scholarly journals Advanced therapy medicinal products: value judgement and ethical evaluation in health technology assessment

2020 ◽  
Vol 21 (3) ◽  
pp. 311-320 ◽  
Author(s):  
Elisabete Gonçalves
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Current Status ◽  
Integrated Analysis ◽  
Medicinal Products ◽  
Real World Data ◽  
Ethical Evaluation ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy

AbstractAdvanced therapy medicinal products (ATMPs) are a heterogeneous class of medicinal products that by offering the potential of cure represent a paradigm shift in the approach of many life-threatening diseases. Although a common regulatory framework for ATMPs has been established in the EU, the health technology assessment (HTA) and financing decisions remain local. The aim of this article is to present an integrated analysis of the current status of the value judgment of ATMPs and the integration of ethical evaluation in the HTA process. It has been identified that approaching the specificities of ATMPs in terms of market access will require a broadening of the definition of value to be able to systematically capture elements of value not traditionally considered. Outcomes modelling will play an important role in the pricing and reimbursement of ATMPs, providing a way to bridge the gap caused by the absence of data from clinical studies or real-world data. Given the nature and disruptive consequences of ATMPs the assessment and adoption of these medicinal products raises important ethical questions, both at a policy and at society level that should be properly addressed. HTA can be made more transparent and reliable, and simultaneously promote robust and accountable decision making, by turning explicit the value judgments implicit in HTA. Ultimately, there should be no core conflict between ethical requirements and HTA in a scenario where the goal is to promote equity and access of patients to truly innovative therapies such as ATMPs, while assuring the sustainability of healthcare systems.

scholarly journals Health Technology Assessment of Advanced Therapy Medicinal Products: Comparison Among 3 European Countries

2021 ◽  
Vol 12 ◽  
Author(s):  
Lucia Gozzo ◽  
Giovanni Luca Romano ◽  
Francesca Romano ◽  
Serena Brancati ◽  
Laura Longo ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
National Level ◽  
Health Technology ◽  
European Countries ◽  
Added Value ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Therapeutic Value ◽  
Advanced Therapy

Even for centrally approved products, each European country is responsible for the effective national market access. This step can result in inequalities in terms of access, due to different opinions about the therapeutic value assessed by health technology assessment (HTA) bodies. Advanced therapy medicinal products (ATMPs) represent a major issue with regard to the HTA in order to make them available at a national level. These products are based on genes, tissues, or cells, commonly developed as one-shot treatment for rare or ultrarare diseases and mandatorily authorized by the EMA with a central procedure. This study aims to provide a comparative analysis of HTA recommendations issued by European countries (France, Germany, and Italy) following EMA approval of ATMPs. We found a low rate of agreement on the therapeutic value (in particular the “added value” compared to the standard of care) of ATMPs. Despite the differences in terms of clinical assessment, the access has been usually guaranteed, even with different timing and limitations. In view of the importance of ATMPs as innovative therapies for unmet needs, it is crucial to understand and act on the causes of disagreement among the HTA. In addition, the adoption of the new EU regulation on HTA would be useful to reduce disparities of medicine’s assessment among European countries.

scholarly journals Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

2018 ◽  
Vol 20 (3) ◽  
pp. 427-438 ◽  
Author(s):  
Bengt Jönsson ◽  
Grace Hampson ◽  
Jonathan Michaels ◽  
Adrian Towse ◽  
J.-Matthias Graf von der Schulenburg ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy

OP119 Advanced Therapy Medicinal Products: Are Current Health Technology Assessment Methods Suitable?

2017 ◽  
Vol 33 (S1) ◽  
pp. 54-55
Author(s):  
Grace Hampson ◽  
Adrian Towse
Keyword(s):  
Cell Therapy ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Medicinal Products ◽  
T Cell Therapy ◽  
Car T Cell Therapy ◽  
Advanced Therapy ◽  
Car T ◽  
Regenerative Medicines

INTRODUCTION:There is considerable excitement around the development of regenerative medicines (or advanced therapy medicinal products, ATMPs), with the expectation that they may bring substantial clinical gains and offer cures for previous debilitating and fatal diseases. However, high costs mean that Health Technology Assessment (HTA) and reimbursement decisions are challenging for payers and manufacturers, even when the therapies are expected to offer good value for money.In Europe, seven ATMPs have market authorization, yet only one has achieved national level reimbursement. Statistics such as these put HTA bodies under pressure to review their methods and consider how these can apply to regenerative medicines.METHODS:We present a review of one example, from the United Kingdom's National Institute for Health and Care Excellence (NICE), who commissioned an external organization to undertake a mock appraisal of a hypothetical ATMP using standard methods. The therapeutic area chosen for the mock appraisal was chimeric antigen receptor (CAR) T-cell therapy for treating relapsed or refractory B-cell acute lymphoblastic leukaemia.RESULTS:The role of uncertainty was a key consideration within the report, yet we found that the presentation of uncertainty within the mock appraisal was misleading for decision makers.We found that the exercise represents a thorough mock HTA of CAR T-cell therapy. However, it focused on testing whether ATMPs could fit into the existing HTA pathway for conventional medicines, rather than seeking to identify the most suitable approach for assessing regenerative medicines. We suggest the latter would have been a more relevant question for the mock appraisal.CONCLUSIONS:Any significant departures from the usual HTA process must be based on solid economic rationale if we are to ensure efficient allocation of resources. Thus, in order for regenerative medicines to be given ‘special treatment,’ it must be demonstrated that societal preferences, or the full extent of health (or non-health) benefits, are not being realised for this group of treatments through existing HTA methods.

scholarly journals Real-world evidence: perspectives on challenges, value, and alignment of regulatory and national health technology assessment data collection requirements

2021 ◽  
Vol 37 ◽  
Author(s):  
Hannah Sievers ◽  
Angelika Joos ◽  
Mickaël Hiligsmann
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Practical Experience ◽  
Added Value ◽  
Real World Data ◽  
Conflicting Demands ◽  
Real World Evidence ◽  
Semistructured Interviews

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.

