Review
Medicine, General & Internal
Alba Nicolas-Boluda, Anne Oppenheimer, Jerome Bouaziz, Arnaud Fauconnier
Summary: Patient Reported Outcome Measures (PROM) allow capturing patients' perspectives on their condition in the context of endometriosis care. Despite their extensive use in research and clinical trials, PROMs are rarely used in clinical practice. The development of digital tools can help facilitate the integration of PROMs into routine endometriosis care, improving patient-centered communication and quality of life.
JOURNAL OF CLINICAL MEDICINE
(2021)
Editorial Material
Dermatology
C. C. Zouboulis, P. V. Chernyshov
Summary: The study by R. Sommer et al. published in the Journal of the European Academy of Dermatology and Venereology in 2021 focused on a specific topic in the field of dermatology.
JOURNAL OF THE EUROPEAN ACADEMY OF DERMATOLOGY AND VENEREOLOGY
(2021)
Review
Health Care Sciences & Services
Priscilla Jia Ling Wee, Yu Heng Kwan, Dionne Hui Fang Loh, Jie Kie Phang, Troy H. Puar, Truls Ostbye, Julian Thumboo, Sungwon Yoon, Lian Leng Low
Summary: This study systematically reviewed 238 unique PROMs for patients with type 2 diabetes, categorized them based on their measurement properties, and may assist clinicians and researchers in selecting appropriate high-quality PROMs for clinical practice and research.
JOURNAL OF MEDICAL INTERNET RESEARCH
(2021)
Article
Clinical Neurology
Marketa Sosnova, Anna Maria Zeitlberger, Michal Ziga, Oliver P. Gautschi, Luca Regli, Astrid Weyerbrock, Oliver Bozinov, Martin N. Stienen, Nicolai Maldaner
Summary: The study found that the majority of patients preferred the smartphone-based outcome measure over conventional paper-based methods, considering it more suitable, convenient, and responsive to their symptoms. Patients also believed that the smartphone app was better at detecting changes in physical performance and differences in symptoms. Most patients indicated a preference for using a smartphone app for future assessment and monitoring of spine-related symptoms.
Article
Orthopedics
Zachary T. Sharfman, Nathan Safran, Eyal Amar, Kunal Varshneya, Marc R. Safran, Ehud Rath
Summary: This study found that hip-specific PROM scores in asymptomatic individuals decrease with age. Comparing a patient's outcome scores with age-normalized scores can help establish an accurate reference frame for interpreting outcomes.
AMERICAN JOURNAL OF SPORTS MEDICINE
(2022)
Article
Multidisciplinary Sciences
Hao Xiong, Shlomo Berkovsky, Mohamed Ali Kaafar, Adam Jaffe, Enrico Coiera, Roneel Sharan
Summary: This study evaluates the performance of cough classification models trained on PCR-confirmed COVID patients' data and data from individuals self-reporting their infective status. The models trained on PCR-confirmed data perform better, utilizing more stable predictive features and converging faster. The study also raises concerns about the reliability of crowd-sourced cough data for developing COVID-19 predictive models.
SCIENTIFIC REPORTS
(2022)
Article
Health Care Sciences & Services
Margaret H. Sandham, Emma A. Hedgecock, Richard J. Siegert, Ajit Narayanan, Mevhibe B. Hocaoglu, Irene J. Higginson
Summary: This study investigates whether machine learning and network analysis can identify different phases of patient palliative status based on symptoms reported on the Integrated Palliative Care Outcome Scale (IPOS). The results show significant variation in symptoms among different phases and clear associations between specific symptoms. Machine learning techniques are also utilized to predict the possible transition between phases. These findings, coupled with advancements in mobile apps and wearable technology, suggest the potential for increased use of digital therapeutics in continuous palliative care monitoring.
JOURNAL OF PAIN AND SYMPTOM MANAGEMENT
(2022)
Editorial Material
Oncology
Derek K. Smith
Summary: This paper presents the lesser-understood implications of using patient-reported outcome measures in clinical research. It demonstrates that the assumptions of common mean-based approaches are often violated and shows the reduction in statistical power due to subjective discretization. It concludes that PRO-based studies must be conscientiously designed and analyses should consider using ordinal statistical models.
