Top

Orphanet Journal of Rare Diseases

Published in:

Open Access 01-12-2022 | Research

Current status and trend of clinical development of orphan drugs in China

Authors: Ziling Xiang, Wengao Jiang, Bo Yan, Junhao Jiang, Hang Zheng

Published in: Orphanet Journal of Rare Diseases | Issue 1/2022

Abstract

Background

Rare diseases have been increasingly recognized as unmet medical and health needs worldwide; a growing demand for the development of orphan drugs emerges subsequently. Therefore, it is of great interest for both the Chinese regulatory agency and pharmaceutical companies to keep tract on the clinical development of orphan drugs in China.

Objective and method

This study aims to reveal the current situation and trend of the clinical development of orphan drugs in China, based on the data collected from the Chinese official platform, dating from January 1, 2013 to December 31, 2021.

Results

A total of 331 clinical trials for orphan drugs were extracted from the platform, covering 31 rare diseases and 124 drugs. Increases were seen in the annual number of clinical trials and drugs being tested, with a sharp increase after 2018. About the disease types of the 331 trials, Parkinson disease (young-onset, early-onset) (86, 26%), hemophilia (70, 21%), homozygote hypercholesterolemia (60, 18%) were the most common. Furthermore, it was also observed that the largest number of clinical trial units for rare disease in east China (90, 41%) and the smallest number located in northwest China (18, 6%) and northeast China (18, 6%).

Conclusions

The growth trends illustrate the progress in clinical trial and drug development of rare diseases from 2013 to 2021. However, promoting orphan drugs development still is an important issue in China; at the same time, further efforts should be made for meet the unmet needs of disease types and balance the uneven distribution of medical resources for clinical trial on rare diseases.

Wakap SN, Lambert DM, Olry A, et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet. 2020;28(2):165–73. https://doi.org/10.1038/s41431-019-0508-0.CrossRef

Melnikova I. Rare diseases and orphan drugs editorial material. Nat Rev Drug Discov. 2012;11(4):267–8. https://doi.org/10.1038/nrd3654.CrossRefPubMed

Zhao H, Cui Y, Zhou X, et al. Study and analysis of the state of rare disease research in Shandong Province, China. Intractable Rare Dis Res. 2012;1(4):161–6. https://doi.org/10.5582/irdr.2012.v1.4.161.CrossRefPubMedPubMedCentral

National Health Commission of the People's Republic of China. Notice on the first official list of rare diseases. June 8, 2018 (in Chinese). https://www.nhc.gov.cn/yzygj/s7659/201806/393a9a37f39c4b458d6e830f40a4bb99.shtml. Accessed 10 Aug 2021.

Giannuzzi V, Conte R, Landi A, et al. Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen. Orphanet J Rare Dis. 2017. https://doi.org/10.1186/s13023-017-0617-1.CrossRefPubMedPubMedCentral

Gong SW, Wang YX, Pan XY, et al. The availability and affordability of orphan drugs for rare diseases in China. Orphanet J Rare Dis. 2016. https://doi.org/10.1186/s13023-016-0392-4.CrossRefPubMedPubMedCentral

Lihua LJZ, Zuo X, Lin L. Fundamental considerations in the clinical review of the registration application for the treatment of rare diseases. Chin J Clin Pharmacol. 2018;34(19):2372–5.

Fonseca DA, Amaral I, Pinto AC, Cotrim MD. Orphan drugs: major development challenges at the clinical stage. Drug Discov Today. 2019;24(3):867–72. https://doi.org/10.1016/j.drudis.2019.01.005.CrossRefPubMed

Kempf L, Goldsmith JC, Temple R. Challenges of developing and conducting clinical trials in rare disorders. Am J Med Genet Part A. 2018;176(4):773–83. https://doi.org/10.1002/ajmg.a.38413.CrossRefPubMed

10.

Yang L, Su C, Lee AM, Bai HX. Focusing on rare diseases in China: are we there yet? Orphanet J Rare Dis. 2015. https://doi.org/10.1186/s13023-015-0361-3.CrossRefPubMedPubMedCentral

11.

Dechartres A, Riveros C, Harroch M, Faber T, Ravaud P. Characteristics and public availability of results of clinical trials on rare diseases registered at Clinicaltrials.gov. JAMA Intern Med. 2016;176(4):556–8. https://doi.org/10.1001/jamainternmed.2016.0137.CrossRefPubMed

12.

Rees CA, Pica N, Monuteaux MC, Bourgeois FT. Noncompletion and nonpublication of trials studying rare diseases: a cross-sectional analysis. PLoS Med. 2019;16(11):e1002966. https://doi.org/10.1371/journal.pmed.1002966.CrossRefPubMedPubMedCentral

13.

Sakate R, Fukagawa A, Takagaki Y, Okura H, Matsuyama A. Trends of clinical trials for drug development in rare diseases. Curr Clin Pharmacol. 2018;13(3):199–208. https://doi.org/10.2174/1574884713666180604081349.CrossRefPubMedPubMedCentral

14.

