PROFITING FROM SCIENCEWWW.MATRIX-NI.ORG MATRIX REPORT : VOL 2 10.2008 LIFE & HEALTH SCIENCES HORIZON PANEL REPORT PROSPERITY AND HEALTH DELIVERED BY SCIENCEPREPARED FOR MATRIX BY
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGMATRIX LIFE & HEALTHPANEL MEMBERS SCIENCES HORIZON PANEL MEMBERSAlan 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)
PAGE 03PROSPERITY AND HEALTHDELIVERED BY SCIENCENORTHERN IRELAND’S LIFE & HEALTH The whole sector employs around 4,000 Over the past 18 months our intensive workSCIENCES COMMUNITY IS READY people, many in genuinely sustainable, with colleagues from academia, industry andTO MEET THE SECTOR’S GLOBAL high-value jobs. Around 30 companies in the healthcare has led to many insights and aCHALLENGES. 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 pleasureThe 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 600that will accelerate the development of R&D staff in the process. It is no secret that We would like to thank all of those whoour vibrant life and health sciences sector, in recent years major opportunities have participated in the compilation of this reportboosting economic beneﬁts for the region, resulted from rising investment in R&D. and to acknowledge the work of Phil Towersand improving the quality of health and and Diana Iacob of PricewaterhouseCooperswellbeing 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 strategicand vitality increasing amid the spectre of recommendations that identify the key R&Dnew or re-emerging infectious diseases and capabilities and future market opportunitiesthe need for new treatments, this sector can to exploit for maximum economic return forbe hugely important. the decades ahead.Our work highlights the strength and depth By implementing these recommendations weof the sector and advances made to date. can ensure not just our continued successComprising around 60 companies with a but look forward to the time when Northerncombined turnover of some £400 million, Ireland will feature in the world rankings ofthe industry is growing. These businesses signiﬁcant participants in the huge globalcreate wealth through products as diverse healthcare and pharmaceuticals market. Thisas medical devices and diagnostics, would provide great economic beneﬁts andpharmaceuticals, bio-pharmaceuticals and position us to meet the challenges that our Bernie Hannigan & Albert Sherrardmedical disposals, as well as biotechnology- healthcare systems are facing. Joint Chairs - Life & Health Sciencesbased services and clinical trials. Horizon Panel
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGLIFE & HEALTH SCIENCESHORIZON PANEL REPORT
PAGE 05EXECUTIVE SUMMARYTHE ECONOMIC IMPORTANCEOF THE HEALTH TECHNOLOGIES& LIFE SCIENCES SECTORImportance to the Northern Ireland (that includes NIBEC and the Centre emergence of new diseases due toeconomy: high value-add, export oriented for Advanced Cardiovascular Research), increased global travel and climatesector 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-forindustry, academia and government have made McClay Research Centre for Pharmaceutical performance. In a bid to control spirallingsigniﬁcant 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 thebudgeted for infrastructure enhancement, expertise, the clinical sector is placing an treatment of disease to its prevention. Atcollaborative research, new product increasing focus on clinical research and the same time, industry will be expected todevelopment and staff development initiatives. development (also known as translational prove to healthcare payers that its productsThis 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 Informationderiving from a long history of technological seven Recognised Research Groups and the Technology (IT). This is anticipated toinnovation, backed by a highly educated, young Northern Ireland Clinical Research Network become a key enabler to more effectiveworkforce 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. Thecompanies, offering a very diverse range of rising demand for effective medicines as the pharmaceutical industry is undergoing aproducts, services and capabilities. Data from population ages, new medical needs emerge period of change as it seeks to increase theInvest NI and DETI suggests that the industry, and the disease burden of the developing world pace of innovation in face of its collapsingas 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 andpeople. This tends to be a high value-add market is projected to be worth some $1.3 gene therapy are anticipated to yield moresector and export-oriented, with around 80% trillion. A number of key trends are anticipated new products than the traditional molecularof sales generated from external markets. to shape-up the future of this industry: chemistry-based R&D activity. • Globalised market place. CollaborativeThe academic sector offers a strong science • Changes in disease patterns put product development across boundariesbase, both in life sciences and related sectors, more pressure on healthcare costs. and companies is anticipated to becomethrough its two world class Universities and Increase in chronic diseases as a result more pervasive. India and China are fastthe six regional Colleges of Further & Higher of ageing population, rise of obesity and becoming preferred centres of productEducation. Examples are the Nanotechnology previously terminal diseases becoming development and manufacturing activity.and Advanced Materials Research Institute chronic through therapy development;
