Clinically Speaking: Gene Therapies: Million-Dollar Miracles

Gene therapies have the potential to treat – and, in some cases, cure – some of the rarest and most debilitating diseases people face. Many diseases with an approved gene therapy have few, if any, treatment options available so these therapies can be life changing.

For families managing one of these conditions, gene therapies bring much-needed hope, but also concerns around safety, durability and especially affordability. Their ultra-high prices can cost people and unprepared plan sponsors millions of dollars. Health plans and employers need the right strategies to make these medications affordable and accessible.

Gene Therapy Definition and Types

Gene therapies are highly targeted medications that edit or replace the faulty or missing genetic material that causes inherited medical conditions. Unlike medications that are taken repeatedly over time, gene therapies are administered once and are expected to provide a long-term benefit.

There are two types of gene therapies: Gene modification, which edits an individual’s DNA at the site of the gene defect by introducing new genetic code to fill a gap or fix a flaw, and cellular therapy, which involves removing cells from a person or a live donor, modifying the genes of those cells in a lab, and then introducing them back into the person. Gene therapies are administered in varying ways, usually requiring extensive training, specific handling procedures and equipment entrusted only to certified sites of care and medical professionals.

Gene Therapy History

While the concept of gene therapy has been around since the early 1970s, practical applications began only recently in the United States when the FDA approved two treatments in 2017. Today, more than 900 potential gene therapies are in the research and development pipeline. Some will be the first to treat the targeted disorder. Some will replace existing high-cost therapies. And some will be reserved for use when other options are exhausted.

Although many will never reach the approval stage, the FDA expects to approve 10 to 20 gene therapies each year by 2025 and today’s $5.2 billion gene therapy market is estimated to grow tenfold by 2031. As more gene therapies are approved, the ability to understand past utilization and project potential utilization and spend will be vital for plan sponsors to make more informed policy and coverage decisions.

Gene Therapy Challenges
Like all treatments, there are some health risks related to gene therapies. However, before they enter the market, they go through rigorous testing and must be FDA-approved. Also, due to the short time gene therapies have been used and the small patient populations with these rare disorders, there are questions about the duration of effectiveness, also known as “durability.” After 10 or 20 years, will individuals need a booster treatment? Or will they need to switch to different treatments?

Above all, plans and patients can be shocked by the cost. A recently approved medication for hemophilia topped $3.5 million for a single treatment —the most expensive drug ever approved. In the case of Type 1 spinal muscular atrophy, the top genetic cause of infant deaths, a one-time curative gene therapy costs $2.1 million. 

Without proper coverage, families are left scrambling for information and assistance at a time when they need to focus their energy on their loved one. Many are forced to crowd-source funds on the web and social media to pay for care.

Typically, the journey to a diagnosis for a rare condition can be long and difficult, taking seven to eight years on average. Individuals and their families often have special needs in addition to appropriate treatment. They may be limited in normal daily activities. Their homes may need costly renovations for accessibility. They may need help from community-based services to afford a wheelchair. And they may be worried about jeopardizing their jobs due to time away from work. Plan sponsors can manage their own risk and control costs, while enabling their covered employees or members and dependents to access gene therapies and the support resources they so desperately need.

Gene Therapy Action Steps

Although gene therapies are expensive, there are steps plan sponsors can take to secure protection and predictability, while keeping the treatments in reach of their members. Here are some key areas they should focus on:

• Monitoring and managing the gene therapy pipeline – Modeling the potential impact to your plan to help predict future costs. Providing exclusive access to gene therapies through a specialty pharmacy. Ensuring patient care is connected across the health care ecosystem. And controlling medication costs with outcomes-based guarantees. After all, for the newest gene therapies, outside of tightly controlled clinical trials, there may be a lack of evidence the treatments work in real-world settings. Plan sponsors need solutions to make sure they only pay for effective therapies.
• Utilization management – In some cases, gene therapies require utilization management to ensure they are used for the right patients at the right time, given the high cost. For example, in the case of hemophilia, gene therapies are only indicated for use in adults who have severe disease.  What’s more, gene therapies are typically adjudicated through the medical benefit, not the pharmacy benefit, so an integrated partner can help keep costs in check.
• A customized approach – Finally, with the complexities and therapeutic nuances specific to genetic conditions, each gene therapy requires a customized, case-by-case approach. The treatments pose new challenges in clinical, procedural, safety and storage support. Many require complex shipping and handling, time-sensitive delivery, and maintenance of an ultra-cold chain of negative 80 degrees Celsius from the manufacturer to the infusion location.

Plan sponsors are wise to demand logistical safeguards to make certain the medication reaches the patient’s site of care at the right time, in compliance with federal regulations, and with timely updates for the physician and patient.

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