Healthcare Innovation: Preparing For The Next Wave Of Promising Gene Therapies

Nov 16,2023

Read Time 4 Minutes

Gene therapy is a transformational success story in 21st-century healthcare. Rare and incurable genetic diseases, such as congenital retinal blindness and spinal muscular atrophy, are now being effectively treated by new genetic therapies. Gene therapy has the potential to treat conditions like cystic fibrosis, cancer, heart disease, diabetes, and HIV — all conditions that affect employee populations and drive considerable employer health costs.

Therapies are also being developed to treat more prevalent genetic conditions, like sickle cell disease, which affects more than 20 million people worldwide. With over 30 gene therapy treatments currently approved by the FDA and an anticipated 10 to 20 added each year, we are at the threshold of a groundbreaking era of genetic medicine.


As the use of gene therapy expands, employers will need to rely on innovative health plan partners to facilitate access to these promising treatments. A recent survey of benefits decision-makers revealed that 81% expect gene therapy affordability to be a significant challenge for their organization in the next two to three years. While employers want to offer these treatments to their employee populations, it will be balanced with a need for effective cost-control strategies.


What To Expect In The Next Two Years


1. A Growing Pipeline


Currently, six therapies targeting populations with a high unmet need are slated for approval in 2024:

  • EXA-cel is a one-time treatment using CRISPR therapeutics to treat two inherited blood disorders: sickle cell disease and transfusion-dependent beta thalassemia (TDT).
  • Lovo-cel targets sickle cell disease.
  • Upstaza™ is used in adults and children aged 18 months and older with severe aromatic L-amino acid decarboxylase (AADC) deficiency. AADC affects the nervous system leading to developmental delays, weak muscle tone, and an inability to control movement of the limbs.
  • Libmeldy® is used to treat children with early-onset metachromatic leukodystrophy (MLD), a rare inherited disorder that causes a progressive loss of motor and cognitive functions affecting the brain, spinal cord, and peripheral nerves.
  • BENEGENE-2 is a one-time treatment for people living with hemophilia B as a means of reducing the long-term clinical treatment burden. This rare genetic bleeding disorder occurs in approximately 1 in 5,000 live births in the U.S.
  • ABO-102 is used to treat Sanfilippo syndrome type B, an autosomal recessive, neurodegenerative disease in children, characterized by profound cognitive impairment and dementia.

A total of 1.09 million people are expected to be treated by gene therapies by December 2034, underlining the importance of staying vigilant about potential new treatments. CarelonRx, Anthem’s pharmacy benefits manager, continues to monitor the expanding drug and biologic pipeline, evaluating the potential efficacy of new treatments and keeping pace with healthcare innovation.


2. Groundbreaking Gene Editing Technology


In the near term, the FDA is considering the first treatments using Vertex Pharmaceutical’s CRISPR tool to edit — rather than just insert — genetic material. This technology is the very definition of healthcare innovation, as CRISPR treatments largely avoid the use of viruses. Instead, they directly make changes in the DNA, using targeted molecular tools. The technique has been compared to the cut-and-paste function in a word processing program, allowing scientists to remove or modify specific problem-causing genes. The company is on track to have two gene therapies approved for sickle cell disease by the end of 2024.


3. Innovative Financing And Healthcare Cost Support


Every bit as noteworthy as the clinical promise of gene therapies is their cost. By 2028, the global gene therapy market is expected to reach over $23.9 billion. This requires a fundamental rethinking of how treatment costs are managed — setting parameters around who can receive these types of treatments, as well as payment options to help cover the expenses.


Anthem’s Gene Therapy Solution works as an extension of its stop loss products to protect self-insured employers from unknown financial risk, while also providing robust care management for employees and their family members with rare and complex conditions. In addition to covering both treatments and medical costs, Anthem will not impose lasers (limited access and restricted services) on future gene therapies that are covered by the Anthem Gene Therapy Solution.


A Whole-Health Approach To Employee Care


People living with genetic diseases often experience impacts on their health and quality of life, including disabilities as well as mental and emotional challenges. To address this, Anthem takes a whole-health approach to care by bringing together health services, medical management, healthcare data, and specialty pharmacy expertise, including access to dedicated Nurse Care Managers who specialize in complex condition management.


Gene therapy’s transformative potential continues to evolve, and with a recent surge of clinical activity targeted at more common conditions, it is well-poised for further growth. Employers can prepare by exploring plan options and coverage strategies vital to connecting their employees with these transformative treatments — while at the same time, mitigating financial risk and protecting their bottom line.