REASONS FOR DIFFICULTY OF THE PHARMACOECONOMIC ANALYSIS FOR ORPHAN DRUGS. WAYS FOR SOLUTIONS
Abstract
Orphan drugs are used for treatment of rare life-threatening diseases. There is no universal definition of a rare disease and no universal approach to orphan drugs legislation. As a result, decision-making in this field requires results of pharmacoeconomic analysis, which structure and interpretation need a specific approach due to high costs of the drugs and limited sample size. Cost-utility analysis, risk management, and international rare diseases patient registries could be used to solve these problems.
About the Authors
Andrey Vladislavovich Pavlysh
First St. Petersburg State Medical University;
The Federal State Budgetary Institute «The Nikiforov Russian Center of Emergency and Radiation Medicine» The Ministry of Russian Federation for Civil Defense, Emergencies and Elimination of Consequences of Natural Disasters
Russian Federation
Russian Federation
MD, intramural doctoral of department of public health and health care,
Leo Tolstoy, 6-8, St. Petersburg, 197022;
head of department of medical supplies and providing medical process – clinical pharmacologist,
Acad. Lebedev St., 4/2, St. Petersburg, 194044
Aleksei Sergeevich Kolbin
First St. Petersburg State Medical University;
First St. Petersburg State University
Russian Federation
Russian Federation
MD, professor, head of department of clinical pharmacology and evidence-based medicine,
Leo Tolstoy, 6-8, St. Petersburg, 197022;
Professor of the Department of Farmakology,
21-Line, 8, VO, St. Petersburg, Russia, 199106
Roman Andreevich Gapeshin
St. Petersburg State University
Russian Federation
Russian Federation
MD, graduate,
21-Line, 8, VO, St. Petersburg, 199106
Stanislav Mikhailovich Malyshev
St. Petersburg State University
Russian Federation
Russian Federation
MD, graduating student,
21-Line, 8, VO, St. Petersburg, 199106
References
1. Ministry of Health data on the number of patients with orphan diseases to re-registers, the number of prescriptions written, in terms of funding. [Dannye Minzdrava po kolichestvu bol'nykh orfannymi zabolevaniyami v registre, po kolichestvu vypisannykh retseptov, po ob'emu finansirovaniya (In Russian)]. URL:https://www.rosminzdrav.ru/opendata (accessed 12.03.2015)
2. Health Technology Assessment: Manual, ed. Ed. YB Belousov. [Otsenka meditsinskikh tekhnologii: metod. posob. Pod obshch. red. Yu.B. Belousova (In Russian)]. Moscow. 2013; 42.
3. Government Decree of 26.04.2012 N 403 (ed. By 04.09.2012) «On the order of the Federal Register of persons suffering from lifethreatening and chronic progressive rare (orphan) diseases that can shorten life expectancy of citizens or their disability, and regional segment. [Postanovlenie Pravitel'stva RF ot 26.04.2012 N 403 (red. ot 04.09.2012) «O poryadke vedeniya Federal'nogo registra lits, stradayushchikh zhizneugrozhayushchimi i khronicheskimi progressiruyushchimi redkimi (orfannymi) zabolevaniyami, privodyashchimi k sokrashcheniyu prodolzhitel'nosti zhizni grazhdan ili ikh invalidnosti, i ego regional'nogo segmenta» (In Russian)]. URL:http://base.consultant.ru/cons/cgi/online.cgi?req=doc;base=LAW;n=135253;div=LAW;dst=100003,0;rnd=0.6197177267167717 (accessed 12.03.2015).
4. Federal Law of 21.11.2011 N 323-FZ (ed. By 06.04.2015) «On the basis of the health of citizens in the Russian Federation» (rev. And ext., Joined. In force from 05.09.2015). [Federal'nyj zakon ot 21.11.2011 N 323-FZ (red. ot 06.04.2015) «Ob osnovah ohrany zdorov'ja grazhdan v Rossijskoj Federacii» (s izm. i dop., vstup. v silu s 09.05.2015) (In Russian)]. URL: http://base.consultant.ru/cons/cgi/online.cgi?req=doc;base=LAW;n=176374;div=LAW;dst=100465,0;rnd=0.0031823667231947184 (accessed 12.03.2015).
