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Damage to the central nervous system (CNS) in adulthood, may lead to cognitive impairments. In Germany, occupational therapy is most often prescribed for neurological diagnoses, including stroke and traumatic brain injury (351 and 343 cases per 100,000, respectively in 2018). For cognitive impairments, the primarily prescribed remedies are sensorimotor-perceptive, motor-functional and neuropsychologically oriented treatment or training of cognitive performance. Here we report the results of a health technology assessment (HTA) report on the clinical efficacy of occupational therapy for patients with cognitive impairments.
To assess clinical efficacy, a systematic overview was conducted based on published systematic reviews and HTA reports from the last ten years summarizing randomized controlled trials (RCTs) retrieved from four bibliographic databases. The target population included adult patients with cognitive impairments caused by diseases of the CNS, excluding moderate to severe dementia. The intervention studied is occupational therapy compared to no occupational therapy. Outcomes were cognitive abilities, independence, self-determination, health-related quality of life (QoL), and participation in activities of daily living (ADL).
Five systematic reviews comprising 1,316 patients were included. There is evidence for a small statistically significant positive effect on “general cognitive function” (10 RCTs, n=470) and on ADL (4 RCTs, n= 405). A non-quantified positive effect was reported on behavior control (1 RCT, n=96), and conflicting evidence on QoL (2 RCTs, n=214). No effect was found for individual components of cognition (5 RCTs, n=202), self-efficacy (1 RCT, n=98) and social participation (2 RCTs, n=194). The level of the evidence was low for all endpoints due to the high risk of bias and small sample sizes.
Based on this systematic overview, it cannot be demonstrated but also not ruled out that occupational therapy for cognitive impairment is an effective therapy for adults with cognitive impairments. The evidence is very uncertain due to small effects and high risk of bias, low statistical power, and heterogeneity of interventions and study populations.
In order to facilitate patient information, patient involvement, and to support patient-centered care, healthcare organizations are increasingly offering access to patient data that are stored in the institution-specific electronic health record (EHR). Patients can access these data, read, and print them, or download and integrate them into any type of patient-held record. This EHR access is typically web-based and called “patient portal” allowing the independent access via the Internet from everywhere. A patient portal may also offer additional features such as prescription requests, appointment booking, messaging, personal health-related reminders, individual therapeutic recommendations, personal diaries, and social networking with other patients. In a Cochrane review, we assessed the effects of providing access to EHR for adult patients on patient empowerment and health-related outcomes compared to usual care.
According to the methods of evidence-based medicine, we developed a protocol for a Cochrane review, which is published in the Cochrane database.
We identified ten randomized controlled trials (RCTs) including 6,668 randomized participants. Seven RCTs took place in the USA, two in Canada, and one in Japan. Additional functionalities of interventions and disease conditions were heterogeneous. Three studies (n = 601) reported on patient empowerment. The risk differences reported were neither statistically significant nor clinically relevant. Eight studies (n = 2,070) reported on nine different risk factors (blood pressure, blood glucose, poor asthma control, 10-year Framingham risk score, cholesterol, body mass index, composite score of eight variables, intraocular pressure, composite score of three variables). The results were heterogeneous. Mostly there were no statistically significant risk differences between study groups.
Overall, there is no evidence for a clear positive effect of patient portals on patient empowerment and health related outcomes (mainly risk factors). However, we identified only a small number of studies. The usage of portals was often low and several studies were older.
Objectives: Treatment switching refers to the situation in a randomized controlled trial where patients switch from their randomly assigned treatment onto an alternative. Often, switching is from the control group onto the experimental treatment. In this instance, a standard intention-to-treat analysis does not identify the true comparative effectiveness of the treatments under investigation. We aim to describe statistical methods for adjusting for treatment switching in a comprehensible way for nonstatisticians, and to summarize views on these methods expressed by stakeholders at the 2014 Adelaide International Workshop on Treatment Switching in Clinical Trials.
Methods: We describe three statistical methods used to adjust for treatment switching: marginal structural models, two-stage adjustment, and rank preserving structural failure time models. We draw upon discussion heard at the Adelaide International Workshop to explore the views of stakeholders on the acceptability of these methods.
Results: Stakeholders noted that adjustment methods are based on assumptions, the validity of which may often be questionable. There was disagreement on the acceptability of adjustment methods, but consensus that when these are used, they should be justified rigorously. The utility of adjustment methods depends upon the decision being made and the processes used by the decision-maker.
