Gilead hep C drug prices blamed for England’s health service rationing treatment

first_img Related: The NHS later tried to completely block coverage for Harvoni, Gilead’s follow-up to Sovaldi, as well as two rival medicines, by questioning the level of evidence for using the new treatments, the BMJ reported. But after NICE recommended the drugs, the NHS then rationed the medicines. The move angered many physicians and patient groups, which disputed the agency’s rationale, according to the medical journal.“The case shows how high prices for high prevalence diseases places huge stress on health systems and reveals the limitations of conventional cost effectiveness analysis,” the BMJ wrote.advertisement [email protected] Eric Risberg/AP Gilead pricing for Sovaldi hepatitis C drug slammed by senators Hepatitis C drugs remain unaffordable in many countries, says WHO study [UPDATE: A day later, an NHS spokesman wrote us this: “These claims are both inaccurate and naive. Actually what the NHS has done is invest an extra $265 million in hHep C treatments last year and again this year, making it the single biggest new treatment investment in years. With that we’ve treated high risk patients, and like many other countries are now working through the backlog of non-urgent patients.“It is utterly naive to pretend that the NHS could instead somehow have ‘magicked up’ several billion pounds in one year, for this one condition, without that meaning damaging cuts in other critical services such as mental health, cancer or primary care. That’s not a failure of planning; that’s just the reality of the financial circumstances facing us.” He added that, as rival drugs reduce prices, NHS expects to expand treatment.]The medical journal noted that the NHS exaggerated the number of patients who might seek treatment as a scare tactic. A study commissioned by the NHS suggested that if $400 million were diverted from its existing budget to pay for the hepatitis drugs, 1,542 lives would be lost because money would not be available to help other patients with other illnesses. Spending $925 million would cause 3,598 deaths.The episode illustrates how the high cost of the Gilead medicines was capable of wreaking havoc, according to the BMJ. Launched in early 2014, Sovaldi had a list price of $84,000 for a 12-week regimen, or a $1,000 a pill, before rebates, and doctors quickly prescribed the drug thanks to a cure rate exceeding 90 percent. About a year later, Gilead introduced Harvoni at a list price of $94,500.Gilead has regularly maintained that the high cure rates will eventually lead to lower health care costs by reducing the need for hospitalizations and other treatments for liver cancer and liver transplants. The NICE recommendations acknowledged such calculations. But such savings are only recognized later and, meanwhile, a growing number of payers in various countries complained the drugs were budget busters.In the US, some state Medicaid programs and private health insurers began restricting access to the medicines, which prompted a warning from federal officials and successful lawsuits from consumers. Gilead, you may recall, has been harshly criticized for its pricing practices; a US Senate Finance Committee report late last year found the company placed profits over patients in establishing prices. Related: Faced with budgetary constraints, England’s National Health Service took several controversial steps to delay coverage of Gilead Sciences’s pricey hepatitis C treatments, but did so at the expense of patients, according to an investigation by the BMJ, the UK medical journal.Specifically, the agency’s moves caused delays in providing treatment to many of the estimated 160,000 hepatitis C patients, while others were unable to obtain the medications due to rationing. As a result, some people are now traveling out of the country to receive treatment. Meanwhile, Gilead has been blamed for igniting the problem due to its pricing practices.The problems began in 2014, when the NHS won a three-month extension to provide coverage for Sovaldi by arguing it needed more time to prepare for a large number of patients expected to seek treatment. The UK’s National Institute for Health and Care Excellence, which is the cost-effectiveness watchdog, had already recommended coverage. But the drug became available 10 months later than expected.advertisement @Pharmalot By Ed Silverman July 28, 2016 Reprints About the Author Reprints Ed Silverman Gilead responded by striking deals to sell the drugs at lower prices in some countries, such as Egypt, and for generic drug makers to manufacture and market versions in dozens of other nations. The medical journal noted, however, that the NHS was unable to negotiate with Gilead or adopt alternative funding mechanisms to lower the cost due to the constraints of NHS procurement laws.An NHS spokesperson told the BMJ the agency is “exploring the potential for a longer term strategic procurement for a supply agreement with the industry to improve the affordability of and access to treatment further.”In a separate editorial, the drug maker was blamed for setting prices too high for its medicines. We asked Gilead for comment and will pass along any reply that we receive.Meanwhile, the BMJ reported there is growing evidence that some frustrated patients are turning to overseas “buyers’ clubs” to purchase the drugs at their own expense.“It is truly ironic that NHS England should choose to start rationing drugs that are so effective they cure almost everyone who is treated,” The Hepatitis C Trust, a patient group in the UK, told the BMJ. The group is seeking a judicial review of the decision to ration the drugs, which the BMJ noted could have “repercussions for other patients as more new drugs increasingly become available at higher prices.”This post was updated to include a belated comment from the NHS. Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. PharmalotGilead hep C drug prices blamed for England’s health service rationing treatment Tags drug pricesGilead Scienceshepatitis Clast_img read more

