It is a good feeling when you pick a winner – whether it is the Super Bowl coin toss, Smarty Jones in the Derby (was that really 10 years ago?), or, oh, a lifesaving melanoma drug in clinical trial.
Following up last April's designation as a Breakthrough Therapy, and the announcement two months ago by Merck of offering expanded access for MK-3475, yesterday the FDA announced more exciting news. The administration granted the drug, rechristened pembrolizumab, a priority review designation that will nearly halve the decision-making window. (Why the change from lambrolizumab to pembrolizumab? No idea, other than to make me learn an additional difficult-to-spell word.)
This means that the cavalry is on its way for late-stage melanoma patients. Details are sure to be forthcoming over the next few months in regards to dose, insurance coverage, availability, and oh yea, price, as I don't think Merck is planning on handing out perm… pambro… argh… MK-3475 for free. Although, there are a lot of Ts to cross before this announcement turns into tangible treatments for public consumption.
Still, the news is extremely positive. The Overall Response Rate from the trial cited is 41%, and 81% of the patients were alive after one year. With melanoma's death rate reaching more than one person every HOUR in the United States, shaving four months off approval of PD-1 will give an extra 3,000 people the opportunity of a promising treatment, who otherwise probably wouldn't see 2015. Granted, not all of these patients will respond to the drug, but some will – as will many others who cannot get access to PD-1 right now. Putting a number on "lives saved" or even affected isn't possible, but whatever that number would be, it is significant.
This priority review is based off a single-arm study – which isn't mine – so the data from the controlled study I am participating in may not even be reviewed until after the drug is approved. Aside from my partial response, the murmurs from the oncology, advocate, and patient worlds all indicate that the larger studies are showing the same or better results. Long-term results will be years away, but since most cancer patients don't have that time, it is great a treatment with obvious sustainable benefits is getting prioritized review and, ultimately, approval.
If you have melanoma right now and for some reason cannot get the expanded access program, you have one job in 2014 – stay alive until Halloween. In all likelihood, the game changes then, and for once, it's in favor of the patient.