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Retricted access

Review article

First published 2021-6

Challenges in Evaluating Safety and Efficacy in Drug Development for Rare Diseases: A Review for Pharmacists

Kanya K. Shah, Stephen Kogut, Angela Slitt

Journal of Pharmacy Practice

Retricted access

Research article

First published 2017-3

Patient Voice in Rare Disease Drug Development and Endpoints

Linda S. Deal, Jonathan C. Goldsmith, Susan Martin [...]

Therapeutic Innovation & Regulatory Science

Open access

Review article

First published 2022

Building cross-border collaborations to increase diversity and accelerate rare disease drug development – meeting report from the inaugural IndoUSrare Annual Conference 2021

Harvinder Kour Khera, Nisha Venugopal, Ramya T. Karur [...]

Therapeutic Advances in Rare Disease

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Review article

First published 2019-9

Impact of Regulatory Incentive Programs on the Future of Pediatric Drug Development

Elizabeth Yen, Jonathan M. Davis, Christopher-Paul Milne

Therapeutic Innovation & Regulatory Science

Open access

Research article

First published 2017-6

Using phase II data for the analysis of phase III studies: An application in rare diseases

Simon Wandel, Beat Neuenschwander, Christian Röver [...]

Clinical Trials

Open access

Case report

First published 2020-12

An Evolved Approach to Advisory Boards in Rare Disease Drug Development: 5-Step Model to Finding and Engaging Patient Advisors

Carlotta Dillon, Joyce Knapp, Mark Stinson

Journal of Patient Experience

Retricted access

Brief report

First published 2021-12

National Institutes of Health,U.S. Food and Drug Administration,15 Partners to Accelerate Development of Rare Disease Gene Therapies

Alex Philippidis

Human Gene Therapy

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Letter

First published 2026-1

Letter: The Internet of Medical Things (IoMT): A New Frontier in the Digital Age for Rare Disease Clinical Trials and Global Drug Development

Aslıgül Kendirci

OMICS: A Journal of Integrative Biology

Open access

Research article

First published 2023

The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies

Katherine L. Beaverson, Daria Julkowska, Mary Catherine V. Letinturier [...]

Therapeutic Advances in Rare Disease

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Other

First published 2000-1

The Orphan Drug Act: Provisions and Considerations

Carson R. Reider

Therapeutic Innovation & Regulatory Science

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