MDM Policy and Practice最新文献

Introduction. Patient decision aids support health care decision making by dynamically integrating evidence-based information with patients' values, goals, and preferences. However, most of these aids are available only in English, limiting accessibility for non-English-speaking populations. Although Portuguese is one of the most spoken languages worldwide, validated decision aids and evaluation instruments in Portuguese remain scarce. Objectives. To translate, culturally adapt, and conduct preliminary content validation of the International Patient Decision Aid Standards (IPDAS) Minimal Criteria instrument for the Portuguese population. Methods. A multidisciplinary team conducted a structured linguistic validation process, including forward and backward translation, synthesis meetings, expert committee review, and pilot testing. Clarity and content relevance were evaluated by a panel of 10 experts using a dichotomous clarity scale and a 4-point relevance scale. Quantitative measures included percentage agreement, Fleiss' kappa, and item-level and scale-level content validity indices (I-CVI, S-CVI/Ave). Qualitative input was gathered through open-ended responses and discussion during the expert panel meeting. Results. Five of the 44 criteria were identified as unclear by more than 20% of the expert panel and were revised. The overall agreement was high (0.93 [0.89; 0.97]), but Fleiss' kappa indicated low interrater agreement (0.03 [-0.01; 0.08]). Eleven criteria were rated by at least 1 panel member as less relevant or in need of revision and were refined accordingly. For all criteria, the I-CVI was >0.79, and the overall S-CVI/Ave was 0.97. Fleiss' kappa for content validity was -0.02 [-0.06; 0.02]. Conclusions. The Portuguese version of the IPDAS Minimal Criteria demonstrated strong content validity and linguistic appropriateness. This adapted instrument will enable more rigorous evaluation of patient decision aids in Portuguese-speaking contexts and support broader implementation of shared decision making.

Highlights: This study provides the first content-validated version of the IPDAS Minimal Criteria in Portuguese, addressing a significant gap in shared decision-making tools.The instrument supports Portuguese-speaking researchers and clinicians in systematically assessing decision aids for quality and usability.These findings highlight the importance of cultural adaptation in ensuring the applicability and effectiveness of decision support tools across different populations.This study advances the field of decision-making research by fostering equitable access to high-quality decision aids in clinical practice.

Background. In primary care, general practitioners (GPs) and community pharmacists (CPs) play pivotal roles in guiding patients' drug choices. Nonprescription drugs (NPDs), which are dispensed with or without a prescription, represent significant health care costs. NPDs are most often used for symptomatic relief and may be subject to shared decision making. We designed shared DECIsion in hEalth (DECIdE), a patient decision aid for NPDs in primary care, via a 3-step user-centered approach. Design. In the first step, a nominal group composed of potential future users (patients, GPs, and CPs) reached a consensus on the prototype's specifications. In the second step, GPs, CPs, and their patients tested the prototype in user tests during simulated consultations based on real-life scenarios, and clinical psychologists conducted individual interviews to improve the prototype. In the third step, international experts used the eDELPHI consensus method to validate DECIdE as a shared decision aid. Results. Sixteen participants in the nominal group reached a consensus on 18 specifications. The user tests involved 16 patients and 4 health care professionals across 2 cycles. The prototypes were improved according to an analysis of 20 individual interviews. Fifteen French-speaking experts, searchers, physicians, and pharmacists from 4 countries reached a consensus on 11 of the 13 propositions inspired by the IPDAS criteria and the French HAS criteria in 2 rounds to validate the final prototype of DECIdE. Conclusion. Interprofessional collaboration at each step of development is the main strength of this user-centered design. DECIdE is currently being evaluated for its effects on decisional conflict in GPs' practices and community pharmacies in a randomized controlled trial. Implications. DECIdE could encourage more rational use of NPDs, particularly in self-medication.

Highlights: DECIdE is the first patient decision aid in French for usual drugs in primary care, based on the latest scientific data in line with an evidence-based medicine approach for general practitioners (GPs) and community pharmacists (CPs).DECIdE's user-centered design includes interprofessional collaboration at each step between searchers, GPs, CPs, and social and clinical psychologists.The original 3 steps of the user-centered design of DECIdE combined user testing with qualitative analysis and 2 successive consensus methods to create and validate the patient decision aid using the nominal group and eDELPHI methods.The free availability of DECIdE on a dedicated Web site will enable GPs and CPs to make widespread use of shared decisions about nonprescription drugs daily.

