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Friday, January 18, 2019

Rafael to Present Plan for Phase 3 Trial of Pancreatic Cancer Combo


American Society of Clinical Oncology (ASCO) in San Francisco, California on Friday, January 18, 2019.
Devimistat is Rafael’s lead altered metabolism directed (AMD) drug candidate, a first-in-class anticancer compound designed to disrupt the altered mitochondrial metabolism in cancer cells.
AVENGER 500 will compare the safety and efficacy of FOLFIRINOX (control arm) with devimistat in combination with modified FOLFIRINOX. Patients 18 – 75 years old of both sexes with metastatic (stage IV) pancreatic adenocarcinoma, not previously treated for metastatic disease and with ECOG performance status of 0 – 1 are eligible for enrollment in this study. This is a prospective, open label, multinational randomized trial and will be conducted in 9 different countries including the United States, six European Union countries, Israel and South Korea. Dr. Philip Agop Philip from Karmanos Cancer Institute of Wayne State University is the principal investigator of this trial. More information on the trial is available at www.clinicaltrials.gov (NCT03504423).
Sanjeev Luther, President and Chief Executive Officer of Rafael Pharmaceuticals, commented: “This is a multinational trial in ~100 sites. We are very excited to present the study design of this trial at the 2019 Annual Gastrointestinal Cancers Symposium.”
Philip A. Philip, MD, PhD, FRCP, the Principal Investigator of this study, commented: “Pancreatic cancer is the deadliest cancer worldwide with very limited treatment options. The current data on devimistat in combination with modified FOLFIRINOX in patients battling pancreatic cancer is immensely promising. I am, therefore, delighted to be involved in this important phase 3 trial.”
Timothy S. Pardee, MD, PhD, FACP, Chief Medical Officer of Rafael Pharmaceuticals, commented: “Devimistat targets tumors in a truly unique way and earlier clinical results suggest it leverages the metabolic vulnerability inherent in pancreatic cancer to improve outcomes in this deadly disease. I am very excited at the opportunity to discuss this important trial at such a preeminent meeting.”
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CMS Seeks to Prod Insurers to Negotiate Lower Drug Prices


The Trump administration on Friday unveiled a proposed Medicare pilot program intended to entice insurers to negotiate lower drug prices for customers of their Part D pharmacy plans.
Medicare also said it is planning to expand a separate test program with an aim of improving coordination of care of chronic illnesses and use of telehealth through insurer-run Advantage programs. The Centers for Medicare and Medicaid Services (CMS) said it intends to test adding a hospice benefit to Advantage in 2021 through this expansion of the Value-Based Insurance Design model.
With these moves, the Trump administration intends to make use of a key feature of the 2010 Affordable Care Act, its creation of the CMS Center for Medicare and Medicaid Innovation (CMMI). This platform allows Medicare officials flexibility to try out new approaches intended to improve the care of the more than 58 million people enrolled in the giant federal health program.
Medicare also intends to use CMMI to partly address the growing chorus of criticism about the generosity of a Part D safety-net feature for insurers. Medicare’s reinsurance covers about 80% of liability for so-called catastrophic spending in Part D. This mechanism originally was intended to shield insurers from the consequences of customers with markedly expensive prescription needs, particularly in the start-up of the Part D program.
The Part D plans are then responsible for 15% of this catastrophic spending, with patients paying 5% out of pocket.
“The structure introduces perverse incentives to push patients to the catastrophic phase and leaves plans with little reason to negotiate lower costs for the highest-spending patients,” CMS Administrator Seema Verma said on a Friday call with reporters.
The tab for this reinsurance almost quadrupled to $37.4 billion in 2017 from $9.4 billion in 2008, according to Medicare’s board of trustees. That far outpaced growth in Part D in general, for which the cost didn’t quite double in the same period. The total tab for Part D rose to $93.9 billion in 2017 from $53.9 billion in 2008, according to the board of trustees.
CMS on Friday said that the high list prices of new medications for cancer, hepatitis C, and rheumatoid arthritis had increased in Part D catastrophic phase spending.

