Kalydeco, for Cystic Fibrosis, wins first annual Forbes Breakthrough Drug Award

Kalydeco, a treatment for cystic fibrosis developed by Vertex Pharmaceuticals VRTX -6.56% with support from the Cystic Fibrosis Foundation , was awarded the first Forbes Breakthrough Drug Award today for dramatically improving the lives of patients and conquering a scientific challenge that had vexed researchers for decades.

The award was presented to Jeffrey Leiden, Vertex’s chief executive, and Fred Van Goor, a scientist who played a key role in inventing Kalydeco at the Forbes Healthcare Summit in New York.

“This drug is the first effective treatment for a deadly disorder (cystic fibrosis),” says Steven Nissen of the Cleveland Clinic, one of an expert panel convened to pick an honoree. “Although I do not care for these children, I am well aware of the devastating effects of this disease on patients and their families.”

Helping me choose, aside from Nissen: Peter Bach, of Memorial Sloan-Kettering, who has criticized the pharmaceutical industry’s pricing practices; Richard Evans, of SSR Research, a long-time industry analyst; Bernard Munos, a consultant who studies why it is so difficult to invent new medicines. I voted, too.

The panel unanimously chose to look at a period of several years, because not every year brings a breakthrough and some bring two. And we chose not to consider medicines until they had been on the market for a few years, because sometimes new drugs turn out to be either less or more important than they seem. Each panelist made his own choices, which we then discussed. We ranked the contenders based on that voting.

Kalydeco was the clear winner, appearing high on each judge’s list and getting a total of four stars on our rating scale.. But there were other important medicines approved during this time period, too. The runners up, in descending order:

*** Eliquis, from Bristol-Myers Squibb BMY -2.00% and Pfizer PFE +0.00%. A whole class of blood-thinning pills offer patients at high risk of blood clots or strokes alternatives to warfarin, the 60-year-old mainstay that requires constant monitoring. Some data indicate they may lower the risk of stroke more than warfarin. The panel opted to pick Eliquis, which it deemed the best of these new blood thinners, over Pradaxa from Boehringer Ingelheim, which was the first to reach the market.

** Xalkori, from Pfizer. This gene-targeted cancer drug has dramatic effects in patients who have non-small cell lung cancer driven by particular sets of genetic mutations. Bach notes that it was really the first cancer drug developed to target a particular cancer driver mutation, and that a diagnostic test to predict which patients would respond was ready when it was approved. It also charted a new path, getting accelerated approval with a companion diagnostic just four years after the driver was identified.

** Yervoy, from Bristol-Myers Squibb. Approved in 2011, Yervoy was the first new treatmet for metastatic melanoma, the deadly skin cancer, in a decade. It is also the opening salvo in a series of new cancer medicines that work by encouraging the immune system to attack tumors.

* Perjeta, from Roche. This drug is an heir to Herceptin, the incredibly effective breast cancer treatment. Perjeta is even more effective.

One concern that made all of the judges uneasy was the issue of price. All of these medicines are expensive, and Kalydeco is the priciest, with a list price of $305,000 per patient per year. Several times, we considered making price a factor in the decision, but at the end of the day we did not factor it in precisely because so many of the drugs were so expensive. What should we do, pick Yervoy, which, in combination with a newer Bristol drug called Opdivo, can cost $256,000 to treat one patient for one year? In the end, drug pricing is a systemic problem.

Here’s what swayed us to pick Kalydeco: it extends the lives of children, and does so dramatically. The gene for cystic fibrosis was discovered in 1989. It took three decades for a treatment to emerge from that work, and even then it only works in a small sliver of patients. (Vertex has a new treatment, Orkambi, that aims to extend this benefit to other types of CF mutations, but the results are less impressive despite a similar cost to Kalydeco.) That’s a major breakthrough, and represents the best that the pharmaceutical industry has to offer.

December 3, 2015
Matthew Herper, FORBES Staff

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