Date: Monday 14th November 2022
Time: 12:00-14:00 GMT | 13:00-15:00 CET
Speakers: Anders Gorst-Rasmussen, Anne Willemsen, Anja Schiel, Dima Samaha, Mihai Rotaru, Lara Wolfson, and Emma Crawford.
Who is this event intended for? Statisticians who either work on regulatory or HTA submissions, or are involved in evaluating HTA submissions. Non-statisticians who want to understand statistical dimensions of HTA.
What is the benefit of attending? We hope this webinar will provide a deeper awareness and understanding of how the new EU HTA regulation may affect not only HTA but regulatory processes.
Registration for this webinar is free, and is open to both Members and Non-Members of PSI. To register for this event, please click here.
Starting in 2025, a system for HTA, not entirely unlike that of EMA for regulatory assessments, will apply to new medicinal products submitted for EMA regulatory review. The system will require manufacturers to submit an HTA evidence dossier broad enough to support each member state’s evaluation of relative effectiveness within the context of their specific healthcare system.
Is this a ripple that will matter mostly for statisticians specializing in HTA – or is it a splash that should be on all pharmaceutical statisticians radar?
|
Speaker |
Biography |
Abstract |
|
|
Anders Gorst-Rasmussen received his PhD in statistics from Aalborg University in 2011 and worked with pharmacoepidemiology and RWE in academia prior to joining the Development organization in Novo Nordisk in 2015. At Novo Nordisk, he has held roles as project statistical lead on various late-phase projects. For the last 2 years, has been working in the HTA area as functional lead for a small team of dedicated HTA statisticians. |
In December 2021, the EU-regulation on HTA (HTAr) was passed. Among other things, HTAr mandates joint EU-level assessment of all new medicines in parallel with the EMA regulatory review, based on a dossier submitted by the manufacturer. Having a joint assessment covering the needs of all 27 EU member states and tied to EMA timelines, is a completely new paradigm that will have major implications for how companies set up and align HTA and regulatory and statistical work. |
 Anne Willemsen |
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute over the past years. Anne has been working for EUnetHTA since 2016 and now heads the Joint Clinical Assessment secretariat of EUnetHTA 21. In her role, she is working to correlate stakeholder interest and maximise efficiency and impact of European collaboration on HTA. She is also involved in developing methods for patient and healthcare professional engagement within the context of these Joint Clinical Assessments. | What is a Joint Clinical Assessment and what requirements are outlined in the HTAR. Furthermore, insights are provided on the scoping process and relevance of the PICO for a JCA. |
|
|
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Norwegian Medicines Agency (NoMA) in 2012. At NoMA she is working as special adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects. She has been Chair of EMA’s Biostatistics Working Party 2017 - 2019 and just finished her 3-year term as Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member of the SAWP and is Member of the new Methodology Working Party (MWP) recently established at EMA. In her role as Team-leader international HTA (iHTA) at NoMA, she has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21. Her particular focus is on parallel EMA-HTA scientific advices, now in the role as Vice-Chair of the JSC-CSCQ. She is furthermore involved in several academic and PPP projects as member of Scientific Advisory boards on subjects such as RWD, Patient reported outcomes, rare diseases, paediatric drug development and decentralized trials and digital tools. |
One of the most discussed issues around the upcoming HTA legislation (HTAR) is the fear of being confronted with an unreasonable number of PICO’s at the time of a joined clinical assessment (JCA). Surprisingly this issue is rarely, if ever, discussed by the applicants during the JSC’s. The assumption must be that the PICO can’t be predicted early in the process and will likely be only determined at the very last moment by the HTAB’s. While there is a scoping process taking place immediately before the start of the JCA it is worth understanding that these PICOs do not arbitrarily change and that they are predictable. I will discuss the theory behind the PICO and how it is supposed to be interpreted by applicants. |
|
|
Dima is a Principal within the Payer Evidence Solution practice at IQVIA. Her recent work has focused on supporting pharmaceutical companies in conceptualizing payer evidence generation strategies, successfully engaging with HTA bodies and decision makers and understanding the implications of the pan -European HTA regulation and the use of real-world evidence in HTA. Prior to joining the consulting world, Dima was an advisor on Innovation and External affairs at l’Institut national d’excellence en sante et en services sociaux (INESSS, Quebec) where she developed strategies to enhance collaborations among key stakeholders and consolidate relationships with International and Canadian HTA bodies. As a consultant to the World Health Organization on HTA, Dima was involved in foundational workstreams on access to medical devices and in the implementation of the first survey on the status and the use of HTA principles following the adoption by the World Health Assembly of the resolution on HTA. Dima has also co-authored several publications on multi-criteria decision analysis ( MCDA) published in peer review medical and scientific journals.Dima has received her Pharm D from the Lebanese American University. She holds a Master of Advances Studies in Health Economics and Management from HEC Universite de Lausanne. |
Dima Samaha will present lessons learned from previously conducted pan-EU assessments under JA3 with a forward looking view on what an additive PICO approach embedding evidence requirements of all Member States could look like. |
 Mihai Rotaru |
