Tag: News Approved

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STAT+: CMS proposes rolling back breakthrough device payment flexibilities

The Centers for Medicare and Medicaid Services is proposing to repeal a pathway that currently allows breakthrough devices to qualify for supplementary payments without proving they provide a substantial clinical improvement over alternatives.

Access to lifesaving new technologies can be stymied when hospitals don’t get paid enough to cover their costs. So since 2001, Medicare has given innovative devices a chance at extra payments when they meet three criteria: they’re new and different from what’s currently available, they offer a clinical improvement over existing options, and they’re especially costly.

Since 2021, devices that receive breakthrough designation from the Food and Drug and Administration have gotten an even sweeter deal: In order to qualify for the extra payments, they only have to demonstrate they’re expensive. 

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Motoneurons Inhibitory Synapses Homeostatically Respond to Neuronal Activity and Modulate Amyotrophic Lateral Sclerosis Pathogenesis

Alterations in excitation/inhibition (E/I) balance and changes in motor neurons (MN) activity may contribute to MN vulnerability in ALS. The balance of pathogenic versus adaptive changes occurring in inhibitory synapses and affecting E/I balance remain unclear. Confocal microscopy of MN from P45 male SOD1G93A mice reveal downregulated GlyR but upregulated GABAR clusters at inhibitory synapses. GlyR and GABAR respond to PSAM and DREADD chemogenetic alterations of MN excitability, with increased activity driving increase in inhibitory clusters. An E3 ligase-conjugated intrabody (GFE3) degrades Gephyrin, decreases GABAR and GlyR clusters, increases net activity, and downregulates disease markers. However, simultaneous decrease of inhibition and increased activity by actPSAM and GFE3 shows no net beneficial effects on disease markers. Thus inhibitory synapses are involved in the early phases of ALS pathogenesis and respond to persistent homeostatic loops, and their suppression delivers a net activity increase, offering potential benefits on disease pathways.

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Development of a Contextualized, Research-Based Flemish Assessment Framework for Digital Care, Assistance, and Support: Delphi Study

<strong>Background:</strong> The rapid evolution of digital technologies has transformed health, mental health, and social care, offering new modalities of digital care, assistance, and support through web-based platforms, mobile apps, extended reality, wearables, and artificial intelligence systems. Despite this proliferation, there is little consensus on what constitutes “high-quality” digital care. Challenges persist regarding data security, interoperability, accessibility, sustainability, and professional competence, whereas existing standards and regulations provide fragmented guidance. <strong>Objective:</strong> This study aimed to develop a contextualized, consensus-based quality assessment framework for digital care, assistance, and support in Flanders, Belgium. For this purpose, perspectives across technology, organizational processes, and professional competencies were integrated. <strong>Methods:</strong> The study used a multiphase design comprising (1) 10 expert interviews with Flemish government officials; (2) a narrative literature review of 303 peer-reviewed and gray literature sources; (3) a 3-round Delphi study with 50 experts across 5 domains (end users, facilitators, technology developers, deontology and ethics experts, and digital inclusion and media literacy experts); and (4) 4 complementary focus groups and 3 interviews with specialists in artificial intelligence, regulation, social work, mental health, and IT. The Delphi rounds gathered iterative feedback through open-ended elicitation, structured rating, and classification of quality criteria. Quantitative data were analyzed using descriptive statistics, whereas qualitative feedback was subjected to thematic analysis. <strong>Results:</strong> A total of 50 experts participated in round 1, a total of 40 (80%) participated in round 2, and 27 (54%) participated in round 3. Round 1 generated 577 unique quality criteria, consolidated into 26 clusters organized under 3 pillars: technology, organization, and professional competencies. The relative importance across pillars was balanced (mean score 37.29, SD 12.38 for technology; 33.33, SD 10.39 for professional competencies; and 29.80, SD 10.45 for organizations). Accessibility, reliability, and safety ranked highest for the technology; vision, quality monitoring, and infrastructure ranked highest for organization; and support, digital competencies, and ethics ranked highest for professional competencies. The finalized framework included 112 criteria, of which 35 (31.3%) were designated as optional and 77 (68.8%) were designated as minimum requirements. Focus groups and interviews validated the framework’s comprehensiveness and usability, emphasizing proportional implementation, user centrality, and alignment with European Union regulations. Stakeholders highlighted the need for tools, training, and governance mechanisms to ensure adoption and sustainability. <strong>Conclusions:</strong> This study produced a codeveloped, context-sensitive quality assessment framework that balances technological robustness, organizational readiness, and professional competence in digital care, assistance, and support. The framework can serve both as a quality safeguard and a developmental road map. Accompanying self-assessment and governance tools enhance practical applicability. Implementation success will depend on governmental support, resource allocation, and structured feedback loops. Future research should pilot the framework in real-world settings, assess its impact, and establish mechanisms for continuous updates to maintain relevance in a rapidly evolving digital landscape. <strong>Trial Registration:</strong>
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STAT+: Novartis CEO joins Anthropic’s board

