The Pharmaceutical Benefits Advisory Committee (PBAC) is an independent expert body that recommends new technologies for listing on the Pharmaceutical Benefits Scheme. Its decision-making process is evidence-based and considers a technology's clinical effectiveness, safety, and cost-effectiveness compared with other technologies. Since 2014, the PBAC has formally taken into account input from those impacted by the technology via an online consumer comments portal and has also reported on received comments in the Public Summary Documents (PSDs). Comments are welcomed from those whose health the technology is trying to improve, as well as carers, clinicians, and organizations. Our objective was to analyze and review consumer comments in the PBAC's decision-making process.

We extracted information about consumer comments from the PBAC PSDs from 2014–9. We conducted simple descriptive analyses.

Our findings reveal that two thirds of all submissions did not receive a single consumer comment. Of the remaining third, eight submissions (less than 1 percent) had a substantial number of consumer comments (>500). For these technologies, multiple submissions were required before a recommendation was issued. Submissions spanned multiple therapeutic areas, the therapeutic areas with the most consumer comments were genetic disease, pediatrics, and oncology.

In the light of our review, we have identified limitations to the current consumer comments process, and after an examination of the processes of other comparable health technology assessment agencies, we have identified a number of improvements that could be made to the PBAC's process to increase consumer engagement.

Written patient evidence is submitted to the National Institute for Health and Care Excellence (NICE) by patient organizations and their nominated patient experts. We reviewed the impact that patient evidence had on the committee's decision making. This local learning can help generate global opportunities for Health Technology Assessment (HTA) bodies and patient groups to further develop their understanding and methodology about how patient evidence can support HTA decision making for ultra-orphan conditions.

There were two phases.

Phase one was an online questionnaire about the impact of patient evidence on the committee's decision making for ultra-orphan HTA evaluations. It was sent to the committee chair, lay committee members and selected other committee members.

Phase 2 developed the initial questionnaire findings using in-depth interviews with the committee chair and the lay members. These gained further understanding of the impact of patient evidence and the themes raised.

Key findings showed patient evidence was helpful to understand the:

• • Burden of disease

• • Patient population

• • Likely uptake of new medicines

• • Impact on carers.

For ultra-orphan conditions, where other forms of evidence are scarcer, patient evidence is fundamental to understanding patient needs, the impact of the disease, patient population and preferences.

Patient evidence was useful for the committee in different ways; it provided the committee with new evidence and it helped the committee understand and interpret the evidence submitted by others. Both are key to committee decision making. It was clear that due to the very small patient population, patient groups knew the patient population, their stage of disease, and their preferences in detail.

The findings will be used to inform an updated patient submission template for ultra-orphan HTAs, and supporting guide. These will be available on the NICE website and offered to the HTAi Interest Group on Patient and Citizen Involvement so they can be shared globally.

Social engagement in health encompasses the idea of involving (parts of) society as full partners in the decision-making regarding both development and implementation of health technologies. Evidence shows that patient engagement is linked with fewer adverse events, better patient self-management, fewer diagnostic tests, decreased use of healthcare services and shorter lengths of stay in hospitals. Matching the escalating healthcare requirements to face the ongoing societal and economic challenges regarding access and coverage to (new) health technologies is not an easy task for health providers.

We conducted a systematic review (CRD42017068714) designed to address the institutional implementation of social engagement by the Brazilian Ministry of Health. All systematic reviews were evaluated using the new version of AMSTAR and, once all findings are synthesized, we will use the GRADE-CERQual approach to assess for confidence.

From 399 publications that met the inclusion criteria, 80 described the implementation of social engagement during the development and implementation of (new) health technologies at various levels (local, regional, national, supranational), countries and for different health technologies and social actors. The remaining 319 publications constitute case studies describing barriers and enablers to implementing social engagement in HTA and coverage decision-making processes. By mapping barriers and facilitators, we explored effectiveness and sustainability, further observing how citizen science-based strategies can ultimately reform health service delivery by innovating the social engagement in health technology development and implementation.

