Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines

Introduction:Bridging gaps between registry-holders, Health Technology Assessment (HTA) producers and users is one of the aims of the European Network for HTA (EUnetHTA) Joint Action 3. In this context, a post-launch evidence generation tool is being developed, including a quality standards tool for registries in HTA. The standards tool for registries in HTA will enable, among others, registry owners to consistently collect high quality registry data, and HTA agencies to use proper registry data collected by others as evidence for their assessments. The objective is to present the first draft version of the tool structure, which is going to be piloted during the forthcoming months.Methods:A review and description of the currently available first version (November 2017) sections, items and criteria for HTA studies.Results:The tool is divided in three sections; “Methodological Information”, “Essential Standards” and “Additional Requirements”. The first section enables users to analyze not only the ability of the registry to answer to research questions but also to check the registry transparency. The second section encloses the essential elements of good practice and evidence quality (therefore all of them must be met before an HTA report can use the registry data). Finally, the third section includes elements of good practice and evidence quality useful to consider in planning and evaluating registries for specific purposes. Although suggestions are defined, the third section item requirements could depend on the individual HTA agency perspectives and needs.Conclusions:There is a clear growing availability and requirement for real world data for health technology assessment. A piloted and robust registry standards tool for HTA can provide a relevant basis to improve both the evidence generation but also to make more trustful and excellent evaluations.

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Web-based toolkit to facilitate European collaboration on evidence generation on promising health technologies

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462309990705 ◽

2009 ◽

Vol 25 (S2) ◽

pp. 68-74 ◽

Cited By ~ 5

Author(s):

Fabienne Quentin ◽

Cédric Carbonneil ◽

Céline Moty-Monnereau ◽

Elena Berti ◽

Wim Goettsch ◽

...

Keyword(s):

Health Technology Assessment ◽

Information Sharing ◽

Technology Assessment ◽

Web Site ◽

Health Technology ◽

Web Based ◽

Health Technologies ◽

European Collaboration ◽

Evidence Generation ◽

The Web

Background: Several countries have developed policy frameworks allowing timely access to promising health technologies on the condition that additional evidence is generated. However, an important barrier to evidence generation is the lack of structured collaboration among health technology assessment (HTA) agencies.Objectives: One of the aims of Work Package 7 (WP7) of the European network for Health Technology Assessment (EUnetHTA) Project was to determine the types of structured collaboration that could facilitate evidence generation and to develop a Web-based toolkit to support such collaboration.Methods: Collaboration modalities were defined by all WP7 Partners. Initial emphasis was on information sharing. Standardized forms for information sharing were developed and tested. An information technology development phase followed with the creation of the Web-based toolkit (Web site).Results: Three levels of collaboration were agreed on: (i) sharing information, (ii) coordinated action, and (iii) joint action. The Web site allows access to structured and standardized forms for requesting information, posting information in response to a request, and posting information spontaneously. An online database contains all of the information requested or posted. Pilot tests on twenty-one promising technologies were satisfactory.Conclusions: This new Web site for sharing information on evidence generation should help countries reach robust decisions on the timely adoption of promising health technologies. It will only become fully operational if EUnetHTA Partners supply relevant, accurate, and updated information, and regularly use the Web site.

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Going beyond routine data collection of clinical trials funded by the health technology assessment programme

Trials ◽

10.1186/1745-6215-16-s2-p42 ◽

2015 ◽

Vol 16 (S2) ◽

Author(s):

James Raftery ◽

Amanda Young ◽

Helen Buxton

Keyword(s):

Clinical Trials ◽

Data Collection ◽

Health Technology Assessment ◽

Technology Assessment ◽

Routine Data ◽

Health Technology ◽

Assessment Programme ◽

Health Technology Assessment Programme ◽

Routine Data Collection

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Primary data collection in health technology assessment

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462307051537 ◽

2007 ◽

Vol 23 (1) ◽

pp. 24-29 ◽

Cited By ~ 7

Author(s):

Michelle L. McIsaac ◽

Ron Goeree ◽

James M. Brophy

Keyword(s):

Data Collection ◽

Health Technology Assessment ◽

Technology Assessment ◽

Health Technology ◽

Primary Data ◽

Evidence Based ◽

Information Need ◽

Methodological Issues ◽

Additional Information ◽

Evidence Based Decision Making

This study discusses the value of primary data collection as part of health technology assessment (HTA). Primary data collection can help reduce uncertainty in HTA and better inform evidence-based decision making. However, methodological issues such as choosing appropriate study design and practical concerns such as the value of collecting additional information need to be addressed. The authors emphasize the conditions required for successful primary data collection in HTA: experienced researchers, sufficient funding, and coordination among stakeholders, government, and researchers. The authors conclude that, under specific conditions, primary data collection is a worthwhile endeavor in the HTA process.

