We are excited to launch our newest blog series: Gene Therapy with CooperGenomics. Think of it like FDR’s fireside chats, only online rather than over the radio, and with a focus on hot topics in genetics instead of New Deal Initiatives and World War II. One of our genetic counselors will be discussing a different genetics article each month.
The first article in this series discusses navigating the current healthcare system so that gene therapy treatments are actually available to the people who need them.
The debate over healthcare in the United States has been a long-standing issue. Countless solutions have been suggested, with the specifics dependent on which involved party is offering them. Everyone can, at the very least, agree that there is no quick and easy fix. Throw in the mix an altogether new medical treatment option, namely gene therapy, and confusion and frustration levels are bound to increase exponentially.
Gene therapy does not only promise to change the lives of countless people affected by rare genetic diseases, it can potentially cure them. The first clinical trial for a gene therapy was in 1990. Since that time, numerous gene therapies have been in the works, including Kymriah, Yescarta, Glybera, and Strimvelis. Later this month, the Food and Drug Administration is expected to approve the first gene therapy, Luxturna, for the treatment of patients with inherited retinal disease caused by changes in the RPE65 gene.
Numerous parties, including patient advocates, payers, manufacturers, healthcare providers, and experts on regulatory affairs, law, and policy, are working together now to guarantee that plans are in place to ensure that patients can actually receive the new treatments. As it turns out, there is a reason why healthcare has been a long-standing issue. There is a lot that goes into the development, production, and validation of a medication.
Some parties have tried to model coverage of gene therapy after existing medical treatments, such as organ transplantation. Unfortunately, this model isn’t perfect as factors including upfront costs, market size, and the potential for booster treatments complicate the issue.
The practicality of gene therapy still has a long and bumpy road ahead of it, but at least the journey has begun. So buckle up!