bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP39 The Real-Option Rate Of Return Approach To Inform The Pricing Of Medicines
Authors: van der Schans Simon , Schöttler Marcel , Postma Maarten , Boersma Cornelis 
Published: December 2022

IntroductionPrices of medicines have increasingly come under payer and societal scrutiny in many countries around the world. As the price-setting process is quite untransparent, the concept of cost-based pricing has been brought forward as an alternative method to inform reimbursement decision-making. A Real-Option rate of Return (ROroR) approach, was recently proposed as a method for a multi-stakeholder driven collaborative investment model. This study showed that there are public-private medicine development opportunities that could lead to lower research and development (R&D) costs for products with a challenging business model. The aim of the current study is to assess the practical use of the ROroR approach and highlight its sensitivity regarding input parameters.MethodsThe ROroR approach incorporates medicine-specific parameters: R&D costs, the number of patients treated per year, the time horizon for recouping the investments set by the stakeholders, the production costs and a predefined profit margin. Three hypothetical case-studies were selected for the ROroR analysis comprising of an orphan, oncology, and a more regular medicine. Parameter input data was derived from the available literature. Cost-based prices were calculated based on applying the ROroR equation under a constant profit margin. Ultimately, the corresponding prices of the case-studies were analyzed for their sensitivity using ten changes of the original value.ResultsThe ROroR approach was most sensitive to the length of the time horizon and the number of patients treated per year. The largest sensitivity was found for the oncological drug, with an asymmetric price change ranging from -25 percent to +271 percent if varying the time horizon or number of patients. The profit margin, and total R&D costs have the least effect on the price: +/-4 percent and +/-45 percent, respectively.ConclusionsThis study shows that cost-based pricing is highly beneficial in uncovering pricing-underlying business or economic mechanisms and suggesting a transparent price. Further research is needed on implementing public-private development models and cost-based price determination using the core parameters.
OP43 Conceptual And Methodological Factors Driving The Integration Of Real-World Evidence In Drugs And Technologies Reimbursement Appraisals
Authors: Plamondon Geneviève , Auclair Yannick , Tremblay Marie-Ève , Beha Sara , Ganache Isabelle 
Published: December 2022

IntroductionReal-world evidence (RWE) can be of value to support comparative effectiveness of drugs and technologies by providing additional information about their use for a variety of patients in real contexts of care. However, the integration of RWE in appraisals can be challenging, and INESSS felt the need to reinforce and explicit the underlying methodological and theoretical foundations.MethodsA comprehensive literature review was carried out, followed by collaborative development work by members of the methodological and assessment teams.ResultsThe literature review led to a common understanding of RWE underlying principles and fed the subsequent phases of the project. Three factors were identified as driving the integration of RWE in reimbursement appraisals at INESSS. Specifically, (i) the design and conduct of the real-world studies are done in accordance with best practices, (ii) the results are presented transparently and include all relevant information to assess the quality of the study and the data, and (iii) the RWE submitted is appropriate and relevant for decision-making. This third component is further ascertained by considering the decisional context (what are the circumstances motivating the submission of RWE and how does it correlate or not with existing evidence?), the data (is the dataset fit for decision needs?) and the study methods (are study design and analytical methods robust enough?). Globally, INESSS considers the integration of RWE in appraisals and its weighting, in relation with the (more traditional) available evidence, to be a case-by-case exercise.ConclusionsThe characterization of the main factors driving the integration of RWE in reimbursement appraisals at INESSS serves as a basis for communicating the requirements for evaluation submissions by sponsors. It further reinforces INESSS capabilities in assessing innovations, which can imply an appraisal of value at various moments along the lifecycle and with a diversity of evidence types. Considering the rapidly evolving literature and international experience, this work is expected to evolve too, and will be updated as needed.
OP48 Interactions Between Regulatory, Health Technology Assessment And Companies: Multi-Stakeholder Survey On The Current Experiences And Future Landscape Evolvement
Authors: Wang Tina 
Published: December 2022

