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CF health economics and public health considerations COLLABORATION WITH OUR NEIGHBORS

CF health economics and public health considerations COLLABORATION WITH OUR NEIGHBORS 28 September, 2017 Session 11.30-12.30 VVSI Sport Hotel Tópart Street 17, Gárdony, Hungary Prof. László Gulácsi Department of Health Economics, Corvinus University of Budapest. Content.

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CF health economics and public health considerations COLLABORATION WITH OUR NEIGHBORS

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  1. CF health economics and public health considerations COLLABORATION WITH OUR NEIGHBORS 28 September, 2017 Session 11.30-12.30 VVSI Sport Hotel Tópart Street 17, Gárdony, Hungary Prof. László Gulácsi Department of Health Economics, Corvinus University of Budapest

  2. Content Rare and ultra rare – the issue Incentives for orphan drug development Pricing, value and reimbursement of orphan drugs CF - Quality of life and disease burden in the Central European countries Rising CF prevalence Access to treatment Considerations for the future

  3. Rare and ultra rare In the EU and US, there are 30 and 25 million people affected by more than 7,000 diseases that are considered rare. (Hall 2014) Orphan drugs target rare diseases - life-threatening or chronically debilitating illnesses with a low prevalence. Despite the small and often heterogeneous populations associated with drugs, the number of orphan drugs entering the market has risen in recent years, because of supportive EU and US policies. Small patient pools mean that R&D costs need to be recouped from smaller sales, resulting in high prices. Small, heterogeneous patient pools often indicate limited and weak clinical and economic evidence at time of product launch. Such combination of high prices and high degrees of uncertainty leads to complex decision making about reimbursement of orphan drugs. To deal with this complex decision making at time of market launch, more and more local data on disease burden, costs, QoL, cost-effectiveness are required by EU countries and conditional reimbursement mechanisms are introduced with re-evaluation after two-three-four years.

  4. Incentives for orphan drug development "Patients suffering from rare conditions should be entitled to the same quality of treatment as other patients". (EP 2000) European regulation 141/2000 introduced a set of commercial incentives in European Union (EU) to try to stimulate the development of products for rare (orphan) diseases, which until then had been largely neglected by the pharmaceutical industry. (Between 1972-1983 only 10 new drugs were approved for rare diseases) Orphan Drug Act: US (1983), EU (2000) Market exclusivity (10+2;-4 EU), Protocol assistance and follow-up, Reduced / waived regulatory fees, Tax credit on clinical trials (US), Specific subsidies for clinical trials (US) European Parliament and Council of the EuropeanUnion. Regulation (EC) No 141/2000 of the EuropeanParliament and of the Council of 16 December 1999on orphan medicinal products. Official Journal of theEuropean Communities. 2000; 22.1.2000.

  5. Pricing, value and reimbursement of orphan drugs Pricing, Price policy – the total market is very small -price law (= international reference pricing) -price tendering -patents ended -> more generics -clawback

  6. Example from The Netherlands (Koopmanschap 2016) 2012 - Press: “CvZ to delist 2 ultra-orphan drugs (Pompe/Fabry)” Myozyme for classic Pompe = 300-700.000 € per QALY. Fueled discussion Ethical to stop treatment? Ethical to value health monetarily? Ethical to deny the scarcity of resources? Better options to limit cost explosion? Why these orphan drugs so expensive? Negotiate on prices? R & D better financed publicly? 2013 - MoH reimbursed drugs lower price negotiated Decisions: -conditional reimbursement scheme introduced (5 years) -WTP/QALY for normal drugs up to 80,000 € per QALY -for ultra orphan drugs WTP = 300,000 € per QALY Message of reimbursement authorities to producers: “Don’t develop drugs with annual treatment costs of more than 225,000 €, we will not even allow conditional reimbursement”.

