tbuckley/cabot-search huggingface dataset:
Added benefit and revenues of oncology drugs approved by the European Medicines Agency between 1995 and 2020: retrospective cohort study
Authors: Francine Brinkhuis, Wim G Goettsch, Aukje K Mantel-Teeuwisse, Lourens T Bloem
Journal: BMJ (2024)

Abstract Objectives To evaluate the added benefit and revenues of oncology drugs, explore their association, and investigate potential discrepancies between added benefit and revenues across different approval pathways of the European Medicines Agency (EMA). Design Retrospective cohort study. Setting Oncology drugs and their indications approved by the EMA between 1995 and 2020. Main outcome measures Added benefit was evaluated using ratings published by seven organisations: health technology assessment agencies from the United States, France, Germany, and Italy, two medical oncology societies, and a drug bulletin. All retrieved ratings were recategorised using a four point ranking scale to indicate negative or non-quantifiable, minor, substantial, or major added benefit. Revenue data were extracted from publicly available financial reports and compared with published estimates of research and development (R&amp;D) costs. Finally, the association between added benefit and revenue was evaluated. All analyses were performed within the overall study cohort, and within subgroups based on the EMA approval pathway: standard marketing authorisation, conditional marketing authorisation, and authorisation under exceptional circumstances. Results 131 oncology drugs with 166 indications were evaluated for their added benefit by at least one organisation within the required timeframe, yielding a total of 458 added benefit ratings; 189 (41%) were negative or non-quantifiable. The median time to offset the median R&amp;D costs ($684m, £535m, €602m, adjusted to 2020 values) was three years; 50 of 55 (91%) drugs recovered these costs within eight years. Drugs with higher added benefit ratings generally had greater revenues. Negative or non-quantifiable added benefit ratings were more frequent for conditional marketing authorisations and authorisations under exceptional circumstances than for standard marketing authorisations (relative risk 1.53, 95% confidence interval 1.23 to 1.89). Conditional marketing authorisations generated lower revenues and took longer to offset R&amp;D costs than standard marketing authorisations (four years compared with three years). Conclusions While revenues seem to align with added benefit, most oncology drugs recover R&amp;D costs within a few years despite providing little added benefit. This is particularly true for drugs approved through conditional marketing authorisations, which inherently appear to lack comprehensive evidence. Policy makers should evaluate whether current regulatory and reimbursement incentives effectively promote development of the most effective drugs for patients with the greatest needs.
Microencapsulated Benzoyl Peroxide for Rosacea in Context: A Review of the Current Treatment Landscape
Authors: Seemal R. Desai, Hilary Baldwin, James Q. Del Rosso, Richard L. Gallo, Neal Bhatia, Julie C. Harper, Jean Philippe York, Linda Stein Gold
Journal: Drugs (2024)

Rosacea, a chronic skin condition affecting millions of people in the USA, leads to significant social and professional stigmatization. Effective management strategies are crucial to alleviate symptoms and improve patients' quality of life. Encapsulated benzoyl peroxide 5% (E-BPO 5%) is a newly FDA-approved topical treatment for rosacea that shows promise in enhancing therapeutic response and minimizing skin irritation. This review aims to assess the role of recently FDA approved E-BPO 5% in the current treatment landscape for rosacea management, as it is not yet included in clinical guidelines that predominantly rely on older approved therapies. The review focuses on randomized controlled trials conducted in English-speaking adults. It evaluates the efficacy, safety, and tolerability of various US Food and Drug Administration (FDA)-approved agents used for rosacea treatment, including E-BPO cream, metronidazole gel, azelaic acid gel and foam, ivermectin cream, minocycline foam, oral doxycycline, brimonidine gel, and oxymetazoline HCl cream. Existing therapies have been effective in reducing papulopustular lesions and erythema associated with rosacea for many years. E-BPO 5% offers a promising addition to the treatment options due to its microencapsulation technology, which prolongs drug delivery time and aims to improve therapeutic response while minimizing skin irritation. Further research is necessary to determine the exact role of E-BPO 5% in the therapeutic landscape for rosacea. However, based on available evidence, E-BPO 5% shows potential as a valuable treatment option for managing inflammatory lesions of rosacea, and it may offer benefits to patients including: rapid onset of action, demonstrated efficacy by Week 2, excellent tolerability, and sustained long-term results for up to 52 weeks of treatment.
Point-of-Care Clinical Trials in Nephrology
Authors: Sarah M. Leatherman, Areef Ishani
Journal: Journal of the American Society of Nephrology (2024)

