In 2017, a Patient Involvement Interest Group (PIIG) was created in the Spanish Network for Health Technology Assessment of the National Health System (RedETS) to facilitate and promote Patient Involvement (PI) in Health Technology Assessment (HTA). The PIIG proposed a decisional flowchart to guide researchers’ in decisions regarding PI methods in HTA. The flowchart proposed a combination of direct involvement and incorporation of patient-based evidence depending on the scope and the aims of the assessment.

This work aims to present the flowchart and the results of the evaluation of the latest experiences in PI in HTA in RedETS (2018–2020), including direct-involvement and patient-based evidence.

A survey was sent to the HTA researchers who implemented PI initiatives in RedETS assessments. The survey asked to describe their experiences, lessons learned, challenges and added value regarding the use of direct-involvement, systematic reviews (SR) and primary studies. A descriptive analysis was performed and the results were discussed in an online PIIG workshop.

Thirty-two assessments included direct PI, twenty-one SR synthesized qualitative and quantitative studies about patient experiences, values and preferences and eight included primary studies, mainly of qualitative design. Recruitment and the lack of methodological resources were the main barriers both for direct PI and primary studies. Relevance of the included studies was the main barrier for SR. Added value was found in all PI methods. Direct-involvement had an impact on the project plan and PICO definition, outcomes relevance, information about the health condition and treatments. SR contributed with relevant patient-based evidence, deeper assessment of patient experiences, values and preferences and implementation factors. Primary studies developed new or contextualized knowledge directly applicable to decision-making.

The PI flowchart has served to facilitate the incorporation of patient input in HTA reports. The different approaches implemented have allowed to provide relevant and well-grounded data in each report to inform decision-making in patient-centered healthcare provision, but it is necessary that specific training and resources are provided to enable adequate and timely implementation.

The European Health Technology Assessment Network (EUnetHTA) Work Package 5B1, is focused on testing the levels of cross-border collaboration on real world data for supporting reimbursement/pricing decision-making. Within this Work Package, we are conducting a pilot on Left Ventricular Assist Device on destination therapy in collaboration with the National Institute for Health and Care Excellence (NICE, UK), the Belgian Health Care Knowledge Centre (KCE, Belgium) and the Italian National Agency for Regional Health Services (AGENAS, Italy). This pilot aims to define the minimum data set for gathering and sharing high quality registry data on key uncertainties found at the time of the health technology assessment (HTA). Furthermore, the pilot will assess the feasibility of carrying out a common analysis or reusing this data for National or Joint Reassessments.

Evidence gaps were based on the four national assessments. Collaborating partners were responsible for agreeing on the key outcomes and proposing the minimum dataset to be registered. European clinical experts and patients rated and prioritized the dataset using a two round Delphi technique (not relevant, important but not critical; critical). The dataset will confirm the basis for the Spanish LVAD registry, implemented at the national health service level to inform inclusion into the healthcare portfolio.

The key outcomes agreed upon by agencies relate to safety, effectiveness, satisfaction and acceptability of the patient and cost-effectiveness, budget impact and organizational impact. Expert cardiologists and cardiac surgeons representing the European and Spanish Society, among others, participated in the prioritization of basic data. The final dataset is expected by December 2020.

The variation in the quality and definition of outcome measures for measuring key evidence gaps reduces the utility of registries for HTA, making it difficult to compare, link, and aggregate data across countries. The EUnetHTA pilot is intended to offer a model for cross-border collaboration on real world data for supporting the decision-making process for pricing and reimbursement.

The COVID-19 pandemic shows that the impact of effective vaccines extends well beyond vaccinated individuals and healthcare systems. Yet, these externalities are not typically considered in health technology assessments (HTA) which may underestimate vaccines’ broader value. We explored to what extent future vaccines relevant to England might exhibit such broader value.

We compared the ten value elements of an existing vaccine evaluation framework to the value elements considered in England according to the Joint Committee on Vaccine and Immunisation (JCVI) and the National Institute for Health and Care Excellence's (NICE) guidelines. Using literature and expert opinion we then explored, for a selection of ten vaccines with an expected UK-launch within five years, on which value elements each vaccine might potentially show added value.

Up to five of ten value elements are unlikely to be considered by JCVI or NICE, including patient and carer productivity, enablement value, impact on antimicrobial resistance and transmission value. Of vaccines studied, 100 percent will potentially generate value on at least one broader value element that is currently ignored; 60 percent to 80 percent may increase vaccinee/patient or carer productivity respectively.

