bluuebunny/crossref_metadata_2025 huggingface dataset:
OP96 Adapting Patient Involvement For Fast Track Appraisals
Authors: Rasburn Mark , Crosbie Helen , Marsden Laura
Published: December 2023

Introduction

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

Methods

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

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

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

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organizing earlier peer support with experienced lay members; and

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

Results

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

Conclusions

Despite shorter timeframes, patient involvement has not been compromised. NICE will use the feedback from patient contributors to review and adapt the induction process and support offered. This will support patient contributors and enable NICE to allocate appropriate resources in the shortened timeframe.
OP102 Towards Universal Health Coverage: Health Technology Assessment Roadmap Development In The Emirate Of Abu Dhabi Involving The Whole Ecosystem
Authors: Alsaeedi Amna , Richter Dirk , Alazeezi Hamda , Nassri Farah , Wael Mahmoud , Oortwijn Wija , Bijlmakers Leon
Published: December 2023

Introduction

The mission of the Department of Health (DoH) of Abu Dhabi in the United Arab Emirates is to provide its population with a healthy life and world leading preventive and curative services. While the DoH has regulations in place to grant market approval to new health technologies, there is a need to develop a clear overall framework for reimbursement and disinvestment decisions. Establishing a structured health technology assessment (HTA) framework is critical for informing decisions on health technologies that offer value for money, with the aim of improving equitable access to health care, financial risk protection, and, ultimately, better health outcomes.

Methods

During 2022, the DoH collaborated with the Radboud University Medical Center to explore the feasibility of applying an evidence-informed deliberative process (EDP) HTA approach through workshops and interviews involving all stakeholders in the ecosystem, such as policy makers, principal investigators, providers, patients and public groups, product manufacturers, payers, and purchasers. A situational analysis was conducted to collect stakeholders’ views and build EDPs. Based on this analysis, a structured roadmap was developed.

Results

The comprehensive five-year roadmap to implement a holistic HTA framework in Abu Dhabi consisted of five major elements, starting with the establishment of an appropriate HTA policy framework as a foundation. Abu Dhabi should firmly establish its HTA structure and program (in one to two years), and at the same time invest in developing and retaining HTA training capacity so that over time (within three to five years) the country can build up its own expertise to sustain the program. This needs to be accompanied by continuous awareness raising among all relevant stakeholders.

Conclusions

This roadmap is the first and most important step toward implementing a holistic HTA framework in Abu Dhabi. Technical work needs to be complemented by continuously raising awareness and involving all relevant stakeholders. Looking at the initial results and international benchmarks, HTA will significantly contribute to achieving a sustainable, high quality healthcare system.
OP106 Standing On The Shoulders Of Giants: Adapting Published Health Technology Assessments On Medical Technologies To Singapore’s Context
Authors: Ooi Lydia , See-Toh Rachel , Soon Swee Sung , Ju Hong , Ng Kwong
Published: December 2023

Introduction

Given time and resource constraints, health technology assessments (HTAs) should avoid duplicating prior HTAs. This abstract describes the experience of the Agency for Care Effectiveness (ACE) with adapting and building on published HTAs of medical technologies to support efficient decision-making.

Methods

Upon defining the scope of an evaluation topic, searches were performed on reference agency websites and HTA networks, such as the International Network of Agencies for Health Technology Assessment, to identify relevant published HTAs. Identified reports were included for evidence synthesis if the inclusion criteria were met. If multiple relevant HTAs were identified, the most recent or comprehensive HTA identified was used as the foundational evidence base and supplemented by additional primary or secondary literature as needed. When deemed necessary, de novo economic models were developed. Where available, reimbursement decisions or recommendations from published HTAs and their applicability to Singapore’s context were also considered. Following ACE’s evaluations, the Ministry of Health Medical Technology Advisory Committee (MTAC) made subsidy recommendations.

Results

To date, ACE has completed 54 HTA evaluations of medical technologies, 47 (87%) of which incorporated published HTA evidence. Although some MTAC recommendations were aligned with published HTAs, discrepancies were observed that were mainly attributed to evolution of the technology or evidence since publication of HTAs; different local clinical needs or management algorithms; and different local costs of the intervention and comparator. Key challenges in adapting published HTAs included differences in the target population and intervention or comparator in published HTAs, mixed recommendations from published HTAs, and non-English language reports, which may underscore the need to adapt published HTAs to Singapore’s context. To overcome some of these challenges, close consultation with stakeholders is critical to understand local clinical need and care pathways and to gather key information such as costs.

