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Trends in Pharmacological Sciences Aug 2019Clinical trials consume the latter half of the 10 to 15 year, 1.5-2.0 billion USD, development cycle for bringing a single new drug to market. Hence, a failed trial... (Review)
Review
Clinical trials consume the latter half of the 10 to 15 year, 1.5-2.0 billion USD, development cycle for bringing a single new drug to market. Hence, a failed trial sinks not only the investment into the trial itself but also the preclinical development costs, rendering the loss per failed clinical trial at 800 million to 1.4 billion USD. Suboptimal patient cohort selection and recruiting techniques, paired with the inability to monitor patients effectively during trials, are two of the main causes for high trial failure rates: only one of 10 compounds entering a clinical trial reaches the market. We explain how recent advances in artificial intelligence (AI) can be used to reshape key steps of clinical trial design towards increasing trial success rates.
Topics: Artificial Intelligence; Clinical Protocols; Clinical Trials as Topic; Clinical Trials, Phase III as Topic; Drug Development; Humans; Patient Compliance; Patient Selection
PubMed: 31326235
DOI: 10.1016/j.tips.2019.05.005 -
Evidence Report/technology Assessment Mar 2011To update a 2004 systematic review of health care service use and health outcomes related to differences in health literacy level and interventions designed to improve... (Review)
Review
OBJECTIVES
To update a 2004 systematic review of health care service use and health outcomes related to differences in health literacy level and interventions designed to improve these outcomes for individuals with low health literacy. Disparities in health outcomes and effectiveness of interventions among different sociodemographic groups were also examined.
DATA SOURCES
We searched MEDLINE®, the Cumulative Index to Nursing and Allied Health Literature, the Cochrane Library, PsychINFO, and the Educational Resources Information Center. For health literacy, we searched using a variety of terms, limited to English and studies published from 2003 to May 25, 2010. For numeracy, we searched from 1966 to May 25, 2010.
REVIEW METHODS
We used standard Evidence-based Practice Center methods of dual review of abstracts, full-text articles, abstractions, quality ratings, and strength of evidence grading. We resolved disagreements by consensus. We evaluated whether newer literature was available for answering key questions, so we broadened our definition of health literacy to include numeracy and oral (spoken) health literacy. We excluded intervention studies that did not measure health literacy directly and updated our approach to evaluate individual study risk of bias and to grade strength of evidence.
RESULTS
We included good- and fair-quality studies: 81 studies addressing health outcomes (reported in 95 articles including 86 measuring health literacy and 16 measuring numeracy, of which 7 measure both) and 42 studies (reported in 45 articles) addressing interventions. Differences in health literacy level were consistently associated with increased hospitalizations, greater emergency care use, lower use of mammography, lower receipt of influenza vaccine, poorer ability to demonstrate taking medications appropriately, poorer ability to interpret labels and health messages, and, among seniors, poorer overall health status and higher mortality. Health literacy level potentially mediates disparities between blacks and whites. The strength of evidence of numeracy studies was insufficient to low, limiting conclusions about the influence of numeracy on health care service use or health outcomes. Two studies suggested numeracy may mediate the effect of disparities on health outcomes. We found no evidence concerning oral health literacy and outcomes. Among intervention studies (27 randomized controlled trials [RCTs], 2 cluster RCTs, and 13 quasi-experimental designs), the strength of evidence for specific design features was low or insufficient. However, several specific features seemed to improve comprehension in one or a few studies. The strength of evidence was moderate for the effect of mixed interventions on health care service use; the effect of intensive self-management inventions on behavior; and the effect of disease-management interventions on disease prevalence/severity. The effects of other mixed interventions on other health outcomes, including knowledge, self-efficacy, adherence, and quality of life, and costs were mixed; thus, the strength of evidence was insufficient.
CONCLUSIONS
The field of health literacy has advanced since the 2004 report. Future research priorities include justifying appropriate cutoffs for health literacy levels prior to conducting studies; developing tools that measure additional related skills, particularly oral (spoken) health literacy; and examining mediators and moderators of the effect of health literacy. Priorities in advancing the design features of interventions include testing novel approaches to increase motivation, techniques for delivering information orally or numerically, "work around" interventions such as patient advocates; determining the effective components of already-tested interventions; determining the cost-effectiveness of programs; and determining the effect of policy and practice interventions.