There is a substantial gap between value generation and value recognition of vaccines in HTA in England. This might lead to undervaluation and underutilization of vaccines, leaving societies more vulnerable than needed when faced with infectious diseases.

Healthcare services, such as cochlear implants and subsequent rehabilitation, aim to increase valuable activities and opportunities of those affected. Their impact may be inferred from the extent that they protect or restore capability, which reflects the real freedoms that people have to be or do things they have reason to value. Capability emerges from the dynamic interaction between available resources, individual, social, and environmental conversion factors, and functionings. This model sets the informational requirements of the capability approach.

On the basis of interviews with thirty-three hearing impaired children and thirty hearing peers, information on capability elements (values, resources, conversion factors, and functionings) was collected. Qualitative results were triangulated with standardized clinical audiological and psycholinguistic quantitative measures.

Hearing impaired children and their hearing peers concurred in terms of the doings and beings they valued, but differed in terms of conversion factors to realize capability. Parents of hearing impaired children played a more upfront role, hearing impairment predominated many areas of life, and communicating through hearing aids required more energy than was usually acknowledged by the people around them.

The capability approach offers opportunities not only to assess impact of technology on dimensions that are important to patients, but also to better understand the mechanisms that are involved in value generation.

Health Technology Wales (HTW) is committed to evaluating the impact of our work. In March 2020, HTW directed efforts to support Welsh Government and health and social care providers in response to the COVID-19 pandemic. We adapted the HTW evaluation framework to specifically capture the impact of our additional COVID-19 work. Here we analyze data collected since the framework was implemented.

Both formal and informal feedback was analyzed. Formal feedback was obtained through the HTW Impact Questionnaire, which was developed to support more formalized data capture for all HTW workstreams and to facilitate feedback from all stakeholder groups. It was piloted with a targeted list of individuals and responses were received for COVID-19 work. Informal feedback included feedback received via email or through word of mouth.

HTW COVID-19 products to date include Topic Exploration Reports, rapid evidence summaries and an Evidence Appraisal Report (EAR) on COVID-19 diagnostic tests (molecular and antibody tests). Stakeholders were positive about these outputs, describing them as valuable and informative. Reported impacts included informing policy and decision making, reducing duplication of efforts and helping to target development. The EAR received national and international focus, leading to HTW involvement in the European Network for Health Technology Assessment (EUnetHTA) COVID-19 reviews. Survey participants who gave feedback on COVID-19 activities included two members of Health Technology Assessment organizations, a health board representative and an industry representative; all agreed that HTW's COVID-19 work was useful, that the methods were reliable and robust and that HTW is responsive. All participants also felt that HTW's COVID-19 work had a positive impact in the wider health and social care context.

HTW was able to respond rapidly to the COVID-19 pandemic and adapt current evaluation practices to capture the impact of COVID-19 work. We will continue to evaluate our COVID-19 activities. Future work will involve following up on the developing impact of our COVID-19 work and expanding our methods for data capture, for example conducting stakeholder interviews.

The expansion of health data offers exciting opportunities to support better and more efficient drug discovery, development and implementation. Data protection and governance provide the legal framework to balance safeguarding patients’ privacy with the benefits to society of medical research. Our aim is to highlight current legal barriers to the better use of health data and propose ways to address them.

Analysis of the relevant legislative texts was supplemented by interviews with external experts in data protection, health research, informatics and cyber security and a workshop with pharmaceutical industry members. We investigated the legal issues arising for six key activities along the pharmaceutical lifecycle, from identifying unmet need through to health technology assessment and pharmacovigilance.

The General Data Protection Regulation (GDPR) was introduced in May 2018 to Harmonise data protection across Europe. However, considerable ambiguity remains, particularly around the appropriate legal bases for data processing in the absence of consent: scientific research, public interest, or provision of health or social care. Other key themes included data subject rights, anonymization, compatibility of primary and secondary (re-)use of data, heterogeneity arising from divergent interpretation, the need for guidance on digital health, and the importance of trust.

We speculate which legal bases are most appropriate for the six pharmaceutical activities studied, but clear guidance and consensus is required. The GDPR was not designed to hamper scientific research, and the issues identified arose from uncertainties rather than barriers per se. Industry and academic researchers should therefore deal proactively with the prevailing uncertainties, share good practice, and engender trust by co-creating a code of conduct and outlining principles of responsible use. Engagement with patients will be critical in encouraging a shared understanding of the value to society of health data for research.

