2 new studies show the FDA is rushing more drugs to market based on shoddy evidence – Updated by Julia Belluz on September 24, 2015, 5:39 p.m. ET

There was a time when the Food and Drug Administration was so sluggish and conservative in approving new drugs that people who desperately needed access to medicines would die waiting.

But by the early 1990s, Congress had created four programs to expedite the development and approval process for new pharmaceuticals (the breakthrough drug designation, priority review, fast track and accelerated approval). These pathways were intended to push innovative new drugs — drugs to treat rare, serious, or life-threatening diseases — through the FDA more quickly.

Since these medicines were sorely needed, the idea was that rushing them through, often on the basis of more limited and preliminary clinical trials data, would help patients languishing with unmet medical needs.

fda jb

(Javier Zarracina)


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An FDA panel just backed the ‘female Viagra.’ Critics say it’s a mistake. – Updated by Julia Belluz on June 4, 2015, 7:21 p.m. ET

1) A panel of advisers to the Food and Drug Administration just recommended the agency approve a new drug designed to boost womens’ sex drives.

2) Flibanserin — a pill dubbed the ‘female Viagra’ — has already been rejected by the FDA twice since 2010 out of concerns that its harms outweigh its benefits.

3) The 18-6 vote is considered a “major victory” for the drug, since the FDA more often than not adopts the advice of the expert group and same panel voted unanimously against flibanserin in the past.

4) Critics have raised questions about the efficacy of flibanserin. They’ve also accused the pharmaceutical industry of inventing the medical condition— hypoactive sexual desire disorder — that the drug would supposedly treat.

American women may be one step closer to the ‘female Viagra’

female viagra

(El Nariz /Shutterstock

Flibanserin (pronounced fluh-BAN-ser-in) is a daily pill made by Sprout Pharmaceuticals designed to treat low libido in premenopausal women. To date, the FDA has not approved it for sale — but the pressure to do so is growing.

On Thursday, a government advisory panel voted 18 to 6 to recommend that the FDA approve the drug — on the condition that Sprout can figure out how to minimize the safety risks to patients. That might entail requiring medical professionals to take a training course before being allowed to prescribe the drug, or making women who want the pink pills sign an agreement saying they won’t use birth control or alcohol while on flibanserin (since these two behaviors increased the risk of side effects).

About one in five women who took the drug have experienced side effects: most commonly, low blood pressure, dizziness, and fainting, increasing the risk of injuries such as concussions.

While Viagra improves men’s sexual performance, by increasing blood flow, the pink pill works on women’s brains, raising dopamine levels.

It’s not clear that the FDA will take the panel’s advice, particularly since the agency has questioned the drug’s effectiveness and safety in the past. As the New York Times reports, the results of clinical trials for flibanserin have been lackluster:

Three clinical trials testing flibanserin were consistent in their results. The women who took part were having an average of two to three of what they defined as “sexually satisfying events” per month when the studies began. Once they started taking the drug, the number of such events increased, but by no more than one event per month more than for women in the trial who got a placebo.

At the advisory committee meeting, the FDA gave a scientific presentation against the drug, arguing that it only resulted in eight more sexually satisfying events per year (when compared to placebo) and that side effects were common.

But the room was also filled with emotional testimony by women who feel they would greatly benefit from the medication. Sprout claims nearly one-tenth of premenopausal women suffer hypoactive sexual desire disorder.

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Biotechnology to the Rescue – By Arlene Weintraub Sept. 2, 2014 | 7:00 a.m. EDT


Wake Forest University’s regenerative medicine lab uses 3-D printing to produce a prototype of a kidney.

The life sciences industry is in the middle of a historic boom, churning out new medical weapons at an unprecedented pace. In 2012, the Food and Drug Administration approved 39 “new molecular entities,” completely novel drugs to treat everything from cancer to tuberculosis to HIV. It topped that off by adding 27 more last year, and there are now more than 4,000 investigational medicines in the pipeline.

Medical device-makers are also in high gear; the 33 products approved in 2013 include replacement hips, new cardiac stents and prosthetic spinal discs. Here’s a sampling of the most innovative developments transforming medical practice and offering patients new hope:

Brain boosters

When the complex communication circuits go awry, the result is a range of neurological disorders, including epilepsy, Parkinson’s disease and paralysis. Now researchers are figuring out how to manipulate neurological signals using tiny implants that, in essence, reset the brain. By targeting very precisely the region where seizures and tremors originate, for example, these minicomputers – sometimes in conjunction with drugs, sometimes on their own – can eliminate debilitating symptoms at their source.