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGWHAT ISPERSONALISED MEDICINE?At its most basic, Personalised Medicine refers - to develop new treatments and to identify theto the use of information about a person’s sub-groups of patients for whom they will workgenetic makeup to tailor strategies for the best. It can also help determine which groupsdetection, treatment, or prevention of disease.* of patients are more prone to developing some diseases and, ideally, help with the selection ofPeople vary from one another in many ways - lifestyle changes and/or treatments that canwhat 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 transformfactors, and their DNA. Many of these healthcare over the next several decades. Newvariations play a role in health and disease. The diagnostic and prognostic tools will increasecombination of these variations across several ability to predict the likely outcomes of druggenes 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 particularin the environment, and can be one of the disease state - could result in more focusedreasons why a drug works for one patient and and targeted drug development. Personalisednot another. medicine also offers the possibility of improved health outcomes and has the potential to makePersonalised medicine aims to use these healthcare more cost-effective.variations - both in the patient and in themolecular underpinnings of the disease itselfTHE PARADIGM OF PERSONALISED MEDICINE RISK PREVENTION TARGETED ASSESSMENT MONITORING DIAGNOSIS THERAPY RESPONSE MONITORING EARLY DETECTION TESTINGSource: Personalised Medicine Coalition
PAGE 07BENEFITS OFPERSONALISED MEDICINEA PERSONALISED APPROACH TO MEDICINE OFFERSSIGNIFICANT BENEFITS FOR EACH OF THE MAJORSTAKEHOLDER GROUPS - PATIENTS, GOVERNMENTS, AS WELLAS INDUSTRY. THESE ARE SUMMARISED BELOW.GOVERNMENT/HEALTHCARE SYSTEMS PATIENTS INDUSTRYDetect disease at an earlier stage, when it is Effective and speciﬁc therapies Improve the selection of targets for drugeasier and more economic to treat effectively discovery Less risk of adverse effectsRational therapeutic decisions based on Reduce the time, cost and failure rate ofpathomechanism of disease rather than on Less time lost compared to trial-and-error clinical trialstrial-and-error approach approach to treatment Monopoly in a speciﬁed segment of theIncorporate diagnostic guidance to treatment Lower cost of treatment (in the longer term) market - early entry and longer dominance of market niche; no competition from genericsReduced adverse drug reactions and Facilitates preventive medicinecomplications of treatment Increased drug effectiveness will command Improvement of quality of life higher pricesShift the emphasis in medicine from reactionto prevention Increased revenues from combination of diagnostics packaged with therapeuticReduce the overall cost of healthcare productsIncreased 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.
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 allKEY 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.
PAGE 09COMMERCIAL PROSPECTS FORPERSONALISED MEDICINEThe market for personalised medicine is • Molecular diagnostics as a stand-alone ineffective and disruptive for patients. Strongmultifaceted, 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; andetc. The diagram below captures the main • Development of new therapies, such as Assuming a scenario where Personalisedconstituents 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 indicatesPersonalised Medicine has the potential a market potential in excess of $200bn.to transform healthcare over the next Companies may specialise in certain diseaseseveral 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. AThe 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 ofviewed 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 DisciplinesMolecular Diagnostics Personalised Therapies Disease Areas GenomicsDNA Sequencing Pharmaceuticals Cancer PharmacogeneticsGene expression proﬁling Recombinant human proteins Diabetes PharmacogenomicsSNP Genotyping Therapeutic monoclonal antibodies Obesity PharmacoproteomicsBiochips and microarrays Gene and cell therapy etc. Cardiovascular PharmacometabonomicsBiomarkers Central nervous system etc.Molecular imaging etc. Systems biology Bioinformatics NanotechnologyTHE PARADIGM OF PERSONALISED MEDICINE RISK PREVENTION TARGETED ASSESSMENT MONITORING DIAGNOSIS THERAPY RESPONSE MONITORING EARLY DETECTION TESTING