Mihai Rotaru works as a Senior Manager Market Access at EFPIA (European Federation of Pharmaceutical Industries and Associations) where he is currently responsible for overall industry engagement in the implementation of the HTA Regulation at EU level. In addition, Mihai is also responsible for oncology policy, biotherapeutics as well as various supply chain issues (Falsified Medicines Directive, shortages and anticounterfeiting efforts). | The EU pharmaceutical industry is keen act as a partner in the development of a fit-for-purpose future system of JCA at EU level. As such, EFPIA is working with its members across a matrix of topics ranging from EU JCA process and implementation at national level, methods, JSC and overall advocacy with an underlying legal support as well. The purpose is to ensure that EFPIA speaks with one voice at EU level but also to ensure that companies are prepared from day 1 to meaningfully participate in the future system and to leverage these efforts into better evidence generation and overall clinical assessment of their innovation. |
|
|
Lara J. Wolfson is Executive Director and Head, Health Technology Assessment (HTA) Statistics in the Biostatistics and Research Decision Sciences (BARDS) organization at MSD Switzerland. Lara holds a PhD in Statistics from Carnegie Mellon University in the US, and an undergraduate degree from Simon Fraser University in Canada. Lara has held academic appointments at University of Waterloo in Canada and Brigham Young University in the US, and has previously worked as a scientist covering health economics, epidemiology, and statistics at the World Health Organization, as well as in the pharmaceutical industry in outcomes research and Market Access at both the Janssen Pharmaceutical Companies of Johnson & Johnson in Belgium and the Merck Center for Observational Research and Real-World Evidence in the US. Lara has authored more than 70 peer-reviewed journal articles, and currently chairs the HTA European Special Interest Group of the PSI (a professional organization for Statisticians in the Pharmaceutical Industry). Lara currently lives in the Greater Zurich Area with her husband, twin sons, and a cat named Hokey Pokey. |
The new paradigm for EU HTA Joint Clinical Assessments will rely heavily on statistical analysis. How can the statistical community prepare for this development? When, where, and how should statisticians be involved? What new capacities and systems needs to be developed? How can academic, government, and industry statisticians effectively collaborate to achieve the EU HTA regulation goals of patient access? In this panel discussion, we will talk with a number of key leaders in the area on these questions and more. |
Date: Monday 14th November 2022
Time: 12:00-14:00 GMT | 13:00-15:00 CET
Speakers: Anders Gorst-Rasmussen, Anne Willemsen, Anja Schiel, Dima Samaha, Mihai Rotaru, Lara Wolfson, and Emma Crawford.
Who is this event intended for? Statisticians who either work on regulatory or HTA submissions, or are involved in evaluating HTA submissions. Non-statisticians who want to understand statistical dimensions of HTA.
What is the benefit of attending? We hope this webinar will provide a deeper awareness and understanding of how the new EU HTA regulation may affect not only HTA but regulatory processes.
Registration for this webinar is free, and is open to both Members and Non-Members of PSI. To register for this event, please click here.
Starting in 2025, a system for HTA, not entirely unlike that of EMA for regulatory assessments, will apply to new medicinal products submitted for EMA regulatory review. The system will require manufacturers to submit an HTA evidence dossier broad enough to support each member state’s evaluation of relative effectiveness within the context of their specific healthcare system.
Is this a ripple that will matter mostly for statisticians specializing in HTA – or is it a splash that should be on all pharmaceutical statisticians radar?
|
Speaker |
Biography |
Abstract |
|
|
Anders Gorst-Rasmussen received his PhD in statistics from Aalborg University in 2011 and worked with pharmacoepidemiology and RWE in academia prior to joining the Development organization in Novo Nordisk in 2015. At Novo Nordisk, he has held roles as project statistical lead on various late-phase projects. For the last 2 years, has been working in the HTA area as functional lead for a small team of dedicated HTA statisticians. |
In December 2021, the EU-regulation on HTA (HTAr) was passed. Among other things, HTAr mandates joint EU-level assessment of all new medicines in parallel with the EMA regulatory review, based on a dossier submitted by the manufacturer. Having a joint assessment covering the needs of all 27 EU member states and tied to EMA timelines, is a completely new paradigm that will have major implications for how companies set up and align HTA and regulatory and statistical work. |
| Anne Willemsen |
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute over the past years. Anne has been working for EUnetHTA since 2016 and now heads the Joint Clinical Assessment secretariat of EUnetHTA 21. In her role, she is working to correlate stakeholder interest and maximise efficiency and impact of European collaboration on HTA. She is also involved in developing methods for patient and healthcare professional engagement within the context of these Joint Clinical Assessments. | What is a Joint Clinical Assessment and what requirements are outlined in the HTAR. Furthermore, insights are provided on the scoping process and relevance of the PICO for a JCA. |
|
|
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Norwegian Medicines Agency (NoMA) in 2012. At NoMA she is working as special adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects. She has been Chair of EMA’s Biostatistics Working Party 2017 - 2019 and just finished her 3-year term as Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member of the SAWP and is Member of the new Methodology Working Party (MWP) recently established at EMA. In her role as Team-leader international HTA (iHTA) at NoMA, she has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21. Her particular focus is on parallel EMA-HTA scientific advices, now in the role as Vice-Chair of the JSC-CSCQ. She is furthermore involved in several academic and PPP projects as member of Scientific Advisory boards on subjects such as RWD, Patient reported outcomes, rare diseases, paediatric drug development and decentralized trials and digital tools. |
