19 research outputs found
Valuing Alzheimer's Disease drugs:A health technology assessment perspective on outcomes
ObjectivesDue to the nature of Alzheimer's disease (AD), health technology assessment (HTA) agencies might face considerable challenges in choosing appropriate outcomes and outcome measures for drugs that treat the condition. This study sought to understand which outcomes informed previous HTAs, to explore possible reasons for prioritizations, and derive potential implications for future assessments of AD drugs.MethodWe conducted a literature review of studies that analyzed decisions made in HTAs (across disease areas) in three European countries: England, Germany, and The Netherlands. We then conducted case studies of technology assessments conducted for AD drugs in these countries.ResultsOverall, outcomes measured using clinical scales dominated decisions or recommendations about whether to fund AD drugs, or price negotiations. HTA processes did not always allow the inclusion of outcomes relevant to people with AD, their carers, and families. Processes did not include early discussion and agreement on what would constitute appropriate outcome measures and cut-off points for effects.ConclusionsWe conclude that in order to ensure that future AD drugs are valued appropriately and timely, early agreement with various stakeholders about outcomes, outcome measures, and cut-offs is important
Conducting public involvement in dementia research:The contribution of the European Working Group of People with Dementia to the ROADMAP project
Background
Dementia outcomes include memory loss, language impairment, reduced quality of life and personality changes. Research suggests that outcomes selected for dementia clinical trials might not be the most important to people affected.
Objective
One of the goals of the âReal world Outcomes across the Alzheimer's Disease spectrum for better care: Multiâmodal data Access Platformâ (ROADMAP) project was to identify important outcomes from the perspective of people with dementia and their caregivers. We review how ROADMAP's Public Involvement shaped the programme, impacted the research process and gave voice to people affected by dementia.
Design
The European Working Group of People with Dementia (EWGPWD) were invited to participate. Inâperson consultations were held with people with dementia and caregivers, with advance information provided on ROADMAP activities. Constructive criticism of survey content, layout and accessibility was sought, as were views and perspectives on terminology and key concepts around disease progression.
Results
The working group provided significant improvements to survey accessibility and acceptability. They promoted better understanding of concepts around disease progression and how researchers might approach measuring and interpreting findings. They effectively expressed difficult concepts through realâworld examples.
Conclusions
The role of the EWGPWD in ROADMAP was crucial, and its impact was highly influential. Involvement from the design stage helped shape the ethos of the programme and ultimately its meaningfulness.
Public contribution
People with dementia and their carers were involved through structured consultations and invited to provide feedback on project materials, methods and insight into terminology and relevant concepts
What outcomes are important to patients with mild cognitive impairment or Alzheimer's disease, their caregivers, and health-care professionals? A systematic review
Introduction: Clinical trials involving patients with Alzheimerâs disease (AD) continue to try to identify disease-modifying treatments. Although trials are designed to meet regulatory and registration requirements, many do not measure outcomes of the disease most relevant to key stakeholders.
Methods: A systematic review sought research that elicited information from people with AD, their caregivers, and health-care professionals on which outcomes of the disease were important. Studies published in any language between 2008 and 2017 were included.
Results: Participants in 34 studies described 32 outcomes of AD. These included clinical (memory, mental health), practical (ability to undertake activities of daily living, access to health information), and personal (desire for patient autonomy, maintenance of identity) outcomes of the disease.
Discussion: Evidence elicited directly from the people most affected by AD reveals a range of disease outcomes that are relevant to them but are not commonly captured in clinical trials of new treatments.</br
Real-world evidence in Alzheimerâs disease: the ROADMAP Data Cube
INTRODUCTION:The ROADMAP project aimed to provide an integrated overview of European real-world data on Alzheimer's disease (AD) across the disease spectrum. METHODS:Metadata were identified from data sources in catalogs of European AD projects. Priority outcomes for different stakeholders were identified through systematic literature review, patient and public consultations, and stakeholder surveys. RESULTS:Information about 66 data sources and 13 outcome domains were integrated into a Data Cube. Gap analysis identified cognitive ability, functional ability/independence, behavioral/neuropsychiatric symptoms, treatment, comorbidities, and mortality as the outcomes collected most. Data were most lacking in caregiver-related outcomes. In general, electronic health records covered a broader, less detailed data spectrum than research cohorts. DISCUSSION:This integrated real-world AD data overview provides an intuitive visual model that facilitates initial assessment and identification of gaps in relevant outcomes data to inform future prospective data collection and matching of data sources and outcomes against research protocols
E-epidemiology : Adapting epidemiological methods for the 21st century
The traditional methods in epidemiological data collection are both
costly and time consuming and less convenient for longitudinal
large-scale studies. During the last decades, epidemiological studies
suffer from low response rates, indicating a need to revise methods used
in epidemiological data collection. e-epidemiology is the science
underlying usage of Information and Communication Technologies (ICT) in
epidemiological studies and enable new possibilities for data collection.
In this thesis four studies evaluating methods including mobile phones,
the web and Interactive Voice Response (IVR) are described.
