Collaborative strategies between the Pharmacy and Therapeutics Committee (Group 1) and hospital-based health technology assessment (HTA) nuclei (Group 2) using HTA tools can promote rational use and medicines access. The aim was to develop a logical model for the drug selection process in Brazilian university hospitals, considering the articulation between the Pharmacy and Therapeutics Committee and hospital-based HTA nuclei (NATS).

A qualitative study created and validated a logical model. The data collection considered documental analysis and semistructured interviews with key informants from both groups about the processes adopted by eight federal university hospitals. The European Project on Hospital-Based Health Technology Assessment (AdHopHTA) was selected as a theoretical reference. The model was discussed in a focal group with the participation of five experts in medicine selection tasks and HTA. The final version of the model was submitted for consistency and vulnerability analysis by the authors. The ethical approval number is 4.784.861.

The logical model was conceived with dimensions of resources, actions, products, results, and impacts. It has allowed a structured and organized view of the 15 actions in the medicine selection. From these 15 actions, seven can be articulated between both groups. The high management support, training, and dissemination of the procedures and flows that organize HTA processes were solutions to minimizing collaborative barriers. The selection of medicines was considered a dynamic process. Some actions, such as the use of pharmacovigilance data, post-selection monitoring, and horizon technology scanning can bring results in terms of treatment innovations and compliance with clinical guidelines.

Collaborative strategies were presented in a logical model with seven joint actions to guide the medicine selection process in hospitals involving tools, flow audits, governance, and human resources. The consistency and vulnerability model analysis defined indicators for HTA collaborative actions.

The Regulation (EU) 2021/2282 on health technology assessment (HTAR) for medicines will come into effect in January 2025; initially, new oncology medicines and advanced therapy medicinal products will be assessed at EU level. How will this work in practice? What does this mean for national HTA bodies, such as the National Centre for Pharmacoeconomics (NCPE) Ireland that uses a cost-effectiveness framework to inform decision-making for medicines?

Joint work to be conducted under the Regulation includes joint clinical assessments (JCA), joint scientific consultations (JSC), and production of procedures and methodological guidance. A review was undertaken of key areas that will be impacted by the HTAR in the Irish HTA process for medicines, including timing, evidence synthesis structure, capacity building, and resource implications.

The HTAR will alter the current process for medicines assessment in Ireland, from early scientific advice to cost-effectiveness assessment post-authorization. JSCs will represent an additional step. Significant training and capacity implications are associated with the JCA, which will require earlier engagement with stakeholders. The NCPE’s pragmatic HTA early triaging process, the “Rapid Review,” may be delayed due to the non-duplication clause in the HTAR. The availability of high-quality comparative effectiveness evidence may help avoid full HTAs in some cases. The benefit of the JCA will be realized if the results can directly inform treatment-effectiveness estimates in cost-effectiveness modeling.

The HTAR will significantly impact on medicines reimbursement procedures in Ireland. For the HTAR to be effective in achieving its aims, sufficient resources will need to be built into the EU HTA system. The balance between the extra resources needed and the resources spared will depend on the quality of the comparative effectiveness evidence available for the JCA.

Most studies on the impact of the COVID-19 pandemic on depression burden focused on the earlier pandemic phase specific to lockdowns, but the longer-term impact of the pandemic is less well studied. In this population-based cohort study with quasi-experimental design, we examined both the short-term and long-term impacts of COVID-19 on depression incidence and healthcare service use among patients with depression.

Using the territory-wide electronic medical records in Hong Kong, we identified patients with new diagnoses of depression from 2014 to 2022. An interrupted time-series (ITS) analysis examined changes in incidence of depression before and during the pandemic. We then divided patients into nine cohorts based on year of incidence and studied their initial and ongoing service use until December 2022. Generalized linear modeling compared the rates of healthcare service use in the year of diagnosis between patients newly diagnosed before and during the pandemic. A separate ITS analysis explored the pandemic impact on the ongoing service use among preexisting patients.

There was an immediate increase in depression incidence (RR=1.21; 95% CI: 1.10, 1.33; p<0.001) in the population since the pandemic with a nonsignificant slope change, suggesting a sustained effect until the end of 2022. Subgroup analysis showed that increases in incidence were significant among adults and the older population, but not adolescents. Depression patients newly diagnosed during the pandemic used 11 percent fewer resources than the prepandemic patients in the first diagnosis year. Preexisting depression patients also had an immediate decrease of 16 percent in overall all-cause service use since the pandemic, with a positive slope change indicating a gradual rebound.