1Cooperative Studies Program Coordinating Center, VA Boston Healthcare System, Boston, Massachusetts 2Department of Biostatistics, Boston University School of Public Health, Boston, Massachusetts 3Minneapolis VA Healthcare System, Minneapolis, Minnesota 4Department of Medicine, University of Minnesota, Minneapolis, Minnesota Correspondence: Dr. Areef Ishani, email: [email protected]
Recommendations to address respondent burden associated with patient-reported outcome assessment
Authors: Olalekan Lee Aiyegbusi, Samantha Cruz Rivera, Jessica Roydhouse, Paul Kamudoni, Yvonne Alder, Nicola Anderson, Robert Mitchell Baldwin, Vishal Bhatnagar, Jennifer Black, Andrew Bottomley, Michael Brundage, David Cella, Philip Collis, Elin-Haf Davies, Alastair K. Denniston, Fabio Efficace, Adrian Gardner, Ari Gnanasakthy, Robert M. Golub, Sarah E. Hughes, Flic Jeyes, Scottie Kern, Bellinda L. King-Kallimanis, Antony Martin, Christel McMullan, Rebecca Mercieca-Bebber, Joao Monteiro, John Devin Peipert, Juan Carlos Quijano-Campos, Chantal Quinten, Khadija Rerhou Rantell, Antoine Regnault, Maxime Sasseville, Liv Marit Valen Schougaard, Roya Sherafat-Kazemzadeh, Claire Snyder, Angela M. Stover, Rav Verdi, Roger Wilson, Melanie J. Calvert
Journal: Nature Medicine (2024)

Patient-reported outcomes (PROs) are increasingly used in healthcare research to provide evidence of the benefits and risks of interventions from the patient perspective and to inform regulatory decisions and health policy. The use of PROs in clinical practice can facilitate symptom monitoring, tailor care to individual needs, aid clinical decision-making and inform value-based healthcare initiatives. Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete PROs, with potential impact on the completeness and quality of the data for decision-making. We therefore conducted an initial literature review to generate a list of candidate recommendations aimed at reducing respondent burden. This was followed by a two-stage Delphi survey by an international multi-stakeholder group. A consensus meeting was held to finalize the recommendations. The final consensus statement includes 19 recommendations to address PRO respondent burden in healthcare research and clinical practice. If implemented, these recommendations may reduce PRO respondent burden. Patient-reported outcomes are invaluable tools, but may impose a burden on patients; this consensus statement provides a set of 19 recommendations to reduce respondent burden.
PolyGR and polyPR knock-in mice reveal a conserved neuroprotective extracellular matrix signature in C9orf72 ALS/FTD neurons
Authors: Carmelo Milioto, Mireia Carcolé, Ashling Giblin, Rachel Coneys, Olivia Attrebi, Mhoriam Ahmed, Samuel S. Harris, Byung Il Lee, Mengke Yang, Robert A. Ellingford, Raja S. Nirujogi, Daniel Biggs, Sally Salomonsson, Matteo Zanovello, Paula de Oliveira, Eszter Katona, Idoia Glaria, Alla Mikheenko, Bethany Geary, Evan Udine, Deniz Vaizoglu, Sharifah Anoar, Khrisha Jotangiya, Gerard Crowley, Demelza M. Smeeth, Mirjam L. Adams, Teresa Niccoli, Rosa Rademakers, Marka van Blitterswijk, Anny Devoy, Soyon Hong, Linda Partridge, Alyssa N. Coyne, Pietro Fratta, Dario R. Alessi, Ben Davies, Marc Aurel Busche, Linda Greensmith, Elizabeth M. C. Fisher, Adrian M. Isaacs
Journal: Nature Neuroscience (2024)

