Novel cell and gene therapy options are significantly improving treatment outcomes, and in some cases providing cures via one-time treatment to rare diseases previously considered untreatable. While these one-time therapies, particularly when curative, have the potential to be beneficial to patients with serious life-threatening diseases, a number of characteristics differentiate them from conventional chronic therapies:
- One-time therapies typically treat an existing prevalent pool of patients at launch.
- One-time therapies typically have treatment regimens of significantly shorter duration relative to chronic therapies.
- The available patient population diminishes, sometimes rapidly, over the course of the curative therapy lifecycle as the pre-launch patient prevalent pool is depleted.
In this paper, we aim to present 7 disruptions and some mitigation strategies related to one-time therapies. Download the PDF to read the paper.