bluuebunny/crossref_metadata_2025 huggingface dataset:
OP94 Is The National Institute for Health And Care Excellence In The United Kingdom More Innovation-Friendly Than The German Institute For Quality And Efficiency in Health Care In Germany?
Authors: Schaefer Ramon , Schlander Michael
Published: January 2018

INTRODUCTION:

Whereas Health Technology Assessments (HTAs) by the National Institute for Health and Care Excellence (NICE) rely heavily on cost utility analysis, HTAs by the German Institute for Quality and Efficiency in Health Care (IQWiG) and the Federal Joint Committee (GBA) focus on an assessment of comparative effectiveness, rejecting a cost per quality-adjusted life year benchmark. The present study aimed to explore the differential impact of methodological choices by NICE and IQWiG/GBA on HTA outcomes.

METHODS:

We extracted data from all GBA decisions between January 2011 (when early benefit assessments were implemented) and April 2015 (cut-off date for the present study), as well as all single technology appraisals (STAs) by NICE published during the same period. We compared early benefit assessment results by IQWiG/GBA and by NICE overall, and by additional criteria including therapeutic area, clinical and incremental cost effectiveness, and patient-relevant endpoints.

RESULTS:

During the study period, NICE issued guidance for 88 technologies (with 125 subgroups). GBA completed 105 appraisals (with 226 subgroups). We identified thirty-seven matched condition-intervention pairs; of these, twenty-four were evaluated differently by NICE and GBA. NICE recommended twenty-nine of thirty-seven interventions (78 percent), whereas GBA confirmed additional benefit for 21/37 only (57 percent; p< .05, two-tailed chi-square test). By therapeutic area, NICE was more likely to evaluate interventions for metabolic and cardiovascular disorders favorably, whereas IQWiG/GBA appraisals were more favorable for treatments of hematological and oncological diseases. Results including all HTAs were consistent with those for matched pairs.

CONCLUSIONS:

Our results suggest that, overall, NICE tends to evaluate new interventions more favorably than IQWiG/GBA. However, our analysis revealed conspicuous differences by therapeutic area. The results are consistent with the hypothesis that different methodological choices may lead to systematic differences in decision making. It seems plausible that the observed differences reflect, at least in part, differences in underlying value judgments.
OP103 CONITEC's Rapid Reports As Technical Support In The Litigation
Authors: Simabuku Eliete , Biella Carla , Catanheide Izamara , Silva Sarah , Canuto Vania
Published: January 2018

INTRODUCTION:

The Unified Health System (SUS) is based on the principle of health as a citizen's right and the state's duty, which must be guaranteed based on public policies. Although there are several legislations, lists of medicines and clinical guidelines, Brazilians who have been prescribed expensive technologies that are not part of the essential drug lists ask judges to issue court orders obliging public health managers to purchase these drugs or to provide elective medical procedures immediately. Due to the health technical inexperience from judges, prosecutors and public lawyers, a partnership has arisen for the National Committee for Health Technology Incorporation (CONITEC) to provide technical assistance to help their decision-making process. Thus the purpose of this study is to describe CONITEC's experiences in communicating with stakeholders in this process.

METHODS:

A case study method was used and information about the rapid reports developed by CONITEC's Executive Secretariat in response to the applicants in the period of 2012 to 2016, was retrieved from CONITEC database.

RESULTS:

Rapid reports (2,773) about health technologies incorporation such as medicines, procedures or medical devices were produced by CONITEC during this period. Most requests covering topics as treatments for diabetes, arterial hypertension, osteoporosis, oncology and epilepsy; diseases for which there are several treatment options in SUS. The data analysis indicated that CONITEC contributed to the evidence based decision-making. On one hand, the Prosecutor's Office has been increasingly requesting information before starting lawsuits and Judiciary Power has increasingly used evidence-based technical information before deciding on the concession of injunctions; on the other hand, from 2012 to 2016 the number of requests decreased for information to State defense in lawsuits that has been already established.

CONCLUSIONS:

There is a growing interest in technical knowledge for fair decision making that respects the current organization of the evidence-based health system.
OP115 Effect Of Multiple Drug Resistance On Costs For Patients With Intra-Abdominal Infections in China
Authors: Zhen Xuemei , Li Yuanyuan , Chen Yixi , Dong Peng , Liu Stephanie , Dong Hengjin
Published: January 2018

INTRODUCTION:

Multiple drug resistance (MDR) intra-abdominal infections (IAIs) are associated with noteworthy direct and societal costs. Compared to previous studies, the present one takes both resistance rate and total medical costs (TMCs) into consideration, focusing on the impact of MDR on TMCs in IAIs, as well as further estimating the additional costs at a national level.

METHODS:

All inpatients discharged between 1 January 2014, and 31 December 2015 from a teaching hospital were included. Due to limits in budget and the large number of inpatients, the randombetween (bottom, top) function was applied to randomly select 40 percent of patients per year. Subsequently, we manually screened out 254 patients with IAIs, according to the International Classification of Disease (tenth revision) and electronic medical records. Eventually, 101 IAIs patients were included, in which 37 were infected by non-MDR bacteria and 64 by MDR bacteria. The Kruskal-wallis non-parametric test and multiple linear regression were employed to analyze the effect of single and multiple variables on TMCs.

