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Gene therapies: The cost to cure is worth it

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Headlines can be misleading, especially when it comes to the price of innovative medicine, and Congress should take note.

Take for instance, recent news coverage of a new gene therapy treatment, known as Zolgensma, which the Food and Drug Administration (FDA) approved on May 24. This novel medical innovation is promising – for the first time – to cure children with a rare disease called spinal muscular atrophy.

The other good news is that Zolgensma is but one of many gene therapies anticipated to reach patients in the near future. According to research by MIT, 15 to 30 new gene therapies are expected to launch within the next five years.

Of course, you must look past the salacious headlines about Zolgensma’s $2.1 million price to recognize the sky isn’t falling. In fact, for the health care system and patients alike, we are at the dawn of a promising new era of medicine.

That’s because these exciting new therapies, which can address the underlying causes of genetic diseases as opposed to treating the symptoms, are often one-time treatments that can cure a disease, avoiding a lifetime of ongoing treatment.

These cures are generally expected to be associated with high price tags. But with so many therapies about to come to market, questions about cost sustainability loom large. Rightly so, stakeholders, including the government, payers, and consumer groups, should be most concerned with how these treatments will be covered, who will be paying for them, and how our health care system can adapt to support getting these cures to the patients who desperately need them.

While the upfront costs for these cures may seem high, it appears that much of the current debate among some health care critics fails to account for the high cost of chronic care our system already supports.

Take, for example, Chronic Granulomatous Disease (CGD), a rare disorder that causes a patient’s immune system to malfunction. Right now, many patients with CGD take Actimmune, a more traditional chronic treatment, three times a week to manage it, which results in $627,840 in medication costs per year. And this does not include the cost of quarterly liver function monitoring, and other health care costs that may be associated with chronic care.

Several public and private insurance companies cover Actimmune, costing insurers millions of dollars over a single patient’s lifetime. Thankfully, there are several gene therapies in the pipeline that may provide a one-time cure for CGD. Even if an approved gene therapy has an initial cost of $2 million, that therapy would present the most cost-efficient treatment option after just over three years, compared to current chronic treatment with Actimmune.

Yet, stakeholders claim the high upfront costs of gene therapies are unsustainable. While the initial costs may invoke sticker shock, these gene therapies certainly are more affordable when weighed against costly, long-term treatments. This affordability measure doesn’t even consider the additional value gene therapies could provide to patients, including improved quality of life or a chance at surviving a disease where no current treatment option exists.

To capture the promise of gene therapies versus the current costs of treating diseases and disorders chronically, the American Consumer Institute compared the current long-term costs for several rare diseases to an assumed $2 million cost for a one-time gene therapy cure.


Current Treatment

Gene Therapy


Severe Combined Immune Deficiency



2.0 years

Paroxysmal Nocturnal Hemoglobinuria



2.9 years

Hereditary Angioedema



3.8 years

Hunter Syndrome



4.0 years

Spinal Muscular Atrophy

$750,000 (Year-1); $375,000 (annually)



4.3 years

What becomes clear is that gene therapies will actually save our health care system precious dollars, and in many cases will do so very quickly.

The cost efficiencies do not stop at rare diseases. Consider hemophilia, which affects 20,000 Americans and costs each individual approximately $270,000 to manage each year. This means our health care system is paying $5.4 billion per year in medications alone to manage the symptoms of the disease, which one day could be cured with a gene therapy.

Beyond pharmaceutical treatments, surgical procedures are another example of the high costs already being paid by the system, with no guarantee of effectiveness. On the other hand, proposed gene therapy treatments are expected not to demand full payment upfront, but rather outcome-based payments tied to the clinical effectiveness of the therapy.

Chronic treatment of symptoms has been the standard in our health care system, and the system continues to pay high costs for chronic treatments and procedures without serious deliberation or consternation. So then why, when presented with a curative option, are we skeptical of a high price and fail to recognize the long-term value and cost savings?

As I mentioned in a co-authored study earlier this year, high up-front costs should not deter policymakers and payers from considering gene therapies viable, and many times, preferable interventions. Rather, all stakeholders should work together to think creatively to determine the best payment structure. Congress should not allow cures to be kept away from patients because we can’t determine the best way to pay for them.

Steve Pociask is president of the American Consumer Institute, a nonprofit educational and research organization. For more information about the Institute, visit www.TheAmericanConsumer.Org or follow us on Twitter @ConsumerPal.

Tags Gene therapy Health care costs

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