Sage Journals: Discover world-class research

Retricted access

Research article

First published 2016-9

A Patient Centricity Team Tool to Enable Patient-Focused Drug Development

Rebecca Ashkenazy, Roslyn F. Schneider

Therapeutic Innovation & Regulatory Science

Open access

Research article

First published 2021-7

Measuring Meaningful Benefit-Risk Tradeoffs to Promote Patient-Focused Drug Development in Prader-Willi Syndrome: A Discrete-Choice Experiment

Jui-Hua Tsai, Norah L. Crossnohere, Theresa Strong [...]

MDM Policy & Practice

Retricted access

Editorial

First published 2018-9

From the Valley of Death to the Crossroads of Opportunity

Peter J. Pitts, Patrick Brady

Therapeutic Innovation & Regulatory Science

Open access

Meeting report

First published 2024-03-05

Draft Guidance for Industry Duchenne Muscular Dystrophy,Becker Muscular Dystrophy,and Related Dystrophinopathies – Developing Potential Treatments for the Entire Spectrum of Disease

Craig McDonald, Eric Camino, Rafael Escandon [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2019

FDA Reported Use of Patient Experience Data in 2018 Drug Approvals

Cameron M. Kieffer, Alexis Reisin Miller, Benjamin Chacko [...]

Therapeutic Innovation & Regulatory Science

Open access

Research article

First published 2023-3

The relevance of fatigue to relapse rate in multiple sclerosis: Applying patient preference data to the OPTIMUM trial

Robert J Fox, Tommi Tervonen, Andrea Phillips-Beyer [...]

Multiple Sclerosis Journal

Open access

Research article

First published 2021-4

Pulmonary hypertension due to interstitial lung disease or chronic obstructive pulmonary disease: a patient experience study of symptoms and their impact on quality of life

Hilary M. DuBrock, Steven D. Nathan, Bryce B. Reeve [...]

Pulmonary Circulation

Open access

Review article

First published 2024

Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trials

JayEtta Hecker, Gabrielle Conecker, Chere Chapman [...]

Therapeutic Advances in Rare Disease

Retricted access

Review article

First published 2022-3

US Food and Drug Administration Facilitated Pediatric Approval Programs: Application to Pediatric Neurological Disorders

Emily Da Cruz, Faraan Rahim, Monica Lemmon [...]

Journal of Child Neurology

Free access

Review article

First published 2016-11

Using High-Intensity Focused Ultrasound as a Means to Provide Targeted Drug Delivery

Ashley McClure

Journal of Diagnostic Medical Sonography

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