Tagged Pharmaceuticals

On The Backs Of Devastating Failures, Biotech Pins Hopes On New Alzheimer’s Drug

It’s the disease that has befuddled drugmakers for decades, and a recent string of high-profile setbacks have rocked the industry. But one company is pushing forward in hopes of breaking into a market worth billions.

Stat: Biotech’s Next Alzheimer’s Test May Answer ‘$25 Billion Question’
Moving on from biopharma’s latest setback in Alzheimer’s disease — and the four that preceded it last year — the industry is turning its attention to a tiny pill made by a small company with hopes to succeed where so many have failed. In the third quarter of this year, Axovant Sciences will release Phase 3 data that will determine whether its drug, intepirdine, can improve cognition and function in patients with mild or moderate Alzheimer’s. Axovant’s readout will follow an outright failure from Merck, disappointing results from Eli Lilly, and a vexing setback for TauRx. (Garde, 2/22)

In other news —

The New York Times: Prolonged Sleep May Be Early Warning Sign Of Dementia
Older adults who started sleeping more than nine hours a night — but had not previously slept so much — were at more than double the risk of developing dementia a decade later than those who slept nine hours or less, researchers report. The increased risk was not seen in people who had always slept more than nine hours. (Rabin, 2/22)

California Healthline: Alzheimer’s Looms Large For Latinos
The number of Alzheimer’s cases in the United States is rising, especially among Latinos — the fastest growing minority in the country. With no cure in sight, diagnoses among U.S. Latinos are expected to increase more than eightfold by 2060, to 3.5 million, according to a report by the University of Southern California’s Edward R. Roybal Institute on Aging and the Latinos Against Alzheimer’s network. (de Marco, 2/23)

This is part of the KHN Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.

Perspectives: Drug Prices Are Meaningless — And That’s The Problem

Read recent commentaries about drug-cost issues.

Los Angeles Times: Big Pharma Really, Really Doesn’t Want You To Know The True Value Of Its Drugs
The latest poster child for cruel and inhuman drug pricing is Kaleo Pharma, maker of an emergency injector for a med called naloxone, which is used as an antidote to save the lives of people who overdose on painkillers. As America’s opioid crisis reaches epidemic levels, Kaleo has jacked up the list price for its Evzio auto-injector by 600%, soaring from $690 several years ago to $4,500, according to lawmakers. (David Lazarus, 2/17)

The Wall Street Journal: How Other Countries Freeload On U.S. Drug Research
President Trump says American companies have been getting “systematically ripped off” by foreign governments and firms. He’s right. Yet he has backed a proposal that would make the problem even worse—permitting Americans to buy prescription drugs from overseas retailers, a practice known as importation. This policy wouldn’t help American consumers much, but it would gut American pharmaceutical companies. The negotiator-in-chief should instead use his skills to open foreign markets for American firms. (Peter Pitts, 2/21)

The Hill: Allowing Medicare To Directly Negotiate Drug Prices Will Hurt Seniors’ Access To New Drugs 
President Trump’s recent attack on pharmaceutical companies as “getting away with murder” is the stuff of headlines. He also commented that the federal Medicare program could save billions of dollars if the program negotiated prices directly with pharmaceutical companies. But lawmakers and the public should understand what direct Medicare drug price negotiations might mean for seniors’ access to needed treatments. (Rafael Fonseca, 2/17)

National Review: Donald Trump & Prescription Drug Costs: Harder To Lower Than They Seem
Keeping track of Donald Trump’s shifting policy positions could make even the most balanced of observers dizzy. Take, for example, the president’s position on prescription-drug prices. Candidate Trump garnered bipartisan plaudits by criticizing the pharmaceutical industry for the high cost of prescription drugs. Shortly after becoming president, he reached out to Democrats such as Representative Elijah Cummings with a promise to lower drug costs. But not long after that, he met with drug company CEOs and backed away from that promise. (Michael Tanner, 2/15)

Health Affairs: Can Employers Take A Bigger Role In Controlling Drug Costs?
An estimated 150 million Americans receive insurance through their employer — and employees and employers alike continue to suffer from “sticker shock” for prices for new drugs, despite several years of debate and threatened congressional action to control the high prices of pharmaceutical products. While considerable attention has been paid to potential actions by Medicare or the Food and Drug Administration (FDA), there has been less focus on the role of private payers to solve the issue. Employers sponsoring health benefits are not bound by the same statutory constraints that apply to Medicare and can decide with fewer restrictions what is covered and how much of the cost employees pay for each service. However, employers are sensitive to making changes to health benefits that could interfere with employee recruitment and this article will discuss employers’ options to address high drug prices more aggressively in that context. (Rober Galvin and Troyen Brennan, 2/17)

Albany Times Union: Why We Get Frustrated With Prescription Drug Prices!
This weekend as I was thumbing through some items in the pharmacy, I came  across the manufacturers information on the reintroduction of Auvi-Q to the market.  Auvi-Q is a autoinjector of epinephrine which is more publicly known as Epi-Pen. The drug is made by Kaleo who you might remember as they recently made news with their new product for narcan for drug overdose know as Evzio.  Evzio as was noted in this interview by Asa Stackel at WNYT 13, comes in at a price of $4,920 per unit which significantly more expensive than other naloxone products which are in the $100 range on average.  As a reminder the Evzio was about $690, came off the market and then back on at $4,920. Well history is repeating itself…..Auvi-Q, which last time we dispensed it was about $465 in early 2016 is back on the market at $4,200.  Yes, a ten fold increase! (New York Assemblyman John T. McDonald, 2/20)

Bloomberg: Alexion Needs New Bets
Alexion Pharmaceuticals Inc. is the latest in a long line of biopharma dealmakers to wish for a time machine. Its $8.4 billion acquisition of Synageva in 2015 was meant to help resolve its dependence on its leading rare-disease drug Soliris. It’s not working. (Max Nisen, 2/16)

The Motley Fool: Thanks, Martin Shkreli, For The Dumbest Advice Ever On Drug Pricing
Martin Shkreli is the epitome of everything the American consumer loves to hate. Shkreli, formerly the CEO of privately held Turing Pharmaceuticals, found himself in the spotlight in Sept. 2015 after his company acquired the rights to Daraprim, a life-saving, infection-fighting drug for AIDS and cancer patients. Despite not changing the formulation of Daraprim (which happened to be more than six decades old), or altering its manufacturing process one iota, Shkreli raised its per-pill price from $13.50 to $750 overnight, a nearly 5,500% increase. (Sean Williams, 2/17)

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When Giving ‘Hope To The Hopeless’ Comes At A Sky-High Price

News outlets report on stories related to pharmaceutical drug pricing.

