Assessing Real-World Data From Electronic Health Records for Health Technology Assessment: The SUITABILITY Checklist: A Good Practices Report of an ISPOR Task Force.

This ISPOR Good Practices report provides a framework for assessing the suitability of electronic health records data for use in health technology assessments (HTAs). Although electronic health record (EHR) data can fill evidence gaps and improve decisions, several important limitations can affect its validity and relevance. The ISPOR framework includes 2 components: data delineation and data fitness for purpose. Data delineation provides a complete understanding of the data and an assessment of its trustworthiness by describing (1) data characteristics; (2) data provenance; and (3) data governance. Fitness for purpose comprises (1) data reliability items, ie, how accurate and complete the estimates are for answering the question at hand and (2) data relevance items, which assess how well the data are suited to answer the particular question from a decision-making perspective. The report includes a checklist specific to EHR data reporting: the ISPOR SUITABILITY Checklist. It also provides recommendations for HTA agencies and policy makers to improve the use of EHR-derived data over time. The report concludes with a discussion of limitations and future directions in the field, including the potential impact from the substantial and rapid advances in the diffusion and capabilities of large language models and generative artificial intelligence. The report's immediate audiences are HTA evidence developers and users. We anticipate that it will also be useful to other stakeholders, particularly regulators and manufacturers, in the future.

A National Hepatitis C Elimination Program in the United States: A Historic Opportunity.

This Viewpoint introduces a proposed 5-year program from the Biden-Harris administration that would use direct-acting antivirals to eliminate hepatitis C in the United States.

Engaging patients and stakeholders in research proposal review: the patient-centered outcomes research institute.

The inaugural round of merit review for the Patient-Centered Outcomes Research Institute (PCORI) in November 2012 included patients and other stakeholders, as well as scientists. This article examines relationships among scores of the 3 reviewer types, changes in scoring after in-person discussion, and the effect of inclusion of patient and stakeholder reviewers on the review process. In the first phase, 363 scientists scored 480 applications. In the second phase, 59 scientists, 21 patients, and 31 stakeholders provided a "prediscussion" score and a final "postdiscussion" score after an in-person meeting for applications. Bland-Altman plots were used to characterize levels of agreement among and within reviewer types before and after discussion. Before discussion, there was little agreement among average scores given by the 4 lead scientific reviewers and patient and stakeholder reviewers. After discussion, the 4 primary reviewers showed mild convergence in their scores, and the 21-member panel came to a much stronger agreement. Of the 25 awards with the best (and lowest) scores after phase 2, only 13 had ranked in the top 25 after the phase 1 review by scientists. Five percent of the 480 proposals submitted were funded. The authors conclude that patient and stakeholder reviewers brought different perspectives to the review process but that in-person discussion led to closer agreement among reviewer types. It is not yet known whether these conclusions are generalizable to future rounds of peer review. Future work would benefit from additional data collection for evaluation purposes and from long-term evaluation of the effect on the funded research.

Review of the economic and quality-of-life burden of cervical human papillomavirus disease.

OBJECTIVE: The purpose of this study was to conduct a systematic literature review on the economic burden and health-related quality-of-life impact of cervical human papillomavirus disease. STUDY DESIGN: A systematic review of cost-of-illness studies and health-related quality-of-life studies was conducted. PubMed, Embase, and PsycINFO databases were searched with the use of predefined terms. RESULTS: Nine economic and 24 quality-of-life studies were identified. The annual health care costs of human papillomavirus-related conditions in the United States range from 2.25-4.6 billion dollars (2005 US dollars). The burden of human papillomavirus is second only to human immunodeficiency virus among sexually transmitted diseases. Health-related quality-of-life areas that are impacted substantially by human papillomavirus include emotional, social, and sexual functioning. CONCLUSION: The economic and quality-of-life burden of cervical human papillomavirus disease is significant and highlights the need for treatment and prevention options for this condition.

Rates and probabilities in economic modelling: transformation, translation and appropriate application.

Economic modelling is increasingly being used to evaluate the cost effectiveness of health technologies. One of the requirements for good practice in modelling is appropriate application of rates and probabilities. In spite of previous descriptions of appropriate use of rates and probabilities, confusions persist beyond a simple understanding of their definitions. The objective of this article is to provide a concise guide to understanding the issues surrounding the use of rates and probabilities reported in the literature in economic models, and an understanding of when and how to transform them appropriately. The article begins by defining rates and probabilities and shows the essential difference between the two measures. Appropriate conversions between rates and probabilities are discussed, and simple examples are provided to illustrate the techniques and pitfalls. How the transformed rates and probabilities may be used in economic models is then described and some recommendations are suggested.

The cost effectiveness of bisphosphonates for the prevention and treatment of osteoporosis: a structured review of the literature.