Conclusions

Published HTAs, when used as an evidence base and supplemented by evidence updates and local contextualization, allow for efficient and robust HTA to support decision-making.
OP107 Streamlining Health Technology Assessment Of Medical Devices Through Development Of The Philippine Essential Medical Device List
Authors: Reyes Patrick Wincy , Liban Lara Alyssa , Victoria Arellano Ma. Cecilia , Santillan Joshua , Samonte Sheena Jasley , Marfori Anne Julienne , Guerrero Anna Melissa , delos Santos Marc James , Lee-Ramos Catherine Manuela , Peralta Agnette ,   Department of Health – Medical Device Unit, Health Technology Assessment Council
Published: December 2023

Introduction

Pursuant to the Universal Health Care Act of 2019, the Health Technology Assessment Council (HTAC) in the Philippines was mandated to make recommendations for government financing of health technologies, including medical devices. The development of a Philippine Essential Medical Device List (PEMDL) will serve as a guide for the procurement of medical devices and supplies in government health facilities and as the basis for creating a price reference index.

Methods

The HTAC and a team of medical device experts (the Expert Advisory Committee) developed a streamlined pathway and guidelines using a Delphi technique with the Philippine Food and Drug Administration (FDA) and the Department of Health (DOH) to determine the necessary criteria for fast tracking the inclusion of medical devices. The pathway was also revised throughout a pilot processing of commonly procured medical devices (e.g., consumables) that were either already covered by existing national health insurance packages or funded by DOH programs, among others.

Results

The final pathway consisted of validating whether the device was already approved by the Philippine FDA and whether it was already part of standard of care. Similar to the usual HTA nomination requirement, the local FDA approval attested to the safety and quality of the device. Meanwhile, the inclusion of the device in the standard of care guaranteed that it was essential in the healthcare setting. This can be written in the World Health Organization technical documents and databases such as the Medical Devices Information System, health facility listing requirements, or locally adopted clinical practice guidelines. The initial PEMDL for release contained a total of 307 medical devices across 16 categories.

Conclusions

Unlike the mandate for the Philippine National Formulary, government health facilities are not yet required to base their procurement on the PEMDL because the list is still in its infancy. Moving forward, the list will include big ticket items and will be updated through consultations with specialty centers and hospitals.
OP109 The Conceptualization And Value Of A Disease Management Approach To HTA In Canada: Findings From A Qualitative Study
Authors: Richardson Marina , Miller Fiona , Daneman Nick , Mighton Chloe , Sander Beate
Published: December 2023

Introduction

Our objective was to conceptualize and assess the potential value of a disease management approach to health technology assessment (HTA) in Canada.

Methods

We conducted 18 semi-structured interviews between April 2022 and October 2022 to elicit informant views on potential opportunities for re-conceptualizing the decision-problem in HTA as a disease management problem versus a technology management problem. Participants were purposefully sampled from national and provincial HTA agencies and related organizations in Canada to achieve representation across the disease pathway including prevention, screening, and treatment, and the decision-making pathway including HTA organizations, expert committee members, and decision-makers. Data were analyzed using thematic organizations (based on the interview guide) and manual line by line coding of the data. Ethics approval was received from the Health Sciences Research Ethics Board at the University of Toronto.

Results

Three key features of a disease management approach to HTA (i.e., disease-based, multi-interventional, and dynamic) emerged from informants that differed from traditional HTA processes in Canada. The concept was generally not perceived to be a new idea – some informants indicating that it was implicit in the HTA analysis framework. There was general support for an explicit disease management approach to HTA if the impact of the approach could be demonstrated, if the assessment could be completed within an appropriate time frame, and if the assessment could include the equity, ethical and implementation domains of HTA. Informants indicated that the reconceptualization of HTA could lead to effective and efficient decision-making throughout a technology’s lifecycle, help breakdown system silos, and offer a platform for greater consideration of non-drug alternatives and upstream interventions. The impact of this approach was anticipated to contribute to a proactive health system that could improve population health, enhance the patient experience, and ensure appropriate stewardship of health care resources.

Conclusions

A disease management approach to HTA has international relevance as an approach that could promote integrated, proactive, sustainable, and resilient health systems.
OP112 Evaluation Of A Self-Administered Smart Phone-Based Application As A Wellness Measure In A Clinical Trial Of Zanubrutinib
Authors: Crescenzo Rocco , Idoine Adam , By Kunthel , Yang Keri
Published: December 2023

Introduction

Assessments self-administered via smart phone-based apps can gather patient wellness data; however, data regarding app utilization in clinical trials are limited. This study evaluated engagement with a self-administered wellness app as a supplemental tool to assess quality-of-life (QoL) in study BGB-3111-215 (NCT04116437), which included patients with B-cell malignancies treated daily with oral zanubrutinib.

Methods

This exploratory analysis evaluated the feasibility of using a voluntary device-based, self-administered activity and QoL questionnaire app (Medable, California, USA) in clinical trial patients. Feasibility was measured by rates of consent, utilization, and compliance. Consent was assessed by the percentage of patients who agreed to use the app. Utilization was assessed by the percentage of patients who engaged with the app. Compliance was assessed by actual versus scheduled engagements. The app also included passive activity tracking and a six-minute walk test.

