A new drug approval offers an opportunity to review the evidence with readers. This news release from the Fred Hutchinson Cancer Research Center about a newly approved drug for Merkel cell carcinoma (MCC), a rare type of skin cancer, missed that chance.
We understand the need to get the news out there and even to highlight a patient story, but there is enough evidence on benefits and risks associated with immunotherapy, not to mention costs, that news releases should give readers a fuller picture. Without that important information, the dramatic experience of the cancer patient in the release provides an overly optimistic picture of patient outcomes for everyone who may be diagnosed with this particular type of cancer.
MCC is a rare, often aggressive type of skin cancer, with about 1,500 new cases diagnosed in the U.S. each year. The 5-year survival rate for MCC is estimated at around 60 percent, according to MayoClinic.org.
This is the first drug approved for treating MCC — but not the only recognized treatment for the disease. Some of the media interest in the approval relates to the rapidity with which the FDA approved it while awarding the drug several special designations — accelerated approval, priority review, breakthrough therapy and orphan drug status, the latter of which “provides incentives to assist and encourage the development of drugs for rare diseases,” according to the FDA’s news release on the approval.
Readers should keep in mind that the evidence for the drug is limited and based on a small study of short duration.
There is no mention of costs in this piece. Even though it calls out the drug avelumab (marketed as Bavencio) by name, the release does not talk about what a typical regimen of that drug or any other would be.
Bloomberg reports that avelumab will be priced at $13,000 a month, or $156,000 for a year of treatment.
The release alludes to a study in the Lancet Oncology but it does not provide any of the measurable benefits found in that study, namely that out of 88 patients who received at least one dose of the drug avelumab, 28 achieved an “objective response” within an average of about 10 months. That means that less than a third of the patients who took the drug saw something positive happen as a result. The release might make you think that, in fact, the majority of patients who take the drug see their tumors shrink. But even when a 90% reduction in tumor mass occurred (as was the case of the patient profiled), it would not automatically translate into longer survival for all patients.
The release states that almost a third of patients in the trial “with no hope at all of surviving” benefited from the drug. What does no hope at all mean? Were all of the patients in the trial at stage 4 of the disease? The release doesn’t tell us.
The same study named in the release shows that multiple patients had side effects from the drugs. These are not mentioned in the release.
The side effects occurred in two patients who developed lymphopenia (abnormally low white blood cells), one who had an increase in blood creatine phosphokinase which could indicate muscle inflammation or damage, and other disorders.
There were also “serious treatment-related adverse events” in five patients, including enterocolitis [inflammation of the small intestine and the colon], infusion-related reaction, aminotransferases [enzymes related to the liver] increased, chondrocalcinosis [calcium deposits in cartilage], synovitis [joint inflammation], and interstitial nephritis [inflammation of the kidneys].
We think that patient stories should be used judiciously and balanced with a full representation of the evidence. In this case, the story of one patient is used as a stand-in for all patient experiences in the absence of any measurements of benefits or risks. We don’t think that it is a fair representation of the two-thirds of patients who may see no benefit from the drug, including the fraction who may actually have a serious adverse reaction.
While the news release notes that this is the first treatment approved by the FDA for MCC, it does not highlight that this is only a phase II study so there’s no direct evidence that the new treatment is better than the existing treatments.
Beyond linking to a Lancet Oncology article, the release doesn’t tell us anything about the study upon which the approval was based. At minimum we’d like news releases to tell us how many patients were enrolled in the study and for how long.
According to the published study, 88 patients were enrolled in the trial and received at least one dose of avelumab over the course of the 14-month trial period. Most patients were followed up at 10.4 months. Among the 88 patients, 28 (31.8%) had an “objective response,” 8 had an “incomplete response” and 20 had “partial responses” by the end of the study. We aren’t provided with data on the average survival for the disease following treatment, but that may be because the 10+-month follow up was of insufficient duration to meaningfully assess the new drug.
As mentioned under harms, there were numerous serious side effects.
Presenting absolute risks and mortality data would have provided important context to the study.
There is no overt disease mongering in the release. It provides some context on MCC and how often it occurs in relation to melanoma, the most common form of skin cancer. However, the comment that MCC is “35 times less common than melanoma, but on average, it is about three times more likely to be deadly,” is difficult to interpret.
The release appropriately explains that the clinical trial in question was for a drug made by Merck and that the lead scientist in the clinical trial receives ongoing funding from other companies. (We should note, too, that one of the reviewers on this news release, Bill Heisel, works at the University of Washington where some of the researchers on the clinical trial work as faculty.)
The release makes no mentions of, or comparisons to, other therapies.
Standard therapies include surgery, radiation and chemotherapy.
The release makes it clear that this is the first therapy approved by the FDA for this particular type of cancer once it has spread.
The release focuses on the fact that “Avelumab is the first FDA-approved treatment for metastatic MCC and the first disease that the drug has ever been approved to treat.” Immunotherapy is an emerging area of research, and the release appropriately explains why this particular approval is important.
The release doesn’t use sensational or unjustified language.