RESULTS:

Compared to patients with non-MDR infections, those with MDR were associated with significantly higher TMCs, higher antimicrobial costs, increased insurance, combination antimicrobial therapy, higher usage of antimicrobial agents, greater number of pathogens, longer length of stay, and longer intensive care unit stays. In addition, the average TMCs among patients with MDR were CNY131,801.17 (1USD was equal to CNY 6.227 in 2015), which were CNY 90,200.99 higher than those with non-MDR infections. If our results are generalizable to the whole country, the total attributable TMCs are estimated to be CNY37.06 billion, and the societal costs of CNY111.18 billion in 2015.

CONCLUSIONS:

This real-world data analysis demonstrated the significant excessive burden MDR infections are posing to the current Chinese healthcare system in terms of both TMCs and healthcare resource utilization. Enhanced antimicrobial stewardship in China is necessary to curb the distribution of MDR bacteria.
OP118 Cost-Effectiveness Analysis Of Molecular Profile Selection For Advanced Head And Neck Cancer
Authors: Rognoni Carla , Bossi Paolo , Licitra Lisa , Quaglini Silvana
Published: January 2018

INTRODUCTION:

Relapsed/metastatic head and neck squamous cell cancer patients are offered a combination of platinum-based chemotherapy (PF, cisplatin-fluorouracil) plus cetuximab regimen (PF+C) according to results of the EXTREME trial (1). However, two economic evaluations showed that addition of cetuximab was not cost-effective.

This study aimed to evaluate the cost-effectiveness of a putative predictive molecular test (MT) to identify and treat only patients potentially responsive to cetuximab when added to PF.

METHODS:

A Markov model was developed to compare both health and economic outcomes of PF+C regimen administered to all patients (PF+C ALL) versus the regimen administered only to MT-positive patients (PF+C POS).

The model considered the following health states: partial/complete response with/out mild/severe adverse events (AEs), progression and death. Rates of progression and survival, response rates to systemic treatment and adverse events were retrieved from the EXTREME trial (1). According to Mesía et al. (2), we assumed that addition of cetuximab to PF would not negatively affect life quality compared to PF alone, and the baseline utility coefficients for disease control and progression were assumed as .67 and .52, respectively.

Only direct costs estimated from the Italian Health Service perspective were included (tariffs and Diagnosis Related Group - DRG - reimbursements).

The model was evaluated according to a cut-off of sensitivity at 85 percent and specificity at 70 percent. A 3 years horizon was chosen. Life expectancy, quality-adjusted life years (QALYs) and costs were discounted at 3.5 percent annually.

RESULTS:

Applying the World Health Organization (WHO) cost-effectiveness threshold of 3 times the gross domestic product for Italy (EUR66,402), PF+C POS resulted a cost-effective choice in comparison to PF+C ALL for a MT cost lower than EUR5,750.

CONCLUSIONS:

Adding cetuximab to PF only to patients positive to a predictive test may be cost-effective. Efforts should be spent to build such a test upon existing evidences in order to save resources for the health systems and spare unnecessary toxicities to patients.
OP132 How A Shared Management Of Home Infusion Can Control Expenditure
Authors: Olivier Cyril , Collingnon Corinne , Fouteau Emmanuelle , Emery Gregory , Belghiti Jacques
Published: January 2018

INTRODUCTION:

In France, medical devices (MDs) for home-based infusion used to be covered by the health insurance system if included on the list of products and services qualifying for reimbursement under a generic description corresponding to a class of products with the same indications. This coverage modality offered no resistance to unnecessary or wasteful spending. Besides, between 2010 and 2015 the expenditure related to these MDs have increased from EUR192million to EUR289million (+50 percent).

METHODS:

The French National Authority for Health (HAS) has assessed the actual benefit of these MDs which have the same indications as the drugs infused at home. This work led to standardize the infusion types (gravity, elastomeric pump or active system requiring an energy source) and the quantities of MDs needed to carry out the different cares (installation, connection, withdrawal) according to the infusion route. At this step, considering that the priority was to redefine the MDs required at home for each care type, no economical assessment had been conducted.

RESULTS:

Based on this medical assessment, the Ministry of Health has distinguished three types of infusion and three types of services (home installation, monitoring and consumables) since 2016. In total, twenty-four packages have been set up for reimbursement with non-cumulative rules. Doctors are in charge to prescribe the appropriate packages; providers and nurses determine together the optimal devices needed for each patient according to his environment.

CONCLUSIONS:

These HAS recommendations on practice standardization have been the keystone for cost negotiations. The new coverage modalities aim to motivate liberal nurses to choose the best fitted products and providers to deliver the right quantities to patients. The expected benefits are an adjusted evaluation of the necessary equipment and a control of health expenditure due to the fixed costs of each infusion package.
OP135 Confirmatory Versus Explorative Endpoints In Drug Approval Versus Health Technology Assessment
Authors: Niehaus Ines , Dintsios Charalabos-Markos
Published: January 2018

INTRODUCTION:

The early benefit assessment of drugs in Germany and their preceded market authorization pursue different objectives, resulting in divergent decision-making strategies. This is reflected inter alia by the diverse inclusion of confirmatory endpoints within the assessments of oncological drugs. The pharmaceutical manufacturers are facing the challenge of meeting the requirements for both evaluation processes by the available evidence and avoiding hereby negative early benefit assessments. This is mainly due to the concept of mutually relevant clinical trials.

