Key Considerations in the Health Technology Assessment of Advanced Therapy Medicinal Products in Scotland, The Netherlands, and England.

OBJECTIVES: Advanced therapy medicinal products (ATMPs) are highly innovative therapies. Their costs and uncertain value claims have raised concerns among health technology assessment (HTA) bodies and payers. Little is known about how underlying considerations in HTA of ATMPs shape assessment and reimbursement recommendations. We aim to identify and assess key considerations that played a role in HTA of ATMPs underlying reimbursement recommendations. METHODS: A review of HTA reports was conducted of all authorized ATMPs in Scotland, The Netherlands, and England. Considerations were extracted and categorized into EUnetHTA Core Model domains. Per jurisdiction, considerations were aggregated and key considerations identified (defined as occurring in >1/assessment per jurisdiction). A narrative analysis was conducted comparing key considerations between jurisdictions and different reimbursement recommendations. RESULTS: We identified 15 ATMPs and 18 HTA reports. In The Netherlands and England most key considerations were identified in clinical effectiveness (EFF) and cost- and economic effectiveness (ECO) domains. In Scotland, the social aspects domain yielded most key considerations, followed by ECO and EFF. More uncertainty in evidence and assessment outcomes was accepted when orphan or end-of-life criteria were applied. A higher percentage of considerations supporting recommendations were identified for products with positive recommendations compared with restricted and negative recommendations. CONCLUSIONS: This is the first empirical review of HTA's using the EUnetHTA Core Model to identify and structure key considerations retrospectively. It provides insights in supporting and opposing considerations for reimbursement of individual products and differences between jurisdictions. Besides the EFF and ECO domain, the social, ethical, and legal domains seem to bear considerable weight in assessment of ATMPs.

An Observational Analysis of a Novel Digital Rectoscope.

BACKGROUND: This is an analysis of the first 50 in-human uses of a novel digital rigid sigmoidoscope. The technology provides digital image capture, telemedicine capabilities, improved ergonomics, and the ability to biopsy under pneumorectum while maintaining the low cost of conventional rigid sigmoidoscopy. The primary outcome was adverse events, and the secondary outcome was diagnostic view. PRELIMINARY RESULTS: Fifty patients underwent outpatient (n = 25) and surgical rectal assessment (n = 25), with a mean age of 60 years. This included 31 men and 19 women with 12 different clinical use indications. No adverse events were reported, and no defects were reported with the instrumentation. Satisfactory diagnoses were obtained in 48 (96%) of 50 uses, images were captured in 48 (96%) of 50 uses, and biopsies were successfully taken in 13 uses (26%). No adverse events were recorded. Independent reviewers of recorded videos agreed on the quality and diagnostic value of the images with a κ of 0.225 (95% CI, 0.144-0.305) when assessing whether the target pathology was adequately visualized. IMPACT OF INNOVATION: The improved views afforded by digital rectoscopy facilitated a satisfactory clinical diagnosis in 96% of uses. The device was successfully deployed in the operating room and outpatients irrespective of bowel preparation method, where it has the potential to replace flexible sigmoidoscopy for specific use cases. The technology provides a high-quality image and video that can be securely recorded for documentation and medicolegal purposes with agreement between blinded users despite a lack of standardized training and heterogenous pathology. We perceive significant impact of this technology for the assessment of colorectal anastomoses, the office management of colitis, "watch and wait," and for diagnostic support in rectal cancer diagnosis. The technology has significant potential to facilitate proctoring and training, and it now requires prospective trials to validate its diagnostic accuracy against more costly flexible sigmoidoscopy systems.

Systematic review automation tools improve efficiency but lack of knowledge impedes their adoption: a survey.

