bluuebunny/crossref_metadata_2025 huggingface dataset:
OD46 Inequalities In Dementia: Identifying Instruments For Measurement
Authors: Hodgson Sian , Hayes Helen , Cubi-Molla Patricia , Garau Martina
Published: January 2025

Introduction

Despite dementia being the seventh leading cause of death globally, there is relatively little discussion of the presence and impact of inequalities in this context. We explore ways to quantify the magnitude and variation over time of inequalities related to people living with dementia (PLWD) and their informal carers.

Methods

We conducted a targeted literature review to identify inequalities faced by PLWD and their informal carers regarding their access to and experience of health and social care in England, Wales, and Northern Ireland. We selected four of the identified inequalities as case studies (CS) to explore data and methods that can be used to measure and monitor progress to tackle them. The CS considered were: (CS1) timely diagnosis in rural areas; (CS2) financial pressures for informal carers; (CS3) timely diagnosis in deprived areas; and (CS4) diagnosis rates for ethnic minority groups. We use data from 2018 to 2023 in England.

Results

We identified 110 inequalities for PLWD and 28 inequalities for carers. For CS1, we proposed two measures: the “rurality gap” (gap in diagnosis rates between the most and least rural areas) and the “concentration index” (the extent to which diagnosis rates are distributed disproportionately between less or more rural areas). The rurality gap suggests that diagnosis rates are five to eight percent lower in rural areas in England. The concentration index supports this finding. CS2 shows that 41 percent of informal carers experience financial difficulties. Due to insufficient data, it was not possible to construct robust measures for CS3 and CS4.

Conclusions

Many inequalities for PLWD and their informal carers are reported in the literature. Our CS highlight the need to improve methods and data to measure a set of inequalities, including those to calculate dementia prevalence and measure timely diagnosis. Better data is crucial now to inform value assessment of the upcoming Alzheimer’s disease treatments and avoid exacerbating existing inequalities.
PP06 Incentives To Incorporate Innovation Into Care Delivery Processes: A Scoping Review And SWOT Analysis
Authors: Haig Madeleine , Main Caitlin , Kanavos Panos
Published: January 2025

Introduction

This study investigated the various incentives employed in Organisation for Economic Co-operation and Development (OECD) and European Economic Area (EEA) countries to enhance access to innovative medical technologies. The literature review encompasses real and theoretical models, offering an overview of strategies to bridge the gap between innovation and access. A strengths, weaknesses, opportunities, and threats (SWOT) analysis was conducted to analyze incentives in health system and therapeutic area context.

Methods

The review methodically examined peer-reviewed articles, reports, and policy documents published between 2000 and 2023. Databases searched include PubMed, Scopus, Web of Science, and EconLit. Grey literature was searched from international organizations’ websites, including the World Bank, World Health Organization (WHO), OECD, Pan American Health Organization (PAHO), and European Commission. Inclusion criteria focused on relevance of financial and non-financial mechanisms to effective implementation of innovative medical technologies, and their application within OECD and EEA countries. COVID-19 research, vaccines, cost-effectiveness studies, and studies that did not discuss implementation were excluded. A SWOT analysis was utilized to categorize the mechanisms by therapeutic area and health system design.

Results

The review identified diverse mechanisms, including reinsurance, impact bonds, outcomes-based agreements, annuity payments, and risk-sharing agreements. Financial mechanisms, such as outcomes-based agreements, were prominent but highlighted implementation obstacles, including a lack of data infrastructure capable of linking outcomes to payments, which ultimately undermines the effectiveness of these strategies. Non-financial mechanisms, such as population health management, were also identified. The effectiveness varied, with some models showing significant improvement in technology accessibility, while others faced implementation and affordability challenges. Comparative analysis highlighted differences in efficacy dependent on the therapeutic area and type of health system in which the incentive is applied.

