Law for rare disease drugs needs revamping, researchers say

first_img By Ed Silverman Nov. 30, 2015 Reprints Makary: We found that an increasing number of drugs approved by the FDA have been orphan drugs. Last year, seven out of 10 blockbuster drugs in the US were passed as orphan drugs, even though they’re used off-label for other conditions. There’s a time when, as physicians, we believe there’s a benefit to patients for an off-label use, and we should be able to give them the drug. The problem isn’t the off-label use. The problem is that some of these drugs may actually have been submitted [for FDA approval] with the ultimate intention for being used more broadly than if they were designed only for the rare disease populations. There’s a pattern of gaming the system to cash in on a taxpayer-funded benefit to the company, and it needs to be addressed in order to preserve the mission of the Act.Pharmalot: How do you mean ‘gaming the system?’Makary: So a rare disease population is defined as affecting less than 200,000 people. But what’s happening is that a drug company develops a medication to treat a specific cancer, and will submit that to the FDA as intended for a very targeted subset of the cancer population. For example, HER2-positive is most commonly associated with breast cancer, but is also found in other cancers. … Once a drug is approved, companies can seek additional indications for populations well exceeding 200,000 people. And this is a pattern with most of the orphan drugs approved by the FDA.Pharmalot: OK, but isn’t that a good thing if more patient populations are ultimately served?Makary: Any time a drug is approved by FDA to help patients, it should be celebrated. But if we create a special mechanism for a drug that ends up being more commonly used after approval, we believe there will be less research conducted for drugs for true orphan diseases. Companies will direct their efforts to wider populations. As a cancer doctor, there are patients that were being helped by the Act, but they may not be in the future.We think this law is being abused, because it suggests taxpayer-funded benefits are no longer truly concentrated on orphan diseases. These subsidies are provided by taxpayer dollars. Should taxpayers subsidize what may ultimately become blockbuster drugs? This is an especially important question, given the climate in America with outsized pricing. The median cost for orphan drugs is $98,534 per patient per year compared with a median cost of $5,153 per patient per year for drugs that are not approved for an orphan population. And the median cost of orphan drugs doubled between 2010 and 2014.Pharmalot: So what do you suggest?Makary: Drug companies should do what’s done in other countries, which is they should pay back the subsidies if their drug becomes a blockbuster. Perhaps a 1 percent tax if a drug grosses $1 billion a year. That seems to be a reasonable way to recoup the taxpayer-funded incentive, since those incentives were never intended for blockbusters. Or maybe if a drug is a $5 billion, the marketing exclusivity is shortened or the company pays a graduated tax.Right now, the public is frustrated with drug pricing. This is a good time to ask if we think the most vulnerable members of our society deserve products developed with some incentives for research and development. As a country, we’ve said the answer is ‘Yes,’ but we believe the mechanism should be re-evaluated.We’re the only country where our program to incentivize rare disease drug development has been abused to the point where 44 percent of all drugs approved, and seven out of 10 blockbuster drugs, are orphans. We hear about scientists asking how they can tailor a drug to gain an orphan indication. We shouldn’t have scientists talk about gaming systems. We want them talking about developing medications. Martin Makary, a cancer surgeon and professor of health policy at Johns Hopkins School of Medicine, is calling for reform to the Orphan Drug Act. Micah Kandros/Johns Hopkins School of Medicine In 1983, the Orphan Drug Act was passed to give drug makers incentives to create medicines for rare diseases, which are defined as maladies that affect fewer than 200,000 people. The incentives include tax credits and seven years of marketing exclusivity. Since then, more than 400 orphan drugs have been approved by the Food and Drug Administration. Last year, though, 41 percent of all FDA approvals were for orphan drugs. And sales of orphan medicines, which carry high price tags, are forecast this year to total $107 billion.In a new paper in the American Journal of Clinical Oncology, a team of researchers argues that drug makers are exploiting loopholes that allow them to widen the market for such drugs and distorting the original purpose of the law. We spoke with Martin Makary, a cancer surgeon and professor of health policy at Johns Hopkins School of Medicine, who is one of the authors, about the need for reform. This is an edited version of the conversation …Pharmalot: What’s the problem with the Orphan Drug Act?advertisement [email protected] Ed Silverman @Pharmalot center_img About the Author Reprints PharmalotLaw for rare disease drugs needs revamping, researchers say Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Makary: The dilemma is this: Pharmaceutical companies have invented and discovered cures for terrible medical conditions, which is wonderful, because generally, the research goes where the largest markets are. The Orphan Drug Act was passed in response to concerns that people with rare diseases were not getting any attention in the drug development community. It was intended to balance out this need with financial awards for companies that normally develop drugs that affect a large population. If we didn’t have the law, there’d be little incentive to develop drugs for rare conditions. Yet at the same time, there are unintended consequences, and this includes creating perverse incentives.Pharmalot: What do you mean by that, exactly?advertisement Tags FDAOrphan Drug Actrare diseaseslast_img read more

