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Regulatory Approvals to HTA Recommendations Outcome and Timing - - PowerPoint PPT Presentation
Regulatory Approvals to HTA Recommendations Outcome and Timing - - PowerPoint PPT Presentation
The Relationship of Conditional Regulatory Approvals to HTA Recommendations Outcome and Timing Pharmaceutical Policy Concurrent Session B3 Oral Presentation Monday, April 15, 2019 Special Thanks to: Jesmine Cai, Tina Wang, and Neil
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Study objective
Examples of Facilitated Regulatory Pathways
US FDA: ‐ Accelerated approval ‐ Breakthrough therapy designation ‐ Fast track ‐ Regenerative Medicine Advanced Therapy Designation (RMAT) ‐ Priority review designation ‐ Orphan designation ‐ Rare pediatric disease priority review voucher ‐ Real Time Oncology Review (RTOR) EU EMA: ‐ PRIME ‐ Accelerated assessment ‐ Conditional marketing authorisation ‐ Orphan designation ‐ Advanced Therapy Medicinal Product Classification ‐ Exceptional Circumstances ‐ Adaptive pathways (via EMA HTA Parallel Scientific Advice) Health Canada
- Priority review
- Conditional (Notice of
Compliance with conditions)
The purpose of the study was to investigate the relationship of conditional approvals (including NOC-cs) to HTA recommendations in terms of the HTA outcomes and timing
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Study method
Data source
CIRS HTAdock Database An ongoing programme to monitor regulatory and HTA performance,;data collected for new active substances (NASs) appraised by key HTA agencies, analysing synchronisation between the regulatory decision and first HTA recommendation in timing and outcome.
Data collected on internationalised NASs)were evaluated in terms of:
- Type of regulatory review pathways (Standard vs. Conditional)
- HTA decisions/ recommendations
- Timing of regulatory submission to HTA decisions/recommendations
Regulatory approval from both of these agencies: (2012 to 2017) EMA Health Canada 1st HTA recommendation by one of the following HTA agencies: (2015 to 2017) CADTH HAS IQWIG SMC TLV The 1st HTA recommendation was then classified into the following categories:
- Positive
- Positive with restrictions
- Negative
- Multiple
NASs were identified from the CIRS Regulatory Approval Times Database that had:
For this presentation, analyses focused on NASs appraised by key HTA agencies from 2015 to 2017
Datasets in this study
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Study method-Trichotomous categorisation of HTA Decisions
Canada England Germany France Scotland Sweden
Positive Positive with restrictions Negative
List
List with clinical criteria and/or conditions
Do not list at the submitted price
Do not list/ reimburse Recommended
Managed Access Scheme Indications differ from Market Authorisation
Not recommended
Major benefit Important benefit Moderate benefit Minor benefit Lesser benefit
Major added benefit
Considerable added benefit
Minor added benefit
Non-quantifiable added benefit
No added benefit proven
Less benefit
Accepted General subsidy Accepted with restrictions Restricted subsidy
Not recommended
No subsidy
Where a green outline indicates that drug reimbursement is possible while a red outline indicates that drug reimbursement is not possible
Classification as described in CIRS R&D Briefing 69: Cai J, McAuslane N, Liberti
- L. 2018. R&D Briefing 69: Review of HTA outcomes and timelines in Australia,
Canada and Europe 2014-2017. Centre for Innovation in Regulatory Science. London, UK
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Conditional vs. standard approvals in Canada
11 6 49 11 4 0% 20% 40% 60% 80% 100%
Standard (64) Conditional (17)
Negative Restriction Positive
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HTA recommendation - timelines
Time from HC submission to CADTH recommendation according to procedure
[CELLRANGE] [CELLRANGE] [CELLRANGE] [CELLRANGE] [CELLRANGE] [CELLRANGE]
- 100
100 200 300 400 500 600 Standard Conditional Standard Conditional Standard Conditional Time (days)
252 218
Median, Box : 25th and 75th percentiles,
Number of products submitted through Standard process: 40 Conditional process: 14
HC submission to HC approval HC approval to CADTH submission CADTH submission to CADTH recommendation
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Comparison with Europe: 1st HTA Recommendations 2015-2017
11 6 7 1 5 29 3 13 3 5 1 49 11 20 6 23 3 17 20 2 25 1 4 29 5 46 9 20 8 22 4 18 3 1 2 2
0% 20% 40% 60% 80% 100%
Standard (64) Conditional (17) Standard (56) Conditional (12) Standard (75) Conditional (12) Standard (68) Conditional (11) Standard (57) Conditional (9) Standard (48) Conditional (5)
Negative Restriction Positive Multiple
England France Germany Scotland Sweden Canada
Number of NASs with 1st HTA recommendation in 2015-2017
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Liberti et al: Frontiers
- Pharmacol. 201
7;8:161
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Comparison with US FDA
[PERCE NTAGE] Out of 12 FDA Standard Approval in this cohort, 100% were approved as standard approval by HC [PERCENTAG E] 48% Out of 12 FDA Accelerated Approval in this cohort, 52% were approved as conditional approval by HC
HC NOC-Cs HC Standard
This cohort contained 24 NASs that were approved by
- Health Canada in 2015-2017
- FDA Accelerated process in 2015-2017
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Summary (2015-2017)
- 21% (17/81) NASs were granted a NOC-C by Health
Canada
- For NOC-Cs the median time for HC approvals was
approximately one month faster than standard approvals.
- Median time to CADTH recommendation was only about 2
weeks longer than for standard, but NOC-Cs were more likely to receive an initial negative recommendation.
- For Conditional Approvals, positive/positive with restriction
recommendations were more frequent in Europe than Canada
- This warrants a better understanding of the factors
underlying the initial negative recommendations, to better align expedited access recommendations with NOC-Cs.
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Recommendation
12
McAuslane et al: he Confluence of Accelerated Regulatory and Health Technology Assessment Access Pathways. CPT Nov 2018
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