Gene therapy is a type of treatment that introduces genetic material to change the course of a disease. This approach can be accomplished by replacing a defective gene, modifying a defective gene, or providing a gene for which a patient may be missing. Some of these therapies have already been introduced in the US market (including treatment for cancer and pediatric blindness) with many more in development.
The latest in drug approval pipeline indicates that there may be many pending innovative treatments available for a variety of rare conditions over the next 1-3 years. In 2021 alone, there are 10 novel treatment options in late term clinical trials expected to be reviewed by the FDA and potentially launched in the area of oncology. Additionally, we anticipate the pipeline for 2022 to be even more robust, specifically in the area of hemophilia, which is a rare bleeding disorder for which there is no cure. Outlined below are some of the potential treatments that may receive approval.
Today, hemophilia is traditionally managed by supplementing patients with blood clotting factor, which their body does not naturally produce. This helps prevent any serious, uncontrolled bleeding events which can be life-threatening. Some patients require “factor” only as needed, and others require prophylactic, maintenance treatment. Today’s treatment options for factor vary – by delivery (IV administration, subcutaneous self-injection), by type, and duration of action (long acting vs. short acting). There are a number of cost and quality considerations to take into account when managing a hemophilia patient.
Let Arxcel help to quantify your financial exposure for future treatments of gene therapy by understanding your patient population.