We look for more critical analysis of FDA expedited approval of a new drug like this.
We understand that the news of the day was the FDA approval. But some review of the data – and some independent critique of the quality of the evidence – would make a story like this more meaningful even on FDA approval day (or especially on FDA approval day). While the results of all the trials submitted to the FDA are not available, the results of one of the trials were published in November of 2011.
One piece of context that the New York Times provided was this:
“The story behind Kalydeco is a cautionary tale for those who believe the sequencing of the human genome will lead to a bonanza of new drugs. While the gene responsible for cystic fibrosis was identified in 1989, it took many years to figure out exactly how mutations led to the disease and even longer to figure out how to counteract the mutations.”
The story explains the cost of $294,000 a year for this drug. And it describes the drug company’s financial assistance plan.
We understand that the focus of this story was the FDA approval. Nonetheless, we wish that even a brief description of the data that led to approval had been provided.
There was no discussion of potential harms from the drug.
The prescribing information for the drug states: “Overall, the most common adverse reactions in 353 patients with CF were headache (17%), upper respiratory tract infection (16%), nasal congestion (16%), nausea (10%), rash (10%), rhinitis (6%), dizziness (5%), arthralgia (5%), and bacteria in sputum (5%).”
The story’s close almost begs for more explanation: “Kalydeco was approved …in about three months and ahead of an April deadline.”
Another story we saw at least reported: “The agency based its expedited approval on two clinical studies involving 213 patients aged 11 and over that lasted nearly a year. In both studies, those treated with Kalydeco had improved lung function, increased weight gain, and fewer acute problems that often require hospital visits and treatment with antibiotics, compared with those who took a placebo, according to Vertex.”
Given the expedited approval process, we wish the story had offered some critical analysis of the evidence.
There was no disease mongering in the story.
There was no independent medical expert interviewed for the story. The story also quoted the same patient we saw profiled in other stories. Did the drug company provide access to her for all who quoted her?
The story at least mentioned that until now patients took “medicines that only tackled the symptoms of the disease, such as antibiotics to treat the infections. Patients typically spent a few hours each day wearing vibrating vests to dislodge mucus build-up.” But there was no comparison of the effectiveness of the new drug with this past approaches.
The story focused on FDA approval. It also mentioned the drug company’s financial assistance plan.
The opening sentence explained that this was “the first therapy to treat an underlying cause of cystic fibrosis.”
It’s clear that the story did not rely solely on a news release.