6 questions for Rep. Tom Price, Trump’s nominee to lead HHS

first_img Log In | Learn More GET STARTED 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. 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. WASHINGTON — It’s called a “courtesy hearing,” but Representative Tom Price, President-elect Donald Trump’s nominee for secretary of health and human services, can expect some of his Senate interrogators to get a little rough.Price, an orthopedic surgeon and six-term GOP congressman from Georgia, will appear before the Senate Committee on Health, Education, Labor, and Pensions to answer questions on Wednesday. It’s a courtesy hearing because a separate panel, the Senate Finance Committee, will actually vote on his nomination. Tags CongressMedicarepolicy Politics What’s included? By Sheila Kaplan Jan. 17, 2017 Reprints What is it? Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Charles Dharapak/AP 6 questions for Rep. Tom Price, Trump’s nominee to lead HHS last_img read more

Pharmalot, Pharmalittle: AstraZeneca hired a fake doctor Down Under

first_img Tags drug pricingpharmaceuticalsSTAT+White House [email protected] By Ed Silverman March 9, 2017 Reprints 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. Hello, everyone, and how are you today? We are doing just fine, thank you, with help from a delightfully shiny sun enveloping the Pharmalot campus and a few needed cups of stimulation. Our flavor today is Cinnamon Dolce, for those keeping track. Meanwhile, here is the usual lineup of tidbits. We should note, by the way, that we will be taking an extended weekend and colleagues will fill in for us. On that note, have a wonderful few days and see you soon …AstraZeneca employed a man in Australia who allegedly falsified his credentials and posed as a doctor who was from India, ABC reports. Shyyam Acharya gained registration with the Medical Board of New South Wales in 2003 and, later, worked for the drug maker for 11 months from 2013 to 2014. The drug makers says that “his stated overseas medical qualification was not a core requirement of the role therefore was not investigated at the time.” GET STARTED What’s included? @Pharmalot Alex Hogan/STAT Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry.center_img About the Author Reprints Pharmalot Log In | Learn More 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. Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED What is it? Ed Silverman Pharmalot, Pharmalittle: AstraZeneca hired a fake doctor Down Under last_img read more

US drug prices are so high that Canada wants other countries as reference points

first_imgPharmalot By Ed Silverman May 22, 2017 Reprints How high are drug prices in the US? So high that the Canadian government may remove the US from its long-standing list of countries that are used as a guide for determining whether prices are excessive.In a proposal issued last week, Health Canada said it wants to overhaul the framework used by the Patented Medicine Prices Review Board, which assesses therapeutic benefits and sets ceiling prices. Right now, this is accomplished, in part, by benchmarking prices against what drug makers currently charge in seven other countries — the US, France, Germany, Italy, Sweden, Switzerland, and the UK. Log In | Learn More Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED 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. Ed Silverman About the Author Reprints What’s included? What is it?center_img GET STARTED @Pharmalot 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. Joe Raedle/Getty Images Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. [email protected] US drug prices are so high that Canada wants other countries as reference points Tags drug pricingpharmaceuticalsSTAT+last_img read more

