Over 20% skip medical care due to cost: Bankrate survey

• Americans continue to worry about paying for healthcare. In a new Bankrate survey of 1,000 adults, 22% said they or a close family member opted not to get necessary medical care because of the cost, while 77% said cost worries had led them to avoid care.
• Of those who admitted skipping a doctor’s visit in the past year, 29% got their insurance coverage in the individual marketplace, such as the ACA exchanges, and 22% were covered under employee benefit plans. About 17% were Medicare and Medicaid beneficiaries.
• Women feel significantly less secure than men when it comes to healthcare. When asked if they had chosen not to get medical care in the past year, 25% of women answered “yes,” compared with 18% of men. Nearly half (47%) of women said a bill they paid in the past 12 months was more expensive than they anticipated, versus 35% of men.

Overall, 41% of those surveyed said a bill paid in the past year was higher than expected.

There’s also widespread concern about access to affordable health insurance. Among all adults, 54% said they are very or somewhat worried that they might not have affordable coverage in the future. That share grew to 67% among younger baby boomers and to 59% for Gen Xers.

“Health care is a huge issue in the run-up to the midterm elections, especially with the effort to eliminate coverage for pre-existing conditions,” Bankrate.com analyst Taylor Tepper said in a statement. “It is incredibly hard to comparison shop, premiums are rising and so are deductibles. The onus has been on the patient to keep prices down.”

This is far from the only survey to show Americans are worried about paying their medical bills. In a recent survey by The Commonwealth Fund, just two-thirds of U.S. adults said they are very or somewhat confident they could afford to pay for a serious illness, down from 70% in 2015. And that confidence dropped to about half when people earned less than $30,150, down from 60% three years ago and 20% below that of adults with higher incomes.

These findings come as CMS has recently issued a final rule requiring hospitals to start posting their price information online. Hospitals must make available a list of their standard charges in a machine-readable format and update it at least annually. But even with mandatory price lists, it can be difficult for patients to anticipate out-of-pocket costs and avoid being blindsided by surprise billings. CMS already required hospitals to make their standard charges public, but a supplementary RFI to the final rule acknowledges pricing transparency issues still need to be addressed.

In the meantime, some states have taken steps to increase visibility around healthcare pricing. In October, Maryland launched a statewide initiative, called Wear the Cost, to help consumers compare prices for common nonemergency procedures at hospitals. The website calculates two types of costs — typical and anticipated costs, such as office visits and surgery, and costs associated with potentially avoidable complications. Consumers are encouraged to look for hospitals with low total costs and low rates of avoidable complications to get the best value.

Massachusetts requires insurers to post healthcare price informationonline, and Arizona requires large medical facilities to post patient costs for the 50 most common medical procedures. Smaller hospitals must provide prices for the top 35 procedures.

But studies have shown most Americans don’t price shop for healthcare. In a national poll by the Bucknell Institute for Public Policy, just over one-fourth of respondents said they request cost information from providers. Those numbers may be higher among younger consumers, though. A 2016 PNC Healthcare survey found 41% of millennials would likely seek cost estimates before getting treatment, versus 21% of baby boomers.

https://bit.ly/2OnuSbg

Gilead Sciences (GILD) Remains a Buy at Mizuho Securities

Mizuho Securities analyst Salim Syed maintained a Buy rating on Gilead Sciences (NASDAQ: GILD) today and set a price target of $94.

https://bit.ly/2B0G1gn

Pfizer, Lilly score reimbursement wins with UK cost regulator

A couple of drugmakers are celebrating this week after favorable decisions from England’s cost watchdog.

The National Institute for Health and Care Excellence (NICE) has recommended both Eli Lilly’s Taltz and Pfizer’s Besponsa—and for the latter drug, the decision marks an about-face from the cost-effectiveness gatekeeper.

For Lilly’s anti-inflammatory med, the endorsement comes in psoriatic arthritis (PsA), Taltz’s newest indication. NICE backed the drug solo or with methotrexate under a couple of conditions—including that patients haven’t responded or have stopped responding to anti-TNF drugs within the first 12 weeks of treatment, and that Lilly provides Taltz with a confidential price break.