Osteoporotic fragility fractures constitute a significant public health concern. The lifetime risk of any osteoporotic fracture is very high (40-50% in women and 13-22% in men). Fractures are associated with significant mortality and morbidity and represent a substantial economic burden to society. Bisphosphonates (alendronate, etidronate, risedronate and ibandronate) are indicated for the treatment and prevention of osteoporosis but are costly compared with other treatments, such as vitamin D and calcium. Our search identified 23 studies evaluating the cost effectiveness of bisphosphonate therapy for the treatment and prevention of fragility fractures; these studies were from five geographical areas and employed a variety of comparators and assumptions. We identified 11 studies investigating bisphosphonates in women with low bone mineral density (BMD) [T-score >2.5 standard deviations {SDs} below normal {mean} peak values for young adults] and previous fractures, five studies investigating bisphosphonates in women with low BMD and no previous fracture, one study of bisphosphonates in women with osteopenia, five studies involving screening and two studies of bisphosphonates in special populations (women initiating corticosteroid treatment and men). In women with low BMD and previous fractures, bisphosphonate therapy was most cost effective in populations aged > or =70 years and was unlikely to be cost effective in populations aged < or =50 years. There was uncertainty concerning the cost effectiveness of bisphosphonates in such populations aged 60-69 years. In women with low BMD without previous fractures, treatment with alendronate or risedronate appeared to be cost effective across countries (UK, US, Denmark), but there was some uncertainty about the cost effectiveness of etidronate in patients in the highest age groups. Identifying risk factors for fractures through means such as spine radiographs to detect vertebral deformities improves the cost effectiveness of treatment. In women with osteopenia, alendronate therapy may be cost effective in women with a T-score of -2.4SD in the US. Screening for low BMD and treatment with alendronate or etidronate appears to be cost effective in postmenopausal women in general and in women with rheumatoid arthritis initiating corticosteroid therapy. Alendronate therapy without screening was also shown to be potentially cost effective in certain at-risk male populations, as well as in women initiating corticosteroid therapy after the age of 40 years. Decision makers in the US, UK and Sweden should consider funding the use of bisphosphonates for the prevention and treatment of osteoporosis in women aged >70 years, particularly if they have other risk factors for fracture. Further studies are required to make more definitive conclusions in other countries and patient populations. Screening strategies for low BMD followed by bisphosphonate treatment should also be considered in the general female population aged >65 years in the UK and US and in patients with rheumatoid arthritis initiating corticosteroid therapy.

Economic outcomes associated with atypical antipsychotics in bipolar disorder: a systematic review.

OBJECTIVE: Bipolar disorder is a serious condition that is costly to the health care system. Atypical antipsychotics are more expensive than conventional treatments. From a policy-making perspective, the additional cost must be justified by improved outcomes. The objective of this study was to conduct a systematic review to determine the relative costs and cost-effectiveness associated with atypical antipsychotics in bipolar disorder. DATA SOURCES: We conducted a systematic review of the literature in PubMed and EMBASE from January 1985 through October 2005, including published studies and conference proceedings. Databases were searched using predefined terms. STUDY SELECTION: Studies were included if they were claims data analyses, trial-based economic evaluations, or cost-effectiveness analyses using models. Data were extracted using predefined tables. DATA SYNTHESIS: Fourteen studies were identified. Seven were medical claims database analyses, 4 were trial-based economic evaluations, and 3 were cost-effectiveness models. Eight of these studies were conference proceedings. The studies did not provide sufficient information to determine any ranking of interventions in terms of least to most costly in overall resource consumption or in terms of their relative cost-effectiveness. Where comparable, results tended to be inconsistent. CONCLUSION: There is a scarcity of economic studies in this field. A reference case outlining how to address the complex interplay between effectiveness, safety, adherence, and quality of life and their impact on resource use and costs is needed to contribute to improving the treatment of patients with bipolar disorder while making the best use of scarce health resources.

Economics of atypical antipsychotics in bipolar disorder: a review of the literature.

Economic evaluations are increasingly being used by policy makers to evaluate the relative costs and benefits of healthcare interventions. These analyses provide economic and clinical evidence to decision makers seeking to make recommendations on treatment alternatives for patients. This article describes the economic evidence on the atypical antipsychotics currently approved for the treatment of bipolar disorder. This area remains under-researched. A literature search identified only six relevant studies of atypical antipsychotics in bipolar disorder: two retrospective database analyses, three economic analyses alongside clinical trials and one cost-effectiveness analysis. Based on the limited available studies, there appears to be no significant difference in healthcare resource use between olanzapine, quetiapine, risperidone and valproate semisodium (divalproex sodium; an antiepileptic drug and a standard treatment for mania associated with bipolar disorder). While a cost-effectiveness study for the UK found haloperidol (a conventional antipsychotic) to be more cost effective than atypical antipsychotics, these results must be considered with caution because of the non-inclusion of adverse effects in the model. No economic data are available for aripiprazole, clozapine or ziprasidone in bipolar disorder. Until more economic evidence becomes available, the economic implications of atypical antipsychotic treatment in patients with bipolar disorder are unlikely to significantly impact on prescribing and treatment patterns. Future economic studies evaluating atypical antipsychotics in bipolar disorder should address the issue of long-term costs and effectiveness to reflect the chronic nature of the disease, the variety of health states that patients may experience and the range of treatments they may receive. A better understanding of the complex interplay between effectiveness, safety, quality of life, adherence and resource use should ultimately contribute to improving the treatment of bipolar disorder.

