Warren losing ground in polls on ‘Medicare for All’

Sen. Elizabeth Warren’s (D-Mass.) main health care proposal is losing support in the polls, posing a challenge to the emerging front-runner as “Medicare for All” comes under fresh attacks from fellow presidential candidates, hospitals, doctors and insurers.
Opposition to Medicare for All was on full display at Tuesday night’s Democratic primary debate, where moderate candidates called the plan a “pipe dream” and an “obliteration” of the private health insurance system.
The bulk of those attacks came from moderates like South Bend, Ind., Mayor Pete Buttigieg, Sen. Amy Klobuchar (D-Minn.) and former Vice President Joe Biden, who accused Warren of being evasive on how she would pay for the proposal.

“There’s still been no explanation for the multitrillion-dollar hole in this plan,” Buttigieg said Wednesday on CNN. “I have a lot of respect for Sen. Warren, but last night she was more specific and forthcoming about the number of selfies she’s taken than about how this plan is going to be funded.”
Several polls showed support dropping or plateauing for Medicare for All throughout the summer. Those shifts in public opinion came amid Democratic debates that spent significant time on the issue, and a well-funded opposition campaign launched by the health care industry.
The declining support creates a challenge for Warren, who gambled on not devising her own health care plan, like other candidates, but instead telling voters “I’m with Bernie” on Medicare for All.
The overall plan, authored by Sen. Bernie Sanders (I-Vt.) and popularized during his 2016 presidential run, has gained more public exposure in the 2020 cycle.
Democratic presidential candidates like Sens. Kamala Harris (D-Calif.) and Cory Booker (D-N.J.)  launched their campaigns in support of Medicare for All but toned down their rhetoric as polls increasingly showed voter discomfort with it.
A Kaiser Family Foundation (KFF) tracking poll released Tuesday showed support has dropped 5 points, and opposition increased 8 points, since April, two months before the first Democratic primary debate.
“We know some arguments commonly made against Medicare for All push people to oppose it. I think it’s a possibility that people hearing more of that discussion and hearing more of those arguments could be leading to a softening in support,” said Liz Hamel, director of public opinion and survey research for KFF.

Arguments from Republicans and moderate Democrats that Medicare for All would increase taxes for the middle class and eliminate private health insurance appear to be breaking through with voters.
“I don’t understand why you believe the only way to deliver affordable coverage to everybody is to obliterate private plans,” Buttigieg said at Tuesday’s debate, addressing Warren.
Support for Medicare for All typically declines when voters are told it would end private insurance. Sanders’s proposal would ban the sale of private insurance plans that cover the same services as the new national health care plan.
Warren and Sanders argue that private insurance companies profit by denying care to their customers, driving up health care costs for millions of Americans.
But Warren sidestepped questions Tuesday, as she did in previous debates, about whether Medicare for All would lead to tax increases for the middle class. She argues overall costs would go down for middle class families because Medicare for All would do away with the deductibles, copays and premiums associated with private insurance.
Meanwhile, industry groups such as the Partnership for America’s Health Care Future, a coalition of doctors, hospitals and insurers opposed to Medicare for All, are spending millions of dollars on ads attacking the proposal in early voting states like Iowa. The group has also aired ads before, during and after some of the debates.
Medicare for All still has the support of the majority of the public — 51 percent said they favored it in October, compared to the 53 percent who said the same in September, according to KFF tracking polls. Fifty-six percent in April said they supported it.
That support mainly comes from Democrats and independents, but it’s dropping among both groups: 71 percent of Democrats and 51 percent of independents said they supported Medicare for All in October, compared with 77 percent of Democrats and 53 percent of independents who said the same last month.
https://thehill.com/policy/healthcare/466205-warren-faces-tougher-sell-with-medicare-for-all

95 percent of baby foods tested had toxic metals, study finds

A test of 168 baby foods from major manufacturers found 95 percent contained toxic metals, according to an investigation published Thursday.
The test found 95 percent of baby foods contained lead, while 73 percent contained arsenic, 75 percent contained cadmium and 32 percent contained mercury, with a quarter containing all four. Twenty percent had more than 10 times the maximum lead level endorsed by public health advocates, which is 1 part per billion.

