bluuebunny/crossref_metadata_2025 huggingface dataset:
PP15 The National Pricing And Reimbursement Process In China, A 2021 Update
Authors: Song Xirong , Kodabuckus Shahela , Patel Preeti
Published: December 2022

Introduction

The Chinese National Reimbursement Drug List (NRDL) was established in the early 2000’s and includes the drugs both fully and partially covered by National Basic Medicine Insurance. As China’s health system has been reformed over the past decade, it is important for manufacturers to understand the ever-changing reimbursement process and its implications on newly launched drugs. This study provides an updated overview of the process based on research conducted in 2021.

Methods

Targeted secondary research was undertaken to evaluate the pricing and reimbursement landscape in China. Primary research was conducted to assess the perspectives of three payers and one policy expert.

Results

National listing remains the most viable and exclusive pathway to get a product reimbursed by public health insurance in China. Since 2017, the NRDL has been updated annually, and revisions are managed by the National Healthcare Security Administration (NHSA). Insights from 2021 suggests that the process of listing a new product on the NRDL lasts five months (July to November). Manufacturers should ensure that submissions are made when the annual NRDL process formally begins, and clinical and health economic evidence is compulsory. If a successful opinion is made by the assessment board, the manufacturer will be invited to negotiate a price with the NHSA. Data from the NHSA indicated that a total of 704 applications were made in 2020. In addition, 138 exclusive drugs were eligible for price negotiation, of which 96 drugs were successful and added to the NRDL. Findings also suggested that the average discount rate increased from 44.0 percent in 2017 to 50.6 percent in 2020.

Conclusions

The national reimbursement process in China has become more transparent overtime. Even so, NRDL listing remains a challenge, with decisions driven by clinical and pharmacoeconomic evidence, and price. Significant price cuts should be considered and anticipated to ensure successful negotiation outcomes.
PP18 Horizon Scanning For Clinical Biosimilar Medicines: Informing The Lifecycle Of Health Technology Assessment And Market Access
Authors: Hussain Amy , Fairbairn Ross , Ogunbayo Dapo
Published: December 2022

Introduction

The National Institute for Health and Care Research (NIHR) Innovation Observatory (IO) national horizon scanning research centre, has a remit to notify its stakeholders, including the National Institute for Health and Care Excellence (NICE), about innovative interventions; including biosimilar medicines in the pipeline. Biosimilar medicines bypass many developmental steps, making them substantially cheaper to manufacture for providers, which increases market availability and improves treatment access for patients.

Methods

Since 2017, the NIHR IO has monitored biosimilars in clinical development that align to the NICE health technology assessment remit. The data set explored was exported from our internal medicines innovation database - MInD.

Data sets were created that included information on the characteristics of biosimilars and their associated clinical trials. Analyses and visualization creation were carried out using Microsoft Excel and Microsoft Power BI.

Results

A total of 100 unique biosimilar medicines in 136 clinical trials were included in the MInD since April 2017. Of these, 44 percent of biosimilars are currently EMA-approved (Nov 2021). Adalimumab was the reference medicine with the most unique biosimilars identified (12%). Seventy-two percent of the biosimilars in MInD were indicated for non-oncology conditions, twenty percent for oncology condition and eight percent for both.

There were 46 biosimilars unapproved, which were in active development. Of these biosimilars 17.4 percent are indicated for an oncology condition, 78.3 percent for non-oncology conditions, and 4.3 percent for biosimilars for both. Aflibercept was the reference product with the most (eight) biosimilars in active development.

There were 56 individual clinical trials in the MInD that list a biosimilar in development. For 26 trials, the primary completion date (PCD) was prior to 2021, whilst 28 trials listed a PCD post-2021, and 2 PCD’s were unavailable

Conclusions

Our analysis identified high levels of active clinical development for biosimilars. The majority of biosimilars being developed are indicated for non-oncology conditions, with many in trials due to readout in the near future. Early identification, monitoring and reporting of biosimilars allows for expedited patient access and benefits, including cost-savings for health services.

This study is funded by the National Institute for Health Research (NIHR) [(HSRIC-2016-10009)/Innovation Observatory].
PP19 Reimbursable Health Apps (DiGA) In Germany: Which Factors Impact The BfArM’s Assessment And Directory Listing? – Updated Research
Authors: Drews Janika , Bonduelle Doreen , Schuller Johanna
Published: December 2022

Introduction

Since May 2020, reimbursement can be requested for Digital healthcare applications (DIGAs) in Germany. The prerequisite for reimbursement is a listing in the Federal Institute for Drugs and Medical Devices (BfArM) DiGA-directory, granted after successful completion of an assessment process. In June 2021, IQVIA first evaluated the BfArM’s decision-making and identified criteria that may positively impact a directory listing. In November 2021, the research was updated to consider the latest developments.

Methods

Published information for each app in the DiGA-directory was qualitatively compared according to pre-specified criteria by two independent reviewers. With no data available for denied apps, only DiGAs with permanent or preliminary listings were compared.