Obama administration declares Zika public health emergency in Puerto Rico

first_img Puerto Rico is being pummeled by Zika, with hundreds of cases of birth defects feared HealthObama administration declares Zika public health emergency in Puerto Rico The emergency declaration will allow the Puerto Rican government to receive more federal aid. Mario Tama/Getty Images According to the Puerto Rico health department, there are now 10,690 confirmed cases of Zika on the island, including 1,035 pregnant women.The Puerto Rican government can now apply for federal funds to train people to help with mosquito control and public education to help people protect themselves from the spread of Zika. It can also reassign public health or agency workers to beef up the response teams.advertisement Related: WASHINGTON — The Obama administration declared a public health emergency in Puerto Rico late Friday over the Zika virus, saying the outbreak on the island has become serious enough to require the extraordinary step.The declaration by the US Department of Health and Human Services allows the Puerto Rican government to receive more federal funds and reassign local public health officials to step up the efforts to fight the virus.“This emergency declaration allows us to provide additional support to the Puerto Rican government and reminds us of the importance of pregnant women, women of childbearing age, and their partners taking additional steps to protect themselves and their families from Zika,” Health and Human Services Secretary Sylvia Burwell said in a statement.advertisement Puerto Rico has been hit hard by the virus, far harder than anywhere in the continental United States. On Thursday, Dr. Vivek Murthy, the US surgeon general, visited the island and warned that as much as 25 percent of residents could be infected with the virus by the end of the year.The move comes a day after HHS announced it will shift money around to free up an additional $81 million, including $34 million at the National Institutes of Health and $47 million at the Biomedical Advanced Research and Development Authority, to fight the virus. By David Nather Aug. 12, 2016 Reprints Tags Barack ObamaCongresspolicyZika Viruslast_img read more

Ohio sues drug makers for spreading the opioid crisis

first_imgPharmalot What is it? STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Ohio sues drug makers for spreading the opioid crisis You can add Ohio to the list of state and local governments that are filing lawsuits against drug makers for spreading the opioid epidemic by allegedly downplaying risks and improperly encouraging prescribing of the addictive painkillers.Attorney General Mike DeWine filed a lawsuit on Wednesday against five companies — Purdue Pharma, Johnson & Johnson’s Janssen unit, Teva Pharmaceuticals, Allergan, and Endo International — for false marketing that included misleading ads in medical journals and the use of physicians and front groups to boost prescriptions for their medicines. What’s included? Log In | Learn More Ed Silverman GET STARTED By Ed Silverman May 31, 2017 Reprintscenter_img Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. About the Author Reprints @Pharmalot Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Toby Talbot/AP Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. [email protected] Tags legalopioidspharmaceuticalsSTAT+last_img read more

Lawmakers urge Trump administration to enact price-gouging penalty

first_img Lawmakers urge Trump administration to enact price-gouging penalty @Pharmalot More than a dozen U.S. senators have asked the Trump administration to proceed “as soon as possible” with a rule that would punish drug makers for overcharging hospitals, clinics, and other providers for medicines purchased under the federal government’s 340B drug discount program.The request comes after the Trump administration recently delayed implementation until July 2018, a move that followed previous delays and prompted fresh criticism that the White House was kowtowing to the pharmaceutical industry. Andrew Harnik/AP By Ed Silverman Oct. 30, 2017 Reprints Ed Silverman [email protected] What is it? Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED What’s included?center_img Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Pharmalot About the Author Reprints STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Log In | Learn More GET STARTED Tags Donald Trumpdrug pricinghospitalspharmaceuticalspolicySTAT+last_img read more