Among the people already benefiting is Janie Norman, 43, of Marietta, Georgia, who was diagnosed with epilepsy at age 11. Norman was able to attend college, get married, and start a family, but she had such frequent seizures that she couldn’t get a driver’s license, go to the movies or grocery store on her own, or play a sport – all were too risky.

Then neurosurgeons at Emory University Hospital in Atlanta placed a tiny implant in her brain that detects the abnormal brain signals that usually precede seizures and responds by delivering short electrical pulses to stop the seizures before they start. Norman has been seizure-free ever since. “It’s a miracle,” she says. “I got my driver’s license. I can take my children to the park. It has really changed my life.”

The NeuroPace RNS System, as the device is called, was approved last year and has been adopted by more than 20 clinics specializing in epilepsy. It also features tiny programmable processors that store and transmit information about what exactly is happening in patients’ brains. By waving a wand over her head, Norman can download the information and transmit it to her doctor. The physicians can then use the information to adjust the device remotely.

An explosion of research that aims to “map” the brain’s function has doctors who treat neurologic disorders predicting a bright future. “The hope is that if we can restore some normal activity, then the natural ability of the brain to use those circuits will keep diseases from getting worse,” says Michael Kilgard, a professor at the University of Texas—Dallas whose research aims to normalize brain activity by sending electrical signals to it via electrodes attached to the vagus nerve in the neck.

Other scientists are developing implants that may someday restore movement in paralyzed patients by repairing damaged connections that instruct the muscles. The brain is great at “reorganizing itself,” Kilgard says. “We’re just guiding it in the right direction.”

Bionic body parts

Whether a patient is an amputee who needs a prosthetic limb or an aging person in need of a new knee or hip joint, the fundamental wish is the same: that the new part will work just as well as the old one did. The device industry is answering the call with myriad new technologies.

In May, the FDA approved the first prosthetic arm that will use special sensors and electrodes to pick up signals transmitted from nearby muscles and translate them to control multiple joints. That will allow users to perform complex tasks, such as grasping small items.

The DEKA Arm System, which goes by the Star Wars-inspired nickname “Luke,” also has sensors in its fingers that can detect how tightly the hand is grasping an object. It communicates that information to the patient through a device touching the skin that vibrates lightly in response to a delicate grip and more intensely as the grasp grows tighter. So a person can shake hands and sense how tight his or her grip is, says Justin Sanchez, program manager in the biological technologies office of the Defense Advanced Research Projects Agency, which funded the arm system’s development.

Retired Chicago police detective John Duffy, who was injured in a cycling accident, wears a prosthetic that flexes in response to signals from his environment.

Retired Chicago police detective John Duffy, who was injured in a cycling accident, wears a prosthetic that flexes in response to signals from his environment.

Prosthetic legs are becoming more lifelike, too. Retired Chicago police detective John Duffy, who lost his leg below the knee in a cycling accident, wears one of the new models that rely on hydraulic cylinders to flex the joints based on signals from a system of sensors and a microprocessor in the leg. As he walks, the sensors continually monitor environmental feedback and send signals to the microprocessor, which controls how much resistance the cylinders apply. The resistance varies according to how fast Duffy walks or whether he is on a flat surface, going up or down stairs, or heading uphill or downhill.

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WHO calls for global crackdown on e-cigs – By Benjamin Goad – 08/26/14 01:55 PM EDT

The World Health Organization (WHO) called Tuesday for tougher regulations around the globe on a burgeoning electronic cigarette industry “filled with promise and threat for tobacco control.”

Screen Shot 2014-08-27 at Aug 27, 2014 12.42
A new WHO report largely mirrors positions taken by public health advocates in the United States and adds to mounting pressure on the Food and Drug Administration (FDA), which is now weighing a final rule to bring e-cigarettes under its supervision.

WHO member states will debate the report during October meetings in Moscow. Among its findings is a need for regulations to stop the marketing of e-cigarettes to children, minimize health risks and block the industry from making unsubstantiated health claims.
Because e-cigarettes produce nicotine vapor instead of smoke and tar, they are sometimes viewed as a healthier alternative to conventional cigarettes and as a potential cessation tool.

However, WHO determined that there is insufficient evidence to conclude e-cigarettes help users quit smoking. Thus, the report’s authors found that the products present an “evolving frontier filled with promise and threat for tobacco control.”

In particular, the group urged member states to “restrict e-cigarette advertising, promotion and sponsorship, to ensure that it does not target youth and non-smokers or people who do not currently use nicotine.”

WHO also calls for legal steps to outlaw the use of e-cigarettes indoors or at workplaces, given research that shows exhaled nicotine vapors push toxic substances into the air.

The report comes a day after the American Heart Association unveiled a similar set of recommendations. 