During the COVID-19 pandemic, service provision for depression was suboptimal in the face of greater demand generated by the increasing depression incidence. Our findings indicate the need to improve mental health resource planning preparedness for future public health crises.

The growing use of real-world evidence (RWE) in pharmaceutical decision-making has prompted various guidelines, including REALISE (REAL World Data In ASia for HEalth Technology Assessment in Reimbursement). We compared RWE guidance from Asian, European, and North American health technology assessment (HTA) and regulatory bodies against the REALISE guidance.

Following a previous search for China/Japan guidelines in 2022, websites of the U.S. Food and Drug Administration (FDA), the National Institute for Health and Care Excellence (NICE), the European Medicines Agency (EMA), and the Canadian Agency for Drugs and Technologies in Health (CADTH) were searched for RWE guidance in May 2023. Sections from each guidance were mapped onto REALISE and categorized as “agree,” “mixed,” “disagree,” or “missing” based on coverage/consistency.

Eleven guidelines were identified: four from Japan, three from China, and one each from FDA, NICE, EMA, and CADTH. No disagreements were found (all mapped sections were tagged “agree”/”mixed”); divergences were in coverage only. Most regulatory guidance had narrower scopes: EMA covered registry-based studies, the FDA’s framework mainly referenced other documents, while Japan had guidance for database and registry data. Conversely, HTA guidelines (CADTH, NICE) were more comprehensive and provided specific recommendations on preferred methods (e.g., transparent reporting) that were “missing” (45 to 46%) from REALISE’s more conceptual discussions. Chinese regulatory guidance was the exception, with similar coverage as REALISE (77% “agree”/“mixed”).

Guidelines varied in scope, but there was overall concordance where recommendations could be mapped across documents. While regulatory bodies could focus on specific types of RWE, reflecting the specific role of RWE in regulatory evaluations (for example demonstrating safety), guidance for HTA was broader to account for different possible use cases in demonstrating comparative effectiveness and value.

Long-acting muscarinic antagonist (LAMA) monotherapy and LAMA in combination with a long-acting beta2 agonist are preferred over inhaled corticosteroid/long-acting beta2-agonist (ICS/LABA) therapy for Global Initiative for Chronic Obstructive Lung Disease (COPD) Group B and C patients. This study assesses impact of the subsidy decision of umeclidinium (umec) and umeclidinium/vilanterol (umec/vil) on Singapore’s healthcare system.

A retrospective cohort study was conducted using national health record databases. Maintenance-naive COPD patients, with no concurrent asthma, initiated on umec, umec/vil, or ICS/LABA from 2016 to 2020, were included. Patient demographics, comorbidities, and clinical characteristics were balanced using propensity score matching. Primary outcomes measured were the rate of severe or moderate COPD exacerbation and pneumonia hospitalization within one-year follow-up. Effect size was estimated using incidence rate ratio (IRR) with 95% confidence intervals (CIs) from Poisson regression. Markov model extrapolated the number of exacerbations and pneumonia hospitalizations avoided arising from the initiation of umec or umec/vil over ICS/LABA.

Patients initiated on umec (n=1,019) were less likely to experience severe (IRR 0.649; 95% CI: 0.438, 0.961) and moderate exacerbations (IRR 0.713; 95% CI: 0.569, 0.892) than ICS/LABA. Similarly, umec/vil-treated patients (n=1,206) had lower rates of severe (IRR 0.713; 95% CI: 0.517, 0.985) and moderate exacerbation (IRR 0.778; 95% CI: 0.642, 0.942). Both therapies were safer than ICS/LABA, with fewer pneumonia hospitalizations for umec (IRR 0.719; 95% CI: 0.532, 0.973) and umec/vil (IRR 0.781; 95% CI: 0.623, 0.980). Coupled with reduced drug cost from value-based pricing, subsidy potentially resulted in SGD53 million (USD39 million) cost savings over 10 years.

As the largest real-world study conducted among COPD patients in Singapore, our findings contribute to the limited real-world evidence in the region. Compared to ICS/LABA, umec or umec/vil were associated with better COPD control and reduced rates of pneumonia hospitalization. This confirms the importance of appropriate prescribing of COPD therapies and validates the subsidy decision.

