It's Not Rocket Science...It's Harder

It's Not Rocket Science...It's Harder

09.14.12 | By John Castellani

This week, PhRMA presented the 2012 Research & Hope Awards to researchers, academics and patient advocates for their work in the fight against Alzheimer's disease (check out video from the event here).

We selected Alzheimer's disease as the focus for this year's honors because of the looming challenge it poses to a growing number of patients, caregivers, our healthcare system and our national economy. But I want to talk for a moment about how Alzheimer's and the work being done to combat it - the successes and the setbacks -- are also examples of the challenges we face in all medical research and when creating innovative medicines and therapies.

When I spoke about these challenges at the awards ceremony, I tried to put them into an understandable context. The recent landing of NASA's Mars Curiosity mission for example.

Like so many, I was excited by the mission's success and the stunning pictures sent back from Mars. But, NASA's success - like all scientific and technological advances - was built upon prior setbacks and the ability of the scientists and engineers to learn how to do it better the next time. The significant statistic that defines this for NASA's Mars endeavors is that more than half of its 40 Mars missions failed.

In other words, there were more than 20 attempts to send a probe to Mars where the rocket didn't launch; where the probe crashed landed; where the probe deployed but we lost communications or where some other difficulty sunk the mission. But, nearly half made it. The rocket got to Mars and the probe did pretty much what the scientists hoped it would.

This is rocket science after all, and the researchers, scientists and engineers took every setback in stride, learned important lessons and made every effort to correct their errors and miscalculations in the next rocket they built.

Now, when a rocket doesn't get to Mars, it is disappointing. Years of work and a huge investment are lost. But the day after, everyone at NASA gets up, goes back to work and tries to glean what lessons they can before starting all over again.

Creating new medicines - whether for Alzheimer's disease or any condition - is a little like rocket science, but magnified. When it comes to Alzheimer's, for example, a new report released by PhRMA shows that since 1998, 101 potential drugs to fight the disease fell short in development and testing while three medicines were approved by the U.S. Food and Drug Administration.

That's 34 setbacks for every approved treatment.

What's true for Alzheimer's disease is true for all biopharmaceutical research. Out of every 5,000-10,000 compounds examined as potential treatments, only one will ever be approved by FDA for patient use. That's a lot of setbacks, with many coming late in the 10 to 15 years of R&D and an average cost of $1.2 billion needed for approved medicines.

Unlike space missions, when a potential new therapy encounters a setback there is an enormous human toll. There are patients hoping for new medicines to help them battle their condition. There are caregivers waiting on new tools to help patients and provide them with relief. Also, there is the economic costs of treatment and continuing care that we all bear - estimated to be $1 trillion a year by 2050 in the case of Alzheimer's.

Today, there are nearly 100 potential new medicines in the pipeline to help treat Alzheimer's disease. Only a handful, if that, will ever be approved for patient use. But, what is critical is what we learn both researching new approved treatments as well as from potential medicines that don't make it.

The odds really do seem stacked against medical progress. Yet over 300 new medicines to treat a wide range of conditions have been created by America's biopharmaceutical research industry and approved by the FDA since 2000. That is both progress and hope.

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