Health Care Technology Assessment Research Articles

ФАРМАКОЭКОНОМИЧЕСКИЕ ПОДХОДЫ К АНАЛИЗУ СТОИМОСТИ АНТИРЕТРОВИРУСНЫХ ПРЕПАРАТОВ, ПРИМЕНЯЕМЫЕ В ТЕРАПИИ ВИЧ-ИНФИЦИРОВАННЫХ ПАЦИЕНТОВ

The legislation in force in the Russian Federation guarantees HIV patients protection of their rights in labor, education and other spheres of life, as well as free counseling, diagnosis and treatment of HIV infection. The continued increase in the number of infected people and, more importantly, the emergence of the HIV epidemic from risk groups into the general population highlights the need for additional improvement in the effectiveness of measures taken to counter the spread of infection. In this regard, it is necessary to use the most rational measures, both from the point of view of efficiency and safety, and from the point of view of costs. The most suitable tool for solving these problems is pharmacoeconomical analysis. At the same time, conducting pharmacoeconomical analysis in the field of HIV infection, as well as in any other nosology, involves the need to take into account certain features at the stages of planning and implementation of the study. It is necessary to highlight some key features of the methodology of pharmacoeconomical analysis in the assessment of healthcare technologies in the field of HIV infection, in particular, cost minimization analysis, which involves choosing a medical technology with minimal costs by comparing the monetary value of alternative technologies, provided the same therapeutic effectiveness. The main object of the study was the documented medical histories of HIV-infected patients, statistical and analytical methods of analysis were used and it was established that in the Russian Federation and in Kuzbass there are all drugs for the treatment of patients with HIV infection included in modern lists of treatment standards according to clinical recommendations. The difference in the cost of antiretroviral drugs at the price of government funding has been established, which affects the costs of treating HIV-infected patients.

Cost of epilepsy in the Russian Federation

Background. Epilepsy underdiagnosis in Russia, population aging, innovative drugs and expensive treatment regimens, the introduction of additional and alternative methods such as neurosurgery, annually increase the cost for treatment of epilepsy patients. In this regard, an issue of maximizing benefits for the largest number of the population becomes relevant. Pharmacoeconomical research within the assessment of healthcare technologies helps to choose the most economically and medically effective treatment strategy among numerous alternatives.Objective: analysis of epilepsy-associated costs in the Russian Federation, aimed at supporting decision-making processes in the field of health policy and financing.Material and methods. The empirical study was based on collecting the data on a continuous sampling of 384 epilepsy outpatient records from subjects aged 19 to 79 years for the period 2019–2022. The issues related to all costs of epilepsy were discussed. While planning, conducting and analyzing these studies, the principles of strengthening the reporting of observational studies in epidemiology (STROBE) were taken into account.Results. The total annual per-patient expenditure on epilepsy treatment comprised a fifth of gross domestic product per capita. Indirect costs exceed direct cost savings. It was found out that the economic damage caused to society, family, and individual related to epilepsy is three times higher than the direct costs.Conclusion. The results obtained are consistent with other publications in which unemployment among epilepsy patients is one of the major cost-determining factors and illustrates a critical need to maintain employment for such patients or to find suitable alternative work through professional retraining.

Validation, adaptation and application of a multi‐criteria decision analysis‐based framework for chemotherapeutic agents in Egypt

AbstractHealth technology assessment is a systematic, transparent, robust, and unbiased assessment of healthcare technology that links evidence and decisions. Healthcare decisions usually require trading‐off, and using a structured approach, such as multi‐criteria decision analysis (MCDA), could improve decision quality. This study aimed to adapt an MCDA‐based framework for oncological drug evaluation in an Egyptian clinical setting to explore the perspectives of different stakeholders. The 10th edition of the Evidence and Value: Impact on decision making framework was translated, validated, adapted, and applied to explore key differences between the priorities of different stakeholders for coverage decisions in the oncology field in Egypt. A direct rating scale or hierarchical point allocation was used to elicit criteria weights. Normalized weights were calculated and compared using the preset cut‐off value of 0.008. Understandability, willingness to use the adopted weighting techniques, and cognitive burden were assessed and compared. Ninety participants completed the criteria‐weighting interview for the adapted tool. The elicited criteria weights differed between the two adopted weighting techniques when comparing stakeholders' priorities using the preset cut‐off (0.008). However, there was no significant difference in the weighting technique understandability, future use willingness, and cognitive burden parameters on categorization by either the weighting technique or stakeholder group. This study provides a specially tailored, straightforward procedure for Egyptian stakeholders to evaluate oncologic drugs in a standardized manner by combining various inputs. It also highlights the main variations across Egyptian stakeholder preferences.

