REAL WORLD EVIDENCE AND
ARTIFICIAL INTELLIGENCE
REAL WORLD EVIDENCE AND ARTIFICIAL INTELLIGENCE
April 28, 2021
7:00 a.m. MDT
Panelists:
Ashley Jaksa, Aetion (USA)
Session Details:
The main goal of this webinar is to promote integration and collaboration among RWEAI IG members, who are invited to speak about their projects and work.
RICC workshop (Saturday 15 June 2019) – “A Primer in Regulatory Interactions and Conditional Coverage – How to Generate Real World Evidence – A Modern Approach”
RICC workshop (Saturday 15 June 2019) – “A Primer in Regulatory Interactions and Conditional Coverage – How to Generate Real World Evidence – A Modern Approach”
Current update: July 2017
The RICC Interest Group has developed the following repository of publically available information. The primary objective of this repository is to provide a regularly updated bibliography of peer-reviewed literature on the key topics in scope of the Interest Group, namely:
- Adaptive pathways
- Early dialogue / scientific advice involving both regulators and HTA bodies
- HTA-Regulatory Interactions relating to evidence development
- Managed entry agreements (focussed on outcomes-based agreements)
- Performance-linked Risk Sharing Agreements (PBRSAs)
- Performance-linked Risk Sharing Agreements (PBRSAs)
- Conditional treatment continuation (CTC)
- Post-marketing additional data collection
Post-marketing additional data collection
Repository Manager: Michelle Mujoomdar (CADTH)
Information Specialist: David Kaunelis (CADTH)
Detail of the literature search methodology
The literature search was performed by an information specialist. Published literature was identified by searching the following bibliographic database: MEDLINE (1946- ) with in-process records & daily updates via Ovid. The search strategy was comprised of both controlled vocabulary, such as the National Library of Medicine’s MeSH (Medical Subject Headings), and keywords. The main search parameters were health technology assessment and regulatory concepts. Additional terms specific to the RICC key topics were also included, such as: coverage with evidence development, scientific advice, adaptive licensing, adaptive pathways, managed entry and conditional approval. A search of specific agencies contributing to this work (TLV, PBAC, NICE, HAS, G-BA and ZIN) was also incorporated into the strategy. No methodological filters were applied to limit retrieval. Retrieval was limited to English-language citations, published from January 1, 2015 to the present. The initial search was completed on February 4, 2016. A monthly alert has been established to update the search results.
- Baird et al. Accelerated access to innovative medicines for patients in need. Clin. Pharm Ther. 2014; 96: 559-571
- Bergmann et al. Actual developments in European regulatory and health technology assessment of new cancer drugs: what does this mean for oncology in Europe? Ann. Oncology. 2014; 25: 303-306
- Berntgen et al. Improving the Contribution of Regulatory Assessment Reports to Health Technology Assessments-A Collaboration between the European Medicines Agency and the European network for Health Technology Assessment. Value in Health. 2014 [Epub ahead of print]
- Cuche M et al. Early dialogue with health technology assessment bodies: a European perspective. IJATC 2014; 30: 571-578
- Elvidge S. EMA’s parallel advice workshop bridges regulatory and reimbursement divide. Nat. Rev. Drug Discovery. 2014; 13:8
- Grainger D. Is this the real life? Is this just fantasy? Int. J. Technol. Assess. Health Care. 2014; 30:239-240Husereau et al. Adaptive approaches to licensing, health technology assessment, and introduction of drugs and devices. Int. J. Technol. Assess. Health Care. 2014; 30:241-249
- Husereau et al. Adaptive approaches to licensing, health technology assessment, and introduction of drugs and devices. Int. J. Technol. Assess. Health Care. 2014; 30:241-249
- Klemp M et al. Transcatheter aortic valve implantation and adaptive / progressive coverage. Int. J. Technol. Assess. Health Care. 2014; 30:250-251
- Klemp M et al. Transcatheter aortic valve implantation and adaptive / progressive coverage. Int. J. Technol. Assess. Health Care. 2014; 30:250-251
- Trusheim et al. The Janus initiative: A multi-stakeholder process and tool set for facilitating and quantifying Adaptive Licensing discussions. Health Policy and Technology. 2014; 3:241-247
- Towse A. Presidents Message: Adaptive pathways to Value Assessment. ISPOR Connections. 2014; 20(5): 3
