Value of Patient-Reported Outcomes Data in Food and Drug Administration Labeling of Oncology Drugs to Payers in the United States
Author(s)
Stephen Ijioma, MSc, PharmD1, Heiko Zettl, PhD1, Nicholas Liu, PharmD2, Audra Gold, MSc3, Mark Price, MA, MEd3, Marci Clark, PharmD3.
1Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, 2Boehringer Ingelheim, Ridgefield, CT, USA, 3RTI Health Solutions, Durham, NC, USA.
1Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, 2Boehringer Ingelheim, Ridgefield, CT, USA, 3RTI Health Solutions, Durham, NC, USA.
OBJECTIVES: The value of patient-reported outcomes (PRO) data included in oncology drug labelling by the United States (US) Food & Drug Administration (FDA) in US payer decision-making is not well understood. The objective of this study was to provide insights into how PRO-based FDA-approved product labeling statements are used by US payers when reviewing and approving new oncology drugs for formulary inclusion.
METHODS: A review of 2 FDA-approved non-small cell lung cancer drug labels including PRO claims and grey and targeted literature searches yielded insights into US payer review processes for new oncology drugs and considerations of PRO data. Literature search findings were used to inform development of a semi-structured discussion guide. One-hour virtual qualitative interviews were conducted with payers/decision-makers (i.e., representatives of regional and national private and public organizations) to gain insights into the perceived value of PRO label data.
RESULTS: Six US payers (1 chief medical officer, 1 former group head, and 4 medical directors) were interviewed separately. Most agreed that PRO data are not a primary driver in formulary decision-making, with clinical safety and efficacy data remaining the gold standard evidence. PRO data are often part of the value proposition, and their absence may be viewed as conspicuous or incomplete. Connecting PRO data to cost savings may be influential. US payers agreed PRO data published in peer-reviewed journals are valued and noted that FDA approval and product inclusion in National Comprehensive Care Network guidelines are the primary influencers of plan coverage and formulary status. PRO label claims may have more influence on oncologists’ drug selections, providing important context of the patient experience.
CONCLUSIONS: While unlikely to directly influence US payer decisions about plan coverage, PRO data in FDA-approved labeling for US oncology drugs may contribute to the overall evidence package, add important context to the value proposition, and inform drug selection.
METHODS: A review of 2 FDA-approved non-small cell lung cancer drug labels including PRO claims and grey and targeted literature searches yielded insights into US payer review processes for new oncology drugs and considerations of PRO data. Literature search findings were used to inform development of a semi-structured discussion guide. One-hour virtual qualitative interviews were conducted with payers/decision-makers (i.e., representatives of regional and national private and public organizations) to gain insights into the perceived value of PRO label data.
RESULTS: Six US payers (1 chief medical officer, 1 former group head, and 4 medical directors) were interviewed separately. Most agreed that PRO data are not a primary driver in formulary decision-making, with clinical safety and efficacy data remaining the gold standard evidence. PRO data are often part of the value proposition, and their absence may be viewed as conspicuous or incomplete. Connecting PRO data to cost savings may be influential. US payers agreed PRO data published in peer-reviewed journals are valued and noted that FDA approval and product inclusion in National Comprehensive Care Network guidelines are the primary influencers of plan coverage and formulary status. PRO label claims may have more influence on oncologists’ drug selections, providing important context of the patient experience.
CONCLUSIONS: While unlikely to directly influence US payer decisions about plan coverage, PRO data in FDA-approved labeling for US oncology drugs may contribute to the overall evidence package, add important context to the value proposition, and inform drug selection.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR236
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling, Reimbursement & Access Policy
Disease
Oncology