Cell and gene therapies are fast turning into a powerful engine of value creation for drugmakers and represent a paradigm shift in the treatment of some of the world’s most devastating and intractable illnesses. In the last five to ten years, for instance, we have witnessed innovative pharma investing deeply in high-risk frontier research involving stem cells and the harnessing of a patient’s own immune system to attack the onset of a disease.
This has become most apparent in fields like oncology where ultra-expensive, yet thoroughly transformative CAR-T therapies are displacing classic treatments and swiftly going mainstream. This featured section strives to interpret the disruptive impact of the gene revolution within medical science and examine its implications for payers, patients, healthcare providers and manufacturers.
On the industry side, we will look into how CRISPR technology, rather like a pair of molecular scissors, is for the first time enabling drug developers to edit genes with unprecedented ease and precision, and as such is radically expanding the parameters of biological-based medicine. So too, the difficulties of integrating cell and gene into conventional portfolios and the fresh challenges to be surmounted in manufacturing, supply chain management and the overall scale-up of this class of medicines.
On the institutional side, we analyze how payers and care providers are going about the tricky task of financing and integrating these cutting edge therapies: from the formulation of first-of-a-kind risk sharing models to the implementation of appropriate surround care. Also of note are the efforts to design an affordable off-the-shelf, gene-edited, universal CAR-T cell.
We finally explore how cell and gene therapy lies right at the heart of the new wave of personalized, precision medicine (PPM) being pioneered across mature pharma markets around the globe, and the ongoing attempts by regulators to rethink the clinical trials and health technology assessment frameworks to adequately take into account the intricacies of these novel treatments, while, at the same time, mitigating possible ethical concerns around genetic modification.