Sacramento Bee: Orphan Drugs Offer Hope For Treating Rare Diseases, Despite High Price Tags 
At Sutter Children’s Center in Sacramento this week, the 7-year-old was given an injection of Spinraza, the first drug approved in the U.S. to treat spinal muscular atrophy, or SMA. … With Spinraza, each thumb-sized 5-milliliter vial – about a teaspoon’s worth – costs $125,000. The recommended dosage is four “loading” injections within about two months, followed by maintenance shots every four months for life. For families such as the Goepperts, that price tag is staggering: roughly half a million dollars just for the initial series of Spinraza shots and $375,000 a year thereafter. (Buck, 2/20)

USA Today: New Patient Group Focuses On Drug Prices Amid Bipartisan Concern
A new patient advocacy group launches Wednesday that distinguishes itself by focusing only on drug prices and eschewing money from the pharmaceutical industry at a time when drug makers are pouring millions into a campaign fighting efforts to regulate them. The formation of Patients for Affordable Drugs (PFAD) comes as local officials and members of Congress are decrying the latest huge drug price hikes, this time by drugmaker Kaléo for its version of the overdose antidote naloxone. (O’Donnell and Shesgreen, 2/22)

Stat: The Latest Voucher Deal Suggests Prices Are Finally Falling
In a noteworthy deal, Gilead Sciences agreed to pay $125 million to Sarepta Therapeutics for a priority review voucher. But one Wall Street analyst expressed disappointment over the price tag and suggested the deal raises questions about how much these controversial vouchers can fetch going forward. (Silverman, 2/21)

San Francisco Chronicle: Pharma CEO Insists $89,000 Drug Will Be Affordable For Patients 
When Marathon Pharmaceuticals’ $89,000 price for a year’s supply of its newly approved muscular dystrophy drug sparked outrage this month, the company’s CEO responded with another figure: $20. That’s how much he expects Duchenne muscular dystrophy patients to pay per prescription of the drug, he wrote in a letter posted on the company’s website. Insurers would cover the medication, and patients would pay only their typical co-pays. He touted the company’s patient assistance program to help patients defray out-of-pocket costs. (Schencker, 2/21)

Stat: Q&A: Pharmacy Middlemen Are ‘Monopolistic Terrorists’
Recent debates about drug pricing have tended to focus on manufacturers and patients, but Doug Collins is among the few legislators looking at the middlemen. Collins, a Republican congressman from Georgia, railed against the country’s three major pharmacy benefit managers in an interview with STAT, expressing his desire for a drug-pricing debate that centers on what he views as the true power centers instead of  “Pharma bro” and the push to allow drug-price negotiation under Medicare Part D. (Facher, 2/20)

Boston Globe: Harvard Pilgrim Expands Use Of Novel Drug Purchasing Deals 
Leveraging its buying power as one of the state’s largest health insurers, Harvard Pilgrim Health Care has struck two more deals to pay for expensive drugs based on how effectively they treat patients, an emerging strategy aimed at reining in medical spending. Harvard Pilgrim, which has 1.3 million members, said the agreements cover the rheumatoid arthritis medicine Enbrel, made by Amgen Inc., and Eli Lilly & Co.’s osteoporosis medicine Forteo. (McCluskey and Woodward, 2/22)

Stat: Here’s Why J&J Won The Bidding For Actelion – And Sanofi Didn’t
As big-game hunting goes, Sanofi chief executive Olivier Brandicourt is 0-for-2. Two months ago, Sanofi appeared to be on the verge of acquiring Actelion Pharmaceuticals, which would have been a notable accomplishment for a couple of reasons: Johnson & Johnson had just backed out of negotiations to acquire Actelion and Sanofi had recently lost out on bidding for Medivation, a stinging defeat in a growth strategy that relies on big deals. (Silverman, 2/16)

Stat: PhRMA CEO: ‘FDA Review Process Is The Gold Standard’
The CEO of the Pharmaceutical Research and Manufacturers of America on Tuesday came out strongly in favor of a rigorous Food and Drug Administration. His remarks come as the Trump administration weighs potential candidates to lead the agency who have suggested they would radically change how it vets new drugs. Asked by STAT about the notion that the FDA should no longer evaluate drugs for effectiveness, the industry trade group’s Stephen Ubl was unambiguous: “We believe that the FDA review process is the gold standard and should remain so.” (Robbins, 2/21)

LancasterOnline: Pennsylvania Proposal Would Make Prescription Drug Manufacturers Reveal Costs
Some state lawmakers are proposing to give the Insurance Department more teeth to tackle the problem of skyrocketing prescription drug prices. House Bill 161 would require pharmaceutical companies to disclose the cost of drugs — including materials, research, clinical trials, marketing and advertising.The bill is co-sponsored by Lancaster city Democrat Mike Sturla and Jim Cox, a Republican who represents part of northern Lancaster County. (Stauffer, 2/21)

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Despite Costly Failures, Pharma Not Giving Up On Research For Alzheimer’s Medication

Meanwhile, Stat reports on the future landscape for new diabetes treatments and KHN looks at the lethal drugs used in the aid-in-dying cases.

The Wall Street Journal: Alzheimer’s: Pharma’s Great White Whale Is Still Worth Hunting
Alzheimer’s disease is both the largest unmet medical need in the U.S. and the most frustrating challenge for the drug industry. Don’t expect pharma companies to get discouraged anytime soon. Last week marked the latest failed trial of an experimental Alzheimer’s disease treatment when Merck & Co. announced results for verubecestat. Eli Lilly’s solanezumab flunked a clinical trial last November, the third failed late-stage trial for the drug. (Grant, 2/20)

Stat: Where Are All The New Diabetes Drugs?
There are few new drugs on the horizon for diabetes, which affects about 29 million Americans. Most of the treatments in late-stage development are simply improved versions of what’s out there — taken weekly versus daily, or orally instead of by injection. So has pharma run out of ideas in diabetes?  Not exactly. But whether its ideas will ever get to market is another question. There’s plenty of promising science in the early stages of research. Available drugs, however, work pretty well. Given the cost of development and a high bar for approval, pharma can only afford to advance true-blue breakthroughs, and those are hard to come by. (Garde, 2/19)

Kaiser Health News: Docs In Northwest Tweak Aid-In-Dying Drugs To Prevent Prolonged Deaths
Two years after an abrupt price hike for a lethal drug used by terminally ill patients to end their lives, doctors in the Northwest are once again rethinking aid-in-dying medications — this time because they’re taking too long to work. The concerned physicians say they’ve come up with yet another alternative to Seconal, the powerful sedative that was the drug of choice under Death with Dignity laws until prices charged by a Canadian company doubled to more than $3,000 per dose. (Aleccia, 2/21)

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Longer Looks: A K Street Renegade; Dismantling Obamacare & Opioids In Sierra Leone

Each week, KHN’s Shefali Luthra finds interesting reads from around the Web.

