Category Archives: Biotechnology

NIH to test Ebola vaccine in humans

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From the US National Institutes of Health

Trial will evaluate vaccine’s safety

Initial human testing of an investigational vaccine to prevent Ebola virus disease will begin next week by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.

ebola

The early-stage trial will begin initial human testing of a vaccine co-developed by NIAID and GlaxoSmithKline (GSK) and will evaluate the experimental vaccine’s safety and ability to generate an immune system response in healthy adults.

The pace of human safety testing for experimental Ebola vaccines has been expedited in response to the ongoing Ebola virus outbreak in West Africa.

Testing will take place at the NIH Clinical Center in Bethesda, Maryland.

The study is the first of several Phase 1 clinical trials that will examine the investigational NIAID/GSK Ebola vaccine and an experimental Ebola vaccine developed by the Public Health Agency of Canada and licensed to NewLink Genetics Corp.

The others are to launch in the fall. These trials are conducted in healthy adults who are not infected with Ebola virus to determine if the vaccine is safe and induces an adequate immune response. Continue reading

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Are your medical records vulnerable to theft?

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This KHN story also ran in .

A decade ago almost all doctors kept paper charts on every patient. That is changing quickly as laptops become as common as stethoscopes in exam rooms. Recent hacking attacks have raised questions about how safe that data may be.

Here are some frequently asked questions about this evolution underway in American medicine and the government programs sparking the change.

Are my medical records stored electronically? Continue reading

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Variation in hospital charges for blood tests called ‘irrational’

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RedBloodCellsBy Roni Caryn Rabin
KHN

One California hospital charged $10 for a blood cholesterol test, while another hospital that ran the same test charged $10,169 — over 1,000 times more.

For another common blood test called a basic metabolic panel, the average hospital charge was $371, but prices ranged from a low of $35 to a high of $7,303, more than 200 times more.

The wide disparity in hospitals’ listed charges for routine blood tests at California hospitals was revealed in a study published in the August issue of BMJ Open. The study examined the listed charges for routine blood tests performed in 2011. Continue reading

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Cut the Crapshoot | RAND

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From the RAND Corporation:

U.S. policymakers need to counteract the perverse financial incentives that induce inventors and investors to bet big on new medical technologies that offer the highest payouts without substantially improving health.

Dice pair

Costly new technology is a primary driver of health care spending. This should come as no surprise, because all players in the dicey U.S. market for new medical technologies — inventors, investors, payers, providers, and patients — face long odds if they want to put money on products that could curb health spending while also boosting health.

The rules of the game need to change.

via Cut the Crapshoot | RAND.

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If you have a stroke, better it should be in Paris

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Frank Browning (Photo by Christophe Sevault)

Frank Browning. (Photo by Christophe Sevault)

Frank Browning writes from Paris
KHN

I had a stroke last month, oh boy.

It’s just that I didn’t know it. Here’s what happened:

Only after three days of flashing, floating visual squiggles — commonly known as ocular migraines that usually last 20 minutes — do I email my old friend Dr. John Krakauer, who helps run stroke recovery at Johns Hopkins Hospital in Baltimore.

After a few questions he told me to get an MRI scan as soon as possible.

In the U.S. that could involve the emergency room (with its hours-long wait) or a complicated process of getting the referral — and then finding a radiologist who would take my coverage.

Here in France, it is so much simpler. Continue reading

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Cord Blood: What You Need to Know – FDA

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Consumer Update from the US Food and Drug Administration

RedBloodCellsCord blood is found in the blood vessels of the placenta and the umbilical cord, cord blood is collected after a baby is born and after the umbilical cord is cut—an important point.

“Because cord blood is typically collected after the baby is delivered and the cord is cut, the procedure is generally safe for the mother and baby,” explains Keith Wonnacott, Ph.D., Chief of the Cellular Therapies Branch in FDA’s Office of Cellular, Tissue, and Gene Therapies. Continue reading

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‘Telepsychiatry’ helps bring mental health care to rural areas

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Ed Spencer, director of South Carolina’s telepsychiatry program, (seated) and Ralph Strickland, program coordinator, (on screen) conduct a simulation of a typical emergency room telepsychiatry consultation at their offices in Columbia.

