Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years

Braun, M. Miles, Farag-El-Massah, Sheiren, Xu, Kui, Coté, Timothy R.·2010·Nature Reviews Drug Discovery
DOI 10.1038/nrd3160

Cited by (149)

Items 101–149 of 149

  1. Rare Diseases and Orphan Drugs: A comprehensive approach of strategic perspectives
    Jan-Cédric Hansen · 2012 · Journal of Communications In Healthcare
  2. Orphan drug approvals in Europe since 2001: an analysis by indication
    Peter Norman · 2013 · Expert Opinion on Orphan Drugs
  3. Trends, lag and characteristics of rare disease drug approval in the USA and China, 1983–2022
    Shaohong Wang, Xin Liu, Yuzhen Zou, Yan Tang, Wei Zuo, Rong Jiang, Junmei Shang, Xin Tian, Q P Liu, Tingting Xu et al. · 2025 · Orphanet Journal of Rare Diseases
  4. Research in Rare Disease: From Genomics to Proteomics
    Jessica Lacoste · 2018 · Assay and Drug Development Technologies
  5. Regulatory environment for novel therapeutic development in mitochondrial diseases
    Michio Hirano, Andrés Berardo, Emanuele Barca, Valentina Emmanuele, Catarina Maria Quinzii, Camilla V. Simpson, Kristin Engelstad, Xiomara Q. Rosales, John L.P. Thompson · 2020 · Journal of Inherited Metabolic Disease
  6. Disease awareness or subtle product placement? Orphan diseases featured in the television series “House, M.D.” - a cross-sectional analysis
    Konstantin Mechler, Juliane Rausch, William K. Mountford, Markus Ries · 2020 · BMC Medical Ethics
  7. Pharmaceutical policy and innovation for rare diseases: A narrative review
    Adrián Alonso Ruiz, Kaitlin Elizabeth Large, Suerie Moon, Marcela Vieira · 2023 · F1000Research
  8. Association between expedited review designations and the US or global burden of disease for drugs approved by the US Food and Drug Administration, 2010–2019: a cross-sectional analysis
    Matthew J. Jackson, Gregory Vaughan, Fred David Ledley · 2024 · BMJ Open
  9. Droga órfã: surgimento de um novo conceito
    Sigride Thomé-Souza, Kette D. R. Valente · 2011 · Journal of Epilepsy and Clinical Neurophysiology
  10. Pharmacogenomic Inequalities: Strategies for Justice in Biomedical Research and Healthcare
    Giovanni De Grandis · 2017 · UCL Discovery (University College London)
  11. Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations
    Andrea Ecker, Segundo Mariz, Frauke Naumann‐Winter, Koen Norga, Ingeborg Barišić, Thomas Girard, Paolo A. Tomasi, Dirk Mentzer, Bruno Miguel Nogueira Sepodes · 2017 · Archives of Disease in Childhood
  12. Comparative analysis of correlations of research and development indicators for rare diseases among Japan, the US, and Europe
    Hirokuni Mizoguchi, Shingo Kano · 2019 · Scientometrics
  13. How Often are Orphan Drugs Orphaned by the Thermochemical Community?
    Kathleen Frances Edwards, Joel Fredric Liebman · 2018 · Current Medicinal Chemistry
  14. The Role of Pharmacoepidemiology in Regulatory Agencies
    Gerald J. Dal Pan, Peter R. Arlett · 2012 · Pharmacoepidemiology
  15. Discovery of New Medicines
    Yves Ribeill · 2013 · Wiley eBooks
  16. The routes to orphan drug designation – our recent experience at the FDA
    Daniel Lev, Chandana Thorat, Ian Phillips, Mathew Thomas, Menfo A. Imoisili · 2011 · Drug Discovery Today
  17. Investigation of quality review issues and the association with application characteristics for new drug applications in first-cycle reviews
    Jhih-Siang Liou, Lin‐Chau Chang · 2019 · Regulatory Toxicology and Pharmacology
  18. Breaking barriers in rare disease research: The RARE-X Open Science Data Challenge as a model for collaborative innovation and community partnership
    Karmen M Trzupek, Ravi Bhargava, Cynthia Kuan, Fanny Sie, Vanessa K. Vogel‐Farley, Katelyn Hobbs, Verena Chung, Maria Diaz, Charlene Son Rigby, Joseph Geraci et al. · 2025 · Human Genetics and Genomics Advances
  19. Discordance in orphan drug approvals between the U.S. Food and Drug Administration and the European Medicines Agency: A retrospective observational analysis
    Jin Ding, Michael M. Hopkins, Paul A. Martin · 2026 · PLoS Medicine
  20. Commercial Aspects of Drug Development
    Edward D. Zanders · 2011 · Springer eBooks
  21. Orphan Drugs
    Maurizio Scarpa, Cinzia Maria Bellettato, Christina Lampe · 2015 · Springer eBooks
  22. Treating Rare Diseases: Business Model for Orphan Drug Development
    Cory S.M. Williams · 2014 · Royal Society of Chemistry eBooks
  23. Orphan Drug Pricing and Cost Trends in USA: An Analysis of Impact of Orphan Drug ACT
    Sangita Mishra, Venkatesh Madhugiri Prakash · 2023 · Indian Journal of Pharmaceutical Education and Research
  24. Entrepreneurship: Drug Discovery Innovation at Start‐Up and Medium‐Sized Biotechnology Companies
    Allen B. Reitz, Kathleen M. Czupich · 2014 · Wiley eBooks