Results

By 26 November 2021, twenty-four apps had received a positive assessment. Permanently listed apps (n=6) focused on health status improvements and/or patients’ health competency. None claimed to improve patients’ quality of life. Results from at least one randomized controlled trial (RCT) showed efficacy with medium or strong significant effect sizes (Cohen’s d > 0.4) versus standard of care (SOC). For preliminarily listed DiGAs (n=18), final results of positive care effects were not yet provided, but applications included study designs for RCTs including definitions of primary endpoints. The BfArM accepted the trial designs to be suitable to measure an app’s positive effects on health care within twelve months after listing. Since the last analysis, no preliminarily listed apps have been granted permanent DiGA-status. Evaluation periods were extended by up to five months for three apps. With none of the trials for preliminary apps being completed, possibilities for assessment remain limited. Claims for either medical benefit and/or patient-relevant structural/procedural improvements do not seem to impact the type of listing.

Conclusions

Updated research confirmed previous findings. For a DiGA-directory listing DiGA, the (planned) provision of RCT data seems to be gold standard. Medium to strong effects on the improvement of health status compared to SOC appear to be important for a permanent listing. Evaluation periods of five preliminarily listed apps are expected to be completed in December 2021, allowing for further analyses in the future.
PP25 Brazilian Collaborative Network For COVID-19 Modeling: Successful Experience Of Using Real-Time Science To Support Evidence-Based Decision-Making
Authors: Bagattini Ângela Maria , Rosa Michelle , Simon Lorena Mendes , Berg de Almeida Gabriel , Ferreira Leonardo Souto , Borges Marcelo Eduardo , Kraenkel Roberto André , Coutinho Renato Mendes , Diniz Filho José Alexandre Felizola , Camey Suzi Alves , de Souza Kuchenbecker Ricardo , Toscano Cristiana Maria
Published: December 2022

Introduction

Modeling is important for guiding policy during epidemics. The objective of this work was to describe the experience of structuring a multidisciplinary collaborative network in Brazil for modeling coronavirus disease 2019 (COVID-19) to support decision-making throughout the pandemic.

Methods

Responding to a national call in June 2020 for proposals on COVID-19 mitigation projects, we established a team of investigators from public universities located in various regions throughout Brazil. The team’s main objective was to model severe acute respiratory syndrome coronavirus 2 transmission dynamics in various demographic and epidemiologic settings in Brazil using different types of models and mitigation interventions. The modeling results aimed to provide information to support policy making. This descriptive study outlines the processes, products, challenges, and lessons learned from this innovative experience.

Results

The network included 18 researchers (epidemiologists, infectious diseases experts, statisticians, and modelers) from various backgrounds, including ecology, geography, physics, and mathematics. The criteria for joining the network were having a communication channel with public health decision-makers and being involved in generating evidence for public policy. During a 24-month period, the following sub-projects were established: (i) development of a susceptible-exposed-infected-recovered-like, individual-based meta-population and Markov chain model; (ii) projection of COVID-19 transmission and impact over time with respect to cases, hospitalizations, and deaths; (iii) assessment of the impact of non-pharmacological interventions for COVID-19; (iv) evaluation of the impact of reopening schools; and (v) determining optimal strategies for COVID-19 vaccination. In addition, we mapped existing COVID-19 modeling groups nationwide and conducted a systematic review of relevant published research literature from Brazil.

Conclusions

Infectious disease modeling for guiding public health policy requires interaction between epidemiologists, public health specialists, and modelers. Communicating modeling results in a non-academic format is an additional challenge, so close interaction with policy makers is essential to ensure that the information is useful. Establishing a network of modeling groups will be useful for future disease outbreaks.
PP33 Molecular Markers For The Detection Of Clinically Significant Prostate Cancer
Authors: del Pino-Sedeño Tasmania , García-Pérez Lidia , Infante-Ventura Diego , Hernández-Yumar Aránzazu , Rodríguez-Rodríguez Leticia , de Armas-Castellano Aythami , Serrano-Aguilar Pedro , del Mar Trujillo-Martín María
Published: December 2022

Introduction

It is estimated that approximately 1.1 million cases of prostate cancer (PCa) are diagnosed in the world every year. In general, PCa is a slow-onset cancer and less than 10 percent of cases are detected in the metastatic phase. In order to identify patients at risk of suffering from clinically significant prostate cancer (csPCa), as well as to avoid unnecessary biopsies, overdiagnosis and overtreatment, a variety of molecular biomarker detection tests have been developed.

Methods

We undertook a systematic review with meta-analyses on the effectiveness of diagnostic tests based on biomarkers in blood or urine samples for the identification of men at risk of csPCa. A cost-effectiveness analysis was conducted using a decision tree model for the short term and a Markov model for the long term, both from the social and the National Health System perspectives. The effectiveness measure was quality-adjusted life years (QALYs). We ran extensive sensitivity analyses, including a probabilistic sensitivity analysis.

