Patient-Reported Outcomes More Common in EU Labeling than FDA Labeling of Drug Approved for Lung Cancer

Neil Canavan

June 2013, Vol 4, No 5 - Economics of Cancer Care


New Orleans, LA—A study comparing patient-reported outcomes (PROs) labeling claims in Europe and in the United States showed that the European Medicines Agency (EMA) is more proactive in considering patient-generated feedback in their drug labeling decisions—this despite the US Food and Drug Administration (FDA)’s 2009 guidance that emphasized the importance of gathering and disseminating such data.

“Our analysis suggests a higher receptivity of the EMA to quality of life as a global concept,” stated Martine Caron, PhD, Development Director at Mapi Research Trust, Lyon, France, and colleagues, who presented their data at the 2013 International Society for Pharmacoeco­nomics and Outcomes Research annual conference.

A PRO is any report that comes directly from patients about how they function or feel in relation to a health condition and its treatment; this patient feedback is not subject to the healthcare provider’s interpretation, but it is a true self-assessment. PROs are considered a vital adjunct in developing the paradigm of personalized medicine, especially in relation to side effects associated with the disease and its treatment. Properly utilized, PROs can be used to help direct patient care.

The investigators looked at the labels of 15 drugs approved by the FDA for the treatment of non–small-cell lung cancer, of which 6 were also approved by the EMA.

Of the 6 EMA-approved drugs, 4 had PROs claims in their labels—docetaxel (Taxotere), erlotinib (Tarce­va), gefitinib (Iressa), and paclitaxel (Paxene). Of the FDA-approved drugs, only 2 had PROs included in their labels—paclitaxel (Taxol) and gemcitabine (Gemzar).

For all the drugs with PROs labeling, claims were related to quality-of- life issues and symptoms. However, the FDA label for paclitaxel lacks a quality-of-life claim.

Except for gefitinib, both agencies granted or denied PROs claims based on data from the same clinical trials; to explain the different decisions made by the FDA and the EMA, the study investigators looked at the agencies’ communications. For the most part, the FDA expressed concerns about quality-of-life study designs and any related analyses.

Will the Trend Continue?
A presumed reluctance on the part of the FDA to grant PROs labeling actually may not be the case. A review published in 2012 found no PROs-containing labels for the 15 oncology agents approved by the FDA between 2006 and 2010 (Gnanasakthy A, et al. Value Health. 2012;15:437-442); however, 3 reasons were suggested to explain the drug manufacturer's role:

  1. The FDA often issues warning letters when PROs are “overemphasized”
  2. Drug manufacturers do not want to spend the extra money in early drug development to collect data on nonprimary (ie, PROs) end points
  3. Increasing use of multinational trials poses too many hurdles for the manufacturer to collect PROs data.

However, 24% of recently approved drugs have PROs labeling—just not in oncology. In oncology, PROs are frequently used to assess treatment benefits and toxicity to evaluate the impact of treatment on quality of life.