ageing population, rise of obesity and becoming preferred centres of product
Education. Examples are the Nanotechnology previously terminal diseases becoming development and manufacturing activity.
and Advanced Materials Research Institute chronic through therapy development;

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WHAT IS
PERSONALISED MEDICINE?
At its most basic, Personalised Medicine refers - to develop new treatments and to identify the
to the use of information about a person’s sub-groups of patients for whom they will work
genetic makeup to tailor strategies for the best. It can also help determine which groups
detection, treatment, or prevention of disease.* of patients are more prone to developing some
diseases and, ideally, help with the selection of
People vary from one another in many ways - lifestyle changes and/or treatments that can
what they eat, the types and amount of stress delay onset of a disease or reduce its impact.
they experience, exposure to environmental Personalised medicine is expected to transform
factors, and their DNA. Many of these healthcare over the next several decades. New
variations play a role in health and disease. The diagnostic and prognostic tools will increase
combination of these variations across several ability to predict the likely outcomes of drug
genes can affect each individual’s risk of therapy, while the expanded use of biomarkers -
developing a disease or reacting to something biological molecules that indicate a particular
in the environment, and can be one of the disease state - could result in more focused
reasons why a drug works for one patient and and targeted drug development. Personalised
not another. medicine also offers the possibility of improved
health outcomes and has the potential to make
Personalised medicine aims to use these healthcare more cost-effective.
variations - both in the patient and in the
molecular underpinnings of the disease itself
THE PARADIGM OF PERSONALISED MEDICINE
RISK PREVENTION TARGETED
ASSESSMENT MONITORING
DIAGNOSIS THERAPY RESPONSE
MONITORING
EARLY DETECTION TESTING
Source: Personalised Medicine Coalition

7. PAGE 07
BENEFITS OF
PERSONALISED MEDICINE
A PERSONALISED APPROACH TO MEDICINE OFFERS
SIGNIFICANT BENEFITS FOR EACH OF THE MAJOR
STAKEHOLDER GROUPS - PATIENTS, GOVERNMENTS, AS WELL
AS INDUSTRY. THESE ARE SUMMARISED BELOW.
GOVERNMENT/HEALTHCARE SYSTEMS PATIENTS INDUSTRY
Detect disease at an earlier stage, when it is Effective and specific therapies Improve the selection of targets for drug
easier and more economic to treat effectively discovery
Less risk of adverse effects
Rational therapeutic decisions based on Reduce the time, cost and failure rate of
pathomechanism of disease rather than on Less time lost compared to trial-and-error clinical trials
trial-and-error approach approach to treatment
Monopoly in a specified segment of the
Incorporate diagnostic guidance to treatment Lower cost of treatment (in the longer term) market - early entry and longer dominance of
market niche; no competition from generics
Reduced adverse drug reactions and Facilitates preventive medicine
complications of treatment Increased drug effectiveness will command
Improvement of quality of life higher prices
Shift the emphasis in medicine from reaction
to prevention Increased revenues from combination of
diagnostics packaged with therapeutic
Reduce the overall cost of healthcare products
Increased professional satisfaction Increase patient compliance with therapy
prescribed
Revive drugs that failed clinical trials or were
withdrawn from the market
Expanding demand for enabling products
and technologies, such as ICT, Agri-food,
nanotechnology.

8. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
KEY DRIVERS AND
CHALLENGES TO DEVELOPMENT
OF PERSONALISED MEDICINE
PROGRESS OF SCIENTIFIC CAPABILITY ADOPTION IN HEALTHCARE SYSTEMS ADOPTION BY INDUSTRY
Scientific progress - whole genome Shift to prevention and early intervention in a The ‘blockbuster’ model of one drug fits all
KEY DRIVERS
sequencing, rapid gene characterisation, bid to manage spiralling costs. is under pressure; improved effectiveness
molecular diagnostics. levels are required for better defined patient
Current drugs are not effective for all patients. populations.
Declining cost of sequencing the human
genome. Bid to reduce costs associated with Adverse The need for enhanced pace of innovation, as
Drug Reactions (ADRs). current R&D methods are yielding fewer new
Advances in information technology and products.
management of health information. The 2004 General Practitioner contract links
remuneration with clinical outcomes. Financial pressure is mounting, as
governments press for lower prices.
Mapping of the genome is still in its infancy. Payment/Reimbursement policies are not Perceived fragmentation of drug markets.
KEY CHALLENGES
tailored to such a system. Personalised
Current availability of bio-banks may restrict medicine is more expensive and more clinical Intellectual property ownership difficult to
the pace of research. data is needed on associated health outcomes secure, particularly if derived from collaborative
and costs benefits. projects.
There are non-genomic factors involved in the
development of personalised medicine, which Education and resources implications Scale of adoption is uncertain - a niche rather
require additional scientific research. - including changes to medical curricula, GP than all-encompassing adoption may be
practices, and other healthcare providers. envisaged, at least in the shorter term.
Policy framework - ethical and privacy concerns
regarding gathering, using and storing genetic
information need addressed.

9. PAGE 09
COMMERCIAL PROSPECTS FOR
PERSONALISED MEDICINE
The market for personalised medicine is • Molecular diagnostics as a stand-alone ineffective and disruptive for patients. Strong
multifaceted, with overlaps between components market; demand is also anticipated from other fields,
and disciplines, and interdependencies with • Remarket of existing drugs (generic and such as central nervous system disorders,
associated sectors - e.g. pharmaceutical, patented); cardiovascular disease and inflammatory disease.
advanced materials, information technology • Development of new biologic drugs; and
etc. The diagram below captures the main • Development of new therapies, such as Assuming a scenario where Personalised
constituents of this complex sector. cell therapy, gene therapy, monoclonal Medicine will grow to account for a quarter of
antibodies etc. total pharmaceutical market value, this indicates
Personalised Medicine has the potential a market potential in excess of $200bn.
to transform healthcare over the next Companies may specialise in certain disease
several decades. areas or provide technologies that cut across However, the evolution of the market
a number of disease groups. Cancer is the is expected to follow a gradual trend. A
The commercial opportunity for companies area generating the greatest interest for personalised approach to medicine has far-
involved in Personalised Medicine can be pharmacogenomic therapies, as it is a very reaching implications, and a re-examination of
viewed as coming from a number of key areas: complex and heterogeneous disease which current approaches to a wide range of industry
requires better classification, and because the practices and policies will be required to allow
therapies currently available are, by and large, its promises to be fully realised.
Markets & Technologies Underlying Disciplines
Molecular Diagnostics Personalised Therapies Disease Areas Genomics
DNA Sequencing Pharmaceuticals Cancer Pharmacogenetics
Gene expression profiling Recombinant human proteins Diabetes Pharmacogenomics
SNP Genotyping Therapeutic monoclonal antibodies Obesity Pharmacoproteomics
Biochips and microarrays Gene and cell therapy etc. Cardiovascular Pharmacometabonomics
Biomarkers Central nervous system etc.
Molecular imaging etc. Systems biology
Bioinformatics
Nanotechnology
THE PARADIGM OF PERSONALISED MEDICINE
RISK PREVENTION TARGETED
ASSESSMENT MONITORING
DIAGNOSIS THERAPY RESPONSE
MONITORING
EARLY DETECTION TESTING