‘Right-to-try’ law threatens patient safety and rational drug development

first_img Andrew Harnik/AP GET STARTED STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Politics By Mark Harrington and Ellen V. Sigal March 12, 2018 Reprints @CancerResrch In President Trump’s first State of the Union address, he challenged Congress to give people who are terminally ill immediate access to experimental treatments without going through the FDA. A number of states have already passed such “right-to-try” laws. The Senate has passed a federal version, and a revised version is now under consideration in the House, with a vote likely on Tuesday.Backers of the right-to-try law would have you believe that the FDA is barring the distribution of all sorts of miracle cures. But many experimental treatments turn out to be totally ineffective. And as it stands, the FDA approves more than 99 percent of the thousand-plus applications it gets each year — usually within days of receiving the application — for access to experimental therapies through an existing program called expanded access or compassionate use. Ellen V. Sigal What is it? Unlock this article — plus daily intelligence on Capitol Hill and the life sciences industry — by subscribing to STAT+. First 30 days free. GET STARTEDcenter_img About the Authors Reprints Log In | Learn More ‘Right-to-try’ law threatens patient safety and rational drug development Mark Harrington @TAGTeam_Tweets Tags Congresspharmaceuticalspolicy What’s included? Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.last_img read more

Long miles, lonely roads: In rural Texas, dying at home means little is easy

first_img Photos by Justin Rex for STAT Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. Please enter a valid email address. Hospice nurse Laura Brown checks Shawn Jordan’s heart in his mother’s house, in Haskell County, Texas. Please enter a valid email address. “I wanted to see everybody one last time, if I was going to pass away,” Jordan said about the moment he decided to go into hospice. “It was going to be in a familiar environment, in my environment. This was going to be a lot more comfortable.” Related: Brown works for Hendrick Hospice Care, affiliated with Hendrick Medical Center in Abilene. After seeing patients at the hospice facility in the city, she talked to Jordan by phone, grabbed supplies, including a colostomy bag, and got on the road.Brown gets supplies from Hendrick Hospice Care in Abilene, before heading out to see patients. As with many hospice nurses, her car is her office — a fuel-efficient, white four-door with a blue cooler for lunch and snacks, and plenty of space for supplies. On the roughly 50-minute drive to Jordan’s house, she took a phone call about another patient who needed help with medications. Such multitasking, she said, is fairly common.Hendrick serves people in 13 counties in a 65-mile radius from Abilene. The standard across the state, according to industry officials, is to be able to get to patients in about an hour.For their home cases, nurses see about four people per shift, said Hendrick director Karen Tucek. They are dispatched in a “spokes on a wheel” formation, moving in one direction and stopping along the way before heading back — Brown started her day 25 miles south of Abilene, then wended her way 50-plus miles north. Jodi Fry, another nurse working that day, headed east from Abilene, seeing patients in a loop that easily reached 100 miles.The roads get rough fast outside small cities like Abilene, and help, while a phone call away, may take a long time to get there.Tucek, like other providers, told stories of nurses and aides getting stuck on the road and needing a vehicle with four-wheel drive to dislodge them. She spoke of run-ins with deer. Nancy Kernell, a nurse and the owner of Area Community Hospice in Plainview, described night runs on dark Panhandle roads.“You drive down roads, you might hit a wild hog, it’s so dark,” she said. “Some people will flicker their lights out in the country so we can find them.” HealthLong miles, lonely roads: In rural Texas, dying at home means little is easy Leave this field empty if you’re human: Since the accident in 2013, he’s had to deal with open wounds that won’t heal, and antibiotic-resistant bacteria that repeatedly find their way inside him. He’d lost weight, and he needed a colostomy bag that would stick well to fragile skin.But what Brown brought wouldn’t do, and after some quick internet sleuthing, she left. First, she hit a pharmacy in town — it didn’t have the right style. Then, she went to a nursing home nearby. A little begging, a little borrowing, she recalled later, and she got the bag.She was back at Jordan’s in under an hour, but the whole trip took more than two hours, not to mention her next stops, and the drive home. She spoke highly of Jordan, and his will to live — and accept death — without regrets. One of his wounds has since started draining fluid — a fearful sign, he said this week, that the eighth infection might be underway.