SUPPORTIVE CARE IN CANCER
(2023)
Article
Medicine, General & Internal
Yongchao Li, Bing Wu, Mao Li, Xiaodong Pang, Liang Yang, Chen Dai, Baogan Peng
Summary: This retrospective study aimed to explore the patient-reported outcome measures (PROM) following coblation nucleoplasty for cervical discogenic dizziness and to compare the therapeutic effect of coblation nucleoplasty with prolonged conservative treatment. The results showed that coblation nucleoplasty significantly improved the symptoms of dizziness and had superior efficacy compared to conservative treatment.
JOURNAL OF CLINICAL MEDICINE
(2023)
Editorial Material
Urology & Nephrology
Findlay MacAskill, Arun Sahai, Majed Shabbir, Tet Yap
Summary: PROMs are increasingly used in healthcare to assess and compare patients, but their limitations in diverse populations should not be overlooked.
NATURE REVIEWS UROLOGY
(2023)
Review
Medicine, General & Internal
Pervez Sultan, Nadir Sharawi, Lindsay Blake, Kazuo Ando, Ellile Sultan, Nima Aghaeepour, Brendan Carvalho, Nishant Sadana
Summary: This study evaluated patient-reported outcome measures (PROMs) for outpatient postpartum recovery and found that the best-performing PROMs currently available are the Maternal Concerns Questionnaire, the Postpartum Quality of Life tool, and the World Health Organization Quality of Life-BREF. Future research should focus on developing and validating a new comprehensive PROM for evaluating outpatient postpartum recovery.
Review
Medicine, General & Internal
Zachary Blood, Anh Tran, Lauren Caleo, Robyn Saw, Mbathio Dieng, Mark Shackleton, H. Peter Soyer, Chris Arnold, Graham J. Mann, Rachael L. Morton
Summary: This systematic review identified PROMs and PREMs used in clinical quality registries for cutaneous melanoma patients, showing that they can enhance care transparency, quality assessment, and research, yet challenges such as resource burden and collection bias may limit their generalizability.
Review
Medicine, General & Internal
Rakhshan Kamran, Liam Jackman, Cynthia Chan, Yujin Suk, Chloe Jacklin, Eve Deck, Nina Wietek, Melissa Stepney, Conrad Harrison, Abhilash Jain, Jeremy Rodrigues
Summary: This systematic review identified barriers and enablers of patient-reported outcome measures (PROMs) implementation in gender-affirming care. A total of 205 different PROMs were used, but there was inconsistency in PROM implementation and a lack of evidence-based approaches. Key barriers included issues with evidence strength and quality of PROMs, while key enablers included using validated PROMs tailored for gender-affirming care and engaging stakeholders in the implementation process.
Article
Rehabilitation
Kurt Kroenke, Timothy A. Miksch, Aaron C. Spaulding, Gina L. Mazza, Christopher C. DeStephano, Shehzad K. Niazi, Allie J. Canoy Illies, Mohamad Bydon, Paull Novotny, Anshit Goyal, Minji K. Lee
Summary: This overview addresses key factors and practical decisions related to the use of patient-reported outcome (PRO) measures, as well as highlights clinical decision support issues.
ARCHIVES OF PHYSICAL MEDICINE AND REHABILITATION
(2022)
Article
Clinical Neurology
Marta Olive-Gadea, David Cano, Marc Rodrigo-Gisbert, Marian Muchada, Estefania Montiel, Maria Baladas, Ester Sanchez-Gavilan, Carolina Paredes, Alvaro Garcia-Tornel, Marta Rubiera, Manuel Requena, Marc Ribo, Carlos A. Molina
Summary: Patient-reported outcome measures (PROMs) provide valuable information for assessing the long-term outcomes of stroke patients. This study used a mobile app to evaluate PROMs in patients with minor stroke or transient ischemic attack and found a correlation between patient-reported outcomes and clinician-assessed outcomes, although some differences were observed.
