Ansgar Hebborn - Academia.edu (original) (raw)

Papers by Ansgar Hebborn

Value in Health, Nov 1, 2004

To compare persistence and compliance of weekly and daily bisphosphonate regimens among postmenop... more To compare persistence and compliance of weekly and daily bisphosphonate regimens among postmenopausal osteoporotic women. METHODS: Postmenopausal osteoporotic women (>45 years) prescribed a once-weekly (QW) bisphosphonate (alendronate 35 or 70 mg) or once-daily (QD) bisphosphonate (alendronate 5 or 10 mg or risedronate 5 mg) were identified from an administrative claims database comprising 30 health plans. The QW and QD study cohorts were followed for 12 months after the index prescription. Medication possession ratio (MPR) was used to estimate compliance while persistence was calculated as the number of days from the initial prescription to a lapse of >30 days after completion of the previous refill. RESULTS: Between 1997 and 2002, a total of 2741 post-menopausal osteoporotic women who were prescribed a bisphosphonate (alendronate QW = 731; alendronate or risedronate QD = 2010) were identified (mean age = 63.7 years). Average MPR for the combined study cohorts was 60.6%. However, QW users had a significantly higher MPR than QD users (69.2% QW vs 57.6% QD, t =-7.51 p < 0.0001). Treatment persistence was significantly longer among the QW users than QD users (227 vs 185 days to discontinuation, respectively, log rank, p < 0.0001). Also, approximately 44% of weekly bisphosphonate users and 32% of daily bisphosphonate users persisted with their therapy at the end of 12 months. CONCLUSIONS: Post-menopausal women prescribed a weekly bisphosphonate regimen had significantly higher rates of compliance and longer persistence compared with those taking a more frequent, daily dosing regimen. However, rates for both regimens were less than desirable. These data demonstrate that less frequent dosing increases persistency, which is needed to obtain maximal long-term therapeutic benefits.

Health Economics Review, Nov 5, 2022

Objectives: Key challenges for a joint European Health Technology Assessment (HTA) include consol... more Objectives: Key challenges for a joint European Health Technology Assessment (HTA) include consolidated approaches towards the choice of adequate comparator(s), selection of endpoints that are relevant to patients with a given disease, dealing with remaining uncertainties as well as transparent and consistent management of related processes. We aimed to further crystallize related core domains within these four areas that warrant further research and scrutiny. Methods: Building on the outcomes of a previously conducted questionnaire survey, four key areas, processes, uncertainty, comparator choice and endpoint selection, were identified. At the inaugural convention of the European Access Academy dedicated working groups were established defining and prioritizing core domains for each of the four areas. The working groups consisted of ~ 10 participants each, representing all relevant stakeholder groups (patients/ clinicians/ regulators/ HTA & payers/ academia/ industry). Story books identifying the work assignments were shared in advance. Two leads and one note taker per working group facilitated the process. All rankings were conducted on an ordinal Likert Response Scale scoring from 1 (low priority) to 7 (high priority). Results: Identified key domains include for processes: i) address (resource-) challenge of multiple PICOs (Patient/ Intervention/ Comparator/ Outcomes), ii) time and capacity challenges, iii) integrating all involved stakeholders, iv) conflicts and aligning between different multinational stakeholders, v) interaction with health technology developer; for uncertainty: i) early and inclusive collaboration, ii) agreement on feasibility of RCT and acceptance of uncertainty, iii) alignment on closing evidence gaps, iv) capacity gaps; for comparator choice: i) criteria for the choice of comparator in an increasingly fragmented treatment landscape, ii) reasonable number of comparators in PICOs, iii) shape Early Advice so that comparator fulfils both regulatory and HTA needs, iv) acceptability of Indirect Treatment Comparisons (ITC), v) ensure broad stakeholder involvement in comparator selection; for endpoint selection: i) approaching new