Often health technology assessment (HTA) products developed by the Scottish Health Technologies Group (SHTG) did not reach clear directive conclusions because the evidence base for a technology was weak. Despite being methodologically robust, these products did not meet the needs of decision-makers and may have had negligible impact.

SHTG set out to equip and empower the recommendation-making council (that is, appraisal committee) to reach clear conclusions. SHTG broadened the HTA components and types of evidence that could be considered. The increased breadth of evidence included: clinicians attending council meetings to respond to questions; patient groups making submissions and presenting at council meetings; Scotland-specific economic modelling; and consultation on draft recommendations. SHTG also restructured the council for improved deliberative decision-making.

Clear directive conclusions were reached in a substantially higher proportion of HTA products (eighty-eight percent in 2019 compared with eight percent in 2017). It became possible for decision-makers to implement findings. It also became feasible to assess the impact and implementation of recommendations.

Broadening SHTG's consideration of HTA components has led to a clearer conclusion being reached and stronger messaging for decision makers. This positions SHTG to increase its influence in the use of health technologies in Scotland.

The Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU) is commissioned to assess ethical aspects in their health technology assessment (HTA) reports, in addition to effects and health economic aspects of the examined interventions. For this purpose, a framework for systematic evaluation of ethical aspects of healthcare technologies has been developed and used at SBU since 2014. With seven years of practice, we decided it was time to evaluate experiences from using the ethical framework and consider possible adjustments to improve future use.

SBU reports in the time period 2014–2020 were systematically screened for ethical content. Focus group meetings with users of the framework (mainly HTA project managers) were held where opinions regarding usability and possible obstacles were collected. A revised version of the document was sent for consultation to relevant stakeholders (possible users, reviewers and recipients) in order to collect additional views.

Of fifty-eight HTA reports produced in the time frame, ethical aspects were evaluated in fifty-five reports (ninety-five percent), and in most cases, the framework had been used as support. In twenty-one cases (thirty-six percent), a professional ethicist had been engaged in the work. In twelve cases (twenty-one percent), ethical aspects were presented in the main conclusions of the report. Opinions from users and reviewers revealed that the framework was generally regarded as a helpful tool, but problems regarding interpretation of specific questions were highlighted and subjected to revision.

The ethical framework is a valuable tool for systematic and transparent identification and discussion of ethical aspects in the HTA context, and it has been well implemented at SBU. A systematic approach to assess ethical aspects can facilitate the communication and dissemination of ethical aspects as principal results from the HTA project.

Since 2013, the coverage of innovative and expensive drugs by the French National Health Insurance considers cost-effectiveness and budget impact, as assessed by the National Authority for Health (HAS) on the basis of an evaluation submitted by the firm. First CAR-T cell therapies were subject to economic evaluation in 2019 in France. We aim at describing the process and results of the economic evaluation of tisagenlecleucel and axicabtagene ciloleucel and the challenges these evaluations raised.

Primary evaluations were submitted by the firms to be reviewed by HAS. The final analyses were submitted to the Committee of Economic Evaluation and Public Health (CEESP), composed of independent economists, clinicians and patients’ representatives. The CEESP issued Opinions related to i) the methodological quality of economic evidence and ii) the cost-effectiveness and budget impact of the drugs under review.

The estimated incremental cost-utility ratio (ICUR) of tisagenlecleucel were rejected, being based on insufficient clinical evidence to estimate and extrapolate the long-term progression and to compare tisagenlecleucel with alternatives. Thus, the CEESP concluded that tisagenlecleucel was not proved cost-effective. The estimated ICUR of axicabtagene ciloleucel at 114,509EUR/QALY vs. chemotherapies was associated with an acceptable level of evidence despite being based on a frail indirect comparison and limited data on quality of life. In a context where France has no official cost-effectiveness threshold, the CEESP considered axicabtagene ciloleucel ICUR to be “very high” and questioned the collective acceptability of the claimed price.

The CEESP stressed that the main source of uncertainty surrounding the ICUR estimates of both drugs was related to the lack of hindsight on effectiveness, especially in terms of overall survival and safety.

The economic evaluation of CAR-T cell therapies highlights the sources of uncertainty underlying the decision and the risk of inefficient resource allocation driven by limited clinical data. It calls for payment schemes accounting for the uncertainty, and effective collection of relevant post-marketing data.