Article
Health Care Sciences & Services
F. C. W. van Krugten, J. J. Busschbach, M. M. Versteegh, L. Hakkaart-van Roijen, W. B. F. Brouwer
Summary: The study aimed to develop and evaluate the psychometric properties of a new quality of life measure, the Mental Health Quality of Life questionnaire (MHQoL), for individuals with mental health problems. Results showed that the MHQoL demonstrated high internal consistency, convergent validity, and test-retest reliability, making it a simple and effective tool to assess quality of life in this population.
QUALITY OF LIFE RESEARCH
(2022)
Article
Economics
Matthijs M. Versteegh, Simone A. Huygens, Beatrijs W. H. Wokke, Joost Smolders
Summary: This study compared 360 different treatment sequences for patients with relapsing-remitting multiple sclerosis (RRMS) in terms of health outcomes and societal costs. The results showed that the optimal treatment sequence for maximizing health outcomes was not necessarily the most cost-effective option. Treatment decisions should be individually tailored.
Article
Economics
Stefan A. Lipman, Arthur E. Attema, Matthijs M. Versteegh
Summary: Time trade-off utilities have been found to be biased upwards, but this bias can be reduced by measuring individuals' discount function and loss aversion and correcting for these individual characteristics.
Review
Economics
Heleen Vellekoop, Matthijs Versteegh, Simone Huygens, Isaac Corro Ramos, Laszlo Szilberhorn, Tamas Zelei, Balazs Nagy, Apostolos Tsiachristas, Rositsa Koleva-Kolarova, Sarah Wordsworth, Maureen Rutten-van Molken
Summary: This study reviewed the cost-effectiveness of personalized medicine (PM) compared to non-PM. The results showed that while PM brings improvements in health, it is often associated with high costs, resulting in an average net monetary benefit of 0 to negative. Pricing policies may be needed to reduce the costs of interventions with negative net monetary benefit.
Article
Medicine, General & Internal
Tobias B. Polak, David G. J. Cucchi, Jonathan J. Darrow, Matthijs M. Versteegh
Summary: This study evaluates the incremental value of new drugs across various disease areas receiving favorable coverage decisions by the UK's NICE. The findings suggest that the median incremental benefit of these drugs, compared to the best alternative therapeutic option, is equivalent to 3-4 months of life in perfect health. The benefits vary across different drug-indication pairs.
Article
Economics
Bram Wouterse, Pieter van Baal, Matthijs Versteegh, Werner Brouwer
Summary: Cost-effectiveness analysis is important but its practical use is different from its theoretical basis. Many assumptions underlying the theoretical models are violated in practice, leading to the potential failure of improving population health or societal welfare by using a single estimate of the threshold.
Article
Medicine, Research & Experimental
Irene Santi, Andrew J. Lloyd, Claudia E. Hastedt, Matthijs M. Versteegh
Summary: This study aimed to evaluate the impact of cognitive impairment on quality of life using the Schizophrenia Cognition Rating Scale (SCoRS). Five key items were selected from the SCoRS and valued through one-on-one video calls using composite time trade-off (cTTO). The results showed that the SCoRS was more sensitive in detecting cognitive changes compared to the EQ-5D.
ADVANCES IN THERAPY
(2023)
Article
Pharmacology & Pharmacy
Gabor Kovacs, David Nagy, Laszlo Szilberhorn, Tamas Zelei, Zsolt Gaal, Heleen Vellekoop, Simone Huygens, Matthijs Versteegh, Maureen Rutten-van Molken, Rositsa Koleva-Kolarova, Apostolos Tsiachristas, Sarah Wordsworth, Balazs Nagy
Summary: This study aimed to assess the cost-effectiveness of MODY screening strategies in Hungary, comparing recent genetic test with no routine screening for MODY. A simulation model combining a decision tree and an individual-level Markov model was used to evaluate the costs per quality-adjusted life year of screening strategies. Stratifying patients based on age and insulin treatment followed by a risk assessment questionnaire, laboratory test, and genetic testing was found to be the most cost-effective strategy, saving EUR 12 and generating 0.0047 quality-adjusted life years gained per screened patient. This strategy could be considered for reimbursement, especially in countries with limited resources.