Aiming for Sustainability

CMS noted that many influential groups, including the Medicare Payment Advisory Commission, have called for action to address the rising reinsurance cost of Medicare Part D’s reinsurance. “Continued growth at this pace may pose a risk to the sustainability of Part D,” the Office of Inspector General for the US Department of Health and Human Services (HHS) said in a 2017 report.
CMS is proposing a voluntary, 5-year model that would target rising federal reinsurance subsidy costs in Part D. It would run from 2020 to 2024. Plans would get a target level of spending for expected catastrophic pharmaceutical bills, Verma said. If the plans spend less, they can share in the savings. They will have to take losses if they exceed the target, she said.
“For the first time, plans will have incentives to lower costs and negotiate down list prices and therefore out-of-pocket expenses for the patients who need relief the most,” Verma said.
Verma pointed out that President Donald J. Trump has asked Congress to consider changes to the Part D structure. She said that the CMMI Part D model is expected to gather information to help lawmakers assess potential revisions to the program.
Elizabeth Carpenter, a practice director at the consulting firm Avalere, told Medscape Medical News that the Part D model fits into HHS’s strategy of using regulatory tools and mechanisms to address drug prices.
There’s also clearly interest in the topic of drug costs from leaders in both chambers of Congress, particularly Senate Finance Chairman Charles E. Grassley (R-IA) and Elijah E. Cummings (D-MD), chairman of the House Oversight and Reform Committee. Still, CMS could prove the faster path for action.
“Regardless of what Congress may or may not do this year, the administration has the authority and the will to take action focused on drug prices, and this is the latest example of that,” Carpenter said about the proposed Part D model.
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Opioid Use in Pregnancy Tied to Severe Birth Defects


More American infants are being born with their intestines outside of their bodies, and the disturbing trend might be linked to the opioid crisis, health officials reported Thursday.
The condition, called gastroschisis, is caused by a hole beside the belly button. The hole can be small or large, and sometimes other organs such as the stomach and liver can also be outside of the baby’s body, according to the U.S. Centers for Disease Control and Prevention scientists.
“The cases of gastroschisis continue to increase, and we did see higher rates of cases in counties that had higher rates of opioid prescriptions,” said lead researcher Jennita Reefhuis. She is chief of the Birth Defects Branch at CDC’s National Center on Birth Defects and Developmental Disabilities.
The researchers found that counties that had higher rates of opioid prescribing had nearly two times more babies born with gastroschisis, compared with counties with low opioid prescribing rates.
“However, we do not know if these two things are directly related,” Reefhuis stressed. “We plan to use this information to guide future research into the effects of opioids used during pregnancy.”
Babies born with gastroschisis need an operation to correct the condition. They also may need other treatments, including nutrients given through an IV, antibiotics to prevent infection and careful monitoring of their body temperature.
Each year, about 1,800 babies in the United States are born with gastroschisis, according to the CDC.
Some studies have found that having a baby at a young age is a powerful risk factor for gastroschisis. But different factors, such as prescription opioid use, might also be linked, the researchers said.
However, the CDC team can’t say that opioid use actually causes gastroschisis, Reefhuis said.
For the study, the researchers looked at cases of gastroschisis in 20 states, from 2006 to 2015, and saw an increase in most age groups.
When they linked opioid prescription data with gastroschisis cases, they found a higher prevalence of gastroschisis where opioid prescription rates were high.
The researchers cautioned that this finding doesn’t mean that women who use opioids during pregnancy will have a baby with gastroschisis.
The finding, however, is an alert that more research on opioid use during pregnancy and its effect on infants is needed.
Peggy Honein is director of the division of congenital and developmental disorders at the CDC’s National Center on Birth Defects and Developmental Disabilities. She said, “The opioid crisis is really the public health emergency of our time.”
The crisis has had a devastating effect in terms of overdose deaths, but it also has a “critical impact on moms and babies,” Honein said. “We want to understand the full impact that prenatal opioid exposure has on maternal health, as well as the health of newborns and children.”
Honein and her colleagues published a report in January in Pediatrics that goes over what is known and not known about “the full impact of the current U.S. opioid crisis on mothers and babies.”
A mother’s opioid addiction can cause her baby to suffer through drug withdrawal, but many other potentially harmful effects, like gastroschisis, aren’t known. These effects could also be developmental and behavioral, Honein said.
Reefhuis said that some women need to take opioids during pregnancy, including for treatment of opioid abuse disorder.
“Women who are pregnant or who are planning to become pregnant, and who are either taking opioids or are planning to take opioids, should talk to their doctor to discuss the risks and benefits to both the mother and the baby,” Reefhuis said.
The report was published Jan. 18 in the CDC’s Morbidity and Mortality Weekly Report.
More information
For more on gastroschisis, visit the U.S. Centers for Disease Control and Prevention.
SOURCES: Jennita Reefhuis, Ph.D., chief, Birth Defects Branch, U.S. National Center on Birth Defects and Developmental Disabilities, U.S. Centers for Disease Control and Prevention; Peggy Honein, Ph.D., M.P.H., director, division of congenital and developmental disorders, U.S. National Center on Birth Defects and Developmental Disabilities, U.S. Centers for Disease Control and Prevention; Jan. 18, 2019, Morbidity and Mortality Weekly Report
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Disrupted Sleep Plagues Hospital Patients, But New Program Might Help