Health Economics Review, Jun 2, 2022

Objectives: We conducted a multi-stakeholder survey to determine key areas where a joint European... more Objectives: We conducted a multi-stakeholder survey to determine key areas where a joint European health technology assessment (HTA) could provide 'additional benefit' compared to the status quo of many parallel independent national and subnational assessments. Methods: Leveraging three iterative Delphi cycles, a semiquantitative questionnaire was developed covering evidence challenges and heterogeneity of value drivers within HTAs across Europe with a focus on hematology/ oncology. The questionnaire consisted of five sections: i) background information; ii) value drivers in HTA assessments today; iii) evolving evidence challenges; iv) heterogeneity of value drivers across Europe; v) impact of Europe's Beating Cancer Plan (EBCP). The questionnaire was circulated across n = 189 stakeholder institutions comprising HTA and regulatory bodies, clinical oncology associations, patient representatives, and industry associations. Results: N = 30 responses were received (HTA bodies: 9; regulators: 10; patients' and physicians' associations: 3 each; industry: 5). Overall, 17 countries and EU level institutions were represented in the responses. Consistency across countries and stakeholder groups was high. Most relevant value drivers in HTAs today (scale 1, low to 5, high) were clinical trial design (mean 4.45), right endpoints (mean 4.40), and size of comparative effect (mean 4.33). Small patient numbers (mean 4.28) and innovative study designs (mean 4.1) were considered the most relevant evolving evidence challenges. Heterogeneity between regulatory and HTA evidence requirements and heterogeneity of the various national treatment standards and national HTA evidence requirements was high. All clinical and patient participants stated to have been with EBCP initiatives. Conclusions: For a European HTA to provide an 'additional benefit' over the multitude of existing national assessments key methodological and process challenges need to be addressed.

Health Economics Review

Objectives Key challenges for a joint European Health Technology Assessment (HTA) include consoli... more Objectives Key challenges for a joint European Health Technology Assessment (HTA) include consolidated approaches towards the choice of adequate comparator(s), selection of endpoints that are relevant to patients with a given disease, dealing with remaining uncertainties as well as transparent and consistent management of related processes. We aimed to further crystallize related core domains within these four areas that warrant further research and scrutiny. Methods Building on the outcomes of a previously conducted questionnaire survey, four key areas, processes, uncertainty, comparator choice and endpoint selection, were identified. At the inaugural convention of the European Access Academy dedicated working groups were established defining and prioritizing core domains for each of the four areas. The working groups consisted of ~ 10 participants each, representing all relevant stakeholder groups (patients/ clinicians/ regulators/ HTA & payers/ academia/ industry). Story books id...

Health Economics Review

Objectives We conducted a multi-stakeholder survey to determine key areas where a joint European ... more Objectives We conducted a multi-stakeholder survey to determine key areas where a joint European health technology assessment (HTA) could provide ‘additional benefit’ compared to the status quo of many parallel independent national and subnational assessments. Methods Leveraging three iterative Delphi cycles, a semiquantitative questionnaire was developed covering evidence challenges and heterogeneity of value drivers within HTAs across Europe with a focus on hematology/oncology. The questionnaire consisted of five sections: i) background information; ii) value drivers in HTA assessments today; iii) evolving evidence challenges; iv) heterogeneity of value drivers across Europe; v) impact of Europe’s Beating Cancer Plan (EBCP). The questionnaire was circulated across n = 189 stakeholder institutions comprising HTA and regulatory bodies, clinical oncology associations, patient representatives, and industry associations. Results N = 30 responses were received (HTA bodies: 9; regulators...

Objectives: The HTA Core Model ® was developed to improve the transferability of health technolog... more Objectives: The HTA Core Model ® was developed to improve the transferability of health technology assessment (HTA) between settings. The model has been used by HTA agencies but is also of interest to manufacturers, for improving internal evidence generation and communicating with other HTA stakeholders. To establish if the model is fit for purpose from an industry perspective, the pharmaceutical company Roche, collaborating with the European Network for HTA (EUnetHTA), conducted an assessment of the model. Methods: A questionnaire was developed to evaluate all assessment elements in the HTA Core Model v2.0 for their usefulness in meeting payers' evidence needs and demonstrating value. The questionnaire was completed by country affiliate teams working in evidence generation and reimbursement submissions for pharmaceuticals. Survey results were discussed in workshops to ensure consistency and alignment between teams. Results: The questionnaire was completed by six teams. An additional team from global pricing and market access participated in workshops. Model domains pertaining to the health problem and current technology use, technology description, clinical effectiveness, and economic value were considered most important because they meet payers' evidence needs. Overall, the model was considered useful to improve the efficiency of HTA evidence generation, share evidence internally, and communicate value to payers and HTA agencies. Conclusions: From an industry perspective, the HTA Core Model provides a useful framework and common terminology for efficient generation of transferable HTA evidence. The timeliness, efficiency, and transparency of HTA processes could be improved by a more standardized approach to HTA across settings.

