scholarly journals Provisions and Special Considerations for Rare Diseases / Orphan Drugs by Health Technology Assessment (HTA) Bodies: Systematic Evaluation in 25 Countries

2016 ◽  
Vol 19 (7) ◽  
pp. A602
Author(s):  
M Mwamburi ◽  
M Nanavaty ◽  
S Gala ◽  
A Nyandege ◽  
Ramesh
Keyword(s):  
Health Technology Assessment ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Health Technology ◽  
Orphan Drugs ◽  
Systematic Evaluation

OP100 How Health Technology Assessment Is Adapting To Orphan Drugs In Canada – Not!

2017 ◽  
Vol 33 (S1) ◽  
pp. 45-46
Author(s):  
Durhane Wong-Rieger ◽  
Ferg Mills
Keyword(s):  
Health Technology Assessment ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Unmet Need ◽  
Health Technology ◽  
Orphan Drugs ◽  
Evaluation Framework ◽  
Lymphocytic Leukemia ◽  
Primary Analysis ◽  
Small Patient Population

INTRODUCTION:Some countries have distinct pathways for drugs for rare diseases (DRDs) (1). In May 2014, the Canadian Agency for Technologies in Health (CADTH) rejected the option of a separate review pathway for DRDs, reiterating that “pharmacoeconomic analyses are critical for all types of drugs”. While the gap between positive recommendations for common and rare drugs may have narrowed, the rejection for DRDs is still proportionally much higher (2). The default has been to provincially negotiate drug access, for patient populations, subgroups or individuals. Still not wishing to create a separate pathway, in March 2016, CADTH produced a revised evaluation framework for “uncertain clinical and pharmacoeconomic evidence” and other considerations representing “significant unmet need” including rarity and difficulty to study because of small patient population”(3). This study analyzes recommendations for DRDs following the two CADTH revisions.METHODS:Methods used were: synthesis of previously conducted analyses of CADTH recommendations for rare and non-rare drugs, primary comparative analysis of CADTH recommendations for DRDs from 2004 to 2016, and qualitative analysis of two drugs submitted for both rare and non-rare conditions: everolimus (breast cancer, pancreatic neuroendocrine tumours, and tuberous sclerosis complex) and ibrutinib (chronic lymphocytic leukemia, small lymphocytic lymphoma, and Waldenström's Macroglobulinemia).RESULTS:Previous analyses found that DRDs received more negative recommendations than did non-rare drugs; both clinical and economic evidence were differentiating factors. The primary analysis provided an additional understanding of reasons for negative recommendations. There is low consistency across assessments and across the two CADTH review committees. The case studies illustrated the challenges for DRDs to overcome barriers of cost-effectiveness and certainty of clinical evidence, even with the revised framework.CONCLUSIONS:This research challenges the premise that Health Technology Assessment for all drugs can result in fair and equitable recommendations for DRDs. Moreover, assessments based on “significant unmet need” do not appear to provide consistent or equitable guidelines for addressing the issues specific to rare diseases.

scholarly journals The application of multi-criteria decision analysis to inform in resource allocation

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 445
Author(s):  
Carina Schey ◽  
Maarten Postma ◽  
Paul Krabbe ◽  
Goran Medic ◽  
Mark Connolly
Keyword(s):  
Decision Analysis ◽  
Health Technology Assessment ◽  
Disease Severity ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Health Technology ◽  
Orphan Drugs ◽  
Analysis Framework ◽  
Multi Criteria Decision Analysis ◽  
Annual Treatment

Background: There is a perception held by payers that orphan products are expensive. As a result, the current health technology assessment systems might be too restrictive for orphan drugs, therefore potentially denying patients access to life-saving medicines. While price is important, it should be considered in relation to a broader range of disease-related product attributes that are not necessarily considered by many health technology assessment agencies. To overcome these challenges, multi-criteria decision analysis has been proposed as an alternative to evaluate technologies. Methods: A targeted literature review was conducted to identify the most frequently cited attributes in multi-criteria decision analysis (MCDA) in rare diseases. From the leading attributes identified, we developed a multi-criteria decision analysis framework with which to aggregate the orphan drug values. We subsequently reviewed and plotted the relationship between single attributes and the average annual treatment costs for 8 drugs used in the treatment of rare endocrine diseases. The annual treatment costs were based on UK list prices for the average daily dose per patient. Results: The five most frequently mentioned attributes in the literature were as follows: Disease severity, Unmet need (or availability of therapeutic alternatives), Comparative effectiveness or efficacy, Quality of evidence and Safety & tolerability. Results from the multi-criteria decision analysis framework indicate a wide range of average annual per-patients costs for drugs intended for the same diseases, and likewise for diseases with a similar level of Disease severity. Conclusions: Multi-criteria decision analysis may offer a viable alternative to support discussion in reimbursement decisions for orphan drugs. The analyses can be used to inform investigations on the application of MCDAs in rare diseases.