Conclusions

The review underscores multifaceted approaches to improve access to innovative medical technologies. While financial incentives play a crucial role, non-financial strategies are also vital. This study provides insights into which incentives are most effective in certain health systems and therapeutic areas. Policymakers can benefit from these insights, leveraging successful models and addressing challenges to ensure equitable access to medical innovations.
PP08 Prognostic Factors For Survival In Adults With Burkitt Lymphoma: A Systematic Review And Meta-Analysis
Authors: De Armas-Castellano Aythami , Infante-Ventura Diego , González-Hernández Yadira , Pino-Sedeño Tasmania del , Leon-Salas Beatríz , Quirós-López Raúl , Trujillo-Martín Mar ,   EuroBloodNet Working Team
Published: January 2025

Introduction

Burkitt lymphoma (BL) is a rare and highly aggressive subtype of non-Hodgkin’s lymphoma. Several studies have identified prognostic factors (PFs) for disease progression and mortality among adults with BL. However, there is no consensus on risk stratification based on PFs. This study aims to identify, critically assess, and synthesize the available evidence on PFs for survival in adults with BL.

Methods

A systematic review of the literature was conducted. MEDLINE, Embase, and CENTRAL were searched from inception to 22 February 2022. Randomized or non-randomized clinical trials and longitudinal observational studies were eligible for inclusion. Reference screening, data extraction, and risk-of-bias assessment using the Quality in Prognosis Studies (QUIPS) tool for prognostic factor studies were conducted independently and in duplicate. Publication bias was examined by visual inspection of funnel plots. Effect measures and the corresponding 95% confidence intervals were pooled with an indirect variance estimation in meta-analyses using Review Manager 5, and sensitivity analyses were conducted. Certainty of evidence was assessed using GRADE.

Results

The search identified 1,119 references after duplicate removal. Of these, 76 potentially relevant papers were selected for full-text assessment and 36 studies (N=10,882) reported in 39 articles were eligible for inclusion. Older age, higher performance status, and central nervous system involvement were associated with poorer overall survival (OS) and progression-free survival (PFS). Black patients exhibited significantly lower OS and relative survival. Bone marrow involvement and higher albumin levels were associated with poorer OS. Treatment with rituximab and treatment with methotrexate were associated with better OS and PFS. No significant differences in survival were found for HIV status, sex, and risk stratification.

Conclusions

This study, framed within a collaboration with European Reference Network EuroBloodNet, provides a comprehensive and methodologically rigorous evidence review on PFs in adults with BL. Several significant associations of PFs and survival estimates were observed, providing data to inform treatment decisions and to improve patient care.
PP09 Fecal Immunochemical Tests For Patients With Symptoms Suggestive Of Colorectal Cancer: A Systematic Review And Multiple-Threshold Meta-Analysis
Authors: Harnan Sue , Hamilton Jean , Simpson Emma , Clowes Mark , Biz Aline Navega , Whyte Sophie , Ren Shijie , Cooper Katy , Ball Alex , Benton Sally , Carten Rachel , Kurien Matt , Monahan Kevin , Heathcote Laura , Stevenson Matt
Published: January 2025

Introduction

Extending fecal immunochemical tests for hemoglobin (FITs) to primary care patients with high-risk symptoms suggestive of colorectal cancer (CRC) could reduce colonoscopy waiting lists, enabling earlier treatment. Higher FIT thresholds could decrease referrals but increase missed disease compared with lower thresholds. We aimed to systematically review and synthesize test accuracy data across thresholds for use in a cost-effectiveness analysis.

Methods

Searches across ten sources were conducted (December 2022). Included were diagnostic accuracy studies of HM-JACKarc, OC-Sensor, FOB Gold, QuikRead go, NS-Prime, and four Immunodiagnostik (IDK) tests in patients presenting to, or referred from, primary care with symptoms suggestive of CRC using any reference standard. Risk of bias was assessed with QUADAS-2. Syntheses of sensitivity and specificity at all reported thresholds were planned for each test to provide summary estimates at all possible thresholds within the observed range. Sensitivity analyses investigating population type and reference standard, and subgroup analyses by patient characteristics (e.g., anemia, age, sex, ethnicity) were conducted.

Results

HM-JACKarc (n=16 studies) sensitivity ranged from 95.9 percent (95 percent credible interval [95% CrI]: 92.7, 97.9) to 46.3 percent (95% CrI: 37.4, 54.9) and specificity from 65.1 percent (95% CrI: 55.6, 74.8) to 97.7 percent (95% CrI: 94.7, 99.2) (thresholds 2 and 400 μg hemoglobin/g feces [μg/g], respectively). OC-Sensor (n=11) sensitivity ranged from 94.2 percent (95% CrI: 91.2, 96.7) to 54.2 percent (95% CrI: 48.4, 60.2) and specificity from 62.7 percent (95% CrI: 47.4, 77.2) to 97.3 percent (95% CrI: 92.9, 99.3) (thresholds 4 and 200 μg/g, respectively). FOB Gold (n=3) sensitivity ranged from 91.4 percent (95% CrI: 71.6, 99.6) to 73.9 percent (95% CrI: 53.8, 91.2) and specificity from 78.1 percent (95% CrI: 70.0, 86.0) to 96.4 percent (95% CrI: 92.6, 98.9) (thresholds 2 and 150 μg/g, respectively). There were limited or no data on the other tests.

