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Volume 6
Issue 1
10.1080/20016689.2018.1542920
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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.
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Article

Patient-Reported Outcome Claims in European and United States Orphan Drug Approvals

by
Szymon Jarosławski
1,
Pascal Auquier
1,
Borislav Borissov
2,
Claude Dussart
3 and
Mondher Toumi
1,*
1
Public Health Department–Research Unit EA 3279, Aix–Marseille University, Marseille, France
2
Prescriptia Ltd., Sofia, Bulgaria
3
Facultéde Médecine Laennec, Claude Bernard University, Laboratoire Parcours Santé Systémique EA 4129, Université de Lyon, Lyon, France
*
Author to whom correspondence should be addressed.
J. Mark. Access Health Policy 2018, 6(1), 1542920; https://doi.org/10.1080/20016689.2018.1542920
Submission received: 6 May 2018 / Revised: 23 October 2018 / Accepted: 26 October 2018 / Published: 7 November 2018

Abstract

Purpose: We aimed to evaluate the rate of usage and the kind of patient-reported outcome (PRO) claims in orphan drug approvals from the European Medicines Agency (EMA) dated between 1/1/2012 and 31/12/2016 and to compare them to those from the US Food and Drug Administration (FDA). Methods: Orphan drug approval documentation was obtained from the EMA website. PRO-related language was extracted from the Summaries of Product Characteristics (SmPCs). Data were compared to a previously published analysis of the FDA approvals from the same time period. Results: Out of 60 approvals that met the inclusion criteria, 12 products approved by the EMA for 13 (21.7%) orphan indications contained PRO language in the Clinical Studies section of the SmPC. Twelve SmPCs contained PRO instruments based on symptoms, five of which also concerned patient functioning. Eight approvals included PRO claims related to quality of life (QoL) most commonly in cancer treatment. Conclusion: The rate of PRO claims was lower for orphan drugs specifically than for all drug approvals by the EMA. However, in accordance with previous findings, the EMA appeared more inclined to grant PRO claims including health-related QoL than the FDA.
Keywords: patient-reported outcomes; quality of life; rare diseases; labelling; EMA; FDA patient-reported outcomes; quality of life; rare diseases; labelling; EMA; FDA

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MDPI and ACS Style

Jarosławski, S.; Auquier, P.; Borissov, B.; Dussart, C.; Toumi, M. Patient-Reported Outcome Claims in European and United States Orphan Drug Approvals. J. Mark. Access Health Policy 2018, 6, 1542920. https://doi.org/10.1080/20016689.2018.1542920

AMA Style

Jarosławski S, Auquier P, Borissov B, Dussart C, Toumi M. Patient-Reported Outcome Claims in European and United States Orphan Drug Approvals. Journal of Market Access & Health Policy. 2018; 6(1):1542920. https://doi.org/10.1080/20016689.2018.1542920

Chicago/Turabian Style

Jarosławski, Szymon, Pascal Auquier, Borislav Borissov, Claude Dussart, and Mondher Toumi. 2018. "Patient-Reported Outcome Claims in European and United States Orphan Drug Approvals" Journal of Market Access & Health Policy 6, no. 1: 1542920. https://doi.org/10.1080/20016689.2018.1542920

APA Style

Jarosławski, S., Auquier, P., Borissov, B., Dussart, C., & Toumi, M. (2018). Patient-Reported Outcome Claims in European and United States Orphan Drug Approvals. Journal of Market Access & Health Policy, 6(1), 1542920. https://doi.org/10.1080/20016689.2018.1542920

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