The Future of Drug Discovery

The Future of Drug Discovery

Who Decides Which Diseases to Treat?

1st Edition - May 18, 2013

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  • Authors: Tamas Bartfai, Graham Lees
  • Paperback ISBN: 9780124071803
  • eBook ISBN: 9780124095199

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The Future of Drug Discovery: Who decides which diseases to treat? provides a timely and detailed look at the efforts of the pharmaceutical industry and how they relate, or should relate, to societal needs. The authors posit that as a result of increasing risk aversion and accelerated savings in research and development, the industry is not developing drugs for increasingly prevalent diseases, such as Alzheimer’s disease, untreatable pain, antibiotics and more. This book carefully exposes the gap between the medicines and therapies we need and the current business path. By analyzing the situation and discussing prospects for the next decade, the The Future of Drug Discovery is a timely book for all those who care about the development needs for drugs for disease.

Key Features

  • Provides an in-depth, broad perspective on the crisis in drug industry
  • Exposes the disconnect between what society needs and what the drug companies are working on
  • Analyses and projects over 10 years into the future
  • Explains what it means for scientists and society
  • Determines what is needed to be done to make sure that the industry responds to society's needs, remains commercially attractive and answers the question as to who decides which diseases to treat


Biomedical researchers involved in drug discovery in industry and academic settings, physicians, patient advocacy groups, health economists and regulatory officials

Table of Contents

  • Expert Reviews for The Future of Drug Discovery: Who Decides Which Diseases to Treat?



    Foreword by Graeme Bilbe PhD

    Foreword by James H Eberwine PhD

    Foreword by Eduard Enrico Holdener MD

    Chapter 00: Introduction

    What medicines you will need & why you might not ever have them

    Aging is the major risk factor for disease

    Painful truth & AD

    More government action required

    The decisiveness & divisiveness of market access

    Government can change the future

    Not all is doom & gloom

    What can society do?

    Chapter 01. Why there will be new drugs despite the ongoing “crisis” of drug development in Big Pharma

    Pharmaceutical industry in crisis & consolidation

    Why is there a strong belief that new future medicines will come?

    Safe drugs in new indications: one does not always need new drugs for new therapies

    Preventive drugs: large, long, expensive, convincing trials

    Age as a risk factor & keeping costs in check

    Newly orphaned diseases: more government needed

    Chapter 02. The need for medicines grows

    Small molecules & biologicals

    The aging population

    Rare or “orphan” diseases

    Drug development: adding biologicals

    Treatment of MS

    Treatment of RA

    Recombinant proteins as treatments

    Future recombinant proteins as treatments

    Pharma, governments, & nongovernmental organizations

    Chapter 03. Medicines are becoming better: some of the breakthrough medicines of the past decades

    Significant progress since WW II

    The great beginning & the feared end of extremely profitable drug discovery

    Drug discovery recovery, phase 1

    Drug discovery arrested?

    Meta-analysis matters: new twist on old information

    New uses for safe drugs

    Off-label use in other indications

    Life cycle extension by repositioning

    Inter- & intra-company competition to replace approved drugs in financially attractive indications

    Pricing policies

    Greater emphasis on long-term drug safety & benefit–risk ratio

    Moving the goalposts

    Weight loss drugs kept waiting

    Evidence-based medicine requires large & long public-funded trials

    Gains of & from EBM

    Numbers of annually approved drugs

    Chapter 04. Which diseases do we want to treat?

    How many diseases are there?

    How pharma companies have selected which diseases to treat

    The funding & direction of biomedical research

    The scientific basis of disease treatment

    New avenues of research & the emergence of biosimilars

    Generic drug makers


    “Firsts in class,” “me–toos,” & “best in class”: marketers’ dreams

    Rescuing successful phase 2 candidates from oblivion

    Una¢¢ounted u$e$ of $afe drug$


    Creating indications in the clinic or at the FDA?

    Off-label use definitely enables approved drugs to treat additional diseases, but is it a desired tactic?

    How can society affect the selection of indications by Big Pharma & by biotech?

    Chapter 05. Therapeutic areas: Strategically important diseases of the future

    Diverging views of society & industry

    The human & economic cost of major diseases

    AD: the most loomingly threatening disease

    Early diagnostic imperatives

    Translational research

    Obesity & type 2 diabetes

    Surgical versus drug interventions

    Neuropathic pain


    Effective governmental & societal intervention

    Chapter 06. Blockbuster proprietary drugs versus generic drugs

    What happened to the blockbuster drug pipeline?

    Where can the industry find new blockbusters?

    More science here, please

    The new blockbusters

    Double-digit growth becomes an unreasonable expectation

    Big Pharma attaches a generic arm

    Generics attach an R&D arm

    Will all drugs become generic?

    Regulating biosimilars

    The future of R&D

    There are still huge, unmet medical needs

    Chapter 07. Why is pharma a special industry?

    Drug discovery is the most regulated human activity

    History & mergers

    Basic & translational research

    Economic & social contribution

    Governmental nonintervention & intervention

    Crisis, what crisis? Tail wagging the dog?

    Academia–industry collaborations

    Gaps in Big Pharma’s research revisited

    Plugging the gaps in Big Pharma’s research

    Regulation & politics

    Clinical trials

    Chapter 08. Diagnosing toward personalized medicine

    “Without diagnostics there can be no treatment; without available treatment, we need not diagnose”

    Growth of diagnostics

    Why is the diagnostics industry becoming so important for the pharmaceutical industry?