The report was compiled by Health Babies, Bright Futures, a coalition of nonprofit and scientific organizations, and echoes the results of a similar study by the Food and Drugs Organization finding at least one of the same metals in 33 of 39 types of food tested.
“Even in the trace amounts found in food, these contaminants can alter the developing brain and erode a child’s IQ. The impacts add up with each meal or snack a baby eats,” the report states. “Despite the risks, with few exceptions there are no specific limits for toxic heavy metals in baby food.”
Rice-based products, fruit juices and sweet potatoes were found to be the products with the biggest danger of neurotoxic harm.
“Arsenic, lead and other heavy metals are known causes of neurodevelopmental harm,” Dr. Philip Landrigan, a pediatrician and director of the Program in Global Public Health and the Common Good in the Schiller Institute for Integrated Science and Society at Boston College, said in a statement.
“Low level exposures add up, and exposures in early life are especially dangerous. The cumulative impact of exposures is what makes this a significant concern that demands action,” Landrigan added.
https://thehill.com/policy/healthcare/466267-study-finds-95-of-168-us-baby-foods-contain-toxic-metals

Cancer Society: Progress in global cancer fight not only possible, but achievable

The Cancer Atlas, 3rd edition, a comprehensive global overview of cancer around the globe, concludes that progress in the fight against cancer is not only possible, but achievable. The report was produced by the American Cancer Society (ACS), the Union for International Cancer Control (UICC), and International Agency for Research on Cancer (IARC). It was released at the World Cancer Leaders’ Summit in Nur-Sultan, Kazakhstan.
The all-new 3rd edition highlights distinct patterns and inequities in the present cancer burden around the world; outlines the risk factors that are driving cancer patterns; and details the prospects for cancer prevention and control. This theme of the current edition is “Access Creates Progress,” drawing attention not only to the problem at hand, but also the means of tackling the cancer burden through access to information and services.
The Cancer Atlas provides information on the global burden of cancer in a user-friendly and accessible form for cancer control advocates, government and public health agencies, and policy makers around the world as well as patients, survivors, and the general public. In addition to the printed report, the information is included on a comprehensive, interactive website.
Cancer is the leading or second-leading cause of premature death (under age 70) in 91 countries worldwide. Based on expected population growth and aging alone, the number of global cancer cases is expected to increase by 60% in 2040. More widespread distribution of lifestyle factors such as smoking, unhealthy diet, and physical inactivity are likely to make that number considerably larger.
Other highlights of the 3rd Edition:

• Tobacco causes more preventable cancer deaths than any other risk factor. In 2017 alone, smoking was responsible for an estimated 2.3 million cancer deaths globally, accounting for 24% of all cancer deaths. There are still 1.1 billion smokers worldwide. Progress in tobacco control legislation over the last decade means 1.5 billion people in 55 countries are now protected by smoke-free legislation.
• While infectious agents are responsible for an estimated 15% of all new cancer cases worldwide, the proportion ranges from around 4% in many very high-income countries to more than 50% in several sub-Saharan African countries. The four major infectious agents (which together account for more than 90% of all infection-related cancers) are Helicobacter pylori , human papillomavirus (HPV), hepatitis B virus (HBV) , and hepatitis C virus (HCV) .
• Excess body weight increases risk of 13 types of cancer and in 2012 accounted for 3.6% of all new cancer cases among adults worldwide. The prevalence of excess body weight is rising worldwide: in 2016, an estimated 39% of men and 40% of women aged 18 years and older, and 27% of boys and 24% of girls aged 5-18 years, were obese. High amounts of sugar-sweetened beverages and sedentary behaviors, including screen-time, increase risk of excess body weight.
• The cancer burden associated with unhealthy diet, excess body weight, and physical inactivity is expected to grow in most parts of the world, particularly in parts of the Middle East and several other low- and middle-income countries in parts of Asia and Oceania because of the obesity epidemic.
• Alcohol is responsible for 4.2% of all cancer deaths globally, with marked variation across countries.
• Breast cancer is the most common cancer in women in almost all countries worldwide, and accounts for almost one in four new cancer cases among women. Lifetime risk of breast cancer among females in high-income countries can be up to three times that in low-income countries.
• Each year, about 270,000 cancer cases are diagnosed in children. Today, five-year survival from childhood cancer in high-income countries is greater than 80%, but it can be as low as 20% in low-income countries. With interventions to improve early diagnosis and adherence to appropriate treatment, childhood cancer survival can be increased to 60% in low-income countries, saving almost 1 million children’s lives over a decade.
• Over the next half century, an estimated 44 million cervical cancer cases will occur worldwide if current trends continue. Effective delivery of combined high coverage screening and vaccination could avert over 13 million cervical cancer cases by 2069, and eventually lead to cervical cancer eliminated as a major public health problem.
• Approximately 3 to 6% of all cancers worldwide are caused by exposures to carcinogens in the workplace.
• Outdoor air pollution causes over half a million lung cancer deaths and millions of deaths from other diseases each year. Outdoor air pollution levels are particularly high in rapidly-growing cities in low- and middle-income countries. Diesel exhaust, classified as a lung carcinogen by IARC, contributes to outdoor air pollution and is also an occupational lung carcinogen.
• Radiotherapy is indicated for about 60% of cancer patients to relieve symptoms, shrink tumors before surgery, or kill remaining cancer cells after surgery to avoid recurrence. Radiotherapy coverage is less than optimal in many low- and middle-income countries. In Ethiopia, for example, a population of approximately 100 million is served by a single radiotherapy center.

“This much is clear,” writes Gary Reedy, ACS chief executive officer in report’s foreword. “We simply must do better to ensure everyone can benefit from advances in the fight against cancer. As you will see in the pages of this Cancer Atlas, Third Edition, progress is not only possible, but also achievable.”
“The Cancer Atlas has proved to be an outstanding publication in the past, helping the cancer community communicate the progress we have or have not made, the challenges we face and the areas of focus for future years,” writes Cary Adams, chief executive officer of UICC. “Its beautifully crafted presentations of facts and evidence help us construct compelling messages to better articulate the problem and present solutions. This new edition will once again be circulated widely and inspire those of us who want to see change happen.”
“Cancer is an issue of sustainable development,” writes Elisabete Weiderpass, director of IARC. “Facing the cancer problem is a prerequisite for addressing social and economic inequities, stimulating economic growth, and accelerating sustainable development. I hope that this book will find widespread use, because prevention is, and should continue to be, the first line of attack in tackling the challenges posed by the global cancer epidemic.”
Article: Jemal A, Torre L, Soerjomataram I, Bray F (Eds). The Cancer Atlas. Third Ed. Atlanta, GA: American Cancer Society, 2019. Also available at: http://canceratlas.cancer.org/
https://www.eurekalert.org/pub_releases/2019-10/acs-rpi101119.php

Whistleblower alleges Medicare fraud at iconic Seattle-based health plan

Group Health Cooperative in Seattle, one of the nation’s oldest and most respected nonprofit health insurance plans, is accused of bilking Medicare out of millions of dollars in a federal whistleblower case.

Teresa Ross, a former medical billing manager at the insurer, alleges that it sought to reverse financial losses in 2010 by claiming some patients were sicker than they were, or by billing for medical conditions that patients didn’t actually have. As a result, the insurer retroactively collected an estimated $8 million from Medicare for 2010 services, according to the suit.
Ross filed suit in federal court in Buffalo, N.Y., in 2012, but it remained under a court seal until July and is in the initial stages. The suit also names as defendants two medical coding consultants, consulting firm DxID of East Rochester, N.Y., and Independent Health Association, an affiliated health plan in Buffalo, N.Y. All denied wrongdoing in separate court motions filed late Wednesday to dismiss the suit.

