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. pmc Estimating the budget impact of orphan medicines in Europe: 2010 - 2020
    Carina Schey
    GMAS, Chiswick Business Park, London, W4 5YA, UK
    Orphanet J Rare Dis 6:62. 2011
  2. pmc Pricing and reimbursement of orphan drugs: the need for more transparency
    Steven Simoens
    Research Centre for Pharmaceutical Care and Pharmaco economics, Katholieke Universiteit Leuven, O and N2 bus 521, Herestraat 49, 3000 Leuven, Belgium
    Orphanet J Rare Dis 6:42. 2011
  3. 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
  4. ncbi Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU
    Roberta Joppi
    Laboratory of Drug Regulatory Policies, Mario Negri Institute for Pharmacological Research, via Giuseppe La Masa, 19, 20156 Milan, Italy
    Eur J Clin Pharmacol 69:1009-24. 2013
  5. ncbi Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007
    Javier Orofino
    Cellerix, Madrid, Spain
    Appl Health Econ Health Policy 8:301-15. 2010
  6. pmc 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
  7. pmc The prevalence and cost of unapproved uses of top-selling orphan drugs
    Aaron S Kesselheim
    Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women s Hospital and Harvard Medical School, Boston, Massachusetts, United States of America
    PLoS ONE 7:e31894. 2012
  8. pmc 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
  9. 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
  10. pmc Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?
    Wills Hughes-Wilson
    EU Committee of Experts on Rare Diseases EUCERD, Global Public Policy and Government Relations, SOBI Swedish Orphan Biovitrum, Tomtebodvägen, 23A, Solna, Sweden
    Orphanet J Rare Dis 7:74. 2012

Detail Information

Publications190 found, 100 shown here

  1. pmc Estimating the budget impact of orphan medicines in Europe: 2010 - 2020
    Carina Schey
    GMAS, Chiswick Business Park, London, W4 5YA, UK
    Orphanet J Rare Dis 6:62. 2011
    ..The aim of this study was to predict the total cost of orphan medicines in Europe between 2010 and 2020 as a percentage of total European pharmaceutical expenditure...
  2. pmc Pricing and reimbursement of orphan drugs: the need for more transparency
    Steven Simoens
    Research Centre for Pharmaceutical Care and Pharmaco economics, Katholieke Universiteit Leuven, O and N2 bus 521, Herestraat 49, 3000 Leuven, Belgium
    Orphanet J Rare Dis 6:42. 2011
    ..Such an approach should be targeted at demonstrating the relative effectiveness, cost-effectiveness and economic viability of orphan drugs with a view to informing pricing and reimbursement decisions...
  3. 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...
  4. ncbi Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU
    Roberta Joppi
    Laboratory of Drug Regulatory Policies, Mario Negri Institute for Pharmacological Research, via Giuseppe La Masa, 19, 20156 Milan, Italy
    Eur J Clin Pharmacol 69:1009-24. 2013
    ..To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers and discuss possible reasons for the small number of products licensed...
  5. ncbi Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007
    Javier Orofino
    Cellerix, Madrid, Spain
    Appl Health Econ Health Policy 8:301-15. 2010
    ..The EU has passed legislation to encourage pharmaceutical companies to invest in research programmes into rare diseases, with the aim of developing new, safe and effective orphan drugs...
  6. pmc 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...
  7. pmc The prevalence and cost of unapproved uses of top-selling orphan drugs
    Aaron S Kesselheim
    Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women s Hospital and Harvard Medical School, Boston, Massachusetts, United States of America
    PLoS ONE 7:e31894. 2012
    ..However, some orphan drugs become top sellers for unclear reasons. We sought to evaluate the extent and cost of approved and unapproved uses of orphan drugs with the highest unit sales...
  8. pmc 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
    ....
  9. 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...
  10. pmc Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?
    Wills Hughes-Wilson
    EU Committee of Experts on Rare Diseases EUCERD, Global Public Policy and Government Relations, SOBI Swedish Orphan Biovitrum, Tomtebodvägen, 23A, Solna, Sweden
    Orphanet J Rare Dis 7:74. 2012
    ..The authors believe that this could, therefore, facilitate the approach for all stakeholders...
  11. 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...
  12. pmc 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
  13. ncbi Drugs for rare diseases: influence of orphan designation status on price
    Eline Picavet
    Research Centre for Pharmaceutical Care and Pharmaco economics, Faculty of Pharmaceutical Sciences, Katholieke Universiteit Leuven, Leuven, Belgium
    Appl Health Econ Health Policy 9:275-9. 2011
    ..In order to gain full insight into orphan drug pricing mechanisms, future research should focus on collecting information about the different factors influencing orphan drug pricing...
