Texas Children's Hospital Puts Gender-Affirming Care on Pause

 Texas Children's Hospital in Houston -- the nation's largest pediatric hospital -- will no longer provide gender-affirming care, including hormone therapies, to its transgender and nonbinary patients.

The decision came in response to a recent order from Texas Gov. Greg Abbott (R) that called upon state welfare agencies to investigate pediatric gender-affirming care as child abuse, adding that "criminal penalties" would be issued to parties who failed to report cases involving such care.

In an emailed statement to MedPage Today, a spokesperson for Texas Children's Hospital wrote that the hospital's mission "is to create a healthier future for all children, including transgender children, within the bounds of the law."

"After assessing the Attorney General's and Governor's actions, Texas Children's Hospital paused hormone-related prescription therapies for gender-affirming services. This step was taken to safeguard our healthcare professionals and impacted families from potential criminal legal ramifications," the spokesperson added.

It remains unclear if any other forms of gender-affirming care will continue to be offered at the facility.

Abbott's order cited a legal opinion from the state's Attorney General Ken Paxton (R), who wrote that certain types of care -- including body modification surgeries, as well as hormone therapies and puberty blockers -- for trans/nonbinary youth are considered abuse under Texas law.

"There is no doubt that these procedures are 'abuse' under Texas law, and thus must be halted," said Paxton in a statement. "The Texas Department of Family and Protective Services (DFPS) has a responsibility to act accordingly. I'll do everything I can to protect against those who take advantage of and harm young Texans."

Surgical interventions for trans/nonbinary youth are extremely rare; any type of surgery typically associated with transitioning is only permitted when a person reaches the age of consent, has lived as their desired gender for at least a year previously, and has been approved by doctors and therapists.

Gender-affirming care for children mainly centers on the "social transition," which involves making non-permanent changes to one's gender expression, such as changing the way you dress, tucking to hide the appearance of genitals, or using a binder to flatten breasts; coming out to friends and family; and/or changing your name on legal documents.

Trans/nonbinary youth entering adolescence may have the option to take puberty blockers, or GnRH agonists, if they meet a number of criteria laid out in the World Professional Association for Transgender Health (WPATH) Standards of Care. According to WPATH, the use of GnRH agonists to suppress estrogen or testosterone production is a fully reversible intervention. Hormone therapies, which are partially reversible depending on what measures are taken, are reserved for older adolescents.

"There is no evidence that long-term mental health outcomes are improved or that rates of suicide are reduced by hormonal or surgical intervention," Paxton wrote in his legal opinion. This claim differs significantly from the existing literature, including a study published last month in JAMA Network Open that found gender-affirming medical interventions for children were associated with lower rates of depression and suicidality.

Paxton's legal opinion also quoted a claim published on the website for the Society for Evidence-Based Gender Medicine -- a group that has been accused of spreading misinformation about gender dysphoria and gender-affirming care -- which stated that "childhood-onset gender dysphoria has been shown to have a high rate of natural resolution, with 61-98% of children reidentifying with their biological sex during puberty."

The statistic cited a 2013 article published in the Journal of the American Academy of Child and Adolescent Psychiatry. However, this study in full showed a strong association between the intensity of a child's gender dysphoria and the persistence of gender dysphoria later in life.

Texas Children's Hospital isn't the first healthcare facility in Texas to bend at the will of conservative backlash and threats of legal action. In November 2021, a Dallas-based program known as GENECIS that provided hormone therapies to trans youth was formally dissolved after being accused of child abuse.

https://www.medpagetoday.com/special-reports/exclusives/97562

CDC needs serious scrutiny to restore Americans' trust in science

 Just when it looked like the Centers for Disease Control and Prevention’s (CDC) COVID management and messaging was going to get some long-overdue oversight, President Biden made a move to protect this gargantuan example of failed bureaucracy with a proposed $22 billion in new spending for the agency, doubling its budget.

Polling last summer showed that many Americans have lost faith in the CDC. Only 32 percent of respondents in an August Gallup poll said they believed the CDC was communicating a clear path to prevent COVID infection. Pew data from January showed 60 percent of respondents found CDC recommendations confusing.

While the CDC was losing credibility, the Government Accountability Office (GAO), Congress’s watchdog, was quietly investigating the agency’s handling of the COVID crisis. On Jan. 27, the GAO delivered a rare “high risk” rating to the Department of Health and Human Services, specifically citing the CDC’s poor data management and ineffective messaging. These findings added fuel to congressional proposals to set up an independent COVID commission to examine the CDC’s performance.

