What a $2 Million Per Dose Gene Therapy Reveals About Drug Pricing
www.propublica.org
Taxpayers and charities helped develop Zolgensma. Then it debuted at a record price, ushering in a new class of wildly expensive drugs. Its story upends the widely held conception that high prices reflect huge industry investments in innovation.

Reporting Highlights

  • A Record Price: The gene therapy Zolgensma helped children born with a fatal disease, spinal muscular atrophy, grow up to run and play. But the cost was stunning: $2 million per dose.
  • Cashing In: While taxpayers and small charities funded the drug’s early development, executives, venture-capital backers and a pharma giant have reaped the profits.
  • Priced Out: The drug’s cost adds to the nation’s ballooning bill for prescription drugs and puts Zolgensma out of reach for kids in many low- and middle-income countries.

~These highlights were written by the reporters and editors who worked on this story.~

  • Neuromancer49@midwest.socialEnglish
    3·
    6 hours ago

    I wish the authors had called out the company by name in the summary - Novartis is one of many pharmaceutical conglomerates raising the price of life-saving therapies. This particular drug is a flagrant example of price gouging, and it will take a lot of effort to fix the problem of cost.

    However, a new system has to carefully skirt around a very real issue - how can we provide market incentive to cure rare diseases? We are going through a quiet Rennaisance where advances in gene therapy and immunotherapy have provided us with the tools to cure most neurodegenerative diseases. However, most are considered rare diseases and the development of these drugs is very expensive (billions of dollars). Under capitalism, companies have to grapple with the horrific fact that curing a disease that affects ~1 in 300,000 with an expensive drug is a “bad investment”.

    Currently, the FDA offers a program where a company that develops a rare disease medication earns a credit for expedited review on a future program. These credits can be sold and traded, and are worth their weight in gold. But, rare disease medicines in the US benefit from longer periods of exclusivity which lets companies maintain high prices for even longer. In the UK, the NHS can rule the price of a drug does not provide enough benefit to society, and elect not to cover the medication, providing incentive to lower prices. I’m less familiar with the EU regulations.