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NORTHERN IRELAND
CURRENT CAPABILITY IN
PERSONALISED MEDICINE
A NUMBER OF KEY BUILDING BLOCKS IN THE DEVELOPMENT OF
PERSONALISED MEDICINE ALREADY EXIST IN NORTHERN IRELAND,
ACROSS THE ACADEMIC, PRIVATE BUSINESS AND CLINICAL (ABC)
SECTORS. OVER THE PAST FEW YEARS, NORTHERN IRELAND
INDUSTRY, ACADEMIA AND GOVERNMENT HAVE MADE SIGNIFICANT
COMMITMENT TO THE DEVELOPMENT OF LIFE SCIENCES CAPABILITIES.
The Northern Ireland business sector has a The health service is placing an increasing Successful development of Personalised
number of leading players, providing highly focus on clinical research and development, Medicine in Northern Ireland is dependent on
innovative products and services. The sector and commercialisation of innovation arising the sector’s ability to draw on and develop
has witnessed a period of growth since 2000, from the clinical sector. It has enormous local capability in complementary sectors
with a number of new companies having been potential to underpin the development of and technologies, particularly ICT (e.g.
set-up to exploit the opportunities offered Personalised Medicine in Northern Ireland, bioinformatics) and Advanced Materials (e.g.
by this exciting sector; they consist of both for example through its seven recognised nanostructures). In turn, developments in life
university spin-outs and pure industry ventures. Research Groups, the Northern Ireland Clinical sciences can be a source of innovation and
Research Support Centre and the Northern development for the Agri-food sector (e.g.
The academic sector offers a strong science Ireland Cancer Research Centre. functional foods, personalised diets etc)
base, both in life sciences and related sectors
through its two world class Universities and the The diagram opposite suggests that in areas
six Colleges of Further and Higher Education. such as clinical trials and biotechnology
Examples are the Nanotechnology and the commercial sector is more advanced in
Advanced Materials Research Institute (that successfully exploiting the local scientific
includes NIBEC and the Centre for Advanced capability. However, a focused effort is
Cardiovascular Research) and Biomedical required to more fully realise the potential
Sciences Research Institute at the University offered by the scientific strengths in areas
of Ulster and the Centre for Cancer Research such as systems biology and diagnostics.
and Cell Biology, the Northern Ireland Clinical This could be achieved through collaborative
Cancer Centre and the McClay Research programmes for knowledge and skill transfer,
Centre for Pharmaceutical Sciences at technology licenses, or creation of well
Queen’s University Belfast. supported spin-off companies.
Source: Technology Capabilities Study for Northern Ireland, MATRIX, 2007

11. PAGE 11
FIGURE 11: LIFESCIENCES CAPABILITY IN NORTHERN IRELAND
Identify how to improve capability Support and Encourage
Clinical Potential impact on NI economy
trials
Biotechnology Small
Strong Medical
disposals
Medium
Agribiology
Exploitation Capability
Biotechnology Major
services
Pharmaceuticals
Medical devices
diagnostics
Timescale to realise impact
Systems Current (<2 years)
biology
Weak 2-5 years
5-10 years
10 years
Consider strategically how to support Build exploitation pathway
Weak Scientific Capability Strong

12. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
REALISING THE
PERSONALISED MEDICINE OPPORTUNITY
FOR NORTHERN IRELAND
As the market intelligence indicates, in the and raising its position in the international
longer term this sector has the potential to arena. The sector would also be able to
transform the way medicine is delivered. Many communicate with a single voice and engage
nations have identified the opportunities offered in a coherent manner with the local support
by this high risk - high reward sector, and mechanisms and stakeholders, for example
investment in research and commercialisation to promote skills development and raise the
in this space has intensified in recent years. profile of the local cluster.
The Life and Health Sciences Horizon Panel
has identified an opportunity for Northern
Ireland to carve out a share of this growing
market, by becoming a centre for Integrated
Research & Development in Personalised
Medicine. This would fuel the development
of Personalised Medicine in Northern Ireland, Northern Ireland to become a centre for Integrated Research & Development
and place the local cluster on the international in Personalised Medicine to:
map in this highly dynamic sector. The Deliver services to the PM industry;
competitive advantage would be conferred by Commercialise innovations & form spin-outs
the integrated nature of the services provided, Achieve global recognition for excellence in a number of strands
harnessing academic scientific expertise, Act as Northern Ireland ‘shopwindow’ to the international community
clinical data and practice and a focus on
commercialisable outputs.
The Panel believes that the sector could be
stimulated locally by creating the environment Local Applied
for the initiation of commercially-targeted ABC Players Research
R&D projects, focussing funding and support
towards applied research not elsewhere funded
and the gap between initial Proof of Concept
and Phase II Clinical trials. Being open to the
full spectrum of the Personalised Medicine Leading Development
technologies and practices, is expected to Pharma & Biotech
stimulate interest from across the sector and Companies
allow the most economically valuable proposals
to emerge.
It is believed that, given the recent Medical Applied Research
developments in local infrastructure, a ‘virtual’ Charities & Development
support mechanism would be sufficient,
providing leadership, funding and expert
support rather than new physical infrastructure.
A highly focused approach to the development Enabled by Northern Ireland Scientific & Clinical Capability
of this exciting sector would help drive closer
cooperation and capability transfer across
the ABC boundaries, thus enhancing the
exploitation capability within the local sector

