Does conditional approval for new oncology drugs in Europe lead to differences in health technology assessment decisions?

Lipska, Iga; Hoekman, Jarno; McAuslane, Neil; Leufkens, Hubert G.M.; Hövels, Anke M.

doi:10.1002/cpt.198

Cited by 32 publications

(48 citation statements)

References 3 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…HTA recommendation outcomes were categorized as positive, positive with restrictions (called restricted), or negative, in line with previous research. 4 CEAs were separated from REAs in jurisdictions performing both. Reported REAs in HTA dossiers may entail three (positive, restricted, negative) to six (ranging from less benefit to major benefit) categories and vary per jurisdiction.…”

Section: Discussionmentioning

confidence: 99%

“…To compare and evaluate HTA practices, many studies exist that investigate HTA recommendations for groups of medicines. [2][3][4][5][6][7][8][9][10][11] They differ in the extent to which they include practice-related factors as explanatory for differences between HTA jurisdictions. Based on our results, it is recommended to take a systematic approach to investigating practice differences between HTA jurisdictions.…”

Section: Discussionmentioning

confidence: 99%

“…Consequently, differences in HTA recommendations have been the subject of extensive research. [2][3][4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19] Studies qualitatively assessing recommendations for groups of drugs in several HTA jurisdictions report that a series of sources contribute to observed differences. These sources can be roughly divided into two categories.…”

Section: Introductionmentioning

confidence: 99%

“…Second, even when jurisdictions apply the same practice (eg, a cost-effectiveness assessment [CEA]), there may still be differences in HTA outcomes, stemming from differences in the inclusion and evaluation of evidence, the impact of evidence on the final recommendation, and the integral leveraging of different types of evidence. [2][3][4][5][6]8 These can be classified as value assessment differences. Within HTA practices, a nonsubmission means that an HTA body provides a recommendation on reimbursement, even though no submission has been provided by the relevant company.…”

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

Differences in Health Technology Assessment Recommendations Among European Jurisdictions: The Role of Practice Variations

et al. 2020

View full text Add to dashboard Cite

Background: Health technology assessment (HTA) plays an important role in reimbursement decision-making in many countries, but recommendations vary widely throughout jurisdictions, even for the same drug. This variation may be due to differences in the weighing of evidence or differences in the processes or procedures, which are known as HTA practices. Objective: To provide insight into the effects of differences in practices on interpretation of intercountry differences in HTA recommendations for conditionally approved drugs. Methods: HTA recommendations for conditionally approved drugs (N = 27) up until June 2017 from England/Wales, France, Germany, the Netherlands, and Scotland were included. Recommendations and practice characteristics were extracted from these five jurisdictions and this data was validated. The effect of nonsubmissions, resubmissions, and reassessments; costeffectiveness assessments; and price negotiations on changes in the percentage of negative recommendations and the interpretation of intercountry differences in HTA outcomes were analyzed using Fisher exact tests. Results: The inclusion of cost-effectiveness assessments led to significant increases in the proportion of negative recommendations in England/Wales (from 4% to 50%, P,.01) and Scotland (from 21% to 71%, P,.01). The subsequent inclusion of price negotiations led to significant reductions in the proportion of negative recommendations in England/ Wales (from 50% to 14%, P,.01), France (from 31% to 3%, P=.012), and Germany (from 34% to 0%, P,.01). Results indicated that the inclusion of nonsubmissions and resubmissions might affect Scottish negative HTA recommendations (from 7% to 21%), but this effect was not significant. No significant effects were observed in the Netherlands, possibly owing to sample size. Conclusion: Variations in HTA practices between international jurisdictions can have a substantial and significant impact on conclusions about recommendations by HTA bodies, as exemplified in this cohort of conditionally approved products. Studies comparing international HTA recommendations should carefully consider possible practice variations between jurisdictions.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Differences in Health Technology Assessment Recommendations Among European Jurisdictions: The Role of Practice Variations

et al. 2020

View full text Add to dashboard Cite

show abstract

“…Uncertainty is rife in health technology assessment (HTA), which leads to increased complexity in decision making. Drug approval processes are typically based on evidence that exhibits several limitations [1][2][3]. These may include, among others, a lack of data on a certain relevant health outcome (e.g.…”

Section: Introductionmentioning

confidence: 99%

Development and Validation of the TRansparent Uncertainty ASsessmenT (TRUST) Tool for Assessing Uncertainties in Health Economic Decision Models

et al. 2019

View full text Add to dashboard Cite

Background An increasing number of technologies are obtaining marketing authorisation based on sparse evidence, which causes growing uncertainty and risk within health technology reimbursement decision making. To ensure that uncertainty is considered and addressed within health technology assessment (HTA) recommendations, uncertainties need to be identified, included in health economic models, and reported. Objective Our objective was to develop the TRansparent Uncertainty ASsessmenT (TRUST) tool for systematically identifying, assessing, and reporting uncertainties in decision models, with the aim of making uncertainties and their impact on cost effectiveness more explicit and transparent. Methods TRUST was developed by drawing on the uncertainty and risk assessment literature. To develop and validate this tool, we conducted HTA stakeholder discussion meetings and interviews and applied it in six real-world HTA case studies in the Netherlands and the UK. Results The TRUST tool enables the identification and categorisation of uncertainty according to its source (transparency issues, methodology issues, and issues with evidence: imprecision, bias and indirectness, and unavailability) in each model aspect. The source of uncertainty determines the appropriate analysis. The impact of uncertainties on cost effectiveness is also assessed. Stakeholders found using the tool to be feasible and of value for transparent uncertainty assessment. TRUST can be used during model development and/or model review. Conclusion The TRUST tool enables systematic identification, assessment, and reporting of uncertainties in health economic models and may contribute to more informed and transparent decision making in the face of uncertainty.

show abstract

Weighing of Evidence by Health Technology Assessment Bodies: Retrospective Study of Reimbursement Recommendations for Conditionally Approved Drugs

Vreman

Bouvy

Bloem

et al. 2018

Clin Pharma and Therapeutics

View full text Add to dashboard Cite

This study assessed whether five Health Technology Assessment ( HTA ) bodies in Europe were more negative about drugs with a Conditional Marketing Authorization ( CMA ) that are approved without controlled studies compared to CMA drugs that are approved based on controlled studies. The HTA recommendations were categorized into positive, restricted, and negative. A total of 92 HTA recommendations were available for 27 drugs. Thirty of 62 (48%) and 17 of 30 (57%) of the recommendations were negative for drugs with and without controlled studies, respectively. Overall, only 12 (13%) recommendations were positive. In all jurisdictions, recommendations between drugs with and drugs without controlled data were comparable, which suggests that the presence of controlled data is not decisive in HTA evaluations. The small proportion of unrestricted positive recommendations highlights difficulties with recommending the drugs in this cohort, which may be caused by scientific uncertainty or other factors. Earlier collaboration between stakeholders is advised in order to improve patient access.

show abstract

Does conditional approval for new oncology drugs in Europe lead to differences in health technology assessment decisions?

Cited by 32 publications

References 3 publications

Differences in Health Technology Assessment Recommendations Among European Jurisdictions: The Role of Practice Variations

Differences in Health Technology Assessment Recommendations Among European Jurisdictions: The Role of Practice Variations

Development and Validation of the TRansparent Uncertainty ASsessmenT (TRUST) Tool for Assessing Uncertainties in Health Economic Decision Models

Weighing of Evidence by Health Technology Assessment Bodies: Retrospective Study of Reimbursement Recommendations for Conditionally Approved Drugs

Contact Info

Product

Resources

About