Results

As of 1 September 2022, 78 patients had enrolled in the study (median age 71 years) and 20 had consented to participate (26% consent rate). Eleven patients (median age 65 years) engaged with the app at least once (14% utilization rate; n=11 for the questionnaire; n=7 for the walk test). Engagement occurred a median two times (range 1 to 17) for the questionnaire and a median seven times (range 1 to 15) for the walk test. Four patients engaged with the questionnaire and six patients engaged in the walk test beyond week 12. Passive activity was collected for eight patients beyond week 12. Among patients who engaged with the QoL questionnaire and walk test, compliance rates were 18 and 21 percent, respectively.

Conclusions

This initial study showed that a subset of patients was willing to participate in the self-administered QoL questionnaire and activity tracker. Further comparison of app results and clinical trial findings are ongoing to explore factors affecting utilization and compliance rates.
OP120 Developing A Call To Action For Patient Involvement In Health Technology Assessment (HTA) In Southern Africa
Authors: Pretorius Lauren , Mueller Debjani , Holtorf Anke Peggy
Published: December 2023

Introduction

In building health technology assessment (HTA) and related decision processes in Southern Africa, institutions and stakeholders face region-specific challenges such as disease prevalence and population makeup. These can be addressed by collaboratively discussing patient engagement solutions that fit the local culture and systems and serve to ensure equitable and sustainable access to patient-relevant health technologies. Our aim is to initiate a collaboration for driving patient involvement (PI) suitable for the Southern African context and Sub-Saharan patient communities. In addition, we explore current experiences in PI, including the stakeholder expectations, gaps, limitations, and new opportunities.

Methods

A one-day hybrid multi-stakeholder PI in HTA workshop was held in Johannesburg, South Africa. Co-created by the participants, the outputs are a call to action and a concept draft for the vital success criteria for PI in the region. The content of the call to action is gathered from pre-workshop surveys, interviews, and outcomes from historic meetings held in conjunction with the Health Technology Assessment International (HTAi) PI workstream as well as facilitated discussion from the actual workshop.

Results

The workshop was attended by 42 participants from nine countries, representing diverse stakeholder groups. The attendees represented multiple PI stakeholder groups. The workshop survey was completed by 44 respondents, while 12 participants completed the post-event survey. A workshop outcomes document highlighting a high level of alignment and identifying seven key success factors was developed. A workshop proceeding detailing the outcomes is now being drafted.

Conclusions

Over 95 percent of respondents to pre-and post-surveys indicated an interest in contributing to a more in-depth description of PI in their country. While the majority of participants were from South Africa, participants from Tanzania, Ethiopia, Zambia, and Zimbabwe emphasized that trans-African-engagement for HTA will provide an additional opportunity for HTA in Africa and patient and community participation in HTA and healthcare decision-making. Hence, a collaborative platform could help all African countries to advance and benefit from improved healthcare decision processes.
OP127 The Cost Effectiveness Of Anti-Vascular Endothelial Growth Factor Treatments For Age-Related Macular Degeneration In The Italian Healthcare Setting
Authors: Basile Michele , Di Brino Eugenio , Rumi Filippo , Cicchetti Americo
Published: December 2023

Introduction

Age-related macular degeneration (AMD) is a common condition that affects the middle part of a patient’s vision. Typically, it first appears in people in their 50s and 60s. While it does not cause total blindness, it can make everyday activities, such as reading and recognizing faces, more difficult. This analysis aimed to define the resource absorption and cost-effectiveness profiles of the anti-vascular endothelial growth factor therapies currently available in the Italian healthcare context.

Methods

A questionnaire was prepared to gather information on specific drivers involved in the provision pathway. The economic analysis was conducted according to activity-based costing methods. A cost-effectiveness analysis was carried out to provide information on the sustainability profile of the treatments available in the Italian setting. Results were reported in terms of the incremental cost-effectiveness ratio (ICER).

Deterministic and probabilistic sensitivity analyses were carried out to test the robustness of the results.

Results

The average absorption of resources per patient along the whole clinical pathway for aflibercept, bevacizumab, ranibizumab, and brolucizumab was EUR6,858, EUR1,420, EUR7,930, and EUR5,667, respectively. Brolucizumab was characterized by an unacceptable cost-effectiveness profile (ICER EUR43,454) versus bevacizumab, considering a willingness-to-pay threshold of EUR40,000 per quality-adjusted life-year (QALY). Compared with ranibizumab, brolucizumab was associated with lower costs (EUR22,368 versus EUR29,333) and higher QALYs (12.8 versus 12.6). Brolucizumab had a higher level of QALYs (12.8 vs 12.7) and lower resources absorbed than aflibercept, with a saving of EUR4,222. Therefore, brolucizumab was a dominant alternative to ranibizumab and aflibercept.

Conclusions

The analysis underlined how brolucizumab is a cost-saving strategy, compared with aflibercept and ranibizumab, and is likely to be cost-effective relative to bevacizumab in the Italian healthcare context.
OP130 Estimating The Economic Burden Of HIV/AIDS In Türkiye Towards The Next Decade
Authors: Erkut Yaren , Kurnaz Mustafa , Balik Ismail , Tibet Birol , Kockaya Güvenc
Published: December 2023

Introduction

Approximately 34.8 million people globally are affected by HIV, 34,000 of whom are in Türkiye, and the impact of HIV continues to grow. Not providing the necessary treatment eventually leads to life threatening AIDS-related consequences. In this study, a descriptive analysis of the published official data on HIV/AIDS was undertaken to assess existing statistics and raise social awareness. The aim was to provide data that will help decision makers in future planning by forecasting the possible number of cases and treatment costs to 2030 using available statistics in Türkiye.

Methods

The forecast of HIV-infected patients, deaths, and healthcare costs to 2030 were calculated using linear regression based on data published between 1985 and 2022 in the literature or by official authorities. The proportion of past deaths caused by AIDS was used to forecast deaths, and the number of patients living with HIV was estimated indirectly using this forecast. Possible treatment expenditures related to HIV/AIDS in Türkiye were estimated based on published healthcare cost data and the number of living patients obtained by considering the possible increase in HIV/AIDS cases, future inflation predictions from the Turkish Central Bank, existing literature, and information from statista.com.

Results

Estimates suggested that 3,002 new cases of HIV/AIDS in 2021 will become 5,709 among a total of 74,227 living patients in 2030. The 598 deaths reported between 1985 and 2022 are predicted to rise to 1,256 by 2030. Estimation of the total treatment cost of HIV/AIDS was TRY1,051,026,183 (USD118,683,580) in 2021, which was estimated to reach TRY15,432,842,049 (USD296,450,696) by 2030. This consisted of outpatient visits (TRY246,710,838 [USD4,739,088]), laboratory tests (TRY2,072,489,815 [USD39,810,623]), inpatient visits (TRY1,352,192,479 [USD25,974,373]), management of complications (TRY3,371,126,960 [USD64,756,247]), and antiretroviral therapy drugs (TRY8,390,321,957 [USD161,170,365]).

Conclusions

The prevalence of HIV/AIDS is expected to rise by 137 percent by 2030, with the economic burden increasing 14.7 times in TRY (2.5 times in USD) from 2021 to 2030 in Türkiye. This proves the severity of the situation and the need for relevant policy measures for society.
OP141 Expert Knowledge Elicitation in Health Technology Assessment: Our Experience Using the Sheffield Elicitation Framework
Authors: Stringer Danielle , Ma Ning , Tivey David
Published: December 2023

Introduction

Expert judgement has an important role in health technology assessment (HTA), including as a source of evidence to inform economic modeling when published data are lacking. Quantitative information may be elicited from experts to inform model inputs and associated uncertainty using one of many expert elicitation methodologies. Here, the feasibility and potential benefits of one expert elicitation method, the Sheffield Elicitation Framework (SHELF), to the HTA process is examined.

Methods

The SHELF method seeks to express the knowledge of multiple experts in the form of a subjective probability distribution. Eliciting a subjective probability distribution allows the uncertainty of experts to be included in probabilistic sensitivity analysis, which is becoming an increasingly prominent feature of HTAs. The individual knowledge of participating experts is combined through behavioral aggregation, where experts participate in a discussion before being asked to provide judgments from the perspective of a rational impartial observer. The whole process is led by a facilitator who ensures all participants contribute and confirm that the final distribution is a product of consensus, not compromise.

Results

We recently conducted two SHELF elicitations as part of an ongoing project aiming to streamline the assessment of positron emission tomography (PET) in Australia. These elicitations provided insight into the usefulness of SHELF within the HTA setting. Given the constraints imposed by the COVID-19 pandemic, the elicitation sessions were conducted online rather than in the ideal face-to-face manner. In collaboration with one of the developers, we successfully adapted the method by making use of video conferencing technology to provide an online environment that mimicked the face-to-face setup as much as possible.

Conclusions

SHELF provides a rigorous and scientific method by which to elicit the knowledge of multiple experts in the form of a probability distribution. However, the method is resource intensive and may be best reserved for when data on key drivers are lacking.
OP143 Association Of Different Venous Access Device With Health‑Related Quality Of Life Among Patients With Breast Cancer In China
Authors: Liu Liu , Xia Zhiyuan , Chen Yingyao , Liu Shimeng
Published: December 2023

Introduction

Few studies have explored the relationship between different venous access devices and health-related quality of life (HRQoL) among patients with breast cancer in China. This study aimed to evaluate the HRQoL of patients with breast cancer in China who underwent different venous access devices and to estimate the association between type of venous access device and HRQoL.

Methods

A multicenter cross-sectional study was conducted in three tertiary hospitals from three major geographical regions in China. The final sample consisted of 472 patients aged from 18 to 78 years. The HRQoL was measured with the EQ-5D-5L scale and the EuroQoL Group visual analog scale. The venous access devices were divided into totally implantable venous access devices (TIVAD), peripherally inserted central catheters (PICC), and other devices. The multivariate regression analyses were used to explore the association between type of venous access device and HRQoL.

Results

Of 472 participants, 352 (75%) used the TIVAD device, 89 (19%) used the PICC device, and 31 (7%) used other devices. The TIVAD group had the highest EQ-5D-5L values (mean 0.89, standard deviation [SD] 0.178), while the PICC group had the lowest values (mean 0.85, SD 0.239). The EQ-5D-5L values for the other venous access device group was in between (mean 0.88, SD 0.127). However, the multivariate analysis indicated that the VAS and EQ-5D-5L scale dimension scores among patients were not significantly different (P>0.05) for the various central venous access devices.

Conclusions

This study demonstrates a non-significant association between the type of venous access device used and the HRQoL of patients with breast cancer in China. Although patients with a TIVAD experienced more pain during device insertion and access for chemotherapy, the negative effect on HRQoL scores was smaller than for PICCs.
OP150 An Inventory Of Policy Levers For Influencing Appropriate Care
Authors: Warkentin Lindsey , Tjosvold Lisa , Bond Ken
Published: December 2023

Introduction

Healthcare reform through appropriate care is a current focus for many jurisdictions. A variety of policy options, or “levers,” are available to decision makers to influence appropriate care. However, these levers are not always identified in advance of developing policy recommendations, and few direct, empirical analyses are available to support their selection. An appropriate care policy lever inventory was developed for health technology assessment (HTA) users in Alberta, Canada, to support HTA scoping and policy development.

Methods

Relevant information was identified by a single reviewer through a scoping search of MEDLINE, forward and backward searching, and targeted gray literature searches. An Excel-based inventory was populated with a list of policy levers and their descriptions, policy effectiveness, and implementation considerations. Filters were developed to identify levers based on key characteristics. The inventory was iteratively refined through presentations to and feedback from key user groups.

Results

The inventory contained 53 policy levers aiming to influence service provision, clinician behavior, fiscal policies, populations or organizations, and patient behavior. The levers varied in how they restrict decision-making. Few levers were considered high impact (>5% change to behavior, utilization, or cost) or well-supported (>10 studies reporting effectiveness). Stakeholders found the inventory information useful, particularly for considering potential levers not frequently utilized within their respective programs. A user guide and case examples were also developed to help users learn to navigate the inventory.

Conclusions

An inventory of policy levers, which can be tailored to specific clinical areas and topics, can be of assistance to healthcare decision makers developing and utilizing HTAs to improve appropriateness of care. With limited indication-specific evidence, policy makers must utilize the broader evidence base on appropriate care policy levers to select and implement strategies that are applicable and transferable to their context. Challenges remain in systematically identifying all relevant literature given the inventory’s breadth, and in updating the inventory to reflect new evidence.
OP154 Horses For Courses: Developing A Proportionate Approach To Health Technology Assessment In England
Authors: Watson Ian , Dilkes Jenna , Ellison-Roberts Geoff , Prescott Jenniffer
Published: December 2023

Introduction

The number and range of technologies that the National Institute for Health and Care Excellence (NICE) evaluates has never been greater, and with that comes an increasing requirement for capacity. However, not all technologies need the full intensity of the current standard evaluation process. This presents an opportunity to differentiate evaluation processes, and in doing so release capacity for more evaluations. NICE has embarked on a project to develop proportionate appraisal processes, allowing promising medicines to move through refined processes that better match the specific needs of individual evaluations.

Methods

The proportionate approach to technology appraisals (PATT) project was initiated in June 2022. Multiple work strands were established to develop and test potential proportionate approaches, focusing mainly on streamlined approaches for appraisals in which a lighter-touch approach is sufficient. By creating an accountability framework which empowered staff across NICE, new processes were developed using test-and-learn principles: piloting key concepts using retrospective reviews and live appraisals and adjusting the approach based on their findings. The impacts of each approach on NICE, on stakeholders and on the individual appraisals were monitored and assessed.

Results

The project has identified a range of procedural, methodological and operational improvements across the NICE technology appraisal process. Substantial efficiencies have been found, including consolidation of activities, reducing duplication of effort and minimizing disproportionate steps. The test-and-learn approach has proved successful, both in rapidly identifying unsuccessful ideas and in refining and adjusting processes in light of new information and challenges.

