A New York Times editorial today on the skyrocketing costs of cancer care asserted that "the Obama administration seems ready to eliminate laws, policies or conflicts of interest that may be driving up costs unnecessarily." That's an overly generous interpretation based on what I heard yesterday from Janet Woodcock, chief of the new drugs division at the rudderless Food and Drug Administration. If there is bold action planned by acting commissioner Frank Torti (a former cancer researcher) to help oncologists, patients and insurers like Medicare figure out the true value of new cancer drugs, she wasn't letting the cat out of the bag.

The venue was an Institute of Medicine forum on "assessing and improving value in cancer care," which brought together many of the leading policy thinkers in this increasingly contentious arena. Barely a day goes by without another major story about the pharmaceutical and biotechnology industry's efforts to beat back efforts at cost control in cancer care, which is increasingly seen as the next big income generator for Big Pharma. This morning's Wall Street Journal, for instance, finally reports (see this GoozNews post) on the "patient advocacy" coalition pushing to eliminate the $1.1 billion for comparative effectiveness research contained in the stimulus plan, or, if that effort fails, precluding any government agency from using the funds to consider comparative cost effectiveness in its studies. Many of the groups in the "patient" coalition receive most of their funds from the pharmaceutical industry.

(Note to Kohl-Grassley: Why don't you add a clause to the Physician Payments Sunshine Act requiring drug companies to disclose payments to non-profit organizations and vice-versa, especially now that some patient advocacy groups are investing in small drug firms willing to pursue promising therapies in their disease category?)

While comparative effectiveness research under the stimulus plan would be conducted, at least in the initial stages, by the National Institutes of Health, the Agency for Healthcare Quality and Research and the Centers for Medicare and Medicaid Services, the FDA could help. How? By requiring drug firms to generate better comparative information when they are conducting their initial proof-of-efficacy trials. It could crack down on companies that fail to follow through on post-approval trials, especially for anti-cancer drugs that gain market access under the FDA's accelerated approval rules, which have been in place since the AIDS crisis of the early 1990s.

And it could actively support the creation of registries where physicians in community practice would have to report the on- and off-label use of anti-cancer drugs and the patient results. These databases could then be used by researchers to evaluate the actual clinical effectiveness and side effects of drugs that in their initial approval or published off-label trials were either marginally effective or signaled safety problems.

The broad possibilities of registries -- and their limitations -- were outlined at the IOM meeting by Daniel Sargent, chief of biostatistics at the Mayo Clinic. "Registries are underutilized," he said. But he quickly added that "you won't pull me away from my belief that we have to stick to randomized data to reach broad conclusions."

One way to use registry data, he said, would be virtual clinical trials based on "cluster randomization," which would compare outcomes in areas of the country with different treatment patterns. Patterns of care diverge because the off-label uses of anti-cancer drugs -- and well over half of all anti-cancer drugs are used for indications not yet approved by the FDA -- are sometimes reimbursed by the local Medicare payment contractor in one area or state but not another.

"We at FDA are very supportive of cluster randomization," Woodcock said. "It's just not done very often." In response to a question about the general use of registry data to measure the effectiveness of off-label uses, she raised a high bar. "Very large registries are critical. Having data on three-quarters of a million cancer patients every year has got to have some benefit."

Meanwhile, Woodcock portrayed the FDA as caught in the middle on the issue of off-label promotion. Oncologists and drug companies back the new system of liberalized compendia listings (often written by physician-researchers with conflicts of interest, as the Times editorial noted). She also backed the liberalized new rules on medical literature distribution. After pointing out that the agency is under pressure from Capitol Hill (Rep. Henry Waxman of California in particular) to crack down on drug salesmen distributing journal article reprints that promote the off-label use of anti-cancer and other drugs, she said simply "they can distribute but not promote." One can only hope she wasn't speaking for the Obama administration on that issue.

So where are we? Compendia can now be paid by drug companies to get their off-label drug uses evaluated and, even with an equivocal rating, get reimbursed. Drug reps can distribute medical literature of dubious quality suggesting such off-label uses are useful. Patient advocacy groups backed by industry are demanding that no one evaluate the use of those drugs -- at least in any way that would be meaningful to patients and payers. Efforts on the Hill to create an effective means of collecting and evaluating data are being fought tooth and nail. And there's no one from the new administration in charge at HHS, CMS or the FDA.

I remain optimistic. The mood at the IOM meeting was that change is necessary, and the blank check trajectory of current policies is unsustainable. But some hint that the Obama administration is ready "to eliminate laws, policies or conflicts of interest that may be driving up costs unnecessarily" would be useful at this point.