From the BIO Convention - Making Medicines Personal

Ronald Bailey | June 28, 2011

At the Biotechnology Industry Organization's annual convention here in D. Herceptin C., the future of personalized medicine is a big topic. New biotechnologies based on genomics, proteomics, metabolomics and so forth are producing diagnostic tests that will help guide researchers and physicians in developing therapies that are targeted precisely at your specific illness. One can see glimmers of the future of personalized medicine in such diagnostic tests for HER2/neu gene in breast cancer. Patients who have that gene respond well to herceptin, whereas those who do not get no benefit from the treatment. Another new genotype test approved by the Food and Drug Administration identifies people (like me) who are more sensitive to the blood thinner Coumadin. A BIO "fun fact" that flashes up on the viewscreens in the each conference room is that there are 1,200 biotech diagnostic tests in use today.

An early Tuesday morning panel addressed the question of how will we afford personalized medicines? John Ridge, the director of Global Reimbursement and Healthcare Economics at Ventana Medical Systems, a division of the global pharmaceutical giant Roche, argued that we are already well down the path toward medical personalization. He pointed out that decades ago, physicians recognized only 5 types of leukemia. Today, there are 51 different types of leukemia and 58 different lymphomas which are all identified using specific biomarkers. Ridge asserted that payers seem reluctant to reimburse for diagnostics even though 70 percent of all health decisions in the U.S. are based on a diagnostic test. Using new personalized diagnostics produces better and more predictable clinical outcomes, reduces patient morbidity caused by side effects, and results in fewer treatments being wasted on patients who won't respond to them.

According to Thomas Bols, vice-president of corporate health policy and market access at Merck Serono in Europe, "personalized medicine is providing the right treatment to the right patient at the right time." Previously, a lot of medicine was based essentially on trial-and-error in which researchers and clinicians dosed patients with compounds to see if they might have a beneficial effect on an illness. Bols noted that most drugs are actually effective for less than 60 percent of treated patients. This means that 40 percent or more are taking treatments which do them no therapeutic good but often do produce harmful side effects."So using a drug is a waste for more than 40 percent of patients," said Bols. Clearly both patients and payers would want to avoid using ineffective drugs.

In addition, about two-thirds of drugs fail in Phase III clinical trials due to lack of efficacy. But it is often the case that a compound actually does work for subsets of patients. The biotech new diagnostics are helping researchers stratify patient populations and thus identify those who would benefit from a treatment while excluding those who don't. Coupling diagnostics with clinical trials could potentially result in smaller more elegant trials reducing research and development costs and getting drugs faster to patients. John Ridge from Roche added that his company was combining new diagnostic biomarkers in clinical trials for over 100 new therapeutics. While the diagnostic tests may be bundled as part of a new therapy, it is important not to lock in the approval of new drugs with specific diagnostics since new and better diagnostics come along all the time.

rather|6.28.11 @ 6:57PM|#

Coupling diagnostics with clinical trials could potentially result in smaller more elegant trials reducing research and development costs and getting drugs faster to patients.

It could potentially lead to discrimination. I'd like to see these issues addressed beforehand

-|6.28.11 @ 7:05PM|#

You'd like a long, drawn-out legal argument over the definition of "discrimination" before seeing anyone who needs help get help? What is it about "getting drugs faster to patients" that bothers you?

rather|6.28.11 @ 7:53PM|#

Why does it have to be long or drawn out?

Why not nip any problems?

|6.29.11 @ 12:04AM|#

Who is being discriminated against that would warrant the delay?

rather|6.29.11 @ 12:12AM|#

Targeted specific treatment would by definition group people by race, sex, and even age. Aids drugs that were toxic in treating children proved to be helpful to adults.

Drug companies would weigh the cost VS. profit in drug engineering. Easily, they could easily decide to go Ferengi on certain populations.

|6.29.11 @ 1:32AM|#

Uh.. so you think the FDA would hold up approval of a drug based on the grouping of test subjects by their targeted demographics?

Every company weighs the costs vs. expected profit in regards to their products. If they can't cover the costs, it's doubtful they would even produce it. I'm not seeing what you're getting at here.

rather|6.29.11 @ 2:00AM|#

No, my concern is not with the FDA. In fact, I was referring to drug development outside the US, and of India in particular. My concern would be that drugs for women would not be a priority in cultures that find less value in girls.

|6.29.11 @ 9:06AM|#

ok...

|6.29.11 @ 10:21AM|#

Nothing the government can't solve!

Wait...

rather|6.29.11 @ 12:48PM|#

Inte skryta John. Det är så amerikansk av dig;-)

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