Bioscience : lost in translation? : how precision medicine closes the innovation gap

241 Pages·2016·1.918 MB·English

by  Barker, Richard

Bioscience— Lost
in Translation?

Bioscience— Lost
in Translation?
How precision
medicine closes
the innovation gap
Professor Richard Barker
Centre for the Advancement of Sustainable Medical
Innovation (CASMI),
Oxford, UK
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1
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Foreword
It’s my pleasure to commend this timely and important book from Richard Barker,
Chair of the Precision Medicine Catapult and one of the UK’s leading figures in
precision medicine.
We are seeing unprecedented breakthroughs here in the UK and across the world
in biomedicine and health technology that have major potential to save or enhance
patients’ lives as never before.
At the basic science level these are extraordinary times with a range of genuinely
transformational new treatments and cures in the pipeline. From cancer to rare
diseases, and to major chronic illnesses that bring such significant costs to individuals
and society.
But the pace, scale and nature of these transformations challenge the traditional
siloed models of both long-chain R+D, and the assessment, adoption and reim-
bursement of new treatments. The quiet revolution of patient centered research and
Precision Medicine drives and demands a much closer integration of treatment and
research.
Having launched the UK’s Life Sciences Strategy with the Prime Minister in 2011,
I am personally and deeply committed to bringing in the changes we need, both
nationally and internationally. Here in the UK I have launched the Early Access
to Medicines Scheme and the Accelerated Access Review for a new model of NHS
partnership in the innovation department, to drive forward crucial reforms to the
UK landscape.
The thesis of this book is that the new era of precision medicine holds the most
important key to revolutionising biomedical science and 21st century healthcare. I
agree. So I am delighted to commend this provocative yet practical book to all who
want to see advances in bioscience benefit patients more rapidly and more effectively.
George Freeman MP,
Minister for Life Sciences