- Tarricone R. et al. Generating appropriate clinical data for value assessment of medical devices: what role does regulation play? Expert Review of Pharmacoeconomics & Clinical Research. 2014; 14: 707-718
- Uguen et al. Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives. Orphanet J Rare Dis. 2014; 9:20
- Wonder M. What can be gained from increased early-stage interaction between regulators, payers and the pharmaceutical industry? Expert Rev Pharmacoecon Outcomes Res. 2014; 14:465-467
- Baird et al. Comparison of Stakeholder Metrics for Traditional and Adaptive Development and Licensing Approaches to Drug Development. Ther. Inn. Reg. Sci. 2013; 47:474-483
- De Jong et al. Appropriate evidence for adaptive marketing authorization. Nat. Rev. Drug Discov. 2013; 12:647-648
- Forda et al. Priorities for improving drug research, development and regulation. Nat. Rev. Drug Discov. 2013; 12:247-248 Technology Assessment. Value in Health. 2014 [Epub ahead of print]
- Henshall et al. Understanding the Role and Evidence Expectations of Health Technology Assessment and Coverage/Payer Bodies: What Are They Looking for, and How and Why Does This Differ From What Regulators Require? Ther. Inn. Reg. Sci. 2013; 48:341-346
- Tsoi et al. Harmonization of reimbursement and regulatory approval processes: a systematic review of international experiences, Expert Rev Pharmacoecon. Outcomes Res. 2013; 13:497-511
- Wonder et al. Early scientific advice obtained simultaneously from regulators and payers: findings from a pilot study in Australia, Value in Health. 2013; 16:1067-1073
- Barker & Garner. Adaptive drug development and licensing. Regulatory Rapporteur. 2012; 9:13-14
Development. Ther. Inn. Reg. Sci. 2013; 47:474-483 - Brekenridge et al. Medicines Regulation and Health Technology Assessment. Clin. Phar. Ther. 2012; 87: 152-154
- Eichler et al. Adaptive Licensing: Taking the Next Step in the Evolution of Drug Approval. Nature. 2012; 91: 426-437
- Frnsdal et al. Interaction initiatives between regulatory, health technology assessment and coverage bodies, and industry. Int. J. Technol. Assess. Health Care. 2012; 28:374-81
- Liberti et al. Preparing for regulatory review and reimbursement decisions: a case for cooperation between regulatory authorities and health technology assessment agencies. Pharm. Med. 2012; 23:263-267
- Messner & Tunis. Current and future state of FDA-CMS parallel reviews. Clin. Pharmacol. Ther. 2012; 91:383-385
Backhouse et al. Early dialogue between the developers of new technologies and pricing and reimbursement agencies: a pilot study. Value in Health. 2011; 14:608-615
Barlas S. Pitting the FDA against CMS: the good, the bad and the ugly about “parallel review”. Biotechnol Health. 2011; 8:29-30
Eichler et al. Bridging the efficacy-effectiveness gap: a regulator’s perspective on addressing variability of drug response. Nat. Rev. Drug Discov. 2011; 10:495-506
Henshall et al. Interactions between HTA, coverage and regulatory processes: emerging issues, goals and opportunities. Int. J. Technol. Assess. Health Care; 2011; 27:253-60.
Pignatti et al. Regulators, payers and prescribers: can we fill the gaps? The Lancet Oncology. 2011; 12: 930-931
Eichler et al. Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers. Nat. Rev. Drug Discov. 2010; 9:277-91.
Battista et al. Health technology assessment and the regulation of medical devices and procedures in Quebec. Int. J. Technol. Assess. Health Care. 1999; 15:593-601
Wang S and Smith J. Potential legal barriers to increasing CMS/FDA collaboration: the law of trade secrets and related considerations. Food and Drug Law Journal. 2003; 58:613-627
The Governmental and non-governmental activities of interest to the RICC provides a list of website links to government and non-government activities that are relevant to the RICC.
At this stage, a simple title and hyperlink is provided although it is intended in the future that a short description will be developed for each of these links. While we endeavour to keep the list current and complete, the information has necessarily been compiled in a non-systematic fashion. If you are aware of activities that are relevant but have not been included in this list we would be grateful if you could let the RICC Repository Manager know so that the appropriate information can be uploaded. If your organisation has been included on the list but you would prefer alternative information presented please also contact us.