Ohio sheriff won’t let deputies carry Narcan, citing safety

first_img Newsletters Sign up for Morning Rounds Your daily dose of news in health and medicine. Associated Press About the Author Reprints Middletown’s city manager responded with a statement that the city continues to respond to every call.Ohio is among the states hit hardest by the opioid crisis. Butler County, near Cincinnati, had a record 192 drug overdose deaths last year. The county coroner has said it is on pace for other record year in 2017.Jones, an early supporter of Donald Trump’s Republican presidential campaign, has gotten national attention before on his tough talk on illegal immigration. He has written to the Mexican government calling for reimbursement for housing in the county jails immigrants in the U.S. illegally. County emergency crews administer Narcan, and the Butler County Health Department has been offering free naloxone kits to relatives and friends of addicts. HAMILTON, Ohio — A sheriff in an Ohio county with record numbers of overdose deaths in recent years is sticking to his long-standing refusal to allow deputies to carry an overdose-reversal drug.Butler County Sheriff Richard Jones says he remains opposed for safety reasons because people can become hostile and violent after being revived with Narcan. Deputies in neighboring counties in southwest Ohio do carry it.“I don’t do Narcan,” Jones told the Cincinnati Enquirer. “They never carried it. Nor will they. That’s my stance.”advertisement Please enter a valid email address. By Associated Press July 7, 2017 Reprints Privacy Policy HealthOhio sheriff won’t let deputies carry Narcan, citing safety Leave this field empty if you’re human: Jones’s latest comments came after a city councilman in Butler County’s Middletown drew national attention with his suggestion that emergency crews should stop responding to people who repeatedly overdose. Councilman Dan Picard also suggested that people who overdose should be forced to perform community service to make up for the cost of an emergency run.advertisement A naloxone nasal injector is demonstrated during a news conference in Cincinnati. John Minchillo/AP Tags addictionopioidslast_img read more