  14. doi 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
  15. ncbi When everyone is an orphan: against adopting a U.S.-styled orphan drug policy in Canada
    Matthew Herder
    Health Law Institute, Faculties of Medicine and Law, Dalhousie University, Halifax, Nova Scotia, Canada
    Account Res 20:227-69. 2013
    ....
  16. ncbi Orphan drug development: an economically viable strategy for biopharma R&D
    Kiran N Meekings
    Thomson Reuters Life Sciences Consulting, The Johnson Building, 77 Hatton Garden, London EC1N 8JS, UK
    Drug Discov Today 17:660-4. 2012
    ..The data support the targeting of rare diseases as an important component of a successful biopharma R&D strategy...
  17. ncbi Perspectives for improving the evaluation and access of therapies for rare lung diseases in Europe
    Maurizio Luisetti
    Department of Molecular Medicine, Section of Pneumology, University of Pavia, IRCCS San Matteo Hospital Foundation, Pavia, Italy
    Respir Med 106:759-68. 2012
    ....
  18. ncbi What is wrong with orphan drug policies?
    André Côté
    Faculté des sciences de l administration, Universite Laval, Quebec, QC, Canada
    Value Health 15:1185-91. 2012
    ....
  19. ncbi What is wrong with orphan drug policies? Suggestions for ways forward
    Panos Kanavos
    Department of Social Policy and LSE Health, London School of Economics, London, UK
    Value Health 15:1182-4. 2012
    ....
  20. pmc The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases
    Brigitta E Miyamoto
    Kakkis EveryLife Foundation For Rare Diseases, 77 Digital Drive, Suite 210, Novato, CA 94949, USA
    Orphanet J Rare Dis 6:49. 2011
    ..Many rare diseases have not utilized AA due to the difficulty in gaining acceptance of novel surrogate endpoints in untreated rare diseases...
  21. 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...
  22. 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...
  23. pmc Building local research and development capacity for the prevention and cure of neglected diseases: the case of India
    H E Kettler
    Office of Health Economics, London, England
    Bull World Health Organ 79:742-7. 2001
    ..Further studies are required on how Indian companies would respond to push and pull incentives originally designed to persuade multinational corporations to do more R&D on neglected diseases...
  24. pmc Pivotal studies of orphan drugs approved for neurological diseases
    Jun Mitsumoto
    College of Physicians and Surgeons, Columbia University, New York, NY, USA
    Ann Neurol 66:184-90. 2009
    ..To identify design elements of clinical trials leading to US Food and Drug Administration approval of drugs for neurological diseases with and without orphan indications...
  25. ncbi EU marketing authorization review of orphan and non-orphan drugs does not differ
    M Putzeist
    Utrecht Institute for Pharmaceutical Sciences, Division of Pharmacoepidemiology and Clinical Pharmacology, WHO Collaborating Centre for Pharmacoepidemiology and Pharmaceutical Policy Analysis, Utrecht University, The Netherlands Medicines Evaluation Board, Utrecht, The Netherlands Electronic address
    Drug Discov Today 18:1001-6. 2013
    ..Differences in deficits were found, but similarities in the way ODs and non-ODs were reviewed and marketing approval decisions were taken, underline that regulatory standards are equally high. ..
  26. pmc Systematic review of available evidence on 11 high-priced inpatient orphan drugs
    Tim A Kanters
    Institute for Medical Technology Assessment, Department of Health Policy and Management, Erasmus University Rotterdam, Burgemeester Oudlaan 50, PO Box 1738, 3000DR Rotterdam, The Netherlands
    Orphanet J Rare Dis 8:124. 2013
    ..This study systematically reviews the available evidence on clinical effectiveness, cost-effectiveness and budget impact for orphan drugs...
  27. ncbi How did uncommon disorders become 'rare diseases'? History of a boundary object
    Caroline Huyard
    Groupe de Sociologie Politique et Morale, EHESS, 10 Rue Monsieur le Prince, Paris, France
    Sociol Health Illn 31:463-77. 2009
    ..In the definition process of the category of rare diseases, the key actors were the patients and public bodies, not medical professionals or the pharmaceutical industry...
