bluuebunny/crossref_metadata_2025 huggingface dataset:
PP47 Experience And Its Implication For Reassessment Of The Transcatheter Aortic Valve Implantation Using Real World Data
Authors: Park Jung Mi , Han Seung Jin , Kim Kyoung Hoon
Published: December 2023

Introduction

South Korea has introduced conditionality to coverage decisions for certain difficult or high-risk procedures. The transcatheter aortic valve implantation (TAVI) was included in the coverage with evidence development (CED) in 2014. This study reviewed the results of reassessment for the TAVI using real world data (RWD) and suggested its implications.

Methods

Healthcare providers authorized to use the promising technologies are required to collect the RWD for suitability evaluation, safety monitoring, and cost-effectiveness, differing from the general reassessment process. In 2021, 45 healthcare providers collected clinical information for TAVI patients. Their registries were linked with the national health insurance claims, which provided data on 19 items to assess safety and effectiveness such as overall mortality, reoperation rates, hospital readmission rates, and degree of functional improvement.

Results

According to the Society of Thoracic Surgeons’ predicted risk of mortality (STS), 988 TAVI patients were classified into three groups; high (STS >8 percent, n=347), intermediate (STS 4-8 percent, n=272), and low (STS <4 percent, n=369); We compared main outcomes and estimated survival probabilities between subgroups. Within 30 days, the overall mortality rates were 4.9 percent (high), 2.6 percent (intermediate), and 1.4 percent (low); major bleeding rates were 7.6 percent (high), 6.2 percent (intermediate),and 1.4 percent (low); incidence of new atrial fibrillation were 6.8 percent (high), 4.2 percent (intermediate), and 3.2 percent (low). Based on the quantitative results using RWD and systematic review for the safety and effectiveness, TAVI is reported to have essential benefits for high-risk group and elderly patients (>80 years). Whereas, intermediate and low-risk groups have out-of-pocket payment rates of 50 percent and 80 percent, respectively.

Conclusions

The reassessment system through RWD accumulation enabled the evidence-based evaluation for the TAVI. Based on the transition to CED for essential benefits, a systematic framework such as RWD collection from treatment commencement should be introduced to broaden RWD use for benefit management of medical technologies with uncertain levels of evidence. Therefore, this ensures overall quality of care and effective coverage in health.
PP64 Cost-Effectiveness Of Fractional Flow Reserve As Diagnostic Tool In Coronary Artery Disease Versus Angiogram Alone In Indian context
Authors: Pusha Monika , Fahim Arif , Kataria Kirti
Published: December 2023

Introduction

Fractional Flow Reserve (FFR) is a diagnostic tool that aids decision-making in the treatment of coronary artery disease (CAD). FFR provides an objective measurement and is used as an adjunct to an angiogram. The clinical and cost-benefit of using FFR have been well established across published literature. This research was aimed at evaluating the economic impact of using FFR as an adjunct to angiogram versus an angiogram alone, in the Indian healthcare context.

Methods

A study from a tertiary care public hospital in India estimated the impact of using FFR as an adjunct to angiogram in management of CAD. This study was used to create a mathematical simulation model to estimate cost-effectiveness and economic impact of using FFR over seven years’ time horizon, from the Indian health systems perspective. A targeted literature review was performed to collect the clinical inputs for the model, and the national public health insurance program data was referenced to obtain the cost inputs.

Results

A hypothetical cohort of 100,000 patients in the model reported 30 percent reduction in unnecessary stenting. Moreover, 14,025 deaths were averted with the adoption of FFR. In addition, there was a cost-saving of INR46,986 (574USD) per death averted and INR5,169 (63USD) per patient treated over a seven-year time horizon. The analysis demonstrated that FFR inclusion in the current clinical practice saves INR2,651 (32USD) per patient in overall upfront cost and INR2,518 (31USD) per patient in overall follow-up cost over a seven year follow-up period owing to improved diagnosis and prognosis.

Conclusions

In conclusion, FFR prevents unnecessary stenting, reduces overall mortality, and proves to be a cost-saving intervention in the long-term when used as a decision-making criterion in CAD patients in the Indian context.
PP65 An Economic Evaluation of Day Care Surgery For Non-Acute Hernia Repair In Government Hospitals Of Sri Lanka
Authors: Gamage Anuji , de Silva Amala , Higashi Hideki , Attygalle Deepika , Sridharan Sathasivam , Senanayake Lakshmen , Dias Ranjan , Dissanayake D M C D , Kim Hansoo
Published: December 2023

Introduction

Early discharge of publicly funded non-acute hernia repair patients may save healthcare costs by reducing inpatient stays. This study reports a cost analysis of establishing day-care surgeries for publicly funded patients undergoing non-acute hernia repair in Sri Lanka.

Methods

A decision tree model was developed to represent the pathway probabilities and costs. Cost data was taken from the Medical Supplies Division, relevant hospitals, and laboratories. Hospital costs per-day were calculated based on WHO-CHOICE model with inflation adjusted to 2022 value. The model assumed that 60 percent of the hernia patients presented to the outpatient department, 39 percent were referred from private clinics, and 1 percent of hernia repairs admitted as inward transfers or emergencies. Of the hernia repairs that were conducted, 95 percent were assumed to be uncomplicated hernias, and the most common post-operative compilation encountered was urine retention accounting for 95 percent of the complicated cases.