METHODS:

Identification and gathering of the endpoints is based on a specifically developed guide. The extracted data from the documents of completed assessments up to July 2015 are used to estimate both separately and together the impact of explorative in relation to confirmatory endpoints on the drug approval and early benefit assessment, by contrasting the European Medicines Agency's risk-benefit-ratio and the benefit-harm-balancing of the national Health Technology Assessment (HTA) jurisdiction.

RESULTS:

Twenty-one of fourty-one studies’ oncological assessments could be included in the endpoint analysis. From a procedural point of view both the drug approval and the early benefit assessment seem to be not confirmatory since they include explorative endpoints as well. Yet, drug approval is in terms of quality of endpoints more confirmatory than early benefit assessment since it contains a higher proportion of primary endpoints. The latter implies only in 67 percent of the assessments a primary endpoint to be relevant for the benefit-harm-balancing. Moreover, explorative mortality endpoints reached the highest agreement and explorative endpoints capturing health-related quality of life no agreement, referring to the mutual relevance of endpoints for the risk-benefit-ratio and the benefit-harm-balancing.

CONCLUSIONS:

The missing information transparency of the assessment reports compared to the information offered within the early benefit assessment makes an assignment of endpoints with respect to the mutually relevant clinical trial sometimes troublesome. To warrant, in the long run, a broader confirmatory basis for decisions in health care supported by HTA, a closer inter-institutional cooperation of approval authorities and German HTA jurisdictions seems favorable.
PP015 Methodological Quality Of Health Technology Assessment Reports
Authors: Hausner Elke , Knelangen Marco , Sanders Laura , Waffenschmidt Siw
Published: January 2018

INTRODUCTION:

Health Technology Assessment (HTA) reports may have a major impact on the health care provided in a country. Hence, one would assume that these reports have a high methodological quality and thus represent a potentially important source of information, for instance, for identifying primary studies for inclusion in the evidence syntheses (for example, systematic reviews, Cochrane reviews, HTA reports). The aim of the present analysis is to evaluate the methodological quality of HTA reports used as a literature source for HTA reports produced by the German Institute for Quality and Efficiency in Health Care (IQWiG).

METHODS:

Eligible IQWiG reports were assessments of drug or non-drug interventions considering HTA reports as the literature source for primary studies and published up to October 2016. An HTA report included in the IQWIG report was considered in the analysis if it was a complete report published in English or German and indexed in the Health Technology Assessment Database (Wiley) or MEDLINE. Only the most current HTA report in an IQWiG report was considered; if more than one current HTA report was available, the one for inclusion in the analysis was randomly selected. The methodological quality of the HTA reports identified was evaluated with the AMSTAR (“Assessment of Multiple Systematic Reviews”) tool (1), which comprises 11 items on methodological quality (meaning a maximum achievable score of 11).

RESULTS:

A total of fifty eligible IQWiG reports using fourty-one eligible HTA reports as literature sources were identified. The mean AMSTAR score of these HTA reports was 5.3 (95 percent Confidence Interval, CI: 4.3, 6.2). None of the HTA reports achieved a score of 11, nineteen (46 percent) had a score between 6 and 10, and twenty-two had a score below 6.

CONCLUSIONS:

HTA reports included in IQWiG reports only have an average methodological quality.
PP017 Social Cost Benefit Analysis Of Cognitive Behavioral Therapy For Alcohol And Cannabis Addiction
Authors: van Gils Paul , Over Eelco , Suijkerbuijk Anita , Lokkerbol Joran , de Wit Ardine
Published: January 2018

INTRODUCTION:

Due to their chronic nature and high prevalence, alcohol and cannabis addiction leads to a significant (disease) burden and high costs, both for those involved and for society. The latter includes effects on health care, quality of life, employment, criminality, education, social security, violence in the public and private domain, and traffic accidents. In the Netherlands, a considerable number of people with an alcohol or cannabis addiction currently do not receive addiction care. Cognitive Behavioral Therapy (CBT) is effective as a treatment for both alcohol and cannabis addiction and is widely used in specialized addiction care centers. This social cost-benefit analysis (SCBA) models costs and benefits of increasing the uptake of CBT for persons with an alcohol addiction and for adolescents with a cannabis addiction, taking into account a wide range of social costs and effects (1).

METHODS:

The method follows general Dutch guidance for performing SCBA. A literature search was conducted to evaluate efficacy of CBT for alcohol and cannabis dependence. In addition, the social costs of alcohol and cannabis addiction for society were mapped, and the costs of enhancing the uptake of CBT were explored. Costs and benefits of increased uptake of CBT for different social domains were modeled for a ten year period, and compared with current (unchanged) uptake during this period. Compliance problems (about 50 percent of clients do not finish CBT) and fall-back to addiction behavior (decrease of effects of CBT over time) were taken into account in model estimations.

