Presentation on theme: "EHC Workshop on Economics and HTA’s for EU Member Organisations"— Presentation transcript:
1 EHC Workshop on Economics and HTA’s for EU Member Organisations Basic economic concepts Health economics, cost-effectiveness and QALYsEHC Workshop on Economics and HTA’s for EU Member OrganisationsSeptember 20th, 2014Keith TolleyDirectorTolley Health Economics Ltd
2 The purpose of the presentation Provide an understanding of key concepts and language of health economics and cost-effectivenessFocus on Quality Adjusted Life Years (QALYs)Set up for session on payer perspective in the afternoon.
3 Climate change in haemophilia Previously littleFunding restrictionBut growth in use and cost of factor VIII (prophylaxis)New expensive recombinant productsPayers concern to control costs
4 Growth in Health Technology Assessment around the globe Organisations assessing clinical and cost- effectiveness of new medicinesThey use the tools of health economicsTo aid decisions about drug and health technology reimbursement and funding
5 HTA bodies in Europe Univ of Tartu VSMTA VASPVT MoH MoH AAZ FinOHTANOKCTVL,SBUSMCUniv of TartuVSMTADACEHTANCPEVASPVTAWMSGNICECVZ/NVTAGAHTAPolDAHTA/IQWiGKCEMoHHASLBI of HTASNHTA, MTU- SFOPHHunHTAMoHAAZAET + AETSA,AVALIA, CAHTA,OSTEBA, UETSAIFA + regional HTAMoHANHTALots of sun, but not so much HTA
6 Developing HTA in Europe Well established in countries such as Sweden, UK, Netherlands, Germany, BelgiumUsing cost-effectiveness criteria in all countries for pharmaceutical reimbursement decisions (including Italy and France recently)With some exceptions (Germany) the main outcome measure being used in assessments of cost-effectiveness is the QALY – “Quality Adjusted Life Years”HTA and the use of cost-effectiveness criteria has emerged in CEE countries in last 5-10 years (e.g. Poland, Estonia, Croatia, Romania, Bulgaria, Czech Republic)
7 Health Economics principles Scarcity of resources: resource allocation decisions in a populationValue of resources used (costs/savings) and benefits of treatments (e.g. survival and quality of life)Efficiency: choosing treatments with the greatest net value for the populationEquity: choosing treatments due to fairness criteria as well as efficiency
8 Health economic evaluation concepts Health economic evaluation is the tool of health economics concerned with assessing value, efficiency and equity in use of resources on health care interventions and technologies.Key principles/terms you will learn about today:Different types of health economic evaluationPerspective and types of costQALYs and the concept of utilityIncremental cost-effectiveness ratio (ICER)Utility (for QALY) measurement – time trade-off, standard gamble, EQ 5D
9 Comparing the Main Methods of Economic Evaluation Type of AnalysisResultMeasurement ofConsequencesCostsCost BenefitCost MinimisationCost UtilityMoneySame outcomes for all interventionsQuality Adjusted Life Years (QALYs)Cost per life year.Cost per QALYCostBenefits valued in money.Net £cost: benefit ratio.Single unit of outcome e.g. life years, blood pressureCost Effectiveness
10 Identification of resources & costs InterventionDirect costIndirect costWider cost implications to society e.g. lost production/wagesNon-health services resource use.eg. patient transportation, informal caregivingHealth services resource use.e.g. inpatient, outpatient, tests, drugs, overhead costs93
11 Perspective The perspective determines the types of costs included A health care perspective would only include direct medical costsCurrently preferred by UK HTA bodies, but likely to change (to some extent) to a broader perspective)A society perspective would also cover non-direct health-related and indirect costsCurrently preferred basis in Sweden
12 Cost-effectiveness and cost utility analysis (CEA and CUA) The tool for assessing value, efficiency (vs equity)Health economic principle is to maximise the population health benefit with the resources availableExpressed as cost per unit of benefit for a new treatment B over current treatment A e.g:Incremental cost per bleed avoided by primary prophylaxis over on-demand treatment (‘cost-effectiveness’ analysis)Incremental cost per QALY gained (‘cost-utility’ analysis)QALY = life years multiplied by quality of life (utility) score on 0-1 scale (the HTA favourite and sometimes enemy !)
