Latest Drugwonks' Blog
From Idea to Commercialization”
An Executive Program for Biotechnology and Medical Device Entrepreneurs and Managers.
The Faculty of Management of Tel-Aviv University, the Israel Life Science Industry Organization (ILSI), The Center for Medicine in the Public Interest and International Institute for Biotechnology Entrepreneurship (a non-profit global contribution by Burrill & Company) are now holding the second program “Health Care Technological Innovation - From Idea to Commercialization.”
This program focuses on the critical elements in the formation, launch, and strategic and operational management of health-related technologies, with a particular focus on companies working in medicinal biotechnology, enabling technologies for life-science research, and medical devices used in human diagnosis and treatment.
We have had many excellent presentations focusing the value-driven reimbursement, personalized medicine as a platform for comparative effectiveness and the role of the Critical Path as a framework for demonstrating risks and benefits of products over the life of a product.
At the same time, Peter Pitts is featured in The Journal of Biolaw and Business Special Commemorative Edition published in honor of Israel's 60th anniversary. His article: "FDA and the Critical Path to 21st Century Medicine" is based on his presentation at the first CMPI sponsored seminar for Israeli life science startups in November 2007.
For the abstract and more info on The Journal go to www.biolawbusiness.com
PS I am also in Israel to see my son receive his beret after completing basic training in the IDF.
Here's the photo.
Everyone, it seems (including my hero, Will Shortz) wants to talk about safety.
And nobody more so (or more appropriately so) than the FDA. Remember -- it was Milton Friedman who said that the FDA was "obsessed with safety." And while Professor Friedman didn't mean it as a badge of honor -- it was taken as such by the 10,000 dedicated public servants at the agency.
But, unfortunately, safety seems only to "sell" when there's a tabloid quality to it. Ask yourself this: What do Vioxx and Avandia and Heparin all have in common? Answer: Page One coverage.
In today's New York Times, reporting on the FDA's new "Sentinel" program appears on Page A18 -- on a slow news day.
It's just another example of tabloid journalism trumping solid reporting. And, for the record, the article in the New York Times by Gardiner Harris is a solid piece of reporting. Have a look :
www.nytimes.com/2008/05/23/washington/23fda.html
Tabloid is easy. Quality is hard.
Tim, as you may already know, has announced his retirement from Eli Lilly & Co. where he served for 22 years (most recently as Vice President for Global Regulatory Affairs).
Tim is a scientist -- and a believer in the art of the possible. Tim-Possible.
When I served at the FDA, Tim offered sage advice and pointed criticism. Both were served with the best interests of the public health in mind -- and his inevitable impish smile.
His one note, high-pitched "HA!" is a trademark.
One of the architects of PDUFA, Tim was also one one of the first people the FDA recruited to discuss the nascent concept of the Critical Path. He loved the idea but also helped to identify some of the problems.
Tim is a guy who, as the old Jewish joke goes, "knows where the rocks are."
His regulatory knowledge is nonpareil and his optimism is contagious.
We wish him well in all of his new endeavors.
Yesterday Jamie Love e-mailed Bruce Sterling (the science fiction) and asked him to help come up with a new term for counterfeiting because, “There are significant problems with overusing the term.” Science Fiction? Give me a break.
In any event, it's a moot point because there is already a word that accurately describes international prescription drug counterfeiting – that word is “crime.” And it translates accurately into most languages.
(As far as Jamie's "significant problems" are concerned, that's another issue for another time.)
Personalized medicine for quicker cures
As we listen to presidential candidates and Congress debate how to control health care and drug costs and raise drug safety standards, most solutions lead to increased government regulation. That is underlined when debate turns to cancer care with high-cost drugs and low returns in quality of life and survival.
But more government regulation on reimbursement rates and overly burdensome drug development requirements will result in less medical innovation and rationing of medical care. What's missing from the debate is the public policy encouragement to produce more "smart" medicine.
One of the hardest parts about being an oncologist is telling patients they have cancer. Even harder, though, is knowing that the drugs prescribed don't work well in most patients and/or will cause severe side effects in others.
That is changing. Scientists working at biotechnology companies are using the knowledge of the human genome to develop drugs targeted to help individual patients. That concept -- known as personalized medicine -- holds the promise of improving care for patients and quickening the pace of drug development so we can "hurt the cancer more than the patient."
The human genome has shown us how our genes affect our health -- including whether we are at risk of disease. Recent advances in DNA research also have taught us how to examine what happens to a cancer tumor's gene or set of genes when they are turned on or off in the presence of a drug.
At my company, Cell Therapeutics Inc., one of our subsidiaries, Systems Medicine, is examining the function of genes in a tumor through a process called genetic profiling. SM examines about 30,000 relevant genes from tumor cells and tests them to determine which gene or genes make the tumor susceptible to the effects of different cancer drugs. Using that technique, we can identify which cancers are sensitive to which drugs. It is a fundamental shift in approaching the dilemma of which is the right drug for the right patient while exposing the least number of patients to a drug's severe side effects.