13. PAGE 13
KEY STAKEHOLDER
INPUTS & OUTPUTS
THE IMPLICATIONS IN TERMS OF COMMITMENT, RESOURCES AND
BENEFITS FOR EACH OF THE LOCAL STAKEHOLDER GROUPS IN
REALISING THE PERSONALISED MEDICINE MARKET OPPORTUNITY
CAN BE SUMMARISED AS FOLLOWS:
GOVERNMENT ACADEMIC STAKEHOLDERS BUSINESS SECTOR CLINICAL SECTOR
Inputs Inputs Inputs Inputs
• Creating an innovation- and • Academic research staff • Business expertise • A commitment towards
business-friendly environment • Enhanced PhD pool • Research expertise & facilities adoption within the local
• Help raise the profile of the • Research facilities • Financial investment clinical practice
local capability • IT capability • Clinical research expertise
• Attract lead scientists from • International research partners • Clinical data
abroad and international • Research facilities
research partners
Outputs Outputs Outputs Outputs
• Development of existing • IP creation, which can be • Extended new product • IP creation, which can be
businesses, by accessing commercialised through pipeline commercialised through
expertise from the local and spin-out companies or • Reduced R&D costs spin-out companies or
international scientific licensing deals • IP creation, which can be licensing deals
community • Spin-out companies commercialised through • Improved patient care,
• Spin-out companies, • Skills development & export spin-out companies or through rational therapeutic
generating associated tax • Enhanced international profile licensing deals decisions rather than trial-
revenues and employment • Attract and retain talent and-error approach
• FDI traction • Industry cross-fertilisation • Cost savings in the long-term,
• Emergence and recycling of (e.g. ICT, Advanced Materials, through increasing emphasis
local entrepreneurs Agri-food) on prevention and early
• International reputation, which intervention
in turn generates new
business.

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WHAT IS HOME-BASED CARE?
THE CORE PROPOSAL FROM THE HORIZON PANEL
CONCERNS THE LINK BETWEEN PATIENTS IN THEIR HOMES
AND THE HEALTH SYSTEM. THIS LINK CAN BE CONSIDERED
TO INCLUDE THE FOLLOWING THREE MARKET COMPONENTS.
1 Telehealth 2 Telecare 3 Secure web messaging and e-visits
Telehealth monitoring is the remote exchange Telecare is the continuous, automatic and This technology usually acts as an enabler
of physiological data between a patient at remote monitoring of real time lifestyle changes for the previous two areas of remote care.
home and medical staff at hospital to assist in and emergencies over time, in order to manage Consumer familiarity with the Internet and
diagnosis, progress monitoring and prevention the risks associated with independent living. e-mail allows for more efficient communication
of various conditions. Specific product Telecare is usually designed to create home with medical staff and for novel healthcare
examples include home-based blood pressure environments which meet the needs of older solutions. A specific example of an e-visit could
monitors that relay information to the patients or disabled people. Specific product examples be an email exchange between a doctor and
GP surgery. Their immediate applications tend include fall or bed sensors. patient. The market also includes technologies
to lie in the management of chronic disease and services based exclusively within the health
(e.g. diabetes, cardiovascular etc). The system (telemedicine) or within the patients’
preventative and personal health management physical home infrastructure (assistive devices).
aspects are expected to become more These do not form part of the Panel’s core
pervasive in the longer term. proposals, but instead are recommended for
consideration in the longer term.

15. PAGE 15
KEY MARKET DRIVERS AND
CHALLENGES TO DEVELOPMENT
KEY MARKET DRIVERS KEY CHALLENGES TO DEVELOPMENT
• Current healthcare systems are unsustainable, in great part due to • Slow adoption by healthcare systems, due to factors such as
an ageing, more chronically-ill population resistance to adoption of information technology and new work
• There have been numerous pilot studies that have demonstrated practices, and difficulties in changing reimbursement systems within
social and financial benefits associated with the home care concept the GP community; significant policy issues therefore remain to be
• Advancement of technologies and IT infrastructures has led to addressed
higher IT spend within healthcare budgets, in a bid to achieve • Integration into existing structures is made more difficult by the need
efficiencies and improve service delivery for a joined-up approach across health, housing and social care
• Rising expectations of consumers with regards to own health status • Maintaining individual privacy - there are concerns regarding
and involvement in their healthcare decisions surveillance and possible loss of privacy and autonomy, and legal
• Adoption of Electronic Health Records (EHR) will act as an enabler issues relating to data confidentiality and protection.
for remote care • Lack of coherent approach - both within health systems (so far
based on regional, small scale pilots) and industry (interoperability
issues among devices and telecommunications providers)
• Level of technology development - there is a need for further
development for example to provide more consumer-friendly devices
• Country variations in policies and structures make exporting more
difficult

16. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
COMMERCIAL PROSPECTS FOR THE
HOME-BASED CARE MARKET
THE CORE PROPOSAL FROM THE HORIZON PANEL CONCERNS
THE LINK BETWEEN PATIENTS IN THEIR HOMES AND THE
HEALTH SYSTEM.
The US is, and will remain, easily the largest In comparison, the entire European market
national market for telehealth, reaching $4.51 is expected to only reach about $1.00 billion
billion in 2010 according to Datamonitor by 2010, despite having a projected 502
forecasts. It will also show faster growth than million people.
Canada at 64% CAGR, compared with 32%
for the latter. This very high growth and large The European market may be smaller than
market share are the result of two that in the North America, but it will still show
main features of the US market: very rapid growth, with an expected CAGR of
• Very high overall US healthcare spending, 60% over the period. The UK is the largest
given the private care and insurance- single market at $23 million and will grow by
based system; 66% CAGR to $286 million in 2010. The ten
• High and rapidly growing rates of chronic new EU nations (NEU 10) will be the fastest
diseases such as diabetes. growing market, with a CAGR of 69%, but will
still only reach a value of $60 million in 2010.

17. PAGE 17
TELEHEALTH MARKET IN NORTH AMERICA BY COUNTRY (2005-2010)*
6,000 100% Canada
United States
5,000
Growth
Percentage Growth
4,000 60%
Million US $
3,000
2,000 30%
1,000
0 0%
2005
2006
2007
2008
2009
2010
TELEHEALTH MARKET IN EUROPE BY COUNTRY (2005-2010)**
1,200 80% Switzerland
70% NEU 10
1,000
Spain
60%
Percentage Growth
800 Rome
50%
Million US $
Italy
600 40%
Benelux
30%
400 Nordics
20%
France
200
10% Germany
0 0% United Kingdom
2005
2006
2007
2008
2009
2010
Growth
* Source: From report
** Source: Datamonitor, Extending the delivery of healthcare beyond the hospital setting, June 2006

18. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
REALISING THE HOME-BASED CARE
OPPORTUNITY FOR NORTHERN IRELAND
The Life and Health Sciences Horizon Panel The overall aim of the model proposed for
recommends that Northern Ireland becomes the Northern Ireland is to join up local capability
first UK region committed to the early adoption across all four of these domains in an
of a telehealth system within the Health and integrated manner and in a real clinical setting.
Social Care practice. This means establishing
a strong local capability across the telehealth The Panel concluded that to maximise the
continuum, and deploying this locally to achieve market opportunity in this area, telehealth
whole connectivity between the health sector technology should be implemented within our
and the home within 15-20 years. own health system.
The Home-based care market encompasses • This would allow the DHSSPS to benefit
a wide range of products and services. The from savings achieved from the delivery
underpinning technologies and systems of health services in this manner, whilst
necessary to deliver those services can be improving patient care; and
sub-divided into four key domains along the • At the same time, it would create an
telehealth continuum, as summarised in the attractive Whole System Integration Test
diagram below. Bed for local and international technology
providers, who could use Northern Ireland
as a gateway to UK and European home
care export markets.
NORTHERN IRELAND POSITION ALONG THE HOMECARE CONTINUUM: WHOLE SYSTEM CONNECTIVITY
Medical Devices Home Telecomms Data Analysis Response System
Assistive devices Broadband Software solutions Family/carer
Sensors ICT Integration Data management NHS - GP, PCT, ACT,
Alert systems Physical structure Social Services
Vital signs monitoring devices Third party service provider

19. PAGE 19
The Panel considered the Northern Ireland Data Analysis Government support
capability’s strengths and gaps against the four • The development of a Home-based care • The announcement made in January
key areas judged to constitute the home based market will require a capability in managing 2008, regarding the development of an
care continuum, and the level of support and and analysing large volumes of data. The European Centre for Connected Health
commitment provided by the local government. Panel judged this was not an area were supported by three Northern Ireland
a significant capability existed locally at government departments, is a powerful
Medical Devices present but from preliminary discussions springboard for future development of
• The panel noted that a local capability with the Horizon ICT Panel it was an area the concept. The project has significant
existed within Northern Ireland within the that could be further explored for future buy-in from the private sector locally and
medical device sector. However, it was investment. internationally.
also noted that these companies lacked
a scale within the market. All of the Response System
companies had to seek an export market • The specific circumstances of each patient
as there was currently little potential for group and the economic case for each
growth domestically. initiative will determine the response
• It was acknowledged that a strategy could system employed. The options to consider
be developed to build upon the existing would include:
capability by seeking additional foreign - Response to be delivered within the
direct investment into the province and by DHSSPS; if so, what are the interface
developing partnership agreements. points, e.g. GPs, Social services,
others;
Home-based Telecommunications - Response to be provided by a family
• The development of a Home-based member;
care market will require a high speed - Response to be delivered by a 3rd
telecommunications infrastructure. The party provider; if so, what
panel members judged that Northern type of activities specifically and
Ireland was already well served in this associated governance; or
area compared to other regions in the - Combination of service delivery;
UK given the existing 100% broadband • The Panel considered that the most
coverage. However, the ability to generate important success factor was not the
a economic capability by developing creation of infrastructure. Instead, it
companies to compete within the was ensuring that the existing response
technology infrastructure sector was not resources (e.g. GP surgeries, District
judged to be strong given the dominance nurses etc) were motivated and
of the existing technology competitors. incentivised to adopt this way of working.

20. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
KEY STAKEHOLDER
INPUTS & OUTPUTS
THE TABLE BELOW SUMMARISES THE COMMITMENT NECESSARY FROM
GOVERNMENT, ACADEMIA, BUSINESS AND THE CLINICAL FIELD TO
SUCCESSFULLY DELIVER HOME-BASED CARE WITHIN THE NORTHERN
IRELAND ECONOMY. THE TABLE ALSO HIGHLIGHTS THE POTENTIAL RETURN
FOR EACH RESPECTIVE GROUP.
GOVERNMENT ACADEMIC SECTOR BUSINESS SECTOR CLINICAL SECTOR
Inputs Inputs Inputs Inputs
• A commitment to a • Social studies • An investment in new • A commitment to changing
‘connected health’ agenda • ICT capability technologies working practices
and establishment of NI as a • Curricula for new disciplines • A long-term contractual • Adjusting reward structures
test-bed for associated in eHealth commitment to the
technology public sector
• A commitment to a joined
up approach to the market
opportunity
• Embrace an attitude of
change
Outputs Outputs Outputs Outputs
• Stimulate indigenous • Graduates to fulfil a new • An opportunity to participate • A new model of healthcare
companies model of health delivery in a locally significant market management
• Create a knowledge base • Growing reputation in the • Potential to export proven • Improved health and quality of
from which export potential Connected Health field technology and know-how to life for relevant patient groups
can be exploited US and European markets through enhanced
• Attract FDI companies, independent living
interested in using NI as a • Improved work practices for
gateway to UK and European health professionals
markets • Realisation of cost savings
• Demonstration of the which can be redistributed
government’s commitment to towards other priority areas,
smarter working practices to thus increasing productivity
improve patient care levels

21. PAGE 21
FRAMEWORK
CONDITIONS
The health technologies and life sciences Personalised medicine specific factors:
sector has the potential to become a major • Participation from the clinical sector
contributor to Northern Ireland’s knowledge- would be critical to help demonstrate
based economy. There are however a number of the economics and clinical benefits of
prerequisites to successfully realising the market pharmacogenetics in clinical practice;
opportunities identified by the Horizon Panel. • Application in existing vs. new drugs would
imply forming public-private partnerships to
General enabling factors: enable enhancements of existing generic
• Most importantly, accomplishing the drugs; and
healthcare opportunities identified by • Addressing the ethical framework -
the Life & Health Sciences Panel is safeguards need to be put in place to
dependent on the sector’s ability to prevent the misuse of genetic information.
exploit and develop local capability in a
number of allied areas. This is an era of Home-based care specific factors:
convergence of technologies, and Life & • Scalable approach to roll-out - it is
Health Sciences sits right at the interplay recommended that DHSSPS retains the
of technologies from related sectors control over the patient groups and the
- Advanced Materials, ICT, Advanced parts of the healthcare system impacted at
Engineering etc; each stage;
• The competitive position of Northern • Defining technology standards and
Ireland is dependent on the ability to protocols - common technology standards
provide an integrated service and product and protocols need developed to provide
portfolio. This means a joined-up approach interoperability of devices;
among the private sector companies, • Stakeholder engagement - existing
but also the use of academic and clinical response resources (e.g. GP surgeries,
capability as a catalyst to innovation and District nurses etc) need motivated and
science. Collaboration with international incentivised to adopt this way of working;
partners, including RoI and GB, would also and
help address some of the gaps that may • Adjusting funding policy - aim to use and
exist adjust the existing reward and operational
in the local capability; structures (e.g. GP contracts) as much
• The availability of an adequate skills base as possible, rather than creating a new,
is of paramount importance. The right parallel system.
quality and volume of skills is necessary
to fuel the growth of the sector. The
education system has been engaging a
lot more with the industry in the last few
years, but even more can be done to align
the curriculum to the changing needs of
the industry; and
• Supportive policy environment - this would
involve the creation of an innovation- and
business- friendly environment, which
would support indigenous existing and new
companies, as well as overseas companies
considering operating in Northern Ireland.

22. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
SUMMARY OF
PROPOSALS
TWO DISTINCT MARKET OPPORTUNITIES HAVE BEEN IDENTIFIED BY THE HEALTH AND LIFE SCIENCES
HORIZON PANEL: PERSONALISED MEDICINE AND HOME-BASED CARE. WHILST THEY HAVE A NUMBER
OF COMMON FEATURES, IN THAT BOTH ADDRESS ISSUES FACING THE GLOBAL HEALTH AND ECONOMY
IN THE LONG-TERM, AND THEY ULTIMATELY COMPLEMENT EACH OTHER WITHIN THE INTEGRATED
HEALTHCARE CONCEPT, THE TWO MODELS OFFER DIFFERENT PROPOSITIONS IN TERMS OF TYPE OF
CAPABILITY IMPACTED LOCALLY, ECONOMIC IMPACT, TIMESCALE AND RISK FACTORS. THE TWO MARKET
PROPOSALS ARE SUMMARISED BELOW.
PERSONALISED MEDICINE HOME-BASED CARE
Market rationale Market rationale
• Large, emerging market across all segments, driven by need for • Developing market, driven by the need for more efficient healthcare
more effective treatments & shift to preventative medicine, and the provision and increasing consumer demand for enhanced quality of
innovation & patent crisis in the pharmaceuticals sector life
• Obstacles: costs, data availability, ethical concerns, reimbursement • Market currently dominated by pilots. Proven benefits, but also show
policy that ‘one size doesn’t fit all’
• Number of players & government investment increasing globally • Main technology platforms exist, albeit requiring further development
• High risk, high reward market • Obstacles to adoption: Device & IT Interoperability, Variation in
• NI has some of the building blocks in place, but lacking critical mass regional policy, Healthcare system resistance to technology &
change, Reimbursement policy
• Northern Ireland capability lagging behind
Northern Ireland Opportunity Northern Ireland Opportunity
• Focus on clear commercialisable applications in the Personalised • Focus on telehealth and telecare segments (excludes telemedicine
Medicine space and house aids)
• Open/Bottom-up approach - based on competitive projects • Adoption within mainstream healthcare system to become Northern
• Create a virtual mechanism, using existing physical infrastructure & Ireland’s competitive differentiator
tech transfer structures • Top-down approach, with decision on policy and strategic approach
• Support collaborative networks, resource development, profile to roll-out resting exclusively with DHSSPS.
raising • Phased roll-out
• Use funding to incentivise collaboration, technology transfer and
attract international players

23. PAGE 23
PERSONALISED MEDICINE HOME-BASED CARE
Advantages Advantages
• Potential for immediate engagement of existing companies and • High FDI traction prospects
impact on their commercially-targeted R&D activity • Technology platforms available internationally, albeit requiring
• Draws on and stimulates R&D in related and complementary fields improvement
• Additional funding leveraged through public sources (UK, RoI and • Lower risk and more immediate economic impact on both patient
European sources) and major international players care and local economy (3 years +)
• Attract PhD and other highly skilled professionals. Retain indigenous • Significant interest in full adoption from DHSSPS locally, which
talent would be a key competitive advantage compared to other pilot-
• Longer term - creation of new companies based schemes.
Draw-backs Draw-backs
• Long-term economic impact (10 years +) • Limited indigenous scientific capability locally
• High risk portfolio • Global market prospects dependent of adoption rates in each
country
Key success factors Key success factors
• Building critical mass and profile through sustained investment • Adjust reimbursement strategy to incentivise adoption
• Combining academic and clinical capability will confer a strong • Embedding behavioural changes - among healthcare professionals
competitive advantage and patients
• Commitment from all stakeholders

24. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
CONTENTS
1 4
25 INTRODUCTION 69 FRAMEWORK CONDITIONS
26 Economic importance of the sector 71 General Enabling Factors
29 The Horizon Panel objectives 73 Personalised Medicine
30 Approach to delivery 74 Home-based Care
2
32 PERSONALISED MEDICINE - THE
OPPORTUNITY FOR NORTHERN
IRELAND
33 What is Personalised Medicine?
34 Applications of Personalised Medicine
36 Benefits of Personalised Medicine
37 Key market drivers and challenges to
development
41 The Market for Personalised Medicine is
Multifaceted
42 Commercial Prospects
48 Northern Ireland Capability
52 The Opportunity for Northern Ireland
3
55 HOME-BASED CARE MARKET - THE
OPPORTUNITY FOR NORTHERN
IRELAND
56 Defining the market
57 Key market drivers and challenges to
development
59 Driving Policies and Iniatives in the
Sector
60 Commercial prospects
62 The Opportunity for Northern Ireland
64 Realising the Home-Based Care
Opportunity for Northern Ireland
66 Northern Ireland Capability
67 Economic Benefits
68 Key Stakeholder Inputs & Outputs

