Opinion: Lifesavers Are Killing With Costs

The market price for gene therapy greatly exceed the costs of the gene editing development and the equipment required for the task.

Michael Steck, OD

Gene therapies have the amazing potential to cure multiple diseases, save lives and give sight to blind children. But the justifications surrounding the costs for these turning-point technologies are laughable. These inflated prices, in an environment of already-crippling healthcare costs, could paralyze the US in the competitive global landscape.

In August, Novartis announced that its CAR-T therapy tisagenlecleucel (Kymriah), a revolutionary treatment for pediatric acute lymphoblastic leukemia, would cost in the neighborhood of $475,000. This tag comes in spite of a federal government investment of $200 million that funded early research of the therapy. It also comes in spite of gene editing equipment costing around $2,000, and plasmids starting at rates below $100.

In spite of the advances made in gene editing technology — in laboratories and clinical settings far beyond their own — Novartis is benefiting from a hiked price to save lives.

What’s worse is the justification some in our field have given to these exaggerated costs. After Spark Therapeutics priced its recently approved potential blindness healer voretigene neparvovec-rzyl (Luxturna) at $850,000, Steve Miller, MD, Chief Medical Officer of Express Scripts, described the decision as “responsible.” Promoting the potential cure for a form of blindness, while also making it inaccessible to most patients by cost is repugnant.

For decades, families, doctors and researchers have dreamed and searched for the cure for cancer and genetic diseases. Now that we are on the cusp of many radical breakthroughs for many of these conditions, the cost to access the therapies are prohibitive for most Americans. It is the responsibility of these companies to bring their product or service to market and have it be affordable, in order to maximize its therapeutic potential.

The cost needs to be related to the therapy’s development, with a profit that allows the company to manage its business and work on developing more therapies. Research and development for a biotech or pharmaceutical company is crucial for innovation. But expensive and misguided marketing campaigns for these potentially unaffordable therapies is diverting resources from R&D. This type of marketing does not promote good medicine, nor work toward the encompassing goal of personalized medicine. Instead, its expenses greatly exceed the costs of the gene editing development, and the equipment required for the task.

The ability to cure cancer or blindness is invaluable. Yet, a medical tourist from the United States can take advantage of a study on gene-editing technologies for cancer at a price of $80,000 and not $475,000. The cost should not be compared to, nor tied to, the cost of other existing therapies with worse outcomes. Nor should the costs be tied to what the families and payers will incur if the child is left blind or resigned to battle a disease that will otherwise kill them.

At these ludicrous prices, several undesirable things will likely happen: people will needlessly die, a black market will form, or regulations will be put in place to set the price.

It is untenable to put the onus on the healthcare insurance industry or the US taxpayer in paying for the therapy at these inflated prices through some sort of installment plan. The problem in healthcare is not how it is paid, but the exorbitant and arbitrary costs of products and services.

Capitalists believe incentives are needed for promoting innovations, driving profits and growing businesses. Experts recently estimated the US is currently paying for 70% on the cost for innovation in healthcare worldwide. Regulations in the US discourage innovation and remove incentive. People living in countries with socialized medicine have regulated pricing and brag of the lower costs and better outcomes for healthcare in their homeland. These same people criticize the US healthcare system for excess spending and lower quality outcomes — while in essence, we are subsidizing their costs.

In the foreseeable future, the increasing costs will be crippling to the US. Being broke and overregulated are causing the US to fall further behind other countries in innovation and in the quality of healthcare, and providing less funding for other necessities.

This is a math problem. On the pricing side of this equation, adjustments are needed to equilibrate the difference. On the spending side of the same equation, the funding for healthcare is limited. Healthcare costs and the rising rate at which these costs are outpacing inflation is unsustainable. We live in amazing times, and should remain optimistic about our future if we act with reason and compassion.

The American healthcare system is very sick. These therapies may very well cure diseases, but the means to get it will kill the patient.

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