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGNORTHERN IRELANDCURRENT CAPABILITY INPERSONALISED MEDICINEA NUMBER OF KEY BUILDING BLOCKS IN THE DEVELOPMENT OFPERSONALISED MEDICINE ALREADY EXIST IN NORTHERN IRELAND,ACROSS THE ACADEMIC, PRIVATE BUSINESS AND CLINICAL (ABC)SECTORS. OVER THE PAST FEW YEARS, NORTHERN IRELANDINDUSTRY, ACADEMIA AND GOVERNMENT HAVE MADE SIGNIFICANTCOMMITMENT TO THE DEVELOPMENT OF LIFE SCIENCES CAPABILITIES.The Northern Ireland business sector has a The health service is placing an increasing Successful development of Personalisednumber of leading players, providing highly focus on clinical research and development, Medicine in Northern Ireland is dependent oninnovative products and services. The sector and commercialisation of innovation arising the sector’s ability to draw on and develophas witnessed a period of growth since 2000, from the clinical sector. It has enormous local capability in complementary sectorswith 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 lifeuniversity 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 sectorsthrough its two world class Universities and the The diagram opposite suggests that in areassix Colleges of Further and Higher Education. such as clinical trials and biotechnologyExamples are the Nanotechnology and the commercial sector is more advanced inAdvanced Materials Research Institute (that successfully exploiting the local scientiﬁcincludes NIBEC and the Centre for Advanced capability. However, a focused effort isCardiovascular Research) and Biomedical required to more fully realise the potentialSciences Research Institute at the University offered by the scientiﬁc strengths in areasof 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 collaborativeCancer Centre and the McClay Research programmes for knowledge and skill transfer,Centre for Pharmaceutical Sciences at technology licenses, or creation of wellQueen’s University Belfast. supported spin-off companies.Source: Technology Capabilities Study for Northern Ireland, MATRIX, 2007
PAGE 11FIGURE 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
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGREALISING THEPERSONALISED MEDICINE OPPORTUNITYFOR NORTHERN IRELANDAs the market intelligence indicates, in the and raising its position in the internationallonger term this sector has the potential to arena. The sector would also be able totransform the way medicine is delivered. Many communicate with a single voice and engagenations have identiﬁed the opportunities offered in a coherent manner with the local supportby this high risk - high reward sector, and mechanisms and stakeholders, for exampleinvestment in research and commercialisation to promote skills development and raise thein this space has intensiﬁed in recent years. proﬁle of the local cluster.The Life and Health Sciences Horizon Panelhas identiﬁed an opportunity for NorthernIreland to carve out a share of this growingmarket, by becoming a centre for IntegratedResearch & Development in PersonalisedMedicine. This would fuel the developmentof Personalised Medicine in Northern Ireland, Northern Ireland to become a centre for Integrated Research & Developmentand 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-outsthe integrated nature of the services provided, Achieve global recognition for excellence in a number of strandsharnessing academic scientiﬁc expertise, Act as Northern Ireland ‘shopwindow’ to the international communityclinical data and practice and a focus oncommercialisable outputs.The Panel believes that the sector could bestimulated locally by creating the environment Local Appliedfor the initiation of commercially-targeted ABC Players ResearchR&D projects, focussing funding and supporttowards applied research not elsewhere fundedand the gap between initial Proof of Conceptand Phase II Clinical trials. Being open to thefull spectrum of the Personalised Medicine Leading Developmenttechnologies and practices, is expected to Pharma & Biotechstimulate interest from across the sector and Companiesallow the most economically valuable proposalsto emerge.It is believed that, given the recent Medical Applied Researchdevelopments in local infrastructure, a ‘virtual’ Charities & Developmentsupport mechanism would be sufﬁcient,providing leadership, funding and expertsupport rather than new physical infrastructure.A highly focused approach to the development Enabled by Northern Ireland Scientiﬁc & Clinical Capabilityof this exciting sector would help drive closercooperation and capability transfer acrossthe ABC boundaries, thus enhancing theexploitation capability within the local sector
PAGE 13KEY STAKEHOLDERINPUTS & OUTPUTSTHE IMPLICATIONS IN TERMS OF COMMITMENT, RESOURCES ANDBENEFITS FOR EACH OF THE LOCAL STAKEHOLDER GROUPS INREALISING THE PERSONALISED MEDICINE MARKET OPPORTUNITYCAN BE SUMMARISED AS FOLLOWS:GOVERNMENT ACADEMIC STAKEHOLDERS BUSINESS SECTOR CLINICAL SECTORInputs 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 partnersOutputs 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.