Conclusions: Treatment switching makes estimating the true comparative effect of a new treatment challenging. However, many decision-makers have reservations with adjustment methods. These, and how they affect the utility of adjustment methods, require further exploration. Further technical work is required to develop adjustment methods to meet real world needs, to enhance their acceptability to decision-makers.
Objectives: External experts can be consulted at different stages of an HTA. When using vague information sources, it is particularly important to plan, analyze, and report the information processing in a standardized and transparent way. Our objective was to search and analyze recommendations regarding where and how to include expert data in HTA.
Methods: We performed a systematic database search and screened the Internet pages of seventy-seven HTA organizations for guidelines, recommendations, and methods papers that address the inclusion of experts in HTA. Relevant documents were downloaded, and information was extracted in a standard form. Results were merged in tables and narrative evidence synthesis.
Results: From twenty-two HTA organizations, we included forty-two documents that consider the use of expert opinion in HTA. Nearly all documents mention experts in the step of preparation of the evidence report. Six documents address their role for priority setting of topics, fifteen for scoping, twelve for the appraisal of evidence and results, another twelve documents mention experts when considering the dissemination of HTA results.
During the assessment step, experts are most often asked to amend the literature search or to provide expertise for special data analyses. Another issue for external experts is to appraise the HTA results and refer them back to a clinical and social context. Little is reported on methods of expert elicitation when their input substitutes study data.
Conclusions: Despite existing recommendations on the use of expert opinion in HTA, common standards for elicitation are scarce in HTA guidelines.
Objectives: In recent years, there has been growth in the use of health technology assessment (HTA) for making decisions about the reimbursement, coverage, or guidance on the use of health technologies. Given this greater emphasis on the use of HTA, it is important to develop standards of good practice and to benchmark the various HTA organizations against these standards.
Methods: This study discusses the conceptual and methodological challenges associated with benchmarking HTA organizations and proposes a series of audit questions based on a previously published set of principles of good practice.
Results and Conclusions: It is concluded that a benchmarking exercise would be feasible and useful, although the question of who should do the benchmarking requires further discussion. Key issues for further research are the alternative methods for weighting the various principles and for generating an overall score, or summary statement of adherence to the principles. Any weighting system, if developed, would need to be explored in different jurisdictions to assess the extent to which the relative importance of the principles is perceived to vary. Finally, the development and precise wording of the audit questions requires further study, with a view to making the questions as unambiguous as possible, and the reproducibility of the assessments as high as possible.
Thank you for the opportunity to respond to the letter by Gibson and Little. The authors raise several points that require a response. First, they claim that we inaccurately characterized the DERP/Washington State Medicaid agency, and did not appreciate that the DERP and the Washington State Medicaid agency have different missions. On the contrary, however, we fully recognized that DERP's mission is to conduct systematic reviews and not to make recommendations, whereas Washington State uses the reviews in making recommendations for their Medicaid enrollees. We specifically noted in the study that “Washington Medicaid is one of fourteen participants in the DERP. DERP researchers conduct health technology assessments for drug classes. Participants in the DERP, such as the Washington Medicaid program, retain local authority for interpreting DERP reports and for decision making regarding which drugs to pay for.” We chose to analyze DERP/Washington State as a single entity for our exercise because we were interested in analyzing the link between the HTA conducted and the decisions that follow them.
Objectives: The aim of this study was to compare the predictive value, clinical effectiveness, and cost-effectiveness of high-sensitivity C-reactive protein (hs-CRP)-screening in addition to traditional risk factor screening in apparently healthy persons as a means of preventing coronary artery disease.
Methods and Results: The systematic review was performed according to internationally recognized methods. Seven studies on risk prediction, one clinical decision-analytic modeling study, and three decision-analytic cost-effectiveness studies were included. The adjusted relative risk of high hs-CRP-level ranged from 0.7 to 2.47 (p < .05 in four of seven studies). Adding hs-CRP to the prediction models increased the areas under the curve by 0.00 to 0.027. Based on the clinical decision analysis, both individuals with elevated hs-CRP-levels and those with hyperlipidemia have a similar gain in life expectancy following statin therapy. One high-quality economic modeling study suggests favorable incremental cost-effectiveness ratios for persons with elevated hs-CRP and higher risk. However, many model parameters were based on limited evidence.
Conclusions: Adding hs-CRP to traditional risk factors improves risk prediction, but the clinical relevance and cost-effectiveness of this improvement remain unclear.
