Concepedia

Orphan Drug Development

Orphan Drug Development is a research framework within regulatory pharmacology that systematically addresses the scientific, economic, and policy challenges inherent in creating therapeutics for rare diseases, distinguished by its reliance on specialized incentives, targeted clinical trial designs, and regulatory pathways established to overcome market failure in rare disease treatment.

312

Publications

13.5K

Citations

1.1K

Authors

574

Institutions

Publications per year

2017–2026

111

Parents

Publications

312

Failure of Investigational Drugs in Late-Stage Clinical Development and Publication of Trial Results

Thomas J. Hwang, Daniel Carpenter, Julie C. Lauffenburger et al. · JAMA Internal Medicine · 2016 · 440 citations

, , +16

FDA Approval and Regulation of Pharmaceuticals, 1983-2018

Jonathan J. Darrow, Jerry Avorn, Aaron S. Kesselheim · JAMA · 2020 · 362 citations

, , +15

Assessing the economic challenges posed by orphan drugs

Michael Drummond, David A. Wilson, Panos Kanavos et al. · International Journal of Technology Assessment in Health Care · 2007 · 278 citations

, , +17

Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

Alessandro Aiuti, Maria Grazia Roncarolo, Luigi Naldini · EMBO Molecular Medicine · 2017 · 256 citations · Full text

, , +17

Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer

Aaron S. Kesselheim · JAMA · 2011 · 229 citations

, , +17

1–5 of 312

Authors

1.1K

  1. CE
    Carla E. M. Hollak

    Amsterdam UMC Location University of Amsterdam, Netherlands

    10 publications · 540 citations

    9

    h-index

  2. SS
    Steven Simoens

    KU Leuven, Belgium

    10 publications · 572 citations

    9

    h-index

  3. AS
    Aaron S. Kesselheim

    Brigham and Women's Hospital, United States

    7 publications · 1.3K citations

    7

    h-index

  4. KL
    Kathleen L. Miller

    United States Food and Drug Administration, United States

    7 publications · 360 citations

    7

    h-index

  5. MD
    Marc Dooms

    KU Leuven, Belgium

    6 publications · 233 citations

    6

    h-index

1–5 of 1.1K

Institutions

574

  1. United States Food and Drug Administration

    Silver Spring, United States

    14 h-index

    41 publications

    1.5K citations

  2. Utrecht University

    Utrecht, The Netherlands

    12 h-index

    30 publications

    1.4K citations

  3. Heidelberg University

    Heidelberg, Germany

    11 h-index

    27 publications

    991 citations

  4. University Hospital Heidelberg

    Heidelberg, Germany

    10 h-index

    25 publications

    956 citations

  5. Harvard University

    Cambridge, United States

    9 h-index

    23 publications

    4.9K citations

  6. KU Leuven

    Leuven, Belgium

    9 h-index

    25 publications

    1.2K citations

  7. Amsterdam UMC Location University of Amsterdam

    Amsterdam, The Netherlands

    8 h-index

    20 publications

    699 citations

  8. Brigham and Women's Hospital

    Boston, United States

    8 h-index

    21 publications

    4.7K citations

1–8 of 574

Venues

  1. Orphanet Journal of Rare Diseases

    OJRD · 38 publications · 2K citations

    24

    h-index

  2. PubMed

    Repository · 15 publications · 535 citations

    13

    h-index

  3. Expert Opinion on Orphan Drugs

    7 publications · 144 citations

    7

    h-index

  4. Clinical Pharmacology & Therapeutics

    CPT · 7 publications · 117 citations

    7

    h-index

  5. PEDIATRICS

    6 publications · 366 citations

    6

    h-index

1–5