The Wall Street Journal: The Rise And Fall Of A K Street Renegade
Few outside Washington had ever heard of Evan Morris. Yet in the capital of wheeling and dealing, he was one of its most gifted operators. From his start as an intern in the Clinton White House, he made powerful friends and at age 27 became a top Washington lobbyist for Roche Holding AG of Switzerland, one of the world’s largest pharmaceutical companies. (Brody Mullins, 2/13)

Vox: No Limits 
Timmy Morrison was delivered by emergency C-section, weighing in at 3 pounds, 9 ounces. Doctors put him under anesthesia within a week and into surgery within a month. Parts of his stomach sometimes made their way to his lungs. Workers in the intensive care unit frequently needed to resuscitate him. He arrived seven weeks premature — but, in a way, just at the right time. (Sarah Kliff, 2/15)

The Dallas Morning News: Severely Disabled Kids’ Lives At Risk, Parents Say, As Texas Enacts Medicaid Cost-Savings Plan
Amy Pratt drove her severely disabled son, Quinten, four-plus hours to Children’s Medical Center Dallas only to learn the insurance company that Texas hired to care for him had suddenly denied payment for an important procedure, one that could potentially save the 9-year-old’s life. In El Paso, 11-year-old Rudy Smith lost most of the therapy services that helped him cope with cerebral palsy and a severe form of epilepsy, which plagues him with 50 to 100 seizures a day. His mother says she’s having trouble getting prescriptions filled, and the insurance company keeps sending her incorrect or faulty medical supplies. (J. David McSwane, 2/13)

The New York Times: Will Obamacare Really Go Under The Knife?
Six days after he was sworn in as America’s 45th president, Donald J. Trump traveled to Philadelphia to address Republican lawmakers at their annual retreat. Standing behind a lectern emblazoned with the presidential seal, Trump predicted, “This Congress is going to be the busiest Congress we’ve had in decades.” Being Trump, he could not resist ad-libbing a superlative: “Maybe ever. Maybe ever. Think of that.” (Robert Draper, 2/14)

The Atlantic: Universal Health Care And The Future Of The Affordable Care Act 
The Senate confirmed Tom Price as secretary of health and human services at 2 a.m. on Friday. After a contentious confirmation process, the Trump administration and the Republican-controlled Congress had finally installed one of the leading generals in its war on Obamacare in the department that oversees its programs. Price is a titan in the GOP camp that wants to repeal the health law, and is perhaps one of the few Republican lawmakers with both the vision and the experience needed to begin the daunting task. (Vann Newkirk, 2/14)

The New Yorker: Another Planned Parenthood Protest Showdown
In January, 1993, the New York City Council moved unanimously to erect a sign at the intersection of Bleecker and Mott streets designating the small corner on the eastern edge of the West Village as Margaret Sanger Square. The bill, introduced by Kathryn Freed, noted that Sanger had opened America’s first birth-control clinic, in Brooklyn, in 1916, and that when she was arrested and jailed on obscenity charges she had taught her fellow-inmates about contraception. Her second birth-control clinic eventually became part of Planned Parenthood in New York City, which now serves more than fifty thousand patients each year and has been headquartered at the corner of Bleecker and Mott since 1992. (Jia Tolentino, 2/13)

Vox: How Repealing Obamacare Could Splinter Neighborhoods
Sure, it was billed as a policy that would make individual people healthier — and it ended up insuring 20 million people. But it also made neighborhoods healthier. It meant communities no longer had to fight over local tax dollars to care for the uninsured. It also meant there were fewer uninsured people who felt cast out and dehumanized by their communities. It lifted up our poorest and most vulnerable neighbors. (Alvin Chang, 2/13)

Al Jazeera: Opioids: Sierra Leone’s Newest Public Health Emergency
The dark street corner would have been silent if not for the grumble of a motorbike. It was nearing midnight, but for Ibrahim Sesay – a 27-year-old motorbike taxi driver in Freetown – the evening had just begun. He pulled four small pills from his breast pocket, gulped them down without water and set off into the night. (Cooper Inveen, 2/13)      

The Wall Street Journal: Computers Turn Medical Sleuths And Identify Skin Cancer
When it comes to melanoma, early detection is a matter of life and death. But it takes a trained eye to distinguish a harmless blemish from cancer, and many people around the world lack ready access to a dermatologist.Scientists have been seeking a solution for some time. In the latest sign that they’re succeeding, researchers at Stanford University have found a way to get a computer to identify skin cancer as reliably as board-certified dermatologists can. The hope is that, eventually, scientists can get this to happen on a smartphone anywhere in the world. (Daniel Akst, 2/10)

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Cleveland Clinic Reports A 2016 Income Drop — Blames Obamacare Reimbursement Rates And High Drug Prices

Meanwhile, in Florida, Republicans are pushing to repeal the state’s certificate-of-need regulations, a Kansas House committee blocks a bill that would allow the Kansas University Health System to ban concealed firearms and the Florida-based Nemours Children’s Hospital awaits state regulators decision about its new heart and lung transplant center.

Cleveland Plain Dealer: Cleveland Clinic CEO Toby Cosgrove Reports Rough Financial Year For Hospital In 2016
The Cleveland Clinic suffered a nearly 50 percent decrease in operating income in 2016, falling to $243 million from a record high of $481 million the year before, according to a report released Wednesday. During his annual State of the Clinic address to employees, Clinic CEO Toby Cosgrove said the decrease was expected and was driven by falling reimbursement brought about by the Affordable Care Act as well as rising drug costs. (Zeltner, 2/15)

Tampa Bay Times: State Approval Of Increased Hospital Beds Could End This Year
When someone wants to build a new hospital or nursing home in Florida or add beds in an existing facility, the state has to agree that their community has a need for expanded health care. It’s a regulation meant to ensure that poor and rich communities alike have equal access to hospitals, hospices and other health facilities. But at $10,000 to $50,000 per facility application, it’s also costly and can lead to lengthy, even pricier lawsuits. (Auslen, 2/15)

KCUR: Bill Allowing KU Health System To Ban Guns Fails In Committee Vote 
A Kansas House committee narrowly rejected a bill Wednesday that would have allowed the University of Kansas Health System to continue banning concealed firearms. It failed to advance on an 11-11 vote. The chairman of the House Federal and State Affairs Committee, Republican Rep. John Barker of Abilene, chose not to vote to break the tie. A state law set to take effect in July will allow concealed weapons on university campuses and in public hospitals and government buildings unless security is in place to keep out guns. The bill would have exempted the KU Health System. (Koranda, 2/15)

Orlando Sentinel: Nemours Awaits State Decision On Heart Transplant Program
Nemours Children’s Hospital will find out on Friday if the state is going to give initial approval to its application for a new heart and lung transplant program, which would be Central Florida’s first and the fifth in the state. This is good news to parents such as Kelly Green, an Orlando resident whose 9-year-old son has congenital heart disease and might one day need a heart transplant. (Miller, 2/15)

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Scott Gottlieb Emerges As Pharmaceutical Industry’s Likely Favorite To Head The FDA

Meanwhile, in Maryland, health care advocates and officials are pushing for drug affordability legislation.