International Journal of Technology Assessment in Health Care, 2020

The European Network for Health Technology Assessment (EUnetHTA) organizes an annual Forum with s... more The European Network for Health Technology Assessment (EUnetHTA) organizes an annual Forum with stakeholders to receive feedback on its activities, processes, and outputs produced. The fourth edition of the EUnetHTA Forum brought together representatives of HTA bodies, patient organizations, healthcare professionals (HCPs), academia, payers, regulators, and industry. The aim of this paper is to provide an overview of the highlights presented at the 2019 EUnetHTA Forum, reporting the main items and themes discussed in the plenary panel and breakout sessions. The leading topic was the concept of unmet medical need seen from different stakeholders' perspectives. Breakout sessions covered the joint production of assessment reports and engagement with payers, patients, and HCPs. Synergies, pragmatism, and inclusiveness across Member States and stakeholders were emphasized as leading factors to put in place a collaboration that serves the interest of patients and public health in a tr...

Health Economics Review, 2018

Background: Health Technology Assessments (HTA) procedures differ substantially across the variou... more Background: Health Technology Assessments (HTA) procedures differ substantially across the various European countries. We reviewed recent appraisals of a pharmaceutical manufacturer in three major European markets (France; Italy; Germany) and identified and categorized related decision drivers. Methods: New marketing authorisation between January 2011 and August 2017, and Roche being the Marketing Authorization Holder, were included. Outcome of HTA appraisals by the Haute Autorité de Santé (HAS), Agenzia Italiana del Farmaco (AIFA), and Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) were reviewed. Respective decision drivers were identified and commonalities and differences across the three countries were determined leveraging the EUnetHTA conceptual taxonomy (i.e. the 9 domains of the EUnetHTA core model). Results: Within that time period Roche received European marketing authorization for eight new molecular entities (10 indications, respectively). Outcome of HTA appraisals was heterogeneous across the three countries. However, the four clinical domains of the EUnetHTA core model were driving the national HTA appraisals, with the clinical effectiveness domain being of most importance. Important drivers related to the other three clinical domains included the target patient population (subgroups, Germany), the current management of the condition (unmet need, Italy), the regulatory status (Orphan Designation, Germany), as well as safety considerations (all three countries). Average time between EMA approval and full commercial availability of new medicines was 63 (Germany), 459 (Italy), and 557 days (France). Conclusions: The clinical domains of the EUnetHTA framework are mainly driven by national HTA appraisals, providing a suitable starting point for further developing a joint European view on value and evidence. Underlying topics and issues still reveal considerable differences.

International Journal of Technology Assessment in Health Care, 2018

Introduction:More than 50 HTA agencies evaluate the clinical value of medicines in Europe, result... more Introduction:More than 50 HTA agencies evaluate the clinical value of medicines in Europe, resulting in duplication of work for HTA agencies and manufacturers, and lengthy and variable time to reimbursement for patients across Europe. A consistent, single European relative clinical scientific benefit assessment of medicines could become a key element in ensuring patients get equitable and timely access across Europe. The European Network for Health Technology Assessment (EUnetHTA) is responsible under Joint Action 3 (JA3, 2016–2020) to pilot more than 30 Relative Effectiveness Assessments (REAs) of medicines. The first EUnetHTA JA3 REA pilots are now being completed and Roche, with its participation in the REA pilot for alectinib, has gathered relevant experience.Methods:The goal of this analysis is to summarize and reflect upon the experience with one of the first EUnetHTA REA assessments in JA3. The authors also propose potential process improvements.Results:The experience with th...

International Journal of Technology Assessment in Health Care, 2018

Objectives:The HTA Core Model® was developed to improve the transferability of health technology ... more Objectives:The HTA Core Model® was developed to improve the transferability of health technology assessment (HTA) between settings. The model has been used by HTA agencies but is also of interest to manufacturers, for improving internal evidence generation and communicating with other HTA stakeholders. To establish if the model is fit for purpose from an industry perspective, the pharmaceutical company Roche, collaborating with the European Network for HTA (EUnetHTA), conducted an assessment of the model.Methods:A questionnaire was developed to evaluate all assessment elements in the HTA Core Model v2.0 for their usefulness in meeting payers’ evidence needs and demonstrating value. The questionnaire was completed by country affiliate teams working in evidence generation and reimbursement submissions for pharmaceuticals. Survey results were discussed in workshops to ensure consistency and alignment between teams.Results:The questionnaire was completed by six teams. An additional team...