Abstract Dipeptide repeat proteins are a major pathogenic feature of C9orf72 amyotrophic lateral sclerosis (C9ALS)/frontotemporal dementia (FTD) pathology, but their physiological impact has yet to be fully determined. Here we generated C9orf72 dipeptide repeat knock-in mouse models characterized by expression of 400 codon-optimized polyGR or polyPR repeats, and heterozygous C9orf72 reduction. (GR)400 and (PR)400 knock-in mice recapitulate key features of C9ALS/FTD, including cortical neuronal hyperexcitability, age-dependent spinal motor neuron loss and progressive motor dysfunction. Quantitative proteomics revealed an increase in extracellular matrix (ECM) proteins in (GR)400 and (PR)400 spinal cord, with the collagen COL6A1 the most increased protein. TGF-β1 was one of the top predicted regulators of this ECM signature and polyGR expression in human induced pluripotent stem cell neurons was sufficient to induce TGF-β1 followed by COL6A1. Knockdown of TGF-β1 or COL6A1 orthologues in polyGR model Drosophila exacerbated neurodegeneration, while expression of TGF-β1 or COL6A1 in induced pluripotent stem cell-derived motor neurons of patients with C9ALS/FTD protected against glutamate-induced cell death. Altogether, our findings reveal a neuroprotective and conserved ECM signature in C9ALS/FTD.
Epigenetic variation impacts individual differences in the transcriptional response to influenza infection
Authors: Katherine A. Aracena, Yen-Lung Lin, Kaixuan Luo, Alain Pacis, Saideep Gona, Zepeng Mu, Vania Yotova, Renata Sindeaux, Albena Pramatarova, Marie-Michelle Simon, Xun Chen, Cristian Groza, David Lougheed, Romain Gregoire, David Brownlee, Carly Boye, Roger Pique-Regi, Yang Li, Xin He, David Bujold, Tomi Pastinen, Guillaume Bourque, Luis B. Barreiro
Journal: Nature Genetics (2024)
A combinatorial genetic strategy for exploring complex genotype–phenotype associations in cancer
Authors: Shan Li, Alicia Wong, Huiyun Sun, Vipul Bhatia, Gerardo Javier, Sujata Jana, Qian Wu, Robert B. Montgomery, Jonathan L. Wright, Hung-Ming Lam, Andrew C. Hsieh, Bishoy M. Faltas, Michael C. Haffner, John K. Lee
Journal: Nature Genetics (2024)