Rationing in an Era of Multiple Tight Constraints: Is Cost-Utility Analysis Still Fit for Purpose?

Cost-utility analysis may not be sufficient to support reimbursement decisions when the assessed health intervention requires a large proportion of the healthcare budget or when the monetary healthcare budget is not the only resource constraint. Such cases include joint replacement, coronavirus disease 2019 (COVID-19) interventions and settings where all resources are constrained (e.g. post-COVID-19 or in low/middle-income countries). Using literature on health technology assessment, rationing and reimbursement in healthcare, we identified seven alternative frameworks for simultaneous decisions about (dis)investment and proposed modifications to deal with multiple resource constraints. These frameworks comprised constrained optimisation; cost-effectiveness league table; 'step-in-the-right-direction' approach; heuristics based on effective gradients; weighted cost-effectiveness ratios; multicriteria decision analysis (MCDA); and programme budgeting and marginal analysis (PBMA). We used numerical examples to demonstrate how five of these alternative frameworks would operate. The modified frameworks we propose could be used in local commissioning and/or health technology assessment to supplement standard cost-utility analysis for interventions that have large budget impact and/or are subject to additional constraints.

Artificial Intelligence in Surgery: The Future is Now.

Background Clinical Artificial intelligence (AI) has reached a critical inflection point. Advances in algorithmic science and increased understanding of operational considerations in AI deployment are opening the door to widespread clinical pathway transformation. For surgery in particular, the application of machine learning algorithms in fields such as computer vision and operative robotics are poised to radically change how we screen, diagnose, risk-stratify, treat and follow-up patients, in both pre- and post-operative stages, and within operating theatres. Summary In this paper, we summarise the current landscape of existing and emerging integrations within complex surgical care pathways. We investigate effective methods for practical use of AI throughout the patient pathway, from early screening and accurate diagnosis to intraoperative robotics, post-operative monitoring and follow-up. Horizon scanning of AI technologies in surgery is used to identify novel innovations that can enhance surgical practice today, with potential for paradigm shifts across core domains of surgical practice in the future. Any AI-driven future must be built on responsible and ethical usage, reinforced by effective oversight of data governance, and of risks to patient safety in deployment. Implementation is additionally bound to considerations of usability and pathway feasibility, and the need for robust healthcare technology assessment and evidence generation. While these factors are traditionally seen as barriers to translating AI into practice, we discuss how holistic implementation practices can create a solid foundation for scaling AI across pathways. Key Messages The next decade will see rapid translation of experimental development into real-world impact. AI will require evolution of work practices, but will also enhance patient safety, enhance surgical quality outcomes, and provide significant value for surgeons and health systems. Surgical practice has always sat on a bedrock of technological innovation. For those that follow this tradition, the future of AI in surgery starts now.

Данные реальной клинической практики: правовое поле в РФ

The real-world data (RWD) constitute a modern trend in the healthcare system and in the structure of taking decisions. The trend is being actively developed in the Russian Federation. Ethical aspects of these trials and legal regulation of RWD are, however, still unresolved. In this paper, development of the legal field and national regulation of RWD in Russia is discussed during registration, assessment of healthcare technologies, management of patient registries, and development of clinical recommendations. It is shown that the acting regulatory field of the Russian Federation contains no bans on the use of RWD to support regulatory decisions. Nevertheless, the existing legal laws set limited opportunities for practical application and accounting of RWD by the competent authority. In conclusion, recommendations on further development of RWD legal field in Russia are provided.

A Preference-Based Value Assessment of the Fear of COVID-19 Contagion.