Results

It was estimated that in the current situation for a cohort of 1,000 patients undergoing non-acute hernia repair, 2,055 overnight in-hospital days were utilized. If day surgery services can be performed with patients observed for less than 24 hours before being discharged the overnight stay can be reduced to 155 patients. In the current scenario the total cost for non-acute hernia repair at a state hospital was estimated LKR170.9M (≈USD529K) per 1000 patients while the same procedure done as a Day Care procedure cost estimate was LKR155.7M (≈USD482K) per 1000 patients. The savings from implementing day-care surgeries for non-acute hernia repair will amount to approximately LKR15M (≈USD40K) when caring for 1,000 patients. The results were sensitive to length of stay and proportion of complicated cases.

Conclusions

Shifting uncomplicated non-acute hernia repair patients from an inward scenario to a day care scenario would lead to a considerable financial saving to the government. More evidence on the value of expanding day care services and observation services capacity should be explored as this would guide efficient and sustainable publicly funded healthcare system in Sri Lanka.
PP67 What Patients Want – Optimizing Oncology Value Assessment To The Goals Of Patients
Authors: Vines Richard
Published: December 2023

Introduction

Cancer is now diagnosed and treated earlier, resulting in improved patient benefit and outcomes. While overall survival (OS) is crucial to patients, there are other value dimensions, such as quality of life (QoL) and reduction in severe side effects, that change patient lives while on treatment. Considering patient reported outcomes (PROs) in value assessments for decision-making can improve individual, population and societal outcomes.

Methods

A multidisciplinary, international group of experts working in the cancer field was brought together to reach consensus on key principles of defining and assessing cancer treatment value. A Delphi-based approach including surveys, virtual panels, interviews and structured online discussions was used to reach consensus. This work was funded by AstraZeneca.

Results

Use of PROs in oncology value assessment is important because it can lead to: improvements in caregiver/patient/physician communication; unmet problem and needs detection; disease and treatment tracking; and better cost effectiveness. While some health technology assessment (HTA) bodies are already accepting QoL data, such as the Institute for Quality and Efficiency in Health Care (IQWiG) in Germany and US Centers for Medicare and Medicaid (CMS), many others do not. It is important that there is consistency in use of QoL data and other PROs, ensuring inclusion and a standardized and simple way of capture. In trials, tolerability data collected via PROs and QoL should be routinely and consistently incorporated and emphasized in HTA value assessments together with safety, efficacy and effectiveness. Data from PROs should be considered in decision-making to help build a better picture of health-related QoL, morbidity and adverse events from the patient perspective.

Conclusions

We are calling on the cancer community to: continue to encourage the use of a broad set of oncology-relevant endpoints in clinical trials to further validate their relevance as endpoints either as predictors of clinical outcomes or endpoints with intrinsic value; collect complementary PRO data in clinical trials and as real-world evidence to better tailor treatment options to the outcomes that individual patients value most; involve patients in HTA processes.
PP86 Systematic Review Toolbox 2.0: Rebuilding Toolbox With A Novel Taxonomy To Classify And Share Evidence Synthesis Tools
Authors: Marshall Christopher , Johnson Eugenie Evelynne , O’Keefe Hannah , Sutton Anthea
Published: December 2023

Introduction

Developed in 2014, the Systematic Review (SR) Toolbox has played a critical role in helping researchers to identify appropriate tools to support systematic reviews. Since the resource was launched, the systematic review and wider evidence synthesis process has evolved considerably. The way in which the SR Toolbox originally classified tools at launch had become dated. We updated and rebuilt the SR Toolbox in 2022 underpinned by a novel taxonomy to reflect the latest review and evidence synthesis landscape.

Methods

All guidance and software tools contained within the SR Toolbox were manually extracted in February 2022. Information contained from tool records were extracted by a single reviewer into an Excel spreadsheet, with a second reviewer checking a sample. The spreadsheet was translated to a Microsoft Access database underpinned with a new taxonomy to reflect the expansion of evidence synthesis methods and new review types (or ‘families’). A brief analysis of the remapped tools was conducted to identify current gaps in software and guidance support for evidence synthesis. A new user interface was also developed.

Results

The updated version of the SR Toolbox was launched 13 May 2022. At that time, the resource included records on 235 software tools and 112 guidance tools. Regarding ‘review families’, most software tools (n = 223) and guidance documents (n = 78) were applicable to supporting systematic reviews. Fewer software (n = 66) and guidance (n = 22) tools were applicable to reviews of reviews, while qualitative reviews were less served by guidance documents (n = 19). In terms of ‘review stages’, most guidance documents were associated with quality assessment (n = 70), while most software was related to searching (n = 84) and synthesis (n = 82). To-date, there is a lack of software (n = 2) and guidance (n = 3) tools to support stakeholder engagement.

