Telerehabilitation shows promise in many fields though strong evidence of benefit has been limited. We reviewed progress in the use of telerehabilitation for persons with chronic obstructive pulmonary disease (COPD). A challenge in caring for persons with this condition is the ability to achieve high levels of patient participation and compliance with rehabilitation processes.

Relevant publications were identified through literature searches from November 2009 to May 2018. We selected those that described studies of telerehabilitation in the management of COPD and reported clinical or administrative outcomes. Study quality was assessed using an approach that considers both study performance and study design. Judgments were made on whether the telerehabilitation application had been successful, if reported outcomes were clinically significant, and if further data were needed to establish the application as suitable for routine use.

Twenty-five publications, on 26 studies, were selected. Twelve were of high or good quality. In 11 studies the telemedicine application was successful. Nine studies had unsuccessful applications, and for six studies success was unclear. Further data before routine use would be required or desirable for all successful applications. In many studies there were difficulties associated with availability of skilled mentors, motivational support for patients and access to reliable remote monitoring and communication technology.

Various types of telerehabilitation are potentially helpful in the management of COPD. Availability and access to these technologies should improve. However, in management of this clinically challenging condition their use must be linked to suitable training and education of patients with COPD and timely support for them from healthcare professionals.

Our aim was to assess the value of nintedanib for non-idiopathic progressive fibrosing interstitial lung disease (non-IPF PF-ILD) and systemic sclerosis-associated ILD (SSc-ILD) in the Spanish context, using a multi-criteria decision analysis (MCDA).

Following an adaptation of the Evidence and Value: Impact on DEcision Making (EVIDEM) MCDA methodology, the estimated value of nintedanib was obtained by means of an additive linear model that combined individual weights (100-points distribution) of criteria with the individual scoring of nintedanib in each criterion for every indication, assigned by a multidisciplinary committee of twelve clinicians, patients, pharmacists, and decision-makers. To assess the reproducibility, an alternative weighting method was applied, as well as a re-test of weights and scores at a different moment of time.

The experts committee recognized nintedanib as an intervention with a positive value contribution in comparison to placebo for the treatment of non-IPF PF-ILD (0.50 ± 0.16, on a scale from −1 to 1) and SSc-ILD (0.40 ± 0.12), diseases which were considered as very severe and with high unmet needs. The drug was perceived as a treatment that provides an added therapeutic benefit for patients (0.06–0.07), given its proven clinical efficacy (0.05–0.06), slight improvements in patient-reported outcomes (0.01–0.02), and similar safety profile than placebo (−0.04–0.00), which will likely be positioned as a recommended therapy in the next clinical practice guidelines updates.

Under this increasingly used methodology, nintedanib has shown to provide a positive value estimate for non-IPF PF-ILD and SSc-ILD when compared to placebo in Spain.

We examined relationships between measures of total knee arthroplasty (TKA) “appropriateness” constructs and surgeon TKA recommendations in people with knee osteoarthritis (OA). Although TKA is highly effective, fifteen to thirty percent of recipients report dissatisfaction and/or little or no symptom improvement. More appropriate selection of surgical candidates may improve both patient outcomes and healthcare resource use, but no validated appropriateness criteria exist currently in Canada.

Patients 30 years of age or older with knee OA referred for surgical consultation at two large joint arthroplasty centres in Alberta, Canada were invited to participate. Participants completed a standardized pre-consult questionnaire, which included the following sociodemographics and validated measures of appropriateness constructs for TKA: knee symptoms; non-surgical management; patient readiness for and expectations of TKA; and net patient benefit. Post-consultation, surgeons were asked to confirm knee OA and their recommendation. We used multivariable logistic regression to examine the relationship between measures of appropriateness constructs and receipt of surgeon TKA recommendation.

Of 3,009 patients approached, 2,360 completed the questionnaire and 2,064 (sixty-nine percent) were eligible at surgical consultation (mean age 65.7 years, standard deviation 9.1; fifty-nine percent were women); 1,495 (seventy-two percent) were recommended for TKA. The likelihood of receiving a TKA recommendation was independently associated with: knee symptoms (odds ratio [OR] per unit increase in pain intensity, 1.19 (95% confidence interval [CI]: 1.11–1.27)); prior non-surgical OA management (OR for prior knee injection, 1.53 (95% CI: 1.21–1.94)); readiness for surgery (OR if definitely/probably willing to undergo TKA, 3.03 (95% CI: 1.99–4.59)); and TKA expectations (OR outcome “very important”: ability to perform daily activities, 1.40 (95% CI: 1.04–1.88); straighten the knee/leg 1.42 (95% CI: 1.13–1.80); participate in exercise/sports 0.75 (95% CI: 0.58–0.98)).