RESULTS:

Per client treated with CBT, the estimated benefits to society are EUR10,000-14,000 and EUR9,700-13,000, for alcohol and cannabis addiction, respectively. These benefits result from reduced morbidity and mortality, improved quality of life, higher productivity, fewer traffic accidents, and fewer criminal activities.

CONCLUSIONS:

This SCBA shows that not only treated clients but also society will benefit from an increase in people treated with CBT in specialized addiction care centers.
PP021 Peer Review Innovations For Grant Applications: Efficient And Effective?
Authors: Frampton Geoff , Shepherd Jonathan , Pickett Karen , Wyatt Jeremy
Published: January 2018

INTRODUCTION:

Peer review of grant applications is employed routinely by health research funding bodies to determine which research proposals should be funded. Peer review faces a number of criticisms, however, especially that it is time consuming, financially expensive, and may not select the best proposals. Various modifications to peer review have been examined in research studies but these have not been systematically reviewed to guide Health Technology Assessment (HTA) funding agencies.

METHODS:

We developed a systematic map based on a logic model to summarize the characteristics of empirical studies that have investigated peer review of health research grant applications. Consultation with stakeholders from a major health research funder (the United Kingdom National Institute for Health Research, NIHR) helped to identify topic areas within the map of particular interest. Innovations that could improve the efficiency and/or effectiveness of peer review were agreed as being a priority for more detailed analysis. Studies of these innovations were identified using pre-specified eligibility criteria and were subjected to a full systematic review.

RESULTS:

The systematic map includes eighty-one studies, most published since 2005, indicating an increasing area of investigation. Studies were mostly observational and retrospective in design, and a large proportion have been conducted in the United States, with many conducted by the National Institutes of Health. An example of an innovation is video training to improve reviewer reliability. Although research councils in the United Kingdom have conducted several relevant studies, these have mainly examined existing practices rather than testing peer review innovations. Full results of the systematic review will be provided in the presentation, and we will assess which innovations could improve the efficiency and/or effectiveness of peer review for selecting health research proposals.

CONCLUSIONS:

Despite considerable interest in, and criticism of, peer review for helping to select health research proposals, there have been few detailed systematic examinations of the primary research evidence in this area. Our evidence synthesis provides the most up-to-date overview of evidence in this important developing area, with recommendations for health research funders in their decision making.
PP022 New Models Are Needed To Optimize The Management Of New Medicines
Authors: Godman Brian , Diogene Eduardo , Fürst Jurij , Garuoliene Kristina , Guerra Augusto , Joppi Roberta , Malmström Rickard , Oortwijn Wija , Selke Gisbert , Timoney Angela , Wettermark Björn
Published: January 2018

INTRODUCTION:

Countries are struggling to fund new premium priced medicines with ever increasing prices. In addition, there are substantial savings as medicines lose their patents. This requires coordinated approaches. Models are being developed centering on three pillars: pre-launch including horizon scanning; peri-launch including pricing and reimbursement (P & R)/ risk sharing; and post-launch including assessing effectiveness (1,2). This will continue to enable access to safe, effective and affordable medicines.

METHODS:

Desk research of regulatory and other relevant policy documents as well as a thorough and extensive literature search in peer-reviewed databases were conducted.

RESULTS:

Models to optimize the use of new medicines are being developed. These center on three pillars: pre-launch activities including horizon scanning with a specific focus on unmet needs, drugs expected place in therapy, drugs preliminary budget impact and forecasting (including medicines likely to lose their patents); peri-launch activities including P & R assessment and assessments of risk sharing arrangements; and post-launch activities include assessing the effectiveness and safety of new medicines in routine clinical care (1,2). Pre-launch activities to agree the number of potential patients for new cancer medicines resulted in hospitals staying within budget (3); and health authorities that had instigated activities pre-launch saw limited excess bleeding with dabigatran (3). Risk-sharing arrangements have increased access to new medicines; however, concerns with their confidential nature and administrative burden (2,3). Qualitative and/or quantitative approaches are also being developed to better value (new) medicines. There is also growing use of patient level data post launch, for example, studies highlighted concerns with dabigatran prescribing in Spain and anti-obesity medicines in Sweden. Long-term follow-up studies have shown greater effectiveness of ciclosporin versus tacrolimus for transplants despite the rhetoric.

CONCLUSIONS:

Stakeholders in the healthcare field are working together and developing methods to increase funding for new valued medicines whilst restricting their use where there are concerns to optimize resource use. This will (need to) continue to enable access to safe, (cost-) effective and affordable medicines.
PP024 Changes In Reporting Characteristics Of Systematic Reviews For The United Kingdom
Authors: Kaltenthaler Eva , Carroll Christopher
Published: January 2018

INTRODUCTION:

A recent publication reported that increasing numbers of systematic reviews are being published and, although standards have improved, many are still poorly conducted and reported, especially non-Cochrane systematic reviews (1). The aim of this study was to assess the quality of the conduct and reporting of systematic reviews undertaken for the United Kingdom (UK) Health Technology Assessment (HTA) programme and published in the International Journal of Technology Assessment in Health Care (2) and compare those undertaken in 2004 and 2014.