Results

Sixty-five studies were included with a total of 34,287 participants. The diagnostic tests analyzed were: PHI, Progensa® PCA3, SelectMDx, MyProstateScore, 4Kscore®, TMPRSS2: ERG, Stockholm3, ExoDx Prostate IntelliScore and Proclarix®. All studies included biopsy as comparator. The sensitivity and specificity of diagnostic tests depended on the test itself and the threshold chosen, and ranged from 42 percent to 99 percent and from 13 percent to 87 percent, respectively. In the cost-effectiveness analysis, the alternative that includes the biomarker, specifically the SelectMDx, led to higher QALYs and healthcare costs with an estimated incremental cost-effectiveness ratio (ICER) of 6,640.21 EUR per QALY. The sensitivity analyses confirmed that the results were robust.

Conclusions

Biomarker testing to select men at risk of csPCa who should undergo prostate biopsy can be a cost-effective strategy depending on its cost per determination and its sensitivity/specificity. The analyses carried out indicate that the SelectMDx biomarker is cost-effective at a cost of EUR 375 per determination.
PP34 Next Generation Sequencing For Informing Lung Cancer Therapy In Europe – Hospital Impact With A Lifecycle Perspective
Authors: Saunders Rhodri , Hedblom Amanda Hansson , Quagliata Luca , Costa Jose Luis , Dumanois Robert
Published: December 2022

Introduction

Next-generation sequencing (NGS) can be run in-house or outsourced to an independent laboratory. It has enabled wider use of deoxyribonucleic acid/ribonucleic acid (DNA + RNA) sequencing in clinical practice. Within oncology, NGS has paved the way for more effective treatment, including personalized medicine. There are, however, large variations in access and reimbursement across Europe. The aim is to understand the European NGS landscape and barriers to access.

Methods

Structured telephone interviews covered topics on NGS perception, guidelines, use-cases, benefits, costs, and future expectations. Twelve experts per country (France, Germany, Italy, Spain): two payers, five oncologists, and five pathologists were interviewed between June and August 2021. Responses were translated into English for qualitative analysis.

Results

NGS was considered most useful when there were approved, targeted treatments. Although often noted that there was a lack of published evidence to support a beneficial link, respondents perceived that NGS has the potential to improve patient quality of life (QoL) and reduce resource use through avoiding suboptimal treatment. All of the payer respondents expected the role of NGS to increase, though it may be held back by lack of reimbursement. Respondents favored in-house NGS over outsourcing in terms of clinical benefit: “Advantages of in-house NGS are turnaround time, results and lean processes” … “you build the expertise in-house. If you have urgent samples, it’s easy to prioritize them”. Reasons for not having in-house NGS included “costs, lack of personnel. Basically, organizational and financial issues.” In-house NGS was perceived to be associated with high setup-costs (acquisition, setup, training), but lower running costs (per-test costs). There was a view that in-house solutions are mainly suitable in larger centers undertaking many tests.

Conclusions

NGS can save costs and provide QoL benefit through enabling optimized, personalized therapy, but published evidence establishing the outcomes link is lacking. From the hospital perspective, investing in NGS requires understanding the cost of NGS over its entire lifecycle, likely entailing a health-technology assessment including health-economic analysis.
PP39 Evidence Generation For Reimbursement Of Digital Health Applications (DiGAs) In Germany
Authors: Fujita-Rohwerder Naomi , Sauerland Stefan
Published: December 2022

Introduction

In 2019, the German government established a new evaluation procedure for digital health applications (DiGAs) to facilitate their reimbursement by statutory health insurance. The procedure involves the assessment of a DiGA’s “positive healthcare effect”, which is defined as a medical benefit and/or “a patient-relevant improvement of structure and processes”. If the available clinical evidence is insufficient to prove the manufacturer’s claim on the positive healthcare effect, but the claim seems plausible, the DiGA is provisionally reimbursed, and further clinical evidence within twelve months must be generated. DiGAs eligible for provisional or permanent reimbursement are publicly listed in the DiGA directory.

In contrast to the usual pathways for reimbursement of healthcare technologies which involve IQWiG as the national HTA agency and the G-BA (Federal Joint Committee) as the decision-making body, the DiGA procedure is currently carried out by the national competent authority (BfArM) and thus outside the joint self-government. Furthermore, legal evidence requirements for DiGAs are comparatively low.

Methods

This work analyzed the suitability of clinical studies that intended to prove a DiGA’s medical benefit. For this purpose, the key elements for clinical studies published in the DiGA directory and clinical trial registries were extracted and compared with the usual evidence requirements in the reimbursement context.

Results

As of October 2020, 20 DiGAs have successfully undergone the application procedure. Fourteen DiGAs (70%) were provisionally accepted. A randomized controlled study (RCT) design was chosen for all clinical studies to be conducted for further evidence generation. However, in four cases (28%), it is questionable whether the clinical study is suitable to demonstrate a medical benefit mainly due to the choice or operationalization of the primary endpoint (n=2), the timing of the endpoint survey (n=2) and/or the choice of the control intervention (n=1).