By Christine Vestal
Stateline

When emergency room patients are deemed “a danger to themselves or others,” every state requires hospitals to hold them until a psychiatrist conducts a face-to-face evaluation to decide whether it is safe to let them leave. In rural hospitals across the country, it can take days for a psychiatrist to show up and perform the exam.

Five years ago, rural hospitals in South Carolina illustrated the problem. On a typical morning, more than 60 people were waiting in the state’s emergency rooms for psychiatric exams so they could either be discharged or admitted for treatment.

Today the scene is quite different, thanks to a “telepsychiatry” program that allows psychiatrists to examine South Carolina patients through videoconferencing, reducing the average wait time from four days to less than 10 hours.

In 2010, North Carolina began rolling out a similar program, and a dozen other states, including Alabama, Kentucky and Wisconsin, plan to follow suit. Continue reading

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Bionic pancreas outperforms insulin pump in adults, youth – NIH

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Diabetes pump

From right, researcher Dr. Steven Russell of Massachusetts General Hospital stands with Frank Spesia and Colby Clarizia, two participants in a type 1 diabetes trial testing an electronic device called a bionic pancreas – the cellphone-sized device shown – which replaces their traditional fingerstick tests and manual insulin pumps. Photo courtesy of Adam Brown,

From the National Institutes of Health

People with type 1 diabetes who used a bionic pancreas instead of manually monitoring glucose using fingerstick tests and delivering insulin using a pump were more likely to have blood glucose levels consistently within the normal range, with fewer dangerous lows or highs.

The report was published online by the New England Journal of Medicine

TThe researchers — at Boston University and Massachusetts General Hospital — say the process of blood glucose control could improve dramatically with the bionic pancreas. Currently, people with type 1 diabetes walk an endless tightrope.

Because their pancreas doesn’t make the hormone insulin, their blood glucose levels can veer dangerously high and low.

Several times a day they must use fingerstick tests to monitor their blood glucose levels and manually take insulin by injection or from a pump.

In two scenarios, the researchers tested a bihormonal bionic pancreas, which uses a removable tiny sensor located in a thin needle inserted under the skin that automatically monitors real time glucose levels in tissue fluid and provides insulin and its counteracting hormone, glucagon, via two automatic pumps. Continue reading

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When doctors need advice, it might not come from a human

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Computer Circuit Board

This story was produced in collaboration with 

Long Island dermatologist Kavita Mariwalla knows well how to treat acne, burns and rashes. But when a patient came in with a potentially disfiguring case of bullous pemphigoid—a rare skin condition that causes large, watery blisters—she was stumped.

The medication doctors usually prescribe for the autoimmune disorder wasn’t available. So she logged in to Modernizing Medicine, a Web-based repository of medical information and insights, for help.

Within seconds, she had the name of another drug that had worked in comparable cases.

“It gives you access to data, and data is king,” she said of Modernizing Medicine. “It’s been very helpful especially in clinically challenging situations.” Continue reading

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There’s a life-saving hepatitis C drug. But you may not be able to afford it.

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Sovaldi logoBy Julie Appleby
KHN Staff Writer

MAR 03, 2014

This KHN story was produced in collaboration with 

There’s a new drug regimen being touted as a potential cure for a dangerous liver virus that causes hepatitis C.  But it costs $84,000 – or $1,000 a pill.

And that price tag is prompting outrage from some consumers and a scramble by insurers to figure out which patients should get the drug —and who pays for it.

Called Sovaldi, the drug is made by California-based Gilead Sciences Inc. and is the latest in handful of new treatments for hepatitis C, a chronic infection that afflicts at least 3 million Americans and is a leading cause of liver failure. It was approved by the U.S. Food & Drug Administration in December. Continue reading

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Science on Stage: Staged readings of “The Sequence” by Seattle playwright Paul Mullin

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dna

Northwest Association for Biomedical Research and Seattle Public Theater will present ‘Science on Stage’ – staged readings with the director and cast of Seattle playwright Paul Mullin’s play ‘The Sequence.’

