bluuebunny/crossref_metadata_2025 huggingface dataset:
OP38 Evaluation Of A High-Cost Medicine For A Rare Disease: 16-Year Cohort Of Imiglucerase Use For Gaucher Disease In Brazil
Authors: Borin Marcus Carvalho , de Assis Acúrcio Francisco , Alvares-Teodoro Juliana , Guerra Junior Augusto Afonso
Published: December 2023

Introduction

Gaucher disease is a lysosomal storage disease of autosomal recessive inheritance that is caused by a deficiency of the enzyme glucocerebrosidase. This deficiency results in accumulation of the enzyme’s main substrate in the lysosomes of macrophages, mainly in the spleen, liver, and bone marrow. In more severe cases it can affect the lung, kidneys, and central nervous system. There are two main treatments available for patients with Gaucher disease: enzyme replacement therapy and inhibition of substrate synthesis. The main enzyme replacement therapy used in Brazil is imiglucerase, an analog of the human β-glucocerebrosidase enzyme. Imiglucerase is produced by recombinant DNA technology using a cell culture derived from the Chinese hamster ovary. It has 497 amino acids and differs from the endogenous enzyme by an amino acid at position 495, where histidine is replaced by arginine. The objective of the study was to analyze the survival of patients treated for Gaucher disease with imiglucerase in Brazil from 2000 to 2015.

Methods

We constructed a retrospective cohort study of patients with Gaucher disease who received imiglucerase through the Brazilian National Health System from 2000 to 2015 using a national database created from the linkage of administrative databases.

Results

A total of 1,241 patients who received imiglucerase were included. The overall survival rates at one, ten, and 15 years were 98.7 percent (95% confidence interval [CI]: 98.1, 99.4), 92.3 percent (95% CI: 90.2, 94.4), and 89.4 percent (95% CI: 85.6, 93.3), respectively.

Conclusions

Our findings advance the understanding of the profile, survival, and risk factors of people with Gaucher disease, adding new data to the discussion regarding pharmaceutical therapies and patient care, and providing data for the development of new public health policies for the use of advanced, high-cost drugs for rare diseases.
OP43 Translating Cell And Gene Therapy HTA Into Practice – Building The Plane As We Fly It
Authors: Fennessy Paul , Ferrie Monica
Published: December 2023

Introduction

Assessing, funding, and implementing cell and gene therapies are usually based on limited evidence. This requires health technology assessment (HTA) agencies to develop new methodologies; payers to accept risk-based funding; industry to consider the right evidence and price; hospitals to consider if they have the necessary requirements; patients to consider their risk appetite for gene therapies; and health departments to consider all the above. Ensuring timely patient access to these therapies is challenging in Australia’s federated health system. To ensure stakeholders are aligned, all must come to the table and share their respective insights, experiences, and expertise to support planning, decision-making, funding, and commissioning of these therapies. It is time to develop a new consultative and decision-making paradigm to expedite HTAs, funding, commissioning, and timely patient access for cell and gene therapies.

Methods

Australian federal, state, and territory government representatives agreed to develop a framework that clarifies processes around information sharing, HTA, funding, commissioning, and monitoring of highly specialized therapies and services (including cell and gene therapies). A draft national framework was developed that addresses assessment, funding, and implementation of high cost, highly specialized therapies and services (e.g., bone marrow transplants). However, it is unclear whether non-government stakeholders have been consulted.

Results

The framework for assessing, funding, and implementing high cost, highly specialized therapies and services across Australia’s public hospital system is pending endorsement by each of the jurisdictional governments. High-level in nature, the framework’s primary audiences are industry and public hospitals. While not all processes are in place, the framework is forward-looking. A detailed implementation plan is warranted to better inform the roles and requirements of each stakeholder.

Conclusions

The framework allows stakeholders to better understand government processes regarding assessment, funding, and implementation of high-cost therapies and services, thereby fostering a collaborative environment that supports timely patient access. Articulating process details in a follow-up implementation plan is essential to gain the trust of, and input from, industry, clinicians, and patient representatives.
OP59 Do Digital Health Terms Provide Sufficient Information To Allow For Health Technology Assessment?
Authors: Zrubka Zsombor , Annette Champion , Di Bidino Rossella , Holtorf Anke-Peggy , Earla Jagadeswara R , Boltyenkov Artem , Tabata-Kelly Masami , Asche Carl Victor , Burrell Anita
Published: December 2023

Introduction

Umbrella digital health term (DHT) (digital health, eHealth, mHealth, telehealth, and telemedicine) definitions contain insufficient information about patient value for health economics and outcomes research and health technology assessment (HTA) purposes. Qualitative content analysis of secondary DHT (e.g., telesurgery and teleradiology) definitions was performed by the ISPOR Digital Health Special Interest Group to determine if they were more useful for health economics and outcomes research purposes.

Methods

Secondary DHT definitions were extracted from a previous scoping review and consolidated by reviewer pairs using uniform rules. Definitions were analyzed for explicit (directly stated) or implicit (inferred) information on 24 categories: Patient, Intervention, Comparator, Outcome, Timing, Setting (PICOTS); the Shannon-Weaver communication model (SWE) (sender, message, encoder, channel, decoder, and receiver, extended with mode of information exchange); the quality domains of Agency for Healthcare Research and Quality (AHRQ) (safe, effective, patient-centered, timely, efficient, and equitable); information related to applied technology or geographic scope; and the World Health Organization (WHO) classification of digital health interventions v1.0 (digital health interventions category, health system challenges, and system categories).

Results

Across 107 unique definitions of 73 secondary DHTs, the number of explicitly or implicitly addressed categories across the frameworks ranged from zero to 15, with references to elements of PICOTS (79.4%), SWE (90.7%), AHRQ (30.8%), applied technology (52.3%), geographic scope (0%), and WHO frameworks (86.9%). PICOTS information was found for Patients in 35 percent of definitions, Intervention in 59 percent, Comparator in 20 percent, and Outcomes in 18 percent.

Conclusions

Secondary DHT definitions do not adequately specify PICOTS or other characteristics of interest for HTA. An online Delphi survey has been launched among a wider group of ISPOR members to identify the minimum information set to define patient facing DHTs for evidence summaries and value assessments. The results of this research should be shared for discussion with other digital health stakeholder groups.
OP71 Road To Public Funding Of Cancer Codependent Technologies In Australia In The Last Ten Years
Authors: Gao Yuan , Laka Mah , Merlin Tracy
Published: December 2023

Introduction

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

Methods

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

Results

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

Conclusions

Most cCDTs obtain funding support, but only after multiple resubmissions to PBAC and MSAC. Polarized decisions are rare. Reasons for rejection primarily relate to uncertain clinical benefit and an unacceptably high incremental cost-effectiveness ratio.
OP78 Cost-effectiveness Of A 20-valent Pneumococcal Conjugate Vaccine To Directly Protect Adults Against Pneumococcal Disease In England
Authors: Mendes Diana , Averin Ahuva , Atwood Mark , Sato Reiko , Vyse Andrew , Campling James , Weycker Derek , Slack Mary , Ellsbury Gillian , Mugwagwa Tendai
Published: December 2023

Introduction

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

Methods

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

Results

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

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

Conclusions

Our findings support replacing PPV23 with PCV20 to directly protect adults against pneumococcal disease, reducing hospitalizations and saving lives in the UK.
OP81 Cost Effectiveness Of Human Papillomavirus Extended Genotyping For Cervical Cancer Screening In Singapore
Authors: Chua Brandon , Lim Li Min , Ng Joseph , Ma Viva , Jaime Caro J. , Wee Hwee Lin
Published: December 2023

Introduction

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

Methods

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

Results

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

Conclusions

XGT can provide a cost effective, risk-based approach to primary cervical cancer screening, with lower resource utilization than PGT.
OP122 Economic Evidence To Support Expanding Use Of Existing Positron Emission Tomography Technology As A Diagnostic Tool For High-Risk Cancer Patients
Authors: Tew Michelle , Douglas Abby , Szer Jeff , Bajel Ashish , Harrison Simon , Tio Shio Yen , Worth Leon , Hicks Rodney , Ritchie David , Slavin Monica , Thursky Karin , Dalziel Kim
Published: December 2023

Introduction

In Australia, 18F-fluorodeoxyglucose positron emission tomography with low-dose computed tomography (FDG-PET/CT) is currently only funded for cancer staging-related indications. A recent multicenter randomized trial demonstrated that FDG-PET/CT, compared with standard of care computed tomography (CT) imaging, improved antimicrobial management and the outcomes of patients with persistent and recurrent neutropenic fever. There is potential value in expanding the use of FDG-PET/CT as a diagnostic tool for this high-risk population. We conducted an economic evaluation from a healthcare perspective alongside the randomized trial and compared FDG-PET/CT with standard CT up to 6 months after the scans.

Methods

Case report forms were used to collect resource utilization data and length of hospitalization. Effectiveness was measured as the number of patients with antimicrobial rationalization and quality-adjusted life-years (QALYs) derived from patient-reported trial-based health-related quality of life. Generalized linear models (GLM) were used to analyze costs and outcomes. Incremental cost-effectiveness ratios (ICERs) for each of the outcomes were calculated and interpreted as the cost per patient with antimicrobial rationalization and cost per QALY gained. To account for sampling, we performed bootstrapping with 1,000 replications using the recycled predictions method.

Results

The adjusted healthcare costs were lower in the FDG-PET/CT group (mean AUD49,563, 95% confidence interval [CI]: 36,867, 65,133; equivalent to USD34,268, 95% CI: 25,490, 45,033) compared with the standard CT group (mean AUD57,574, 95% CI: 44,837, 73,347; equivalent to USD39,807, 95% CI: 31,000, 50,712). The magnitude of differences in QALYs between the two groups was small (0.001; 95% CI: -0.001, -0.001). When simulated 1,000 times, our analysis showed that across both outcomes FDG-PET/CT was the dominant strategy as it was cheaper and had better outcomes than standard CT in 74 percent of simulations.

Conclusions

FDG-PET/CT is cost effective when compared with standard CT for investigating persistent or recurrent neutropenic fever in high-risk patients. Aligning economic evaluations with clinical studies is key to an integrated evidence generation approach for supporting funding for FDG-PET/CT in this patient group.
OP124 Cost Effectiveness Of End-Stage Renal Disease Treatment Methods In Türkiye
Authors: Kılıç Mustafa , Yigit Vahit
Published: December 2023

Introduction

Chronic kidney disease is an important public health problem and is a leading cause of morbidity and mortality worldwide. Hemodialysis (HD), peritoneal dialysis (PD), and kidney transplantation (Tx) are the main treatments for this disease. The aim of this research was to determine the cost effectiveness of treatments for end-stage renal disease from the perspective of a reimbursement institution in Türkiye.

Methods

A Markov model was developed to measure costs and health outcomes in terms of quality-adjusted life-years (QALYs). The model parameters were based on a six percent discount rate, lifetime time horizon, and a reimbursement agency perspective. The main outcome measures were the incremental cost-effectiveness ratio (ICER) and the cost per QALY. One-way and probabilistic sensitivity analyses were performed to determine parameter uncertainty.

Results

The lifetime costs of HD, PD, and Tx were USD26,883, USD37,672, and USD31,227, respectively. The lifetime QALYs gained with HD, PD, and Tx were 5.21, 6.77, and 9.73, respectively. The cost per QALY of HD, PD, and Tx were USD5,161, USD5,567, and USD3,211, respectively. Compared with Tx, the ICERs for HD and PD were USD961 and USD2,178, respectively.

Conclusions

Cost differences have occurred between the treatment options for end-stage renal disease due to the increase in drug costs in Türkiye in recent years. As seen in the Markov model in this research, HD, PD, and Tx are complementary rather than rival treatments. This study found that the cost effectiveness of Tx is higher than HD or PD. However, the rate of Tx, which has a higher quality of life compared with HD, is around 22 percent in Türkiye; the rate for PD is four percent. It is therefore recommended that a health policy be developed to encourage kidney donation and promote PD as a superior alternative to HD for eligible patients.
OP138 Navigating High-Cost Medicines: Promoting Consistent, Evidence-based Use Of High-Cost Medicines In A Fiscally And Equitable Responsible Manner
Authors: Pulver Lisa , Barclay Peter , Bennett Dr Sasha , Burgess Naomi , Dartnell Jonathan , Drake Catherine , Laba Tracey-Lea , Liew David , Mason Kylie , Melocco Terry , O’Reilly Mary , Sheahan Linda , Subramaniam Kavitha , Hill Catherine
Published: December 2023

Introduction

Hospitals play a significant and important role in funding high-cost medicines so patients can access treatments they need. High-cost medicines are often specialty medicines, which contribute to a significant and increasing portion of the hospital budget. It is imperative that these expensive medicines are governed and managed with a fair, standardized evidence-based process. We aim to provide a framework for Drugs and Therapeutics Committees (DTCs).

Methods

During 2021, Guiding Principles were developed following a literature review and survey of current practices by DTCs in Australia. An Expert Advisory Group (EAG) was convened, comprising individuals with expertise in quality use of medicines, evidence-based medicine and medicines governance. The guiding principles were drafted by the EAG, in consultation with a range of stakeholders and relevant external organizations. All feedback was collated, reviewed and discussed to refine the content of the final Guiding Principles released in January 2022.

Results

Seven overarching principles provide key recommendations for the governance of high-cost medicines:

(i)

A definition of high‑cost medicines should be determined and clearly articulated for use by each medicines governance committee.

(ii)

Review of high-cost medicines requires members with relevant expertize to facilitate good and effective decision-making.

(iii)

The committee should engage directly with the applicant prior to review to ensure a full understanding of the rationale for the request.

(iv)

consistent, robust and transparent procedure for the assessment of high-cost medicine applications should be defined and implemented for use by each medicines governance committee to ensure fair process.

(v)

Ethical considerations fundamentally underpin deliberations around high-cost medicines.

(vi)

The decisions and outcomes of the decision making should be transparent and appropriately communicated to the various audiences.

(vii)

The high-quality assessment of high-cost medicines requires appropriate training and resourcing.

Conclusions

These national Guiding Principles promote consistent, evidence-based use of high-cost medicines and provide a framework for DTCs to assess and achieve effective governance for the quality use of high‑cost medicines.
OP148 Influence Of The Hospital-Based Health Technology Assessment Unit On New Technologies Transfer At The National Level In Kazakhstan
Authors: Avdeyev Andrey , Saduakassova Aigul , Tleulessova Indira , Akhmedullin Ruslan , Lyugay Ekaterina , Fet Maхim , Albayev Rustam , Benberin Valeriy , Shanazarov Nasrulla , Okesh Makhabbat , Aimanova Tansolpan , Akhmetova Makpal , Bariyeva Gulzada , Turar Olzhas
Published: December 2023

Introduction

The hospital-based Health Technology Assessment (HB-HTA) Unit in the Hospital of the President’s Affairs Administration has been operating since 2015 and is the first example of the implementation of the HB-HTA system in Kazakhstani hospitals. In addition to assessing the feasibility of implementing new health technologies (HTs) into the hospital’s practice, the Unit also interacts with the National Regulatory Authority (NRA) to transfer new technologies into the National Reimbursement System (NRS). We report on the transformation of the HB-HTA Unit from a stand-alone entity to an integrated, specialized agency.

Methods

Data were drawn from reports of the HB-HTA Unit and internal NRA documents. Inclusion of new HTs into the NRS consists of the following sequential stages: (i) implementing the technology in at least one hospital; (ii) filing an application with the Ministry of Health Care (MoH) in the field of HTA to resolve the issue of reimbursement at the national level; (iii) in case of a positive decision, approval at the national level of the clinical protocol for using the medical intervention; (iv) agreement on the reimbursement price of the technology.

Results

Based on positive recommendations from the HB-HTA report in 2015, the hospital implemented 19 new nuclear medicine technologies. In 2016, the hospital initiated an application to the MoH to include these technologies in the NRS (previously, these technologies were carried out only for a fee or with private insurance). From 2016 to 2020, a positive decision from the MoH was received, protocols for medical interventions were published at the national level, and cost estimates were formulated. In 2021, 19 new medical services in nuclear imaging and scintigraphy were included in the NRS.

Conclusions

Despite the long and bureaucratic process of including new HTs in the NRS, the HB-HTA Unit managed to speed up this process. One of the main priorities of the integrated HB-HTA Unit is to promote the transfer of HTs into the health system at the national level.
OP157 Evaluating The Clinical And Economic Impact Of Adopting A Closed Peripheral Intravenous Catheter System In A Japanese Hospital
Authors: Xian Tan Kristin Hui , Tomaru Takeshi , Gala Smeet , Ma Yan
Published: December 2023

Introduction

Up to 90 percent of inpatients require an intravenous catheter during their hospitalization. A closed, integrated peripheral intravenous catheter (PIVC) system has been shown to protect veins for longer and reduce the risk of complications and unnecessary restarts when compared with an open system. This study evaluated the annual clinical and economic outcomes of adopting a closed, integrated PIVC system, instead of an open system, for inpatients in a Japanese hospital.

Methods

A budget impact analysis was developed to estimate the clinical and economic impact for a 500-bed hospital with an 85 percent occupancy rate and a 96-hour catheter replacement protocol. For the analysis, the average length of stay for patients was 12 days and 90 percent of inpatients required a PIVC. Inputs such as catheter failure rate, complication rate, consumables costs, and complication management costs were informed by global and local data sources. The outcomes evaluated included consumables utilization, complication events, nurse time, and overall cost impact.

Results

The analysis estimated that 12,604 patients required PIVCs. Moving from an open to a closed, integrated PIVC system resulted in a 68 percent reduction in consumables (3,786 fewer catheters and 36,315 fewer connecting accessories). Complications (occlusion, extravasation, phlebitis, and bending) were reduced by 62 percent (3,682 fewer episodes). Blood exposure was reduced by 98 percent (3,565 fewer episodes), and nurse time decreased by 17 percent (786 fewer hours). This resulted in a potential overall cost saving of JPY3,955,140 (USD28,756) annually, after offsetting the acquisition cost of JPY888,247 (USD6,458) associated with the closed system.

Conclusions

PIVC is the most common vascular access device used in hospitals, and insertion and maintenance are often performed by nurses. Fewer complications can be expected with a closed system, leading to better patient outcomes. In addition, nurses spend less time managing complications and replacing PIVCs, and consumables utilization is reduced. This results in improved operational efficiency and potential cost savings for hospitals.
OP159 Quality Of Evidence For Clinical Benefit Of Cancer Medicines Assessed For Funding In Australia
Authors: Vitry Agnes , Caird Cathy
Published: December 2023

Introduction

This study aimed to describe the type of evidence available for and the clinical benefit of cancer medicines assessed for funding in Australia by the Pharmaceutical Benefits Advisory Committee (PBAC). The evidence was assessed with the European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS).

Methods

All data on applications submitted to PBAC between 2010 and 2020 were independently extracted in duplicate from PBAC Public Summary Documents available online. Any disagreements were resolved through discussion. ESMO-MCBS ratings were retrieved from the ESMO-MCBS website. Substantial benefit for the ESMO-MCBS was defined as a grade A or B for (neo)adjuvant intent and four or five for palliative intent.

Results

In the study period, 182 cancer indications for 100 cancer medicines were examined by PBAC, including 124 (68%) for solid tumors (116 in the palliative setting) and 58 (32%) for hematological cancers. A total of 138 (76%) indications were recommended for public funding, 40 (22%) were rejected, and four (2%) were deferred. Randomized controlled trials (RCTs) were the main source of evidence in 154 indications (85%) and single-arm studies in 27 (15%) indications. RCTs were available in 113 (91%) and 41 (71%) of the solid tumor and hematological cancer indications, respectively. In submissions with RCTs, mature overall survival (OS) was reported in 81 (53%) indications. For indications with a statistically significant improvement in OS, the median gain was 3.0 months (range 0.9 to 17.0) for solid tumors and 8.2 months (range 1 to 9.1) for hematological cancers. The ESMO-MCBS score was available for 99 solid tumor indications. For indications in the palliative setting, 47 (52%) had substantial clinical benefit according to ESMO-MCBS v1.1, including 35 (51%) indications recommended by PBAC and six (35%) indications that were rejected.

Conclusions

These results show that only a minority of cancer medicine indications considered by PBAC are supported by a good level of evidence and provide a modest extension of patient survival.
OP162 Making Local Economic Evaluation More Relevant: Using Expert Elicitation To Adjust Published Intervention Effects To Reflect Local Context
Authors: Gray Jodi , Karnon Jonathan
Published: December 2023

Introduction

Expert elicitation is often used in economic analyses to estimate uncertain or unobserved parameters for decision models. However, it has rarely been used in the context of local decision-making. A pragmatic elicitation process was used during a local economic evaluation to prompt local experts to assess the relevance of the published evidence to their setting, and to adjust the published effect estimates to better reflect the intervention effect expected in their setting.

Methods

Elicitation was undertaken for two interventions that targeted the prevention of hospital-acquired hypoglycemia. Six clinical experts from within the Southern Adelaide Local Health Network (SALHN) were systematically presented with information on the setting of the published evaluation and their local setting. This included information on the hospital and quality of care, patient characteristics, and the research context. After comparing the settings, the experts were asked to estimate the most realistic, most pessimistic, and most optimistic intervention effects for their local context.

Results

The local intervention effect was estimated to be smaller than the published estimate for both interventions. For one intervention, this was driven by the lower complexity of the local patient cohort. For the other intervention, it was driven by differences in the scope of implementation, with hospital-wide local implementation expected to reduce staff buy-in relative to the targeted implementation used in the published evaluation. The elicited local intervention effects were used in a cost-consequence analysis to estimate the likely costs and effects of the interventions if they were implemented locally.

Conclusions

The pragmatic elicitation process provides a feasible and acceptable way to assess and transparently adjust the published effect estimates to better reflect the expected intervention effect in the local setting.

Including this step in local economic evaluations can increase the relevance of these evaluations to local decision makers. Further development and application of these methods may facilitate greater use of economic evaluation in local settings.
OP174 Health Technology Assessment And Economic Evaluations For A Genomic Strategy In Italy
Authors: Di Brino Eugenio , Antonini Debora , Falasca Giulia , Basile Michele , Rumi Filippo , Cicchetti Americo
Published: December 2023

Introduction

The challenge to health systems is sustainability, not only in the economic and financial sense of compatibility of spending with allocated resources, but also in terms of equity in access to services and care, quality, safety, innovation and research, that is, in terms of the effectiveness of the right to health. Some countries, such as Italy, do not have formal health technology assessment (HTA) or other similar processes that take into account views outside the decision maker.

Methods

In the Italian national context, there is currently no single tariff for the reimbursability and pricing of genomic technologies. In fact, although genomic tests have been in clinical practice for many years now, to date they have not yet been included in the LEA (Minimum healthcare provision), especially in view of the fact that a defined and transparent process for updating the Essential Levels of Care has been operational since 2018. With the goal of structuring guidelines for the adaptability of economic evaluations to currently available and developing genomic technologies, a literature review was conducted.

Results

The literature review showed that there are some methodological and practical issues that need to be carefully considered when designing and conducting economic evaluations of genomic tests. In more detail, five key concepts were identified in order to implement the most comprehensive economic evaluation of the technologies under study: the PICO model, the survey perspective, the costs included in the analysis, the effectiveness analysis, the time horizon, and the discount rate.

Conclusions

Adequate definition of these concepts appears to be of paramount importance in view of the fact that genomic testing may have important consequences for future generations as well. For the purpose of sustainability of access of genomic technologies, the use of Budget Impact Analysis (BIA) is recommended in all analysis settings being essential for the regulator to tie access to its available budget capacity.
PP17 Rare Diseases: An Analysis Of Assessments Carried Out By The National Committee For Health Technology Incorporation In Brazilian Public Health System (Conitec)
Authors: Rodrigues Fernanda , Borges Stefani , Pires Wolney , da Costa Nathalia , Louly Priscila , Prado Clementina , Santos Vania
Published: December 2023

Introduction

The treatment of rare diseases has been a challenge for the Brazilian Unified Health System. In addition to the high costs of treatments, the characteristics inherent to this type of disease bring weaknesses to the scientific evidence of efficacy and safety. The National Committee for Health Technology Incorporation In Brazilian Public Health System (Conitec) is formed by a plenary of experts who monthly assess demands for incorporation into the public health system.

Methods

This exploratory, descriptive, and retrospective study aims to gather qualitative and quantitative data on criteria considered by healthcare decision-makers from Conitec and analyzes which rare diseases were benefited by the commission recommendations. Data from June 2012 to November 2022 were collected from the Conitec website to a specific extraction form and analyzed using descriptive statistics.

Results

A total of 763 technologies were evaluated from June 2012 to November 2022, with 158 being drugs for rare diseases. Among these, those with the highest number of diseases benefited were multiple sclerosis 13.3 percent (n=21), cystic fibrosis 6.3 percent (n=10) and pulmonary hypertension 5.7 percent (n=9). About 70 (44.3%) technologies were incorporated into the Unified Health System to treat rare diseases. In these incorporations, 25 technologies initially had an unfavorable recommendation, and only after the public consultation they were recommended for incorporation. Reasons that contributed to this change in recommendation were the new scientific evidence presented (64%), new negotiation of the drug price (28%), and new budgetary impact by revising the calculation of the target population (8%).

Conclusions

The criteria for evaluating technologies for rare diseases are similar to those adopted for other clinical conditions. However, it is important to adopt specific criteria in analysis of drugs targeted at diseases considered rare for the population. Negotiating prices with industry is an important factor that was highlighted, potentially favoring access to new treatments that can modify the progress of these diseases.
PP21 Are We Ready For It? Developing Criteria To Include Artificial Intelligence Medical Devices (AI-MDs) For Health Technology Assessment
Authors: See-Toh Rachel , Ooi Lydia , Ng Zhen Long , Soon Swee Sung , Ju Hong , Ng Kwong
Published: December 2023

Introduction

The increasing pace at which artificial intelligence medical devices (AI-MDs) or digital health technologies (DHTs) have been introduced and integrated in healthcare has not been matched with appropriate selection criteria for health technology assessment (HTA) to inform funding decision-making. To align with international best practice and local regulatory guidance, the Agency for Care Effectiveness (ACE) developed criteria to include AI-MDs as part of its 2022 topic prioritization process for medical technologies. This abstract describes ACE’s approach to develop the inclusion criteria.

Methods

To develop key principles for including AI-MDs in ACE’s topic prioritization process, relevant information from overseas HTA agencies, local regulatory guidelines, and ACE’s existing topic selection criteria were reviewed. A search of international HTA agency websites was conducted in September 2022 to identify relevant information on inclusion of AI-MDs in healthcare for reimbursement recommendations.

Additionally, local regulatory guidelines for AI-MDs in healthcare were also identified. The inclusion criteria were then piloted with AI-MDs identified from ACE’s horizon scanning workstream to examine their feasibility for HTA topic selection.

Results

One overseas framework on DHTs from the National Institute for Health and Care Excellence (NICE) and two local regulatory guidelines were identified. Based on the key finding that the purpose of AI-MD use in guiding clinical management and its associated risks were important considerations, the following criteria were developed: (i) full registration with the regulatory body;(ii) device characteristics should be interventional, have direct impact on patient safety, or support accurate diagnosis or treatment which is critical to avoid death and serious health deterioration; and (iii) the AI algorithm should be fixed as opposed to adaptable as per regulatory requirements. Using this inclusion criteria, eight AI-MDs surfaced from horizon scanning were screened with the above criteria and deemed suitable for HTA topic selection.

Conclusions

As AI technologies are increasingly used to replace or supplement current clinical practice, continuous adaptation of HTA method is needed to ensure appropriate topic selection.
PP30 The Fast Track In Drug Registration By ANVISA – Brazil And Possible Consequences
Authors: Kelles Silvana , Pereira Camila , Martins Carina , Reis Daniel , Azevedo Ernesto , Ribeiro Geraldo , Zocrato Karina , Carvalho Lélia , Freitas Marcela , Horta Maria , Barbosa Mariana , Talim Mariza , Borin Marcus
Published: December 2023

Introduction

The fast track in drug registration by the Brazilian Health Regulatory Agency, ANVISA, began in 2017 and is intended to prioritize analysis related to drugs relevant to public health. This process is appropriate in situations where there are no therapeutic alternative available or for technologies show significant improvement in safety, efficacy, or adherence to treatment.

Methods

The Brazilian public administration has a tool for accessing information called Transparency Portal. Thus, data on the number of drugs approved by fast track between 2018 and 2021 were requested through this tool and evaluated.

Results

The data received by the Brazilian transparency portal shows that the number of requests for fast track had an increase from one in 2018 to 32 in 2021. There is an important increase of registrations, being drugs with phase II trials and with single-arm clinical trials. With registration based on a phase II trial, these patients are in fact receiving drugs in a context similar to clinical trials, but with funding from the healthcare system. Given that phase II studies are conducted in a limited population, there has been an increase in the registration of drugs with suboptimal efficacy and safety concerns. This scenario of uncertainty leads to non-adherence to treatment and a discrepancy in real-world outcomes in comparison to the clinical trial. About 70 percent of phase II trials, show no benefit, and only 30 percent proceed to later phases. It is noteworthy that about 50 percent of the studies that move on to later phases fail to show benefits. Nowadays the number of drugs approved by the fast track has increased, many probably with phase II studies and no comparator group.

Conclusions

Given the uncertainties in the efficacy and safety of a drug registered via fast track, often based on phase II studies, implementing provisional registration with real-world evaluation of outcomes, and coupled with financing based on risk-sharing agreements, may be a sustainable alternative for health systems.
PP61 Cost-Effectiveness Analysis Of Trastuzumab Deruxtecan Versus Chemotherapy For Previously Treated HER2-Positive Gastric Cancer In Singapore
Authors: Koh Ivan , Tan Ling Eng , Abdul Aziz Mohamed Ismail , Lin Liang , Ng Kwong
Published: December 2023

Introduction

The phase two DESTINY-Gastric-01 trial demonstrated that trastuzumab deruxtecan (T-DXd) improved overall survival in Asian patients with human epidermal growth factor receptor 2 (HER2)-positive, advanced gastric or gastroesophageal adenocarcinoma that had progressed following two or more treatments, compared with chemotherapy (irinotecan or paclitaxel monotherapy). Considering the high cost of T-DXd, we assessed the cost-effectiveness of T-DXd versus chemotherapy from the Singapore healthcare system’s perspective.

Methods

A partitioned survival model with three health states (progression-free, progressed disease and death) was developed, with a five-year time horizon. Survival curves from DESTINY-Gastric-01 were extrapolated beyond the trial duration using parametric functions. Health state utilities were obtained from published literature and direct costs were sourced from public healthcare institutions in Singapore. Utility decrements for adverse events such as interstitial lung disease was incorporated into the model for the differences in safety profiles. A discount rate of three percent was applied to costs and outcomes. One-way deterministic sensitivity analyses (OWSA) and scenario analyses were conducted to assess parameter and model uncertainties.