Preface and acknowledgements
This book is written to focus the minds of medical professionals, life science research-
ers, policy- makers, and industry executives on a problem that frustrates so many of
their efforts to advance medical innovation. Much too much promising bioscience has
been— and continues to be— lost in translation. Over more than 25 years of observing,
managing, and studying innovation in biomedicine, I have become convinced we can
and must overcome these gaps in translation, if we set out to understand their origins.
Other domains of human endeavour, especially the world of information techno-
logy, have seen dramatic practical advances over this same time period: products with
better performance, at lower cost, reaching the consumer faster each year. In contrast,
in medical innovation we have seen huge investments yield fewer products, slower and
at greater cost, year on year. The result, of course, is a process that is unsustainable. We
all need this situation to change radically, both in our professional roles and as patients
and tax- paying citizens. I have written this book as my contribution to driving the
changes we need to close the innovation gap.
I have been privileged to work in a wide variety of healthcare organizations on both
sides of the Atlantic. They have included universities, private companies, public sector
bodies, and the voluntary sector. I see the innovation challenge as a call to teamwork
across all of them.
The groundwork for my thinking was laid in leadership roles at IBM, Chiron,
iKnowMed, Molecular Staging, and the Association of the British Pharmaceutical
Industry (ABPI). While working on medical innovation issues in each of them I have
benefited greatly from the insights of colleagues far too numerous to mention. But
I would like to pay particular tribute to a few who have particularly influenced my
recent thinking—t o my senior Centre for the Advancement of Sustainable Medical
Innovation (CASMI) colleagues John Bell and Hugh Watkins at Oxford, John Tooke
and Rob Horne at UCL, and to a very stimulating group of (largely) young CASMI
members and associates, notably Jack Scannell, David Brindley, Natasha Davie, Liz
Morrell, Megan Morys, Elin Haf Davies, Sarah Garner, Rosie Pigott, Stuart Faulkner,
Suzanne Ii, and my CASMI co- founder, Nick Edwards.
I have drawn freely on work that CASMI has done on areas such as open innovation,
new adaptive models of development, and the spread and adoption of new technology.
This work has been supported by the Wellcome Trust, the Oxford Biomedical Research
Centre, the UK government, the European Innovative Medicines Initiative, and Nesta.
I’m grateful for their support.
I have also learnt many useful lessons from colleagues at the Health Innovation
Network as they seek to spread valuable innovations across South London, including
Chris Streather, Tara Donnelly, Zoe Lelliott, Anna King and Guy Boersma. Precision
medicine is a major theme of this book, and I’ve gained insights from the team starting
up the UK Precision Medicine Catapult. I have the honour to chair both these innova-
tion enterprises; also Image Analysis, led by Olga Kubassova, a digital health company
viii PRefACe And ACKnOwledgeMentS
that is transforming the productivity of MRI. Other inspirational individuals further
afield include Aubrey de Grey, Mike Cope, Liz Parrish, and Avi Roy, all of whom are
dedicated to tackling the process and diseases of aging.
My particular thanks to Jack Scannell, Anoop Maini, and Avi Roy for reading and
commenting on all or part of the book.
I have also had the privilege of observing great innovators in the pharmaceutical
industry, in recent years as a board member of Celgene. They have convinced me that
speed, open- mindedness, and creativity are quite compatible with the challenging,
long- term business of bringing new drugs to patients.
In contrast to much popular supposition, regulators are largely supporting change
rather than resisting it. Among current and former regulators, I’d highlight Hans-G eorg
Eichler, Thomas Longgren, and Tomas Salmonson at the European Medicines Agency,
and Janet Woodcock and Mac Lumpkin (now with the Gates Foundation) at the US Food
& Drug Administration. Successive leaders at the Medicines and Healthcare Products
Regulatory Agency, including Alasdair Breckenridge, Kent Woods, Ian Hudson, Gordon
Duff, and Mike Rawlins have helped in pioneering thinking that is reflected in this book.
And many progressive life science industry leaders are partners in change.
I would like to particularly acknowledge the leadership of David Cooksey, whose
seminal 2006 report paved the way for many efforts in this area, including my own.
He has been an unstinting force behind creating a future in which life sciences make
greater practical impact on our lives, faster and more affordably.
This is a book about the pressing need for change, to make the medical innova-
tion process sustainable. It does not set out to explain in detail how things are done
today: I will refer readers to some useful sources for this background. Instead it explores
where and why current models are unsustainable and what we can do about it.
I have deliberately sought to look at the innovation process ‘from soup to nuts’—
from the initial idea of a product to the final benefit felt by the patient. I hope the book
will be read by experts in the various stages of this long process, but I ask their under-
standing where— in my aim of encompassing it all— I don’t do justice to the details that
they know better than me. The references I include are sometimes to specific original
research but also to reviews or even popular accounts where they seem accurate.
I have sought to take a sector-w ide perspective, including pharmaceuticals, diagnos-
tics, medical devices, and digital health. These are converging in this emerging era of
precision medicine. However, much of the ‘gap analysis’ and many of the prescriptions
for closing the gaps focus on pharmaceuticals. This is for two reasons: there has been
much more public discussion and debate about drugs than about the other life science
industry areas; and I believe there are genuinely more problems to be solved. Some of
the solutions, though, apply across the sector and I will try to draw this out.
Despite its analysis of ‘gaps in translation’, this is fundamentally an optimistic book.
I see many signs that the changes we need are starting to be made, but inertia is strong,
so clear sight and determination are critical. I hope to reinforce both.
As always, I am immensely grateful for the support and patience of my wife,
Michaela. I also greatly appreciate the enthusiastic sponsorship and guidance of Nic
Wilson, my editor at OUP, who urged me to include lots of instructive examples in the
book - it is the richer for it - and warmly thank her colleague James Cox for skilfully
piloting me through the publication process.
Endorsements
‘It is not easy to keep up with the rapid pace of biomedical innovation — nor changes in
society and markets. This book captures exactly where we are right now: an unprecedented
wave of innovation that arrives in health systems that are not prepared. And neither is
the business model of the pharmaceutical industry.’
Richard Bergström, Director, European Federation of Pharmaceutical
Industry and Associations, Belgium
‘This is a timely and provocative analysis. It is crucially important that researchers,
clinicians, industry, and regulators adapt effectively to the new era of stratified medicine.
This book is a welcome contribution to the debate.’
Professor Jeremy Farrar, Executive Director, The Wellcome Trust, UK
‘Richard Barker presents a provocative and thoughtful review of the health care
ecosystem, demonstrating time and again that communication and collaboration across
the stakeholder community are essential to realizing patient benefits from biomedical
innovation.’
Dr Barbara Lopez Kunz, Global Chief Executive,
Drug Information Association, Switzerland
‘Richard Barker, drawing on a quarter of a century of deep practical involvement in the
topic, has written an invaluable synthesis of the factors that account for the disappointing
rate of translation of science into affordable treatments to address unmet clinical need.
Crucial will be forging of transparent productive relationships between academia,
industry and an enlightened regulatory system. Essential reading for all those interested
in making the medical innovation process faster, more productive and sustainable.’
Professor Sir John Tooke, co- Chair of the Centre for the Advancement
of Sustainable Medical Innovation, UK, former Vice- Provost of University
College London, and former President of the UK Academy of Medical Sciences