13 Incremental CE ratio (ICER)* [Cost (B) - Cost (A)] i.e Difference in Cost[Effect (B) - Effect (A)] Difference in EffectIt reveals the additional cost per unit of benefit of switching from one treatment option (current practice) to another or new treatment option, i.e. from A to B*Sometimes known as the incremental cost-utility ratio (ICUR)
14 Cost-effective footballers? :€330k (£275k) weekly salaryAnnual salary of €17,160,00060 goals in 50 games€286k per goal:€288k (£240k) weekly salaryAnnual salary of €14,976,00055 goals in 55 games€272k per goalIncremental annual cost per extra goal by Messi = €437kGoal per game adjusted: = €218k
16 Calculating pre and post treatment cost per QALY figures Without treatment (A)Estimated survival= 20 yearsEstimated utility = 0.5QALY = (20 x 0.5) = 10Cost = €4,000With treatment (B)Estimated survival= 20 yearsEstimated utility = 0.6QALYs = (20 x 0.6) = 12Cost = €36,000ICER = €36,000- €4,000/12-10 QALYs= €32,000/2 QALYs= €16,000/QALY gained
17 QALYs Whereas clinical outcomes only allow comparisons within diseases e.g. Cost per bleed avoided for different haemophilia treatmentsQALYs allow comparisons across disease arease.g. cost per QALY for haemophilia treatments vs cost per QALY for cancer treatments v cost per QALY for migraine treatments
19 UK Benchmark for decisions: incremental cost per QALY gained A = <£20,000 perQALY gained:Considered anefficient use ofresourcesB = >£30,000 perQALY gainedWould need specialcircumstances toacceptProbability of rejection on grounds of cost ineffectivenessIncreasing cost/QALY (log scale)Source: Rawlins and Culyer, BMJ 2005;329:
20 How does disease affect patients’ health related quality of life? General Health PerceptionsPsychological Distress/Well beingDiseasePhysical FunctioningSocial/Role FunctioningEssentially, when we talk about HRQoL we are appreciating that there is more to disease than clinical signs and symptoms. If you ask a patient about asthma, they do not talk about inflammation in the lungs and bronchoconstriction, they talk about not being able to run for a bus, being bothered by pollen which means they stay indoors to avoidPersonal FunctioningAdapted fromWare, 1984
21 QALYs...The ‘Quality adjusted Life Year’ (QALY)Survival (e.g. 10 years)Health related QoL (Utility 0-1)QALY = survival weighted by utility (10 life years x 0.5 = 5 QALYs)To derive a utility need a health state and a valuation of that health state
22 UtilityThe utility weight (typically on a 0-1 scale ) reflects the preference (or value) people have for different health statesHRQoL or Utility = preference or valueThe more preferable a health state the more utility associated with it (i.e. health state with a value = is preferred to a health state with a value = 0.125).
23 Example of health states on the 0-1 utility scale PERFECT HEALTH0.9Normal health0.8Headache once a week0.7Migraine once a week0.63 migraines per week0.5Daily migraines0.4Regular severe migraines0.3Regular severe migraines and other pain0.2Regular pain (including severe migraines)0.1Constant migraines and painDEATHUsing migraine/pain as an exampleUtility scales are typically bounded by 0 (death) and 1 (perfect health)
24 How is a ‘utility measured’? Time Trade-offStandard gambleVisual analogue scaleGeneric questionnaire – e.g. EQ 5D, SF 6D
25 Time trade-off (TTO) - valuation Ask respondent:How much time in perfect health would you give up to avoid a longer time with the health state - haemophilia patient with 5 bleeds per 3 monthsFor example, accepting 6 years in perfect health followed by death instead of 10 years with the health state gives the utility of 0.6 (6 divided by 10)
26 Direct measurement using Time Trade-off Applying TTOStep 1:Full health state description typically requiredStep 2: Completing the TTO exerciseDirect measurement using Time Trade-offHow many years in perfect health followed by death would you accept?Years YearsVersus10 years in Health State AResponse:= 6 years
27 Standard Gamble (SG) - valuation Ask respondent would you:Accept a gamble which gives perfect health (e.g. 10 yrs) or deathOrChoose a health state for 10 yrs – general health state with mobility etc problems, or haemophilia with 5 bleeds per 3 monthsIf accept gamble at odds of 60% chance of perfect health, the utility of the health state is 0.6
28 Visual analogue scale (VAS)-valuation Ask respondent to:Place a cross on a scale marked (0 being death and 100 perfect health) for the state - haemophilia patient with 5 bleeds per 3 monthsIf the cross is placed at 60, (then rescaled 0-1) the utility is 0.6.
29 Applying the VAS Step 2: Completing the VAS Step 1: Full health state description typically requiredStep 2: Completing the VASVisual Analogue Scale100 Perfect Health755025DeathResponse = 30 on VAS – equates to a utility of 0.3Health State A
30 Example health state in haemophilia Few published examples - Naraine et al (Canada)Health Related quality of life for severe haemophilia: used standard gamble technique.Conducted in 30 healthy adults, 30 parents of children with haemophilia, and 28 adults with haemophilia.Seven scenarios/health states for on-demand or prophylaxis, low, medium or high dose, level of bleeding frequency and infection risk were valued.