Real-world evidence (RWE) is increasingly used in healthcare research to address evidence gaps, reduce uncertainty about medical technology benefits, and provide real-world insights. Efforts to integrate RWE in regulatory and health technology assessment (HTA) processes are growing. However, variations among countries pose challenges. The objective is to analyze and compare various (inter)national RWE guidelines, focusing on real-world hospital data utilization.

We conducted a review to identify RWE guidance published from 2016 to 2023, with a focus on the EU5 nations (UK, France, Germany, Italy, and Spain) and the ONCOVALUE consortium affiliates (Finland, the Netherlands, Denmark, Italy, and Portugal). To ensure a comprehensive overview, we also investigated Canada, the European Medicines Agency (EMA), ISPOR, and the European Society for Medical Oncology (ESMO). We conducted in-depth interviews with HTA experts of all included countries, focusing on real-world hospital data within the European HTA context. The interviews underwent thematic analysis related to the utilization of RWE in HTA.

We identified nine guidance reports: six focused on HTA-RWE (Medicinrådet/Denmark, NICE/UK, AQuAS/Spain, HAS/France, IQWiG/Germany, CADTH/Canada), one from EMA, and two international (ISPOR, ESMO). Only NICE, IQWiG, and CADTH offered recommendations covering hospital data, emphasizing the data curation process. HAS addressed considerations in choosing secondary data sources, while IQWiG established robust criteria for registries to ensure data quality. Regarding patient-reported outcomes data, only HAS and NICE provided recommendations in their guidance. The HTA experts acknowledged the value of hospital data but expressed caution due to its unstructured nature, noting that the use of hospital-based RWE is more accepted in descriptive studies.

Guidances prioritize the clinical domain, emphasizing transparency, fitness for purpose, reproducibility, robustness, bias minimization, and generalizability. Notably, there’s a lack of comprehensive source-specific guidance for real-world data sources, including registries, hospitals, claims, and wearables. Enhanced guidance on the total data generation process, (data mapping, data federation), cost data, quality of life, and cross-border data usage would strengthen hospital-based RWE assessments.

For early benefit assessment in Germany, drugs must be assessed at market entry as to whether they provide an added benefit versus the current standard therapy. This retrospective analysis focuses on assessment reports prepared by the independent Institute for Quality and Efficiency in Health Care (IQWiG). The objective was to describe the relationship between available evidence and assessment outcome.

Data were retrospectively extracted from IQWiG’s early benefit assessment reports of oncological drugs that entered the market between June 2021 and December 2021. The underlying evidence for the benefit assessments was divided into three groups: direct comparison (based on a randomized controlled trial [RCT]), indirect comparison, and other sources (e. g., only a single-arm trial was submitted). Afterwards, the outcome of the assessment was described by type of available evidence.

In the period under review, IQWiG worked on 27 oncology projects addressing 40 different oncological research questions resulting in 46 conclusions. About half of the available evidence in the benefit assessments was a direct comparison with standard therapy based on an RCT (n=22/46). From this level of evidence, an added benefit was derived for the drug under assessment in 64 percent of cases. Indirect comparisons (n=7/46) mostly led to an added benefit not proven (86%). If only other sources were submitted (n=17/46), the added benefit was assessed as not proven in all projects examined.

This retrospective analysis highlights the pivotal role of RCTs in providing strong evidence for health technology assessment. Conversely, single-arm and observational studies fail to yield substantial evidence. In the future, RCTs should be made simpler, faster, and cheaper; for example, by integrating them into existing infrastructures in routine practice (e.g., registry-based RCTs) or by conducting randomized adaptive platform trials.

The widespread adoption of digital health technologies (DHTs) brings new methodological challenges to health technology assessment (HTA). Different tools, guidelines, and methods have been defined by different stakeholders at national/international levels. In this scenario, the real thread for HTA is the lack of coordination and harmonization in the efforts to finalize the definition of an HTA framework for DHTs.

The European Digital Health Technology Assessment (EDiHTA) project aims to provide the first framework co-created by all stakeholders along the value chain for DHTs. The consortium is comprised of 15 partners from nine countries (Italy, the Netherlands, Denmark, Spain, Germany, Norway, UK, Belgium, and Poland). Stakeholders included are HTA agencies, regulators, hospitals, patient/citizen representatives, academics/universities, notified bodies, and developers. The project covers all types of DHTs and Technology Readiness Levels (TRLs). A multistakeholder, multidomain, and modular approach is adopted with the final goal to provide a validated, ready-to-use digital HTA framework for DHTs for the European ecosystem.