Conclusions

The SR Toolbox has received a significant update to ensure that tools are classified and shared based on the latest systematic review and evidence synthesis methods. As part of the update, analysis of the contents of the toolbox highlighted potential gaps in tool support for certain review types/stages.
PP88 An Exploratory Analysis Of The Potential Cost-Benefit Of Delaying Kidney Disease Progression In Australia
Authors: Kim John , Dehle Francis , Teal Alex , Brydon Scott , Stevens Vanessa , Anselmo Anthony
Published: December 2023

Introduction

Chronic Kidney Disease (CKD) is a condition that leads to end-stage renal disease (ESRD), characterized by a gradual loss of kidney function. In 2021, the healthcare system expenditure of CKD in Australia was estimated to be over AUD2.3 billion (USD1.5 billion), largely attributed to Kidney Replacement Therapy (KRT, dialysis or kidney transplantation). This exploratory analysis aims to calculate the cost-benefit to the Australian healthcare system should KRT be delayed.

Methods

The prevalence of ESRD with and without KRT between 2016 and 2021 was estimated, and a simple linear regression model was created to estimate the prevalence of ESRD with KRT between 2022 and 2026. The projected cost of KRT management in 2022 was calculated, enabling an approximate cost benefit presented as the number of patients needed to reduce expenditure by AUD1 million (USD0.7 million).

Results

In 2021, it was calculated that 34,554 patients live with ESRD in Australia, of which 28,542 patients are on KRT. The number of new patients on KRT increases linearly by an average of 943 patients per year and provided a model with a strong goodness-of-fit (R2 = 0.99); predicting that the prevalence of patients on KRT is estimated to increase to 33,417 patients by 2026. Dialysis accounts for the highest cost associated with ESRD management, estimated to be AUD87,975/year/patient (USD58,253), and accounts for over AUD1.3 billion (USD0.9 billion) in annual expenditure. When considering the proportion of patients receiving KRT undergoing dialysis (52.6%), first-year renal transplant (3.4%), and post-kidney transplantation (43.9%), in 2022, the average annual cost per patient receiving KRT is estimated to be AUD57,565 (USD38,109). The prevention of KRT in 17.4 patients in 2022, decreasing to 15.4 patients in 2026, has the potential to save AUD1 million/year (USD0.7 million).

Conclusions

The prevalence of ESRD in Australia increases linearly and contributes to a significant cost to the Australian healthcare system. In 2022, preventing KRT in 17.4 patients (0.06%) can equate to a saving of AUD1 million/year (USD0.7 million), further decreasing to 15.4 patients (0.05%) in 2026.
PP93 Health Technology Assessments For Rare Diseases In Australia: A Case Study On Cystic Fibrosis
Authors: Jaiswal Himani , D’Ausilio Anna , Bending Matthew
Published: December 2023

Introduction

Currently, no cure exists for the 1 in 2,500 Australian babies born with potentially fatal cystic fibrosis (CF). The authors conducted a health technology assessment (HTA) case study analysis of all regulatory approved CF treatments in Australia from January 1994 to July 2022. Submissions were also made under the Therapeutics Goods Administration and Pharmaceutical Benefits Advisory Committee (TGA-PBAC) parallel process.

Methods

Public summary and source materials were researched to understand relevant clinical and health economic evidence requirements, and access decisions from Australia’s lead HTA body, PBAC.

Results

The review found that there are more than seven approved products in Australia. Of those, all four novel CF transmembrane conductance regulator (CFTR) modulating medications, which treat the underlying disease, received an orphan drug designation and were eventually listed. However, initial HTA decisions were mixed, with one recommended (25%), one not recommended (25%), and two deferred (50%). Clinical efficacy, cost-effectiveness, clinical need, as well as patient/carer-centric perspectives were most influential in HTA recommendations. Like other rare disease treatments, price, high incremental cost-effectiveness ratios (ICERs), uncertainty around cost-effectiveness and/or efficacy were key barriers to positive decisions. Notably, Australian stakeholders did not recommend CF medicines when their ICERs significantly exceeded a threshold of AUD200,000 (USD134,700) per quality-adjusted life year (QALY) gained. Administratively, Australia addresses risks associated with poor cost-effectiveness and high costs through managed access programs, risk-sharing agreements (RSA) and special pricing arrangements.

Recently approved elexacaftor-tezacaftor-ivacaftor would be inaccessible to many Australian patients without inclusion in the Pharmaceutical Benefits Scheme (PBS); this placement increases access by limiting patients’ payments to AUD42.50 (USD28.62) maximum per prescription. Alternatively, manufacturers of therapies for other chronic or rare life-threatening conditions can participate in Australia’s Highly Specialised Drugs Program and/or Life Saving Drugs Program to facilitate access.

Conclusions

Companies can accelerate and optimize market access by using the TGA-PBAC parallel process. Other Asia-Pacific countries can model components of Australia’s approach to advancing access to innovative, live-saving therapies.
PP94 Robotic-Assisted Thoracoscopic Surgery Versus Video-Assisted Thoracoscopic Surgery And Open Thoracotomy: A Systematic Review And Meta-Analysis
Authors: Lee Jiyeon , Jung Youjin
Published: December 2023

Introduction

Robot-assisted surgery is one of the minimally invasive surgical approaches that has been increasingly utilized across a wide range of surgeries. However, there is limited evidence of robotic-assisted thoracic surgery (RATS) for patients with lung cancer. This study aims to evaluate the safety and effectiveness of RATS in lung cancer patients compared with video-assisted thoracoscopic surgery (VATS) and open thoracotomy.