Topics: Cause of Death; Emergency Medical Services; Health Literacy; Health Status; Hospitalization; Humans; Influenza Vaccines; Mammography; Outcome Assessment, Health Care; Patient Compliance; Randomized Controlled Trials as Topic
PubMed: 23126607
DOI: No ID Found -
The Cochrane Database of Systematic... Jan 2021Pulmonary rehabilitation is a proven, effective intervention for people with chronic respiratory diseases including chronic obstructive pulmonary disease (COPD),... (Meta-Analysis)
Meta-Analysis
BACKGROUND
Pulmonary rehabilitation is a proven, effective intervention for people with chronic respiratory diseases including chronic obstructive pulmonary disease (COPD), interstitial lung disease (ILD) and bronchiectasis. However, relatively few people attend or complete a program, due to factors including a lack of programs, issues associated with travel and transport, and other health issues. Traditionally, pulmonary rehabilitation is delivered in-person on an outpatient basis at a hospital or other healthcare facility (referred to as centre-based pulmonary rehabilitation). Newer, alternative modes of pulmonary rehabilitation delivery include home-based models and the use of telehealth. Telerehabilitation is the delivery of rehabilitation services at a distance, using information and communication technology. To date, there has not been a comprehensive assessment of the clinical efficacy or safety of telerehabilitation, or its ability to improve uptake and access to rehabilitation services, for people with chronic respiratory disease.
OBJECTIVES
To determine the effectiveness and safety of telerehabilitation for people with chronic respiratory disease.
SEARCH METHODS
We searched the Cochrane Airways Trials Register, and the Cochrane Central Register of Controlled Trials; six databases including MEDLINE and Embase; and three trials registries, up to 30 November 2020. We checked reference lists of all included studies for additional references, and handsearched relevant respiratory journals and meeting abstracts.
SELECTION CRITERIA
All randomised controlled trials and controlled clinical trials of telerehabilitation for the delivery of pulmonary rehabilitation were eligible for inclusion. The telerehabilitation intervention was required to include exercise training, with at least 50% of the rehabilitation intervention being delivered by telerehabilitation.
DATA COLLECTION AND ANALYSIS
We used standard methods recommended by Cochrane. We assessed the risk of bias for all studies, and used the ROBINS-I tool to assess bias in non-randomised controlled clinical trials. We assessed the certainty of evidence with GRADE. Comparisons were telerehabilitation compared to traditional in-person (centre-based) pulmonary rehabilitation, and telerehabilitation compared to no rehabilitation. We analysed studies of telerehabilitation for maintenance rehabilitation separately from trials of telerehabilitation for initial primary pulmonary rehabilitation.
MAIN RESULTS
We included a total of 15 studies (32 reports) with 1904 participants, using five different models of telerehabilitation. Almost all (99%) participants had chronic obstructive pulmonary disease (COPD). Three studies were controlled clinical trials. For primary pulmonary rehabilitation, there was probably little or no difference between telerehabilitation and in-person pulmonary rehabilitation for exercise capacity measured as 6-Minute Walking Distance (6MWD) (mean difference (MD) 0.06 metres (m), 95% confidence interval (CI) -10.82 m to 10.94 m; 556 participants; four studies; moderate-certainty evidence). There may also be little or no difference for quality of life measured with the St George's Respiratory Questionnaire (SGRQ) total score (MD -1.26, 95% CI -3.97 to 1.45; 274 participants; two studies; low-certainty evidence), or for breathlessness on the Chronic Respiratory Questionnaire (CRQ) dyspnoea domain score (MD 0.13, 95% CI -0.13 to 0.40; 426 participants; three studies; low-certainty evidence). Participants were more likely to complete a program of telerehabilitation, with a 93% completion rate (95% CI 90% to 96%), compared to a 70% completion rate for in-person rehabilitation. When compared to no rehabilitation control, trials of primary telerehabilitation may increase exercise capacity on 6MWD (MD 22.17 m, 95% CI -38.89 m to 83.23 m; 94 participants; two studies; low-certainty evidence) and may also increase 6MWD when delivered as maintenance rehabilitation (MD 78.1 m, 95% CI 49.6 m to 106.6 m; 209 participants; two studies; low-certainty evidence). No adverse effects of telerehabilitation were noted over and above any reported for in-person rehabilitation or no rehabilitation.
