bluuebunny/crossref_metadata_2025 huggingface dataset:
OP56 Are Therapeutic Positioning Reports Driving Pharmaceutical Reimbursement Outcomes In Spain?
Authors: Dacosta Raquel Fernandez , Berardi Andrea , Macaulay Richard
Published: December 2019

Introduction

Following marketing authorization in Spain, new medicines are assessed by the Inter-Ministerial Pricing Commission for Pharmaceuticals (CIPM), which provides reimbursement recommendations with a maximum ex-factory price. However, there are 17 autonomous regions, which can make distinct reimbursement decisions. To drive consistency, the Spanish Agency for Medicines and Health Products has issued national Therapeutic Positioning Reports (TPRs) for new medicines since 2012. Since November 2017, CIPM recommendations have been published monthly, giving the opportunity to analyze the impact of TPRs on the speed and outcome of CIPM decisions, which this research evaluates.

Methods

Publicly-available CIPM and TRP decisions were identified from www.msssi.gob.es and www.aemps.gob.es, respectively. Marketing authorization dates were identified from www.ema.europa.eu or www.aemps.gob.es (10 March 2007-11 February 2018). Pearson's chi-squared and Mann-Whitney U statistical tests were performed using R.

Results

One hundred and ninety-three drug-indication pairings with an associated TPR were identified. The majority (62% [120/193]) were recommended as alternative treatment options with only 19 percent (36/193) deemed to be superior and 19 percent (37/193) not recommended. One hundred and eight CIPM recommendations were identified across seven monthly reports, issued a mean of 12.2 months after market approval, 59 percent (64/108) were positive and 41 percent (44/108) were negative recommendations. There were 34 drug-indication pairings with both CIPM and TPR recommendations available. Of these, 24 percent, 56 percent and 21 percent had TPR outcomes of ‘superior’, ‘alternative’ and ‘not recommended’, respectively and 71 percent and 29 percent had positive and negative CIPM outcomes. Drug-indication pairings with ‘negative’ TPRs were significantly more likely to have negative CIPMs than those with either ‘alternative’ or ‘superior’ TPRs (71% vs. 19%, respectively, χ2 = 5.16, p = 0.02) and were more likely to experience significantly longer delays to CIPM recommendation (23.9 vs. 13.5 months, respectively, U = 50, p = 0.03).

Conclusions

Drug-indication pairings with ‘positive’ and ‘alterative’ TPR outcomes are associated with significantly better and faster CIPM recommendations than those with ‘not recommended’ TPR outcomes
OP74 Stoma Cover Use By Fully Laryngectomized Patients
Authors: Deblois Simon , L'Espérance Sylvain , Coulombe Martin , Lepanto Luigi , Rhainds Marc
Published: December 2019

Introduction

The use of stoma covers has been a common clinical practice for laryngectomized patients for several years. In the province of Québec, Canada, laryngectomized patients can obtain stoma covers through a dedicated program providing them with medical supplies and voice re-education services. For many years, the program's supply has included cloth and/or foam covers, but the supply of Heat and Moisture Exchangers (HME) has been limited. Two hospital-based HTA units joined their expertise to assess the opportunity of providing HMEs to more patients, depending on their clinical characteristics.

Methods

Their joined assessment rested on a systematic review (SR) and a field assessment. The systematic review aimed at assessing the efficacy, clinical effectiveness and safety of various types of stoma covers. The field inquiries intended to assess the perceptions of clinicians and managers towards stoma covers in clinical practice.

Results

We included 27 studies in the SR. Most of them appraised the clinical effectiveness or safety of HME filters. Their methodological quality was very low with potential conflicts of interest whereas many studies were financed by the industry. The heterogeneity of study designs, expected outcomes and paucity of comparative studies prevented the pooling of results. Industry sponsorship appeared to be an important issue, since 17 of the included studies were sponsored. The SR did not provide conclusive evidence concerning the efficacy, clinical effectiveness and safety of the various types of stoma covers. The field inquiries intended to assess the perceptions of clinicians and managers towards stoma covers in clinical practice. It showed that industry representatives are quite active in clinical settings, promoting their products. Clinicians’ opinions and preferences were coherent with the systematic review main observations: in a context where the quality of the evidence is low, clinicians’ recommendations of stoma protectors for laryngectomies are mainly based on their professional experience and academic training.

Conclusions

Future research of high methodological quality would strengthen the evidence concerning the relative efficacy and safety of different stoma protectors. These studies would help define evidence-based allocation criteria and set parameters so that the choice of a stoma protector is best adapted to a laryngectomized patients’ condition.
OP77 Nudging In Non-Invasive Prenatal Testing: Ethical Guidance
Authors: Stanak Michal
Published: December 2019

Introduction

Non-Invasive Prenatal Testing (NIPT) has already established itself in many European countries (either via public or private institutions) as an option at hand that pregnant women can choose. Based on mother's blood, NIPT claims to “quasi-diagnose” among other things the presence of chromosomal abnormalities caused by an aneuploidy of a chromosome (such as Trisomy 13, 18, and 21). Apart from normative issues concerning the question of “whether to fund NIPT by universal coverage”, NIPT gives rise also to normative issues concerning the question of “how to put NIPT into practice” – the analysis of which is the goal of this study.

