An overview of cancer drugs approved through expedited approval programs and orphan medicine designation globally between 2011 and 2020

Wang, Shuhang; Yang, Qi; Deng, Lei; Liu, Qi; Yang, Yuqi; Ma, Peiwen; Men, Yuxin; Yung, Bryant C.; Lee, Robert J.; Zhang, Mengzi; Li, Ning

doi:10.1016/j.drudis.2021.12.021

Cited by 22 publications

(24 citation statements)

References 13 publications

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“…6,7 Furthermore, we noted, as observed in prior publications, that drugs with oncology indications were approved through 1 or more expedited programs more often than were drugs in other therapeutic areas. 7,14 The use of expedited programs is likely to continue to increase over time. During the past 3 decades, the number of drugs receiving FDA orphan drug designation increased 4 fold, 12 increasing the pool of drugs disproportionately likely to qualify for expedited programs.…”

Section: Discussionmentioning

confidence: 99%

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Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021

2022

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ImportanceThe US Food and Drug Administration (FDA) has 4 programs that can be used alone or in combination to expedite drug availability: Accelerated Approval, Breakthrough Therapy, Fast Track, and Priority Review. Drugs using these programs can include novel drugs, which do not contain a previously FDA-approved active moiety, and orphan drugs, intended for diseases or conditions affecting fewer than 200 000 people; to date, no comprehensive evaluation of how these programs have been used in combination has been published.ObjectiveTo assess how often and in what combinations expedited programs are used in the development and review of approved novel biologics and small-molecule drugs, stratified by orphan drug status and indication.Design, Setting, and ParticipantsThis cross-sectional study evaluated all novel drugs that were FDA approved between January 1, 2008, and December 31, 2021.Main Outcomes and MeasuresThe main outcome was the frequency with which expedited programs were used and in what combinations, stratified by orphan drug status and drug type (small molecule vs therapeutic biologic). The unit of analysis was the novel drug–indication pair because a drug can be approved for multiple indications, each of which may use a different expedited program or differ in orphan drug status.ResultsThe study included 581 novel drug–indication pairs approved during the 14-year study period; 252 (43.4%) were orphan drugs, 139 (23.9%) were therapeutic biologics, and 442 (76.1%) were small-molecule drugs. Use of at least 1 expedited program increased from 11 of 26 drug-indication pairs (42.3%) in 2008 to 41 of 55 (74.5%) in 2021. Of the 363 approved drug-indication pairs using at least 1 expedited program, 225 (62.0%) were orphan drugs; at least 1 expedited program was used by 97 of the 139 approved biologic drugs (69.8%) and by 266 of the 442 approved small-molecule drugs (60.2%). Eighty-two of the 581 novel drug–indication pairs (14.1%) used the Accelerated Approval Program; of those, 65 (79.3%) were oncology drugs and 70 (85.4%) had an orphan designation.Conclusions and RelevanceThe study showed that use of the FDA’s expedited programs to bring novel drugs to market in the US increased from 2008 to 2021. The findings suggest that this trend is likely to continue.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021

2022

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“…Timely approval of new oncology drugs saves lives and has attracted a lot of public attention. Drug regulatory agencies strive to approve novel oncology drugs in an expeditious way [ 17 ]. We found that oncology drugs were associated with shorter NDA approval time than non-oncology drugs, regardless of whether or not the drug was designated as PR, UNOD, or standard review.…”