The EDiHTA is articulated in eight working packages and covers four years. Starting from the mapping of the current scenario, after the definition of a common taxonomy, EDiHTA will develop a DHT-dedicated HTA framework. A common digital platform to support the assessment will be developed. Both the HTA framework and platform will be tested, refined, and validated through a piloting process involving disparate DHTs (mApp, telemedicine, artificial intelligence, and others) with different TRLs, available in different countries, with the participation of all stakeholders. As a consequence, evidence requirements will be defined and made more transparent; this will support health technology.

The EDiHTA project will deliver the first European digital HTA framework able to support and inform decision-making (at the macro [policy], meso [management providers], and micro [clinicians] level) towards faster and safer access to DHTs of added value, taking European regulations and legal requirements into account and assessing all HTA domains.

Multiple sclerosis (MS) is characterized by a significant humanistic burden, as this is a chronic progressive disease associated with high levels of disability and need for caregiver support. Our objective was to explore to what extent “softer” outcome measures are considered as part of the health technology assessment (HTA) process of medications evaluated for the management of MS.

We performed a review of all Technology Appraisal Guidance (TAs) available at the National Institute for Health and Care Excellence (NICE) website, relating to the management of MS, in order to identify outcomes used either by the manufacturer or discussed by the Committee, that were beyond the typical ones around the clinical evidence, side effects, and costs associated with each technology under evaluation. The review focused on outcomes relating to the caregiver burden, the ease of use of the medication under evaluation, and the fatigue reported by patients.

Fifteen TAs were identified. Eight (53%) of the TAs included information on the caregiver burden, where the manufacturers applied caregiver disutilities in the cost-effectiveness analyses, which were deemed appropriate by the Committee. Six (40%) of the TAs further included a discussion on the ease of use for specific medications where the Committee concluded that the benefits of the mode of administration may not have been captured in the cost-effectiveness analysis. Fatigue was discussed in two TAs; the Committee provided conflicting conclusions around this measure, but in the most recent TA, it recognized that fatigue was an important outcome measure.

Based on the above findings, it appears that “softer” outcome measures are deemed relevant in the HTA process for treatments aimed for MS, on top of the measures typically used in the HTA process; this highlights the importance of recording the humanistic burden of disease in a holistic way.

Behavioral economics (BE) offers a spectrum of underutilized tools that could significantly enhance health technology assessment (HTA). This study proposes integration of BE into HTA, recognizing that understanding behavioral drivers can help in evaluating health technologies’ sustainability and adoption rates, thereby aligning HTA more closely with real-world healthcare dynamics and patient needs.

The study reviews existing key BE concepts and proposals for integrating BE concepts such as loss aversion, present bias, framing effects, and social norms influence into HTA framework. Loss aversion refers to the tendency of people to prefer avoiding losses rather than acquiring equivalent gains, while present bias is the tendency to prioritize immediate rewards over future benefits. Framing effect is the cognitive bias where people decide on options based on whether they are presented with positive or negative connotations.

Loss aversion can increase patient adherence to chronic disease management technologies by emphasizing the avoidance of negative health outcomes. Present bias limits adopting preventive technologies, where immediate costs overshadow long-term benefits. Framing effects may determine how the presentation of technologies influences acceptance; for example, by framing a new surgical device as reducing the risk of postoperative complications (a positive frame), rather than not increasing the risk (a negative frame), it may be more readily accepted. Additionally, use of default options in electronic health records can improve data accuracy, and recognizing social norms can drive broader adoption and success of telehealth solutions.

Integrating BE with HTA methodology offers a pathway to more patient-centered and sustainable health technology implementation, with an emphasis on patient behavior patterns. This approach enables HTA to more effectively address human behavior intricacies, ensuring that health technology evaluations are both comprehensive and relevant to the evolving needs of healthcare systems and patients.

Increasing resources are devoted to osteoarthritis surgical care in Australia annually, with significant expenditure attributed to hip and knee arthroplasties. Safe, efficient, and sustainable models of care are required. This study aimed to determine the impact on healthcare costs of implementing an enhanced short-stay model of care (ESS-MOC) for arthroplasty at a national level.