PERSONALIZED MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Laszlo Szilberhorn, Tamas Zelei, Heleen Vellekoop, Simone Huygens, Matthijs Versteegh, Maureen Rutten-van Molken, Rositsa Koleva-Kolarova, Apostolos Tsiachristas, Sarah Wordsworth, Balazs Nagy
Summary: Correct diagnosis of MODY is important to provide appropriate treatment. Screening with the MODY calculator then genetic testing is considered cost-effective, and the addition of autoantibody testing can replace no screening. The budget impact ranges from 0.001% to 0.025% of annual public healthcare spending.
PERSONALIZED MEDICINE
(2023)
Editorial Material
Pharmacology & Pharmacy
Maureen Rutten-van Moelken, Matthijs Versteegh, Balazs Nagy, Sarah Wordsworth
PERSONALIZED MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Rositsa Koleva-Kolarova, Heleen Vellekoop, Simone Huygens, Matthijs Versteegh, Maureen Rutten-van Molken, Laszlo Szilberhorn, Tamas Zelei, Balazs Nagy, Sarah Wordsworth, Apostolos Tsiachristas
Summary: The cost-effectiveness and budget impact of introducing extended DPYD testing with ToxNav prior to capecitabine/5-fluorouracil in metastatic breast cancer patients in the UK, The Netherlands, and Hungary were examined. ToxNav was cost-effective in all three countries and resulted in budget savings in the UK and The Netherlands.
PERSONALIZED MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Rositsa Koleva-Kolarova, Heleen Vellekoop, Simone Huygens, Matthijs Versteegh, Maureen Rutten-van Moelken, Laszlo Szilberhorn, Tamas Zelei, Balazs Nagy, Sarah Wordsworth, Apostolos Tsiachristas
Summary: This study aimed to assess the cost-effectiveness of ToxNav (c), a multivariant genetic test, for screening DPYD and personalized chemotherapy dosing in metastatic breast cancer patients in the UK. Results showed that ToxNav (c) was dominant over standard of care, providing additional quality-adjusted life years and cost savings per patient. Sensitivity analysis further supported the effectiveness of the ToxNav (c) strategy.
PERSONALIZED MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Heleen Vellekoop, Simone Huygens, Matthijs Versteegh, Laszlo Szilberhorn, Tamas Zelei, Balazs Nagy, Rositsa Koleva-Kolarova, Sarah Wordsworth, Maureen Rutten-van Molken
Summary: This study investigated the cost-effectiveness of the histology-independent treatment entrectinib for cancer patients with NTRK gene fusions. The analysis conducted in England, Hungary, and The Netherlands found that all testing strategies resulted in a negative net benefit to society. This could be partly explained by the rarity of NTRK gene fusions.
PERSONALIZED MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Rositsa Koleva-Kolarova, Laszlo Szilberhorn, Tamas Zelei, Heleen Vellekoop, Balazs Nagy, Simone Huygens, Matthijs Versteegh, Maureen Rutten-van Molken, Sarah Wordsworth, Apostolos Tsiachristas
Summary: This study examines the financing and reimbursement barriers of personalized medicine in Europe and proposes important recommendations, such as establishing legal foundations, creating databases, using financial-based agreements, regulating price and reimbursement transparency, as well as fostering a business-friendly environment and attractive market for innovation.
PERSONALIZED MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Balazs Nagy, Tamas Zelei, Heleen Vellekoop, Simone Huygens, Matthijs Versteegh, Maureen Rutten-van Moelken, Rositsa Koleva-Kolarova, Apostolos Tsiachristas, Sarah Wordsworth, Laszlo Szilberhorn
Summary: The HEcoPerMed consortium developed a methodological guidance for economic evaluations in personalized medicine, and health economic models were developed to analyze the recommendations. The models addressed most of the recommendations and identified essential recommendations in each study.
PERSONALIZED MEDICINE
(2023)