Conclusions

Even though all currently ongoing or planned clinical studies with DiGAs are RCTs, not all of them are adequate to demonstrate a medical benefit according to the usual evidence requirements.
PP41 COVID-19 Modeling To Support Decision Making In Brazil: A Scoping Review
Authors: Rosa Michelle , Bagattini Angela Maria , Simon Lorena Mendes , de Almeida Gabriel Berg , da Rosa Isabella Inês Rodrigues , Toscano Cristiana Maria
Published: December 2022

Introduction

In the context of the COVID-19 pandemic, which required urgent responses from health systems, and ongoing decision making in a context of limited and evolving evidence, modeling played a significant role in supporting public policy making. Nonetheless, particularly in low and middle-income countries, modeling groups are scarce, and usually not routinely involved in supporting public health policy making. We aimed to appraise COVID-19 modeling work in Brazil during the pandemic.

Methods

We performed a scoping review following PRISMA guidelines to identify groups conducting COVID-19 modeling to support health decision-making in Brazil. Search strategies were applied to MEDLINE, LILACS, Embase, ArXiv, and also included National data repositories and gray literature. We excluded reports of models without modeling results. Titles, abstracts, data repository descriptions and full-text articles identified were read and selected by two reviewers. Data extracted included modeling questions, model characteristics (structure, type, and programming), epidemiologic data sources, main outcomes reported, and parameters. To further identify modeling groups that might have not yet published results, snowball sampling was performed, and a short survey was sent electronically. Investigators and policymakers were invited to an online interview, to obtain further information on how they interacted, communicated, and used modeling results.

Results

We retrieved 1,061 references. After removing duplicates (127), 1,016 abstracts and titles were screened. From an initial selection of 142 abstracts, 133 research groups were identified, of which 67 didn’t meet the eligibility criteria. Of these, 66 groups were invited for an interview, of which 24 were available, including 18 modeling groups from academic institutions, and four groups from State Health departments. Most models assessed the impact of mitigation measures in cases/hospitalization/deaths and healthcare service demand. Interaction and communication with decision-makers were not well established in most groups.

Conclusions

Despite a large number of modeling groups in Brazil, we observed a significant gap in modeling demand and communicating its results to support the decision-making process during the COVID-19 pandemic.
PP45 The Cost-Of-Illness Of Diabetic Macular Edema In Italy
Authors: Basile Michele , Calabrò Giovanna Elisa , Bandello Francesco , Varano Monica , Castronovo Giuseppe , Amore Filippo , Melchiorre Tiziano , Cicchetti Americo
Published: December 2022

Introduction

Diabetic Macular Edema (DME) is an important complication of Diabetic Retinopathy (DR). Intravitreal steroids in slow-release systems represent a safe and effective therapeutic option for the management of DME, capable of improving patients’ quality of life by reducing the number of injections thus increasing the therapeutic adherence and the effectiveness of the treatment. This study aims to determine the economic impact of DME and the consequences, in terms of both expenditure and organizational impact, associated with a greater use of the intravitreal dexamethasone implant.

Methods

The analysis entailed the comparison between two scenarios: a first scenario based on the current use of therapeutic alternatives available in the Italian healthcare setting (as is) and an alternative scenario based on the assumption of an increased use of intravitreal dexamethasone implant (to be). The results of the analysis are expressed in terms of resource absorption associated with the two scenarios as well as in terms of the cost differential given by their comparison.

Results

Despite an increase in expenditure in terms of acquisition costs of pharmacological alternatives (EUR 898,362) and interventions provided (EUR 22,093,160), the greater use of prolonged-release dexamethasone allows for significant savings in terms of healthcare professionals’ time, follow-up and productivity losses incurred by patient/caregiver. These reductions in healthcare costs resulted in a saving of EUR 1,987,678 over a 5-year period. Such a reduction would allow, considering a total annual management cost of EUR 6,115 for the intravitreal dexamethasone, to treat 325 more patients at the same cost of the as is scenario based on the current rate of use of dexamethasone.

Conclusions

In a context characterized by the need to increase the allocative efficiency of economic resources, the recourse to therapeutic alternatives, such as prolonged release dexamethasone, allowing the reduction of costs for the management of a given pathology is crucial to generate more value for patients and the entire society.
PP56 The Use of Computed Tomography for Detecting COVID-19 Pneumonia: Rapid Evidence Review
Authors: Mideksa Samson , Ababor Sabit , Gebreyohannes Yosef , Bogale Firmaye , Solomon Dagmawit , Getachew Tsegaye , Ararso Desalegn , Wolde Ermias , Kebede Zelalem , Tollera Getachew
Published: December 2022

Introduction

The reverse transcription polymerase chain reaction (RT-PCR) test is the gold standard for detecting coronavirus disease 2019 (COVID-19), but recent studies have reported false negative results for RT-PCR. Consequently, several countries are looking at chest computed tomography (CT) for the diagnostic workup of patients with suspected or probable COVID-19 to inform clinical management. Therefore, this rapid review provides the best available evidence on the use of chest CT for detecting COVID-19 pneumonia.