Budget impact analysis was conducted for hospitals providing arthroplasty surgery over the years 2023 to 2030. Population-based sample projections obtained from clinical registry and administrative datasets of individuals receiving hip or knee arthroplasty for osteoarthritis were applied. The ESS-MOC assigned 30 percent of eligible patients to a shortened acute-ward-stay pathway and outpatient rehabilitation. The remaining 70 percent received a current practice pathway. The primary outcome was total healthcare cost savings post-implementation of the ESS-MOC, with return on investment (ROI) ratio and hospital bed-days utilized also estimated. Costs are presented in Australian dollars (AUD) and United States dollars (USD), at 2023 prices.

Estimated hospital cost savings for the years 2023 to 2030 from implementing the ESS-MOC were AUD641 million (USD427 million) (95% CI: AUD99 million [USD66 million] to AUD1,250 million) [USD834 million]). This corresponds to a ROI ratio of 8.88 (1.3 to 17.9) dollars returned for each dollar invested in implementing the care model. For the period 2023 to 2030, an estimated 337,000 (261,000 to 412,000) acute surgical ward bed-days, and 721,000 (471,000 to 1,028,000) rehabilitation bed-days could be saved. Total implementation costs for the ESS-MOC were estimated at AUD72 million (USD46 million) over eight years.

Implementation of an ESS-MOC for eligible arthroplasty patients in Australia would generate significant cost and healthcare resource savings. This budget impact analysis demonstrates a best practice approach to comprehensively assessing value, at a national level, of implementing sustainable models of care in high-burden healthcare contexts. Findings are relevant to other settings where hospital stay following joint arthroplasty remains excessively long.

The exploration of molecular characteristics has emerged as a prominent trend to advance precision medicine. The utilization of genetic testing to guide therapy is integral to precision medicine. This study aims to investigate the potential patient populations for the reimbursement of next-generation sequencing (NGS) and assess the budget impact from the perspective of Taiwan’s single insurer, the National Health Insurance Administration.

To comprehend the scope for medicines with companion diagnostics (CDx) involved, we analyze the U.S. Food and Drug Administration-approved/cleared diagnostic tests, conduct a literature review to identify medicines approved by the European Medicines Agency that require a CDx, and identify the medicines with CDx involved covered by the National Health Insurance (NHI) in Taiwan. Subsequently, we explore the potential reimbursement indications for NGS testing and conduct a budget impact analysis to evaluate the expected financial impact for the NHI over a five-year period. Furthermore, sensitivity analyses are conducted to deal with uncertainty.

We have compiled 13 cancer types for which NGS can serve as a companion diagnostic. These encompass non-small-cell lung cancer, colorectal cancer, breast cancer, ovarian cancer, biliary tract cancer, acute myeloid leukemia, acute lymphoblastic leukemia, melanoma, cholangiocarcinoma, prostate cancer, pancreatic cancer, gastrointestinal stromal tumor, and thyroid cancer/medullary thyroid cancer. The implementation of NGS reimbursement in NHI will benefit 25,000 to 30,000 patients undergoing targeted therapies. The projected incremental budget impact ranges from TWD570 million to TWD650 million (USD19 million to USD22 million) over five years.

This study focuses on evaluating the financial impact of incorporating NGS testing into NHI reimbursement for relevant cancer drug indications. The findings can serve as references for the planning of reimbursement policies. However, with the advancement of precision medicine, it is foreseeable that there will be a broader range of applications for NGS, and its cost will gradually decrease.

Core outcome sets (COS) are increasingly being developed for routine care, but it is unclear how they compare to COS for research for a given condition, particularly regarding recommended outcomes. If similar, embedding COS within clinical practice creates opportunities for improving data for real-world evidence. This work aims to compare outcomes in COS for different settings.

Cancer, neurology, cardiovascular, rheumatology, and orthopedic COS within the Core Outcomes Measures in Effectiveness Trials (COMET) database were reviewed to create matched sets of COS (COS that were developed for the same condition but different settings). Recommended outcomes were extracted along with information on COS scope (condition, population, and intervention), patient involvement, and year of publication. Specific outcome matches (e.g., cognition and executive capacity) and general outcome matches (e.g., mobility and physical function) were identified within each matched COS set to report the number and percent of distinct outcomes recommended for both settings.