It’s a boost for Taltz, which is competing head-to-head with Novartis giant Cosentyx in both the psoriasis and psoriatic arthritis categories. And while Cosentyx had a big head start in both, Lilly thinks the positive trial data Taltz generated in PsA patients who had already been treated with anti-TNF drugs will provide its contender an edge.

“Up until now, the prescriber won’t have had a clinical trial to refer to that will give the prescriber confidence” that a therapy would perform well in the previously treated population, Pete Salzmann, Lilly VP of immunology, said in a December interview. And that population’s a large one, considering that “only about half of the patients will get a fully satisfactory response to their first-line agent.”

Meanwhile, for Pfizer, new guidance recommending acute lymphoblastic leukemia drug Besponsa is also a welcome development. NICE gave the cancer-fighter the cold shoulder last August after determining it wasn’t cost effective, and Pfizer had plenty to say about that verdict.

“Today’s frustrating decision for inotuzumab ozogamicin is another example of how NICE is not appropriately assessing the value of modern cancer medicines, leaving patients without access to new treatments that could transform their lives,” the company said in a statement at the time.

Now, though, NICE is in favor, and in its updated guidance it pointed to a the fact that “more people having” Besponsa “are able to go on to have a stem cell transplant when compared with people having … other treatments” for relapsed or refractory CD22- positive B-cell precursor forms of the disease. Besponsa also meets NICE’s criteria for qualifying as a life-extending end-of-life treatment, another factor that scored it points with the body.

As Lilly does, though, Pfizer has to provide Besponsa with a secret discount on the £8,048-per-vial drug if it wants to stay in NICE’s good graces, NICE said.

https://bit.ly/2OstNix

CMS Proposes Rule for 2018 Risk Adjustment Payments

The Centers for Medicare & Medicaid Services (CMS) continued its push to reinstate risk adjustment payments to health insurance plans, releasing a proposed ruleto issue risk adjustment payments for the 2018 plan year.

Last month, the agency posted a final rule to reinstate the risk adjustment payments for the 2017 plan year. “The rule proposed today would allow the program to continue for the 2018 benefit year,” the agency said Wednesday in a press release.

The risk adjustment payments reimburse health insurers who sell individual or small-group policies both inside and outside the Affordable Care Act’s (ACA’s) health insurance exchanges. Health plans whose enrollees are healthier than average pay into the risk adjustment program, which then spreads the funds among plans with less healthy enrollees. The agency stopped the payments in early July due to an adverse federal court decision.

First, in January, the U.S. District Court for the District of Massachusetts ruled against a health insurance co-op that had sued over the risk adjustment formula. Judge Dennis Saylor held that the Department of Health and Human Services (HHS) was within its rights to set up the risk adjustment program. “In substance, the Court concludes that HHS acted within the bounds of its authority, even when the consequences of its choices may not always have been optimal,” Saylor wrote.

But in late February, the U.S. District Court for the District of New Mexico invalidated the payments, ruling in a case involving a New Mexico co-op that the use of statewide average premium data to calculate the payments was invalid. “The Court concludes that … HHS’ use of statewide average premiums in its risk adjustment methodology is not contrary to law, but is arbitrary and capricious,” wrote Judge James O. Browning.

At the time, CMS administrator Seema Verma said in a statement issued July 7th that she was “disappointed” by the New Mexico ruling. “As a result of this litigation, billions of dollars in risk adjustment payments and collections are now on hold,” she said. “CMS has asked the court to reconsider its ruling, and hopes for a prompt resolution that allows CMS to prevent more adverse impacts on Americans who receive their insurance in the individual and small group markets.”

“The New Mexico district court’s ruling currently bars CMS from collecting or making payments under the current methodology, which uses the statewide average premium,” the press release continued. “This aspect of the risk adjustment methodology was promulgated as part of a regulation first issued by the Obama administration in 2013. CMS will provide additional guidance shortly on how it will handle other issues relating to risk adjustment payments.”

