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Gene Patents and Licensing Practices and Their Impact on Patient Access to Genetic Tests Report of the Secretary’s Advisory Committee on Genetics, Health, and Society April 2010 A pdf version of this report is available at http://oba.od.nih.gov/oba/SACGHS/reports/SACGHS_patents_report_2010.pdf Secretary’s Advisory Committee on Genetics, Health, and Society 6705 Rockledge Drive Suite 750, MSC 7985 Bethesda, MD 20892-7985 301-496-9838 (Phone) 301-496-9839 (Fax) http://www4.od.nih.gov/oba/sacghs.htm March 31, 2010 The Honorable Kathleen Sebelius Secretary of Health and Human Services 200 Independence Avenue, S.W. Washington, D.C. 20201 Dear Secretary Sebelius: In keeping with our mandate to provide advice on the broad range of policy issues raised by the development and use of genetic technologies as well as our charge to examine the impact of gene patents and licensing practices on access to genetic testing, the Secretary’s Advisory Committee on Genetics, Health, and Society (SACGHS) is providing to you its report Gene Patents and Licensing Practices and Their Impact on Patient Access to Genetic Tests. The report explores the effects of patents and licensing practices on basic genetic research, genetic test development, patient access to genetic tests, and genetic testing quality and offers advice on how to address harms and potential future problems that the Committee identified. It is based on evidence gathered through a literature review and original case studies of genetic testing for 10 clinical conditions as well as consultations with experts and a consideration of public perspectives. Based on its study, SACGHS found that patents on genetic discoveries do not appear to be necessary for either basic genetic research or the development of available genetic tests. The Committee also found that patents have been used to narrow or clear the market of existing tests, thereby limiting, rather than promoting availability of testing. SACGHS found that patients have been unable to obtain testing when a patent-protected sole provider does not accept particular payers, particularly state Medicaid insurance. SACGHS also found that when there is a patent- enforcing sole provider, patients cannot obtain independent second-opinion testing, and sample sharing as a means of ensuring the quality of testing is not possible. The substantial number of existing patents on genes and methods of diagnosis also pose a threat to the development of multiplex testing, parallel sequencing, and whole-genome sequencing, the areas of genetic testing with the greatest potential future benefits. The six recommendations contained in this report identify steps that the Department of Health and Human Services could take to help address existing harms and to help eliminate potential barriers to development of promising new testing technologies. The statutory changes the Committee has proposed are narrowly tailored to directly address the identified problems without altering patent rights for therapeutics. We appreciate the opportunity to serve you and the Department and hope the report will help you and the Department in achieving equitable access to health care and stimulating progress in health care technology. Sincerely, Steven Teutsch, M.D., M.P.H. SACGHS Chair SACGHS ROSTER Chair Steven Teutsch, M.D., M.P.H. (2010)* David Dale, M.D. (2012) Chief Science Officer Professor of Medicine Los Angeles County Department of Health University of Washington 313 North Figueroa Street 1959 NE Pacific Street Los Angeles, CA 90012 Seattle, WA 98195 Members Gwen Darien (2012) Executive Director Mara Aspinall, M.B.A. (2011) Samuel Waxman Cancer Research Foundation President and CEO 1249 Fifth Avenue, Suite 907 On-Q-ity New York, NY 