Information and decision support needs of parents considering amniocentesis: interviews with pregnant women and health professionals

Objective Our aim was to clarify and categorize information and decision support needs of pregnant women deciding about amniocentesis. Background Prenatal screening for Down’s syndrome (implemented in routine practice) generates a quantifiable risk of chromosome abnormality. To increase certainty, chromosomal material needs to be obtained through amniocentesis or other diagnostic test. Amniocentesis carries risks of pregnancy loss. Methods Semi‐structured interviews were conducted with health professionals and pregnant women who had considered amniocentesis. The data were qualitatively analysed using a two‐step thematic content analysis. Results A sample of 17 health professionals and 17 pregnant women were interviewed. Professionals demonstrated little consensus regarding the miscarriage rate, the potential consequences of amniocentesis testing and the uncertainty associated with the tests. Furthermore, methods employed to communicate risks varied between professionals. Pregnant women reported heightened stress and anxiety. Twelve out of 17 women described the decision as complex and difficult to make while five participants were satisfied with the information and support provided. Women would have liked more information about the risks involved, the results, the consequences of an amniocentesis and associated emotional difficulties. Women highlighted the need for personalized information, presented in multiple ways, while remaining simple and unbiased. Conclusions There is variation in the provision of information related to amniocentesis testing. The majority of pregnant women reported difficulties making a decision and identified dimensions of information and decision support where improvements were needed.     

Expect the unexpected: patients’ and families’ expectations and experiences of new clinical procedures

Background

Early stage clinical innovation often occurs ‘under the radar’ of governance systems for established procedures. Previously impossible or unavailable techniques still being developed involve additional uncertainty and unknown risks and benefits compared with standard procedures. Patient and family expectations, perceptions and experiences of these new procedures and their possible impacts on aspects of patient safety are under‐researched.

Objective

To explore patient and family expectations and experiences of undergoing new clinical procedures.

Setting

A large UK Hospital NHS Foundation Trust with a range of clinically innovative specialties.

Methods

We interviewed 15 patients who received new clinical procedures in a variety of medical and surgical specialties. Qualitative interviews were used to facilitate in‐depth exploration of patient and family views and experiences. Topics included patient and family access to and expectations of the procedure, informed decision making regarding acceptance of new procedures and post‐procedure experiences.

Results

Some patients sought out specific interventions, while others accepted new treatment options that clinicians proposed. Most participants reported that explanations about the procedure and risks were clear, and there were opportunities to ask questions prior to the procedure. Most participants also regarded their procedures as successful. However, post‐procedure information follow‐up was often reported as lacking and some outcomes were considerably problematic and raised patient safety issues.

Discussion

The importance of patients’ and family views in evaluating health care are increasingly recognized. We discuss the implications of our findings for informed decision making and post‐intervention follow‐up.     

Cost-Effectiveness and Budget Impact of Future Developments With Whole-Genome Sequencing for Patients With Lung Cancer

ObjectivesThis study aimed to investigate the cost-effectiveness, budget impact (BI), and impact of uncertainty of future developments concerning whole-genome sequencing (WGS) as a clinical diagnostic test compared with standard of care (SoC) in patients with locally advanced and metastatic non–small cell lung cancer.MethodsA total of 3 likely scenarios to take place within 5 years (according to experts) were simulated using a previously developed, peer reviewed, and published decision model. The scenarios concerned “WGS results used for treatment selection” (scenario 1), “WGS-based biomarker for immunotherapy” (scenario 2), and “off-label drug approval for WGS results” (scenario 3). Two diagnostic strategies of the original model, “SoC” and “WGS as a diagnostic test” (base model), were used to compare our scenarios with. Outcomes were reported for the base model, all scenarios separately, combined (combined unweighted), and weighted by likelihood (combined weighted). Cost-effectiveness, BI, and value of information analyses were performed for WGS compared with SoC.ResultsTotal costs and quality-adjusted life-years for SoC in metastatic non–small cell lung cancer were €149 698 and 1.235. Incremental outcomes of WGS were €1529/0.002(base model), ?€222/0.020(scenario 1), ?€2576/0.023(scenario 2), €388/0.024(scenario 3), ?€5041/0.060(combined unweighted), and ?€1715/0.029(combined weighted). The annual BI for adopting WGS for this population in The Netherlands ranged between €682 million (combined unweighted) and €714 million (base model). The consequences of uncertainty amounted to €3.4 million for all scenarios (combined weighted) and to €699 000 for the diagnostic yield of WGS alone (combined weighted).ConclusionsOur findings suggest that it is likely for WGS to become cost-effective within the near future if it identifies more patients with actionable targets and show the impact of uncertainty regarding its diagnostic yield. Modeling future scenarios can be useful to consider early adoption of WGS while timely anticipating on unforeseen developments before final conclusions are reached.     

Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

Background

Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them.

Objectives

To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology—specifically economic evaluation methods—in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges.

Methods

An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs.

Results

Three key topics were identified and prioritised for discussion—uncertainty, discounting, and health outcomes and value. The panel discussed that evidence challenges linked to increased uncertainty may be mitigated by collection of follow-on data, use of value of information analysis, and/or outcomes-based contracts. For discount rates, an international, multi-disciplinary forum should be established to consider the economic, social and ethical implications of the choice of rate. Finally, consideration of the feasibility of assessing the value of ATMPs beyond health gain may also be key for decision-making.

Conclusions

ATMPs face a challenge in demonstrating their value within current HTA frameworks. Consideration of current HTA principles and practices with regards to the specific characteristics of ATMPs and continued dialogue will be key to ensuring appropriate market access.

Classification code

I.

     

Previvors’ Uncertainty Management Strategies for Hereditary Breast and Ovarian Cancer

Individuals with a genetic predisposition to develop hereditary breast and ovarian cancer (HBOC), but who have not been diagnosed with cancer, are referred to as previvors. Although genetic testing may reduce previvors’ worries about whether or not they have a high genetic cancer risk, testing positive produces negative emotions and long-term uncertainty—thus requiring the management of uncertainty. Existing research indicates family, friends, and social support networks are limited in their assistance for previvors’ uncertainty management. Therefore, this study examined how health care providers may assist previvors in uncertainty management by asking: What strategies do BRCA-positive previvors enact with their health care providers to help manage their uncertainty about HBOC? Purposive sampling was employed to recruit participants via online social media. The final sample consisted of 34 BRCA-positive women. Interviews revealed four uncertainty management strategies—seeking health care providers as informational sources, seeking health care providers as partners for decision making, seeking health care providers for supportive communication, and seeking referrals from health care providers for social support networks. Findings indicate that health care providers who are knowledgeable about BRCA, provide information, answer questions, check understanding, and provide additional resources assist previvors in managing their uncertainties by distinguishing options and fostering meaning.     

Prioritizing Additional Data Collection to Reduce Decision Uncertainty in the HIV/AIDS Response in 6 US Cities: A Value of Information Analysis

ObjectivesThe ambitious goals of the US Ending the HIV Epidemic initiative will require a targeted, context-specific public health response. Model-based economic evaluation provides useful guidance for decision making while characterizing decision uncertainty. We aim to quantify the value of eliminating uncertainty about different parameters in selecting combination implementation strategies to reduce the public health burden of HIV/AIDS in 6 US cities and identify future data collection priorities.MethodsWe used a dynamic compartmental HIV transmission model developed for 6 US cities to evaluate the cost-effectiveness of a range of combination implementation strategies. Using a metamodeling approach with nonparametric and deep learning methods, we calculated the expected value of perfect information, representing the maximum value of further research to eliminate decision uncertainty, and the expected value of partial perfect information for key groups of parameters that would be collected together in practice.ResultsThe population expected value of perfect information ranged from $59 683 (Miami) to $54 108 679 (Los Angeles). The rank ordering of expected value of partial perfect information on key groups of parameters were largely consistent across cities and highest for parameters pertaining to HIV risk behaviors, probability of HIV transmission, health service engagement, HIV-related mortality, health utility weights, and healthcare costs. Los Angeles was an exception, where parameters on retention in pre-exposure prophylaxis ranked highest in contributing to decision uncertainty.ConclusionsFunding additional data collection on HIV/AIDS may be warranted in Baltimore, Los Angeles, and New York City. Value of information analysis should be embedded into decision-making processes on funding future research and public health intervention.     

Decision analysis for resource allocation in health care

This paper addresses the use of economic evaluation to inform resource allocation decisions within health care systems about which interventions to reimburse and whether additional research should be funded. A social decision-making view of economic evaluation, that is to maximize health gains subject to an exogenous budget constraint, is adopted. A brief overview of the components of an economic evaluation is presented. Particular attention is paid to how uncertainty is inherent to decisions about resource allocation, the consequences of that uncertainty and how it can be incorporated informatively into economic evaluation. A Bayesian approach to uncertainty is used as it meets the needs of social decision-making, allowing analysts to quantify the probability that an intervention is cost-effective given the available evidence and to quantify the expected value of further research. The discussion covers methods to represent parameter and structural uncertainty and considers the role of formal elicitation of expert judgements. The association between decisions to approve interventions for reimbursement and decisions about future research funding, and how value of information analysis can be used to formalize this link, is explained. Recent developments in the UK highlight the evolving policy environment for economic evaluation, such as the Cooksey report on the funding of UK health research, the review of the Pharmaceutical Price Regulation Scheme by the Office of Fair Trading and the update of the methodological guidelines issued by the National Institute for Health and Clinical Excellence. The paper concludes by describing ongoing methodological work designed to meet the challenges of undertaking decision analysis for resource allocation in health care.     