25. PAGE 25
INTRODUCTION
1

26. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
THE ECONOMIC IMPORTANCE OF
THE HEALTH TECHNOLOGIES & LIFE
SCIENCES SECTOR
NORTHERN IRELAND
CONTEXT
As technologies converge, ‘life sciences’ • The need for stronger links with academia Over the past five years Northern Ireland
represents an all-encompassing title capturing and clinicians in order to bolster technology industry, academia and government have made
a wide range of industry sub-sectors such transfer and innovation significant commitment to the development of
as pharmaceuticals, biotechnology, medical • Shortage of ‘veteran’ entrepreneurs in life sciences capabilities. In excess of $160
devices and diagnostics, drug delivery, clinical Northern Ireland, with the experience and million has been budgeted for infrastructure
trials etc. The sector in Northern Ireland time to mentor life science businesses enhancement, collaborative research, new
comprises organisations across the Academic, • Need to remain at the forefront of R&D, product development and staff development
Business and Clinical (ABC) sectors. in light of increasing competition from low initiatives.
cost manufacturing regions
The business sector counts approximately 60 In the same timeframe, Northern Ireland
companies, offering a very diverse range of The academic sector offers a strong science life science companies have committed to
products, services and capabilities. Data from base, both in life sciences and related sectors strategic investment expenditure in the order
Invest NI and DETI suggests that the industry through its two world class Universities and of $340 million including approved government
as a whole has a combined turnover of around the six regional Colleges of Further & Higher assistance of $70 million. In a country with a
£310 million and employs approximately 4,000 Education. Examples are the Nanotechnology population of just 1.7 million people, employing
people. This tends to be a high value-add and Advanced Materials Research Institute (that around 4,000 in life sciences, this level of
sector and export-oriented, with around 80% includes NIBEC and the Centre for Advanced investment (almost $125,000 per employee) is
of sales generated from external markets. Cardiovascular Research), Biomedical Sciences impressive.
Research Institute, Centre for Cancer Research
The top five companies by size account for and Cell Biology, the Northern Ireland Clinical This illustrates the local stakeholders’
some two thirds of the sector’s turnover Cancer Centre and the McClay Research Centre determination to maximise the opportunities
and 3,838 of its employees. The majority for Pharmaceutical Sciences. deriving from a long history of technological
of companies in the sector are small, with innovation, backed by a highly educated, young
turnovers of under £500,000. In addition to its intrinsic clinical practise workforce and an internationally recognised
expertise, the clinical sector is placing an research base.
The issues facing local industry include: increasing focus on clinical research and
• Difficulty of gaining access to early stage development and commercialisation of
funding, due to the higher risk and longer innovation arising from the clinical sector. Its
lead times to commercialisation that apply strengths lie in the seven recognised Research
to this sector Groups, the Northern Ireland Clinical Research
• Skill shortages in certain areas Support Centre and multiple joint programmes
• Long and costly product development, with equivalent organisations in Ireland and the
due to compliance with a strict regulatory UK as well as the local academic and business
regime sectors.

27. PAGE 27
GLOBAL
CONTEXT
A growing market Growing antibiotic resistance However, the human genome has proved more
At global level, the sector is set to benefit from in existing diseases. complex and less amenable to mechanistic
rising demand for effective medicines as the The overuse of antibiotics, and the limited analysis than many scientists anticipated,
population ages, new medical needs emerge number of new ones in the pipeline, has when the draft map was completed in 2001.
and the disease burden of the developing world left little defence against several previously Hence the fact that Pharma is still struggling
increasingly resembles that of the developed contained pathogens. Examples include to apply the insights it has gleaned from the
world. By 2020, the global pharmaceuticals hospital infections and tuberculosis. molecular sciences - genomics, proteomics,
market is projected to be worth some $1.3 metabonomics and the like - to improve
trillion. The market is anticipated to be shaped Industry innovation crisis its performance. The industry requires
up by a number of key trends: Currently, it appears that the global a fresh approach to its R&D processes,
pharmaceutical industry is not in a strong and identification of target molecules and
Changes in disease patterns put position to capitalise on the market compounds.
pressure on healthcare costs opportunities, unless it can change the way in
Increase in chronic diseases. The global which it operates. Its core problem is the slowing Globalising marketplace
population is projected to rise from 6.5 billion pace of innovation in providing effective new Globalisation will bring fundamental changes,
in 2005 to 7.6 billion in 2020. It is also aging therapies for the world’s unmet medical needs. particularly in the area of product development.
rapidly; by 2020, about 719.4m people - 9.4% With an inexpensive supply of highly trained
of the world’s inhabitants - will be 65 or more, The industry’s best hope of earning higher researchers and improving intellectual
compared with 477.4 million (7.3%) two years returns lies in the development of packages of property norms, China and India look likely to
ago. Older people typically consume more products and services targeted at patients with become a focus of product development and
healthcare products and services than younger specific disease subtypes and that, if it was to manufacturing activity in life sciences.
people, as they tend to develop multiple make such “targeted treatments”, it would have
chronic diseases. Obesity, especially in the US, to start by focusing on diseases rather than Changing relationships
is another driver of higher costs, as it tends compounds. Co-operation will be a critical element of
to lead to chronic diseases such as diabetes, success in life sciences. Biotech and big
coronary heart disease and osteoarthritis. Biotechnology research is anticipated to pharma will work together to bring new
deliver more new products than the traditional products to market. Teams will co-operate
More new diseases. molecular R&D activities. Gene therapy is globally on processes such as product
Greater population density, increased travel likely to move from treatment of conditions to development or test analysis. Increasing levels
and climate changes have increased the cures by modifying patient DNA. Previously of patient choice and knowledge will place a
appearance of new pathogens. These are untreatable conditions could therefore find premium on successful relationships between
often viral, initially difficult to treat, and highly a treatment, as is, for example, the case of physicians and those in their care.
dangerous. Examples include ebola, SARS and cancer and other genetic conditions.
potentially avian flu.

28. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
Increasing Role of Information Pay-for-performance
Technology (IT) The provision of healthcare is not all that
More pervasive use of IT will lead to more is changing; so is the way in which it is
effective storage and retrieval of patient measured. Several countries have set up
records, improved tracking of medical agencies specifically to compare the safety and
outcomes and better remote care. Automation efficacy of different forms of intervention and
of simpler processes is also anticipated to promote the use of evidence-based medicine.
free up physicians’ time to focus on higher- The US Agency for Healthcare Research and
value activities such as diagnosis and patient Quality is one such body, as is the UK Centre
communication. for Health Technology Evaluation - a division
of the National Institute for Clinical Health
Blurring Healthcare Boundaries and Effectiveness (NICE). The industry will
Changes in the way healthcare is delivered have to prove to healthcare payers, who are
will arguably play an a critical role in shaping increasingly interested in establishing best
the industry’s future. The primary-care sector medical practice, that its products really work
is expanding and becoming more regimented, and provide value for money.
as general practitioners perform more minor
surgical procedures and healthcare payers Focus on prevention
increasingly mandate the treatment protocols A growing number of governments in both
they must follow, including the drugs they can developed and developing countries are trying
prescribe. Conversely, the secondary-care to shift the focus from the treatment of disease
sector is contracting, as clinical advances to its prevention. For example, at least 18
render previously terminal diseases chronic; countries have already introduced nationwide
healthcare providers like Clinovia in the UK, bans on smoking in enclosed public places, as
and Gentiva in the US, deliver secondary care have a number of US states. Similarly, some
at home; and hospitals focus on the specialist countries are waging war against rising levels
care that cannot be supplied anywhere else. of obesity. The role of genetics in identifying
The self-medication sector is also growing, as those at risk and in early interventions is also
more and more products that would once have expected to play a major role in preventative
been available only on prescription are sold in medicine.
OTC formats.

