“FDA Challenges and Obstacles
“We heard a resounding chorus frommost stakeholders of the enormouschallenges with the regulatory burdensplaced on cell therapy in general, andstem cell therapy even more so, by theFDA. Instead of the ever growing bodyof work in cell therapy, with its overallexcellent safety record, making thepathway to approval smoother, it seemsto many that the requirements imposedby the FDA are increasing.
“A recent therapy touted by the FDAas a success had such a high clinicaldevelopment burden placed on it thatby the time it was finally approved,standard of care had evolved andits market was significantly reduced,leading to liquidation of the company.Companies, and sadly patients, mustgo outside the United States, as faraway as Japan, to find regulatoryagencies willing to work successfullytowards approval.
“Ultra-orphan diseases still must reachthe same statistical burden, whichrequires larger effect sizes than seenin the majority of approved, successfuldrugs and biologics. Everyone hastheir own list of how the FDA makesit seemingly impossible to take stemcell therapy through the regulatoryprocess. In 2014, Japan recognizedthe differences in regulatory approachesneeded for regenerative medicine andtook action. However, the FDA does notappear to have the same motivation. Infact, in a disturbing turn of events, FDAhas recently began providing certainmembers of the U.S. Congress withcompletely one-sided information on thedangers of cell therapies encounteredin clinical trials. However, FDA failedto provided any context or balancedinformation regarding the safety recordof cell therapies that comprises the vastmajority published clinical literature. TheFDA appears to be literally lobbyingagainst the very therapeutic modalitythey are responsible for promoting.
“CIRM needs to join with Congress,academia, and patients, to bring aboutreal change to meaningfully balancerisk-benefit in FDA regulations andmore importantly, the FDA’s behaviorand willingness to grant licensure toeffective therapies.”
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