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.

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.

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.

VP08 Real-World Data Use In Health Technology Assessments: A Comparison Of Five Health Technology Assessment Agencies

2017 ◽  
Vol 33 (S1) ◽  
pp. 149-150
Author(s):  
Amr Makady ◽  
Ard van Veelen ◽  
Anthonius de Boer ◽  
Hans Hillege ◽  
Olaf Klunger ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Relative Effectiveness ◽  
Internal Validity ◽  
Health Technology ◽  
Real World Data ◽  
World Data ◽  
The Impact

INTRODUCTION:Reimbursement decisions are usually based on evidence from randomized controlled trials (RCT) with high internal validity but lower external validity. Real-World Data (RWD) may provide complimentary evidence for relative effectiveness assessments (REA's) and cost-effectiveness assessments (CEA's) of treatments. This study explores to which extent RWD is incorporated in REA's and CEA's of drugs used to treat metastatic melanoma (MM) by five Health Technology Assessment (HTA) agencies.METHODS:Dossiers for MM drugs published between 1 January 2011 and 31 December 2016 were retrieved for HTA agencies in five countries: the United Kingdom (NICE), Scotland (SMC), France (HAS), Germany (IQWiG) and the Netherlands (ZIN). A standardized data-extraction form was used to extract data on RWD mentioned in the assessment and its impact on appraisal (for example, positive, negative, neutral or unknown) for both REA and CEA.RESULTS:In total, fourty-nine dossiers were retrieved: NICE = 10, SMC = 13, IQWiG = 16, HAS = 8 and ZIN = 2. Nine dossiers (18.4 percent) included RWD in REA's for several parameters: to describe effectiveness (n = 5) and/or the safety (n = 2) of the drug, and/or the prevalence of MM (n = 4). CEA's were included in 25/49 dossiers (IQWiG and HAS did not perform CEA's). Of the twenty-five CEA's, twenty (80 percent) included RWD to extrapolate long-term effectiveness (n = 19), and/or identify costs associated with treatments (n = 7). When RWD was included in REA's (n = 9), its impact on the appraisal was negative (n = 4), neutral (n = 2), unknown (n = 1) or was not discussed in the appraisal (n = 2). When RWD was included in CEA's (n = 11), its impact on the appraisal varied between positive (n = 2), negative (n = 5) and unknown (n = 4).CONCLUSIONS:Generally, RWD is more often included in CEA's than REA's (80 percent versus 18.4 percent, respectively). When included, RWD was mostly used to describe the effectiveness of the drug (REA) or to predict long-term effectiveness (CEA). The impact of RWD on the appraisal varied greatly within both REA's and CEA's.

OP135 Confirmatory Versus Explorative Endpoints In Drug Approval Versus Health Technology Assessment

2017 ◽  
Vol 33 (S1) ◽  
pp. 63-64
Author(s):  
Ines Niehaus ◽  
Charalabos-Markos Dintsios
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Drug Approval ◽  
Health Technology ◽  
Benefit Assessment ◽  
Benefit Ratio ◽  
Early Benefit Assessment ◽  
The Impact ◽  
Early Benefit

INTRODUCTION:The early benefit assessment of drugs in Germany and their preceded market authorization pursue different objectives, resulting in divergent decision-making strategies. This is reflected inter alia by the diverse inclusion of confirmatory endpoints within the assessments of oncological drugs. The pharmaceutical manufacturers are facing the challenge of meeting the requirements for both evaluation processes by the available evidence and avoiding hereby negative early benefit assessments. This is mainly due to the concept of mutually relevant clinical trials.METHODS:Identification and gathering of the endpoints is based on a specifically developed guide. The extracted data from the documents of completed assessments up to July 2015 are used to estimate both separately and together the impact of explorative in relation to confirmatory endpoints on the drug approval and early benefit assessment, by contrasting the European Medicines Agency's risk-benefit-ratio and the benefit-harm-balancing of the national Health Technology Assessment (HTA) jurisdiction.RESULTS:Twenty-one of fourty-one studies’ oncological assessments could be included in the endpoint analysis. From a procedural point of view both the drug approval and the early benefit assessment seem to be not confirmatory since they include explorative endpoints as well. Yet, drug approval is in terms of quality of endpoints more confirmatory than early benefit assessment since it contains a higher proportion of primary endpoints. The latter implies only in 67 percent of the assessments a primary endpoint to be relevant for the benefit-harm-balancing. Moreover, explorative mortality endpoints reached the highest agreement and explorative endpoints capturing health-related quality of life no agreement, referring to the mutual relevance of endpoints for the risk-benefit-ratio and the benefit-harm-balancing.CONCLUSIONS:The missing information transparency of the assessment reports compared to the information offered within the early benefit assessment makes an assignment of endpoints with respect to the mutually relevant clinical trial sometimes troublesome. To warrant, in the long run, a broader confirmatory basis for decisions in health care supported by HTA, a closer inter-institutional cooperation of approval authorities and German HTA jurisdictions seems favorable.

