A complete report of Stratified Medicine as the new approach to deliver healthcare and fully Personalised Medicines. Market and key drivers, industry needs and the Scottish strengths, are all presented in this report.

1. OPPORTUNITIES IN LIFE SCIENCES
Scottish Enterprise
Stratified and Personalised Medicine:
An Overview
Supporting a globally competitive Scotland

2. OPPORTUNITIES 1
Scottish Enterprise
SUMMARY
Stratified medicine encompasses the following approaches:
1) The use of a range of genomic and other biomarkers
to improve the drug development process by significantly
reducing cost and risk.
2) The use of companion diagnostics (CDx) to identify patient
populations into those most likely to respond favourably,
and without adverse effects, to a particular therapeutic
(Rx) treatment option. The pace of change will be
3) The use of biomarkers to optimise dosage of drug, for accelerated yet further by the
prognosis and monitoring outcome of treatments. imminent arrival of affordable
Successfully deployed stratified medicine approaches serve
to increase margins for pharmaceutical companies, markedly
genome based personalised
reducing cost and speeding drug development. This is particularly medicine.
important at a time when major pharmaceutical companies are
coming under intense pressure as patents on key products expire
and these products are substituted by generics.
To date the application of stratified medicine approaches has
been primarily associated with drug based therapeutics and
molecular or protein based biomarkers. However the process is
generic and is likely to develop and encompass a wider range of
both therapeutic modalities (cellular, surgical & behavioural) and
analytical techniques (physiological measurements and functional
imaging).
Stratified medicine can also generate significant savings for
healthcare providers and most importantly deliver better clinical
outcomes to the patient.
Full reports on the markets covered here can be downloaded from the Scottish Enterprise website www.scottish-enterprise.com/lifesciences

3. OPPORTUNITIES 2
BACKGROUND
Stratified medicine is a new approach to delivering
healthcare, and can be seen as a step on the road to
delivering fully personalised medicines. It encompasses
biomarkers derived from all “omics” technologies (genomics,
proteomics and metabolomics) and increasingly other
analytical technologies such as imaging. To date such
biomarkers have been principally used as a tool to accelerate
drug discovery and development and develop a small group
of Rx-CDx products to stratify patient populations based on Personalised medicine is very much in
disease status and response to therapy. process and will lead to the sequencing of
The pace of change will be accelerated yet further by the
imminent arrival of affordable genome based personalised
individual genomes becoming a routine.
medicine. The commercial availability of a “$1,000 genome” was
announced by a major research tools company in early 2012.
An affordable “$100 genome” by 2018 is a realistic proposition.
Thus the transition from the current era of stratified medicine to
one of personalised medicine is very much in process and will
lead to the sequencing of individual genomes becoming a routine
and accepted part of clinical practice.
Stratified medicine has already significantly changed the
pharma, diagnostics, biotechnology and impacted IT industries by
generating a much greater “evidence base” for informed decision
making. Personalised medicine is likely to similarly change
clinical practice. This will present not only major challenges for
the life science sectors and NHS in Scotland but also tremendous
commercial and societal opportunities.