Purpose. Health technology assessment (HTA) focuses on overall survival (OS) as the key clinical endpoint when making oncology treatment decisions. However, capturing robust OS estimates in early-stage breast cancer (eBC) is challenging, and patients may value other endpoints. This study assessed patient preferences for treatment attributes and endpoints in neoadjuvant therapy where mature and nonconfounded OS is rarely available. Patients and Methods. An online discrete choice experiment (DCE) was developed with health care professionals and patient advisory groups and conducted in Germany, France, Italy, and Spain with patients with HER2+ eBC. Patients were presented with 15 tasks and in each asked to choose between 2 hypothetical treatment options or to opt out in the neoadjuvant setting. Treatment attributes included OS at 5-years, disease-free survival (DFS) at 5-years, pathological complete response (pCR), impact of side effects on quality of life (QoL), and ability to receive breast-conserving surgery (BCS). Data were analyzed using multinomial logit and random parameters logit models. Results. Three hundred thirty-four patients with HER2+ eBC responded. The most valued attribute was achieving pCR (no invasive cancer in the breast and lymph nodes in all patients after treatment), followed by 5-y DFS in 95% of patients. OS ranked third in importance. The ability to undergo BCS and the impact of side effects on QoL were less important. Preferences varied by hormone receptor status, time since diagnosis, cancer stage, and age. Conclusion. Our findings are in-line with regulatory reviews, which accept pCR, DFS, and OS as clinically valid endpoints. When recommending reimbursements for treatments, HTA bodies and payers should consider patient preferences for treatment attributes and endpoints in their decision making when valuing new cancer treatments.

Highlights: Traditional cancer treatment assessments focus on overall survival (OS), but in early-stage breast (eBC) cancer, patients may prioritize other treatment outcomes, since robust OS data may be unavailable.A discrete choice experiment was conducted with 334 patients with HER2+ eBC in the neoadjuvant setting in Germany, France, Italy, and Spain.Patients considered achieving pathological complete response (no invasive cancer in the breast and lymph nodes in all patients after treatment) to be the most important attribute when making a treatment decision, followed by disease free-survival (DFS) and OS; the ability to undergo breast-conserving surgery and the impact of side effects on quality of life were less important.These insights highlight the need for health authorities and reimbursement bodies to consider patient-valued outcomes beyond OS in eBC treatment evaluations.

Introduction. Limited evidence guides pediatric rheumatologists on when to withdraw biologic therapy in children with juvenile idiopathic arthritis, resulting in wide variation in clinical practice. This study aimed to develop and evaluate a decision support tool (DST) based on expert opinion to support pediatric rheumatologists in making withdrawal decisions. Methods. A literature review, focus groups, interviews, and prior research informed the design of the prototype DST. Evaluation of the DST's face validity, content validity, acceptance, and feasibility was conducted through user testing interviews and a survey among pediatric rheumatologists from the Netherlands and Canada. Findings were summarized using descriptive and qualitative content analyses. Results. The prototype DST requires input on relevant patient, disease, and treatment characteristics. Its primary output is the predicted likelihood of biologic therapy withdrawal. Pediatric rheumatologists can adjust the importance of characteristics and observe the resulting impact on withdrawal likelihood. Eleven pediatric rheumatologists participated in testing. Key themes identified included the need for 1) clear terminology to ensure consistent interpretation of model inputs, 2) concise instructions on how and when to adjust the relative importance of characteristics, and 3) practice rounds to build trust among pediatric rheumatologists in the DST's output. Participants found the DST feasible for clinical use, with its main value in explaining decisions to patients and engaging them in the decision-making process. Suggested future improvements include tracking the outcomes of withdrawal decisions and integrating predictive models based on clinical data. Conclusions. The DST developed in this study was well-received. Its main value lies in helping pediatric rheumatologists explain their decisions to patients and parents. The top priority for further development is integrating scientific evidence on successful withdrawal decisions.

Highlights: Decision support tools that provide structure to decisions based on expert opinion can increase transparency and consistency in medical decision making in the absence of clinical evidence.Data from clinical vignette studies that use an experimental design to elicit treatment preferences can be used to predict treatment decision making.A decision support tool to support biologic therapy withdrawal decisions has the most value in explaining the decision to children with nonsystemic juvenile idiopathic arthritis and their parents.

Background: Parkinson's disease (PD) is a neurodegenerative disease characterized by motor and nonmotor symptoms that worsen over time. In some cases, an advanced treatment may be needed. The use of levodopa-carbidopa intestinal gel (LCIG) is one of these options. However, deciding whether to receive it can be difficult. A patient decision aid (PDA), a tool designed to inform about treatment options, can help and promote patients' participation in decision making.

Objectives: This study was conducted to develop a PDA on LCIG and assess its acceptability.

Methods: The International Patient Decision Aid Standards framework was used to develop the PDA. An advisory committee (n = 5) gave feedback on the PDA prototype. Acceptability was evaluated using a cross-sectional descriptive design. A convenience sample of 36 participants (including persons with PD receiving and not receiving LCIG, caregivers, and health care professionals) was used. Acceptability data, sociodemographics, and health literacy were collected using questionnaires and a focus group.

