SEJ393 - Artículos
https://hdl.handle.net/10481/22289
2024-03-28T15:19:13ZA systematic review of the impact of spinal cord injury on costs and health-related quality of life
https://hdl.handle.net/10481/87712
A systematic review of the impact of spinal cord injury on costs and health-related quality of life
Epstein, David; Epstein, David Mark
OBJECTIVES
To systematically review the health-related quality of life (HRQoL) burden and costs of spinal cord injury (SCI) on health services, patients, and wider society.
METHOD
A systematic review guided by the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Statement was conducted through Scopus, PubMed and Embase databases. Descriptive analyses, random-effects direct meta-analysis and random-effects meta-regression were conducted.
RESULTS
A total of 67 studies were eligible for inclusion. SCI individuals tend to report higher HRQoL in mental than physical dimensions of the Short-Form 36. Neurological level of SCI negatively affects HRQoL. Cross-sectional studies find employment is associated with better HRQoL, but the effect is not observed in longitudinal studies. The estimated lifetime expenditure per individual with SCI ranged from $0.5 million to $2.0 million, with greater costs associated with earlier age at injury, neurological level, United States (US) healthcare setting, and the inclusion of non-healthcare items in the study.
CONCLUSION
SCI, and neurological level of injury, are associated with low HRQoL on mobility and physical dimensions. Mental health scores tend to be greater than physical scores, and most dimensions of HRQoL appear to improve over time, at least over the first year. These conditions are associated with high costs which vary by country.
Challenges for Economic Evaluations of Advanced Therapy Medicinal Products: A Systematic Review
https://hdl.handle.net/10481/80488
Challenges for Economic Evaluations of Advanced Therapy Medicinal Products: A Systematic Review
Olry de Labry Lima, Antonio; García Mochón, Leticia; Pérez Troncoso, Daniel; Epstein, David Mark
Objectives: Advanced therapy medicinal products (ATMPs) are drugs for human use for the treatment of chronic, degenerative,
or life-threatening diseases that are based on genes, tissues, or cells. This article aimed to identify and critically review
published economic analyses of ATMPs.
Methods: A systematic review of economic analyses of ATMPs was undertaken. Study characteristics, design, sources of data,
resources and unit costs, modeling and extrapolation methods, study results, and sensitivity analyses were assessed.
Results: A total of 46 economic analyses of ATMP (from 45 articles) were included; 4 were cell therapy medicinal products, 33
gene therapy medicinal products, and 9 tissue-engineered products. 30 therapies had commercial marketing approval; 39
studies were cost-utility analysis, 5 were cost-effectiveness analysis, and 2 were cost only studies. Four studies predicted
that the ATMP offered a step change in the management of the condition and 10 studies estimated that the ATMP would
offer a lower mean cost.
Conclusions: Comparison with historical controls, pooling of data, and use of techniques such as mixture cure fraction models
should be used cautiously. Sensitivity analyses should be used across a plausible range of prices. Clinical studies need to be
designed to align with health technology assessment requirements, including generic quality of life, and payers should aim
for clarity of criteria. Regulators and national payers should aim for compatibility of registers to allow interchange of data.
Given the increasing reliance on industry-funded economic analyses, careful critical review is recommended.
Study protocol for a multicentre comparative diagnostic accuracy study of tools to establish the presence and severity of peripheral arterial disease in people with diabetes mellitus: the DM PAD study
https://hdl.handle.net/10481/80376
Study protocol for a multicentre comparative diagnostic accuracy study of tools to establish the presence and severity of peripheral arterial disease in people with diabetes mellitus: the DM PAD study
Normahani, Pasha; Epstein, David Mark; DM PAD Study
Introduction Peripheral arterial disease (PAD) is a key risk
factor for cardiovascular disease, foot ulceration and lower
limb amputation in people with diabetes. Early diagnosis of
PAD can enable optimisation of therapies to manage these
risks. Its diagnosis is fundamental, though challenging in
the context of diabetes. Although a variety of diagnostic
bedside tests are available, there is no agreement as to
which is the most accurate in routine clinical practice.
The aim of this study is to determine the diagnostic
performance of a variety of tests (audible waveform
assessment, visual waveform assessment, ankle brachial
pressure index (ABPI), exercise ABPI and toe brachial
pressure index (TBPI)) for the diagnosis of PAD in people
with diabetes as determined by a reference test (CT
angiography (CTA) or magnetic resonance angiography
(MRA)). In selected centres, we also aim to evaluate the
performance of a new point-of-
care
duplex ultrasound
scan (PAD-scan).
