Clinically meaningful benefit and real‐world evidence in Alzheimer's disease research and care
Abstract In the realm of medical research, assessing novel therapies extends beyond statistical significance. The concept of meaningful benefits plays a pivotal role in determining the practical impact of interventions on patient outcomes. Clinical trials, which form the bedrock of evidence‐based me...
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| Format: | Article |
| Language: | English |
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Wiley
2025-04-01
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| Series: | Alzheimer’s & Dementia: Translational Research & Clinical Interventions |
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| Online Access: | https://doi.org/10.1002/trc2.70090 |
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| author | Robert Perneczky Lutz Froelich |
| author_facet | Robert Perneczky Lutz Froelich |
| author_sort | Robert Perneczky |
| collection | DOAJ |
| description | Abstract In the realm of medical research, assessing novel therapies extends beyond statistical significance. The concept of meaningful benefits plays a pivotal role in determining the practical impact of interventions on patient outcomes. Clinical trials, which form the bedrock of evidence‐based medicine, guide treatment decisions and shape health‐care practices. While statistical significance remains a fundamental criterion, it falls short in fully evaluating the clinical relevance of therapeutic interventions. Clinically meaningful benefits focus on tangible improvements in patient health and well‐being, transcending mere statistical thresholds. Key considerations include survival rates, symptom relief, functional status, and other patient‐oriented outcomes. Determining meaningful benefits varies across diseases, patient populations, and available treatments. Balancing statistical rigor with clinical relevance is crucial. Overpowered trials may detect smaller differences than anticipated, necessitating careful interpretation. Researchers must view trial results through a patient‐centric lens. Beyond survival, evaluating quality of life and side effects is equally relevant. Quantifying meaningful benefits involves metrics like numbers needed to treat and progression‐free survival. Consistency across outcomes matters, as clinicians weigh gains in survival against improvements in quality of life. The pursuit of meaningful benefits elevates clinical trials from mere statistical exercises to patient‐centered endeavors. Researchers, clinicians, and regulators must prioritize outcomes that genuinely matter to patients, ensuring that medical progress translates into meaningful improvements for them and their families. |
| format | Article |
| id | doaj-art-8ab5978ce3f4464f9bb89b964865fe74 |
| institution | OA Journals |
| issn | 2352-8737 |
| language | English |
| publishDate | 2025-04-01 |
| publisher | Wiley |
| record_format | Article |
| series | Alzheimer’s & Dementia: Translational Research & Clinical Interventions |
| spelling | doaj-art-8ab5978ce3f4464f9bb89b964865fe742025-08-20T02:22:09ZengWileyAlzheimer’s & Dementia: Translational Research & Clinical Interventions2352-87372025-04-01112n/an/a10.1002/trc2.70090Clinically meaningful benefit and real‐world evidence in Alzheimer's disease research and careRobert Perneczky0Lutz Froelich1Department of Psychiatry and Psychotherapy University Hospital, LMU Munich Munich GermanyDepartment of Geriatric Psychiatry Central Institute of Mental Health Mannheim GermanyAbstract In the realm of medical research, assessing novel therapies extends beyond statistical significance. The concept of meaningful benefits plays a pivotal role in determining the practical impact of interventions on patient outcomes. Clinical trials, which form the bedrock of evidence‐based medicine, guide treatment decisions and shape health‐care practices. While statistical significance remains a fundamental criterion, it falls short in fully evaluating the clinical relevance of therapeutic interventions. Clinically meaningful benefits focus on tangible improvements in patient health and well‐being, transcending mere statistical thresholds. Key considerations include survival rates, symptom relief, functional status, and other patient‐oriented outcomes. Determining meaningful benefits varies across diseases, patient populations, and available treatments. Balancing statistical rigor with clinical relevance is crucial. Overpowered trials may detect smaller differences than anticipated, necessitating careful interpretation. Researchers must view trial results through a patient‐centric lens. Beyond survival, evaluating quality of life and side effects is equally relevant. Quantifying meaningful benefits involves metrics like numbers needed to treat and progression‐free survival. Consistency across outcomes matters, as clinicians weigh gains in survival against improvements in quality of life. The pursuit of meaningful benefits elevates clinical trials from mere statistical exercises to patient‐centered endeavors. Researchers, clinicians, and regulators must prioritize outcomes that genuinely matter to patients, ensuring that medical progress translates into meaningful improvements for them and their families.https://doi.org/10.1002/trc2.70090Alzheimer's disease and neurodegenerationbiomarker‐based early and accurate diagnosisdisease prevention and early interventiondisease‐modifying and symptomatic treatmentmild cognitive impairment and dementiapatient autonomy and independence |
| spellingShingle | Robert Perneczky Lutz Froelich Clinically meaningful benefit and real‐world evidence in Alzheimer's disease research and care Alzheimer’s & Dementia: Translational Research & Clinical Interventions Alzheimer's disease and neurodegeneration biomarker‐based early and accurate diagnosis disease prevention and early intervention disease‐modifying and symptomatic treatment mild cognitive impairment and dementia patient autonomy and independence |
| title | Clinically meaningful benefit and real‐world evidence in Alzheimer's disease research and care |
| title_full | Clinically meaningful benefit and real‐world evidence in Alzheimer's disease research and care |
| title_fullStr | Clinically meaningful benefit and real‐world evidence in Alzheimer's disease research and care |
| title_full_unstemmed | Clinically meaningful benefit and real‐world evidence in Alzheimer's disease research and care |
| title_short | Clinically meaningful benefit and real‐world evidence in Alzheimer's disease research and care |
| title_sort | clinically meaningful benefit and real world evidence in alzheimer s disease research and care |
| topic | Alzheimer's disease and neurodegeneration biomarker‐based early and accurate diagnosis disease prevention and early intervention disease‐modifying and symptomatic treatment mild cognitive impairment and dementia patient autonomy and independence |
| url | https://doi.org/10.1002/trc2.70090 |
| work_keys_str_mv | AT robertperneczky clinicallymeaningfulbenefitandrealworldevidenceinalzheimersdiseaseresearchandcare AT lutzfroelich clinicallymeaningfulbenefitandrealworldevidenceinalzheimersdiseaseresearchandcare |