orphan drug production

Summary

Summary: Production of drugs or biologicals which are unlikely to be manufactured by private industry unless special incentives are provided by others.

Top Publications

  1. ncbi Endovasc establishes orphan drug test
    Irene M Walczak
    Diabetes Technol Ther 4:740-1. 2002
  2. ncbi Unresolved issues in contraceptive health policy
    Natalie D Reaves
    Economics Department, Rowan University, Glassboro, New Jersey 08028, USA
    Health Care Women Int 23:854-60. 2002
  3. ncbi Orphan drugs and orphan tests in the USA
    Jess G Thoene
    Hayward Genetics Center, Tulane University School of Medicine, New Orleans, LA 70112, USA
    Community Genet 7:169-72. 2004
  4. ncbi Clinical research for rare disease: opportunities, challenges, and solutions
    Robert C Griggs
    Department of Neurology, University of Rochester, 1351 Mt Hope Avenue, Suite 203, Rochester, NY 14620, USA
    Mol Genet Metab 96:20-6. 2009
  5. ncbi Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years
    M Miles Braun
    Office of Orphan Products Development, US Food and Drug Administration, Silverspring, Maryland 20993, USA
    Nat Rev Drug Discov 9:519-22. 2010
  6. ncbi Rare diseases, orphan drugs and their regulation: questions and misconceptions
    Erik Tambuyzer
    Genzyme, Ikaroslaan 53, B 1930, Zaventem, Belgium
    Nat Rev Drug Discov 9:921-9. 2010
  7. ncbi Potential interactions of the Orphan Drug Act and pharmacogenomics: a flood of orphan drugs and abuses?
    David Loughnot
    Am J Law Med 31:365-80. 2005
  8. ncbi Does orphan drug legislation really answer the needs of patients?
    Marlene E Haffner
    Office of Orphan Products Development, Food and Drug Administration, Amgen, Washington, DC, USA
    Lancet 371:2041-4. 2008
  9. ncbi Expanding global research and development for neglected diseases
    David J Winters
    The Global Fund to Fight AIDS, Tuberculosis and Malaria, Geneva, Switzerland
    Bull World Health Organ 84:414-6. 2006
  10. ncbi Pharmaceutical innovation in the 21st century: new drug approvals in the first decade, 2000-2009
    K I Kaitin
    Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA
    Clin Pharmacol Ther 89:183-8. 2011

Research Grants

  1. Dichlorphenamide vs Acetazolamide for Periodic Paralysis
    Robert C Griggs; Fiscal Year: 2010
  2. Microarray analysis of gene expresion in T. cruzi
    Rick Tarleton; Fiscal Year: 2006
  3. IMMUNOREGULATION IN EXPERIMENTAL CHAGAS DISEASE
    Rick Tarleton; Fiscal Year: 2006
  4. Dichlorphenamide vs Acetazolamide for Periodic Paralysis
    ROBERT GRIGGS; Fiscal Year: 2009
  5. Dichlorphenamide vs Acetazolamide for Periodic Paralysis
    ROBERT GRIGGS; Fiscal Year: 2007
  6. CD8+ T cell and Trypanosoma cruzi infection
    Rick Tarleton; Fiscal Year: 2007
  7. Plan forTrial to find Optimum Steroid Regimen in Duchenne Muscular Dystrophy
    ROBERT GRIGGS; Fiscal Year: 2006
  8. IMMUNOREGULATION IN EXPERIMENTAL CHAGAS DISEASE
    Rick Tarleton; Fiscal Year: 2007
  9. Nervous System Channelopathies: Pathogenesis & Treatment
    ROBERT GRIGGS; Fiscal Year: 2007
  10. EXPERIMENTAL THERAPEUTICS IN NEUROLOGICAL DISEASE
    ROBERT GRIGGS; Fiscal Year: 2007

Detail Information

Publications166 found, 100 shown here

  1. ncbi Endovasc establishes orphan drug test
    Irene M Walczak
    Diabetes Technol Ther 4:740-1. 2002
  2. ncbi Unresolved issues in contraceptive health policy
    Natalie D Reaves
    Economics Department, Rowan University, Glassboro, New Jersey 08028, USA
    Health Care Women Int 23:854-60. 2002
    ..S. federal legislation known as the Food and Drug Administration (FDA) defense could provide the economic incentives needed to spark the second contraceptive revolution...
  3. ncbi Orphan drugs and orphan tests in the USA
    Jess G Thoene
    Hayward Genetics Center, Tulane University School of Medicine, New Orleans, LA 70112, USA
    Community Genet 7:169-72. 2004
  4. ncbi Clinical research for rare disease: opportunities, challenges, and solutions
    Robert C Griggs
    Department of Neurology, University of Rochester, 1351 Mt Hope Avenue, Suite 203, Rochester, NY 14620, USA
    Mol Genet Metab 96:20-6. 2009
    ....