Setting priorities for research.

Setting priorities for research should be conducted in order to make the most efficient use of scarce resources. Yet the uptake in practice of such methods by researchers and commissioners of research alike has been slow, in part because the methodologies available to do so have not been widely disseminated. This paper argues that an appropriate priority-setting methodology should meet the objectives of the health system, that is to provide the most health benefits to the population that it serves within the budget constraint and while respecting equity considerations. A condition for these criteria to be met is to construct and operationalise an appropriate definition of the value of research. Five different ways that have been used in practice to value research and set priorities were reviewed. Shortcomings in the ways research is valued make it unlikely that the application of subjective methods, burden of disease methods, and clinical variations and payback methods meet the objectives of the health system. Using the fifth method, value of information, priority-setting can meet the objectives of the health system because it expresses the value of research using the same overall cost-effectiveness framework that is employed for decisions on service provision. However, this method still requires further work to evaluate how research outcomes can then be communicated to clinical practitioners and how practitioners can be encouraged to implement them.

Patient-powered research networks aim to improve patient care and health research.

The era of big data, loosely defined as the development and analysis of large or complex data sets, brings new opportunities to empower patients and their families to generate, collect, and use their health information for both clinical and research purposes. In 2013 the Patient-Centered Outcomes Research Institute launched a large national research network, PCORnet, that includes both clinical and patient-powered research networks. This article describes these networks, their potential uses, and the challenges they face. The networks are engaging patients, family members, and caregivers in four key ways: contributing data securely, with privacy protected; including diverse and representative groups of patients in research; prioritizing research questions, participating in research, and disseminating results; and participating in the leadership and governance of patient-powered research networks. If technical, regulatory, and organizational challenges can be overcome, PCORnet will allow research to be conducted more efficiently and cost-effectively and results to be disseminated quickly back to patients, clinicians, and delivery systems to improve patient health.

Launching PCORnet, a national patient-centered clinical research network.

The Patient-Centered Outcomes Research Institute (PCORI) has launched PCORnet, a major initiative to support an effective, sustainable national research infrastructure that will advance the use of electronic health data in comparative effectiveness research (CER) and other types of research. In December 2013, PCORI's board of governors funded 11 clinical data research networks (CDRNs) and 18 patient-powered research networks (PPRNs) for a period of 18 months. CDRNs are based on the electronic health records and other electronic sources of very large populations receiving healthcare within integrated or networked delivery systems. PPRNs are built primarily by communities of motivated patients, forming partnerships with researchers. These patients intend to participate in clinical research, by generating questions, sharing data, volunteering for interventional trials, and interpreting and disseminating results. Rapidly building a new national resource to facilitate a large-scale, patient-centered CER is associated with a number of technical, regulatory, and organizational challenges, which are described here.

A systematic review of adherence, treatment satisfaction and costs, in fixed-dose combination regimens in type 2 diabetes.

OBJECTIVE: Oral antidiabetics have comparable safety and efficacy when used as fixed-dose combination therapies (FDCT) or loose-pill combination therapies (LPCT) for patients with T2DM. To evaluate alternative outcomes to safety and efficacy with FDCT, a systematic review of literature was conducted. METHODS: Searches of Medline/Embase databases from 1998 to 2009 used predefined terms: 'fixed-dose combination', 'loose-dose combination' and 'diabetes'. Abstracts were reviewed from ISPOR, ADA, and EASD meetings (1998-2009). T2DM studies reporting adherence, patient-reported outcomes, costs, resource use or cost effectiveness were included. RESULTS: Seventeen studies met the search criteria. Seven studies reported adherence. Adherence was 10-13% higher for FDCT than LPCT in patients starting combination therapy. Adherence decreased 1.5% and 10.0% when switching from monotherapy to combination therapy for FDCT and LPCT respectively (p < 0.001). Switching to FDCT increased adherence 3.5%-12.4%, while remaining on LPCT changed adherence -1.5% to 5.0% (p < 0.005). For patients newly initiating OAD medication, one study found no adherence advantage for FDCT compared with monotherapy or LPCT. Five RCTs reported treatment satisfaction. Four publications reported patients preferred FDCT using the Diabetes Treatment Satisfaction Questionnaire (DTSQ). One publication reported improved satisfaction for one DTSQ subscale. Five abstracts reported economic outcomes. Two abstracts determined patients on FDCT used fewer healthcare resources and had decreased direct monthly healthcare costs versus LPCT. Two cost-effectiveness analyses determined clinical benefits from clinical trials translate into cost savings and increased life expectancy. One budget impact model reported minimal budget impact. LIMITATIONS: (1) There was limited published literature identified in this review. (2) FDCT are oral medications; these findings may only be relevant to those individuals taking an oral antidiabetic therapy. (3) Publication and reporting biases may exist. CONCLUSIONS: The published literature suggested that T2DM patients treated with FDCT may have better adherence, improved satisfaction, and lower direct medical costs, compared to those treated with LPCT.