5. Aballea S., Toumi M., Vataire A., et al. Quantitative Analysis of the Influence of Disease and Product Characteristics on Orphan Drug Prices in Europe. Value In Health. 2010; 13 (3): A82.
6. Batshaw M., Groft S., Krischer J. Research Into Rare Diseases of Childhood. JAMA. 2014; 311 (17): 1729-1730.
7. Cohen D., Reynolds M. Interpreting the Results of CostEffectiveness Studies. J Am Coll Cardiol. 2008; 52: 2119-2126.
8. Commission of the European Communities (2000). Commission Regulation (EC) No 847/2000 of 27 April 2000 laying down the provisions for implementation of the criteria for designation of a medicinal product as an orphan medicinal product and definitions of the concepts «similar medicinal product» and «clinical superiority». Official Journal of the European Communities. 2000; 28 (4).
9. Congress Public Law 280, 107th. From the U.S. Government Printing Office. Page 1987. RARE DISEASES ACT OF 2002. URL: http://www.gpo.gov/fdsys/pkg/PLAW-107publ280/html/PLAW-107publ280.htm (accessed: 12.03.2015)
10. Coyle D., Cheung M., Evans G. Opportunity Cost of Funding Drugs for Rare Diseases: The Cost-Effectiveness of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. Med Decis Making. 2014; 34: 1016-1029.
11. Denis A., Mergaert L., Fostier C., et al. A comparative study of European rare disease and orphan drug markets. Health Policy. 2010; 97: 173-179.
12. Douglas C., Wilcox E., Burgess M., et al. Why orphan drug coverage reimbursement decision-makingneeds patient and public involvement. Health Policy. 2015; 119: 588-596.
13. Drummond M., Towse A. Orphan drugs policies: a suitable case for treatment. Eur J Health Econ. 2014; 15: 335-340.
14. Ferner R., Hughes D. The problem of orphan drugs. BMJ. 2010; 341: c6456.
15. Finkel M. The Orphan Drug Act and the Federal Government's Orphan Products Development Program. Public Health Reports. 1984; 99 (3): 313-316.
16. Fox J. Rare-disease drugs boosted by new Prescription Drug User Fee Act. Nature Biotechnology. 2012; 30 (8): 733-734.
17. Hall A., Carlson M. The current status of orphan drug development in Europe and the US. Intractable & rare diseases research. 2014; 3 (1): 1-7.
18. Hollis A. In: Health services restructuring in Canada: new evidence and new directions. Beach C, editor. Montreal: McGillQueen's University Press; 2006. Drugs for rare diseases: paying for innovation.
19. Hughes D., Tunnage B., Yeo S. Drugs for exceptionally rare diseases: do they deserve special status for funding? Q J Med. 2005; 98: 829-836.
20. Hyry H., Stern A., Cox T., et al. Limits on use of health economic assessments for rare diseases. Q J Med. 2014; 107: 241-245.
21. Jakubiak-Lasocka J., Jakubczyk M. Cost-effectiveness versus Cost-Utility Analyses: What Are the Motives Behind Using Each and How Do Their Results Differ? – A Polish Example. Value In Health Regional. 2014; 4C: 66-74.
22. Jessop N. The Dilemma with Orphan Drugs. Pharmaceutical Technology Europe. 2013; 25 (8): 8-9.
23. Joppi R., Bertele V., Garattini S. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur J Clin Pharmacol. 2013; 69: 1009-1024.
24. Kanters T., Steenhoek A., Hakkaart L. Budget Impact of Orphan Drugs In the Netherlands In The Period 2006-2012. Value In Health. 2014; 17 (7): A525.
25. Kesselheim A., Gagne J. Strategies for Postmarketing Surveillance of Drugs for Rare Diseases. Clinical pharmacology & Therapeutics. 2014; 95 (3): 265-268.
26. Logviss K., Krievins D., Purvina S. Rare diseases and orphan drugs: Latvian story. Orphanet Journal of Rare Diseases. 2014; 9 (1): 147.