Conclusions

Sensitivity and specificity were synthesized for three tests only, since data for the remaining tests were extremely limited or absent. Even at the lowest threshold, none of the tests had perfect sensitivity. Future studies should further investigate comparative accuracy and the impact of patient characteristics, patient recruitment criteria, and the reference standard on estimates of diagnostic test accuracy.
PP14 Recombinant Human Erythropoietin For Sickle Cell Disease And Brazilian Healthcare System Sustainability: Cost-Effectiveness And Budget Impact Analysis
Authors: Gargano Ludmila , Lobo Clarisse , De Oliveira Haliton Alves , Lucchetta Rosa
Published: January 2025

Introduction

Sickle cell disease (SCD) is a rare disease, including renal complications. Erythropoiesis-stimulating agents (i.e., recombinant human erythropoietin, rHuEPO) are recommended for SCD and renal impairment. Evidence suggests its effectiveness in raising hemoglobin (Hb), which may reduce the need for regular blood transfusions. Cost-effectiveness analysis (CEA) and budget impact analysis (BIA) of rHuEPO were performed from the Brazilian Unified Health System (SUS) perspective.

Methods

A decision tree was created to assess rHuEPO’s impact on reducing blood transfusions. Quality-adjusted life year (QALY) for transfusion dependency and direct medical costs related to rHuEPO and transfusion were considered. SCD patients with worsening kidney function and Hb levels enter the model with an indication of receiving regular transfusions; those who proceed to the rHuEPO+standard care arm are likely to have clinically relevant elevation in Hb levels (i.e., <1.5 g/dL), suspending transfusions, and those who do not respond to treatment continue to receive regular transfusions. BIA population was estimated based on epidemiological data, considering direct costs over a five-year horizon.

Results

rHuEPO+standard care compared to standard care generated 36.8 percent less need for transfusions, resulting in an increase of 0.033 QALY and a saving of BRL11,564 (USD2,362) per patient/year. rHuEPO was the dominant alternative; that is, there was greater clinical benefit and lower total cost. At BIA, the eligible population was 5,274 patients per year, on average. The direct cost of acquiring rHuEPO for the total eligible population summed BRL13,737,129 (USD2,806,016) in five years. However, considering the estimated effectiveness of CEA in reducing transfusions, BIA demonstrated savings of BRL96,545,791 (USD19,720,936) accumulated over five years.

Conclusions

Health technology assessments showing the new alternative as dominant are uncommon, especially in rare diseases, where patented orphan drugs usually have increased costs, which is not the case of rHuEPO. In this analysis, rHuEPO remained the dominant alternative and its incorporation would result in savings that may contribute to the sustainability of the Sistema Único de Saúde, Brazil’s national health system.
PP18 Red Flags For The Early Diagnosis Of Rare And Complex Connective Tissue And Musculoskeletal Diseases
Authors: García Guillermo Pérez , Vicente Edo Maria José , Valero Montserrat Salas , Remón Lucía Prieto , Benavent Patricia Gavin
Published: January 2025

Introduction

In collaboration with a European Reference Network for rare diseases, we aimed to identify red flags for the diagnosis of rare and complex connective tissue and musculoskeletal diseases (rCTDs). Some indicators, presented as red flags, might raise clinicians’ awareness about the presence of rCTDs. Their identification is critical in primary care, where they are most likely to be first observed.

Methods

Firstly, we conducted a scoping review to identify red flags already published in the scientific literature. We included studies about people with rCTDs that described red flags, warning signs, alarm symptoms, and pathognomonic signs identifiable in a primary care setting. Then, we conducted a systematic review of evidence pointing out which signs and symptoms should arouse suspicion specifically for IgG4-related disease. We included studies providing estimates of diagnostic precision or prevalence of signs and symptoms, and we assessed their quality and applicability to the review question. We conducted systematic searches in major medical databases and manual searches in rare disease resources.