OBJECTIVE: We investigated systematic review automation tool use by systematic reviewers, health technology assessors and clinical guideline developerst. STUDY DESIGN AND SETTING: An online, 16-question survey was distributed across several evidence synthesis, health technology assessment and guideline development organizations. We asked the respondents what tools they use and abandon, how often and when do they use the tools, their perceived time savings and accuracy, and desired new tools. Descriptive statistics were used to report the results. RESULTS: A total of 253 respondents completed the survey; 89% have used systematic review automation tools - most frequently whilst screening (79%). Respondents' "top 3" tools included: Covidence (45%), RevMan (35%), Rayyan and GRADEPro (both 22%); most commonly abandoned were Rayyan (19%), Covidence (15%), DistillerSR (14%) and RevMan (13%). Tools saved time (80%) and increased accuracy (54%). Respondents taught themselves to how to use the tools (72%); lack of knowledge was the most frequent barrier to tool adoption (51%). New tool development was suggested for the searching and data extraction stages. CONCLUSION: Automation tools will likely have an increasingly important role in high-quality and timely reviews. Further work is required in training and dissemination of automation tools and ensuring they meet the desirable features of those conducting systematic reviews.

Opportunities and challenges for applying model-informed drug development approaches to gene therapies.

As part of the US Food and Drug Administration (FDA)'s Prescription Drug User Fee Act (PDUFA) VI commitments, the Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) are conducting a model-informed drug development (MIDD) pilot program. Sponsor(s) who apply and are selected will be granted meetings that aim to facilitate the application of MIDD approaches throughout the product development lifecycle and the regulatory process. Due to their complex mechanisms of action and limited clinical experience, cell and gene therapies have the potential to benefit from the application of MIDD methods, which may facilitate their safety and efficacy evaluations. Leveraging data that are generated from all stages of drug development into appropriate modeling and simulation techniques that inform decisions remains challenging. Additional discussions regarding the application of quantitative modeling approaches to drug development decisions, such as through the MIDD pilot program, may be crucial for both the sponsor(s) and regulatory review teams. Here, we share some perspectives on the opportunities and challenges for utilizing MIDD approaches for product review, which we hope will encourage investigators to publish their experiences and application of MIDD in gene therapy product development.

Extrapolating Parametric Survival Models in Health Technology Assessment: A Simulation Study.

Extrapolations of parametric survival models fitted to censored data are routinely used in the assessment of health technologies to estimate mean survival, particularly in diseases that potentially reduce the life expectancy of patients. Akaike's information criterion (AIC) and Bayesian information criterion (BIC) are commonly used in health technology assessment alongside an assessment of plausibility to determine which statistical model best fits the data and should be used for prediction of long-term treatment effects. We compare fit and estimates of restricted mean survival time (RMST) from 8 parametric models and contrast models preferred in terms of AIC, BIC, and log-likelihood, without considering model plausibility. We assess the methods' suitability for selecting a parametric model through simulation of data replicating the follow-up of intervention arms for various time-to-event outcomes from 4 clinical trials. Follow-up was replicated through the consideration of recruitment duration and minimum and maximum follow-up times. Ten thousand simulations of each scenario were performed. We demonstrate that the different methods can result in disagreement over the best model and that it is inappropriate to base model selection solely on goodness-of-fit statistics without consideration of hazard behavior and plausibility of extrapolations. We show that typical trial follow-up can be unsuitable for extrapolation, resulting in unreliable estimation of multiple parameter models, and infer that selecting survival models based only on goodness-of-fit statistics is unsuitable due to the high level of uncertainty in a cost-effectiveness analysis. This article demonstrates the potential problems of overreliance on goodness-of-fit statistics when selecting a model for extrapolation. When follow-up is more mature, BIC appears superior to the other selection methods, selecting models with the most accurate and least biased estimates of RMST.

Early Health Technology Assessment during Nonalcoholic Steatohepatitis Drug Development: A Two-Round, Cross-Country, Multicriteria Decision Analysis.

Background. The assessment of value along the clinical development of new biopharmaceutical compounds is a challenging task. Complex and uncertain evidence has to be analyzed, considering a multitude of value preferences from different stakeholders. Objective. To investigate the use of multicriteria decision analysis (MCDA) to support decision making during drug development while considering payer and health technology assessment (HTA) value concerns, by applying the Advance Value Framework in nonalcoholic steatohepatitis (NASH) and testing for the consistency of the results. Design. A multiattribute value theory methodology was applied and 2 rounds of decision conferences (DCs) were organized in 3 countries (England, France, and Germany), with the participation of national key experts and stakeholders using the MACBETH questioning protocol and algorithm. A total of 51 health care professionals, patient advocates, and methodologists, including (ex-) committee members or assessors from national HTA bodies, participated in 6 DCs in the study countries. Target Population. NASH patients in fibrosis stages F2 to 3 were considered. Interventions. The value of a hypothetical product profile was assessed against 3 compounds under development using their phase 2 results. Outcome Measures. DC participants' value preferences were elicited involving criteria selection, options scoring, and criteria weighting. Results. Highly consistent valuation rankings were observed in all DCs, always favoring the same compound. Highly consistent rankings of criteria clusters were observed, favoring therapeutic benefit criteria, followed by safety profile and innovation level criteria. Limitations. There was a lack of comparative treatment effects, early evidence on surrogate endpoints was used, and stakeholder representativeness was limited in some DCs. Conclusions. The use of MCDA is promising in supporting early HTA, illustrating high consistency in results across countries and between study rounds.