CQ Roll Call: Drug Companies Throwing Weight Behind Gottlieb To Lead FDA
The pharmaceutical industry is privately urging the Trump administration to tap Scott Gottlieb, a former official at the Food and Drug Administration, to be the agency’s new commissioner, according to several lobbyists and individuals with knowledge of the discussions. Drug companies, which thrive on a high level of regulatory consistency given the long development timelines for their products, are growing concerned that President Donald Trump may choose an outsider with no scientific background who would make drastic changes to the current drug approval system. (Williams, 2/16)

The Baltimore Sun: Health Care Advocates Make Pitch For Bills To Block Drug Price Hikes 
Healthcare advocates who are pushing for bills to combat rising prescription drug prices in Maryland drew sympathy but also skepticism from state lawmakers Wednesday. The General Assembly is considering a pair of bills. One would require drug companies to give notice of and explain significant price hikes. The other would authorize the attorney general to sue makers of generic drugs who engage in price gouging. (Woods, 2/15)

The Associated Press: Md. Health Officials Call For Drug Affordability Legislation
When Bonnita Spikes, of Prince George’s County, learned her Alzheimer’s medication’s price had skyrocketed to $500, she was no longer able to afford the essential medicine. Diagnosed in 2013, Spikes quit her job after she determined her condition had progressed to the point where she was no longer able to fulfill her duties, leaving her without insurance or the ability to pay the soaring drugs’ costs. “I just can’t imagine needing something that important and not being able to get it,” Spikes said. (Schwartz, 2/15)

And in other drug pricing news —

Kaiser Health News: 5 Reasons Why An $89,000 Drug Has Congress Fuming
The latest flashpoint in the ongoing debate over high drug prices is Emflaza, an $89,000-a-year drug that treats Duchenne muscular dystrophy. People who have been watching the drug price issue closely, however, can reasonably ask why there is so much heat at that price tag? Late last year, two drugs went on the market for six-figure prices. Exondys 51 sells for $300,000 a year and Spinraza for a whopping $750,000. (Tribble and Lupkin, 2/16)

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How Long You Stay On Opioids May Depend On The Doctor You See In the E.R.

Which doctor a person happens to see at a local emergency room can have long-term consequences when it comes to opioid use.

Within the same hospital, some doctors are three times more likely to prescribe an opioid than other doctors, and patients treated by high-prescribing doctors are more likely to become long-term opioid users, according to a study published Wednesday in the New England Journal of Medicine.

“Physicians are just doing things all over the map,” says Dr. Michael Barnett, an assistant professor at the Harvard T. H. Chan School of Public Health and one of the study’s authors. “This is a call to arms for people to start paying a lot more attention to having a unified approach.”

The study looked at how many opioid prescriptions emergency physicians gave to about 377,000 Medicare beneficiaries from 2008 through 2011. The lowest-prescribing quartile of doctors prescribed opioids to just 7 percent of patients, while the highest prescribed opioids to 24 percent — more than three times as often.

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Patients who saw a high-intensity prescriber were about 30 percent more likely to end up with a long-term opioid prescription of at least six months within the year following their hospital visit. They were also more likely to return to the hospital in the next 12 months with an opioid-related fall or fracture, a risk factor for seniors who take the powerful painkillers.

Overall, about one in every 48 Medicare patients prescribed an opioid in the study were likely to become a long-term opioid user.

There is a growing consensus among doctors that opioids have long been overprescribed. In 2010, there were enough prescriptions written to supply every American adult with hydrocodone for a month, according to the Centers for Disease Control and Prevention.

Part of the problem, Barnett believes, is that there isn’t enough guidance for doctors on when it’s appropriate to prescribe an opioid. Much of the evidence for when they are appropriate comes from small studies sponsored by drug companies.

“It’s kind of a grey area and there’s not very clear evidence around what you should do, so we use our own judgement. And there’s a huge gulf between what one doctor thinks and another,” Barnett explains.

Take, for example, a patient who comes to the emergency room complaining of back pain. There’s evidence that opioids are not necessary in that situation, but many doctors prescribe them anyway, said Barnett. “The world of pain treatment outside of opioids is limited and can take time to figure out. Opioids are an easy fix.”

The problem, he said, is that “even one prescription for opioids carries risks with it, that from my own experience as a provider, we tend to underestimate and under-explain to patients.”

“It is very, very plausible that well-intentioned but perhaps overly aggressive prescribing of opioids makes it likely that a patient will continue a medication long-term even if they don’t truly need it,” said Dr. David Juurlink, a professor of medicine at the University of Toronto. He was not involved in the study. “The doctors in the lowest quartile are the ones whose prescribing we should seek to be emulating.”

The study did not look at whether the opioids were correctly prescribed in each incidence. Dr. Carla Perissinotto, a geriatrician at the University of California San Francisco, worries that some of the doctors in the lowest quartile might be under-prescribing. “We have to be careful to not make assumptions too quickly and assume they’re bad prescribers, because it could be the opposite,” said Perissinotto, who also was not involved in the study.

Usually, a patient is prescribed just a handful of pills by a doctor at the emergency department to tide them over until the patient can visit his or her primary care physician.

But many primary care doctors simply refill the opioid prescription for another 30 days or longer, a phenomenon Barnett calls clinical inertia. “There’s this cognitive bias to keep going with the flow especially if the patient still feels they’re in pain.”

That can have long-term implications: One-third of people who have taken prescription opioids for at least two months say they became addicted to or physically dependent on them, a recent Washington Post-Kaiser Family Foundation survey found.

“We know there is a population of people who will potentially get addicted if they’re exposed to an opioid,” said Dr. Lewis Nelson, chair of the department of emergency medicine at Rutgers New Jersey Medical School, who was not involved with the study. “It’s a numbers game. The more people you expose, the more people you are likely to hit in that population likely to get addicted.”

Some hospitals are starting to help doctors prescribe opioids more judiciously. Many of those efforts have taken place after 2011, and therefore any changes would not be seen in the New England Journal of Medicine study.

For the past year, St. Joseph’s Regional Medical Center in New Jersey has been instructing doctors to prescribe opioids only as a last resort, said Dr. Mark Rosenberg, who runs the hospital’s emergency department and is on the board of the American College of Emergency Physicians. Rosenberg helped institute protocols that have doctors try other methods of pain relief first, such as a Novocain injection. In just over a year, the hospital has managed to reduce the number of opioid prescriptions written in the emergency room by 50 percent.

New York City and Washington state have also tried to introduce opioid prescribing guidelines into hospitals, and the ACEP expects to release new nationwide guidelines for emergency physicians in 2018.

Categories: Pharmaceuticals, Public Health, Syndicate

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Perspectives: Drugmakers Hike Prices Because They Can. The How Gets A Little More Complicated.

Read recent commentaries about drug-cost issues.

