August 2019 

Will gene therapy for HIV cause financial toxicity?

The technology of gene editing through CRISPR has the potential to clear people’s cells of HIV’s genetic material, treat some cancers and correct inherited disorders such as hemophilia.

The different teams of scientists working to apply CRISPR or other forms of gene therapy for treating diseases in humans issue press releases from time to time that are often reproduced by some media. What is not mentioned in those press releases is the cost of CRISPR treatment should it one day be approved by regulatory agencies in Canada and other high-income countries.

The high cost of cancer treatment

CRISPR is cutting-edge technology and is likely to be expensive. To begin to form an idea of what CRISPR might cost, it may be worth looking at the cost of cutting-edge cancer treatment. The list prices for some approved immune-based therapies (so-called “checkpoint inhibitors”) for cancer are between US$100,000 and $200,000 per person per year. The cost can vary depending on the checkpoint inhibitor used, the dose administered, and whether or not it is prescribed in combination. Some people may require at least two years of treatment.

Looking at immune-based therapies for cancer that include some degree of genetic manipulation, such as Kymriah (produced by Novartis), list prices are higher still: around US$500,000 per person per year, not including the high cost of managing side effects. The high price of medicines, particularly the latest treatments for cancer, causes what some researchers refer to as “financial toxicity” for patients. By “financial toxicity,” the researchers mean that the catastrophic expense of cancer care has the potential to “diminish quality of life and impede delivery of the highest quality care”. They note that “existing data have identified both objective financial burden and subjective financial distress as key components of financial toxicity.”

The issue of cost is one that will bedevil the emerging field of gene therapy.

Gene therapy—the world’s most expensive drug?

In May 2019, the U.S. Food and Drug Administration (FDA) approved the sale and use of a gene therapy called Zolgensma. This therapy is meant to be a one-time treatment for people with spinal muscular atrophy, an inherited condition that occurs in about 1 in every 11,000 births. The pharmaceutical company Novartis has priced the gene therapy at around $US 2.1 million per person—$425,000 a year spread over five years. This is possibly the world’s most expensive drug. According to Novartis, the corporation is “working closely with insurers to create five-year agreements based on success of the treatment as well as other novel pay-over-time options.”

At this time, absent a major breakthrough, it is unlikely that a quick, simple and safe method of curing HIV will become widely available in the next five years. However, should regulatory agencies subsequently approve a combination of gene therapy and other technologies that can cure HIV, the cost will likely be very high.

It is possible that through public pressure on pharmaceutical companies, governments will one day obtain a substantially reduced price for gene-based or other therapies that can cure HIV. Such substantial reductions in cost are necessary if an HIV cure is to be widely distributed, particularly in low- and middle-income countries.


TreatmentUpdate 231

The Canadian HIV Cure Enterprise (CanCURE)

—Sean R. Hosein


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