AUTHORS' CONCLUSIONS
This review suggests that primary pulmonary rehabilitation, or maintenance rehabilitation, delivered via telerehabilitation for people with chronic respiratory disease achieves outcomes similar to those of traditional centre-based pulmonary rehabilitation, with no safety issues identified. However, the certainty of the evidence provided by this review is limited by the small number of studies, of varying telerehabilitation models, with relatively few participants. Future research should consider the clinical effect of telerehabilitation for individuals with chronic respiratory diseases other than COPD, the duration of benefit of telerehabilitation beyond the period of the intervention, and the economic cost of telerehabilitation.
Topics: Bias; Chronic Disease; Controlled Clinical Trials as Topic; Dyspnea; Exercise Tolerance; Humans; Internet; Non-Randomized Controlled Trials as Topic; Patient Compliance; Pulmonary Disease, Chronic Obstructive; Quality of Life; Randomized Controlled Trials as Topic; Respiration Disorders; Telephone; Telerehabilitation; Videoconferencing; Walk Test
PubMed: 33511633
DOI: 10.1002/14651858.CD013040.pub2 -
The Cochrane Database of Systematic... Feb 2019International clinical practice guidelines routinely recommend that cardiac patients participate in rehabilitation programmes for comprehensive secondary prevention.... (Meta-Analysis)
Meta-Analysis
BACKGROUND
International clinical practice guidelines routinely recommend that cardiac patients participate in rehabilitation programmes for comprehensive secondary prevention. However, data show that only a small proportion of these patients utilise rehabilitation.
OBJECTIVES
First, to assess interventions provided to increase patient enrolment in, adherence to, and completion of cardiac rehabilitation. Second, to assess intervention costs and associated harms, as well as interventions intended to promote equitable CR utilisation in vulnerable patient subpopulations.
SEARCH METHODS
Review authors performed a search on 10 July 2018, to identify studies published since publication of the previous systematic review. We searched the Cochrane Central Register of Controlled Trials (CENTRAL); the National Health Service (NHS) Centre for Reviews and Dissemination (CRD) databases (Health Technology Assessment (HTA) and Database of Abstracts of Reviews of Effects (DARE)), in the Cochrane Library (Wiley); MEDLINE (Ovid); Embase (Elsevier); the Cumulative Index to Nursing and Allied Health Literature (CINAHL) (EBSCOhost); and Conference Proceedings Citation Index - Science (CPCI-S) on Web of Science (Clarivate Analytics). We checked the reference lists of relevant systematic reviews for additional studies and also searched two clinical trial registers. We applied no language restrictions.
SELECTION CRITERIA
We included randomised controlled trials (RCTs) in adults with myocardial infarction, with angina, undergoing coronary artery bypass graft surgery or percutaneous coronary intervention, or with heart failure who were eligible for cardiac rehabilitation. Interventions had to aim to increase utilisation of comprehensive phase II cardiac rehabilitation. We included only studies that measured one or more of our primary outcomes. Secondary outcomes were harms and costs, and we focused on equity.
DATA COLLECTION AND ANALYSIS
Two review authors independently screened the titles and abstracts of all identified references for eligibility, and we obtained full papers of potentially relevant trials. Two review authors independently considered these trials for inclusion, assessed included studies for risk of bias, and extracted trial data independently. We resolved disagreements through consultation with a third review author. We performed random-effects meta-regression for each outcome and explored prespecified study characteristics.