Results: Sample characteristics were a mean age of 64.4 y (s = 14 y), university level of education (46.7%), and duration of illness of less than 10 y (80%). The health literacy score was judged as very good ( $\overline{x}$ = 55.2/70, s = 7.3). Qualitative data analysis allowed for final adjustments to the published PDA version. Conclusions. This study is the first to report the development of a PDA on LCIG and its acceptability testing. Participants found the PDA to be useful and would recommend it. All health care professionals indicated they intended to use it in their practice. More research will be needed to evaluate the PDA's implementation and its effects on users.

Highlights: Deciding whether to opt for an advanced Parkinson's disease treatment such as levodopa-carbidopa intestinal gel can be challenging for many patients.A patient decision aid on levodopa-carbidopa intestinal gel for Parkinson's disease persons, caregivers, and health care professionals was developed to support a decision-making process.This article summarizes the steps used for its development and its acceptability testing.

Background. A barrier to early-stage oral cavity cancer detection is the lack of a defined population and screening regimen satisfying risk-benefit considerations. Methods. We constructed a microsimulation model, Simulation of Cancers of the Oral cavity and Risk Exposures (SCORE), that incorporates risk profiles defined by smoking and alcohol exposure. SCORE simulates the development and progression of oral potentially malignant disorders (OPMD) representing benign, dysplastic, or malignant lesions in the US population starting at age 40 y. OPMD high-risk characteristics of malignant transformation informed a biopsy decision rule. SCORE was calibrated to national cancer registry data. We compared life expectancy in those aged 40 to 60 y with OPMDs, cancer incidence, and cancer-specific deaths across screening strategies with and without the biopsy decision rule, assuming screening every 3 y starting at age 50 y. Results. In US men, all screening strategies reduced cancer incidence and cancer-specific mortality by at least 26% and 20% compared with no screening. Whether with or without a biopsy decision rule, life expectancy among those aged 40 to 60 y with OPMDs was 36.37 ± 0.01 life-years, a gain of 0.03 life-years. However, the use of the biopsy rule improved diagnostic efficiency with 8 biopsies per treatable diagnosis. Screening with or without the biopsy decision rule in high-risk men demonstrated comparable benefit, reducing cancer-specific deaths by 27% and incidence by 20% compared with no screening. Meanwhile, in the non-high-risk subpopulation, applying the biopsy rule avoided the harms of excess procedures, reducing lifetime biopsies by 38% versus biopsy of all OPMDs while preserving reductions in cancer burden. Conclusions. SCORE enables virtual trials of various screening regimens and target populations. Given the time and cost of clinical trials, SCORE may facilitate the evaluation of new technologies and clinical recommendations.

Highlights: A new oral cancer simulation model with risk factors including degrees of smoking and alcohol exposure, oral lesion features, and sex incorporates more accurate and precise representation of patient risk categories.We evaluated screening strategies for oral potentially malignant disorders with or without risk-stratified biopsy referral in both the general population and subpopulations defined by degrees of smoking and alcohol exposure.Men with a high degree of both smoking and alcohol exposure exhibited a significant reduction in cancer-specific deaths and cancer incidence from screening programs for oral potentially malignant disorders.Screening with risk-stratified biopsy, using a surgical treatment threshold of moderate dysplasia or worse, yielded the greatest efficiency in term of biopsies needed to detect 1 treatable case.

Objective. The patient's perspective in shared decision making has expanded to regulatory decision making for medical devices under the Food and Drug Administration's Patient Preference Initiative. Methods. Using choice-based conjoint (CBC) procedures, a discrete-choice experiment measure describing the risks and benefits of osseointegration was designed and used in a preference study among 188 adults with lower-limb loss. Our measure included 8 attributes of 1) risks: chance of infection, complete device failure rate, time without prosthesis, activity limitations, and 2) benefits: avoidance of socket problems, limb perception, improved motion with less fatigue, and chance of limiting daily pain, with 3 to 4 levels each. We used a random, full-profile, balanced-overlap design in which 18 CBC conjoint pairs, sociodemographic, and clinical questions were completed. The analysis included random parameters logit with 1,000 Halton draws and latent class. Results. The least important levels, when compared with their respective attribute baseline level, were for avoiding the highest chance (50%) of serious infection (β = -1.32, P < 0.001), highest chance (40%) of complete device failure (β = -0.96, P < 0.001), and longest (9 mo) time without prosthesis (β = -1.12, P < 0.001). The most preferred levels, when compared with their respective attribute baseline level, were to eliminate daily pain (β = 0.87, P < 0.001; β = 0.62, P < 0.001). The preference for avoiding current osseointegration infection risk (10%) was much lower (β = 0.51, P < 0.001), showing that preferences to avoid the actual infection risk are offset by osseointegrations benefits. Latent class analysis showed 2 distinct classes with some risk averse and some with more balanced preferences. Conclusions. The strongest preferences were seen for attributes avoiding complications; however, individuals demonstrated a willingness to make risk-benefit tradeoffs at current risk levels. These findings can guide future regulatory prosthetic decisions and allow better shared decision making to decrease prosthetic abandonment.