“He’s trying to take care of himself,” Brown said. “He’s probably outlived people who have had the same thing happen to them.”And Jordan is equally respectful of the nurses who come to see him.“You don’t realize you get that attached, but you do,” he said of the nurse who treated him most often in 2017. “As far as I’m concerned, she’s family.”Hospice nurses are often referred to as angels, because of when they enter people’s lives and the work that they do. But Brown bristled at the idea that she was an angel. Her work, she said, is a privilege.“You just get to be there, to hold their hand, and to love them,” she said, before a long pause. “I’m not really sure what an angel is supposed to feel like.” HASKELL COUNTY, Texas — To get to the house where Shawn Jordan wants to die, you drive a hypnotic road along miles of furrowed cotton fields, gnarly mesquite trees, low-to-the-ground cactus, and cattle perpetually in search of food.This iconic land of open spaces and oil pumps, where the Panhandle meets west Texas, is where the 43-year-old came home after a car accident he said should have killed him. He’s since had his paralyzed legs amputated, and survived seven MRSA infections.He’s been in hospice care since that last infection, because he knows the eighth could be his last. And he wants to spend his remaining time here, with family, surrounded by the rocking horse and toys of his toddler grandson, as well as emblems of the working west — antique spurs and ranch hooks on the wall, tin stars, and a saddle in a corner by the door. It’s his country. It’s home.advertisement Shawn Jordan Most hospices fare well in first public release of Medicare quality scores Reimagining hospice care — for the living Related: Privacy Policy “I wanted to see everybody one last time, if I was going to pass away. It was going to be in a familiar environment, in my environment. ” Kassner said missed calls and appointments, especially at the very end of life, should prompt a discussion of how hospice providers are paid by the Centers for Medicare and Medicaid Services. Medicare is the primary payment vehicle for hospice services and agencies are paid a day rate for patients, even if, as the Kaiser story showed, no one comes to the house. Kassner suggested that a two-tier system might work better, where on top of the flat rate, hospices would get paid extra for visits in those last few days.He and others also said that while not excusing outright neglect, the burden falls on providers that serve rural patients to set expectations as early on in the process as possible.“In rural areas, it’s a completely different setup,” Kassner said.Hendrick Hospice nurse Jodi Fry heads east from Abilene to see her first patient in Clyde, Texas. Hospice managers send staff to home visits in a “spokes on a wheel” fashion to minimize travel time. The search for the right colostomy bagBrown had to navigate from memory to find the gravel road that led to Jordan’s house; her GPS struggled with the address. Dodging toys and a new litter of kittens lounging in the shade of the front porch, she knocked on Jordan’s door. There was no answer. She called. Again, no answer.Concerned, she tried the door. It opened, and after a couple of tense minutes, she found Jordan in his bedroom, where he was trying to dress himself for the visit. Eventually, he wheeled into the living room, and the two began to talk.His eyes darted around the room, as he sipped watermelon lemonade and frequently shifted in his wheelchair. Occasionally, he’d make a joke. “At some point today, I’ll have to go get a Dr Pepper, because it’s the nectar of life,” he said, laughing hoarsely about the unofficial state beverage.Jordan shares his home with his mother, his son, his grandson, and his daughter-in-law, who, as his main caretaker, has had to learn things like how to correctly pick him up. His two other children live out of state. They were in the car with him when it flipped over a guardrail a few miles from his home. Newsletters Sign up for Morning Rounds Your daily dose of news in health and medicine. “We want the expectation to be set up front,” said Gina Councilman, executive director of Hospice of San Angelo, which serves 15 counties in west Texas that covers an area bigger than Massachusetts and Connecticut combined. “We’ll talk on the phone, but it will be a lag to get out there.”On a recent afternoon, a routine nursing visit to check on Jordan seemed straightforward — he needed a new colostomy bag. It turned out to be anything but.The once-paved road that leads to the house where Jordan lives in Haskell County, Texas, is flanked with mesquite, cotton, and wild grasses. A shortage of nurses, an excess of milesIt’s not easy to find people who will do hospice nursing in this part of Texas. The hours are irregular, and nurses are often on-call. A 250-mile day isn’t unheard of.For registered nurse Laura Brown, it’s what you have to do to care for sick people. Before joining hospice, she was an infusion nurse, giving cancer patients chemotherapy. She was used to patients dying as much as living.