Methods

Complemented by a hand search, we have conducted a systematic literature search in Ovid MEDLINE and PsycINFO for combinations of NIPT and nudging, NIPT and participation, and NIPT and ethics. Screening was based on content analysis of titles, abstracts, and articles. Writing of the study is in progress.

Results

We identified 83 references of which 39 were included. The main instance of nudging (or also of unintentional choice design) was the use of default bias (the application or reduction of friction cost/hassle factor) that influenced the turnout to NIPT. In establishing NIPT in universal coverage systems, further potential biases identified were the use of authority bias, bandwagon effect, sunk-cost bias, and framing effect. The core ethical challenges with nudging in NIPT derive from the lack of transparency of the methods applied and the challenge of paternalism.

Conclusions

Along the line of accountability for reasonableness, four specific recommendations are suggested as the ethical guidance to using of the tool of nudging in NIPT: (i) decision makers should recognize that some choice design is inevitable, (ii) nudging should be done transparently, (iii) rationales for nudging should be publicly accessible. (iv) revision procedures should be put in place.
OP79 Improving Public Understanding Of Scottish Medicines Consortium Advice
Authors: Dickson Jennifer , Taylor Louise , McCormack Jackie , Downes Noreen , Lockhart Lindsay , Walker Laura
Published: December 2019

Introduction

Transparency of processes and decision making is important to the Scottish Medicines Consortium (SMC). An independent review of access to new medicines in Scotland in 2016 recommended that SMC should review its communication of decisions with a view to achieving greater transparency. SMC therefore began to develop plain English summaries of advice on each new medicine.

Methods

A multi-stakeholder approach was adopted to develop the summary documents, with patient groups involved. Firstly, a review of communications for the public from other HTA organizations was conducted. The public involvement team then held a workshop to find out what patient groups felt would be important to include when explaining SMC decisions to patients and the public. The process was also informed by reviewing examples of good practice from other parts of NHSScotland, including patient versions of Scottish Intercollegiate Guidelines Network (SIGN) clinical guidelines. Exemplar documents were then developed and feedback sought from the Public Involvement Network Advisory Group.

Results

A format was developed for the SMC ‘Decision Explained’ summaries consisting of a question and answer format for each medicine decision in a two page document. The summaries were piloted internally over a six month period, during which the development process and layout were finalized. Since September 2018 these summaries have been published on the website alongside the technical advice.

Conclusions

Partnership working between SMC and patient groups has helped to develop a new way of communicating SMC's decisions to patients and the public in a clear way, helping to improve transparency and understanding. Evaluation of the summaries will be undertaken from six months of publication.
OP96 Assessing Impact Of UK Health Technology Assessment Programme Trials
Authors: Carroll Christopher , Tattersall Andy
Published: December 2019

Introduction

Citation analysis is a standard tool for measuring the impact and influence of scientific work. One purpose behind controlled trials is to answer clinical and policy questions and to contribute directly or indirectly (contributing to systematic review and meta-analyses) to the production of practice guidance. The citation of trials within systematic reviews and policy or guidance documents therefore represents an authentic and meaningful measure of impact.

Methods

All 136 randomized controlled trials published by the United Kingdom (UK) Health Technology Assessment (HTA) programme in a 10-year period (2006-2015) were identified. Web of Science citation index was used to collect citation data relating to each trial. Altmetrics were used to identify additional policy and guidance documents. Citation data were collected and tabulated, and descriptive statistics produced. Additional data were collected for principal ‘spin-off’ publications.

Results

Eighty-eight percent of trials were cited by at least one Cochrane or non-Cochrane systematic review or meta-analysis; 37 percent by at least one Cochrane review (90 Cochrane reviews in total); 85 percent by at least one non-Cochrane systematic review or meta-analysis (365 in total). Forty-four percent of trials were cited by at least one unique piece of published policy or guidance. Mean number of review citations per published trial: 25.30; mean number of systematic reviews/meta-analyses per trial: 3.34; mean number of guidance documents per trial: 0.85. Trial investigators published the primary clinical outcome data in 27 additional peer-reviewed journal articles, generating citations in a further 66 unique reviews and 22 unique guidance documents.

Conclusions

Based on the payback model, this sample of 136 UK HTA trials represent meaningful impact: 88 percent of trials were cited in systematic reviews and 44 percent in guidance documents. Chronological data indicate that there might be a sizeable time-lag between publication and impact, especially for policy documents and Cochrane reviews.
OP103 Incorporating Health Technology Assessment In The Development Of A Clinical Care Pathway
Authors: Benkhalti Maria , Dagenais Pierre
Published: December 2019

Introduction

Clinical care pathways (CPWs) provide a step-wise multidisciplinary care plan for patients with a particular health condition. Their aim is to optimize patient outcomes and organization of care by supporting evidence-based practice. It therefore seems inevitable that health technology assessment (HTA) should be incorporated within the development process of a CPW. As CPWs become increasingly utilized, there is a need to understand the added value and strategies to integrating HTA in the development of a CPW.