METHODS:

A comparative sample of all systematic reviews published in 2004 and 2014 in the UK HTA monograph series was identified by a structured search of MEDLINE in August 2016. After piloting of the form, two reviewers each extracted relevant data. These data were tabulated and summarized.

RESULTS:

The search identified twenty-three systematic reviews from 2004 and thirty from 2014. By 2014, compared with 2004, a smaller proportion of treatment (53 percent versus 70 percent) and pharmaceutical (20 percent versus 57 percent) reviews were being published. In 2014, there were much higher percentages of review registrations (70 percent versus 0 percent) and available protocols (90 percent versus 17 percent); increased explicit inclusion of unpublished literature (65 percent versus 39 percent); less frequent use of local checklists (32 percent versus 61 percent) for critical appraisal; more complete reporting of study flow for inclusion (97 percent versus 57 percent) and exclusion (91 percent and 65 percent) of studies; and there were more reviews reporting limitations affecting the review itself (73 percent versus 49 percent). The process had clearly become more reflective and rigorous. However, some previous weaknesses persisted, including the general absence of any assessment of publication bias and the failure to report overall numbers of patients in the review.

CONCLUSIONS:

Marked improvements can be seen in the conduct and reporting of systematic reviews published by the UK HTA programme as a result of the publication and general acceptance of the PRISMA statement (3) and the increased application of a smaller number of relevant standards.
PP028 Hyperhidrosis Quality Of Life Measures: Review And Patient Perspective
Authors: Jones-Diette Julie , Wade Ros , Wright Kath , Llewellyn Alexis , Rice Stephen , Moloney Eoin , Stoniute Julija , Layton Alison , Levell Nick , Stansby Gerard , Craig Dawn , Woolacott Nerys
Published: January 2018

INTRODUCTION:

Primary hyperhidrosis has no discernible cause and is characterised by uncontrollable excessive and unpredictable sweating, which occurs at rest, regardless of temperature. The symptoms of hyperhidrosis can significantly affect quality of life, and can lead to social embarrassment, loneliness, anxiety and depression.

The aim of this literature review was to identify the tools used to measure quality of life in studies of hyperhidrosis. Patient advisors provided insight and their perspective.

METHODS:

Studies were identified through searches undertaken in January 2016. The search strategies combined topic terms for hyperhidrosis with a recognised search filter for “quality of life”. All studies that reported measuring quality of life or described a quality of life measure/tool in the context of primary hyperhidrosis were included. The information on the tools and their use in hyperhidrosis was summarized in a narrative synthesis. Patient advisors contributed to the interpretation of the findings.

RESULTS:

The review included 184 studies and many studies used multiple tools. Twenty-two individual tools were identified. The review identified disease specific, dermatology specific, and general health/utility tools. The most commonly identified tools were the Dermatology Life Quality Index (DLQI), the Hyperhidrosis Disease Severity Scale (HDSS), and the Hyperhidrosis Quality of Life Questionnaire (HQLQ). The Hyperhidrosis Quality of Life index (HidroQoL©) is recently designed and validated, and therefore was used only in its validation study.

When asked about these four quality of life tools patient advisors agreed that the HidroQoL© tool covered disease-specific quality of life dimensions relevant to them most comprehensively and was easy to complete. The DLQI was considered to be too general and too focussed on the skin. The HDSS was considered to be too basic and not sufficiently discriminating.

CONCLUSIONS:

Future studies of the effectiveness of interventions for hyperhidrosis on health-related quality of life may benefit from including the HidroQoL© tool.
PP044 Adherence To Enzyme Replacement Therapy In Gaucher Disease
Authors: Quevedo Amanda , Dornelles Alicia , Paskulin Livia , Alegra Taciane , Krug Barbara , Vairo Filippo , Picon Rafael , Shwartz Ida , Picon Paulo
Published: January 2018

INTRODUCTION:

Gaucher disease (GD) is a genetic autosomic disorder for which treatment has been funded by the Brazilian government since the 1990s. In our state most patients are treated with enzyme replacement therapy (ERT) and followed by our Reference Center under the recommendation of the Ministry of Health Brazilian guidelines. There is a lack in the literature about adherence of patients to treatment. The objective was to describe adherence to the treatment in a cohort of all GD patients in the southern state of Brazil.

METHODS:

This was a cohort study of all GD patients treated with velaglucerase α, taliglucerase α and imiglucerase from January 2010 to January 2015. Adherence was measured as recommended by the Brazilian guidelines as to perform more than 50 percent of the anticipated infusions per year.

RESULTS:

Our study included thirty-seven patients of both genders. Doses of ERT varied from 15 to 45IU/kg for type 1 patients and from 30 to 60 IU/kg for type 3 patients. A mean of 83 percent of anticipated infusions were performed and from all patients only one did not adhere to the treatment during the 5 years of our study. The majority of the patients performed at least 50 percent of all anticipated infusions.

