The Food and Drug Administration approved a new immunotherapy drug that acts as a “checkpoint” inhibitor in patients whose bladder cancer (urothelial carcinoma) has not responded to other chemotherapy. The drug, atezolizumab (marketed as Tecentriq), showed effectiveness in about 14 percent of 310 patients in a very preliminary study.
The release notes that the trial measured tumor shrinkage as the main outcome. It didn’t note, as a NYT article did, and important for this type of trial, that it is unknown whether the treatment makes a difference in survival. A larger study is being done to examine this. Whether it makes a difference in quality of life is also important and will presumably be examined in the larger trial.
The National Cancer Institute (NCI) estimates 76,960 new cases of bladder cancer and 16,390 deaths from this disease in 2016. This approval of a new drug for treating bladder cancer is part of a wider area of research into immunotherapy, in which drugs target mechanisms of the human immune response. Patients with the PD-L1 protein highly expressed in their tumors appear to be responding best to the drug. This drug is an example of targeted chemotherapy which is becoming more important as treatments can be fine-tuned to the cancers of individual patients.
In patients with intractable cancers, immunotherapy appears to offer some new hope while also bringing sobering questions about affordability and disparity of access by income. The drugs are typically very expensive — with some estimates putting the treatment cost for a single patient as high as $1 million.
When the FDA gives a compound a “breakthrough” designation, some physicians and patients can misunderstand what that means. That’s a topic recently explored in this post.
The release does not mention cost. The FDA evaluates drugs without consideration of cost, so it’s understandable that they wouldn’t comment on it — and that’s why we’re rating this Not Applicable. But let’s be real here: The value of a new drug is a function not just of how well it works, but its cost-effectiveness. So we look forward to the day when any news release about a new drug — even one from the FDA — will include some mention of cost in its message.
This release gives the preliminary results from a single study of 310 patients. Because the drug is experimental, long-term results haven’t been studied. Of the 310 patients enrolled in the lone trial upon which the approval is based, 14.8 percent showed some shrinkage of tumors during the assessment period lasting from 2.1 to about 13.8 months. For a subset of patients who were classified as having a positive response to PD-L1 expression, the response was 26 percent.
As noted above, tumor shrinkage doesn’t always lead to improvements in survival or quality of life. It is a surrogate endpoint — a substitute — for the main outcome which is survival. Although this drug looks promising, to be fully transparent about its benefits as currently known, the release should have explained this. As noted in the “Summary,” another larger study is underway to assess the drug’s impact on extending life and improving quality of life.
The release includes a description of side effects of this treatment. The most common ones included fatigue, decreased appetite, nausea, urinary tract infection, fever (pyrexia) and constipation. The release also mentions that patients taking the drug have an increased risk of infection and serious side effects that are “immune-mediated.” We would have liked a bit more context on what this term means for patients.
The release outlines that this is the very first clinical trial of this compound for safety and efficacy, and under which regulatory paths the drug was assessed, including “breakthrough therapy designation, priority review status and accelerated approval.” We’re giving this release the benefit of the doubt and rating it Satisfactory for its bare bones description of the trial (and since we already addressed the lack of long-term survival data in the “Benefits” section.) As it stands, the results from the small trial were preliminary and short-term and did not reflect long-term survival. We would have liked to see a reference or link to the completed trial where one could look at the results to assess independently or learn more about the outcomes. We think the release should have provided more data on the patient demographics, the treatment regimen (dosing amount and schedule) as well as the range in tumor reduction.
This release describes the approval of a new drug by a regulatory agency, but we’d still like to know who sponsored the study that’s the basis for the approval. It’s not enough to state who markets the drug.
The release does not give us very much information about alternatives. These patients had already received other therapy and their cancer had recurred, so it is implied that they had no other alternatives. But this isn’t stated very clearly. We can assume (and it seems evident) that the platinum-based therapy that the patients didn’t respond to initially is the usual treatment.
The FDA’s approval of a drug clears the way for the drug sponsor to scale up production and begin marketing and selling the product. (The FDA doesn’t state this explicitly but it can be inferred.) A NYT article said the drug would be available in about two months.
The release clearly states this product is first of its kind for this type of cancer.
We found no unjustifiable language.
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Comments (1)
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Sally James (@jamesian)
May 26, 2016 at 2:32 pmVinay Prasad, a researcher, questions how we use “surrogate endpoints” such as tumor shrinkage in a new paper. Read more here: http://scienmag.com/surrogate-endpoints-poor-proxy-for-survival-in-cancer-drug-approval-process/
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