Evaluation of distance facilitation and technology in an interprofessional simulation exercise.

INTRODUCTION: This paper explores two objectives: (1) effectiveness of interprofessional education facilitators utilizing distance technology to facilitate and debrief an interprofessional exercise with students located on the islands Oahu and Hawai'i Island (Hilo), and (2) impact of technology on the simulation experience for facilitators and students. METHODS: Four disciplines participated in an interprofessional hospital discharge exercise for the same geriatric patient case. Two questions administered to facilitators and students after each session focused on: (1) satisfaction with the students' ability to work through the simulation, and (2) satisfaction with the use of distance technology. RESULTS: Results showed no significant differences when comparing students to facilitators for the students' ability to work through the simulation. Students gave significantly lower satisfaction scores with distance technology than faculty. There were no significant differences in scores among disciplines between either facilitators or students. Pharmacy distance students had significantly lower scores for satisfaction with the simulation exercise and for the role of distance technology compared to other students. Qualitative analyses showed trend improvements over five semesters in four technology areas; volume, noise, difficulty with clarity/understanding, and seating location/placement issues for the on-site group, but only significant improvement with clarity/understanding in the distance group. CONCLUSIONS: Interprofessional simulation exercises can be successfully facilitated from both on-site and distance site without compromising students' ability to work through the exercise. Satisfaction with distance technology was lower for distance student groups.

Uptake of methodological advances for synthesis of continuous and time-to-event outcomes would maximize use of the evidence base.

OBJECTIVE: The objective of the study is to establish how often continuous and time-to-event outcomes are synthesized in health technology assessment (HTA), the statistical methods and software used in their analysis and how often evidence synthesis informs decision models. STUDY DESIGN AND SETTING: This is a review of National Institute of Health Research HTA reports, National Institute for Health and Care Excellence (NICE) technology appraisals, and NICE guidelines reporting quantitative meta-analysis or network meta-analysis of at least one continuous or time-to-event outcome published from April 01, 2018 to March 31, 2019. RESULTS: We identified 47 eligible articles. At least one continuous or time-to-event outcome was synthesized in 51% and 55% of articles, respectively. Evidence synthesis results informed decision models in two-thirds of articles. The review and expert knowledge identified five areas where methodology is available for improving the synthesis of continuous and time-to-event outcomes: i) outcomes reported on multiple scales, ii) reporting of multiple related outcomes, iii) appropriateness of the additive scale, iv) reporting of multiple time points, and v) nonproportional hazards. We identified three anticipated barriers to the uptake and implementation of these methods: i) statistical expertise, ii) software, and iii) reporting of trials. CONCLUSION: Continuous and time-to-event outcomes are routinely reported in HTA. However, increased uptake of methodological advances could maximize the evidence base used to inform the decision making process.

Reweighting Randomized Controlled Trial Evidence to Better Reflect Real Life - A Case Study of the Innovative Medicines Initiative.

Evidence from randomized controlled trials available for timely health technology assessments of new pharmacological treatments and regulatory decision making may not be generalizable to local patient populations, often resulting in decisions being made under uncertainty. In recent years, several reweighting approaches have been explored to address this important question of generalizability to a target population. We present a case study of the Innovative Medicines Initiative to illustrate the inverse propensity score reweighting methodology, which may allow us to estimate the expected treatment benefit if a clinical trial had been run in a broader real-world target population. We learned that identifying treatment effect modifiers, understanding and managing differences between patient characteristic data sets, and balancing the closeness of trial and target patient populations with effective sample size are key to successfully using this methodology and potentially mitigating some of this uncertainty around local decision making.

