scholarly journals A comparative analysis of international health technology assessments for novel gene silencing therapies: patisiran and inotersen

2021 ◽  
Vol 8 ◽  
pp. 14-21
Author(s):  
Sergio Iannazzo
Keyword(s):  
Comparative Analysis ◽  
Technology Assessment ◽  
International Health ◽  
Standard Of Care ◽  
Health Technology ◽  
Comprehensive Overview ◽  
Multisystemic Disease ◽  
Assessment Decisions ◽  
The Impact

Objectives: Using the case study of patisiran and inotersen, we conducted a narrative comparative analysis of the health technology assessment (HTA) agency appraisals of these two first-in-class transthyretin gene silencers, which represent exceptional advances in the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis, a rare and multisystemic disease. Despite the impact of each product on the treatment landscape, the majority of HTAs are only considered standard of care as a comparator, resulting in a void of information and limited comprehension of the clinical and pharmacoeconomic differences between the two treatments. Methods: A search was conducted internationally for HTA reports, and only instances where assessment decisions for both treatments were publicly available were included in the present analysis. The HTA reports were analyzed broadly for the assessment of clinical and pharmacoeconomic evidence. Only economic models considering both patisiran and inotersen were included in this analysis. Results: A total of nine agencies with public assessment reports for both treatments were identified. HTA agency assessments for both treatments were essentially positive; however, differences were noted in the final recommendations, place in treatment or reimbursed indications, and in the narrative of the evaluations. Only the Canadian Agency for Drugs and Technologies in Health (CADTH) assessment for patisiran evaluated an economic model comparing the two treatments. Conclusions: The differences summarized in this comparative analysis may provide a more comprehensive overview of the two treatments.

scholarly journals Validity of Surrogate Endpoints and Their Impact on Coverage Recommendations: A Retrospective Analysis across International Health Technology Assessment Agencies

2021 ◽  
pp. 0272989X2199455
Author(s):  
Oriana Ciani ◽  
Bogdan Grigore ◽  
Hedwig Blommestein ◽  
Saskia de Groot ◽  
Meilin Möllenkamp ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
International Health ◽  
Surrogate Endpoint ◽  
Health Technology ◽  
Surrogate Endpoints ◽  
Patient Centered ◽  
Statistical Validation ◽  
Level Of Evidence ◽  
The Impact

Background Surrogate endpoints (i.e., intermediate endpoints intended to predict for patient-centered outcomes) are increasingly common. However, little is known about how surrogate evidence is handled in the context of health technology assessment (HTA). Objectives 1) To map methodologies for the validation of surrogate endpoints and 2) to determine their impact on acceptability of surrogates and coverage decisions made by HTA agencies. Methods We sought HTA reports where evaluation relied on a surrogate from 8 HTA agencies. We extracted data on the methods applied for surrogate validation. We assessed the level of agreement between agencies and fitted mixed-effects logistic regression models to test the impact of validation approaches on the agency’s acceptability of the surrogate endpoint and their coverage recommendation. Results Of the 124 included reports, 61 (49%) discussed the level of evidence to support the relationship between the surrogate and the patient-centered endpoint, 27 (22%) reported a correlation coefficient/association measure, and 40 (32%) quantified the expected effect on the patient-centered outcome. Overall, the surrogate endpoint was deemed acceptable in 49 (40%) reports ( k-coefficient 0.10, P = 0.004). Any consideration of the level of evidence was associated with accepting the surrogate endpoint as valid (odds ratio [OR], 4.60; 95% confidence interval [CI], 1.60–13.18, P = 0.005). However, we did not find strong evidence of an association between accepting the surrogate endpoint and agency coverage recommendation (OR, 0.71; 95% CI, 0.23–2.20; P = 0.55). Conclusions Handling of surrogate endpoint evidence in reports varied greatly across HTA agencies, with inconsistent consideration of the level of evidence and statistical validation. Our findings call for careful reconsideration of the issue of surrogacy and the need for harmonization of practices across international HTA agencies.

