P329: Observations following Implementation of Accelerated Approval Pathway for New Drugs in Taiwan
Poster Presenter
Yu Fa Su
Project Manager
Taiwan Center For Drug Evaluation Taiwan
Objectives
This study aimed to examine the current status of drugs granted accelerated approval (AA) in Taiwan and whether they were converted to regular approval. Our findings provide a reference for drug companies submitting an application for accelerated approval.
Method
An internal database of the Taiwan Center for Drug Evaluation (CDE) was used to evaluate accelerated approval drug-indication pairs from 2013 to 2024. We assessed surrogate endpoints, the status of confirmatory trials, and whether indications were converted to regular approval.
Results
There were 33 drugs granted accelerated approval from 2013 to 2024 in Taiwan. The types of drugs were categorized as follows: 18 under chemical drugs and 15 under biological drugs. Since a drug can be approved for more than one indication through the accelerated approval pathway, we further analyzed drug-indication pairs and identified a total of 58 pairs. Treatment areas were categorized as follows: 54 pairs were antineoplastic, 1 was anti-infective, 1 was vaccine, 1 was anti-inflammatory, and 1 was anti-lymphoproliferative. At the end of 2024, 41% (24 pairs) had been converted to regular approval following a median of 2.25 years, 12% (7 pairs) had been withdrawn, and 47% (27 pairs) remained ongoing after a median of 2 years.
Among the 54 antineoplastic drug-indication pairs granted accelerated approval, most of them (50 pairs) used overall response rate (objective response rate, ORR) as surrogate endpoints, 3 pairs used progression-free survival (PFS), and 1 pair used ORR combined with PFS. A total of 22 out of these 54 antineoplastic drug-indication pairs were converted to regular approval, 8 of which were converted based on PFS, 4 on PFS plus overall survival (OS), 4 on ORR combined with duration of response (DOR), 3 on OS, 1 on durable response rate, 1 on pathological complete response rate and event free survival, and 1 on an unmet medical need with narrowed indication because the confirmatory trial only showed positive trend without reaching statistical significance. A comparison of accelerated and regular approval indications revealed that 16 of 22 (73 %) were unchanged (accelerated approval notices were removed), while 6 of 22 (27 %) were revised.
The one vaccine granted accelerated approval was an Enterovirus (EV) 71 vaccine developed by a local Taiwanese company. The approval was based on immunogenicity criteria which was consistent with the guideline released by the Taiwan CDE in 2018.
Conclusion
The Taiwan Food and Drug Administration has implemented the AA pathway since 2013 to expedite the overall drug development process. This allows the approval of new drugs addressing unmet medical needs based on surrogate endpoints, which indicate clinically meaningful improvements. Confirmatory trials are then conducted to verify clinical benefits prior to regular approval.
Our findings show that at the end of 2024, 41% had been converted to regular approval. Without the AA pathway, these drugs would not have been available earlier to patients.
However, the public should be aware that AA approval isn’t equivalent to regular approval. Should clinical trials fail to verify clinical benefits, indications may be withdrawn or narrowed.
The majority of drugs granted AA in Taiwan are antineoplastic, due to a good understanding of surrogate endpoints, such as ORR, and their ability to reasonably predict clinical benefits. In Taiwan, EV 71 infection was a serious public health problem. To facilitate the development of EV vaccines, the Taiwan CDE released a development strategy guideline in 2018, in which AA based on immunogenicity criteria were clearly described along with the requirements of a vaccine efficacy confirmatory study. As a result, two EV 71 vaccines were developed by local Taiwanese companies. For both products, the CDE provided thorough consultations throughout the development process. One vaccine was initially approved through the AA pathway before a confirmatory study was completed. For the other vaccine, a vaccine efficacy study was completed and granted via regular approval.
The AA pathway allows earlier approval of new drugs targeting unmet medical needs. Drug companies are encouraged to consult regulatory authorities earlier in the development process to streamline the future conversion to regular approval.