  7. Cost /QALY threshold Cost / QALY (Qality Adjusted Life Years) The financingthreshold is an important issue: a 3x GDP/capitathreshold was implemented officially in Poland and inHungary, and tends to be used in the Czech Republic,Romania and Bulgaria. (Gulácsi et al. 2014) The financing threshold affects all medical technologies claiming public funding. In Hungary the current version of the health economics guideline was issued in 2013. In this guideline, technologies claiming for public funding are declared as cost-effective under the threshold of 2x GDP per capita/QALY, and proclaimed not cost-effective if the ICER is higher than 3xGDP per capita/QALY. The WHO-recommended 3 timesGDP/QALY

  8. CEE EQ-5D what do we have?

  9. CEE rare diseases EQ-5D what do we have? Austria:0 Bulgaria: 5 epidermolysis bullosa (Angelis et al. 2016), Duchenne muscular dystrophy (Cavazza et al. 2016), haemophilia (Cavazza et al. 2016),cystic fibrosis (Chevreul et al. 2016), histiocytosis (Iskrov et al. 2016), Prader-Willi syndrome (Lopez-Bastida et a. 2016) Croatia: 0 Czech Republic 0 Hungary: 10 cystic fibrosis&caregivers (Péntek et al. 2014; Chevreul et al. 2016), scleroderma (Minier et al. 2010; Lopez-Bastida et al. 2016) Duchenne muscular dystrophy & caregivers (Péntek et al. 2015; Cavazza et al. 2016), haemophilia (Cavazza et al. 2016), fragile X syndrome (Chevreul et al. 2016), Prader-Willi syndrome (Lopez-Bastida et a. 2016), mucopolysaccharidosis (Péntek et al.) Poland: 5 Gaucher disease (Machaczka et al.2015), Wilson disease (Masełbas et al.2010), hidradenitis suppurativa (Matusiak et al. 2010), haemophilia (Noone et al. 2013), Fabry disease (Żuraw et al. 2011) Romania:0 Slovak Republic:0 Slovenia: 0

  10. CEE rare diseases Cost-of-Illness what do we have? Austria: 0 Bulgaria:5 mucopolysaccharidosis (Péntek et a. 2016), haemophilia (Cavazza et al. 2016), Prader-Willi syndrome (Lopez-Bastida et a. 2016), epidermolysis bullosa (Angelis et al. 2016), cystic fibrosis (Chevreul et al. 2016) Czech Republic 0 Hungary: 10 mucopolysaccharidosis (Péntek et a. 2016), haemophilia (Cavazza et al. 2016), Prader-Willi syndrome (Lopez-Bastida et a. 2016), epidermolysis bullosa (Angelis et al. 2016), cystic fibrosis (Péntek et al. 2014; Chevreul et al. 2016)Duchenne muscular dystrophy & caregivers (Péntek et al. 2015; Cavazza et al. 2016), fragile X syndrome (Chevreul et al. 2016), scleroderma (Lopez-Bastida et al. 2016) Poland:0 Romania:0 Slovak Republic:0 Slovenia:0

  11. CF in eight European countries Cost es QALY Conditional reimbursement

  12. CF QoL (Chevrul et al. 2016)

  13. Average annual costs per patients(Chevrul et al. 2016)

  14. cF cikek

  15. Data on disease burden of cystic fibrosis in Hungary (Péntek et al. 2014) Aim: To assess quality of life and resourceutilisations of patients with cystic fibrosis. Method: cross-sectional survey (BURQOL-RD project), EQ-5D-5L questionnaire, healthcare utilisations were retrospectively surveyed Results: 110 patients(age-groups, year: 0–13, N = 48; 14–17, N = 12; ≥18, N = 50). EQ-5D-5L score in age-groups 18–24 and 25–34 was significantly lower than in the general population(p<0.05). 75 patients (68%) attended pulmonology care, 55 patients (50%) were hospitalised in the past 6 and 12 months, respectively, and 57 patients (52%) were taking dornase alpha. Five adult patients (10%) received helpfrom non-professional caregiver. Conclusions: Cystic fibrosis leads to significant deterioration of quality of life. Thisstudy is the first from the Central Eastern European region that provides basic inputs for further health economicevaluations of cystic fibrosis care.