Abstract Available genetically defined cancer models are limited in genotypic and phenotypic complexity and underrepresent the heterogeneity of human cancer. Here, we describe a combinatorial genetic strategy applied to an organoid transformation assay to rapidly generate diverse, clinically relevant bladder and prostate cancer models. Importantly, the clonal architecture of the resultant tumors can be resolved using single-cell or spatially resolved next-generation sequencing to uncover polygenic drivers of cancer phenotypes.
Neutrophils bearing adhesive polymer micropatches as a drug-free cancer immunotherapy
Authors: Ninad Kumbhojkar, Supriya Prakash, Tatsuya Fukuta, Kwasi Adu-Berchie, Neha Kapate, Rocky An, Solomina Darko, Vineeth Chandran Suja, Kyung Soo Park, Alexander P. Gottlieb, Michael Griffith Bibbey, Malini Mukherji, Lily Li-Wen Wang, David J. Mooney, Samir Mitragotri
Journal: Nature Biomedical Engineering (2024)
Critically ill patients having time outdoors: prevalence and resources in Australia and New Zealand
Authors: Matthew J. Maiden, Michelle Horton, Paul Power, Serena Knowles, Naomi E. Hammond, Naomi Hammond, Serena Knowles, Amy Freeman-Anderson, Belinda Howe, Diane Mackle, Manoj Saxena, Elizabeth Yarad, Ed Litton, Subodh Ganu, Ian Seppelt, Miriam Towns, Graeme Duke, Stephanie Hunter, Julie Evans, Dianne Parker, Clare Loughnan, Blessy Thomas, Nicole Robertson, Kym Gellie, Rachael Parke, Magdalena Butler, Keri-Anne Cowdrey, Melissa Robertson, Ellie McMahon, Zahn Koorts, Shay McGuinness, Brittany Mason, Colin McArthur, Yan Chen, Lauren West, Caroline O’Connor, Hiromi Nakamuro, Anna Richardson, Emily Rigby, Matthew Thackery, Jemma Trickey, Claire Cattigan, Michelle Horton, Matthew Maiden, Cameron Knott, Julie Smith, Catherine Boschert, Treena Sara, Graeme Duke, Stephanie Hunter, Julie Evans, Dianne Parker, Clare Loughnan, Blessy Thomas, Kym Gellie, Nicole Robertson, Mahesh Ramanan, Prashanti Marella, Julia Affleck, Lisa Dougherty, Katrina Ellem, Toni McKenna, Mary Nourse, Bree McDonald, Natasha Edmunds, Jan Mehrtens, Rosalba Cross, Helen Wong, Pawel Twardowski, Robyn Hutchinson, Gabrielle Hanlon, Jonathan Barrett, Shailesh Bihari, Julia Brown, Laura Grear, Craig French, Samantha Bates, Miriam Towns, James McCullough, Mandy Tallott, Maimoonbe Gough, Marek Nalos, Laura Younger, Ravi Krishnamurphy, Khaled El-Khawas, Dianne Hill, Louise Trent, Janet How, Anne Stuart, Llesley Chadwick, Elizabeth Thomas-Vuilleumier, Neeraj Bhadange, Steven Tyler, Kellie Sosnowski, Lynette Morrison, Joanne Sutton, Natalie Soar, Graeme Duke, Stephanie Hunter, Julie Evans, Dianne Parker, Clare Loughnan, Kym Gellie, Nicole Robertson, Katherine Jongebloed, Mackenzie Finnis, Tony Williams, Rima Song, Vivian Lai, Dinu Girijadevi, Alex Browne, Jette Koelle, Charlotte McNab, Kristy Masters, Rebecca Gresham, Julie Lowrey, Janet Liang, Sarah Bridges, Sandra Peake, Tricia Williams, Catherine Kurenda, Alexis Tabah, Maree Duroux, Kylie Jacobs, Megan Ratcliffe, Stuart Baker, Ravikiran Sonawane, Stephanie O’Connor, Nerissa Brown, Kathleen Glasby, Justine Rivett, Lewis Campbell, Brittney Mackenzie, Lizzy Ross, Elizabeth Yarad, Frances Bass, Naomi Hammond, Anne O’Connor, Tom Burbidge-King, Sharon Waterson, Jennifer Coles, Heidi Buhr, Matthew Anstey, Bradley Wibrow, Bhaumik Mevavala, Sarah Hardy, Anthadene Endemann, Marlene Kolybaba, Janet Ferrier, Annamaria Palermo, John Santamaria, Jennifer Holmes, Craig French, Samantha Bates, Miriam Towns, Jonathan Chen, Amanda Miller, Adam Deane, Kathleen Byrne, Deborah Barge, Kim Dooley, Adam Visser, Judith Smith, Robert Martynoga, Amelia Butler, Kara Trask, Kirsha Delaney, Eden Lesona, Umesh Kadam, Maree Monaghan, Mainak Majumdar, Naomi Hammond, Serena Knowles, Yang Li, John Myburgh, Ian Seppelt, Conrad Nangla, Fatima Butt
Journal: Intensive Care Medicine (2024)
Emerging roles of nerve‐bone axis in modulating skeletal system
Authors: Jingya Li, Zhuoyuan Zhang, Jinru Tang, Zeyu Hou, Longjiang Li, Bo Li
Journal: Medicinal Research Reviews (2024)

Over the past decades, emerging evidence in the literature has demonstrated that the innervation of bone is a crucial modulator for skeletal physiology and pathophysiology. The nerve-bone axis sparked extensive preclinical and clinical investigations aimed at elucidating the contribution of nerve-bone crosstalks to skeleton metabolism, homeostasis, and injury repair through the perspective of skeletal neurobiology. To date, peripheral nerves have been widely reported to mediate bone growth and development and fracture healing via the secretion of neurotransmitters, neuropeptides, axon guidance factors, and neurotrophins. Relevant studies have further identified several critical neural pathways that stimulate profound alterations in bone cell biology, revealing a complex interplay between the skeleton and nerve systems. In addition, inspired by nerve-bone crosstalk, novel drug delivery systems and bioactive materials have been developed to emulate and facilitate the process of natural bone repair through neuromodulation, eventually boosting osteogenesis for ideal skeletal tissue regeneration. Overall, this work aims to review the novel research findings that contribute to deepening the current understanding of the nerve-bone axis, bringing forth some schemas that can be translated into the clinical scenario to highlight the critical roles of neuromodulation in the skeletal system.
Author Correction: Combined effects of host genetics and diet on human gut microbiota and incident disease in a single population cohort
Authors: Youwen Qin, Aki S. Havulinna, Yang Liu, Pekka Jousilahti, Scott C. Ritchie, Alex Tokolyi, Jon G. Sanders, Liisa Valsta, Marta Brożyńska, Qiyun Zhu, Anupriya Tripathi, Yoshiki Vázquez-Baeza, Rohit Loomba, Susan Cheng, Mohit Jain, Teemu Niiranen, Leo Lahti, Rob Knight, Veikko Salomaa, Michael Inouye, Guillaume Méric
Journal: Nature Genetics (2024)