Massachusetts AG launches probe of addiction treatment fraud

first_imgOne focus of the investigation appears to be the way in which insurance benefits were obtained for the patients sent out of state, according to people familiar with the matter.Peter SanAngelo, of Malden, Mass., died of a carfentanil overdose last fall in Delray Beach, Fla., where he was allegedly brokered for treatment using a Capital Blue Cross insurance plan purchased with a phony Pennsylvania address. He was 33.Peter SanAngelo FacebookHis cousin Samantha Herring said she has been contacted by Massachusetts investigators.“With the anniversary of Peter’s death approaching, it is of course my greatest hope that the people who were involved in this are held accountable,” she said.SanAngelo had been homeless and jobless in July 2016 when he was offered free insurance and luxury rehab that he hoped would rid him of his decade-long dependence on heroin. A sober life with his 4-year-old son suddenly seemed possible. He jumped at the opportunity.Text messages on his phone, obtained by the Globe and STAT through Herring, laid out the scheme that brought him south. A broker signed him up for insurance using the address of a Pennsylvania sober house where he had never been, and then someone else bought him a plane ticket. Shortly after he got to the treatment center, however, his Blue Cross policy was canceled for nonpayment. He got a job managing a Florida sober house, and relapsed.On the morning of Oct. 16, 2016, police found his body slumped in the driver’s seat of a van that belonged to the sober house where he worked. He was alone, with a needle in his pocket. HealthMassachusetts AG launches probe of addiction treatment fraud Massachusetts Attorney General Maura Healey, whose office is investigating drug treatment fraud. Jessica Rinaldi/The Boston Globe The Massachusetts attorney general’s office is investigating a far-reaching insurance scheme that recruited drug users and sent them to treatment centers in other states to exploit their benefit payments, according to people contacted by the office and others familiar with the matter.Jillian Fennimore, a spokeswoman for Attorney General Maura Healey, confirmed the office is conducting a criminal investigation of addiction treatment scams, which have proliferated amid the national opioid addiction crisis. She would not provide details of the probe, including whether particular entities or individuals were being targeted.The investigation follows reporting by STAT and the Boston Globe on a national network of insurance fraud that preyed upon people desperate to break their addiction to opioids.advertisement Related: Related: The addict brokers: Middlemen profit as desperate patients are ‘treated like paychecks’ Desperate for addiction treatment, patients are pawns in lucrative insurance fraud scheme Investigators have also contacted the family of Patrick Graney, according to a person with direct knowledge of the communications.Graney spent about two months in south Florida in the summer of 2016 after an alleged broker offered him free insurance and a free plane ticket.The 30-year-old from Milton, Mass., who had first tried OxyContin at a high school dance and later graduated to heroin, relapsed just a few weeks after he arrived. He left treatment to sleep on the beach for a period before returning to the facility.But by late August, the insurance plan purchased for him had been canceled. By early September, he was back out on the street.Graney’s mother was trying desperately to get him home, and made plans to buy him a bus ticket.Graney didn’t make it. He checked into a hotel room on Sept. 9 with two strangers he had met during a failed attempt to get into a detox center. Just after 2 a.m. on Sept. 10, he overdosed on cocaine and died. The Greyhound he was supposed to take to Boston left 10 hours later.Patrick Graney (center) with his parents, Maureen and Jack. Graney familyIn a statement, Healey urged anyone victimized in a drug treatment scam to call her office’s Health Care Helpline at 888-830-6277 or file a complaint online at www.eform.ago.state.ma.us.“It is critical that people struggling with addiction can safely access treatment services,” Healey said. “Unfortunately, those seeking to make a profit off of this epidemic are targeting vulnerable patients with illegal treatment and recovery scams.”The attorney general’s office also indicated it is taking a broader look at sober home operators in Massachusetts. Sober homes offer group living for people in recovery from drug addiction. Healey’s office said it was looking into allegations of poor living conditions in some sober homes, false advertising, and the failure of some operators to maintain a sober environment. The families of two Massachusetts men who died in Florida after allegedly being sent there for treatment by patient brokers are among those who have been contacted by investigators in the attorney general’s office. In both cases — detailed by STAT and the Globe in May and July — brokers or their associates allegedly helped the men obtain private insurance benefits and paid for them to fly to Florida for treatment.advertisement Tags addictionlegalopioidsstates [email protected] About the Authors Reprints @evanmallen By David Armstrong and Evan Allen — Boston Globe Sept. 29, 2017 Reprints Many were sent to treatment centers hundreds of miles from home for expensive and often shoddy care paid for by insurance benefits obtained by using fake addresses. Patient brokers are often paid as “marketers” by treatment centers to sign up patients with private insurance plans. Evan Allen — Boston Globelast_img read more

A scrappy crew of biotechs is working on a new wave of schizophrenia medicines

first_img [email protected] About the Author Reprints By Meghana Keshavan Aug. 17, 2018 Reprints Biotech Correspondent Meghana covers biotech and contributes to The Readout newsletter. Tags biotechnologySTAT+ Biotech @megkesh Adobe What is it? Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTEDcenter_img GET STARTED Log In | Learn More 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. A scrappy crew of biotechs is working on a new wave of schizophrenia medicines Meghana Keshavan 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. What’s included? Schizophrenia is a devastating, all-too-common neuropsychiatric illness — and it’s long bedeviled scientists.It’s one thing to keep the hallucinations in check: Decades-old antipsychotics will generally do the trick. But there is no treatment for other debilitating symptoms of the disease: listlessness, memory loss, a general inability to find any joy in life.last_img read more

