Amber Freed, a mother of boy-and-girl twins, has high hopes and aspirations for her son, Maxwell, who was diagnosed with an incredibly rare, unnamed debilitating disease, SLC6A1. The disease is progressive and neurodevelopmental. It causes speech apraxia, intellectual disability, and progresses into a debilitating form of epilepsy.

When the diagnosis was first given to Freed, she immediately sought answers, but doctors had devastating news: not only was there no cure for Maxwell’s disease, there were no existing drugs or therapies to help with the symptoms.

After tracking down a well-respected scientist, Dr. Steven Gray of the University of Texas Southwestern Medical Center, Freed flew to Washington, D.C., for a conference she knew he was attending and the two sat down for a four-hour-long dinner. During the dinner, Freed learned that Maxwell was a good candidate for gene replacement therapy and thus discussed a treatment plan.

Unfortunately, this therapy does not come at a bargain. Patient organizations must raise upwards of seven million dollars to simply begin phase one of a clinical trial. 

That’s because the cost of developing and bringing new drugs to the market is so high. In fact, the total cost of developing a new drug from start to finish is a staggering 2.6 billion dollars

Freed is well aware of this. As she puts it, “It’s incredibly expensive to research and develop drugs especially for a rare group of patients.” 

But Freed recognizes that price controls aren’t the answer to high drug prices, saying, “Just because you put a price cap on a drug doesn’t mean it’s any cheaper.” 

Implementing drug price caps is an excellent approach in theory but price controls stifle innovation and delay the time in which drugs, including those for patients with incurable diseases, reach the market.

Rather than utilize drug-price controls, Freed believes policies should focus on expanding access to life-saving treatments and allow a free, competitive market to drive prices down. For more information, check out IWF’s Two Truths And A Lie: Pharmaceutical Price Controls.