Before the coronavirus pandemic, children who were on the Early Years Neurodevelopment (EYND) assessment pathway and suspected to have possible Autism Spectrum Disorder (ASD), received clinic based appointments. This process included a parental interview by a doctor, a specialist speech and language therapy assessment, autism diagnostic observation schedule (ADOS), which were all carried out on hospital sites. These were postponed in March following national guidance. Our aim was to continue providing accurate evidence-based service for ASD diagnosis.

We utilised evidence-based telehealth methods to perform a specialist speech and language assessment in a child's home via video call. Parents were also invited to share videos of everyday activities via a secure portal. We could observe the child in a meaningful setting and witness functional impact of their needs. Each case is discussed by a multiagency panel based on DSM-V criteria.

Online training was undertaken by professionals to deliver the Brief Observation of Autism Symptoms (BOSA) based on the ADOS for COVID times. Parents were coached by the therapist to enable them to become the administrator, rather than a professional.

Telephonic feedback from the first ten parents whose children underwent a telehealth assessment has been positive; the home was deemed more natural and for some less distressing than clinic. Formal patient surveys have been devised for both the telehealth and BOSA clinic assessments. Analysis is expected by the end of March.

To date we have been able to reach an outcome for thirty children, the diagnosis of ASD for twenty-four children and the other six received a diagnosis of global developmental delay or language disorder.

We expect that telehealth will reduce the number of assessments before an ASD diagnosis is made resulting in more prudent healthcare. The new methods have demonstrated clear increased parental participation.

The Scottish Health Technologies Group (SHTG) set out to assess the impact of HTA products. Two questions were posed: Does advice from SHTG have influence? How is SHTG advice used?

SHTG adapted a tool developed by the International Network of Agencies for Health Technology Assessment (INAHTA). The INAHTA framework investigates indications of impact and categorizes outputs into levels of impact. Over three years, potential users of SHTG advice were contacted six to twelve months after advice was published and asked how the advice had been used. HTA outputs were categorized into the four levels of influence they achieved: ‘major influence’, ‘some influence’, ‘some consideration’ and ‘no known influence’.

HTA products were found to have been used in four main ways: ‘informed discussion’, ‘referenced’, ‘informed policy’ or ‘directly informed practice’. Levels of influence had steadily increased over the three years assessed. The findings were well received by internal audiences, with particular interest in the various ways HTA recommendations had been used. There was also feedback about ‘marking our own homework’. These results have informed a new SHTG strategy and supported clear messaging around the value of HTA.

SHTG has found a pragmatic, resource-light way to explore the impact of HTA outputs, which has proved valuable for driving strategy and messaging.

The Department of Management and Incorporation of Technologies and Innovation in Health (DGITIS) acts as Conitec's Executive Secretariat. Among its attributions, it promotes the public/patient involvement in the health technology assessment (HTA) process. Recently, Conitec has been working on the inclusion of patient's testimonials about their illness experience in the plenary sessions, that is, the monthly meeting where technologies are assessed.

To support the action of including patient reporting in Conitec's HTA process, DGITIS developed research on HTA agencies websites worldwide. The main criteria was the inclusion of patients’ reports in their Committee meetings. DGITIS contacted some of these agencies and requested a listserv question to the International Network of Agencies for Health Technology Assessment (INAHTA) members. These findings supported the DGITIS for the inclusion of patient participation in Conitec's meetings, from the selection process to the actual participation.

For the Conitec's HTA process, the patients’ participation should occur in the prior session to the public consultation, guaranteeing the inclusion of their perspective since the recommendation process beginning. Hence, every demand for incorporation to be discussed at Conitec's meeting should be preceded by a public call for patients with the clinical condition. The DGITIS will also hold preparatory meetings, which will serve as moments for shared construction of knowledge and literacy.

The nomination process, so far, has been grounded as a consensus among the patients. Thus, Conitec acts as a mediator, connecting the involved stakeholders, in a way that they can autonomously organize themselves and indicate the main representative and an alternate one. With the inclusion of the patient's perspective in the Conitec's meeting, another form of patient participation was opened in the HTA process. Therefore, the consolidation of this participation space is feasible and contributes to enrich the Brazilian HTA process.

Medical devices play an essential role in health care, but they are also a leading causes of increasing healthcare expenditures. The purchase of technologies and the determination of how and when they should be used are among the most important decisions made by decision-makers, at the institutional level.

