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PP216 Indirect Treatment Comparison Assessment: An Improvement Intervention In The Scottish Medicines Consortium
- John Scott, Moira McMurray, Rickie O'Connell, Pauline McGuire, Noreen Downes
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 37 / Issue S1 / December 2021
- Published online by Cambridge University Press:
- 03 December 2021, pp. 27-28
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Introduction
The Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of the National Health Service Scotland based on pharmaceutical company submissions. As the appraisals are conducted close to the point of marketing authorization, there is often a lack of direct head-to-head data. In 2019, assessment of relevant comparative efficacy was informed via indirect treatment comparisons (ITC) in 55 percent (36/66) of submissions. While the ITCs are essential to the decision-making process, they are frequently incomplete.
MethodsA focus group was conducted with the clinical assessment team (n = 11) to explore problems in the submission process and to identify areas for improvement. It was agreed that providing improved guidance to companies prior to submission may prevent future inconsistencies. A working group (n = 5) was tasked with identifying and implementing potential solutions. The group reviewed the focus group findings, relevant literature, and guidance from other organizations. Draft guidance was developed that was reviewed by two pharmaceutical industry representatives (SMC subcommittee members).
ResultsFindings from the focus group highlighted issues broadly related to the incomplete presentation and reporting of ITCs. The improved guidance document outlined specific requirements in a checklist format for reporting and presenting the results of different ITC data. This guidance was published in February 2020. To evaluate the impact of the updated guidance and to identify any further changes required, a follow-up focus group and survey of industry representatives is planned for March 2021.
ConclusionsThe aim of the ITC guidance is to provide pharmaceutical companies with direction to improve the quality and transparency of reporting, which will in turn improve the quality of HTAs and thus strengthen the recommendations provided by the SMC. The follow-up focus groups and survey will assess the impact of the guidance. It is acknowledged that the results of this process may be limited by the small sample size and short duration of the assessment.
PP215 An Evaluation Of The Scottish Medicine Consortium Detailed Advice Document
- Solveiga Zibaite, Pamela Andrews, Fiona McTaggart, Pauline McGuire, Scott Hill
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 37 / Issue S1 / December 2021
- Published online by Cambridge University Press:
- 03 December 2021, p. 27
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Introduction
The Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines. The advice is implemented at the local level by 14 Health Board Area Drug and Therapeutics Committees (ADTCs). The primary output is a published document, the Detailed Advice Document (DAD), which aims to describe the strengths and weaknesses of the evidence considered and the rationale for the decision. We examined how the DAD is being used to determine areas for improvement.
MethodsWe conducted semi-structured interviews with a purposive sample of SMC and ADTC members and formulary pharmacists, who are one of the key audiences. Interviews were recorded and transcribed using Microsoft Teams and coded in NVivo. The results were assessed via thematic analysis, which included major themes such as the structure and content of the DAD and its usefulness in supporting implementation of the advice from an ADTC perspective.
ResultsFollowing initial interviews (n = 7), some early themes have emerged. The DAD is a valued tool describing the assessment of a medicine's clinical and cost effectiveness. The current length of the DADs and the technical language used can limit the accessibility of information, and there have been suggestions on how to improve the structure and content. Additional interviews are still being completed and full interview results (available early 2021) will be analyzed to identify key themes.
ConclusionsThe DAD is the primary output of SMC's HTA process, which includes decisions on whether a medicine can be routinely prescribed in the National Health Service Scotland. DADs have increased in length over the years, reflecting the increasing complexity of new medicines and a corresponding increase in the size of pharmaceutical company submissions. The interviews conducted to date suggest that the DADs are highly regarded and support implementation of new medicines advice by the ADTC. The findings of this evaluation will lead to an action plan for improvement.
PP52 Interim Decision-Making To Address Uncertainty At Early Assessment
- Noreen Downes, Jan Jones, Anne Lee, Pauline McGuire
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, pp. 46-47
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Introduction
Medicines regulation has become increasingly adaptive to support earlier patient access but the immature clinical data is often challenging for health technology assessment decision-makers due to high levels of uncertainty on long term risks and benefits. Scottish Medicines Consortium (SMC) is therefore exploring new, more adaptive approaches to help manage this challenge.
MethodsSMC consulted with key stakeholders including clinicians, the pharmaceutical industry and patient groups on a number of options that would allow the committee to make an interim decision that would be revisited based on later evidence. The ability to collect robust patient level data given data capabilities in National Health Service Scotland (NHSScotland) was an important consideration.
ResultsTo ensure that additional evidence would be available to inform a re-assessment, the new approach applies to medicines with a Conditional Marketing Authorisation (MA) from the European Medicines Agency (EMA). This obligates the company to provide specified clinical data to the regulator within a pre-set timeframe. For these medicines, the SMC decision-making committee can accept or not recommend the medicine as at present but can also accept the medicine on an interim basis, if the regulator's mandated Specific Obligations are likely to address the uncertainties in the clinical evidence. When the regulator converts the MA from conditional to standard, the company is required to make a further SMC submission to allow a reassessment and a final decision. The company can also provide additional supplementary post-licensing patient level evidence at reassessment.
ConclusionsThis new decision option allows SMC to test an approach to managing uncertainty targeted at a small number of promising new medicines where there is unmet patient need, with the reassurance that a final decision will be supported by additional clinical data.