Methods

Literature searches were conducted in the PubMed, Cochrane Library, JBI Library, Epistemonikos, and Evidence Aid databases for relevant papers published from 1 December 2019 to 12 June 2020. The search strategy was based on the identified population, concept, and context with respect to the objective of the review. The methodological quality of the seven systematic reviews identified was appraised using the AMSTAR checklist.

Results

The use of CT as a first-line screening tool for COVID-19 is not beneficial. Nevertheless, for patients with suspected COVID-19 pneumonia who had a false-negative RT-PCR results, cross-checking with CT and a repeated RT-PCR test is essential to avoid misdiagnosis. Chest CT offered high sensitivity for detecting COVID-19 pneumonia among symptomatic individuals, especially in those with severe disease. It was also useful for evaluating the potential complications, disease severity, and progression of COVID-19 pneumonia. The features of CT images differed at the early, intermediate, and late stages of COVID-19 pneumonia, but the most common imaging findings were patchy and ground glass opacities. It was also found that diagnosing COVID-19 in children using CT is challenging.

Conclusions

Even though chest CT is not useful for primary screening of COVID-19, it is useful for detecting later stage disease and for evaluating patients with COVID-19 pneumonia. For patients with suspected COVID-19 pneumonia and a false negative RT-PCR result, cross-checking with a chest CT and a repeat RT-PCR test is essential to avoid misdiagnosis.
PP59 Multidimensional Evaluation Of The Reducer Device In Patients With Refractory Angina
Authors: Cicchetti Americo , Rumi Filippo , Siviero Ludovica , Fortunato Agostino
Published: December 2022

Introduction

Treatments for coronary heart disease patients have had major developments in recent decades, both in the pharmacological and interventional fields, and this has helped to prolong the survival of these patients. However, the growing number of patients who show persistent and disabling symptoms of angina proves that at the same time their quality of life has not been equally improved.

Methods

We conducted a multidimensional assessment coherent with health technology assessment methodology on the Coronary Sinus Reducer System (CSRS). CSRS is the latest line of therapy for patients with coronary artery disease who are ineligible for revascularization, demonstrate reversible ischemia, and have refractory angina pectoris (AP) despite optimal standard medical therapy. We performed a literature review in order to gather evidence on efficacy and safety of the device and on the economic and organizational impact of the procedure. In the economic domain we developed a cost-utility model based on a decision tree and a five-year time horizon budget impact model.

Results

Several studies in the literature have shown that this therapy is related to an increase in quality of life and an improvement in symptoms of refractory angina. The economic evaluations conducted show how the therapy, despite an increase in the resources absorbed in the first years of implementation, reaches a cost saving profile in the medium term due to positive outcomes, while leading to an increase in the quality of life in patients suffering from refractory angina.

Conclusions

The treatment of refractory angina remains a challenge for today’s medicine. Patients suffering from this condition are often described as “no option” patients. Thus, despite there is a need of further evidence to establish even more robustly the economic sustainability of the device, especially on its effectiveness in the medium-long term, the device should be taking into account in those patients who could benefit from it in terms of relieving the symptoms of angina and improving their quality of life.
PP68 Dexcom G6® Device For Diabetes During Pregnancy
Authors: Ramos-García Vanesa , Rivero-Santana Amado , Perestelo-Pérez Lilisbeth , Duarte-Díaz Andrea , Álvarez-Pérez Yolanda , Torres-Castaño Alezandra , Toledo-Chávarri Ana , Wägner Ana María , Rodríguez-Rodríguez Leticia , González-Rodríguez Carlos , Serrano-Aguilar Pedro
Published: December 2022

Introduction

Diabetes mellitus (DM) is one of the most frequent metabolic complications associated with pregnancy, affecting both the prognosis of the pregnant woman and the newborn. Pregestational DM type 1 (T1DM) and type 2 (T2DM) and gestational DM (GDM) are associated with an increased risk of pregnancy complications such as miscarriage, fetal malformations, macrosomia, preeclampsia, and neonatal hypoglycemia, among others. The aim of this review was to evaluate the efficacy and safety of using the Dexcom G6 device (Dexcom, Co., USA) to continuously self-monitor blood glucose levels during pregnancy. This report was requested by the Spanish Ministry of Health.

Methods

We systematically searched for articles published to July 2021 in the MEDLINE, Embase, and Web of Science databases. We included experimental and observational primary studies addressing the safety, efficacy, and cost effectiveness of the Dexcom G6 device for gestational and pregestational diabetes.

Results

Two non-comparative prospective studies were identified. One study of 25 pregnant women with T1DM, which evaluated glycemic control and complications during pregnancy and postpartum, reported stable hemoglobin A1c levels during gestation in women using the Dexcom G6 device. The percentage of time spent in the therapeutic glucose range (63 to 140 mg/dL) was 59 percent; 38 percent was in the hyperglycemic range and 3 percent was in the hypoglycemic range. Although some patients reported mild erythematous and edematous reactions to the sensor, no moderate or severe reactions or infections occurred at the sensor insertion site. The other study in pregnant women with T1DM (n=20), T2DM (n=3), or GDM (n=9) showed adequate accuracy of the Dexcom G6 device, compared with the reference method, especially when the sensor is placed on the arm.