“I go wherever they send me,” she said, listing off small towns that rise up after miles of open land, just to disappear a minute later in a rearview mirror.Brown had not seen Jordan in a while — his regular nurse was on vacation, and she was filling in. He would be her second stop on a day that eventually covered 220 miles from home to patients and back again. Jordan’s wish is not unusual — most people who enter hospice want to die at home, even if they often don’t. But in rural Texas, where rugged individualism defines both the relationship to the land and the hesitation to ask for outside help, many don’t sign up for hospice until their very last days. And when they do, the long distances and often empty miles mean long hours for nurses, arduous trips to the hospital, and extra work for caregivers who have to learn basic medical care.Hospice providers in the more remote western and southern parts of the state shared stories with STAT of nursing shortages, scheduling gymnastics, run-ins with wildlife, and the wear and tear of long days in cars for the men and women who treat people at home. Distance sometimes forces hard conversations: At the moment they and their caregivers need help most, a nurse may not quickly be at their side.advertisement By Megha Satyanarayana March 15, 2018 Reprints Privacy Policy Leave this field empty if you’re human: Kernell said she’s been looking for a nurse for two months, and there are times when her hospice is short-staffed. When that happens, she adds herself to the call schedule.“We don’t ever go down on care because of nurses,” she said. “We just work longer hours.”Councilman, of Hospice of San Angelo, said to better cover her sprawling service area, she tries to find nurses who live outside the city. Still, there are gaps, so her organization uses telemedicine.“We are constantly on the phone with our patients, triaging, giving medication suggestions, while we are on the way out there,” she said.The living room of Jordan’s home. He began living there with his mother after an accident that eventually cost him his legs. Before his wreck, he helped build out the living room with one of his sons. Jordan waits for his hospice nurse to return with a colostomy bag. He’s been fighting MRSA infections since the 2013 accident and fears the next one will be his last. Hospice isn’t available to everyone who might need itTexas has nearly 500 hospice providers, but 60 percent of its counties do not have a single provider, according to 2015 data — the latest available — compiled by Cordt Kassner, CEO of  Hospice Analytics. Most of those counties are classified as rural by the U.S. Department of Agriculture, but even many of the counties classified as urban are really not — they are one county over from a large town or small city, and the distances are still significant.Use of hospice by eligible patients is only slightly less in rural counties than in urban ones, but Kassner’s data make clear that rural residents enter hospice much later than those living in or near cities: The average length of stay for rural residents is 63 days. For urban residents, it’s 81.Overall, about half the people who are eligible for hospice services in the state actually use them, which is slightly higher than the national average of 46 percent.“Texas, as a whole, in my opinion, has many more hospices than are needed,” said Kassner. There are 254 counties in the state of Texas, and the largest five, by population, have more than 40 percent of the state’s providers. “The distribution of those hospices is not necessarily appropriate.”This is one reason why individual hospices treat people in so many counties, said Larry Farrow, the executive director of the Texas & New Mexico Hospice Organization.“It’s hard. I wish I could say that no matter where you reside in Texas, you have a hospice within 50 miles of you, but it’s not true, and it may never be true,” he said.The lack of nurses and huge areas served by Texas hospices raise possible concerns about the quality of care that people who want to die at home receive. Kaiser Health News recently reported on egregious oversights in hospice care throughout the nation, describing cases that happened in rural parts of the country. In some cases, families called for help at odd hours, and no one came. In another complaint, a provider who showed up was inexperienced, and her calls for backup went unanswered.Hospice experts told STAT that while such situations happen, it’s hard to know how often. In general, most patients and family members who contribute comments to Medicare’s Hospice Compare website seem to be pleased with the quality of care they receive. Tags end of lifenursingpatientslast_img read more

