Granting conditional marketing approval would allow cell therapy developers to recoup some of the high costs of manufacturing, Jacques Galipeau, chair of the MSC Scientific Committee at industry think tank ISCT, told us.
“The cost of goods per patient is orders of magnitude greater than that of a pill [and] this must be borne by the entity striving to test safety & efficacy of cell technology,” he told us.
And with many cell technologies being developed by academic health centres or small biotechs “the very high upfront costs of manufacturing therefore becomes a substantial obstacle to engage in innovation and development of an emerging technology.”
“Early conditional approval of therapies deemed safe, yet still in the process of more onerous ‘efficacy’ studies may allow for a financially viable mechanism - eg mitigation of cost of goods - to demonstrate efficacy and spur activity in this space.”
He continued: “A streamlined manufacturing cost recovery regulatory path which at least permits accelerated access to promising cell technologies to accredited hospitals would help all players in this space address the unmet needs of patients.”
His comments come off the back of the REGROW Act, proposed US legislation introduced in March which is looking to expedite cell therapy development through regulatory pathway 351 and 361 of the Public Health Service Act.
While ISCT is in favour of efforts to provide affordable and accessible cell therapy treatments to patients, the group opposes several features in the Act it believes fail to do this.
“As written, the Act relaxes the regulatory requirements for 351 technologies in a manner that allows conditional FDA marketing approval without scientific evidence of efficacy, as per current standards,” Miguel Forte, chair of the ISCT’s Commercialisation Committee, told Biopharma-Reporter.com
But while“the legislation aims to promote faster access to new therapies, the method in which it has proposed access is inadequate and incomplete.”
He said that treatments for patients should have adequate documentation of the safety and efficacy on a proper risk-benefit assessment. “This means that from a regulatory perspective it should not be a significant difference.”
“The question is the adequacy of the assessment of the risk-benefit and the opportunity to provide patient access to new medications with an advantage of that being done within the system even if the system could be improved with faster decision and potentially less risk-aversion.”