Medical innovation brings the potential to alter and even save lives. One recent category of innovation is gene and cell therapies, which came to market in 2010. 15 years later, their development shows no signs of slowing down.

While the medical upside of these therapies is significant, they come with significant price tags. They often top $1M before considering additional supportive care cost. Those responsible for pharmacy benefits should keep a close eye on this category. It is branching from its origins in rare and ultra-rare conditions to more common conditions with far larger eligible populations.

What are Gene and Cell Therapies

Gene and cell therapies both involve the potential to impact genetic and acquired diseases. The U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research approves gene and cell therapies. A complete list of approved therapies is updated regularly by the FDA and can be found on their website.

Most gene therapies have come to market with multimillion-dollar price tags. But it is important to note that these prices only encompass the actual therapy. Preparations for gene and CAR-T therapy sometimes include chemotherapy and supportive care with and after therapy. These can add upwards of $1M to the cost of treatment.

How can you respond?

With the high prices and potential risks of gene therapies, plan sponsors are looking for ways to protect their costs while also providing potential life-changing care for their members. There currently is no perfect solution, but a few options do exist:

  • Stop loss and reinsurance: this protection is becoming less reliable as vendors flag patients and preemptively increase lasers
  • Gene therapy solutions: third party vendors offer per member per month fee-based coverage for gene therapies, but some have significant exclusions
  • Work with manufacturers: some manufacturers offer outcomes-based contracts to reimburse costs when medications do not achieve the desired results
  • Tough decisions: for smaller plan sponsors, the cost of these therapies could cause financial collapse and they may have to exclude the category from their plans

Gene and Cell Therapy Outlook

With the potential for dozens of FDA approvals in the coming years, gene and cell therapies remain a top industry trend to monitor. So far in 2024, three agents have received approval. Three more agents have anticipated FDA approval dates and additional agent approvals are anticipated.

As gene and cell therapies move into more common disease states, the population eligible for these groundbreaking innovations will expand exponentially.

As innovation continues in a category with potentially plan-busting price tags, working with the right pharmacy benefits consultant is critical. It allows plan sponsors to mitigate risk and strike a balance between plan member care and pharmacy cost.

Our team monitors market trends and the pharmaceutical pipeline and the latest clinical research to ensure our clients receive the information and insights they need to make the best decisions possible for their plans.

Download the briefing below for a summary of the current landscape of gene and cell therapies, along with our recommendations.

Download the Briefing on Gene and Cell Therapies