Conclusions

The proportionate approach to technology appraisals project represents an important part of improving and streamlining NICE’s approach to health technology assessment, reflecting the increasing demands on the program. Developing proportionate approaches allows efficient use of limited evaluation resources to continue supporting rapid, evidence-based access to innovative technologies. The project also provides a valuable demonstration of an agile, flexible approach to process improvement, paving the way to rapid, dynamic updates in the future.
OP168 Costs And Effectiveness Of Whole Exome Sequencing (WES) In Patients With Unsolved Rare Disease Through The Diagnostic Pathway
Authors: Marshall Deborah A , Degeling Koen , Tagimacruz Toni , Seeger Trevor A. , Boycott Kym M , Bernier Francois , Mendoza-Londona Roberto , MacDonald Karen V. , Hartley Taila , Hayeems Robin Z.
Published: December 2023

Introduction

Patients suspected of having a rare genetic disease often experience lengthy and costly diagnostic odysseys. The timing of whole exome sequencing (WES) in the testing sequence, its diagnostic yield and test costs in the sequence all factor into estimates of cost-effectiveness analysis for health technology assessment.

Methods

We modeled the diagnostic pathway using a discrete event simulation model, starting with the first test result. We defined and populated the simulation based on data from the electronic medical records of n=307 from the Care-for-Rare SOLVE multi-center Canadian observational cohort. Five alternative diagnostic pathways were modeled based on the observed data: no WES, and WES as the first, second, third or fourth test in the sequence. WES as the second test in the sequence is considered standard of care in medical genetic centers in Canada. We assessed effectiveness of WES in terms of diagnostic yield, time to diagnosis, and costs as patient-level overall test costs (2020 CAD/USD) across the diagnostic pathway.

Results

Compared to molecular and specialized diagnostic tests only (i.e., no WES), WES increased diagnostic yield from 5 percent to 40 percent. The shortest time to diagnosis for those with a diagnosis was 1.82 years in the diagnostic pathway with WES as the second test. Test costs for each pathway were CAD2,800 (USD2,087, no WES), CAD2,700 (USD2,013, WES as first test), CAD3,500 (USD2,609, WES as second test), CAD4,500 (USD3,354, WES as third test), and CAD5,300 (USD3,951, WES as fourth test).

Conclusions

Placing WES earlier in the diagnostic pathway for patients suspected of having a rare disease is associated with an increased diagnostic yield, reduced time to diagnosis and lower overall test costs with the benefits being greater the earlier in the pathway that WES is implemented.
OP172 International Collaboration For Translating The Peer Review Of Electronic Search Strategies (PRESS) Checklist: A Harmonized Approach
Authors: Hodgson Amanda , Masterson Daniele , Ferreira T. P. , Laguardia Josué , da Silva Cicera Henrique , Martinez Silveira Martha Silvia , McGowan Jessie , Rabb Danielle
Published: December 2023

Introduction

The PRESS guideline and checklist provides a set of recommendations concerning the information that should be used by librarians and other information specialists when they are asked to evaluate electronic search strategies. CADTH and PRESS authors were approached for permission to translate this checklist into various languages.

Methods

The team from Fundação Oswaldo Cruz and the Universidade Federal do Rio de Janeiro asked CADTH for permission to translate the PRESS guideline and checklist for their research work. They translated PRESS following the steps advocated by the scientific literature on the translation of standardized questionnaires.

Results

CADTH is now sharing and actively disseminating the PRESS translations in French and Portuguese (and a forthcoming version in Spanish) via the Finding the Evidence website and through related presentation activities.

Conclusions

The coordinated translation of key health technology assessment (HTA) tools provides an avenue for international uptake and improvement of best practice in information retrieval, which is a foundational feature of HTA work. With the absence of formal translation guidelines on the translation of protocols such as PRESS, CADTH would benefit from developing guidance for HTA teams requesting to translate our tools. CADTH is currently conducting research on the uptake and use of PRESS, which will inform future knowledge mobilization strategies such as translation standards and communications.
PP01 Health Technology Assessment Of Cervical Artificial Disc Replacement: Highlighting The Need For A Consistent International Approach
Authors: Zhang Enti , Annoni Elena , Strachan Liesl
Published: December 2023

Introduction

Cervical artificial disc replacement (C-ADR) is not a new technology but one that has seen many technological advances in the past 10 years. Indeed, a recent review described total disc arthroplasty as the most innovative development in the history of spinal surgery. The primary goals of C-ADR are to reduce or eliminate pain, and restore normal segmental motion. The aim of this analysis was to identify, extract and examine key health outcomes and economic data from published health technology assessment (HTA) reports on C-ADR, with the aim of understanding how the evolution of this technology has influenced assessments internationally.

