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

The US FDA has advocated the patient-reported outcome (PRO) draft guidance as the main vehicle for evaluating PRO-based labeling claims for oncology drugs.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.