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White paper: Gene therapy

Keeping costs from negating its unprecedented potential


Troy Brennan, MD, Former Executive Vice President and Chief Medical Officer, CVS Health

White Paper

White Paper: Gene Therapy

Keeping Costs from Negating Its Unprecedented Potential

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After many years of scientific promise but few real results, gene therapies are now a reality. These exciting new interventions have the potential to relieve tremendous suffering for patients with genetic disorders.


What are Gene Therapies?

Gene therapies insert DNA containing a functioning gene into a cell, to replace a faulty or missing one, to correct the effects of a disease-causing mutation. In order for gene therapy to work, once administered, the new DNA must reach the damaged cell, enter the cell, and either express or disrupt a protein. Gene therapies fall into two categories: in vivo and ex vivo.


The Massive Economic Implications

Along with the promise of a potential cure that gene therapies offer, come unprecedented costs. Zolgensma, a treatment for spinal muscular atrophy (SMA), debuted at a cost of more than $2 million — by far the most costly single-dose medication ever – is a great example of this challenge. Other gene therapies, all likely carrying very high price tags, are currently under development.

If future therapies continue to carry similarly high price tags and would add a heavy financial burden on the nation’s health care system, they could bankrupt small employer and self-insured plans. Even if we assumed just 5,000 individuals were being treated by gene therapy each year, with treatments priced at $2 million each, it would add $10 billion per year to the nation’s health care bill — an additional $50 per year of health care costs for every insured American.


Ensuring Access, Mitigating Cost Impact

The role of CVS Health — and Aetna as a health insurer — is to reduce the costs of therapy, while ensuring appropriate utilization of cutting-edge therapies. CVS Health will continue to build a rigorous, evidence-based formulary and apply clinically appropriate utilization management rules to gene therapy. However, there is a lot more that needs to be done and it will take a concerted effort from a range of stakeholders to ensure the price of these treatments doesn’t negate their potential. 

In this white paper, we highlight additional solutions we are developing to help mitigate the financial impact for our clients and their plan members.

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This document contains references to brand-name prescription drugs that are trademarks or registered trademarks of pharmaceutical manufacturers not affiliated with CVS Health.

Image source: Licensed from Getty Images, 2020.