California's Affordability Plan for Cell/Gene Therapy Could be National Model

CIRM proposal nears launch in two clinical award rounds totaling $295 million

CIRM consulted with BlueRidge Life Sciences to develop a plan aimed at helping the agency meet the voters’ mandate that the state agency make its potential treatments affordable and accessible. CIRM/Blueridge graphic

California’s $12 billion stem cell and gene therapy program is on track to delve deeply into matters such as pricing studies for multimillion-dollar therapies, as well as patient “pain points,” as it moves to fulfill a voter mandate to make its potential therapies affordable and accessible.

Over the next few years, the effort is likely to take the state program ever deeper into the business details of commercialization, as well as the science of the programs it funds. In the process, the California Institute for Regenerative Medicine (CIRM), as the state agency is officially known, will generate a pathway that could serve as a model nationally.

“The goal is to support thoughtful planning across CIRM’s preclinical and clinical-stage programs to ensure that innovative treatments reach the patients who need them most,” said a CIRM memo prepared for a public, online meeting on Wednesday..

“Early understanding of the product’s economic value to health systems and key stakeholders is important for successful adoption in the market,” the memo said. It also called for the creation of a plan to “ensure the ‘right’ data will be available for regulators and reimbursement authorities to justify value and pricing.”

The CIRM effort includes a scorecard to evaluate grantees’ activities aimed at commercializing research. The scorecard will include milestones for the work. Failing to meet milestones on CIRM awards can result in the loss of funding.

Another feature of the CIRM plan is