Supreme Court weighs whether to review a whistleblower case against Gilead

first_img Supreme Court weighs whether to review a whistleblower case against Gilead @Pharmalot Log In | Learn More Pharmalot Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED About the Author Reprints What’s included? Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. What is it? Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. By Ed Silverman April 18, 2018 Reprints [email protected] In a case fraught with implications for the pharmaceutical industry, the U.S. Supreme Court has asked the solicitor general for its views on a lawsuit involving Gilead Sciences (GILD) and what constitutes a material representation by companies that bill the federal government.The request came in a long-running whistleblower suit that accused the company of misleading regulators about contaminated ingredients used in various HIV medicines and falsifying data to win marketing approval for the drugs. Ed Silverman GET STARTED Tags infectious diseaselegalpharmaceuticalspolicySTAT+last_img read more

Vertex and U.K. health officials reach an impasse over pricey cystic fibrosis drug

first_img What’s included? Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED @Pharmalot Log In | Learn More A long-running effort to provide coverage in the U.K. for a cystic fibrosis drug has reached an impasse as health officials maintain the price offered by Vertex Pharmaceuticals (VRTX) is “unsupportable,” while the company called their position “outrageous” and “unconscionable.”At issue is the Orkambi treatment, which was determined not to be cost effective by the U.K.’s National Institute for Health and Care Excellence. As a result, the National Health Service in England is refusing to provide coverage unless Vertex lowers the price. About 10,400 people suffer from cystic fibrosis in the U.K., and more than 116,000 people have signed an online petition demanding NHS coverage. Craig F. Walker/The Boston Globe Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Tags drug pricingpharmaceuticalsSTAT+center_img GET STARTED By Ed Silverman July 5, 2018 Reprints Pharmalot About the Author Reprints What is it? [email protected] Ed Silverman Vertex and U.K. health officials reach an impasse over pricey cystic fibrosis drug last_img read more

Startup Spotlight: This biotech is attracting attention, even without a broad portfolio

first_img Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Kate Sheridan STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. By Kate Sheridan Nov. 15, 2018 Reprints Biotech About the Author Reprints What is it? What’s included? General Assignment Reporter Kate covers biotech startups and the venture capital firms that back them. Cadent Therapeutics — a Cambridge, Mass.-based startup with a complicated history — announced Thursday that it has raised about $40 million for its neuroscience programs in a series B round backed by Atlas Venture, Cowen Healthcare Investments, and Novartis Institutes for BioMedical Research, among others.Among biotech startups, Cadent is bucking a trend. Lately, entrepreneurs have gravitated toward working on platforms instead of individual potential drugs. In theory, that strategy lessens some of the risk inherent in early-stage biotechs. If a company is based around one particular drug and that drug fails, that could blow up a company’s entire future.center_img Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Startup Spotlight: This biotech is attracting attention, even without a broad portfolio Log In | Learn More [email protected] Alex Hogan/STAT GET STARTED @sheridan_kate Tags biotechnologyBostonneuroscienceSTAT+last_img read more

What’s holding pharma back from the next antibiotic breakthrough?