This systematic review addresses the know-do gap on social engagement in health technology development and implementation, from a global perspective, as a way of improving the Brazilian Ministry of Health's HTA activities and enabling a Brazilian strategy to reform health service delivery. Enabling social engagement as early as possible, during all the stages of the development cycle, grants a more effective and sustainable health care system.

The National Institute for Health and Care Excellence (NICE) may recommend temporary funding through managed access agreements (MAAs) for oncology drugs (via the Cancer Drugs Fund [CDF]) and highly specialized therapies for rare diseases. MAAs allow for the collection of evidence to address key areas of clinical uncertainty, while providing access of medicines to patients, prior to re-appraisal by NICE. Observational data and other real-world evidence (RWE) are crucial requirements for all MAAs and herein we examine the extent these data are being used to inform HTA decisions at re-appraisal.

Existing MAAs entered into between the National Health Service (NHS) England and manufacturers as of 30 October 2018 were identified; for drug:indication pairings with NICE re-appraisals, all information was reviewed and the key data extracted.

Of the twenty-two MAAs identified, only two drug:indication pairings have been subsequently re-appraised by NICE: BV(brentuximab vedotin):non-Hodgkin lymphoma (’recommended’) and pembrolizumab:relapsed or refractory classical Hodgkin lymphoma (’recommended’). Data from a retrospective questionnaire regarding the proportion of patients that received curative stem cell transplant (SCT) post-BV (from patients who received BV in the old CDF) were accepted to provide sufficient evidence on the post-BV SCT rate by NICE. Meanwhile, for pembrolizumab, long-term survival benefit was the key clinical uncertainty; the primary data collection source was updated phase III randomized controlled trial data. At re-appraisal no reference was made to the observational data component; more mature survival data reduced uncertainty over survival benefits and were sufficient to support a positive NICE recommendation.

Of the twenty-two MAAs to date, only two drugs have been re-appraised thus far, with both receiving positive NICE recommendations. Observational data were successfully used to address key clinical uncertainties regarding subsequent real-world treatment patterns for BV, but observational data were not referred to in the NICE recommendation for pembrolizumab. The re-appraisal of more drugs in the future will clarify the importance being placed on observational data collection requested by NICE for existing MAAs.

Health technology assessment (HTA) is scientifically incorporating the demand, effectiveness and expedience of adopting innovative technologies, supported by evidence-based medicine. The rapid development of innovations led to an accelerated need to comprehend their benefit and satisfaction through actual utilization. ‘Real World’ Experience (RWE) in action is a source for collecting information through practice that has not yet been formally published. The target is to identify challenges and barriers in adoption, considerations on significant investment and local adjustment. This study aimed to assess the benefit of RWE as a tool for gathering information understanding and evaluating new technologies as candidates for public funding.

Twelve new technologies were assessed by 36 technology-assessors from the Ministry of Health (MOH) and the hospital, and were ranked using two scales: (i) the contribution of using RWE to benefit the assessment mechanism, and (ii) the perceptual “value” of the technology.

Analyzing the contribution of RWE to HTA, 67 percent of the technologies reviewed were recommended for adoption. Low RWE ranking correlated with a decision to reject technology adoption. The most important “value” among all assessors on RWE was “innovation”, and among hospital assessors, the parameter “local adjustment” was cardinal. MOH assessors highlighted RWE to increase knowledge of regulators to encourage adoption. The aspect relating to economic burden received only medium ranking.

RWE provides an essential contribution to HTA throughout all stages in the life of the technology, especially at early phases. It is an important methodology to gather evidence from the ongoing experience of clinicians, increasing awareness towards promising innovations and amplifying the confidence of decision-making. In this new era of HTA, new concepts arise on the horizon: multi-dimensional evaluations. Expert opinion and RWE are paving the way towards better understanding of beneficial technologies, even in the absence of formal published evidence.

Co-production relates to patients and health professionals working in equal partnership with shared decision-making. Patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) are increasingly being used to involve patients and measure healthcare quality. We set out to develop a set of universal experience questions for use across Wales. These will be used in various settings, including the national electronic PROMs and PREMs platform, which is already collecting outcome data across Wales and has received over 7,000 responses to date.

