January 31, 2020

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It’s no secret that new drugs are expensive to create. And not long ago, drug companies shied away from creating cures for diseases that affected only a few thousand people — fewer patients meant smaller profits. But in 1983, Congress passed the Orphan Drug Act (ODA) which incentivized the development of treatments for rare diseases.

And it worked. Since it passed, the legislation has helped to create hundreds of new treatments for rare diseases. But it may have also caused some unexpected side effects. According to Peter Bach, a pulmonologist and intensive care physician at Memorial Sloan Kettering Cancer Center, the push towards finding cures for rare diseases has been so strong that drug companies are paying little attention to more common illnesses, including some of the leading causes of death in the United States, like heart disease, cancer, and diabetes.

Three Takeaways:

  • Bach points to extended market exclusivity as one of the biggest drivers of orphan drug creation.Typical exclusivity periods usually only give developers a monopoly over the sale of a drug for five years, versus seven years for orphan drugs, making the latter much more profitable. Bach says that this has pulled companies too far towards orphan drugs, causing them to neglect more widespread diseases.
  • Bach argues that the ODA allowed pharmaceutical companies to drastically shift their business model. Instead of focusing on drugs that will have the widest use, many companies transitioned to what Bach calls a more “boutique business model” where they can sell incredibly expensive drugs to patients with the best insurance. The high price tag allows companies to ignore other patients in need. Bach points to large gaps in life expectancy as proof that the rich are receiving health care that works, but such health care is often impossible for poorer Americans to access.
  • Bach believes the solution is pretty simple: rewarding research and development that aligns with what most people need. In much the same way that Congress incentivized cures for orphan drugs, it needs to incentivize cures for diseases that impact more people. In response, Bach thinks drug companies will shift to create treatments and programs that hundreds of thousands of Americans desperately need.

More reading:

  • Read Bach’s column in Bloomberg to learn more about orphan drugs, like a new therapy called Zolgensma, which costs $2.1 million per treatment.
  • The Opioid Crisis Response Act of 2018 attempted to address the opioid epidemic, which takes tens of thousands of lives every year. But according to this article from Vox, experts are worried the bill falls short.
  • Learn more about the history behind orphan drugs in this episode of 99% Invisible.

medical research, peter bach, disease, medicine, health, pharmaceuticals

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