27. Loughnot D. Potential interactions of the Orphan Drug Act and pharmacogenomics: A flood of orphan drugs and abuses? Am J Law Med. 2005; 31 (2-3): 365-380.
28. Luzatto L., Hollak C., Cox T., et al. Rare diseases and effective treatments: are we delivering? Lancet. 2015; 385 (9970): 750-752.
29. Messori A., Cicchetti A., Patregani L. Orphan drugs. Relating price determination to disease prevalence. BMJ. 2010; 341: c4615.
30. Michel M., Toumi M. Access to orphan drugs in Europe: current and future issues. Expert Rev. Pharmacoeconomics Outcomes Res. 2012; 12 (1): 23-29.
31. Miners A. Estimating 'Costs' for Cost-Effectiveness Analysis. PharmacoEconomics. 2008; 26 (9): 745-751.
32. Murphy S., Puwanant A., Griggs R., et al. Unintended Effects of Orphan Product Designation for Rare Neurological Diseases. Ann Neurol. 2012; 72: 481-490.
33. Picavet E., Cassiman D., Simoens S. What is known about the cost-effectiveness of orphan drugs? Evidence from cost-utility analyses. Journal of Clinical Pharmacy and Therapeutics. 2015; 40: 304-307.
34. Picavet E., Dooms M., Cassiman D. et al. Orphan Drugs for Rare Diseases: Grounds for Special Status. Drug Development Research. 2012; 73:115-119.
35. Picavet E., Dooms M., Cassiman D., et al. Drugs for rare diseases: Influence of orphan designation status on price. Appl Health Econ Health Policy. 2011; 9 (4): 275-279.
36. Picavet E., Morel T., Cassiman D., et al. Shining a light in the black box of orphan drug pricing. Orphanet J Rare Dis. 2014; 9 (1): 62.
37. Rare Disease Act of 2002. http://history.nih.gov/research/downloads/PL107-280.pdf (accessed 12.03.2015).
38. Roos J., Hyry H., Cox T. Orphan drug pricing may warrant a competition law investigation. BMJ. 2010; 341 (7782): 1084-1086.
39. Schunemann H., Hill S., Guyatt G., et al. The GRADE approach and Bradford Hill’s criteria for causation. J Epidemiol Community Health. 2011; 65: 392-395.
40. Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011; 6 (1): 42.
41. Simoens S., Cassiman D., Dooms M., et al. Orphan Drugs for Rare Diseases. Is it Time to Revisit Their Special Market Access Status? Drugs. 2012; 72 (11): 1437-1443.
42. Simoens S., Cassiman D., Picavet E., et al. Are some orphan drugs for rare diseases too expensive? A study of purchase versus compounding costs. Drugs Ther Perspect. 2011; 27 (10): 24-26.
43. Simoens S., Picavet E., Dooms M., et al. Cost-Effectiveness Assessment of Orphan Drugs. Appl Health Econ Health Policy. 2013; 11: 1-3.
44. US Code of Federal Regulations (CFR) Title 21 Food and Drugs Part 316.31 Scope of 44. Orphan Drug Exclusive Approval. URL http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=316.31. (accessed: 12.03.2015)
45. Vegter S., Rozenbaum M., Postema R., et al. Review of Regulatory Recommendations for Orphan Drug Submissions in the Netherlands and Scotland: Focus on the Underlying Pharmacoeconomic Evaluations. Clinical Therapeutics. 2010; 32 (9): 1651-1661.
46. Winquist E., Bell C., Clarke J., et al. An Evaluation Framework for Funding Drugs for Rare Diseases. Value In Health. 2012; 15: 982-986.
For citation:
Pavlysh A.V., Kolbin A.S., Gapeshin R.A., Malyshev S.M. REASONS FOR DIFFICULTY OF THE PHARMACOECONOMIC ANALYSIS FOR ORPHAN DRUGS. WAYS FOR SOLUTIONS. FARMAKOEKONOMIKA. Modern Pharmacoeconomic and Pharmacoepidemiology. 2015;8(2):3-10. (In Russ.) https://doi.org/10.17749/2070-4909.2015.8.2.003-010
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