Conclusions

Randomized controlled trials are required to assess the effectiveness and safety of the Dexcom G6 device in maintaining adequate glucose control during pregnancy in women with DM. Studies are also needed to compare the Dexcom G6 device with conventional capillary blood glucose self-monitoring or other monitoring methods. No cost-effectiveness studies have been conducted for the Dexcom G6 device in this patient population.
PP74 Taking A Lifecycle Approach To Scottish Medicines Consortium Budget Impact Analysis
Authors: Booth Corinne , Dimitrova Maria , Henriquez Alex , Hislop Jennifer , Manson Jan , Wright Helen
Published: December 2022

Introduction

The Scottish Medicines Consortium (SMC) conducts early health technology assessment of new medicines in Scotland. While budget impact is not a factor in decisions on reimbursement, budget impact information is provided initially through horizon scanning reports for high impact medicines (estimated net budget impact >GBP 500,000 [EUR 585,710] per annum) to aid financial planning and implementation of advice at the local level, and later through budget impact templates from the submitting company issued alongside SMC advice. This research aimed to understand how the information is used and to evaluate the benefits of a lifecycle approach to budget impact analysis.

Methods

Health Board users of the budget impact templates were surveyed to explore the degree of utilization and identify areas for improvement, including the need to cross-validate the horizon scanning estimates with those of the submitting company. Responses were analyzed quantitatively and qualitatively, with comments coded in Nvivo (QSR International) and themes established through thematic analysis.

Results

The initial responses received (n=17) provided representation from 57 percent of Health Boards (i.e., payers) covering 79 percent of the population. Preliminary results showed that while the budget impact templates were valued, 69 percent of respondents found them ‘somewhat useful’, suggesting scope for improvement. Almost half (48%) of the respondents used the templates for high impact medicines, with only 30 percent using them for all medicines. The majority (76%) of those surveyed thought there would be value in linking budget impact information throughout the SMC process. An emerging theme was that some users found the templates complex and inflexible, and that a simpler, more adaptable tool to aid the planning process would be welcomed. Priorities identified for improving template included adapting them to the local population and adjusting medicine prices to reflect confidential discounts.

Conclusions

This research suggests that budget impact information is valued by Health Boards and that there is strong support for linking budget impact estimates and engaging stakeholders throughout a medicine’s lifecycle. Simplifying the templates, increasing their adaptability, and providing guidance and training in their use will be key steps in improving this important part of SMC process.
PP84 Change Management Of Patient Associations In Italy: From Emergency Response To Organizational Learning
Authors: Di Brino Eugenio , Morandi Federica , Cicchetti Americo
Published: December 2022

Introduction

The role of associations dedicated to patient advocacy has assumed strategic importance within the most advanced health systems, including the Italian NHS. In this period of strong national and international emergency, the associations of citizens and patients have also changed their actions and have implemented others to alleviate the discomforts of sick people in Italy, collaborating with institutions and health services.

Methods

Data were collected using a semi-structured survey, with both yes / no and open questions, developed and administered by the Patient Advocacy LAB (ALTEMS- Catholic University of Sacred Heart) to 150 patient advocacy associations. The organizational changes and the initiatives adopted by patient associations during the COVID-19 emergency was investigated.

Results

The majority of the initiatives adopted by patient advocacy associations during the COVID-19 pandemic have been introduced during the first wave (March- June 2020), and that most of them have been maintained to (December 2020). These initiatives included improvements and updating of the communication tools aimed at reaching the higher number of patients. Thanks to these new approaches, the empathy and the assistance to patients have been increased. In addition, a number of training initiatives have been developed online and they have been followed by a large number of patients and caregivers. At an operative level, during the pandemic, many patient advocacy associations have provided their support to distribute masks, gloves, and therapies to patients. Unfortunately the fundraising activities suffered due to the lockdown, and the main problems were related to social distancing and lack of public campaigns. The institutional relationships have increased during the COVID-19 pandemic and they mainly regarded the participation in legislative interventions. Among the initiatives adopted, our sampled associations affirmed that they would like to maintain the social support provided to patients experienced during the pandemic.

Finally we calculated a resiliency score and we discovered that the majority of the sampled associations developed an intermediate level of resiliency and that it is positively correlated with their tenure.

Conclusions

Our results provide a fresh view about the role of patient advocacy associations during the pandemic indicate their important role within the NHS.
PP88 Bayesian Joint Models For Cost-Effectiveness Analyses Based On Clustered Participant Data, With Implementation In Stan
Authors: Esser Jonas , Varga Anita , Alili Mohamed El , Van Dongen Johanna , Bosmans Judith
Published: December 2022

Introduction

Cost-effectiveness analyses of empirical participant data are frequently complicated by irregularly distributed and correlated observations, which are not well approximated by normal distributions. Things get even more difficult when observations are clustered within higher level units (for example, hospitals) or the participant (that is, multiple measurements at different timepoints). Therefore, we developed a flexible Bayesian approach to jointly model costs and effects of two competing interventions with a multilevel structure.