ER patients given ketamine, other powerful drugs in clinical trials without their consent, FDA finds

first_imgHospitalsER patients given ketamine, other powerful drugs in clinical trials without their consent, FDA finds By Sharon Begley Oct. 29, 2018 Reprints [email protected] African-Americans are disproportionately enrolled in studies that don’t require informed consent A hospital spokesperson said the findings by inspectors from the FDA “are neither formal nor conclusory.” Hennepin “has provided formal written responses to each” of the inspectors’ findings, she said, adding that the hospital “has undertaken several reviews by independent experts to evaluate its research and emergency medical practices with the intent to improve our processes. …We understand our mission includes providing the highest standards of medical care, and also conducting studies that help to continuously improve the health and wellness of our patients.” Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. About the Author Reprints Among other violations identified by FDA inspectors, the IRB ruled that researchers did not need consent to make patients part of a clinical trial in which they were given antipsychotic drugs that they might not receive as part of usual care. The Public Citizen letter said the IRB, which is legally obligated to protect research participants, “appears to lack even a basic understanding of federal regulations for the protection of human subjects and is clearly incapable of fulfilling its obligation” to do so.advertisement Senior Writer, Science and Discovery (1956-2021) Sharon covered science and discovery. Privacy Policy Leave this field empty if you’re human: Just as concerning, Turner said, it’s not clear that the patients were in fact too agitated to be asked for their consent, as the scientists argued to the IRB. According to the evaluation form’s scoring, “it’s not like they had acute psychosis,” Turner said. “These investigators were basically drugging up individuals with a degree of agitation many of us could experience, like after being pulled over for speeding.”In the first study cited by the FDA inspectors, researchers injected either ketamine or haloperidol into people taken to the ER, to reduce their “agitation.” Ketamine is not approved by the FDA for that use. The unwitting participants were treated not according to clinicians’ best judgment but according to the study’s protocol: Those arriving during certain months got ketamine and those arriving in other months got haloperidol.Among the trial’s results was that some patients given ketamine suffered breathing problems; 39 percent required intubation, compared to 4 percent given haloperidol. Before the trial began, its leaders had warned in a 2013 paper that ketamine can impair breathing and so should be reserved for only the most severely agitated patients.The identities of five of the seven studies the FDA report flagged are redacted, but one corresponds to the second ketamine clinical trial questioned in Public Citizen’s letter. It compared ketamine to midazolam, also in “agitated” ER patients given one or the other drug essentially randomly, not according to which — if any — might help them. The study was suspended by the medical center in June, for unclear reasons, but it also failed to obtain informed consent from participants, the FDA inspectors found.A third clinical trial compared the safety and efficacy of four drugs (the antipsychotics olanzapine, haloperidol, and ziprasidone and the sedative midazolam) in agitated patients. The hospital scientists had initially requested an FDA waiver from the informed consent requirement. FDA denied the request. The scientists then requested IRB approval, calling their study observational (meaning patients would be treated according to their doctors’ best judgment rather than receiving drugs as per the trial protocol) and therefore entitled to “expedited