Patients at the core

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

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.

The coreHEM endpoints and the process used to select them were published in Haemophilia on May 21.

“We felt we had a unique opportunity here where the precompetitive space could align on not just what regulators want, but also what would be important to clinicians in their decision-making and for payers and how they would reimburse it,” said Mark Skinner, a hemophilia patient and a consultant to the National Hemophilia Foundation.

“We felt we had a unique opportunity here where the precompetitive space could align.”

Mark Skinner, hemophilia patient

In total, there were 39 organizations represented on the voting panel including patient groups, gene therapy companies, regulators, payers and health technology assessment (HTA) agencies, and researchers (see “coreHEM Voters”).

The coreHEM group used a process that gave priority to patients’ concerns, ensuring the inclusion of endpoints that patients considered critical but that were not rated as highly by other stakeholders (see “Assembling the Core”).

The resulting coreHEM outcomes include the traditional Phase III primary endpoint of bleed frequency. coreHEM adds improvements in mental health status and chronic pain, which were considered critical by patient representatives on the voting panel.

It also includes outcomes that are specific to gene therapies, such as the duration of factor expression and factor activity level. And it adds utilization of the healthcare system measured via direct care costs.

Participating companies had to agree in advance to use the endpoints.

Endpoint selection was just the first step, however. The group must

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