One of the most discussed issues around the upcoming HTA legislation (HTAR) is the fear of being confronted with an unreasonable number of PICO’s at the time of a joined clinical assessment (JCA). Surprisingly this issue is rarely, if ever, discussed by the applicants during the JSC’s. The assumption must be that the PICO can’t be predicted early in the process and will likely be only determined at the very last moment by the HTAB’s. While there is a scoping process taking place immediately before the start of the JCA it is worth understanding that these PICOs do not arbitrarily change and that they are predictable. I will discuss the theory behind the PICO and how it is supposed to be interpreted by applicants. |
|
|
Dima is a Principal within the Payer Evidence Solution practice at IQVIA. Her recent work has focused on supporting pharmaceutical companies in conceptualizing payer evidence generation strategies, successfully engaging with HTA bodies and decision makers and understanding the implications of the pan -European HTA regulation and the use of real-world evidence in HTA. Prior to joining the consulting world, Dima was an advisor on Innovation and External affairs at l’Institut national d’excellence en sante et en services sociaux (INESSS, Quebec) where she developed strategies to enhance collaborations among key stakeholders and consolidate relationships with International and Canadian HTA bodies. As a consultant to the World Health Organization on HTA, Dima was involved in foundational workstreams on access to medical devices and in the implementation of the first survey on the status and the use of HTA principles following the adoption by the World Health Assembly of the resolution on HTA. Dima has also co-authored several publications on multi-criteria decision analysis ( MCDA) published in peer review medical and scientific journals.Dima has received her Pharm D from the Lebanese American University. She holds a Master of Advances Studies in Health Economics and Management from HEC Universite de Lausanne. |
Dima Samaha will present lessons learned from previously conducted pan-EU assessments under JA3 with a forward looking view on what an additive PICO approach embedding evidence requirements of all Member States could look like. |
Mihai Rotaru |
Mihai Rotaru works as a Senior Manager Market Access at EFPIA (European Federation of Pharmaceutical Industries and Associations) where he is currently responsible for overall industry engagement in the implementation of the HTA Regulation at EU level. In addition, Mihai is also responsible for oncology policy, biotherapeutics as well as various supply chain issues (Falsified Medicines Directive, shortages and anticounterfeiting efforts). | The EU pharmaceutical industry is keen act as a partner in the development of a fit-for-purpose future system of JCA at EU level. As such, EFPIA is working with its members across a matrix of topics ranging from EU JCA process and implementation at national level, methods, JSC and overall advocacy with an underlying legal support as well. The purpose is to ensure that EFPIA speaks with one voice at EU level but also to ensure that companies are prepared from day 1 to meaningfully participate in the future system and to leverage these efforts into better evidence generation and overall clinical assessment of their innovation. |
|
|
Lara J. Wolfson is Executive Director and Head, Health Technology Assessment (HTA) Statistics in the Biostatistics and Research Decision Sciences (BARDS) organization at MSD Switzerland. Lara holds a PhD in Statistics from Carnegie Mellon University in the US, and an undergraduate degree from Simon Fraser University in Canada. Lara has held academic appointments at University of Waterloo in Canada and Brigham Young University in the US, and has previously worked as a scientist covering health economics, epidemiology, and statistics at the World Health Organization, as well as in the pharmaceutical industry in outcomes research and Market Access at both the Janssen Pharmaceutical Companies of Johnson & Johnson in Belgium and the Merck Center for Observational Research and Real-World Evidence in the US. Lara has authored more than 70 peer-reviewed journal articles, and currently chairs the HTA European Special Interest Group of the PSI (a professional organization for Statisticians in the Pharmaceutical Industry). Lara currently lives in the Greater Zurich Area with her husband, twin sons, and a cat named Hokey Pokey. |
The new paradigm for EU HTA Joint Clinical Assessments will rely heavily on statistical analysis. How can the statistical community prepare for this development? When, where, and how should statisticians be involved? What new capacities and systems needs to be developed? How can academic, government, and industry statisticians effectively collaborate to achieve the EU HTA regulation goals of patient access? In this panel discussion, we will talk with a number of key leaders in the area on these questions and more. |
Date: Monday 14th November 2022
Time: 12:00-14:00 GMT | 13:00-15:00 CET
Speakers: Anders Gorst-Rasmussen, Anne Willemsen, Anja Schiel, Dima Samaha, Mihai Rotaru, Lara Wolfson, and Emma Crawford.
Who is this event intended for? Statisticians who either work on regulatory or HTA submissions, or are involved in evaluating HTA submissions. Non-statisticians who want to understand statistical dimensions of HTA.
What is the benefit of attending? We hope this webinar will provide a deeper awareness and understanding of how the new EU HTA regulation may affect not only HTA but regulatory processes.
Registration for this webinar is free, and is open to both Members and Non-Members of PSI. To register for this event, please click here.
Starting in 2025, a system for HTA, not entirely unlike that of EMA for regulatory assessments, will apply to new medicinal products submitted for EMA regulatory review. The system will require manufacturers to submit an HTA evidence dossier broad enough to support each member state’s evaluation of relative effectiveness within the context of their specific healthcare system.
