OP07 Real World Evidence: How Can It Improve Health Technology Assessment?

Author(s):  
John Gillespie ◽  
Sebnem Erdol ◽  
Chris Foteff ◽  
Liesl Strachan

Introduction:Health Technology Assessment (HTA) considers the question of whether evaluated technologies are cost-effective in real world settings. As observed in HTA conducted by the Australian Medical Services Advisory Committee (MSAC), questions regarding the validity of data inputs to economic analyses that reflect real-world practice is a common reason for uncertainty on the cost effectiveness of new technologies. In addition to resource use and costs, there may be other uncertainties regarding the eligible patient population, patient management pathways and comparator selection. Our objective in this study was to present case studies from Australia where real world linked datasets could be better utilized to inform HTA conducted by the MSAC.Methods:For selected therapy areas, assessment reports and public summary documents of HTA conducted by the MSAC published between January 2015 and November 2017 were reviewed. Our analysis identified HTAs where uncertainties around the inputs for health economic evaluations, as well as uncertainties in defining eligible patient numbers or current patient pathways of care were shown to exist. We then explored whether these uncertainties could have been addressed through real world linked datasets.Results:Our preliminary investigations identified two assessments: MSAC assessment of capsule endoscopy and transcatheter aortic valve implantation - where availability of real world linked data could have addressed uncertainties around the inputs required for the health economic evaluations.Conclusions:Australia has a range of real world datasets with the potential to be used to inform HTA conducted by the MSAC. This can only be achieved if the datasets could be better linked and accessible for use by key stakeholders in the MSAC HTA process (e.g. industry, clinician, patient societies). Use of these data sets in HTA will enable timelier patient access to cost-effective technologies and more effective implementation and review of technologies after adoption into clinical practice.

2020 ◽  
Vol 36 (5) ◽  
pp. 474-480
Author(s):  
Jing Lou ◽  
Sarin KC ◽  
Kai Yee Toh ◽  
Saudamini Dabak ◽  
Amanda Adler ◽  
...  

There is growing interest globally in using real-world data (RWD) and real-world evidence (RWE) for health technology assessment (HTA). Optimal collection, analysis, and use of RWD/RWE to inform HTA requires a conceptual framework to standardize processes and ensure consistency. However, such framework is currently lacking in Asia, a region that is likely to benefit from RWD/RWE for at least two reasons. First, there is often limited Asian representation in clinical trials unless specifically conducted in Asian populations, and RWD may help to fill the evidence gap. Second, in a few Asian health systems, reimbursement decisions are not made at market entry; thus, allowing RWD/RWE to be collected to give more certainty about the effectiveness of technologies in the local setting and inform their appropriate use. Furthermore, an alignment of RWD/RWE policies across Asia would equip decision makers with context-relevant evidence, and improve timely patient access to new technologies. Using data collected from eleven health systems in Asia, this paper provides a review of the current landscape of RWD/RWE in Asia to inform HTA and explores a way forward to align policies within the region. This paper concludes with a proposal to establish an international collaboration among academics and HTA agencies in the region: the REAL World Data In ASia for HEalth Technology Assessment in Reimbursement (REALISE) working group, which seeks to develop a non-binding guidance document on the use of RWD/RWE to inform HTA for decision making in Asia.


Author(s):  
Orna Tal ◽  
Inbal Tal

Introduction:Health technology assessment (HTA) is scientifically incorporating the demand, effectiveness and expedience of adopting innovative technologies, supported by evidence-based medicine. The rapid development of innovations led to an accelerated need to comprehend their benefit and satisfaction through actual utilization. ‘Real World’ Experience (RWE) in action is a source for collecting information through practice that has not yet been formally published. The target is to identify challenges and barriers in adoption, considerations on significant investment and local adjustment. This study aimed to assess the benefit of RWE as a tool for gathering information understanding and evaluating new technologies as candidates for public funding.Methods:Twelve new technologies were assessed by 36 technology-assessors from the Ministry of Health (MOH) and the hospital, and were ranked using two scales: (i) the contribution of using RWE to benefit the assessment mechanism, and (ii) the perceptual “value” of the technology.Results:Analyzing the contribution of RWE to HTA, 67 percent of the technologies reviewed were recommended for adoption. Low RWE ranking correlated with a decision to reject technology adoption. The most important “value” among all assessors on RWE was “innovation”, and among hospital assessors, the parameter “local adjustment” was cardinal. MOH assessors highlighted RWE to increase knowledge of regulators to encourage adoption. The aspect relating to economic burden received only medium ranking.Conclusions:RWE provides an essential contribution to HTA throughout all stages in the life of the technology, especially at early phases. It is an important methodology to gather evidence from the ongoing experience of clinicians, increasing awareness towards promising innovations and amplifying the confidence of decision-making. In this new era of HTA, new concepts arise on the horizon: multi-dimensional evaluations. Expert opinion and RWE are paving the way towards better understanding of beneficial technologies, even in the absence of formal published evidence.