Bloomberg: Games Drugmakers Play
One of my goals when I joined Bloomberg View a month ago was to dive into the contentious subject of drug pricing. I’d like to explain to readers why the pharmaceutical companies have been raising prices so relentlessly these past half-dozen years, even as the practice has become a huge issue on Capitol Hill and in the country. My friend Barry Werth, who has written two fine books about the biotech company Vertex, says the answer is simple: “Because they can.” But the details of how they do it can be murky, complicated — and sometimes underhanded. (Joe Nocera, 2/13)

The Washington Post: Why People Should Be Able To Buy Drugs Approved In Other Countries
For years, muscular dystrophy patients in the United States have been purchasing the drug deflazacort — used to stabilize muscle strength and keep patients mobile for a period of time — from companies in the United Kingdom at a manageable price of $1,600 a year. But because an American company just got approval from the Food and Drug Administration to sell the drug in the United States, the price of the drug will soar to a staggering $89,000 annually, the Wall Street Journal reported last week. (Robert Gebelhoff, 2/14)

The Wall Street Journal: Don’t Ignore Politicians’ Ire Over Orphan Drugs
The latest drug-pricing drama has pushed the debate in a new direction. It would be a mistake for biotech shareholders to dismiss the uproar as the same old story. Marathon Pharmaceuticals’ decision to list the old steroid deflazacort at an $89,000 annual price to treat Duchenne muscular dystrophy, and the resulting outcry, has had an immediate impact on the company. Marathon said Monday that it plans to delay the drug’s U.S. launch, scheduled for next month, after politicians in Washington expressed anger over the company’s plans. (Charley Grant, 2/14)

Los Angeles Times: Trump Needs To Be Careful In Deregulating The Drug Industry
In a recent meeting with pharmaceutical-industry bigwigs, President Trump declared that “we’re going to be cutting regulations at a level that nobody’s ever seen before.” He also said that “we’re going to have tremendous protection for the people.”It’s hard to see how he can do both. (David Lazarus, 2/10)

Fayette Tribune: President Trump Misunderstands What Government Drug Price Negotiations Entail
President Donald Trump recently pledged to let federal officials negotiate the prices of drugs covered under Medicare. He claims this will save taxpayers billions of dollars. Nobody doubts that Trump and his team are shrewd negotiators. But the sorts of “negotiations” that Trump refers to have nothing in common with haggling over a real estate deal. Instead, the action that Trump has proposed — repealing the non-interference clause, originally drafted by Democratic Senators Ted Kennedy and Tom Daschle — would result in Medicare drug prices going up and patient choice going down. (Peter J. Pitts, 2/10)

North Jersey: Overdose Rescue Drug Overpriced
There’s no dispute opioid addiction is an epidemic.More than 183,000 people have died from opioid overdoses since 1999, according to the federal Centers for Disease Control and Prevention. In New Jersey, Gov. Chris Christie and legislators have responded with proposals to expand treatment for addicts and to limit the amount of drugs physicians can prescribe. Other states are following suit. Unfortunately, not all responses are as constructive. (2/13)

Scranton Times-Tribune: End Gouging For Antidote
The national opioid epidemic now claims more lives each day than guns or car crashes — about 91 a day nationwide, according to the Centers for Disease Control and Prevention. As appalling as that is, the toll would be far worse but for naloxone, a drug approved in 1971 that stops the respiratory arrest produced by opioid overdoses. Public health agencies have credited the drug with saving many thousands of people who might otherwise have died due to heroin or prescription opioid overdoses. (2/14)

Stat: The Creative Science Of Coining Drug Names
Cialis, Eliquis, Jevtana, Xgeva. These drug names may sound silly, but the process of creating and testing them is anything but. With more than 30,000 proprietary drugs in the United States alone, coming up with a unique brand name is no easy task. And while these names may seem like they were created by over-caffeinated Scrabble players, they are usually the result of intense focus by creative name development professionals coupled with clear-eyed research designed to enhance the prospects of Food and Drug Administration approval. (Mike Pile, 2/8)

Bloomberg: Teva Tries Out ‘Alternative Guidance’
Teva Pharmaceutical Industries Ltd. gave investors a nice surprise on its fourth-quarter earnings call Monday by maintaining its 2017 revenue and earnings guidance. Teva shares jumped about 4 percent on the news. Many investors had assumed Teva’s guidance, already cut in January, would have to come down again after a U.S. court knocked out patents on its best-selling drug Copaxone, making generic competition much more likely. The company’s CEO stepped down shortly afterwards. (Max Nisen, 2/13)

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Next Up On The Pharma Rebranding Bandwagon: Generics

News outlets report on stories related to pharmaceutical drug pricing.

Stat: Generic Drug Lobby Rebrands Itself As Pricing Politics Intensify
The Generic Pharmaceutical Association is no more. Meet the Association for Accessible Medicines. Every industry group invested in the drug-pricing debate is gearing up and burnishing its brand. PhRMA, the brand-name pharmaceuticals lobby, has its “Go Boldly” campaign. Now the generics lobby is launching its own “education campaign” under a whole new name. (Scott, 2/14)

Stat: Will Pharma Use A Tax Break To Create Jobs? It Didn’t Last Time
Drug makers are promising to create tens of thousands of American jobs if President Donald Trump follows through on his promise to give them a big tax break if they “repatriate” cash they’ve stashed overseas. But that’s not what happened last time pharma got a tax holiday. Instead, drug makers used the tens of billions they brought back to the US to enrich their CEOs and drive up their stock prices. Rather than adding jobs, they laid off thousands of workers. (Garde, 2/10)

Stat: What Might Pharma Buy With Its Repatriated Cash?
If President Donald Trump keeps his promise, the world’s biggest drug makers will soon get access to billions of dollars siloed overseas. And if investors and analysts are right, much of that money will go toward acquisitions. So what might pharma buy with its freed-up capital? According to a recent investor survey by EvercoreISI, these are 2017’s most likely takeout targets. (Garde, 2/10)

Reuters: Pharma Industry Shuns Trump Push For Radical Shift At FDA
U.S. President Donald Trump’s vow to roll back government regulations at least 75 percent is causing anxiety for some pharmaceutical executives that a less robust Food and Drug Administration would make it harder to secure insurance coverage for pricey new medicines. The prospect of big change at the regulatory agency comes as drugmakers are under fire for high prices, including Marathon Pharmaceuticals LLC, which said Monday it was “pausing” the launch of its Duchenne muscular dystrophy drug after U.S. lawmakers questioned its $89,000 a year price. (2/15)

Bloomberg: CEO Under Fire For $89,000 Drug Has A History Of Steep Price Hikes 
The CEO of the latest drugmaker to face criticism over a product’s high price has a history of steep hikes on other drugs and at past companies. Marathon Pharmaceuticals LLC Chief Executive Officer Jeffrey Aronin, under fire for setting an $89,000 price on the company’s drug for a rare, deadly muscle disease, was questioned in a letter more than two years ago by Washington lawmakers about mark-ups on two heart drugs. Years earlier, as the leader of another company, Aronin took high price increases on a drug used to treat babies with a congenital defect. (Greifeld and Langreth, 2/14)

Bloomberg: Big Pharma Is Pointing Fingers, And Hoping Trump Will Listen 
In the fast-moving Washington game of who’s to blame for high U.S. drug prices, an often-overlooked industry is readying its defenses against pharmaceutical companies that fault other parts of the health sector for the costs faced by patients. Known as pharmacy benefits managers, or PBMs, the industry includes giants such as Express Scripts Holding Co. and CVS Health Corp., which negotiate prices with drugmakers, work with pharmacies and help set the co-pays patients pay out of pocket. Now these middlemen are now taking it from all sides. (Tracer, Langreth and Edney, 2/8)