The FDA issued a proposed rule to assert authority over the largely unregulated industry, which is worth an estimated $3 billion worldwide. The agency has completed a public comment period that attracted more than 70,000 comments and is now preparing a final rule.

Read more: http://thehill.com/regulation/215996-who-calls-for-global-crackdown-on-e-cigs#ixzz3BZmXZqBW  Follow us: @thehill on Twitter | TheHill on Facebook

“All-natural” labels on food are meaningless. Let’s get rid of them. – Updated by Brad Plumer on June 23, 2014, 9:30 a.m. ET

Walk down the aisle of a grocery store and you’re bound to see all sorts of bizarre foods labeled as “natural” or “all-natural.” There are “natural” Cheetos and “natural” cookies. There are “all-natural” fruit drinks that contain high-fructose corn syrup.

The ‘natural’ label is meaningless — but many shoppers take it seriously

The “natural” label is basically meaningless — there are very few rules for how it’s used and companies will slap it on all sorts of things.

And yet a lot of shoppers seem to take the label seriously, assuming it means the food is somehow better for you or healthier. Over at Grist, Nathanael Johnson points to a new Consumer Reports survey finding that 59 percent of those polled check for a “natural” label when shopping for food. As he laments, “When will the vague ‘natural’ food label die?”

It’s worth expanding on Johnson’s point. There aren’t really any clear definitions for what counts as “natural” food, which is one reason why you see it pop up in so many odd places. But that’s partly because the word itself is inapt — very little about modern agriculture is “natural,” and it’s just not a good way of assessing the health or sustainability of our food system.

There’s no clear definition for “natural” food


But is it natural? BSIP/UIG/Getty Images

The Food and Drug Administration has no official definition of “natural food” — in part, they say, because a great many foods in the grocery store have usually been processed or altered in some way and so it’s difficult to draw a clear line.

The FDA tried to find a precise definition of ‘natural’ in the early 1990s and gave up

Back in 1991, the FDA actually tried to come up with a more precise standard. But after two years of trying, the agency gave up. “It’s too complex,” one FDA official lamented in 2008.

(By the way, this is in contrast to the term “organic,” a term that is more precisely defined and regulated by the US Department of Agriculture.)

By and large, the FDA doesn’t regulate most uses of “natural” labels, though it will occasionally send warning letters — if, say, a product is labeled “all-natural” but contains citric acid or calcium chloride or potassium sorbate. (Though, as one investigation by the Center for Science in the Public Interest found, those warning letters often go ignored.)

Things are a little different with fresh meat, which is regulated by the Department of Agriculture. There, “natural” is defined as meaning the meat contains no artificial ingredients and is minimally processed. But even here, some artificial additives are allowed (such as chicken flavored with a salt broth). And meat from animals raised on antibiotics or hormones can still be called “natural.”

Consumer groups have often complained that the lack of a clear definition means that lots of odd things get misleadingly labeled “all-natural” or “100% natural” even when they include chemical ingredients. The Center for Science in the Public Interest has a long list of oddities over the years, including a Hunt’s “100% Natural” tomato sauce that contained citric acid or a Minute Maid “All-Natural” cranberry cocktail that contained high-fructose corn syrup.

That group called for stricter definitions and regulations on the practice, arguing that the label misleads consumers. But even that’s not as easy as it sounds. A more precise definition would still be fairly misleading — in part because the word “natural” isn’t a very helpful way to think about food.

Our food system isn’t “natural” to begin with

Underlying this broader issue is the widespread belief that “anything natural is good, and anything unnatural is bad,” as Cambridge geneticist Ottoline Leyser put it.

‘The cereal crops we eat bear little resemblance to their naturally selected ancestors’

In a recent essay in PLOS Biology, Leyser argues that it’s time to kill this mistaken idea once and for all. Basically everything in modern agriculture is unnatural. “The cereal crops we eat bear little resemblance to their naturally selected ancestors, and the environments in which we grow them are equally highly manipulated and engineered by us,” she writes. “We have, over the last 10,000 years, bred out of our main food plants all kinds of survival strategies that natural selection put in. ”

How is GMO food different from regular food?

It might help to distinguish genetic engineering from traditional techniques for producing food.

Humans have been selectively breeding plants and animals for tens of thousands of years to get certain desired traits. Over time, for example, farmers (and scientists) have bred corn to become larger, to hold more kernels on an ear, and to flourish in different climates. That process has certainly altered corn’s genes. But it’s not usually considered “genetic engineering.”

Genetic engineering, by contrast, involves the direct manipulation of DNA, and only really became possible in the 1970s. It often takes two different forms: There’s “cisgenesis,” which involves directly swapping genes between two organisms that could otherwise breed — say, from wheat to wheat. Or there’s “transgenesis,” which involves taking well-characterized genes from a different species (say, bacteria) and transplanting them into a crop (say, corn) to produce certain desired traits.