Eighteen matched sets were identified. The median (IQR) number of distinct outcomes recommended for both settings was 6 (4, 8) and the median (IQR) percent of all distinct outcomes that were recommended for both settings, of those included across both settings, was 20 percent (12%, 33%), ranging from nine percent for stroke rehabilitation to 50 percent for psoriatic arthritis. Variation due to potential factors such as outcome granularity, number of matched COS per condition, and scope discrepancies will be discussed. Case studies will be presented for localized and advanced prostate cancer, which demonstrated high levels of overlap.

Within a given condition, there appears to be a subset of outcomes relevant to both research and care, but the degree of overlap varies by disease area. Capturing these overlapping subsets of outcomes within routine data collection could provide a starting point to support generation of real-world evidence, thereby ensuring focus on outcomes of crucial importance to key stakeholders.

Minimally invasive procedures (MIPs) shorten procedural, hospital, and patient recovery time, providing patient benefits and reducing healthcare resource use (HCRU). This study explored the economic impact of two MIPs as discussed in published literature: transcatheter aortic valve replacement (TAVI), a step-change in cardiac clinical practice (“revolutionary”); and water vapor thermal therapy (WVTT), a progressive innovation in benign prostatic hyperplasia (BPH) (“evolutionary”).

Two pragmatic literature reviews were conducted to identify studies reporting comparative HCRU for TAVI versus surgical aortic valve replacement (SAVR) in aortic stenosis (AS), and WVTT versus other procedures in BPH. Searches were conducted on 20 October 2023 in the MEDLINE and MEDLINE plus Embase databases for the AS and BPH reviews, respectively. Studies from Asia-Pacific (AS review) and all geographies (BPH review) were included. HCRU data reported in the literature were identified and extracted.

Forty eligible studies were included (14 AS, 26 BPH). Commonly reported outcomes were hospital length of stay (LOS) (AS, BPH studies), intensive care unit (ICU) LOS (AS studies), and procedure duration (BPH studies). In AS studies, hospital LOS was shorter for TAVI (8.00 to 19.96 days) than SAVR (13.09 to 29.50 days). ICU LOS was shorter with TAVI (0.00 to 6.40 days) than SAVR (1.00 to 8.13 days). In BPH, WVTT had shorter hospital LOS (0.00 to 1.10 days) than other procedures (1.00 to 3.00 days). Of five studies reporting procedure duration, four showed shorter procedure time with WVTT (4.00 to 30.00 mins) than other procedures (5.20 to 148.00 mins).

MIPs, whether “revolutionary” or “evolutionary,” could offer notable time-savings (both in terms of procedure duration and length of stay) at the hospital level, which may also lead to cost savings. This highlights the importance of establishing mechanisms in payer-level economic evaluations or health technology assessments that can account for the time-saving advantages of MIPs at the hospital level.

Target product profiles (TPPs) can be used to outline the minimal and desirable characteristics of a health technology to meet a particular health need. They have been used by industry, regulators, and international health agencies to guide product development and facilitate communication across stakeholders, but there appears to have been limited consideration of their utility within HTA.

We completed a narrative review of academic and grey literature on uses of TPPs across the product development, regulatory, and HTA pathway and considered areas within HTA where TPPs could have a role. We then developed a case study alongside an assessment of virtual ward platforms for acute respiratory infection. This case study aimed to explore whether it was possible to construct a TPP to support guidance based on HTA committee discussions and to consider associated opportunities and challenges. We also compared our TPP with other product specifications used within NHS England to support commissioning.

There has been discussion about how industry can build HTA perspectives into TPPs. However, we did not identify use of TPPs led by HTA agencies. Two use cases of TPPs were identified: (i) to support demand signaling for areas of unmet need and (ii) to provide a framework for class-based recommendations. HTA committee discussions supported the development of a TPP for virtual ward platforms with domains relating to patient population, technological specification, evidence requirements, and other factors (e.g., patient choice). Challenges around the time needed to consider both evidence and desired attributes and overlap with approaches used in commissioning were identified.

TPPs may have a role to play within HTA to highlight areas of unmet need or to support innovative approaches to guidance. Digital health technologies may be particularly amenable to these approaches due to the larger range of similar products and ease of replicability. Further work should define how TPPs can be used to their full potential.

Asthma affects individuals of all ages and is the most common chronic disease among children. The Brazilian Unified Health System (SUS) reimburses the use of mepolizumab as an additional maintenance treatment for severe eosinophilic asthma in adults. This study aimed to evaluate the cost-effectiveness of expanding the reimbursement of mepolizumab to patients aged six years and older.