Bathhouse Theater at Green Lake with pre- or post-play discussions

Oct. 5 -   Saturday,   2pm          post-play discussion for NWABR members    - 3:45pm

Oct. 12 – Saturday,   2pm           post-play discussion for the public                   – 3:45pm

Oct. 13 - Sunday,     7pm           pre-play discussion for NWABR donors           – 6:00pm

Tickets:  $20 for NWABR members, $22 for all others, available online or at the door.

Play Synopsis:  Sequencing of the human genome was a public race – and a personal war!  Strong personalities, emerging technology and differing views on public vs. private DNA ownership fueled the race to obtain the first human DNA sequence.  Covering the story was a young journalist worried about her own destiny with breast cancer and the information that her genome sequence would uncover.

More information and online ticket information are available at http://nwabr.org/science-on-stage or contact Reitha Weeks at rweeks@nwabr.org for information about group sales.

Proceeds support NWABR programs.

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FDA moves to reduce use of long-acting opioid pain drugs

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The US Food and Drug Administration has changed the labeling on long-acting opioids, such as OxyContin, in an effort to limit the use of these drugs to patients with severe refractory pain. Here’s is the Consumer Update from the FDA released today.

FDA Consumer Update

FDA logojpgConsumers and health care professionals will soon find updated labeling for extended-release and long-acting opioid pain relievers to help ensure their safe and appropriate use.

In addition to requiring new labeling on these prescription medications, the Food and Drug Administration (FDA) is also requiring manufacturers to study certain known serious risks when these drugs are used long-term.

“The new labeling requirements and other actions are intended to help prescribers and patients make better decisions about who benefits from the use of these medications. They also are meant to reduce problems associated with their use,” says Douglas Throckmorton, M.D., deputy director of regulatory programs in FDA’s Center for Drug Evaluation and Research.

“Altogether, the actions we’re now announcing are part of FDA’s efforts to make opioids as safe as possible for those who need them,” Throckmorton adds.

He noted that the actions come after careful analysis of new safety information, including reviews of medical literature, and consideration of input from patients, experts and many other interested parties.

How Labeling Will Change

Opioids work by changing the way the brain perceives pain. They are available by prescription as pills, liquids, and skin patches.

Extended-release and long-acting (ER/LA) forms pose a greater safety concern because—as their names suggest—they produce their effects for a longer period, and many contain higher doses compared with immediate release or opioid/non-opioid combination products.

They include, to name a few, long acting versions of opioids such as morphine, oxycodone, and fentanyl.

Currently, labeling on these ER/LA opioids indicate they are for “the relief of moderate to severe pain in patients requiring continuous, around-the-clock opioid treatment for an extended period of time.”

However, the updated indication for when to prescribe and take these medicines will, when finalized, emphasize that other, less potentially addictive, treatment options should be considered first.

FDA is requiring labeling that says the drugs are “indicated for the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate.”

The “limitations of use” portion of the new labeling retains language indicating that the drugs are not intended for use as an “as-needed” pain reliever.

Furthermore, the new labeling adds: “Because of the risks of addiction, abuse and misuse with opioids, even at recommended doses, and because of the greater risks of overdose and death with extended-release opioid formulations, reserve [Tradename] for use in patients for whom alternative treatment options (e.g., non-opioid analgesics or immediate-release opioids) are ineffective, not tolerated, or would be otherwise inadequate to provide sufficient management of pain.”

This new labeling language emphasizes that patients in pain should be assessed not only by their rating on a pain intensity scale, but also based on a more thoughtful determination that their pain—however it may be defined—is severe enough to require daily, around-the-clock, long-term opioid treatment, and for which alternative treatment options are inadequate.

This framework better enables prescribers to make decisions based on a patient’s individual needs, given the serious risks associated with ER/LA opioids, against a backdrop of alternatives such as immediate release (IR) opioids and non-opioid pain relievers.