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGWHAT IS HOME-BASED CARE?THE CORE PROPOSAL FROM THE HORIZON PANELCONCERNS THE LINK BETWEEN PATIENTS IN THEIR HOMESAND THE HEALTH SYSTEM. THIS LINK CAN BE CONSIDEREDTO INCLUDE THE FOLLOWING THREE MARKET COMPONENTS.1 Telehealth 2 Telecare 3 Secure web messaging and e-visitsTelehealth monitoring is the remote exchange Telecare is the continuous, automatic and This technology usually acts as an enablerof 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 anddiagnosis, progress monitoring and prevention the risks associated with independent living. e-mail allows for more efﬁcient communicationof various conditions. Speciﬁc product Telecare is usually designed to create home with medical staff and for novel healthcareexamples include home-based blood pressure environments which meet the needs of older solutions. A speciﬁc example of an e-visit couldmonitors that relay information to the patients or disabled people. Speciﬁc product examples be an email exchange between a doctor andGP surgery. Their immediate applications tend include fall or bed sensors. patient. The market also includes technologiesto 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 corepervasive in the longer term. proposals, but instead are recommended for consideration in the longer term.
PAGE 15KEY MARKET DRIVERS ANDCHALLENGES TO DEVELOPMENTKEY 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
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGCOMMERCIAL PROSPECTS FOR THEHOME-BASED CARE MARKETTHE CORE PROPOSAL FROM THE HORIZON PANEL CONCERNSTHE LINK BETWEEN PATIENTS IN THEIR HOMES AND THEHEALTH SYSTEM.The US is, and will remain, easily the largest In comparison, the entire European marketnational market for telehealth, reaching $4.51 is expected to only reach about $1.00 billionbillion in 2010 according to Datamonitor by 2010, despite having a projected 502forecasts. 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 thanmarket share are the result of two that in the North America, but it will still showmain 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.
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
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGREALISING THE HOME-BASED CAREOPPORTUNITY FOR NORTHERN IRELANDThe Life and Health Sciences Horizon Panel The overall aim of the model proposed forrecommends that Northern Ireland becomes the Northern Ireland is to join up local capabilityﬁrst UK region committed to the early adoption across all four of these domains in anof a telehealth system within the Health and integrated manner and in a real clinical setting.Social Care practice. This means establishinga strong local capability across the telehealth The Panel concluded that to maximise thecontinuum, and deploying this locally to achieve market opportunity in this area, telehealthwhole connectivity between the health sector technology should be implemented within ourand the home within 15-20 years. own health system.The Home-based care market encompasses • This would allow the DHSSPS to beneﬁta wide range of products and services. The from savings achieved from the deliveryunderpinning technologies and systems of health services in this manner, whilstnecessary to deliver those services can be improving patient care; andsub-divided into four key domains along the • At the same time, it would create antelehealth continuum, as summarised in the attractive Whole System Integration Testdiagram 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
PAGE 19The Panel considered the Northern Ireland Data Analysis Government supportcapability’s strengths and gaps against the four • The development of a Home-based care • The announcement made in Januarykey areas judged to constitute the home based market will require a capability in managing 2008, regarding the development of ancare continuum, and the level of support and and analysing large volumes of data. The European Centre for Connected Healthcommitment 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 powerfulMedical 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.
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGKEY STAKEHOLDERINPUTS & OUTPUTSTHE TABLE BELOW SUMMARISES THE COMMITMENT NECESSARY FROMGOVERNMENT, ACADEMIA, BUSINESS AND THE CLINICAL FIELD TOSUCCESSFULLY DELIVER HOME-BASED CARE WITHIN THE NORTHERNIRELAND ECONOMY. THE TABLE ALSO HIGHLIGHTS THE POTENTIAL RETURNFOR EACH RESPECTIVE GROUP.GOVERNMENT ACADEMIC SECTOR BUSINESS SECTOR CLINICAL SECTORInputs 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 changeOutputs 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
PAGE 21FRAMEWORKCONDITIONSThe health technologies and life sciences Personalised medicine speciﬁc factors:sector has the potential to become a major • Participation from the clinical sectorcontributor to Northern Ireland’s knowledge- would be critical to help demonstratebased economy. There are however a number of the economics and clinical beneﬁts ofprerequisites 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 toGeneral 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.
