ARTICLE | Product Development

Patients at the core

How patients, payers, companies identified core outcomes for hemophilia gene therapies

May 25, 2018 1:01 PM UTC

With excitement mounting for the seven hemophilia gene therapies in the clinic, the National Hemophilia Foundation wanted to avoid a scenario in which patients, physicians and payers had no good way to compare them.

The foundation therefore teamed up with the Center for Medical Technology Policy (CMTP) and McMaster University to assemble a precompetitive collaboration to develop a standardized set of endpoints and ensure that stakeholders would commit to using them...