The present research focuses on the Portuguese health system and sheds light on the characterization of decision-making process by those involved in Magnetic Resonance Imaging (MRI) purchases.

To characterize the decision-making process, results from forty questionnaires and twenty-seven semi-structured interviews with key decision-makers were merged, using a mixed method approach. To assess competences for decision-making, a questionnaire was applied, and Exploratory and Confirmatory Factorial Analysis conducted.

Cost and suppliers’ characteristics are seen as the most important indicators to guide decisions. The decision is undertaken by a committee, in a bottom-up process, characterized by a bounded rationality, influenced by intuition and a consultant decision-maker. The reasoning and justification for selection of the committee members is unclear. The decision process is considered to be bureaucratic, time-consuming and long. Patients are negatively perceived as stakeholders in the process. Few studies were performed (mostly related to the workload of the Radiology Department) to support the decision and no national or international health technology assessment (HTA) study was used in the process, to guide decisions. Decision-makers have limited knowledge and training in areas of decision-making in the areas of health informatics, health economics and especially HTA. This may limit their ability to truly understand the future implications of their purchase decisions.

To foster HTA in decision-making processes, recommendations are made, in particular, to: (i) establish an HTA in-house unit, able to carry out studies considering the hospital context and aiming to inform managerial local decisions (ii) promote a team comprised of technology assessment multidisciplinary researchers but also professionals from the health institution able to carry out HTA studies (iii) foster common languages and values to increase uptake of HTA studies.

Prescribing of medicines in primary care in Wales has been exceptional in 2020 due to COVID-19 and the associated changes to the delivery of health services. The changes are likely to have harmful, albeit unintended, consequences, including disruption of pharmacy stock management; unpredictable changes in prescribing; and interruption to patients’ supply of medicines and reduced medication adherence. Changes in prescribing are unlikely to be distributed evenly across the country or population. Therefore, this study aimed to identify changes in GP prescribing compared with previous years, the variation of these changes, and factors related to the variation in changes, to identify patient subgroups for whom the impact is disproportionate.

We identified medicines of interest where concerns around prescribing have been raised and, for each of these medicines, retrieved monthly prescribing data for each GP practice in Wales (N = 492). We then linked these data with other publicly available data (for example, practice size, indices of multiple deprivation, disease prevalence).

We developed a novel approach to measure the impact of COVID-19 on GP prescribing. We compared observed with expected prescribing volume projected via time series modelling and differences were related to patient and practice characteristics using general estimating equations.

There was evidence of stockpiling of medicines during March 2020 (for example, oral-contraceptives and oral-anticoagulants with 11.6 and 18.5 percent increases from March 2019), followed by a short-term reduction in prescribing for oral-contraceptives (a reduction of 12.9 percent), but not oral-anticoagulants (an increase of 6.5 percent). However, GP level data show considerable deviation from the national trend for several GPs, which may be due to health and socio-demographic factors.

COVID-19 has had a major impact on primary care prescribing in Wales. The distribution of changes in prescribing will not be even across the country or the population. Identification of systematic variation in impacts on prescribing could identify geographical areas or patients in need of additional support to ensure uninterrupted and appropriate access to medicines.

Gaining the perspective of patients is invaluable in the design, management and reporting of research. As part of the process of facilitating clinical research into the effectiveness of a digital colposcope in a cervical cancer pathway, patients were involved from the outset.

Using funding made available by a Public Involvement Fund, a patient consultation group was established. The group's initial discussions informed the design of a feasibility study and funding application, which was submitted to the UK National Institute of Health Research (NIHR). A Patient and Public Involvement (PPI) representative was recruited and along with the consultation group, contributed to the ethical approvals for the study. The Patient Information Sheet and Consent Form were reviewed by the patients, to ensure readability, understandability and accessibility. The patient questionnaires and interview topics that are part of the feasibility study were also developed in conjunction with the PPI group, to make sure that women's concerns are being addressed in the research design and protocols.

The PPI consultation group's contributions helped strengthen the funding application and funding for a feasibility study was granted as part of the NIHR's Research for Patient Benefit funding scheme. Part of the grant will be used for training and reimbursement for time spent for the PPI representative. Data collection for the study is due to commence in the summer of 2021. The PPI group will be consulted at the beginning and end of the data collection period and will contribute to the data analysis and dissemination of the research output, including a Plain English Summary.