Patient experience leads and clinical leads were invited to a workshop to discuss standardized PREMs collection in Wales, with all health boards and trusts represented. It was agreed that quantitative patient experience data collection, while limited, would be a pragmatic way to collect responses from a large cohort. It was agreed that a previously developed set of PREMs questions could be adapted for use in all healthcare settings. Patient focus groups reduced the number of questions to a shortlist of those considered most important by patients. Wording was improved and an additional question was added.

In partnership with stakeholders we developed and agreed on a set of universal PREMs questions. These have been added to the national electronic platform, with collection commencing imminently. This will allow patients accessing secondary care in Wales to provide PREMs and PROMs responses.

Development of a standardized set of PREMs has allowed us to initiate collection on a national basis. Addition of PREMs to the national electronic platform provides a unique means of collecting large volumes of data consistently, allowing us to benchmark across and within organizations. It will also allow experience teams to target improvement initiatives and identify good practice. Together with outcomes responses, the data will be used to measure experience of care in Wales.

A literature review concerning the economic evaluation of telemonitoring was requested by the authority in charge of health evaluation in France, in a context of deployment of remote patient monitoring and identification of its financing. Due to the heterogeneity of existing telemonitoring solutions, it was necessary to stratify the evaluation according to patient involvement. Three levels of patient involvement are considered: weak (automated monitoring), medium (monitoring supported by a professional), and strong (active remote participation).

We performed a scoping review to provide a comprehensive overview of different systems of telemonitoring and their reported cost-effectiveness.

Following PRISMA-ScR guidelines, a search was performed in four databases: PubMed, MEDLINE, EMBASE, and Cochrane Library between January 1, 2013 and May 19, 2020. Remote patient monitoring should include the combination of three elements: a connected device, an organizational solution for data analysis and alert management, and a system allowing personalized interactions, and three degrees of involvement.

We identified 61 eligible studies among the 489 records identified. Heart failure remains the pathology most represented in the studies selected (n = 24). The cost-utility analysis was chosen in a preponderant way (n = 41). Forty-four studies (72 percent) reported that the intervention was expected cost-effective. Heterogeneity has been observed in the remote monitoring solutions but all systems are reported cost-effective. The small number of long-term studies does not allow conclusions to be drawn on the transposability.

Remote patient monitoring is reported to be cost-effective whatever the system and patient involvement.

Hemophilia is a rare, inherited bleeding disorder affecting an estimated 400,000 people worldwide (1). Characterized by spontaneous bleeding and long-term, irreversible joint damage, persons with hemophilia are often limited in normal day-to-day activities, including work/school, and require comprehensive care at specialized treatment centers. With replacement therapies extending survival by decades and vastly improving quality of life (QoL), routine prophylaxis is considered the standard-of-care in developed countries. However, due to the cost of replacement factor, access to treatment remains a challenge, and increased scrutiny over funding has been augmented by growing demands on healthcare budgets (2). Thus, the hemophilia community shares a unified goal of objectively defining patient-centered value in hemophilia care.

Using a three-tiered outcomes hierarchy model initially described by Porter (3), an international, multidisciplinary panel of health economics outcomes researchers and hemophilia experts developed a value framework for decision makers to assess value of various healthcare interventions in hemophilia.

The three tiers for assessing value are: (i) Health status achieved/retained; (ii) Process of recovery; and (iii) Sustainability of health. Tier one measures survival, quality of life (QoL), and hemophilia-specific outcomes of bleeding frequency, musculoskeletal complications, and severe bleeds, as well as function/activity (that is, lifestyle impairment). Tier two measures time to initial treatment or recovery and time missed at education/work, as well as disutility of care (that is, inhibitor development, pathogen transmission/infections, orthopedic intervention, and venous access). Tier three measures avoidance of bleeds, maintenance of productive lives, and long-term health, while capturing long-term consequences of insufficient therapy or age-related complications. Applicability of the framework can be demonstrated in areas of healthcare delivery, treatment regimen, and innovation for new therapies.