Patient value: Perspectives from the advocacy community

All health‐care systems are under financial pressure and many have therefore developed value frameworks to assist decision making regarding access to treatment. Unfortunately, many frameworks simply reflect the clinically focused values held by health‐care professionals rather than outcomes that also matter to patients. It is difficult to define one single homogeneous set of patient values as these are shaped by social, religious and cultural factors, and health‐care environment, as well as many factors such as age, gender, education, family and friends and personal finances. Instead of focusing on an aggregated set of values, frameworks should attempt to incorporate the broader range of outcomes that patients may regard as more relevant. Patient advocates are well placed to advise assessment bodies on how particular therapies will impact the patient population under consideration and should be closely involved in developing value frameworks. In this paper, a group of patient advocates explore the varying definitions of patient value and make positive recommendations for working together to strengthen the patient voice in this area. The authors call on framework developers, the patient advocacy and research communities, the health‐care industry and decision‐makers to undertake specific actions to ensure patient value is included in current and future value frameworks. This is justified on compassionate and economic grounds: better health outcomes result when patients receive treatment tailored to individual needs. Paying attention to the patient perspective also results in better use of resources—a goal that should appeal to all stakeholders.     

Communication Tools to Support Advance Care Planning and Hospital Care During the COVID-19 Pandemic: A Design Process

The COVID-19 pandemic has exposed the medical and social vulnerability of an unprecedented number of people. Consequently, there has never been a more important time for clinicians to engage patients in advance care planning (ACP) discussions about their goals, values, and preferences in the event of critical illness. An evidence-based communication tool—the Serious Illness Conversation Guide—was adapted to address COVID-related ACP challenges using a user-centered design process: convening relevant experts to propose initial guide adaptations; soliciting feedback from key clinical stakeholders from multiple disciplines and geographic regions; and iteratively testing language with patient actors. With feedback focused on sharing risk about COVID-19–related critical illness, recommendations for treatment decisions, and use of person-centered language, the team also developed conversation guides for inpatient and outpatient use. These tools consist of open-ended questions to elicit perception of risk, goals, and care preferences in the event of critical illness, and language to convey prognostic uncertainty. To support use of these tools, publicly available implementation materials were also developed for clinicians to effectively engage high-risk patients and overcome challenges related to the changed communication context, including video demonstrations, telehealth communication tips, and step-by-step approaches to identifying high-risk patients and documenting conversation findings in the electronic health record. Well-designed communication tools and implementation strategies can equip clinicians to foster connection with patients and promote shared decision making. Although not an antidote to this crisis, such high-quality ACP may be one of the most powerful tools we have to prevent or ameliorate suffering due to COVID-19.     

Approaches to Aggregation and Decision Making—A Health Economics Approach: An ISPOR Special Task Force Report [5]

The fifth section of our Special Task Force report identifies and discusses two aggregation issues: 1) aggregation of cost and benefit information across individuals to a population level for benefit plan decision making and 2) combining multiple elements of value into a single value metric for individuals. First, we argue that additional elements could be included in measures of value, but such elements have not generally been included in measures of quality-adjusted life-years. For example, we describe a recently developed extended cost-effectiveness analysis (ECEA) that provides a good example of how to use a broader concept of utility. ECEA adds two features—measures of financial risk protection and income distributional consequences. We then discuss a further option for expanding this approach—augmented CEA, which can introduce many value measures. Neither of these approaches, however, provide a comprehensive measure of value. To resolve this issue, we review a technique called multicriteria decision analysis that can provide a comprehensive measure of value. We then discuss budget-setting and prioritization using multicriteria decision analysis, issues not yet fully resolved. Next, we discuss deliberative processes, which represent another important approach for population- or plan-level decisions used by many health technology assessment bodies. These use quantitative information on CEA and other elements, but the group decisions are reached by a deliberative voting process. Finally, we briefly discuss the use of stated preference methods for developing “hedonic” value frameworks, and conclude with some recommendations in this area.