In issuing the final rule for the 2017 benefit year, the agency “has determined that taking immediate action to allow for the continued operation of the risk adjustment program is imperative to maintain stability and predictability in the individual and small group health insurance markets,” CMS said in a statement. “Quick resolution also helps to preserve the significant investment made by states, issuers, and the federal government to stand up the program. This final rule reissues the risk-adjustment methodology previously established for the 2017 benefit year.”

The rule itself notes that the agency didn’t make any changes regarding its use of statewide average premium data — it’s just explaining the reasoning for its use better. “The district court recognized that use of [the] statewide average premium maintained the budget neutrality of the program, but concluded that HHS had not adequately explained the underlying decision to adopt a methodology that kept the program budget neutral, that is, that ensured that amounts collected from issuers would equal payments made to issuers for the applicable benefit year,” the rule stated. “Accordingly, HHS is providing additional explanation herein.”

The 2018 proposed rule “continues our effort to help stabilize the individual and small group markets,” Verma said in Wednesday’s press release. “Our goal has been, and will continue to be, to stabilize the market and provide American consumers with more affordable health coverage options.”

https://bit.ly/2Mjxn0S

Regenxbio’s wet AMD gene therapy shows promise in phase 1

Regenxbio reported promising interim data from a phase 1 trial of its gene therapy for wet age-related macular degeneration (AMD). The treatment, designed to combat vision loss caused by the formation of new blood vessels in the eye, reduced the need for in-eye injections of anti-VEGF drugs. The company is now looking to move the treatment into phase 2.

Considered incurable, AMD is treated with intraocular injections of drugs—such as Regeneron’s Eylea and Roche’s Avastin and Lucentis—that target VEGF (vascular endothelial growth factor), a protein that stimulates the growth of blood vessels. These drugs prevent the growth of leaky blood vessels in the eye that cause wet AMD’s signature scarring, death of photoreceptors and ultimately, vision loss. While these injections are the most effective treatment for wet AMD, getting regular injections into the eye can be unattractive for patients.

A one-time gene therapy would address this concern. Regenxbio’s RGX-314 uses an adeno-associated virus to deliver a gene that encodes a protein that neutralizes VEGF activity. The phase 1 study divided 18 patients into three groups that received three different doses of the gene therapy. The patients must have had a history of frequent anti-VEGF treatment, as well as a history of responding to these treatments, to qualify for the trial.

The study found the gene therapy to be safe and well-tolerated at all doses. All three groups had dose-dependent protein expression levels and dose-dependent reduction in anti-VEGF injections. The mean protein levels measured increased dramatically as the doses increased: the group receiving the lowest dose had a mean protein level of 2.4 ng/ml, while the higher doses posted levels of 12.8 and 160.2 ng/ml. Three patients in the group that received the highest dose did not need anti-VEGF injections in the six months after treatment. When compared to the six months prior to their most recent anti-VEGF injection, the injection rate for this group after gene therapy was reduced by 53%.

“We are very encouraged by the positive interim data for RGX-314 and the potential of NAV gene therapy as a one-time treatment for wet AMD, particularly as this is a nonrare patient population with a significant treatment burden,” said Kenneth Mills, Regenxbio’s president and CEO, in a release. “Regenxbio looks forward to applying what we are learning from this trial to expand the RGX-314 clinical program into a phase 2 trial and bring this novel therapy to patients as quickly as possible.”

Regenxbio will move the highest dose, 6 x 10¹⁰ genome copies (GC) per eye, into phase 2 and will introduce an even higher dose, 1.6 x 10¹¹ GC per eye, into the phase 1 trial. It plans to start the phase 2 study in 2019.

Despite the early promise of its wet AMD program, Regenxbio’s shares dipped Tuesday morning as the company reported some safety issues in a trial testing its gene therapy for homozygous familial hypercholesterolemia. Two patients had elevated levels of liver enzymes called transminases and one of them had to be hospitalized.

https://bit.ly/2OYwPvO

Perrigo Needs ‘Meet-Or-Beat’ Quarters, Deutsche Bank Says In Downgrade

Perrigo Company PLC, PRGO 0.21% was hit with a downgrade from Deutsche Bank after an eventful week that included the announced separation of its prescription pharmaceutical business and its second-quarter report.