  5. ncbi Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years
    M Miles Braun
    Office of Orphan Products Development, US Food and Drug Administration, Silverspring, Maryland 20993, USA
    Nat Rev Drug Discov 9:519-22. 2010
    ..The implications of such findings for future development and marketing of therapies for rare diseases are discussed...
  6. ncbi Rare diseases, orphan drugs and their regulation: questions and misconceptions
    Erik Tambuyzer
    Genzyme, Ikaroslaan 53, B 1930, Zaventem, Belgium
    Nat Rev Drug Discov 9:921-9. 2010
    ..This article provides an industry perspective on some of the common questions and misconceptions related to orphan drug development and its regulation, with the aim of facilitating future progress in the field...
  7. ncbi Potential interactions of the Orphan Drug Act and pharmacogenomics: a flood of orphan drugs and abuses?
    David Loughnot
    Am J Law Med 31:365-80. 2005
  8. ncbi Does orphan drug legislation really answer the needs of patients?
    Marlene E Haffner
    Office of Orphan Products Development, Food and Drug Administration, Amgen, Washington, DC, USA
    Lancet 371:2041-4. 2008
  9. ncbi Expanding global research and development for neglected diseases
    David J Winters
    The Global Fund to Fight AIDS, Tuberculosis and Malaria, Geneva, Switzerland
    Bull World Health Organ 84:414-6. 2006
  10. ncbi Pharmaceutical innovation in the 21st century: new drug approvals in the first decade, 2000-2009
    K I Kaitin
    Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA
    Clin Pharmacol Ther 89:183-8. 2011
    ..1 and 6.9 years, respectively). The results provide the underpinnings of a fundamental shift in the structure of the research-based industry...
  11. ncbi Assessing the economic challenges posed by orphan drugs
    Michael F Drummond
    Centre for Health Economics, University of York, Heslington, York, UK
    Int J Technol Assess Health Care 23:36-42. 2007
    ....
  12. ncbi Translation of rare disease research into orphan drug development: disease matters
    Harald E Heemstra
    Utrecht Institute for Pharmaceutical Sciences, Utrecht University, The Netherlands
    Drug Discov Today 14:1166-73. 2009
    ..Consequently, additional incentives should focus on stimulating the specific needs of rare disease research at disease class level...
  13. ncbi Drugs for exceptionally rare diseases: do they deserve special status for funding?
    D A Hughes
    Centre for Economics and Policy in Health, Institute of Medical and Social Care Research, University of Wales, Bangor, UK
    QJM 98:829-36. 2005
    ..Mechanisms are discussed for creating a policy that would reduce geographical inequalities in provision across Europe...
  14. ncbi Why rare diseases are an important medical and social issue
    Arrigo Schieppati
    Clinical Research Centre for Rare Disease Aldo e Cele Dacc, Mario Negri Institute for Pharmacological Research, Bergamo, Italy
    Lancet 371:2039-41. 2008
  15. ncbi Predictors of orphan drug approval in the European Union
    Harald E Heemstra
    Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, P O Box 80 082, 3508 TB, Utrecht, The Netherlands
    Eur J Clin Pharmacol 64:545-52. 2008
    ..Recent literature discusses factors that may influence the development of new orphan medicinal products in the EU. This study aims to identify predictors for successful marketing authorisation of potential orphan drugs in the EU...
  16. ncbi The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?
    Olivier Wellman-Labadie
    Division of Dermatology, Department of Medicine, University of British Columbia, 835 West 10th Ave, Vancouver, BC, Canada
    Health Policy 95:216-28. 2010
    ..This study investigates issues associated with the United States Orphan Drug Act...
  17. ncbi Is orphan drug status beneficial to tropical disease control? Comparison of the American and future European orphan drug acts
    P Trouiller
    Centre Hospitalier Universitaire de Grenoble, France
    Trop Med Int Health 4:412-20. 1999
    ..However, some provisions of the European text may be relevant to tropical diseases, admitting the need for a more specific rule for evaluations of this kind of drug and recognizing the existence of 'diseases of exception'...
  18. ncbi Safety-related regulatory actions for orphan drugs in the US and EU: a cohort study
    Harald E Heemstra
    Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, 3508 TB Utrecht, The Netherlands
    Drug Saf 33:127-37. 2010
    ..Several studies have been conducted on safety-related regulatory actions for drugs, but none of these have specifically focused on orphan drugs...
  19. ncbi A comparative study of European rare disease and orphan drug markets
    Alain Denis
    Yellow Window Management Consultants, Antwerp, Belgium
    Health Policy 97:173-9. 2010
    ..This article aims to compare regulatory aspects of rare disease and orphan drug markets in Belgium, France, Italy, the Netherlands, Sweden and the United Kingdom...
