Gene Therapy Could Change Rare Disease Treatment Forever, But Will Anyone be Able to Afford It?

According to a story from BNN Bloomberg, gene therapies are without a doubt an upcoming advancement in care and treatment for many diseases that have a genetic origin. This includes a major portion of rare diseases as well, which is many cases are caused by genetic abnormalities or mutations. These therapies could vastly improve outcomes for these rare patients. However, there is still a significant problem that has yet to be answered: how much will these drugs therapies cost?

An Opportunity for Reduced Costs

Many gene therapies are being developed as a single use treatment in which a single dose or procedure is all that is necessary to treat a disease effectively. This means that gene therapies could be replacing treatments that a patient was often expected to take for a longer period of time, sometimes indefinitely. This undoubtedly presents the possibility of reduced overall costs for patients, even if the single therapy itself is still highly priced. Still, the prospect of high costs is already causing concern.

The drug company Novartis has selected an installment style payment approach for Zolgensma, a gene therapy that is in development as a treatment for spinal muscular atrophy. This decision seems to be indicative of the company’s intention to charge a steep price for the therapy. With the US Food and Drug Administration (FDA) predicting potentially 20 cell or gene therapy approvals per year by 2025, reasonable and responsible pricing is going to be critical if these treatments are actually going to be accessible to patients and have the impact that is anticipated.

Or A New Level of Exorbitant Pricing

Predictions from the UBS group projects that Zolgensma will cost $2 million per treatment. This would represent a new escalation in the trend of extremely high drug prices for rare diseases. Novartis has even suggested the possibility of charging as much as $5 million. Meanwhile, the Institute for Clinical and Economic Review (ICER) claims that Zolgensma’s true value could range from $1.5 million to $310,000.

Amidst the debate about pricing, there is still some uncertainty about the long term impact of single use gene therapies. There still isn’t any data that proves that the benefits will truly last for the rest of a patient’s lifetime. If these drugs become more widely used and available, there is little chance that multi-million dollar prices are going to be practical (not that they really are now anyway). Installment plans are also of limited use if the therapy is still overpriced as a whole.

To maximize the usefulness and impact of gene therapies, a reasonable and fair starting price is essential.


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