Stat: SEC Plans To Review Some Pharma Industry Accounting Practices
Concerned about the way that some drug makers report earnings in their financial statements, the US Securities and Exchange Commission recently indicated plans to evaluate pharmaceutical industry accounting practices.The plans were disclosed in a Jan. 11 letter that the agency sent to Allergan over GAAP, or generally accepted accounting principles. (Silverman, 2/13)

The Baltimore Sun: Cost Of Overdose Drug Could Hamper Access In Maryland And Elsewhere 
The price of a drug that has saved the lives of more than 800 people overdosing on heroin or other opioids in Baltimore is rising rapidly.The antidote known as naloxone revives addicts after they’ve stopped breathing, with either a simple spray in their nose or an injection. The use of naloxone is a centerpiece of Baltimore public health officials’ wide-ranging efforts to battle the growing heroin epidemic, but the rising price of the antidote could constrain the campaign to stop or at least slow the rate of overdose deaths. (Cohn, 2/13)

Stat: Former PBM CEO Pleads Guilty To Paying Kickbacks
Aformer head of a pharmacy benefits manager pleaded guilty in federal court in Texas on Monday to paying kickbacks in order to win business from government health plans. Between 2001 and 2013, Douglas Pick, who was once chief executive at Pharmaceutical Technologies, orchestrated nearly $3.6 million in combined payments to the head of a health plan, as well as several individuals who were hired to boost business for the PBM, according to court documents. Pick faces up to three years in federal prison, according to the US Department of Justice. (Siverman, 2/14)

Stat: Gilead Faces New Patent Challenges To Hepatitis C Drugs In India
After losing one challenge to Gilead Sciences patents on hepatitis C drugs in India, patient advocacy groups are now challenging still other patents the company holds for its drugs in the country. At the same time, the groups are also challenging Gilead patents in Argentina, moves that reflect an ongoing strategy to widen patient access to the medicines. (Silverman, 2/14)

Nashville Tennessean: Tennessee Bill Would Nix Drug Swaps By Insurers, Benefit Drug Industry
A coalition of Tennessee patient and health care provider groups backed by major U.S. drug companies is behind a bill that would stop insurance companies from switching the medications they cover to cheaper substitutes midway through a coverage year. The Reliable Coverage Act would require insurance companies to keep providing the same medication coverage they promise when enrollees sign up each year. Currently insurers can change the medications they cover midway through an enrollment year by ending coverage of a particular drug entirely, which increases out-of-pocket costs for a drug or requires additional approval by the insurance company before a drug is covered. (Wadhwani and Boucher, 2/13)

Kaiser Health News: Former FDA Chief Cites 5 Things To Watch On Drug Approvals, And Keeping Drugs Safe
The just-departed commissioner of the Food and Drug Administration has concerns about plans to speed up drug approvals and dramatically reduce regulations at the agency, as advocated recently by President Donald Trump. Dr. Robert Califf, who stepped down last month, shared his thoughts about keeping Americans safe — and making sure drugs actually work — after about a year overseeing the federal agency. (Lupkin and Tribble, 2/14)

The Associated Press: Ex-Drug Company CEO Shkreli To Speak At Harvard
Controversial former pharmaceutical executive Martin Shkreli is set to speak at Harvard while out on bail awaiting his federal securities fraud trial. The former CEO of Turing Pharmaceuticals is expected to talk about investing and healthcare at an event organized by the Harvard Financial Analysts Club. The talk on Wednesday is open to the Harvard community only. (2/12)

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Decades-Long Quest To Battle Alzheimer’s Dealt Latest Blow As Merck Pulls Plug On Anticipated Drug

Similar treatments from Eli Lilly, Pfizer, and Johnson & Johnson have all come up short in recent years. Meanwhile, treatments for mental illnesses have seen few advances despite increased understanding of the diseases.

Stat: The Next Big Alzheimer’s Trial Came Up A Dud
Merck pulled the plug on a closely watched trial in Alzheimer’s disease after finding out its in-development drug had no effect on patients with mild forms of the disorder. The drug, verubecestat, showed “virtually no chance” of meeting its goal in a 2,200-patient study, Merck said. The trial was meant to read out later this year, but Merck, on the advice of its independent data monitors, pulled the plug early. (Garde, 2/14)

Stat: Scouring The Brain For Clues To New Treatments For Mental Illness
While demand for mental health drugs has surged, big pharmaceutical companies have largely backed away from investing in the field; the number of psychopharmacological drug research programs has shrunk 70 percent in the last decade, according to NeuroPerspective…To advance the field, researchers say they need to find biomarkers — tangible biological clues that can help diagnose mental illness, just the way high blood glucose levels can signal diabetes. The hope is that those biomarkers could help pinpoint what’s gone wrong in the circuitry of a particular patient’s brain and offer clues for drug development — and, perhaps one day, even precision psychiatric therapies. (Keshavan, 2/15)

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Drugmaker Hits Pause After Receiving Blistering Criticism Over $89,000 Medication

Hours after receiving a letter from Sen. Bernie Sanders and Rep. Elijah Cummings calling the pricing strategy on the muscular dystrophy drug unconscionable, Marathon Pharmaceuticals announced it was reevaluating the product launch.

The Associated Press: Company Announces Pause For Drug After Price Criticism
Marathon Pharmaceuticals announced Monday that it will temporarily halt the rollout of a drug to treat genetic muscle deterioration just hours after two members of Congress expressed outrage that the company planned to charge $89,000 a year for a drug that’s widely available abroad for about $1,000 a year. Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., urged the company earlier in the day to lower the drug’s price. (Freking, 2/13)

The Wall Street Journal: Firm Delays Muscular Dystrophy Drug U.S. Launch Amid Criticism Of $89,000 Price
In a statement posted on the website of a nonprofit group involved with muscular dystrophy, Marathon CEO Jeffrey Aronin said the company was “pausing our launch,” which had been scheduled for March. The company will meet with “caregivers and explain our commercialization plans, review their concerns, discuss all options, and move forward with commercialization based on an agreed plan of action,” he said in the statement. (Walker and Pulliam, 2/13)

Stat: Sanders Blasts Marathon Over The High Price For Old Medicine
The [delay] comes just hours after two lawmakers, including Senator Bernie Sanders (I-Vt.), sent a harshly worded letter, blasting Marathon for “unconscionable” pricing for the drug, which would be used to treat a rare form of muscular dystrophy. And beyond urging the drug maker to lower its $89,000 price, they demanded the company fork over a raft of information, including development expenses and profit projections. (Silverman, 2/13)

The Hill: Sanders, Cummings Hit Pharma Company For Drug’s $89K Price Tag
Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings (D-Md.), two vocal critics of high drug prices, demanded answers from Marathon Pharmaceuticals Monday, calling the price of Deflazacort “unconscionable.” “Marathon’s apparent abuse of government-granted exclusivity periods and incentives to sell what should be a widely available drug for $89,000 a year is unconscionable,” Sanders and Cummings wrote in the letter to the CEO of Marathon. (Hellmann, 2/13)

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Former FDA Chief Cites 5 Things To Watch On Drug Approvals, And Keeping Drugs Safe

The just-departed commissioner of the Food and Drug Administration has concerns about plans to speed up drug approvals and dramatically reduce regulations at the agency, as advocated recently by President Donald Trump.