  20. ncbi Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy
    Joe T R Clarke
    Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, Ont
    CMAJ 174:189-90. 2006
  21. ncbi A new model to evaluate the long-term cost effectiveness of orphan and highly specialised drugs following listing on the Australian Pharmaceutical Benefits Scheme: the Bosentan Patient Registry
    Aj Owen
    Centre for Clinical Research Excellence in Therapeutics, Department of Epidemiology and Preventive Medicine, Monash University, Melbourne, Australia
    J Med Econ 11:235-43. 2008
    ..Risk-sharing schemes between drug sponsor and government may provide an acceptable method of balancing the uncertainty of longer-term cost effectiveness with the public demand for equitable and timely access to new drugs...
  22. ncbi Exploring emerging technologies using metaphors--a study of orphan drugs and pharmacogenomics
    Wouter Boon
    Department of Innovation Studies, Copernicus Institute for Sustainable Development and Innovation, Utrecht University, Heidelberglaan 2, 3584 CS Utrecht, The Netherlands
    Soc Sci Med 66:1915-27. 2008
    ....
  23. ncbi The orphan drug backlash
    Thomas Maeder
    Sci Am 288:80-7. 2003
  24. ncbi Choosing the right incentive strategy for research and development in neglected diseases
    Stephen M Maurer
    Goldman School of Public Health, University of California, Berkeley, CA 94720 7320, USA
    Bull World Health Organ 84:376-81. 2006
    ..The efficiency of pay-as-you-go methods depends on sponsors' willingness to withdraw funding from failed drug discovery programmes...
  25. ncbi Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67
    Arna S Desser
    Department of Health Management and Health Economics, University of Oslo, PO Box 1089, Blindern, N 0317 Oslo, Norway
    BMJ 341:c4715. 2010
    ..To determine whether a general societal preference for prioritising treatment of rare diseases over common ones exists and could provide a justification for accepting higher cost effectiveness thresholds for orphan drugs...
  26. ncbi Orphan drug legislation: lessons for neglected tropical diseases
    Stefano Villa
    Centre for Research on Health and Social Care Management CERGAS, Universita Bocconi, Milan, Italy
    Int J Health Plann Manage 24:27-42. 2009
    ..Second, small-sized companies, which have played a successful role in the development of orphan drugs for rare diseases, may also represent a good business strategy for the case of tropical diseases...
  27. ncbi Incorporating a rapid-impact package for neglected tropical diseases with programs for HIV/AIDS, tuberculosis, and malaria
    Peter J Hotez
    Department of Microbiology, Immunology, and Tropical Medicine, The George Washington University, Washington, District of Columbia, USA
    PLoS Med 3:e102. 2006
  28. ncbi Adopting orphan drugs--two dozen years of treating rare diseases
    Marlene E Haffner
    Office of Orphan Products Development at the Food and Drug Administration, Rockville, MD, USA
    N Engl J Med 354:445-7. 2006
  29. ncbi A journey of hope: lessons learned from studies on rare diseases and orphan drugs
    M Wästfelt
    Strategy and Development Office, Karolinska Institutet, Stockholm, Sweden
    J Intern Med 260:1-10. 2006
    ..These lessons, along with the crucial importance of cooperation between academic institutions, pharmaceutical companies, patient advocacy groups and society in the elucidation of rare diseases, are highlighted in the present review...
  30. ncbi Orphan drugs revisited
    C McCabe
    Health Economics and Decision Science, School of Health and Related Research, Regent Court, 30 Regent Street, Sheffield S1 4DA
    QJM 99:341-5; discussion 350-1. 2006
  31. ncbi Adopting an orphan
    Andrea Rinaldi
    EMBO Rep 6:507-10. 2005
  32. ncbi Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products
    James W Dear
    Clinical Pharmacology Unit, Centre for Cardiovascular Science, University of Edinburgh, Queen s Medical Research Institute, Edinburgh, UK
    Br J Clin Pharmacol 62:264-71. 2006
    ..In this article we review OMPs and the incentives for their development and discuss the challenges presented by funding these treatments...
  33. ncbi Assessing the economic challenges posed by orphan drugs: a comment on Drummond et al
    Christopher McCabe
    Int J Technol Assess Health Care 23:397-401; author reply 401-4. 2007
  34. ncbi The US orphan drug programme 1983-1995
    S R Shulman
    Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA
    Pharmacoeconomics 12:312-26. 1997
    ....
  35. ncbi Orphan drugs and the NHS: should we value rarity?