Methods

A comprehensive search of over 90 HTA organization websites and the INAHTA HTA database using key terms for C-ADR surgical procedures was coupled with a literature search of recent systematic reviews. No language restrictions were applied.

Results

Twenty HTA reports of C-ADR surgery published from 2005 to 2022 were included for review. Several HTAs (4/20) were updates or reassessments by the same agency and one was an update across agencies (Italy update of Belgian HTA). While many of the HTAs concluded C-ADR is as effective as standard care and superior in certain outcomes, there was no pattern or consistency in the conclusions or recommendations from these assessments, even as the evidence base expanded over time. Our analysis found this was largely due to variations in HTA approaches among agencies including: differences in research questions asked, PICO (Population, Intervention, Comparator, Outcomes) criteria and methods performed, such as: rapid versus full systematic reviews; inclusion of economic evaluations and/or budget impact analyses.

Indeed, one of the only predictive factors for a positive HTA was a favorable cost-effectiveness analysis.

Conclusions

C-ADR is an established technology with extensive HTA investigation internationally. The lack of a consistent approach taken by HTA bodies made prediction of successful HTA outcomes difficult. Future alignment of key evaluation processes and methods may help address current international variations and support consistent decision making on patient access.
PP13 Results Of An International Survey About Barriers In Patient Involvement In Health Technology Assessment Of Digital Health Technologies
Authors: Gallastegui-Calvache Edurne , Moltó-Puigmartí Carolina , Segur-Ferrer Joan , Vivanco-Hidalgo Rosa Maria
Published: December 2023

Introduction

In recent years, several efforts have been initiated by health technology assessment (HTA) agencies to increasingly incorporate patient involvement (PI) into their assessment processes. The rationale behind PI in HTA is that patients can give their perspective and experiences about health, illness and the use of health technologies, complementing clinical and healthcare system standpoints.

A recent systematic review summarized current evidence on the barriers and facilitators of PI in HTA. Barriers and facilitators were classified according to the context in which they appear, as organizational, decision-making, political and community-related.

Digital health technologies offer a singular opportunity to address some challenges faced by healthcare systems. However, we hypothesized that their intrinsic characteristics may have some implications for PI in digital HTA. We conducted a survey to gain knowledge about barriers in PI in digital HTA.

Methods

The survey was elaborated using Microsoft Forms and consisted of 31 questions divided into four blocks (organizational, decision-making, political and community-related). It was sent to three Spanish, one Catalan and one International patient umbrella organizations. The results were analysed using Microsoft Excel.

Results

A total of four responses were received from three Spanish and one Catalan patient umbrella organizations. Results showed high, partial, and low concordance among organizations in 65 percent, 19 percent and 16 percent of the answers received, respectively. According to the results, most of the barriers for PI seem to be the same for HTA of digital health technologies as for other types of health technologies. However, two or three of the four umbrella organizations consider that HTA of digital health technologies might pose greater challenges in terms of achieving significant patient participation, sufficient patient training and knowledge of HTA processes, proper preparation and tools of the HTA experts and logistics during the HTA process.

Conclusions

Barriers in PI are mostly the same for digital or other types of health technologies. Few differences are related to patient training, tools, and logistics during HTA process.
PP18 Unlocking The Potential Of Medical Device Reimbursement In India For Better Health Outcomes
Authors: Goel Ashwin , Sunderland Stephen , Srinivasan Shruti , Fahim Arif , Pusha Monika , Kataria Kirti
Published: December 2023

Introduction

The Indian healthcare landscape has witnessed several promising changes including the introduction of a comprehensive medical technology inclusion process, Diagnosis-related group (DRG)-pilot and value-based incentives for hospital services under the national public health insurance scheme. Realizing the need for a more patient-centric stance towards improving healthcare outcomes as the way forward, we propose incremental changes including greater participation of public and private care-providers in topic prioritization and the appraisal committee. We also propose a unique evidence-driven approach using reimbursement as a lever for rewarding quality and innovation in medical technologies.

Methods

We developed two discussion guides to capture the ideas around deeper involvement of care-providers and patient societies, and introduction of value-based reimbursement for incentivizing high-quality implantable medical devices in India. The guides were prepared using secondary research and key informant interviews. Over 25 key stakeholders representing payers, regulatory agencies, government authorities, clinical experts, and industry players selected through quota sampling participated in a roundtable meeting. Based on the meeting outcomes, key recommendations for leveraging medical device reimbursement for better health outcomes were developed.

Results

This qualitative research was carried out with participation of key stakeholders across the medical device reimbursement process. The group proposed recommendations for bringing care-providers closer to the process through a structured and inclusive nomination approach involving therapy users and patient groups at various stages of evaluation. Complementing the existing value-based incentives framework for hospital services, we proposed a similar two-step pathway for incentivizing quality of implantable medical devices. The proposal includes the introduction of certification-based and outcome-based incentives built on a scientific and holistic evaluation criterion.