It allows prescribers to make an assessment of pain relative to a patient’s ability to perform daily activities or enjoy a reasonable quality of life.

FDA-approved labeling of these pain relievers already describes the effects on newborns of exposure to these drugs while in the mother’s womb and warns against use by women during pregnancy and labor and while nursing.

The new labeling, however, will provide more detail and will elevate the risk of neonatal opioid withdrawal syndrome (NOWS) to the most prominent position in labeling—a boxed warning. Symptoms of NOWS may include poor feeding, rapid breathing, trembling, and excessive or high-pitched crying.

Postmarket Studies

Recognizing the need for more scientific data about the benefits and risks of ER/LA opioids when used over long periods, FDA also decided to require drug companies to conduct longer term studies and trials of ER/LA opioid pain relievers on the market.

The companies must evaluate long-term use, with the goal of assessing a variety of known serious risks, including misuse, abuse, addiction, overdose, and death, as well as the risks of developing increasing sensitivity to pain.

Education to Reduce Risk

Following implementation of the safety labeling changes, certain educational materials for patients and health care professionals will be modified to reflect the new labeling for the ER/LA opioid pain relievers.

As part of the new labeling changes, opioid manufacturers also must revise a paper handout patients receive with their prescription.

The ER/LA Opioid Analgesics Risk Evaluation and Mitigation Strategy (REMS) will also be updated after the labeling changes are finalized.

The ER/LA Opioid Analgesics REMS requires manufacturers to make available continuing education courses for health care professionals who prescribe these drugs.

The courses, from accredited sources, teach about risks and safe prescribing and safe use practices of these medications.

“By improving information about the risks of ER/LA opioid pain relievers and by clarifying the populations for whom the benefits outweigh the risks, we aim to improve the safe and appropriate use of these products,” says Throckmorton.

He adds: “This is not the first or last initiative, and we will continue supporting broader efforts to solve the serious public health problems associated with the misuse and abuse of opioids.”

This article appears on FDA’s Consumer Updates page, which features the latest on all FDA-regulated products.

Sept 10, 2013

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Drug companies battle against generic biologics state-by-state.

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By Michael Ollove
Stateline Staff Writer

The year 2013 began with the promise of a state-by-state, coast-to-coast battle between the makers of brand-name medications derived from living organisms—known as biologics—and those who to make and sell copies of those drugs.

The battle has turned into a rout, at least for now. In state after state, the brand-name makers—led by the pharmaceutical giants Amgen and Genentech—have been unable to convince state legislatures to require pharmacists to notify doctors (and sometimes patients) when they substitute generics for brand-name biologic drugs.

Manufacturers of copies fiercely opposed such a requirement, which they said would put them at a competitive disadvantage.

Notification measures died in Arizona, Arkansas, Colorado, Indiana, Illinois, Maryland, Mississippi, Nevada, Texas and Washington.

pills-spill-out-of-bottle

Oregon, Utah and Virginia all adopted notification requirements, but with sunsetting provisions that will take effect before any of the knock-offs reach the market. Only one state, North Dakota, passed the measure the brand-name manufacturers sought.

“State legislators accepted that there was absolutely no need to push this type of legislation” said Brynna Clark, director of state affairs for the Generic Pharmaceutical Association, which took the lead in organizing the effort against notification requirements. “Nobody’s hair was on fire.”

Measures are still alive in California, where the prospects for passage look bright, and in Pennsylvania and Massachusetts.

Two Years Away

Most believe it will be at least two years before any copies of biologics receive approval from the Food and Drug Administration (FDA). But the skirmishes in 18 states this year give a sense of the high stakes involved.

Biologics already account for roughly a quarter of the $320 billion spent annually on medications in the U.S., according to IMS Health, and biologic medicines are expected to command ever larger portions of the prescription drug marketplace.

The Congressional Budget Office has estimated that substituting biologic doppelgangers for brand names would reduce federal Medicaid and Medicare spending by $25 billion over 10 years.

The CBO estimates that total federal spending on prescription drugs during the same period will total $500 billion.

