Expert Opinion on Orphan Drugs [e-ISSN 2167-8707] is an international journal publishing rigorously peer-reviewed review articles and original papers on rare diseases and orphan drugs. Each article is structured to incorporate the author’s own expert opinion on the impact of the topic on research and clinical practice and the scope for future development.

Expert Opinion on Orphan Drugs aims to draw a spotlight on diseases that do not have a high prevalence but have a huge impact on patients and their clinicians, through valuable and high-quality expert insights. In a field which is ever-growing and changing, Expert Opinion on Orphan Drugs is committed to acting as a reliable beacon of guidance and support for researchers, clinicians, patients and advocacy groups in order to optimise research, development, management and quality of life of rare disease patients.

Articles focus on the following key areas:

    Diagnostics, pharmacogenomics and screening;
    Public health and epidemiology;
    Drug development;
    Lead generation and discovery;
    Early or late phase clinical trials;
    Regulatory issues;
    Post-marketing safety and treatment outcomes.

The journal also welcomes solicited and unsolicited papers including Drug Evaluations, specifically reviewing the pharmacological properties, clinical potential/clinical efficacy, safety and, if applicable, HE/OR data on a particular orphan drug, as well as Original Research papers and Editorials.
Once your paper has been assessed for suitability by the Editorial Team, it will then be double-anonymous peer-reviewed by independent, expert referees.

 For any pre-submission enquiries, please contact the Commissioning Editor.

Please note, from 2023 the Print ISSN is not in active use as this journal is no longer published in print.

Editor in Chief:
M. Ian Phillips, Director of the Center for Rare Disease Therapies, Keck Graduate Institute

Editorial Board:
Sabina Antoniu, Pulmonary Disease Division, University of Medicine and Pharmacy, Romania
Martin Braddock, Global Medicines Development, AstraZeneca R&D, UK
Maria Luisa Brandi, Unit of Bone and Mineral Metabolism, University of Florence, Italy
Roberto Giugliani, Department of Genetics, Federal University of Rio Grande do Sul, Brazil
Stephen Groft, Director, Office of Rare Diseases, National Institutes of Health, USA
Aaron S. Kesselheim, Harvard Medical School and Brigham and Women's Hospital, USA
Stephen Kingsmore, Center for Pediatric Genomic Medicine, Children's Mercy Hospitals and Clinics, USA
Alex MacKenzie, Pediatrics, CHEO, University of Ottawa, Canada
Maria Mavris, EURORDIS - European Organisation for Rare Diseases, France
Khue Nguyen, University of California, San Diego, USA 
Daniel O'Connor, Medicines and Healthcare Products Regulatory Agency, UK
Manuel Posada, Director, Research Institute for Rare Diseases, Instituto de Salud Carlos III, Spain
Peter L. Saltonstall, President and CEO, National Organization for Rare Disorders (NORD), USA
Michael Shy, Division of Neuromuscular Medicine, University of Iowa, USA
Ali Taher, Division of Hematology/Oncology, American University of Beirut Medical Centre, Lebanon
Eleftherios Tsiridis, Academic Department of Trauma and Orthopaedics, University of Leeds, UK