Methods

A comprehensive search for studies that compared RATS versus VATS or open thoracotomy published until 12 April 2022, was conducted. Two review authors independently assessed studies for inclusion and risk of bias, and extracted data. We used results of reported perioperative outcomes, oncological outcomes, and survival outcomes. When more than two studies contributed data, meta-analyses were performed.

Results

Four randomized controlled trials (RCT) were included. Firstly, three RCTs comparing RATS with VATS were identified. Compared with the VATS group, the RATS group had significantly lower blood loss, more harvested lymph nodes and lymph node stations. However, there were no significant differences in operative time, transfusion rates, hospital stay, drainage duration, reoperation, readmission, postoperative pain, and postoperative complications. Survival outcomes were not reported. Secondly, one RCT comparing RATS with open thoracotomy was identified. Compared with open thoracotomy group, the RATS group had significantly lower blood loss, less postoperative pain, and shorter chest drainage duration. On the other hand, there were no significant differences in operative time, hospital stay, postoperative complications, number of harvested lymph nodes and lymph node stations, and survival outcomes (disease-free survival, overall survival).

Conclusions

Evidence on the effectiveness and safety of RATS compared with VATS or open thoracotomy for lung cancer is of low certainty, but we suggest that RATS is a feasible and safe alternative to conventional thoracic surgeries for lung cancer patients on the basis of current data. Additionally, more and better studies are required to provide evidence on the benefits and cost-effectiveness of RATS.
PP96 Continuous Innovation In Neurostimulation Therapies For The Management Of Chronic Pain: Challenges For Health Technology Assessment Policy
Authors: Joglekar Rashmi , Strachan Liesl
Published: December 2023

Introduction

Chronic pain is a debilitating condition with a high burden of disease. Neurostimulation therapy is an established modality for patients with chronic pain refractory to pharmacological based approaches and conservative interventional therapies. The therapy has evolved over the decades, based on improved understanding of the mechanisms of action, as well as technological advancement in device design.

Our objective is to conduct a review of the innovation in neurostimulation therapy for chronic pain, in the context of health technology assessment (HTA), and its implications on policies related to patient access.

Methods

A qualitative literature review was conducted to identify published HTAs, systematic reviews, clinical guidelines and other relevant articles and reports on neurostimulation therapies used in pain management. Searches were limited to the past 10 years to ensure that a contemporary analysis was conducted.

Results

Our review indicates that there has been continuous innovation in neurostimulation therapies for chronic pain. This includes improvements in battery longevity and reduced size, advances in the design of leads, the development of novel stimulation waveforms and personalized programming using sophisticated algorithms including sensing and feedback loops, and remote management to name a few. Clinical research has also enabled an expansion in the range of neural targets and indicated subpopulations. The literature shows that apart from reduction in pain, neurostimulation therapy facilitates improvements in the quality of life, and reduction in opioid dependence, carer burden and disability, which are outcomes important to patients as well as to society at large. Clinical guidelines are largely supportive of neurostimulation for the management of chronic refractory pain in carefully selected patients.

Conclusions

The range and complexity of neurostimulation devices and the variety of study designs presents a challenge for evidence synthesis. HTA bodies need to ensure that the methodologies for evaluating a heterogeneous therapy such as neurostimulation for pain management are robust, and that the policies for determining access to such innovative therapies are patient-centric and fit-for-purpose.
PP102 Impact Of Placing Peripherally Inserted Central Catheters At Patient Bedside Versus Radiology Suite In A Private Australian Hospital
Authors: Xian Tan Kristin Hui , Gala Smeet , Ma Yan
Published: December 2023

Introduction

A peripherally inserted central catheter (PICC) is an important vascular access device to administer certain intravenous (IV) therapies, which is traditionally placed using fluoroscopy in radiology suites. With advancing tip-confirmation technology, PICCs can be placed at the patient bedside by nurses, without any delays arising from wait time for the radiology suite or the need of transferring patients to the radiology suite, leading to time savings and allowing prompt start to IV therapy. This study aims to estimate the cost and time impact of placing PICCs at the bedside using tip-confirmation technology led by nurses versus in the radiology suite using fluoroscopy by radiologists.

Methods

A budget impact analysis was developed using Microsoft Excel to estimate the annual impact of inserting PICCs at the bedside versus in the radiology suite. The base case scenario was modelled for 1,000 PICCs placed in a private Australian hospital. Impact on bed days, labor time and overall cost was estimated by using global and local data sources for inputs. It was assumed that 100 percent PICC are placed in a radiology suite in current practice, while 95 percent are placed at the bedside and 5 percent in the radiology suite in future practice.

