3:39 PM
 | 
Dec 22, 2017
 |  BioCentury  |  Regulation

Preference pioneers

How Rituxan Hycela became the first drug with patient preference data on its U.S. label

Rituxan Hycela subcutaneous rituximab provides the first example of how companies can get patient preference data on an FDA-approved drug label.

The new formulation is intended to reduce the treatment burden of rituximab therapy, which was previously available only as an IV infusion that took 1.5-6 hours. The subcutaneous formulation can be administered by a healthcare professional in 5-7 minutes.

In addition to pharmacokinetic bridging studies and head-to-head Phase III studies that showed no difference between the safety and efficacy of the two formulations, Roche conducted the Phase IIIb PrefMab study to generate data about patients’ experiences and preferences.

“Typically in a clinical trial, we collect a lot of safety and efficacy information. But rarely do we ask patients what it’s like to undergo treatment, how they experienced it and how it impacted their life,” Nancy Valente told BioCentury. Valente is VP of global product development in hematology and oncology at Roche’s Genentech Inc. unit.

“Rarely do we ask patients what it’s like to undergo treatment, how they experienced it and how it impacted their life.”

Nancy Valente, Genentech

PrefMab met the primary endpoint assessed by the Patient Preference Questionnaire (PPQ), a set of three questions that asked which formulation patients preferred, how strongly and why. Over three-quarters of patients in PrefMab preferred Rituxan Hycela over Rituxan on the PPQ.

Valente said Roche wanted to ensure the data were robust enough to submit to regulators and payers to help make the case for access, but the company didn’t design the study expecting that its data would make the label.

FDA concluded patients could make use of the preference information and placed the PPQ data in a new section of the label called “Patient Experience.”

“We expected that patients might want to know about the experiences of patients receiving the product through this route of administration,” said FDA spokesperson Jeremy Kahn in an email to BioCentury.

Learning a new instrument

Valente said the 743-patient study, which enrolled treatment-naïve patients with diffuse large B cell lymphoma (DLBCL) or follicular lymphoma, is the largest preference study conducted in oncology.

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