CRISPR-Engineered Gene Therapy for Sickle Cell Disease: Hidden Costs May Make Treatment Beyond Reach of Most Patients

While venture capital and then the public directed billions of dollars into complex, precision-based therapies such cell and gene therapies, the pathway to return on investment becomes a direct function of patient outcomes. A prerequisite for patient outcomes, fundamentally, are programs for access. If for example the products once approved by the U.S. Food and Drug Administration (FDA) or other national level regulators become too expensive then access to this new form of care becomes quite limited. If the process of accessing the gene therapy becomes too cumbersome, so too is the access to care limited. Also targeted gene therapies with a price tag of $2+ million dollars must have substantial outcomes, otherwise uptake by health systems will be limited. An interesting case in point—representing a troubling sign—is access to the recent FDA approved gene therapy Casgevy. A gene-based therapy for sickle cell disease designed and developed by both CRISPR Therapeutics and Vertex Pharmaceuticals, using gene editing technology.