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Go-to-market Challenges of CAR-T Therapies

CAR-T therapies are set to revolutionise cancer treatment. With the first curative therapies gaining market access across the globe, CAR-T therapies are subject to numerous commercial and non-commercial challenges. Despite positive clinical responses, the ability to overcome these challenges will determine the future of CAR-T therapies and show whether pharmaceutical companies can make true on the promise of initiating a new era of cancer care.

Setting the Scene

In 2019, the commercial pharmaceutical industry recorded global sales of ~1300bn USD and is expected to grow at a CAGR of 6.8% until 2024. Even though the majority of sales are still generated with traditional single-compound medicines, biologics are the major growth driver of the pharmaceutical industry. The development of simple compound medicines has reached its limits and research in this area is almost at peak. Today, biologics are the most promising technology which, combined with increased knowledge of medicinal chemistry and new methods of production, enable companies to develop completely new drugs and treatments.1 In 2019, biologics accounted for ~21% of global drug sales2 and represented 21 of 69 Drug License Application Approvals granted by the FDA.3,4

Within the field of biologics, advanced therapies have emerged as the innovative spearhead focusing on novel curative treatments. They comprise cell and gene therapies, as well as tissue engineering, as shown in Figure 1.5 Curative cell therapies today are mostly oncology-focused treatments where genetically modified T-cells are transferred into a patient to target a specific protein and destroy the malignant cells. Gene therapies are treatments that focus on delivering therapeutic DNA into a patient´s cells to cure the underlying disease. Current methods comprise the editing of genetic material, the addition of genetic material and the targeted silencing of genes. Tissue engineering is the umbrella term for therapies that restore, maintain and/or improve damaged tissue or organs by combining scaffolds, cells and biologically active molecules.6

As illustrated in Figure 1, there are currently ~1000 registered clinical trials for advanced therapies, accounting for ~15% of all clinical trials conducted globally. The trial landscape is coined by gene therapies focussing on rare diseases across numerous indications and curative cell therapies focussing on oncology. The majority of the ~625 cell therapy trials is investigating the potential of CAR-T treatments, which are regarded to be a revolution in cancer therapy.7

CAR-T Therapies at a Glance

CAR-T therapy refers to chimeric antigen receptor (CAR)-T-cell therapy, an innovative and individualised cancer treatment method that combines the capabilities of cell, immune and gene therapy into one therapy concept. T-cells are ex-vivo genetically modified to express a chimeric antigen receptor on their surface that recognises and binds to a specific antigen on the surface of malignant cells. Once the receptor binds to an antigen, the T-cell is stimulated to attack and destroy the malignant cell. Due to the fact that CARs have the ability to combine both antigen-binding and T-cell-activating functions into a single receptor, they are defined as chimeric.8,9