scholarly journals Added Therapeutic Value of Medicinal Products for French and German Health Technology Assessment Organizations: A Systematic Comparison

2020 ◽  
Author(s):  
Denis Boucaud-Maitre ◽  
Driss Berdaï ◽  
Francesco Salvo
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Medicinal Products ◽  
Systematic Comparison ◽  
German Health ◽  
Therapeutic Value

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.

OP171 Does Parallel Regulatory-Health Technology Assessment Reviews Affect Time To Health Technology Assessment Decisions?

2018 ◽  
Vol 34 (S1) ◽  
pp. 62-63
Author(s):  
Jesmine Cai ◽  
Tina Wang ◽  
Neil McAuslane ◽  
Lawrence Liberti
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Review Process ◽  
Health Technology ◽  
Access To Medicines ◽  
Regulatory Approval ◽  
Regulatory Submission ◽  
Therapeutic Value ◽  
Assessment Decisions ◽  
Health Canada

Introduction:Timely recommendation by Health Technology Assessment (HTA) agencies for drug reimbursement is critical to ensure patient access to medicines of therapeutic value. In this study, HTA performance was examined in terms of their outcome and timing by looking at how 103 drugs, which gained regulatory approval from 2013 to 2015, were assessed by HTA agencies from 2014 to 2016.Methods:Products must have received regulatory approval from one of the following regulatory agencies: EMA (Europe), Health Canada (Canada) and TGA (Australia). The first HTA recommendations were then collected from PBAC (Australia), CADTH (Canada), HAS (France), IQWiG (Germany), SMC (Scotland) and TLV (Sweden). The HTA decisions were classified as positive, positive with restrictions, negative and multiple.Results:Eighty-four drugs were approved in Europe before Australia and Canada. Of the studied HTA agencies, PBAC had the highest percentage of products recommended within a year from regulatory approval (93 percent). In addition, Australia had the shortest median time between first regulatory submission by any of the three agencies and HTA recommendation (553 days) as compared to Europe (616 days) and Canada (722 days). This can be attributed to the TGA/PBAC parallel process. However, Australia has the highest proportion of products receiving a negative PBAC recommendation (62 percent).Conclusions:The majority of drugs were first submitted for reimbursement in Europe, but the time from regulatory submission to HTA decision was the fastest in Australia. This can be attributed to the TGA/PBAC parallel review process, which showed its benefit in reducing the overall time. A parallel review process is also available in Canada; however, it is not utilized as frequently by companies as in Australia.

PRICING, REIMBURSEMENT, AND HEALTH TECHNOLOGY ASSESSMENT OF MEDICINAL PRODUCTS IN BULGARIA

2017 ◽  
Vol 33 (3) ◽  
pp. 365-370 ◽  
Author(s):  
Tatyana Benisheva-Dimitrova ◽  
Dobriana Sidjimova ◽  
Daniela Cherneva ◽  
Nikolay Kralimarkov
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Objective Assessment ◽  
Historical Analysis ◽  
Health Technology ◽  
National Council ◽  
The European Union ◽  
Medicinal Products ◽  
Policy Objective ◽  
Pricing And Reimbursement

Objectives: The aim of this study was to investigate the analysis, discussion, and challenges of the price and reimbursement process of medicinal products in Bulgaria in the period 2000–15 and health technology assessment (HTA) role in these processes.Methods: The dynamics of the reform, with respect to the healthcare and pharmaceutical sectors, are tracked by documentary review of regulations, articles, and reports in the European Union (EU), as well as analytical and historical analysis.Results: Pricing and reimbursement processes have passed through a variety of committees between 2003 and 2012. Separate units for pricing and reimbursement of medicinal products were established in Bulgaria for the first time, in 2013, when an independent body, the National Council at Prices and Reimbursement of Medicinal Products, was set up to approve medicinal products with new international nonproprietary names (INN) for reimbursement in Bulgaria. Over the course of 2 years (2013–14), thirty-three new INNs were approved for reimbursement. In December 2015, a new HTA body was introduced, and assigned to the National Centre for Public Health and Analyses.Conclusions: Although Bulgaria has current legislation on pricing and reimbursement which is in accordance with the EU rules, there is no mechanism for reporting and monitoring these processes or the financial resources annually, so as to provide an overall objective assessment and analysis by year. Therefore, this financial assessment should become a national policy objective for the future.

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.

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