Section: Discussionmentioning

confidence: 99%

Trends and Characteristics of New Drug Approvals in China, 2011–2021

Liu

et al. 2022

Ther Innov Regul Sci

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Background In the past decade, the Chinese drug regulatory system has undergone many changes. A major reform starting in 2015 has significantly reshaped the regulatory processes. It was important to assess the impact of the reform on new drug approvals in China. Method We analyzed the temporal trends of regulatory characteristics of the new drugs approved by the Chinese regulatory agency from 2011 to 2021, using data collected in the Pharmcube database. Results A total of 353 new drugs were approved, including 220 small molecule drugs, 86 biological products and 47 vaccines. The annual number of new drug approvals increased dramatically since 2017, reaching a record high of 70 in 2021. The median NDA approval time was 15.4 months in 2017-2021, the shortest in the decade, and was significantly shorter than that in the pre-reform period. The newly instituted expedited pathways such as priority review (PR) and accelerated approval for urgently needed overseas drugs (UNOD) significantly reduced new drug application (NDA) approval times compared with standard review. For imported drugs, in 2017-2021, the median time difference between the first approval in the world and the approval in China was 5 years, representing significant “drug lag”. However, the proportion of the imported drugs approved in China within 3 years of its first foreign approval has increased to 24.4% in 2017-2021. Conclusion The regulatory reform has produced significant, positive immediate outcomes in several metrics of drug regulatory approval. China’s regulatory system will continue to evolve as there still are many areas requiring further reform and improvement.

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“…To overcome the drug lag and cultivate a more innovationfriendly drug R&D ecosystem in China, the NMPA has implemented priority examination and approval since 2015, emphasizing clinical value-oriented approval (18). The milestone policy "Opinions on Reforming the Review and Approval System of Drugs and Medical Devices" was also issued to improve clinical development efficiency in China, together with many compatible regulatory reforms, such as restrictions on imported drug approval, which loosened that clinical trial data acquired abroad were accepted (11,19).…”

Section: Discussionmentioning

confidence: 99%

“…In 2020, a series of expedited programs were launched and updated to accelerate drug development and review in China, including breakthrough therapy, conditional approval, and priority review, which caused an increase in annual approvals (19). In contrast, the innovation system of R&D in the US has long been established, including expedited programs of drug reviews, which is why annual approvals were relatively stable (20).…”

Section: Discussionmentioning

confidence: 99%

Availability and Affordability of Oncology Drugs in 2012-2021 in China and the United States

Huang

Zhu²,

Ga³

et al. 2022

Front. Oncol.

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ObjectiveTo systematically summarize the landscape and characteristics of all approved new anticancer drugs for the last 10 years in China and the United States (US) to further inform the trend, current state, and existing gap in the availability and affordability of cancer medicine between the two countries.MethodsMainly based on the Pharmcube database, a list and detailed information of anticancer drugs approved in China and the United States were acquired. The annual number, time lag, and basic characteristics, including drug type, mechanism, enterprise type, indication population, drug target, and cancer type of approved drugs were compared.ResultsEighty-seven and 118 new anticancer drugs were approved in China and the US, respectively, showing a stable trend in the US, while a significant increase was observed after 2016 in China. Of the 42 cancer medicines launched in both countries, the US took precedence, and the median time lag markedly decreased, from 6.53 years in 2012 to 0.88 years in 2020. A total of 14.4% of drugs were applicable to children in the US, while only 2.3% were applicable in China, and there was no difference in drug type and enterprise. Thirty-one and 43 targets were explored, with respect to 27 and 36 cancer types in China and the US, respectively, during the period. In addition, the expenditure of drugs on PD-1 and PD-L1 in China was generally lower than that in America.ConclusionThe availability of new anticancer drugs has increased dramatically in the past decade, particularly in China. Compared with the US, the launch of new anticancer drugs in China lags behind, but the time lag has been shortened significantly, and better affordability is observed in immune drugs. More attention should be given to differentiated innovation, and unmet medical needs in special populations like childhood tumors, which are important directions of new drug R&D in China.

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An overview of cancer drugs approved through expedited approval programs and orphan medicine designation globally between 2011 and 2020

Cited by 22 publications

References 13 publications

Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021

Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021

Trends and Characteristics of New Drug Approvals in China, 2011–2021

Availability and Affordability of Oncology Drugs in 2012-2021 in China and the United States

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