Results

By shifting PICC insertion to the bedside using tip-confirmation technology, the model estimated a reduction of labor time by 221 hours and bed days by 113 days. Despite an increase in the cost of consumables by AUD34,041 (USD22,760) and reduction of Medicare Benefits Schedule rebate by AUD260,730 (USD174,328), overall cost savings of AUD1.01million (USD675,660) was observed due to significant savings due to the t reduced utilization of the radiology suite.

Conclusions

PICC insertion at the patient bedside using tip-confirmation technology by nurses may lead to time and cost savings as compared to placing them in the radiology suite. This can help alleviate the burden on radiology suites and reduce their wait times, potentially leading to timely treatment initiation and discharge. Since PICCs at the bedside are typically placed by specialized vascular access nurses, these cost savings can be redirected to employ and train them.
PP109 The Use Of Health Technology Assessment In Decision Making: Evidence From The Balkan Countries
Authors: Mechili Enkeleint A. , Petelos Elena , Bucaj Jorgjia , Gallos Parisis
Published: December 2023

Introduction

According to the most recent definition health technology assessment (HTA) “is a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The purpose is to inform decision-making in order to promote an equitable, efficient, and high-quality health system”. This article aimed to evaluate implementation of HTA in decision making in the Balkan countries.

Methods

A scoping review of the existing literature took place to locate relevant scientific articles, policy papers and documents released by the respective Ministries. We searched data for 6 Western Balkan countries (Albania, Bosnia and Herzegovina, Kosovo, Montenegro, North Macedonia and Serbia) and didn’t focus on those countries that are part of the European Union (EU). For the literature search key words were used, while documents only in English were included in the analyses. Additionally, the search was conducted for the period January 2010 until October 2022.

Results

The Western Balkan countries are in process of integration to the EU and based on this they are trying to make improvement in different sectors including health services. However, the use of HTA in most of the studied countries is in its preliminary phase. Most of the countries have established HTA bodies or specific authorities but with limited resources (both human and financial). Additionally, their reports are non-binding for policy makers and healthcare decisions are taken based on experts’ opinions and not an extensive HTA analyses.

Conclusions

Despite their efforts, the Western Balkan countries need to improve and considerably increase the use of HTA in decision-making. Its use can help in provision of better healthcare services as well as to decrease costs. Specific attention should be put on human and financial resources that are lacking in all settings.

Western Balkan countries need to put much more efforts for harmonization of their legal framework with that of the EU countries.
PP113 Common Methodological Issues In Systematic Reviews Supporting Single Technology Appraisal Submissions To The National Institute for Health and Care Excellence
Authors: Fitzgerald Anita , Wilson Katy , Wood Hannah
Published: December 2023

Introduction

This project aimed to identify methodological issues reported by Evidence Review Groups (ERGs) in the systematic reviews (SR) supporting single technology appraisal (STA) submissions to the National Institute for Health and Care Excellence (NICE). STA submissions contain SRs related to the clinical and cost-effectiveness of the proposed intervention and NICE require the methods for these reviews to be clearly detailed in the submission. The intention of this project was to identify methodological aspects of submissions where companies may need additional guidance or support to provide the evidence required for efficient and effective decision-making and in turn facilitate timely access to clinical innovation.

Methods

From 2019, 61 STAs were identified from the NICE website, of which 46 were included. We extracted information about the data requests or clarification questions raised by the ERG in relation to the methodological section of both the clinical and cost effectiveness SRs reported in the STA. We then categorized these data and grouped by theme to determine the most common methodological issues faced by companies. We did not assess whether comments made by the ERG were accurate or justified.

Results

For both clinical and cost-effectiveness SRs, the most frequent clarification questions arose from the search methods section, specifically seeking information about missing intervention or comparator terms, the use of search filters and search platforms. Clarification questions were also commonly asked about the appropriateness of interventions and comparators. There were very few clarification questions asked about screening, data extraction or risk of bias assessment.

Conclusions

Companies looking to submit an STA should align their submission methodology to established best practice guidance for systematic reviewing to ensure their methods are fit for purpose and avoid unnecessary delays to the STA process. Consistency with the PRISMA reporting standards would help ensure that the ERG is provided with the information needed to assess the appropriateness of the STA methodology and is likely to reduce the need for clarification questions.
PP130 The Effectiveness Of Extracorporeal Shock Wave Therapy For Plantar Fasciitis: A Systematic Review And Meta-analysis
Authors: Jung Youjin , Lee Seul Ki
Published: December 2023

Introduction

Extracorporeal shock wave therapy (ESWT) has been used since the 1990s to treat various musculoskeletal disorders, but there is considerable controversy regarding the effectiveness of ESWT. Our aim was to conduct a systematic review of randomized controlled trials (RCT) to investigate the effectiveness of ESWT for plantar fasciitis.

Methods

A comprehensive search was conducted via electronic databases including MEDLINE, Embase, the Cochrane Controlled trials register, and 5 Korean databases from inception date to April 2022. Two review authors independently assessed studies for inclusion and risk of bias, and extracted study data. Major outcomes were pain relief, function, and quality of life.