The Affordable Care Act calls for an abbreviated approval process for the biologic copies, as a means to promote competition and reduce costs.

While the federal government will determine whether particular biosimilars are interchangeable with the originals, it will be up to the states to determine the exact policies regarding substitutions.

Companies that make the copies say their products should be treated no differently than generic chemical drugs. By state law, pharmacists can (and in some cases, must) substitute cheaper generics for brand-name medications once the original patent has expired, unless otherwise directed by the doctor or, in some states, the patient.

Complex Substances

But, there are big differences between copying chemical drugs and copying biologic drugs.

Until the 1970s, virtually all manufactured medicines were derived from chemicals. They are sometimes called “small molecule” drugs because they are composed of relatively few atoms and have stable, well-defined chemical structures, making it relatively easy to produce exact duplicates.

Biologics, which are used to treat patients with cancer, blood irregularities, psoriasis, and auto-immune and neurological disorders, are the result of complicated biological manufacturing and testing processes. They have as many as 20,000 atoms, and they interact with the body in different ways from chemically-derived medicines.

As a result, it is virtually impossible to make exact copies, but only something very similar. Hence, the generic versions are often referred to as “biosimilars,” “bioequivalences” “biocomparables,” “me-too biologics” or “biobetters.”

The expense and complexity of manufacturing biosimilars also means they won’t be as deeply discounted as generic chemical drugs, which generally cost 50 percent to 90 percent less than the originals. Biologic copies are expected to cost between 10 percent and 20 percent less than the originals.

Although biosimilars are already on the market in Europe, none have yet been approved for the U.S. market (U.S. original patent periods are longer). The FDA has, however, issued draft rules for the approval process. Most expect the first biosimilars to gain FDA approval in 2015.

Notification of Substitutes

But battle lines have already been drawn. Amgen, which makes both biologics and biosimilars, and Genentech have led the efforts to promote passage of state notification laws. Together the two companies contributed about $650,000 to state legislative races in 2012.

Gino Grampp, director of research and development policy for Amgen, said that because biosimilars are not identical to the originals, it is important that physicians be alerted when there is a substitution.

Otherwise, if a patient has an adverse reaction to a biosimilar, the doctor may not be able to identify the cause of the problem.

“We aren’t saying the substitution shouldn’t be made,” Grampp said. “We’re saying the doctor should have a record of that substitution.”

State Sen. Jerry Hill, the Democrat who sponsored the notification bill in California, was persuaded by that argument. “In California, there’s real sensitivity to the patient’s right to know and patients controlling their own bodies,” Hill said.

But the generic association argues the proposed legislation is a ruse by the brand-name manufacturers to cause anxiety about biosimilars.

“It’s going to raise trust issues and concerns about these medicines,” said Clark of the Generic Pharmaceutical Association. “Why does my doctor have to be specially notified if these drugs are really OK?”

Clark argues that if the FDA approves a biosimilar as interchangeable with the original product, there is no need to create obstacles at the state level. Biosimilars, she argues, should be treated no differently from generic chemical drugs.

Level of Acceptance

Few doctors object to the substitution of generics for chemical medications, but “there’s a real question whether there will be the same level of acceptance among doctors of biosimilars as there are with generics,” said Jack Hoadley, a research  professor at the Health Policy Institute at Georgetown University.

Any signal, such as a notification requirement, that suggests biosimilars are not the equal of the originals could diminish confidence in them among doctors and patients.

Meanwhile, the Generic Pharmaceutical Association has found a potent ally in pharmacists, who fear the notification requirement would be a burden. Their opposition was instrumental in the defeat of Maryland state Sen. Joan Carter Conway’s notification bill this year.

“They said it was just too much work,” said Conway, a Democrat.

Stateline logo

Stateline is a nonpartisan, nonprofit news service of the Pew Center on the States that provides daily reporting and analysis on trends in state policy.

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FDA warns of rare acetaminophen risk

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A Consumer Update from the US Food and Drug Administration

ucm363013Acetaminophen, a fever and pain reliever that is one of the most widely used medicines in the U.S., can cause rare but serious skin reactions, warns the Food and Drug Administration (FDA).