  28. doi Why rare diseases are an important medical and social issue
    Arrigo Schieppati
    Clinical Research Centre for Rare Disease Aldo e Cele Daccò, Mario Negri Institute for Pharmacological Research, Bergamo, Italy
    Lancet 371:2039-41. 2008
  29. ncbi Raising orphans: how clinical development programs of drugs for rare and common diseases are different
    M Orfali
    Specialty Care Business Unit, Pfizer, Cambridge, Massachusetts, USA
    Clin Pharmacol Ther 92:262-4. 2012
    ..We conclude that small studies of appropriate design can support US FDA approval of new medicines for rare diseases...
  30. ncbi Systematic review of comparative effectiveness data for oncology orphan drugs
    Mindy M Cheng
    Pharmaceutical Outcomes Research and Policy Program, University of Washington, Seattle, WA 98195 7630, USA
    Am J Manag Care 18:47-62. 2012
    ..To systematically assess clinical and economic evidence for oncology orphan drugs marketed in the United States and to highlight the challenges and opportunities for evidence development within this pharmaceutical category...
  31. ncbi Characteristics of orphan drug applications that fail to achieve marketing approval in the USA
    Harald E Heemstra
    Medicines Evaluations Board, PO Box 16229, 2500 BE Den Haag, The Netherlands
    Drug Discov Today 16:73-80. 2011
    ..Sponsors, therefore, should engage in dialogue with the FDA and thoughtfully design pivotal clinical trials in accordance with FDA guidance documents...
  32. 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
    ....
  33. pmc Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?
    Anne E M Brabers
    NIVEL, Netherlands Institute for Health Services Research, PO Box 1568, 3500 BN Utrecht, The Netherlands
    Orphanet J Rare Dis 6:59. 2011
    ..In the interest of rare disorder patients better understanding of the effect of the market exclusivity incentive on follow-on OMP development is warranted...
  34. 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
    ....
  35. 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...
  36. 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
    ....
  37. 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
    ....
  38. 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
    ....
  39. pmc 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...
  40. 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
  41. pmc 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
  42. pmc 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...
  43. ncbi Mission possible
    Andrew L Hopkins
    Pfizer Global Research and Development, Sandwich, UK
    Nature 449:166-9. 2007
  44. ncbi Patent sense
    Paul Herrling
    Corporate Research, Novartis International, Basel, Switzerland
    Nature 449:174-5. 2007
  45. pmc 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
  46. pmc 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...
  47. 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
  48. pmc 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...
  49. 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...
  50. 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...
  51. ncbi The orphan drug backlash
    Thomas Maeder
    Sci Am 288:80-7. 2003
  52. pmc 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...
  53. 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...
  54. 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
  55. pmc Adopting an orphan
    Andrea Rinaldi
    EMBO Rep 6:507-10. 2005
  56. pmc 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
  57. 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
  58. 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...
  59. 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...
  60. 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...
  61. 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...
  62. 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...
  63. 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
  64. 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...
  65. ncbi Neglected diseases: progress in drug development
    Simon L Croft
    Curr Opin Investig Drugs 8:103-4. 2007
  66. 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...
  67. 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
  68. 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...
  69. ncbi Law and research could add up to profitable niche drugs
    Burkhard Haefner
    Nature 446:856. 2007
  70. doi FDA program could boost treatments for neglected diseases
    Kate Traynor
    Am J Health Syst Pharm 65:1595-6. 2008
  71. 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
  72. 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...
  73. 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...
  74. doi Orphan Drug Act passes quarter-century milestone in fight against rare diseases
    Greg Lavine
    Am J Health Syst Pharm 65:1205, 1210. 2008
  75. doi Orphan economics: the downside of supplyside pharmacology
    Kurt Samson
    Ann Neurol 64:A13-6. 2008
  76. 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...
  77. ncbi Orphan drugs
    Gary D Novack
    Ocul Surf 6:52-3. 2008
  78. ncbi Marlene Haffner
    Catherine Goodman
    Nat Chem Biol 2:657. 2006
  79. ncbi PPPs succeeding in tackling neglected diseases
    Simon Frantz
    Nat Rev Drug Discov 4:796-7. 2005
  80. 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...
  81. ncbi New products highlight ambiguity of orphan drug law
    Brian Reid
    Nat Biotechnol 21:6-7. 2003
  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 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...
  84. ncbi This Dutchman is flying
    Noshua Watson
    Fortune 147:89-90. 2003
  85. 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...
