Carole Longson - NICE

Taking a tough, and closely watched, payer approach to new drugs

Name: Carole Longson
Title: Director, Center for Health Technology Evaluation, NICE

Since its inception in 2000, NICE has stirred up a furor, touched off a firestorm, inspired an outcry--choose any metaphor for controversy, and it's been used to describe the reactions to yes-or-no decisions by the cost-effectiveness watchdog. Though Sir Andrew Dillon is the titular head of NICE and its usual high-level face, someone else stands in the thick of the treatment-assessment process: Carole Longson, who directs the unit in charge of sifting the evidence on drugs--and crunching the numbers on their costs.

Longson is the person who publicly explains why NICE recommends a treatment--or not. Her statements are the measured, intelligent words of a researcher-turned-government official--she worked for 8 years at GlaxoSmithKline ($GSK)--but they're quite blunt as well. In rejecting Savient Pharmaceuticals' gout drug recently, she said, "There was considerable uncertainty about the long-term efficacy and safety of [Krystexxa], and a very high cost compared with the known benefit." In other words, we're not sure it works, not sure it's safe--and it's expensive, too.

Longson has said similar things about other drugs. And because NICE's blessing is required for National Health Service patients to have easy access to a treatment, those statements trigger complaint and criticism from the media, from patient groups, and from drugmakers. Nowhere has this been more evident than in oncology, where NICE and its assessment committee have rejected one pricey treatment after another, unsatisfied with their cost-benefit balance.

When Longson speaks at conferences and meetings, she likes to talk about "consulting with stakeholders" and "maintaining an innovation-friendly environment." What that means is working with drugmakers to get the right kind of data, which isn't as easy as it may sound. The agency called InterMune's ($ITMN) trials on Esbriet too short for comfort, and it took issue with analyses put forward by Bristol-Myers Squibb ($BMY) and AstraZeneca ($AZN) in support of their new diabetes drug Forxiga.

Increasingly, Longson's work with stakeholders involves negotiating discount after discount. Once upon a time, drugmakers waited for a rejection to cough up a cost cut. Johnson & Johnson ($JNJ), for instance, initially failed to win NICE's blessing for its Velcade blood-cancer drug. But the company came back with a "patient access scheme," as these pricing arrangements are called, and won the agency over.

The Velcade scheme was groundbreaking in another way as well: It was a risk-sharing agreement, in which J&J agreed to bear a portion of the treatment costs for patients who didn't respond to the drug. That agreement spawned others in a similar vein. We're not sure how many, because these access schemes are now confidential. And now, drugmakers are as likely to offer a price-cutting scheme upfront--whatever its form--to shorten the path to approved use by the National Health Service. That's not to say that the upfront offer is always enough, however. Novartis ($NVS) recently persuaded NICE to change its mind on the eye drug Lucentis by offering a "revised patient access scheme" and new data backing up its effectiveness in certain patients.

Drugmakers are so eager to please Longson's group not only because it influences use on the NHS. Other countries keep an eye on NICE's decisions and use them in their own assessments. Some governments don't have the capacity to analyze a treatment's cost-effectiveness, and they simply adopt NICE's decisions as their own. So, when Longson offers her polite-yet-frank assessments to constituents in the U.K., the world is listening.

-- Tracy Staton (email | Twitter)

Carole Longson - NICE