The value assessment criteria of orphan drugs in medical insurance access: a qualitative systematic analysis_Chinese Journal of Evidence-Based Medicine

Authors：

ZHOU Jing ¹ , YANG Nan ¹ , SHI Lizheng ² ,  HU Ming ¹ ,  CHANG Rangdan ³

1. West China School of Pharmacy, Sichuan University, Chengdu, 610041, P.R.China;
2. School of Public Health and Tropical Medicine, Tulane University, LA 70112, USA;
3. Sichuan Medical Device Bidding Service Center, Chengdu, 610021, P.R.China;

Corresponding?author：

HU Ming, Email: huming@scu.edu.cn; CHANG Rangdan, Email: changranger@163.com

Keywords：

Orphan medical product; Value assessment; Qualitative systematic analysis

DOI：

10.7507/1672-2531.201907043

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Objectives To provide a useful framework for improved understanding of international value drivers in the decision-making process of medical insurance access, and to explore the value assessment criteria of orphan drugs from stakeholders’ perspective.Methods PubMed, EMbase, CINAHL Plus, ProQuest, Web of Science, CNKI and WanFang Data databases were electronically searched to collect studies from January 1^st, 1983 to December 31^st, 2018. Research questions were constructed based on SPIDER model. We established the inclusion and exclusion criteria to filter studies. Study quality was evaluated using the Critical Appraisal Skills Programme (CASP) checklist. A thematic synthesis was undertaken to develop descriptive themes, analytical constructs and third-level themes of value drivers by NVivo 11 software, and confidence in the findings was assessed using the CERQual method.Results A total of 10 studies including 20 research countries were included. Fifty descriptive themes were interpreted and embedded within 14 analytical constructs and 3 third-level themes after induction. Specifically, 3 broad themes were disease-related influence factors, which included severity, unmet requirements, disease burden, affected individuals, and moral and ethical considerations; drug-related influence factors, which included safety, efficacy, economics, innovation, quality of evidence; and some external and non-pharmaceutical intrinsic properties factors, which included reimbursement status in other countries or regions, government goals and priorities, confirmed drug supply and impact on environment.Conclusions In addition to conventional considerations such as efficacy and pharmacoeconomics, stakeholders are willing to take a broader perspective when in the case of the value assessment of orphan drugs. Comprehensive understanding of these value drivers is important to shape policy and enhance decision-making.

Citation： ZHOU Jing, YANG Nan, SHI Lizheng, HU Ming, CHANG Rangdan. The value assessment criteria of orphan drugs in medical insurance access: a qualitative systematic analysis. Chinese Journal of Evidence-Based Medicine, 2019, 19(11): 1292-1299. doi: 10.7507/1672-2531.201907043 Copy

1.	Jaime CJ. Editorial: Pursuing efficiency: a dead end for HTA? Value Health, 2009, 12(Suppl 2): S49.
2.	Friedmann C, Levy P, Hensel P, et al. Using multi-criteria decision analysis to appraise orphan drugs: a systematic review. Expert Rev Pharmacoecon Outcomes Res, 2018, 18(2): 135-146.
3.	Cohen JP, Felix A. Are payers treating orphan drugs differently? J Mark Access Health Policy, 2014, 2: 23513.
4.	胡娟娟, 陳昕, 龔時薇. 英國, 加拿大和澳大利亞國家衛生服務保障體系下罕用藥遴選原則分析. 醫學與社會, 2016, 29(1): 10-12+16.
5.	黃崇斐, 拜爭剛, 吳淑婷, 等. 定性系統評價的撰寫方法介紹. 中國循證醫學雜志, 2015, 15(9): 1106-1111.
6.	陳敏, 張伶俐, 李幼平, 等. 罕見疾病治療藥物政策的循證評價. 中國循證醫學雜志, 2015, 15(7): 802-809.
7.	Critical Appraisal Skills Programme (CASP) quality-assessment tool. Available at: http://www.casp-uk.net.
8.	拜爭剛, 劉少堃, 黃崇斐, 等. 定性系統評價證據分級工具—系統評價證據分簡介. 中國循證醫學雜志, 2015, 15(12): 1465-1470.
9.	Nicod E, Kanavos P. Scientific and social value judgments for orphan drugs in health technology assessment. Int J Technol Assess Health Care, 2016, 32(4): 218-232.
10.	Nicod E, Berg BK, Durand-Zaleski I, et al. Dealing with uncertainty and accounting for social value judgments in assessments of orphan drugs: evidence from four European countries. Value Health, 2017, 20(7): 919-926.
11.	Rosenberg-Yunger ZR, Daar AS, Thorsteinsdóttir H, et al. Priority setting for orphan drugs: an international comparison. Health Policy, 2011, 100(1): 25-34.
12.	Picavet E, Cassiman D, Simoens S. Reimbursement of orphan drugs in Belgium: what (else) matters? Orphanet J Rare Dis, 2014, 9: 139.
13.	Schlander M, Garattini S, Kolominsky-Rabas P, et al. Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement. J Mark Access Health Policy, 2016, 4: 33039.
14.	Torrent-Farnell J, Comellas M, Poveda JL, et al. The view of experts on initiatives to be undertaken to promote equity in the access to orphan drugs and specialised care for rare diseases in Spain: a Delphi consensus. Health Policy, 2018, 122(6): 590-598.
15.	Annemans L, Aymé S, Le Cam Y, et al. Recommendations from the European working group for value assessment and funding processes in rare diseases (ORPH-VAL). Orphanet J Rare Dis, 2017, 12(1): 50.
16.	Nicod E, Kanavos P. Developing an evidence-based methodological framework to systematically compare HTA coverage decisions: A mixed methods study. Health Policy, 2016, 120(1): 35-45.
17.	Kamusheva M, Manova M, Savova AT, et al. Comparative analysis of legislative requirements about patients' access to biotechnological drugs for rare diseases in central and eastern European countries. Front Pharmacol, 2018, 9: 795.
18.	Menon D, Clark D, Stafinski T. Reimbursement of drugs for rare diseases through the public healthcare system in Canada: where are we now? Healthc Policy, 2015, 11(1): 15-32.
19.	張抒揚. 關愛罕見病, 從保障用藥開始. 中國衛生, 2019, 34(5): 64-65.
20.	Zelei T, Molnár MJ, Szegedi M, et al. Systematic review on the evaluation criteria of orphan medicines in central and eastern European countries. Orphanet J Rare Dis, 2016, 11(1): 72.
21.	Paulden M, Stafinski T, Menon D, et al. Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework. Pharmacoeconomics, 2015, 33(3): 255-269.
22.	Schnipper LE, Davidson NE, Wollins DS, et al. American society of clinical oncology statement: a conceptual framework to assess the value of cancer treatment options. J Clin Oncol, 2015, 33(23): 2563-2577.
23.	A framework for payer assessment of the value of new technologies: a US appoach. Available at: https://www.ohe.org/news/framework-payer-assessment-value-new-technologies-us-approach.

