Patient-reported outcomes in support of oncology product labeling claims: regulatory context and challenges

Abstract

The US FDA has advocated the patient-reported outcome (PRO) draft guidance as the main vehicle for evaluating PRO-based labeling claims for oncology drugs. In addition, FDA-affiliated researchers have identified factors inhibiting acceptance of health-related quality of life (HRQoL)-based claims for oncology product labels. The views of the FDA on PRO claims are extensive and prescriptive. By contrast, the European Medicines Agency (EMA) has conducted authorizations without an explicitly defined approach for evaluating HRQoL data. A reflective paper released in 2005 offered only broad recommendations on HRQoL labeling claims. The different approaches between the two regulatory agencies partly stem from underlying, divergent organizational characteristics. Moreover, general issues inherent in PRO research in oncology trials including trial design, missing data, multiplicity of end points and inconsistent findings of HRQoL data are discussed.

Keywords::

• EMA
• health-related quality of life
• HRQoL
• oncology product labeling claims
• patient-reported outcomes
• PRO
• US FDA

Acknowledgments

The views expressed in this article are the result of independent work and do not necessarily represent the views and findings of Mapi Values.

Financial & competing interests disclosure

The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.