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Sunday, October 2, 2022

Court orders HHS to vacate 340B payment discounts through 2022

 340B hospitals picked up a win Wednesday when a federal judge ordered that “defective” payment cuts for the drug discount program be tossed for the remainder of 2022.

U.S. District Judge Rudolph Contreras’ ruling addresses the first of two remedy questions stemming from June’s Supreme Court decision, in which the top court overturned a nearly 30% rate cut adjustment the Department of Health and Human Services (HHS) first introduced in 2018.

The Supreme Court unanimously rejected HHS’ argument that it did not need to survey hospitals’ acquisition costs before introducing the adjustment. The case was returned to lower courts to address potential remedies.

Hospital industry plaintiffs including the American Hospital Association (AHA), the Association of American Medical Colleges and America’s Essential Hospitals filed motions seeking to remedy years of unlawful underpayments to 340B hospitals and to vacate the 340B reimbursement rate of the Outpatient Prospective Payment System (OPPS) rule still in effect through the end of the year.

Addressing the latter, Contreras granted hospitals’ motion to vacate the 2022 reimbursement rate without injunction.  

The judge said HHS’ claims that vacatur of the 2022 rate would be disruptive were persuasive. HHS itself admitted that the remainder of 2022 accounts for “only … a small sliver of the overall time periods challenged in this action,” Contreras wrote, and the department has already begun taking steps to address a potential budgetary shortfall resulting from the Supreme Court’s ruling.

“In short, the Court finds that any disruption that will be caused by vacating the prospective portion of the 2022 OPPS Rule's 340B reimbursement rate does not rise to the level of justifying remand without vacatur,” the judge wrote.

Hospital industry stakeholders applauded the court’s decision.

“This is an important victory for 340B hospitals that have been fighting these unlawful Medicare cuts for nearly six years,” Maureen Testoni, president and CEO of 340B Health, which counts 1,400 hospitals as members. “The Centers for Medicare & Medicaid Services (CMS) has the clear responsibility to restore the appropriate payments for 340B drugs immediately, and now a federal court has ordered it to do so without delay.”

The immediate halt will help hospitals continue to provide services to their communities, AHA General Counsel and Secretary Melinda Hatton said in a statement already looking ahead to the court’s next decision.

“We continue to urge the administration to promptly reimburse all the hospitals that were affected by these unlawful cuts in previous years and to ensure the remainder of the hospital field is not penalized for their prior unlawful policy, especially as hospitals and health systems continue to deal with rising costs for supplies, equipment, drugs and labor,” she said.

AHA submitted several court filings in August warning against a budget-neutral recoupment plan that would require hospitals unaffected by the cuts to foot the bill.

https://www.fiercehealthcare.com/providers/court-orders-hhs-vacate-340b-payment-discounts-through-2022-recoupment-prior-years-yet-be

Lexicon: New Data on Sotagliflozin's Effect on Reducing Recurrent Heart Failure

  • Data presented at the Heart Failure Society of America annual scientific meeting
  • New analysis adds to growing body of evidence in support of the use of sotagliflozin, an investigational dual SGLT1 and SGLT2 inhibitor, in the treatment of heart failure


MeiraGTx: Phase 1/2 Data Show Safety, Vision Improvement in Retinitis Pigmentosa Gene Therapy

 MeiraGTx Holdings plc (Nasdaq:MGTX), a vertically integrated, clinical-stage gene therapy company, announced today the primary results from the Phase 1/2 study evaluating the investigational gene therapy botaretigene sparoparvovec (formerly AAV-RPGR) in patients with the inherited retinal disease X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene.

Treatment with botaretigene sparoparvovec was found to have an acceptable safety profile and efficacy assessments in this proof-of-concept study demonstrated improvements in retinal sensitivity, visual function and functional vision.1 These findings were presented in a late-breaking oral presentation today at the Retina Subspecialty Day program of the American Academy of Ophthalmology (AAO) 2022 Annual Meeting (Abstract #30071754) by Professor Michel Michaelides.

https://www.marketscreener.com/quote/stock/MEIRAGTX-HOLDINGS-PLC-44154954/news/Late-Breaking-Phase-1-2-Data-Demonstrates-Safety-Profile-of-Investigational-Gene-Therapy-Botaretigen-41905060/

Legend Biotech to Host 2022 R&D Day on Monday, October 3

 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, today announced it will host its in-person and virtual Research & Development Day on Monday, October 3, 2022 at 10:00 a.m. ET at Andaz 5th Avenue in New York City. Please click here to register for the event.

The event will feature a comprehensive overview of our platforms and the latest developments in our pipeline through presentations from members of Legend’s senior leadership team:

  • Ying Huang, Ph.D., Chief Executive Officer

  • Guowei Fang, Ph.D., Senior Vice President, Global Head of Research and Early Development

  • Lida Pacaud, M.D., Vice President, Clinical Development

  • Steve Gavel, Vice President, Commercial Development, US & Europe

A live webcast and presentation will be available to investors and other interested parties on the Legend Biotech website under "Events and Presentations". A replay of the webcast can be viewed as early as 24 hours after the event.