Dr. Robert Califf, who stepped down last month, shared his thoughts about keeping Americans safe — and making sure drugs actually work — after about a year overseeing the federal agency. His takeaways:

1. Faster drug approvals, being advocated by Trump and others, don’t necessarily mean less expensive drugs.

“What I’m concerned about is that when people hear ‘faster approval,’ you get an image of the FDA sitting on this application and, you know, twiddling thumbs,” Califf said. “That’s not what happens.”

Califf explained that the slow part of getting a drug into patients’ hands happens well before the drugmaker submits an application to the FDA. It can take decades to discover, tweak and test a new drug molecule, and that development process is what needs a boost.

“The real action [happens] before the application gets submitted and that can be a time frame from years to decades as people try to figure out what will work and what will not,” Califf said.

Although faster drug development could bring development costs down, Califf said, “There’s not a direct relationship between the cost of development and the price of drugs or devices.”

“The concept of safety is much more complex than most people think about until they look into it deeply,” said Robert Califf, just-departed FDA administrator. (Courtesy of the FDA)

“The concept of safety is much more complex than most people think about until they look into it deeply,” said Robert Califf, just-departed FDA administrator. (Courtesy of the FDA)

2. A law passed in December should streamline drug approvals — in a positive way.

The 21st Century Cures Act, a bipartisan bill signed by President Barack Obama late last year, is a “very well-rounded piece of legislation that will speed up product development.”

To begin with, it encourages the FDA to consider pieces of information in its approval process in addition to traditional clinical trials, including “real world evidence” and biomarkers. Biomarkers are used in studies in place of outcomes that are more difficult to measure. For example, tumor shrinkage is sometimes a biomarker for surviving cancer.

“Using real world evidence in clinical trials is one that I’m particularly excited about as a way to get better answers at a lower cost and faster at the same time,” Califf said.

3. But Califf has concerns about maintaining drug safety standards and ensuring thorough clinical trials.

“The concept of safety is much more complex than most people think about until they look into it deeply,” Califf said. “All drugs have risk. None of them are absolutely safe. And the actual safety risks are only revealed through clinical trials with the same quality and number of patients involved as it takes to look at efficacy.”

About 92 percent of drugs that get into human clinical trials don’t make it to market because they fail to show any benefit or, worse, they have unexpected toxicity, he said.

“Declaring a drug is safe after very little information is treacherous,” Califf said.

Califf recently wrote for JAMA on the FDA’s balancing act of protecting the public and encouraging innovation. And the FDA released a paper last month documenting examples of a promising drug, vaccine or device that each did well in a Phase 2 clinical trial but “bombed out” in Phase 3, Califf said. Drugs typically go through three “phases” of studies called clinical trials before gaining FDA approval. With each new phase, researchers test drugs or other products on more people and on more measures of safety and effectiveness.

“It’s 22 examples of why it’s a big mistake to think that you can judge the balance of risk and benefit from a small amount of data,” Califf said.

4. Faster, better drugs can’t be approved if the FDA is understaffed, he argued.

Califf called a potential FDA hiring freeze “unfortunate” and said new staff is needed to meet the faster approval timelines and to give advice to those developing drugs so “bad mistakes” aren’t made.

The FDA has been steadily hiring staff to keep up with the growing industry it oversees. But hundreds of openings are still left to be filled.

Califf said the drug industry’s most valued interaction with the FDA is “during the process of [drug] development,” which begins before a drug application is submitted for approval. FDA staff can provide timely feedback and advice that can help a company choose the right studies needed for approval and, effectively, speed up drug development.

“A hiring freeze at this time when we are just revving up with 21st Century Cures is unfortunate. I hope that the dust will settle soon and the FDA can get back to its hiring,” Califf said.

5. The FDA is using big data to track the safety of drugs already on the market — and it plans to do that a lot more.

The FDA has an established adverse event database but after pain reliever Vioxx was pulled from the market in 2004, the agency began working on a system called Sentinel.

The initiative uses claims data to look at drug safety. It has 180 million individual electronic records and tracks every dispensed prescription, hospitalization as well as serious outcomes, Califf said. In addition, Sentinel is now being opened up to industry and academia.

“It’s used every day by the FDA,” Califf said, adding that the system is “getting better and better.”

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

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Drugmaker Marathon ‘Pausing’ Delivery Of $89,000-a-year Muscular Dystrophy Drug

In a surprise move Monday, Marathon Pharmaceuticals told patient advocates that it would “pause” the launch of their drug Emflaza because of pricing concerns expressed by patients and advocacy groups.

The drugmaker had announced an $89,000 annual price tag for their newly-approved drug last week — but patients and lawmakers immediately cried foul.

“What you’re doing is robbing my insurance company,” said Dana Edwards, a mother from New Jersey whose 12-year-old has taken deflazacort, the generic version of the drug, since he was five years old.

Newly approved Emflaza treats Duchenne muscular dystrophy, a rare, devastating neuromuscular disorder. Patients have been importing the generic version of the drug from overseas for about $1,200 a year.

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A statement read to a conference of parents, patients and advocates in Washington, D.C., and attributed to Marathon CEO Jeffrey Aronin said: “Our goal in commercializing Emflaza all along has been to make it available to that broader set of patients who prior to FDA approval have not had access to the therapy… We are pausing our launch, which has not yet taken place. We have not sold any new product and will pause that process.”

Pat Furlong, the president and founder of Parent Project Muscular Dystrophy, which sponsored the Monday morning conference, read the statement to an outraged crowd in a conference room at the Mayflower Hotel.

The company will continue to offer patients an expanded access program, Furlong read, which allows about 800 patients to receive the drug from the company. More can join that program for free, and patients can continue importing drugs from Canada or “wherever they are getting it,” the statement said.

Furlong told the audience that complaints from patients helped to prompt Marathon’s action: “Since last week’s approval, they’ve heard from all of us,” he said.

Late Monday, Marathon released a statement and open letter to the Duchenne community, elaborating on its goals of patient access and promising that “price should not be a barrier.”

“Put simply we expect patients will pay a standard copay of typically $20 or less per prescription,” the statement reads.

A Marathon Pharmaceutical executive speaks at the Parent Project Muscular Dystrophy advocacy conference. (Sarah Jane Tribble/KHN)

A Marathon Pharmaceutical executive speaks at the Parent Project Muscular Dystrophy advocacy conference. (Sarah Jane Tribble/KHN)

The company also heard from Capitol Hill. On Monday morning, Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., sent a letter to Marathon demanding answers about the $89,000 price tag for a drug that has been routinely used by Duchenne patients since at least 2005.

“We believe Marathon is abusing our nation’s ‘orphan drug’ program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases — not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades,” Sanders and Cummings wrote.

Orphan drugs are medicines targeted at people with rare diseases, which often carry very high price tags. Emflaza was approved last Thursday for treating Duchenne disease. The drug is a steroid that manages symptoms for Duchenne’s sufferers; it does not cure the underlying disease.