Impact of CEDIT recommendations: An example of health technology assessment in a hospital network

2006 ◽  
Vol 22 (2) ◽  
pp. 161-168 ◽  
Author(s):  
Florence Bodeau-Livinec ◽  
Emmanuelle Simon ◽  
Catherine Montagnier-Petrissans ◽  
Marie-Eve Joël ◽  
Elisabeth Féry-Lemonnier
Keyword(s):  
Decision Making ◽  
Technology Assessment ◽  
Health Technology ◽  
Technological Innovations ◽  
Administrative Staff ◽  
Good Match ◽  
French Hospital ◽  
The Impact ◽  
Widespread Interest

Objectives: The objective of this study is to assess the impact of CEDIT (French Committee for the Assessment and Dissemination of Technological Innovations) recommendations on the introduction of technological innovations within the AP-HP (Assistance Publique–Hôpitaux de Paris), the French hospital network to which this body is attached.Methods: In 2002, a study based on semidirective interviews of fourteen people affected by these recommendations and a case study relating to thirteen recommendations issued between 1995 and 1998 were conducted.Results: The CEDIT is very scientifically reputable among interviewees. There is generally widespread interest for the recommendations. They are used as decision-making tools by administrative staff and as negotiating instruments by doctors in their dealings with management. Based on the case study, ten of thirteen recommendations had an impact on the introduction of the technology in health establishments. One recommendation appears not to have had an impact. Furthermore, the impact of two technologies was impossible to assess.Conclusions: This study highlights the significant impact of recommendations arising from a structure that is attached to a hospital network and the good match between CEDIT's objectives and its assignments.

Integration of health technology assessment recommendations into organizational and clinical practice: A case study in Catalonia

2006 ◽  
Vol 22 (2) ◽  
pp. 169-176 ◽  
Author(s):  
Marie-Pierre Gagnon ◽  
Emília Sánchez ◽  
Joan M. V. Pons
Keyword(s):  
Decision Making ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Complex Dynamics ◽  
Scientific Evidence ◽  
Health Technology ◽  
Health Technologies ◽  
The Impact ◽  
Support Decision Making

Objectives:Evaluating the impact of recommendations based upon health technology assessment (HTA) represents a challenge for both HTA agencies and healthcare policy makers. This research sought to understand factors affecting the uptake of HTA recommendations to support decision making with respect to the introduction of three health technologies.Methods:Using a multidimensional framework, based upon a combination of theoretical models, a case study was conducted. A total of twenty-eight semistructured interviews were done with physicians from fifteen hospitals and other stakeholders in Catalonia. Interview content was analyzed iteratively and classified according to theoretical dimensions and contextual factors.Results:At the sociopolitical level, factors related to the organization and financing of the health system were found to affect the utilization of HTA recommendations. At the healthcare organization level, existing collaborations between the hospital and the HTA agency favored the integration of recommendations into practices. Formalism in the organization also influenced the utilization of HTA recommendations. At the professional level, the high degree of autonomy of specialists, the importance of peers and collegial control, and the definition of professional roles and responsibilities influenced physicians' willingness to integrate HTA recommendations into their practice.Conclusions:This study offers a comprehensive framework to understand the complex dynamics that affect adoption of health technologies in organizational and professional practices. The findings suggest some avenues to promote the integration of HTA recommendations into practices and, thus, increase the utilization of scientific evidence to support decision making in health care.

scholarly journals MENTORING A HEALTH TECHNOLOGY ASSESSMENT INITIATIVE IN KAZAKHSTAN

2014 ◽  
Vol 30 (2) ◽  
pp. 147-152 ◽  
Author(s):  
Sergei Muratov ◽  
David Hailey ◽  
Vicki Foerster ◽  
Bruce Brady ◽  
Don Juzwishin ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
International Health ◽  
Policy Decision ◽  
Health Technology ◽  
Organization Culture ◽  
Train The Trainer ◽  
Staff Members ◽  
Health Policy Decision ◽  
The Republic

