Drug development can be time-consuming and expensive. Recent estimates suggest that, on average, it takes 10 years and at least $1 billion to bring a drug to market. Given the time and expense of developing drugs de novo, pharmaceutical companies have become increasingly interested in finding new uses for existing drugs—a process referred to as drug repurposing or repositioning.

Historically, drug repurposing has been largely an unintentional, serendipitous process that took place when a drug was found to have an offtarget effect or a previously unrecognized on-target effect that could be used for identifying a new indication. Perhaps the most recognizable example of such a successful repositioning effort is sildenafil. Originally developed as an anti-hypertensive, sildenafil, marketed as Viagra and under other trade names, has been repurposed for the treatment of erectile dysfunction and pulmonary arterial hypertension. Viagra generated more than $2 billion worldwide in 2012 and has recently been studied for the treatment of heart failure.

Given the widespread interest in drug repurposing, the Roundtable on Translating Genomic-Based Research for Health of the Institute of Medicine hosted a workshop on June 24, 2013, in Washington, DC, to assess the current landscape of drug repurposing activities in industry, academia, and government. Stakeholders, including government officials, pharmaceutical company representatives, academic researchers, regulators, funders, and patients, were invited to present their perspectives and to participate in workshop discussions. Drug Repurposing and Repositioning is the summary of that workshop. This report examines enabling tools and technology for drug repurposing; evaluates the business models and economic incentives for pursuing a repurposing approach; and discusses how genomic and genetic research could be positioned to better enable a drug repurposing paradigm.

Contents

• THE NATIONAL ACADEMIES
• PLANNING COMMITTEE
• ROUNDTABLE ON TRANSLATING GENOMIC-BASED RESEARCH FOR HEALTH
• Reviewers
• Acknowledgments
• Abbreviations and Acronyms
• 1. Introduction and Themes of the Workshop
  • WORKSHOP THEMES
• 2. The State of the Science
  • INDUSTRY REPURPOSING EFFORTS
  • RARE DISEASES
  • POTENTIAL OPPORTUNITIES FOR DRUG DISCOVERY
• 3. Enabling Tools and Technology
  • DISCOVERY USING PUBLICLY AVAILABLE DATA
  • ROLE OF BASIC SCIENCE FOR TRANSLATION
  • DATA MINING
  • REPURPOSING AT NCATS
  • PATIENT-REPORTED DATA
• 4. Value Propositions for Drug Repurposing
  • BUSINESS MODELS
  • REPURPOSING DRUGS CURRENTLY IN DEVELOPMENT
  • THALIDOMIDE: REPURPOSING A DRUG THAT WAS NOT SUCCESSFUL FOR ITS FIRST INDICATION
• 5. Policy Approaches and Legal Framework
  • OVERCOMING BARRIERS TO DRUG REPURPOSING AT NCATS
  • OVERCOMING BARRIERS TO DRUG REPURPOSING AT THE MRC
  • COMPOUND AVAILABILITY
  • LEGAL AND INTELLECTUAL PROPERTY ISSUES
• 6. Increasing the Efficiency and Success of Repurposing
  • THE ROLE OF GENOMICS
  • USE OF ELECTRONIC MEDICAL RECORDS
  • EXISTING BARRIERS
  • POSSIBLE NEXT STEPS
  • FUTURE REPURPOSING EFFORTS
• References
• APPENDIXES
  • A Workshop Agenda
  • B Speaker Biographical Sketches
  • C Statement of Task
  • D Registered Attendees

Rapporteurs: Sarah H. Beachy, Samuel G. Johnson, Steve Olson, and Adam C. Berger.

This project was supported by contracts between the National Academy of Sciences and the American Academy of Nursing (unnumbered contract); American College of Medical Genetics and Genomics (unnumbered contract); American Heart Association (unnumbered contract); American Medical Association (unnumbered contract); American Society of Human Genetics (unnumbered contract); Association for Molecular Pathology (unnumbered contract); Blue Cross and Blue Shield Association (unnumbered contract); College of American Pathologists (unnumbered contract); Department of the Air Force (Contract No. FA7014-0-P-0072); Department of Veterans Affairs (Contract No. VA248-P-1528); Eli Lilly and Company (unnumbered contract); Genetic Alliance (unnumbered contract); Health Resources and Services Administration (Contract No. HHSH250201100119P and Contract No. HHSH25034017T); International Society for Cardiovascular Translational Research (unnumbered contract); Johnson & Johnson (unnumbered contract); the Kaiser Permanente Program Offices Community Benefit II at the East Bay Community Foundation (Contract No. 20121257); Life Technologies (unnumbered contract); National Cancer Institute (Contract No. HHSN263201200074I, TO#5); National Coalition for Health Professional Education in Genetics (unnumbered contract); National Heart, Lung, and Blood Institute (Contract No. N01-OD-4-2139, TO#275); National Human Genome Research Institute (Contract No. N01-OD-4-2139, TO#264 and Contract No. HHSN263201200074I, TO#5); National Institute of Mental Health (Contract No. N01-OD-4-2139, TO#275 and Contract No. HHSN263201200074I, TO#5); National Institute of Nursing Research (Contract No. HHSN263201200074I, TO#5); National Institute on Aging (Contract No. N01-OD-4-2139, TO#275 and Contract No. HHSN263201200074I, TO#5); National Society of Genetic Counselors (unnumbered contract); Northrop Grumman Health IT (unnumbered contract); Office of Rare Diseases Research (Contract No. N01-OD-4-2139, TO#275); Pfizer Inc. (unnumbered contract); and PhRMA (unnumbered contract). The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the activity.

Suggested citation:

IOM (Institute of Medicine). 2014. Drug repurposing and repositioning: Workshop summary. Washington, DC: The National Academies Press.

NOTICE: The workshop that is the subject of this workshop summary was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine.