Htadock R&D Briefing
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Review of HTA outcomes and timelines in Australia, Canada and Europe 2015-2019 Figure 1: First HTA recommendations: comparisons across key jurisdictions in 2019 Positive Restriction Negative Multiple © CIRS, R&D Briefing 78 Australia Canada England France N=13 N=26 N=22 N=32 5% 22% 31% 36% 41% 69% 59% 46% 54% 37% Germany Poland Scotland Sweden N=26 N=19 N=21 N=14 5% 11% 21% 31% 21% 19% 36% 54% 43% 33% 15% 68% 43% Contents Summary 2 Overview of new drug recommendations 3 Synchronisation of regulatory and HTA recommendations 4 37 NASs appraised by seven jurisdictions in 2015-2019 5 Focus on Orphan Designation 6 Focus on ATMPs 7 Features of Australia 8 Features of Canada 9 Features of Europe 10 Overview of HTA implementation 12 Methodology and definitions 13 R&D BRIEFING 78 SUMMARY Introduction Timely recommendation for drug reimbursement by health technology assessment (HTA) agencies is critical to ensure that patient access to medicines of therapeutic value is not delayed. As part of an ongoing study to monitor regulatory and HTA performance, CIRS has been collecting data on new active substances (NASs) appraised between 2015 and 2019 by eight health technology assessment (HTA) agencies, analysing synchronisation between the regulatory decision and first HTA recommendation in timing and outcome. Recommendations were collected from the Australian Pharmaceutical Benefits Advisory Committee (PBAC), Canadian Agency for Drugs and Technologies in Health (CADTH; both Common Drug Review [CDR] and pan-Canadian Oncology Drug Review [pCODR]), English National Institute for Health and Care Excellence (NICE), French Haute Autorité de Santé (HAS), German Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), Polish Agencja Oceny Technologii Medycznych i Taryfikacji (AOTMiT), Scottish Medicines Consortium (SMC) and Swedish Tandvårds- & läkemedelsförmånsverket (TLV), for NASs approved 2012-2019 by the respective jurisdictional regulatory agencies, the Australian Therapeutic Goods Administration (TGA), Health Canada and European Medicines Association (EMA). Using a methodology outlined on page 13, the HTA recommendations in this report have been classified as positive, positive with restrictions or negative. Figure 32 illustrates how the specific recommendations by the eight HTA systems are captured within this trichotomous categorisation. In cases in which more than one HTA dossier was submitted by a company for the same drug based on different sub-indications within an approved regulatory label and the final HTA outcome for these individual sub-indications differed, the outcome was classified as multiple. Observations • In 2019, 46% to 95% of NASs approved In Australia, more than 60% of NASs were listed in the by regulatory agencies received a PBS list in 2015-2019 after receiving a positive or positive or positive with restrictions positive with restrictions first recommendation. first recommendation by HTA agencies across all of the studied jurisdictions. Of 103 drugs with PBAC recommendations in Australia from 2015- England had the highest proportion of 2019, 63 were listed in the PBS list. Of those drugs in the PBS list, 38% positive/positive with restrictions took more than one cycle to receive a positive/ positive with recommendations for NASs appraised restrictions PBAC recommendation. by HTA agencies. • Of all studied HTA agencies, Germany had the highest proportion of In Canada, the HC/CADTH parallel process reduced products recommended within one the time from regulatory approval to HTA year of regulatory approval (92% in recommendation. 2019). Approximately half of the NASs submitted for HTA recommendation underwent the Health Canada/ CADTH parallel review process. The • Australia had the shortest median parallel review process was a success in reducing the time taken to time between regulatory approval and reach the first HTA recommendation. In 2018-2019, the median time HTA recommendation (24 days) in taken from regulatory approval to HTA recommendation was reduced 2015-2019, followed by Germany (132 by using the parallel process (144 days), compared to the sequential days). process (426 days). • CIRS analysed NASs rolled out to seven jurisdictions, excluding Poland due to variation, and identified 37 In Europe, the lag between EMA approval and HTA NASs that received a recommendation recommendations varied across the European by all HTA agencies during the period jurisdictions, from 132 median days in Germany to of 2015-2019. Germany provided the 538 median days in Poland. highest number of recommendations as the first country of appraisal (30%), In the studied European jurisdictions, the time from EMA approval to followed by Australia (24%). England HTA recommendation was generally longer for those NASs that