Results

For the scoping review, 49 studies out of 1,656 records met the inclusion criteria. Two reported red flags for autoimmune diseases altogether, and 14 described red flags for systemic sclerosis. For the systematic review, seven studies out of 4,477 records met the criteria, comprising five diagnostic precision studies and two large case series. These were generally rated as having a high risk of bias and were included as indirect evidence. We identified 32 potential IgG4-related disease red flags, 10 related to clinical history findings and basic signs or symptoms, and eight belonging to common laboratory findings and basic imaging techniques.

Conclusions

Red flags for rCTDs have generally been established through expert consensus and lack valid indicators for diagnosis, such as sensitivity, specificity, or predictive values. They frequently overlap among different rCTDs. Potential red flags are prone to change as further evidence emerges. This shows the need to collaborate with reference networks to address rare diseases where the evidence is still scarce.
PP27 Evidence Quality and HTA outcomes in Reappraisals For Drugs For Rare Diseases In Germany
Authors: Wiedmann Lea , Cairns John , Nolte Ellen
Published: January 2025

Introduction

Evidence on re-appraisals of health technologies in Germany is limited, and for rare disease treatments (RDTs) the Federal Joint Committee (GBA) uses different processes depending on whether the annual revenue threshold has been exceeded. We analyze RDTs for which an initial appraisal and a reappraisal were conducted to better understand processes used to determine the clinical benefit rating and their outcomes.

Methods

We review appraisal documents for 55 RDT indications published between January 2011 and September 2023. We extract information for the change in the maturity of survival data, the type of evidence, the risk of bias, and the availability of additional evidence as proxies for evidence quality. Specifically, we review the reasons for conducting reappraisals; examine how evidence quality and the clinical benefit rating differed between initial appraisals and re-appraisals; and explore the association between evidence quality and the clinical benefit rating following reappraisal.

Results

Most reappraisals were conducted because of exceeding the revenue threshold of EUR50 million (USD54 million) per year or reaching the review date when an initial decision was time limited. Almost all initial appraisals used the limited process, while the majority of reappraisals used the regular process. While nine out of 55 reappraisals achieved a higher benefit rating in reappraisals compared to initial appraisals, in 21 out of 55 reappraisals the benefit rating decreased. There was some evidence that reappraisals with accepted randomized controlled trial evidence were significantly more likely to achieve a higher clinical benefit rating.

Conclusions

Our findings confirm that reasons and processes for completing reappraisals of RDTs in Germany differ. Moreover, the quality of the evidence submitted for both initial appraisals and reappraisals of RDTs was limited and achieving a high clinical benefit rating in reappraisals was rare.
PP31 Logical Model Articulating Pharmacy Commissions And Hospital-Based Health Technology Assessment Nuclei: Collaborative Strategies For The Drug Selection
Authors: Stopatto Denise Souza , Silva Elias Flávia Tavares
Published: January 2025

Introduction

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).

Methods

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.

Results

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.

Conclusions

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.
PP34 Impact Of The COVID-19 Pandemic On Depression Incidence And Healthcare Service Use Among Patients With Depression
Authors: Yi Chan Vivien Kin , Chai Yi , Man Chan Sandra Sau , Luo Hao , Jit Mark , Knapp Martin , Bishai David Makram , Ni Michael , Kei Wong Ian Chi , Li Shirley Xue
Published: January 2025

Introduction

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.

Methods

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.

Results

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.

Conclusions

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.
PP35 Keeping It Real Around The World: Comparing Real-World Evidence Guidance From Regulatory And Health Technology Assessment Bodies
Authors: Wee Yan Ran , Ng Alvin , Dinkar Eesha , Evans Jennifer Sara
Published: January 2025

Introduction

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.

Methods

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.

Results

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”).

Conclusions

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.
PP41 Enhancing Health Technology Assessment Through Behavioral Economics: A Strategic Approach To Sustainable Healthcare Innovation
Authors: Swami Shilpi
Published: January 2025

Introduction

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.

Methods

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.

Results

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.

Conclusions

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.
PP45 Core Outcome Sets For Research And Core Outcome Sets For Routine Care: Do They Overlap?
Authors: Kearney Anna , Gorst Sarah , Williamson Paula , Dodd Susanna
Published: January 2025

Introduction

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.