    Christopher McCabe
    White Rose Initiative in Health Technology Assessment, Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Sheffield S1 4DA
    BMJ 331:1016-9. 2005
  36. ncbi Patent sense
    Paul Herrling
    Corporate Research, Novartis International, Basel, Switzerland
    Nature 449:174-5. 2007
  37. ncbi Mission possible
    Andrew L Hopkins
    Pfizer Global Research and Development, Sandwich, UK
    Nature 449:166-9. 2007
  38. ncbi Rare essentials: drugs for rare diseases as essential medicines
    Pieter Stolk
    Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, 3508 TB Utrecht, Netherlands
    Bull World Health Organ 84:745-51. 2006
    ..In this paper we propose selection criteria for an Orphan Medicines Model List that could form a departure point for future work towards an extensive WHO Orphan Medicines Programme...
  39. ncbi Market incentives and pharmaceutical innovation
    Wesley Yin
    University of Chicago, 1155 E 60th Street, Chicago, IL 60637, United States
    J Health Econ 27:1060-77. 2008
    ..Tax credits can stimulate R & D; yet because they leave revenue margins unaffected, tax credits appear to have a more limited impact on private innovation in markets with smaller revenue potential...
  40. ncbi Two decades of orphan product development
    Marlene E Haffner
    Office of Orphan Products Development, US Food and Drug Administration, Room 15A08, 5600 Fishers Lane, Rockville, Maryland 20857, USA
    Nat Rev Drug Discov 1:821-5. 2002
    ....
  41. ncbi Orphan Drug Act passes quarter-century milestone in fight against rare diseases
    Greg Lavine
    Am J Health Syst Pharm 65:1205, 1210. 2008
  42. ncbi [Hope for patients with rare diseases--"orphan" drugs]
    M Kuzelova
    Katedra farmakologie a toxikologie FaF UK, Bratislava, SR
    Cas Lek Cesk 145:296-300. 2006
    ..The real availability of "Orphan" medicinal products in the particular EU member states is analysed...
  43. ncbi Improving access to medicines in poor countries: the role of universities
    Dave A Chokshi
    University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA
    PLoS Med 3:e136. 2006
  44. ncbi Clinical trials of orphan medicines
    Brendan M Buckley
    European Centre for Clinical Trials in Rare Diseases, University College Cork, Cork, Ireland
    Lancet 371:2051-5. 2008
  45. ncbi [European incentives for orphan medicinal products]
    H Enzmann
    Bundesinstitut fur Arzneimittel und Medizinprodukte, Bonn, BRD
    Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz 51:500-8. 2008
    ..This article describes the procedure of orphan drug designation and highlights problems regarding coverage of treatment costs and increasingly individualized medicine...
  46. ncbi The quantity and quality of worldwide new drug introductions, 1982-2003
    Henry G Grabowski
    Department of Economics, Duke University, Durham, North Carolina, USA
    Health Aff (Millwood) 25:452-60. 2006
    ..S. firms overtook their European counterparts in innovative performance or the introduction of first-in-class, biotech, and orphan products. The United States also became the leading market for first launch...
  47. ncbi Developing drugs for developing countries
    David B Ridley
    Fuqua School of Business, Duke University, Durham, North Carolina, USA
    Health Aff (Millwood) 25:313-24. 2006
    ..In a well-functioning market, the voucher would speed access to highly valued treatments. Thus, the voucher could benefit consumers in both developing and developed countries at relatively low cost to the taxpayer...
  48. ncbi FDA program could boost treatments for neglected diseases
    Kate Traynor
    Am J Health Syst Pharm 65:1595-6. 2008
  49. ncbi Orphan economics: the downside of supplyside pharmacology
    Kurt Samson
    Ann Neurol 64:A13-6. 2008
  50. ncbi Access to information supporting availability of medicines for patients suffering from rare diseases looking for possible treatments: the EuOrphan Service
    Donatas Stakisaitis
    Mykolas Romeris University, Ateities 20, Vilnius, Lithuania
    Medicina (Kaunas) 43:441-6. 2007
    ..In this perspective, EuOrphan could become a very valuable tool for globalizing the information about the availability of treatment (authorized or under development) of orphan patients...
  51. ncbi Promoting, improving and accelerating the drug development and approval processes
    A I Graul
    Drug News Perspect 21:36-43. 2008
    ..This article reviews the three programs and provides a capsule view of new developments in these vital areas during the year 2007...
  52. ncbi Law and research could add up to profitable niche drugs
    Burkhard Haefner
    Nature 446:856. 2007
  53. ncbi Promoting, improving and accelerating the drug development and approval processes
    A I Graul
    Drug News Perspect 20:45-55. 2007
    ..This article reviews the three programs and provides a capsule view of new developments in these vital areas during the year 2006...
