| | | |   By Elizabeth Cooney | Good morning. Gene therapies for sickle cell disease sound like a home run. But their advent could end up being a “so close, yet so far” scenario. Read on. | | | ‘Playing with an infectious disease fire’: Empty Covid coffers court disaster, experts say The failure of Congress last week to approve additional pandemic response funding could have potentially devastating consequences, prominent Covid-19 experts are warning, leaving the federal government unable to invest in more therapeutics, vaccines, testing, and other initiatives. Congress may yet approve more funding after a deal to provide $15 billion to continue Covid-19 response activities imploded, but the impasse could leave the country stuck yet again in a cycle of under-preparedness, several experts told STAT’s Rachel Cohrs. It’s not entirely certain a new variant could be coming, or when it could emerge, or how severe it could be, they said, but if lawmakers wait to find out, it’s going to be too late. “You are playing with an infectious disease fire, and it will burn you,” epidemiologist Micheal Osterholm said. “Unfortunately, people will unnecessarily have to die.” Read more. | Gene therapies could be coming for sickle cell. Will patients be able to pay for them?  (ALEX HOGAN/STAT; ADOBE) In the next year or so, U.S. regulators may approve gene therapies — one developed by Vertex and another by Bluebird Bio — to treat sickle cell disease, an inherited group of blood disorders that affect an estimated 100,000 Americans and are particularly prevalent among Black people. Any treatment would be welcome for a condition that causes significant pain, multiple infections, and leads to an early death, especially because a gene therapy could potentially end the illness with one treatment. But gene therapies come with high price tags — and it’s estimated that roughly 40% of sickle cell patients are covered by Medicaid. The advent of sickle cell gene therapies could be a “so close, yet so far” scenario, if the treatments become available but are out of reach for the people on Medicaid who could most benefit. Read more in STAT+. | Texas judge halts investigations into medical treatments for transgender children A judge has blocked investigations of gender-affirming care for transgender youth as child abuse, temporarily preventing the state from enforcing Texas Gov. Greg Abbott’s order to consider medically accepted treatments for transgender youth — including hormones and puberty-suppressing drugs — as abuse. District Judge Amy Clark Meachum said the governor’s directive had been improperly adopted and violated the state constitution. The injunction follows a legal challenge by the parents of a 16-year-old transgender girl, among the first to be investigated by the state’s Department of Family and Protective Services under Abbott’s order. The ruling applied to all investigations initiated across Texas under Abbott’s order, which the court said could no longer be enforced pending a trial on the issue, set for July. The state immediately appealed Friday’s decision. | Four steps to design innovative medicines for all Improving diversity in clinical trials is a critical goal for the biopharmaceutical industry. To ensure that our work yields innovative medicines for all, we must embed D&I where science starts — in the lab. Taking diversity into account long before potential therapies enter the clinic; Recognizing and rooting out social biases that could skew research; Lowering barriers to inclusive collaboration. As an industry, we must follow these four steps to make research truly diverse and inclusive. | Closer look: He made chemical compounds for global drugmakers. Then bombs fell in Kyiv  Enamine's headquarters are in a former Soviet chemical plant in Kyiv. (COURTESY IVAN KONDRATOV/ENAMINE) It was just last month, with Covid having calmed down in Kyiv, that Ivan Kondratov returned to his office. He managed medicinal chemistry projects, building “target libraries” for clients, mostly large biopharma companies around the world. His employer, Enamine, had become a pillar of global drug development, with its catalog of chemical compounds for drugmakers to test in creating potential treatments. But on Feb. 24, all of that came to a halt as Ukrainians were stunned awake by the sound of explosions. And everything changed for the chemists and scientists. “The situation developed really, really quickly,” Kondratov told STAT’s Isabella Cueto, speaking by phone from northwest Ukraine, where he had sought refuge. Suddenly, reading the latest published research, figuring out what chemical building blocks his team could synthesize, mattered little. “This was my routine. And now I don’t even think about that,” he said. Read more. | Staffing shortages and health care workers' mental health lead list of safety concerns Medical errors and device malfunctions have historically topped the list of safety concerns compiled by ECRI, a nonprofit that tracks safety, quality, and cost-effectiveness in health care. This year staffing shortages and mental health among health care workers lead the latest rankings, issued today. Those interrelated issues aren’t new, but they have surged to prominence with the pandemic, accelerated by burnout, emotional exhaustion, and depression. They are now jeopardizing patient safety, the analysts say. Staffing shortages mean many patients are turned away or wait longer for care, even if they’re facing life-threatening emergencies. The full list: - Staffing shortages
- Covid-19 effects on health care workers’ mental health
- Bias and racism in addressing patient safety
- Vaccine coverage gaps and errors
- Cognitive biases and diagnostic error
- Nonventilator health care-associated pneumonia
- Human factors in operationalizing telehealth
- International supply chain disruptions
- Products subject to emergency use authorization
- Telemetry monitoring
| Quality of older adults’ diet is deteriorating When a large national study surveyed older U.S. adults’ diets, they found that almost 20 years ago, just over half were eating poorly. Now that proportion has risen to 61%, new research in JAMA Network Open reports, based on data from 2001 through 2018. To determine overall diet quality, the analysts culled responses from more than 10,000 people over age 65 and matched them to goals set by the American Heart Association and the USDA Healthy Eating Index. Consumption of total fruits and vegetables, nuts, seeds, and legumes decreased from 2001 to 2018, while consumption of fish and shellfish, processed meat, and saturated fat showed an increasing trend. Overall, the proportion of older adults with poor diet quality grew by 10%, and the proportion with an ideal diet quality remained consistently low. Also, “the low cost of empty calories relative to the higher cost of nutrient-rich foods exacerbated social inequalities in dietary health,” the authors write. | | | | | What to read around the web today - Texas Supreme Court deals final blow to federal abortion law challenge. Texas Tribune
- As a crisis hotline grows, so do fears it won't be ready. New York Times
- What to know about Amylyx’s ALS drug and a highly anticipated advisory panel meeting. STAT+
- Jury: California health system did not abuse market power. Associated Press
- In New York appearance, CMS administrator pushes for Congress to act on drug prices. STAT+
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