Patient-reported outcome labelling still rare in the US

2016 ◽  
Vol 752 (1) ◽  
pp. 31-31
Keyword(s):  
Patient Reported Outcome ◽  
Patient Reported ◽  
The Us

scholarly journals Responder Definition of a Patient-Reported Outcome Instrument for Laryngopharyngeal Reflux Based on the US FDA Guidance

2015 ◽  
Vol 18 (4) ◽  
pp. 396-403 ◽  
Author(s):  
Han-Chung Lien ◽  
Chen-Chi Wang ◽  
Shou-Wu Lee ◽  
Jeng-Yuan Hsu ◽  
Hong-Zen Yeh ◽  
...  
Keyword(s):  
Laryngopharyngeal Reflux ◽  
Patient Reported Outcome ◽  
Fda Guidance ◽  
Outcome Instrument ◽  
Patient Reported ◽  
The Us ◽  
Us Fda ◽  
Definition Of

scholarly journals End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings

2019 ◽  
Vol 3 (23) ◽  
pp. 4002-4020 ◽  
Author(s):  
Ann T. Farrell ◽  
Julie Panepinto ◽  
Ankit A. Desai ◽  
Adetola A. Kassim ◽  
Jeffrey Lebensburger ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcome ◽  
Cell Disease ◽  
End Points ◽  
Low Resource Settings ◽  
Low Resource ◽  
Patient Reported ◽  
The Us ◽  
American Society

Abstract To address the global burden of sickle cell disease and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to patient-reported outcome, pain (non–patient-reported outcomes), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the end-organ considerations, measurement of cure, and low-resource settings panels as well as relevant findings and recommendations from the biomarkers panel.

Patient-Reported Outcomes Supporting Anticancer Product Approvals

2007 ◽  
Vol 25 (32) ◽  
pp. 5094-5099 ◽  
Author(s):  
Edwin P. Rock ◽  
Dianne L. Kennedy ◽  
Melissa H. Furness ◽  
William F. Pierce ◽  
Richard Pazdur ◽  
...  
Keyword(s):  
Clinical Benefit ◽  
Patient Reported Outcome ◽  
Functional Domain ◽  
Draft Guidance ◽  
Treatment Benefit ◽  
Regulatory Review ◽  
Single Item ◽  
Patient Reported ◽  
The Us ◽  
Support Treatment

In 2006, the US Food and Drug Administration (FDA) published draft guidance to provide recommendations for development, validation, implementation, and interpretation of patient-reported outcome (PRO) measures that can support treatment benefit claims in product labeling. Here, we summarize and discuss FDA approvals of anticancer products in the context of the draft guidance. We identified anticancer product approvals having efficacy claim(s) based at least in part on a PRO. In addition, we collated limitations of PRO instruments commonly submitted for regulatory review over the period from October 1, 2004 to September 30, 2006. From 1995 onward, nine indications were approved for seven anticancer products based at least in part on a PRO. In eight of nine approvals, PRO data supplemented other evidence of clinical benefit. In seven approvals, the PRO measured a single symptom or functional domain that was directly attributable to the treatment benefit observed in the disease. The FDA's draft PRO guidance describes principles that have been used in anticancer product approvals for more than a decade. PRO end points typically support treatment benefit claims that refer to a patient's symptoms or ability to function. Single-item PROs may be acceptable. PRO data should be both internally consistent and aligned with other evidence of clinical benefit. The FDA encourages sponsors to consult with the FDA early in the process of PRO development.

scholarly journals Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration

2018 ◽  
Vol 6 (1) ◽  
pp. 1433426
Author(s):  
Szymon Jarosławski ◽  
Pascal Auquier ◽  
Borislav Borissov ◽  
Claude Dussart ◽  
Mondher Toumi
Keyword(s):  
Drug Administration ◽  
Food And Drug Administration ◽  
Orphan Drugs ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
The Us

P041 DEVELOPMENT OF PROMIS PEDIATRIC PATIENT-REPORTED OUTCOME SHORT FORMS FOR FATIGUE AND PAIN INTERFERENCE IN CHILDREN WITH CROHN’S DISEASE

2020 ◽  
Vol 158 (3) ◽  
pp. S101-S102
Author(s):  
Julia Schuchard ◽  
Michael Kappelman ◽  
Andrew Grossman ◽  
Jennifer Clegg ◽  
Christopher Forrest
Keyword(s):  
Crohn’S Disease ◽  
Crohn's Disease ◽  
Pediatric Patient ◽  
Patient Reported Outcome ◽  
Pain Interference ◽  
Short Forms ◽  
Patient Reported
Sign in / Sign up

Export Citation Format

Share Document