Harmonizing Core Platforms In The Delivery of Patient-Specific Cell Therapies To Accelerate Patient Access To Life-Changing Treatments | Biomedgrid

As our knowledge of human biology has grown, the newly found information has been leveraged to devise increasingly complicated treatments. This began with small molecule treatments that impact cell functions and has led to the rapidly maturing biologics space that largely aims to upregulate and downregulate those functions. This, in turn, has led to the emerging field of cell and gene therapy, where the aim is to create, renew, or replace cell functions and directly affect human tissue. Significant excitement has led this field to evaluate its' benefits in a breadth of disease areas with leading candidates in oncology and congenital blood diseases. However, along with this anticipation comes questions about how the pharmaceutical industry will support these complicated and, therefore, expensive treatments to ensure patients are able to benefit from these potentially life-changing therapies. While the indications that are best treated by these medicines are still to be determined, clinical trials have shown enough early success to make the industry confident in the long-term clinical impacts of this therapeutic avenue. With that in mind, current therapies are sheltered from the institutional inefficiencies of their delivery pathways by servicing small patient populations in medically competent locales. To understand the influences on patient access, developers must look at the delivery pathway for cell and gene therapies from a needle to needle viewpoint, especially for autologous therapies, as seen in (Figure 1).