CONCLUSIONS:

We noted a very high rate of adherence to treatment with a very few adverse effects. Our data might be showing that the very high rate of adherence in these chronic disease patients may be attributed to the value of treatment by patients and their family, and also due to the existence of a multidisciplinary team at the reference center. These data might be useful for public health policy making in other countries.
PP050 Analysis Of Pharmacoeconomic Studies Published In The Scientific Electronic Library “eLIBRARY.RU” (RSCI)
Authors: Yagudina Roza , Kulikov Andrey , Zubarev Pavel , Protsenko Marina , Ugrekhelidze Dzhumber
Published: January 2018

INTRODUCTION:

During the information search revealed that there is no published analysis reflecting the actual status of pharmacoeconomic and clinical-economic research, but there are databases that allow you to do so. The platform for searching research data was scientific electronic library “eLIBRARY.RU”. This portal is Russia's largest internet library of scientific publications, with rich search capabilities and the timely receipt of necessary information.

METHODS:

A search was carried out during the period from 2005 to 2015 of research papers on Russian and foreign pharmacoeconomic and clinical-economic research was carried out as at 1 September 2016 on the words “pharmacoeconomic*”, “clinical and econom*” with the appropriate endings. The criteria for inclusion in this analysis were general accessibility and availability of full text scientific material on the portal.

RESULTS:

Over the last ten years the number of publications increased by 4 times. The leading destinations included cardiovascular, pulmonary, oncological and endocrinological diseases. According to published reports pharmacoeconomic and clinical-economic studies carried out in fifty-two subjects of the Russian Federation on the basis of sixty-three Universities. In addition to researchers from the Russian Federation, has placed the representatives from eleven countries. Only one third of the published studies are generally cited. Among the methods of pharmacoeconomic analysis of the most popular in published studies is the analysis of “cost–effectiveness”, which is used in about 45 percent of the available studies.

CONCLUSIONS:

As a result, 1,425 articles were identified and analyzed. With the aim of providing concrete data that clearly illustrate the situation with the Russian scientific, pharmacoeconomic and clinical-economic writings to date all studies were classified according to the following parameters: geographical, temporary, local and nosological. In the framework of the analysis highlighted the authors whose works are most RISC (Russian Science Citation Index) with leading positions in the number of publications.
PP071 Health Technology Assessment In Bulgaria: A Review Of The First Fifteen Reports Assessed
Authors: Raycheva Ralitsa , Iskrov Georgi , Stefanov Rumen
Published: January 2018

INTRODUCTION:

In Bulgaria, the regulatory body sets for the first time legal requirements for Health Technology Assessment (HTA) in Law on Medicinal Products in Human Medicine (LMPHM) on 27 June 2015. The next essential step for HTA capacity building was the promulgation of Ordinance 9 / December 1, 2015 on the conditions and procedures for conducting health technology assessment by the Ministry of Health (1). In the beginning of 2016, the Main Price and Reimbursement Committee was set and launched a process for establishing the small working groups with the task of reviewing the first applicants reports of pharmaceuticals for inclusion in the Positive Drug List (PDL).

METHODS:

The objective of this study is to summarize the recommendations of the newly established HTA Committee in Bulgaria and to examine the characteristics of the technologies and the key considerations that led to those decisions. We systematically read all published by the Committee recommendations for 2016 and analyzed them under: type of recommendations (positive or negative for inclusion in PDL), population, specialization, type of service, type of justification and the impact on final conclusions.

RESULTS:

For the first year of its work the HTA Committee was able to assess fifteen technologies (pharmaceuticals) and only one received a negative recommendation (6 percent) from the working group. All the rest (n = 14; 94 percent) were recommended for funding. The final recommendation from the Main Price and Reimbursement Committee is available for four (27 percent) technologies – all positive for inclusion in PDL. All recommendations were connected with adults and in oncology (n = 4; 27 percent); heart diseases (n = 4; 27 percent); Chronic Obstructive Pulmonary Disease, COPD (n = 2; 13 percent); diabetes (n = 2; 13 percent); psoriasis (n = 2; 13 percent); Hepatitis C (n = 1; 7 percent). The only negative recommendation was justified due to lack of robust evidence, safety issues and credibility of HTA analysis (2).

CONCLUSIONS:

The information about the number of applications received from the Committee is not available and correct conclusions about the capability is not possible, but indirect circumstances, as the lack of well-trained HTA experts, certainly impede establishment of the small working groups and slow the assessment process (3). At this point it is clear that additional efforts are need to overcome the barriers and smooth adoption and implementation of HTA methods in Bulgaria.
PP100 Economic Evaluation Of A New Non-Antibiotic First-line Treatment Of Recurrent Urinary Tract Infections
Authors: Guglielmo Annamaria , Damiano Rocco , Ciani Oriana , Tarricone Rosanna , Cicione Antonio , Giancotti Monica , Mauro Marianna
Published: January 2018

INTRODUCTION:

Urinary tract infections (UTIs) are common in female patients in general practice. These bacterial infections affect half of all women at least once in their life. Antibiotics are usually prescribed for UTIs, and continuous low antimicrobial prophylaxis is administered to patients at high risk of recurrent UTI (rUTIs) (1). However, a major concern arises due to the increased rates of severe treatment-related side effects and emergence of antimicrobial resistance, which makes rUTIs management more challenging while seeking the use of more expensive alternatives. On this basis, clinical evaluations of rUTI interventions should be accompanied by economic evaluations in order to guide healthcare policy and decision processes about healthcare resources allocation. The aim of this work was to perform a cost-effectiveness analysis of a novel effective non-antibiotic treatment option for prophylaxis of female patients with a history of rUTIs, based on intravesical administration of hyaluronic acid (HA) plus chondroitin sulfate (CS), as compared to recommended 1st-line antibiotic therapy (2).