Objectives: The aim of this study was to assist in the development of a health technology assessment (HTA) program for the Ministry of Health (MOH) of the Republic of KazakhstanMethods: Mentoring of an initial HTA program in Kazakhstan was provided by the Canadian Society for International Health (CSIH) by means of a partnership with the Kazakhstan MOH. HTA materials, courses, and one-on-one support for the preparation of a series of initial HTA reports by MOH HTA staff were provided by a seven-member CSIH team over a 2.5-year project.Results: Guidance documents on HTA and institutional strengthening were prepared in response to an extensive set of deliverables developed by the MOH and the World Bank. Introductory and train-the-trainer workshops in HTA and economic evaluation were provided for MOH staff members, experts from Kazakhstan research institutes and physicians. Five short HTA reports were successfully developed by staff in the Ministry's HTA Unit with assistance from the CSIH team. Challenges that may be relevant to other emerging HTA programs included lack of familiarity with some essential underlying concepts, organization culture, and limited time for MOH staff to do HTA work.Conclusions: The project helped to define the need for HTA and mentored MOH staff in taking the first steps to establish a program to support health policy decision making in Kazakhstan. This experience offers practical lessons for other emerging HTA programs, although these should be tailored to the specific context.

CAPACITY BUILDING IN AGENCIES FOR EFFICIENT AND EFFECTIVE HEALTH TECHNOLOGY ASSESSMENT

2016 ◽  
Vol 32 (4) ◽  
pp. 292-299 ◽  
Author(s):  
Debjani Mueller ◽  
Iñaki Gutiérrez-Ibarluzea ◽  
Tara Schuller ◽  
Marco Chiumente ◽  
Jeonghoon Ahn ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Capacity Building ◽  
Technology Assessment ◽  
Health Technology ◽  
Group Discussions ◽  
Middle Income ◽  
Working Together ◽  
Effective Decision ◽  
Effective Decision Making ◽  
The Impact

Objectives: Health technology assessment (HTA) yields information that can be ideally used to address deficiencies in health systems and to create a wider understanding of the impact of different policy considerations around technology reimbursement and use. The structure of HTA programs varies across different jurisdictions according to decision-maker needs. Moreover, conducting HTA requires specialized skills. Effective decision making should include multiple criteria (medical, economic, technical, ethical, social, legal, and cultural) and requires multi-disciplinary teams of experts working together to produce these assessments. A workshop explored the multi-disciplinary skills and competencies required to build an effective and efficient HTA team, with a focus on low- and middle-income settings.Methods: This proceeding summarizes main points from a workshop on capacity building, drawing on presentations and group discussions among attendees including different points of view.Results and Conclusions: The workshop and thus this study would have benefited from a larger variety of stakeholders. Therefore, the conclusions arising from the workshop are not the opinion of a representative sample of HTA professionals. Nonetheless, organizations and speakers were carefully selected to provide a valuable approach to this theme. Thus, these proceedings highlight some of the gaps and needs in the education and training programs offered worldwide and calls for further investigation.

scholarly journals History of health technology assessment in Belgium

2009 ◽  
Vol 25 (S1) ◽  
pp. 82-87 ◽  
Author(s):  
Irina Cleemput ◽  
Philippe Van Wilder
Keyword(s):  
Health Care ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Good Clinical Practice ◽  
Health Technology ◽  
Services Research ◽  
Drug Reimbursement ◽  
History Of ◽  
The Impact ◽  
Further Development

Objectives: This paper gives an overview of health technology assessment (HTA) in Belgium.Methods: The information included in the overview is based on legal documents and publicly available year reports of the Belgian Health Care Knowledge Centre (KCE).Results: Belgium has a relatively young history in HTA. The principle of evidence-based medicine (EBM) was introduced in the drug reimbursement procedure in 2001, with the establishment of the Drug Reimbursement Committee (DRC). The DRC assesses the efficacy, safety, convenience, applicability, and effectiveness of a drug relative to existing treatment alternatives. For some drugs, relative cost-effectiveness is also evaluated. The activities of the DRC can, therefore, be considered to be the first official HTA activities in Belgium. Later, in 2003, KCE was established. Its mission was to perform policy preparing research in the healthcare and health insurance sector and to give advice to policy makers about how they can obtain an efficient allocation of limited healthcare resources that optimizes the quality and accessibility of health care. This broad mission has been operationalized by activities in three domains of research: HTA, health services research, and good clinical practice. KCE is independent from the policy maker. Its HTAs contain policy recommendations that may inform policy decisions but are not binding.Conclusions: Although the Belgian history of HTA is relatively short, its foundations are strong and the impact of HTA increasing. Nevertheless KCE has many challenges for the future, including continued quality assurance, further development of international collaboration, and further development of methodological guidance for HTA.

