Assessment of PRO Label Claims Granted by the FDA as Compared to the EMA (2006–2010)

DeMuro, Carla; Clark, Marci; Doward, Lynda; Evans, Emily; Mordin, Margaret; Gnanasakthy, Ari

doi:10.1016/j.jval.2013.08.2293

Cited by 72 publications

(50 citation statements)

References 6 publications

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“…Additionally, 21.7% of orphan-designated approvals in the studied sample presented PRO claims in their SmPCs. This is significantly below the 47% reported PRO claims for all new drug approvals by the EMA from 2006 to 2010 [11] and the 46% reported from 2008 to 2012 [12]. Conversely to the above mentioned studies, which analysed the entire content of the European Public Assessment Reports (EPARs), we focused only on the SmPCs, because they include PRO claims granted by the EMA.…”

Section: Discussionmentioning

confidence: 96%

“…This can be partially explained by the fact that, while primary endpoints were typically based on disease symptoms, the FDA-approved PRO claims were symptom-based. Also, the EMA is more inclined to approve HRQoL and functioning measures, although not as primary endpoints [5,11]. All but one approval with a non-primary-endpoint-based PRO claims in our sample involved higher degree outcomes, such as HRQoL or functioning.…”

Section: Discussionmentioning

confidence: 99%

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Patient-reported outcome claims in European and United States orphan drug approvals

Jarosławski

Auquier

Borissov³

et al. 2018

Journal of Market Access & Health Policy

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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.

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Section: Discussionmentioning

confidence: 96%

Section: Discussionmentioning

confidence: 99%

Patient-reported outcome claims in European and United States orphan drug approvals

Jarosławski

Auquier

Borissov³

et al. 2018

Journal of Market Access & Health Policy

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show abstract

“…Overall, over the last decade or so, both the EMA and the FDA have shown increasing levels of interest towards PRO usage during MA procedures [20,24]. EMA appeared, however, to be more flexible and open to the inclusion of validated and clinically/methodologically appropriate PROs [33]. Indeed, many EMA disease-specific guidelines now require PRO endpoints to be included as secondary endpoints (e.g., the Ankylosing Spondylitis Quality of Life; ASHRQoL) [34].…”

Section: Discussionmentioning

confidence: 99%

Role of patient-reported outcomes and other efficacy endpoints in the drug approval process in Europe (2008–2012)

Bansal¹,

Bhagat²,

Schifano³

et al. 2015

JEGH

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The present study aimed at systematically reviewing the role and extent of patient-reported outcomes (PROs) usage within the package of scientific evidence considered for marketing authorization (MA). All regulatory information published by the European Medicines Agency (EMA) for products authorized between January 2008 and December 2012 and appearing in the European Public Assessment Report (EPAR) database was examined for efficacy endpoints. The endpoints here considered included: PROs, clinician reported outcomes (CROs), and laboratory reported outcomes (LROs). LROs were the most frequently reported endpoints. Out of the 180 products here selected, 99 (55%), 67 (37%), and 30 (17%), respectively, used LROs, CROs and PROs as primary endpoints (PEs). PROs as any endpoints were used in 82 (46%) products. Out of these, PROs were documented as PE in 30 (37%), with 27 (33%) products having used PROs both as primary and non-PEs. PRO usage was most frequently identified with nervous system and antineoplastic agents. During the study period, the use of all the three types of endpoints appeared to be static. Both the regulatory bodies and the industry should ensure complete and clear reporting of all endpoints used, including PROs, to improve transparency.

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“…proved product labels 2006-2010, identified that 24% were granted PRO claims [2], with a comparison of FDA and EMA labelling showing that the EMA is more likely than the FDA to approve PRO claims [3]. The majority of PRO data that is captured does not make it onto the regulatory approved label, this is particularly noted in oncology [4] which is currently being actively addressed by industry and regulators [5].…”

mentioning

confidence: 99%

Patient reported outcomes to support drug development decision making

Nixon

Wild

Muehlhausen

2015

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Assessment of PRO Label Claims Granted by the FDA as Compared to the EMA (2006–2010)

Cited by 72 publications

References 6 publications

Patient-reported outcome claims in European and United States orphan drug approvals

Patient-reported outcome claims in European and United States orphan drug approvals

Role of patient-reported outcomes and other efficacy endpoints in the drug approval process in Europe (2008–2012)

Patient reported outcomes to support drug development decision making

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