Developing medicines is a minefield that Tranzyme Pharma has navigated well so far. But the Durham company is about to embark on one of its trickiest missions.
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Buyers looking for a kitchen appliance, computer or car can turn to a variety of product reviews and ratings, but there’s no version of Consumer Reports that helps physicians or patients with information about which treatments pack the most bang for the buck.
For that matter, in cases where multiple treatments are available, there may not even be any research to see which treatment does better under what circumstances.
Sure, health care providers like Duke University Health System and health insurers like Blue Cross Blue Shield keep extensive medical records to track an allergic reaction to a medication, length of stay in intensive care and other treatment results for each patient. Kaiser Permanente, the largest managed care organization in the U.S., developed a reputation early on for denying coverage of treatments that are not as effective as their cost might suggest. But in general, health insurers and providers don’t use comparative information extensively. They also rarely share the data.
In the United Kingdom, regulators look at how new medicines fare against existing ones in clinical and cost effectiveness before they approve the new medicines for sale.
In the U.S., the standard test to get regulatory approval for a new medicine is a comparison with a placebo, a drug that is designed to do nothing, Scott Evangelista, a Deloitte consultant who lives in Chapel Hill and helps life science companies with tough problems nationwide, told an audience of hundreds this week at the North Carolina Biotech conference in Raleigh.
And when the new medicine passes the test, the drugmaker can claim, “We’re better than the sugar pill,” Evangelista said. The test results provide few clues how effective the new medicine is in a real-world situation.
But the pressure is rising to get the right medicines to the right people at the right time and the right value, he said.
For the more than 500 biotech companies in North Carolina – the majority of them call the Research Triangle area home – this could mean significant changes.
“You have to get your customers involved and you have to listen to things you don’t really want to hear,” Evangelista told the crowd at the conference.
Things like comparing a new drug head-to-head with existing ones, which can end favorably for the competition. Zeroing in on a small group of patients who stand to benefit most from a new drug, thereby limiting the drug’s sales potential. Adding a test to make sure only the patients who benefit from a drug get it, which makes the treatment more expensive and less convenient. Or suspending development of a drug, because it will cost a lot more than existing ones but is only a little bit more effective.
A 2010 report in the Journal of the American Medical Association suggested that of more than 300 studies the top six medical journals published over 15 months in 2008 and 2009, 43 percent included comparisons of different medications. Only 2 percent of the studies analyzed which treatments were better values.
Indeed, of the more than $2 trillion in annual U.S. health expenditures, only an estimated 0.1 percent is spent on researching the comparative effectiveness of medical treatments, according to a 2007 report of the Congressional Budget Office.
The lack of information has contributed to health care costs that have little to do with how well patients do.
Case in point: Blood-pressure lowering drugs.
An 8-year-long study that followed more than 32,000 high-risk Americans age 55 and older found that diuretics were not only superior but also cheaper than newer enzyme inhibitors and calcium-channel blockers, according to outcome results published 2002 in JAMA.
With annual health care expenditures doubling to 16 percent of the U.S. economy in about 30 years and projected to reach 20 percent by 2016, this disconnect between costs and outcomes has insurers, employers and politicians pushing for a change in course.
In the past two years, Congress approved health care reform that stresses patients’ rights, earmarked $1.1 billion for comparative effectiveness research as part of the federal stimulus bill and established the Patient-Centered Outcomes Research Institute to set research priorities, oversee clinical trials and hand out money.
“None of this would be happening if cost wasn’t an issue,” Evangelista said.
Of course, comparative effectiveness isn’t a new concept in U.S. health care. It’s been around for more than 30 years. Two federal institutes to evaluate health care technology have opened and closed since 1978.
But this time around, America is graying – 10,000 baby boomers turn 60 every day, said Deidre Connelly, president of GSK North America Pharmaceuticals and a featured speaker at the biotech conference – treatments for chronic diseases account for an estimated 75 percent of U.S. health care spending and the rapid switch to electronic medical records will make comparative effectiveness analyses easier within a few years.
One Midwest health insurer has already launched its own research arm with plans to analyze its medical records for comparative effectiveness and conduct its own clinical trials, according to a report by CenterWatch, a trade publication that tracks the contract medical research industry.
This time, Evangelista said, “it’s going to happen.”
GlaxoSmithKline wants to scale back research and development and the cuts could affect jobs at the British drugmaker’s U.S. headquarters in Research Triangle Park, IBM unveils the $360 million cloud computing center it established on its RTP campus and a Durham startup reels in $10.5 million in venture capital and a deal with Burlington-based medical testing giant LabCorp. Read more…
Imagine a chemist, a bioethicist, a pharmacist and a physician come together to discuss personalized medicine. The last thing you expect them to talk about is the need to wear matching jackets like TV weather forecasters.
But that’s exactly what a panel of experts did Monday at the RTI Fellows Symposium at the University of North Carolina’s Friday Center in Chapel Hill.
Micell Technologies receives $15 million from St. Jude Medical, GlaxoSmithKline curbs its funding educational programs that bring doctors up-to-date, Duke Genome Center gets a $19.5 million grant and a consortium of area universities and nonprofit organizations are chasing a piece of the $63 billion the Obama Administration wants to spend over the next six years on global health care programs.
Salix Pharmaceuticals, a Morrisville company that specializes in gastrointestinal treatments, has seen its shares rise nearly 50 percent in the past two days.
Investors rushed to trade the stock after Salix announced results from two late-stage studies Monday that showed rifaximin works better than placebo in treating non-constipation irritable bowel syndrome. Salix sells rifaximin for traveler’s diarrhea under the name Xifaxan.
A report offers hope that federal funds could become available for economic development in innovation hot spots such as the Research Triangle area, Bayer CropScience adds a research collaboration to recent efforts of creating better biotech seeds and Family Health International, a Durham organization that aims to improve public health worldwide, uses realty shows to stem the spread of HIV/AIDS.
Tranzyme Pharma is one of dozens of drug development companies in North Carolina’s Research Triangle area, a biotech hot spot that is ranked third in the nation by number of companies. The Durham company has diligently advanced therapies based on a hormone that was discovered a decade ago, a technology also used by two rivals. Now, Tranzyme’s Board of Directors has to decide how to pay for the final development step and get its drugs to market: Go public, sell the company or go back to its investors hat in hand one more time.
This is the first part of three.