METHODS:

A cost-utility analysis was performed in order to estimate the effectiveness of each treatment, according to the number of UTIs annual episodes, and the incremental cost-effectiveness (ICER) for patients with UTI, starting from data collected during a multicentric observational case-cross-over clinical trial involving seven European centers (2). The economic model includes the costs of HA treatment and the costs associated with each UTI, such as costs of UTI diagnostics and antibiotic treatment, additional care by the elderly-care physician, additional nursing care, and hospitalizations, as well as the expected QALY, measured through the Short Form Health Survey (SF-36) questionnaires administered to patients, for both groups (3).

RESULTS:

At this stage, preliminary findings suggest that HA plus CS is a cost-effective alternative to antibiotics for the treatment of recurrent UTIs, that could reduce UTIs events in female patients with a history of recurrent UTI at an acceptable cost.

CONCLUSIONS:

The results of this study support the use of HA plus CS against antimicrobials as 1st-line therapy in the management of rUTIs.
PP142 A Mental Health Hospital-based Health Technology Assessment In Quebec, Canada: Structure And Products
Authors: Gheorghiu Ionela , Lesage Alain , Mongodin Adam , Galdin Marlène
Published: January 2018

INTRODUCTION:

Our Hospital-based Health Technology Assessment unit (HB-HTA) was founded in 2011 following the nomination of Louis-H. Lafontaine hospital as the Montreal University Mental Health Institute (IUSMM). From the beginning, the HB-HTA has been supporting and advising the Chief Executive Officer of IUSMM in the decision-making process concerning the implementation of new technologies and practices in mental health. Since 2015, the HB-HTA is part of the East of Montreal Regional Integrated Health and Social Services Centre (CIUSSS de l'Est-de-l’Île de Montréal), continuing to support decisions in mental health. Currently, the HB-HTA unit is nested in the Quality, Performance and Ethics department.

METHODS:

Formed by a coordinator, a scientific advisor and a manager, the HB-HTA team plans, organizes and sets up the evaluation activities. The unit benefits from the support of a Steering Committee which consists of representatives of clinical, administrative and research directions, as well as of health users and families. This committee determine the strategic orientation of the HB-HTA unit, prioritize the projects, approves the evaluation products and gives indications on the knowledge transfer process.

RESULTS:

To answer the decision questions, our HB-HTA unit employs two types of products: evaluation reports and informative notes. Based on an exhaustive literature search and consultations with stakeholders, the evaluation reports offer recommendations to support the decision-making process. The informative notes are rapid responses based on a partial literature search. The nature of this type of analysis does not allow the formulation of recommendations, however, a conclusion of the consulted literature is offered.

CONCLUSIONS:

Based on the work of our HB-HTA unit, some important decisions were made by the IUSMM. As an example, the systematic screening of psychiatric patients for drug and alcohol was not favored by our institution; rather than this, priority was given to staff training, in order to better identify and treat psychiatric patients with substance abuse comorbidity.
VP08 Real-World Data Use In Health Technology Assessments: A Comparison Of Five Health Technology Assessment Agencies
Authors: Makady Amr , van Veelen Ard , de Boer Anthonius , Hillege Hans , Klunger Olaf , Goettsch Wim
Published: January 2018

INTRODUCTION:

Reimbursement decisions are usually based on evidence from randomized controlled trials (RCT) with high internal validity but lower external validity. Real-World Data (RWD) may provide complimentary evidence for relative effectiveness assessments (REA's) and cost-effectiveness assessments (CEA's) of treatments. This study explores to which extent RWD is incorporated in REA's and CEA's of drugs used to treat metastatic melanoma (MM) by five Health Technology Assessment (HTA) agencies.

METHODS:

Dossiers for MM drugs published between 1 January 2011 and 31 December 2016 were retrieved for HTA agencies in five countries: the United Kingdom (NICE), Scotland (SMC), France (HAS), Germany (IQWiG) and the Netherlands (ZIN). A standardized data-extraction form was used to extract data on RWD mentioned in the assessment and its impact on appraisal (for example, positive, negative, neutral or unknown) for both REA and CEA.

