Gilead-Kite Yescarta 1st CAR T-cell Therapy to Get Euro OK for Use in Second-Line Lymphomas

 -- First Treatment in 30 Years to Improve Upon Standard of Care (SOC) for Second-Line Treatment of DLBCL –

-- Based on Landmark ZUMA-7 Study, Patients with DLBCL Treated Second-Line with Yescarta Had Event-Free Survival of 8.3 Months versus Two Months for SOC [4-fold greater improvement] --

-- In ZUMA-7, Yescarta Patients with DLBCL were 2.5 Times More Likely than SOC to be Alive at Two Years Without Cancer Progression or Need for Additional Treatments --

https://finance.yahoo.com/news/kite-yescarta-first-car-t-203000030.html

Pfizer takes Vyndaqel copay fight to SCOTUS, decrying 'staggeringly overbroad' anti-kickback rule

 After two legal defeats, Pfizer is taking its copay assistance lawsuit for costly rare heart disease drug tafamidis to the Supreme Court. Once again, the company is challenging the U.S. Department of Health and Human Services’ (HHS') interpretation of anti-kickback law.

Pfizer is asking the Supreme Court to decide whether a proposed program to help Medicare patients pay out-of-pocket costs for its tafamidis drugs—Vyndaqel and Vyndamax—would violate U.S. anti-kickback laws. An HHS opinion in 2019 ruled against Pfizer, finding that such an arrangement would break a criminal ban on financial support to patients for a federally reimbursed healthcare product. Pfizer sued to overturn the ruling, but two courts have since sided with the U.S. government.

As Pfizer sees it, the case represents “an optimal vehicle” for the Supreme Court to address the HHS’ “staggeringly overbroad” interpretation of U.S. anti-kickback laws, the company said in its petition (PDF). But critics have viewed Pfizer’s case as an attempt to weaken the HHS’ safeguards against illegal payment to Medicare patients.

The argument centers on whether the existing anti-kickback law requires an element of corrupt intent for financial support to Medicare patients to be considered illegal. Both the HHS and two lower courts believe ill intent is not needed for such payments to be deemed kickbacks, while Pfizer thinks it is.

The Vyndaqel/Vyndamax case is an ideal vehicle, Pfizer said, as its patient-assistance program for the therapy “poses no risk of corrupting independent medical decision-making.” That’s because the Vyndaqel/Vyndamax brands are currently the only FDA-approved drugs to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a rare, life-threatening heart disease that mostly happen in elderly people, or Medicare beneficiaries. So, the company argues, there’s no element of seeking to alter treatment decisions in favor of the Pfizer drugs over a rival.

But that window may be quickly closing. Alnylam looks on track to break into ATTR-CM after reporting positive phase 3 data for its Onpattro. The RNA silencing drug came to the U.S. market in 2018 bearing a list price of about $450,000 for another even rarer form of ATTR called neuropathy that affects the peripheral nerve system. Alnylam didn’t immediately respond to a Fierce Pharma request for comment.

Vyndaqel/Vyndamax currently cost $225,000 a year, and, under Medicare’s formula, patients are responsible for a co-pay of about $13,000 per year. Pfizer’s proposed assistance program would lower the out-of-pocket cost to $35 per month, according to the company. 

Pfizer argues that its copay assistance program would not “induce” improper utilization of the tafamidis drugs, but merely “influences” a patient’s ability to access the treatment. The HHS, however, argues it breaks the law because it would induce a patient to purchase tafamidis by removing a financial obstacle.

To Pfizer, HHS is presenting an overly broad interpretation of the anti-kickback statue that “outlaws a wide swath of routine, beneficial conduct” within Medicare. The statute’s “text, structure, and history,” such as the Congress’ use of the words “kickback, bribe, or rebate,” show that the law “targets only payments made to corrupt decisionmaking,” Pfizer contends. HHS’ move is also “out of step” with the Supreme Court’s “longstanding efforts to ensure that criminal laws do not sweep more broadly than Congress intended,” the New York drugmaker said.

The Supreme Court has until Nov. 14 to decide whether to take on the case. A win for Pfizer would have detrimental effects on drug pricing controls in the U.S., lawyers previously told Fierce Pharma.

Since cost sharing is “really the only check against pricing,” if drugmakers were allowed to “greatly reduce or even eliminate those cost-sharing obligations, there would be essentially no economic cap on pricing,” Bass, Berry & Sims lawyer Jennifer Michael said in an interview last year. Michael previously led the industry guidance branch counseling the HHS' Office of Inspector General.

https://www.fiercepharma.com/pharma/pfizer-takes-vyndaqel-copay-fight-supreme-court-crying-staggeringly-overbroad-anti-kickback

Salix report uncovers climbing death rates from chronic liver disease

 Hospitalizations and deaths from chronic liver disease (CLD) continue to rise, according to a new report from Salix Pharmaceuticals.