Value in Health, 2001

also resulted in a positive short-and long-term health economic benefit in acutely ill medical pa... more also resulted in a positive short-and long-term health economic benefit in acutely ill medical patients. The health-economic benefit of enoxaparin was positively related with the length of the follow-up period and a higher risk for recurrence of VTE and mortality in asymptomatic patients.

Value in Health, 2014

The German market access system for drugs have been changed significantly in the last years, by i... more The German market access system for drugs have been changed significantly in the last years, by introducing a similar focus on benefit assessment as in the French system. The research question remains whether they produce consistent results in terms of additional benefit (AB) for pharmaceuticals which have passed the assessment in both systems. MethOds: The G-BA and IQWiG as well as the Transparency Commission (TC) databases were searched systematically to identify those products, which have been processed in both systems between Jan 2011 and Dec 2013. For further comparison a data grid consisting of 26 items for evaluation has been developed including study comparator, primary clinical endpoints, health related quality of life inclusion. Results: Overall, 140 new therapies have been assessed in France by TC, and 80 in Germany by the G-BA. According to inclusion criteria, 44 products could be identified which have passed through both systems including 7 orphan drugs. Thirteen products (30%) had no AB granted by both Agencies, whereas 9 (20%) were in both cases granted with a minor AB, (assuming that "minor" values are equivalent between the two systems), amounting to 22/44 cases with a similar resolution. Five cases (11%) showed a discrepancy in added benefit, all times TC = no and G-BA = yes. However, varying magnitudes appeared to be the greatest difference (n = 17 (39%) remaining drugs), conditioned by lacking concordance of both scale grade systems. cOnclusiOns: Decisions of the agencies in both countries show partial heterogeneity in driving criteria like benefit levels (ASMR and AB). Although the evidence package for initial assessment in both countries is largely similar, preliminary results suggest their contextualization and scales are different. Further analysis based on results of the grid is needed to better assess criteria leading to different benefit levels and their reimbursement impact.

Value in Health, 2005

Post-menopausal osteoporosis has significant health care costs and impacts quality of life. The o... more Post-menopausal osteoporosis has significant health care costs and impacts quality of life. The objective of this analysis was to assess the cost-effectiveness of risedronate compared to calcium + vitamin D, etidronate, alendronate, and ibandronate in high-risk osteoporotic patients in Germany. METHODS: A validated model (Tosteson, 2001) was used to estimate the impact of therapy on hip and vertebral fractures, costs, and quality adjusted life years (QALYs). The analysis included women 70 years with a BMD T-Score of <-2.5 and a history of vertebral fracture, treated over 3 years. The model further simulated downstream costs and QALYs for a 10-year period. Country-specific data included general population mortality, hip and hospitalized vertebral fracture rates, fracture costs, and daily drug prices (risedronate €1.50; etidronate €1.17; alendronate €1.50; ibandronate €1.38). Ibandonate price based on U.S. pricing relative to risedronate, as German pricing not available. Hip and vertebral fracture reductions (risedronate 60%, 49%; etidronate 34%, 37%; alendronate 51%, 47%; ibandronate 0% [No efficacy demonstrated], 52% [Using 62% for efficacy does not change results]) were based on published clinical trials. RESULTS: In a cohort of 1000 postmenopausal women with 3 years of treatment the model predicted the following costs, total hip and hospitalized vertebral fractures and QALYs: risedronate (€8.22M, 139, 5451); alendronate (€8.41M, 142, 5447); etidronate (€8.42M, 149, 5441); ibandronate (€9.33M, 159, 5429); calcium + vitamin D (€7.91M, 164, 5427). All bisphosphonates were dominated by risedronate, which was less costly and had better outcomes. Risedronate had a cost per fracture averted of €12,389 and a cost per QALY gained of €13,253, compared to calcium + vitamin D. CONCLUSIONS: The analysis favors the adoption of risedronate therapy for the treatment of postmenopausal osteoporosis compared to other bisphosphonates.