Is this a ripple that will matter mostly for statisticians specializing in HTA – or is it a splash that should be on all pharmaceutical statisticians radar?
|
Speaker |
Biography |
Abstract |
|
|
Anders Gorst-Rasmussen received his PhD in statistics from Aalborg University in 2011 and worked with pharmacoepidemiology and RWE in academia prior to joining the Development organization in Novo Nordisk in 2015. At Novo Nordisk, he has held roles as project statistical lead on various late-phase projects. For the last 2 years, has been working in the HTA area as functional lead for a small team of dedicated HTA statisticians. |
In December 2021, the EU-regulation on HTA (HTAr) was passed. Among other things, HTAr mandates joint EU-level assessment of all new medicines in parallel with the EMA regulatory review, based on a dossier submitted by the manufacturer. Having a joint assessment covering the needs of all 27 EU member states and tied to EMA timelines, is a completely new paradigm that will have major implications for how companies set up and align HTA and regulatory and statistical work. |
| Anne Willemsen |
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute over the past years. Anne has been working for EUnetHTA since 2016 and now heads the Joint Clinical Assessment secretariat of EUnetHTA 21. In her role, she is working to correlate stakeholder interest and maximise efficiency and impact of European collaboration on HTA. She is also involved in developing methods for patient and healthcare professional engagement within the context of these Joint Clinical Assessments. | What is a Joint Clinical Assessment and what requirements are outlined in the HTAR. Furthermore, insights are provided on the scoping process and relevance of the PICO for a JCA. |
|
|
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Norwegian Medicines Agency (NoMA) in 2012. At NoMA she is working as special adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects. She has been Chair of EMA’s Biostatistics Working Party 2017 - 2019 and just finished her 3-year term as Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member of the SAWP and is Member of the new Methodology Working Party (MWP) recently established at EMA. In her role as Team-leader international HTA (iHTA) at NoMA, she has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21. Her particular focus is on parallel EMA-HTA scientific advices, now in the role as Vice-Chair of the JSC-CSCQ. She is furthermore involved in several academic and PPP projects as member of Scientific Advisory boards on subjects such as RWD, Patient reported outcomes, rare diseases, paediatric drug development and decentralized trials and digital tools. |
One of the most discussed issues around the upcoming HTA legislation (HTAR) is the fear of being confronted with an unreasonable number of PICO’s at the time of a joined clinical assessment (JCA). Surprisingly this issue is rarely, if ever, discussed by the applicants during the JSC’s. The assumption must be that the PICO can’t be predicted early in the process and will likely be only determined at the very last moment by the HTAB’s. While there is a scoping process taking place immediately before the start of the JCA it is worth understanding that these PICOs do not arbitrarily change and that they are predictable. I will discuss the theory behind the PICO and how it is supposed to be interpreted by applicants. |
|
|
Dima is a Principal within the Payer Evidence Solution practice at IQVIA. Her recent work has focused on supporting pharmaceutical companies in conceptualizing payer evidence generation strategies, successfully engaging with HTA bodies and decision makers and understanding the implications of the pan -European HTA regulation and the use of real-world evidence in HTA. Prior to joining the consulting world, Dima was an advisor on Innovation and External affairs at l’Institut national d’excellence en sante et en services sociaux (INESSS, Quebec) where she developed strategies to enhance collaborations among key stakeholders and consolidate relationships with International and Canadian HTA bodies. As a consultant to the World Health Organization on HTA, Dima was involved in foundational workstreams on access to medical devices and in the implementation of the first survey on the status and the use of HTA principles following the adoption by the World Health Assembly of the resolution on HTA. Dima has also co-authored several publications on multi-criteria decision analysis ( MCDA) published in peer review medical and scientific journals.Dima has received her Pharm D from the Lebanese American University. She holds a Master of Advances Studies in Health Economics and Management from HEC Universite de Lausanne. |
Dima Samaha will present lessons learned from previously conducted pan-EU assessments under JA3 with a forward looking view on what an additive PICO approach embedding evidence requirements of all Member States could look like. |
Mihai Rotaru |
Mihai Rotaru works as a Senior Manager Market Access at EFPIA (European Federation of Pharmaceutical Industries and Associations) where he is currently responsible for overall industry engagement in the implementation of the HTA Regulation at EU level. In addition, Mihai is also responsible for oncology policy, biotherapeutics as well as various supply chain issues (Falsified Medicines Directive, shortages and anticounterfeiting efforts). | The EU pharmaceutical industry is keen act as a partner in the development of a fit-for-purpose future system of JCA at EU level. As such, EFPIA is working with its members across a matrix of topics ranging from EU JCA process and implementation at national level, methods, JSC and overall advocacy with an underlying legal support as well. The purpose is to ensure that EFPIA speaks with one voice at EU level but also to ensure that companies are prepared from day 1 to meaningfully participate in the future system and to leverage these efforts into better evidence generation and overall clinical assessment of their innovation. |
|
|
Lara J. Wolfson is Executive Director and Head, Health Technology Assessment (HTA) Statistics in the Biostatistics and Research Decision Sciences (BARDS) organization at MSD Switzerland. Lara holds a PhD in Statistics from Carnegie Mellon University in the US, and an undergraduate degree from Simon Fraser University in Canada. Lara has held academic appointments at University of Waterloo in Canada and Brigham Young University in the US, and has previously worked as a scientist covering health economics, epidemiology, and statistics at the World Health Organization, as well as in the pharmaceutical industry in outcomes research and Market Access at both the Janssen Pharmaceutical Companies of Johnson & Johnson in Belgium and the Merck Center for Observational Research and Real-World Evidence in the US. Lara has authored more than 70 peer-reviewed journal articles, and currently chairs the HTA European Special Interest Group of the PSI (a professional organization for Statisticians in the Pharmaceutical Industry). Lara currently lives in the Greater Zurich Area with her husband, twin sons, and a cat named Hokey Pokey. |
The new paradigm for EU HTA Joint Clinical Assessments will rely heavily on statistical analysis. How can the statistical community prepare for this development? When, where, and how should statisticians be involved? What new capacities and systems needs to be developed? How can academic, government, and industry statisticians effectively collaborate to achieve the EU HTA regulation goals of patient access? In this panel discussion, we will talk with a number of key leaders in the area on these questions and more. |
Date: Monday 14th November 2022
Time: 12:00-14:00 GMT | 13:00-15:00 CET
Speakers: Anders Gorst-Rasmussen, Anne Willemsen, Anja Schiel, Dima Samaha, Mihai Rotaru, Lara Wolfson, and Emma Crawford.