scholarly journals Building a trusted framework for uncertainty assessment in rare diseases: suggestions for improvement (Response to “TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases”)

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Sabine E. Grimm ◽  
Xavier Pouwels ◽  
Bram L. T. Ramaekers ◽  
Ben Wijnen ◽  
Saskia Knies ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Research Agenda ◽  
Health Technology ◽  
Orphan Drugs ◽  
Uncertainty Assessment ◽  
Letter To The Editor ◽  
Research Prioritisation ◽  
Evidence Generation

AbstractThe aim of this letter to the editor is to provide a comprehensive summary of uncertainty assessment in Health Technology Assessment, with a focus on transferability to the setting of rare diseases. The authors of “TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases” presented recommendations for reducing uncertainty in rare diseases. Their article is of great importance but unfortunately suffers from a lack of references to the wider uncertainty in Health Technology Assessment and research prioritisation literature and consequently fails to provide a trusted framework for decision-making in rare diseases. In this letter to the editor we critique the authors’ tool and provide pointers as to how their proposal can be strengthened. We present references to the literature, including our own tool for uncertainty assessment (TRUST; unrelated to the authors’ research), apply TRUST to two assessments of orphan drugs in rare diseases and provide a broader perspective on uncertainty and risk management in rare diseases, including a detailed research agenda.

scholarly journals Digi-HTA: Health technology assessment framework for digital healthcare services

2019 ◽  
Vol 11 (4) ◽  
Author(s):  
Jari Haverinen ◽  
Niina Keränen ◽  
Petra Falkenbach ◽  
Anna Maijala ◽  
Timo Kolehmainen ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
State Of The Art ◽  
Health Technology ◽  
Healthcare Services ◽  
The State ◽  
Systematic Evaluation ◽  
Health Technologies ◽  
Digital Healthcare ◽  
And Robotics

Health technology assessment (HTA) refers to the systematic evaluation of the properties, effects, and/or impacts of health technology. The main purpose of the assessment is to inform decisionmakers in order to better support the introduction of new health technologies. New digital healthcare solutions like mHealth, artificial intelligence (AI), and robotics have brought with them a great potential to further develop healthcare services, but their introduction should follow the same criteria as that of other healthcare methods. They must provide evidence-based benefits and be safe to use, and their impacts on patients and organizations need to be clarified. The first objective of this study was to describe the state-of-the-art HTA methods for mHealth, AI, and robotics. The second objective of this study was to evaluate the domains needed in the assessment. The final aim was to develop an HTA framework for digital healthcare services to support the introduction of novel technologies into Finnish healthcare. In this study, the state-of-the-art HTA methods were evaluated using a literature review and interviews. It was noted that some good practices already existed, but the overall picture showed that further development is still needed, especially in the AI and robotics fields. With the cooperation of professionals, key aspects and domains that should be taken into account to make fast but comprehensive assessments were identified. Based on this information, we created a new framework which supports the HTA process for digital healthcare services. The framework was named Digi-HTA.

scholarly journals SCIENTIFIC AND SOCIAL VALUE JUDGMENTS FOR ORPHAN DRUGS IN HEALTH TECHNOLOGY ASSESSMENT

2016 ◽  
Vol 32 (4) ◽  
pp. 218-232 ◽  
Author(s):  
Elena Nicod ◽  
Panos Kanavos
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Orphan Drugs ◽  
Social Value ◽  
European Medicines Agency ◽  
Final Decision ◽  
Value Judgments ◽  
Accountability For Reasonableness ◽  
The Impact

Objectives: We explore how broader aspects of a treatment's value and the impact of the condition on patients not captured by routine health technology assessment (HTA) methods using clinical and economic evidence, defined as “other considerations,” may influence HTA processes in different settings.Methods: Countries included were England, Scotland, Sweden, and France. Data sources were the publicly available reports on HTA recommendations. Ten drugs with European Medicines Agency orphan designation and appraised in England were selected. Qualitative thematic analysis was used to systematically identify and code all “other considerations” based on a previously developed methodological framework, which also coded whether it was provided by stakeholders, and how it influenced the decision.Results: A classification framework of scientific and social value judgments was developed and used throughout the study. A total of 125 “other considerations” were identified and grouped into ten subcategories based on the information provided. Eighteen to 100 percent of these, depending on the agency, were put forward as one of the main reasons for the final decision potentially contributing to accepting a higher incremental cost-effectiveness ratio or uncertain evidence. Some of these were nonquantified or nonelicited and pertained to the assessor's judgment. A taxonomy of these value judgments was created to be used in future cases. Results also contributed to better defining the determinants of social value and improving accountability for reasonableness.Conclusions: The systematic identification of the scientific and social value judgments enables to better understanding the dimensions of value, which can be used to improve their transparency and consistent use across decisions and settings.

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