Out of Order: If you build it …

Where companies traditionally have had to jump through hoops to impress regulators to approve the drugs they develop, they will increasingly find themselves jumping through even narrower hoops to convince payors that they have made significant strides in improving patient care.

Randall C Willis
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It seems that every year, the song is the same, and moreoften than not, the opening refrains appear with an English accent.
In June, the United Kingdom's National Institute for Healthand Clinical Excellence (NICE) rejected reimbursement of Roche's melanoma drugZelboraf not because it didn't work, but because they didn't show it madeenough of a difference in overall survival to warrant its premium price overexisting therapies.
Explaining the decision at the time, NICE's CEO said, "Weneed to be sure that new treatments provide sufficient benefits to patients tojustify the significant cost the NHS is being asked to pay. [Zelboraf] is anexpensive drug and its long-term benefits are difficult to quantify."
Last December, Novartis faced a similar issue when NICErejected reimbursement for its multiple sclerosis treatment Gilenya. Again,Dillon argued, "While Novartis submitted evidence that shows Gilenya can reducerelapses, our independent committee has not been convinced that it is acost-effective treatment option, even with the proposed Patient Access Scheme."
In both cases, of course, the companies argued that NICE waswrong in their interpretations of the impact of these drugs on patientpopulations.
Fine, NICE isn't very nice when it comes to gettingexpensive drugs to market in the U.K. Nothing new under the sun. Similar pressuresare felt by drug companies in countries throughout Europe and in Canada, andthey are increasingly feeling the pressure from payors within the UnitedStates.
So why do I bring this up now?
Because, as I begin research for two upcoming featurearticles on personalized medicine (which will appear in our October andNovember issues), I realize that the current challenges to convince payors to …well, pay, will likely escalate as pharmaceutical firms develop a wider arrayof drugs designed to more effectively and safely treat smaller and smallerpatient communities.
The math speaks for itself. If the cost of developing thedrug doesn't change (or at least not appreciably), but it is being delivered tofewer patients, then the cost of treatment per patient will need to be higherto recoup the investment. This is something payors aren't happy to hear as theyface tightening budgets and expanding patient rolls.
And Zelboraf is something of a poster-boy for thischallenge.
Touted by some as the prime example of personalized medicineat work, Zelboraf is targeted at a specific subset of metastatic melanomapatients who share a common genetic profile (positive for the V600E mutation ofB-Raf), and was co-developed with a diagnostic test (also from Roche) toidentify patients with this biomarker.
Despite its clear efficacy in clinical trials, however, NICEexpressed concern over the uncertainty of long-term survival benefits as manypatients in the trial switched treatment as their disease progressed, somethingthat happens commonly in cancer trials when one arm is shown to be particularlyeffective.
In this case, it seems that Roche did everything that hasbeen asked of them in the co-development of a targeted therapy with adiagnostic test to ensure only those patients most likely to benefit wouldreceive treatment, and yet they are left scratching their heads, while patientsare left with what some might now consider suboptimal treatment options.
And this discussion is being held over treatment of patientswith a marker that occurs in a large percentage of the metastatic melanomapopulation. What does the future hold for Vertex Pharmaceuticals' Kalydeco,approved in January as a treatment for a rare form of cystic fibrosis (carryingthe G551D mutation) that occurs in only 4 percent of CF patients (about 1200patients)?
Where companies traditionally have had to jump through hoopsto impress regulators to approve the drugs they develop, they will increasinglyfind themselves jumping through even narrower hoops to convince payors thatthey have made significant strides in improving patient care.
From a business perspective, that will mean paying evencloser attention to identifying the drug targets most likely to produce notjust safe and effective treatments, but safe and cost-effective treatments. Itwill mean rethinking the clinical trial process to ensure that the numbers lookas good as possible—not just better—when compared to current standards of care,which will rely on finding those patient subpopulations as early as possible.And it may even mean repeating clinical trials once a select patient populationhas been identified as achieving a particularly outstanding benefit.
Ironically, we may inadvertently increase the costs of drugdevelopment while trying to ensure cost-effectiveness of the drugs that finallymake it to patients. For the sake of payors, I hope the money they save todayby refusing payment will help offset some of the potential excesses later.
And for the pharmaceutical companies, the lesson might be:If you build it, they will come … but they won't necessarily pay for it, sobuild wisely.
Willis is the features editor of ddn. He has worked at bothends of the pharmaceutical industry, from basic research to marketing, and haswritten about biomedical science for almost two decades.
EDITOR'S NOTE: We have recently been informed thatthe NICE guidance regarding Zelboraf was not a final guidance, but rather a first-draft decision. The final guidance is not expected to bepublished before December, according to NICE. Similarly, theGilenya discussion was centered on a first-draft decision, and in itsfinal guidance, NICE recommended Gilenya for use by the NHS. Weregret any confusion we may have caused, and thank officials at NICE forthe clarification.

Randall C Willis

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