Value in Health, Nov 1, 2004

To compare persistence and compliance of weekly and daily bisphosphonate regimens among postmenop... more To compare persistence and compliance of weekly and daily bisphosphonate regimens among postmenopausal osteoporotic women. METHODS: Postmenopausal osteoporotic women (>45 years) prescribed a once-weekly (QW) bisphosphonate (alendronate 35 or 70 mg) or once-daily (QD) bisphosphonate (alendronate 5 or 10 mg or risedronate 5 mg) were identified from an administrative claims database comprising 30 health plans. The QW and QD study cohorts were followed for 12 months after the index prescription. Medication possession ratio (MPR) was used to estimate compliance while persistence was calculated as the number of days from the initial prescription to a lapse of >30 days after completion of the previous refill. RESULTS: Between 1997 and 2002, a total of 2741 post-menopausal osteoporotic women who were prescribed a bisphosphonate (alendronate QW = 731; alendronate or risedronate QD = 2010) were identified (mean age = 63.7 years). Average MPR for the combined study cohorts was 60.6%. However, QW users had a significantly higher MPR than QD users (69.2% QW vs 57.6% QD, t =-7.51 p < 0.0001). Treatment persistence was significantly longer among the QW users than QD users (227 vs 185 days to discontinuation, respectively, log rank, p < 0.0001). Also, approximately 44% of weekly bisphosphonate users and 32% of daily bisphosphonate users persisted with their therapy at the end of 12 months. CONCLUSIONS: Post-menopausal women prescribed a weekly bisphosphonate regimen had significantly higher rates of compliance and longer persistence compared with those taking a more frequent, daily dosing regimen. However, rates for both regimens were less than desirable. These data demonstrate that less frequent dosing increases persistency, which is needed to obtain maximal long-term therapeutic benefits.

Health Economics Review, Nov 5, 2022

Objectives: Key challenges for a joint European Health Technology Assessment (HTA) include consol... more Objectives: Key challenges for a joint European Health Technology Assessment (HTA) include consolidated approaches towards the choice of adequate comparator(s), selection of endpoints that are relevant to patients with a given disease, dealing with remaining uncertainties as well as transparent and consistent management of related processes. We aimed to further crystallize related core domains within these four areas that warrant further research and scrutiny. Methods: Building on the outcomes of a previously conducted questionnaire survey, four key areas, processes, uncertainty, comparator choice and endpoint selection, were identified. At the inaugural convention of the European Access Academy dedicated working groups were established defining and prioritizing core domains for each of the four areas. The working groups consisted of ~ 10 participants each, representing all relevant stakeholder groups (patients/ clinicians/ regulators/ HTA & payers/ academia/ industry). Story books identifying the work assignments were shared in advance. Two leads and one note taker per working group facilitated the process. All rankings were conducted on an ordinal Likert Response Scale scoring from 1 (low priority) to 7 (high priority). Results: Identified key domains include for processes: i) address (resource-) challenge of multiple PICOs (Patient/ Intervention/ Comparator/ Outcomes), ii) time and capacity challenges, iii) integrating all involved stakeholders, iv) conflicts and aligning between different multinational stakeholders, v) interaction with health technology developer; for uncertainty: i) early and inclusive collaboration, ii) agreement on feasibility of RCT and acceptance of uncertainty, iii) alignment on closing evidence gaps, iv) capacity gaps; for comparator choice: i) criteria for the choice of comparator in an increasingly fragmented treatment landscape, ii) reasonable number of comparators in PICOs, iii) shape Early Advice so that comparator fulfils both regulatory and HTA needs, iv) acceptability of Indirect Treatment Comparisons (ITC), v) ensure broad stakeholder involvement in comparator selection; for endpoint selection: i) approaching new