  54. ncbi Neglected diseases: progress in drug development
    Simon L Croft
    Curr Opin Investig Drugs 8:103-4. 2007
  55. ncbi Proposal ties financial incentives to development of drugs for neglected diseases
    Donna Young
    Am J Health Syst Pharm 63:694, 696. 2006
  56. ncbi Biologics for inflammatory bowel disease: drug approval and monitoring in the United States
    William J Tremaine
    Division of Gastroenterology and Hepatology, Mayo Clinic College of Medicine, 200 1st Street, SW, Rochester, MN 55905, USA
    Gastroenterol Clin North Am 35:735-41. 2006
    ..FDA regulation of a new drug or biologic can be divided into three stages: the commercial IND, the NDA, and postmarketing (phase 4) studies. It is unclear if generic versions of biologics can be approved within the current legislation...
  57. ncbi Marlene Haffner
    Catherine Goodman
    Nat Chem Biol 2:657. 2006
  58. ncbi The Minor Use and Minor Species Animal Health Act: past, present, and future
    Carol J Haley
    Pfizer Inc, New York, NY, USA
    Food Drug Law J 61:13-43. 2006
  59. ncbi Vouchers for FDA priority reviews
    Ian D Spatz
    Health Aff (Millwood) 25:1184; author reply 1184-5. 2006
  60. ncbi Should the patent system for new medicines be abolished?
    J A DiMasi
    Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA
    Clin Pharmacol Ther 82:488-90. 2007
  61. ncbi Orphan drugs
    Gary D Novack
    Ocul Surf 6:52-3. 2008
  62. ncbi [Orphan drugs--medications for patients with rare diseases]
    Desirée Thielke
    Laegehuset, NIVA, Amtssygehuset i Gentofte, Dermatologisk afdeling, og Swedish Orphan International AB
    Ugeskr Laeger 168:2236-8. 2006
    ..S. Congress passed the Orphan Drug Act, creating financial incentives for manufacturers. Since 2000 the EU has had a similar regulation, resulting in more than 20 ODs being granted marketing authorisation in the EU...
  63. ncbi EU to review rare disease drugs market exclusivity
    Cormac Sheridan
    Nat Biotechnol 22:1061. 2004
  64. ncbi Adoption agent
    Carmen Peota
    Minn Med 88:8-9. 2005
  65. ncbi Gleevec for the treatment of chronic myelogenous leukemia: US. Food and Drug Administration regulatory mechanisms, accelerated approval, and orphan drug status
    Martin H Cohen
    Division of Oncology Drug Products HFD 150, Center for Drug Evaluation and Research and Office of Orphan Products Development, Office of the Commissioner, Food and Drug Administration, Rockville, Maryland 20857, USA
    Oncologist 7:390-2. 2002
    ..The purpose of this report is to summarize FDA regulatory mechanisms, i.e., accelerated approval and orphan drug regulations, that have permitted patients to receive this drug as rapidly as possible...
  66. ncbi An overview of drug development in the United States and current challenges
    Sharon Wyatt Moore
    Medical and Regulatory Affairs, Clinical Trial Management Services, Inc, Bristol, TN 37620, USA
    South Med J 96:1244-55; quiz 1256. 2003
    ..Although the ultimate goal is to bring safer and more effective medical products to patients in a timely manner, multiple challenges face those who participate in drug development...
  67. ncbi Understanding orphan drug regulations: an EU and U.S. comparative analysis
    Aurelie Grienenberger
    Regulatory Affairs and Drug Development, Voisin Consulting, Paris, France
    J Biolaw Bus 7:58-61. 2004
    ..An update of the orphan drugs designations is provided as well as the upcoming challenges that orphan regulations will meet with the development of pharmaco-genomics and personalized medicines...
  68. ncbi [Orphan drugs: some legal, ethical and economics aspects]
    J Y Pabst
    , , 74, route du Rhin, B.P. 24, 67401 Illkirch-Graffenstaden Cedex, France
    Rev Epidemiol Sante Publique 49:387-96. 2001
    ..This document implements the dispositions available to pharmaceutical firms inciting them to invest in the development of orphan drugs...
  69. ncbi Developing treatments for inborn errors: incentives available to the clinician
    Marlene E Haffner
    FDA Office of Orphan Products Development, Food and Drug Administration, Rockville, MD 20857, USA
    Mol Genet Metab 81:S63-6. 2004
    ..Today, international support for rare disease research is providing stimulus and motivation to overcome the financial barriers and encourage development of treatment for very rare diseases throughout the world...
  70. ncbi Medicines for children licensed by the European Agency for the Evaluation of Medicinal Products
    A Ceci
    Department of Pharmacology and Human Physiology, University of Bari, Italy
    Eur J Clin Pharmacol 58:495-500. 2002
    ....
