Poster Presentation Eradicate Cancer 2018

FDA approved the first two CAR-T therapies in 2017, offering new hope for cancer patients with treatment refractory disease and confirming the maturation of the cellular immunotherapy modality from research to commercial/clinical readiness. 

Transformational therapeutic outcomes will however require transformation in manufacturing and supply chains, research translation and funding. The centralised, big batch, make-to-stock, sell-and-forget pharmaceutical supply chains of today are inadequate. Supply chains must accommodate just in time production initiated only when a clinician identifies an eligible patient and manufacturing confirms an available production slot. Clinical sites become part of the regulated supply chain. Manufacturing is labour intensive and in many cases each production run is for a single patient treatment. New translational research approaches are emerging to accommodate these new supply chains, and a highly variable, living therapeutic product with a very long duration of action. Production costs are significantly higher than traditional pharmaceuticals and prices are extremely high, frequently US$250,000+ per treatment before hospital costs. Potentially curative outcomes are driving pay-for-performance models.

Is Australia ready to take full advantage of this therapeutic and technical revolution? Australia has a proud history in cell therapy and cellular immunotherapy research, however lacks the commitment to succeed being shown by other countries and lacks critical pieces of supply chain capability essential to ensure early adoption of late stage and commercial products here.

By making a commitment to a “cell and gene therapy ready” Australia, and investing a modest $100m in three initiatives, Australia can reap a triple bottom line benefit from this revolution in the form of:

1. >500 direct advanced manufacturing jobs in the near term, potentially double this in the mid-term and significant spill-over jobs, all difficult to move off-shore;
2. Improved, earlier access to cost effective, breakthrough therapies delivering transformational health outcomes for >3,000 Australian terminal blood cancer and single genetic defect patients in the near term alone; and
3. Improved translational research efficiency and company formation providing greater economic return on the world-class science and substantial dollars invested in R&D over previous decades.

The three initiatives are:

1. Establishing commercial scale cell and gene therapy manufacturing and supply chain capability (cell processing, viral vectors, clinical centres of excellence, critical materials security)
2. Establishing a national “venture catalyst” organisation as the focal point for research translation and commercialisation
3. Market access (regulatory and reimbursement) innovation