'The problem is small enough, the problem is big enough': a qualitative study of health technology assessment and public policy on drug funding decisions for children.

BACKGROUND: Public policy approaches to funding paediatric medicines in developed public health systems remain understudied. Current approaches to HTA present a variety of conceptual, methodological and practical problems in the context of child health. This study explores the technical and sociopolitical determinants of public funding decisions on paediatric drugs, through the analysis of interviews with stakeholders involved in or impacted by HTA for child health technologies at the provincial and national levels in Canada. METHODS: We undertook in-depth interviews with a purposive sample (n = 22) of stakeholders involved with or affected by drug funding decisions for children at the provincial (Ontario) and national levels in Canada. Grounded theory methods were employed to guide data collection and analysis. Theory on 'technology-as-policy' and the sociopolitics of health technologies served as sensitizing concepts for inductive data coding and analysis. Emergent themes informed the development of conceptual and practical insights on social values and system dynamics related to child HTA, of relevance to public policymaking on the coverage of health technologies for children in Canada. RESULTS: Participant reflection on the normative and systems dimensions of drug funding for children formed two broad categories: HTA paradigms and sociopolitical context. Our analysis revealed notable differences of context and substance related to child health technology production, evaluation and use. These differences spanned the major phases of HTA (from assembly to assessment to integration) and the surrounding sociopolitical milieu (from markets to governance to politics). Careful analysis of these differences sets in relief a number of substantive and procedural shortcomings of current HTA paradigms in respect of child health. Our findings suggest a need to rethink how HTA is structured and operationalized for child health technologies. CONCLUSIONS: Current approaches to health technology assessment are not well calibrated to the realities of child health and illness. Our study presents a nuanced and contextually grounded analysis of concepts instrumental to drug funding decisions for children. The insights generated are directly applicable to the Canadian and Ontario contexts, but also yield fundamental knowledge about HTA for children that are germane to drug policy in other health systems.

Value of Information Analytical Methods: Report 2 of the ISPOR Value of Information Analysis Emerging Good Practices Task Force.

The allocation of healthcare resources among competing priorities requires an assessment of the expected costs and health effects of investing resources in the activities and of the opportunity cost of the expenditure. To date, much effort has been devoted to assessing the expected costs and health effects, but there remains an important need to also reflect the consequences of uncertainty in resource allocation decisions and the value of further research to reduce uncertainty. Decision making with uncertainty may turn out to be suboptimal, resulting in health loss. Consequently, there may be value in reducing uncertainty, through the collection of new evidence, to better inform resource decisions. This value can be quantified using value of information (VOI) analysis. This report from the ISPOR VOI Task Force describes methods for computing 4 VOI measures: the expected value of perfect information, expected value of partial perfect information (EVPPI), expected value of sample information (EVSI), and expected net benefit of sampling (ENBS). Several methods exist for computing EVPPI and EVSI, and this report provides guidance on selecting the most appropriate method based on the features of the decision problem. The report provides a number of recommendations for good practice when planning, undertaking, or reviewing VOI analyses. The software needed to compute VOI is discussed, and areas for future research are highlighted.

Analysis of sponsor hearings on health technology assessment decision making.

Objective The aim of this study was to get a better understanding of the frequency of Pharmaceutical Benefits Advisory Committee (PBAC) hearings, the factors that influence a sponsor's decision to proceed with a hearing and to assess the impact hearings may have had on PBAC decision making. Methods All public summary documents (PSDs) from March 2014 to November 2016 PBAC meetings, obtained from the Pharmaceutical Benefits Scheme (PBS) website, were examined to identify major submissions for which sponsor hearings were conducted. Each PSD was analysed to determine the topics discussed at the sponsor hearing and the 'usefulness' of a sponsor hearing from the PBAC's perspective. Results During the study period there were 472 PSDs. 74 sponsor hearings (28% of major submissions) were conducted during the study period. A clinician external to the sponsor presented at the majority of the hearings (78%) and accordingly, the main topics presented related to clinical positioning/use and clinical benefit/use. Conclusion The PBAC considered approximately 45% of sponsor hearings to be informative or moderately informative whereas 18% were classed as uninformative. What is known about the topic? Although the sponsors of medicines being considered by the Pharmaceutical Benefits Advisory Committee (PBAC) for public subsidy have been able to give a 10 min presentation to the Committee at the time of decision making for several years, it is unknown whether these hearings are beneficial. What does this paper add? We present what is believed to be the results of the first analysis of PBAC sponsor hearings. What are the implications for practitioners? All stakeholders should consider the findings of our research and associated recommendations to ensure that future sponsor hearings enhance PBAC decision making and promote good public health policy.