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGSUMMARY OFPROPOSALSTWO DISTINCT MARKET OPPORTUNITIES HAVE BEEN IDENTIFIED BY THE HEALTH AND LIFE SCIENCESHORIZON PANEL: PERSONALISED MEDICINE AND HOME-BASED CARE. WHILST THEY HAVE A NUMBEROF COMMON FEATURES, IN THAT BOTH ADDRESS ISSUES FACING THE GLOBAL HEALTH AND ECONOMYIN THE LONG-TERM, AND THEY ULTIMATELY COMPLEMENT EACH OTHER WITHIN THE INTEGRATEDHEALTHCARE CONCEPT, THE TWO MODELS OFFER DIFFERENT PROPOSITIONS IN TERMS OF TYPE OFCAPABILITY IMPACTED LOCALLY, ECONOMIC IMPACT, TIMESCALE AND RISK FACTORS. THE TWO MARKETPROPOSALS ARE SUMMARISED BELOW.PERSONALISED MEDICINE HOME-BASED CAREMarket 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 behindNorthern 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
PAGE 23PERSONALISED MEDICINE HOME-BASED CAREAdvantages 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 countryKey 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
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGCONTENTS1 425 INTRODUCTION 69 FRAMEWORK CONDITIONS26 Economic importance of the sector 71 General Enabling Factors29 The Horizon Panel objectives 73 Personalised Medicine30 Approach to delivery 74 Home-based Care232 PERSONALISED MEDICINE - THE OPPORTUNITY FOR NORTHERN IRELAND33 What is Personalised Medicine?34 Applications of Personalised Medicine36 Beneﬁts of Personalised Medicine37 Key market drivers and challenges to development41 The Market for Personalised Medicine is Multifaceted42 Commercial Prospects48 Northern Ireland Capability52 The Opportunity for Northern Ireland355 HOME-BASED CARE MARKET - THE OPPORTUNITY FOR NORTHERN IRELAND56 Deﬁning the market57 Key market drivers and challenges to development59 Driving Policies and Iniatives in the Sector60 Commercial prospects62 The Opportunity for Northern Ireland64 Realising the Home-Based Care Opportunity for Northern Ireland66 Northern Ireland Capability67 Economic Beneﬁts68 Key Stakeholder Inputs & Outputs
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGTHE ECONOMIC IMPORTANCE OFTHE HEALTH TECHNOLOGIES & LIFESCIENCES SECTORNORTHERN IRELANDCONTEXTAs technologies converge, ‘life sciences’ • The need for stronger links with academia Over the past ﬁve years Northern Irelandrepresents an all-encompassing title capturing and clinicians in order to bolster technology industry, academia and government have madea wide range of industry sub-sectors such transfer and innovation signiﬁcant commitment to the development ofas pharmaceuticals, biotechnology, medical • Shortage of ‘veteran’ entrepreneurs in life sciences capabilities. In excess of $160devices and diagnostics, drug delivery, clinical Northern Ireland, with the experience and million has been budgeted for infrastructuretrials etc. The sector in Northern Ireland time to mentor life science businesses enhancement, collaborative research, newcomprises organisations across the Academic, • Need to remain at the forefront of R&D, product development and staff developmentBusiness and Clinical (ABC) sectors. in light of increasing competition from low initiatives. cost manufacturing regionsThe business sector counts approximately 60 In the same timeframe, Northern Irelandcompanies, offering a very diverse range of The academic sector offers a strong science life science companies have committed toproducts, services and capabilities. Data from base, both in life sciences and related sectors strategic investment expenditure in the orderInvest NI and DETI suggests that the industry through its two world class Universities and of $340 million including approved governmentas 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, employingpeople. This tends to be a high value-add and Advanced Materials Research Institute (that around 4,000 in life sciences, this level ofsector and export-oriented, with around 80% includes NIBEC and the Centre for Advanced investment (almost $125,000 per employee) isof sales generated from external markets. Cardiovascular Research), Biomedical Sciences impressive. Research Institute, Centre for Cancer ResearchThe 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 opportunitiesand 3,838 of its employees. The majority for Pharmaceutical Sciences. deriving from a long history of technologicalof companies in the sector are small, with innovation, backed by a highly educated, youngturnovers 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.