Methods and analysis A prospective multicentre
diagnostic accuracy study ( ClinicalTrials. gov Identifier
NCT05009602). We aim to recruit 730 people with
diabetes from 18 centres across the UK, covering primary
and secondary healthcare. Consenting participants will
undergo the tests under investigation. Reference tests
(CTA or MRA) will be performed within 6 weeks of the
index tests. Imaging will be reported by blinded consultant
radiologists at a core imaging lab, using a validated
scoring system, which will also be used to categorise PAD
severity. The presence of one or more arterial lesions of
≥50% stenosis, or tandem lesions with a combined value
of ≥50%, will be used as the threshold for the diagnosis
of PAD. The primary outcome measure of diagnostic
performance will be test sensitivity.
Ethics and dissemination The study has received
approval from the National Research Ethics Service (NRES)
(REC reference 21/PR/1221). Results will be disseminated
through research presentations and papers.
Examining the benefit of graduated compression stockings in the prevention of hospital-associated venous thromboembolism in low-risk surgical patients: a multicentre cluster randomised controlled trial (PETS trial)
https://hdl.handle.net/10481/80116
Examining the benefit of graduated compression stockings in the prevention of hospital-associated venous thromboembolism in low-risk surgical patients: a multicentre cluster randomised controlled trial (PETS trial)
Machin, Matthew; Epstein, David Mark
Introduction Hospital-acquired
thrombosis (HAT) is
defined as any venous thromboembolism (VTE)-related
event during a hospital admission or occurring up to 90
days post discharge, and is associated with significant
morbidity, mortality and healthcare-associated
costs.
Although surgery is an established risk factor for VTE,
operations with a short hospital stay (<48 hours) and
that permit early ambulation are associated with a low
risk of VTE. Many patients undergoing short-stay
surgical
procedures and who are at low risk of VTE are treated
with graduated compression stockings (GCS). However,
evidence for the use of GCS in VTE prevention for this
cohort is poor.
Methods and analysis A multicentre, cluster randomised
controlled trial which aims to determine whether GCS are
superior in comparison to no GCS in the prevention of VTE for
surgical patients undergoing short-stay
procedures assessed
to be at low risk of VTE. A total of 50 sites (21 472 participants)
will be randomised to either intervention (GCS) or control
(no GCS). Adult participants (18–59 years) who undergo
short-stay
surgical procedures and are assessed as low risk
of VTE will be included in the study. Participants will provide
consent to be contacted for follow-up
at 7-days
and 90-days
postsurgical procedure. The primary outcome is the rate of
symptomatic VTE, that is, deep vein thrombosis or pulmonary
embolism during admission or within 90 days. Secondary
outcomes include healthcare costs and changes in quality of
life. The main analysis will be according to the intention-to-
treat
principle and will compare the rates of VTE at 90 days,
measured at an individual level, using hierarchical (multilevel)
logistic regression.
Barriers for the evaluation of advanced therapy medicines and their translation to clinical practice: Umbrella review
https://hdl.handle.net/10481/78945
Barriers for the evaluation of advanced therapy medicines and their translation to clinical practice: Umbrella review
Aguilera Cobos, Lorena; Epstein, David Mark
Advanced therapy medicinal products (ATMPs) are a fast-growing field of medicine with wide potential application.
Nevertheless, so far, only 19 have obtained European Union (EU) marketing authorisation and only 13 of
these have translated successfully into clinical practice. This study conducts an umbrella review to identify the
main barriers for the evaluation of ATMPs and their translation into clinical practice across the development
lifecycle. 71 systematic reviews were included, of which 50 dealt primarily with effectiveness and safety, 13 with
translation from pre-clinical to human subjects. Others dealt with economic issues and translation from health
technology assessment to market access. The literature highlights the importance of synergistic research groups
or networks that collaborate across the in-vitro science, preclinical and clinical investigation phases, and the role
of private investor capital and public-private collaborations. Most ATMPs reviewed seem to have a favourable
safety profile although considerable uncertainties remain. Randomised controlled trials are not always feasible in
these patient groups. Greater sharing of data is recommended, both at preclinical and post-marketing real world
evidence. There are considerable variations between EU countries in how they regulate hospital exemption for
ATMPs, and this can lead to inequitable access for patients.