  86. ncbi EU to review rare disease drugs market exclusivity
    Cormac Sheridan
    Nat Biotechnol 22:1061. 2004
  87. 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
  88. 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...
  89. ncbi Diagnostic challenges and new therapies for rare lysosomal storage diseases
    Barbara Hesselgrave
    Case Manager 14:48-52. 2003
  90. ncbi Orphan anticancer drug rejected
    Adrian Burton
    Lancet Oncol 4:524. 2003
  91. pmc 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
  92. pmc The European rare diseases therapeutic initiative
    Alain Fischer
    Department of Pediatric Immunology, Necker University Hospital, Paris, France
    PLoS Med 2:e243. 2005
  93. ncbi Adoption agent
    Carmen Peota
    Minn Med 88:8-9. 2005
  94. ncbi Proposal ties financial incentives to development of drugs for neglected diseases
    Donna Young
    Am J Health Syst Pharm 63:694, 696. 2006
  95. pmc 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
  96. 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...
  97. 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...
  98. ncbi EMEA approves OGS drug rejected by FDA
    Peter Mitchell
    Nat Biotechnol 20:858-9. 2002
  99. 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...
  100. 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
  101. ncbi Vouchers for FDA priority reviews
    Ian D Spatz
    Health Aff (Millwood) 25:1184; author reply 1184-5. 2006

Research Grants20

  1. Dichlorphenamide vs Acetazolamide for Periodic Paralysis
    Robert C Griggs; Fiscal Year: 2010
    ..abstract_text> ..
  2. Indo-US Research Conference on Rare Diseases and Orphan Drugs
    James C Cloyd; Fiscal Year: 2010
    ....
  3. Inhaled dopamine agonists for late stage Parkinsonism
    Patrik Munzar; Fiscal Year: 2005
    ..Eventual FDA approval of inhaled dopamine agonist product for treatment of motor fluctuations in late stage Parkinson's will substantially improve the treatment of this serious and common neurodegenerative disease. ..
  4. RADIOPHARMACEUTICALS: DIAGNOSIS AND TREATMENT OF CANCER
    Richard Wahl; Fiscal Year: 1990
    ..Through this series of mutually reinforcing projects supported by the core labs, the end result should be improved diagnosis and treatment of cancer...
  5. TOXICOLOGY OF IRON ORAL CHELATING COMPOUNDS
    Charles Tyson; Fiscal Year: 2000
    ..The drugs under development are considered to be orphan drugs. ..
  6. Experimental Therapeutics of Neuromuscular Disease
    ROBERT GRIGGS; Fiscal Year: 2008
    ..unreadable] [unreadable] [unreadable] [unreadable]..
  7. Drug Discovery Consortium for Chagas Disease
    Rick L Tarleton; Fiscal Year: 2010
    ..The focus of this proposal is the discovery and development of drugs to treat this infection. ..
  8. Plan forTrial to find Optimum Steroid Regimen in Duchenne Muscular Dystrophy
    ROBERT GRIGGS; Fiscal Year: 2006
    ....
  9. 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. ..
  10. 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. ..
  11. Novel treatment for muscle disease: Fueling the pipeline and finding the product
    ROBERT GRIGGS; Fiscal Year: 2006
    ..unreadable] [unreadable] [unreadable]..
  12. Vaccine Discovery for Chagas Disease
    Rick Tarleton; Fiscal Year: 2008
    ..Genes cloned in this process will be used to produce and test protective cocktails of genes in the vaccination studies described in Project 3...
  13. TREATMENT OF EBV ASSOCIATED LYMPHOPROLIFERATIVE DISEASE
    Denis Moss; Fiscal Year: 2002
    ....
  14. PHASE III TRIAL OF TETRATHIOMOLYBDATE IN PRIMARY BILIAR*
    George Brewer; Fiscal Year: 2007
    ..Abstract Not Provided ..
  15. 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. [unreadable] [unreadable] [unreadable]..
  16. Integrated Human Research Subjects Protection Project
    Ross McKinney; Fiscal Year: 2003
    ..abstract_text> ..
  17. Pathogenesis and treatment of the periodic paralyses
    ROBERT GRIGGS; Fiscal Year: 2004
    ....
  18. 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. ..
  19. Nervous System Channelopathies: Pathogenesis & Treatment
    ROBERT GRIGGS; Fiscal Year: 2005
    ..They may also offer a window for understanding common disorders likely to be caused by CNS channel mutations/dysfunction such as migraine and epilepsy. ..