To assess the preference-based value of the fear of COVID-19 contagion. We conducted a web-based, cross-sectional discrete choice experiment among 544 US adults. We used a Bayesian efficient design to generate choice sets. Each choice set comprised two hypothetical COVID-19 vaccine options characterized by seven attributes: chance of COVID-19 infection, chance of having severe symptoms from COVID-19 infection, vaccine protection duration, chance of mild to moderate adverse events from vaccination, chance of serious adverse events from vaccination, chance of future exposure to COVID-19 after vaccination, and out-of-pocket cost. We used mixed logit (ML) and latent class (LC) models to analyze data. Furthermore, we calculated the willingness-to-pay for eliminating the chance of future exposure to COVID-19, shedding light on the value attributed to the fear of contagion. The ML model demonstrated all attributes, including the chance of future exposure to COVID-19, were statistically significant. The participants were willing to pay approximately $13,046 to eliminate the chance of future exposure to COVID-19 or their fear of contagion when COVID-19 was still pandemic. The LC model unveiled two participant classes with distinct preference weights for the chance of future exposure to COVID-19 and out-of-pocket cost attributes. Nevertheless, the chance of future exposure to COVID-19 exposure held a significant degree of importance in both classes. The chance of future exposure to COVID-19 exposure or fear of contagion was a significant element in the value assessment of COVID-19 vaccines. Further studies should be conducted to verify the value of fear of contagion and include it in the value assessment of healthcare technologies for infectious diseases.

A Bayesian change-point detection approach to the economic evaluation of risky projects: an application to healthcare technology assessment

Abstract We propose a Bayesian hypothesis testing framework that allows for the assessment of evidence collected during a clinical trial about the cost-effectiveness of a healthcare technology. The model exploits a Bayesian updating rule that makes the link between the evidence collected in clinical research and the expected payoffs of adoption to the healthcare system. The framework takes into account the cost of decision errors in the payoff function, allowing the decision maker to compute the cost of taking a decision when evidence is far from the optimal decision triggers. We show, using a real-world cost-effectiveness study based on clinical trial evidence, how rules derived from a sequential adaptive design approach can lead to quicker decisions when compared to the value of information decision framework. Our application shows that a sequential approach has the potential to lead to quicker decisions, higher payoffs, and better health outcomes.

EE447 Methods of Incorporating Changes in Drug Price over Time into Health Economic Evaluations: A Targeted Literature Review

Drug prices are innately dynamic, yet conventional economic evaluation assumes a constant price over time. Some healthcare technology assessment agencies, including the Institute for Clinical and Economic Review, do permit the inclusion of life-cycle drug pricing (LCDP) under certain circumstances. However, little guidance is given when this should be implemented and how it should be done. Our aim was to identify methods of incorporating LCDP into cost-effectiveness models, and characterize the current state-of-the-art.

Pulp fiction or pulp fact-does orthodontics damage pulpal tissue?

Electronic databases PubMed, Scopus and Embase were systematically searched and restricted to articles published between February 2009 and 2022. Studies were categorized using the modified method by the Swedish Council of Technology Assessment in Health Care. 20 studies were included, one of which was categorized as high quality (Grade A) and 19 of which were of moderate quality (Grade B). Exclusion criteria included articles with insufficient descriptions of reliability and reproducibility testing, review articles and case reports, and studies including traumatised teeth. Three independent authors examined titles, abstracts and full texts of relevant articles against the inclusion criteria. Disagreements were resolved by discussion. Retrieved studies were assessed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Data extracted included tooth movement performed, the appliance and force used, follow-up of subjects, changes in pulpal blood flow (PBF), tooth sensibility, expression of inflammation-related proteins, along with pulpal histology and morphology changes during tooth movement (intrusion, extrusion and tipping). Overall risk of bias was unclear. A reduction in pulpal blood flow and tooth sensibility due to the application of orthodontic forces was reported by studies included in the review. Increases in the activity of proteins and enzymes related to inflammation of the pulp was reported. Two studies reported histological changes of the pulpal tissues related to orthodontic treatment. Orthodontic forces cause multiple temporary detectable changes in the dental pulp. The authors conclude there are no clear signs of permanent pulpal damage to healthy teeth due to the application of orthodontic forces.

HTA49 Implementation of Equity as an Element of Value in Health Care Technology Assessments in Europe

HTA49 Implementation of Equity as an Element of Value in Health Care Technology Assessments in Europe

Integrating Empirical Analysis and Normative Inquiry in Health Technology Assessment: The Values in Doing Assessments of Health Technologies Approach.