 Anyone who has cared for a hospitalized loved one knows that frequent nighttime sleep interruptions — caused by noise or nursing checks — are a big concern.
But in a new study, a Chicago hospital adopted sleep-friendly measures for patients that led to fewer nighttime awakenings without compromising care.
Nighttime room entries dropped by 44 percent after researchers educated doctors and nurses on the health consequences of in-hospital sleep deprivation. The researchers also tweaked the hospital’s electronic health records system to avoid unnecessary overnight disruptions.
Over a year, patients in the so-called SIESTA unit also experienced an average of four times fewer disruptions for medication dosing and three times fewer for routine vital signs.
“We’ve known [inpatient sleep deprivation] is a problem since Florence Nightingale in the 1800s, so why hasn’t it been fixed? It’s a very patient-centered problem that also has health implications,” said study author Dr. Vineet Arora. She’s a professor of medicine at University of Chicago.
Arora’s prior research showed that even short amounts of sleep loss among hospital patients were associated with higher blood pressure and higher blood sugar levels during hospitalization. Other research has focused on delirium in sleep-deprived patients, as well as hospital readmission rates, she said.
A prior survey of Medicare patients also showed that only 62 percent reported their room was kept quiet at night, Arora noted.
The new study was conducted on two 18-room general medicine units. About 1,100 patients were admitted to either a standard unit or a SIESTA-enhanced unit. In the SIESTA (Sleep for Inpatients: Empowering Staff to Act) unit, clinicians were coached on improving patient sleep, while those in the standard unit were not.
The SIESTA program also used “nudges” through patients’ electronic health records to have staff skip unnecessary nighttime vital signs checks or medication doses.
While sleep-friendly orders rose in both inpatient units, the SIESTA unit logged more significant changes. Decisions to forgo unnecessary vital signs checks every four hours rose from 4 percent to 34 percent. Meanwhile, sleep-friendly timing of nighttime medications such as anti-clotting drugs jumped from 15 percent to 42 percent.
Dr. Seun Ross is director of nursing practice and work environment at the American Nurses Association. She said, “I believe all hospitals and clinicians err on the side of caution when choosing to assess their patients during specific intervals at night.” Ross was familiar with but wasn’t involved in the new research.
“Based on clinical judgment and a conversation with the patient, nighttime disruptions can be decreased,” Ross added. “This initiative is practical for patients that qualify — meaning they are clinically stable and not in critical condition. Sleep is healing, which is important for every human, but in an inpatient setting, communication is paramount.”
Arora and Ross agreed that many hospital nurses would be open to suggestions or a formal program that minimizes patients’ sleep disruptions.
“I actually view nurses as natural partners to improve patients’ sleep,” Arora said, noting that the American Academy of Nursing recommends nurses reduce unnecessary overnight care.
But, “while sleep is considered in the domain of nursing, it’s important to have doctors and nurses on the same page. It’s absolutely a team effort,” she added.
“I hope patients in the future, when they go to the hospital, are guaranteed their sleep will be considered as part of their recovery,” Arora said.
The study is in the January issue of the Journal of Hospital Medicine.
More information
The Palo Alto Medical Foundation offers more on the effects of sleep deprivation.
SOURCES: Vineet Arora, M.D., professor, medicine, and associate chief medical officer, clinical learning environment, University of Chicago; Seun O. Ross, D.N.P., R.N., director, nursing practice and work environment, American Nurses Association, Silver Spring, Md.; January 2019, Journal of Hospital Medicine
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Novartis and Adamis’ EpiPen challenger launches at double-digit discount


