PDUFA

question: What is your take on the Prescription Drug User Fee Act? Do you think it is helping or hurting the FDA in the drug approval process, especially in the recent market removals and queries to drug safety? -- Dee

Dear Dee,
Definitely a negative. The bottom line impact is that the FDA is now beholden to the industry it is supposed to regulate for its funding. A business should please its customers. A federal regulator should have an arm's length relationship.

Rep. Maurice Hinchey (D-NY) introduced legislation this week that would send all the user fees to the U.S. Treasury, and force the Congress to appropriate funds to the FDA to carry out its tasks. This is a far better way to go. -- Merrill

Avastin

Please tell me what I'm missing. I'm having a hard time understanding the hullabaloo over Genentech's Avastin drug. Their stock has skyrocketed because of its 'success' in treating colon and now lung cancer patients. But in both trials survival with Avastin + standard Chemo drugs vs. just Chemo was 3 more stinking months!! So what?? I'm sure Avastin isn't going to be cheap... and it buys you 3 months. While all the euphoria over this drug? It's not curing anything.. the cancer figures how this momemtary road block and kills you 3 mos. later. I guess the 'war against cancer' has been so lacking in success that any news not matter how meager is good. --Kurt

Dear Kurt,

Alas, many cancer therapies provide only a temporary respite from the ravages of the disease. The FDA approves drugs that are better than nothing or better when added on to what already doesn't do much. Still, they charge quite a bit of money for those extra few months, and that probably accounts for the hubbub in the stock market. -- Merrill

A better way to fund drug innovation?

Reader Aidan Hollis writes:
What do you think of proposals to reward drug innovators directly based on the incremental therapeutic effect of their innovation?

My response:
Readers interested in University of Calgary economics professor Aidan Hollis' proposal for a government-run innovation rewards system should read his draft paper.

There is a growing movement among economists to scrap monopoly patents as the basis for fostering innovation and substitute awards systems or government or non-profit-run research institutes. The great drawback to most such ideas is that they are a direct affront to the intellectual property establishment. The primary virtue of Hollis' proposal is that he avoids the politically impossible task of eliminating patents. Instead, he bases his rewards on their utility (as measured by improvements in quality-adjusted life years, an complicated but useful tool used by economists to measure healthcare outcomes). He would set up a major government agency to evaluate the quality improvements generated by new drugs (a proposal that echoes my own in "The $800 Million Pill").

I can see some problems with his scheme. How would it deal with rare diseases? When patient populations are very small, even very large improvements in quality-of-life and extended life would not generate enough rewards to interest corporate innovators. It also doesn't deal with the growing problem of early stage research patents on genes and proteins and their projected (as opposed to defined) uses. This fencing of the basic science intellectual commons is emerging as a major roadblock to medical innovation, which, again as my book points out, is dependent on research at these early stages.

But the proposal goes a long toward suggesting an alternative to the current system that rewards anything patentable that can be successfully marketed, which defines much of what Big Pharma does. It would be interesting to hear others' comments.

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