About Legend Biotech

Legend Biotech is a global biotechnology company dedicated to treating, and one day curing, life-threatening diseases. Headquartered in Somerset, New Jersey, we are developing advanced cell therapies across a diverse array of technology platforms, including autologous and allogenic chimeric antigen receptor T-cell and natural killer (NK) cell-based immunotherapy. From our three R&D sites around the world, we apply these innovative technologies to pursue the discovery of cutting-edge therapeutics for patients worldwide.

https://finance.yahoo.com/news/legend-biotech-host-2022-r-123000880.html

Universal Health Services : Patriot Support Programs Expands Military Advisory Board

 The Universal Health Services Patriot Support Programs provide treatment for active duty military, veterans and their families. Programs and services are specifically designed to address the effects of combat stress, post-traumatic stress, depression, substance use disorder and other behavioral health issues.

We announce the expansion of the Patriot Support Programs Advisory Board, welcoming new member Major General (Ret) Jerry L. Fenwick, U.S. Air Force, and Air National Guard. General Fenwick retired in 2021 after 35 years of service in both the U.S. Army and Air Force. His final assignment was as Director, Office of the Joint Surgeon and Surgeon General of the National Guard, Washington, D.C. His career as an Emergency Medicine Physician has included multiple deployments with a focus on austere medicine and patient movement. Following retirement, Dr. Fenwick became the Chief Medical Officer for Disaster and International Medicine for Longview Technology, Herndon, VA. He is also CEO of Marshall Creek Medical Consulting and Medical Director of 417 Healthcare, Springfield, MO.

"During 2021, we served more than 18,000 active-duty military personnel, veterans and family members across the Behavioral Health division, including through our designated Patriot Support Programs," said Matt Peterson, Executive Vice President and President, Behavioral Health Division. "With a focused and dedicated Advisory Board, further expanded to include the wisdom and talents of Major General (Ret) Fenwick, we will continue to enhance our offerings to ensure military personnel receive the support and care they need to succeed. I have personally served with General Fenwick and can speak to his relentless commitment to saving lives."

The Advisory Board is comprised of:

  • Master Chief Petty Officer of the Navy (Ret) Joe Campa, U.S. Navy
  • David Frost, Vice Admiral (Ret), U.S. Navy
  • The Honorable Michael Kussman, MC, Under Secretary for VA Health (Ret), Brigadier General, U.S. Army
  • Rear Admiral (Ret) William McDaniel, MC, U.S. Navy
  • James Roudebush, MC, Lieutenant General (Ret), U.S. Air Force

Telehealth startup allegedly treated minors without parents' consent

 Telehealth startup Cerebral has been accused of prescribing minors prescriptions without the consent of their parents, The Wall Street Journal reported Sept. 29. 

Cerebral has a software that verified customers' IDs, but the software didn't identify patients' ages, according to former employees and documents reviewed by the Journal. Clinicians were allegedly supposed to verify patient ages during 30-minute video chats. 

"We have provided much-needed care to hundreds of thousands of patients, many of whom would not have had access to critical mental-health support without Cerebral's telemental health services," Cerebral told the Journal

Cerebral also said it tested different ways to verify IDs in its registration process and has always met legal requirements.

A 17-year-old from Missouri, Anthony Kroll, signed up for Cerebral and went through the ID software, which showed he was a minor. 

He proceeded to meet with a clinician, stating that he had suicidal thoughts, and she prescribed him an antidepressant that had a warning label for adolescents, according to medical records reviewed by the Journal. Mr. Kroll later died by suicide.

Mr. Kroll's parents said they did not know he was seeking mental health treatment nor were they made aware that he was taking an antidepressant. 

Missouri law prohibits clinicians from providing mental health treatment to people under 18 without parental consent. 

Cerebral said Mr. Krol misrepresented his age. 

The company later identified 17 minors who enrolled in their services between May 2021 and April 2022, including Mr. Krol. 

According to the documents and employees, the company enrolled people with bipolar disorder, suicidal thoughts and other serious conditions, giving some patients appointments without properly vetting their IDs. Cerebral has said it no longer prescribes most controlled substances to new patients, according to the Journal.

https://www.beckershospitalreview.com/telehealth/telehealth-startup-allegedly-treated-minors-without-parents-consent.html

Higher Dose, Longer Treatment With Eylea Shows Promise in Eye Disease

 Bumping up the dose of aflibercept (Eylea) led to prolonged treatment intervals for neovascular eye disease without a loss in efficacy or major increase in adverse events, two randomized trials showed.

Dosing the VEGF inhibitor at 8 mg every 12 or 16 weeks achieved non-inferiority to treatment with 2 mg every 8 weeks with respect to best-corrected visual acuity (BCVA) in patients with diabetic macular edema (DME). More than 90% of patients randomized to 8 mg maintained the 12-16 week dosing interval through 48 weeks, according to a presentation at the American Academy of Ophthalmology (AAO) meeting.

