The Philippine National Formulary (PNF) System preceded the health technology assessment (HTA) process in the Philippines, which was institutionalized in 2019. The transition led to previously prioritized topics of expert bodies overseeing the PNF System being endorsed to the HTA Council. However, the advent of COVID-19 forced the HTA Philippines to focus on emergency assessment needs and financing recommendations for the national government, resulting in limited capacity to assess non-public health emergency topics. To address this and improve patient access to medicines, we adopted the World Health Organization (WHO) process for evaluating and selecting medicines in the National Essential Medicines List (NEML).

In assessing the pre-pandemic topics, we matched the population, intervention, comparator, and outcomes of the WHO clinical evidence reviews with those scoped with relevant stakeholders and performed local costing analyses to ensure applicability of findings to the Philippine setting. When needed, we subjected the topics to price negotiation or conducted qualitative assessments.

We found the method efficient in expediting the decision-making process of the HTA Council. However, given the limited internal capacity of the HTA Philippines to conduct assessments for all ongoing HTA tracks, some of the topics responsive to Universal Health Care will be outsourced to the HTA Research Network, which is yet to be established. There is also a need to improve alignment among the topics being assessed, since the priorities of the proponents, national health program, and national payer have already evolved.

It is important to identify the priority areas for stakeholders as part of the topic nomination process, account for analytic capacity when setting the number of topics for HTA, establish mechanisms to allow proponents to conduct HTAs based on the HTA Council’s methodological standards, and proactively work with the national regulatory agency on horizon scanning and early HTA. We also recommend efficient monitoring, evaluation, and updating of the Philippine HTA guidelines so that they are more responsive to the needs of the healthcare system and the Filipino people.

Patients receiving endocrine therapy for hormone-sensitive cancers, such as men with prostate cancer (MPC) on hormone ablation therapy (HAT) and women with breast cancer (WBC) on adjuvant aromatase inhibitor therapy (AAIT), have an increased risk of developing osteoporosis. The aim of this study was to compare the safety and effectiveness of denosumab (Prolia®) with selective estrogen receptor modulators (SERMs) (raloxifene and bazedoxifene), bisphosphonates (zoledronate, ibandronate, alendronate, and risedronate), and placebo for the treatment of osteoporosis in patients receiving endocrine therapy for hormone-sensitive cancer.

Systematic literature searches were conducted in three biomedical databases (PubMed, the Cochrane Library, and Embase) to identify randomized controlled trials (RCTs). Only RCTs that investigated MPC on HAT or WBC on AAIT allocated to denosumab, SERMs, bisphosphonates, or placebo were included. RCTs were appraised using the Cochrane Risk of Bias 2.0 tool. Frequentist network and pairwise meta-analyses were performed on predetermined outcomes of vertebral or nonvertebral fractures, treatment-related adverse events (AEs), bone mineral density (BMD), mortality, withdrawal due to treatment-related AEs, and serious AEs.

A total of 14 RCTs (15 publications, 6,463 participants) were included. Relative to placebo, denosumab was found to prevent vertebral fractures in cancer patients receiving endocrine therapy. Moreover, denosumab, alendronate, and zoledronate increased femoral neck (FN) and lumbar spine (LS) BMD in MPC on HAT, compared with placebo, whereas denosumab, risedronate, and ibandronate improved LS and total hip BMD in WBC on AAIT. Similarly, denosumab and risedronate increased trochanteric BMD in WBC on AAIT, compared with placebo. In WBC on AAIT, only denosumab increased FN BMD relative to placebo.

Denosumab was more effective than placebo in preventing vertebral fractures and improving BMD at the LS and FN in MPC on HAT, and in preventing vertebral fractures and improving FN, trochanteric, total hip, and LS BMD in WBC on AAIT. From a policy perspective, the continued reimbursement of denosumab needs to be reviewed.

In Australia, cancer codependent technologies (cCDTs) mostly comprise a biomarker targeting medicine and a companion diagnostic test (CDx). Health technology assessment (HTA) of cCDTs is carried out to inform funding deliberations on CDxs by the Medical Services Advisory Committee (MSAC) and on personalized medicine by the Pharmaceutical Benefits Advisory Committee (PBAC). To understand the strengths and weaknesses of this dual assessment mechanism, we studied the journey of cCDTs in getting funding support from the two committees since the introduction of the codependent technology evaluation framework.

