2011REP ORTU.S. INTELLECTTO THE PREUALSI DENTPROPERTY ON ENFORCEMENT COORDINATOR PROPELLING INNOVATION IN COVER ANNUAL T IRTLEEPORT HERE ON DRUGINTELLECT DISCOVERYUAL, DEVELO PROPERTYPMENT , ANEDNFORCEMENT EVALUATION Executive Office of the President President’s Council of Advisors on Science and Technology SEPTEMBER 2012 2011REP ORTU.S. INTELLECT TO THE PREUALSI PRODENTPERTY ON ENFORCEMENT COORDINATOR PROPELLING INNOVATION IN ANNUAL REPORT ON DRUGINTELLECT DISCOVERYUAL, DEVELO PROPERTYPMENT , ANEDNFORCEMENT EVALUATION Executive Office of the President President’s Council of Advisors on Science and Technology SEPTEMBER 2012 About the President’s Council of Advisors on Science and Technology The President’s Council of Advisors on Science and Technology (PCAST) is an advisory group of the nation’s leading scientists and engineers, appointed by the President to augment the science and tech­ nology advice available to him from inside the White House and from cabinet departments and other Federal agencies. PCAST is consulted about and often makes policy recommendations concerning the full range of issues where understandings from the domains of science, technology, and innovation bear potentially on the policy choices before the President. For more information about PCAST, see www.whitehouse.gov/ostp /pcast. The President’s Council of Advisors on Science and Technology Co-Chairs John P. Holdren Eric Lander Assistant to the President for President Science and Technology Broad Institute of Harvard and MIT Director, Office of Science and Technology Policy Vice Chairs William Press Maxine Savitz Raymer Professor, Computer Science and Vice President Integrative Biology National Academy of Engineering University of Texas at Austin Members Rosina Bierbaum S. James Gates, Jr. Professor, Natural Resources and John S. Toll Professor of Physics Environmental Policy Director, Center for String and Particle Theory School of Natural Resources and Environment University of Maryland, College Park and School of Public Health Mark Gorenberg University of Michigan Managing Director Christine Cassel Hummer Winblad Venture Partners President and CEO Shirley Ann Jackson American Board of Internal Medicine President Christopher Chyba Rensselaer Polytechnic Institute Professor, Astrophysical Sciences and International Affairs Richard C. Levin Director, Program on Science and Global President Security Yale University Princeton University ★ i ★ REPORT TO THE PRESIDENT ON PROPELLING INNOVATION IN DRUG DISCOVERY, DEVELOPMENT, AND EVALUATION Chad Mirkin Barbara Schaal Rathmann Professor, Chemistry, Materials Mary­Dell Chilton Distinguished Professor of Science and Engineering, Chemical and Biology Biological Engineering and Medicine Washington University, St. Louis Director, International Institute for Vice President, National Academy of Sciences Nanotechnology Eric Schmidt Northwestern University Executive Chairman Mario Molina Google, Inc. Professor, Chemistry and Biochemistry Daniel Schrag University of California, San Diego Sturgis Hooper Professor of Geology Professor, Center for Atmospheric Sciences Professor, Environmental Science and Engineering at the Scripps Institution of Oceanography Director, Harvard University Center for Director, Mario Molina Center for Energy and Environment Environment, Mexico City Harvard University Ernest J. Moniz David E. Shaw Cecil and Ida Green Professor of Physics and Chief Scientist, D.E. Shaw Research Engineering Systems Senior Research Fellow, Center for Computational Director, MIT’s Energy Initiative Biology and Bioinformatics Massachusetts Institute of Technology Columbia University Craig Mundie Ahmed Zewail Chief Research and Strategy Officer Linus Pauling Professor of Chemistry and Physics Microsoft Corporation Director, Physical Biology Center Ed Penhoet California Institute of Technology Director, Alta Partners Professor Emeritus, Biochemistry and Public Health University of California, Berkeley Staff Deborah Stine Danielle Evers Executive Director AAAS Science and Technology Policy Fellow Amber Hartman Scholz Assistant Executive Director ★ ii ★ EXECUTIVE OFFICE OF THE PRESIDENT PRESIDENT’S COUNCIL OF ADVISORS ON SCIENCE AND TECHNOLOGY WASHINGTON, D.C. 20502 President Barack Obama EXECUTIVE OFFICE OF THE PRESIDENT The WhiteEXECUTIVE House OFFICE OF THEEXECUTIVE PRESIDENT OFFICE OF THE PRESIDENT PRESIDENT’S COUNCIL OF ADVISORS ON SCIENCE AND TECHNOLOGY PRESIDENT’SWashington, COUNCIL DC 20502 OF ADVISORS ON SCIENCE AND TECHNOLOGY WASHINGTON,PRESIDENT’S D.C. 20502 COUNCIL OF ADVISORS ON SCIENCE AND TECHNOLOGY WASHINGTON, D.C. 20502 WASHINGTON, D.C. 20502 March 25, 2010 March 25, 2010 March 25, 2010 Dear Mr. President, President Barack Obama President Barack Obama President Barack Obama The White House