Health technology assessment (HTA) aims, through empirical analysis, to shed light on the value of health technologies (O'Rourke et al. [2020, International Journal of Technology Assessment in Health Care 36, 187-90]). HTA is, then, where facts and values meet. But how, where, and when do facts and values meet in HTA? Currently, HTA is usually portrayed as a sequential process, starting with empirical analysis (assessment), followed by a deliberation on the implications of the findings for a judgment of a health technology's value (appraisal). In this paper, we will argue that in HTA, empirical analysis and normative inquiry are much more closely entwined. In fact, as we hope to show, normative commitments act as an indispensable guide for the collection and interpretation of empirical evidence. Drawing on policy sciences, we will suggest a concrete methodology that can help HTA practitioners to integrate empirical analysis and normative inquiry in a transparent way. The proposed methodology can be conceived as a concrete means for conducting a scoping exercise in HTA. Moreover, it offers a distinct way of giving stakeholders a structural and constructive role in HTA. This paper outlines the approach developed by the values in doing assessments of health technologies project, a project funded by the Erasmus+ program (contract number 2018-1-NL01-KA203-038960), which is the European Union's program to support education, training, youth, and sport in Europe. The project has resulted in an E-learning course, an accompanying handbook, and a consensus statement, all freely available from the project's website www.validatehta.eu.

Scoping review of rapid health technology assessment

This study aims to summarize the research on rapid health technology assessment(RHTA) based on scoping review, which is expected to serve as a reference for future research on RHTA. First, articles on RHTA were retrieved from both Chinese and English databases. After data screening and extraction by two personnel independently, descriptive analysis was conducted on the results, and evidence distribution was analyzed based on tables, bar charts, line charts, radar charts, and pie charts. Finally, a total of 82 eligible articles were included and the characteristics were as follows.(1) Articles on RHTA were mainly published from 2011 to 2022. The number of articles showed an obvious increase from 2014 and surged in 2019.(2) Among the journals with the above RHTA papers published, Chinese Journal of New Drugs(21.5%), Evaluation and Analysis of Drug-Use in Hospitals of China(16.9%), and Chinese Pharmacy(15.4%) topped the Chinese journals in the number of the papers, while International Journal of Technology Assessment in Health Care(23.5%), Reviews in Medical Virology(11.8%), and Value in Health(11.8%) came out on top in the English journals.(3)The RHTA of drugs(especially western medicine)(68.7%) dominated the eligible articles, followed by the RHTA of therapy technology(13.1%), detection technology(5.1%), and diagnosis technology(1.0%). There was a significant gap in the number of studies among different health technology categories and an imbalance in the types of health technologies involved in the overall RHTA studies.(4) RHTA of tumors has been the research focus in recent years. In summary, RHTA plays a positive role in rapid health decision-making, but there is a lack of primary data sources at present. Compared with the systematic review, Meta-analysis, and pharmacoeconomic studies, a few reports on health technology assessment(HTA) were included. In the future, research on the evaluation of the safety, efficacy, cost effectiveness and social applicability of health technologies should be further strengthened.

Overview of Trending Medical Technologies

Healthy population is regarded as the most valuable asset of any country. Unfortunately, the health challenges that hinder mankind's wellbeing are enormously increasing. Examples include but are not limited to: the diversity of emerging diseases afflicting the global population, the projected demographic growth of elderly people who need consistent monitoring, the deficiency in medical staff, the lower density of physicians, and the challenging geographical location of the population from healthcare providers. The mitigation of such health challenges calls for novel technologies to improve patient outcomes. In this article, seven emerging technologies, namely: Wearable Devices and Internet of Things, Artificial Intelligence, Blockchain Technology or Distributed Ledger Technology, Robotics Technology, Telehealth and Telemedicine, Big Data Technology and Nanomedicine have been highlighted. For each discussed technology, its historical background, development drivers, market status and trends, significance to healthcare, key player companies, and associated challenges have been presented. The information contained in this paper was collected from different journal articles, websites, reports, conference proceedings, and books. It was observed that though the technologies discussed in this article show growth at different rates, healthcare technology development and implementation are very promising in revolutionizing the health sector and improving the health of the population. Therefore, healthcare providers and countries are recommended to put in place Healthcare Technology Assessment Programs to help them collect data regarding the technology efficacy, relevance, safety, outcomes, and alternative technologies towards better planning for healthcare services improvement.

Documentary Practices of Hospital Librarians in Evidence-based Medicine: the Example of Health Technology Assessment in Swedish Healthcare

Today’s healthcare rely on a basis of evidence-based medicine (EBM) and in modern healthcare there are demands for rational decision-making about new methods, technology and treatments. HTA (Health Technology Assessment) supports decision-making in healthcare and in this study we turn to documentary practices of hospital librarians in HTA, as well as how documentary practices shape and are shaped by the work and roles of hospital librarians. Five central documentary practices were identified as initial searching, negotiating a search strategy, the main searching, making a selection, and documenting the search process. These practices construct the work and roles of hospital librarians through different documents, for example formal guidelines for systematic reviews and various tools used for searching, selecting and documenting the search process.