The Justice Department has thus far declined to take over the case, but said in a June 21 court filing that “an active investigation is ongoing.”

The whistleblower suit is one of at least 18 such cases documented by KHN that accuse Medicare Advantage managed-care plans of ripping off the government by exaggerating how sick its patients were. The whistleblower cases have emerged as a primary tool for clawing back overpayments. While many of the cases are pending in courts, five have recovered a total of nearly $360 million.
“The fraudulent practices described in this complaint are a product of the belief, common among MA organizations, that the law can be violated without meaningful consequence,” Ross alleges.
Medicare Advantage plans are a privately run alternative to traditional Medicare that often offer extra benefits such as dental and vision coverage, but limit choice of medical providers. They have exploded in popularity in recent years, enrolling more than 22 million people, just over one in three of those eligible for Medicare.
Word of another whistleblower alleging Medicare Advantage billing fraud comes as the White House is pushing to expand enrollment in the plans. On Oct. 3, President Donald Trump issued an executive order that permits the plans to offer a range of new benefits to attract patients. One, for instance, is partly covering the cost of Apple Watches as an inducement.
Group Health opened for business more than seven decades ago and was among the first managed-care plans to contract with Medicare. Formed by a coalition of unions, farmers and local activists, the HMO grew from just a few hundred families to more than 600,000 patients before its members agreed to join California-based Kaiser Permanente. That happened in early 2017, and the plan is now called Kaiser Foundation Health Plan of Washington. (Kaiser Health News is not affiliated with Kaiser Permanente.)

In an emailed statement, a Kaiser Permanente spokesperson said: “We believe that Group Health complied with the law by submitting its data in good faith, relying on the recommendations of the vendor as well as communications with the federal government, which has not intervened in the case at this time.”
Ross nods to the plan’s history, saying it has “traditionally catered to the public interest, often highlighting its efforts to support low-income patients and provide affordable, quality care.”
The insurer’s Medicare Advantage plans “have also traditionally been well regarded, receiving accolades from industry groups and Medicare itself,” according to the suit.
But Ross, who worked at Group Health for more than 14 years in jobs involving billing and coding, said that from 2008 through 2010 GHC “went from an operating income of almost $57 million to an operating loss of $60 million. Ross said the losses were “due largely to poor business decisions by company management.”
The lawsuit alleges that the insurer manipulated a Medicare billing formula known as a risk score. The formula is supposed to pay health plans higher rates for sicker patients. But Medicare estimates that overpayments triggered by inflated risk scores have cost taxpayers $30 billion over the past three years alone.
According to Ross, a GHC executive attended a meeting of the Alliance of Community Health Plans in 2011 where he heard from a colleague at Independent Health about an “exciting opportunity” to increase risk scores and revenue. The colleague said Independent Health “had made a lot of money” using its consulting company, which specializes in combing patient charts to find overlooked diseases that health plans can bill for retroactively.
In November 2011, Group Health hired the East Rochester firm DxID to review medical charts for 2010. The review resulted in $12 million in new claims, according to the suit. Under the deal, DxID took a percentage of the claims revenue it generated, which came to about $1.5 million that year, the suit says.
Ross said she and a doctor who later reviewed the charts found “systematic” problems with the firm’s coding practices. In one case, the plan billed for “major depression” in a patient described by his doctor as having an “amazingly sunny disposition.” Overall, about three-quarters of its claims for higher charges in 2010 were not justified, according to the suit. Ross estimated that the consultants submitted some $35 million in new claims to Medicare on behalf of GHC for 2010 and 2011.