VP75 Improving Access To Ultra-Orphan Medicines In NHS Scotland
- Noreen Downes, Jan Jones, Anne Lee, Ailsa Brown, Pauline McGuire, Helen Wright
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 35 / Issue S1 / 2019
- Published online by Cambridge University Press:
- 31 December 2019, p. 93
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Introduction
Medicines for very rare conditions present challenges for healthcare globally due to uncertain evidence and often extremely high costs. In 2014, SMC introduced an ultra-orphan framework placing less emphasis on the cost per quality adjusted life year (QALY). Despite this, many medicines continued to be not recommended. A new pathway aimed at improved patient access based on further evidence collection is now being implemented.
MethodsThe development of the new pathway has involved collaboration with key stakeholders including patient groups, the pharmaceutical industry, and clinicians. Medicines that meet a new definition (based on four criteria including the prevalence of the condition treated) will be appraised by the SMC committee and a data collection plan will then be agreed with the pharmaceutical company.
ResultsFrom April 2019, medicines validated as ultra-orphans will initially be appraised using the broader decision-making framework and the SMC committee will outline key uncertainties in the clinical effectiveness. The medicine will then be available for a period of at least three years while further data are gathered, potentially comprising ongoing clinical trials, registry data, and patient reported outcome measures. SMC will then re-assess the clinical and economic evidence to inform a final decision on routine use of the medicine in NHS Scotland.
ConclusionsThe new pathway for ultra-orphan medicines will allow further evidence on their longer-term clinical benefits to be collected before a final decision on routine use. This approach reflects the current direction of travel in medicines regulation, by making medicines that address an unmet need available to patients at an earlier stage of development.
Contributors
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- By Ghazi Al-Rawas, Vazken Andréassian, Tianqi Ao, Stacey A. Archfield, Berit Arheimer, András Bárdossy, Trent Biggs, Günter Blöschl, Theresa Blume, Marco Borga, Helge Bormann, Gianluca Botter, Tom Brown, Donald H. Burn, Sean K. Carey, Attilio Castellarin, Francis Chiew, François Colin, Paulin Coulibaly, Armand Crabit, Barry Croke, Siegfried Demuth, Qingyun Duan, Giuliano Di Baldassarre, Thomas Dunne, Ying Fan, Xing Fang, Boris Gartsman, Alexander Gelfan, Mikhail Georgievski, Nick van de Giesen, David C. Goodrich, Hoshin V. Gupta, Khaled Haddad, David M. Hannah, H. A. P. Hapuarachchi, Hege Hisdal, Kamila Hlavčová, Markus Hrachowitz, Denis A. Hughes, Günter Humer, Ruud Hurkmans, Vito Iacobellis, Elena Ilyichyova, Hiroshi Ishidaira, Graham Jewitt, Shaofeng Jia, Jeffrey R. Kennedy, Anthony S. Kiem, Robert Kirnbauer, Thomas R. Kjeldsen, Jürgen Komma, Leonid M. Korytny, Charles N. Kroll, George Kuczera, Gregor Laaha, Henny A. J. van Lanen, Hjalmar Laudon, Jens Liebe, Shijun Lin, Göran Lindström, Suxia Liu, Jun Magome, Danny G. Marks, Dominic Mazvimavi, Jeffrey J. McDonnell, Brian L. McGlynn, Kevin J. McGuire, Neil McIntyre, Thomas A. McMahon, Ralf Merz, Robert A. Metcalfe, Alberto Montanari, David Morris, Roger Moussa, Lakshman Nandagiri, Thomas Nester, Taha B. M. J. Ouarda, Ludovic Oudin, Juraj Parajka, Charles S. Pearson, Murray C. Peel, Charles Perrin, John W. Pomeroy, David A. Post, Ataur Rahman, Liliang Ren, Magdalena Rogger, Dan Rosbjerg, José Luis Salinas, Jos Samuel, Eric Sauquet, Hubert H. G. Savenije, Takahiro Sayama, John C. Schaake, Kevin Shook, Murugesu Sivapalan, Jon Olav Skøien, Chris Soulsby, Christopher Spence, R. ‘Sri’ Srikanthan, Tammo S. Steenhuis, Jan Szolgay, Yasuto Tachikawa, Kuniyoshi Takeuchi, Lena M. Tallaksen, Dörthe Tetzlaff, Sally E. Thompson, Elena Toth, Peter A. Troch, Remko Uijlenhoet, Carl L. Unkrich, Alberto Viglione, Neil R. Viney, Richard M. Vogel, Thorsten Wagener, M. Todd Walter, Guoqiang Wang, Markus Weiler, Rolf Weingartner, Erwin Weinmann, Hessel Winsemius, Ross A. Woods, Dawen Yang, Chihiro Yoshimura, Andy Young, Gordon Young, Erwin Zehe, Yongqiang Zhang, Maichun C. Zhou
- Edited by Günter Blöschl, Technische Universität Wien, Austria, Murugesu Sivapalan, University of Illinois, Urbana-Champaign, Thorsten Wagener, University of Bristol, Alberto Viglione, Technische Universität Wien, Austria, Hubert Savenije, Technische Universiteit Delft, The Netherlands
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- Book:
- Runoff Prediction in Ungauged Basins
- Published online:
- 05 April 2013
- Print publication:
- 18 April 2013, pp ix-xiv
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