2021 ◽  
Author(s):  
Alina Weise ◽  
Roland Brian Büchter ◽  
Dawid Pieper ◽  
Tim Mathes

Abstract Objective: For assessing cost-effectiveness, Health Technology Assessment (HTA) organisations may use primary economic evaluations (P-HEs) or Systematic Reviews of Health Economic evaluations (SR-HEs). The latter pose the question whether the results from existing P-HEs are transferable across decision contexts (e.g. jurisdictions). A particularly pertinent issue is the high variability of costs and resource needs across jurisdictions. Our objective was to review the methods documents of HTA organisations and compare their recommendations on considering transferability in SR-HE. Methods: We systematically hand searched the webpages of 158 HTA organisations for relevant methods documents from January to March 2019. Two independent reviewers performed searches and selected documents according to pre-defined criteria. One reviewer extracted data in standardised and piloted tables and a second reviewer checked them for accuracy. We synthesized data using tabulations and in a narrative way. Results: We identified 155 potentially relevant documents from 63 HTA organisations. Of these, 7 were included in the synthesis. The included organisations have different aims when preparing a SR-HE (e.g. to determine the need for conducting their own P-HE). The recommendations vary regarding the underlying terminology (e.g. transferability/generalisability), the assessment approaches (e.g. structure), the assessment criteria and the integration in the review process.Conclusion: Only few HTA organisations address the assessment of transferability in their methodological recommendations for SR-HEs. Transferability considerations are related to different purposes. The assessment concepts and criteria are heterogeneous. Developing standards to consider transferability in SR-HEs is desirable.


2016 ◽  
Vol 7 (3) ◽  
Author(s):  
Paul C Langley

The European Network for Health Technology Assessment (EUnetHTA) guidelines for health economic evaluations represent a consolidated view of non-binding recommendations for assessments of the relative effectiveness of pharmaceuticals or other health technologies. EUnetHTA views itself as the scientific and technological backbone of the development of health technology assessment in the European Union and among its member states and other partners. Unfortunately, the standards for health technology assessment proposed by EUnetHTA do not meet the standards of normal science. They do not support credible claims for the clinical and comparative cost-effectiveness of pharmaceuticals. In rejecting the standards of normal science the guidelines put to one side the opportunity not only to re-assess and replicate clinical and cost-effectiveness claims but to provide meaningful feedback on claims assessment to health care decision makers. The purpose of this review is to make the case that, in failing to support standards for experimentation, EUnetHTA is advocating its partners support the creation of modeled or simulated imaginary or false worlds. While EUnetHTA is not alone in recommending the construction of imaginary worlds to support formulary decisions, there is still the opportunity to revisit these recommendations and decide whether or not to encourage a scientifically rigorous approach to health technology assessments - to abandon a commitment to intelligent design in favor of natural selection.  Conflict of Interest None   Type: Commentary  


2017 ◽  
Vol 33 (S1) ◽  
pp. 223-224
Author(s):  
Michela Tinelli ◽  
Andrea Manfrin ◽  
Martin Knapp ◽  
Americo Cicchetti ◽  
Andrea Mandelli