April 28, 2021
7:00 a.m. MDT
Panelists:
Ashley Jaksa, Aetion (USA)
Session Details:
The main goal of this webinar is to promote integration and collaboration among RWEAI IG members, who are invited to speak about their projects and work.
RICC workshop (Saturday 15 June 2019) – “A Primer in Regulatory Interactions and Conditional Coverage – How to Generate Real World Evidence – A Modern Approach”
RICC workshop (Saturday 15 June 2019) – “A Primer in Regulatory Interactions and Conditional Coverage – How to Generate Real World Evidence – A Modern Approach”
Current update: July 2017
The RICC Interest Group has developed the following repository of publically available information. The primary objective of this repository is to provide a regularly updated bibliography of peer-reviewed literature on the key topics in scope of the Interest Group, namely:
- Adaptive pathways
- Early dialogue / scientific advice involving both regulators and HTA bodies
- HTA-Regulatory Interactions relating to evidence development
- Managed entry agreements (focussed on outcomes-based agreements)
- Performance-linked Risk Sharing Agreements (PBRSAs)
- Performance-linked Risk Sharing Agreements (PBRSAs)
- Conditional treatment continuation (CTC)
- Post-marketing additional data collection
Post-marketing additional data collection
Repository Manager: Michelle Mujoomdar (CADTH)
Information Specialist: David Kaunelis (CADTH)
Detail of the literature search methodology
The literature search was performed by an information specialist. Published literature was identified by searching the following bibliographic database: MEDLINE (1946- ) with in-process records & daily updates via Ovid. The search strategy was comprised of both controlled vocabulary, such as the National Library of Medicine’s MeSH (Medical Subject Headings), and keywords. The main search parameters were health technology assessment and regulatory concepts. Additional terms specific to the RICC key topics were also included, such as: coverage with evidence development, scientific advice, adaptive licensing, adaptive pathways, managed entry and conditional approval. A search of specific agencies contributing to this work (TLV, PBAC, NICE, HAS, G-BA and ZIN) was also incorporated into the strategy. No methodological filters were applied to limit retrieval. Retrieval was limited to English-language citations, published from January 1, 2015 to the present. The initial search was completed on February 4, 2016. A monthly alert has been established to update the search results.
- Baird et al. Accelerated access to innovative medicines for patients in need. Clin. Pharm Ther. 2014; 96: 559-571
- Bergmann et al. Actual developments in European regulatory and health technology assessment of new cancer drugs: what does this mean for oncology in Europe? Ann. Oncology. 2014; 25: 303-306
- Berntgen et al. Improving the Contribution of Regulatory Assessment Reports to Health Technology Assessments-A Collaboration between the European Medicines Agency and the European network for Health Technology Assessment. Value in Health. 2014 [Epub ahead of print]
- Cuche M et al. Early dialogue with health technology assessment bodies: a European perspective. IJATC 2014; 30: 571-578
- Elvidge S. EMA’s parallel advice workshop bridges regulatory and reimbursement divide. Nat. Rev. Drug Discovery. 2014; 13:8
- Grainger D. Is this the real life? Is this just fantasy? Int. J. Technol. Assess. Health Care. 2014; 30:239-240Husereau et al. Adaptive approaches to licensing, health technology assessment, and introduction of drugs and devices. Int. J. Technol. Assess. Health Care. 2014; 30:241-249
- Husereau et al. Adaptive approaches to licensing, health technology assessment, and introduction of drugs and devices. Int. J. Technol. Assess. Health Care. 2014; 30:241-249
- Klemp M et al. Transcatheter aortic valve implantation and adaptive / progressive coverage. Int. J. Technol. Assess. Health Care. 2014; 30:250-251
- Klemp M et al. Transcatheter aortic valve implantation and adaptive / progressive coverage. Int. J. Technol. Assess. Health Care. 2014; 30:250-251
- Trusheim et al. The Janus initiative: A multi-stakeholder process and tool set for facilitating and quantifying Adaptive Licensing discussions. Health Policy and Technology. 2014; 3:241-247
- Towse A. Presidents Message: Adaptive pathways to Value Assessment. ISPOR Connections. 2014; 20(5): 3
