What Has Five Hats and a Hundred Thousand Hands?

So I’m with my family and 50,000 of our closest friends at the Live Earth concert near New York City when these five college aged guys with backward hats start whooping and hollering like they had found gold or were giving birth to those little creatures in Alien. I was hesitant to turn around and inquire “Are you ok? Have you been invaded?” Soon I noticed that the noise level was way beyond five guys, in fact it had started to spread in a pattern. Great, I thought, we’re all being invaded.

Soon the noise was way away from us and we could see it and it was…The Wave…

Let’s just say those five guys were pretty proud of themselves for moving 50,000 people to follow their lead. Five hats moved 100,000 hands. Go figure. We understand that challenge at FasterCures. We have been trying to move a $100 billion research enterprise in this country on a budget of only a few million dollars a year. Now, with our grant from Sumner Redstone of $35 million, we have a chance to start a real wave. But, it is still a huge challenge for even $35 million to move $100 billion. And, that is why we are gathering our “five hats” -- smart friends and advisors to help us determine how to create a wave that will move $100 billion to accelerate the discovery of cures. Seems impossible, but then again…I was at a concert the other day and there were these five guys with backward hats…

Greg

The FDA and You

You might be shocked to hear that the Superintendent of the Montgomery County schools has a budget akin to what FDA Commissioner von Eschenbach has to run an agency that regulates a whopping 25% of American consumer spending. Think about how you use FDA-regulated products each day -- when shampooing, brushing our teeth, taking aspirin or prescription drugs, applying makeup, putting on sunscreen, talking on a cell phone, cooking food in a microwave, and even watching TV. It starts to seem like there is little in our daily lives that isn't affected.

In recent weeks, I've been participating in hill visits with the FDA Alliance (I also sit on their Board) to talk to Congressional staff about FDA's resources. We've been making headway to increase awareness about the need for increased appropriations for FDA. We have yet to meet with anyone who disagrees with the argument that the agency needs more money. How much of an increase we'll see remains to be seen, but the next few weeks will be critical.

The FDA budget has been going backwards not forwards because of inflation, increasing costs, expanded missions, and scientific advancement. The current budget levels are far from adequate. We're asking for $2 billion for fiscal year 2008 (plus user fees on top of that).

I think we'd all agree we look to the FDA to be at the ready as scientific innovation flows through the pipeline. Otherwise what's the point of investing in innovation in the first place? We're expecting cures, and we'd like them sooner than later. The agency needs to be a cutting-edge regulator, one that is a "Gold Standard" worldwide. We need to be maximizing the potential of things like large datasets of electronic health records. This will aid in monitoring drug safety.

The list of things that FDA staff are thinking about now is no doubt long, but they can't scratch the surface without adequate funding. We are all either patients now, or will be one at some point. We need a strong and robust FDA. The 111 members of the FDA Alliance representing consumers, patients, professional societies, and industry all uniformly agree on that point. Increasing FDA's budget is a win-win for public health and the economy. Wouldn't you agree?

Margaret Anderson, COO, FasterCures

Working For You?

I had all of 4 minutes on CNBC this week – which I shared with Merrill Goozner -- to answer the question, “Should the FDA be speeding cancer drugs to market?” What a question.

Answer: YES -- if they work, NO -- if they don’t! (Merrill agreed. that left 3 minutes of dead space.)

But there’s the rub. What does it mean for a drug to “work?” Is extending life three months at a cost of ten to twenty thousand dollars per patient “working?” Is a drug that has a 92% chance of not being a placebo effect “working” compared to a drug that has a 95% chance?

This issue came to a head at the recent meeting of the American Society of Clinical Oncologists – otherwise known as “cancer doctors.” According to the New York Times, several people who had spoken out against a particular cancer drug received threats, even death threats. Apparently some patients felt they had the right to have this drug approved as a last resort even if many scientists felt it did not “work.” And if those scientists didn’t support “saving their lives”, these angry patients felt comfortable threatening them. This is an example of a system that is not working.

Or rather, the system is working just as we set it up to work. We have a $100 billion research enterprise feeding products into a $1 billion drug review system, funded to an ever growing degree by industry user fees. At the front end, we have some pretty strict rules for what constitutes “working.” And anything less than a high “non-randomness” score (i.e. 95% chance of not being random) won’t “work.” Try telling that to terminally ill patients who want to take a drug that has a score of 90 or 85.

Meanwhile, those user fees have been restricted for years from being applied to post market surveillance of newly approved drugs. If you can’t do post market surveillance, you don’t know how a drug is working in the real world until someone reviews random adverse event reports from doctors and starts comparing them to previously obtained clinical trial data that may or may not have tested for the reported effect. And then when deadly effects are discovered in some people, all hell breaks loose because nobody was monitoring whether the drug “worked.”

Avandia. Vioxx. Accutane. Thalidomide. All these drugs work for some people in some very important ways. For other people, they don’t just not work, they’re deadly. Until we have a system that works, it will be hard to tell whether the drugs are working ….or wasting both money …and lives.

Greg

Why We Won the War

Welcome to this, my inaugural post, on the new Fastercures blog. I hope you'll check in with us early and often and bring your friends. Comments are welcome and ideas even more so.

So maybe you heard the news that FasterCures received a $35 million (It feels really good when you put your little finger in your cheek and turn it when you say $35 million dollars) grant from Sumner Redstone. I sat with him the day of the announcement and asked a question that had been on my mind for a long time -- or ever since I finished reading "Cryptonomicon" by Neal Stephenson a month ago.

"So, Mr. Redstone, " I asked, "When you were breaking codes in the Pacific in WWII, (Gentle Reader, you probably didn't know that Redstone was a code breaker in the War, stick with us for more surprises) how did you act on what you learned without giving away to the Japanese that you had broken their codes?"

"Not a problem," said he. "They didn't believe it was possible to break their codes so nothing we did looked suspicious to them. The Chicago Tribune even published that we had broken their codes and the government was going to indict them for treason until we learned the Japanese didn't believe it."

Glad I asked.

You see, people get confused with this "seeing is believing" business. I have always felt it was the other way around. You can't see what you don't believe in. People who believe in something the rest of us can't see and then make it visible to us, we call magicians, or inventors, or visionaries. People who can't believe in something until they see it, we call "objective, " or "realists," or "practical." (Some historians argue that the Native Americans couldn't see, much less grok (look it up, Stranger in a Strange Land, by Robert Heinlein) the pilgrims approaching their shores because they had no mental concept of ships and people crossing the water.)

You see (sorry), here at FasterCures we believe in something we can't see -- that we can cure many of the diseases that cripple and kill us in this generation. Say that at a science convention and see what happens. A group of very objective practical people will guffaw and turn away. Ignore them. Find the one or two people who come up to you and say "yes, I know." They are the ones who believe it and will make it happen.

We won WWII because our enemies could not believe that we could break their codes and they couldn't see what we were doing as a result. The war on disease is one we can win, but you have to believe it first in order to see it later....or sooner.

That's it for now.
This is Greg Simon at FasterBlogs for FasterCures