In our cohort of patients with confirmed knee OA who consulted a surgeon for TKA, appropriateness constructs were significantly associated with receipt of a TKA recommendation. Research is ongoing to evaluate the predictive validity of these measures for patient-reported outcomes associated with TKA.

The reimbursement of medicines by the Spanish National Health System (NHS) is based on a set of criteria included in the Royal Legislative Decree 1/2015 (RDL 1/2015). The Interministerial Committee on Pricing of Medicines and Healthcare Products (CIPM) is responsible for the final price and reimbursement (P&R) decision, including on its resolutions the criteria listed in the law by which the reimbursement of a drug is approved or denied. Nevertheless, the information behind its reasoning is not provided. The present study aims to characterize the P&R criteria of the RDL 1/2015 through criteria definitions from other countries to improve the P&R evaluation in Spain.

A multidisciplinary experts panel with relevant experience in drug evaluation and decision making at national, regional, and local level in Spain was selected for this study. A literature review to characterize the criteria listed in the RDL 1/2015 was performed based on the most relevant and recognized Health Technology Assessment (HTA) agencies in Europe, UK, and Canada. Eventually, a feasibility study was performed to evaluate the novel drug cefiderocol using the characterized criteria, including a reflective discussion of the results.

Consensus was reached among the multidisciplinary experts on the characterization of the criteria set by the law. The feasibility of their application to a new drug was exploratory, notwithstanding it showed the potential to improve the transparency as well as to offer a more structured rationale for the CIPM to evaluate the inclusion of new drugs in the Spanish NHS.

Precision medicines rely on companion diagnostics to identify patient subgroups eligible for receiving the pharmaceutical product. Until recently, the Belgian public health payer, RIZIV-INAMI, assessed precision medicines and companion diagnostics separately for reimbursement decisions. As both components are considered co-dependent technologies, their assessment should be conducted jointly from a health technology assessment (HTA) perspective. As of July 2019, a novel procedure was implemented accommodating for this joint assessment practice. The aim of this research was to formulate recommendations to improve the assessment in the novel procedure.

This study evaluated the precision medicine assessment reports of RIZIV-INAMI of the last 5 years under the former assessment procedure. The HTA framework for co-dependent technologies developed by Merlin et al. for the Australian healthcare system was used as a reference standard in this evaluation. Criteria were scored as either present or not present.

Thirteen assessment reports were evaluated. Varying scores between reports were obtained for the domain establishing the co-dependent relationship between diagnostic and pharmaceutical. Domains evaluating the clinical utility of the biomarker and the cost-effectiveness performed poorly, whereas the budget impact and the transfer of trial data to the local setting performed well.

Based on these results we recommend three amendments for the novel procedure. (i) The implementation of the linked evidence approach when direct evidence of clinical utility is not present, (ii) incorporation of a bias assessment tool, and (iii) further specify guidelines for submission and assessment to decrease the variability of reported evidence between assessment reports.

To investigate the behavior of restricted mean survival time (RMST) and designs of a two-state Markov microsimulation model through a 2 × 4 × 2 full factorial experiment.

By projecting patient-wise 15-year-post-trial survival, we estimated life-year-gained between an intervention and a control group using data from the Cardiovascular Outcomes for People Using Anticoagulation Strategies Study (COMPASS). Projections considered either in-trial events or post-trial medications. They were compared based on three factors: (i) choice of probability of death, (ii) lengths of cycle, and (iii) usage of half-a-cycle age correction. Three-way analysis of variance and post-hoc Tukey's Honest Significant Difference test compared means among factors.

When both in-trial events and post-trial study medications were considered, monthly, quarterly, or semiannually were not different from one other in projected life-year-gained. However, the annual one was different from the others: mean and 95 percent confidence interval 252.2 (190.5–313.9) days monthly, 251.8 (192.0–311.6) quarterly, 249.1 (189.7–308.5) semiannually, and 240.8 (178.5–303.1) annually. The other two factors also impacted life-year-gained: background probability (269.1 [260.3–277.9] days projected with REACH-based-probabilities, 227.7 [212.6–242.8] with a USA life table); half-a-cycle age correction (245.5 [199.0–292] with correction and 251.4 [209.1–293.7] without correction). When not considering post-trial medications, only the choice of probability of death appeared to impact life-year-gained.

For a large trial or cohort, to optimally project life-year-gained, one should consider using (i) annual projections, (ii) life table probabilities, (iii) in-trial events, and (iv) post-trial medication use.