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Good morning. A reminder that if you’re ever feeling down about a mistake you made, there is always a way to turn it around — like how this delivery robot company has turned the issue of its robot crashing into my local bus stop into a marketing opportunity.

The need-to-know this morning

  • Revolution Medicines raised $2 billion in concurrent stock and debt offerings. The mammoth financings — double what the company intended to raise — come just days after daraxonrasib, its experimental treatment for advanced pancreatic cancer, was shown to double the median overall survival of patients in a Phase 3 clinical trial.

Bain Capital again creates a startup with older pharma drugs

After Bain Capital last summer said it licensed five immunology drugs from Bristol Myers Squibb, it’s now unveiling the company to take those treatments forward: a startup called Beeline Medicines.

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STAT+: FDA panel will meet to discuss allowing broader access to certain peptides

WASHINGTON — The Food and Drug Administration will convene an outside panel of advisers to discuss whether to allow compounding pharmacies to manufacture certain peptides, the agency announced on Wednesday. The meetings will take place July 23 and 24. Another will be held before the end of February 2027.

In 2023, the FDA removed 19 peptides from a list of drugs the agency allows compounding pharmacies to produce.  The July panel will discuss whether to add back seven peptides. At a future meeting that has yet to be scheduled, the panel will discuss whether to add back five.

There is limited data on the effectiveness and safety of many of these products, but they have become increasingly popular, promoted by online influencers and health secretary Robert F. Kennedy Jr. The move comes after Kennedy told podcaster Joe Rogan in February that he wanted to make those peptides more accessible. 

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STAT+: A $15 AI test, Project Glasswing, and how Doctronic pilot blindsided Utah medical board

You’re reading the web edition of STAT’s AI Prognosis newsletter, our subscriber-exclusive guide to artificial intelligence in health care and medicine. Sign up to get it delivered in your inbox every Wednesday. 

This newsletter will be taking a break next week as I head to the Grand Canyon. Please send must-see northern Arizona sights, and — you know the drill — Phoenix ice cream recommendations: aiprognosis@statnews.com

There are so many important things going on that we’re bringing you a big ol’ roundup this week.

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STAT+: Pharmalittle: We’re reading about FDA seeking more data on a Lilly obesity pill, a pharma 340B win, and more

Top of the morning to you. The middle of the week is upon us and, since you made it this far, why not forge ahead? After all, there is always light at the end of the proverbial tunnel. You never know what you may accomplish. So please join us as we celebrate this notion with a cup or three of delicious stimulation. Our choice today is chocolate raspberry. Meanwhile, we have assembled the latest menu of tidbits to help you along. So please dig in. Have a smashing day, and please feel free to forward any secrets you come across. Our “in basket” is always open. …

The U.S. Food and Drug Administration asked Eli Lilly for more data on liver injury linked to its newly approved ​obesity pill, Reuters says, citing a letter posted on the agency website. The April 1 letter also said Lilly must conduct post-marketing trials to assess risks related to cardiovascular events and delayed gastric emptying. The drugmaker is required to also conduct a milk-only lactation study in ​lactating women who have received a dose of the pill to assess concentrations of the drug ​in breast milk using a validated assay. The weight loss pill, ‌branded Foundayo, a once-daily oral medication that targets the GLP-1 hormone, won approval earlier this month under the Commissioner’s National Priority voucher program, which aims to speed FDA decisions on drugs deemed critical to public health or ​national security.

AbbVie, Novartis, AstraZeneca, and the Pharmaceutical Research & Manufacturers of America, the industry trade group, notched a victory after a U.S. appeals court vacated an order rejecting their request to block a Maryland drug discount law, remanding the decision for review, Bloomberg Law reports. The U.S. Court of Appeals for the Fourth Circuit ruled that a lower court erred when it denied a motion filed by the companies and the trade group for a preliminary injunction against a Maryland law. H.B. 1056, currently in effect, requires manufacturers to distribute discounted drugs to an unlimited number of pharmacies that contract with health providers under the 340B Drug Discount Program. They argued the law improperly forces drug companies to supply so-called contract pharmacies as part of the program, and that the law is illegal because it is preempted by federal law and also violates the U.S. Constitution.