The model included patients aged six years and older refractory to inhaled corticosteroid (ICS) plus long-acting beta-agonist (LABA) treatment. Baseline characteristics and outcomes were based on clinical trials and national hospitalization data. A Markov model with monthly cycles considered the health states without exacerbation, with exacerbation (including the need for oral steroids, emergency room admission, or hospitalization), and death. Direct medical costs (rate: USD1=BRL4.93) associated with all treatments were estimated from public health reimbursement sources. Quality-adjusted life years (QALY) was the primary outcome. Deterministic (Tornado) and probabilistic (Monte Carlo simulations) sensitivity analyses were conducted using Microsoft Excel.

Considering the current mepolizumab price (USD964.49) reimbursed by the Ministry of Health (MOH), the incremental cost-effectiveness ratio (ICER) was USD137,384.92/QALY (95% confidence interval: USD92,625.64, USD208,542.44), exceeding the USD24,333.86 threshold proposed by the National Committee for Health Technology Incorporation (Conitec). Using the average price from local public purchases (USD417.74), the ICER was USD96,673.51/QALY. Considering the past proposed discount from the manufacturer (USD390,93) and dose fractionation in children up to 12 years, the ICER was estimated in USD50,574.06/QALY. The most impactful parameters were the utility without exacerbation, mepolizumab cost, and relative reduction in exacerbation rate.

Considering current reimbursement prices, mepolizumab use in children and adolescents with refractory severe eosinophilic asthma is not cost effective in Brazil. Alternative scenarios with price discounts and dose fractionation may alter these conclusions.

First-generation somatostatin analogues (SSA) are indicated as a first-line medical treatment for acromegaly in patients with persistent disease after surgery or who are not eligible for surgery. These drugs are the largest contributor to the direct medical cost of acromegaly management worldwide. We analyze the patterns of prescription and costs of SSA for the treatment of acromegaly in Brazil.

The first-generation SSA, octreotide LAR (OCT-LAR) and lanreotide autogel (LAN-ATG), are available in Brazil to treat acromegaly through the Specialized Component of Pharmaceutical Assistance policy. The public records of the nationwide database (DATASUS) were accessed to identify the drug use patterns during the year 2022. The current values of the drug acquisition costs of each medication in 2022 were consulted at the national prices database (BPS). Results were converted in purchase power parity (PPP) dollars according to the World Bank rates.

The acquisition cost of each octreotide (OCT-LAR) ampoule was USD492.33 (10 mg), USD537.04 (20 mg), and USD703.79 (30 mg); for lanreotide (LAN-ATG), each ampoule was USD394.74 for the 60 mg dosage, and USD418.48 for the 90 mg and 120 mg dosages. The average annual cost of a monthly 30 mg dosage of OCT-LAR would be estimated as USD8,445,48 against an average annual cost of USD5,021.76 for a monthly 120 mg dosage of LAN-ATG. Thus, we estimate that USD15,520,747.59 was spent on first-generation SSA to treat acromegaly in Brazil’s Unified Health System (SUS) in the year 2022; of this, 79 percent was attributed to OCT-LAR.

Life-long treatment with SSA is related with a high economic burden in Brazil. There is a predominance of OCT-LAR prescription, where expanding the use of LAN-ATG may help reduce costs to the SUS. Nevertheless, studies and investments in other treatments, such as pituitary surgery and radiotherapy access, at a national level are also essential to improve acromegaly treatment costs.

Academic life scientists often struggle to develop and commercialize concrete medical products based on their discoveries. Early health technology assessment (eHTA) can help innovators to define target product profiles (TPPs) with strong value propositions. To understand how eHTA can best help facilitate the clinical translation of university-based inventions, we conducted a survey of stakeholders in the life science innovation ecosystem.

Our 10-minute online survey includes questions on respondents’ location, organizational affiliations, experiences in health technology development, and awareness and perceptions of eHTA. eHTA is broadly defined as the use of tools from health economics, epidemiology, management, and related disciplines to assess the potential value of a medical product candidate for patients, payers, providers, manufacturers, and other stakeholders. The survey is being advertised using social media and email, and it will be followed up with semistructured interviews. Data on 51 complete responses were summarized using frequency tables and cross-tabulations, and the statistical significance of subgroup differences was evaluated using Fisher’s exact test.