Public summary documents summarizing deliberations by each committee were reviewed from 2012 to 2022. Information was retrieved on the patient indication, date, biomarkers related to the tests, and PBAC or MSAC funding outcomes. The alignment of HTA decisions, time taken until dual funding approval (if approved), and the reasons for discrepant and negative decision-making were determined.

From 2012 to 2022, a total of 26 cCDT applications were submitted to PBAC and MSAC, corresponding with 43 paired PBAC/MSAC considerations and 11 single committee considerations. Non-small cell lung cancer and programmed cell death ligand 1 were the most frequently nominated cancer and biomarker test, respectively. When a cCDT was submitted in the same decision round to both committees, 60 percent of funding decisions were aligned, reaching 73 percent when the considerations were made separately (resubmissions). Only 9 percent of considerations received polarized, where one committee supported and the other committee rejected funding. After multiple resubmissions, 73 percent of cCDTs obtained dual funding support after an average of 34.8 weeks, with considerations by PBAC and MSAC occurring an average of 2.3 and 1.9 times, respectively.

Most cCDTs obtain funding support, but only after multiple resubmissions to PBAC and MSAC. Polarized decisions are rare. Reasons for rejection primarily relate to uncertain clinical benefit and an unacceptably high incremental cost-effectiveness ratio.

Interest in early detection of complications in hospitals has increased recently. Complications after elective or urgent surgery are frequent and are associated with higher mortality rates, longer hospital stays, and more resource utilization. The ZERO project implemented an educational nursing program and developed an innovative algorithm that assesses a patient’s complication risk based on clinical parameters to prevent complications and reduce hospital burden. Our aim was to present the results from one year of implementing ZERO at the Clinic Barcelona University Hospital.

A comparative effectiveness and cost study was conducted. Data from patients admitted after elective or urgent surgery were collected for one year retrospectively (n=8,844 from January 2019 to December 2019) and prospectively (ZERO) (n=8,163 from October 2021 to October 2022). Effectiveness was measured in terms of mortality, complications, and life-years gained (LYG). Length of stay (LoS) at conventional, intermediate, and intensive care units and rates of readmissions were collected for resource use. The chi-square test was used to compare categorical variables. The t-test and Wilcoxon test were used for normally and non-normally distributed continuous variables, respectively.

There was a significant decrease in the rate of complications (7.8%, 95% confidence interval [CI]: -8.46, -7.19; p<0.001) with ZERO. Moreover, there were statistically significant reductions in mean LoS for readmissions to conventional wards (-5.04 days, 95%CI: -9.9, -0.18; p=0.04) and to the intensive care ward within the same episode (-4.68 days, 95%CI: -9.26, -0.14; p=0.02). The mean cost per patient was EUR2,772.92 and EUR2,591.57 before and after ZERO implementation, respectively. After accounting for the cost of implementing ZERO, there was a cost saving of EUR147.76 per patient (p=0.048), which yielded a yearly impact of EUR1,206,165 for the hospital budget.

This one-year analysis of the effect of ZERO on surgical patients shows that it decreases complication rates and all types of LoS, leading to overall cost savings for the hospital.

With the rapid development of innovative health technologies, evidence increasingly shows that overdiagnosis is harmful to a person’s health and that it is a global public health issue. This study aimed to analyze the current research status and corresponding foci in the field of overdiagnosis in Chinese and English databases using bibliometric methods.

A search was conducted in the English Web of Science Core Collection database and the Chinese China National Knowledge Infrastructure database for literature published from inception to 31 December 2021. CiteSpace (version 5.8 R1) software was used to perform bibliometric analysis on the countries, institutions, and keyword clusters of the included literature on overdiagnosis and to draw a corresponding visual knowledge map.

A total of 2,841 English and 43 Chinese publications were included. There was an increasing trend in the annual publication volume of both Chinese and English literature, with the publication volume of English research increasing significantly since 2010. In terms of countries and institutions, the top ten in English research on overdiagnosis were all from high income countries. The cooperation among these countries and institutions was close, unlike in China where the cooperation was relatively limited. Analysis of keyword clustering showed that the potential research foci for English literature on overdiagnosis included breast cancer, thyroid cancer, prostate cancer, lung cancer, and other tumor types, whereas the clustering in Chinese records was relatively scattered and mainly focused on overdiagnosis of thyroid cancer.

The research topics in the Chinese literature on overdiagnosis lag significantly behind English research. It is suggested that more research on overdiagnosis and related fields should be actively promoted and conducted in China in the future.