This value framework represents an initial collaboration with stakeholders to define and organize an array of patient-centric outcomes of importance in hemophilia into a practical tool that can influence treatment and funding decisions in hemophilia care.

In 2020, the Swedish regions cemented a national managed-introduction project for medical devices, to ensure equal, cost-effective, and appropriate use. Health technology assessment (HTA) is an important component of the project. Swedish national health registers have world-class coverage and completeness but may lack information enabling adequate evaluation of medical devices. This study reviews the current situation and ongoing initiatives. Additionally, the potential for medical device health economics and outcomes research (HEOR) using Swedish registers is assessed.

A review of Swedish national health registers was undertaken, focusing on available data, and contextualized for the purpose of HEOR for medical devices. Additionally, the review included an evaluation of the Swedish reimbursement authority's (The Dental and Pharmaceutical Benefits Agency, TLV) ongoing initiatives to improve the potential to follow-up the impact of the technologies they assess and develop value-based pricing schemes.

Five registers were deemed the most relevant national health registers for device research. They include high-quality longitudinal data and are linkable on a per-patient basis. For devices, main limitations include limited data on specialized outpatient care, lags in updating certain registers, lack of laboratory data, and challenges in identifying the specific device used. Reports indicate that certain limitations are being addressed, including pilot-studies investigating the opportunity for automated reporting of data from regional systems, and app-collected patient-reported health data.

Swedish registers provide comprehensive sources for HEOR studies, but limitations related to the assessment of medical device impact remain. As is common with register data reporting grouped diagnoses and interventions, specific devices are not directly identifiable in the national health registers. For some devices, this might be addressable through linkage with other data-sources. Swedish authorities are undertaking several initiatives that will likely improve the potential for HTA and follow-up of medical devices using national health register data.

The fact that HTA is a value-laden process is recognized in the literature. This is one of the reasons for promoting a better integration of ethics in HTA processes. Although what is meant by value-judgment (VJ) and how it can be used in HTA is not clear for some authors; others have proposed the elicitation of implicit VJs, to make them more explicit, as one way for clarifying the role ethics may play in HTA. In order to clarify what a VJ is, a conceptual analysis is needed to distinguish it from a factual-judgment and see how they diverge on certain aspects and converge on others.

The distinction between VJs and factual-judgments was debated in the fifties. At the core of the philosophy of language was a distinction between factual-scientific assertions about facts, considered objective, and VJs on what is right/wrong-good/bad, considered subjective. In speech-act theory these distinctions were treated as two different operations: assertive and evaluative. A conceptual analysis of VJs, considering them as specific speech-acts, was used for clarifying/deciphering the role of VJs in HTA.

VJs are intrinsically embedded in decision-making since they are the reasons justifying decisions. This is why implicit VJs can be identified at every decision-step in the HTA process. Assessment is usually considered objective while appraisal seems subjective. Since VJs are entrenched in the decisions taken throughout the assessment process, the results are not completely objective. Ethical analysis also distinguishes two types of VJs, those based on normative criteria and those based on various degrees of value actualization. Furthermore, since evaluation requires criteria based on a rational process, VJs are not totally subjective.

Elicitation of VJs in HTA is one way of integrating ethics in HTA and offers decision-makers a more thorough picture of the ethical issues involved in their decision.

Uveitis is inflammation inside the eye whose underlying cause may be infectious or non-infectious. The objective of our study was to assess the cost-effectiveness of the dexamethasone implant and adalimumab compared with current practice (immunosuppressants and systemic corticosteroids) in patients with non-infectious intermediate, posterior or pan-uveitis.

A Markov model was built to estimate costs and benefits of the interventions. Systematic reviews were performed to identify the relevant evidence. Quality of life data collected in three key randomized-controlled trials (1-3) was used to estimate the interventions effectiveness compared with the trials comparator arms, which consisted of placebo plus limited current practice (LCP). An indirect treatment comparison between adalimumab and dexamethasone was considered inappropriate due to lack of necessary evidence. For adalimumab, patients with active and inactive uveitis were considered separately. Due to the short duration of the trials, the rate of blindness, an important complication of uveitis, was highly uncertain. Substantial exploratory analyses were therefore undertaken. The analysis was performed from the perspective of the National Health Service (NHS) and Personal Social Services (PSS). Costs were calculated based on standard United Kingdom sources.