The CDC’s antiquated information system, designed in 1925, could not support modeling to show how a pandemic might spread, or a real-time picture of how the COVID crisis was developing. This information would have been useful to developing an effective containment strategy. Yet, the CDC’s estimates were always days old.  

By itself, this merits bad marks. We now know, however, from reporting by The New York Times, that the CDC was purposefully hiding information all the while on infection rates, hospitalizations and deaths attributable to COVID, likely making the crisis appear even more severe. And a recent study by economists at Johns Hopkins found that masking and social distancing — central in the administration’s approach to containing the spread of COVID — were completely ineffective. 

Ironically, while the CDC has yet to acknowledge that much of its advice was misleading, not grounded in science, or simply wrong, the U.S. surgeon general, who heads the U.S. Public Health Service’s uniformed corps, just announced his intention to force social media companies to disgorge sources of “misinformation” that appear on their platforms.

The CDC is in dire need of top-to-bottom reform. Consider the damage it did to the public’s trust in science with its mantra of (in effect) “We follow the science and it is always changing.” This rhetorical cover for political science — not virology — rightly could cause citizens to doubt expert guidance in the future.

The problem may be that the agency no longer appears to believe in its own special brand of science. The modern era’s public health knowledge base began in 1854, when a London physician stopped a cholera epidemic by mapping infected people and concluding that water in a specific well was causal. This began a long process of learning by trial and error, leading to a universally agreed-upon set of steps to control communicable diseases: case-finding, contact-tracing and quarantine. But in attempting to contain COVID, contemporary public health officials pursued these steps with a half-heartedness that would have scandalized previous generations of epidemiologists. Instead, they hung their hopes on a new vaccine. 

The failure of the CDC to manage COVID-19 more effectively points to an endemic problem of bureaucracy — namely, seeking to appear more relevant as a means to grow bigger. Over the last three decades, the CDC has de-emphasized its principal mission of protecting Americans from novel microbes and viruses that, if uncontrolled, can kill millions. Instead of modernizing its information systems, to choose but one example, the agency has sought a role in solving more high-profile social problems. Shamefully, it has encouraged a promiscuous use of the term “epidemic” to describe conditions that its methodologies are powerless to affect, including racism, gun violence, child abuse, sex trafficking, opioid abuse, obesity and loneliness. The CDC won’t be the agency that ends those crises, and trying to do so only distracts from its critical core mission. 

The enormous infusion of funds headed to the CDC is part of the president’s newly unveiled COVID National Preparedness Plan, a document that seems a little late in coming. With its publication, the White House spares the CDC critical examination of its performance. Rewarding failure, however, will surely result in an even less effective response to the next pandemic.  

Carl Schramm is a professor at Syracuse University and is a senior adviser to the COVID Commission Planning Group. His most recent book is “Burn The Business Plan” (Simon and Schuster, 2018).

https://thehill.com/opinion/healthcare/597393-cdc-needs-serious-scrutiny-to-restore-americans-trust-in-science

New Generic will Cut into BMS's Top-Earning Cancer Drug

 Bristol Myers Squibb's moneymaker cancer drug Revlimid finally has a competitor. Israel-based generic medicines leader Teva Pharmaceuticals announced a generic version of Revlimid called lenalidomide, which could significantly cut into BMS’s earnings.

Celgene initially developed Revlimid, and the U.S. Food and Drug Administration approved it in 2006. When BMS bought out Celgene in 2019 in a massive $74 billion deal, it took over the entire oncology portfolio, and Revlimid quickly became a cash cow.

Revlimid ranked No.2 on the biggest cancer drugs by sale globally in 2021, with a whopping $12.8 billion. It is BMS’s top-earning product. In the company’s 2021 end-of-year/Q4 report, BMS listed Revlimid as both a quarterly and yearly winner, up 1% from Q3 2021 and 6% from Q4 2020. That same report projected Revlimid’s 2022 revenue to top $10 billion.

The drug is used to treat cancers, such as multiple myeloma, follicular lymphoma, myelodysplastic syndromes, and mantle cell lymphoma. Lymphomas affect about 825,651 people in the United States, and another 90,000 will be diagnosed each year. About 35,000 people in the United States are diagnosed with multiple myeloma each year. Multiple myeloma is a dismal diagnosis, as the five-year survival rate is only about 54%. Between these two types of blood cancers, Revlimid has been in high demand - until now at least. 

Teva’s introduction of a generic version to the market could be a literal lifesaver. About 56% of all American adults will experience financial hardship from medical costs alone. More than 25% of Americans say that they or a family member have delayed medical treatment for a severe illness because they are worried about the cost. In cases of cancers like lymphomas and multiple myeloma, waiting to see a doctor could be a deadly delay.