IntroductionThe interactions between regulators, health technology assessment (HTA), and companies play a significant role in the process of getting medicine to patients. These have evolved at a product level as well as at a policy and cross-jurisdictional level; however, it is important these activities are adding value for stakeholders involved. A survey conducted in March 2021 assessed the current interactions from multi-stakeholders, and their perceptions on the added value these interactions bring to better decision-making.MethodsThree separate questionnaires containing nine questions were developed to assess the perceptions from pharmaceutical companies, regulators, and HTA agencies. The three questionnaires contained analogous questions where appropriate. The company questionnaire was sent to senior management at 19 international pharmaceutical companies, the agency survey was sent to 32 agencies (17 regulatory agencies and 15 HTA agencies) in Australia, Canada, Europe, and Asia.ResultsSeven regulators, seven HTA agencies, and nine companies responded to the survey. All regulators and HTAs indicated they have interactions with their peer agencies, as well as between regulators and HTA. The top areas of interactions for regulators were formal work-sharing between regulators during review (86% response) and regulatory strengthening (86%), whilst for HTAs, interactions between HTA on methodology/framework (83%) and HTA capacity building (67%). Regulatory-HTA interactions were seen to have fewer practical benefits, which may suggest areas for improvement. Both companies and agencies believed an effective engagement model should support evidence generation; agencies also viewed an aligned process and improved decision-making as important. Respondents believed that an ideal ecosystem for interactions should facilitate separate remits for stakeholders, converged requirements, aligned process and increased transparency and trust.ConclusionsThis survey provided a snapshot of the current landscape interactions between stakeholders during the life cycle of new medicines, identified the areas where value is added and improvement are needed. Suggested building blocks to improve future interactions included early scientific advice, alignment of evidence requirements, and a collaborative approach among all stakeholders.
OP52 Health Technology Assessment Of Pain-Free Blood Draw Devices In Pediatrics
Authors: Cristiano Ilaria , Faggiano Francesco , Esposito Maria Teresa , Porzio Ottavia , Ritrovato Matteo 
Published: December 2022

IntroductionBlood collection in a pediatric population is a time-consuming activity and an unpleasant experience. Moreover, many laboratory tests require only small amounts of blood while larger quantity of blood is usually drawn, generating excess waste that must be properly disposed of. To solve patient concerns and workflow inefficiencies biomedical companies developed Pain-Free Blood Draw (PFBD) devices. The aim of this health technology assessment (HTA) study is to compare the performances of PFBD devices with the standard venipuncture to evaluate the potential benefits of introducing PFBD devices into clinical practice.MethodsPFBD devices use microneedles that breach the stratum corneum, significantly reducing the pain perception due to the superficial skin penetration. Decision-oriented HTA method, was applied to conduct the HTA process. It is an analytical instrument that integrates the EunetHTA CoreModel with the analytic hierarchy process, to choose the best technology solution by identifying the main evaluation criteria and defining the weightsof system and performance values. Eight professionals have been involved to define the evaluation criteria and to measure the two technologies’ performance. As the method requires, a literature review was conducted to define the evaluation scheme represented by a multilevel decision tree composed of evaluation areas (domains) and key performance indicators (KPI).ResultsFive evaluation domains were included in the analysis (clinical effectiveness, safety, costs, organizational aspects, and technical characteristics), described by 35 KPIs. Preliminary clinical effectiveness results showed diagnostic concordance between blood samples obtained with PFBD and venipuncture. Even if the additional costs of PFBD, these devices seem to improve the safety by reducing the biological risks for operators. Moreover, considering pediatric patients, organizational aspects would benefit by the use of PFBD in terms of ease of use, compliance of patients, and time reduction for blood collection.ConclusionsResults showed that PFBD not only have great repercussions in terms of clinical benefits, especially for pediatric patients, but also a significant impact in terms of organizational aspects.
OP56 A Life Cycle Approach To Horizon Scanning Outputs - From Signals To Guidelines
Authors: Gonzalez Moral Sonia Garcia , Khan Sarah Khalid , Lanyi Katherine , Ogunbayo Dapo , Craig Dawn 
Published: December 2022