10029 610 Lincoln Street Waltham, MA 02451 Rochelle Dreyfuss, M.S., J.D. (2011) Pauline Newman Professor of Law Sylvia Mann Au, M.S., CGC (2010)** New York University School of Law Hawaii State Genetics Coordinator 40 Washington Square South Genetics Program New York, NY 10012-1099 Hawaii Department of Health 741 Sunset Avenue Charis Eng, M.D., Ph.D. (2013) Honolulu, HI 96816 Chair and Founding Director Genomic Medical Institute Janice V. Bach, M.S. (2013) Cleveland Clinic Foundation State Genetics Coordinator and Manager 9500 Euclid Avenue Michigan Department of Community Health Cleveland, OH 44195 Genomic and Genetic Disorders Section 201 Townsend Street James P. Evans, M.D., Ph.D. (2010) Lansing, MI 48909 Professor of Genetics and Medicine Director of Clinical Cancer Genetics and the Paul Billings, M.D., Ph.D., FACP, FACMG Bryson Program in Human Genetics (2011) Departments of Medicine and Genetics Director and Chief Scientific Officer The University of North Carolina at Chapel Hill Genomic Medicine Institute Chapel Hill, NC 27599 El Camino Hospital Mountain View, CA 94040 Andrea Ferreira-Gonzalez, Ph.D. (2010) Professor of Pathology and Director of Molecular Diagnostics Laboratory Virginia Commonwealth University 403 North 13th Street Richmond, VA 23298 * The year indicates the year in which the member’s term expires. ** Term ended following the February 2010 SACGHS meeting. i Julio Licinio, M.D. (2010)** Director The John Curtin School of Medical Research The Australian National University Canberra, ACT 2601 Acton, Australia Barbara Burns McGrath, R.N., Ph.D. (2010) Research Associate Professor School of Nursing University of Washington Box 357263 Seattle, WA 98195 Samuel Nussbaum, M.D. (2012) Executive Vice President Clinical Health Policy and Chief Medical Officer Wellpoint 120 Monument Circle Indianapolis, IN 46204 Charmaine D. M. Royal, Ph.D. (2012) Associate Research Professor Institute for Genome Sciences and Policy Duke University Box 91009 450 Research Drive Durham, NC 27708 Sheila Walcoff, J.D (2012) Partner McDermott, Will & Emery, LLP 600 13th Street, NW, Suite 1200 Washington, DC 20005 Marc S. Williams, M.D., FAAP, FACMG (2010) Director Intermountain Healthcare Clinical Genetics Institute 324 10th Avenue Salt Lake City, UT 84103 Paul Wise, M.D., M.P.H. (2011) Richard E. Behrman Professor of Child Health and Society CHP/PCOR Stanford University 117 Encina Commons Stanford, CA 94305 * The year indicates the year in which the member’s term expires. ** Term ended following the February 2010 SACGHS meeting. ii Ex Officio Members Muin J. Khoury, M.D., Ph.D. Director Department of Commerce Office of Public Health Genomics Centers for Disease Control and Prevention Michael Amos, Ph.D. 1825 Century Center Boulevard Scientific Advisor Atlanta, GA 30345 Chemical Science and Technology Laboratory National Institute of Standards and Technology Jeffrey Roche, M.D. 100 Bureau Drive Medical Officer Gaithersburg, MD 20899 Office of Clinical Standards and Quality Centers for Medicare & Medicaid Services Department of Defense MS S3-02-01 7500 Security Boulevard LTC Adam Kanis, MC, USA Baltimore, MD 21244 Department of Pediatrics Tripler Army Medical Center Alberto Gutierrez, Ph.D. MCHK-PE Deputy Director 1 Jarrett White Road Office of In Vitro Diagnostic Device Evaluation Tripler AMC (Honolulu), HI 96859 and Safety Center for Devices and Radiological Health Department of Energy Food and Drug Administration 2098 Gaither Road Daniel Drell, Ph.D. Rockville, MD 20850 Biologist Life Sciences Division Denise Geolot, Ph.D., R.N. Office of Biological and Environmental Director Research Center for Quality 1000 Independence Avenue, SW Health Resources and Services Administration Washington, DC 20585 5700 Fishers Lane Rockville, MD 20857 Department