Methods

Through a case study of an HTA on treatments for chronic low back pain requested as part of the development of a CPW for chronic musculoskeletal pain, we demonstrated the three key strategies to include HTA in CPWs described by Rehaluk 2016 and added a fourth one. We then showed how these strategies contribute to the development of a CPW which answers the quality criteria outlined by the Cochrane Effective Practice of Care group through a strength, weaknesses, opportunities, and threats analysis.

Results

We confirmed four key strategies to including HTA in CPWs (organizational positioning of the HTA unit, partnership and communication with stakeholders, tailoring the integration of contextual data with evidence from the literature, explore tools to facilitate the use of HTA findings). The inclusion of HTA through these strategies contributes to the development of a CPW which meets the ten criteria to evaluate the quality of a CPW outlined by the Cochrane Effective Practice of Care group. Through a strength, weaknesses, opportunities, and threats analysis, we describe how each of the criteria were met and how this led to recommendations influencing our regional organization of care.

Conclusions

The inclusion of HTA in CPW development increases its capacity to directly influence organization of care. HTA can represent a pivotal vehicle to ensure good quality CPWs.
OP124 Disinvestment – A Global Challenge Requiring Collaboration?
Authors: Warner Mary
Published: December 2019

Introduction

Australia has had some success at utilizing Health Technology Assessment (HTA) to disinvest and reassess medical services. This has been achieved through a range of methods including identifying services through initiatives such as ‘Choosing Wisely’, examining real world service data and seeking expert clinical opinion. This presentation will discuss how better international collaboration in disinvestment and reassessment methods using HTA could lead to more efficient health care systems.

Methods

Both the Australian and South Korean governments have a particular interest in disinvestment and reassessment in their health care systems. These countries have been sharing information over the past two years with a common goal of improving their health systems through a rigorous reassessment process. The Australian Government is in the process of reviewing all publicly funded services utilizing expert clinical committee advice, often referring the reassessment of services to a HTA process. A similar process is also being undertaken in South Korea.

Results

Australia has disinvested in a wide range of services using HTA, including hip arthroscopy, lipectomy and hyperbaric oxygen therapy. It is also undertaking an extensive reassessment of 5,700 services. Reassessment may not lead to HTA, but it often includes an examination of whether a service should be subjected to HTA to remain publicly funded. Australia and South Korea have similar approaches in undertaking disinvestment and reassessment. HTA disinvestment and reassessment strategies have generated good outcomes for consumers, health care providers and funders in both countries.

Conclusions

Disinvestment and reassessment of medical services require funders that support the continual improvement of health care systems. Disinvestment and reassessment HTA can be difficult, mainly due to external interests - an issue experienced by many countries. Further international collaboration in this area may provide a more supportive environment to undertake HTA for disinvestment.
OP152 Pharmacoeconomic Assessment And Drug Expenditure Reduction In Ireland
Authors: Kennedy Cormac
Published: December 2019

Introduction

All new products to be reimbursed from the Irish health budget are subject to a rigorous assessment by the National Centre for Pharmacoeconomics (NCPE). Following assessment, a recommendation is made regarding its cost-effectiveness at the submitted price. This may lead a reduction in the drug price. This study aimed to determine the reduction in expenditure due to the pharmacoeconomic assessment process in Ireland.

Methods

Product details, submitted price and gross budget impact were recorded for each NCPE submission from 2012 to 2015. The latter was chosen as reimbursement data are currently available until 2016. A product was included if its assessment suggested price reduction was required and the product was reimbursed under the High-Tech Drug Scheme (HTDS), a scheme for high cost drugs in a primary care setting. The utilization and actual expenditure of each product was extracted from national reimbursement data for the year after approval. The expected expenditure, calculated using the submitted price, was then compared to the actual expenditure.

Results

A total of 162 products were assessed during the study period. There was a potential price reduction for 65 products based on the assessment outcome. Of these, 15 were reimbursed under the HTDS. A reduction in expenditure was evident for eight of the 15 products (53 percent). The average reduction was eight percent of the expected expenditure. All products showed an actual expenditure greater the predicted budget impact submitted by the applicant.

Conclusions

To the authors’ knowledge, this is the first report of expenditure reduction due to a pharmaco-economic assessment process. With the ever-increasing utilization of high cost drugs, the study demonstrates the importance of a process to assess and negotiate cost-effective drug prices. However, the study underestimates reductions, as it is yet to include commercial rebates returned to a central budget. Future research will aim to capture these reductions.
OP169 Implementation Of A Quality Management System In The Spanish Network Of Health Technology Assessment
Authors: Márquez-Peláez Sergio , Viguera-Guerra Isabel , Molina-Lopez Teresa , Amaro Juan-Antonio Blasco ,   Workgroup REDETS
Published: December 2019

Introduction

The Spanish network of health technology assessment (HTA) agencies (REDETS) is a collaboration of eight agencies, units and services, commissioned by the national and regional governments. The network coordinates work within a common methodological and work framework, guided by the principles of mutual recognition and cooperation. In 2016, a common self-assessment quality tool was developed for the implementation of an overall Quality Management System. Currently we are working on the second step that deals with actions about management, joint activities as a network, and organizational aspects of the network.