Previously, our group—the International Working Group for HTA Advancement—proposed a set of fifteen Key Principles that could be applied to health technology assessment (HTA) programs in different jurisdictions and across a range of organizations and perspectives. In this commentary, we investigate the extent to which these principles are supported and used by fourteen selected HTA organizations worldwide. We find that some principles are broadly supported: examples include being explicit about HTA goals and scope; considering a wide range of evidence and outcomes; and being unbiased and transparent. Other principles receive less widespread support: examples are addressing issues of generalizability and transferability; being transparent on the link between HTA findings and decision-making processes; considering a full societal perspective; and monitoring the implementation of HTA findings. The analysis also suggests a lack of consensus in the field about some principles—for example, considering a societal perspective. Our study highlights differences in the uptake of key principles for HTA and indicates considerable room for improvement for HTA organizations to adopt principles identified to reflect good HTA practices. Most HTA organizations espouse certain general concepts of good practice—for example, assessments should be unbiased and transparent. However, principles that require more intensive follow-up—for example, monitoring the implementation of HTA findings—have received little support and execution.
Objectives: The objectives of this study were (i) to develop a systematic framework for describing and comparing different features of health technology assessment (HTA) agencies, (ii) to identify and describe similarities and differences between the agencies, and (iii) to draw conclusions both for producers and users of HTA in research, policy, and practice.
Methods: We performed a systematic literature search, added information from HTA agencies, and developed a conceptual framework comprising eight main domains: organization, scope, processes, methods, dissemination, decision, implementation, and impact. We grouped relevant items of these domains in an evidence table and chose five HTA agencies to test our framework: DAHTA@DIMDI, HAS, IQWiG, NICE, and SBU. Item and domain similarity was assessed using the percentage of identical characteristics in pairwise comparisons across agencies. Results were interpreted across agencies by demonstrating similarities and differences.
Results: Based on 306 included documents, we identified 90 characteristics of eight main domains appropriate for our framework. After applying the framework to the five agencies, we were able to show 40 percent similarities in “dissemination,” 38 percent in “scope,” 35 percent in “organization,” 29 percent in “methods,” 26 percent in “processes,” 23 percent in “impact,” 19 percent in “decision,” and 17 percent in “implementation.”
Conclusion: We found considerably more differences than similarities of HTA features across agencies and countries. Our framework and comparison provides insights and clarification into the need for harmonization. Our findings could serve as descriptive database facilitating communication between producers and users.
Objectives: Progression-free survival (PFS) has not been validated as a surrogate endpoint for overall survival (OS) for anthracycline (A) and taxane-based (T) chemotherapy in advanced breast cancer (ABC). Using trial-level, meta-analytic approaches, we evaluated PFS as a surrogate endpoint.
Methods: A literature review identified randomized, controlled A and T trials for ABC. Progression-based endpoints were classified by prospective definitions. Treatment effects were derived as hazard ratios for PFS (HRPFS) and OS (HROS). Kappa statistic assessed overall agreement. A fixed-effects regression model was used to predict HROS from observed HRPFS. Cross-validation was performed. Sensitivity and subgroup analyses were performed for PFS definition, year of last patient recruitment, line of treatment, and constant rate assumption.
Results: Sixteen A and fifteen T trials met inclusion criteria, producing seventeen A (n = 4,323) and seventeen T (n = 5,893) trial-arm pairs. Agreement (kappa statistic) between the direction of HROS and HRPFS was 0.71 for A (p = .0029) and 0.75 for T (p = .0028). While HRPFS was a statistically significant predictor of HROS for both A (p = .0019) and T (p = .012), the explained variances were 0.49 (A) and 0.35 (T). In cross-validation, 97 percent of the 95 percent prediction intervals crossed the equivalence line, and the direction of predicted HROS agreed with observed HROS in 82 percent (A) and 76 percent (T). Results were robust in sensitivity and subgroup analyses.
Conclusions: This meta-analysis suggests that the trial-level treatment effect on PFS is significantly associated with the trial-level treatment effect on OS. However, prediction of OS based on PFS is surrounded with uncertainty.