Adult vaccination with 13-valent pneumococcal conjugate vaccine is currently recommended in the UK only for very high-risk individuals, with 23-valent pneumococcal polysaccharide vaccine (PPV23) being recommended to all adults 65 years or older and those 18 years or older with specified risk conditions. A 20-valent pneumococcal conjugate vaccine (PCV20) has recently become available for use in adults with potential to address a substantial proportion of the current adult pneumococcal disease burden in the UK. We evaluated the cost-effectiveness of PCV20 vaccination compared with PPV23 in adults in England currently eligible for pneumococcal vaccination.

A probabilistic model with a Markov-type process was used to depict lifetime risks and costs of pneumococcal disease among a cohort of English adults. Epidemiologic parameters, serotype coverage, costs, vaccine effectiveness and coverage were based on published literature or publicly available data. The National Health Service perspective was adopted, health effects were expressed in quality-adjusted life years (QALYs), and future costs and QALYs were discounted at 3.5 percent.

Results suggest that under reasonable assumptions concerning disease burden, vaccine, effectiveness, and vaccine cost, PCV20 implementation of an age-and risk-based strategy targeting all adults aged 65 years or older and younger risk group adults aged 18 to 64 years would reduce a large number of pneumococcal disease hospitalizations and pneumococcal-related deaths compared to currently recommended PPV23.

The incremental cost-effectiveness ratio was well below the current willingness-to-pay range of GBP20,000-GBP30,000 per QALY gained, with PCV20 being cost saving compared with PPV23 in base case and most sensitivity analyses. Probabilistic sensitivity analysis suggests high certainty in recommending PCV20 for vaccination of adults aged 18 to 64 years in risk groups and all aged 65 years or older instead of PPV23.

Our findings support replacing PPV23 with PCV20 to directly protect adults against pneumococcal disease, reducing hospitalizations and saving lives in the UK.

The addition of gene expression profiles (GEP) to the current clinicopathological diagnosis of aggressive large B cell lymphomas may lead to the reclassification of patients, treatment changes and improved outcomes. A GEP test is in development using TempoSeq technology to distinguish Burkitt Lymphoma (BL) and Primary Mediastinal Large B Cell lymphoma (PMBCL) from Diffuse Large B Cell Lymphoma (DLBCL). This study aims to inform developers about the potential impact of the test on costs and health outcomes, and pricing and evidence generation strategies.

Decision models compared current diagnosis with current plus GEP signatures over a lifetime horizon using a UK health and social care perspective. Inputs were taken from the literature and based on assumptions. Threshold estimates were made of the maximum price of the test and impact of incorrect disease classification using a threshold of GDP30,000 (USD37,155) per Quality Adjusted Life year (QALY). One way sensitivity analysis was conducted.

At base case values the BL signature delivers incremental QALYs of 0.0249 at an additional cost per patient of GBP508 (USD629). This results in a net monetary benefit (NMB) of GBP239 (USD296). The PMBCL signature delivers 0.0011 QALYs, a cost saving of GBP202 (USD250) and an NMB of GBP236 (USD292). The maximum threshold price for a combined test to be cost effective is GBP776 (USD961) (base case GBP400 (USD495)). Results are sensitive to cost differences in first line treatments and impact of false diagnoses.

A combined test could be cost-effective in a UK context at a price around GBP750 (USD929). The developers can use this estimate to inform return on investment calculations. The number of patients who were reclassified as a result of the addition of GEP in our model was taken from small retrospective studies and the impact of false diagnoses was based on limited evidence. If the developers choose to proceed with the development, these aspects should be incorporated in evidence generation strategies.

The recent release of powerful next-generation sequencing platforms, which can provide whole exome sequencing (WES) or whole genome sequencing (WGS) in quicker timelines and at reduced costs, has resulted in proposals for these diagnostic testing methods to be routinely integrated into clinical practice in multiple settings. However, the complexities of these diagnostic approaches, and the minimal comparative evidence available on them, creates difficulties in the evaluation of their diagnostic performance. Novel approaches need to be developed to improve the health technology assessment (HTA) of WES and WGS.

Several HTAs on genetic testing and the use of WES or WGS in fetal medicine were reviewed. Information on factors associated with this diagnostic modality that affect typical test accuracy assessment (e.g., sensitivity and specificity) was extracted. The multiple steps required for completing a WES or WGS test, and the potential for the introduction of errors (type I or type II) at each of these steps, were mapped and examples provided. The clinical and economic implications associated with imperfect and uncertain test accuracy were described.