Methods

A structured search strategy in the main electronic databases and a manual search in websites of networks national and international agencies were carried out in June 2017, in order to gather previous knowledge and developed standards. Through the information included in this review, and with the collaboration of all members, a group of standards for REDETS was developed. Finally, standards proposed were discussed in a face-to face meeting until an agreement was reached.

Results

A proposal of 31 standards was put forward taking all the collected information. The aim of each standard was defined, and its level of compliance was specified. Those standards were grouped under nine quality criteria structured in four dimensions: (i) Responsibility and Resources, (ii) Performance and Membership, (iii) Procedures, and (iv) Relations.

Conclusions

Based on the gathered information and the agreement of the all members, we developed a toolkit embracing a group of standards for the joint activities within the Spanish Network, network administration and management. It is a complementary instrument of the previous self-evaluating tool, following the establishment of an overall quality management system and under the philosophy of continuous improvement processes.
PP21 High Risk Class Medical Devices Evaluation In Germany: Another Arzneimittelmarkt-Neuordnungsgesetz?
Authors: Müller Elvira , Oelze Ilse-Barbara , Neeser Kurt
Published: December 2019

Introduction

In 2011 the Arzneimittelmarkt-Neuordnungsgesetz (AMNOG) evaluation process for new drugs was implemented in Germany. Since then, the evidence requirements follow high standards and results impact reimbursement price negotiations. More recently, in 2016, a legal norm (§137h SGBV) to evaluate new treatment and diagnostic methods (MDs) of high risk classes by the Federal Joint Committee (G-BA) was introduced. The requirements, involved stakeholders, timing and results for both processes are outlined and compared.

Methods

Methodological guidelines from G-BA and Institute for Quality and Efficiency in Health Care (IQWiG), consultations and evaluations for MDs according to §137h and for drugs according to AMNOG were reviewed and compared. Published assessment results were analyzed according the decision criteria and impact on price negotiations with Statutory Health Insurance.

Results

Hospitals need to submit jointly with the manufacturer comparative evidence on clinical efficacy, safety and cost when applying for additional compensation (Neue Untersuchungs- und Behandlungsmethoden [NUB] application) for new high risk class MDs being subject to §137h. A fast track assessment by IQWiG/G-BA follows within four months resulting in benefit proven, potential benefit or no benefit compared to alternatives. The latter can lead to exclusion from reimbursement. Until now one MD was granted a benefit, two treatments were assigned a potential benefit and six MDs no benefit, while 55 percent of drugs evaluated under AMNOG were granted an additional benefit. Compared to drugs, the required evidence for MDs is similar. Whereas assessment time is shorter, manufacturers can seek advice from G-BA upfront for free and need to collaborate closely with hospitals.

Conclusions

Half of MDs examined did not qualify for an assessment under §137h. Unlike for drugs evaluated under AMNOG, the majority of new MDs failed to be granted potential benefit as a treatment alternative and might be excluded from reimbursement. Manufacturers are challenged to generate high quality, comparative evidence within their studies.
PP26 Shift From Regional To Federal Funding: Methodological Considerations
Authors: Newton Skye , Kessels Sharon , Vogan Arlene , Merlin Tracy
Published: December 2019

Introduction

Australia has a two-tier public funding system, and many genetic tests are funded by different states and territories prior to being considered for public funding by the Federal government. In this context, health technology assessments (HTAs) of genetic tests for heritable conditions are problematic. We aimed to discuss the possible impacts on HTA methodology of a shift from regional to federal funding for genetic testing for heritable conditions.

Methods

Several HTA reports and economic models on genetic tests considered by the Medical Services Advisory Committee (MSAC) were reviewed and compared to ‘real world’ clinical practice.

Results

Every HTA of germline testing performed for the MSAC have so far compared genetic testing versus no genetic testing. However, testing for BRCA1/2 for patients with breast cancer currently occurs in Familial Cancer Centres, and testing for germline mutations for familial hypercholesterolaemia currently occurs through specialist lipid clinics. In both settings, the index patient and family members are given multidisciplinary support, including genetic counselling. The HTA comparison therefore did not reflect what the true clinical and cost-effectiveness impact of federal funding would be. Federal funding means that tests may be ordered by a broader range of specialists or general practitioners. The evidence identified was predominantly sourced from specialised centres, where knowledge regarding how to interpret the tests is high. The clinical utility of these tests largely depended on how clinicians understood and conveyed the results.