Conclusions

Through this process, we created a pragmatic and concrete call for a stronger voice from care-providers and patient groups in the evaluation process. Consecutively, the proposed innovative framework introducing value-based incentives for implantable medical devices will be instrumental in enabling access to quality health care to poor patients. These strategies follow the principles of value-based care and will go a long way in achieving better health outcomes for the population. The scientific initiative has been made possible with the support of St. Jude Medical India Pvt Ltd. (now Abbott).
PP24 Methodological Frameworks And Assessment Domains For Digital Health Technology Assessment: Scoping Review And Thematic Analysis
Authors: Segur-Ferrer Joan , Moltó-Puigmartí Carolina , Pastells-Peiró Roland , Vivanco-Hidalgo Rosa Maria
Published: December 2023

Introduction

Experts highlight the need to reconsider the frameworks used in health technology assessment (HTA) in order to cover the particularities of digital health technologies (DHT). However, there is a large heterogeneity in the literature about terminologies and definitions. Our objective was to identify the methodological frameworks used worldwide for HTA of DHT and generate a proposal of domains to consider in the assessment through a thematic analysis, a qualitative data analysis method that consists of identifying, analysing and reporting patterns across the data.

Methods

Methodological frameworks were identified through a scoping review (ScR) in accordance with the PRISMA extension for ScR. We searched five different databases for peer-reviewed and grey literature published between 2011 and 2021. The retrieved references were screened using Rayyan by two authors in a single-blind manner. References included were analyzed thematically by three reviewers using ATLAS.ti.

Results

The systematic search retrieved 3,061 references (2,238 unique), of which 26 were included. These, in turn, synthesized 102 frameworks designed for the assessment of DHT. The included documents contained a wide variety of items to consider in HTA of DHT and often used different wording to refer to similar concepts. Through the thematic analysis, we reduced this heterogeneity. Specifically, in the first analysis phase we established 176 provisional codes related to different assessment domains. In the second one, these codes were grouped in 86 descriptive themes that, in turn, were clustered in the third phase in 61 analytical themes. The latter were organized through a vertical hierarchy of three levels: level 1 (13 domains), level 2 (38 dimensions), and level 3 (11 sub-dimensions).

Conclusions

There is a need to reconsider the frameworks and assessment domains used for HTA of DHT. Our thematic analysis of 26 references led to the definition of 13 domains, 38 dimensions and 11 sub-dimensions to consider in HTA of DHT. From our perspective, thematic analysis is the most appropriate method to identify assessment items for HTA of DHT from literature.
PP26 Critical Review Of The Reimbursement Process For Software As A Medical Device And Challenges In South Korea
Authors: Kwon Soontack , Han Seung Jin , Kim Kyung-Hoon
Published: December 2023

Introduction

The global artificial intelligence (AI) healthcare market is predicted to grow rapidly. Various technologies for AI-based Software as Medical Device (SaMD) have been developed, and demand for their health insurance reimbursement coverage is increasing. Reimbursement policies for new medical technologies need to be thoroughly examined, despite their role in stimulating the market. The reason is that health insurance finance can have significant impact on the entire country, including patients, providers, and industry.

Methods

Based on guidelines for applying Korea’s innovative medical technologies, especially AI-based imaging medical technology, to health insurance, we examined outcome factors such as procedures and benefits. After the guidelines’ publication in 2019, we examined their impact on the medical device market through changes in the number of clinical trials and identified cases in which health insurance was listed.

Results

The process of registering SaMD’s health insurance occurs in accordance with the existing medical technology evaluation system, and it can take up to 460 days from application to approval. If new technologies, including SaMDs demonstrate significant improvement in diagnostic capabilities and cost-effectiveness compared to existing practices, separate health insurance claims are available. Since the scheme’s announcement in 2019, items approved for SaMD clinical trials have increased (2018: ‘n=4; 2020: n=44; 2021: n=37). However, as of November 2022, only one was listed for health insurance benefits (VUNO Med-DeepBrain®), and one case was not listed on benefits but was recognized for its innovation and entered the market on the premise of suspending the health technology assessment process and accumulating real-world data (VUNO Med-DeepCARS®).™ DeepBrain® is a deep learning-based image reading technology costing about KRW80,000 (USD60) higher than conventional brain-magnetic resonance imaging and readings.

Conclusions

The number of SaMDs attempting clinical trials is increasing, but there is a low number of cases of reimbursement because most technologies are often classified as existing technologies and do not receive additional compensation. Since SaMD continuously is developed by accumulating data and feedback, a flexible system that can reflect this is required.