Health technology assessment (HTA) is a dynamic, rapidly evolving process, embracing different types of assessments that inform real-world decisions about the value (i.e., benefits, risks, and costs) of new and existing technologies. Historically, most HTA agencies have focused on producing high quality assessment reports that can be used by a range of decision makers. However, increasingly organizations are undertaking or commissioning HTAs to inform a particular resource allocation decision, such as listing a drug on a national or local formulary, defining the range of coverage under insurance plans, or issuing mandatory guidance on the use of health technologies in a particular healthcare system. A set of fifteen principles that can be used in assessing existing or establishing new HTA activities is proposed, providing examples from existing HTA programs. The principal focus is on those HTA activities that are linked to, or include, a particular resource allocation decision. In these HTAs, the consideration of both costs and benefits, in an economic evaluation, is critical. It is also important to consider the link between the HTA and the decision that will follow. The principles are organized into four sections: (i) “Structure” of HTA programs; (ii) “Methods” of HTA; (iii) “Processes for Conduct” of HTA; and (iv) “Use of HTAs in Decision Making.”
Our goal in formulating the proposed principles was to stimulate discussion of HTA program goals and procedures to enhance the rigor, validity, and usefulness of HTA. The thoughtful and constructive comments by David Banta, David Hailey, and Duncan Neuhauser are an excellent beginning to what we hope will be an extended and interactive process. We appreciate the opportunity to respond to some of their comments, suggestions, and critiques.
Objectives: The aim of this study was to summarize the current evidence for the cost-effectiveness of primarily human papillomavirus (HPV) -based cervical cancer screening in settings with already established Papanicolaou test (Pap) programs. Emphasis was placed on the German situation with annual Pap screening.
Methods: Medical, economic, and health technology assessment (HTA) databases were systematically searched for cost-effectiveness studies comparing HPV to Pap screening. Study data were extracted, standardized, and summarized in cost-effectiveness plots contrasting HPV strategies to Pap screening with 1-, 2-, 3-, and 5-years interval. For each Pap setting, the likelihood of cost-effective HPV screening was assessed depending on willingness-to-pay.
Results: We reviewed twelve decision-analytic cost-effectiveness models. Study results showed wide variation due to methodical heterogeneity. Data synthesis revealed that the cost-effectiveness of HPV screening depends on the interval of the established Pap screening strategy. In comparison with Pap screening every 2 years, only 25 percent of the HPV-based screening strategies were cost-effective. However, in comparison with Pap screening every 1, 3, or 5 years, 83 percent, 55 percent, and 92 percent of HPV screening strategies were cost-effective, respectively. Results for settings with annual Pap screening are based on models assuming 100 percent screening coverage.
Conclusions: The introduction of HPV-based screening programs is cost-effective if the screening interval of the established Pap program exceeds 2 years. In settings with biennial Pap screening, introduction of HPV-based screening is unlikely to be cost-effective. Results also suggest cost-effectiveness of HPV-based screening in settings with annual Pap screening; however, this finding should be confirmed under realistic screening adherence assumptions.
Objectives: The purpose of this health technology assessment (HTA), commissioned by the German Federal Ministry of Health and Social Security, was to systematically review the evidence for the effectiveness and cost-effectiveness of antiviral treatment (AVT) with interferon (INF) or peginterferon (PegIFN) in combination with ribavirin (RBV) in treatment-naïve patients with chronic hepatitis C (CHC) and to apply these data in the context of the German health-care system.
Methods: We performed a systematic literature search on effectiveness and cost-effectiveness of AVT and summarized results using meta-analysis and evidence tables. We applied the German Hepatitis C Model (GEHMO), a decision-analytic Markov model, to determine long-term clinical effectiveness, costs, and incremental cost-effectiveness ratios (ICER) of the examined treatment strategies. Model parameters were derived from German databases, published international randomized clinical trials (RCT), and a Cochrane Review.
Results: Overall, nine RCTs, two HTA reports, one Cochrane review, two meta-analyses, and seven economic evaluations met the inclusion criteria. These studies indicate that PegIFN+RBV achieved the highest sustained virological response rates (SVR) (54–61 percent), followed by IFN+RBV (38–54 percent) and IFN monotherapy (11–21 percent). Based on our meta-analysis, PegIFN+RBV reduced cases without SVR by 17 percent compared with INF+RBV. International cost-effectiveness studies indicate that INF+RBV is cost-effective when compared with INF monotherapy. For PegIFN+RBV, our decision analysis yielded an ICER of €9,800 per quality-adjusted life-year gained.
Conclusions: This HTA suggests that initial combination therapy prolongs life, improves quality of life, and is cost-effective in patients with CHC. Peginterferon plus ribavirin is the most effective and efficient treatment among the examined options. However, because not all chronic hepatitis C patients will develop progressive liver disease, a thorough assessment of the eligibility and appropriateness of treatment with combination therapy must be performed in each individual patient.
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