Results

We identified a total of 48 RCTs comparing ESWT with corticosteroid injection (n=14), conventional therapies (n=19), and sham control (n=21). Most studies included participants with chronic heel pain diagnosed as plantar fasciitis. All trials were susceptible to bias. In terms of pain results, ESWT showed no significant difference when compared with the steroid injection group and the conventional therapy group, and significant pain relief was confirmed only compared to the sham control group (Mean Difference -1.71; 95% confidence interval [CI] -2.44,-0.98; I2=70%;). Functional outcomes were significantly improved in the ESWT group compared to the steroid injection group (standardized mean difference 0.45; 95% CI 0.27,0.63; I2=0%) and the sham control group (SMD 0.84, 95% CI 0.23,1.45; I2=91%), but no significant difference was found when compared to the conventional therapy group.

Conclusions

Based upon the currently available low certainty evidence because of wide clinical diversity and varying treatment protocols of included trials, ESWT is associated with improved function and may be associated with pain reduction in plantar fasciitis. Further evidence is needed from well-designed studies with a standard dose and treatment protocol.
PP132 Health Technology Assessment Agencies’ Expectations Regarding Patient Experience Data in Australia, China, And Japan
Authors: Alonzo Carolina , Ding Ding , Poon Jiat-Ling , Zschocke Juergen , Zhang Lei , Toshihiko Aranishi , Myrick Shane , Hill Jennifer , Larkin Louise , Perez Nancy , Delbecque Laure
Published: December 2023

Introduction

Health technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to review HTA agencies’ expectations with regards to utilizing PED to support drug reimbursement in Australia, Japan, and China.

Methods

Published HTA guidance documents were reviewed in 2021 to identify any PED-specific information. If available, recommendations related to the type of PED (e.g., generic vs. disease-specific clinical outcomes assessment (COA)); COA validation, analyses, endpoints and interpretation; and the interest in PED beyond COA in HTA decision-making (e.g., patient preference information) were reviewed. Literature review and semi-structured interviews with key opinion leaders (KOLs) were conducted to further explore these themes and future trends with regards to PED.

Results

Australia’s Pharmaceutical Benefits Advisory Committee guidance document includes a dedicated section on patient-reported outcomes (PRO), providing details on preferred PRO instruments; validation expectations; and recommended methods to explore score interpretation, assess and report PRO results and handle missing data. While PED derived from non-PRO sources are not discussed in the guidance, the KOL noted that they should not be ruled out. Japan’s Center for Outcomes Research and Economic Evaluation for Health guideline includes a section dedicated to PROs without details related to instrument validation, analyses and interpretation, however, is focused on the use of PRO to inform health economic assessments. In China, the HTA center of China National Health Commission drafted two disease-specific technical guidance documents recommending the inclusion of PROs in efficacy assessments and use of instruments relevant in the Chinese population; these points were echoed by the KOL interviewed.

Conclusions

There are recommendations on PED use included in country-specific guidance documents, however their level of detail varies greatly. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development.
PP133 What Services And Products Should A Health Technology Assessment Agency Provide?
Authors: Faraldo-Valles Maria-Jose , Maceira-Rozas Maria-Carmen , Casal-Acción Beatriz , Gomez Patricia , Trinanes Yolanda
Published: December 2023

Introduction

Health technology assessment (HTA) bodies support healthcare decision-making by producing different kind of products. The high speed of the healthcare innovations and the scenarios such as the COVID-19 pandemic challenge HTA organizations to adapt their services to better respond to these demands. The Spanish Network of HTA Agencies (RedETS) is redefining the services and the products in its portfolio. The first step has been conducting a review in order to identify the most relevant HTA products.

Methods

A scoping review with two sections was conducted: (i) analysis of results from a bibliographic search performed in the main biomedical databases; and (ii) analysis of results from a manual review of the official websites of seven international HTA agencies: CADTH (Canada), INESSS (Canada), SBU (Sweden), NICE (United Kingdom), IQWIG (Germany), HAS (France), IECS (Argentina) and IETS (Colombia). The EUnetHTA website was also reviewed.

Results

The search identified 1,311 references; 21 studies were considered relevant. The main topic found was about rapid responses services. The standard timeline for these should be less than six months, with even some produced in days. Transparency about methodology and involvement of decision-makers were considered key points to be included. Website analysis revealed similar HTA reports production but variation in the domains and elements considered. The timeframe for conducting a full HTA report can be up to 24 months, with a median of 12 months. Agencies also offer some kinds of rapid response services. Scientific consultation and horizon scanning systems for emerging technologies are other services performed by some agencies.

Conclusions

The review reveals that agencies have different products to address different needs throughout the life cycle of technologies: from scientific advice to full HTA. In addition, HTA agencies have incorporated rapid responses into their services. According to literature, these products could support short-term decision-making.
PP135 The Cost-Of-Illness Of The Management Of Patients Affected By Hyperparathyroidism In The Italian Healthcare Setting
Authors: Basile Michele , Antonini Debora , Fortunato Agostino , Cicchetti Americo
Published: December 2023

Introduction

Hyperparathyroidism is a medical condition characterized by an excess of parathyroid hormone (PTH) in the blood. This hormone is produced by the parathyroid glands, located behind the thyroid gland. PTH plays a crucial role in regulating calcium levels in the body, which is necessary for muscle activity, nerve transmission, and maintaining bone density. In this study, the authors aimed to determine the parameters necessary for defining the delivery process of interventions for managing hyperparathyroidism.