Conclusions

The benefit of testing may have been overestimated due to the comparator and setting used (i.e. specialised and centralized care, associated with high clinical utility). Any HTA of genetic testing for heritable conditions, which could result in a shift in the delivery of testing or care for the patient, should consider the applicability of the evidence identified. Further, it should assess the subsequent impact this may have on the effectiveness and cost-effectiveness of the test and the quality of care provided for patients and their family.
PP28 Adoption Of Non-Pharmaceuticals In Galicia: Beyond Conventional Health Technology Assessment
Authors: Varela-Lema Leonor , Zapata-Cachafeiro Maruxa , Pego Yolanda Triñanes , Vallés Maria José Faraldo
Published: December 2019

Introduction

The specificities of non-pharmaceuticals can require adapting classical health technology assessment (HTA) methodologies and developing additional regional approaches to support decision-making processes. However, little information exists regarding the explicit approaches used in different countries. The aim of this work is to provide an overview of the role and activities of the Galician HTA agency (avalia-t, Spain) regarding assessment, appraisal and continued evaluation across the whole life cycle of non-pharmaceutical technologies.

Methods

In depth review and analysis of the activities undertaken by avalia-t during the past five years to support the introduction and appropriate use of non-pharmaceutical health care technologies at the regional level.

Results

A multidisciplinary Commission judges the added value of new non-pharmaceuticals and establishes the indications and conditions for use. HTAs, which are mandatory for all relevant technologies, rely on the best available evidence on safety and effectiveness but also provide fit for purpose contextualized information based on organizational data and administrative registers. Interaction with multidisciplinary stakeholders is commonly needed to complement the evidence base (ad hoc working groups, face to face discussions), and post-launch studies can be implemented to analyze the utilization and results in real world practice. Performance indicators and other HTA based products can also be required to ensure the quality of health care (e.g., appropriate use indications, quality indicators, evidence based patient information). In addition, technical and scientific advice/support can be provided at different decision levels of the health organization to promote the quality of care and appropriate use of technologies (e.g., regional mental health program, suicide management strategy, bariatric surgery surveillance registry).

Conclusions

Rigorous, comprehensive and systematic processes for supporting non-pharmaceutical technology adoption and implementation are required. Although it is acknowledged that core information does not differ substantially within countries, contextualized information is recognized as essential for establishing the conditions for use at the regional level.
PP31 Medical Device Regulation: What Is New?
Authors: Oelze Ilse-Barbara , Neeser Kurt , Müller Elvira
Published: December 2019

Introduction

In 2017, the European Union (EU) commission released the final versions of the Medical Device Regulation (MDR) and In-vitro Diagnostic Device Regulation. These regulations will replace the EU directives (Medical Device Directive [MDD], In-vitro Diagnostic Device [IVDD], and Active Implantable Medical Device [AIMD]). EU regulations are effective in all EU countries at date of publication. In contrast, the EU directives must be implemented in national law first.

Methods

Guidelines and respective legislation, consultation results and methods/medical device (MD) evaluations were reviewed and analyzed. Decision criteria and reasoning, assessment outcomes and potential impact on price negotiations were the main aspects for comparison.

Results

Manufacturers have to be aware of the importance of clinical data for demonstrating the compliance of their products. This applies both to the approval of the products and the “post-market activities” and particularly to the “post-market clinical follow-up” for which requirements for Class I and II products need to be further developed. The MDR requires manufacturers to collect clinical data before and after approval, which could lead to excessive documentation requirements. The term “sufficient clinical data” from the MDR is unclear. A functional Eudamed specification is necessary, which enables an automated processing of relevant data. A stronger involvement in the evaluation process is needed as well as more transparency in the Joint Federal Committee (G-BA) and faster evaluation processes.

Conclusions

The MDR increases the burden especially for small businesses, and it is doubtable that the ultimate goal – improving patient safety – will be achieved. The increased demands and rising costs of the new EU MDR and bottlenecks at Notified Bodies can be a risk for the MD industry. Due to the general reduction in the remuneration for services with a high proportion of technical services, it is feared that products will be withdrawn from the market for economic reasons or that they will not be marketed.
PP66 Increasing Burden Of Out-Of-Pocket Healthcare Expense On Patients
Authors: Arora Rhythm , Dugar Nikhil , Saxena Vandit , Jaiswal Sunil K. , Kumari Chitresh , Cook Nigel , Furio Olga
Published: December 2019

Introduction

We conducted an analysis of the key factors triggering cost-sharing mechanisms to understand the status of out-of-pocket (OOP) healthcare expense in the United States (US), Europe, and emerging markets and better appreciate the implications of OOP healthcare expense on patients’ health management.

Methods

A review of literature and databases including The Organisation for Economic Co-operation and Development (OECD) and World Bank was performed to understand different cost-sharing mechanisms, factors triggering OOP expenditure and the country-wise trends of OOP expenditure. Additionally, the impact of OOP expenditure on healthcare budget and on patients in terms of medication adherence, uptake of newer therapies and generic substitution was explored.