first_img [email protected] By Peter Bak Dec. 10, 2018 Reprints Cost pressure is one formidable obstacle. Any new antibiotic will face substantial pressure from competitors, as most antibiotic classes have now lost market exclusivity and relatively affordable generic options are available. Related: Peter Bak Related: Tags antibioticsdrug developmentfinanceinfectious diseaseresearch The O157:H7 strain of the E. coli bacteria Janice Carr/CDC via AP These models require the coordination and alignments of governments, hospitals, insurers, and others — a daunting task. As such, they have been mostly academic exercises.FDA Commissioner Scott Gottlieb announced serious consideration for reforming reimbursement for antibiotics. What transpires from that could be the watershed moment. But even if it is, any action remains years in the future.Questions remainWith a clear and present danger of the growing need for new antibiotics, questions abound:When will big players re-enter the licensing or merger and acquisition space, if at all? Following the launch of antibiotics targeting complicated urinary tract infections (e.g. Achaogen’s Zemdri and Melinta’s Vabomere), many in the industry are waiting to see if the market will sustain them. The clinical uptake and commercial success of these brands could be the data point that larger companies are waiting to access.Who will become the market leaders, given the lack of interest by big pharmaceutical companies? Smaller companies are currently the dominant sponsors of novel and recently launched antibiotics. As larger companies have divested their anti-infective assets, their portfolios have found their way into public corporations, such as Melinta’s acquisition of The Medicines portfolio and AstraZeneca spinning out its portfolio to Entasis, which recently closed a $75 million IPO (after lowering its offered share price). The market, though, has been skeptical: Despite Achaogen’s launch of Zemdri, the company slimmed payrolls to focus on commercialization activities and essentially put out the “for sale” sign.Will enabling technologies, such as rapid diagnostics, help get novel antibiotics more quickly and efficiently to the patients who need them? While rapid, sensitive, and specific diagnostics have long been a holy grail, the field is still waiting for them to appear. Structures to spur innovation in this space have popped up, like CARB-X eligibility for diagnostic companies and the Longitude Prize for the development of an inexpensive, sensitive, and rapid point-of-care test for bacteria sensitivity. Such advances will enable hospital systems to confidently use costly novel antibiotics for the patients who most need them without the fear of unnecessarily speeding the development of antibiotic resistance.The next few years will prove critical for organizations developing novel antibiotics. Commercial successes could be the tipping point that pulls together stakeholders to reinvigorate the commercial model for anti-infectives.But as we have seen with other efforts to reorganize health care delivery in the U.S., that could prove as difficult as eradicating a superbug.Peter Bak, Ph.D., is vice president for life science strategy at Back Bay Life Science Advisors. Related: About the Author Reprints In the five years since the alarm sounded about so-called superbugs, the world has continued to grapple with the staggering health and economic impacts of antibiotic resistance and mounting resistance to antibiotics of last resort.In the U.S. alone, antibiotic resistance adds $20 billion to $35 billion in direct health care costs each year, along with 8 million extra days in the hospital. In the European Union, multidrug-resistant infections kill more than 30,000 people every year. While scientists work to uncover new ways to combat antibiotic-resistant microbes — which harbor a dizzying array of tools to evade antibiotics — the question of how to best develop and commercialize novel antibiotics while at the same time creating a return for investors remains just as puzzling as the most recalcitrant bacteria.Any company considering investing in novel anti-infectives faces substantial commercial headwinds despite a clear and urgent need for them.advertisement The population of potential recipients is another impediment. A pharmaceutical company developing an antibiotic for a multidrug-resistant bacterial strain targets a relatively small pool of infections. For example, a study that assessed bloodstream infections between 2009 and 2013 revealed that only 1 percent of bacteria were resistant to the most commonly used antibiotics. It’s worth noting, however, that the report also demonstrates the medical need within patients unlucky enough to be infected by that 1 percent of bacteria. These resistant strains were associated with a 40 percent increase in the risk of dying and more than doubled the average hospital stay.Creating an investment strategy around this relatively small patient population is further confounded by the increasing role of antibiotic stewardship committees. They develop guidelines to make sure that specific classes of antibiotics, particularly the often-expensive novel classes, are used only for appropriate infections as a way to prevent further development of antibiotic resistance. As a result, novel antibiotics targeting multidrug-resistant microbe strains are often deployed only after extensive laboratory tests have confirmed that a bad actor is present — at which point it may be too late for the patient.Although these hurdles may hold back any investor considering an anti-infective opportunity, several incentives and economic models have been proposed to reduce clinical development time and costs for anti-infectives, and ensure a commercially viable payment and reimbursement structure. These concepts are generally known as “push” and “pull” incentives. Watch as a superbug is bornVolume 90%Press shift question mark to access a list of keyboard shortcutsKeyboard ShortcutsEnabledDisabledPlay/PauseSPACEIncrease Volume↑Decrease Volume↓Seek