Who is this event intended for? Statisticians who either work on regulatory or HTA submissions, or are involved in evaluating HTA submissions. Non-statisticians who want to understand statistical dimensions of HTA.
What is the benefit of attending? We hope this webinar will provide a deeper awareness and understanding of how the new EU HTA regulation may affect not only HTA but regulatory processes.
Registration for this webinar is free, and is open to both Members and Non-Members of PSI. To register for this event, please click here.
Starting in 2025, a system for HTA, not entirely unlike that of EMA for regulatory assessments, will apply to new medicinal products submitted for EMA regulatory review. The system will require manufacturers to submit an HTA evidence dossier broad enough to support each member state’s evaluation of relative effectiveness within the context of their specific healthcare system.
Is this a ripple that will matter mostly for statisticians specializing in HTA – or is it a splash that should be on all pharmaceutical statisticians radar?
|
Speaker |
Biography |
Abstract |
|
|
Anders Gorst-Rasmussen received his PhD in statistics from Aalborg University in 2011 and worked with pharmacoepidemiology and RWE in academia prior to joining the Development organization in Novo Nordisk in 2015. At Novo Nordisk, he has held roles as project statistical lead on various late-phase projects. For the last 2 years, has been working in the HTA area as functional lead for a small team of dedicated HTA statisticians. |
In December 2021, the EU-regulation on HTA (HTAr) was passed. Among other things, HTAr mandates joint EU-level assessment of all new medicines in parallel with the EMA regulatory review, based on a dossier submitted by the manufacturer. Having a joint assessment covering the needs of all 27 EU member states and tied to EMA timelines, is a completely new paradigm that will have major implications for how companies set up and align HTA and regulatory and statistical work. |
| Anne Willemsen |
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute over the past years. Anne has been working for EUnetHTA since 2016 and now heads the Joint Clinical Assessment secretariat of EUnetHTA 21. In her role, she is working to correlate stakeholder interest and maximise efficiency and impact of European collaboration on HTA. She is also involved in developing methods for patient and healthcare professional engagement within the context of these Joint Clinical Assessments. | What is a Joint Clinical Assessment and what requirements are outlined in the HTAR. Furthermore, insights are provided on the scoping process and relevance of the PICO for a JCA. |
|
|
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Norwegian Medicines Agency (NoMA) in 2012. At NoMA she is working as special adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects. She has been Chair of EMA’s Biostatistics Working Party 2017 - 2019 and just finished her 3-year term as Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member of the SAWP and is Member of the new Methodology Working Party (MWP) recently established at EMA. In her role as Team-leader international HTA (iHTA) at NoMA, she has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21. Her particular focus is on parallel EMA-HTA scientific advices, now in the role as Vice-Chair of the JSC-CSCQ. She is furthermore involved in several academic and PPP projects as member of Scientific Advisory boards on subjects such as RWD, Patient reported outcomes, rare diseases, paediatric drug development and decentralized trials and digital tools. |
One of the most discussed issues around the upcoming HTA legislation (HTAR) is the fear of being confronted with an unreasonable number of PICO’s at the time of a joined clinical assessment (JCA). Surprisingly this issue is rarely, if ever, discussed by the applicants during the JSC’s. The assumption must be that the PICO can’t be predicted early in the process and will likely be only determined at the very last moment by the HTAB’s. While there is a scoping process taking place immediately before the start of the JCA it is worth understanding that these PICOs do not arbitrarily change and that they are predictable. I will discuss the theory behind the PICO and how it is supposed to be interpreted by applicants. |
|
|
Dima is a Principal within the Payer Evidence Solution practice at IQVIA. Her recent work has focused on supporting pharmaceutical companies in conceptualizing payer evidence generation strategies, successfully engaging with HTA bodies and decision makers and understanding the implications of the pan -European HTA regulation and the use of real-world evidence in HTA. Prior to joining the consulting world, Dima was an advisor on Innovation and External affairs at l’Institut national d’excellence en sante et en services sociaux (INESSS, Quebec) where she developed strategies to enhance collaborations among key stakeholders and consolidate relationships with International and Canadian HTA bodies. As a consultant to the World Health Organization on HTA, Dima was involved in foundational workstreams on access to medical devices and in the implementation of the first survey on the status and the use of HTA principles following the adoption by the World Health Assembly of the resolution on HTA. Dima has also co-authored several publications on multi-criteria decision analysis ( MCDA) published in peer review medical and scientific journals.Dima has received her Pharm D from the Lebanese American University. She holds a Master of Advances Studies in Health Economics and Management from HEC Universite de Lausanne. |
Dima Samaha will present lessons learned from previously conducted pan-EU assessments under JA3 with a forward looking view on what an additive PICO approach embedding evidence requirements of all Member States could look like. |
Mihai Rotaru |