The Challenge of Sustainable Access to Telemonitoring Tools for People with Diabetes in Europe: Lessons from COVID-19 and Beyond.

Telemedicine in diabetes care has been evolving over several years, particularly since the advent of cloud-connected technologies for diabetes management, such as glucose monitoring devices, including continuous glucose monitoring (CGM) systems, that facilitate sharing of glucose data between people with diabetes and their healthcare professionals in near-real time. Extreme social distancing and shielding in place for vulnerable patients during the COVID-19 pandemic has created both the challenge and the opportunity to provide care at a distance on a large scale. Available evidence suggests that glucose control has in fact improved during this period for people with diabetes who are able to use CGM devices for remote glucose monitoring. The development of telemedicine as part of the standard of care in diabetes faces significant challenges in the European context, particularly in terms of providing consistent and effective care at a distance to large populations of patients while using robust systems that can be supported by large regional and national healthcare services. These challenges include a fragmented approach to healthcare technology assessment and reimbursement, lack of eHealth education and literacy, particularly amongst healthcare professionals, lack of data integration, as well as concerns about electronic health records, patient consent and privacy. Here we review the benefits of and challenges to wider application of telemedicine and telemonitoring in the post-pandemic future, with the aim to ensure that the value of these eHealth services is provided to patients, healthcare providers and health systems.

How does HTA addresses current social expectations? An international survey.

Integration of ethics into technology assessment in healthcare (HTA) reports is directly linked to the need of decision makers to provide rational grounds justifying their social choices. In a decision-making paradigm, facts and values are intertwined and the social role of HTA reports is to provide relevant information to decision makers. Since 2003, numerous surveys and discussions have addressed different aspects of the integration of ethics into HTA. This study aims to clarify how HTA professionals consider the integration of ethics into HTA, so an international survey was conducted in 2018 and the results are reported here. A survey comprising twenty-two questions was designed and carried out from April 2018 to July 2018. Three hundred and twenty-eight HTA agencies from seventy-five countries were invited to participate in this survey. Eighty-nine participants completed the survey, representing a participation rate of twenty-seven percent. As to how HTA reports should fulfill their social role, over 84 percent of respondents agreed upon the necessity to address this role for decision makers, patients, and citizens. At a lower level, the same was found regarding the necessity to make value-judgments explicit in different report sections, including ethical analysis. This contrasts with the response-variability obtained on the status of ethical analysis with the exception of the expertise required. Variability in stakeholder-participation usefulness was also observed. This study reveals the importance of a three-phase approach, including assessment, contextual data, and recommendations, and highlights the necessity to make explicit value-judgments and have a systematic ethical analysis in order to fulfill HTA's social role in guiding decision makers.

Efficacy of Clear Aligners in Correcting Anterior Open Bite- A Systematic Review

Objective:The aim was to systematically search the literature and assess the available evidence regardingtheclinical effectiveness of clear aligners in correcting anterior open bite.Materials and Methods:PubMed, PubMed Central, MEDLINE, Embase, National Library of Medicine,Cochrane Library, Web of Knowledge, and LILACS were searched electronically to identify all peerreviewed articles till date potentially relevant to the review.The reference lists of all eligible articles wereexamined for additional studies. Additional manual search was also conducted in many orthodontic journalsof interest, and unpublished articles were also searched for. To rate the methodological quality of the selectedarticles, a grading system described by the Swedish Council on Technology Assessment in Health Care wasused.Results:Ten relevant articles were selected to be included in this systematic review. According to the SBUtool, among the selected studies, the methodological quality was moderate for four studies and limited forthe others. Thus, conclusions with a limited level of evidence could be arrived at from the review process.The most recurrent sources of bias were related to the study design, lack of blinding procedure, the samplesize, and the lack of control group. However, there was substantial consistency among studies that clearaligner therapy is a viable option to correct anterior open-bite.Conclusion:No definite conclusion could be derived due to the heterogeneity of the studies. Due to thelimitations of this study, further clinical and experimental research is needed to statistically assess long-termstability of occlusion and skeletal relationships in treated open-bite cases.