Buckle up, Mylan, another epinephrine injection is in town—and its sticker price is lower than your own generic EpiPen’s cut-rate cost.
Adamis Pharmaceuticals and its commercial partner, Novartis’ Sandoz unit, announced Wednesday they had officially launched the 0.3 mg dose of Symjepi, a prefilled epinephrine syringe meant for the emergency treatment of allergic reactions.
Sandoz has set the wholesale acquisition cost for a Symjepi two-pack at $250, lower than the $300 price of Mylan’s authorized generic EpiPen and Teva’s generic version. And it’s touting its injector design as a plus.
“The Symjepi device is small in size and fits into the palm of your hand, with the goal of a simple-to-use application and intuitive, user-friendly design,” Sandoz President Carol Lynch said in a statement.
Symjepi’s launch comes a year and half after Adamis won its FDA nod—and more than two years after its initial FDA rejection in mid-2016. When it was approved, Adamis CEO Dennis Carlo said the firm was looking for potential partners to “facilitate broad patient access to this new epinephrine treatment option and to maximize the value of our important asset.” Enter Sandoz, which earned exclusive U.S. commercialization rights to Symjepi in July 2018.
Sandoz is now rolling out Symjepi through a phased launch, the Novartis subsidiary said in a statement. First, it will focus on the institutional setting, “an established channel where Sandoz Inc. has significant experience and knowledge.” After the professional sector introduction, Sandoz will then expand to the patient-facing retail market.

In addition to the 0.3 mg injection intended for patients who weigh 30 kg (about 66 pounds) or more, the pair also snagged FDA backing last September for a 0.15 mg pediatric dose. Novartis is now “actively preparing” for the launch of that kid-friendly version, a Novartis spokesman told FiercePharma, but declined to provide a timeline for competitive reasons.
With Teva’s generic already aiming to steal share and the new, even cheaper Symjepi hitting the market, Mylan sales are likely to take a hit. In the third quarter prior to Teva’s launch, Mylan suffered a 14% decrease in North American sales, which it attributed “primarily to lower volumes on existing products, including the EpiPen Auto-Injector.” Executives on the Q3 call in early November said the volume drop was simply the result of the timing of purchases; they said they expect EpiPen demand would “rebalance” itself in Q4 to the usual volume it has seen in the past. That remains to be seen, however, until the company unveils its 2018 results next month.
Now that Teva and Sandoz have both launched, there’s a chance Mylan might never see EpiPen bounce back to its traditional size. But Mylan might not fret about the competition too much, because EpiPen has already shrunk to the point where the company no longer relies on it for a big chunk of sales. No single Mylan product accounts for more than 3% of its revenue, the company has said.
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FDA clears Verily’s prescription-only ECG smartwatch


Verily, the Alphabet research division formerly known as Google Life Sciences, has received a 510(k) clearance from the FDA for its own clinical study smartwatch, complete with an on-demand ECG. But as a prescription-only device, it’s far from a competitor to Apple’s publicly availablesmartwatch.
Verily’s Study Watch was first launched as an investigational device in April 2017 to help capture health information from participants in studies such as Project Baseline—a longitudinal venture by Google, Stanford Medicine and Duke University that aims to track 10,000 volunteers for four years using a variety of metrics.
The FDA cleared a new version of the watch as a class II device for adults, including those with known or suspected heart conditions, for use as a single-lead ECG.
“One area of focus for Study Watch has been cardiovascular health, as heart disease remains the No. 1 killer of men and women in the United States,” Verily’s cardiovascular health innovations head, Michael McConnell, wrote in a company blog post.