Mihai Rotaru works as a Senior Manager Market Access at EFPIA (European Federation of Pharmaceutical Industries and Associations) where he is currently responsible for overall industry engagement in the implementation of the HTA Regulation at EU level. In addition, Mihai is also responsible for oncology policy, biotherapeutics as well as various supply chain issues (Falsified Medicines Directive, shortages and anticounterfeiting efforts). | The EU pharmaceutical industry is keen act as a partner in the development of a fit-for-purpose future system of JCA at EU level. As such, EFPIA is working with its members across a matrix of topics ranging from EU JCA process and implementation at national level, methods, JSC and overall advocacy with an underlying legal support as well. The purpose is to ensure that EFPIA speaks with one voice at EU level but also to ensure that companies are prepared from day 1 to meaningfully participate in the future system and to leverage these efforts into better evidence generation and overall clinical assessment of their innovation. |
|
|
Lara J. Wolfson is Executive Director and Head, Health Technology Assessment (HTA) Statistics in the Biostatistics and Research Decision Sciences (BARDS) organization at MSD Switzerland. Lara holds a PhD in Statistics from Carnegie Mellon University in the US, and an undergraduate degree from Simon Fraser University in Canada. Lara has held academic appointments at University of Waterloo in Canada and Brigham Young University in the US, and has previously worked as a scientist covering health economics, epidemiology, and statistics at the World Health Organization, as well as in the pharmaceutical industry in outcomes research and Market Access at both the Janssen Pharmaceutical Companies of Johnson & Johnson in Belgium and the Merck Center for Observational Research and Real-World Evidence in the US. Lara has authored more than 70 peer-reviewed journal articles, and currently chairs the HTA European Special Interest Group of the PSI (a professional organization for Statisticians in the Pharmaceutical Industry). Lara currently lives in the Greater Zurich Area with her husband, twin sons, and a cat named Hokey Pokey. |
The new paradigm for EU HTA Joint Clinical Assessments will rely heavily on statistical analysis. How can the statistical community prepare for this development? When, where, and how should statisticians be involved? What new capacities and systems needs to be developed? How can academic, government, and industry statisticians effectively collaborate to achieve the EU HTA regulation goals of patient access? In this panel discussion, we will talk with a number of key leaders in the area on these questions and more. |
Date: Monday 14th November 2022
Time: 12:00-14:00 GMT | 13:00-15:00 CET
Speakers: Anders Gorst-Rasmussen, Anne Willemsen, Anja Schiel, Dima Samaha, Mihai Rotaru, Lara Wolfson, and Emma Crawford.
Who is this event intended for? Statisticians who either work on regulatory or HTA submissions, or are involved in evaluating HTA submissions. Non-statisticians who want to understand statistical dimensions of HTA.
What is the benefit of attending? We hope this webinar will provide a deeper awareness and understanding of how the new EU HTA regulation may affect not only HTA but regulatory processes.
Registration for this webinar is free, and is open to both Members and Non-Members of PSI. To register for this event, please click here.
Starting in 2025, a system for HTA, not entirely unlike that of EMA for regulatory assessments, will apply to new medicinal products submitted for EMA regulatory review. The system will require manufacturers to submit an HTA evidence dossier broad enough to support each member state’s evaluation of relative effectiveness within the context of their specific healthcare system.
Is this a ripple that will matter mostly for statisticians specializing in HTA – or is it a splash that should be on all pharmaceutical statisticians radar?
|
Speaker |
Biography |
Abstract |
|
|
Anders Gorst-Rasmussen received his PhD in statistics from Aalborg University in 2011 and worked with pharmacoepidemiology and RWE in academia prior to joining the Development organization in Novo Nordisk in 2015. At Novo Nordisk, he has held roles as project statistical lead on various late-phase projects. For the last 2 years, has been working in the HTA area as functional lead for a small team of dedicated HTA statisticians. |
In December 2021, the EU-regulation on HTA (HTAr) was passed. Among other things, HTAr mandates joint EU-level assessment of all new medicines in parallel with the EMA regulatory review, based on a dossier submitted by the manufacturer. Having a joint assessment covering the needs of all 27 EU member states and tied to EMA timelines, is a completely new paradigm that will have major implications for how companies set up and align HTA and regulatory and statistical work. |
| Anne Willemsen |
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute over the past years. Anne has been working for EUnetHTA since 2016 and now heads the Joint Clinical Assessment secretariat of EUnetHTA 21. In her role, she is working to correlate stakeholder interest and maximise efficiency and impact of European collaboration on HTA. She is also involved in developing methods for patient and healthcare professional engagement within the context of these Joint Clinical Assessments. | What is a Joint Clinical Assessment and what requirements are outlined in the HTAR. Furthermore, insights are provided on the scoping process and relevance of the PICO for a JCA. |
|
|
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Norwegian Medicines Agency (NoMA) in 2012. At NoMA she is working as special adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects. She has been Chair of EMA’s Biostatistics Working Party 2017 - 2019 and just finished her 3-year term as Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member of the SAWP and is Member of the new Methodology Working Party (MWP) recently established at EMA. In her role as Team-leader international HTA (iHTA) at NoMA, she has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21. Her particular focus is on parallel EMA-HTA scientific advices, now in the role as Vice-Chair of the JSC-CSCQ. She is furthermore involved in several academic and PPP projects as member of Scientific Advisory boards on subjects such as RWD, Patient reported outcomes, rare diseases, paediatric drug development and decentralized trials and digital tools. |