PAGE 27GLOBALCONTEXTA growing market Growing antibiotic resistance However, the human genome has proved moreAt global level, the sector is set to beneﬁt from in existing diseases. complex and less amenable to mechanisticrising 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 strugglingincreasingly resembles that of the developed contained pathogens. Examples include to apply the insights it has gleaned from theworld. 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 improvetrillion. The market is anticipated to be shaped Industry innovation crisis its performance. The industry requiresup 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 andChanges in disease patterns put position to capitalise on the market compounds.pressure on healthcare costs opportunities, unless it can change the way inIncrease in chronic diseases. The global which it operates. Its core problem is the slowing Globalising marketplacepopulation 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 trainedof the world’s inhabitants - will be 65 or more, The industry’s best hope of earning higher researchers and improving intellectualcompared with 477.4 million (7.3%) two years returns lies in the development of packages of property norms, China and India look likely toago. Older people typically consume more products and services targeted at patients with become a focus of product development andhealthcare 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 havechronic diseases. Obesity, especially in the US, to start by focusing on diseases rather than Changing relationshipsis another driver of higher costs, as it tends compounds. Co-operation will be a critical element ofto lead to chronic diseases such as diabetes, success in life sciences. Biotech and bigcoronary 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-operateMore new diseases. molecular R&D activities. Gene therapy is globally on processes such as productGreater population density, increased travel likely to move from treatment of conditions to development or test analysis. Increasing levelsand climate changes have increased the cures by modifying patient DNA. Previously of patient choice and knowledge will place aappearance of new pathogens. These are untreatable conditions could therefore ﬁnd premium on successful relationships betweenoften 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.
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGIncreasing Role of Information Pay-for-performanceTechnology (IT) The provision of healthcare is not all thatMore pervasive use of IT will lead to more is changing; so is the way in which it iseffective storage and retrieval of patient measured. Several countries have set uprecords, improved tracking of medical agencies speciﬁcally to compare the safety andoutcomes and better remote care. Automation efﬁcacy of different forms of intervention andof 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 andvalue activities such as diagnosis and patient Quality is one such body, as is the UK Centrecommunication. for Health Technology Evaluation - a division of the National Institute for Clinical HealthBlurring Healthcare Boundaries and Effectiveness (NICE). The industry willChanges in the way healthcare is delivered have to prove to healthcare payers, who arewill arguably play an a critical role in shaping increasingly interested in establishing bestthe industry’s future. The primary-care sector medical practice, that its products really workis expanding and becoming more regimented, and provide value for money.as general practitioners perform more minorsurgical procedures and healthcare payers Focus on preventionincreasingly mandate the treatment protocols A growing number of governments in boththey must follow, including the drugs they can developed and developing countries are tryingprescribe. Conversely, the secondary-care to shift the focus from the treatment of diseasesector is contracting, as clinical advances to its prevention. For example, at least 18render previously terminal diseases chronic; countries have already introduced nationwidehealthcare providers like Clinovia in the UK, bans on smoking in enclosed public places, asand Gentiva in the US, deliver secondary care have a number of US states. Similarly, someat home; and hospitals focus on the specialist countries are waging war against rising levelscare that cannot be supplied anywhere else. of obesity. The role of genetics in identifyingThe self-medication sector is also growing, as those at risk and in early interventions is alsomore and more products that would once have expected to play a major role in preventativebeen available only on prescription are sold in medicine.OTC formats.
PAGE 29THE HORIZONPANEL OBJECTIVESThe Life and Health Sciences Horizon Panel The Life and Health Sciences Horizon Panel was tasked to deliver on theis one of the ﬁve technology horizon scanning following objectives:panels established under the auspices of theNorthern Ireland Science Industry Panel -MATRIX. The other four Horizon panels i Identify the sustainable market opportunities that could berepresent 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 NorthernMATRIX is an expert advisory panel Ireland science and industry base relevant to the identiﬁedreporting to DETI and the DETI Minister onmatters pertinent to the exploitation and market opportunities;commercialisation of science, technology andR&D. It is led by high-technology and R&D iii Determine the opportunities for external collaboration thatintensive industry and advises Northern IrelandGovernment on the development of improved would supplement critical gaps in NI research and technologyinterfaces between Northern Ireland business capability in ensuring early delivery of potential outputs;and the research, science and technologybase, with a view to ensuring the region’s iv Engage with key stakeholders and partners critical to thescience and R&D strengths are exploitedfor maximum economic and commercial success of relevant market opportunities (public, private andadvantage. 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.