Health Economics Review, Jun 2, 2022

Objectives: We conducted a multi-stakeholder survey to determine key areas where a joint European... more Objectives: We conducted a multi-stakeholder survey to determine key areas where a joint European health technology assessment (HTA) could provide 'additional benefit' compared to the status quo of many parallel independent national and subnational assessments. Methods: Leveraging three iterative Delphi cycles, a semiquantitative questionnaire was developed covering evidence challenges and heterogeneity of value drivers within HTAs across Europe with a focus on hematology/ oncology. The questionnaire consisted of five sections: i) background information; ii) value drivers in HTA assessments today; iii) evolving evidence challenges; iv) heterogeneity of value drivers across Europe; v) impact of Europe's Beating Cancer Plan (EBCP). The questionnaire was circulated across n = 189 stakeholder institutions comprising HTA and regulatory bodies, clinical oncology associations, patient representatives, and industry associations. Results: N = 30 responses were received (HTA bodies: 9; regulators: 10; patients' and physicians' associations: 3 each; industry: 5). Overall, 17 countries and EU level institutions were represented in the responses. Consistency across countries and stakeholder groups was high. Most relevant value drivers in HTAs today (scale 1, low to 5, high) were clinical trial design (mean 4.45), right endpoints (mean 4.40), and size of comparative effect (mean 4.33). Small patient numbers (mean 4.28) and innovative study designs (mean 4.1) were considered the most relevant evolving evidence challenges. Heterogeneity between regulatory and HTA evidence requirements and heterogeneity of the various national treatment standards and national HTA evidence requirements was high. All clinical and patient participants stated to have been with EBCP initiatives. Conclusions: For a European HTA to provide an 'additional benefit' over the multitude of existing national assessments key methodological and process challenges need to be addressed.

Health Economics Review

Objectives Key challenges for a joint European Health Technology Assessment (HTA) include consoli... more Objectives Key challenges for a joint European Health Technology Assessment (HTA) include consolidated approaches towards the choice of adequate comparator(s), selection of endpoints that are relevant to patients with a given disease, dealing with remaining uncertainties as well as transparent and consistent management of related processes. We aimed to further crystallize related core domains within these four areas that warrant further research and scrutiny. Methods Building on the outcomes of a previously conducted questionnaire survey, four key areas, processes, uncertainty, comparator choice and endpoint selection, were identified. At the inaugural convention of the European Access Academy dedicated working groups were established defining and prioritizing core domains for each of the four areas. The working groups consisted of ~ 10 participants each, representing all relevant stakeholder groups (patients/ clinicians/ regulators/ HTA & payers/ academia/ industry). Story books id...

Health Economics Review

Objectives We conducted a multi-stakeholder survey to determine key areas where a joint European ... more Objectives We conducted a multi-stakeholder survey to determine key areas where a joint European health technology assessment (HTA) could provide ‘additional benefit’ compared to the status quo of many parallel independent national and subnational assessments. Methods Leveraging three iterative Delphi cycles, a semiquantitative questionnaire was developed covering evidence challenges and heterogeneity of value drivers within HTAs across Europe with a focus on hematology/oncology. The questionnaire consisted of five sections: i) background information; ii) value drivers in HTA assessments today; iii) evolving evidence challenges; iv) heterogeneity of value drivers across Europe; v) impact of Europe’s Beating Cancer Plan (EBCP). The questionnaire was circulated across n = 189 stakeholder institutions comprising HTA and regulatory bodies, clinical oncology associations, patient representatives, and industry associations. Results N = 30 responses were received (HTA bodies: 9; regulators...

Objectives: The HTA Core Model ® was developed to improve the transferability of health technolog... more Objectives: The HTA Core Model ® was developed to improve the transferability of health technology assessment (HTA) between settings. The model has been used by HTA agencies but is also of interest to manufacturers, for improving internal evidence generation and communicating with other HTA stakeholders. To establish if the model is fit for purpose from an industry perspective, the pharmaceutical company Roche, collaborating with the European Network for HTA (EUnetHTA), conducted an assessment of the model. Methods: A questionnaire was developed to evaluate all assessment elements in the HTA Core Model v2.0 for their usefulness in meeting payers' evidence needs and demonstrating value. The questionnaire was completed by country affiliate teams working in evidence generation and reimbursement submissions for pharmaceuticals. Survey results were discussed in workshops to ensure consistency and alignment between teams. Results: The questionnaire was completed by six teams. An additional team from global pricing and market access participated in workshops. Model domains pertaining to the health problem and current technology use, technology description, clinical effectiveness, and economic value were considered most important because they meet payers' evidence needs. Overall, the model was considered useful to improve the efficiency of HTA evidence generation, share evidence internally, and communicate value to payers and HTA agencies. Conclusions: From an industry perspective, the HTA Core Model provides a useful framework and common terminology for efficient generation of transferable HTA evidence. The timeliness, efficiency, and transparency of HTA processes could be improved by a more standardized approach to HTA across settings.

