Patient-Reported Outcomes Labeling for Products Approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014)

open access:
 JCO April 11, 2016 JCO636480
Patient-Reported Outcomes Labeling for Products Approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014)

Table 3 Oncology Products With Potential for PRO Labeling (FDA OHOP, 2010-2014) (Olaparib/Ovarian)

Although all 13 registration trials included established PROMs (Patient Reported Outcome Measures), only 5 (38.4%) were RCTs. Significant deficiencies noted by the FDA reviewers within the DAP included unacceptable levels of missing values, use of inappropriate PROMs, and a lack of clinical significance.

 Two major factors may contribute to the smaller number of PRO labeling instances in oncology drug development. First, oncology studies are significantly more likely to be small, single arm, and open label. Thus, these studies may not be perceived to be candidates for inclusion of PROs because of potential bias introduced by the open-label design.¹ Second, given that most oncology drugs are marketed first in the United States, oncology trials are likely to go through various regulatory pathways designed to speed up the development and regulatory process in the United States.²² Pressed for time and given the challenges involved in integrating PROs in clinical trials, sponsors may be unlikely to prioritize PRO end points.23,2

Conclusion Although symptoms and functional decrements are common among patients with cancer, PRO (Patient Reported Outcomes) labeling is rare in the United States, likely because of logistical hurdles and oncology study design. Recent developments within the FDA OHOP to capture PROs in oncology studies for the purpose of product labeling are encouraging.

 In conclusion, the small number of PRO labeling instances granted by the FDA OHOP is a reflection of the difficulties faced by regulators and the industry to capture the patient’s voice in oncology drug development. However, recent developments within the FDA, and particularly within OHOP, to better capture PROs in oncology studies for the purpose of product labeling is encouraging. Sponsors should also make every effort to capture PROs in cancer drug development, and product labeling should not be the only goal for including PROs in drug development. PROs should be included in development programs to capture a comprehensive evaluation of the study participants’ experience, which can provide useful information on the impact of the new therapy for patients, regulators, health care providers, caregivers, and payers who need to choose between competing therapies.