Methods

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.

Results

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.

Conclusions

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.
PP47 The Role Of Target Product Profiles Within Health Technology Assessment: A Narrative Review And Case Study
Authors: Hopkin Gareth , Chalkidou Anastasia , Ismail Farhan , Pelekanou Charlotte , Salmon Mark , Dawoud Dalia
Published: January 2025

Introduction

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.

Methods

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.

Results

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.

Conclusions

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.
PP51 Pharmaceutical Innovativeness Index (PII): A New Framework For Assessing Health Technology Innovation Based On Therapeutic And Social Relevance
Authors: Gargano Ludmila , André Ariane , Freitas Isabela , de Assis Acurcio Francisco , Alvares-Teodoro Juliana , Guerra Augusto
Published: January 2025

Introduction

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.

Methods

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.

Results

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.

Conclusions

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.
PP57 Outcomes Model For Assessing Strategies Improving In Vitro Fertilization Birth Rates
Authors: Mongan Suzanna , Lyu Juntao , Kim Hansoo
Published: January 2025

Introduction

Infertility affects one-sixth of women worldwide, with over seven million assisted reproductive cycles performed annually. Oral dydrogesterone is recommended alternatively for luteal phase support in in vitro fertilization (IVF), preventing miscarriages and improving live birth rates. This study aims to develop an outcomes model comparing oral dydrogesterone treatment with the standard of care in the IVF cycle over a 10-year period.

Methods

A two-level Markov cohort model in Microsoft Excel includes six health states: IVF, pregnancy, miscarriage, live birth, perinatal death, and maternal death. Miscarriage, live birth, and perinatal death are sub-states of pregnancy. Transition probabilities are based on published rates with medical intervention limited to the first 12 weeks of gestation. A sensitivity analysis of treatment was performed. Data from a published meta-analysis of nine dydrogesterone studies for IVF luteal phase support were used. The baseline cohort is 10,000 Australian females undergoing IVF annually over a 10-year period.

Results

Over the 10-year time horizon, compared to standard care, the group treated with dydrogesterone was estimated to increase the number of live births by 3.5 percent (range: 3.4 to 3.7%), reduce the number of miscarriages by 69.4 percent (range: 66.2 to 72.7%), reduce the perinatal death by 10.9 percent (range: 10.4 to 11.4%), reduce the IVF cycles by 11.56 percent (range: 11.0 to 12.11%), and reduce the death of the mothers by 10.9 percent (range: 10.4 to 11.4%).

Conclusions

The outcomes model projected that treatment with oral dydrogesterone significantly reduced the number of miscarriages and improved the number of live births compared to the standard of care used for IVF patients in Australia.
PP60 Enhancing Patient Empowerment: European Capacity Building Initiative And Curriculum Development In Health Technology Assessment Training
Authors: Muehler Milena , Siebert Silke , Schnell-Inderst Petra , Roßmann Sonja , Puntscher Sibylle , Planinschitz Tanja , Hallsson Lára R. , Houÿez François , Delaye Julien , Strammiello Valentina , Siebert Uwe
Published: January 2025

Introduction

The new Regulation (EU) 2021/2282 on health technology assessment (HTAR) highlights the increasing importance of patient perspectives, emphasizing their crucial role in HTA. The European Capacity Building for Patients (EUCAPA) project aims to empower patients for active involvement in HTA. EUCAPA focuses on equipping patients with capacities through the development of HTA training, including introductory, fast-track, and extended training programs.

Methods

Within the EU-funded EU4Health program, the EUCAPA consortium was tasked with capacity building for patients and patient representatives. Training programs were developed using a coproduction approach, integrating patient representatives’ perspectives into both curriculum development and the design of training sessions. This innovative approach involved close collaboration between patient representatives and HTA scientists, jointly shaping the program content. The curriculum was designed to incorporate the heterogeneous medical and cultural backgrounds and varying levels of prior knowledge among participants, recognizing diverse experiences that individuals bring to the training. To enhance accessibility, a blended learning approach with offline, virtual, and in-person parts was adopted.