Ultimately, genetic engineering tries to accomplish the same goals as traditional breeding — create plants and animals with desired characteristics. But genetic engineering allows even more fine-tuning. It can be faster than traditional breeding and it allows engineers to transfer specific genes from one species to another. In theory, that allows for a much greater array of traits.

Here’s a diagram from the Food and Drug Administration:





How Much Does It Hurt? – By Stephen S. Hall Published Jun 8, 2014

Zohydro is the new FDA-approved painkiller that some doctors think the FDA had no business approving. And in ERs across America, they’re anxiously awaiting the fallout.

(Photo: Bobby Doherty/New York Magazine)

Even before a single doctor in the United States had written a prescription for Zohydro, the controversial long-acting painkiller approved by the Food and Drug Administration last October, potential users were already dreaming up possible street names. “How many times will this be said in the future,” someone posted on Opiophile, an online forum for people who like to share their drug experiences and expertise. “Got any of dem Zoh’s?” There were other possibilities: Zs, Zodros, and Zorros.

Another voiced chimed in: “I like Zorros … Yeah, has a ring to it.” This was on October 26, 2013, less than 24 hours after the FDA announced its decision.

And in April, even before Nima Majlesi, an emergency-room physician at Staten Island University Hospital, had seen a single report of an overdose or death related to Zohydro in the borough, he and his fellow doctor Amit Gupta were searching websites for the first anecdotal, and decidedly unofficial, accounts of its recreational use. Majlesi logged on to another well-known drug-use site to see if anyone had posted an opening-night review.

“How do you spell it again?” he asked Gupta, with whom he shares an unadorned office a few feet from the emergency room.

“Z-O-H-Y-D-R-O,” Gupta said, and Majlesi repeated the letters aloud as he typed them into a site called Erowid.

If the prescription-painkiller epidemic in America is a heartland phenomenon, as is often said, the heartland begins just beyond the toll plaza of the Verrazano-Narrows Bridge. As New York City Department of Health epidemiologists have helplessly documented over the past ten years, opioid abuse has skyrocketed on Staten Island, with three times the rate of overdose deaths of the rest of the city. In a borough where police have busted illegal prescription-drug-selling operations in everything from a neighborhood deli to an ice-cream truck, Majlesi and Gupta have seen and heard it all: the urgent calls of EMS teams phoning in reports of suspected overdoses as ambulance crews race to the hospital, lethargic or comatose patients (often young adults in their 20s and 30s) wheeled into the ER. Sometimes the overdose is accidental. Often there’s nothing to do because first responders didn’t arrive in time.

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New gene tests may give cancer patients quicker path to treatment – BY JULIE STEENHUYSEN CHICAGO June 6 , 2014 1:45am EDT

A view shows the U.S. Food and Drug Administration (FDA) logo at the lobby of its headquarters in Silver Spring, Maryland August 14, 2012.   REUTERS/Jason Reed

A view shows the U.S. Food and Drug Administration (FDA) logo at the lobby of its headquarters in Silver Spring, Maryland August 14, 2012.


A view shows the U.S. Food and Drug Administration (FDA) logo at the lobby of its headquarters in Silver Spring, Maryland August 14, 2012.

Credit: Reuters/Jason Reed

(Reuters) – A new way of evaluating tumors may soon help cancer patients identify the underlying genetic link to their disease – and the best possible treatment – all in a single test.

Researchers are set to begin clinical trials using a more comprehensive testing method that looks for all of the known genes that may be active in a tumor.

The new method could guide patients to the right drug earlier, potentially replacing current tests known as companion diagnostics that only look for a specific biological trait or “biomarker.” The presence of a biomarker can predict whether a new class of drugs called targeted therapies will work on particular tumors.

Results of these broader tests could even be used to quickly identify which patients might benefit from experimental drugs being tested in clinical trials. U.S. health officials see it as the future direction of cancer diagnostics.

“We really are moving away from this one drug, one biomarker, one companion diagnostic,” said Dr Richard Pazdur, the U.S. Food and Drug Administration’s oncology chief.

In advanced melanoma, for example, about half of patients’ tumors have a mutation in the BRAF gene. Roche makes a drug called Zelboraf that blocks that pathway, at least for a time. To get Roche’s drug, patients need to be evaluated with an FDA-approved companion diagnostic test. One of the tests is also made by Roche.

In many cases, the FDA requires single-biomarker companion diagnostics as part of the drug approval process, but the broader testing model opens the door to additional players in the diagnostics space, including U.S.-based Foundation Medicine Inc and Thermo Fisher’s Life Technologies.

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