(Press-News.org) Only about half of new drugs approved in the last decade had comparative effectiveness data available at the time of their approval by the U.S. Food and Drug Administration, and approximately two-thirds of new drugs had this information available when alternative treatment options existed, according to a study in the May 4 issue of JAMA.
In 2009, Congress allocated $1.1 billion to comparative effectiveness research. According to the Institute of Medicine, such research is defined as the "generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care." Comparative effectiveness information on drugs is most useful to decision-makers shortly after marketing approval, when observational data from routine care and data from large head-to-head trials comparing multiple treatments are not yet available. "Comparative effectiveness is taking on an increasingly important role in U.S. health care, yet little is known about the availability of comparative efficacy data for drugs at the time of their approval in the United States," according to background information in the study.
Nikolas H. Goldberg, and colleagues from Brigham and Women's Hospital and Harvard Medical School, Boston, conducted a study to determine the proportion of recently approved drugs that had comparative efficacy data available at the time of market authorization in the United States and to examine trends in availability of this information over time and by therapeutic indication. Data for the study were derived from approval packages publicly available through the online database of drug products (new molecular entities [NMEs]) approved by the U.S. Food and Drug Administration (FDA) between 2000 and 2010. The researchers analyzed whether eligible efficacy studies were head-to-head active controlled trials and whether the results of such studies were available in the approval packages.
The authors identified 197 eligible approved NMEs between 2000 and 2010, of which 100 (51 percent) had comparative efficacy data available at the time of market authorization. After excluding orphan products (n = 37; products or drugs that may be useful for common or rare diseases but which are not considered commercially viable) and other NMEs approved for indications for which no alternative treatments existed (n= 17), the proportion with available comparative efficacy data increased to 70 percent. On a yearly basis, the proportion of NMEs with comparative efficacy data (excluding orphan drugs and those for which no alternative treatment existed) varied between 50 percent in 2008 and 92 percent in 2010.
The researchers found that availability of comparative efficacy data was more common for some therapeutic indications, including diabetes mellitus (89 percent) and infectious diseases (73 percent), than others, such as hormones and contraceptives (33 percent), and cancer (35 percent). After excluding orphan drugs and products approved for indications for which no alternative treatments existed, the proportions by therapeutic indications were more similar. The authors also found that compared with those drugs that received standard review designations, NMEs that received priority review designations were much less likely to have comparative efficacy data.
The researchers note that although comparative efficacy data meeting their minimal criteria were available for approximately half of all newly approved NMEs, they did not assess the extent to which the publicly available data are informative enough to provide a basis for prescribing and coverage decisions.
"In conclusion, we found that publicly available documents include results of at least 1 head-to-head trial with an approved alternative for approximately half of all newly approved NMEs. Strategies are needed to enhance the accessibility of, and ultimately the use of, this information, particularly in the early marketing experience, when comparative effectiveness data from other sources are scarce or nonexistent."
###
(JAMA. 2011;305[17]1786-1789. Available pre-embargo to the media at www.jamamedia.org)
Editor's Note: Please see the article for additional information, including other authors, author contributions and affiliations, financial disclosures, funding and support, etc.
To contact corresponding author Joshua J. Gagne, Pharm.D., M.S., call Kevin Myron at 617-534-1605 or email kmyron@partners.org.
Many new drugs did not have comparative effectiveness information available at time of FDA approval
2011-05-04
ELSE PRESS RELEASES FROM THIS DATE:
Structured exercise training associated with improved glycemic control for patients with diabetes
2011-05-04
Implementing structured exercise training, including aerobic, resistance or both, was associated with a greater reduction in hemoglobin A1c levels (a marker of glucose control) for patients with diabetes compared to patients in the control group, and longer weekly exercise duration was also associated with a greater decrease in these levels, according to results of an analysis of previous studies, published in the May 4 issue of JAMA.
"Exercise is a cornerstone of diabetes management, along with dietary and pharmacological interventions. Current guidelines recommend that ...
Turning 'bad' fat into 'good': A future treatment for obesity?
2011-05-04
By knocking down the expression of a protein in rat brains known to stimulate eating, Johns Hopkins researchers say they not only reduced the animals' calorie intake and weight, but also transformed their fat into a type that burns off more energy. The finding could lead to better obesity treatments for humans, the scientists report.
"If we could get the human body to turn 'bad fat' into 'good fat' that burns calories instead of storing them, we could add a serious new tool to tackle the obesity epidemic in the United States," says study leader Sheng Bi, M.D., an associate ...