We are pleased to send you this Report To The President On Propelling Innovation In Drug Discovery, The White House The White House Washington, DC 20502 Washington, DC 20502Development, andWashington, Evaluation DC, prepared 20502 by the President’s Council of Advisors on Science and Technology (PCAST). This report responds to your request for recommendations on this topic. Dear Mr. President, Dear Mr. President, Dear Mr. President, The past quarter-century has seen tremendous progress in biomedical research, leading to an increas- We are pleased to send you this “Report to the President and Congress on the Third Assessment of the We are pleased to send you this “ReportWe areto thepleased President to send and you Congress this “Report on the to Third the President Assessment and of Congress the on the Third Assessment of the National Nanotechnologying understanding Initiative,” preparedof cancer, by heart the President’sdisease, diabetes, Council and of otherAdvisors devastating on Science diseases. and The Nation has led National Nanotechnology Initiative,”National prepared Nanotechnology by the President’s Initiative,” Council preparedof Advisors by theon SciencePresident’s and Council of Advisors on Science and Technology (PCAST).the worldThis report in such reflects progress, a PCAST due in decision significant to advise part to you wise on investments this topic and by fulfills the Federal PCAST’s Government in Technology (PCAST). This reportst reflectsTechnolo a PCASTgy (PCAST). decision This to reportadvise reflectsyou on athis PCAST topic anddecision fulfills to PCAST’sadvise you on this topic and fulfills PCAST’s responsibilities under the 21st Century Nanotechnology Research and Development Act (Public Law 108-153) responsibilities underbasic the 21 biomedical Centuryresponsibilities research.Nanotechnology These under breakthroughs Research the 21st andCentury areDevelopment beginning Nanotechnology Actto pay (Public off Research in Law terms 108 and of- 153)Developmentnew therapies Actfor (Public Law 108-153) and Executive Order 13349 to provide periodic updates to Congress. and Executive OrderAmerican 13349 to providepatients.and periodic Executive updates Order to 13349 Congress. to provide periodic updates to Congress. To provide a solid scientific basis for our recommendations, the Council assembled a PCAST Working Group To provide a solid scientificStill, the basis pace for ofTo newour provide recommendations, therapeutic a solid developmentscientific the Councilbasis has for not assembled our kept recommendations, up witha PCAST the explosion Working the Council inGroup scientific assembled knowledge. a PCAST Working Group of three PCAST members and 12 non-governmental members with broad expertise in nanotechnology. The of three PCAST members and 12 nonof- governmentalthree PCAST members withand 12 broad non -expertisegovernmental in nanotechnology. members with broadThe expertise in nanotechnology. The Working Group addressedThe number the requirements of novel drugs of Publichas remained Law 108 constant-153, with for additionalseveral decades, efforts evenaimed as in R&D four budgets have sub- Working Group addressed the requirementsWorking of Group Public addressed Law 108 -the153, requirements with additional of Public efforts Law aimed 108 in-153, four with additional efforts aimed in four areas: NNI programstantially management; increased. the outputs As you of know, nanotechnology; this situation environment, poses an increasing health, and challenge safety research;for ensuring the creation of areas: NNI program management; theareas: outputs NNI of program nanotechnology; management; environment, the outputs health, of nanotechnology; and safety research; environment, health, and safety research; and the vision for NNI for the next ten years. The Working Group’s deliberations were informed by and the vision for NNIinnovative for the next therapies tenand years. the for vision patients.The Working for NNIIt is also forGroup’s thecausing next deliberations tenserious years. stresses Thewere Working informedthroughout Group’s by the biomedical deliberations ecosystem. were informed by discussions with 37 government officials, industry leaders, and technical experts from a wide range of fields discussions with 37 government officials,discussions industry with leaders, 37 government and technical officials, experts industry from aleaders, wide range and technicalof fields experts from a wide range of fields involving nanotechnology. involving nanotechnology.To identify and involvidevelopng constructive nanotechnology. solutions to this challenge, PCAST engaged a wide range of stake- holders in discussions over the past twelve months—including senior leaders from the biopharmaceutical The