IntroductionThe National Institute for Health and Care Research Innovation Observatory (IO) is a horizon scanning centre based at Newcastle University, United Kingdom. The IO provides horizon scanning intelligence on new and innovative medicinal products to the National Institute for Health and Care Excellence (NICE) as technology briefing notifications (TBNs). We present an analysis of how TBNs produced between April 2017 and October 2021 feed into the NICE HTA process and used to inform their Technology Appraisal (TA) programme.MethodsTBNs were mapped to relevant published NICE TA guidance and time from horizon scanning identification to NICE recommendation was studied. For mapping technologies undergoing appraisal, provisional guidance-in-development (GID) identification numbers (IDs) were used. For technologies that had not reached the NICE scoping stage yet, the NICE Topic Selection decision and ID was used.ResultsSix hundred and ninety-three TBNs were submitted to NICE between April 2017 and October 2021; 653 were prioritised for TA. Of those, eleven percent mapped to a published NICE TA guidance; forty-three percent to a GID, twenty-two percent were undergoing consultation, and three percent were not traced. Further twenty-one percent mapped to a suspended or terminated TA. Reasons for this included HTA timeliness, regulatory issues or companies unwilling to submit evidence to NICE. Time from technology identification to TA guidance publication ranged from twenty-two to 115 months. The average time from TBN submission to NICE recommendation was thirty months.ConclusionsTimely notification is key in achieving TA recommendation aligned with market authorization but not the only influencing factor. After issuing a TBN, the NICE appraisal process might be terminated, suspended or withdrawn due to unforeseen factors. Horizon scanning plays a key role triggering the NICE TA process; understanding factors that influence the successful TA completion would streamline processes and find efficiencies.
OP62 Patients’ Opinion On Health Technology Assessment Reports: An Analysis Of Brazilian Health Technology Assessment In 2021
Authors: Komoda Denis Satoshi , Correa Carlos Roberto , Visacri Marilia Berlofa , Santos Daniela , Maia Flavia 
Published: December 2022

IntroductionThe Brazilian health technology assessment (HTA) process includes calls for public consultations, in which society can give its opinion on reports emitted by the National Committee for Health Technology Incorporation (CONITEC). Open and closed queries for public consultation are performed by official formularies and can be accessed online at CONITEC webpage. Queries are divided into two categories of reports: clinical protocols and guidelines, and incorporation/exclusion demands. Incorporation/exclusion queries are subdivided in two additional categories: opinion and experience, or technical. In this study we analyze the weight of patients’ participation in opinion and experience queries and their opinion (pro or con) on inclusion/exclusion of health technologies.MethodsFormularies concerning concluded public consultations on health technology incorporation/exclusion reports were extracted from CONITEC website from 1 January to 26 November 2021. Entries on the opinion and experience formularies included amongst others, a close-ended question about the opinion of participants on health technology incorporation/exclusion reports (“favorable”/“against”/“neither”). In this study, we analyzed patients’ opinion contained within concluded public consultations on incorporation/exclusion of health technologies.ResultsA total of 63 health technology incorporation/exclusion queries were performed in the analyzed period, of which there were only four exclusions. A total of 32,209 contributions were registered. “Patients”, “Health professionals”, “Family or caregivers”, “Interest on the theme”, accounted for 99.4 percent (13.5, 16.7, 32.3, 36.7%, respectively). Patient participation accounted for 4,367 (13.5%) entries. The total number of opinions in favor of the presented documents by the “Patients” was 4,268 (97.7%), 59 (1.4%) disagreed and 40 (0.9%) had no opinion.ConclusionsPublic consultation of official HTA reports is a very useful tool to legitimize decisions through social participation. Although patient participation is not numerically the most important category to contribute on public consultation queries, patients are, if not the most influential stakeholder, the main recipient of decisions concerning health technologies incorporations. Further analyses shall investigate experience narratives included in public consultation queries.
OP63 Patient and Public Perspectives On The Scottish Medicines Consortium Detailed Advice Document
Authors: Pierre Miranda , Dickson Jennifer , Lockhart Lindsay , McCormack Jackie 
Published: December 2022

IntroductionThe Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines, the primary output of which is a document referred to as the Detailed Advice Document (DAD). This comprises an overview of all data considered on the clinical and cost-effectiveness of the medicine, as well as the input from patient groups (PGs), patients, and carers. In 2020, SMC commenced a stakeholder evaluation of the DAD including a workshop with PGs and public partners (members of the public who volunteer with SMC) to explore the potential for using the DAD more widely.MethodsPGs and public partners, all having significant experience of engaging with SMC, participated in the workshop. Feedback was gathered using virtual post-it notes, collated and analysed for key themes. We also gathered oral feedback from participants. Sample DADs were distributed for two medicines recently appraised, one of which included a Patient and Clinician Engagement (PACE) meeting. These were chosen because they reflect different aspects of public and patient involvement at SMC, including how this is presented in the DAD.ResultsOverall, the workshop participants (n=7) recognised the DAD was a useful document for the clinicians who are its primary audience. Its language was perceived to be challenging, including complex information that is not accessible to a wide audience and may only be fully understood by those with a good understanding and knowledge of HTA. This was a key barrier to using the DAD more widely, in particular the health economics information. Suggestions for broadening the audience of the DAD included summaries of key points and an introductory section clarifying the purpose of the DAD and its intended audience, along with signposting to the plain language summary produced by SMC. These will be implemented where possible.ConclusionsImproving how SMC communicates decisions to patients and the public, by working in partnership with these stakeholders, will help strengthen public involvement throughout the HTA process.
OP68 Real-World Experience With The National Institute For Health And Care Excellence’s Willingness To Accept Less Costly, Less Effective Healthcare Technologies
Authors: Caverly Suzanne , Falla Edel , Gaultney Jennifer , Boyers Dwayne , Jacobsen Elisabet 
Published: December 2022

