A PROfessional trial

FDA: Incyte's Jakafi is exemplar for patient-reported outcomes

Cancer companies have struggled to gain FDA approvals based on patient-reported outcomes, or even to get data from a PRO instrument on a drug's label. Incyte Corp. accomplished both in November with Jakafi ruxolitinib for myelofibrosis, and the agency says other companies would be wise to follow the biotech's example.

The Jakafi experience shows how patient-reported outcome (PRO) tools should be integrated into drug development programs and rebuts concerns that doing so requires large investments of time or money, according to Richard Pazdur, director of FDA's Office of Hematology Oncology Products.

"It is remarkable that you had a small company with a relatively rare disease being able to address the whole issue of a PRO in an effective manner," Pazdur told BioCentury.

In November, FDA approved Jakafi to treat myelofibrosis based on two Phase III trials, including one that used total symptom score, a PRO measure that used a novel tool tailored to the myelofibrosis population, as a secondary endpoint.

Reduction in spleen volume was the primary endpoint in both trials. But Pazdur said the PRO was a decisive factor in FDA's decision to grant full approval to Jakafi.

Incyte's recipe for success was to follow the agency's 2009 guidance on PRO development, work with patients to develop the instrument, and start the PRO process early while the drug was in Phase I/II trials.

Starting early allowed Incyte to work with FDA for nearly two years to finalize the measurement tool and iron out an SPA for a Phase III trial. An SPA will often be necessary for companies tailoring a PRO to a new indication, Pazdur noted.

Two other factors were key to Jakafi's approval: Incyte ensured

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