To patients grappling with incurable diseases, new therapies can’t come quickly enough. A bill that hopes to address this need sailed through the House in the summer with the goal of getting more “21st Century Cures” through the drug approval pipeline. But a pair of new studies found that speeding up this process could put drugs that are ineffective or harmful on the market.
At least for complex neurological diseases, such as Alzheimer’s, Parkinson’s and depression, recent history suggests that approving drugs faster based on biomarkers — early signs that a drug could be working — might make drugs that ultimately don’t work available to patients, two teams of researchers found.
These early biomarkers are one of several ways the House bill could speed up the drug approval process. A Senate version of the bill is expected to be presented soon, with many wondering how similar it will be to the House version. The Food and Drug Administration has said the House legislation wouldn’t override its ability to approve safe and effective drugs.
A second study published in the British Medical Journal this week examined three Alzheimer’s drugs that progressed to late-stage trials based on positive data in earlier stage trials that suggested the drugs were working. In each case, the team found that the phase two trials suggested the drugs would work — two showed encouraging decreases in measurements of beta amyloid, a protein that builds up in the brains of Alzheimer’s patients. One drug, semagacestat, was then tested in 1,537 patients and was found to cause skin cancer and even worsen patients’ ability to perform activities of daily life. The other two drugs looked promising in intermediate trials, but also failed once tested in large numbers of patients.
The researchers projected that if the semagacestat had been approved based on its encouraging results in intermediate trials, the treatment would likely have cost thousands of dollars a year, treated 234,000 patients, and caused 19,000 cases of skin cancer.
“This is just yet another example where, if you look at these very reasonable surrogate endpoints … you can get results that are completely different when you look at things you really care about, which, in this case, is memory loss and the ability to function, day-to-day,” said Diana Zuckerman, president of the National Center for Health Research.
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