One of the most discussed issues around the upcoming HTA legislation (HTAR) is the fear of being confronted with an unreasonable number of PICO’s at the time of a joined clinical assessment (JCA). Surprisingly this issue is rarely, if ever, discussed by the applicants during the JSC’s. The assumption must be that the PICO can’t be predicted early in the process and will likely be only determined at the very last moment by the HTAB’s. While there is a scoping process taking place immediately before the start of the JCA it is worth understanding that these PICOs do not arbitrarily change and that they are predictable. I will discuss the theory behind the PICO and how it is supposed to be interpreted by applicants. |
|
|
Dima is a Principal within the Payer Evidence Solution practice at IQVIA. Her recent work has focused on supporting pharmaceutical companies in conceptualizing payer evidence generation strategies, successfully engaging with HTA bodies and decision makers and understanding the implications of the pan -European HTA regulation and the use of real-world evidence in HTA. Prior to joining the consulting world, Dima was an advisor on Innovation and External affairs at l’Institut national d’excellence en sante et en services sociaux (INESSS, Quebec) where she developed strategies to enhance collaborations among key stakeholders and consolidate relationships with International and Canadian HTA bodies. As a consultant to the World Health Organization on HTA, Dima was involved in foundational workstreams on access to medical devices and in the implementation of the first survey on the status and the use of HTA principles following the adoption by the World Health Assembly of the resolution on HTA. Dima has also co-authored several publications on multi-criteria decision analysis ( MCDA) published in peer review medical and scientific journals.Dima has received her Pharm D from the Lebanese American University. She holds a Master of Advances Studies in Health Economics and Management from HEC Universite de Lausanne. |
Dima Samaha will present lessons learned from previously conducted pan-EU assessments under JA3 with a forward looking view on what an additive PICO approach embedding evidence requirements of all Member States could look like. |
Mihai Rotaru |
Mihai Rotaru works as a Senior Manager Market Access at EFPIA (European Federation of Pharmaceutical Industries and Associations) where he is currently responsible for overall industry engagement in the implementation of the HTA Regulation at EU level. In addition, Mihai is also responsible for oncology policy, biotherapeutics as well as various supply chain issues (Falsified Medicines Directive, shortages and anticounterfeiting efforts). | The EU pharmaceutical industry is keen act as a partner in the development of a fit-for-purpose future system of JCA at EU level. As such, EFPIA is working with its members across a matrix of topics ranging from EU JCA process and implementation at national level, methods, JSC and overall advocacy with an underlying legal support as well. The purpose is to ensure that EFPIA speaks with one voice at EU level but also to ensure that companies are prepared from day 1 to meaningfully participate in the future system and to leverage these efforts into better evidence generation and overall clinical assessment of their innovation. |
|
|
Lara J. Wolfson is Executive Director and Head, Health Technology Assessment (HTA) Statistics in the Biostatistics and Research Decision Sciences (BARDS) organization at MSD Switzerland. Lara holds a PhD in Statistics from Carnegie Mellon University in the US, and an undergraduate degree from Simon Fraser University in Canada. Lara has held academic appointments at University of Waterloo in Canada and Brigham Young University in the US, and has previously worked as a scientist covering health economics, epidemiology, and statistics at the World Health Organization, as well as in the pharmaceutical industry in outcomes research and Market Access at both the Janssen Pharmaceutical Companies of Johnson & Johnson in Belgium and the Merck Center for Observational Research and Real-World Evidence in the US. Lara has authored more than 70 peer-reviewed journal articles, and currently chairs the HTA European Special Interest Group of the PSI (a professional organization for Statisticians in the Pharmaceutical Industry). Lara currently lives in the Greater Zurich Area with her husband, twin sons, and a cat named Hokey Pokey. |
The new paradigm for EU HTA Joint Clinical Assessments will rely heavily on statistical analysis. How can the statistical community prepare for this development? When, where, and how should statisticians be involved? What new capacities and systems needs to be developed? How can academic, government, and industry statisticians effectively collaborate to achieve the EU HTA regulation goals of patient access? In this panel discussion, we will talk with a number of key leaders in the area on these questions and more. |
Date: Monday 14th November 2022
Time: 12:00-14:00 GMT | 13:00-15:00 CET
Speakers: Anders Gorst-Rasmussen, Anne Willemsen, Anja Schiel, Dima Samaha, Mihai Rotaru, Lara Wolfson, and Emma Crawford.
Who is this event intended for? Statisticians who either work on regulatory or HTA submissions, or are involved in evaluating HTA submissions. Non-statisticians who want to understand statistical dimensions of HTA.
What is the benefit of attending? We hope this webinar will provide a deeper awareness and understanding of how the new EU HTA regulation may affect not only HTA but regulatory processes.
Registration for this webinar is free, and is open to both Members and Non-Members of PSI. To register for this event, please click here.
Starting in 2025, a system for HTA, not entirely unlike that of EMA for regulatory assessments, will apply to new medicinal products submitted for EMA regulatory review. The system will require manufacturers to submit an HTA evidence dossier broad enough to support each member state’s evaluation of relative effectiveness within the context of their specific healthcare system.
