<Linking pharmacogenomics to genomics will lead to increased output.
Lehman/McKinsey sort of referred to this in the Q&A after their presentation. Didn't fit their thesis, so they sort of brushed it off as best I recall.
Personally, I think your point will eventually have the single most dramatic effect on drug development expense of anything I can think of. If you can identify a subgroup ahead of time for which the drug works (or doesn't work) it has a huge effect on the size of the Phase III efficacy studies you need.>
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Pharmacogenomics Streamlines Clinical Trials
Pharmacogenomics won't replace current standards and methods — but it will mean more efficient trials.
By Kelly Kagamas Tomkies
for Office.com
Feb. 6, 2001 — There is no question that pharmacogenomics is gaining momentum, but what impact the new technology will have on the clinical trials industry remains to be seen. While experts say that pharmacogenomics will speed up the process for clinical trials, it will not allow scientists to eliminate any steps of drug discovery.
Pharmacogenomics builds on the results of the Human Genome Project and advanced genetic tools. The goal is twofold: to determine what the differences between patients' DNA could mean for drug efficiency and to develop diagnostic tests. Meeting this goal would allow pharmaceutical companies to produce drugs that are personalized in accordance with an individual's unique genetic sequence.
"I think pharmacogenomics will mean more efficient trials," says Anne Bailey, VP of diagnostic development with Variagenics, a pharmacogenomics firm in Cambridge, Mass., adding that it allows scientists to see who are responders and who are nonresponders earlier in the trial process. "Then the efficacy data is much more powerful when presented to the FDA."
Specifically how pharmacogenomics effects clinical trials depends on the phase, says Bonnie Gould Rothberg, M.D., pharmacogenomics group leader at CuraGen Corp. in New Haven, Conn. "There are two ways pharmacogenomics can have a positive impact on clinical trials: in the preclinical stage and Phase I, and at the end of the process in Phase III," says Rothberg.
"There are two ways pharmacogenomics can have a positive impact on clinical trials: in the preclinical stage and Phase I, and at the end of the process in Phase III."
— Bonnie Gould Rothberg, M.D.
group leader for pharmacogenomics
CuraGen
Companies would go bankrupt trying to do the chemistry involved on 100 new targets to determine which is the most promising, notes Rothberg, but with pharmacogenomics, scientists can create a database that can screen targets for the worrisome gene.
"Let's say you gave your new target to mice during a 28-day toxicity study," she says. "They were healthy and happy. Then you go to Phase I, and in 48 hours, patients' liver insulin has increased. Why?"
Pharmacogenomics allows scientists to return to gene-expression profiles and find the offending gene — if the cause is genetic. That means potential new drugs can get to Phase I faster. But the biggest impact, says Rothberg, will be in Phase III trials.
"In a Phase III trial, you are testing the drug against a known therapy to see if it has equal or better efficacy," she says. Pharmacogenomics studies could help scientists determine why a drug has a less-than-expected efficacy rate. Companies can then decide whether they want to proceed or cut their losses.
Bailey notes that the efficacy of a new drug often charts like a bell curve. "With pharmacogenomics, we can find out earlier and are better off, whether the drug has the efficacy we are looking for. Let's say only 30 percent of trial patients respond," she says. "Companies can decide whether they want to capture 100 percent of 30 percent of the market or if the trial needs to be stopped."
Bailey says companies are finding the 30 percent markets more and more appealing.
"We are currently conducting a Phase II trial with a partner," she says. "There is a small percentage of patients that are responding very well, while others are responding marginally or poorly. Pharmacogenomics studies will help the company decide whether it will continue or use that genetic factor as a criteria for patients in a more extensive new trial. Pharmacogenomics is opening up new areas for companies."
Rothberg cautions, however, that pharmacogenomics will not replace current trial practices.
"It will not supplant good clinical practice," she says. "I don't see massive changes in the process. You are dealing with conservative entities: toxicologists, physicians, and the FDA and regulatory officials. They are not going to delete any steps because of pharmacogenomics." |