Results

The training comprised introductory (two hours), fast-track (eight hours), and extended (four days) training sessions. Training modules include competencies such as a profound understanding of HTA processes and key principles, domains, impact of HTA, outcome measurements, and study designs to raise awareness, promote, and enhance patient engagement as foreseen in the HTAR. The training modules focus on empowering patients to advocate for their rights and communities and to contribute meaningfully to comprehensive treatment evaluation. Specifically, modules on communication and personal skills, patient involvement, and engagement at both national and European levels underscore promotion of patient empowerment. Training sessions can be accessed online (EUCAPA.eu).

Conclusions

The training development within the EUCAPA project addresses the evolving landscape of joint HTA by emphasizing the significance of patient perspectives, fostering core competencies, and responding to the increasing demand for active patient involvement and advocacy at both national and supranational levels.
PP61 A Decade Of Enhanced Patient And Citizen Involvement In Health Technology Appraisal In Scotland
Authors: Dickson Jennifer , Cairns Daniel , McCormack Jackie , Russell Kate
Published: January 2025

Introduction

Over the past 10 years, the Scottish Medicines Consortium (SMC) has strived to strengthen the involvement of patients and citizens in its work and improve public understanding of health technology appraisal (HTA). A series of innovations have been brought in since 2014 to increase levels of engagement and satisfaction of participants. This work describes these and their impact.

Methods

SMC has introduced numerous transformational changes since 2014. Innovations include a new Patient Group Partner (PGP) registration system, strengthened patient group submission process, participation of patient representatives at Patient and Clinician Engagement (PACE) and SMC Committee meetings, embargoed early release of SMC decisions to PGPs, provision of Summary Information for PGPs (from submitting company), and revised role of public representatives. The creation of the SMC Public Involvement Network Advisory Group has underpinned these achievements, as has the provision of comprehensive one-to-one support, information and education to PGPs. SMC has continually evaluated the satisfaction of participating PGPs using an online questionnaire.

Results

There has been a sustained increase over the past 10 years in both the number of patient groups engaging with SMC and the number of PGP submissions that SMC receives. Adopting a continuous improvement approach, working in partnership with PGPs and public representatives, has helped SMC to ensure that stakeholders in the HTA process are effectively engaged and informed about the HTA of new medicines in Scotland. Surveys of public involvement in SMC consistently show an extremely high level of satisfaction from PGPs who work with SMC, with most PGPs consistently rating their experience of working with SMC as excellent.

Conclusions

Over the past 10 years, SMC has strived to strengthen how it involves citizens and patient representatives in HTA. Various innovations and a continuous improvement approach have helped to ensure that there are high levels of satisfaction and understanding of the HTA process from patient groups who engage with SMC. This is underpinned by a partnership approach to working.
PP62 Improving Patients’ Access To Medicines Through Health Technology Assessment In Ukraine: Measuring The Impact Of The National HTA Roadmap
Authors: Piniazhko Oresta , Khmelovska Marharyta , Serediuk Valeriia , Babenko Mykhaylo , Lobas Mykhailo , Kosyachenko Kostyantyn , Sucu Rabia , Valuieva Nataliia
Published: January 2025

Introduction

In 2020, health technology assessment (HTA) became mandatory for any new medicine covered by public funds. The aim of the review is to present the impact of HTA implementation despite Russia’s full-scale war against Ukraine. Thanks to the introduced reforms, including HTA introduction, the coverage from the public payer in providing patients with medicines increased from 11 percent in 2013 to 34 percent in 2023.

Methods

This review summarizes all Department of HTA conclusions with recommendations made during 2023, decisions made by the Ministry of Health (MOH) based on these recommendations and their impact on patient access, and the steps made to further institutionalize HTA in Ukraine. In 2023, the HTA guidelines for medicines were revised based on the lessons learned and global experience along with knowledge exchange with the United Kingdom HTA agency, NICE International.

Results

In 2023, the HTA Department prepared 19 HTA conclusions. The majority concerned treatment of oncologic diseases (32%), orphan diseases (21%), and multiple sclerosis (16%). HTA assessments resulted in recommendations for listing (84% cases). Fifty-three percent of medicines were recommended for managed entry agreement, and the MOH decided to forward 10 medicines for negotiations in 2023. Thirty-two percent of recommendations considered inclusion into the National Essential Medicines List (NEML); 16% with further access through the reimbursement program. Almost 70 percent of total need in NEML is currently covered by public funds, compared to 40 percent in 2020 (according to the Support in Market Development (SMD) analytical database).