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGAPPROACH TO DELIVERYWe adopted a step driven approach to 1 Project Mobilisationachieving the project deliverables, underpinned An initial meeting was held with the Matrixby monthly Panel working meetings. The secretariat to agree the approach to delivering12-strong Panel brought together leading and managing the project. This was thenrepresentatives from across the local captured in the Project Initiation Document.industry - academics (6), clinical sectorR&D representatives (2), and private sector 2 Project Scopingcompanies (2 large businesses and 2 SMEs). Given the broad and complex nature of theThe 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
PAGE 31approach to identifying the relevant market The proﬁle of the local sector capability wasopportunities for Northern Ireland. It was also analysed, drawing information from DETI’sultimately agreed that the most effective means parallel study on Northern Ireland Technologyto achieving this was through a brainstorming capability, and the BioBusiness and Invest NIsession, which would harness the collective Sector review conducted in 2006.knowledge of the Panel members and themarket acumen of the PwC core team and In light of the research ﬁndings, a number ofexpert advisors. The event was held in May suggestions were made on potential areas2007 and elicited views on ‘Sustainable Market of focus for the Northern Ireland cluster, andOpportunities for Northern Ireland plc’ from models for implementation.a wide number of standpoints: World diseaseprevalence, Key industry trends, Emerging and 4 Consolidation Of Optionscross-cutting technologies, Policy & regulation, The options on the best approach forand Northern Ireland competitive position. Northern Ireland approaching this market and establishing a competitive position wereOver 100 innovative ideas were generated, deliberated with the Panel members. A numberwhich were then analysed and found to cluster of options to the implementation models werearound 11 principal market-related themes. The discussed, together with the implications forPanel ﬁltered these further and selected the top the local stakeholders.priorities through a voting process. The resultswere discussed at the Panel meeting in June, The emerging thoughts were shared and testedwhen the top two key areas of focus for Northern with a limited number of key sector leaders inIreland were agreed as: Personalised Medicine the local economy. These discussions helpedand Home-based Care. gauge their views on how the proposed models could be effectively deployed, and initiated3 In-depth Market Analysis early buy-in and engagement across theIn-depth market research was carried out industry community.within the two selected themes, to explorespeciﬁc technology application opportunities 5 Reportingwithin each. The research used analysis of The ﬁnal report explores the recommendationsextensive market intelligence and interviews of the Life and Health Sciences Horizon Panelwith international and local industry experts, in the areas of Personalised Medicine andto establish: Key areas of market growth, Home-based Care and summarises the marketEmerging technology applications, Key players, rationale for these recommendations.and Potential collaboration partners.
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGPERSONALISED MEDICINE -THE OPPORTUNITY FORNORTHERN IRELAND2
PAGE 33WHAT IS PERSONALISEDMEDICINE?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 ofgenetic makeup to tailor strategies for the lifestyle changes and/or treatments that can diagnostics and therapeutics is the availabilitydetection, 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 arePeople 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. Newthey experience, exposure to environmental diagnostic and prognostic tools will increase Although scientiﬁc advances such asfactors, and their DNA. Many of these the ability to predict the likely outcomes of drug the mapping of the human genome andvariations play a role in health and disease. The therapy, while the expanded use of biomarkers computation technology are underpinningcombination of these variations across several - biological molecules that indicate a particular fast scientiﬁc developments, adoption in thegenes can affect each individual’s risk of disease state - could result in more focused mainstream clinical system lags behind. Ifdeveloping 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 regulatorynot another. healthcare more cost-effective. approaches, revamped medical education curricula, integrated health informationPersonalised Medicine aims to use these The illustration arrow reﬂects the current and systems, legislation to protect againstvariations - both in the patient and in the anticipated ﬂow of healthcare services, and genetic discrimination, insurance coveragemolecular 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 encouragessub-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 detectingTHE PARADIGM OF PERSONALISED MEDICINE** RISK PREVENTION TARGETED ASSESSMENT MONITORING DIAGNOSIS THERAPY RESPONSE MONITORING EARLY DETECTION TESTING* (Source: Personalised Medicine Coalition)** (Source: Personalised Medicine Coalition)
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGAPPLICATIONS OFPERSONALISED MEDICINEA PERSONALISED APPROACH TO MEDICINE PROMISES A NUMBEROF BENEFITS IN ADDRESSING THE KEY CHALLENGES FACINGHEALTHCARE SYSTEMS WORLDWIDE:Currently, physicians often have to use trial Governments and medical communities are screening programmes and diagnostic testsand error methods to ﬁnd the most effective increasingly stressing preventive medicine as at personal and General Practitioner levelmedication for each patient. As more is learnt the most cost-effective approach to improving would enable people to be more in charge ofabout which molecular variations best predict the quality of life. Developments in molecular their own health and health practitioners tohow a patient will react to a treatment, and diagnostics and genetic testing will drive this recommend life style, nutritional and medicaldevelop accurate and cost-effective tests, market. The design and validation of preventive action at an early stage.doctors will have more information to guidetheir decision about which medications arelikely to work best. Testing is already beingused to ﬁnd the one in four women likely torespond to a particular breast cancer drug. Inaddition, testing could help predict the best BETTER DIAGNOSESdosing schedule or combination of drugs for a AND EARLIERparticular patient. INTERVENTIONMolecular analysis could determine preciselywhich variant of a disease a person has, orwhether they are susceptible to drug toxicities,to help guide treatment choices. For preventivemedicine, such analysis could improve the abilityto identify which individuals are predisposed INCREASED MORE BENEFIT ADOPTION OFto develop a particular condition - and guide EFFECTIVE FOR HEALTHCARE PREVENTATIVEdecisions about interventions that might THERAPIES SYSTEMS CAREprevent it, delay its onset or reduce its impact.Toxicity associated with inappropriate use ofapproved drugs is a real problem and a cost tohealthcare systems, that Personalised Medicinecan provide a partial solution to. In the USAalone, adverse drug reactions (ADRs) are IMPROVEDresponsible for approximately 100,000 drug- SAFETYrelated deaths and 2.2 million hospitalisations OF DRUGSper year, representing a cost of roughly$100 billion.