Of 51 respondents, a majority lived in Canada (38/51; 75%) and had an academic affiliation (39/51; 76%). A “lack of commercialization skills among academic life science teams” was identified as a barrier to clinical translation by 41 percent (21/51), though this varied by academic affiliation (33% vs 67%; p=0.051) and industry experience (65% vs 29%; p=0.033). While 31 percent (16/51) reported familiarity with eHTA, this also varied by academic affiliation (23% vs 58%; p=0.033). Only 20 percent (10/51) had previously used eHTA, but a majority expressed an interest in learning more (39/51; 76%) and in using eHTA in the future (31/51; 61%).

Making eHTA more accessible for academic life scientists who lack commercialization experience may mitigate an important barrier to clinical translation of university-developed health technologies. While awareness of eHTA is relatively low in this group, they are interested in learning more about and using eHTA, and efforts should be made to integrate eHTA with existing product development tools like the TPP.

The value of a new pharmaceutical product should rely on its therapeutic benefits while also considering social health needs. Adapting health technology assessment (HTA) tools, we propose a transparent framework, with well-defined criteria and script, to determine the value of innovation (i.e., innovativeness) with clinical and methodological parameters, based on the social relevance and therapeutic value of new medicines.

The study was developed by adapting HTA-based methods identified in the literature: the Italian Medicines Agency (AIFA) and the EValuation of pharmaceutical Innovations with regard to Therapeutic Advantage (EVITA) tool. A sample of oncology drugs approved by the U.S. Food and Drug Administration (FDA) between 2011 and 2021 were assessed by researchers trained in HTA tools using those methods. After assessing the sample, researchers discussed the results, difficulties, and issues experienced. Those issues were addressed, resulting in the creation of the new framework, which included the redefinition of domains, definition of classification criteria for each domain, and scores according to relevance.

The Pharmaceutical Innovativeness Index (PII) was proposed, with a script, domains, criteria, and an algorithm. The evaluation begins defining indication, outcomes, therapeutic alternatives, time perspective, and data sources. Four domains were considered: Therapeutic Need, which evaluates the existence and benefits of alternatives; Added Therapeutic Value, which talks about the incremental clinical benefit when compared to those alternatives (these first two domains were graded into five levels ranging from absent to maximum); Study Design and Methodological Quality, both classified into three levels. Classification criteria for each domain can be adapted according to the indication and relevant outcomes.

The PII framework considers clinical and social value weighted by methodological limitation of available evidence to determine the value of innovation of pharmaceutical products. It stands out as a transparent, adaptable, and reproducible tool that aims to reduce the subjectivity of analyses and assist with decision-making. PII has the potential to inform decision-making processes involving value-based pricing, reimbursement, and research and development investment.

Prostate cancer (PC) constitutes 13.5% of all cancer cases globally. Treatment is individualized and depends on various factors. In recent years, there has been an increase in the approval of drugs for the disease by the U.S. Food and Drug Administration (FDA). The study aimed to assess the innovativeness of FDA-approved drugs for PC treatment from 2011 to 2021.

A search was conducted in the FDA database to identify medications approved from 2011 to 2021 and their supporting studies for approval. The assessment of the value of innovations was performed through the Pharmaceutical Innovativeness Index (PII), a methodology that quantitatively evaluates innovations in four domains: Therapeutic Need and Added Therapeutic Value of the new medication (graded on five levels from important to absent), and Study Design and Methodological Quality (graded on three levels) of the pivotal studies used as a data source for evaluation. Medications are assessed for a specific clinical indication and compared to available therapeutic alternatives.

Seven medications were identified for the treatment of PC, targeting different stages of the disease. The drugs were evaluated with a score ranging from 0 to 100, measuring the degree of innovativeness across the four assessed domains. Five (70%) medications scored above 50.0. The majority of the medications addressed a significant Therapeutic Need (n=4; 56%). The Added Therapeutic Value was assessed based on the survival gain compared to the comparator, with five medications considered poor (70%) and two moderate (30%). Regarding the quality of evidence, most studies showed a low risk of bias and a partially adequate design.

In recent years, there has been an increase in the development of drugs for prostate cancer. However, the Added Therapeutic Value shows a small to moderate increase in survival compared to existing treatments. Many studies used a placebo instead of comparing new medications with available therapies. The PII indicates that, despite advancements, new technologies are needed to improve patient survival.