Methods

To achieve this, a group of Key Opinion Leaders (KOLs) was surveyed using a questionnaire to investigate specific drivers such as the duration of interventions, drug therapies employed, and materials required. The authors also estimated the indirect costs associated with patients and caregivers. The economic analysis considered the perspectives of both the Italian National Health Service (SSN) and the community. The analysis was conducted using Activity Based Costing (ABC) methods to determine the full cost sustained for a parathyroidectomy surgery and pharmacological therapies per patient treated, as well as the average resources absorbed by a patient managed through pure surveillance.

Results

The results showed that the average annual cost of parathyroidectomy and pharmacological therapies per patient was EUR5,193.20 and EUR1,726.96, respectively. Productivity losses due to the interventions amounted to EUR858.21 and EUR66.80 for the patient and caregiver, respectively. The pure surveillance strategy incurred an average yearly cost of EUR197.42.

Conclusions

The study’s major limitation was the lack of evidence available concerning the therapies under analysis, particularly in the Italian context. Nonetheless, the survey of clinicians provided useful insights into the expenditures associated with implementing interventions for managing hyperparathyroidism. In conclusion, managing hyperparathyroidism involves considerable costs, and healthcare providers must consider the perspectives of both the SSN and the community when estimating the economic impact of interventions. The authors’ analysis provides insights into the cost of different interventions, which could help healthcare providers make informed decisions when managing hyperparathyroidism.
PP140 Burden of Illness And Health Care Costs In People with Alzheimer’s Disease
Authors: Edwards Sophie , Hahn-Pedersen Julie , Robinson Danielle , Chan Mei Sum , Bray Benjamin , Clark Alice , Ivkovic Milana , Michalak Wojciech , Wichmann Christian , Berry Sasha , Evans Marc
Published: December 2023

Introduction

Alzheimer’s disease (AD), the most common cause of dementia, is becoming increasingly prevalent worldwide. Understanding the current burden of AD is important in health economic evaluations of new therapies. We aimed to estimate the burden of illness, and healthcare costs of people living with AD using a large, comprehensive real-world database in England.

Methods

A retrospective cohort study was undertaken in the Discover-NOW dataset, a real-world database containing the linked primary and secondary care electronic health records of ˜3 million people living in North West London, England. Patients diagnosed with AD were followed from the later of 1 January 2010 or AD diagnosis date, to the earlier of 31 December 2021 or end of follow up (maximum 10 years). Baseline prevalence of 33 comorbidities, incidence of 7 outcomes (survival, cardiovascular, care home admission, hepatic and renal outcomes), healthcare resource utilisation and total direct healthcare costs (using National Health Service tariffs and unit cost approaches) were calculated.

Results

Of 18,116 patients diagnosed with AD, at baseline the mean age was 81 years, 62 percent were female, 65 percent were White, 16.5 percent Asian and 8.9 percent Black. At baseline, hypertension prevalence was 60.2 percent, chronic kidney disease 35.5 percent and Type 2 diabetes 22.4 percent. The highest incidence rates across these outcomes were 13.4 (95% confidence interval [CI]:12.2,14.7) per 1,000 person years for stroke, 7.5 (95% CI: 6.6, 8.5) for myocardial infarction, and 83.6 (95% CI: 80.1, 87.0) for care home admission. Median survival was 4.9 years from diagnosis. Their annual total direct healthcare cost was GBP4,547 per patient, of which 58 percent were from hospital admissions. The majority (75%) of healthcare contacts were from primary care. AD patients had an average length of stay of 11.5 days per inpatient admission, and spent on average one week per year as inpatients.

Conclusions

AD is associated with high direct healthcare costs, with patients’ annual costs ˜1.7 times that of the UK population. The majority of these costs are associated with inpatient hospital admissions.
PP146 Seeing Eye-To-Eye on Real-World Evidence: Are Guidance from Japan and China Consistent with Recommendations from REALISE in Asia?
Authors: Wee Yan Ran , Fujita Natsumi , Evans Jennifer S
Published: December 2023

Introduction

The REAL World Data (RWD) In Asia for Health Technology Assessment (HTA) guidance was developed by a regional working group to facilitate the increasing acceptance of real-world evidence (RWE) in Asia. We compared the consistency of REALISE against guidance from Japan and China.

Methods

Country-specific guidance for RWE/RWD use in pharmaceutical development were identified in May 2022 through governmental websites, with validation searches via Google. Sections from local guidance were mapped onto REALISE and categorized as “agree”, “mixed”, “disagree” or “missing” based on coverage and consistency.

