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A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved by the FDA and the EMA (2012-2016).
Value Health. 2019 02; 22(2):203-209.VH

Abstract

OBJECTIVES

To compare US Food and Drug Administration (FDA) and European Medicines Agency (EMA) labeling for evidence based on patient-reported outcomes (PROs) of new oncology treatments approved by both agencies.

METHODS

Oncology drugs and indications approved between 2012 and 2016 by both the FDA and the EMA were identified. PRO-related language and analysis reported in US product labels and drug approval packages and EMA summaries of product characteristics were compared for each indication.

RESULTS

In total, 49 oncology drugs were approved for a total of 64 indications. Of the 64 indications, 45 (70.3%) included PRO data in either regulatory submission. No FDA PRO labeling was identified. PRO language was included in the summary of product characteristics for 21 (46.7%) of 45 indications. European Organisation for Research and Treatment of Cancer and Functional Assessment of Cancer Therapy measures were used frequently in submissions. FDA's comments suggest that aspects of study design (eg, open labels) or the validity of PRO measures was the primary reason for the lack of labeling based on PRO endpoints. Both agencies identified missing PRO data as problematic for interpretation.

CONCLUSIONS

During this time period, the FDA and the EMA used different evidentiary standards to assess PRO data from oncology studies, with the EMA more likely to accept data from open-label studies and broad concepts such as health-related quality of life. An understanding of the key differences between the agencies may guide sponsor PRO strategy when pursuing labeling. Patient-focused proximal concepts are more likely than distal concepts to receive positive reviews.

Authors+Show Affiliations

RTI Health Solutions, Research Triangle Park, NC, USA. Electronic address: gnanasakthy@rti.org.RTI Health Solutions, Research Triangle Park, NC, USA.RTI Health Solutions, Research Triangle Park, NC, USA.Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.RTI Health Solutions, Research Triangle Park, NC, USA.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't
Review

Language

eng

PubMed ID

30711065

Citation

Gnanasakthy, Ari, et al. "A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved By the FDA and the EMA (2012-2016)." Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research, vol. 22, no. 2, 2019, pp. 203-209.
Gnanasakthy A, Barrett A, Evans E, et al. A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved by the FDA and the EMA (2012-2016). Value Health. 2019;22(2):203-209.
Gnanasakthy, A., Barrett, A., Evans, E., D'Alessio, D., & Romano, C. D. (2019). A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved by the FDA and the EMA (2012-2016). Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research, 22(2), 203-209. https://doi.org/10.1016/j.jval.2018.09.2842
Gnanasakthy A, et al. A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved By the FDA and the EMA (2012-2016). Value Health. 2019;22(2):203-209. PubMed PMID: 30711065.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved by the FDA and the EMA (2012-2016). AU - Gnanasakthy,Ari, AU - Barrett,Amy, AU - Evans,Emily, AU - D'Alessio,Denise, AU - Romano,Carla DeMuro, PY - 2018/04/12/received PY - 2018/08/27/revised PY - 2018/09/25/accepted PY - 2019/2/4/entrez PY - 2019/2/4/pubmed PY - 2019/3/23/medline KW - European Medicines Agency (EMA) KW - Food and Drug Administration (FDA) KW - labeling KW - oncology KW - patient-reported outcome (PRO) SP - 203 EP - 209 JF - Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research JO - Value Health VL - 22 IS - 2 N2 - OBJECTIVES: To compare US Food and Drug Administration (FDA) and European Medicines Agency (EMA) labeling for evidence based on patient-reported outcomes (PROs) of new oncology treatments approved by both agencies. METHODS: Oncology drugs and indications approved between 2012 and 2016 by both the FDA and the EMA were identified. PRO-related language and analysis reported in US product labels and drug approval packages and EMA summaries of product characteristics were compared for each indication. RESULTS: In total, 49 oncology drugs were approved for a total of 64 indications. Of the 64 indications, 45 (70.3%) included PRO data in either regulatory submission. No FDA PRO labeling was identified. PRO language was included in the summary of product characteristics for 21 (46.7%) of 45 indications. European Organisation for Research and Treatment of Cancer and Functional Assessment of Cancer Therapy measures were used frequently in submissions. FDA's comments suggest that aspects of study design (eg, open labels) or the validity of PRO measures was the primary reason for the lack of labeling based on PRO endpoints. Both agencies identified missing PRO data as problematic for interpretation. CONCLUSIONS: During this time period, the FDA and the EMA used different evidentiary standards to assess PRO data from oncology studies, with the EMA more likely to accept data from open-label studies and broad concepts such as health-related quality of life. An understanding of the key differences between the agencies may guide sponsor PRO strategy when pursuing labeling. Patient-focused proximal concepts are more likely than distal concepts to receive positive reviews. SN - 1524-4733 UR - https://neuro.unboundmedicine.com/medline/citation/30711065/A_Review_of_Patient_Reported_Outcomes_Labeling_for_Oncology_Drugs_Approved_by_the_FDA_and_the_EMA__2012_2016__ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1098-3015(18)36165-5 DB - PRIME DP - Unbound Medicine ER -