Forward→Seek Backward←Captions On/OffcFullscreen/Exit FullscreenfMute/UnmutemSeek %0-9 facebook twitter Email Linkhttps://www.statnews.com/2018/12/10/holding-pharma-back-from-next-antibiotic-breakthrough/?jwsource=clCopied EmbedCopiedLive00:0002:2502:25  The superbugs are growing in strength and it’s our fault. Hyacinth Empinado/STAT Pushing anti-infectives through clinical developmentA variety of mechanisms are currently in place to help push an anti-infective through the clinical development process.From a regulatory perspective, the Food and Drug Administration and the European Medicines Agency have done their part devising programs that help pharmaceutical companies quickly and efficiently shepherd their anti-infective drugs through clinical trials.As part of the 2012 Generating Antibiotic Incentives Now (GAIN) provision, the FDA created the designation of qualified infectious disease product. It includes several incentives for antibiotics that meet certain criteria, including eligibility for priority review, fast track designation, and a five-year extension of exclusivity. Not to be outdone, the EMA is exploring “adaptive pathways” for high-need areas in which current care is unsatisfactory (including infections), granting marketing approval based on targeted studies in circumscribed patient populations with continued post-marketing data gathering.Both the FDA and EMA have been flexible about the totality of evidence required for approval. For most drugs, companies are required to conduct large, randomized, controlled Phase 3 trials. For some anti-infectives, recent approvals were based on one randomized, controlled Phase 3 trial and a small open-label, single-arm study in a population of high unmet need.While programs like these may grease the wheels of clinical development, it is still up to the sponsor to find money to pay for these efforts. As a result, public-private partnerships have emerged to help push anti-infective therapies onto the market. One is CARB-X, a nonprofit organization funded in part by the National Institutes of Health, the Bill and Melinda Gates Foundation, and the Wellcome Trust, among others. With a target investment budget of $500 million, the organization aims to make non-dilutive investments in antibiotics to prevent and treat life-threatening bacterial infections. So far, CARB-X has deployed $91 million of funding across 17 programs for antibiotics. First OpinionWhat’s holding pharma back from the next antibiotic breakthrough? Novartis is the latest big drug maker to exit antibiotic research Innovative ways to pay for new antibiotics will help fight superbugs A case study in the fast-rising threat of antibiotic resistance How the treatment of infections is paid for is another obstacle. This treatment is often covered by a diagnosis related group, under which a hospital is paid a lump sum for an episode of patient care. That leaves the hospital on the hook for any additional costs, including pharmaceuticals like antibiotics, that exceed the prespecified payment rate. The upshot is that institutions have an incentive to use the least costly option available — not necessarily the best in class.advertisement Not to be left out, traditional VCs have also entered the game. Novo Holdings launched the REPAIR Impact Fund in 2018, with a budget of $165 million to invest in companies focused on treating drug-resistant organisms. These efforts have paid off, as there has been a spate of company formation and venture capital investment around developing novel anti-infectives.As highlighted by a recent CARB-X report, non-dilutive funding may beget further investment. As of the third quarter of 2018, 15 companies in the CARB-X portfolio had raised $762 million in private funds since their awards were announced. Beyond these examples, an estimated $800 million in venture capital funding for anti-infective companies was spent between 2016 and 2017, according to an analysis conducted by my company, Back Bay Life Science Advisors (the report is available by request).This spark of interest in drug development is encouraging, but large pharma is hardly fanning the flames. Several commercial-stage companies, including AstraZeneca, The Medicines Company, and Novartis, have shuttered their antibiotic operations. Companies that still have anti-infective franchises remain on the sidelines of the merger and acquisitions game — compared to other therapeutic areas, there has been a dearth of anti-infective licensing and merger and acquisition deals compared with the previous five years. While there are multiple reasons for this, one is that the economic model for developing novel antibiotics to treat resistant bugs has yet to be clearly defined.Pull incentives aren’t yet robustWhat pull incentives might look like in the future remains cloudy, though some groundwork has been established. For instance, the qualified infectious disease product designation mentioned earlier offers an extra five years of exclusivity during which sponsors can market their drugs without threat of generic competition. While this extends the runway to generics, it does little to address the current reimbursement landscape, and so may do little to lure investors toward anti-infective development.Some existing mechanisms are in place to provide additional reimbursement for novel anti-infectives, such as Medicare’s new technology add-on payment program. This offers an additional payment for the use novel in-patient products that are an improvement over the current clinical standard of care and are otherwise cost prohibitive under normal reimbursement structures. Although these payments have been granted to novel antibiotics, they have done little to increase their uptake and utilization.Innovative economic models have been suggested, ranging from a subscription-based model, under which hospitals or governments pay sponsors for licenses to access antibiotics, to a framework in which a drug developer is granted a minimum level of annual revenue for bringing an anti-infective through FDA approval.last_img read more