Gene therapies can change lives, but sometimes the hurdles to receiving them can seem insurmountable. The reasons are twofold: cost and coverage.
When gene therapy is an option and is prescribed, plans and patients alike may be stunned by the cost. Take Type 1 spinal muscular atrophy (SMA). Babies are diagnosed with this type of SMA shortly after birth, and those who aren’t treated typically die before they reach their second birthday. Untreated, they eventually need specialized wheelchairs and are on ventilators around the clock. Gene therapy can alter the course of SMA in significant ways for these patients, but could cost millions.
When families face a rare and devastating diagnosis such as SMA, they should be able to rely on proper coverage. Instead, many are forced to scramble for information and assistance, including turning to GoFundMe or other campaigns to pay for the life altering drugs they require.
For patients (or their parents), fear and frustration
The journey to a diagnosis for a rare condition can be long and difficult: on average, 7.6 years.
In addition to identifying appropriate treatments, patients and their families often have numerous special needs. They may not be able to afford a wheelchair without help from community-based services, their homes may not be accessible without costly renovations, or they may require assistance figuring out the patient’s dietary restrictions.
The uncertainty can even extend to the family’s life decisions: Can parents change jobs without jeopardizing insurance coverage? Are their jobs in jeopardy due to time away from work or other issues related to a diagnosis?
Faced with the diagnosis of a rare or ultra-rare condition, some people try to navigate the health care system themselves, struggling to understand their coverage and options. Some families discover they don’t have coverage or lack adequate coverage for the medications they need. They may turn to social media in a desperate bid to raise money or contact newspapers and other traditional media, hoping to pressure their plan to cover the needed medications and services.
What plan sponsors can do
When families face a rare and devastating diagnosis, they should be able to begin treatment promptly, without worries about how to afford vitally needed medications.
By covering rare and gene therapies, plan sponsors can manage their own risk and control costs while enabling patients to access the therapies that help to treat or control their conditions. And while these therapies are known to be expensive, there are solutions that can help plan sponsors with protection and predictability, while keeping these medications in reach of patients.