- Tarricone R. et al. Generating appropriate clinical data for value assessment of medical devices: what role does regulation play? Expert Review of Pharmacoeconomics & Clinical Research. 2014; 14: 707-718
- Uguen et al. Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives. Orphanet J Rare Dis. 2014; 9:20
- Wonder M. What can be gained from increased early-stage interaction between regulators, payers and the pharmaceutical industry? Expert Rev Pharmacoecon Outcomes Res. 2014; 14:465-467
- Baird et al. Comparison of Stakeholder Metrics for Traditional and Adaptive Development and Licensing Approaches to Drug Development. Ther. Inn. Reg. Sci. 2013; 47:474-483
- De Jong et al. Appropriate evidence for adaptive marketing authorization. Nat. Rev. Drug Discov. 2013; 12:647-648
- Forda et al. Priorities for improving drug research, development and regulation. Nat. Rev. Drug Discov. 2013; 12:247-248 Technology Assessment. Value in Health. 2014 [Epub ahead of print]
- Henshall et al. Understanding the Role and Evidence Expectations of Health Technology Assessment and Coverage/Payer Bodies: What Are They Looking for, and How and Why Does This Differ From What Regulators Require? Ther. Inn. Reg. Sci. 2013; 48:341-346
- Tsoi et al. Harmonization of reimbursement and regulatory approval processes: a systematic review of international experiences, Expert Rev Pharmacoecon. Outcomes Res. 2013; 13:497-511
- Wonder et al. Early scientific advice obtained simultaneously from regulators and payers: findings from a pilot study in Australia, Value in Health. 2013; 16:1067-1073
- Barker & Garner. Adaptive drug development and licensing. Regulatory Rapporteur. 2012; 9:13-14
Development. Ther. Inn. Reg. Sci. 2013; 47:474-483 - Brekenridge et al. Medicines Regulation and Health Technology Assessment. Clin. Phar. Ther. 2012; 87: 152-154
- Eichler et al. Adaptive Licensing: Taking the Next Step in the Evolution of Drug Approval. Nature. 2012; 91: 426-437
- Frnsdal et al. Interaction initiatives between regulatory, health technology assessment and coverage bodies, and industry. Int. J. Technol. Assess. Health Care. 2012; 28:374-81
- Liberti et al. Preparing for regulatory review and reimbursement decisions: a case for cooperation between regulatory authorities and health technology assessment agencies. Pharm. Med. 2012; 23:263-267
- Messner & Tunis. Current and future state of FDA-CMS parallel reviews. Clin. Pharmacol. Ther. 2012; 91:383-385
Backhouse et al. Early dialogue between the developers of new technologies and pricing and reimbursement agencies: a pilot study. Value in Health. 2011; 14:608-615
Barlas S. Pitting the FDA against CMS: the good, the bad and the ugly about “parallel review”. Biotechnol Health. 2011; 8:29-30
Eichler et al. Bridging the efficacy-effectiveness gap: a regulator’s perspective on addressing variability of drug response. Nat. Rev. Drug Discov. 2011; 10:495-506
Henshall et al. Interactions between HTA, coverage and regulatory processes: emerging issues, goals and opportunities. Int. J. Technol. Assess. Health Care; 2011; 27:253-60.
Pignatti et al. Regulators, payers and prescribers: can we fill the gaps? The Lancet Oncology. 2011; 12: 930-931
Eichler et al. Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers. Nat. Rev. Drug Discov. 2010; 9:277-91.
Battista et al. Health technology assessment and the regulation of medical devices and procedures in Quebec. Int. J. Technol. Assess. Health Care. 1999; 15:593-601
Wang S and Smith J. Potential legal barriers to increasing CMS/FDA collaboration: the law of trade secrets and related considerations. Food and Drug Law Journal. 2003; 58:613-627
The Governmental and non-governmental activities of interest to the RICC provides a list of website links to government and non-government activities that are relevant to the RICC.
At this stage, a simple title and hyperlink is provided although it is intended in the future that a short description will be developed for each of these links. While we endeavour to keep the list current and complete, the information has necessarily been compiled in a non-systematic fashion. If you are aware of activities that are relevant but have not been included in this list we would be grateful if you could let the RICC Repository Manager know so that the appropriate information can be uploaded. If your organisation has been included on the list but you would prefer alternative information presented please also contact us.