International Journal of Technology Assessment in Health Care, 2020

The European Network for Health Technology Assessment (EUnetHTA) organizes an annual Forum with s... more The European Network for Health Technology Assessment (EUnetHTA) organizes an annual Forum with stakeholders to receive feedback on its activities, processes, and outputs produced. The fourth edition of the EUnetHTA Forum brought together representatives of HTA bodies, patient organizations, healthcare professionals (HCPs), academia, payers, regulators, and industry. The aim of this paper is to provide an overview of the highlights presented at the 2019 EUnetHTA Forum, reporting the main items and themes discussed in the plenary panel and breakout sessions. The leading topic was the concept of unmet medical need seen from different stakeholders' perspectives. Breakout sessions covered the joint production of assessment reports and engagement with payers, patients, and HCPs. Synergies, pragmatism, and inclusiveness across Member States and stakeholders were emphasized as leading factors to put in place a collaboration that serves the interest of patients and public health in a tr...

Health Economics Review, 2018

Background: Health Technology Assessments (HTA) procedures differ substantially across the variou... more Background: Health Technology Assessments (HTA) procedures differ substantially across the various European countries. We reviewed recent appraisals of a pharmaceutical manufacturer in three major European markets (France; Italy; Germany) and identified and categorized related decision drivers. Methods: New marketing authorisation between January 2011 and August 2017, and Roche being the Marketing Authorization Holder, were included. Outcome of HTA appraisals by the Haute Autorité de Santé (HAS), Agenzia Italiana del Farmaco (AIFA), and Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) were reviewed. Respective decision drivers were identified and commonalities and differences across the three countries were determined leveraging the EUnetHTA conceptual taxonomy (i.e. the 9 domains of the EUnetHTA core model). Results: Within that time period Roche received European marketing authorization for eight new molecular entities (10 indications, respectively). Outcome of HTA appraisals was heterogeneous across the three countries. However, the four clinical domains of the EUnetHTA core model were driving the national HTA appraisals, with the clinical effectiveness domain being of most importance. Important drivers related to the other three clinical domains included the target patient population (subgroups, Germany), the current management of the condition (unmet need, Italy), the regulatory status (Orphan Designation, Germany), as well as safety considerations (all three countries). Average time between EMA approval and full commercial availability of new medicines was 63 (Germany), 459 (Italy), and 557 days (France). Conclusions: The clinical domains of the EUnetHTA framework are mainly driven by national HTA appraisals, providing a suitable starting point for further developing a joint European view on value and evidence. Underlying topics and issues still reveal considerable differences.

International Journal of Technology Assessment in Health Care, 2018

Introduction:More than 50 HTA agencies evaluate the clinical value of medicines in Europe, result... more Introduction:More than 50 HTA agencies evaluate the clinical value of medicines in Europe, resulting in duplication of work for HTA agencies and manufacturers, and lengthy and variable time to reimbursement for patients across Europe. A consistent, single European relative clinical scientific benefit assessment of medicines could become a key element in ensuring patients get equitable and timely access across Europe. The European Network for Health Technology Assessment (EUnetHTA) is responsible under Joint Action 3 (JA3, 2016–2020) to pilot more than 30 Relative Effectiveness Assessments (REAs) of medicines. The first EUnetHTA JA3 REA pilots are now being completed and Roche, with its participation in the REA pilot for alectinib, has gathered relevant experience.Methods:The goal of this analysis is to summarize and reflect upon the experience with one of the first EUnetHTA REA assessments in JA3. The authors also propose potential process improvements.Results:The experience with th...

International Journal of Technology Assessment in Health Care, 2018

Objectives:The HTA Core Model® was developed to improve the transferability of health technology ... more Objectives:The HTA Core Model® was developed to improve the transferability of health technology assessment (HTA) between settings. The model has been used by HTA agencies but is also of interest to manufacturers, for improving internal evidence generation and communicating with other HTA stakeholders. To establish if the model is fit for purpose from an industry perspective, the pharmaceutical company Roche, collaborating with the European Network for HTA (EUnetHTA), conducted an assessment of the model.Methods:A questionnaire was developed to evaluate all assessment elements in the HTA Core Model v2.0 for their usefulness in meeting payers’ evidence needs and demonstrating value. The questionnaire was completed by country affiliate teams working in evidence generation and reimbursement submissions for pharmaceuticals. Survey results were discussed in workshops to ensure consistency and alignment between teams.Results:The questionnaire was completed by six teams. An additional team...

