Overview of Clinical Pharmacology Packages of New Drug Applications Approved for the Treatment of Rare Diseases

Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases

(2022) Carla Epps et al.   Therapeutic Innovation & Regulatory Science

Clinical Pharmacology in Drug Development for Rare Diseases in Neurology: Contributions and Opportunities

(2021) Bilal Abuasal et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Physiologically‐Based Pharmacokinetic Modeling in Renal and Hepatic Impairment Populations: A Pharmaceutical Industry Perspective

(2020) Tycho Heimbach et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis

(2020) Adrienne Y.L. Chan et al.   VALUE IN HEALTH

How many rare diseases are there?

(2019) Melissa Haendel et al.   NATURE REVIEWS DRUG DISCOVERY

From scientific discovery to treatments for rare diseases – the view from the National Center for Advancing Translational Sciences – Office of Rare Diseases Research

(2018) Petra Kaufmann et al.   Orphanet Journal of Rare Diseases

The promise and challenges of rare cancer research

(2016) Kimberly M Komatsubara et al.   LANCET ONCOLOGY

Rare cancers: a sea of opportunity

(2016) Niki Boyd et al.   LANCET ONCOLOGY

Rare diseases, orphan drugs and their regulation: questions and misconceptions

(2010) Erik Tambuyzer   NATURE REVIEWS DRUG DISCOVERY