Social participation in the health technology incorporation process into Unified Health System.

OBJECTIVE: To describe the current process of social participation in the incorporation of health technologies in Brazil, within the context of the Unified Health System (SUS). METHODS: A descriptive study was conducted based on the analysis of official records of the actions of the National Committee for Health Technology Incorporation into Unified Health System and its website, from the beginning of its activities in January 2012 until December 2017. RESULTS: The findings indicate that, in Brazil, there are legal instruments related to social participation in health, including the health technology assessment (HTA) field. However, its implementation is relatively recent and has been carried out gradually. In addition to the legal instruments (National Health Council representative, public consultation and public hearing forecast), other information and transparency strategies have been shown to be allied to social participation in the incorporation of health technologies. However, activities such as legally provided public hearings have not yet been carried out. CONCLUSIONS: Several actions to foster social participation were developed over the analyzed period, but they need to be evaluated in order to maintain or improve them. In addition, there is a need for more qualified social participation in the various existing spaces, including those prescribed by law.

Health technology assessment (HTA) of optoelectronic biosensors for oncology by analytic hierarchy process (AHP) and Likert scale.

BACKGROUND: The multicriteria decision method (MCDM) aims to find conflicts among alternatives by comparing and evaluating them according to various criteria to reach the best compromise solution. The evaluation of a new health technology is extremely important in the health sciences field. The aim of this work is to evaluate a new health technology to assay thyroglobulin in patients with differentiated thyroid cancer to improve its service from an organizational point of view, by planning new and appropriate training activities, ensuring proper use of resources and satisfying the needs of different users. METHODS: The evaluation was performed using two methodologies: the analytic hierarchy process (AHP) and the Likert scale. The AHP is a multicriteria decision approach that assigns a weight to each evaluation criterion according to the decision maker's pairwise comparisons of the criteria. The Likert scale is a psychometric scale employed to study the degree of user satisfaction by measuring opinions. RESULTS: Results show the need of particularly improving clinical efficiency, effectiveness, and return on sales (ROS) related to the technology; technological safety, human resources and other parameters do not need to be improved because of the high satisfaction results of the users. CONCLUSIONS: The application of both methods provided the necessary information to improve the quality of the service, allowing the decision maker to identify the most valuable service features and to improve these to ensure user satisfaction and to identify possible service improvements.

Delayed Patient Access to Innovative Medical Technologies in South Korea: A Lead-Time Analysis of Reimbursement Coverage Determinations.

Background and ObjectivesTimely access to innovative medical technologies driven by accelerated patient access pathways can substantially improve the health outcomes of patients who often have few therapeutic alternatives. We analyzed lead-times for the medical procedure reimbursement coverage process undertaken in South Korea from 2014 to 2017, which is considered one of the most important factors contributing to delays in patient access to new medical technologies. METHODS: This analysis was performed using the open datasets source of "Medical Procedure Expert Evaluation Committee (MPEEC)" meeting results and medical procedure coverage application information published on the Health Insurance Review and Assessment Service Web site. RESULTS: From 2014 to 2017, 90 percent of all new coverage determinations took on average >250 days with almost 20 percent taking more than 2 years (>750 days), The average lead-time from the medical procedure coverage application to MPEEC meeting in 2015 was 435.0 ± 214.7 days (n = 26), which was significantly shorter than the average lead-time in 2014 (624.9 ± 290.3 days, n = 16) (p < .05). The average lead-time from application to official enforcement in 2015 was significantly shorter than that of 2014 (540.8 ± 217.4; n = 16 versus 734.1 ± 299.7 days; n = 26, respectively) (p < .05). CONCLUSIONS: While this analysis showed a general trend of a reduction in the time taken to receive a positive coverage determination for a new medical technology, the average lead-time remains well over the government mandated 100 days. To continue this trend and further enhance the patient access pathway for medical procedure coverage determinations, some measures can be applied. In particular, the extended "One-Stop Service" program encompassing coverage determinations is one such recommendation that could be considered.