The Analyst

Deutsche Bank analyst Gregg Gilbert downgraded Perrigo from Buy to Hold and lowered the price target from $93 to $78.

The Thesis

In light of soft Q2 results and a lowered outlook, Deutsche Bank is moving to the sidelines for Perrigo’s generics and consumer businesses, Gilbert said in the downgrade note. (See the analyst’s track record here.)

“The company’s strategic decision to separate the Rx business from the consumer business could be a good long-term decision, but it will at a minimum take some time for the market to fully assess and understand the financial and operational merits of this move and whether and how it can affect shareholder value,” the analyst said.

It’s important for Perrigo to reestablish “meet-or-beat” quarterly performance, Gilbert said.

In Q2, Perrigo reported an adjusted EPS of $1.22, a 4-percent decrease in revenue and a lowered prescription sales outlook.

The Deutsche Bank analyst outlined other key takeaways from the print:

• Perrigo recognizes the advantage of separating the companies.
• The pharma company is disappointed by the performance of its animal health business, but has not made any decisions on its future.
• The launch of gProair and scopolamine are confirmed.
• “The weakness in generics was based in part on pricing dynamics that were somewhat more negative than the company had expected,” Gilbert said.

https://bit.ly/2OZMxa1

Astellas Expands Gene Therapy Investment, Buys UK Quethera for $109M

Japan-based Astellas plunked down nearly $109 million to snap up privately-held gene therapy company Quethera Limited and its novel treatments for glaucoma and other ocular disorders.

The acquisition of U.K.-based Quethera strengthens Astellas’ gene therapy platforms for eye disease. In 2016, the Japanese company entered into a licensing agreement with CLINO Corporation for its gene therapy treatments for retinitis pigmentosa. CLINO was developing adeno-associated virus-modified volvox channelrhodopsin-1 for the treatment. Two years prior, Astellas forged a research agreement with Harvard Medical School investigator Constance L. Cepko for genetic treatments for retinitis pigmentosa, a degenerative disease caused by genetic mutations and characterized by loss of peripheral and night vision.

With the Quethera deal in hand, Astellas now has that company’s recombinant adeno-associated viral vector system (rAAV) platform to target glaucoma. Quethera’s lead pre-clinical candidate of the program has demonstrated significantly improved survival of retinal ganglion cells (RGCs) in pre-clinical models, Astellas said this morning. Quethera’s treatment is designed to be administered as a single injection on one occasion into the affected eye. On its website, Quethera said the rAAV vector system it is using has “been shown to be safe and does not produce a large inflammatory response after injection” That makes it an ideal platform for the Quethera gene therapies, the company said.

Kenji Yasukawa, president and chief executive officer of Astellas, said the deal for Quethera demonstrates his company’s commitment to harness state-of-the-art technologies and develop them for patient value.

“We believe the rAAV program has potential as a new therapeutic option for the treatment of refractory glaucoma through an intraocular pressure (IOP)-independent mechanism. It would address a high unmet medical need in glaucoma patients who are at risk of losing their eyesight,” Yasukawa said in a statement.

Peter Widdowson, chief executive officer of Quethera, said the deal with Astellas will enable the company to accelerate its evaluation of the rAAV technology program to see if it is successful in slowing or preventing disease progression. Widdowson, who co-founded Quethera in 2013, said his company’s novel approach is focused on exploring potential treatment options for common ophthalmic diseases, such as glaucoma, that can cause blindness and severely affect quality of life for patients.

Under terms of the deal, Astellas ponied up £85 million, just under $109 million U.S., in upfront and contingent payments. The company declined to provide information on what those milestone contingencies might be. When the deal is completed, Quethera will become a wholly-owned subsidiary of Astellas.

Astellas said the acquisition of Quethera will have “immaterial” impact on the company’s financial results for this year.

https://bit.ly/2MiGLlr