Limited data on analytical and clinical validity were identified. WES and WGS are multistep processes and errors were found in sampling, molecular sequencing, bioinformatic filtering, and variant interpretation; therefore, the assumption that WES or WGS is 100 percent sensitive or specific is not reasonable. Although alternative evidence-based estimates are unlikely to be available, the inevitability of such errors, and their implications in terms of comparative effectiveness, safety, and cost effectiveness, should be described in HTAs.

While unknown diagnostic accuracy remains an issue with WES and WGS testing, formal sensitivity analysis of test performance characteristics should be conducted as part of HTAs. A checklist has been developed to assist those involved in HTA and policy to understand the potential for inaccurate test results in clinical practice, and the risk-benefit implications of these diagnostic errors for patients.

The World Health Organization recommends the human papillomavirus (HPV) test for cervical cancer screening. HPV partial genotyping (PGT) identifies HPV16 and HPV18 individually and the 12 other high-risk HPV genotypes (hrHPV) collectively. In contrast, HPV extended genotyping (XGT) identifies six hrHPV individually (HPV16,18,31,45,51, and 52) and the other eight in three groups (HPV33/58, HPV56/59/66, and HPV35/39/68). XGT allows better risk stratification for patient management and monitoring of persistent same-genotype infections (PSGI), which convey a higher risk for cervical cancer. This study compared the cost, quality-adjusted life-years (QALYs), and resource use of XGT with PGT when used as the primary cervical cancer screening method in Singapore.

A discretely integrated condition event simulation was developed for screening 500,122 women aged 30 to 69 years over five years from the health system perspective, using a three percent annual discount. For XGT, women with HPV35/39/51/56/59/66/68 and reflex cytology of atypical squamous cells of undetermined significance were recalled for a repeat screening in one year, instead of the immediate colposcopy referral that occurs with PGT. At repeat screening, colposcopy was only provided for women with PSGI on XGT. Published data from Singapore were used for inputs and supplemented with data from international literature. Deterministic and probabilistic uncertainty analyses were conducted. Scenario analysis was conducted to simulate various HPV burdens among women. XGT was cost effective when the incremental cost-effectiveness ratio (ICER) relative to PGT was below SGD100,000 (USD118,906) (gross domestic product per capita in 2021).

XGT was cost effective relative to PGT (ICER SGD16,370 [USD19,465]), with fewer colposcopies (n=7,130; 19%), liquid-based cytology tests (n=6,027; 7%), and clinic consultations (n=9,787; 2%) but more HPV tests (n=2,446; 0.5%). The ICER was most sensitive to the relative cost of XGT and the cost of PGT. XGT remained cost effective in one-way sensitivity analysis in all 1,000 probabilistic uncertainty simulations and in scenario analysis with various HPV burdens.

XGT can provide a cost effective, risk-based approach to primary cervical cancer screening, with lower resource utilization than PGT.

Positron emission tomography combined with computed tomography (PET/CT) using 18F-sodium fluoride (18F-NaF) is used for functional imaging in diseases to detect abnormally altered osteogenic activity, such as benign and malignant bone diseases and inflammatory or traumatic changes in skeletal bones.

A systematic search of literature using keywords in the MEDLINE database was conducted to identify literature on the clinical and cost effectiveness of using PET/CT with 18F-NaF-based radiopharmaceuticals in the diagnosis of bone and cartilage cancer. The search retrieved 323 publications. The analysis included 11 publications that met the selection criteria, including one meta-analysis and ten literature reviews.

The pooled sensitivity, specificity, diagnostic odds ratio (DOR) and area under the receiver operating characteristic curve of 18F-NaF-based PET/CT for the detection of bone metastases were 0.98 (95% confidence interval [CI]: 0.95, 0.99), 0.90 (95% CI: 0.86, 0.93), 123.2, and 0.97, respectively. 18F-NaF-based PET/CT was highly effective in detecting bone metastases during staging and restaging of patients with high-risk prostate cancer. The effectiveness of 18F-NaF-based PET/CT was superior to bone scintigraphy with technetium-99m and single-photon emission computed tomography (SPECT) and was comparable to diffusion-weighted imaging.

PET/CT with 18F-NaF is a more accurate method of localizing and characterizing malignant bone lesions than SPECT. This method has improved clinical accuracy and provides greater convenience for patients and referring physicians. PET/CT with 18F-NaF in malignant neoplasms is a more specific, sensitive study than 18F-fluorodeoxyglucose PET/CT. These results were submitted to the Ministry of Health for a decision on the inclusion of 18F-NaF-based PET/CT in the state reimbursement system.