In its motion to dismiss Ross’ case, GHC called the matter a “difference of opinion between her allegedly ‘conservative’ method for evaluating the underlying documentation for certain medical conditions and her perception of an ‘aggressive’ approach taken by Defendants.”
Independent Health and the DxID consultants took a similar position in their court motion, arguing that Ross “seeks to manufacture a fraud case out of an honest disagreement about the meaning and applicability of unclear, complex and often conflicting industry-wide coding criteria.”
In a statement, Independent Health spokesman Frank Sava added: “We believe the coding policies being challenged here were lawful and proper and all parties were paid appropriately.”
Whistleblowers sue on behalf of the federal government and can share in any money recovered. Typically, the cases remain under a court seal for years while the Justice Department investigates.
https://www.fiercehealthcare.com/payer/whistleblower-alleges-medicare-fraud-at-iconic-seattle-based-health-plan

How 4 states are handling surprise medical bills

States across the country have passed laws designed to protect patients from surprise medical bills.
These laws typically address bills that occur after patients unintentionally receive out-of-network care in emergency situations or at in-network hospitals.
Four things to know about the laws:
1. New Mexico’s surprise-billing law takes effect Jan. 1. It requires insurers to pay for nonemergency care by an out-of-network provider in certain situations, such as when medically necessary care is unavailable within the insurance company’s network, according to JDSupra. The news service reported that the law, by July 1, will also require licensed healthcare facilities to post consumers’ rights information.
2. Texas’ new surprise-billing law, signed by Gov. Greg Abbott June 14, also takes effect Jan. 1. The law bans surprise medical bills in circumstances where patients are unable to choose the provider they see or the facility they visit, said one of the bill’s authors, Sen. Kelly Hancock, R-North Richland Hills. The law covers medical emergencies and out-of-network lab and imaging work.
3. California’s surprise-billing law, in effect since 2017, addresses unexpected patient bills for out-of-network, nonemergency physician services at in-network hospitals by paying out-of-network health professionals a benchmark, locally negotiated market rate.
4. Florida passed a surprise-billing law in 2016. Patients who receive out-of-network care at an in-network facility are only on the hook for the in-network fee. Public radio station WUSF reported that the rest of the charges must be worked out between physicians and insurance companies, via a dispute resolution process.
https://www.beckershospitalreview.com/finance/how-4-states-are-handling-surprise-medical-bills.html