In the version of the article initially published, the descriptions of two FUT2 genotypes were inverted.In Fig. 4c and Extended Data Fig. 7b and their legends,
Deaths from Excessive Alcohol Use — United States, 2016–2021
Authors: Marissa B. Esser, Adam Sherk, Yong Liu, Timothy S. Naimi
Journal: MMWR Morbidity and Mortality Weekly Report (2024)

Deaths from causes fully attributable to alcohol use have increased during the past 2 decades in the United States, particularly from 2019 to 2020, concurrent with the onset of the COVID-19 pandemic.However, previous studies of trends have not assessed underlying causes of deaths that are partially attributable to alcohol use, such as injuries or certain types of cancer.CDC's Alcohol-Related Disease Impact application was used to estimate the average annual number and agestandardized rate of deaths from excessive alcohol use in the United States based on 58 alcohol-related causes of death during three periods (2016-2017, 2018-2019, and 2020-2021).Average annual number of deaths from excessive alcohol use increased 29.3%, from 137,927 during 2016-2017 to 178,307 during 2020-2021; age-standardized alcohol-related death rates increased from 38.1 to 47.6 per 100,000 population.During this time, deaths from excessive alcohol use among males increased 26.8%, from 94,362 per year to 119,606, and among females increased 34.7%, from 43,565 per year to 58,701.Implementation of evidence-based policies that reduce the availability and accessibility of alcohol and increase its price (e.g., policies that reduce the number and concentration of places selling alcohol and increase alcohol taxes) could reduce excessive alcohol use and alcohol-related deaths.* https://www.cdc.gov/nchs/products/databriefs/db448.htmalcohol sales † also increased during this time.Previous studies of trends have not included underlying causes of death that are partially attributable to alcohol (1,3), such as injuries or certain types of cancer, for which drinking is a substantial risk factor (4,5).A comprehensive assessment of changes in deaths from excessive alcohol use that includes conditions that are fully and partially attributable to alcohol can guide the rationale for and implementation of effective prevention strategies. Methods
Mortality Risks Over 20 Years in Men With Stage I to III Hormone Receptor–Positive Breast Cancer
Authors: Julieta Leone, Michael J. Hassett, Rachel A. Freedman, Sara M. Tolaney, Noah Graham, Nabihah Tayob, Carlos T. Vallejo, Eric P. Winer, Nancy U. Lin, José P. Leone
Journal: JAMA Oncology (2024)

In women with hormone receptor-positive (HR+) breast cancer, the risk of distant recurrence and death persists for at least 20 years from diagnosis. The risk of late mortality in men with HR+ breast cancer has not been reported.
Progression-Free Survival Analysis With a Graphical Estimand Approach in the Phase 2 SAMCO-PRODIGE 54 Trial—Reply
Authors: Olayidé Boussari, Julien Taïeb
Journal: JAMA Oncology (2024)

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The Transplant Experience for Undocumented Immigrant Patients Formerly Receiving Emergency Dialysis and Caregivers
Authors: Katherine Rizzolo, Nathan Rockey, Claudia Camacho, Colin Gardner, Sixto Giusti, Lilia Cervantes
Journal: JAMA Network Open (2024)

Individuals of undocumented immigration status with kidney failure face barriers to receiving transplants due to lack of health insurance despite no regulatory barriers. Little is known about the perspectives on kidney transplant among individuals with undocumented immigration status with kidney failure who relied on emergency hemodialysis.
Learning From the Experiences of Undocumented Immigrant Kidney Transplant Recipients—From Exceptional Individuals to Equitable Systems
Authors: Anna Morenz, Yue-Harn Ng, Catherine R. Butler
Journal: JAMA Network Open (2024)

Katherine Rizzolo, MD; Nathan Rockey, MD; Claudia Camacho, BA; Colin Gardner, BA; Sixto Giusti, MD; Lilia Cervantes, MD, MSCS