The pharma’s annual "Liver Health Annual Trends Report" also revealed a lack of knowledge and proper care for patients with liver disease, resulting in CLD overtaking diabetes and stroke as the No. 1 cause of death among people ages 25 to 54.

As many as 34% of gastroenterologists reported increases in patient hospitalizations due to alcoholic cirrhosis (scarring) of the liver in the past year.

More than half (55%) of primary care physicians and 49% of nurse practitioners/physician assistants surveyed reported feeling uncomfortable treating overt hepatic encephalopathy (HE) and preferring outside consult. Salix, which markets Xifaxan for HE, wants to overcome this.

These data reveal a gap in the adoption of national treatment guidelines, particularly among primary care physicians. The report surveyed 400 healthcare providers who treat patients with CLD, along with qualitative and secondary research, to try to reveal the biggest hurdles to improving diagnosis and treatment for patients with CLD.

Perceived issues with payer coverage and the high cost of out-of-pocket treatment were the leading factors impacting treatment initiation and treatment interruptions. In addition, 50% of providers report that the ability of a patient to pay out-of-pocket for treatment is extremely important for improving CLD patient outcomes.

The annual report was released in October during Liver Health Awareness Month as part of an effort to better educate physicians on identifying and managing patients with CLD and cirrhosis. This report is timely, as 38% of respondents admitted to not knowing the national guidelines for the management of CLD.

“It is our hope the Salix Liver Health Annual Trends Report will increase awareness of this growing public health concern, help bridge the knowledge gap, and provide tools for healthcare providers that may improve identification and management of liver disease,” Nicola Kayel, vice president, marketing at Salix Pharmaceuticals, told Fierce Pharma Marketing.

“[It] may help bring a better understanding of the impact of chronic liver disease and potentially help address under-diagnosis and under-treatment among the approximately 4.5 million adults in the U.S. living with the disease.”

While the full annual report can be found at LiverHEalthNow.com, the platform also houses resources for healthcare providers to help identify and manage patients with CLD and cirrhosis and foster positive physician-patient conversations.

A second site, UnderstandingHE.com, offers patients/caregivers information and resources on working with their providers to manage their HE and complications with cirrhosis. A third site, UnseenEpisodes.com, is for the healthcare provider side of treatment of patients with HE.  

“Salix remains committed to delivering resources that inform, educate and help healthcare providers improve the care of people living with chronic liver disease, cirrhosis and complications such as hepatic encephalopathy,” Kayel said.

Moving forward, Salix remains committed to providing education not only to physicians but all to healthcare providers along with patients and caregivers.

“Supporting a wide range of GI educational programs that aim to improve GI health, we view education as a key underpinning to who we are as a company,” Kayel said. “We look to support both patients and physicians with impactful resources so that all healthcare providers can continue to support their patients to the best of their abilities as well as provide resources and educational materials to patients so that they can have informed discussions with their providers.”

https://www.fiercepharma.com/marketing/salix-report-uncovers-climbing-death-rates-chronic-liver-disease

Prosecutor confirms probe into Europe's COVID-19 vaccine purchasing

 Amid intense public scrutiny of the European Commission's large deal to purchase Pfizer COVID-19 vaccine doses, prosecutors there appear to be doing some digging of their own.

The European Public Prosecutors Office (EPPO) has confirmed that it has an “ongoing investigation” into the acquisition of COVID-19 vaccines in the EU. The “exceptional confirmation” comes after “extremely high” public interest in the issue, the EPPO’s statement said. The prosecutor didn't name any vaccine company in its brief statement.

The intense public interest likely refers to the situation around the Pfizer COVID-19 supply deal in Europe. In recent weeks, some EU officials have pressed for answers about how authorities ended up striking a massive supply deal with the drugmaker and its partner BioNTech for up to 1.8 billion doses.

Last week at a European Parliament hearing, a Pfizer executive who participated in vaccine contract negotiations with the European Commission categorically denied allegations that the company's CEO, Albert Bourla, Ph.D., negotiated the contract through text messages. The executive was Janine Small, the company’s president of international developed markets.

Such vaccine negotations are typically too detailed and involve too many parties to be conducted via text messages, Small noted.

At the hearing, Small was sitting in for Bourla, who was originally slated to testify before the parliament’s COVID-19 committee before he pulled out.

The hearing came a few weeks after a European Court of Auditors report found that a joint negotation team was not involved in striking the Pfizer vaccine deal. That was a violation of a previously agreed protocol, the Court of Auditors said.