Novant Health leaders talk Walgreens partnership goals for 2020

Novant Health’s Oct. 1 announcement that it will open retail health clinics in several Walgreens pharmacies in North Carolina comes amid a stream of retailers taking on healthcare.
The 15-hospital system, headquartered in Winston-Salem, said the retail clinics will be staffed with Novant Health physician assistants and nurse practitioners who will treat common illnesses and injuries and offer chronic care follow-ups. Walgreens also will acquire nine Novant Health retail pharmacies.
Here, Pam Oliver, MD, president of Novant Health Physician Network, and Cedric Terrell, senior vice president of pharmacy for Novant Health, discuss the Walgreens partnership as well as the potential effects of other major retailers such as Walmart and Amazon moving into the healthcare space.
Editor’s note: Responses were lightly edited for length and clarity. 
Question: What prompted Novant Health to consider the Walgreens partnership?
Pam Oliver: This partnership with Walgreens is really about our commitment to making healthcare more convenient, accessible, affordable for not only our patients but also for our team members. The retail healthcare clinic collaboration is also about giving patients options about how they can get healthcare [and] stay well, depending on what their needs are. From a global perspective, this collaboration will allow us to quickly scale our patient access for and to high-quality cost-effective pharmacy services and clinical care. It aligns with our overall growth strategy and how we are thinking through developing our clinics in the future to not be just the traditional bricks-and-mortar strategy.
Q: What are the primary goals behind the partnership?
PO: Access points to improve our attachment to and capture of patients and consumers within our market. We also hope it will help us deliver on our population health objective, and for pharmacy, to enhance our speed to value for pharmacy and services. This is a way that can be both cost-efficient and scalable.
Q: What is the timeline for the partnership?
PO: Walgreens will assume ownership of all Novant Health nonspecialty retail and mail-order pharmacies within the next 60 days. Our specialty pharmacy will continue to be operated by Novant Health. We expect that the first retail clinics will be online and seeing patients in the first half of 2020.
Q: How do you think efforts by major retailers like Walgreens, Walmart and Amazon will ramp up the demand for healthcare talent? 
PO: I think for anyone working in healthcare, these new models and collaborations may offer just more choice. We continue to see, and as news has shown, shortages of primary care physicians, nurses and many others that are needed to support our healthcare system. And we know as others enter [healthcare], we will compete for talent.
There is more choice on the side of the providers. We have a large medical group, so we think we have a competitive advantage because of the resources we have committed to addressing burnout and making this the most attractive place to work. The team member experience has been part of our mission, vision and values consistently as we gauge how our team members feel about being employed or working as part of Novant Health. We see they also value being part of the system and validate we are committed to having an inclusive team. I think we are positioned well in the competition for talent, but there will be more competition as people have more options that didn’t exist previously.
Cedric Terrell: At Novant we moved to residency-trained pharmacists about two years ago. You had to be residency-trained or have at least about two to three years of experience in a particular area for the new hires. Also, with our workforce development with our [pharmacy] technicians, we set a tier process so our technicians could take on more of the operational or dispensary roles as our pharmacists move closer to the patient touchpoints. Whether they’re prescribing or they’re making interventions at Novant Health, what I see is more specialization of that, and so as we bring on new team members, we continue to develop them in more of the specialty and subspecialty space. And then probably on a broader scale externally to Novant, you’re going to see the same thing as the workforce changes with these new care delivery models.
Q: Which major corporations do you think will be next to offer their own healthcare? 
PO: I think it’s important to acknowledge that every major employer is looking for ways to have more affordable and accessible healthcare for their team members. And healthcare organizations are looking beyond traditional mergers and acquisitions to gain new services and skills for our patients’ needs both now and in the future. Partnerships aren’t a market differentiator anymore. It’s been more of the norm. Walmart [and] Amazon are entering into what we would consider nontraditional partnerships. We see great potential in collaborating with companies that would traditionally be seen as competitors because we think that is the model to drive high quality and more efficient care. And we also think that it’s a way to produce savings on the healthcare side that will help us to reinvest in our communities and in our public health efforts in our communities. I don’t have a healthcare corporation in mind as the next, but I do think that is the norm.
CT: That’s hard to call. As you look at it, the major players are the ones to be focused on as the disruption [in healthcare] continues. The point of the matter is to bring services closer to consumers’, customers’ and patients’ home[s]. As we continue to look at ways to do that, you’re going to have the digitalization of therapy as well as digitization of pharmaceuticals. That’s truly a new approach to management of a patient population. I think the major players is now the norm, but with that is going to be some refinement, and probably some new goals, that develop.
https://www.beckershospitalreview.com/pharmacy/novant-health-leaders-talk-walgreens-partnership-goals-for-2020.html

Upcoming events – Novartis’s asthma Zeal and Alnylam’s lumasiran tes

Welcome to your weekly roundup of approaching clinical readouts. In the fourth quarter Novartis will report the first phase III data with fevipiprant, an oral DP2 inhibitor, in severe asthma.
The company hopes that the project can bridge the gap between inhaled therapies and biologicals. However, previous failures with this class have made investors wary. If Novartis might be able to brush off a clinical trial flop, the same might nto be true for Gossamer Bio, whose lead project, GB001, also hits DP2.
The first pivotal data with fevipiprant will come from the Zeal 1 and 2 trials, in patients with moderate to severe asthma. The primary endpoint of both is change in forced expiratory volume in one second (FEV₁), a measure of lung function, at 12 weeks versus placebo.
Leerink analysts reckon a best-case scenario for both Novartis and Gossamer would be FEV₁ improvements versus placebo of 200ml or more – putting fevipiprant on a par with Sanofi/Regeneron’s Dupixent in asthma – although they added that a 100ml improvement could also hit statistical significance.
But history is not on the companies’ side. Several DP2 inhibitors have been discontinued after failing to show a benefit in asthma, including Astrazeneca’s AZD1981 and Amgen’s vidupiprant/AMG 853. Leerink blamed “suboptimal pharmacology” with these older agents. Meanwhile, Novartis believes that it could have a better chance with fevipiprant as it is targeting more severe patients and those with high eosinophils, a marker of allergic asthma.
However, even in allergic asthma data have been mixed, with no clear dose response seen on FEV₁ in a phase II trial, despite an overall win. Gossamer is already trying to play down the importance of FEV₁ as an endpoint, but this looks like clutching at straws.
Fevipiprant is in two more pivotal trials, Luster 1 and 2, which Gossamer and its backers believe could be more relevant for GB001. However, investors in the smaller group might still be in for a rocky ride if the Zeal trials fall short.

