Novel Financing Approaches are Needed to Capitalize on Life-Saving Gene Therapies

Today, the American Consumer Institute Center for Citizen Research (ACI) announced the release of its latest ConsumerGram, highlighting the promise of new gene replacement therapies and urging health care stakeholders to develop innovative ways of financing these important treatments.

The report, authored by Dr. Joseph Fuhr, Liam Sigaud and Steve Pociask, describes the economic benefits of gene therapies, which unlike most conventional treatments, can deliver lifesaving cures for debilitating genetic disorders by fixing their underlying causes.

In 2017, the FDA approved the first gene therapies for use in the United States, and dozens more are expected to gain approval in the years ahead.

Despite high upfront costs, gene therapies generate significant savings by replacing expensive long-term pharmaceutical treatments, reducing hospitalizations, preventing further health deterioration, and improving patients’ quality of life. A gene therapy for severe hemophilia, for example, is expected to cost about $1 million per patient. But this one-time cure eliminates approximately $690,000 in annual costs of conventional treatments. In less than two years, the gene therapy pays for itself. Other gene therapies for spinal muscular dystrophy, Best Disease (a rare form of blindness), and lymphoblastic leukemia offer similar savings over the long-term.

“Curing a disease, rather than treating it, can save insurers and taxpayers money in the long-run, even if the initial investment is significant, and provide an immense improvement to longevity and quality of life,” said Dr. Joseph Fuhr.

However, under the present healthcare system, insurance companies are often reluctant to pay for therapies that have high upfront costs and whose benefits are spread over a lifetime. “Payers, policymakers, and manufacturers must recognize that existing mechanisms to finance medical treatments and manage affordability may be inadequate to cope with the growing number of gene therapies being introduced,” said Mr. Sigaud.

New, value-based payment strategies need to be developed to make the growing number of lifesaving gene therapies broadly accessible to patients.

You can read the ConsumerGram by visiting here.

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