Is this a ripple that will matter mostly for statisticians specializing in HTA – or is it a splash that should be on all pharmaceutical statisticians radar?
|
Speaker |
Biography |
Abstract |
|
|
Anders Gorst-Rasmussen received his PhD in statistics from Aalborg University in 2011 and worked with pharmacoepidemiology and RWE in academia prior to joining the Development organization in Novo Nordisk in 2015. At Novo Nordisk, he has held roles as project statistical lead on various late-phase projects. For the last 2 years, has been working in the HTA area as functional lead for a small team of dedicated HTA statisticians. |
In December 2021, the EU-regulation on HTA (HTAr) was passed. Among other things, HTAr mandates joint EU-level assessment of all new medicines in parallel with the EMA regulatory review, based on a dossier submitted by the manufacturer. Having a joint assessment covering the needs of all 27 EU member states and tied to EMA timelines, is a completely new paradigm that will have major implications for how companies set up and align HTA and regulatory and statistical work. |
| Anne Willemsen |
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute over the past years. Anne has been working for EUnetHTA since 2016 and now heads the Joint Clinical Assessment secretariat of EUnetHTA 21. In her role, she is working to correlate stakeholder interest and maximise efficiency and impact of European collaboration on HTA. She is also involved in developing methods for patient and healthcare professional engagement within the context of these Joint Clinical Assessments. | What is a Joint Clinical Assessment and what requirements are outlined in the HTAR. Furthermore, insights are provided on the scoping process and relevance of the PICO for a JCA. |
|
|
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Norwegian Medicines Agency (NoMA) in 2012. At NoMA she is working as special adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects. She has been Chair of EMA’s Biostatistics Working Party 2017 - 2019 and just finished her 3-year term as Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member of the SAWP and is Member of the new Methodology Working Party (MWP) recently established at EMA. In her role as Team-leader international HTA (iHTA) at NoMA, she has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21. Her particular focus is on parallel EMA-HTA scientific advices, now in the role as Vice-Chair of the JSC-CSCQ. She is furthermore involved in several academic and PPP projects as member of Scientific Advisory boards on subjects such as RWD, Patient reported outcomes, rare diseases, paediatric drug development and decentralized trials and digital tools. |
One of the most discussed issues around the upcoming HTA legislation (HTAR) is the fear of being confronted with an unreasonable number of PICO’s at the time of a joined clinical assessment (JCA). Surprisingly this issue is rarely, if ever, discussed by the applicants during the JSC’s. The assumption must be that the PICO can’t be predicted early in the process and will likely be only determined at the very last moment by the HTAB’s. While there is a scoping process taking place immediately before the start of the JCA it is worth understanding that these PICOs do not arbitrarily change and that they are predictable. I will discuss the theory behind the PICO and how it is supposed to be interpreted by applicants. |
|
|
Dima is a Principal within the Payer Evidence Solution practice at IQVIA. Her recent work has focused on supporting pharmaceutical companies in conceptualizing payer evidence generation strategies, successfully engaging with HTA bodies and decision makers and understanding the implications of the pan -European HTA regulation and the use of real-world evidence in HTA. Prior to joining the consulting world, Dima was an advisor on Innovation and External affairs at l’Institut national d’excellence en sante et en services sociaux (INESSS, Quebec) where she developed strategies to enhance collaborations among key stakeholders and consolidate relationships with International and Canadian HTA bodies. As a consultant to the World Health Organization on HTA, Dima was involved in foundational workstreams on access to medical devices and in the implementation of the first survey on the status and the use of HTA principles following the adoption by the World Health Assembly of the resolution on HTA. Dima has also co-authored several publications on multi-criteria decision analysis ( MCDA) published in peer review medical and scientific journals.Dima has received her Pharm D from the Lebanese American University. She holds a Master of Advances Studies in Health Economics and Management from HEC Universite de Lausanne. |
Dima Samaha will present lessons learned from previously conducted pan-EU assessments under JA3 with a forward looking view on what an additive PICO approach embedding evidence requirements of all Member States could look like. |
Mihai Rotaru |
Mihai Rotaru works as a Senior Manager Market Access at EFPIA (European Federation of Pharmaceutical Industries and Associations) where he is currently responsible for overall industry engagement in the implementation of the HTA Regulation at EU level. In addition, Mihai is also responsible for oncology policy, biotherapeutics as well as various supply chain issues (Falsified Medicines Directive, shortages and anticounterfeiting efforts). | The EU pharmaceutical industry is keen act as a partner in the development of a fit-for-purpose future system of JCA at EU level. As such, EFPIA is working with its members across a matrix of topics ranging from EU JCA process and implementation at national level, methods, JSC and overall advocacy with an underlying legal support as well. The purpose is to ensure that EFPIA speaks with one voice at EU level but also to ensure that companies are prepared from day 1 to meaningfully participate in the future system and to leverage these efforts into better evidence generation and overall clinical assessment of their innovation. |
|
|
Lara J. Wolfson is Executive Director and Head, Health Technology Assessment (HTA) Statistics in the Biostatistics and Research Decision Sciences (BARDS) organization at MSD Switzerland. Lara holds a PhD in Statistics from Carnegie Mellon University in the US, and an undergraduate degree from Simon Fraser University in Canada. Lara has held academic appointments at University of Waterloo in Canada and Brigham Young University in the US, and has previously worked as a scientist covering health economics, epidemiology, and statistics at the World Health Organization, as well as in the pharmaceutical industry in outcomes research and Market Access at both the Janssen Pharmaceutical Companies of Johnson & Johnson in Belgium and the Merck Center for Observational Research and Real-World Evidence in the US. Lara has authored more than 70 peer-reviewed journal articles, and currently chairs the HTA European Special Interest Group of the PSI (a professional organization for Statisticians in the Pharmaceutical Industry). Lara currently lives in the Greater Zurich Area with her husband, twin sons, and a cat named Hokey Pokey. |
The new paradigm for EU HTA Joint Clinical Assessments will rely heavily on statistical analysis. How can the statistical community prepare for this development? When, where, and how should statisticians be involved? What new capacities and systems needs to be developed? How can academic, government, and industry statisticians effectively collaborate to achieve the EU HTA regulation goals of patient access? In this panel discussion, we will talk with a number of key leaders in the area on these questions and more. |