What's more, Europe's contract for the Pfizer doses was the biggest COVID-19 contract signed by the bloc. Those doses will dominate the EU’s vaccine portfolio until the end of 2023, the Court of Auditors said.

The EPPO concluded its statement by noting that no further details will be made public at this stage of the investigation.

https://www.fiercepharma.com/pharma/european-public-prosecutors-office-confirms-ongoing-investigation-european-unions-covid-19

White House: 8M have applied for student loan debt forgiveness

 President Biden on Monday lauded the official launch of student loan forgiveness applications, with the White House touting that more than 8 million Americans have already applied for the administration’s program during an online soft launch that began late last week.

“Today marks a big step, among others, that my administration is taking to make education a ticket to the middle class that folks can actually afford,” Biden said. “The new student loan application is now open. If you have federal student debt, please visit StudentAid.gov. It’s easy, simple and fast and it’s a new day for millions of Americans all across our nation.”

Biden said the website, which soft launched on Friday, has handled applications “without a glitch or any difficulty.” The president, who was joined for the announcement by Education Secretary Miguel Cardona, called the portal a “game changer” that would allow the government to better serve the public.

The beta testing phase that launched on Friday allowed applications to be submitted, but they were not processed until the formal launch.

The president’s student loan forgiveness program, which he unveiled in August, is set to forgive $10,000 in federal student loan debt for borrowers earning under $125,000 and $20,000 for borrowers who received Pell Grants.

Since the announcement, the administration updated the plan to exclude borrowers with privately held federal student loans, saying loans not held by the Education Department are no longer eligible. Cardona said on Monday they are “working on pathways” to support those borrowers who are excluded.

“We’re moving as quickly as possible to provide relief to as many as possible,” the secretary added.

Several conservative groups and Republican state officials have filed lawsuits seeking to end the loan forgiveness program, though the filings have thus far failed to proceed because of lack of standing over who is harmed by the policy, which is optional. Biden noted in his remarks that litigation against the program is underway, adding “our legal judgment is that it won’t” stop the program.

Biden also took a jab at Republicans for criticizing the student loan forgiveness program for being too costly, arguing that it will be paid for because of his efforts to bring down the deficit.

White House press secretary Karine Jean-Pierre earlier on Monday said the administration has “seen an overwhelmingly positive response as well as a strong website performance since we began testing the site this past Friday night.”

Jean-Pierre added that the Department of Education’s technical team will continue to monitor the site’s performance in real time.

https://thehill.com/homenews/administration/3693141-white-house-says-8-million-americans-have-applied-for-student-loan-debt-forgiveness/

US warned to get ready as Europe deals with new COVID-19 rise

 Rising COVID-19 cases in Europe are setting off warnings that the U.S. could experience a new surge this winter. 

Previous jumps in the U.S. have followed a pattern in which cases first rise in Europe, making officials nervous they could see a spike in U.S. cases as the weather turns.

The most recent data from the European Centre for Disease Prevention and Control shows that cases began going up around the beginning of September in Europe.  

The seven-day average is roughly 230,000 cases per day, reflecting rates that were seen in late July when Europe was still dealing with the omicron BA.4/BA.5 subvariant wave. 

World Health Organization Director-General Tedros Adhanom Ghebreyesus stated last week that a rise in cases in Europe was expected due to cooling temperatures, but stressed that hospitalizations and deaths did not have to rise as well due to the viral therapeutics that are now available. 

Confirmed coronavirus-related deaths across the European Union have remained low, with a seven-day average of 280 as of last week. 

In the U.S., cases and deaths have continued to trend down, but dropping temperatures that push people indoors, dismal booster vaccination numbers and an overall disregard for pandemic mitigation practices are setting the stage for a winter wave resembling the one across the Atlantic. 

The seven-day moving average for cases in the U.S. is around 38,000, while the seven-day moving average for deaths is about 330. 

Ali Mokdad, epidemiologist and professor of Health Metrics Sciences at the Institute for Health Metrics and Evaluation at the University of Washington, told The Hill the contrast in the regions can be attributed to multiple factors, including warmer temperatures in the U.S. and differing levels of community immunity. 

“In the U.S., we have a higher infection rate than many European countries, where more people have been infected here. So we have a little bit more immunity than they do, but still we have waning immunity,” Mokdad said. 

COVID-19 cases, deaths and hospitalizations in the U.S. will likely begin going up in three to four weeks, Mokdad said, though they won’t reach the same levels seen during the omicron wave last winter. He emphasized that this projection is contingent on a situation in which new coronavirus variants that are better at escaping immunity don’t rise in dominance. 

Researchers and virologists have continually stated that the risk of a more infectious variant still looms as the SARS-CoV-2 virus continues to spread and mutate. 