  25. The Unaccounted Insurance Value of Medical Innovation
    Anushree Subramaniam · 2016 · SSRN Electronic Journal
  26. Perspective from the Literature on the Role of Expert Judgment in Scientific and Statistical Research and Practice
    Naomi C. Brownstein · 2018 · arXiv (Cornell University)
  27. Association between expedited review designations and the US or global burden of disease for drugs approved by the US Food and Drug Administration 2010–2019
    Matthew J. Jackson, Gregory Vaughan, Fred David Ledley · 2023 · medRxiv
  28. Review of: "The Plight of Rare Diseases in Southern Africa: Health and Social Services Policy Recommendations"
    Laura Magazzini · 2023
  29. Aspects réglementaires des médicaments orphelins destinés à la population pédiatrique : contraintes et opportunités pour l'industrie pharmaceutique
    Aurélie Tireford · 2010 · HAL
  30. Drugs for Cancer
    James F. Spicer, Johann Sebastian De Bono · 2013
  31. Views from Academia, Industry, Regulatory Agencies, and the Legal System
    J. Avorn, Jingping Mo, R. Reynolds, G. Dal Pan, P. Arlett, A. Kesselheim · 2013 · Textbook of Pharmacoepidemiology
  32. Medicines for rare (orphan) diseases: an enquiry and proposal for tax-incentives
    Jacob E. John · 2011 · Drug Discovery Today
  33. Regulators Should Better Leverage Effectiveness Standards to Enhance Drug Value
    Huseyin Naci, George Alexander · 2014 · Pharmacotherapy The Journal of Human Pharmacology and Drug Therapy
  34. Providing Medical Information for Orphan Drugs
    Sean D. Turbeville, David A. Wells, Carl S. Hornfeldt · 2012 · Drug Information Journal
  35. 14. The Drug Development of Orphan Drug
    Harumasa Nakamura · 2013 · Nihon Naika Gakkai Zasshi
  36. Future of Medical Research in Rare Diseases and Cancers: Shift from Pharma to Biotech and the Golden Age of Medical Advancement
    Abhith Pallegar · 2017 · Cancer and Clinical Oncology
  37. 희귀질환 연구와 치료제 개발 동향
    이신행, 이장익 · 2013 · Korean Journal of Clinical Pharmacy
  38. Views from Academia, Industry, Regulatory Agencies, and the Legal System
    Joshua J. Gagne, Jerry Avorn, Nicolle M. Gatto, Jingping Mo, Gerald J. Dal Pan, June M. Raine, Shinobu Uzu, Aaron Seth Kesselheim, Kerstin Noëlle Vokinger · 2021
  39. Orphan Drugs
    Maurizio Scarpa, Cinzia Maria Bellettato, Christina Lampe · 2015 · Springer eBooks
  40. The Role of Pharmacoepidemiology in Regulatory Agencies
    Gerald J. Dal Pan, June M. Raine, Shinobu Uzu · 2019 · Pharmacoepidemiology
  41. 14．希少疾患への取り組み，オーファンドラッグの開発にむけて
    治雅 中村 · Institutional Repositories DataBase (IRDB)
  42. Investigation of the diagnostic importance and accuracy of CT in the chest compared to the RT-PCR test for suspected COVID-19 patients in Jordan
    Haytham Alewaidat, Ziad Bataineh, Mohammad Bani-Ahmad, Manar Alali, Ali Almakhadmeh · 2023 · F1000Research
  43. Personalized precision medicine of strategic health action in Niemann-Pick type A/B disease: Case report
    Juan Manuel Sánchez-Vargas · 2023 · Journal of Clinical Images and Medical Case Reports
  44. Investigation of the diagnostic importance and accuracy of CT in the chest compared to the RT-PCR test for suspected COVID-19 patients in Jordan
    Haytham Alewaidat, Ziad Bataineh, Mohammad Bani-Ahmad, Manar Alali, Ali Almakhadmeh · 2023 · F1000Research
  45. Precision Personalized Medicine of Strategic Health Action in Niemann-Pick Disease type A/B
    Juan Manuel Sánchez-Vargas, Lina Johanna Moreno Giraldo · 2023 · Genetics and Clinical Genomics
  46. Determinants of orphan drug health technology assessment in South Korea: an empirical analysis
    Se Hee Lee, Sung Kyung Lee, Jong Hyuk Lee, Joon Seok Bang · 2025 · Frontiers in Pharmacology
  47. Additional file 1 of Protocol paper: a multi-center, double-blinded, randomized, 6-month, placebo-controlled study followed by 12-month open label extension to evaluate the safety and efficacy of Saracatinib in Fibrodysplasia Ossificans Progressiva (STOPFOP)
    Smilde, Bernard J., Clemens Stockklausner, Richard W. Keen, Andrew Whittaker, Alex N. Bullock, von Delft, Annette, Natasja M. van Schoor, Yu, Paul B., Elisabeth M. W. Eekhoff · 2022 · Figshare
  48. Additional file 2 of Protocol paper: a multi-center, double-blinded, randomized, 6-month, placebo-controlled study followed by 12-month open label extension to evaluate the safety and efficacy of Saracatinib in Fibrodysplasia Ossificans Progressiva (STOPFOP)
    Smilde, Bernard J., Clemens Stockklausner, Richard W. Keen, Andrew Whittaker, Bullock, Alex N., von Delft, Annette, Natasja M. van Schoor, Yu, Paul B., Elisabeth M. W. Eekhoff · 2022 · Figshare
  49. Additional file 2 of Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases
    Kathleen L. Miller, Lewis J. Fermaglich, Janet W. Maynard · 2021 · Figshare