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Most of the time, news organizations announce medical breakthroughs as if they were medical miracles. The meaning of the term to the FDA is quite different from what it means to the public.

How many times have we all heard some development in medical research described as a "medical breakthrough"?

Here are some examples:

  • In 2016, multiple news services announced a breakthrough in treating strokes. Instead of using potentially deadly "clot busting" drugs, surgeons had mastered a technique  called embolectomy. The surgeon threads a catheter with a tiny mesh tip through an artery into the brain, and when the catheter is removed, the clot comes out. What the stories in the news failed to mention was that the procedure was introduced as a "last resort therapy," that had actually been performed, although with great success, on just one patient two years earlier.
  • In 2015 the CBS news program Sixty Minutes ran a story on a clinical trial of using polio virus to treat the aggressive form of brain cancer glioblastoma with a screen reading "Cancer Breakthrough." Then the reporter revealed that the person who inspired the headline had died.

  • In 2014, over 10,000 people died of Ebola in West Africa. In 2015, the respected medical journal Lancet reported that a vaccine had been tested for treating Ebola exposure. News organizations heralded a "medical breakthrough," but failed to note that 16 out of 21 people who were given the vaccine more than 10 days after exposure to the virus developed the generally fatal infection.

The dictionary definition of a breakthrough is “a sudden, dramatic, and important discovery or development." The FDA definition, however, is quite different. The FDA Fact Sheet states that a breakthrough is:

  • A drug
  • Intended to be used by itself or with other drugs to treat a life-threatening condition
  • That offers a significant improvement over currently available treatments.

If the best currently available treatment is successful 50 percent of the time and carries a 20 percent risk of sudden death from the treatment itself, and the new treatment is successful 50 percent of the time and carries a 2 percent risk of sudden death, like the "clot catcher" treatment for stroke, then the new treatment is a breakthrough. If the best currently available treatment extends life three months and the new treatment extends life for a year, as seems to be the case with the polio vaccine for brain cancer, then the new treatment is a breakthrough. If the best available treatment results in a 90 percent death rate and the new treatment results in an 80 percent death rate, like the Ebola vaccine, then the new treatment is a breakthrough.

A breakthrough treatment is a better treatment. It's almost never a miracle treatment, although it may seem to be a miracle at first. So why does the FDA bother to designate some treatments as breakthroughs?

The breakthrough designation just means that the FDA will fast-track its decision on approving further testing of the drug. When a drug receives breakthrough status, the FDA will rule in 60 days whether to approve or deny further testing. When research seems to have genuine potential for treating a life-threatening disease, the FDA generally goes out of its way to make sure the drug is tested sooner so, if it really works, and that's not a given, then it can be put on the market sooner.

Continue reading after recommendations

  • Kesselheim AS, Woloshin S, Eddings W, Franklin JM, Ross KM, Schwartz LM. Physicians' Knowledge About FDA Approval Standards and Perceptions of the "Breakthrough Therapy" Designation. JAMA. 2016 Apr 12. 315(14). 1516-8. doi: 10.1001/jama.2015.16984. No abstract available. PMID: 27115269.
  • John S, Burgess R, Cheng-Ching E, Wisco D, Taqui A, Bain M, Toth G, Uchino K, Hui F, Hussain MS. Last resort: case of clot translocation in intra-arterial stroke therapy. BMJ Case Rep. 2014 Jan 6
  • 2014. pii: bcr2013010958. doi: 10.1136/bcr-2013-010958. PMID: 24395869.
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