Next Article in Journal
Cost-Effectiveness of Solifenacin Compared with Oral Antimuscarinic Agents for the Treatment of Patients with Overactive Bladder in the UK
Previous Article in Journal
Sales Promotion by Wholesalers Affects General Practitioners’ Prescription Behaviours in Japan
 
 
Search for Articles:
Title / Keyword
Author / Affiliation / Email
Journal
Article Type
 
 
Advanced Search
 
Section
Special Issue
Volume
Issue
Number
Page
 
Journals
JMAHP
Volume 6
Issue 1
10.1080/20016689.2018.1433426
jmahp-logo
Submit to this Journal Review for this Journal Propose a Special Issue
► Article Menu

Article Menu

share Share announcement Help format_quote Cite question_answer Discuss in SciProfiles thumb_up
...
Endorse textsms
...
Comment
Journal of Market Access & Health Policy (JMAHP) is published by MDPI from Volume 12 Issue 1 (2024). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with Taylor & Francis.
first_page
settings
Order Article Reprints
Font Type:
Arial Georgia Verdana
Font Size:
Aa Aa Aa
Line Spacing:
Column Width:
Background:
Article

Low Rates of Patient-Reported Outcome Claims for Orphan Drugs Approved by the US Food and Drug Administration

by
Szymon Jarosławski
1,
Pascal Auquier
1,
Borislav Borissov
2,
Claude Dussart
3,4 and
Mondher Toumi
1
1
Public Health Department–Research Unit EA 3279, Aix–Marseille University, 27 bd Jean Moulin, Marseille, France
2
Prescriptia Ltd, Sofia, Bulgaria
3
Laboratoire Parcours Santé Systémique EA 4129, Claude Bernard University, Lyon, France
4
Faculté de Médecine Laennec, Université de Lyon, Lyon, France
J. Mark. Access Health Policy 2018, 6(1), 1433426; https://doi.org/10.1080/20016689.2018.1433426
Submission received: 6 October 2017 / Revised: 1 January 2018 / Accepted: 12 January 2018 / Published: 12 February 2018

Abstract

Background: Claims included in package inserts (PIs) for medicinal products approved by the US Food and Drug Administration (FDA) constitute the regulatory definition of drugs’ benefits and risks. Objective: We sought to assess the usage of patient-reported outcome (PRO) claims in a comprehensive set of US FDA orphan drug approvals dated between 1/1/2012 and 31/12/2016, and characterize them. Study design: Orphan drug approval documentation was obtained from the US FDA website. Drug Package Inserts (PI) were analyzed to extract information on PRO-related language. Results: Among 178 drugs that met inclusion criteria, 16 (9%) products approved for 16 orphan indications contained PRO language in the Clinical Studies section of the PI. All PRO instruments concerned disease symptoms, and two also referred to patient functioning. The most common PRO instrument was a bleed-specific rating scale for four products approved for the treatment or prevention of bleeding episodes in patients with genetic bleeding disorders. Conclusions: There is a need to implement public incentives for academic development of PRO instruments for rare conditions and for regulatory policies that mandate the collection of PRO endpoints in pivotal trials of orphan drugs.
Keywords: patient-reported outcomes; quality of life; rare diseases; orphan drugs; labelling patient-reported outcomes; quality of life; rare diseases; orphan drugs; labelling

Share and Cite

MDPI and ACS Style

Jarosławski, S.; Auquier, P.; Borissov, B.; Dussart, C.; Toumi, M. Low Rates of Patient-Reported Outcome Claims for Orphan Drugs Approved by the US Food and Drug Administration. J. Mark. Access Health Policy 2018, 6, 1433426. https://doi.org/10.1080/20016689.2018.1433426

AMA Style

Jarosławski S, Auquier P, Borissov B, Dussart C, Toumi M. Low Rates of Patient-Reported Outcome Claims for Orphan Drugs Approved by the US Food and Drug Administration. Journal of Market Access & Health Policy. 2018; 6(1):1433426. https://doi.org/10.1080/20016689.2018.1433426

Chicago/Turabian Style

Jarosławski, Szymon, Pascal Auquier, Borislav Borissov, Claude Dussart, and Mondher Toumi. 2018. "Low Rates of Patient-Reported Outcome Claims for Orphan Drugs Approved by the US Food and Drug Administration" Journal of Market Access & Health Policy 6, no. 1: 1433426. https://doi.org/10.1080/20016689.2018.1433426

APA Style

Jarosławski, S., Auquier, P., Borissov, B., Dussart, C., & Toumi, M. (2018). Low Rates of Patient-Reported Outcome Claims for Orphan Drugs Approved by the US Food and Drug Administration. Journal of Market Access & Health Policy, 6(1), 1433426. https://doi.org/10.1080/20016689.2018.1433426

Article Metrics

Back to TopTop