Analysis of risks and countermeasures for the temporary import of clinically urgent drugs based on focus group interviews and Delphi expert consultation method_Chinese Journal of Evidence-Based Medicine

Authors：

YI Qiusha ^1,2,3 , LU Runxin ^1,2,3 , QIU Panda ^1,2,3,4 , ZENG Linan ^1,2,3,5 , ZHANG Faqiong ⁶ , ZOU Kun ^1,2,3 , SHI Yuqing ^1,2,3 ,  HAN Lu ^1,2,3 ,  ZHANG Lingli ^1,2,3,5,7

1. Department of Pharmacy/Evidence-Based Pharmacy Center, West China Second University Hospital, Sichuan University; Children's Medicine Key Laboratory of Sichuan Province, Chengdu, 610041, P.R.China;
2. NMPA Key Laboratory for Technical Research on Drug Products in vitro and in vivo Correlation, Chengdu, 610041, P.R.China;
3. Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu, 610041, P.R.China;
4. West China School of Pharmacy, Sichuan University, Chengdu, 610041, P.R.China;
5. West China Biomedical Big Data Center, West China Hospital, Sichuan University, Chengdu, 610041, P.R.China;
6. West China School of Public Health, Sichuan University, Chengdu, 610041, P.R.China;
7. Chinese Evidence-based Medicine Center, West China Hospital, Sichuan University, Chengdu, 610041, P.R.China;

Corresponding author：

HAN Lu, Email: luhan830301@sina.com; ZHANG Lingli, Email: zhanglingli@scu.edu.cn

Keywords：

Urgently needed in clinical practice; Temporary import; Drugs; Focus group

DOI：

10.7507/1672-2531.202502060

Video：

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Objective To analyze the issues and risks associated with the policy of temporary import of urgently needed clinical drugs and offer optimized suggestions for this domain in our country. Methods The focus group interview method and in-depth interview method were employed, and 34 individuals related to the temporary import of urgently needed clinical drugs were selected for the interviews. Based on the results of the review of domestic and international policies for the temporary import of urgently needed clinical drugs and the conclusions drawn from the interviews, a questionnaire was formulated. The Delphi expert consultation method was utilized to select 14 experts from various relevant fields, and questionnaires were distributed and collected by mail for objective presentation. Through an expert consensus meeting, the final suggestions were formed. Results Initially, 20 suggestions were proposed for each stage of the temporary import of urgently needed clinical drugs. After expert consultation, 32 questionnaires were retrieved (a response rate of 91%), and all were valid (a validity rate of 100%). Through discussions at the expert consensus meeting, 17 policy suggestions were ultimately formed. Conclusion It is recommended that the National Health Commission, in collaboration with the National Medical Products Administration and the customs department, conduct in-depth discussions and refine the specific implementation measures for the temporary import of urgently needed clinical drugs to guarantee the safety, efficacy, and timeliness of clinical drug use.

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7.	Joshi C, Nickels K, Demarest S, et al. Results of an international Delphi consensus in epilepsy with myoclonic atonic seizures/ Doose syndrome. Seizure, 2021, 85: 12-18.
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10.	Dressler A, Trimmel-Schwahofer P, Reithofer E, et al. Efficacy and tolerability of the ketogenic diet in Dravet syndrome - comparison with various standard antiepileptic drug regimen. Epilepsy Res, 2015, 109: 81-89.
11.	Trivisano M, Striano P, Sartorelli J, et al. CHD2 mutations are a rare cause of generalized epilepsy with myoclonic-atonic seizures. Epilepsy Behav, 2015, 51: 53-56.
12.	Vlaskamp DR, Rump P, Callenbach PM, et al. Haploinsufficiency of the STX1B gene is associated with myoclonic astatic epilepsy. Eur J Paediatr Neurol, 2016, 20: 489-492.
13.	Song L, Liu F, Liu Y, et al. Clonazepam add-on therapy for drug-resistant epilepsy. Cochrane Database Syst Rev, 2020, 4: Cd012253.
14.	晏国文, 曹学平. 国产罕见病药品氯巴占加速入院. 中国经营报, 2022-11-14(B16).
15.	Resolution of the government of the Russian federation No. 230 of March 5, 2020. 2020.
16.	Resolution of the government of the Russian federation No. 593 of April 5, 2022. 2022.
17.	Resolution of the government of the Russian federation No. 3341-r of November 26, 2021. 2021.
18.	Resolution of the government of the Russian federation No. 853 of June 1, 2021. 2021.
19.	Federal state unitary enterprise moscow endocrine plant imported 3000 packages of the unregistered medicine with INN clobazam for treatment of patients with epilepsy. 2021.
20.	FSUE “endopharm” imported “ethosuximide” not registered in the Russian federation for treatment of children with epilepsy. 2021.