Results

The findings reveal that patients are concerned about rising healthcare OOP costs, and we observed an increase of 134 percent in the number of articles published on OOP from 2005 to 2017. The percentage of household spending that goes OOP as healthcare expense is higher in Brazil, Russia, India, and China (BRIC countries; ~11 percent) compared to France, Germany, Italy, United Kingdom, US, Japan, and Canada (G7 countries; ~2 percent). In addition, OOP expenditure increased with age (1.9 percent of take home income in 55-64 age group versus 1.2 percent in 18-25 age group) and is higher in the low-income population (2.8 percent of take home income versus 1 percent in high-income group). Whereas, increasing OOP expenditure reduces the overall healthcare expenditure due to generic substitution (28 percent reduction) and reduction in excessive consumption of supplementary medicines, it also reduces patient adherence (~20 percent decline in dispensed prescriptions) and may foster a reluctance to adopt newer therapies.

Conclusions

The population groups most impacted by increasing OOP expense are the older population, those in the low-income bracket and in poorer countries. While OOP expense may help in the effective and judicious utilization of healthcare system resources and medicines usage, its implementation requires a cautious and considered approach.
PP83 A Conceptual Decision-Making Framework For Pharmaceutical Innovations
Authors: Boersma Cornelis , Geenen Joost , Postma Maarten
Published: December 2019

Introduction

The trend of growing healthcare expenditures is unsustainable in many countries. The increasing pressure on healthcare budgets due to, for example, population ageing, increasing numbers of patients with chronic diseases (including multimorbidity), and the introduction of new pharmaceutical innovations, leads to political and societal debate. In particular, the introduction of expensive pharmaceutical innovations causes a lot of discussion and uncovers various paradoxes and dilemmas. There is a societal demand for innovation focused on existing medical needs (e.g., oncological, immune-mediated inflammatory, and orphan diseases), but the price of pharmaceutical innovations is a barrier to patient access. As a consequence, systems try to introduce measures or incentivize market forces to improve access for patients, while also containing budget impact. This does not always lead to better access and affordability. The aim of this study was to develop and test a conceptual decision-making framework for pharmaceutical innovations.

Methods

A retrospective study was conducted to identify the successes and challenges of decision-making systems across Europe. A conceptual decision-making framework, including proposed procedures, criteria, and health technology assessment (HTA) requirements (including tools), was developed and tested based on specific case examples (e.g. oncology and hepatitis C).

Results

The conceptual decision-making framework comprised an algorithm for relevant decision-making criteria (e.g. clinical evidence, medical need, cost-effectiveness, and budget impact). The algorithm was developed hierarchically and ranked the criteria in order to optimally inform various types of investment decisions. This novel approach to conducting budget impact analyses resulted in more realistic predictions of the burden of pharmaceutical innovations on healthcare budgets, and can be used as part of horizon-scanning processes to inform healthcare decision making. Results from selected case examples are presented.

Conclusions

The conceptual decision-making framework and proposed method for budget impact predictions will allow for more balanced future healthcare investment decisions.
PP84 Different Interpretation Of Evidence By A Health Technology Assessment Body And A Decision Maker
Authors: Dintsios Charalabos-Markos , Ruof Jörg , Worm Franziska , Herpers Matthias
Published: December 2019

Introduction

Within early benefit assessment of pharmaceuticals in Germany, addenda can be commissioned by the Federal Joint Committee (FJC) to the health technology assessment (HTA) agency, mainly as a result of a hearing. Our aim was to analyze the issues for and impact of commissioned addenda, as well as the agreement between HTA agency recommendations and FJC decisions.

Methods

All available relevant documents on addenda commissioned up to the end of 2017 were screened and their essential content extracted. Differences between the HTA agency and FJC recommendations were tested, and concordance was analyzed using agreement statistics (Cohen's kappa and Fleiss’ kappa).

Results

Most of the 90 addenda commissioned up to the end of 2017 concerned oncological products. In all contingent comparisons, positive changes in added benefit or evidence level on a subpopulation basis (n = 124) were more common than negative changes. Agreement of assessments, addenda, and appraisals reached a moderate strength for added benefit (Fleiss’ kappa 0.47, range 0.41 - 0.54). Overall agreement between addenda and appraisals on a binary nominal basis was poor for added benefit (Cohen's kappa 0.18, range 0.01 - 0.36) and fair for evidence quality (Cohen's kappa 0.35, 0.19–0.52). Cohen's kappa ranged from “less than by chance” (respiratory diseases) to “perfect” (neurological diseases), but was only statistically significant for neurological and other diseases. Three addenda are presented in detail as examples.

Conclusions

Addenda have a high impact on decision-makers’ appraisals, offering additional analyses of supplementary evidence submitted by the manufacturers. Nevertheless, the agreement between addenda and appraisals varies, highlighting different methodological approaches and decision-making factors between the HTA agency and the FJC.
PP86 Reimbursement of Combination Oncology Products: Can Two (Companies) Tango?
Authors: Turkstra Erika , Liu Lok Wan , Berardi Andrea , Macaulay Richard
Published: December 2019

Introduction

A range of innovative, targeted anti-cancer therapies have been developed over the past 20 years. More recently, companies have been developing combinations of these drugs. While this promises substantial efficacy benefits, dual-brand oncology therapy combinations may potentially create substantial economic burden. Obtaining a positive health technology assessment (HTA) recommendation and public reimbursement can be a major challenge, and may be more difficult when each constituent monotherapy is marketed by a different company. We evaluated whether dual-brand oncology therapies developed by a single manufacturer had faster or better outcomes than those developed by two separate manufacturers.