Conclusions

The HTA function in Ukraine has evolved since 2020 with a visible impact on patients’ access. Introduction of new procedures, such as HTA for medical devices, may improve patient access to health technologies and provide evidence for rational and transparent decision-making processes, with gradual transformation of the Department of HTA at the State Expert Centre into an independent HTA agency by 2026.
PP66 International Horizon Scanning Initiative (IHSI) : Time to Review the Need for Stand-Alone National Horizon Scanning?
Authors: Eames Heather , Harte Marie , Choitir Caitríona Ní , McCullagh Laura , Tilson Lesley , Adams Roisin
Published: January 2025

Introduction

Ireland is a member of the International Horizon Scanning Initiative (IHSI) and has access to the full horizon for medicines across multiple disease areas and specialized reports on high-impact technologies within disease areas (IHSI High Impact Reports [HIRs]). Health technology developers (HTDs) provide a notification to HTA agencies and payers on intent to seek reimbursement for a technology in the preceding two years.

Methods

Information on technologies submitted to the national HTA agency in 2023 was selected for comparison to information in the IHSI database. The IHSI system detects products prior to regulatory approval. The national notification system (NNS) can receive information post-licensing. We examined how many products were received in the NNS that were either in the regulatory process or licensed in the EU. We sought to compare key information in the NNS and the IHSI database to identify areas of duplication. Areas where information from NNS could complement the IHSI database were highlighted. HIRs were not included in this assessment.

Results

In 2023, 20 percent of products submitted by HTDs via the NNS were already licensed. To compare the information provided in IHSI and the NNS, a sample of 35 technologies common to both systems was selected. The IHSI and the NNS contain multiple data fields (154 in IHSI and 72 in NNS) across domains including information on product characteristics, regulatory information, clinical trial data, and cost. The exceptional fields specific to the Irish context were compassionate access scheme availability in Ireland, Ireland-specific patient population estimates, and local pricing and reimbursement proposals.

Conclusions

Receipt of information from HTDs remains important, particularly country-specific information. There are efficiencies in identifying technologies via an independent horizon scanning system, not reliant on submissions by HTDs. Such a system could facilitate joint assessment, joint early advice, and joint problem-solving between countries. Joint efforts can also facilitate broader scope on how horizon scanning can be used to increase health system preparedness.
PP71 Organizations Responsible For The Evaluation Of Health Technologies Globally: A Scoping Review
Authors: MigliavacaCelina Borges Migliavaca Celina Borges , Colpani Verônica , Zago Marcolino Miriam Allein , Falavigna Maicon , Polanczyk Carisi Anne
Published: January 2025

Introduction

The process of health technology assessment (HTA) is a valuable tool for the pursuit of equitable and sustainable healthcare systems. Various countries have established organizations dedicated to conducting HTAs, adapting such institutions to local healthcare ecosystems. The aim of this study was to evaluate the structure, methods, and processes of organizations responsible for national-level HTAs globally.

Methods

A scoping review was conducted assessing organizations responsible for conducting HTAs for national-level decision-making in any country. Identification of eligible organizations was performed through a review of member organizations of INAHTA, EUnetHTA, RedETSA, and HTAsiaLink networks, as well as organizations evaluated in reviews with a similar scope. For each organization, the following data were searched: country, year of foundation, organizational nature, role in decision-making, funding, technologies assessed, criteria considered for decision-making (such as efficacy and safety, costs, impact on equity, among others), type of economic evaluation, and patient involvement.

Results

We identified 69 organizations, from 56 countries, mainly European (n=39; 56%). Fifty-three (77%) are government-affiliated; most (n=51; 74%) have a consultative role. Public funding is the main funding, and 12 (17%) organizations charge fees for conducting HTA. Technologies assessed include drugs (n=61; 88%), devices (n=47; 68%), and procedures (n=33; 48%). HTA is usually initiated upon request from the manufacturer (n=45; 65%). Patient involvement is not clearly described in 32 organizations (46%); in 29 organizations (42%), the role of patients is to provide information that is considered during decision-making.

Conclusions

Among the evaluated organizations, it is observed that the majority are government-affiliated, have public funding, and play a consultative role. The results of this study serve as an important reference for the development and improvement of organizations responsible for conducting HTAs.