1. 中华人民共和国国家卫生健康委员会法规司. 中华人民共和国药品管理法. 2019.
2. 中华人民共和国国家卫生健康委员会. 临床急需药品临时进口工作方案. 2022.
3. 中华人民共和国国家卫生健康委员会. 氯巴占临时进口工作方案. 2022.
4. 曹颖, 王森, 邹锟, 等. 罕见病用药医保现状分析及思考. 赣南医学院学报, 2023, 43(6): 620-625,636.
5. 张波, 张抒扬, 许婷婷, 等. 氯巴占治疗难治性癫痫专家共识(2022). 协和医学杂志, 2022, 13(5): 768-782.
6. Nakajima H. A pharmacological profile of clobazam (Mystan), a new antiepileptic drug. Nihon Yakurigaku Zasshi, 2001, 118: 117-122.
7. Joshi C, Nickels K, Demarest S, et al. Results of an international Delphi consensus in epilepsy with myoclonic atonic seizures/ Doose syndrome. Seizure, 2021, 85: 12-18.
8. Montouris G, Aboumatar S, Burdette D, et al. Expert opinion: proposed diagnostic and treatment algorithms for Lennox-Gastaut syndrome in adult patients. Epilepsy Behav, 2020, 110: 107146.
9. Wirrell EC, Laux L, Franz DN, et al. Stiripentol in Dravet syndrome: results of a retrospective U. S. study . Epilepsia, 2013, 54: 1595-1604.
10. Dressler A, Trimmel-Schwahofer P, Reithofer E, et al. Efficacy and tolerability of the ketogenic diet in Dravet syndrome - comparison with various standard antiepileptic drug regimen. Epilepsy Res, 2015, 109: 81-89.
11. Trivisano M, Striano P, Sartorelli J, et al. CHD2 mutations are a rare cause of generalized epilepsy with myoclonic-atonic seizures. Epilepsy Behav, 2015, 51: 53-56.
12. Vlaskamp DR, Rump P, Callenbach PM, et al. Haploinsufficiency of the STX1B gene is associated with myoclonic astatic epilepsy. Eur J Paediatr Neurol, 2016, 20: 489-492.
13. Song L, Liu F, Liu Y, et al. Clonazepam add-on therapy for drug-resistant epilepsy. Cochrane Database Syst Rev, 2020, 4: Cd012253.
14. 晏国文, 曹学平. 国产罕见病药品氯巴占加速入院. 中国经营报, 2022-11-14(B16).
15. Resolution of the government of the Russian federation No. 230 of March 5, 2020. 2020.
16. Resolution of the government of the Russian federation No. 593 of April 5, 2022. 2022.
17. Resolution of the government of the Russian federation No. 3341-r of November 26, 2021. 2021.
18. Resolution of the government of the Russian federation No. 853 of June 1, 2021. 2021.
19. Federal state unitary enterprise moscow endocrine plant imported 3000 packages of the unregistered medicine with INN clobazam for treatment of patients with epilepsy. 2021.
20. FSUE “endopharm” imported “ethosuximide” not registered in the Russian federation for treatment of children with epilepsy. 2021.

Chinese Journal of Evidence-Based Medicine

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