The power of this technology is enormous. Rather than imposing new regulations on manufacturers or physicians, the most effective way to ensure safety is to use scientific advances to better understand why some patients react badly to some drugs, and encourage development of new diagnostics and therapies that take that into account.
Adoption of personalized medicine will mean that patients will get quicker access to the newest cancer therapies. The current time and cost of drug development is about 16 years and $1.7 billion. Cancer drug development is actually the least-efficient in all of medicine. Genetic profiling could shorten clinical development time and costs because only those patients likely to benefit will be enrolled in clinical trials, making it easier for regulators to make safety and efficacy decisions more quickly.
But, federal regulators need to understand the value personalized medicine can bring and develop policies that encourage development of these new drugs and diagnostics, while doctors need to understand the power of genetic profiling so they can make good prescribing decisions. Some day, instead of physicians diagnosing lung cancer or colon cancer based on where the tissue is from and the way a tissue looks under the microscope, we may be referring to a genetic profile of a tumor in your lung or colon tissue recognizing that the genes that make each of us unique also makes our cancers unique.
The product is Ivory Soap -- and it's only 99 and 44/100th% pure. Should it be taken off the market?
Silly? Not when you consider that the current debate over drug safety is being hijacked by the Panjandrums of the Precautionary Principle. What we need is a debate on smart safety.
Have a look at this new op-ed in The Journal of Life Sciences, "A Balancing Act:."
Here's the link:
www.tjols.com/article-662.html
And here's a taste:
His video interview can be found here:
www.vimeo.com/1045899
(Other "newsmakers" interviews can be found at www.cmpi.org)
Dr. Weber, we are proud to say, serves as Chairman of the Board for the Center for Medicine in the Public Interest (the public policy home of www.drugwonks.com).
According to Bloomberg, two companies that produce different types of stem cells have signed contacts to sell their products to drugmakers, showing the new technology will be used to help discover medicines not just to repair or replace damaged cells.
California Stem Cells Inc., an Irvine, California, biotechnology company that turns embryonic stem cells into neurons, said today it's selling the brain cells to researchers trying to find drugs to treat Lou Gehrig's disease. CellDesign Inc., of
The efforts of these two closely held companies indicate stem cells will aid in the search for old-fashioned drugs long before they're infused into patients. It also suggests that the first businesses to benefit from stem cell technology will be traditional pharmaceutical companies and their suppliers not developers of new kinds of therapies.
``It's similar to what happened in the last century with molecular biology'' and gene therapy, said John Hambor, CellDesign's founding chief executive officer, in a telephone interview yesterday. ``We're now going down a similar path with stem cell biology. While it may lead to cures by itself, it will drive the next generation of drug discovery.''
Here's a link to the complete story:
http://www.bloomberg.com/apps/news?pid=20601124&sid=aYKfThhsUVwI&refer=home
At a recent European Commission-sponsored high level conference, Internal Market Commissioner Charlie McCreevy, commented that the EC wants practical, pragmatic suggestions for fighting the “modern-day highway robbery” known as piracy and counterfeiting. McCreevy stressed that more regulation is not the answer, and that solutions lie with public-private cooperation.
Only governments can put into place enhanced investigatory assets and harsher penalties.
Public/Private cooperation? Certainly. But only governments can catch and keep criminals in jail.
When it comes to discouraging international prescription drug counterfeiting, what we need is a more robust cell culture.
Now it turns out that Avastin, the drug that Goozner and Mahar love to hate shows considerable promise in extending average progression free survival by up to nine months. Contrary to the twisted narrative put forth by critics, the discovery of the off-label use of Avastin for gliomas came from academia without industry funding (which of course Goozner also criticizes...either way industry is damned, pilloried for inventing useless new uses and attacked for not being innovative)
Here's what happened:
"In the pilot study, the researchers found that dual therapy with Avastin and the chemotherapy drug irinotecan either shrank the tumors or restricted their growth in nearly all cases for up to three months longer than comparative therapies. Three months is a significant advance when dealing with these aggressive tumors, Vredenburgh said; common current treatment normally offers only six to 12 weeks of halted growth before the tumor grows and spreads, ultimately destroying cognitive and physical function and leading to death."
Following this discovery, Genentech, Duke University and NIH worked together to conduct an expanded study.. The FDA initially resisted because of side effects such as bleeding, but since 40 percent of all brain cancer patients experience bleeding for a fatal illness the risk seemed worth it (to everyone except Maggie Mahar www.healthbeatblog.org/2008/02/the-wall-street.html
James Vredenburgh, M.D., a brain cancer specialist at Duke's Preston Robert Tisch Brain Tumor Center and lead researcher on the study noted: "Going forward, we will also explore the efficacy of this treatment in newly diagnosed patients," he said. "Ultimately, our hope is that this will offer a real weapon in what is now a very limited arsenal for treating a very challenging cancer."
www.cancer.duke.edu/btc/modules/news/article.php
That includes patients like Senator Kennedy. Let's hope that his doctors rely upon experts like Vredenburgh in making treatment decisions, not cynical second guessers like Goozner and Mahar who would let hatred of drug companies color life and death decisions.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