INTRODUCTION:There is a lack of Health-Technology-Assessment (HTA) tools in pharmacy practice and the collection of real-world-evidence (RWE) in community pharmacy to populate longer-term-disease-progression-modelling (1). This project is looking at the development and application of a novel Patient-Reported-Outcome- Measure (PROM) in community pharmacy that can enable: the evaluation of the quality of care delivered from the patient perspective in terms of economic impact, patient health outcomes and ‘utilities’; the collection of RWE and evaluate long-term effect of care; to provide different stakeholders with unique evidence-based information that help formulate health policies in community pharmacy that are safe, effective, patient-focused and cost-effective, balancing access to innovation and cost containment.METHODS:Evidence from the Italian-Medicine-Use-Review (I-MUR) trial (2) showed that the I-MUR intervention provided by community pharmacists to asthma patients is effective, cost-saving and cost-effective (3). The trial allowed to model a framework (I-MUR-HTA) that would enable to routinely deliver the intervention, but also collect and analyse PROM data on its clinical-effectiveness, quality-of-life and cost-effectiveness. I-MUR-HTA was discussed within three expert-panel discussions including policy-makers, commissioners, academics, healthcare-professionals and patient-representatives in Italy, United Kingdom and Europe. Current plan include testing the use of the tool in the real world environment.RESULTS:Evidence collected from the panel discussions confirmed that I-MUR-HTA evidence-based information is relevant to meet current National-Health-Care-System plans and this is what is needed to support the evaluation of innovative effective and cost-effective health policies and promote their implementation across nations. Current Italian law on pharmacy services provides the appropriate institutional framework to regulate the introduction of I-MUR-HTA across the territory. Its implementation is underway and a real-world pilot is planned to take place in Italy.CONCLUSIONS:I-MUR-HTA appears to be an innovative tool to promote active patient involvement into policy-decision-making and pharmacy-service.


2021 ◽  
Author(s):  
Piyush Kumar

The covid-19 pandemic had disclosed the truth that health care resources are limited and the medical interventions applied to deal with the pandemic such as ventilators are not available to most of the population and there are needs of suitable cheaper alternative to be assessed to be cost effective than the available interventions to reduce the economic burden as well. Only looking at the benefits of intervention avoiding the cost effective analysis will do harm definitely in short as well as long run as the economic burden must need to be addressed in order to make an intervention reach from top to bottom strata of the population. The health technology assessment will help policy makers to achieve the goal of primary health care as well as universal health coverage by making the healthcare system affordable, accessible , available to everyone particularly poor populations. A simple question is: are the ventilators available to all who required it? Of course the answer is no. Limiting the medical interventions only to rich people is not going to solve the problems of nations; the interventions should be available, accessible, and affordable to everyone who needs it. This is the only way and for this we must have HTA (health technology assessment) added with economic evaluations of medical interventions to search alternatives which can be better cost effective than existing one. In this article I am discussing significance of HTA and economic evaluations of medical interventions in Low & Middle Income countries.


Author(s):  
Hannah Sievers ◽  
Angelika Joos ◽  
Mickaël Hiligsmann

Abstract Objective This study aims to assess stakeholder perceptions on the challenges and value of real-world evidence (RWE) post approval, the differences in regulatory and health technology assessment (HTA) real-world data (RWD) collection requirements under the German regulation for more safety in drug supply (GSAV), and future alignment opportunities to create a complementary framework for postapproval RWE requirements. Methods Eleven semistructured interviews were conducted purposively with pharmaceutical industry experts, regulatory authorities, health technology assessment bodies (HTAbs), and academia. The interview questions focused on the role of RWE post approval, the added value and challenges of RWE, the most important requirements for RWD collection, experience with registries as a source of RWD, perceptions on the GSAV law, RWE requirements in other countries, and the differences between regulatory and HTA requirements and alignment opportunities. The interviews were recorded, transcribed, and translated for coding in Nvivo to summarize the findings. Results All experts agree that RWE could close evidence gaps by showing the actual value of medicines in patients under real-world conditions. However, experts acknowledged certain challenges such as: (i) heterogeneous perspectives and differences in outcome measures for RWE generation and (ii) missing practical experience with RWD collected through mandatory registries within the German benefit assessment due to an unclear implementation of the GSAV. Conclusions This study revealed that all stakeholder groups recognize the added value of RWE but experience conflicting demands for RWD collection. Harmonizing requirements can be achieved through common postlicensing evidence generation (PLEG) plans and joint scientific advice to address uncertainties regarding evidence needs and to optimize drug development.


Author(s):  
Alex Simpson ◽  
Sreeram V Ramagopalan

In this round up, we cover how COVID-19 has been beneficial for improved access to real-world data, as well as how real-world data can be used to address health inequity, an area of increasing interest for health technology assessment.


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