  16. QoL general population vs. CF patients (Péntek et al. 2014)

  17. Important issues Care givers perspective, societal perspective Rare diseases are becoming ‚Treatment related’ chronic diseases (similar to Chronic lymphocytic leukemia) The increasing prevalence due to improvements in treatment has led to an increase in the number of patients requiring treatment. The life expectancy of the (well) treated patients are rising Long term financial commitments Multiple co-morbidities due to the increased life expectancy Integrated care is needed. Impossible to set up professional guidelines (in a ‚classic’ way) Enormous data requirement

  18. Important issues 2 We need to know more about the: -Productivity loss: absenteeism, presenteeism, informal care -potential health and economic gain International collaboration Difficulties in international collaboration: the Bartel index and Zarit Scale example Hungary Access to therapy

  19. Access to treatment „Medicines willnot workif you do nottake them.” (by: Dr. Everet Koop) „…… more health benefits worldwide …… would result from improving adherence to existing treatments than developing any new medical technology!” (by: Hayden Barry Bosworth) adherence determining factors: • reimbursement • … and many more important factors

  20. Access (use) biologicals in RA in CEE (Péntek et al. 2014)

  21. Access (use) biologicals in Crohn’s disease in CEE(Rencz et al. 2015)

  22. Access to RTX (NHL, CLL, RA and other indications) in 28 EU countries (Gulácsi et al. 2017)

  23. Conclusion The prevalence and disease burden of rare diseases are rising We need to know more about the: -productivity loss: absenteeism, presenteeism, informal care -potential health and economic gain Medical, ethical, methodological, economic, health policy and political challenges … where we are now? projections about the next 5-10 years , modeling … International collaboration: COLLABORATION WITH OUR NEIGHBORS!

  24. Thank you! Prof. László Gulácsi, MD, MSc, PhD, PhD, PhD, PhD, habil, DSc Department of Health Economics, Corvinus University of Budapest http://hecon.uni-corvinus.hu laszlo.gulacsi@uni-corvinus.hu

  25. EQ-5D questionnaire 3-level responses No problem (1) Mild problem (2) Severe problem (3) 5 domains: • Mobility • Self-care • Usual activities • Pain / dicomfort • Anxiety / depression

  26. Time trade off Case 1 30 years old, renal failure, haemodialysis 3 times a week How many years would you offer of your 40 remaining life years to return into perfect health? Case 2 30 years old, wearing glasses How many years would you offer of your remaining 40 life years to return into perfect health?

  27. Time trade off (cont.) Case 1 (renalfailure): answerse.g. 10 years 1 – (10/40) = 0.75 Case 2 (glasses): answerse.g. 1 year 1 – (1/40)= 0.975 Renal failure Glasses 0 1

  28. Standard gamble Choose between • remaining ill • medical intervention that either restores your health or kills you (certain death risk) Case 1: 15% mortality risk of an operation to recover from renal failure to perfect health. 1 – 0.15 = 0.85 Case 2: 1% mortality risk to recover perfect vision (no glasses). 1 – 0.01 = 0.99

  29. Indirect health state (243 HSs) utility measures: EQ-5D EQ-5D descriptive (patients): 1=no problem 2=some problem 3=severe problem Utility of the health state (society) 1 1 1 1 1 1 1 1 2 1 1 0,883 1 1 1 2 1 0,796 2 2 2 2 3 0,082 3 2 2 2 2 -0,594 conversion Population, value (TTO)

  30. Health status of the population in Hungary, 2000-2010 (Baji et al. 2015) Survey in 2000, N=5503 ; Survey in 2010: N=2281

  31. Disease progression: HRQoL and costs (Péntek et al. 2007) Disease progression Disease progression

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