Breaking News

Bitter fights over pricing threaten to keep gene therapies out of Europe

October 4, 2023
Reporter, Morning Rounds Writer

Good morning. Don't miss a report from our Europe correspondent, Andrew Joseph, on the tension between cost-conscious national health systems on the continent and companies setting seven-figure prices for gene therapies meant to improve health for a lifetime.

science

Nobel in chemistry goes dotty

The 2023 Nobel Prize in chemistry was awarded Wednesday to three scientists for the discovery and synthesis of quantum dots, tiny particles that have fueled innovations in nanotechnology from televisions to mapping different tissues in the body. 

The prize went to Moungi Bawendi of MIT, Louis Brus of Columbia University, and Alexei Ekimov, formerly the chief scientist at Nanocrystals Technology in New York. They will split 11 million Swedish kronor, about $1 million, and have their names added to a list of chemistry Nobel winners that prior to this year included 189 names (two of whom have won the prize twice). Eight women have received the honor.

Quantum dots are so tiny that their properties are influenced by their size. In particular, the dots of different size produce different colors. 

The trio of scientists, in research in the 1980s and 1990s, advanced the field by demonstrating such quantum effects and figuring out how to produce quantum dots. Now, the dots are used in applications from computer screens to surgery. 


pandemic

Another updated Covid shot wins FDA authorization

Americans seeking an updated Covid-19 vaccine now have another choice. The FDA authorized Novavax's version yesterday, three weeks after approving the updated Moderna and Pfizer-BioNTech shots. Novavax has struggled to gain a foothold in the now-diminishing Covid vaccine market — globally and within the U.S. — but the company caught a break. Its shots were previously limited to people who had either not had a primary series of Covid vaccinations or who had received a primary series and only one booster, and who would not agree to get additional mRNA boosters, the technology used by Pfizer and Moderna. (Novavax uses a sub-unit protein platform.)

While the Novavax vaccine is still only for people aged 12 and older, it's now available to anyone in that age group who wants to use it — as long as they have gone at least two months since they had a previous Covid vaccination. STAT's Helen Branswell explains.


first opinion

Why a geriatrician thinks term limits are a terrible idea

Mitch McConnell's momentary freezing. President Biden's tripping. And before her death at age 90, Dianne Feinstein's declining health. Each instance renewed calls for term limits for our aging leaders. "As a geriatrician, I think that the idea of maximum age for politicians is terrible," Anna Chodos of UCSF writes in a STAT First Opinion. "The very concept is rooted in ageism. Conflating age and health or age and ability cannot actually help us discern who's truly fit for office and who's not."

Aging is a varied, unpredictable process whose blows are softened by advantages and luck, she argues. Researchers believe both Biden and former President Trump may be superagers, people who maintain their mental and physical functioning in older age. They're not airline pilots, who face a different kind of scrutiny, but for politicians, Chodos suggests, judgments should be made based on their actual work, their record, their policies, and their proposals. Read more.



leftCloser Look

When sky-high gene therapy prices make health systems in Europe balk, patients can be left behind

https://www.statnews.com/2023/10/04/gene-therapy-makers-wonder-if-they-can-make-a-profit-in-europe-patients-fear-being-left-behind-again/Eros Dervisji for STAT

Stella Pelteki was born with beta thalassemia, a disorder that inhibits oxygen's flow through the blood and requires frequent blood transfusions. When she heard about Bluebird Bio's gene therapy Zynteglo, which was likened to a cure and won European approval in 2019, her hopes soared. "I had been waiting 18 years — imagine how I felt," Pelteki, now 32 and splitting her time between Germany and Greece, told STAT's Europe correspondent Andrew Joseph. But after Bluebird halted talks with Germany over its seven-figure pricing, the company left Europe, and left Pelteki out of luck.

When a company promoting an expensive, possibly life-changing treatment collides with a cost-conscious, skeptical national health system, it can crush patients' hopes. Bluebird's experience brings to mind difficult negotiations between Vertex and the U.K. over its cystic fibrosis drugs, but Novartis had an easier path with Zolgensma. People wonder what will happen to the next product, said Androulla Eleftheriou of the Thalassaemia International Federation. "Will it have the same fate?" Read more.


mental health

Use of mental health services for kids jumped since the pandemic began, with telehealth still strong

The pandemic placed extraordinary pressure on our mental health, especially in its earliest months and for children. Telehealth filled some lockdown gaps in mental health services, and it still plays a significant role, a new RAND study in JAMA Network Open says. Looking at visits and spending for pediatric mental health services covered by families' employer-provided insurance from January 2019 through August 2022, the analysis found in-person services for kids and adolescents fell 42% in 2020, while telehealth services surged 30-fold for such diagnoses as anxiety disorders, ADHD, and major depressive disorder. 

By August 2022, in-person mental health services rebounded to 75% of pre-pandemic levels, but telehealth was still 23 times higher. All told, use of mental health services is 22% higher than before the pandemic. While children covered by commercial insurance may differ from those with other kinds of coverage, the authors say telehealth care for kids' mental health needs continues to fill an important gap.


health

Sugary drink consumption varies with where you live

Sugar-sweetened beverages are bad for our health, linked to conditions ranging from obesity and cardiovascular disease to cavities in our teeth. Medical advice warns against these drinks and some countries have imposed taxes on them, cutting sales especially for people with lower income. But a new study in Nature Communications reports that around the world, overall consumption still rose at least 16% since 1990, with wide variations depending on where you live.

The Global Dietary Database, which tracks soft drinks, energy drinks, fruit juices, punch, lemonade, and aguas frescas containing more than 50 calories per serving, found that overall consumption ranged from 0.7 servings per week in South Asia to 7.8 servings per week in Latin America and the Caribbean. Slicing the data more closely, intake was higher in males than females, and in younger than older people; the highest intake was among urban, highly educated adults in Sub-Saharan Africa (12.4 servings per week).


The First Opinion podcast is back for a new season. First Opinion editor Torie Bosch talks with physicians Alison Block and Nikki Zite about the frustrating and heartbreaking consequences of living in a post-Roe America for providers, trainees, and patients. Listen here.


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What we're reading

  • America's epidemic of chronic illness is killing us too soon, Washington Post
  • Idaho banned abortion. Then it turned down supports for pregnancies and births, ProPublica
  • FDA finds 'potential systemic bias' in Amgen's KRAS drug trial ahead of advisory meeting, STAT
  • Court tosses $223.8 million verdict against J&J in talc cancer case, Reuters 
  • Lilly bolts another one on, acquiring Point Therapeutics for $1.4 billion, STAT

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