In January, Kaiser Health News published an investigation that found that the orphan drug program intended to help desperate patients is being manipulated by drugmakers. While the companies aren’t breaking the law, they are using the Orphan Drug Act to secure lucrative incentives and gain monopoly control of rare disease markets, the investigation found.

Republican Rep. Robert Aderholt, the chairman of the Appropriations subcommittee that funds the FDA, said the 6000 percent increase in deflazacort’s price “makes me question whether the current construct of how FDA approves orphan drugs does more harm than good if companies have found a way to game the system.”

In addition to orphan status, the FDA gave Marathon a rare pediatric disease priority review voucher, which the company can sell for millions of dollars to another drugmaker who can use it to expedite their own drug’s approval.

At Monday’s meeting, Marathon executives presented a slideshow to a packed room that detailed different patient financial assistance programs as well as the drug’s expected availability. Emflaza was expected to be on the market by mid-March, said Eric Messner, vice president of sales and marketing for Marathon.

Messner explained that the drug would cost between $50,000 to $54,000 after rebates and discounts. And he emphasized that Marathon did not expect patients to pay that price. “We expect that patients will experience a low or zero out-of-pocket experience. I know there has been a lot of discussion about that. How did we arrive at the price?” Messner said. An audience member interrupted Messner asking if the company had talked to patients and families about the price.

He offered assurance that they did and said there would be an ongoing dialogue with patient groups.

Edwards, the New Jersey mother, said she hopes President Donald Trump will fulfill his vow to bring down high drug prices.

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

Categories: Pharmaceuticals, Syndicate

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Viewpoints: Drug Companies ‘Risky’ Idea; Scrapping Rule On Guns And Mental Illness Is ‘A Bad Move’

A selection of opinions on health care from around the country.

Chicago Tribune: Guns And Mental Illness: Don’t Scrap This Rule
If someone has a mental illness severe enough that he cannot work or manage his own money, should he be allowed to own a gun? In the waning weeks of his presidency, Barack Obama answered that question. Motivated by Adam Lanza’s bloody rampage at an elementary school in Newtown, Conn., that killed 20 children and six educators in 2012, Obama imposed a rule that barred gun ownership for people who qualify for Social Security disability insurance because their mental illness keeps them from working, and who cannot manage their benefits. That pool is small — just 75,000 Americans. (2/10)

The Wichita Eagle: Services For Disabled Also In Violation
Not only did federal officials determine last month that KanCare was “substantively out of compliance with federal statutes and regulations,” they determined the previous month that services for people with disabilities were also out of compliance. Yet state lawmakers had to learn about both decisions by reading about them in the newspaper. (2/12)

The Des Moines Register: Legislators Should Repay State For Cheap Health Care
Gov. Terry Branstad said in 2012 he would begin voluntarily paying 20 percent of his state-funded health insurance premiums. He encouraged other state workers, including lawmakers, to follow suit. The next year, the governor said some legislators, who had previously enjoyed premium-free health insurance, had started contributing 20 percent. But not a single lawmaker is paying that share, according to December 2016 data obtained by The Des Moines Register. And it appears they are violating state law. (2/11)

The Columbus Dispatch: More Needed To Fight Overdoses
Ohio lawmakers are asking good questions about the money that Gov. John Kasich’s proposed budget would devote to the state’s opioid epidemic and the collateral damage from it. Last week, Republican and Democrat legislators said they are concerned that the budget does not do enough. The concern is warranted. Ohio leads the nation in drug-overdose deaths, with 3,050 recorded in 2015, the most recent year with complete statistics. (2/13)

The New York Times: LSD To Cure Depression? Not So Fast
Psychedelics, the fabled enlightenment drugs of the ’60s, are making a comeback — this time as medical treatment. A recent study claimed that psilocybin, a mushroom-derived hallucinogenic, relieves anxiety and depression in people with life-threatening cancer. Anecdotal reports have said similar things about so-called microdoses of LSD. … I fear that in our desire to combat suffering, we will ignore the potential risks of these drugs, or be seduced by preliminary research that seems promising. (Richard A. Friedman, 2/13)

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In Midst Of Pharma Trying To Rebrand Image, Company Jacks Up Old Drug’s Price To $89,000

Marathon has set the list price of its muscular dystrophy drug at $89,000 a year, 50 to 70 times higher than patients were paying to import the drug from the U.K.

The Wall Street Journal: Drug Industry Goes Boldly Into New Minefield
Last month, the pharmaceutical industry, under pressure from the president among others for persistent price hikes, launched an ad campaign to highlight its drug research. Called “Go Boldly,” an allusion to poet Dylan Thomas, the campaign was intended to bolster the industry’s reputation after pricing scandals involving companies such as Valeant Pharmaceuticals International and Turing Pharmaceuticals. It took just a few weeks for another pharma company to ignite a controversy and set off the requisite tales of patients unable to afford the treatment. (Grant, 2/10)

The Washington Post: An Old Drug Gets A New Price To Fight A Rare Disease: $89,000 A Year
An old steroid treatment, long available outside the United States, received approval this week for a rare disease that afflicts about 15,000 Americans. Though not previously approved in the United States, the drug, deflazacort, has for years been available to patients suffering from the devastating and fatal disease Duchenne muscular dystrophy; families can import it from abroad for about $1,200 per year on average. The new list price for the drug? $89,000 a year. (Johnson, 2/10)

The Wall Street Journal: Marathon Pharmaceuticals To Charge $89,000 For Muscular Dystrophy Drug After 70-Fold Increase
The U.S. Food and Drug Administration on Thursday approved Marathon’s drug, a corticosteroid called deflazacort, to treat a rare type of muscular dystrophy that affects some 12,000 boys in the U.S., most of whom die in their 20s and 30s. The drug isn’t a cure, but it has been shown to improve muscle strength, the FDA said in a statement announcing the approval. The drug wasn’t sold in the U.S. mainly because no company thought it would be profitable enough to warrant the effort of seeking FDA approval. (Walker, 2/10)

Meanwhile, one senator is looking into loopholes surrounding orphan drugs —

Stat: Grassley Opens Investigation Into Loopholes In Orphan Drug Law
Amid rising concern over prescription drug pricing, Senator Chuck Grassley (R-Iowa) has begun an investigation into whether drug makers are exploiting loopholes to widen the market for so-called orphan drugs. The move comes after accelerating scrutiny of this lucrative corner of the pharmaceutical market. Under the Orphan Drug Act, which was passed in 1983, the Food and Drug Administration began approving medicines to treat rare diseases that affect fewer than 200,000 people. The incentives to fill these unmet medical needs include tax credits and seven years of exclusive marketing rights. (Silverman, 2/10)

Kaiser Health News: Grassley Launches Inquiry Into Orphan Drugs After KHN Investigation
Republican Sen. Chuck Grassley, chairman of the Senate Judiciary Committee, has opened an inquiry into potential abuses of the Orphan Drug Act that may have contributed to high prices on commonly used drugs. In a statement, Grassley said the inquiry is “based on reporting from Kaiser Health News” and strong consumer concern about high drug prices. (Tribble, 2/10)

This is part of the KHN Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.