Methods

Our new model is presented in mathematical form and discussed in detail. We model costs and Quality-Adjusted Life-Years effects through Gamma and Beta distributions, and account for the dependency between costs and effects by adding the effects as a predictor for the costs. We further include hurdle models to account for costs of for the presence of zero costs and perfect health scores.

The full model is implemented in the probabilistic programming language Stan. To compare the performance of our Bayesian model to a frequentist approach (linear mixed model combined with bootstrapping), we simulate 1000 datasets consisting of 400 participants and 20 clusters. Performance of both models is assessed in terms of variance, bias and coverage probability with respect to the costs and effects defined in the simulation.

Results

We ran a preliminary simulation with high intraclass correlation, strong negative correlation for patient-level costs and effects, and positive correlation of cluster effects on both outcomes. The analysis shows that the Bayesian model exhibits a slightly larger bias for estimated costs, but smaller errors and higher coverage probability compared to the frequentist alternative. We will explore different scenarios where we vary the parameters of the simulations and assess whether the results are robust to change.

Conclusions

It is very important that economic evaluations in health care produce precise and reliable results. Our Bayesian approach is able to handle multiple statistical complexities at once and performs better than a comparable frequentist model. Whether this conclusion holds for different simulation scenarios will be explored in further stages of this study.
PP90 Startup And Inclusion Problems In Healthcare Efficiency Studies: A Quantitative And Qualitative Analysis
Authors: Drenth Jennifer , Van Liere-visser Karen , Warmerdam Daniël , Zijp Inge , van Zessen Ruud
Published: December 2022

Introduction

Aging populations and specialized medicine are leading to increasing healthcare costs which are expected to rise in the next decades. The Netherlands Organization for Health Research and Development (ZonMw) funds trials that address the efficiency of healthcare interventions in order to evaluate new and existing interventions. These studies have led to considerable cost savings and increased health outcomes. However, efficiency studies often face setbacks during the start-up and inclusion which limit the available research capacity and postpone the availability of novel findings. Here, we investigate the scope of these problems and identify common causes.

Methods

Records from efficiency research trials funded by ZonMw from 2014-2020 were combined with information provided by project leaders through a survey. The combined dataset was explored through statistical analysis. Next, a subset of 30 selected projects was evaluated qualitatively to gain a better understanding of the possible underlying reasons for the experienced problems.

Results

The response rate among project leaders was 73 percent (146/201). Data indicate that 61 percent of projects started as planned and 35 percent included the first patient as scheduled. The complexity of setting up a multicenter study and legal procedures like local ethical approval were associated with delays in starting inclusions. In addition, 56 percent of studies had to extend the inclusion period by more than 6 months. Possible reasons that were identified include the limited numbers of patients available, and treatment preferences of the doctor, the patient, or the participating center.

Conclusions

Our results indicate that the majority of trials face setbacks and the main reasons include time to procure legal and ethical approval, limited patient numbers, as well as unforeseen treatment preferences. More streamlined procedures regarding approvals could speed up trial initiation, and better knowledge of eligible patients and treatment preferences could lead to more realistic planning. The results and conclusions from this study can be applied by ZonMw and other relevant stakeholders to resolve the identified problems in order to accelerate healthcare efficiency research.
PP92 Should Missing Data Be Multiply Imputed Prior To Longitudinal Linear Mixed-Model Analyses In Trial-Based Economic Evaluations?
Authors: Ben Ângela J , van Dongen Johanna M , Alili Mohamed El , Heymans Martijn W , Twisk Jos W R , MacNeil-Vroomen Janet L , de Wit Maartje , van Dijk Susan E M , Oosterhuis Teddy , Bosmans Judith E
Published: December 2022

Introduction

For the analysis of clinical effects, multiple imputation (MI) of missing data was shown to be unnecessary when using longitudinal linear mixed-models (LLM). It remains unclear whether this also applies to cost estimates from trial-based economic evaluations, that are generally right-skewed. Therefore, this study aimed to assess whether MI is required prior to LLM when analyzing longitudinal cost-effectiveness data.

Methods

Two-thousand complete datasets were simulated containing five time points. Incomplete datasets were generated with 10 percent, 25 percent, and 50 percent missing data in costs and effects, assuming a Missing At Random (MAR) mechanism. Statistical performance of six different methodological strategies was compared in terms of empirical bias (EB), root-mean-squared error (RMSE), and coverage rate (CR). Six strategies were compared: (i) LLM (LLM), (ii) MI prior to LLM (MI-LLM), (iii) mean imputation prior to LLM (M-LLM), (iv) complete-case analysis prior to seemingly unrelated regression (CCA-SUR), (v) MI prior to SUR (MI-SUR), and (vi) mean imputation prior to SUR (M-SUR). To evaluate the impact on the probability of cost-effectiveness at different willingness-to-pay [WTPs] thresholds, cost-effectiveness analyses were performed by applying the six strategies to two empirical datasets with 9% and 50% of missing data, respectively.