R WE ready for reimbursement? A round up of developments in RWE relating to health technology assessment: part 2

2021 ◽  
Author(s):  
Alex Simpson ◽  
Sreeram V Ramagopalan
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Health Inequity ◽  
Real World Data ◽  
World Data

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.

ETHICS IN HEALTH TECHNOLOGY ASSESSMENT: A SYSTEMATIC REVIEW

2018 ◽  
Vol 34 (5) ◽  
pp. 447-457 ◽  
Author(s):  
Christian A. Bellemare ◽  
Pierre Dagenais ◽  
Suzanne K.-Bédard ◽  
Jean-Pierre Béland ◽  
Louise Bernier ◽  
...  
Keyword(s):  
Systematic Review ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Policy Decision ◽  
Health Technology ◽  
Ethical Analysis ◽  
Social Needs ◽  
Inductive Method ◽  
Value Judgments ◽  
Ethical Evaluation

Objectives:Integration of ethics into health technology assessment (HTA) remains challenging for HTA practitioners. We conducted a systematic review on social and methodological issues related to ethical analysis in HTA. We examined: (1) reasons for integrating ethics (social needs); (2) obstacles to ethical integration; (3) concepts and processes deployed in ethical evaluation (more specifically value judgments) and critical analyses of formal experimentations of ethical evaluation in HTA.Methods:Search criteria included “ethic,” “technology assessment,” and “HTA”. The literature search was done in Medline/Ovid, SCOPUS, CINAHL, PsycINFO, and the international HTA Database. Screening of citations, full-text screening, and data extraction were performed by two subgroups of two independent reviewers. Data extracted from articles were grouped into categories using a general inductive method.Results:A list of 1,646 citations remained after the removal of duplicates. Of these, 132 were fully reviewed, yielding 67 eligible articles for analysis. The social need most often reported was to inform policy decision making. The absence of shared standard models for ethical analysis was the obstacle to integration most often mentioned. Fairness and Equity and values embedded in Principlism were the values most often mentioned in relation to ethical evaluation.Conclusions:Compared with the scientific experimental paradigm, there are no settled proceedings for ethics in HTA nor consensus on the role of ethical theory and ethical expertise hindering its integration. Our findings enable us to hypothesize that there exists interdependence between the three issues studied in this work and that value judgments could be their linking concept.

PP14 Development Of The European Network For Health Technology Assessment Standards Tool For Registries In Health Technology Assessment

2018 ◽  
Vol 34 (S1) ◽  
pp. 70-70
Author(s):  
Emmanuel Gimenez Garcia ◽  
Mireia Espallargues ◽  
Jae Long ◽  
Maja Valentic ◽  
Irena Guzina ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Good Practice ◽  
Health Technology ◽  
Registry Data ◽  
European Network ◽  
Real World Data ◽  
The Third ◽  
Evidence Quality ◽  
Evidence Generation

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.

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.

scholarly journals Application of Health Technology Assessment (HTA) to Evaluate New Laboratory Tests in a Health System: A Case Study of Bladder Cancer Testing

2020 ◽  
Vol 7 ◽  
pp. 237428952096822
Author(s):  
Erik J. Landaas ◽  
Ashley M. Eckel ◽  
Jonathan L. Wright ◽  
Geoffrey S. Baird ◽  
Ryan N. Hansen ◽  
...  
Keyword(s):  
Bladder Cancer ◽  
Pilot Study ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Test System ◽  
Real World Data ◽  
World Data

We describe the methods and decision from a health technology assessment of a new molecular test for bladder cancer (Cxbladder), which was proposed for adoption to our send-out test menu by urology providers. The Cxbladder health technology assessment report contained mixed evidence; predominant concerns were related to the test’s low specificity and high cost. The low specificity indicated a high false-positive rate, which our laboratory formulary committee concluded would result in unnecessary confirmatory testing and follow-up. Our committee voted unanimously to not adopt the test system-wide for use for the initial diagnosis of bladder cancer but supported a pilot study for bladder cancer recurrence surveillance. The pilot study used real-world data from patient management in the scenario in which a patient is evaluated for possible recurrent bladder cancer after a finding of atypical cytopathology in the urine. We evaluated the type and number of follow-up tests conducted including urine cytopathology, imaging studies, repeat cystoscopy evaluation, biopsy, and repeat Cxbladder and their test results. The pilot identified ordering challenges and suggested potential use cases in which the results of Cxbladder affected a change in management. Our health technology assessment provided an objective process to efficiently review test performance and guide new test adoption. Based on our pilot, there were real-world data indicating improved clinician decision-making among select patients who underwent Cxbladder testing.

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