Methods

Recent combination oncology drug products were screened in November 2018 to identify whether one or two manufacturers were involved. The websites of various HTA organizations were screened and the relevant data extracted.

Results

A total of 78 recommendations for dual-brand oncology treatments were identified across the HTA agencies screened: 26 of these were for combinations by the same manufacturer and 52 were for combinations with two manufacturers. Dual-brand therapies developed by a single manufacturer were more likely to receive full or optimized/conditional recommendations (58% “recommended” and 12% “optimized/conditional”) than those marketed by two separate manufacturers (42% “recommended” and 8% “optimized/conditional”). Dual-brand therapies with two manufacturers were more likely to receive negative HTA recommendations than those marketed by a single manufacturer (50% versus 31%). However, the median time from marketing authorization to recommendation in European countries was the same (6 months), regardless of whether each constituent monotherapy was marketed by one or two manufacturers.

Conclusions

HTA agencies were more likely to issue negative recommendations for dual-brand oncology treatments marketed by two separate companies, compared with those marketed by a single company. A single company may have more flexibility in price setting, which may facilitate more positive HTA recommendations.
PP89 Cost Effectiveness Of Hepatitis A Vaccination In India
Authors: Gurav Yogesh , Bagepally Bhavani Shankara , Thavorncharoensap Montarat , Chaikledkaew Usa
Published: December 2019

Introduction

Due to epidemiological transition, a rise in hepatitis A outbreaks among adults in the state of Kerala, India has been noted. This has intensified the need for hepatitis A vaccination (HAV), but evidence regarding the cost effectiveness of HAV, which is essential to guide policy decisions, is lacking. This study was undertaken to evaluate the cost effectiveness of HAV among adults in Kerala state.

Methods

To determine the cost effectiveness of HAV from a societal and a payer perspective, a Markov model was constructed with a cycle length of two months. The lifetime costs and outcomes for HAV and no vaccination were compared using a discount rate of 3 percent. Data for the model input parameters of cost, coverage, and effectiveness were derived from the published literatures. One-way and probabilistic sensitivity analyses were applied. A threshold based on the per capita gross domestic product (GDP) was used (1 GDP = INR 127,702.48 [USD 1,886.03]).

Results

The incremental cost-effectiveness ratios for both societal and payer perspectives were negative, indicating that HAV was dominant, being less costly and more effective than no vaccination. The discount rates and utility values for adults with HAV were the most sensitive parameters.

Conclusions

A HAV strategy would be cost-saving, compared with no vaccination, in the Kerala state of India.
PP121 How To Involve Patients In Decisions About Antibiotic Prophylaxis After Tick Bite
Authors: Bouchard Sylvie , Gernigon Gaelle , Karam Fatiha , Daigle Jean-Marc , Morrow Genevieve , Freitas Adriana , Tardif Mélanie
Published: December 2019

Introduction

Antibiotic prophylaxis with a single dose of doxycycline after a tick bite is one of the tools for preventing Lyme disease, which is becoming increasingly prevalent in Quebec. The aim of this work was to revisit this practice in adults and children younger than 8 years of age.

Methods

To assess the safety and absolute risk reduction (ARR) of doxycycline for preventing Lyme disease in contraindicated populations, two systematic reviews were conducted with a re-analysis of the original efficacy data. A knowledge mobilization framework was used to consider the scientific, contextual, and experiential evidence, taking into account information on patients’ and clinicians’ experiences.

Results

A single dose of doxycycline prescribed within 72 hours of being bitten by a tick (Ixodes scapularis) could prevent cutaneous manifestation of Lyme disease (ARR -2.8%, 95% confidence interval: -11.7–6.1; p = 0.06), without serious side effects, provided that the bite occurred in a geographical region where at least 25 percent of nymph and 50 percent of adult ticks are infected with the disease. However, the level of evidence was low and its generalizability to other contexts was doubtful. The decision to prescribe antibiotic prophylaxis may be based more on the fear of Lyme disease, rather than on effectiveness data and the real risk of contracting Lyme disease.

Conclusions

It may be challenging for clinicians to discuss Lyme disease prophylaxis with patients and their families in contexts where people are fearful of the disease, and the risk of contracting it from a tick bite is uncertain. Decision aids that provide scientific evidence on the real risk of developing Lyme disease after a tick bite, particularly in Quebec, can promote informed decisions based on patient preferences and values by supporting discussion between clinicians and patients.
PP170 Quantifying the Life-Cycle Value of Second Generation Antipsychotics
Authors: Berdud Mikel , Wallin-Bernhardsson Niklas , Zamora Bernarda , Lindgren Peter , Towse Adrian
Published: December 2019

Introduction

We estimate the life-cycle value of risperidone – Second-Generation Antipsychotics (SGA) – to balance the view that cost per Quality-Adjusted Life Year (QALY) estimates at launch are enough to guide access decisions. Study results will also drive discussion on access and price to recognize the dynamic nature of pharmaceutical pricing over the long-run.

