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Breakthrough Therapy Designation Criteria Identify Drugs that Improve Clinical Outcomes for Patients: A Case for More Streamlined Coverage of Promising Therapies.

ABSTRACT: The breakthrough therapy designation (BTD) process was created to expedite clinical development timelines for drugs intended to treat serious conditions and preliminary clinical evidence indicates the drug may demonstrate substantial improvement over existing therapies. This analysis demonstrates that BTD is a valuable tool for expediting approval of promising therapies in oncology. By comparing drugs indicated to treat non-small cell lung cancer (NSCLC) approved with BTD or without BTD between January 2013 and October 2021, BTD drugs reduced the risk of death by a median of 31% and progression by a median of 48%, while drugs never receiving BTD reduced the risk of death and progression by a median of 15% and 41.9%, respectively. These findings show that BTD criteria accurately identify drugs that improve long-term outcomes for patients with cancer and warrant coordinated efforts to ensure timely coverage decisions and access for patients.

SUBMITTER: Collins G

PROVIDER: S-EPMC10324637 | biostudies-literature | 2023 Jul

REPOSITORIES: biostudies-literature

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Breakthrough Therapy Designation Criteria Identify Drugs that Improve Clinical Outcomes for Patients: A Case for More Streamlined Coverage of Promising Therapies.

Collins Grace G Stewart Mark M McKelvey Brittany B Stires Hillary H Allen Jeff J

Clinical cancer research : an official journal of the American Association for Cancer Research 20230701 13

The breakthrough therapy designation (BTD) process was created to expedite clinical development timelines for drugs intended to treat serious conditions and preliminary clinical evidence indicates the drug may demonstrate substantial improvement over existing therapies. This analysis demonstrates that BTD is a valuable tool for expediting approval of promising therapies in oncology. By comparing drugs indicated to treat non-small cell lung cancer (NSCLC) approved with BTD or without BTD between ...[more]

PMID: 35616593

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Project description:ImportanceThe China National Drug Administration (NMPA) established the breakthrough therapy designation (BTD) in 2020 to encourage the accelerated development of drugs for the prevention and treatment of diseases that are life-threatening. However, the differences between BTD and non-BTD cancer drugs regarding clinical benefit, regulatory approval, and price are unclear.ObjectivesTo compare BTD and non-BTD cancer drugs in clinical benefit (defined as efficacy and safety), novelty, time to approval, and average monthly treatment price.Design, setting, and participantsThis cross-sectional study analyzes the original indication of BTD and non-BTD novel cancer drugs approved by the NMPA between July 8, 2020, and July 8, 2024. Data on efficacy, safety, regulatory approval, and price of cancer drugs were extracted from pivotal clinical trials based on review reports published by the NMPA, peer-reviewed articles or meeting reports, and winning bid prices for cancer drugs in the Chinese provincial-level centralized procurement process.Main outcomes and measuresThe main outcome was the efficacy and safety associated with BTD vs non-BTD cancer drugs, including progression-free survival (PFS), response rate (RR), duration of response, serious adverse events, grade 3 or higher adverse events, and treatment-related deaths. In addition, the time to approval, novelty, and initial and latest average monthly treatment prices were evaluated, as well as the average annual reduction rate (AARR; the sum of the reduction rates divided by the number of years for the monthly treatment price) for these cancer drugs.ResultsBetween July 2020 and July 2024, 18 BTD (36%) and 32 non-BTD (64%) cancer drugs were approved by the NMPA. The median (IQR) clinical development time for BTD drugs was significantly shorter than for non-BTD drugs (5.6 [95% CI, 4.3-7.3] vs 6.6 [95% CI, 6.0-8.5] years; P = .02). No significant differences were observed in PFS (HR, 0.44 [95% CI, 0.38-0.52] vs HR, 0.51 [95% CI, 0.40-0.65]; P = .20), PFS gained (median [IQR], 5.4 [3.9-7.0] vs 2.7 [2.6-5.9] months; P = .77), RR (58% [95% CI, 45%-74%] vs 59% [95% CI, 51%-69%]; P = .85), and duration of response (median [IQR], 18.0 [15.0-21.6] vs 11.1 [7.4-17.4] months; P = .09) between BTD and non-BTD drugs. The rates of serious adverse events (37% [95% CI, 26%-52%] vs 32% [95% CI, 27%-36%]; P = .45), adverse events grade 3 or higher (64% [95% CI, 53%-77%] vs 55% [95% CI, 45%-68%]; P = .31), and treatment-related deaths (2% [95% CI, 1%-4%] vs 1% [95% CI, 1%-2%]; P = .10) were similar between BTD and non-BTD drugs. BTD drugs are more likely to be first-in-class drugs (5 of 18 [28%] vs 1 of 32 [3%]; P = .02). Differences in the median (IQR) initial ($5665 [$3542-$9321] vs $3361 [$2604-$5474]; P = .06) and latest ($5665 [$1553-$9321] vs $2145 [$1318-$4276]; P = .18) average monthly treatment prices for BTD drugs and non-BTD drugs were not significant. The median (IQR) AARRs for BTD drugs and non-BTD drugs were 15.2% (0%-46.9%) and 19.8% (1.0%-42.9%), respectively.Conclusions and relevanceThe findings of this cross-sectional study suggest that BTD has facilitated faster time to market for cancer drugs and improved novelty, but the price of treatment is relatively higher. There was no significant difference on comparative efficacy and safety.

Project description:BackgroundRegulatory pathways adopted by the United States Food Drug and Administration (FDA) and Australian Therapeutic Goods Administration (TGA) enable expedited approval of medicines that are thought to offer significant clinical advantage over existing options for severe diseases.ObjectivesTo review Australian accessibility to medicines approved through the FDA breakthrough therapy designation (BTD) process including timelines and approvals by the TGA and Pharmaceutical Benefits Advisory Committee (PBAC) for listing on the Pharmaceutical Benefits Scheme (PBS).MethodsRetrospective review of published reports from the FDA, TGA, PBAC and PBS for BTDs from 1 January 2013-31 August 2023. Uniform data about BTD and milestone dates were collected. Analysis included all BTDs approved by FDA until 31-August-2022. Main outcome measures: Rates of approval by TGA and PBAC, and PBS-listing; and median (interquartile range, IQR) time from FDA submission to FDA approval, and FDA approval to TGA approval, PBAC approval and PBS listing for cancer and non-cancer medicines.ResultsOf 237 BTDs across 156 medicines, 68% were approved by the TGA, and 37% were listed on the PBS. Median (IQR) time from FDA submission to FDA approval was shorter for cancer compared to non-cancer; 179 days (140-210) vs 232 days (181-245), p < 0.02. Time from FDA approval to PBS listing was similar for cancer and non-cancer; median 744 days (IQR, 549-1136) and 733 days (IQR 440-960) respectively, with improvements for cancer BTDs noted for 2018-2022 compared to 2013-2017; 566 days (IQR 319-831) vs 880 days (IQR 620-1362), p < 0.02 but not for non-cancer BTDs.ConclusionBTD medicines are accessible in Australia approximately 2 years after FDA approval. Since 2018, time to PBS listing for cancer therapies improved, mirroring shorter FDA approval times for this category. Further understanding of clinical studies and context by therapeutic area may improve timely and safe access to life-saving medicines.

Dataset Information

Breakthrough Therapy Designation Criteria Identify Drugs that Improve Clinical Outcomes for Patients: A Case for More Streamlined Coverage of Promising Therapies.

Publications

Breakthrough Therapy Designation Criteria Identify Drugs that Improve Clinical Outcomes for Patients: A Case for More Streamlined Coverage of Promising Therapies.

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