1. Jaime CJ. Editorial: Pursuing efficiency: a dead end for HTA? Value Health, 2009, 12(Suppl 2): S49.
2. Friedmann C, Levy P, Hensel P, et al. Using multi-criteria decision analysis to appraise orphan drugs: a systematic review. Expert Rev Pharmacoecon Outcomes Res, 2018, 18(2): 135-146.
3. Cohen JP, Felix A. Are payers treating orphan drugs differently? J Mark Access Health Policy, 2014, 2: 23513.
4. 胡娟娟, 陳昕, 龔時薇. 英國, 加拿大和澳大利亞國家衛生服務保障體系下罕用藥遴選原則分析. 醫學與社會, 2016, 29(1): 10-12+16.
5. 黃崇斐, 拜爭剛, 吳淑婷, 等. 定性系統評價的撰寫方法介紹. 中國循證醫學雜志, 2015, 15(9): 1106-1111.
6. 陳敏, 張伶俐, 李幼平, 等. 罕見疾病治療藥物政策的循證評價. 中國循證醫學雜志, 2015, 15(7): 802-809.
7. Critical Appraisal Skills Programme (CASP) quality-assessment tool. Available at: http://www.casp-uk.net.
8. 拜爭剛, 劉少堃, 黃崇斐, 等. 定性系統評價證據分級工具—系統評價證據分簡介. 中國循證醫學雜志, 2015, 15(12): 1465-1470.
9. Nicod E, Kanavos P. Scientific and social value judgments for orphan drugs in health technology assessment. Int J Technol Assess Health Care, 2016, 32(4): 218-232.
10. Nicod E, Berg BK, Durand-Zaleski I, et al. Dealing with uncertainty and accounting for social value judgments in assessments of orphan drugs: evidence from four European countries. Value Health, 2017, 20(7): 919-926.
11. Rosenberg-Yunger ZR, Daar AS, Thorsteinsdóttir H, et al. Priority setting for orphan drugs: an international comparison. Health Policy, 2011, 100(1): 25-34.
12. Picavet E, Cassiman D, Simoens S. Reimbursement of orphan drugs in Belgium: what (else) matters? Orphanet J Rare Dis, 2014, 9: 139.
13. Schlander M, Garattini S, Kolominsky-Rabas P, et al. Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement. J Mark Access Health Policy, 2016, 4: 33039.
14. Torrent-Farnell J, Comellas M, Poveda JL, et al. The view of experts on initiatives to be undertaken to promote equity in the access to orphan drugs and specialised care for rare diseases in Spain: a Delphi consensus. Health Policy, 2018, 122(6): 590-598.
15. Annemans L, Aymé S, Le Cam Y, et al. Recommendations from the European working group for value assessment and funding processes in rare diseases (ORPH-VAL). Orphanet J Rare Dis, 2017, 12(1): 50.
16. Nicod E, Kanavos P. Developing an evidence-based methodological framework to systematically compare HTA coverage decisions: A mixed methods study. Health Policy, 2016, 120(1): 35-45.
17. Kamusheva M, Manova M, Savova AT, et al. Comparative analysis of legislative requirements about patients' access to biotechnological drugs for rare diseases in central and eastern European countries. Front Pharmacol, 2018, 9: 795.
18. Menon D, Clark D, Stafinski T. Reimbursement of drugs for rare diseases through the public healthcare system in Canada: where are we now? Healthc Policy, 2015, 11(1): 15-32.
19. 張抒揚. 關愛罕見病, 從保障用藥開始. 中國衛生, 2019, 34(5): 64-65.
20. Zelei T, Molnár MJ, Szegedi M, et al. Systematic review on the evaluation criteria of orphan medicines in central and eastern European countries. Orphanet J Rare Dis, 2016, 11(1): 72.
21. Paulden M, Stafinski T, Menon D, et al. Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework. Pharmacoeconomics, 2015, 33(3): 255-269.
22. Schnipper LE, Davidson NE, Wollins DS, et al. American society of clinical oncology statement: a conceptual framework to assess the value of cancer treatment options. J Clin Oncol, 2015, 33(23): 2563-2577.
23. A framework for payer assessment of the value of new technologies: a US appoach. Available at: https://www.ohe.org/news/framework-payer-assessment-value-new-technologies-us-approach.

Chinese Journal of Evidence-Based Medicine

The value assessment criteria of orphan drugs in medical insurance access: a qualitative systematic analysis

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