review.” The IRB agreed, including giving the okay to forgo consent. A Minneapolis hospital tested powerful antipsychotics and the potent anesthetic ketamine on emergency room patients without their knowledge or consent, violating regulations on human research, federal inspectors have determined.Based on those findings, a health watchdog group on Monday urged federal regulators to suspend all clinical trials at the hospital. In a letter to the Food and Drug Administration and the Department of Health and Human Services office that protects human research subjects, Public Citizen also called for regulators to immediately launch an investigation into the conduct and oversight of the studies and “impose severe sanctions for the serious ethical and regulatory lapses that have occurred in the ketamine clinical trials and other studies” at Minneapolis’s Hennepin County Medical Center.The hospital committee that green-lighted the studies, called an institutional review board (IRB), “appears incapable of doing its job,” said Dr. Michael Carome, director of Public Citizen’s Health Research Group, who organized the letter. It acted unethically and placed patients in danger, he said, “including by waiving the requirement for informed consent in situations where that is not allowed.”advertisementcenter_img Trending Now: Related: In fact the study was not observational: Patients received one of the four drugs based on when they arrived in the ER. They were not informed of this, and so were unwitting participants, the FDA inspectors found.“When the investigators didn’t get FDA clearance, they did an end-run around the agency,” Turner said. “They claimed it was no longer the randomized controlled trial they’d planned, and then pushed it through the IRB as a waiver-of-consent study,” a strategy he called “dodgy.”The FDA inspectors found that “some or all of the subjects [in four studies] were likely to be vulnerable to coercion or undue influence,” yet the IRB did not require the researchers to do anything to safeguard the participants’ safety.In addition to asking HHS to suspend all clinical trials at Hennepin unless doing so would harm participants, Public Citizen called it “imperative” that the hospital disband its current IRB and train a new one, retrain researchers involved in human research, and inform the unwitting participants in past trials of steps “to redress these violations.” Adobe Tags pharmaceuticalsresearch Comparing the Covid-19 vaccines developed by Pfizer, Moderna, and Johnson & Johnson In July, Public Citizen and 64 bioethicists, physicians, and other scholars submitted a complaint about two of Hennepin’s ketamine studies to the FDA and HHS’s Office of Human Research Protections. In August, FDA sent inspectors to the hospital.Their report, obtained by Public Citizen through a public records request and shared with STAT, examined additional clinical trials beyond those initially flagged. It found that in four, the hospital IRB “did not determine that informed consent would be sought from each prospective subject” as required by law, while in another five, the IRB granted fast-track review to studies that didn’t qualify for it.At least three of the studies cited by the FDA inspectors involved people brought to the emergency room with “severe” agitation, as assessed by emergency technicians using criteria developed by the researchers. The study leaders apparently persuaded the IRB that such patients could not provide informed consent, and so could be swept into the trial unknowingly.In fact, such patients are considered “vulnerable,” said bioethicist Leigh Turner of the University of Minnesota, who signed the Public Citizen letter. According to federal law, they are supposed to receive special safeguards, such as having a family member or other representative give or decline consent. That did not happen. Sharon Begley @sxbegle Please enter a valid email address.last_img read more