MAIN RESULTS
Overall, we included 26 studies with 5299 participants (29 comparisons). Participants were primarily male (64.2%). Ten (38.5%) studies included patients with heart failure. We assessed most studies as having low or unclear risk of bias. Sixteen studies (3164 participants) reported interventions to improve enrolment in cardiac rehabilitation, 11 studies (2319 participants) reported interventions to improve adherence to cardiac rehabilitation, and seven studies (1567 participants) reported interventions to increase programme completion. Researchers tested a variety of interventions to increase utilisation of cardiac rehabilitation. In many studies, this consisted of contacts made by a healthcare provider during or shortly after an acute care hospitalisation.Low-quality evidence shows an effect of interventions on increasing programme enrolment (19 comparisons; risk ratio (RR) 1.27, 95% confidence interval (CI) 1.13 to 1.42). Meta-regression revealed that the intervention deliverer (nurse or allied healthcare provider; P = 0.02) and the delivery format (face-to-face; P = 0.01) were influential in increasing enrolment. Low-quality evidence shows interventions to increase adherence were effective (nine comparisons; standardised mean difference (SMD) 0.38, 95% CI 0.20 to 0.55), particularly when they were delivered remotely, such as in home-based programs (SMD 0.56, 95% CI 0.37 to 0.76). Moderate-quality evidence shows interventions to increase programme completion were also effective (eight comparisons; RR 1.13, 95% CI 1.02 to 1.25), but those applied in multi-centre studies were less effective than those given in single-centre studies, leading to questions regarding generalisability. A moderate level of statistical heterogeneity across intervention studies reflects heterogeneity in intervention approaches. There was no evidence of small-study bias for enrolment (insufficient studies to test for this in the other outcomes).With regard to secondary outcomes, no studies reported on harms associated with the interventions. Only two studies reported costs. In terms of equity, trialists tested interventions designed to improve utilisation among women and older patients. Evidence is insufficient for quantitative assessment of whether women-tailored programmes were associated with increased utilisation, and studies that assess motivating women are needed. For older participants, again while quantitative assessment could not be undertaken, peer navigation may improve enrolment.
AUTHORS' CONCLUSIONS
Interventions may increase cardiac rehabilitation enrolment, adherence and completion; however the quality of evidence was low to moderate due to heterogeneity of the interventions used, among other factors. Effects on enrolment were larger in studies targeting healthcare providers, training nurses, or allied healthcare providers to intervene face-to-face; effects on adherence were larger in studies that tested remote interventions. More research is needed, particularly to discover the best ways to increase programme completion.
Topics: Adult; Angina Pectoris; Angioplasty, Balloon, Coronary; Cardiac Rehabilitation; Coronary Artery Bypass; Coronary Disease; Exercise; Female; Heart Failure; Humans; Male; Middle Aged; Myocardial Infarction; Patient Acceptance of Health Care; Patient Compliance; Randomized Controlled Trials as Topic; Secondary Prevention
PubMed: 30706942
DOI: 10.1002/14651858.CD007131.pub4 -
Journal of Medical Internet Research Feb 2015Adherence to chronic disease management is critical to achieving improved health outcomes, quality of life, and cost-effective health care. As the burden of chronic... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Adherence to chronic disease management is critical to achieving improved health outcomes, quality of life, and cost-effective health care. As the burden of chronic diseases continues to grow globally, so does the impact of non-adherence. Mobile technologies are increasingly being used in health care and public health practice (mHealth) for patient communication, monitoring, and education, and to facilitate adherence to chronic diseases management.
OBJECTIVE
We conducted a systematic review of the literature to evaluate the effectiveness of mHealth in supporting the adherence of patients to chronic diseases management ("mAdherence"), and the usability, feasibility, and acceptability of mAdherence tools and platforms in chronic disease management among patients and health care providers.
METHODS
We searched PubMed, Embase, and EBSCO databases for studies that assessed the role of mAdherence in chronic disease management of diabetes mellitus, cardiovascular disease, and chronic lung diseases from 1980 through May 2014. Outcomes of interest included effect of mHealth on patient adherence to chronic diseases management, disease-specific clinical outcomes after intervention, and the usability, feasibility, and acceptability of mAdherence tools and platforms in chronic disease management among target end-users.
RESULTS
In all, 107 articles met all inclusion criteria. Short message service was the most commonly used mAdherence tool in 40.2% (43/107) of studies. Usability, feasibility, and acceptability or patient preferences for mAdherence interventions were assessed in 57.9% (62/107) of studies and found to be generally high. A total of 27 studies employed randomized controlled trial (RCT) methods to assess impact on adherence behaviors, and significant improvements were observed in 15 of those studies (56%). Of the 41 RCTs that measured effects on disease-specific clinical outcomes, significant improvements between groups were reported in 16 studies (39%).
CONCLUSIONS
There is potential for mHealth tools to better facilitate adherence to chronic disease management, but the evidence supporting its current effectiveness is mixed. Further research should focus on understanding and improving how mHealth tools can overcome specific barriers to adherence.