IntroductionResearch suggests a different willingness to pay for more effective interventions than willingness to accept (WTA) for less effective interventions which has prompted debate as to whether the threshold in the southwest (SW) quadrant should be kinked to reflect this disparity. Acceptance of less costly, less effective interventions with incremental cost-effectiveness ratios (ICERs) in the SW-quadrant presents an opportunity for resource-constrained healthcare systems by releasing resources for other purposes, which is of particular importance during a pandemic. The National Institute for Health and Care Excellence (NICE)’s methods guide suggests the threshold for decision-making for SW-quadrant interventions be the same as for more expensive, more effective interventions. To assess NICE’s WTA less effective treatments, the objective was to review the outcomes and decision drivers for interventions presenting SW-quadrant ICERs.MethodsA review of NICE health technology appraisals (HTAs) containing SW-quadrant ICERs identified from 2015-2021 was conducted. Appraisal details were extracted and analyzed to identify trends in the WTA and decision drivers.ResultsThe HTA review identified twenty-one submissions containing SW-quadrant ICERs in the base-case/scenario analysis. Eighty-one percent received a positive recommendation, with ICERs ranging from GBP 30,000-GBP 4.2m (EUR 35,264-EUR 5m) compared to a range of GBP 789-GBP 50,905 (EUR 927-EUR 59,837) for negative recommendations. The HTAs covered a wide range of therapeutic areas including psoriasis, multiple sclerosis and multiple oncology indications. Decision drivers identified that may have had a positive influence on final outcomes included a high net monetary benefit, a small QALY difference, clinical unmet need, poor tolerability of existing treatments, and oral administration route.ConclusionsThe analysis suggests that there is a high rate of acceptance of interventions with ICERs in the SW-quadrant, however, the threshold for acceptance is unclear. The high frequency of HTAs with SW-quadrant ICERs identified in this review indicates the need for further guidance on such interventions in the NICE reference case.
OP70 Gaps In The Evaluation Of Clinical Decision Support Software (CDSS): Interviews With Australian Policymakers
Authors: Laka Mah , Carter Drew , Merlin Tracy 
Published: December 2022

IntroductionClinical Decision Support Software (CDSS) can improve the quality and safety of care by providing patient-specific diagnostic and treatment recommendations. However, robust evaluation is required to ensure that the recommendations provided are clinically valid, up-to-date, and relevant to a specific clinical context. Most evaluation studies assess CDSS performance from the perspective of end-user requirements. But only occasionally is CDSS subject to stringent pre- and post-market evaluation, making it difficult to determine the safety and quality in practice. This study aimed to assess CDSS evaluation in Australia to identify gaps in evaluation approaches.MethodsWe conducted 11 semi-structured interviews with different policymakers from committees involved in digital health activities in Australia. Data were thematically analyzed using both theory-based (deductive) and data-driven (inductive) approaches.ResultsOur findings indicated that evaluating CDSS as a purely technical intervention has overly narrowed the assessment of benefits and risks by inadequately capturing the sociotechnical environment. Existing evaluation methods, adopting a static view of the implemented system, cannot discern the impact of the dynamic clinical environment and rapidly evolving technology on CDSS performance. The timeframe of evaluation studies are also incongruent with fast software upgrade cycles, with clinical practices and software potentially changing by the time evaluation is complete. The regulation of software as a medical device depends on the intended use. CDSS are exempt from regulation because they only ‘produce advice’; however, this ignores the fact that they can transition to specifying a diagnosis and treatment after a software update. There is no framework for continuous post-market monitoring, and this is especially important when a CDSS algorithm can change and impact on patient management.ConclusionsThe sociotechnical environment is a significant factor influencing the impact of CDSS on clinical practice, therefore evaluation approaches must acknowledge the dynamic nature of clinical and organizational contexts. Pragmatic and data-driven methodologies are required for CDSS evaluation that acknowledge the evolving landscape of clinical practice and its relationship to technology.