Involving patients greatly amplified the quality of the funding and ethical applications and will continue to benefit the ongoing research. Resources were widely available within the researcher's University and also through UK-wide schemes. Such resources are crucial and should be encouraged as part of all clinical research.

With unprecedented times, comes accelerated change. Hospitals in our region have begun to facilitate safe discharge for COVID-19 patients in the form of “The virtual COVID ward”. This has enabled patients to be monitored safely in the community using pulse oximetry, Florence (a telehealth mobile app) and remote consultations. Our objective is to expand upon this model by providing home oxygen therapy for these patients facilitated by telemedicine.

Patients were discharged with an oxygen concentrator if they had an oxygen requirement equal to or less than four litres/minute. Fraction of inspired oxygen needed to be stable and an early warning score of less than four was also required. Once admitted, the Florence app and daily remote consultations were crucial to closely monitor the patient's clinical status. The patient was instructed to enter oxygen saturations and heart rate into the app four times daily. The app would then alert our team if any patients observations deteriorate, triggering immediate assessment.

We have discharged ninety patients to the virtual ward, fifty-six of these with home oxygen. The average age was fifty-seven and the Clinical Frailty Score ranged between one and six. At present, ten patients have been re-admitted, four with increasing oxygen requirements, and six with unrelated symptoms. Two patients had oxygen concentrators installed at home after we were alerted to their desaturation by the Florence App. The re-admission rate is eleven percent, which mirrors that of other virtual wards (who do not provide home oxygen). In total, the ward has saved the trust 627 hospital inpatient ‘days’. Patients report increased satisfaction at playing a meaningful role in monitoring their own healthcare using the app.

Our novel model of supported discharge with oxygen therapy using telehealth demonstrates that it is possible to manage such patients, safely, in the community. Other trusts could utilise this model to reduce inpatient bed occupancy. Looking to the future, could telehealth be utilised further to facilitate other “Virtual wards” in the community?

Wearable Digital Health Technologies (WDHTs) can support and enhance self-management by giving individuals with chronic conditions more control over their health, safety and wellbeing. Involving patients early on in the design of these technologies facilitates the development of person-centered products. It may increase the potential uptake of (and adherence to) any intervention they are designed to deliver. This research aims to elicit chronic kidney disease (CKD) patients’ preferences for WDHTs that may help patients manage their conditions.

We used discrete choice experiments (DCE) to elicit preferences for WDHTs characterized by their generalizable characteristics. The study design was informed by a multi-stage mixed-method approach (MSMMA). This included a review of the published literature, focus group interviews and one-to-one interactions with CKD patients to identify relevant characteristics (that is, attributes and levels) associated with wearable DHTs. We collected the data from 113 patients (age ≥18 years) with stage 3 or above CKD. The analysis started with a conventional multinomial logit model and was extended by investigating heterogeneity in preferences via latent class models.

Our MSMMA yielded ten potential attributes for consideration in a choice task. The final list included five attributes, cross-checked and validated by the research team, and patient representatives. The most preferred attributes of WDHTs were device appearance, format and type of information provided, and mode of engagement with patients. Respondents preferred a discreet device, which offered options that individuals could choose from and provided medical information.

We show how to use MSMMA to elicit user preferences in (and to inform the) early stages of the development of WDHTs. Individuals with CKD preferred specific characteristics that would make them more likely to engage with the self-management support WDHT. Our results provide valuable insights that can be used to inform the development of different WDHTs for different segments of the CKD patients population, moving away from a one-size-fits-all provision and resulting in population health gains.

Zio XT Service was one of five Digital Health Technologies (DHTs) to be assessed by the National Institute for Health and Care Excellence (NICE) as part of their evaluation pilot. The King's Technology Evaluation Centre (KiTEC) act as an External Assessment Centre for NICE and worked on this pilot evaluation. The service comprises a I-Lead ECG patch, an inbuilt software that makes use of artificial intelligence (AI) algorithms to record, store and analyze ECG traces, and a team of cardiac physiologists.

Although the methods were based on NICE's existing Medical Technologies Guidance Process, they were modified to suit the assessment of DHTs. The process was split into two sections, with the option to discontinue the assessment if it was considered that insufficient evidence was available for the technology. Clinical experts and patients were consulted through the process and clinical, economic and technical evidence was considered. Costs for three care pathways were modelled.