PAGE 35PERSONALISED MEDICINE ALSO HAS THE POTENTIAL TO HELPTHE PHARMACEUTICAL INDUSTRY TO IMPROVE ITS PERFORMANCEAROUND 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 throughdrugs’ that have failed clinical trials because genotypic characteristics, patients who are pharmacogenomics can extend the patentof lack of efﬁcacy and toxicity problems. non-responders and those liable to adverse life of a drug. A drug with redeﬁned newNew indications can be explored in deﬁned reactions, can be excluded. indications for a particular group of patientspopulations groups where the drug use is safe would start a new patent life.and effective.Redeﬁning the patient population for marketeddrugs would enhance effectiveness and safety.Upper ﬁgures for efﬁcacy of medical treatmentof most diseases are less than 100%. Examplepercentage of patients that show lack of MORE EFFICIENT DRUGresponse to current therapy in key disease DEVELOPMENTareas: Epilepsy - 70%, Hypertension - 65%, PROCESSDepression - 60%, Diabetes mellitus - 55%,Rheumatoid arthritis - 50%, Alzheimer’sdisease - 30%. Improvement in treatment isan opportunity for development of PersonalisedMedicines.A better understanding of genetic variations EXPAND RESCUING BENEFIT MARKET OFcould help scientists identify new disease ‘DEAD DRUGS’ FOR EXISTINGsubgroups and their associated molecular INDUSTRY DRUGSpathways, and design drugs that target them.Molecular analysis could also help selectpatients for inclusion in, or exclusion from,late stage clinical trials - helping gain approvalfor drugs that might otherwise be abandonedbecause they appear to be ineffective in thelarger patient population. EXTEND IMPROVED PATIENT LIFE BY EFFICACY OF IDENTIFYING NEWPharmacogenomics may be applied to expand TREATMENTS APPLICATIONSmarkets for approved drugs with generallyrestricted use because of limited efﬁcacy
PROFITING FROM SCIENCE WWW.MATRIX-NI.ORGBENEFITS OFPERSONALISED MEDICINEA PERSONALISED APPROACH TO MEDICINE THEREFORE OFFERS THEPROSPECT FOR SIGNIFICANT BENEFITS FOR EACH OF THE MAJORSTAKEHOLDER GROUPS - PATIENTS, GOVERNMENTS, AND INDUSTRY.THESE ARE SUMMARISED BELOW.GOVERNMENT/HEALTHCARE SYSTEMS PATIENTS INDUSTRYDetect disease at an earlier stage, when it is Effective and speciﬁc therapies Improve the selection of targets for drugeasier and more economic to treat effectively discovery Less risk of adverse effectsRational therapeutic decisions based on Reduce the time, cost and failure rate ofpathomechanism of disease rather than on Less time lost compared to trial and error clinical trialstrial-and-error approach approach to treatment Monopoly in a speciﬁed segment of theIncorporate diagnostic guidance to treatment Lower cost of treatment (in the longer term) market - early entry and longer dominance of market niche; no competition from genericsReduced adverse drug reactions and Facilitates preventive medicinecomplications of treatment Increased drug effectiveness will command Improvement of quality of life higher pricesShift the emphasis in medicine from reaction toprevention Increased revenues from combination of diagnostics packaged with therapeuticReduce the overall cost of healthcare productsIncreased 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.