Leg ulcers are usually treated with compression therapy unless there is evidence of peripheral arterial disease (PAD). Compression may lead to vascular complications in people with PAD. Timely diagnosis and appropriate treatment are important to ensure the best patient outcomes. PAD is usually assessed using a manual ankle brachial pressure index (ABPI). Automated devices may reduce time to diagnosis and treatment of leg ulcers. This study investigated the diagnostic accuracy, clinical effectiveness, and cost effectiveness of automated ABPI measurement, compared with manual doppler testing, to detect PAD in people with leg ulcers.

We conducted a systematic review of studies of any design assessing automated devices in any population. Meta-analyses were conducted where possible. A decision tree and Markov model was used to capture lifetime costs, quality-adjusted life-years (QALYs), and cost effectiveness from a UK National Health Service perspective. Sensitivity analyses captured the uncertainty surrounding model assumptions and probabilistic sensitivity analyses described parameter uncertainty. Value of information analysis was conducted to identify future research priorities.

A total of 22 studies provided diagnostic accuracy data for five automated devices, but there were no studies in people with leg ulcers. Meta-analysis of 12 studies demonstrated a pooled sensitivity of 64 percent (95% confidence interval [CI]: 57, 71) and a specificity of 96 percent (95% CI: 92, 98) for detecting PAD. Automated devices were cheaper to complete due to shorter test times, but the increased risk of inappropriate or delayed treatment due to inaccurate test results offset the cost savings and reduced the QALYs for automated devices.

Given the current limited evidence base, automated devices would only be cost effective if they can demonstrate substantial reductions in time to diagnosis in clinical practice. Value of information analysis identified the following research priorities: (i) to determine diagnostic accuracy in a population with leg ulcers; (ii) to determine the impact of test results on ulcer healing times, and (iii) to determine the risk of providing inappropriate treatment based on inaccurate test results.

The cost effectiveness of treating Bell’s palsy with prednisolone in children is unknown. This study aimed to assess the cost effectiveness of prednisolone, compared with placebo, in treating Bell’s Palsy in children from a healthcare sector perspective.

This economic evaluation was a prospectively planned secondary analysis of a triple-blind randomized superiority trial conducted from 2015 to 2020 that compared prednisolone with placebo. The time horizon was six months after randomization. The 180 participants were aged from six months to 17 years and presented within 72 hours of onset of clinician diagnosed Bell’s palsy. Interventions were oral prednisolone (1 mg per kg daily) or taste-matched placebo administered for ten days. Incremental cost-effectiveness ratios comparing prednisolone with placebo were estimated. Costs included medication costs, doctor visits, and medical tests over the six-month study period. Effectiveness was measured using quality-adjusted life-years (QALYs) derived from the Child Health Utility 9D instrument. Nonparametric bootstrapping was performed to capture uncertainties. Prespecified subgroup analyses by age (12 to 17 years versus <12 years) were performed.

The mean cost per patient was USD188 in the prednisolone group and USD121 in the placebo group over the six-month period (difference USD66, 95% confidence interval [CI]: 47, 179). The mean QALYs gained over six months were 0.45 in the prednisolone group and 0.44 in the placebo group (difference 0.01, 95%CI: -0.01, 0.03). Prednisolone was very likely cost effective given a conventional willingness-to-pay threshold of USD 50,000 per QALY gained (the cost per additional QALY gained was USD6,625 using prednisolone compared with placebo). Subgroup analysis suggested that this was primarily driven by the high probability of prednisolone being cost effective in children aged 12 to 17 years (98%), compared with those younger than 12 years (51%).

This study provides new evidence to stakeholders and policy makers who are considering whether to make prednisolone available for treating Bell’s palsy in children aged 12 to 17 years.

The number of patient preference studies in health has increased dramatically. There is growing use of patient preferences in a wide variety of contexts, including health technology assessment. Patient preference studies can help inform decision makers on the needs and priorities of patients and the tradeoffs they are willing to make about health technologies.

This International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force included international experts, health preference researchers and others from diverse backgrounds, including regulatory, health technology assessment, medicine, patient advocacy, and the pharmaceutical industry. The report underwent two rounds of written reviews by ISPOR Preferences Special Interest Group members until a final consensus was reached. The Task Force focused on developing a roadmap that would: (i) apply to the wide variety of preference methods, (ii) identify key domains to guide researchers and other stakeholders in making patient preference studies more useful to decision makers, and (iii) detail important questions to guide researchers conducting preference studies and those critically appraising them.

