At a time when melanoma is the deadliest skin cancer on record, the Federal Drug Administration was still slow-walking its decision-making over a new drug, Replimune (RP1). The staff members used inappropriate criteria to make their decision about rejecting the drug, and at a time when one of the most lethal cancers is not responding to other treatments, they were showing poor judgment:
Read more Iran strikes water systems — media strikes Trump
Skin cancer is the most common form of cancer in the United States, with as many as 3 million people being diagnosed with skin cancer every year, according to the American Cancer Society. The most dangerous form of skin cancer is melanoma, which accounts for the large majority of skin cancer deaths.
According to data from the U.S. Centers for Disease Control and Prevention, melanoma rates have steadily increased in the U.S. since 1999. And while the risk of melanoma increases as we age, it is the third most commonly diagnosed cancer between the ages of 25 and 39.
The FDA staff were rejecting the results of a clinical trial for the second time in two years, because there was only one study conducted without using a control group. Some experts agreed that there were some issues with the study; the manufacturer refused to establish a control group, which would have meant that some patients would have been deprived of the treatment, which would have been ethically questionable.
Still the results of the study were impressive:
RP1 is a re-engineered herpes virus that is injected into melanoma tumors and causes cancer cells to burst. This releases flares that train the immune system to track down and kill cancer cells hiding around the body. Many patients with metastatic melanoma don’t respond to, or develop resistance to, other front-line immunotherapies.
Replimune’s therapy turbo-charges the response to other immunotherapies by unmasking cancer cells. In this way it shrinks both superficial and distant tumors, with far fewer side effects than chemotherapy or radiation. A third of patients whose cancer worsened on other immunotherapies experienced remission on RP1, with cancers vanishing in one of six.
The FDA staff were dissatisfied with the level of remission, but were contradicted by two oncologists who were part of the advisory committee that was formed:
While FDA staffers downplayed this benefit, one oncologist at the advisory committee hearing pointedly noted that most physicians consider a 30% response rate in metastatic patients whose cancer worsened on other treatments to be significant. Another oncologist chided the FDA for failing to establish a precise benchmark for determining what constitutes a benefit of ‘large magnitude.’
Due to the difficulty in treating this type of melanoma, many physicians encouraged its approval:
Read more Identity formation and predestination
JPMorgan’s Anupam Rana said, ‘Our thesis remains that Replimune and the physician/patient community will stress the unmet need in refractory melanoma and the overall benefit/risk profile of RP1 (we do sense a lot of support for the product in the physician community based on our channel checks).’
Since these patients have so few options available to them for treatment, another doctor on the advisory committee to the FDA thought the benefits were worth the risks:
‘The way the study was conducted was maybe not perfect,’ Hussein Tawbi, M.D., a melanoma expert at MD Anderson Cancer Center, said when explaining his ‘yes’ vote. ‘But at the end, there were tumors that shrank, both the injected ones and the non-injected ones, that resulted in clinical benefit for patients that otherwise don’t have any other options available to them.’
The FDA must continue to streamline its approval process, especially for those patients who have run out of options. The agency had set August 2 as its target action date for RP1, but as of early August 3, no final decision had been announced publicly.
A significant sidenote to the story is the growing cases of melanoma in young adults, who are listening to myths that have arisen about sunscreens. Many have decided that they are less at risk if they refrain from using sunscreen than if they apply them:
As well as misinformation regarding the safety of sunscreen, the study also highlighted several concerning trends promising ‘natural’ sun protection. For example, nearly 1 in 4 under-35s believe drinking water prevents sunburn.
The numbers of young people who are suffering from melanoma are already rising. Let’s hope that use of Replimune will be approved to provide benefits for this deadly cancer.
Read more Don’t let the rank-and-file science reporters off the COVID hook either
These young people may be the future patients demanding the drug.