VP60 The Importance Of Patient Organizations Involvement In Health Technology Assessment

2017 ◽  
Vol 33 (S1) ◽  
pp. 176-176
Author(s):  
Kathi Apostolidis ◽  
Lydia Makaroff ◽  
Francesco De Lorenzo ◽  
Francesco Florindi
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Patient Preferences ◽  
Health Technology ◽  
White Paper ◽  
Public Consultation ◽  
Policy Position ◽  
List Type ◽  
Patient Organizations ◽  
The Impact

INTRODUCTION:In Europe, the work done by the European Commission and the European Network for Health Technology Assessment (EUnetHTA) has consolidated the role of Health Technology Assessment (HTA), and promises to better harmonize its impact across European Union (EU) countries. However, more work is needed to improve patient involvement in assessing new health technology, and in developing research priorities.METHODS:The European Cancer Patient Coalition (ECPC) developed a model for engagement of patients in HTA based on the experience from: •ECPC's ‘Value of Innovation in Oncology’ White Paper, which includes input from ECPC's membership•ECPC's leading role in the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) study, funded by the Innovative Medicines Initiative, to develop guidelines on how patient-preference studies should be performed throughout the development of new medical treatments.RESULTS:The ECPC ‘Value of Innovation in Oncology’ White Paper was launched in 2017. The paper provides ECPC's policy position on key obstacles to equitable access to meaningful innovation. The paper recommends the establishment of an EU-wide HTA body to reduce delays and variations in access and to avoid duplication of effort by individual Member States. The paper also recommends that patients should be formally and routinely included in HTA policy and operations at EU and at national levels. These recommendations were also submitted to the European Commission's public consultation on strengthening EU cooperation on HTA.Through its work in PREFER, ECPC is helping to improve how patient preferences are measured and valued to capture the impact of health technology on patients daily life. Patient preferences are concerned with measuring how patients value components such as treatment end points, route of administration, treatment duration, treatment frequency, frequency of side-effects, price, and quality of life.CONCLUSIONS:Patient organization involvement in HTA is vital. Patient organizations offer unique insights, experiences, identify unmet needs, and can help to produce practical recommendations

scholarly journals The development of health technology assessment in Brazil

2009 ◽  
Vol 25 (S1) ◽  
pp. 255-259 ◽  
Author(s):  
David Banta ◽  
Rosimary T. Almeida
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Descriptive Analysis ◽  
Institutional Development ◽  
Health Technology ◽  
National Agency ◽  
Private Companies ◽  
Considerable Impact ◽  
The Impact ◽  
Growing Network

Objectives: The aim of this study was to describe the developing health technology assessment (HTA) activities in Brazil, both historically and in the present day.Methods: This report is a descriptive analysis based on personal experiences of the authors and on selected literature.Results: Interest in HTA in Brazil began in the mid-1980s. Several seminars and consultations were held, often with invited foreign participants. A cadre of people with knowledge and expertise in HTA was gradually developed. In 2003, several policies were developed by the federal government of Brazil to encourage HTA and base clinical, management, and policy decisions on HTA. During the past 5 years, institutional development has been rapid in government, private companies (mainly prepaid health plans), academia, and research institutes. Further policy changes are needed to maximize the impact of these developments. Nevertheless, although the growing network of HTA programs will have a considerable impact on Brazilian health care, further institutional development could stimulate this change.Conclusions: It would be desirable if the Federal Ministry of Health of Brazil were to proceed to develop a national agency for HTA.

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