4. OPPORTUNITIES 3
Market and Key Drivers Industry Needs
The leading market for both CDx and biomarkers has been cancer. • Validated biomarkers: New technical breakthroughs
The market value of targeted cancer therapies, where there is a have produced a huge pipeline of prospective biomarkers
companion diagnostic element, is now $40 billion and is growing at all of which require better and faster methods of
CAGR of 15%. Landmark companion diagnostic (CDx) products such validation, a major opportunity for Scottish CRO base and
as the Her2 test for breast cancer patient stratification are now in translational research base.
extensive use in both Europe and US and generating significant • Data handling (IT & Bioinformatics): The two major
revenues ($100 million). needs and opportunities are for seamless and secure
The market for cancer biomarkers in general (i.e. Non CDx), employed medical records and data recording systems to efficiently
in cancer clinical trials and discovery, was ten times greater at $1 deal with the sheer volume of data. This is an opportunity
billion in 2010. This in turn was part of the $20 billion market for all for Scottish IT companies to specialise in medical
biomarkers used in drug development (BCC Research 2010). related data handling. Related to this are opportunities
to statistically analyze data and put the data in a context
Biomarkers for drug development and clinical trials for other relating biomarker changes to underlying biology.
indications are coming on line, and Rx-Dx combinations are
evidently negotiating the complexities of regulatory approval for • Adaptation to changes in the regulatory processes:
both diagnostics and therapeutics. Stratified and personalised medicine is going to produce
major changes in regulatory authorities requiring more
In short, stratified medicine is here to stay. Key drivers include complex data submission packages which incorporate
needs for: biomarker data within a regulatory submission for a new
• Pharmaceutical companies to reduce the cost and drug. There is an opportunity for Scottish companies,
increase the speed of the drug development process; particularly specialist CRO’s and consultancies, to gain
market share by anticipating and adapting to these
• Cost savings for healthcare providers; changes quickly.
• Improved clinical outcomes for patients. • Development of new business models & processes:
Thus the ability of countries, life science industry sectors, New therapeutics have traditionally been priced by value
companies and healthcare systems to quickly anticipate adapt – where a perceived monetary value is assigned to the
and successfully exploit the opportunities created by advances impact of the treatment. In contrast diagnostics have been
in stratified medicine is of immediate commercial importance. priced according to production cost with a margin added.
Success in adapting to the changes brought by stratified medicine This has created an as yet unresolved business challenge
is strategically important. It will provide a foundation and between the in vitro diagnostics providers and pharma
bridgehead to take advantage of what may be much greater long- partners. Similarly optimised processes to effectively
term opportunities created as personalised medicine becomes a incorporate novel biomarker and diagnostics development
technical and commercial reality. into the drug development process are required.

5. OPPORTUNITIES 4
Solving these issues are both opportunities for Scottish Scottish Strengths
companies, particularly in vitro diagnostic & CRO
companies, to develop novel business pricing solutions • A strong presence of major international research
re-engineered workflow processes and strategic tools companies: Major research tools companies are
partnerships. implanted in Scotland (Life Technologies, Gen-Probe,
Millipore now Merck, Agilent and a number of SMEs). This
• Adaptations within Healthcare Provider Systems: cluster is capable of developing and manufacturing kits
Change in clinical evaluation bodies (equivalent to NICE and services which provide stratified medicine tools for a
in UK), to evaluate cost effectiveness of new personalised global Pharma and biotechnology market.
medicines are needed. Adapting NHS and other healthcare
systems to deliver stratified and personalised medicine, • An internationally competitive cluster of pharma CRO’s
in particular handling of genomic data and long term with presence of majors such as Charles River, Aptuit &
education of clinicians and patients is required and will Quintiles: As with the research tools companies this cluster
create new opportunities for prepared companies. is competitive and capable of adapting its processes and
services to exploit the growth of stratified medicine.
• A vibrant group in vitro diagnostic (IVD) and imaging
companies: Scottish IVD and imaging companies have the
technical capability of developing and providing companion
diagnostics co-marketed with new pharmaceuticals. To
date most marketed CDx products have been molecular
biomarker based tests using DNA/RNA or proteins.
However in future other diagnostic modalities such as
imaging based CDx tests will be deployed.
• An internationally competitive clinical translational
research base: Scotland clinical translational researchers
are working on most of the disease areas that stratified
medicine is predicted to impact upon in the coming
decade. The major Wyeth TMRC investment created
infrastructure, and more importantly a cohort of clinical
researchers (now within Health Science Scotland), with
extensive experience of industry lead stratified medicine
development. Other initiatives which will support stratified
medicine include biobanks notably Generation Scotland,
significant initiatives in bioinformatics through Scottish
Bioinformatics Forum (SBF) and coordinated mechanism
of patient recruitment through Scottish Health Informatics
Partnership (SHIP).

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February 2012