RESULTS:

In total, fourty-nine dossiers were retrieved: NICE = 10, SMC = 13, IQWiG = 16, HAS = 8 and ZIN = 2. Nine dossiers (18.4 percent) included RWD in REA's for several parameters: to describe effectiveness (n = 5) and/or the safety (n = 2) of the drug, and/or the prevalence of MM (n = 4). CEA's were included in 25/49 dossiers (IQWiG and HAS did not perform CEA's). Of the twenty-five CEA's, twenty (80 percent) included RWD to extrapolate long-term effectiveness (n = 19), and/or identify costs associated with treatments (n = 7). When RWD was included in REA's (n = 9), its impact on the appraisal was negative (n = 4), neutral (n = 2), unknown (n = 1) or was not discussed in the appraisal (n = 2). When RWD was included in CEA's (n = 11), its impact on the appraisal varied between positive (n = 2), negative (n = 5) and unknown (n = 4).

CONCLUSIONS:

Generally, RWD is more often included in CEA's than REA's (80 percent versus 18.4 percent, respectively). When included, RWD was mostly used to describe the effectiveness of the drug (REA) or to predict long-term effectiveness (CEA). The impact of RWD on the appraisal varied greatly within both REA's and CEA's.
VP15 A Comparison Of Reporting In United Kingdom Health Technology Assessment And Other Systematic Reviews
Authors: Carroll Christopher , Kaltenthaler Eva
Published: January 2018

INTRODUCTION:

A recent study claimed that increasing numbers of reviews are being published and many are poorly conducted and reported (1). The aim of the present study was to assess how well reporting standards in systematic reviews published in 2014 in the United Kingdom Health Technology Assessment (UK HTA) monograph series compared with the reporting in Cochrane and other “non-Cochrane” systematic reviews from the same year, as reported by Page et al. (1).

METHODS:

All relevant UK HTA programme systematic reviews published in 2014 were identified. After piloting of the form, two reviewers each extracted relevant data on conduct and reporting from these reviews. These data were compared with data for Cochrane and “non-Cochrane" systematic reviews from 2014, as published by Page et al. (1). All data were tabulated and summarized.

RESULTS:

There were 30 UK HTA programme systematic reviews and 300 other systematic reviews, including Cochrane reviews (n = 45). Fewer UK HTA reviews covered therapeutic and pharmaceutical topics (53 percent and 20 percent respectively) than Cochrane (100 percent and 51 percent). The percentage of HTA reviews with required elements of conduct and reporting was frequently very similar to Cochrane and much higher than all other systematic reviews: for example, availability of protocols (90 percent, 98 percent and 16 percent respectively); the specification of study design criteria (100 percent, 100 percent, 79 percent); the reporting of outcomes (100 percent, 100 percent, 78 percent), quality assessment (100 percent, 100 percent, 70 percent) and other processes; the searching of trial registries for unpublished data (70 percent, 62 percent, 19 percent); reporting of reasons for excluding studies (91 percent, 91 percent and 70 percent) and reporting of authors' conflicts of interest (100 percent, 100 percent, 87 percent). However, HTA reviews compare less favourably with Cochrane and other reviews in the assessment of publication bias and reporting overall numbers of patients in the review.

CONCLUSIONS:

UK HTA systematic reviews are often produced within a specific policy-making context and cover a greater variety of topics than Cochrane reviews. This has implications for timelines, tools and resources. However, they still tend to present standards of conduct and reporting equivalent to “gold standard” Cochrane reviews and are superior to systematic reviews more generally.
VP26 Comparing Statistical Methods For Meta-Analysis Of Rare Event Data
Authors: Sharma Tarang , Gøtzsche Peter , Kuss Oliver
Published: January 2018

INTRODUCTION:

We aimed to identify the validity and robustness of effect estimates for serious rare adverse events in clinical study reports of antidepressant trials, across different meta-analysis methods for rare binary events data (1,2).

METHODS:

Four serious rare adverse events (all-cause mortality, suicidality, aggressive behaviour and akathisia) were meta-analyzed using different methods (3). The Yusuf-Peto odds ratio (OR), which ignores studies with no events in the treatment arms, was compared with the alternative approaches of generalized linear mixed models (GLMM), conditional logistic regression, a Bayesian approach using Markov Chain Monte Carlo (MCMC) and a beta-binomial regression model.

RESULTS:

Though the estimates for the four outcomes did not change substantially across the different analysis methods, the Yusuf-Peto method underestimated the treatment harm and overstimated its precision, especially when the estimated odds ratio (OR) deviated greatly from 1. For example the OR for suicidality for children and adolescents was 2.39 (95 percent Confidence Interval, CI 1.32 to 4.33, using the Yusuf-Peto method), but increased to 2.64 (95 percent CI 1.33 to 5.26) using conditional logistic regression, to 2.69 (95 percent CI 1.19 to 6.09) using beta-binomial, to 2.73 (95 percent CI 1.37 to 5.42) using the GLMM and finally to 2.87 (95 percent CI 1.42 to 5.98) using the MCMC approach.

CONCLUSIONS:

The method used for meta-analysis of rare events data influences the estimates obtained and the exclusion of double zero-event studies can give misleading results. To ensure reduction of bias and erroneous inferences, sensitivity analyses should be performed using different methods and we recommend that the Yusuf-Peto approach should no longer be used. Other methods, in particular the beta-binomial method that was shown to be superior, should be considered instead.