This Task Force report provides a novel roadmap that invites patient-preference researchers to work with decision makers, patients and other stakeholders to do even more to ensure that studies are useful and impactful. The ISPOR Roadmap consists of five key elements: (i) Context; (ii) Purpose; (iii) Population; (iv) Method; and (v) Impact. In this report, we define these five elements and provide good practices on how patient-preference researchers can actively contribute to increasing the usefulness and impact of patient preference studies in decision-making. We also present a set of key questions that can support researchers and other stakeholders in assessing efforts that promote preference studies’ intended and unintended impact.

This roadmap can help increase the usefulness and impact of patient preference studies in decision-making by challenging researchers to engage and partner with decision makers, patients and others, and together consider the intended and unintended impacts of patient preference studies on decision-making while actively fostering positive impact.

To facilitate communication of clinical study results among patients, clinicians, and payers, this post-hoc analysis examined the association between achieving the Health Assessment Questionnaire-Disability Index (HAQ-DI) clinical meaningful improvement (CMI) and normalization of individual daily activities using pooled clinical trial data of patients with moderate-severe psoriatic arthritis (PsA).

Patients with active PsA (n=1,120) who participated in two Phase III trials were administered the HAQ-DI at baseline and weeks (W) 24 and 52. The HAQ-DI is a patient-reported instrument measuring functional status that is validated for rheumatological diseases including PsA. It contains 20-items assessing activities of daily living using a 0-3 Likert scale [0=able to perform activity without difficulty (normalization) through to 3=unable to perform activity]; 0.35 or more improvement in total HAQ-DI represents the CMI in PsA patients. Study participants with baseline item-specific scores more than zero (indicating some level of impairment) who experienced both 0.35 or more improvement in total HAQ-DI and item-specific score equal to zero, were compared over time.

The proportion of patients with compromised ability to perform individual activities at baseline ranged from 42.3 percent (Difficulty Turning Faucet) to 84.1 percent (Difficulty Doing Chores). Fourteen of 20 activities with 60 percent or more of patients reporting impairment at baseline decreased to one of 20 activities in patients reaching the CMI at W24. Fourteen of 20 activities with 50 percent or more of patients reporting no impairment at W24 increased to 18 of 20 activities at W52. Notably, the proportion of patients reporting impairment for the two outlier activities at W52 (Reach-Get Down 5lb Item/Doing Chores) decreased during the follow-up period by 28 percent and 30 percent, respectively, despite presenting as very challenging at baseline.

PsA patients achieving a clinically meaningful change in total HAQ-DI over time were more likely to achieve normalization of individual daily activities. Translating changes in patient-reported outcome scores to specific symptom or functional improvements are meaningful to help patients understand clinical trial results, as well as to communicate with payers the value of accessing treatment for specific health conditions.

Within Australia and New Zealand (ANZ) there is limited evidence regarding the experience and satisfaction across the healthcare system of people living with multiple myeloma (MM). We aimed to quantify the patient experience across the healthcare system to help identify potential areas of the healthcare pathway that could be targeted for improvement to maximize patient satisfaction.

A 30- to 40-minute online survey was completed by adults in ANZ diagnosed with MM. Anchored best-worst scaling (ABWS) is a technique that takes advantage of an individual’s ability to reliably identify extremes (‘best’ and ‘worst’) in sets of items, eliciting discriminating rankings free of scale bias. This study implemented a novel anchoring process to rescale importance and satisfaction best-worst scores for factors across the MM healthcare pathway, which could be compared and combined to form a patient experience index (PEI). There were 15 factors or ‘moments that matter’ (MTM), each describing a different aspect of the patient journey, such as time to diagnosis, treatment logistics, and side effects. The MTM were derived from qualitative research with patients as well as a workshop with key opinion leaders. Additional survey questions were included to help identify potential ways to improve patient satisfaction.

The results were based on 62 patients with MM. The overall median PEI score was 63.1. The top three MTM that were most important to patients, but they were least satisfied with (calculated by combining the top four of each most important/least satisfied factor for each participant), were side effects of medication, effectiveness of medication, and medication access.