Ovarian Cancer is usually diagnosed at an advanced stage. Extensive or ultra-radical surgery aims to improve the outcome by removing all visible tumour. National Institute for Health and Care Excellence UK 2013 Guidance expressed concern about its efficacy and safety, recommending research comparing complication rates, survival and quality of life with those following standard surgery. We present prospective observational data on quality of life and survival following surgery for advanced ovarian cancer. Innovative methods were used to collect patient reported outcomes and complex surgical information to compare outcomes of surgery of greater or lesser complexity used in routine practice.

A cohort study collected disease, surgical, complications, survival and quality of life data (validated instruments including EURO-QOL, EORTC-30 and OVA28) across a 2-year period in 12 United Kingdom sites and in parallel studies in Melbourne, Australia and Kolkata, India.

Two hundred and sixty patients undergoing cytoreductive surgery were recruited in 12 months. Centres varied in utilisation of complex surgical procedures. Excluding patients with inoperable disease, 125 patients underwent low, 70 intermediate and 63 high Surgical Complexity Score (SCS) procedures. Complete cytoreduction with < 1cm residual disease was achieved in 100/125 (80 percent) low, 65/70 (92 percent) intermediate, and 57/63 (90 percent) high SCS groups (p = 0.023). Compliance with 12 months questionnaires was 89%. All surgical groups had improved EORTC QLC 30 Global at 12 months compared with prior to operation, with overlapping 95% confidence intervals and no between group differences at 12 months. Complications, survival and quality of life adjusted for disease burden and surgical complexity over 2 years’ follow-up will be described.

Results will inform the update of NICE Interventional Procedures guidance recommendations on clinical governance arrangements for ovarian cancer surgery and enable clinicians and patients to better understand the outcomes of surgery, informing the consent process.

To better understand the process of hospital acquisition of innovative medical devices (MDs) and the hospital-based health technology assessment (HB-HTA) pathways in France, an in-depth study based on a quantitative approach is needed. The aim of the present study was to assess through a national survey how HB-HTA is currently implemented in French hospitals and to identify its level of formalization.

A quantitative online survey was conducted among hospitals performing HB-HTA in France, with a focus on the acquisition of innovative MDs for individual use. The survey, conducted between March and June 2022, was developed by a scientific board composed of members of the French-speaking Society for HB-HTA.

Sixty-seven out of 131 surveyed hospitals with HB-HTA activities responded, including 29 university hospitals, 24 nonprofit private hospitals, and 14 local hospitals. Sixty-one respondents (91 percent) reported the existence of a process dedicated to evaluating innovative MDs; of these, 16 declared that their hospitals had a formalized unit with HB-HTA activity. These units were more frequently found in larger hospitals with more than 500 inpatient beds (n = 16, p = 0.0160) and in university hospitals (n = 12, p = 0.0158). No hospital reported any collaboration with HAS, the French national HTA agency.

A diverse range of HB-HTA organizations with different structural levels exist in France for MD procurement linked to the category of hospitals. The study highlights the need for recognition of HB-HTA activity at the regulatory level in France and for direct collaboration between HTA activities performed at local and national levels.

While patient participation in individual health technology assessments (HTAs) has been frequently described in the literature, patient and citizen participation at the organizational level is less described and may be less understood and practiced in HTA bodies. We aimed to better understand its use by describing current practice.

To elicit descriptive case studies and insights we conducted semi-structured interviews and open-ended questionnaires with HTA body staff and patients and citizens participating at the organizational level in Belgium, France, Quebec, Scotland, and Wales.

We identified examples of organizational participation in managerial aspects: governance, defining patient involvement processes, evaluation processes and methods, and capacity building. Mechanisms included consultation, collaboration, and membership of standing (permanent) groups. These were sometimes combined. Participants were usually from umbrella patient organizations and patient associations, as well as individual patients and citizens.

Although the concept, participation at the organizational level, is not well-established, we observed a trend toward growth in each jurisdiction. Some goals were shared for this participation, but HTA bodies focused more on instrumental goals, especially improving participation in HTAs, while patients and citizens were more likely to offer democratic and developmental goals beyond improving participation processes.

Our findings provide rationales for organizational-level participation from the perspectives of HTA bodies and patients. The case studies provide insights into how to involve participants and who may be seen as legitimate participants. These findings may be useful to HTA bodies, the patient sector, and communities when devising an organizational-level participation framework.