Although rare, possible reactions to acetaminophen include three serious skin diseases whose symptoms can include rash, blisters and, in the worst case, widespread damage to the surface of skin.

If you are taking acetaminophen and develop a rash or other skin reaction, stop taking the product immediately and seek medical attention right away.

Used for decades by millions of people, acetaminophen is the generic name of a common active ingredient included in numerous prescription and non-prescription medicines.

Tylenol is one brand name of the pain reliever sold over the counter, but acetaminophen is also available as a generic under various names.

It is also used in combination with other medicines, including opioids for pain and medicines to treat colds, coughs, allergy, headaches and trouble sleeping.

“This new information is not intended to worry consumers or health care professionals, nor is it meant to encourage them to choose other medications,” says Sharon Hertz, M.D., deputy director of FDA’s Division of Anesthesia, Analgesia and Addiction. “However, it is extremely important that people recognize and react quickly to the initial symptoms of these rare but serious, side effects, which are potentially fatal.”

Other drugs used to treat fever and pain, such as nonsteroidal anti-inflammatory drugs including ibuprofen and naproxen, already carry warnings about the risk of serious skin reactions.

Advil and Motrin are among the common brand names that include ibuprofen as an active ingredient. Aleve and Midol Extended Relief are among the best-known brand names that include naproxen as an active ingredient.

FDA is requiring that a warning about these skin reactions be added to the labels of all prescription medicines containing acetaminophen.

FDA will work with manufacturers to get the warnings added to the labels of over-the-counter (OTC) medicines containing acetaminophen.

On OTC medicines, the word “acetaminophen” appears on the front of the package and on the Drug Facts label’s “active ingredients” section.

On prescription medications, the label may spell out the ingredient or use a shortened version such as “APAP,” “acet,” “acetamin” or “acetaminoph.”

Ingredient Linked to Several Conditions

Stevens-Johnson Syndrome (SJS) and toxic epidermal necrolysis (TEN) are the two most serious skin reactions linked in rare cases to acetaminophen. They usually require hospitalization and can cause death.

Problems usually begin with flu-like symptoms followed by rash, blistering and extensive damage to the surfaces of the skin. Recovery can take weeks or months, and possible complications include scarring, changes in skin pigmentation, blindness and damage to internal organs.

A third skin reaction, acute generalized exanthematous pustulosis (AGEP), usually resolves within two weeks of stopping the medication that caused the problem.

A serious skin reaction can occur at any time, even if you’ve taken acetaminophen previously without a problem. There is currently no way of predicting who might be at higher risk.

If you’ve ever had a skin reaction when taking acetaminophen, don’t take the drug again and discuss alternate pain relievers/fever reducers with your health care professional.

Evidence of Link

Prior to deciding to add a warning about skin reactions to products containing acetaminophen, FDA reviewed medical literature and its own database, the FDA Adverse Event Reporting System (FAERS).

A search of FAERS uncovered 107 cases from 1969 to 2012, resulting in 67 hospitalizations and 12 deaths. Most cases involved single-ingredient acetaminophen products; the cases were categorized as either probable or possible cases associated with acetaminophen.

A small number of cases, just over two dozen, are documented in medical literature, with cases involving people of various ages.

FDA has examined—and continues to examine—acetaminophen for safety issues, just as it does with all approved drugs. The warning comes two years after FDA took new steps to reduce the risk of liver injury from acetaminophen.

In that case, FDA asked all makers of prescription products to limit acetaminophen to 325 milligrams per tablet or capsule. FDA also required all prescription acetaminophen products to include a Boxed Warning—FDA’s strongest warning, used for calling attention to serious risks.

The agency continues to consider the benefits of this medication to outweigh the risks.

“FDA’s actions should be viewed within the context of the millions who, over generations, have benefited from acetaminophen,” says Hertz. “Nonetheless, given the severity of the risk, it is important for patients and health care providers to be aware of it.”

This article appears on FDA’s Consumer Updates page, which features the latest on all FDA-regulated products.

August 1, 2013

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