Topics: Cardiovascular Diseases; Chronic Disease; Diabetes Mellitus; Disease Management; Humans; Lung Diseases; Patient Compliance; Randomized Controlled Trials as Topic; Telemedicine; Text Messaging; Treatment Outcome
PubMed: 25803266
DOI: 10.2196/jmir.3951 -
The Cochrane Database of Systematic... Jun 2011Schizophrenia can be a severe and chronic illness characterised by lack of insight and poor compliance with treatment. Psychoeducational approaches have been developed... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Schizophrenia can be a severe and chronic illness characterised by lack of insight and poor compliance with treatment. Psychoeducational approaches have been developed to increase patients' knowledge of, and insight into, their illness and its treatment. It is supposed that this increased knowledge and insight will enable people with schizophrenia to cope in a more effective way with their illness, thereby improving prognosis.
OBJECTIVES
To assess the effects of psychoeducational interventions compared with standard levels of knowledge provision.
SEARCH STRATEGY
We searched the Cochrane Schizophrenia Group Trials Register (February 2010).
SELECTION CRITERIA
All relevant randomised controlled trials focusing on psychoeducation for schizophrenia and/or related serious mental illnesses involving individuals or groups. We excluded quasi-randomised trials.
DATA COLLECTION AND ANALYSIS
At least two review authors extracted data independently from included papers. We contacted authors of trials for additional and missing data. We calculated risk ratios (RR) and 95% confidence intervals (CI) of homogeneous dichotomous data. We used a fixed-effects model for heterogeneous dichotomous data. Where possible we also calculated the numbers needed to treat (NNT), as well as weighted means for continuous data.
MAIN RESULTS
This review includes a total of 5142 participants (mostly inpatients) from 44 trials conducted between 1988 and 2009 (median study duration ˜ 12 weeks, risk of bias - moderate). We found that incidences of non-compliance were lower in the psychoeducation group in the short term (n = 1400, RR 0.52 CI 0.40 to 0.67, NNT 11 CI 9 to 16). This finding holds for the medium and long term. Relapse appeared to be lower in psychoeducation group (n = 1214, RR 0.70 CI 0.61 to 0.81, NNT 9 CI 7 to 14) and this also applied to readmission (n = 206, RR 0.71 CI 0.56 to 0.89, NNT 5 CI 4 to 13). Scale-derived data also suggested that psychoeducation promotes better social and global functioning. In the medium term, treating four people with schizophrenia with psychoeducation instead of standard care resulted in one additional person showing a clinical improvement. Evidence suggests that participants receiving psychoeducation are more likely to be satisfied with mental health services (n = 236, RR 0.24 CI 0.12 to 0.50, NNT 5 CI 5 to 8) and have improved quality of life.
AUTHORS' CONCLUSIONS
Psychoeducation does seem to reduce relapse, readmission and encourage medication compliance, as well as reduce the length of hospital stay in these hospital-based studies of limited quality. The true size of effect is likely to be less than demonstrated in this review - but, nevertheless, some sort of psychoeducation could be clinically effective and potentially cost beneficial. It is not difficult to justify better, more applicable, research in this area aimed at fully investigating the effects of this promising approach.
Topics: Awareness; Family; Female; Humans; Male; Patient Compliance; Patient Education as Topic; Psychotic Disorders; Randomized Controlled Trials as Topic; Schizophrenia; Schizophrenic Psychology
PubMed: 21678337
DOI: 10.1002/14651858.CD002831.pub2 -
The Journal of Allergy and Clinical... 2017Clinical studies to prevent the development of food allergy have recently helped reshape public policy recommendations on the early introduction of allergenic foods.... (Review)
Review
Clinical studies to prevent the development of food allergy have recently helped reshape public policy recommendations on the early introduction of allergenic foods. These trials are also prompting new research, and it is therefore important to address the unique design and analysis challenges of prevention trials. We highlight statistical concepts and give recommendations that clinical researchers may wish to adopt when designing future study protocols and analysis plans for prevention studies. Topics include selecting a study sample, addressing internal and external validity, improving statistical power, choosing alpha and beta, analysis innovations to address dilution effects, and analysis methods to deal with poor compliance, dropout, and missing data.