Value in Health, 2001

also resulted in a positive short-and long-term health economic benefit in acutely ill medical pa... more also resulted in a positive short-and long-term health economic benefit in acutely ill medical patients. The health-economic benefit of enoxaparin was positively related with the length of the follow-up period and a higher risk for recurrence of VTE and mortality in asymptomatic patients.

Value in Health, 2014

The German market access system for drugs have been changed significantly in the last years, by i... more The German market access system for drugs have been changed significantly in the last years, by introducing a similar focus on benefit assessment as in the French system. The research question remains whether they produce consistent results in terms of additional benefit (AB) for pharmaceuticals which have passed the assessment in both systems. MethOds: The G-BA and IQWiG as well as the Transparency Commission (TC) databases were searched systematically to identify those products, which have been processed in both systems between Jan 2011 and Dec 2013. For further comparison a data grid consisting of 26 items for evaluation has been developed including study comparator, primary clinical endpoints, health related quality of life inclusion. Results: Overall, 140 new therapies have been assessed in France by TC, and 80 in Germany by the G-BA. According to inclusion criteria, 44 products could be identified which have passed through both systems including 7 orphan drugs. Thirteen products (30%) had no AB granted by both Agencies, whereas 9 (20%) were in both cases granted with a minor AB, (assuming that "minor" values are equivalent between the two systems), amounting to 22/44 cases with a similar resolution. Five cases (11%) showed a discrepancy in added benefit, all times TC = no and G-BA = yes. However, varying magnitudes appeared to be the greatest difference (n = 17 (39%) remaining drugs), conditioned by lacking concordance of both scale grade systems. cOnclusiOns: Decisions of the agencies in both countries show partial heterogeneity in driving criteria like benefit levels (ASMR and AB). Although the evidence package for initial assessment in both countries is largely similar, preliminary results suggest their contextualization and scales are different. Further analysis based on results of the grid is needed to better assess criteria leading to different benefit levels and their reimbursement impact.

Value in Health, 2005

Post-menopausal osteoporosis has significant health care costs and impacts quality of life. The o... more Post-menopausal osteoporosis has significant health care costs and impacts quality of life. The objective of this analysis was to assess the cost-effectiveness of risedronate compared to calcium + vitamin D, etidronate, alendronate, and ibandronate in high-risk osteoporotic patients in Germany. METHODS: A validated model (Tosteson, 2001) was used to estimate the impact of therapy on hip and vertebral fractures, costs, and quality adjusted life years (QALYs). The analysis included women 70 years with a BMD T-Score of <-2.5 and a history of vertebral fracture, treated over 3 years. The model further simulated downstream costs and QALYs for a 10-year period. Country-specific data included general population mortality, hip and hospitalized vertebral fracture rates, fracture costs, and daily drug prices (risedronate €1.50; etidronate €1.17; alendronate €1.50; ibandronate €1.38). Ibandonate price based on U.S. pricing relative to risedronate, as German pricing not available. Hip and vertebral fracture reductions (risedronate 60%, 49%; etidronate 34%, 37%; alendronate 51%, 47%; ibandronate 0% [No efficacy demonstrated], 52% [Using 62% for efficacy does not change results]) were based on published clinical trials. RESULTS: In a cohort of 1000 postmenopausal women with 3 years of treatment the model predicted the following costs, total hip and hospitalized vertebral fractures and QALYs: risedronate (€8.22M, 139, 5451); alendronate (€8.41M, 142, 5447); etidronate (€8.42M, 149, 5441); ibandronate (€9.33M, 159, 5429); calcium + vitamin D (€7.91M, 164, 5427). All bisphosphonates were dominated by risedronate, which was less costly and had better outcomes. Risedronate had a cost per fracture averted of €12,389 and a cost per QALY gained of €13,253, compared to calcium + vitamin D. CONCLUSIONS: The analysis favors the adoption of risedronate therapy for the treatment of postmenopausal osteoporosis compared to other bisphosphonates.