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

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.

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 Development of a checklist to guide equity considerations in health technology assessment

2021 ◽  
Vol 37 ◽  
Author(s):  
Maria Benkhalti ◽  
Manuel Espinoza ◽  
Richard Cookson ◽  
Vivian Welch ◽  
Peter Tugwell ◽  
...  
Keyword(s):  
Knowledge Translation ◽  
Health Technology Assessment ◽  
Priority Setting ◽  
Technology Assessment ◽  
Literature Search ◽  
Health Inequities ◽  
Health Technology ◽  
Decision Makers ◽  
Added Value ◽  
Pilot Test

Abstract Objectives Health technology assessment (HTA) can impact health inequities by informing healthcare priority-setting decisions. This paper presents a novel checklist to guide HTA practitioners looking to include equity considerations in their work: the equity checklist for HTA (ECHTA). The list is pragmatically organized according to the generic HTA phases and can be consulted at each step. Methods A first set of items was based on the framework for equity in HTA developed by Culyer and Bombard. After rewording and reorganizing according to five HTA phases, they were complemented by elements emerging from a literature search. Consultations with method experts, decision makers, and stakeholders further refined the items. Further feedback was sought during a presentation of the tool at an international HTA conference. Lastly, the checklist was piloted through all five stages of an HTA. Results ECHTA proposes elements to be considered at each one of the five HTA phases: Scoping, Evaluation, Recommendations and Conclusions, Knowledge Translation and Implementation, and Reassessment. More than a simple checklist, the tool provides details and examples that guide the evaluators through an analysis in each phase. A pilot test is also presented, which demonstrates the ECHTA's usability and added value. Conclusions ECHTA provides guidance for HTA evaluators wishing to ensure that their conclusions do not contribute to inequalities in health. Several points to build upon the current checklist will be addressed by a working group of experts, and further feedback is welcome from evaluators who have used the tool.

Qualitative comparative analysis of health economic evaluation guidelines for health technology assessment in European countries

2020 ◽  
pp. 1-8
Author(s):  
Konstantinos Zisis ◽  
Panagiota Naoum ◽  
Kostas Athanasakis
Keyword(s):  
Sensitivity Analysis ◽  
Content Analysis ◽  
Economic Evaluation ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Evaluation Method ◽  
Outcome Measure ◽  
Health Technology ◽  
European Countries ◽  
Common Practices

Abstract Objective To classify, analyze, and compare published guidelines for economic evaluation within health technology assessment (HTA) in European countries and highlight differences and similarities. Methods We performed a literature review to identify published guidance for the conduct and assessment of economic evaluation studies that are undertaken within the context of HTA processes in European countries. Organizations and working groups were identified via the ISPOR, INAHTA, and EUnetHTA databases. Following the identification of official documents, we performed a qualitative content analysis to highlight discrepancies or common practices under the following categories: comparator, perspective on costs/benefits, time horizon, economic evaluation method, instrument used for utility measurement, outcome measure, source for efficacy, modeling, sensitivity analysis, discounting, and incremental cost-effectiveness ratio. Results A total of nineteen guidance documents were identified (in English) providing data for the analysis in nineteen countries. The comparative content analysis identified common practices in most countries regarding the approaches to the choice of comparator, source of data, the preferred economic evaluation method, the option for a lifetime analytical horizon, discounting, and the choice of key outcome measure—for which, most countries recommend the use of the EQ-5D instrument. Differences were mainly found in the choice of perspective, dealing with uncertainty and sensitivity analysis, the use of end points, and the required use of modeling. Conclusions The use of economic evaluation constitutes one of the key pillars of the HTA process in Europe. Although a methodological convergence has occurred during the last few years, notable differences still remain.

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.

HEALTH TECHNOLOGY ASSESSMENT IN THE NETHERLANDS

2000 ◽  
Vol 16 (2) ◽  
pp. 485-519 ◽  
Author(s):  
Michael Bos
Keyword(s):  
Health Care ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Private Insurance ◽  
National Level ◽  
Health Technology ◽  
Care Quality ◽  
High Tech ◽  
Fee For Service ◽  
Equal Distribution

The Dutch healthcare system is not a single overall plan, but has evolved from a constantly changing mix of institutions, regulations, and responsibilities. The resulting system provides high-quality care with reasonable efficiency and equal distribution over the population. Every Dutch citizen is entitled to health care. Health insurance is provided by a mix of compulsory national insurance and public and private insurance schemes. Hospitals generally have a private legal basis but are heavily regulated. Supraregional planning of high-tech medical services is also regulated. Hospitals function under fixed, prospective budgets with regulation of capital investments. Independent general practitioners serve a gatekeeper role for specialist and hospital services and are paid by capitation or fee for service. Specialists are paid by fee for service. All physicians' fees are controlled by the Ministry of Economic Affairs. Coverage of benefits is an important method of controlling the cost of services. There is increasing concern about health care quality. Health technology assessment (HTA) has become increasingly visible during the last 15 years. A special national fund for HTA, set up in 1988, has led to many formal and informal changes. HTA has evolved from a research activity into policy research for improving health care on the national level. In 1993 the government stated formally that enhancing effectiveness in health care was one of its prime targets and that HTA would be a prime tool for this purpose. The most important current issue is coordination of HTA activities, which is now undertaken by a new platform representing the important actors in health care and HTA.

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