In study I, the feasibility of using an Internet-based hearing test
combined with a web-based questionnaire was evaluated in a pilot study
among Swedish hunters. The response rate was very low with a bias toward
older individuals (40-60 years) who had access to the correct equipment
at study start. Though a number of limitations, the hearing-test
demonstrates a possibility of using the web in epidemiological data
collection. In study II, repeated measures of physical activity level
(PAL) through a Java-based questionnaire in mobile phones were compared
to a gold standard of measuring energy expenditure. The Java-based
physical activity questionnaire sent repeatedly through mobile phones
produced average PAL estimates that agreed well with PAL reference
values, indicating that the method may be a feasible and cost effective
method for data collection on physical activity. Study III compared data
collected through Short Message Service (SMS) to traditional telephone
interviews in a population-based sample. Though the study produced very
low response rate, the results on influenza vaccination status was not
statistically significantly different from data collected through
telephone interviews. Study IV compared data on self-reports on
infectious disease where the participants could choose between web and
IVR. The web was more popular than IVR and attracted more men and younger
individuals with a higher completed education compared to IVR. There was
no statistically significantly difference of reported infections or
Influenza-Like Illness (ILI) between the two techniques after adjusting
for available confounders.
Studies I, III and IV were affected by low response rates, effecting both
the validity and precision of the results. All studies were affected by
bias and all but study II were probably confounded by age. The mechanisms
behind these factors are important to evaluate further in order to
understand how it affects the collected data. However, when possible to
adjust for confounders, the techniques per se did not seem to influence
data negatively compared to reference data. All studies were evaluated on
a Swedish population with high access to the Internet and mobile phones,
and the results might not be generalizable to populations with less
access. This thesis has demonstrated a fraction of the possibilities
using ICT in epidemiological data collection and e-epidemiology is still
in its youth. Once the techniques have been thoroughly evaluated, there
are probably endless possibilities to ensure high quality data collection
through methods adapted to a modern society
Estimation of Physical Activity Levels Using Cell Phone Questionnaires: A Comparison With Accelerometry for Evaluation of Between-Subject and Within-Subject Variations
Background: Physical activity promotes health and longevity. Further elaboration of the role of physical activity for human health in epidemiological studies on large samples requires accurate methods that are easy to use, cheap, and possible to repeat. The use of telecommunication technologies such as cell phones is highly interesting in this respect. In an earlier report, we showed that physical activity level (PAL) assessed using a cell phone procedure agreed well with corresponding estimates obtained using the doubly labeled water method. However, our earlier study indicated high within-subject variation in relation to between-subject variations in PAL using cell phones, but we could not assess if this was a true variation of PAL or an artifact of the cell phone technique. less thanbrgreater than less thanbrgreater thanObjective: Our objective was to compare within-and between-subject variations in PAL by means of cell phones with corresponding estimates using an accelerometer. In addition, we compared the agreement of daily PAL values obtained using the cell phone questionnaire with corresponding data obtained using an accelerometer. less thanbrgreater than less thanbrgreater thanMethods: PAL was measured both with the cell phone questionnaire and with a triaxial accelerometer daily during a 2-week study period in 21 healthy Swedish women (20 to 45 years of age and BMI from 17.7 kg/m(2) to 33.6 kg/m(2)). The results were evaluated by fitting linear mixed effect models and descriptive statistics and graphs. less thanbrgreater than less thanbrgreater thanResults: With the accelerometer, 57% (95% confidence interval [CI] 40%-66%) of the variation was within subjects, while with the cell phone, within-subject variation was 76% (95% CI 59%-83%). The day-to-day variations in PAL observed using the cell phone questions agreed well with the corresponding accelerometer results. less thanbrgreater than less thanbrgreater thanConclusions: Both the cell phone questionnaire and the accelerometer showed high within-subject variations. Furthermore, day-to-day variations in PAL within subjects assessed using the cell phone agreed well with corresponding accelerometer values. Consequently, our cell phone questionnaire is a promising tool for assessing levels of physical activity. The tool may be useful for large-scale prospective studies.Funding Agencies|Swedish Research Council, FORMAS||Magnus Bergvall Foundation||Thuring Foundation||Swedish Society of Medicine|
The Validity of Self-Initiated, Event-Driven Infectious Disease Reporting in General Population Cohorts
<div><p>Background</p><p>The 2009/2010 pandemic influenza highlighted the need for valid and timely incidence data. In 2007 we started the development of a passive surveillance scheme based on passive follow-up of representative general population cohorts. Cohort members are asked to spontaneously report all instances of colds and fevers as soon as they occur for up to 9 months. Suspecting that compliance might be poor, we aimed to assess the validity of self-initiated, event-driven outcome reporting over long periods.</p><p>Methods</p><p>During two 8 week periods in 2008 and 2009, 2376 and 2514 cohort members in Stockholm County were sent one-week recall questionnaires, which served as reference method.</p><p>Results</p><p>The questionnaires were completed by 88% and 86% of the cohort members. Whilst the false positive proportion (1âspecificity) in the reporting was low (upper bound of the 95% confidence interval [CI] â€2% in each season), the false negative proportion (failure to report, 1âsensitivity) was considerable (60% [95% CI 52%â67%] in each season). Still, the resulting epidemic curves for influenza-like illness compared well with those from existing General Practitioner-based sentinel surveillance in terms of shape, timing of peak, and year-to-year variation. This suggested that the error was fairly constant.</p><p>Conclusions</p><p>Passive long-term surveillance through self-initiated, event-driven outcome reporting underestimates incidence rates of common upper respiratory tract infections. However, because underreporting appears predictable, simple corrections could potentially restore validity.</p></div
Epidemic curves for influenza-like illness (ILI).
<p>The curves are derived from the passive follow-up with self-initiated, event-driven outcome reporting in population-based surveillance cohorts (solid line) and adjusted for imperfect sensitivity (dashed line). The upper graph represents 2008, the lower one 2009.</p