Results

Five Japanese and three Chinese documents were mapped. Most sections in Chinese guidance (77%) and 36 percent of sections in Japanese guidance were tagged “agree” or “mixed”, with general alignment on definitions and good practice considerations (study design, accountability); however, 63 percent of Japanese sections were tagged “missing” from REALISE. As local documents took the regulatory perspective, they lacked REALISE’s discussion of translating RWD to RWE for HTA/economic evaluations specifically. Local guidance focused on practicalities of RWD collection in local contexts, including descriptions of specific actions (e.g., evaluating RWD sources, ensuring data security) rather than overarching principles described in REALISE; specifically, Japanese guidance described how to access and analyze databases/registries, reflecting Japan’s landscape of robust sources of national healthcare data, but lacked discussion of other RWE study types, data sources and specialized analytical methods. While Chinese guidance had a broader view of RWD types (more similar to REALISE), they also contained discussions on pharmacovigilance and omics data, communication with regulatory bodies, and incorporation of RWE into the approval pathway for traditional Chinese medicines.

Conclusions

Despite differing purposes (with no RWE guidance from local HTA bodies), local and regional guidance align on general principles/good practice in generating/using RWE, providing common ground for increasing usage of RWE in HTA in Asia.
PP152 Evaluation Of Reimbursement Periods In The Turkish Biosimilar Product Market (1995-2022)
Authors: Deger Sena Unluler , Vural Elif Hilal
Published: December 2023

Introduction

Biosimilar products that would enhance the patient’s access to treatments have emerged as a product group that is becoming more widespread globally. Since Turkiye is an emerging market in the pharmaceutical area in the Middle East and North Africa region, and being a reference country in pricing processes for some countries, the number of products in the market and reimbursement decisions are important. The aim of this study is to evaluate duration of inclusion of biosimilars into reimbursement lists after registration in Turkiye.

Methods

This study used website-based information (Turkish Medicine and Medical Devices Agency Registered Products List and Detailed Pharmaceutical Price List, Social Security Institution Reimbursed Product List) to analyze the reimbursement approval duration for registered biosimilars in Turkiye after receiving registration approval. A study has been conducted on the launch period of biosimilar products to patients access with reimbursement and the evaluation period of reimbursement applications after registration approval. Even though they might not be active on the reimbursement list right now, products that have previously been approved for payment have been included.

Results

Between 1995-2022, biosimilars of 13 active substances in total were registered by the Ministry of Health in Turkiye. Thirty-three different brands and 105 biosimilars with all pharmaceutical forms are registered. As of November 2022, 72 biosimilars were in the reimbursement list. Twenty-two of reimbursed biosimilars were deactivated or excluded from reimbursement. It is calculated that the average evaluation and approval timeline for reimbursement of biosimilars between 2009 and 2022 is 9 months. When biosimilars in the European Medicines Agency (EMA) and Turkiye are compared, 13 biosimilar active substances are licensed in Turkiye compared to 19 for the EMA. When the total number of brands is compared, it has been observed that while 33 brands are registered in Turkiye, this number is 73 for the EMA.

Conclusions

This study, in which reimbursement approval dates for registered biosimilars in Turkiye have been compared based on years and access timelines of biotechnological products, shows that biosimilars have been launched to patients access with reimbursement much faster than biotechnological products.
PP153 Efficacy And Safety of Onasemnogene Abeparvovec For The Treatment Of Patients With Spinal Muscular Atrophy Type 1: Meta-Analysis
Authors: Borges Stefani , Fernandes Brígida , Rodrigues Fernanda , Krug Bárbara , Cirilo Hérica Núbia , Schwartz Ida Vanessa , Probst Livia , Zimmermann Ivan
Published: December 2023

Introduction

Onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q (SMA) type 1 in several countries, which calls for an independent assessment of its evidence regarding efficacy and safety.

Methods

This study results from searches conducted on databases MEDLINE, Embase, LILACS and Cochrane Library up to November 2022, supported by additional searches on registry databases and by manual searches of references listed in eligible studies. Outcomes of interest were global survival and mechanical-ventilation-free survival, improvement in motor function and treatment-related adverse events. Risk of bias was assessed via ROBINS-I and certainty of evidence via GRADE. Proportional meta-analysis models were performed when applicable.

Results

Four reports of three open-label, non-comparative clinical trials (START, STR1VE-US and STR1VE-EU) covering 67 patients were included in review. Meta-analyses of data available in a 12-month follow-up estimate a global survival of 97.6% (95% confidence interval [CI]: 92.6, 99.9; I2 = 0%, n=67), an event-free survival of 96.5% (95%CI: 90.8, 99.5; I2 = 32%, n=66) and a CHOP-INTEND score of 40 points or less proportion of 87.3% (95%CI: 69.8, 97.8; I2 = 69%, n=67). Proportions of 61.1% (95%CI: 40, 80.2; I² = 62%, n=67) of serious adverse events and of 58.4% (95%CI: 46.5, 69.8; I2 = 78%, n=67) of treatment-related adverse events are estimated. Despite the significant effect magnitude, reviewed studies were assessed as high risk of bias and as having very low certainty of evidence due to imprecision and risk of bias.

Conclusions

Reduced sample size and follow-up time offer uncertainties as regards the long-term benefits of the gene therapy, which strongly calls for the monitoring and assessment of results in clinical practice.