The estimated incremental cost-effectiveness (ICER) of dexamethasone compared with LCP was GBP19,509 per quality-adjusted life year (QALY) gained. The estimated ICER of adalimumab compared with LCP was GBP94,523 and GBP317,547 per QALY in patients with active and inactive uveitis respectively. The factors with the largest impact upon the ICERs were the rate of blindness and the proportion of cases of blindness avoided by interventions.

Dexamethasone and adalimumab resulted in health gains, but at significant extra costs, especially adalimumab which is unlikely to be considered a cost-effective use of NHS resources. The results of the analysis are highly uncertain due to the limited availability of evidence on: the comparative effectiveness of dexamethasone, adalimumab and current practice; the effectiveness of treatments in avoiding blindness; and, the effectiveness of interventions in different subgroups.

Urothelial Bladder Cancer (UBC) is the ninth worldwide most common cancer. In Italy the prevalence of the disease is about 10 percent, representing the third most prevalent cancer with 180,775 cases in men and 42,757 cases in women. The increase in the incidence requires continuous surveillance and care, resulting in a significant burden on Italian Healthcare System, making any improvement to the strategy for diagnosing and treating this disease important to the medical and scientific community. The aim of this study was to evaluate the burden of UBC in the Italian context, collecting and measuring the total costs of the disease.

An economic analysis in the National Health Service perspective was carried out, evaluating in six centers direct costs in terms of outpatient, inpatient and emergency care, pharmaceuticals and follow up procedures and indirect costs in terms of productivity losses. Data were collected through aggregated form reports, focusing on patients with an existing diagnosis of UBC who were taken in charge in the last year. Statistical analysis was conducted in order to explore variations between centers.

Mean total annual cost per patients was EUR11,310, increasing for disease severity from EUR6,954 for superficial disease to EUR24,896 for metastatic stage. The analysis confirmed a proportional relation between disease severity and disability grade. The total burden of the disease considering all patients, including prevalence and incidence data coming from AIOM guidelines 2015, was EUR2,833,655,822, of which 15 percent is represented by estimated productivity losses.

Our analysis represents the first economic burden study of UBC in the Italian context as well as the first real life evidence of the current therapeutic algorithm. This study opens the possibility for further analysis on the indirect costs components that represent a great burden for the society, especially for the stages of the disease with high disability grade.

For the assessment of videorasterstereography compared with radiography for monitoring idiopathic scoliosis, new tools given by the INTEGRATE-HTA methodology were included within the frame set by the authority. In contrast to the usual linear approach by covering the single domains' safety and efficacy, costs, and ethical, legal, socio-cultural and organizational issues (ELSOI) separately, the methodology is based on the assumption of interactions of different aspects of the domains. From the very beginning, these interactions are captured systematically using various tools and are repeatedly discussed with stakeholders.

Relevant databases were systematically searched for studies and question catalogues were processed in order to be able to search for specific aspects in orienting bibliographic research. An initial logic model was developed and successively expanded. A synoptic table showing multiple mapping of aspects to domains, a complexity checklist, and a semantic complex highlighting the socio-cultural impact of the disease were newly developed.

Four diagnostic studies with high risk of bias were included. For the clinical outcome, Cobb angle standard deviations up to 8.2 degrees and average measurement differences up to 8.8 degrees were determined. These differences in measurement accuracy correspond to those observed for purely radiographic measurements in other studies. In addition to freedom from radiation as the greatest advantage, there were clear arguments in favor of videorasterstereography when considering ELSOI, for example less harm.

Using INTEGRATE-HTA contributes to identify interactions between domains and to significantly expand the perspective on a technology. It improves patient-centered understanding and facilitates discussions.