The findings of the study, conducted under the leadership of researchers Ian Liu, Aaron Kesselheim and Edward Shaffer Cliff, indicate that of the 46 cancer treatment drugs that received approval through the same accelerated procedure in 2013-2017, 63 percent received permanent approval from the FDA In later years, in only 43 A percentage of the drugs presented evidence that they are indeed clinically effective – that is, only 20 drugs out of the 46.
“Drugs that receive temporary approval through the FDA’s expedited procedure are usually drugs that are intended for the treatment of advanced, terminal diseases in patients who have no other response,” explains Prof. Ido Wolff, director of the oncology department at Ichilov Hospital and head of the Tel Aviv University School of Medicine. According to him, in many cases there are promising initial results regarding any medicine before a comprehensive examination or criticism of the findings has been made. “In such a situation, the FDA has two options: either wait for a larger and orderly study that will confirm or contradict the initial findings, or give the drug a ‘conditional’ approval, and over the years examine its effectiveness in depth and decide on a permanent approval.”
According to the professor, while comprehensive clinical studies to examine drugs and their effectiveness can take five to seven years, preliminary studies are published within one or two years. “In the case of terminal cancer or diseases that have no cure and doctors have no other solutions to offer, it is right to make drugs that show promising initial results more accessible and give patients some hope.” The problem, he explains, lies in the lack of proper examination and control of the subsequent clinical studies after receiving the temporary approval. “Sometimes it happens that the approval remains and the efficiency turns out not to exist.” According to him, another problem relates to the marketing of the drug. “There are cases where marketers will present the FDA approval – without admitting that it is a temporary and not a permanent approval. This is enough for people all over the world to want to use the drug. Over the years, until the effectiveness of the drug is ascertained, it already receives the status of a legitimate treatment.”
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And what is happening in Israel?
Prof. Wolff, who also serves as the head of the National Council for the Prevention, Diagnosis and Treatment of Malignant Diseases, says that even in Israel there are drugs on the market that received only temporary approval through the FDA’s expedited procedure, but control mechanisms also exist. “The registration procedure is done separately in Israel, so that not every drug approved by the American Food and Drug Administration will necessarily be approved here, and rightfully so.” Another control mechanism is the medicine basket. “A medicine enters the basket only after lengthy professional discussions. Usually it is a medicine whose effectiveness has already been fully proven. The medicine basket gives another measure of security.” And yet, the professor testifies, he too has come across cases where, even though the evidence regarding the drug’s effectiveness is only partial, marketers try to encourage their use.
The professor emphasizes that the doctors must make it clear to the patients, especially if it is an expensive drug that is not in the medicine basket, that this is a treatment whose effectiveness has not yet been proven. “One can understand the need of a patient who is dealing with terminal cancer to cling to some medicine, with some hope, but he must receive all the information.”