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Aberrant local and global neural activation patterns in pediatric Prader–Willi syndrome

PurposeAlthough cognitive disorders in children with Prader–Willi syndrome (PWS) are linked to abnormalities in spontaneous neural activation and functional connectivity (FC), the specific neural activation patterns remain uncertain, especially in young children with PWS.MethodsThe current study set out to explore specific local and global neural activation in pediatric PWS using the amplitude of low-frequency fluctuations (ALFF), regional homogeneity (ReHo), and seed-based whole brain FC. Information was gathered from 35 pediatric PWS patients and 33 healthy controls (HC). Both groups’ ALFF and ReHo values were computed, and FC were constructed on the basis of altered ALFF and ReHo regions. The relationships between altered ALFF, ReHo, and FC and the Griffiths Developmental Scales (GDS) of the PWS group were analyzed using partial correlation analysis.ResultsBoth ALFF and ReHo exhibited decreases in occipital lobe, temporal lobe, and cingulate gyrus, and altered ReHo was present in parietal lobe, frontal lobe, and basal ganglia areas. Moreover, ALFF and ReHo also exhibited increases in occipital and temporal lobes. Decreased FC was detected in the visual network (VN), sensorimotor network (SMN), salience network (SAN), and default mode network (DMN). The SMN-, cingulate-, and occipital lobe-related neural activation patterns were significantly positively correlated with the GDS score.ConclusionThe PWS group was characterized mainly by decreased neuronal physiological function and the ReHo was similar to ALFF but more extensive. The decreased local and global brain neural activation patterns may serve as early physiological indicators of cognitive abnormalities.
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Early tinnitus burden and subjective hearing are candidate markers of 2-year quality of life after cochlear implantation in single-sided deafness

BackgroundCochlear implantation is a common treatment for adults with single-sided deafness (SSD), but patient-reported benefits vary. The relationships among tinnitus burden, perceived hearing ability, psychological distress, disease-specific health-related quality of life, and whether early postoperative outcomes predict later results are not well understood.ObjectiveThis study explores how disease-specific quality of life relates to tinnitus burden, hearing, stress, depression, and anxiety after cochlear implantation in SSD. It also seeks early markers linked to 2-year outcomes.MethodsThis secondary complete-case analysis was based on a previously reported prospective longitudinal SSD cohort. Of 70 adults with postlingual SSD, 36 (51.4%) had complete Nijmegen Cochlear Implant Questionnaire (NCIQ) data at baseline and at 6 months, 1 year, and 2 years after unilateral cochlear implantation and were included. Additional measures included the Tinnitus Questionnaire (TQ), Oldenburg Inventory (OI), PerceivFed Stress Questionnaire (PSQ), General Depression Scale (ADS-L), Generalized Anxiety Disorder 7-item scale (GAD-7), and Freiburg Monosyllable Test (FMT) at 65 dB. Timepoint-specific correlations with the NCIQ were analyzed using Spearman’s rank correlations. Exploratory multivariable analyses employed linear regression on rank-transformed variables to assess whether baseline and 6-month patient-reported profiles were associated with 2-year NCIQ outcomes. Longitudinal within-patient comparisons were conducted as a secondary descriptive analysis.ResultsHigher NCIQ scores were linked to lower tinnitus burden and better hearing across all assessments. Associations with depression and anxiety persisted, while connections with perceived stress emerged after surgery. At baseline, higher tinnitus burden was associated with lower 2-year NCIQ scores. At 6 months, higher tinnitus is still associated with lower 2-year NCIQ scores, whereas better hearing is associated with higher 2-year NCIQ scores. Early postoperative improvement was followed by stabilization over 2 years.ConclusionImprovement in health-related quality of life after cochlear implantation in adults with SSD is complex and extends beyond hearing alone. Tinnitus was the most consistent negative factor, while improved subjective hearing at 6 months was associated with better outcomes at 2 years. These results support a structured, multidimensional approach to patient-reported follow-up after cochlear implantation in SSD and suggest that early postoperative patient-reported status may serve as an early candidate marker for later quality-of-life outcomes.