    Targeted cancer therapies

    Companion diagnostics

    Tougher regulation of diagnostics

    What makes a diagnostic test good?

    Chapter 09. Personalized medicine

    Personalized medicine basics


    Emerging personalized medicine

    Adverse drug reactions may rank as high as the fifth leading cause of death

    The “hair-dryer” & “toaster” drugs

    The future of personalized medicine

    Knowledge management

    What does this mean for drug treatments?

    Knowledge mining & learning algorithms

    Chapter 10. How much can drugs cost?

    Will the sums spent on medical care and new medicines grow?

    The “number to treat”

    The price of the most expensive drugs

    Eroding the high-priced drugs

    Quality adjusted life year costs: the QALY

    How is the QALY calculated in the UK?

    What about cost effectiveness?

    Chapter 11. Modeling drug discovery until 2025

    Early twenty-first century realities

    Long-term safety (gets) in the face of short-term pharma

    The path away from innovation

    Evidence-based medicine

    Risk of failure leads to risk aversion

    Filling the strategic vacuum

    VCs: less capital leads to less venture

    Society-funded safety net

    The history of the future

    How will we then make new drugs in these areas of medical need abandoned by pharma?

    What would be the best way to dispense governmental support?

    Which diseases are international, urgent priorities?

    Step 1: intensify research

    Step 2: new results, new targets, new biotechs

    Step 3: government-backed initiatives

    Outsourcing & what parts of the drug discovery process are scalable?

    Real really beats virtual

    Manufacturing in the near future

    Clinical trials in the near future

    Positive outcome of the near future

    Negative outcome of the near future

    Perfect is not perfect

    Room, & time, for optimism?

    Faster, longer regulatory practices

    Chapter 12. Drug development models between 2010 & 2025

    Why the pharma industry is where it is

    Emerging economies & pharma

    Intellectual property & quality

    Switzerland’s opportunity

    Drug development is moving, but not completely

    But where does this leave us when we ask who will develop new drugs?

    Medical need motivates

    Past & future trends

    Rescue of phase 2 drugs

    The fourth idea for a big incentive: rewrite patent guidelines

    A “Biotech Projects Stock Market”

    Safeguarding the future of safe medicines


Product details

  • No. of pages: 376
  • Language: English
  • Copyright: © Academic Press 2013
  • Published: May 18, 2013
  • Imprint: Academic Press
  • Paperback ISBN: 9780124071803
  • eBook ISBN: 9780124095199

About the Authors

Tamas Bartfai

Tamas Bartfai was a student of mathematics, physics, and chemistry before translating his skills into biochemistry, pharmacology and neuroscience. Trained in Stockholm University, Yale University, and The Rockefeller University, he is presently a professor at The Scripps Research Institute, the University Oxford, and the University of Pennsylvania, and an expert in medicinal chemistry and the neurological sciences. He has been working in the development of new medicines and vaccines for many years as a former Sr.VP of Hoffmann La Roche, and long-term consultant at Astra, Novartis and, presently, Pfizer. Eight of the drugs Dr. Bartfai developed are in clinical use and three are in trials. He has trained and collaborated with many scientists throughout his scientific work on the topics of fever, neuropeptides, and prostaglandins, while publishing over 400 articles in over 80 journals.

Dr. Bartfai has held many prestigious academic positions. He is a member of Academia Europae and the Hungarian Academy of Sciences, a fellow of AAAS for pioneering work on neuropeptides, and a member of the Royal Swedish Academy of Sciences, which awards the Nobel Prizes in Chemistry and Physics. He was professor of the Karolinska Institute, which awards the Nobel Prize in Medicine or Physiology. He has been awarded a number of prestigious prizes including Eötvös Medal for mathematics in 1966, Budapest, Hungary; Royal Swedish Academy’s Svedberg Prize for biochemistry in 1985 and Ericsson Prize in 1996; and the Ellison Medical Foundation Senior Scholar Award 2002.

Affiliations and Expertise

PhD, The Scripps Research Institute, La Jolla, CA Harold L. Dorris Neurological Research Center and Scripps Research Institute, La Jolla, California, USA

Graham Lees

Graham V Lees acquired his BA, MA and PhD degrees at the University of Cambridge. His postdoctoral work on the biophysics of ion channels was followed by a lengthy career in scientific, technical and medical publishing with Elsevier (Amsterdam), Raven Press (New York), Academic Press (San Diego & London) and TheScientificWorld (San Diego, Boynton Beach, Newbury & Helsinki). His scientific writing has been actively increasing, including contributions to the textbook Fundamental Neuroscience; co-editing with Edward G. Jones and Lorne Mendell, and contributing to Neuroscience to Neurological Recovery for the Society for Neuroscience (SfN); and co-authoring The Future of Drug Discovery: Who Decides Which Diseases to Treat and Drug Discovery: From Bedside to Wall Street with Tamas Bartfai. He is interested in politics and social aspects of drug discovery. He has a profound ability to translate complex processes and ideas into more simple English that can be widely read.

The authors’ previous book Drug Discovery: from Bedside to Wall Street, Elsevier/Academic Press, 2006, has been published in Japanese (Chem-Bio Informatics Society) and Mandarin (Science Press). Their later book’s Japanese and Mandarin editions are in preparation.

Affiliations and Expertise

PhD, Publishing Consultant, Corpus Alienum Oy, Finland

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