29. PAGE 29
THE HORIZON
PANEL OBJECTIVES
The Life and Health Sciences Horizon Panel The Life and Health Sciences Horizon Panel was tasked to deliver on the
is one of the five technology horizon scanning following objectives:
panels established under the auspices of the
Northern Ireland Science Industry Panel -
MATRIX. The other four Horizon panels
i Identify the sustainable market opportunities that could be
represent the following sectors: ICT, Advanced exploited distinctively by the NI science and industry base;
Materials, Agri-food and Advanced Engineering
(Transport).
ii Identify the research and technology strengths of the Northern
MATRIX is an expert advisory panel
Ireland science and industry base relevant to the identified
reporting to DETI and the DETI Minister on
matters pertinent to the exploitation and market opportunities;
commercialisation of science, technology and
R&D. It is led by high-technology and R&D iii Determine the opportunities for external collaboration that
intensive industry and advises Northern Ireland
Government on the development of improved
would supplement critical gaps in NI research and technology
interfaces between Northern Ireland business capability in ensuring early delivery of potential outputs;
and the research, science and technology
base, with a view to ensuring the region’s iv Engage with key stakeholders and partners critical to the
science and R&D strengths are exploited
for maximum economic and commercial
success of relevant market opportunities (public, private and
advantage. academic) in Northern Ireland to assist in the prioritisation of
the key technology enabled business opportunities for the
sector;
v Identify internal and external networks (including those
networks wider than the life and health sciences sector)
essential to deliver technology and business growth and
establish processes to build and sustain these networks; and
vi Identify investment opportunities in the sector.

30. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
APPROACH TO DELIVERY
We adopted a step driven approach to 1 Project Mobilisation
achieving the project deliverables, underpinned An initial meeting was held with the Matrix
by monthly Panel working meetings. The secretariat to agree the approach to delivering
12-strong Panel brought together leading and managing the project. This was then
representatives from across the local captured in the Project Initiation Document.
industry - academics (6), clinical sector
R&D representatives (2), and private sector 2 Project Scoping
companies (2 large businesses and 2 SMEs). Given the broad and complex nature of the
The Panel was co-chaired by leaders from health technologies and life sciences sector,
business and academia. it was important to select the most appropriate
Project mobilisation
MONTHLY MEETINGS WITH THE HORIZON LIFE & HEALTH SCIENCES PANEL
Project scoping
Define key areas of focus
Market Opportunities Assessment Market Opportunities Assessment
Personalised Medicine Home-based Care
Consolidation of options
Roadmap to Development
5. Report production

31. PAGE 31
approach to identifying the relevant market The profile of the local sector capability was
opportunities for Northern Ireland. It was also analysed, drawing information from DETI’s
ultimately agreed that the most effective means parallel study on Northern Ireland Technology
to achieving this was through a brainstorming capability, and the BioBusiness and Invest NI
session, which would harness the collective Sector review conducted in 2006.
knowledge of the Panel members and the
market acumen of the PwC core team and In light of the research findings, a number of
expert advisors. The event was held in May suggestions were made on potential areas
2007 and elicited views on ‘Sustainable Market of focus for the Northern Ireland cluster, and
Opportunities for Northern Ireland plc’ from models for implementation.
a wide number of standpoints: World disease
prevalence, Key industry trends, Emerging and 4 Consolidation Of Options
cross-cutting technologies, Policy & regulation, The options on the best approach for
and Northern Ireland competitive position. Northern Ireland approaching this market
and establishing a competitive position were
Over 100 innovative ideas were generated, deliberated with the Panel members. A number
which were then analysed and found to cluster of options to the implementation models were
around 11 principal market-related themes. The discussed, together with the implications for
Panel filtered these further and selected the top the local stakeholders.
priorities through a voting process. The results
were discussed at the Panel meeting in June, The emerging thoughts were shared and tested
when the top two key areas of focus for Northern with a limited number of key sector leaders in
Ireland were agreed as: Personalised Medicine the local economy. These discussions helped
and Home-based Care. gauge their views on how the proposed models
could be effectively deployed, and initiated
3 In-depth Market Analysis early buy-in and engagement across the
In-depth market research was carried out industry community.
within the two selected themes, to explore
specific technology application opportunities 5 Reporting
within each. The research used analysis of The final report explores the recommendations
extensive market intelligence and interviews of the Life and Health Sciences Horizon Panel
with international and local industry experts, in the areas of Personalised Medicine and
to establish: Key areas of market growth, Home-based Care and summarises the market
Emerging technology applications, Key players, rationale for these recommendations.
and Potential collaboration partners.

32. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
PERSONALISED MEDICINE -
THE OPPORTUNITY FOR
NORTHERN IRELAND
2

33. PAGE 33
WHAT IS PERSONALISED
MEDICINE?
At its most basic, Personalised Medicine refers of patients are more prone to developing some specific genetic risk for certain diseases.
to the use of information about a person’s diseases and, ideally, help with the selection of A key factor that will drive the integration of
genetic makeup to tailor strategies for the lifestyle changes and/or treatments that can diagnostics and therapeutics is the availability
detection, treatment, or prevention of disease.* delay onset of a disease or reduce its impact. of improved and more precise diagnostic
methods, which are easy to perform and are
People vary from one another in many ways - Personalised Medicine is expected to transform not prohibitively expensive.
what they eat, the types and amount of stress healthcare over the next several decades. New
they experience, exposure to environmental diagnostic and prognostic tools will increase Although scientific advances such as
factors, and their DNA. Many of these the ability to predict the likely outcomes of drug the mapping of the human genome and
variations play a role in health and disease. The therapy, while the expanded use of biomarkers computation technology are underpinning
combination of these variations across several - biological molecules that indicate a particular fast scientific developments, adoption in the
genes can affect each individual’s risk of disease state - could result in more focused mainstream clinical system lags behind. If
developing a disease or reacting to something and targeted drug development. Personalised Personalised Medicine is to realise its potential,
in the environment, and can be one of the Medicine also offers the possibility of improved it will require an extensive system of support.
reasons why a drug works for one patient and health outcomes and has the potential to make This system will include new regulatory
not another. healthcare more cost-effective. approaches, revamped medical education
curricula, integrated health information
Personalised Medicine aims to use these The illustration arrow reflects the current and systems, legislation to protect against
variations - both in the patient and in the anticipated flow of healthcare services, and genetic discrimination, insurance coverage
molecular underpinnings of the disease itself changing points of intervention, as medicine for sophisticated molecular diagnostic tests,
- to develop new treatments and to identify the becomes more personalised. Early detection and a reimbursement system that encourages
sub-groups of patients for whom they will work testing will play an increasing role, with proactive care.
best. It can also help determine which groups expanded screening programmes for detecting
THE PARADIGM OF PERSONALISED MEDICINE**
RISK PREVENTION TARGETED
ASSESSMENT MONITORING
DIAGNOSIS THERAPY RESPONSE
MONITORING
EARLY DETECTION TESTING
* (Source: Personalised Medicine Coalition)
** (Source: Personalised Medicine Coalition)

34. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
APPLICATIONS OF
PERSONALISED MEDICINE
A PERSONALISED APPROACH TO MEDICINE PROMISES A NUMBER
OF BENEFITS IN ADDRESSING THE KEY CHALLENGES FACING
HEALTHCARE SYSTEMS WORLDWIDE:
Currently, physicians often have to use trial Governments and medical communities are screening programmes and diagnostic tests
and error methods to find the most effective increasingly stressing preventive medicine as at personal and General Practitioner level
medication for each patient. As more is learnt the most cost-effective approach to improving would enable people to be more in charge of
about which molecular variations best predict the quality of life. Developments in molecular their own health and health practitioners to
how a patient will react to a treatment, and diagnostics and genetic testing will drive this recommend life style, nutritional and medical
develop accurate and cost-effective tests, market. The design and validation of preventive action at an early stage.
doctors will have more information to guide
their decision about which medications are
likely to work best. Testing is already being
used to find the one in four women likely to
respond to a particular breast cancer drug. In
addition, testing could help predict the best BETTER
DIAGNOSES
dosing schedule or combination of drugs for a
AND EARLIER
particular patient. INTERVENTION
Molecular analysis could determine precisely
which variant of a disease a person has, or
whether they are susceptible to drug toxicities,
to help guide treatment choices. For preventive
medicine, such analysis could improve the ability
to identify which individuals are predisposed INCREASED
MORE BENEFIT
ADOPTION OF
to develop a particular condition - and guide EFFECTIVE FOR HEALTHCARE
PREVENTATIVE
decisions about interventions that might THERAPIES SYSTEMS
CARE
prevent it, delay its onset or reduce its impact.
Toxicity associated with inappropriate use of
approved drugs is a real problem and a cost to
healthcare systems, that Personalised Medicine
can provide a partial solution to. In the USA
alone, adverse drug reactions (ADRs) are IMPROVED
responsible for approximately 100,000 drug- SAFETY
related deaths and 2.2 million hospitalisations OF DRUGS
per year, representing a cost of roughly
$100 billion.

35. PAGE 35
PERSONALISED MEDICINE ALSO HAS THE POTENTIAL TO HELP
THE PHARMACEUTICAL INDUSTRY TO IMPROVE ITS PERFORMANCE
AROUND BOTH NEW PRODUCT R&D AND EXISTING DRUGS.
Genotyping may be used for rescuing ‘dead and toxicity in some patients. By defining the Repositioning of a drug through
drugs’ that have failed clinical trials because genotypic characteristics, patients who are pharmacogenomics can extend the patent
of lack of efficacy and toxicity problems. non-responders and those liable to adverse life of a drug. A drug with redefined new
New indications can be explored in defined reactions, can be excluded. indications for a particular group of patients
populations groups where the drug use is safe would start a new patent life.
and effective.
Redefining the patient population for marketed
drugs would enhance effectiveness and safety.
Upper figures for efficacy of medical treatment
of most diseases are less than 100%. Example
percentage of patients that show lack of MORE
EFFICIENT DRUG
response to current therapy in key disease
DEVELOPMENT
areas: Epilepsy - 70%, Hypertension - 65%, PROCESS
Depression - 60%, Diabetes mellitus - 55%,
Rheumatoid arthritis - 50%, Alzheimer’s
disease - 30%. Improvement in treatment is
an opportunity for development of Personalised
Medicines.
A better understanding of genetic variations EXPAND
RESCUING BENEFIT
MARKET OF
could help scientists identify new disease ‘DEAD DRUGS’ FOR
EXISTING
subgroups and their associated molecular INDUSTRY
DRUGS
pathways, and design drugs that target them.
Molecular analysis could also help select
patients for inclusion in, or exclusion from,
late stage clinical trials - helping gain approval
for drugs that might otherwise be abandoned
because they appear to be ineffective in the
larger patient population. EXTEND
IMPROVED PATIENT LIFE BY
EFFICACY OF IDENTIFYING NEW
Pharmacogenomics may be applied to expand TREATMENTS APPLICATIONS
markets for approved drugs with generally
restricted use because of limited efficacy

36. PROFITING FROM SCIENCE WWW.MATRIX-NI.ORG
BENEFITS OF
PERSONALISED MEDICINE
A PERSONALISED APPROACH TO MEDICINE THEREFORE OFFERS THE
PROSPECT FOR SIGNIFICANT BENEFITS FOR EACH OF THE MAJOR
STAKEHOLDER GROUPS - PATIENTS, GOVERNMENTS, AND INDUSTRY.
THESE ARE SUMMARISED BELOW.
GOVERNMENT/HEALTHCARE SYSTEMS PATIENTS INDUSTRY
Detect disease at an earlier stage, when it is Effective and specific therapies Improve the selection of targets for drug
easier and more economic to treat effectively discovery
Less risk of adverse effects
Rational therapeutic decisions based on Reduce the time, cost and failure rate of
pathomechanism of disease rather than on Less time lost compared to trial and error clinical trials
trial-and-error approach approach to treatment
Monopoly in a specified segment of the
Incorporate diagnostic guidance to treatment Lower cost of treatment (in the longer term) market - early entry and longer dominance of
market niche; no competition from generics
Reduced adverse drug reactions and Facilitates preventive medicine
complications of treatment Increased drug effectiveness will command
Improvement of quality of life higher prices
Shift the emphasis in medicine from reaction to
prevention Increased revenues from combination of
diagnostics packaged with therapeutic
Reduce the overall cost of healthcare products
Increased professional satisfaction Increase patient compliance with therapy
prescribed
Revive drugs that failed clinical trials or were
withdrawn from the market
Expanding demand for enabling products
and technologies, such as ICT, Agri-food,
nanotechnology.