The COVID-19 Scenario Modeling Hub, a U.S.-based data collective operated by researchers and public health experts, publishes possible directions in which the pandemic could go based on multiple models. 

The most recent projections from the data hub present four scenarios: two in which no new variants occur and two in which they do.

Shaun Truelove, assistant scientist at the Johns Hopkins Bloomberg School of Public Health and a member of the COVID-19 Scenario Modeling Hub team, said he is leaning away from the potential scenarios where no new variants arise. 

“We are seeing variants, we are seeing that they are emerging and it seems like we will have one of those,” said Truelove. 

The BA.5 omicron subvariant, the dominant strain in the U.S., has begun losing its foothold in the U.S., with sublineages like BA.4.6 and BQ.1.1 growing in prevalence. In some parts of the Midwest, BA.4.6 now accounts for a fifth of COVID-19 cases. 

The differences in community immunity levels between the U.S. and Europe presents a challenge in projecting where the American infection rates will go based on overseas observations, according to Truelove. 

“We have a situation where people are constantly being infected, so their immunity from that infection or from vaccination is waning. And then at the same time as all that’s happening, we’re also seeing the influx of these new variants that have these immune escape properties,” he said. 

Apart from COVID-19, there are other bits of information that can be gleaned from Europe’s situation that could help forecast what the U.S. will experience in the upcoming months. 

Health officials have warned that the flu will likely be worse than it has been in recent years due to lack of exposure, possibly resulting in a “twindemic” of both viruses. Mokdad noted that a spike of influenza infection in Europe has not yet been observed amid the coronavirus surge. 

“We haven’t seen a spike of cases in Europe right now. … So some encouraging news for the flu, but I wouldn’t bet on it,” said Mokdad. 

While U.S. influenza cases have been rising, only 3.3 percent of lab-tested specimens have come back as positive, according to the Centers for Disease Control and Prevention. Recent polling from the National Foundation for Infectious Diseases found that 49 percent of U.S. adults plan on getting the flu shot this year, only 5 points lower than the percent of adults who got the shot last year.

The prevalence of people getting vaccinated and practicing viral mitigation methods like masking and social distancing has largely fallen out of favor. President Biden said in September that the pandemic was “over,” a move that Mokdad partly cited for the slow booster uptake. 

“President Biden said the pandemic is over, but that’s a mistake for me at a time when we’re trying to push a booster for a new vaccine designed for BA.4 and BA.5 and he goes and says it’s over. So why would people go and get the vaccine?” Mokdad said. “It’s very hard for me to say COVID-19 is over. It’s not over, especially right now coming into winter.” 

Experts who spoke with The Hill strongly encouraged people to get the updated bivalent booster ahead of the holiday season. 

“The best holiday present that you can give — whatever you celebrate — that you give for yourself and your family members is protection and safety. And the best way to do it is to go and get your booster and your flu shot,” Mokdad said. 

https://thehill.com/policy/healthcare/3693142-us-warned-to-get-ready-as-europe-deals-with-new-covid-19-rise/

House Dems move to reaffirm FDA authority on abortion pill access

 House Democrats on Monday introduced a resolution to reaffirm the Food and Drug Administration’s (FDA) authority to preempt state law and ensure patients continue to have access to reproductive health care products.

The resolution from Reps. Diana DeGette (D-Colo.) and Mondaire Jones (D-N.Y.) reaffirms the FDA’s authority to prevent states from enacting regulations that limit or prohibit patients from accessing reproductive health products approved by the agency, including abortion pills delivered directly to patients.

It also emphasizes the authority of the U.S. Attorney General to take action against any state that enacts a law that prohibits or limits a patients’ ability to access or use such products.

There are two pills needed for a medication abortion within the first 10 weeks of pregnancy. The FDA first approved such pills nearly 20 years ago.

Medication abortion accounted for 54 percent of all pregnancy terminations in the U.S. in 2020.

Attorney General Merrick Garland in June indicated the Justice Department will take action against states that ban abortion pills, though it’s unclear if the federal government has that authority. 

So far, more than a dozen states have enacted new laws to limit, or outright ban, patients’ access to reproductive care in the wake of the Supreme Court’s decision to overturn Roe v. Wade.  

The FDA temporarily lifted a requirement that mifepristone — a common abortion medication — be dispensed in-person at a clinic or hospital because of the COVID-19 pandemic, and the Biden administration made the change permanent in December 2021, paving the way for doctors to prescribe the drug digitally and then mail the pills to patients.

The resolution is endorsed by Planned Parenthood Federation of America, the National Women’s Law Center, the Center for Reproductive Rights, and other national organizations, and was sponsored by 28 other House members.

https://thehill.com/policy/healthcare/3693202-house-democrats-move-to-reaffirm-fda-authority-on-abortion-pill-access/