He J, Tang M, Zhang X, et al. Incidence and prevalence of 121 rare diseases in China: current status and challenges. Intractable Rare Dis Res. 2019;8(2):89–97. https://doi.org/10.5582/irdr.2019.01066.CrossRefPubMedPubMedCentral

15.

National Health Committee. Notice on printing and distributing the catalogue of the first batch of drugs to encourage imitation (no. 744 of 2019). Oct 9, 2019 (in Chinese). http://www.nhc.gov.cn/yaozs/s7656/201910/38e3961482c04b59a2aa3e36106b1a4f.shtml. Accessed 3 June 2021.

16.

Sartor O, Halabi S. Independent data monitoring committees: an update and overview. Urol Oncol Semin Orig Invest. 2015;33(3):143–8. https://doi.org/10.1016/j.urolonc.2014.12.013.CrossRef

17.

Bell SA, Smith CT. A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov. 2014. Orphanet J Rare Dis. https://doi.org/10.1186/s13023-014-0170-0.

18.

The General Office of the Central Committee of the Communist Party of China, the General Office of the State Council. The General Office of the Central Committee of the Communist Party of China and the General Office of the State Council issued the “Opinions on deepening the reform of the examination and approval system and encouraging the innovation of pharmaceutical medical devices”. Oct 8, 2017 (in Chinese). http://www.gov.cn/zhengce/2017-10/08/content_5230105.htm. Accessed 3 June 2021.

19.

China Food and Drug Administration. Opinions of the General Administration on encouraging drug innovation to implement priority review and approval. Food and Drug Administration chemical tube (no. 126 of 2017). Dec 28, 2017 (in Chinese). http://www.nmpa.gov.cn/WS04/CL2196/324193.html. Accessed 3 June 3 2021.

20.

The State Council. State Council’s opinions on reforming the examination and approval system for drug and medical devices. Aug 18, 2015 (in Chinese). http://www.gov.cn/zhengce/content/2015-08/18/content_10101.htm. Accessed 3 June 2021.

21.

General Office of the State Council. State Council General Office’s opinion on the evaluation of the consistency of quality and efficacy of generic drugs. March 5, 2016 (in Chinese). http://www.gov.cn/zhengce/content/2016-03/05/content_5049364.htm. Accessed 21 Mar 2022.

22.

Center For Drug Evaluation. Notice on Issuing the "Technical Guidelines for Clinical Research and Development of Drugs for Rare Diseases". January 6, 2022 (in Chinese). https://www.cde.org.cn/main/news/viewInfoCommon/c4e1ef312a0a0c039a7a4ca55b91d4e8. Accessed 22 Mar 2022.

23.

National Medical Products Administration. Drug Administration Law of the People's Republic of China. December 1, 2019 (in Chinese). https://www.nmpa.gov.cn/xxgk/fgwj/flxzhfg/20190827083801685.html. Accessed 21 Mar 2022.

24.

. National Medical Products Administration. Working procedure on the priority review of drug approval (interim). July 7, 2020 (in Chinese). https://www.nmpa.gov.cn/zhuanti/ypqxgg/ggzhcfg/20200708151701834.html. Accessed 21 Mar 2022.

25.

Mullard A. FDA approves ultra-orphan drug on a 4-patient trial. Nat Rev Drug Discov. 2015;14(10):669–669. https://doi.org/10.1038/nrd4756.CrossRefPubMed

26.

Shi XM, Liu H, Wang L, et al. Study on the current situation of China’s First List of Rare Diseases based on 15 million hospitalizations. Zhonghua yi xue za zhi. 2018;98(40):3274–8. https://doi.org/10.3760/cma.j.issn.0376-2491.2018.40.012.CrossRefPubMed

27.

National Heath Committee. Notice on printing and distributing the requirements for the work of the construction of the major clinical drug evaluation technology platform for major new drugs. Jan 21, 2019 (in Chinese). http://www.nhc.gov.cn/qjjys/s3593k/201901/f4b2ff724a564b35964b296b427b9002.shtml. Accessed 3 June 2021.

Title: Current status and trend of clinical development of orphan drugs in China
Authors: Ziling Xiang
Wengao Jiang
Bo Yan
Junhao Jiang
Hang Zheng
Publication date: 01-12-2022
Publisher: BioMed Central
Published in: Orphanet Journal of Rare Diseases / Issue 1/2022
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/s13023-022-02440-4

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Current status and trend of clinical development of orphan drugs in China

Abstract

Background

Objective and method

Results

Conclusions

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Background

Objective and method

Results

Conclusions

Please log in to get access to this content

Other articles of this Issue 1/2022

Identifying adult hypophosphatasia in the rheumatology unit

Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study

Literature review and expert opinion on the impact of achondroplasia on medical complications and health-related quality of life and expectations for long-term impact of vosoritide: a modified Delphi study

Factors of family impact in a Swedish–German cohort of children born with esophageal atresia

A retrospective cohort study on European Reference Network for Rare Vascular Diseases 5 outcome measures for Hereditary Haemorrhagic Telangiectasia in Denmark

Impact of a digital manual for guidance on malignant hyperthermia: patient education