of Health and Human Services Eric Green, M.D., Ph.D. Naomi Goldstein, Ph.D. Director Director National Human Genome Research Institute Office of Planning, Research, and Evaluation MSC 2152 Administration for Children and Families 31 Center Drive 370 L’Enfant Promenade, SW Bethesda, MD 20892 Washington, DC 20447 Robinsue Frohboese, J.D., Ph.D. Gurvaneet Randhawa, M.D., M.P.H Principal Deputy Director Medical Officer Office for Civil Rights Center for Outcomes and Evidence (COE) 200 Independence Avenue, SW Agency for Healthcare Research & Quality Washington, DC 20201 540 Gaither Road Rockville, MD 20850 Michael A. Carome, M.D. Associate Director for Regulatory Affairs Office for Human Research Protections 1101 Wootton Parkway, Suite 200 Rockville, MD 20852 iii Department of Labor Phyllis Borzi, J.D., M.A. Assistant Secretary of Labor Employee Benefits Security Administration 200 Constitution Avenue, NW, Suite S-2524 Washington, DC 20210 Department of Veterans Affairs Ellen Fox, M.D. Director National Center for Ethics in Health Care 810 Vermont Avenue, NW Washington, DC 20420 Equal Employment Opportunity Commission Stuart Ishimaru, J.D. Acting Chair 1801 L Street, NW Washington, DC 20507 Federal Trade Commission Sarah Botha, J.D. Attorney Division of Advertising Practices Bureau of Consumer Protection 600 Pennsylvania Avenue, NW Washington, D.C. 20580 iv ACKNOWLEDGMENTS The Committee wishes to thank the members of the SACGHS Task Force on Gene Patents and Licensing Practices, which served from October 2006 to September 2009, for their assistance in the development of this report. The Task Force was chaired by James Evans and included the following SACGHS members: Mara Aspinall, who served on it before becoming a Committee member and then continued as a Member, Rochelle Dreyfuss, and Andrea Ferreira-Gonzalez. The Task Force also included former SACGHS members Chira Chen, a patient representative; Joseph Telfair, a public health and consumer advocacy expert; Sylvia Au, a state genetics program coordinator; Debra
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  • Nr EC Aandoening Orphacode Patiëntenorganisatie Vragenlijst

    Nr EC Aandoening Orphacode Patiëntenorganisatie Vragenlijst

    Nr EC Aandoening Orphacode Patiëntenorganisatie Vragenlijst P PNR ingediend? G-24-12 ABeta amyloidosis, Dutch type ORPHA:100006 Vereniging HCHWA-D Nee P 14 G-8-7 Tubular duplication of the esophagus ORPHA:100048 Vereniging voor Ouderen en Kinderen met Slokdarmafsluiting (VOKS) Ja P 124 G-17-10 Neurogenic thoracic outlet syndrome ORPHA:100073 RSI-vereniging Nee P 305 G-11-43 Laryngeal neuroendocrine tumor ORPHA:100083 St NET-Groep Ja P 62 G-11-43 Middle ear neuroendocrine tumor ORPHA:100084 St NET-Groep Ja P 62 G-11-1 Thyroid tumor ORPHA:100087 Schildklier Organisatie NL (SON) Ja P 60 G-11-13 Thyroid tumor ORPHA:100087 Schildklier Organisatie NL (SON) Ja P 60 G-11-35 Thyroid Tumor ORPHA:100087 Schildklier Organisatie NL (SON) Ja P 60 G-11-13 Thyroid carcinoma ORPHA:100088 Schildklier Organisatie NL (SON) Ja P 60 G-11-35 Thyroid carcinoma ORPHA:100088 Schildklier Organisatie NL (SON) Ja P 60 G-3-11 Thyroid carcinoma ORPHA:100088 Schildklier Organisatie NL (SON) Ja P 60 G-3-17 Thyroid carcinoma ORPHA:100088 Schildklier Organisatie NL (SON) Ja P 60 G-3-13 Adrenal/paraganglial tumor ORPHA:100091 Nlse Vereniging voor patiënten met Paragangliomen (NVPG) Ja P 29 G-3-13 Adrenal/paraganglial tumor ORPHA:100091 Bijniervereniging (NVACP) Nee P 64 G-3-2 Adrenal/paraganglial tumor ORPHA:100091 Nlse Vereniging voor Patiënten met Paragangliomen (NVPG) Ja P 29 G-3-2 Adrenal/paraganglial tumor ORPHA:100091 Bijniervereniging (NVACP) Nee P 64 G-11-29 Gastroenteropancreatic neuroendocrine neoplasm ORPHA:100092 St NET-Groep Ja P 62 G-11-27 Thymic tumor ORPHA:100100