Regulatory Framework for Drug Development in Rare Diseases

Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy

(2021) Stephen L. Lake et al.   STATISTICS IN MEDICINE

Rare Breast Cancer Subtypes

(2021) Sarah Jenkins et al.   Current Oncology Reports

Evolving Acceptance and Use of RWE for Regulatory Decision Making on the Benefit/Risk Assessment of a Drug in Japan

(2021) Kinue Nishioka et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease

(2021) Kevin Hammon et al.   CTS-Clinical and Translational Science

Perspective on model‐informed drug development

(2021) Lawrence J. Lesko   CPT-Pharmacometrics & Systems Pharmacology

Real‐World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value

(2021) Peter Arlett et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

The U.S. Food and Drug Administration’s Complex Innovative Trial Design Pilot Meeting Program: Progress to date

(2021) Dionne Price et al.   Clinical Trials

Quantitative systems pharmacology: Landscape analysis of regulatory submissions to the US Food and Drug Administration

(2021) Jane P. F. Bai et al.   CPT-Pharmacometrics & Systems Pharmacology

Use of real‐world evidence in regulatory decisions for rare diseases in the United States—Current status and future directions

(2020) Jasmanda Wu et al.   PHARMACOEPIDEMIOLOGY AND DRUG SAFETY

Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases

(2020) Joëlle Micallef et al.   THERAPIE

The evolution of patient-focused drug development and Duchenne muscular dystrophy

(2020) Norah L. Crossnohere et al.   Expert Review of Pharmacoeconomics & Outcomes Research

Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?

(2020) Katherine A. High   Nature Communications

Model-Informed Drug Development: Current U.S. Regulatory Practice and Future Considerations

(2019) Yaning Wang et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

A human-on-a-chip approach to tackling rare diseases

(2019) Camilly P. Pires de Mello et al.   DRUG DISCOVERY TODAY

Determinants of Market Exclusivity for Prescription Drugs in the United States

(2017) Aaron S. Kesselheim et al.   JAMA Internal Medicine

The European Union Committee of Experts on Rare Diseases: three productive years at the service of the rare disease community

(2014) Ségolène Aymé et al.   Orphanet Journal of Rare Diseases

Rare-disease genetics in the era of next-generation sequencing: discovery to translation

(2013) Kym M. Boycott et al.   NATURE REVIEWS GENETICS

Identification and management of poor response to growth-promoting therapy in children with short stature

(2012) Peter Bang et al.   CLINICAL ENDOCRINOLOGY