“This work may ultimately give us insights into the utility of integrating mobile health data into the clinical care environment, and how these data can support the physician-patient relationship,” McConnell said. Verily plans use both the investigational and the FDA-cleared versions in studies going forward.
Verily recently announced a $1 billion investment round to catapult new global partnerships, business development and potential acquisitions.

“We are taking external funding to increase flexibility and optionality as we expand on our core strategic focus areas,” Verily CEO Andrew Conrad said at the time. “Adding a well-rounded group of seasoned investors, led by Silver Lake, will further prepare us to execute as healthcare continues the shift towards evidence generation and value-based reimbursement models.”
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Oversight urged for unproven consumer ‘neurotechnologies’


The marketing of direct-to-consumer “neurotechnologies” can be enticing: apps that diagnose a mental state, and brain devices that improve cognition or “read” one’s emotional state. However, many of these increasingly popular products aren’t fully supported by science and have little to no regulatory oversight, which poses potential health risks to the public. In a new piece published in the journal Science this week, two bioethicists from Penn Medicine and the University of British Columbia suggest the creation of a working group that would further study, monitor, and provide guidance for this growing industry—which is expected to top $3 billion by 2020.
“There’s a real thirst for knowledge about the efficacy of these products from the public, which remains unclear because of this lack of oversight and gap in knowledge,” said lead author Anna Wexler, Ph.D., an instructor in the department of Medical Ethics and Health Policy at the Perelman School of Medicine at the University of Pennsylvania. “We believe a diverse, dedicated group would help back up or refute claims made by companies, determine what’s safe, better understand their use among consumers, and address possible ethical concerns.”
The group, made up of researchers, ethicists, funders, and industry experts, among others, the authors wrote, would serve as a clearinghouse for , such as the U.S. Food and Drug Administration (FDA) and the Federal Trade Commission (FTC), third-party organizations that monitor advertising claims, industry, social and medical scientists, , and the public at large.
While some of these techniques are used in clinical and research laboratory settings—for example, electroencephalography (EEG) devices are used to diagnose and treat epilepsy—many consumer-grade versions of neurotechnology devices are only loosely based in science. It is unclear whether the laboratory data collected to test them is applicable to consumer-grade products, leading many in the scientific world to question the efficacy of, and advocate for increased regulation of these readily available techniques and products.
For example, some consumer neurostimulation devices may pose dangers, such as skin burns. There are also potential psychological harms from many consumer EEG devices that purport to “read” one’s emotional state.
“If a consumer EEG device erroneously shows that an individual is in a stressed state, this may cause him or her to become stressed or to enact this stressed state, resulting in unwarranted psychological harm,” the authors wrote. Also, a smartphone wellness app that diagnoses symptoms of depression does so without medical support structures, such as a psychologist or mental health counselor.
The devices have thrived in part because of minimal . Many fall outside of FDA jurisdiction because they are categorized as “low-risk” wellness products, paving an easier path to the market. Also, investors interested in financing these devices have publicly stated that it would be difficult to invest in them if they required an FDA approval, the authors said, which would mean rigorous testing and time.
Currently, most of the regulatory burden for consumer neurotechnology falls to the FTC, which has the authority to act on claims of false advertising. However, with thousands of health and wellness apps and devices, that oversight is ill-suited to monitor and regulate the industry effectively, they said.
The authors’ proposal is two-fold: create an independent working group that would survey the main domains of direct-to-consumer neurotechnologies and provide succinct appraisals of potential harms and probable efficacy. Rather than evaluating each and every product or providing overarching framing questions, the proposed working group’s appraisals would outline the evidence base and potential risks, and identify gaps in current knowledge.
This working group would be responsible for disseminating those appraisals to the public and partnering with organizations well positioned to communicate with key consumer groups.
“Given that government agencies and private enterprises are actively funding research into new methods of modulating brain function,” the authors wrote, “the present generation of [direct-to-consumer] neurotechnologies may be only the tip of the iceberg—making it all the more imperative to create an independent body to monitor developments in this domain.”
Explore further
More information: Anna Wexler et al, Oversight of direct-to-consumer neurotechnologies, Science (2019). DOI: 10.1126/science.aav0223
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