Anxiety and depression are major drivers of health-related quality of life, adherence to therapy, and motivation to self-manage chronic conditions. A number of studies have shown rates of anxiety and depression to be higher among individuals with hemophilia compared to that of the general population (1). As the primary point of care for persons with hemophilia, hematologists are well-placed to assess the mental health state of their patients (2). The aim of this study is to explore physician- and patient-reported rates of anxiety and depression among a cohort with severe hemophilia.

Data were drawn from the Cost of Haemophilia across Europe – a Socioeconomic Survey (CHESS), a cost-of-illness study in severe hemophilia A and B across five European countries (France, Germany, Italy, Spain, and the UK) (3). Physicians provided clinical and sociodemographic information for 1,285 adult patients, 551 of whom completed corresponding questionnaires, including EuroQol EQ-5D-3L. We compared the self-reported EQ-5D with physician reports of anxiety and depression.

Across the five countries, physicians recorded a diagnosis of anxiety disorder in 189 patients (15 percent; range 4 percent-28 percent) and depression in 178 patients (14 percent; range 10 percent-28 percent). Seventy-three patients (6 percent) recorded comorbid anxiety and depression. Forty-six percent of patients with anxiety and 58 percent of patients with depression were receiving some form of treatment for their condition.

Within the EQ-5D measure, 42 percent of individuals recorded problems with anxiety or depression, with 6 percent of patients reporting “extreme” anxiety or depression. Twenty-two percent of individuals with a self-reported problem were not recorded with a corresponding diagnosis by their hematologist; 39 percent of patients reporting “extreme” anxiety or depression were absent of any physician-reported diagnosis.

Anxiety and depression are notable conditions within the hemophilia community, particularly so among those with severe condition. The mental health of individuals with hemophilia is an important aspect in ensuring therapy adherence and overall wellbeing and should be considered as part of a multidisciplinary approach to management of the condition.

The objective of the European Network for Health Technology Assessment (EUnetHTA) Joint Action 3 (JA3) was to develop a sustainable European model for future collaboration on HTA, by reducing duplication in HTA production and increasing patient access to health technologies. Compared to the previous JA2, several procedural changes were made aiming to increase usability, transparency, and inclusiveness of relative effectiveness assessments (REAs). This article presents and highlights these changes, explains their rationale as well as their influence on HTA production.

Feedback from REA teams and project managers was collected. At the end of JA3, all lessons learned were mapped, resulting in a set of recommendations for a future REA production process.

In JA3, forty-three EUnetHTA REAs have been produced. Efforts to increase the usability of the REAs were made by focussing on the needs of REA producers and users (HTA agencies) and by increasing stakeholder involvement. Huge steps were taken with regard to transparency, which was achieved through publication of guidances, templates, and up-to-date information on the EUnetHTA website. In an attempt to improve inclusiveness, (stakeholder) interaction and involvement as well as feedback procedures were enhanced and streamlined. The fine-tuned project management brought all aspects together and facilitated a consistent and reliable workflow.

Despite that HTA agencies have different national requirements, the procedural changes made in JA3 proved to counteract some of these challenges. Nevertheless, it is of utmost importance that further perceived methodological differences are being resolved to ensure a strong base for future European collaboration on REA production.

Mobile health (mHealth) is increasingly being seen as a promising solution to the multiple health system challenges that Vietnam faces, including inadequate health service provision. Nevertheless, there is limited published information describing the sustainability of such mHealth solutions in the Vietnamese context. This study reviewed the available projects and interventions to evaluate factors challenging sustainability of mHealth initiatives in Vietnam.

A multimodal scoping study was designed to collect data from various sources: published literature, government reports, unpublished literature, conference presentations, online documents, and key informant interviews. Relevant mHealth initiatives were identified and selected through electronic searches and informal discussions with key stakeholders. Collected data was charted and classified by thematic analysis. Challenges to the sustainability of mHealth initiatives were discussed in the context of the strengths, weaknesses, opportunities, and threats (SWOT).

Twenty mHealth initiatives with relevant information and available data were collected, covering the period from 2010 to 2017. Fifteen (75 percent) were primarily funded by external donors, one (5 percent) was government supported, and four (20 percent) were self-funded projects. Five (25 percent) were on-going, and fifteen (75 percent) were completed at the time of data collection. Four (20 percent) out of the completed initiatives were continuing to use materials, infrastructure, and technology to engage end-users.

The high percentage of external funding, complicated governmental bureaucracy and lack of government commitment, electronic medical record standardization, and legislation relating to mHealth are amongst the largest challenges to mHealth sustainability in Vietnam. In addition, findings demonstrate it is crucial for project managers of mHealth initiatives to build strong relationships with the Vietnam government and advocate for their mHealth initiatives in order to promote sustainability.