One of India’s biggest drug makers has a ‘deeply troubling’ pattern of manufacturing problems

first_img [email protected] APStock For the third time in four months, Lupin Pharmaceuticals, one of India’s biggest suppliers of generic drugs to foreign markets, has been admonished by the Food and Drug Administration for a series of troubling quality-control issues at a manufacturing facility.In a recent inspection report, the regulator cited the company for failing to thoroughly review unexplained deficiencies in batches of medicines and not taking “scientifically sound” steps to evaluate samples; delays in assessing health hazards; insufficiently investigating customer complaints, including an instance involving mold on medicines shipped to the U.S.; and a lack of oversight and training. About the Author Reprints GET STARTED What is it? By Ed Silverman Feb. 11, 2019 Reprints Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED One of India’s biggest drug makers has a ‘deeply troubling’ pattern of manufacturing problems Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry.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. What’s included? @Pharmalot Pharmalot 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. Log In | Learn More Ed Silverman Tags government agenciespharmaceuticalsSTAT+last_img read more

NASA twins study explores body’s response to long space travel

first_img In space, NASA heard astronaut’s immune system scream By Associated Press April 11, 2019 Reprints Related: Please enter a valid email address. More importantly, the study “represents more than one small step for mankind” by pointing out potential risks of longer-duration spaceflight that need study in more astronauts, said Markus Lobrich of Germany’s Darmstadt University and Penny Jeggo of the University of Sussex, who weren’t involved in the work. Key findingsNASA already knew some of the toll of space travel, such as bone loss that requires exercise to counter. This time, NASA-funded scientists looked for a gamut of physiologic and genomic changes that Scott Kelly experienced in space, comparing them to his DNA double on the ground, former astronaut Mark Kelly. Some results had been reported in February.Possibly the weirdest finding had to do with something called telomeres, the protective ends of chromosomes. Those tips gradually shorten as we get older, and are thought to be linked to age-related diseases including some cancers.But in space, Scott Kelly’s telomeres got longer. “We were surprised,” said Colorado State University telomere expert Susan Bailey. She can’t explain it although it doesn’t mean Kelly got younger. Back on Earth, his telomeres mostly returned to preflight average although he did have more short telomeres than before.Next, Kelly’s DNA wasn’t mutated in space but the activity of many of his genes — how they switch on and off — did change, especially in the last half of the voyage, which ended in March 2016.Immune system genes especially were affected, putting it “almost on high alert as a way to try and understand this new environment,” said study co-author Christopher Mason, a Weill Cornell Medicine geneticist in New York. Privacy Policy Related: NASA’s lab rat: What astronaut Scott Kelly’s year in space can teach us The findings were published in Friday’s edition of the journal Science, on some notable space anniversaries — when Soviet cosmonaut Yuri Gagarin became the first person in space in 1961, and the first launch of the space shuttle in 1981.advertisement Associated Press Tags geneticsresearch Leave this field empty if you’re human: Again, most gene expression returned to normal back home, but some of the immune-related genes were hyperactive six months later.Other findings:Some changes in the structure of Kelly’s eye and thickening of his retina suggested that, like about 40% of astronauts, he experienced symptoms of “spaceflight-associated neuro-ocular syndrome.” It may be caused by fluids shifting in the absence of gravity.He experienced some chromosomal instability that might reflect radiation exposure in space.A flu shot given in space worked as well as one on Earth.Kelly aced cognitive tests in space but slowed down after his return, maybe as more things competed for his attention.Ultra long-distance testingResearchers needed months’ worth of blood, urine, and fecal samples, along with cognitive and physical tests and ultrasound scans. That meant getting creative: Some blood samples required analysis so rapidly that Kelly would time collection so the blood could travel on Russian Soyuz capsules carrying other astronauts back to Earth.That wouldn’t be an option on a three-year trip to Mars. One of the study’s technological advances: Portable DNA-sequencing equipment that will let astronauts run some of their own genomic analyses on future missions, said Weill Cornell’s Mason. Newsletters Sign up for Morning Rounds Your daily dose of news in health and medicine. HealthNASA twins study explores body’s response to long space travel About the Author Reprints NASA astronaut Scott Kelly (left) and his identical twin, Mark, who participated in a twins study that was a never-before opportunity to track the biological consequences of spaceflight in genetic doubles. Pat Sullivan/AP WASHINGTON — From his eyes to his immune system, astronaut Scott Kelly’s body sometimes reacted strangely to nearly a year in orbit, at least compared to his Earth-bound identical twin — but newly published research shows nothing that would cancel even longer space treks, like to Mars.The good news: Kelly largely bounced back after returning home, say scientists who released final results from NASA’s “twins study,” a never-before opportunity to track the biological consequences of spaceflight in genetic doubles.It marks “the dawn of human genomics in space,” said Dr. Andrew Feinberg of Johns Hopkins University. He led one of 10 teams of researchers that scrutinized the twins’ health down to the molecular level before, during and after Kelly’s 340-day stay at the International Space Station.advertisement What’s next?Studying one pair of twins can’t prove risks of spaceflight, researchers cautioned. And longer missions, to the moon or Mars, will mean greater stress and radiation exposure.Colorado State’s Bailey plans to study 10 additional astronauts on yearlong missions, using the twin findings as a road map.“We need to get outside of low-Earth orbit and we need for the astronauts to spend longer periods of time to really evaluate some of these health effects,” she said.— Lauran Neergaardlast_img read more