Breast cancers found between mammograms more likely to be aggressive
2011-05-04
Breast cancers that are first detectable in the interval between screening mammograms are more likely to be aggressive, fast-growing tumors according to a study published online May 3rd in the Journal of the National Cancer Institute.
Other studies have shown that cancers diagnosed between mammograms, known as interval cancers, tend to have a worse prognosis than those detected during routine screening. This study examined the difference between "true" interval cancers—those not detectable on the previous mammogram—and "missed" interval cancers—those not detected on ...
UCLA scientists discover new way to wake up the immune system using nano vaults to deliver drugs
2011-05-04
UCLA scientists have discovered a way to wake up the immune system to fight cancer by delivering an immune system-stimulating protein in a nanoscale container called a vault directly into lung cancer tumors, harnessing the body's natural defenses to fight disease growth.
The vaults, barrel-shaped nanoscale capsules found in the cytoplasm of all mammalian cells, were engineered to slowly release a protein, the chemokine CCL21, into the tumor. Pre-clinical studies in mice with lung cancer showed that the protein stimulated the immune system to recognize and attack the ...
Thylacine hunting behavior: Case of crying wolf?
2011-05-04
PROVIDENCE, R.I. [Brown University] — Its head and body looked like a dog, yet its striped coat was cat-like. It carried its young in a pouch, like a kangaroo. No wonder the thylacine — the enigmatic, iconic creature of Australia and Tasmania — was the object of so much confusion, alternately called the "marsupial wolf" and the "Tasmanian tiger."
So what was it? By studying bones of thylacines and 31 other mammals, researchers at Brown University have the answer: The thylacine was a Tasmanian tiger — more cat than dog, although clearly a marsupial. In a paper published ...
Penn study shows drop off in coronary artery bypass surgeries for heart patients
2011-05-04
Philadelphia – New research from the University of Pennsylvania School of Medicine shows a substantial decrease in one type of revascularization procedure, coronary artery bypass graft (CABG) surgery, while rates of utilization of the other form, percutaneous coronary intervention (PCI), has remained unchanged. Coronary revascularization, the process of restoring the flow of oxygen and nutrients to the heart, is among the most common hospital-based major interventional procedures performed in the United States. Over the past decade, the field of coronary revascularization ...
Physicians suggest how airlines can better respond to in-flight emergencies
2011-05-04
BOSTON – The concepts now at the center of the health care quality movement, adopted in large part from the airline industry, should be used to standardize the processes and the equipment for in-flight medical emergencies, according to two Beth Israel Deaconess Medical Center physicians.
Writing in an online release for the May 11 Journal of the American Medical Association, Melissa Mattison, MD and Mark Zeidel, MD, note that because the airline industry has adopted root cause analysis of accidents and near misses "most individual flight attendants will never experience ...
Why the eye is better than a camera
2011-05-04
The human eye long ago solved a problem common to both digital and film cameras: how to get good contrast in an image while also capturing faint detail. Nearly 50 years ago, physiologists described the retina's tricks for improving contrast and sharpening edges, but new experiments by neurobiologists at University of California, Berkeley and the University of Nebraska Medical Center in Omaha show how the eye achieves this without sacrificing shadow detail. These details will be published next week in the online, open access journal PLoS Biology.
"Lateral inhibition" (when ...
Nicotine and cocaine leave similar mark on brain after first contact
2011-05-04
The effects of nicotine upon brain regions involved in addiction mirror those of cocaine, according to new neuroscience research.
A single 15-minute exposure to nicotine caused a long-term increase in the excitability of neurons involved in reward, according to a study published in The Journal of Neuroscience. The results suggest that nicotine and cocaine hijack similar mechanisms of memory on first contact to create long-lasting changes in a person's brain.
"Of course, for smoking it's a very long-term behavioral change, but everything starts from the first exposure," ...
World's smallest atomic clock on sale
2011-05-04
ALBUQUERQUE, N.M. — A matchbook-sized atomic clock 100 times smaller than its commercial predecessors has been created by a team of researchers at Symmetricom Inc. Draper Laboratory and Sandia National Laboratories. The portable Chip Scale Atomic Clock (CSAC) — only about 1.5 inches on a side and less than a half-inch in depth — also requires 100 times less power than its predecessors. Instead of 10 watts, it uses only 100 milliwatts.
"It's the difference between lugging around a device powered by a car battery and one powered by two AA batteries," said Sandia lead investigator ...