A total of thirty relevant clinical studies were identified, with a further study being reviewed as part of a separate technical assessment, focusing on the AI component of the technology. Four of the studies were considered to be pivotal to the decision problem, one of which was a Randomized Controlled Trial. The technology was found to have a greater diagnostic yield than a standard ambulatory monitor, however diagnostic accuracy measures were absent in the literature. Three economic models were developed to represent three care pathways: patients with syncope, patients who have had a stroke or transient ischaemic attack and a third model assessing downstream costs associated with stroke treatment.

Digital Health Technologies and Artificial Intelligence Technologies pose novel and unique challenges to health technology assessment (HTA) bodies. Zio XT Service is a diagnostic tool, with both human and AI input, making it a particularly complex technology to assess. This work serves as a case-study in the evaluation of DHTs and AI and the lessons learned may contribute to the development of guidelines for such technologies.

Wearable digital health technologies (WDHTs) offer several solutions in terms of disease monitoring, management and delivery of specific interventions. In chronic conditions, WDHTs can be used to support individuals’ self-management efforts, potentially improving adherence to (and outcomes resulting from) interventions. Early health technology assessment (HTA) methods can inform considerations about the potential clinical and economic benefits of technology in the initial phases of the product's lifecycle, facilitating identification of those Research & Development (R&D) investments with the greatest potential stakeholders’ payoff. We report our experience of using early HTA methods to support R&D decisions relating to novel WDHT being designed to support self-management of chronic kidney disease (CKD).

We performed a literature review, focus-group interviews with patients, and qualitative interviews with the prototype development team to understand the relevant characteristics of WDHTs, quantify relevant clinical indications and existing technological constraints. An early economic evaluation was used to identify the key drivers of value for money, and a discrete choice experiment shed light onto patient preferences towards what key features the WDHT should have for the users to adopt it. Then a model-based cost-effectiveness analysis was undertaken incorporating headroom analysis, return on investment, one-way sensitivity analysis and scenario analyses using data from secondary sources.

The review of the literature, focus groups with CKD patients, and qualitative interviews with technology developers helped to understand relevant characteristics of WDHT and user preferences helped inform the next R&D iteration. Compared to the standard care, WDHT that support stage ≥3 CKD patients self-management at home by measuring blood pressure and monitor mobility has the potential to be cost-effective at conventional cost-effectiveness threshold levels. From the headroom analysis, novel WDHT can be priced up to GBP280 (EUR315, USD360) and still be cost-effective compared to standard home blood pressure monitoring.

Our study provides valuable information for the further development of the WDHT, such as defining a go/no-go decision, as well as providing a template for performing early HTA of Digital Health Interventions.

The MAVORIC trial compared mogamulizumab to vorinostat in patients with mycosis fungoides (MF) or Sézary syndrome (SS), subtypes of cutaneous T-cell lymphoma. However, the treatment comparison within MAVORIC may not represent an HTA relevant population from a UK perspective: (i) 72.6 percent of patients randomized to vorinostat switched to mogamulizumab and (ii) vorinostat is not used in current clinical practice in the UK. This study explores methods to adjust treatment effect estimates using different crossover adjustment methods and Real-World Evidence.

This medicine is subject to additional monitoring. This will allow quick identification of new safety information. See www.mhra.gov.uk/yellowcard for how to report side effects.

An advanced stage (stage ≥IIB MF and all SS) population was included. Three methods were considered for treatment crossover adjustment. A synthetic control arm was created using the Hospital Episodes Statistics (HES) dataset. Predicted survival for the MAVORIC control arm, post-crossover adjustment, was compared to the HES to inform the selection of the appropriate methods for adjustment. A direct comparison between mogamulizumab (reweighted to represent the distribution of MF/SS patients in the HES) and the synthetic control was also conducted.

Following crossover adjustment of the vorinostat arm, using the inverse probability of censoring weighting method, the overall survival (OS) hazard ratio (HR) estimate for mogamulizumab vs. vorinostat was 0.45 (95% confidence interval (CI): 0.19, 1.07). This adjustment method was considered the most appropriate based on an assessment of assumptions and a comparison of OS between the adjusted vorinostat data and the HES data. The OS HR estimate for reweighted mogamulizumab vs. synthetic control from HES was 0.33 (CI: 0.21, 0.50).

Real World Evidence from the HES database can be used to validate crossover adjustment methods and to better reflect current clinical practice in the UK. Results using both methods support each other.