Topics: Allergens; Clinical Trials as Topic; Food Hypersensitivity; Humans; Patient Compliance; Public Policy; Research Design
PubMed: 28283152
DOI: 10.1016/j.jaip.2016.12.007 -
Drug Delivery and Translational Research Dec 2017Sexual intercourse (vaginal and anal) is the predominant mode of human immunodeficiency virus (HIV) transmission. Topical microbicides used in an on-demand format (i.e.,... (Review)
Review
Sexual intercourse (vaginal and anal) is the predominant mode of human immunodeficiency virus (HIV) transmission. Topical microbicides used in an on-demand format (i.e., immediately before or after sex) can be part of an effective tool kit utilized to prevent sexual transmission of HIV. The effectiveness of prevention products is positively correlated with adherence, which is likely to depend on user acceptability of the product. The development of an efficacious and acceptable product is therefore paramount for the success of an on-demand product. Acceptability of on-demand products (e.g., gels, films, and tablets) and their attributes is influenced by a multitude of user-specific factors that span behavioral, lifestyle, socio-economic, and cultural aspects. In addition, physicochemical properties of the drug, anatomical and physiological aspects of anorectal and vaginal compartments, issues relating to large-scale production, and cost can impact product development. These factors together with user preferences determine the design space of an effective, acceptable, and feasible on-demand product. In this review, we summarize the interacting factors that together determine product choice and its target product profile.
Topics: Administration, Rectal; Administration, Topical; Anti-Infective Agents; Clinical Trials as Topic; Female; HIV Infections; Humans; Male; Patient Compliance; Patient Preference; Sexual Behavior; Vaginal Creams, Foams, and Jellies
PubMed: 28589452
DOI: 10.1007/s13346-017-0385-4 -
Cancer Treatment Reviews Jun 2018Adolescents and Young Adults (AYA) with cancer are young people developing serious illness when at the interface between the responsibilities of paediatric and adult... (Review)
Review
Adolescents and Young Adults (AYA) with cancer are young people developing serious illness when at the interface between the responsibilities of paediatric and adult cancer services. Personally, they are in a period of transition both biologically and in major social roles (Sawyer et al., 2018) [1]. For these and other reasons they present a unique set of clinical challenges in their management. Over the last 20 years the requirement for specific services to address their needs has been identified and this has become a growing field of research. Despite this survival rates still lag behind those of children and older adults with cancer (Gatta et al., 2009) [2]. Why do AYA patients have worse outcomes? The observation is that the reason is multifactorial with path to diagnosis, unique cancer biology, uncertainty of treatment protocol, compliance issues and poor recruitment to clinical trials all playing a part. In this review we will discuss the unique challenges faced by healthcare professionals when managing AYA patients who are commonly and accurately described as being in an 'interface' position.
Topics: Adolescent; Clinical Trials as Topic; Fertility; Humans; Neoplasms; Palliative Care; Patient Compliance; Patient Selection; Young Adult
PubMed: 29753962
DOI: 10.1016/j.ctrv.2018.05.001 -
Respiratory Medicine Sep 2003Education about asthma and self-management of asthma are now key recommendations of asthma management guidelines. A Cochrane systematic review of 12 RCTs found that...
Education about asthma and self-management of asthma are now key recommendations of asthma management guidelines. A Cochrane systematic review of 12 RCTs found that limited education programmes that offer information about asthma but not self-management skills did not reduce hospitalisation rates or visits to the doctor for asthma. The positive outcomes from limited asthma education were a reduction in symptoms. Asthma self-management education which consists of information, self-monitoring, regular medical review, and a written action plan is effective and leads to a reduction in hospitalisation and ER visits for asthma, unscheduled doctors visits, days lost from work, episodes of nocturnal asthma, indirect costs and an improvement in quality of life. The effects were large enough to be of both clinical and statistical significance. While a structured asthma self-management programme is effective in a hospital setting, attempts to deliver these programmes in primary care have met with varying success.
Topics: Asthma; Clinical Trials as Topic; Humans; Patient Compliance; Patient Education as Topic; Primary Health Care; Self Care; Teaching Materials
PubMed: 14509558
DOI: 10.1016/s0954-6111(03)00134-3