Results

For costs and effects, LLM, MI-LLM, and MI-SUR performed better than M-LLM, CCA-SUR, and M-SUR, as indicated by smaller EBs and RMSEs, as well as CRs closer to the nominal levels of 0.95. However, even though LLM, MI-LLM, and MI-SUR performed equally well for effects, MI-LLM and MI-SUR were found to perform better than LLM for costs at 10 percent and 25 percent missing data. At 50 percent missing data, all strategies resulted in relatively high EBs and RMSEs for costs. In both empirical datasets, LLM, MI-LLM, and MI-SUR all resulted in similar probabilities of cost-effectiveness at different WTPs.

Conclusions

When opting for using LLM for analyzing trial-based economic evaluation data, researchers are advised to multiply impute missing values first. Otherwise, MI-SUR may also be used.
PP93 The Impact Of Using Different EQ-5D Scoring Methods On Cost-Utility Outcomes: A Simulation Study
Authors: Ben Ângela J , van Dongen Johanna M , Finch Aureliano P , Alili Mohamed El , Bosmans Judith E
Published: December 2022

Introduction

Patients’ EQ-5D health states are preferably valued using country-specific value sets. If value sets are not available, crosswalks may be used to estimate utility values. However, up until now the impact of using crosswalks instead of value sets on cost-utility outcomes remains unclear.

Methods

Trial-based cost-utility data were simulated for four conditions (depression, low back pain, osteoarthritis, and cancer), three levels of disease severity (mild, moderate, and severe), and three treatment effect sizes (small, medium, and large), resulting in 36 scenarios. For all scenarios, utility values were estimated using four scoring methods (EQ-5D-3L value set, EQ-5D-5L value set, 3L-to-5L crosswalk, and 5L-to-3L crosswalk) for three countries (the Netherlands, the United States, and Japan). Mean utility values, quality-adjusted life years (QALYs), incremental QALYs, and cost-utility outcomes (incremental cost-effectiveness ratios [ICER], probabilities of cost-effectiveness at willingness-to-pay [WTP] thresholds) were compared between value sets and crosswalks.

Results

Differences between value sets and crosswalks ranged from -0.33 to 0.13 for mean utility values, from -0.18 to 0.13 for QALYs, and from -0.01 to 0.08 for incremental QALYs. Because of the small differences in incremental QALYs, ICERs between scoring methods were considerably different. For small effect sizes, at a WTP of EUR 20,000/QALY gained, the largest difference in the probability of cost-effectiveness was found for moderate cancer between the 5L value set and 3L-to-5L crosswalk (difference 0.63) using Japanese valuations. For medium effect sizes, the largest difference was found for mild cancer between the 3L value set and the 5L-to-3L crosswalk (difference 0.06) using Japanese valuations. For large effect sizes, the largest difference was found for mild osteoarthritis between the 3L value set and 5L-to-3L crosswalk (difference 0.08) using Japanese valuations.

Conclusions

Our findings indicate that reimbursement decisions may change depending on the use of crosswalks. Crosswalks are justifiable in absence of country-specific value sets but should not be considered a sustainable alternative for value sets.
PP94 Exploring The Relationship Between Price And Outcome Of The Health-Economic Assessment In Japan
Authors: Song Xirong , Sejpal Nehal , Konar Rangaraja , Patil Saurabh , Gusain Sapana , Sonigra Preksha , Tikone Rutuja
Published: December 2022

Introduction

This study reviewed all publicly available Japanese cost-effectiveness appraisals delivered by the Center for Outcomes Research and Economic Evaluation for Health (C2H) from the national institute of public health (NIPH) and analyzed the relationship between the outcomes of the health-economic assessments and the final price adjustment decisions made by the Ministry of Health, Labour and Welfare (MHLW).

Methods

Data were extracted from all official health-economic assessments published by C2H website for the analysis. The extractions were structured based on the following items: indication, assessment methodology, appropriate comparators, health-economic outcomes, and key uncertainties identified by C2H. The analysis was performed on 29th November 2021. A threshold of 5 million JPY per Quality Adjusted Life Year gained (JPY/QALY) was used for the incremental cost-effectiveness ratio (ICER) analysis cut-off.

Results

Up to the time of this analysis, six health-economic assessments had been conducted for five products: three assessments performed cost-effectiveness analysis, one performed cost-minimization analysis and two performed cost-effectiveness and cost-minimization analysis for different comparators and different patient subgroups respectively. Among the five assessments’ reported ICER values, four of them are under the 5 million JPY/QALY threshold, ranging from 328,585 JPY/QALY to 483,056 JPY/QALY. However, price adjustments were still implemented on three out of the four products which were deemed to be cost-effective, ranging from -0.5 percent to -4.3 percent (mean: -3.0%). For the only product deemed to be not cost-effective, a price adjustment of -4.3 percent was implemented.

Conclusions

A price discount could be implemented regardless of whether the ICER value falls under the 5 million JPY/QALY threshold. However, a lower magnitude of price discount is likely to be applied by MHLW for more cost effective treatments. The outcome of this analysis may be limited by the small sample size and continuous monitoring of further HTA publications in Japan is needed.