Results

Treatment with T-Dxd, compared to chemotherapy, had a high base case incremental cost-effectiveness ratio (ICER) of over SGD450,000 (USD334,900) per quality-adjusted life-year gained. The cost of T-DXd greatly influenced the results according to OWSA. Seventy-three percent of the total costs accrued in the T-DXd arm was due to the cost of the drug, compared to seven percent in the chemotherapy arm. The ICER was also sensitive to the assumptions around extrapolation of the survival curves, but when tested across all scenario analyses, the results remained unfavorable.

Conclusions

At the current cost, T-DXd does not represent good value compared to chemotherapy for previously treated HER2-positive gastric cancer in Singapore. The findings from our cost-effectiveness analysis, alongside other considerations, will be useful to inform policy makers on funding decisions.
PP75 Using Real World Evidence To Support The Reimbursement Of Proton Therapy Across A Broad Range Of Rare Cancers
Authors: Arora Nimita , Tilden Dominic , Penfold Scott
Published: December 2023

Introduction

The Australian Bragg Centre for Proton Therapy and Research in Adelaide will be Australia’s first center with the capacity to deliver proton beam therapy (PBT). PBT uses energy from protons to target cancer cells while minimizing damage to surrounding healthy tissue, including vital organs. Compared to X-ray (photon) radiation therapy (PRT), PBT reduces the risk of serious and long-term complications.

To improve access to PBT, the South Australian Health and Medical Research Institute (SAHMRI) submitted an application to the Medical Services Advisory Committee (MSAC) including a cost utility analysis (CUA) comparing PBT with PRT to treat specific pediatric/adolescent and young adult (AYA) and rare adult tumors.

Methods

A systematic review identified 28 comparative, mostly real-world studies to support the conclusion that PBT has superior safety and non-inferior efficacy to PRT in the requested indications. The key challenge for the CUA was to quantify the cost and quality of life implications of the superior safety profile across a wide range of indications with a limited comparative evidence base. A simple lifetime decision analytic model was developed which modeled the rates, costs and utilities associated with relevant toxicities. The complications of radiotherapy are often chronic and included secondary malignancies, visual impairments, endocrine dysfunction, dysphagia, hearing loss and intellectual disability. Some of these toxicities are only applicable to patients with cranial cancers. Therefore, the event rates applied in the evaluation were adjusted to account for the proportion of patients within each population estimated to have extracranial cancers.

Results

When results in the adult and pediatric/AYA populations were weighted across the expected utilization of PBT (34% adults, 66% pediatric/AYA) in each population, PBT was dominant relative to PRT.

Conclusions

In November 2020, MSAC recommended funding PBT in specific populations at high risk of long-term side effects from PRT. To address uncertainties around the evidence base, MSAC further requested the following:

•

All patients receive comparative photon/proton plans to determine eligibility

•

A national registry is established for patients treated with PBT.
PP76 From Hospital-based Health Technology Assessment (HTA) To Treatment Decision: What Decisions Actually Result After HTA In A Resource Constrained Environment?
Authors: Fitzgerald Anita , Fevre James Le , Morgan Gina , Streat Stephen , Munn Stephen
Published: December 2023

Introduction

The Northern Region Clinical Practice Committee (NRCPC) conducts hospital-based health technology assessments (HTA) to provide advice to hospital managers regarding both the implementation of new technologies and the configuration of existing services. To assist in the comparison of dissimilar health technologies applied across different disciplines and different hospitals, the NRCPC developed a prioritization tool. This abstract reports the use of the tool over the 17-year period that the committee has been in operation.

Methods

The score given to each HTA depends on cost-utility, predicted health improvements and the quality of evidence. In addition to the scoring tool, editorial notes are provided to contextualize the agreed score and to explain the NRCPCs interpretation of the evidence.

Results

Most of the time hospital managers have made decisions concordant with the recommendations of the NRCPC; submissions are recommended to be implemented, declined, or receive interim approval with data collection. The latter often occurs when there are uncertainties about efficacy, but no (or very few) safety concerns, or where there are uncertainties about whether the proposed costs are reproducible in the hospital setting. In these cases, management responses often require submitters to undertake a limited number of cases and collect data for audit over a one-to two-year period. Low-scoring submissions are often declined, whereas high-scoring submissions have not been declined to date. The interim approval (with data collection) strategy has had variable outcomes based on the willingness of the implementing clinicians to collect accurate data about both costs and outcomes. From 2005 to 2022, the NRCPC received 146 submissions. This poster reports graphical representations of the decisions made over the NRCPCs period of operation.

Conclusions

The NRCPC scoring tool has been successful to date in providing a framework for decision makers to allow consistent, unbiased and objective assessments of dissimilar technologies. Prioritization tools in hospital-based HTA are beneficial to decision makers in hospital settings.