Also at AAO, investigators presented results from a separate randomized trial involving patients with neovascular age-related macular degeneration (AMD). It showed that 8 mg every 12 or 16 weeks achieved BCVA similar to 2 mg every 8 weeks at 48 weeks.

PHOTON

"The 48-week results show the non-inferiority of 8 mg Q12 or Q16, despite greatly increased dosing intervals," said David Brown, MD, of Retina Consultants of Texas in Houston. "Remarkably, 91% of patients can be maintained on Q12 and 89% of Q16 and overall, 93% on Q12 or greater. We hope to get this benefit out to our patients in the coming future."

Brown reported initial results from the phase II/III PHOTON randomized trial evaluating a novel 8-mg formulation of aflibercept. The four-times higher molar dose compared with standard aflibercept dosing is hypothesized to achieve a longer effective vitreal concentration and more sustained inhibition of VEGF signaling.

Investigators in the PHOTON trial randomized 658 patients with untreated DME 1:2:1 to intravitreal aflibercept 2 mg every 8 weeks (2q8), 8 mg every 12 weeks (8q12), or 8 mg every 16 weeks (8q16). Patient age was around 62, about 39% were female, and about 72% were white.

Patients in the 2q8 arm initially receive five monthly doses of aflibercept whereas patients in the two 8-mg arms received three monthly injections. Baseline characteristics did not differ significantly among treatment groups, including a mean hemoglobin A1c of 8% across the three groups.

The primary endpoint was change in BCVA from baseline at 48 weeks, and the trial was statistically powered for non-inferiority. The primary analysis showed improvement in BCVA of 8.7 letters with the 2q8 group, 8.1 letters in the 8q12 group (P<0.0001) and 7.2 letters in the 8q16 group (P=0.0031), meeting prespecified statistical criteria for non-inferiority.

Maintenance of dosing intervals in the experimental arms was a secondary endpoint. The results showed that 91% of patients randomized to 8q12 remained at that interval, as did 89% of patients randomized to 8q16. Overall, 93% of patients randomized to 8 mg maintained dosing intervals ≥12 weeks, said Brown.

Change in central retinal thickness also did not differ significantly among the three groups, ranging from -148 µm in the 8q16 arm to -172 µm in the 8q12 arm.

Rates of adverse events (AEs, all grades) averaged 31.0% across the three treatment arms and did not differ significantly. Rates of individual types of AEs also did not differ across the treatment groups. The incidence of intraocular inflammation averaged 0.8% and did not differ significantly among the treatment groups.

AAO panelist Shlomit Schaal, MD, PhD, of the University of Massachusetts in Worcester, pointed out that the higher dose of aflibercept produces the same number of anti-VEGF molecules as bevacizumab (Avastin) after 4 weeks.

"Wouldn't you think that rather than increasing the dose, you should focus on other mechanisms of the disease in DME, not just increasing the same molecule?" she asked Brown.

Acknowledging the presence of "millions of cytokines produced by diabetic eyes" as potential treatment targets, Brown said anti-VEGF remains the "big gorilla." Any strategies that can increase dosing intervals, even modestly, will make a "big difference in this working-class population that has to take off school or get a substitute teacher. Anything we can do to decrease treatment burden [is helpful]."

PULSAR

The same three dosing intervals were evaluated in the phase III PULSAR trial, which included 1,009 patients with untreated neovascular AMD, randomized 1:1:1 to the three dosing schedules. The primary endpoint was change from baseline to 48 weeks in BCVA, said Paolo Lanzetta, MD, of the University of Udine in Milan. Overall, 93.3% of patients (age around 74, more than half female, more than three-fourths white) completed the 48 weeks of treatment and follow-up.

At 48 weeks, all three groups achieved improvement in BCVA from baseline: 7.0 letters with 2q8, 6.1 letters with 8q12, and 5.9 letters with 8q16. The 8q12 (P=0.0009) and 8q16 (P=0.0011) treatment groups demonstrated non-inferiority to the 2q8 group. Overall, 83% of patients randomized to 8 mg injections of aflibercept maintained ≥12-week dosing intervals through 48 weeks.

Significantly more patients in both 8-mg groups had no retinal fluid in the center subfield at 16 weeks, a secondary endpoint. The percentage of patients without fluid in the center subfield was 63% in the two 8-mg groups combined versus 52% in the 2q8 group (P=0.0002).

Mean reduction in central retinal thickness from baseline was 147 µm in the 8q16 group, 142 µm in the 8q12 group, and 126 µm in the 2q8 group.

The incidence of AEs was similar across the treatment groups.


Disclosures

The trials were supported by Regeneron/Bayer.

Brown disclosed relationships with Regeneron/Bayer and Genenteh/Roche.

Lanzetta disclosed relationships with Aerie, Allergan, Apellis, Bausch & Lomb, Bayer, Biogen, Boehringer Ingelheim, I-Care, Genentech, Novartis, Outlook Therapeutics, and Roche.