Highlights: Importance shown to avoid the actual infection risk of osseointegration can be offset by the individual's importance for the benefits of osseointegration to avoid pain and socket problems and to have rapid device snap on.Individuals also showed they are willing to trade the actual osseointegration device failure rate risks for the likely benefits of osseointegration.Individuals strongly preferred avoiding time without the use of a prosthetic for the time it takes to undergo and recover from the osseointegration procedure, informing the debate for favoring a faster procedure and recovery time if it is safe. However, these preferences were still in the tradeoff range for the benefits of osseointegration.Individuals showed the strongest importance for a potential benefit of

Background. For localized esophageal cancer, more than 1 curative treatment option is available. As these different options are associated with substantially different treatment outcomes, decision making can be complex. Moreover, treatment decision making for a patient involves multiple health care providers (HCPs) from different disciplines over time, who might have their own role and perspective on the decision-making process. This study aims to describe how HCPs communicate during treatment decision consultations with patients with localized esophageal cancer. Methods. Audio recordings of 20 preintervention scripted standardized patient assessments (SPAs) from the SOURCE trial were used. Using 2 highly similar cases, acted by a simulated patient, considerably reduced variation at patient level. Audio recordings were content coded using open coding and rated on the degree of patient involvement in decision making using the OPTION-12. Results. Radiation and surgical HCPs discussed 1 to 4 different treatment options, from a total of 5 different options observed over all consultations. They discussed 0 to 11 different side effects and complications, from a total of 28. While some HCPs explicitly presented a choice, many used various implicit forms of suggesting a choice and either implicitly or explicitly marked their own preferences for treatment. Consultations showed a mean OPTION-12 score of 40.11 (range 0-100). Conclusions. This study shows extensive practice variation in how and to what extent standardized patients with localized esophageal cancer were involved in decision making and in the number and type of treatment options and pros and cons that were presented to them. Implications. The findings suggest a need for mutual alignment within oncologic HCPs treating patients with esophageal cancer.

Highlights: Practice variation was found in how and to what extent health care providers involved standardized patients with localized esophageal cancer in decision making.Health care providers varied in the number and type of treatment options and pros and cons they presented.These findings suggest a need for multidisciplinary alignment.

Background. Liver cancer is the only cancer in Australia with rising incidence and mortality rates, despite the potential for early detection through surveillance of high-risk individuals. Hepatocellular carcinoma (HCC), the most common form of primary liver cancer, has curative treatment options available if detected early. Six-monthly HCC surveillance is recommended for people with liver cirrhosis and was proposed for inclusion in the 2023 Cancer Council Australia Clinical Practice Guidelines for Hepatocellular Carcinoma Surveillance for People at High Risk in Australia. To evaluate the proposed 2023 guideline recommendation, we developed Policy1-Liver, a novel mathematical model of liver disease, HCC, and surveillance. We then assessed the health and economic implications of 6-monthly HCC surveillance in Australia via ultrasound, with or without alpha-fetoprotein. Methods. Policy1-Liver was calibrated to existing data sources on liver disease, HCC, and health care costs in Australia. We assessed the impact of 6-monthly routine HCC surveillance with ultrasound with or without alpha-fetoprotein testing as well as a range of other sensitivity analyses and alternative scenarios such as varying surveillance adherence and intervals to assess potential future modifications to surveillance. Results. We estimated that 6-monthly HCC surveillance, with or without alpha-fetoprotein, can increase early-stage diagnoses to up to 81% and reduce HCC mortality by 22% in people with cirrhosis. We estimate an incremental cost-effectiveness ratio of $28,423 per quality-adjusted life-year for 6-monthly surveillance with ultrasound alone compared with no surveillance. Conclusions. These findings support guideline-recommended 6-monthly HCC surveillance with ultrasound, affirming its health benefits and cost-effectiveness, and demonstrate the potential to improve cost-effectiveness by refining surveillance intervals and improving early-stage HCC survival. Supporting implementation of the surveillance guidelines will play a key role in improving HCC mortality rates in Australia.

Highlights: Routine surveillance can improve the likelihood of early-stage detection of liver cancer, improving survival.Our modeling found that routine HCC surveillance with ultrasound would be cost-effective for people with liver cirrhosis in Australia.These findings can inform guidelines and investment in liver cancer control for high-risk patients.