POBOLJŠANJE PROCENE ODNOSA TROŠKOVA I EFEKATA LEKOVA ZA RETKE BOLESTI
Sažetak
Pozadina: Inkrementalni odnos cena/efikasnost (ICER) mnogih lekova za retke bolesti je često mnogo veći od prihvaćenog praga isplativosti za refundaciju, prvenstveno zbog njihovih izuzetno visokih cena, što postavlja pitanje njihove dostupnosti.
Cilj: Cilj ovog rada bio je da se sagleda neophodna prilagođavanja metoda koje se koriste za analizu troškova/efikasnosti lekova za retke bolesti.
Metode: Ovaj članak predstavlja narativni pregled metoda za prilagođavanje analize cene/efikasnosti lekova za retke bolesti kako bi se dobila realnija procena ICER praga, što je suštinska informacija za donosioce odluka.
Rezultati: Ulazne podatke u analizu troškova/efikasnosti leka za retke bolesti treba prilagoditi promenom diskontne stope, preciznijom procenom komunalnih usluga, isključujući troškove koji nisu povezani sa lečenjem, izračunavanjem lokalnog C/E praga, i što je najvažnije, pregovaranjem o leku cena dok se ne pređe C/E prag. Sa intenziviranim prilagođenim istraživanjem troškova/efikasnosti u ovoj oblasti, mnoge neizvesnosti će biti okončane, a stvarna vrednost mnogih lekova za retke bolesti biće poznata, što će uticati na cene u održivom pravcu.
Zaključak: Sa prilagođavanjima će se pristupiti stvarnoj ceni/efikasnosti leka za retke bolesti, omogućavajući na osnovu dokaza i potpuno transparentne odluke o nadoknadi.
Reference
1. Silva EN da, Sousa TRV. Economic evaluation in the context of rare diseases: is it possible? Cad Saude Publica. 2015;31(3):496–506.
2. Shafie AA, Chaiyakunapruk N, Supian A, et al. State of rare disease management in Southeast Asia. Orphanet J Rare Dis. 2016;11(1):107.
3. Giugliani L, Vanzella C, Zambrano MB, et al. Clinical research challenges in rare genetic diseases in Brazil. Genet Mol Biol. 2019;42(1 suppl 1):305–11.
4. Jayasundara K, Hollis A, Krahn M, et al. Estimating the clinical cost of drug development for orphan versus non-orphan drugs. Orphanet J Rare Dis. 2019;14(1):12.
5. Zajdel J, Zajdel R. Brand-name drug, generic drug, orphan drug. Pharmacological therapy with biosimilar drugs – provision of due diligence in the treatment process. Contemp Oncol. 2013;17(6):477.
6. Ollendorf DA, Chapman RH, Pearson SD. Evaluating and Valuing Drugs for Rare Conditions: No Easy Answers. Value Health J Int Soc Pharmacoeconomics Outcomes Res. 2018;21(5):547–52.
7. 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):24–6.
8. Ehni HJ. Expensive cancer drugs and just health care. Best Pract Res Clin Gastroenterol. 2014;28(2):327–37.
9. Annemans L, Aymé S, Cam YL, 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.
10. Blonda A, Denier Y, Huys I, et al. How to Value Orphan Drugs? A Review of European Value Assessment Frameworks. Front Pharmacol. 2021;12:631527.
11. Rubin JL, Lopez A, Booth J, et al. Limitations of standard cost-effectiveness methods for health technology assessment of treatments for rare, chronic diseases: a case study of treatment for cystic fibrosis. J Med Econ. 2022;25(1):783–91.
12. Zanichelli A, Azin GM, Cristina F, et al. Safety, effectiveness, and impact on quality of life of self-administration with plasma-derived nanofiltered C1 inhibitor (Berinert®) in patients with hereditary angioedema: the SABHA study. Orphanet J Rare Dis. 2018;13(1):51.
13. Cohen JS, Biesecker BB. Quality of life in rare genetic conditions: A systematic review of the literature. Am J Med Genet A. 2010;152A(5):1136–56.
14. Green C. On the societal value of health care: what do we know about the person trade-off technique? Health Econ. 2001;10(3):233–43.
15. Robinson S. Test-retest reliability of health state valuation techniques: the time trade off and person trade off. Health Econ. 2011;20(11):1379–91.
16. Richardson J, Iezzi A, Sinha K, et al. An instrument for measuring the social willingness to pay for health state improvement. Health Econ. 2014;23(7):792–805.
17. Lakdawalla D, MacEwan JP, Dubois R, et al. What do pharmaceuticals really cost in the long run? Am J Manag Care. 2017;23(8):488–93.
18. Cole AL, Dusetzina SB. Generic Price Competition For Specialty Drugs: Too Little, Too Late? Health Aff (Millwood). 2018;37(5):738–42.
19. Schlander M, Garattini S, Holm S, et al. Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders. J Comp Eff Res. 2014;3(4):399–422.
20. Yeung K, Bloudek L, Ding Y, et al. Value-Based Pricing of US Prescription Drugs. JAMA Health Forum. 2022;3(12):e224631.
21. Zanotto BS, Etges APB da S, Marcolino MAZ, et al. Value-Based Healthcare Initiatives in Practice: A Systematic Review. J Healthc Manag Am Coll Healthc Exec. 2021;66(5):340–65.
22. Angelis A, Polyakov R, Wouters OJ, et al. High drug prices are not justified by industry’s spending on research and development. BMJ. 2023;380:e071710.
23. Kazibwe J, Gheorghe A, Wilson D, et al. The Use of Cost-Effectiveness Thresholds for Evaluating Health Interventions in Low- and Middle-Income Countries From 2015 to 2020: A Review. Value Health J Int Soc Pharmacoeconomics Outcomes Res. 2022;25(3):385–9.
24. Santos AS, Guerra-Junior AA, Godman B, et al. Cost-effectiveness thresholds: methods for setting and examples from around the world. Expert Rev Pharmacoecon Outcomes Res. 2018;18(3):277–88.
25. Iino H, Hashiguchi M, Hori S. Estimating the range of incremental cost-effectiveness thresholds for healthcare based on willingness to pay and GDP per capita: A systematic review. PLOS ONE. 2022;17(4):e0266934.
26. Vallejo-Torres L, García-Lorenzo B, Castilla I, et al. On the Estimation of the Cost-Effectiveness Threshold: Why, What, How? Value Health. 2016;19(5):558–66.
27. Vanness DJ, Lomas J, Ahn H. A Health Opportunity Cost Threshold for Cost-Effectiveness Analysis in the United States. Ann Intern Med. 2021;174(1):25–32.
28. Hernandez-Villafuerte K, Zamora B, Feng Y, et al. Estimating health system opportunity costs: the role of non-linearities and inefficiency. Cost Eff Resour Alloc. 2022;20(1):56.
29. Charlton V. Does NICE apply the rule of rescue in its approach to highly specialised technologies? J Med Ethics. 2022;48(2):118–25.
30. Lasalvia P, Prieto-Pinto L, Moreno M, et al. International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review. Expert Rev Pharmacoecon Outcomes Res. 2019;19(4):409–20.
31. Stafinski T, Glennie J, Young A, et al. HTA decision-making for drugs for rare diseases: comparison of processes across countries. Orphanet J Rare Dis. 2022;17(1):258.
