## The $2 Million Cure: Gene Therapies and the Future of Healthcare Costs

Ciji Green’s daughter, Maisie, was diagnosed with spinal muscular atrophy, causing her muscles too waste away. The disease is often fatal before age 2 without treatment. In 2019, a genetic therapy was approved by the Food and Drug administration to treat SMA, but the single-dose therapy costs $2 million. Green’s insurance said it wouldn’t pay for the new, expensive drug. 

“I became very angry, to know that ther was something that could help her. And I knew without a shadow of a doubt I was burying my daughter before she was 2,” Green said. 

The Zolgensma medication Maisie needed is part of a new breakthrough class of gene therapies often treating rare diseases where patients have few, if any options.But, as the high-cost drugs treat more common diseases, they could have wider impacts on the U.S. health care system.

“I liken it to a coming tsunami, which is basically gonna overwhelm the employer-sponsored insurance system,” said economist Jonathan Gruber, who helped develop the Affordable Care Act.Today, more than 300 high-cost genetic therapies are in clinical trials, and some are aimed to treat diseases suffered by millions.Companies who provide insurance for employees could be called on to cover the new, costly medications, and many employers are not prepared.”The first problem is that many companies in America are what we call self-insured. They pay their own medical bills. About two-thirds of the insured in America are in such arrangements,” Gruber said. “They can’t afford to pay this, so they’re facing a tough financial decision, which is, ‘Do I cover this drug and potentially go bankrupt? or do I not help my unlucky employee?'”

## Why the drugs are so expensive

Doug Ingram is CEO of Sarepta Therapeutics, which is among those charging millions for a drug. Sarepta’s Elevidys costs $3.2 million for a single-dose treatment, but it might potentially be all that’s needed to slow Duchenne muscular dystrophy, a muscle wasting disease.

There’s an urgent unmet need for treatments for children who have the disease. It is so dire doctors have told the parents of children with duchenne to go home and love their child, because the degenerative disease is ultimately fatal, Ingram said.His company uses an engineered gene that instructs the cells to make a protein to protect the child’s muscles. 

“So the first question is could you even do this and do this safely? The second question that was obvious is if you could, you have to manufacture th

Why New Therapies Cost So Much – and how to Bring Prices Down

New medical treatments are often incredibly expensive. This isn’t a simple case of pharmaceutical companies being greedy. Developing a single therapy is a massive undertaking, riddled with risk and requiring billions of dollars. But change is possible, and industry leaders are speaking out about the need for reform.

The Staggering Costs of Drug Development

Bringing a new therapy to market typically takes over a decade and costs nearly $3 billion. Think about that: ten years and three billion dollars before a single patient even benefits. And the odds are stacked against success. The probability of a therapy making it through the entire development process is extremely low. This inherent risk is a major driver of high prices. Companies need to recoup their investment and fund future research.

According to pharmaceutical CEO Richard Ingram, prices will come down as manufacturing processes improve.He also believes streamlining federal regulations is crucial. “Today, on average, it takes more than 10 years to develop a therapy. It costs nearly $3 billion on average to make a therapy. And at the beginning of that journey, the probability of it being successful is nearly zero. and in the context of that, of course therapies when they’re eventually approved are going to be very expensive. So what we need to do is fix that,” Ingram stated.

The Regulatory Burden

Ingram points to decades of accumulating regulations as a notable problem. While these regulations were initially intended to ensure safety and efficacy, they’ve become overly complex. “We’ve had 60-70 years of layering and layering and layering of requirements, all for the laudable goal of ensuring that the therapies that are approved in the United States are both safe and effective. We have to do the hard work of getting under that and stripping it down to those things that are absolutely necessary, informed by the science that we have today, not the science we had in the ’60s, and find a way to make therapies less than $3 billion with a higher probability of success.”

Essentially, the regulatory process needs a major overhaul. It needs to be updated to reflect modern scientific understanding and eliminate unnecessary hurdles. This isn’t about lowering safety standards; it’s about making the process more efficient and less costly.

Getting treatment for maisie

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