The findings from this research contribute to the understanding of patient experiences of treatment and care for MM. The results can inform healthcare decisions for prioritizing interventions that align with patient experiences. In the future, the study could be executed longitudinally to assess shifts in satisfaction within the MM healthcare journey, which would be especially worthwhile if new programs are implemented to improve patient satisfaction.

There are more than 7,000 rare diseases (RDs), which are individually rare but have a large collective impact on patients and families, the health system, and society. There are few treatments for RD; where treatments do exist, they are often exceptionally expensive. Understanding the socioeconomic burden (SEB) of RD is crucial to properly valuing these treatments and informing health technology assessment. Our team has developed a framework of cost elements for inclusion in studies of the costs of RDs using an evidence-informed consensus-based approach.

We conducted a scoping review to identify published economic evaluations studies in RD, searching five electronic databases to identify English language RD studies published 2010-2021. We applied our framework of cost elements to assess studies regarding what cost elements were included.

Of 4,890 records identified, 48 studies were screened for inclusion. Most were from the US (n=27), UK (n=6), and Canada (n=6), and focused on hemophilia (n=14) or cystic fibrosis (n=11). Healthcare system and payer perspectives were most often reported (n=41), with only seven studies reporting a societal perspective. Cost elements most often included were medications (n=41), hospitalizations (n=35), surgery (n=20) medical tests (n=16), and outpatient (unspecified) visits (n=16). Costs to patients, families, and society were less commonly included: productivity (n=5), travel/accommodation (n=3), government benefits (n=2), family impacts (n=0), or other costs relevant to RD (n=1). While unsurprising, given that most analyses focused on healthcare or payer perspectives, this finding illustrates the extent to which the burden of RD is largely unstudied.

Our scoping review demonstrated that most studies are conducted from a healthcare system/payer perspective, and largely consider only medical costs. These studies undercount the hidden burden of rare disease borne by patients and families leading to a gap in our global understanding of the full impact of rare diseases on families. To properly account for the these and value emerging treatments for RD, patient- and family-borne costs must be considered in economic evaluations for health technology assessment.

People living with HIV/AIDS (PLWHA) frequently struggle to maintain optimal adherence to antiretrovirals (ARVs). Different adherence-improving interventions have been developed and examined through decision analytic model-based health technology assessments. Therefore, we aimed to conduct a systematic review of all decision analytic models developed to improve adherence in PLWHA.

The review protocol was registered on Prospero (CRD42022270039) and relevant studies published from inception to 23 October 2022 were identified through searches of the following databases: PubMed, Embase, the National Health Service Economic Evaluation Database, PsycINFO, the Health Economic Evaluations Database, and EconLit. Studies were included if they were modeling works of full economic evaluations, including cost-effectiveness analyses (CEA), cost-utility analyses, cost-benefit analyses, and cost-consequence analyses. The primary outcome was the cost effectiveness of adherence interventions reported as the incremental cost per additional quality-adjusted life-year (QALY). Study quality was assessed with the Quality of Health Economics Studies instrument. Due to the heterogeneity of the data, a permutation matrix was used for quantitative data synthesis rather than a meta-analysis.

The 15 studies identified were conducted in North America (8/15), Africa (4/15), and Europe (3/15). The time horizon was one year in one study, ten years in one study, 20 years in three studies, and a lifetime horizon in ten studies. The types of interventions were smartphone-based (5/15), nurse involved (2/15), directly observed therapy (2/15), case manager involved (1/15), simplification of regimens (1/15), Link4Health (1/15), and others (3/15) that involved multicomponent intervention. The interventions gained higher QALYs with cost savings in all 15 studies and gained QALYs at a higher cost at an acceptable incremental cost-effectiveness ratio in 80 percent (12/15) of studies. The studies were of fair (13%) to high quality (87%).

This study is the first systematic review of decision analytic model-based CEAs of adherence interventions in the management of PLWHA. Most of the identified studies recently published good quality cost-effectiveness analyses with an adequate timeframe.

It is important to create and maintain durable hemodialysis (HD) access in health systems to reduce morbidity and maintain overall cost control in patients with end-stage kidney disease (ESKD). To evaluate the choice of HD vascular access creation procedures and their related economic costs, we aimed to identify economic evaluations on vascular access (VA) creation procedures in patients with ESKD.

A systematic literature review was conducted using the Cochrane methodology to identify cost-effectiveness analyses (CEAs), budget impact analyses, and cost analyses of various HD access creation procedures. Eligible publications published from 2012 onwards were retrieved by searching PubMed, Embase, and the Cochrane Library. The Consolidated Health Economic Evaluation Reporting Standards 2022 checklist and ISPOR Task Force guidelines were used to appraise the quality of the economic evaluations and budget impact analyses, respectively. Costs were adjusted for inflation and purchasing power parity and standardized to US dollars.