  71. ncbi U.S. Food and Drug Administration drug approval summaries: imatinib mesylate, mesna tablets, and zoledronic acid
    Martin H Cohen
    Division of Oncology Drug Products, Center for Drug Evaluation and Research, U S Food and Drug Administration, Rockville, Maryland 20857, USA
    Oncologist 7:393-400. 2002
    ..Information provided includes rationale for drug development, study design, efficacy and safety results, and pertinent literature references...
  72. ncbi Enzyme replacement therapy for Fabry disease: lessons from two alpha-galactosidase A orphan products and one FDA approval
    Robert J Desnick
    Department of Human Genetics, Box 1498, Mount Sinai School of Medicine, Fifth Avenue at 100th St, New York, NY 10029, USA
    Expert Opin Biol Ther 4:1167-76. 2004
    ..The process also highlighted important issues in the evaluation of drugs to treat life-threatening genetic diseases for which the pathological basis is well-defined...
  73. ncbi Increasing the use of orphan drugs in clinical practice
    Sally-Anne Hulton
    Birmingham Children's Hospital NHS Trust, Birmingham
    Hosp Med 65:400-3. 2004
    ..Over the next few years, orphan drugs will probably be increasingly adopted into everyday clinical practice, partly because of genomic stratification...
  74. ncbi EMEA approves OGS drug rejected by FDA
    Peter Mitchell
    Nat Biotechnol 20:858-9. 2002
  75. ncbi Orphan anticancer drug rejected
    Adrian Burton
    Lancet Oncol 4:524. 2003
  76. ncbi PPPs succeeding in tackling neglected diseases
    Simon Frantz
    Nat Rev Drug Discov 4:796-7. 2005
  77. ncbi New products highlight ambiguity of orphan drug law
    Brian Reid
    Nat Biotechnol 21:6-7. 2003
  78. ncbi Economic and regulatory considerations in pharmacogenomics for drug licensing and healthcare
    Jai Shah
    Department of Social Policy, London School of Economics and Political Science, and is now at the Canadian Institutes of Health Research, 410 Laurier Avenue West (11th Floor, Ottawa, Ontario K1A 0W9, Canada
    Nat Biotechnol 21:747-53. 2003
  79. ncbi From club drug to orphan drug: sodium oxybate (Xyrem) for the treatment of cataplexy
    David E Fuller
    Orphan Medical, Inc, Minnetonka, Minnesota 55305, USA
    Pharmacotherapy 23:1205-9. 2003
    ..This program has been successful in satisfying the needs of patients and physicians while ensuring responsible distribution of the drug...
  80. ncbi A breakthrough in R&D for neglected diseases: new ways to get the drugs we need
    Mary Moran
    London School of Economics and Political Science, London, United Kingdom
    PLoS Med 2:e302. 2005
  81. ncbi The European rare diseases therapeutic initiative
    Alain Fischer
    Department of Pediatric Immunology, Necker University Hospital, Paris, France
    PLoS Med 2:e243. 2005
  82. ncbi The Food and Drug Administration's Office of Orphan Products Development: incentives, grants, and special designations speed therapies for orphan diseases
    Marlene E Haffner
    The FDA Office of Orphan Products Development and Public Health Service, USA
    Retina 25:S89-S90. 2005
  83. ncbi Diagnostic challenges and new therapies for rare lysosomal storage diseases
    Barbara Hesselgrave
    Case Manager 14:48-52. 2003
  84. ncbi This Dutchman is flying
    Noshua Watson
    Fortune 147:89-90. 2003
  85. ncbi Congress, the FDA, and the fair development of new medications for children
    Ross E McKinney
    Department of Pediatrics, Division of Infectious Diseases, Duke University School of Medicine, Durham, NC 27710, USA
    Pediatrics 112:669-70. 2003
  86. ncbi Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia
    Carl Rudolf Blankart
    German Research Center for Environmental Health, Munich, Germany
    Pharmacoeconomics 29:63-82. 2011
    ....
  87. ncbi Adverse drug reactions to anthelmintics
    Haleh Bagheri
    Department of Pharmacology, Midi Pyrenees Center of Pharmacovigilance, Faculty of Medicine, Toulouse University Hospital, Toulouse, France
    Ann Pharmacother 38:383-8. 2004
    ..Anthelmintics are commonly used in tropical areas, but are also prescribed in Western countries with other climates. However, pharmacoepidemiologic studies of these drugs are rare...
  88. ncbi Anecdotal therapies
    L E Millikan
    Department of Dermatology, Tulane University Medical Center, New Orleans, Louisiana 70112, USA
    Adv Exp Med Biol 455:407-11. 1999
    ..The formulation of a low concentration of zinc pyrithione seemed unusual, and this truly was an anecdotal approach, using a homeopathic dosage of a commonly used p..