But now, patients can access the same medication for a much lower price. The generic drug, called lenalidomide, is now available from Teva in 5 mg, 10 mg, 25 mg and 25 mg.

“The launch of our first generic version of Revlimid in the U.S. provides patients with another important treatment option for these extremely challenging conditions, demonstrating Teva’s commitment to making complex generic drugs available to the patients who need them,” Christine Baeder, senior vice president and chief operating officer of U.S. Generics at Teva USA, said.

Teva is a global leader in generic medicines. It has the largest portfolio of FDA-approved generic drugs on the market. Chances are that you’ve had a Teva product - the company makes 1 in 12 generic medicines in the U.S. Teva also holds first place when it comes to first-to-file opportunities, and it has more than 100 more currently pending in the U.S.

While this is excellent news for people with blood cancers, it may not be welcome news for BMS. The company knew that a generic version of Revlimid was in the pipeline. In its Q4 2021 report, it estimated a drastic $10.5 billion loss in 2022 due to “loss of exclusivity” for Revlimid and another popular drug, Abraxane.

https://www.biospace.com/article/new-generic-cuts-into-bristol-myers-squibb-s-top-earning-cancer-drug/

Cortexyme Posts Safety Data From SAD Portion Of COR588 Trial In P.Gingivalis

 Cortexyme Inc (NASDAQ: CRTX) has announced results from the single ascending dose (SAD) portion of the Phase 1 trial of COR588 for diseases related to P. gingivalis infection.

• The SAD trial was designed to evaluate the safety and pharmacokinetics of COR588 in healthy volunteers.

• In the SAD portion of the Phase 1 trial, preliminary results indicate COR588 was well-tolerated across all four cohorts in the dose range from 25 mg to 200 mg with no serious adverse events.

• No clinically significant findings were observed on other safety measures, including vital signs, laboratory findings, telemetry, or ECGs.

• COR588 exhibited an 11-to-12-hour half-life consistent with once-daily dosing and a dose-proportional pharmacokinetic profile.

• The Company will share the complete data set from the COR588 Phase 1 trial in Q2 of 2022 once the multiple ascending dosing phase is complete.

https://finance.yahoo.com/news/cortexyme-posts-safety-data-sad-162705439.html

Biogen cut to Hold from Buy by Stifel

 Target to $223 from $304

https://finviz.com/quote.ashx?t=BIIB&ty=c&ta=1&p=d

BioNTech, Regeneron Expand Collaboration on Combo for Lung Cancer

 

• BioNTech and Regeneron plan to jointly conduct clinical trials evaluating FixVac candidate BNT116 in combination with Libtayo for the treatment of advanced non-small cell lung cancer (NSCLC)

• The development costs for the trials will be equally shared between the parties

• The companies advance clinical development of FixVac and Libtayo combination to third tumor type building on their existing agreements in melanoma and prostate cancer by combining their complementary immunotherapies to pave the way for new treatment options in high medical need indications

https://finance.yahoo.com/news/biontech-regeneron-expand-strategic-collaboration-132700302.html

Kala Med Now Covered on UnitedHealthcare Commercial and Cigna Medicare

 Kala Pharmaceuticals, Inc., (NASDAQ:KALA), a commercial-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies for diseases of the eye, today announced that UnitedHealthcare, one of the largest commercial health care plans in the United States, covering approximately 13 million lives, has added EYSUVIS (loteprednol etabonate ophthalmic suspension) 0.25% as a covered brand on its commercial formularies effective March 2022. EYSUVIS now has coverage for approximately 118 million lives, or 70% of all commercial lives.

Also today, Kala announced that Cigna Medicare has added EYSUVIS as a preferred brand effective February 2022, adding an additional 1.9 million Medicare lives. EYSUVIS now has Medicare preferred coverage for select plans with Express Scripts, Prime Therapeutics and Cigna. Total EYSUVIS Medicare coverage is now approximately 7.1 million lives, or 15% of all Medicare lives.

“Today’s announcement marks significant progress toward our goal of expanding access to EYSUVIS for the millions of people who suffer from dry eye disease, including dry eye flares,” said Todd Bazemore, President and Chief Operating Officer of Kala Pharmaceuticals. “We look forward to continuing to engage with other commercial and Medicare Part D health plans as we execute on our goal of providing broad market access to EYSUVIS for eye care professionals and their patients.”

EYSUVIS became commercially available in January 2021 as the first and only FDA-approved medicine for the short-term (up to two weeks) treatment of the signs and symptoms of dry eye disease.

https://finance.yahoo.com/news/kala-pharmaceuticals-announces-eysuvis-now-130000614.html