Upcoming readouts with DP2 inhibitors
Project	Company	Trial	Indication	Primary endpoint	Data due	2024e sales ($m)
Fevipiprant/ QAW039	Novartis	Zeal 1	Moderate to severe asthma (Gina 3/4)	FEV1 at 12 weeks	Q4 2019	619
		Zeal 2	Moderate to severe asthma (Gina 3/4)	FEV1 at 12 weeks	Q4 2019
		Luster 1	Severe eosinophilic asthma (Gina 4/5)	Exacerbations at one year	Q1 2020
		Luster 2	Severe eosinophilic asthma (Gina 4/5)	Exacerbations at one year	Q1 2020
GB001	Gossamer Bio	Leda (phII)	Moderate to severe eosinophilic asthma (Gina 4/5)	Asthma worsening composite	2020	272
Gina: Global Initiative on Asthma; Source: EvaluatePharma, clinicaltrials.gov.

Alnylam sees the light
Alnylam is due to report from its pivotal Illuminate-A trial of lumasiran in primary hyperoxaluria type 1 (PH1), a rare inherited disease, by the end of the year.
Lumasiran is an RNAi therapeutic designed to reduce levels of the enzyme glycolate oxidase, preventing the formation of oxalate; in PH1 this substance builds up in combination with calcium to form kidney and bladder stones, causing kidney problems and, eventually, end-stage renal disease.
There are three types of primary hyperoxaluria that differ in severity and genetic cause. Lumasiran targets only PH1, which is caused by a mutation in the AGXT gene. There are no approved therapies for the disease, and some patients have to undergo liver and kidney transplants.
The Illuminate trial compares subcutaneous lumasiran versus placebo in 30 adults and children. The primary measure is the percentage change in 24-hour urinary oxalate excretion from baseline to 6 months, a surrogate endpoint. Key secondary endpoints include additional measures of urinary oxalate, estimated glomerular filtration rate, safety and tolerability.
In terms of competition lumasiran is neck and neck with Dicerna’s DCR-PHXC, another RNAi contender that is in a pivotal trial called Phyox2 in 36 patients with PH1 and PH2. Dicerna hopes that its candidate could be used in all genetic subtypes of PH. DCR-PHXC targets a different enzyme, lactate dehydrogenase A, inhibition of which is said to reduce oxalate levels.
Dicerna’s trial also uses the 24-hour urinary oxalate excretion as an endpoint, but the study measures levels at 3, 4, 5 and 6 months to give an average value over time; this could help mitigate variability and placebo effect.
If Illuminate-A proves positive Alnylam expects to file early next year.

Selected trials in primary hyperoxaluria type 1
Trial	Details	Data?
Illuminate-A NCT03681184	30 adults and children, three monthly 3mg/kg doses or placebo, followed by quarterly maintenance doses	YE 2019
Illuminate-B NCT03905694	20 patients aged under six, dosing based on weight	Mid 2020
Illuminate-C	Single arm study in patients with severe renal impairment	Initiate by YE 2019
Lumasiran Phase I/II NCT02706886	20 patients, 1mg/kg monthly, 3mg/kg monthly and 3mg/kg quarterly	Reported: mean maximal reduction in urinary oxalate of 75% relative to baseline across all cohorts
Phyox2 NCT03847909	36 patients with PH1 or PH2. Multiple fixed doses of DCR-PHXC	Study completion May 2020
Source: EvaluatePharma, clinicaltrials.gov.

https://www.evaluate.com/vantage/articles/events/upcoming-events/upcoming-events-novartiss-asthma-zeal-and-alnylams