scholarly journals WORK-LIFE BALANCE: A CASE STUDY ANALYSIS ON GOOGLE AND A COMPARATIVE STUDY OF PRACTICES FOLLOWED IN NORWAY AND INDIA

2020 ◽  
Vol 5 (8) ◽  
Author(s):  
Deepa Singh
Keyword(s):  
Comparative Analysis ◽  
Work Life ◽  
Work Life Balance ◽  
Survey Method ◽  
Study Analysis ◽  
Life Balance ◽  
Case Study Analysis ◽  
Secondary Sources ◽  
The Impact

The concept of work life balance emerged during the Industrial Revolution. In those days Industrialists agreed upon labour union’s demand of ‘One day off’. Later on, this is converted into ‘two day off’ which is followed in all the western countries. Grady et al (2008) stated that the term’ work-life balance’ is more comprehensive and includes “family, community, recreation and personal time.” The objectives of the research are to study the impact of practices followed in Norway and India on work-life balance, to gain insight on Google company’s work-life balance practices, to identify and suggest vital work-life balance practices that can be applied in Indian context. The research is descriptive in nature. The data has been collected mainly from secondary sources – research papers, annual report of companies (employee engagement) and articles. The tools used for achieving objectives and analysing are comparison method, case study analysis and survey method. The comparative and case study analysis shows that the education system, job security, income inequality are some of the factors which hinder the work-life balance in India. Flexi hours, time for creativity, family participation in workplace boost the enthusiasm and morale of the employees. The research is based on secondary sources. The primary data would more effectively convey the problems faced in work life balance. The work-life balance practices followed by only one company is taken into the study due to time constraint. The comparative analysis of work-life balance practices of big films would give better insight and would help in deriving dynamic solution. The sector centred, institution centred or gender centred study will give broader overview of the work-life balance. The search acts a base for comparative analysis.

TeCHO+ program in Gujarat: a protocol for health technology assessment

2020 ◽  
Vol 6 (4) ◽  
pp. 209-214
Author(s):  
Somen Saha ◽  
Priya Kotwani ◽  
Apurvakumar Pandya ◽  
Deepak Saxena ◽  
Tapasvi Puwar ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Community Health ◽  
Technology Assessment ◽  
Tracking System ◽  
Newborn Care ◽  
Health Technology ◽  
The State ◽  
Family Welfare ◽  
The Impact

The Health and Family Welfare Department, Government of Gujarat, is implementing a program named Technology for Community Health Operation or TeCHO+ addressing state’s priority health issues. This program envisages replacing the existing mother and child tracking system or e-Mamta application in the state. This program is based on ImTeCHO—Innovative Mobile Technology for Community Health Operations—which was piloted in Jhagadia, Bharuch district of Gujarat in 2013. The program showed improvements not only in terms of coverage of maternal and newborn care packages averting malnutrition but also was cost-effective. This paper details the protocol for health technology assessment to assess the impact of TeCHO+ program on data quality, improvement in service delivery coverage, reduction in morbidity and mortality as well as assess the cost-effectiveness. The study will be conducted in five districts of the state. A mixed-method approach will be adopted. Data will be validated in a phased manner over a period of 3 years along with an assessment of key outcome indicators. Additionally, key informant interviews will be conducted and cost data will be gathered to perform cost-effectiveness analysis. The study will inform policymakers about the impact of TeCHO+ program on quality, access and cost-effectiveness of healthcare services.

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