  89. ncbi Gaucher disease: clinical profile and therapeutic developments
    Timothy M Cox
    Department of Medicine, University of Cambridge, Cambridge, UK
    Biologics 4:299-313. 2010
    ....
  90. ncbi Development of antidotes: problems and strategies
    Ladislaus Szinicz
    Bundeswehr Institute of Pharmacology and Toxicology, Neuherbergstr 11, 80937 Munich, Germany
    Toxicology 233:23-30. 2007
    ....
  91. ncbi A review on clinical management and pharmacological therapy on hereditary haemorrhagic telangiectasia (HHT)
    Roberto Zarrabeitia
    Hospital de Sierrallana, Reference Hospital for HHT in Spain, Torrelavega, Santander, Spain
    Curr Vasc Pharmacol 8:473-81. 2010
    ..In this context, the use of hormones, immunosuppresants and anti-angiogenic agents are under preclinical study in our laboratory...
  92. ncbi A decade of safety-related regulatory action in the Netherlands: a retrospective analysis of direct healthcare professional communications from 1999 to 2009
    Peter G M Mol
    University Medical Center Groningen, Groningen, The Netherlands
    Drug Saf 33:463-74. 2010
    ..This challenge has led to a life-cycle approach but, so far, few data are available on post-approval safety issues requiring regulatory action...
  93. ncbi Prader-Willi syndrome in Taiwan
    Hsiang Yu Lin
    Department of Pediatrics, Mackay Memorial Hospital, and Mackay Medicine, Nursing and Management College, Taipei, Taiwan
    Pediatr Int 49:375-9. 2007
    ..Prader-Willi syndrome (PWS) is a congenital disorder caused by absent expression of paternal genes in 15q11-13 affecting multiple systems. The information concerning the clinical features of this genetic disorder is incomplete in Taiwan...
  94. ncbi Regulatory considerations for developing drugs for rare diseases: orphan designations and early phase clinical trials
    Anne R Pariser
    Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland 20993, USA
    Discov Med 11:367-75. 2011
    ....
  95. ncbi Access to orphan drugs despite poor quality of clinical evidence
    Alain G Dupont
    Commission for Reimbursement of Medicines, Vrije Universiteit Brussel, Brussels, Belgium
    Br J Clin Pharmacol 71:488-96. 2011
    ..We analysed the Belgian reimbursement decisions of orphan drugs as compared with those of innovative drugs for more common but equally severe diseases, with special emphasis on the quality of clinical evidence...
  96. ncbi Drug development for orphan diseases in the context of personalized medicine
    George J Brewer
    Department of Human Genetics, University of Michigan Medical School, Ann Arbor, Mich 48109 0534, USA
    Transl Res 154:314-22. 2009
    ....
  97. ncbi Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders
    Carla E M Hollak
    Department of Internal Medicine, Division of Endocrinology and Metabolism, Academic Medical Center, Amsterdam, The Netherlands
    Orphanet J Rare Dis 6:16. 2011
    ..We argue that disease specific (rather than drug specific) registries, supervised by independent clinicians are urgently needed for the best long-term evaluation of treatments of these rare diseases...
  98. ncbi 'Orphan drugs' in cardiology: nadolol and quinidine
    Giuseppe Inama
    Division of Cardiology, Cardiocerebrovascular Department, Ospedale Maggiore, Crema, Italy
    J Cardiovasc Med (Hagerstown) 11:143-4. 2010
    ..We hope that scientific societies can influence healthcare and pharmaceutical institutions, in order to restore the availability of two cardiovascular drugs that are extremely important in the care of arrhythmic patients...
  99. ncbi Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer
    Aaron S Kesselheim
    Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women s Hospital, 1620 Tremont St, Ste 3030, Boston, MA 02120, USA
    JAMA 305:2320-6. 2011
    ..Orphan products constitute most new drug approvals in oncology, but safety and efficacy questions have emerged about some of these agents...
  100. ncbi Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013
    Alain Denis
    Yellow Window Management Consultants, Antwerp, Belgium
    J Med Econ 13:295-301. 2010
    ..This article aims to calculate the impact of orphan drugs on the Belgian drug budget in 2008 and to forecast its impact over the following 5 years...
  101. ncbi Rare diseases and orphan drugs
    Domenica Taruscio
    Centro Nazionale Malattie Rare, Istituto Superiore di Sanita, Rome, Italy
    Ann Ist Super Sanita 47:83-93. 2011
    ..This paper illustrates mainly the European initiatives and will discuss the problematic and controversial aspects surrounding orphan drugs. Finally, activities and measures adopted in Italy are presented...

Research Grants72

  1. Dichlorphenamide vs Acetazolamide for Periodic Paralysis
    Robert C Griggs; Fiscal Year: 2010
    ..abstract_text> ..
