(CNN)President Donald Trump used his first address to a joint session of Congress on Tuesday to, among other things, promise to “slash the restraints” put on drug development by the Food and Drug Administration and across the government so the “slow and burdensome (drug) approval process” doesn’t hinder medical advances.
To illustrate his point, he invited “miracles” like Megan Crowley to attend the speech.
Crowley is a 20-year-old sophomore at the University of Notre Dame who wasn’t expected to live past age 5. She has Pompe disease, a serious and rare inherited disorder that leads to severe muscle weakness and heart problems. It is typically fatal. The disease had no pharmaceuticaltreatment until her father founded a company to develop an enzyme replacement therapy that would help save her life.
In 1992, the FDA created the “accelerated approval” process, which allows drugs to go ahead with even earlier-stage data if they would be used to treat a life-threatening or serious illness. That year, the FDA also created the “priority review” system.
The 1983 law called the Orphan Drug Act gives tax breaks and an exclusive market for a period to drugs that treat patients with a disease that may be too rare for researchers to find enough subjects for typical large randomized trials.
Drug companies would often not develop drugs for these markets because there may not have been enough incentive to invest in a product that would be used by so few people.
Crowley’s father was able to set up his company and make the drug that saved her under the authority of this act.
Has this faster process inspired innovation?
Since the FDA created this faster process, an increasing number of companies have used it to get their drugs to market, but there aren’t necessarily an abundance of “miracles” and breakthrough drugs. In fact, a 2015 independent analysis of drugs approved during this faster processing time found that the trend”is being driven by drugs that are not first in class and thus potentially are less innovative.”
What drugs have benefited from the sped-up process?
Imatinib, a drug used to treat chronic myelogenous leukemia, benefited from all four programs created to speed the drug approval process and got through in a mere 2 months of FDA review, one study showed.
Have there been any problems related to a quick approval process?
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Gemtuzumab, a drug that went through the accelerated approval process and was approved in 2000 to treat myeloid leukemia, a bone marrow cancer, was voluntarily withdrawn in 2010. The additional studies required by the FDA after expedited approval contradicted earlier research, finding that people who took it had no improvement and that there were a greater number of deaths among patients who took it. The drug was also associated with a potentially fatal liver condition.
Studies also show that some drugs approved using this quicker process had a number of significant adverse events and required an additional warning labels called black-box warnings.
That’s what happened with the drug rosiglitazone. Eight years after it was approved, the maker had to put black-box labels because research showed that taking it raised the risk of congestive heart failure and death from cardiovascular causes.