and Scotland had the highest received a negative HTA outcome. Among 39 commonly appraised congruence (73%) of first HTA NASs approved by EMA, drugs with accelerated regulatory approval recommendations. generally had the fastest median time from regulatory approval to HTA recommendation. R&D Briefing 78 July 2020, © Centre for Innovation in Regulatory Science, Ltd. 2 OVERVIEW OF NEW DRUG RECOMMENDATIONS Figure 2: First HTA recommendation comparison across key jurisdictions by year of HTA recommendation Between 2015-2019 Positive Restriction Negative Multiple 100% 1 1 1 1 1 1 1 2 2 2 2 2 2 3 3 3 3 2 2 5 4 4 5 7 3 10 8 10 4 7 5 4 8 80% 10 1011 6 10 7 14 14 8 1411 12 13 13 13 8 4 13 8 60% 10 1915 1312 14 7 11 17 13 7 6 12 8 17 5 6 19 8 1 19 40% 3 4 1 24 18 27 12 13 7 16 18 12 8 12 20% 10 10 9 13 11 9 6 7 8 11 8 4 9 8 5 8 9 8 5 8 4 5 6 4 4 Proportion recommendedof NASs 2 2 2 0% 1 1 1 Australia Canada England France Germany Poland Scotland Sweden N= Number of NASs © CIRS, R&D Briefing 78 In 2019, England had the highest proportion (95%) of positive/positive with restrictions recommendations for NASs appraised by HTA agencies (Figure 1). Within this cohort, France appraised the highest number of NASs in 2019 (32 NASs), while Australia appraised the fewest (13 NASs) (Figure 1). In 2019, three Australian PBAC recommendations obtained TGA approval in 2020 and were outside of the criteria of this briefing. The year-on-year variation in the number of recommendations is due to a number of reasons, including the number of regulatory approvals, the company submission strategy and the review time by the HTA agencies. More than 45% of NASs approved by relevant regulatory agencies received a positive or positive with restrictions recommendation by HTA agencies in all of the studied jurisdictions in 2019 (Figure 2). Over these five years, Australia and Poland had a gradual increase in the proportion of positive or positive with restrictions recommendations (increase from 42% in 2015 to 54% in Australia and increase from 11% in 2015 to 89% in Poland). Australia had the fastest median rollout time from regulatory submission to first HTA recommendation in 2019 (408 days), followed by Canada (556 days). There were less variation in rollout time in these two countries in 2019 compared with 2018, which indicated an improved consistency (Figure 3). The median rollout time has been relatively constant in 2015-2019 across all jurisdictions with the exception of England and Poland, which showed a decrease of 83 and 103 median days respectively from 2018 to 2019. In Poland, a decrease in 246 median days was observed over the last 3 years, from 1188 days in 2016 to 942 days in 2019. However, it still took the longest time for products to roll out in Poland compared with other jurisdictions. Figure 3: Rollout time from regulatory submission to HTA recommendation by year of HTA recommendation th th Median 25 and 75 percentiles, Overall median (1744) 1400 762 1200 673 857 971 723 1000 774 694 743 706 467 609 679 674 700 800 609 1188 1045 571 521 538 942 350 803 600 383 709 Time, Time, days 821 687 692 665 658 581 590 572 597 585 535 400 556 518 408 362 200 © CIRS, R&D Briefing 78 0 3 Australia Canada England France Germany Poland Scotland Sweden (75th percentile of regulatory submission to HTA recommendation for Poland in 2017) R&D Briefing 78 July 2020, © Centre for Innovation in Regulatory Science, Ltd. 3 SYNCHRONISATION OF REGULATORY AND HTA RECOMMENDATIONS Figure 4: Breakdown of rollout time across all jurisdictions in 2015-2019 2015 380 15 Australia 2016 351 32 2017 360 126 Regulatory authority review time 2018 364 17 2019 346 25 Regulatory approval to HTA 2015 391 251 recommendation (national level) Canada 2016 348 175 2017 356 182 2018 338 162 2019 344 207 Time taken from regulatory approval 2015 409 314 to HTA recommendation includes England 2016 441 341 2017 402 386 • Company submission strategy 2018 444 288 • Company time for pre-submission 2019 415 321 preparation 2015 417 228 France 2016 418 202 • HTA agency review time 2017 421 194 2018 407 246 2019 436 276 2015 437 139 Germany 2016 406 129 2017 388 116 2018 450 142 2019 435 140 2015 394 444 2016 427 524 Poland 2017 422 802 2018 388 603 2019 419 564 2015 418 257 2016 369 203 Scotland 2017 419 293 2018 430 259 2019 417 257 Median time, days 2015 415 184 2016 406 159 Sweden 2017 406 217 2018 423 241 © CIRS, R&D Briefing 78 2019 433 222 0 200 400 600 800 1000 1200 Median time, days Australia had the shortest median time between regulatory approval and HTA recommendation of 24 days in 2015- 2019 (Figure 4). PBAC had the shortest overall median time between regulatory approval and HTA recommendation, suggesting the proactive approach within Australia to move toward synchronising the timing of HTA and regulatory recommendation is achieving its purpose.