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PP385 Using Common Data Models And Data Networks For Evidence Generation In Health Technology Assessment

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462320001683 ◽

2020 ◽

Vol 36 (S1) ◽

pp. 32-32

Author(s):

Seamus Kent ◽

Jacoline Bouvy

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Healthcare Delivery ◽

Health Technology ◽

Data Models ◽

Medical Decision ◽

Data Networks ◽

Common Data Model ◽

Analytical Tools ◽

Evidence Generation

IntroductionDifferences between healthcare datasets in structure, content, and coding systems are widely recognized as significant barriers to generating robust evidence for regulatory and medical decision making. As a result, there is a growing interest in using common data models embedded within large data networks. By standardizing the structure, contents, and semantics of disparate healthcare databases, common data models like the Observational and Medical Outcomes Partnerships common data model (OMOP-CDM) enable multidatabase studies to be undertaken at speed and in a transparent way. To date, little attention has been given to their potential role in health technology assessment (HTA).MethodsWe identify the uses of observational data in generating evidence in HTA, some common analytical challenges faced in their estimation, and the infrastructural, technical, and data reusability constraints that limit its wider use. We discuss where and how the OMOP-CDM could overcome these barriers in relation to different types of evidence requirements.ResultsThe OMOP-CDM increases the interoperability of otherwise disparate datasets, allowing reliable evidence to be generated from multidatabase studies at speed and transparently. The current analytical tools are best suited for clinical characterization and population-level effect estimation. Further developments to these tools are required to support analyses common in HTA like parametric survival modeling. Differences in costing methods as well as the structure of healthcare delivery between countries may limit the feasibility and value of standardization.ConclusionsThe OMOP-CDM has the potential to support reliable and timely evidence generation in HTA. The analytical tools should be further developed to support common HTA use cases.

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PP32 Joint Early Dialogs Between Medical Device Regulation and Health Technology Assessment

International Journal of Technology Assessment in Health Care ◽

10.1017/s026646231900196x ◽

2019 ◽

Vol 35 (S1) ◽

pp. 43-43

Author(s):

Petra Schnell-Inderst ◽

Claudia Wild

Keyword(s):

High Risk ◽

Health Technology Assessment ◽

Medical Device ◽

Technology Assessment ◽

Health Technology ◽

European Medicines Agency ◽

Scientific Advice ◽

Market Approval ◽

Evidence Generation ◽

Medical Device Regulation

IntroductionIn Europe, the new Medical Device Regulation (MDR) and In Vitro Diagnostics Regulation (IVDR) that entered into force 2017 will have to be applied until 2020 and 2022, respectively. Under the old regulation, there was a large gap between evidence requirements for market approval and market access for high risk (class IIb and III) medical devices (MD). The MDR/IVDR will require appropriate clinical investigations for these MD classes. Despite the different purpose of market approval and surveillance and reimbursement decisions, there are possible synergies with regard to evidence generation, for example, design of pivotal trials and post-launch evidence generation with observational data. In the MDR, early scientific advice can be provided by expert panels of the European Commission if requested by MD developers. For medicinal products, the European network for Health Technology Assessment (EUnetHTA) has established joint early dialogs (JED) of HTA agencies with the European Medicines Agency and manufacturers. A similar approach might be possible with the Medical Device Coordination Group (MDCG). The objective was to explore possible synergies for JED with the MDCG and EUnetHTA.MethodsIn 2018, EUnetHTA established a task force for HTA and MDR/IVDR. A workshop, which will explore possible synergies and activities on JED as well as the viewpoints of stakeholders will be held in May 2019. Participants will be Directorate-Generals GROW (Internal Market, Industry, Entrepreneurship and SME) and SANTE (Health and Food Safety), EUnetHTA members assessing MD, representatives of national competent authorities, Team Notified Bodies, MedTech Europe, patient representatives and academia.ResultsA report on the presentations, the results of the discussion, and next steps in a possible collaboration will be presented.ConclusionsJoint early scientific advice to manufacturers on the European level for evidence generation by HTA agencies and the MDCG has the potential to streamline evidence generation in the life cycle of high risk MD.

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