Methods

We estimated number of patients treated for schizophrenia with risperidone in Sweden and the United Kingdom (UK) between 1994-2017 based on usage data form national statistics and volume sales data from IQVIA. We collected data from literature on the effectiveness (QALYs) and costs (EUR 2017) of risperidone (SGA) and haloperidol – First-Generation antipsychotic (FGA). We estimate the life-cycle value added by risperidone versus haloperidol, and the life-cycle distribution of the social surplus between the payer (consumer surplus) and the innovator (producer surplus).

Results

We estimated the consumer surplus, the producer surplus, the Net Monetary Benefit (NMB) and Incremental Cost-Effectiveness Ratio (ICER) at each year and in aggregate terms (1993-2017). For the UK the producer surplus was ~28 percent out of the total surplus before patent expiration and five percent after patent expiration. In Sweden, producer surplus was around 6 percent out of the total surplus before patent expiration and one percent thereafter. In both countries, during the life-cycle of risperidone, the NMB per patient increased and the ICER decreased as a response to: (i) the launch of Risperidone Long-Acting Injectable (RLAI); and (ii) the generic entry.

Conclusions

The value added by risperidone increased during the life-cycle due to the launch of RLAI and the generic competition. This suggests that, considering the entire life-cycle, the value added by SGAs to the system is higher than the expected value estimated using cost-effectiveness analysis at launch. Pricing and reimbursement decisions should take into account the dynamic nature of pharmaceutical markets and the value added by innovative medicines over the long-run.
PP179 Health Technology Assessment Of Pediatric Intensive Care Ventilators
Authors: Di Mauro Roxana , Faggiano Francesco , Andellini Martina , Derrico Pietro , Ritrovato Matteo
Published: December 2019

Introduction

A health technology assessment (HTA) process to evaluate the best intensive care ventilator manufacturers has been carried out in different pediatric intensive care units (ICUs) of Bambino Gesù Children's Hospital (OPBG). The purpose of this study is to determine: (i) the most relevant features of a ventilator to be considered between different manufacturers, and (ii) the methodology to conduct the assessment to support the decision-making process about the choice to adopt the suitable technology for OPBG.

Methods

The decision-oriented HTA method (Do-HTA), developed by the HTA unit of OPBG, was applied to conduct the assessment. Do-HTA involves the integration of the European Network for HTA (EUnetHTA) CoreModel and the Analytic Hierarchy Process with the support of an informatics tool. It provides the definition and numerical evaluation of assessment parameters to evaluate the performance of technologies. A literature review involving ICU professionals was used to define and weight the assessment elements on clinical, technical, organizational, economic, and safety domains. In particular, a subgroup of these domains has been included in a checklist for the comparative evaluation of different ventilator models, each of which was tested in three independent runs performed in three different ICUs.

Results

Results show that safety and clinical effectiveness had highest the impact within the evaluation, followed by organizational, technical and economic aspects. A percentage value per each ventilator has been assigned, representing the global performances regarding the assessment elements.

Conclusions

This study presents and discusses the benefits and drawbacks of innovative features of ventilators, all characteristics to be taken into account during the evaluation process and a methodology to conduct it. The project identified the best performing ventilator model through a collective decision, giving a reliable recommendation to the Hospital Decision Makers.
PP186 Telemonitoring With Pacemakers For Patients With Heart Failure
Authors: Myles Susan , Poole Ruth Louise , Facey Karen
Published: December 2019

Introduction

Evidence supporting the use of pacemakers is well established. However, evidence about the optimal use of pacemaker telemonitoring for disease management in heart failure is not. Health Technology Wales (HTW) held a national adoption event to encourage implementation and best practice in use of pacemaker telemonitoring in the National Health Service (NHS) Wales to improve patient outcomes in heart failure.

Methods

Multi-stakeholder national adoption workshop using a mixture of expert presentations, case studies and interdisciplinary group and panel discussions to agree key actions to understand the value and promote optimal use of pacemakers for remote disease monitoring in patients with heart failure in Wales.

Results

The workshop was attended by forty-five senior professionals with an interest in improving care of patients with heart failure. Actions to progress included: providing a centralized Welsh system to support technical issues that arise with telemonitoring; considering interoperability with other NHS Wales systems; encouraging value-based procurement with collection of a core outcome set; agreeing implementation issues with both professionals and patients; audit to understand experience, resource use and outcomes; and sharing manufacturer evidence on the accuracy of telemanagement algorithms. It was suggested that these actions be progressed via an All-Wales multi-stakeholder approach, led by the Welsh Cardiac Network.

Conclusions

Developing a more agile, lifecycle approach to technology appraisal is currently advocated; recalibrating the focus from technology assessment to technology management across the complete technology lifecycle. HTW will endeavour through regular adoption events to facilitate such a paradigm shift that aims to understand value and optimise use of evidence-based technologies.