A total of 40 economic evaluations met the inclusion criteria, including 28 cost analyses, three budget impact analyses, and nine CEAs. Widely evaluated procedures in the published literature were endovascular and surgical arteriovenous fistula (AVF), arteriovenous graft (AVG), and central venous catheterization (CVC). The results indicated that AVF was the most cost-effective strategy, followed by AVG, and CVC. Three studies showed that endovascular AVF was cost effective, compared with surgical AVF, and resulted in overall cost savings of about USD53 million dollars over a five-year period. Results of the quality assessment showed that budget impact analyses scored 63 percent, while the average score for economic evaluations was 58 percent.

It was challenging to identify a single effective method of managing vascular access due to the substantial heterogeneity among VA creation techniques. However, most of the included economic evaluations showed that AVF was a cost-effective method of VA creation relative to other identified techniques for patients with ESKD on HD.

Patients with hematological malignancies are likely to develop hypogammaglobulinemia (HGG) and subsequent infections. Immunoglobulin (Ig) replacement is commonly given to prevent infections, but the total costs and cost effectiveness of its use are unknown.

A systematic review was conducted following PRISMA guidelines to assess evidence on the costs and cost effectiveness of Ig replacement, administered intravenously (IVIg) or subcutaneously (SCIg), in adult patients with hematological malignancies. This review was registered with PROSPERO (CRD42022321908).

Six studies were included out of a total of 3,612 citations. A narrative synthesis was conducted because of the high level of heterogeneity across the included studies. Two economic evaluations were identified: one cost-utility analysis (CUA) of IVIg versus no Ig and one comparing IVIg with SCIg. The quality of the evidence was low, with most studies having small patient numbers and a high risk of bias. Compared with no treatment, Ig replacement reduced the hospitalization rate in patients with hematological malignancies.

One study reported no change in hospitalization rates following a program to reduce IVIg use, and an observational study comparing IVIg with SCIg found more hospitalizations with SCIg but lower total costs per patient. The CUA comparing IVIg with no IVIg suggested that IVIg treatment was not cost effective, but this study was published in 1991 and had significant limitations. The other CUA found that home-based SCIg was more cost effective than IVIg, but model inputs were derived from unpublished data in a very small patient cohort with HGG and different malignancies.

Our review highlights key gaps in the literature. The cost effectiveness of Ig replacement in patients with hematological malignancies is still very uncertain. Despite the increasing use of Ig replacement there are limited data regarding its direct and indirect costs, and its optimal use and implications for healthcare resources remain unclear. Given the paucity of data on the cost and cost effectiveness of Ig treatment in this population, further health economic research is warranted.

The National Institute for Health and Care Excellence (NICE) is piloting a new innovative approach to the way digital products, devices, and diagnostics that most reflect system need and demand are assessed. This early value assessment (EVA) approach will allow a more rapid assessment to enable patients to benefit from promising technologies sooner. Involving patients in the health technology assessment (HTA) lifecycle is a core principle at NICE, but established methods are not suitable for a rapid timeframe. NICE needs to adapt the approach to ensure that patients are supported to participate in EVAs and that their involvement is meaningful.

Due to the rapid timeframe, it was important to ensure patient contributors were not overloaded with information and that contact points were aligned. NICE reviewed the standard induction, support documents, and contact points to adapt the support provided. This included:

• • updating recruitment documents to communicate the role of the committee and the EVA process;

• • combining induction meetings between various NICE teams and providing recorded presentations;

• • organizing earlier peer support with experienced lay members; and

• • advising which of NICE’s nine online modules were most relevant.

Support for patient contributors has been an important part of the HTA process, so enabling people to prepare and confidently deliver content at a committee meeting is vital. There has been some variation in the processes for different topics, but the feedback received from patient contributors indicated that their involvement was meaningful and valued. This was attributed to their close working relationship with the project team. NICE is collecting feedback from all patient contributors using an online survey. The findings of this survey and the evaluation of the support mechanisms will be presented.

Despite shorter timeframes, patient involvement has not been compromised. NICE will use the feedback from patient contributors to review and adapt the induction process and support offered. This will support patient contributors and enable NICE to allocate appropriate resources in the shortened timeframe.