First responder communication in CBRN environments: FIRCOM-CBRN study.

INTRODUCTION: Recent terror attacks and assassinations involving highly toxic chemical weapons have stressed the importance of sufficient respiratory protection of medical first responders and receivers. As full-face respirators cause perceptual-motor impairment, they not only impair vision but also significantly reduce speech intelligibility. The recent introduction of electronic voice projection units (VPUs), attached to a respirator, may improve communication while wearing personal respiratory protection. OBJECTIVE: To determine the influence of currently used respirators and VPUs on medical communication and speech intelligibility. METHODS: 37 trauma anaesthetists carried out an evaluation exercise of six different respirators and VPUs including one control. Participants had to listen to audio clips of a variety of sentences dealing with scenarios of emergency triage and medical history taking. RESULTS: In the questionnaire, operators stated that speech intelligibility of the Avon C50 respirator scored the highest (mean 3.9, ±SD 1.0) and that the Respirex Powered Respiratory Protective Suit (PRPS) NHS-suit scored lowest (1.6, 0.9). Regarding loudness the C50 plus the Avon VPU scored highest (4.1, 0.7), followed by the Draeger FPS-7000-com-plus (3.4, 1.0) and the Respirex PRPS NHS-suit scored lowest (2.3, 0.8). CONCLUSIONS: We found that the Avon C50 is the preferred model among the tested respirators. In our model, electronic voice projection modules improved loudness but not speech intelligibility. The Respirex PRPS NHS-suit was rated significantly less favourably in respect of medical communication and speech intelligibility.

Comparing access to orphan medicinal products in Europe.

OBJECTIVES: The primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to their full or partial reimbursement by the public health service. METHODS: Data were collated on: marketing authorisations, Health Technology Assessment (HTA) decisions, commissioning, and reimbursement decisions, and respective dates of these events for all the OMPs centrally authorised. Indicators of availability of and access to OMPs were calculated in each country and compared. RESULTS: We found that since the implementation of the OMPs Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorisation in the European Union. These OMPs are most widely accessible in Germany and France. In the other countries between 30 and 60% of OMPs are reimbursed. In particular in England, less than 50% of centrally authorised OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after authorisation - but since 2011, there is an evaluation and potentially a pricing negotiation between companies and sickness funds (third party payers). In the other countries, the shortest time from authorisation to a reimbursement decision is observed in Italy and France where it takes 18.6 and 19.5 months respectively on average. CONCLUSIONS: Marketing authorisation granted to OMPs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-OMPs too). We found that more than a half of centrally authorised OMPs were available in the five selected countries, but that access to patients was further restricted by different national reimbursement policies, especially in the UK, Italy and Spain.

The mHealth App Usability Questionnaire (MAUQ): Development and Validation Study.

BACKGROUND: After a mobile health (mHealth) app is created, an important step is to evaluate the usability of the app before it is released to the public. There are multiple ways of conducting a usability study, one of which is collecting target users' feedback with a usability questionnaire. Different groups have used different questionnaires for mHealth app usability evaluation: The commonly used questionnaires are the System Usability Scale (SUS) and Post-Study System Usability Questionnaire (PSSUQ). However, the SUS and PSSUQ were not designed to evaluate the usability of mHealth apps. Self-written questionnaires are also commonly used for evaluation of mHealth app usability but they have not been validated. OBJECTIVE: The goal of this project was to develop and validate a new mHealth app usability questionnaire. METHODS: An mHealth app usability questionnaire (MAUQ) was designed by the research team based on a number of existing questionnaires used in previous mobile app usability studies, especially the well-validated questionnaires. MAUQ, SUS, and PSSUQ were then used to evaluate the usability of two mHealth apps: an interactive mHealth app and a standalone mHealth app. The reliability and validity of the new questionnaire were evaluated. The correlation coefficients among MAUQ, SUS, and PSSUQ were calculated. RESULTS: In this study, 128 study participants provided responses to the questionnaire statements. Psychometric analysis indicated that the MAUQ has three subscales and their internal consistency reliability is high. The relevant subscales correlated well with the subscales of the PSSUQ. The overall scale also strongly correlated with the PSSUQ and SUS. Four versions of the MAUQ were created in relation to the type of app (interactive or standalone) and target user of the app (patient or provider). A website has been created to make it convenient for mHealth app developers to use this new questionnaire in order to assess the usability of their mHealth apps. CONCLUSIONS: The newly created mHealth app usability questionnaire-MAUQ-has the reliability and validity required to assess mHealth app usability.