  2. Microarray analysis of gene expresion in T. cruzi
    Rick Tarleton; Fiscal Year: 2006
    ..Completion of this study will provide valuable data on the expression of genes in T. cruzi, especially the many that are "hypothetical," and provide a starting point for elucidating the functions of these genes. ..
  3. IMMUNOREGULATION IN EXPERIMENTAL CHAGAS DISEASE
    Rick Tarleton; Fiscal Year: 2006
    ..cruzi infection, including the extent of polyclonal activation in the infection and the degree to which an over-aggressive immune response contributes to disease. ..
  4. Dichlorphenamide vs Acetazolamide for Periodic Paralysis
    ROBERT GRIGGS; Fiscal Year: 2009
    ..abstract_text> ..
  5. Dichlorphenamide vs Acetazolamide for Periodic Paralysis
    ROBERT GRIGGS; Fiscal Year: 2007
    ..abstract_text> ..
  6. CD8+ T cell and Trypanosoma cruzi infection
    Rick Tarleton; Fiscal Year: 2007
    ..cruzi will be determined and the biological significance of the effect of this antagonism of the generation and effector function of CD8+ T cells will be determined. ..
  7. Plan forTrial to find Optimum Steroid Regimen in Duchenne Muscular Dystrophy
    ROBERT GRIGGS; Fiscal Year: 2006
    ....
  8. IMMUNOREGULATION IN EXPERIMENTAL CHAGAS DISEASE
    Rick Tarleton; Fiscal Year: 2007
    ..The results of these studies also have strong parallels with human T. cruzi infection and implications for possible treatment of infection and disease in infected subjects. ..
  9. Nervous System Channelopathies: Pathogenesis & Treatment
    ROBERT GRIGGS; Fiscal Year: 2007
    ..They may also offer a window for understanding common disorders likely to be caused by CNS channel mutations/dysfunction such as migraine and epilepsy. ..
  10. EXPERIMENTAL THERAPEUTICS IN NEUROLOGICAL DISEASE
    ROBERT GRIGGS; Fiscal Year: 2007
    ..Training facilities include: the General Clinical Research Center, Clinical Trials Coordination Centers, Biostatistics Department, Gorell Molecular Biology Laboratory, Center for Aging & Developmental Biology. ..
  11. PHASE III TRIAL OF TETRATHIOMOLYBDATE IN PRIMARY BILIAR*
    George Brewer; Fiscal Year: 2007
    ..Abstract Not Provided ..
  12. Novel treatment for muscle disease: Fueling the pipeline and finding the product
    ROBERT GRIGGS; Fiscal Year: 2006
    ....
  13. Dichlorphenamide vs Acetazolamide for Periodic Paralysis
    ROBERT GRIGGS; Fiscal Year: 2004
    ....
  14. IMMUNOREGULATION IN EXPERIMENTAL CHAGAS' DISEASE
    Rick Tarleton; Fiscal Year: 1993
    ....
  15. HOOKWORM LARVAL INFECTIVITY AND DEVELOPMENT
    Peter Hotez; Fiscal Year: 2002
    ..The pharmacologic manipulation of parasite-derived protein kinases may offer a new approach to chemotherapy by taking larvae out of arrest. ..
  16. TREATMENT OF EBV ASSOCIATED LYMPHOPROLIFERATIVE DISEASE
    Denis Moss; Fiscal Year: 2002
    ....
  17. CD8+ T CELLS AND TRYPANOSOMA CRUZI INFECTIONS
    Rick Tarleton; Fiscal Year: 2002
    ..The completion of this project could provide the information and the tools necessary to determine whether or not targets of the anti-T. cruzi CTL response would be useful as vaccines. ..
  18. North Carolina Collaborative PPRU Network
    Ross McKinney; Fiscal Year: 2004
    ..6) To train physicians and pharmacologists in clinical and developmental pharmacology: the centerpiece will be a 3-year pediatric pharmacology fellowship. Drs. Brouwer and Lindley (UNC) will lead this program. ..
  19. Novel Designs and Outcome Measures for Bench to Bedside Research on NMD
    ROBERT GRIGGS; Fiscal Year: 2007
    ..In this effort to provide new treatments for neuromuscular disease, this conference will help to further public health goals. ..
  20. Integrated Human Research Subjects Protection Project
    Ross McKinney; Fiscal Year: 2003
    ..abstract_text> ..
  21. Pathogenesis and treatment of the periodic paralyses
    ROBERT GRIGGS; Fiscal Year: 2004
    ....
  22. CD8+ T cell and Trypanosoma cruzi infection
    Rick Tarleton; Fiscal Year: 2009
    ..cruzi will be determined and the biological significance of the effect of this antagonism of the generation and effector function of CD8+ T cells will be determined. ..