29.C. Workshop: Assessment, regulation and use of apps for health management: where we are and where we are going?

Abstract Maintaining and improving health is one of the fundamental human right that may be pursued through the assurance of an equal and affordable access to health services to everyone. In this light, the growing use of mobile communication technology in the health sector is helping people to use preventive, diagnostic and therapeutic services more easily. In fact, through mobile communications technologies, also known as mHealth, people can have access to different services, namely doctors' consultations, follow-up visits, and medication management. From the side of health professionals, mHealth could allow to streamline tracking patients' data and making a timely use of patients' health data. Eventually, mHealth could also affect citizens, patients and health service as a whole, improving monitoring, reducing hospital admissions and enhancing education. Available data on the use of mobile technology among population let mHealth, and in particular mobile health applications (health apps), be a cutting-edge tool for the management of population health. In witness of this, the NICE is developing a system for evaluating health apps following its experience in the healthcare technology assessment (HTA) program. Furthermore, new HTA-like framework, i.e. The Model for Assessment of Telemedicine Applications (MAST), and new guidance for health apps assessment have been proposed. In fact, health apps may be considered as a health technology and may be classified as medical device considering that the new regulation of the European parliament 2017/745 includes among medical devices software intended to be used, alone or in combination, for human beings for one or more specific medical purposes. Even though current statistics show that health apps are not among the top downloaded apps, it may be guessed that their use will increase in the next years because of observed general trend and health digitalization. In this light, where do we are in terms of regulation and assessment of health apps? From the regulatory viewpoint, Germany is playing a forerunner role as it is working in order to make health apps prescribed for patients. Anyway, this policy calls the attention on the value of using apps in the management of citizens' and patients' health. Considering the implications of the use of health apps not only in terms of effectiveness and safety, but also with respect to - among the others - privacy, usability and interoperability, a multidisciplinary approach is envisaged to assess their impact. HTA could therefore foster a comprehensive evaluation and enable the involvement of health apps users in the assessment process. This workshop will address these topics through a) a description of the current state of the art of health apps regulation; b) an overview of available HTA evaluation of health apps: c) a focus on the available evidence on the impact of health apps on clinical outcomes, with specific reference to the field of mental health. Key messages Health apps can improve population’s health management. International lessons on designing evaluation pathways to reimbursement can offer insights for the establishment of new systems. The evaluation of health apps deserves further development as only effectiveness and technical aspects are commonly evaluated.

Affordability of in vivo gene therapy. Problems and potential solutions

Aim. To review foreign approaches to the evaluation and financing of in vivo gene therapy drugs. Materials and Methods. The first stage of the review included the systematic search for publications of clinical-economic studies (CES) on in vivo gene therapy drugs registered on the international pharmaceutical market. The analysis of the methodology used in the found CESs was performed based on the given criteria. The second and third stages of the review included the search for the information on the approaches and results of the evaluation of in vivo gene therapy drugs by foreign agencies on the assessment of healthcare technologies (AHT) and the implemented approaches to the financing of expensive medical technologies. Results. Totally, four in vivo gene therapy drugs registered on the foreign markets were found: talimogene laherparepvec (Imlygic), voretigene neparvovec (Luxturna), onasemnogene abeparvovec (Zolgensma), and alipogene tiparvove (Glybera). The analysis of the methodology of CES on in vivo gene therapy drugs did not reveal general methodological peculiarities. The analysis of the approaches to the reimbursement for the cost of these four drugs in the European countries showed that there were no special procedures intended for gene drugs. However, in the majority of countries, there are some mechanisms of reimbursement for orphan drugs and the majority of the above-mentioned in vivo gene therapy drugs are orphan. It was revealed that the most perspective methods of payment for in vivo gene therapy drugs were installment payments, risk-sharing agreements, and their combinations. Still, none of these payment methods are implemented in Russia because of the lack of regulatory and legal framework. Conclusion. The design and methodological peculiarities of CES on in vivo gene therapy drugs are similar to orphan drugs (in some countries, there is a special flexible procedure). In the Russian Federation, there is no special procedure of ATH for orphan drugs, which can provide difficulties in the reimbursement for these drugs. Besides, in the Russian Federation, the issue of the implementation of innovative models for drugs provision (risk-sharing, installment payments is acute). The changes in this area would also lead to the optimization of the influence of expensive healthcare technologies on the budget of the system of healthcare.