Measuring Survival Benefit in Health Technology Assessment in the Presence of Nonproportional Hazards.

BACKGROUND: Proportional hazards (PH) is an assumption often made by researchers, despite evidence of nonproportionality in a significant proportion of clinical trials. In the presence of non-PH, the interpretation of hazard ratios, medians, and landmark survival as summary measures of treatment effect can become problematic. Several recent studies have recommended restricted mean survival time (RMST) as an alternative metric for survival analysis, particularly where non-PH may apply. OBJECTIVES: To determine the current approaches of health technology assessment (HTA) agencies to value assessment in the presence of non-PH, and the extent to which RMST is accepted as an alternative measure of treatment benefit. METHODS: Methodological guidelines published by 10 HTA agencies were reviewed to establish recommended approaches for presenting survival benefit from clinical trials. Published HTA reports for 23 oncology agents approved by the US Food and Drug Administration and the European Medicines Agency since 2014 were reviewed to determine how guidelines are implemented in practice and identify instances where the PH assumption was tested and RMST analyses reported. RESULTS: Testing for non-PH is not widely incorporated into HTA except by the UK National Institute for Health and Care Excellence. RMST is used infrequently but has been used in a number of countries, particularly by agencies that focus on cost effectiveness. CONCLUSIONS: HTA agencies vary in their approaches to non-PH. Most do not routinely check the PH assumption. RMST has played a role in assessing clinical benefit within HTA, although not consistently within countries (across drugs) or across countries (for the same drug).

Experience of Using an App in HIV Patients Older Than 60 Years: Pilot Program.

BACKGROUND: New technologies can promote knowledge of HIV infection among patients suffering from this disease. Older patients with HIV infection represent an increasingly large group that could benefit from the use of specific apps. OBJECTIVE: The aim of the study was to observe the acceptability and use of a mobile app on HIV infection in patients at least 60 years old and offer them the possibility of anonymously establishing contact with their peers. METHODS: A series of clinical and psychosocial parameters were studied in 30 HIV-infected patients of over 60 years. The patients must be at least 60 years old, with a follow-up in the outpatient clinic for at least 1 year and without pathologies that limit his or her life expectancy to less than a year. They must know how to read and write. To be part of the group assigned to the app, they had to have their own smartphone and confirm that they were connected to the internet from that device. Overall, 15 of them were randomized to use an app and 15 were in the control group. All tests were repeated after 6 months. RESULTS: The median age of patients was 66.5 years. Among them, 29 patients had an undetectable viral load at baseline. The median number of comorbid diseases was 2. Overall, 11 of them lived with their partners and 19 lived alone. They spent an average of 5 hours a day sitting down, and 56% (17/30) of them referred high physical activity. They scored 4 out of 5 for general quality of life perception. Moreover, 80% (24/30) presented high adherence to their treatment, and the average number of concomitant medications was 5. In the 6-min walking test, they covered a distance of 400 meters, and 3 of them desaturated during the test. The 15 patients made frequent use of the app, with 2407 sessions and an average of 7 min and 56 seconds time of use with a total of 13,143 screen views. During the 6 months of the trial, 3 non-AIDS events took place. There were no significant modifications to body mass index, blood pressure measurements, lipid profile, or immuno-virology information data. There were no differences in the questionnaire scores for perception of quality of life, confessed physical activity, or antiretroviral treatment (ART) and non-ART treatment adherence. CONCLUSIONS: Significant differences between studied parameters were not objectified in these patients, possibly because this trial has significant limitations, such as a small sample size and only a brief follow-up period. However, patients did use the app frequently, making this a possible intervention to be proposed in future subsequent studies.