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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3.	中華人民共和國國家衛生健康委員會. 氯巴占臨時進口工作方案. 2022.
4.	曹穎, 王森, 鄒錕, 等. 罕見病用藥醫保現狀分析及思考. 贛南醫學院學報, 2023, 43(6): 620-625,636.
5.	張波, 張抒揚, 許婷婷, 等. 氯巴占治療難治性癲癇專家共識(2022). 協和醫學雜志, 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.	晏國文, 曹學平. 國產罕見病藥品氯巴占加速入院. 中國經營報, 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. 中華人民共和國國家衛生健康委員會法規司. 中華人民共和國藥品管理法. 2019.
2. 中華人民共和國國家衛生健康委員會. 臨床急需藥品臨時進口工作方案. 2022.
3. 中華人民共和國國家衛生健康委員會. 氯巴占臨時進口工作方案. 2022.
4. 曹穎, 王森, 鄒錕, 等. 罕見病用藥醫保現狀分析及思考. 贛南醫學院學報, 2023, 43(6): 620-625,636.
5. 張波, 張抒揚, 許婷婷, 等. 氯巴占治療難治性癲癇專家共識(2022). 協和醫學雜志, 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. 晏國文, 曹學平. 國產罕見病藥品氯巴占加速入院. 中國經營報, 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.

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