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» Gene Therapy Drugs – A Big Change in the Market

February 28, 2023

Employee Benefits, Healthcare, Learning and Development, Prescription Drugs

Gene Therapy Drugs

What are they: Gene therapy drugs are medications that use genebased techniques to treat diseases or medical conditions. They involve introducing genetic material into cells to replace or suppress malfunctioning or missing genes. This can be done through a variety of methods, such as viruses carrying the desired genetic material, or through direct injection of the genetic material. Gene therapy can be used to treat genetic disorders, cancer, and some infectious diseases.


Why do the drugs matter: Gene therapy drugs matter because they offer the potential to treat, and in some cases cure, many genetic diseases and disorders that are caused by a single gene mutation. This could have farreaching implications, as many rare and debilitating diseases have no effective treatments or cures. Gene therapy drugs have the potential to change the course of medicine, allowing individuals to live healthier and more productive lives.


What is the big issue with the drugs: The main issue with gene therapy drugs is that they are expensive and difficult to develop. Developing a gene therapy drug involves a great deal of research, clinical testing, and regulatory approval, which can take many years and cost hundreds of millions of dollars. Additionally, gene therapy drugs can sometimes be difficult to deliver to the target cells, and the effects may not be longlasting.


Example: The FDA recently approved two gene therapies, doubling the number on the market for diseases other than cancer. (

  • Bluebird bio’s Zynteglo is for a blood disorder called beta-thalassemia that’s customarily treated in the most severe cases through regular transfusions. Zynteglo is marketed as a one-time, custom-designed injection that takes aim at the underlying genetic causes of the disorder, and costs $2.8 million.
  • Zynteglo was the most expensive drug on the market for a few weeks, until the FDA approved bluebird’s Skysona for a rare neurological disorder called cerebral adrenoleukodystrophy. Skysona had a list price of $3 million.
  • Bluebird estimates there are 1,300–1,500 people in the U.S. who are eligible for Zynteglo. CVS estimates there are only 700 potential U.S. candidates for Skysona.

The Big Picture:

  • The average gene therapy is likely to cost at least $1.5 million, said Colin Young, director of drug development pipeline research at Tufts Medical Center’s NEWDIGS Initiative.
  • Including oncology drugs, gene therapies could end up costing the U.S. around $30 billion a year, Young said. Total U.S. prescription drug spending was $348.4 billion in 2020, according to CMS.
  • “On a macro scale, it’s not a big problem,” Young said. “On a micro scale, it’s still quite a problem, because if you’re a self-insured company and somebody suddenly requires a $3 million gene therapy, that could wipe out the money you have for the rest of your employees.”
  • Even experts and groups generally critical of high drug prices say the cost could be justified if it delivers a lifelong cure that saves the health care system a lot of money in the long run.
  • When the modern health system came into existence, “there wasn’t an expectation at the time for potentially durable, potentially curable class of therapeutics like gene therapies,” said David Barrett, CEO of the American Society of Gene and Cell Therapy.
  • “That’s creating some issues trying to fit a transformative class of therapeutics into a system that was designed to address almost a more transactional approach with prescription drugs, where the benefit is tied to the administration of the therapy.”


The biggest concerns of how these drugs could impact our highly fragmented healthcare systems stems from the misalignment of interests. With the drugs new and unknown characteristics many are wondering if they could be worth the cost. Others are more hopeful and see the opportunity to create a new form of healing in the healthcare industry. Overall, it is something that all markets of the healthcare system are monitoring.


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Eleanor Schroeder

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Posted by in Employee Benefits, Healthcare, Learning and Development, Prescription Drugs