31 An illustration of a health state description Scenario 1: Baseline on demand-therapy: low to moderate joint bleeding frequency (<3 in 3 months)Your son was diagnosed with severe haemophilia shortly after birth.He bleeds frequently and easily into muscles and joints, especially after a bang or fall and requires one or two infusions of FVIII.Infusion means a needle is inserted into his vein. The needle is connected to a syringe containing the FVIII. You have to wait until all the FVIII is in the vein (15–20 min). He may need to go to the hospital for an infusion, if you’re not on homecare treatment.With minor bleeding events, he has moderate pain that prevents a few activities.Generally, he is able to walk around without difficulty or walking equipment.He avoids activities and bodily contact sports that may result in a bleed.A few times a year, he has a serious bleed into a joint and he experiences severe pain that prevents most activities for a few days.He misses a week or more of schoolAfter the joint has healed, he is able to walk around without difficulty
32 Utility valuation survey - Respondents Can be patients, patient carers, members of publicIf public complete the exercise, said to represent community based or society values for the health states
33 Generic utility questionnaires Broad description of quality of lifeUse direct measurement techniques (TTO/SG) to value general health statesQuestionnaire then applied to find which health state a patient is inMost popular questionnaires are:EQ-5D (EuroQoL)SF-36 (SF6D)Health Utilities Index (HUI)
34 EQ 5D utility questionnaire 100 = best assessmentMobilitySelf-careUsual activities100 mm Visual analogue scalePain/ discomfortAnxiety/ depression0 = worst assessment
35 Applying the EQ 5DStep 1:Patient complete the EQ 5D questionnaire to reflect their health stateEQ-5D QuestionnaireMobilityI have no problems in walking aboutI have some problems in walking aboutI am confined to bedSelf-CareI have no problems with self-careI have some problems washing or dressing myself 2I am unable to wash or dress myself 3Usual Activities (e.g. work, study, housework,family or leisure activities)I have no problems with performing my usual activities 1I have some problems with performing my usual activities 2I am unable to perform my usual activities 3Pain/DiscomfortI have no pain or discomfort 1I have moderate pain or discomfort 2I have extreme pain or discomfort 3Anxiety/ DepressionI am not anxious or depressed 1I am moderately anxious or depressed 2I am extremely anxious or depressed 3Step 2: Apply valuation system (tariff):Valued by members of the general public (in Europe, but available for other countries) using time trade-off (TTO) techniquesn=3,235 respondents (cross section of the public).
36 EQ 5D utility score and QALY calculation Response to questionnaire = UtilityTTO tariff = 0.49= 0.42= 0.25Step 3QALY:Calculation Life years = x 0.42Utility = 0.42QALYs = 4.2Step 4
37 How are utilities for generic instrument health states derived? Valuation systemTime trade-off (TTO)Standard gamble (SG)Rating scale/visual analogue scale (VAS)QuestionnaireEQ 5DSF 6DHealth Utilities IndexEQ 5D VAS
38 EQ 5D utilities EQ-5D - utility values 0,0 0.8 0.6 0.4 0.2 1) Kobelt G et al. HEPAC 2001, 2: 60-682) Meads et al. Value in Health 2004; Poster Presenation at ISPOR 20043) Groen et al. Am J Transplantation 2004; 4:4) Lloyd A et al. Primary Care Respiratory J 2007,5) Miners, Haemophilia, 20090.20.60.80.40,00.50 – patients before lung transplantation (3)EQ-5D - utility values1.00 – best health status0.87 – severe haemophilia receiving prophylaxis (5)0.76 – patients with mild multiple sclerosis (EDSS ≤ 3.0) (1)0.66 – severe haemophilia receiving on-demand treatment (5)0.58 – patients with pulmonary arterial hypertension NYHA III (2)0.43 – patients with pulmonary arterial hypertension NYHA IV (2)0.33 – patients hospitalized for exacerbation with asthma (4))0.23 – patients with severe multiple sclerosis (EDSS ≥ 6.5) (1)death
39 Comparing the generic instruments EQ 5DSF 6DHUI5 health domains (mobility, self care, usual activities, pain/discomfort, anxiety depression)Utilities based on TTO methodsValued by general population (in several countries)Only adult version currently validatedHas a greater range of scores than SF 6DMay lack sensitivity due to fewer domains especially in less severe health problemsQuick questionnaire to completeCeiling effects found (large percentage of respondents report no problem)6 health domains (physical functioning, role limitation, social functioning, pain, mental health, vitality)Derived from the validated generic quality of life questionnaireSF36Based on SG methodsValued by general population (in UK)Only adult version currently validatedFloor effects found (large proportion of respondents report low scores)7-8 health domains (HUI3) (speech, vision, ambulation, dexterity, emotion, cognition, pain)Based on SG and VAS methodsValued by general population (in Canada)Includes a version available for use in children (HUI2)Has stronger emphasis on sensory elements than other instruments
40 Some observationsHTA general preference is for societal valuation (general public) and/or using a generic questionnaireThe different techniques can easily produce different utility scoresWhat is important is difference between health state scores as that measures the QoL benefits of treatment:If on-demand treatment utility is 0.6If prophylaxis utility is 0.8Means there is a 0.2 gain in utility score/quality of life
41 Whose QALY are we interested in? The Patient certainlyThe family caregivers?
42 QALY is a QALY is a QALY“An assumption that underlies most of NICE's technology appraisals has been that “a QALY is a QALY is a QALY.” By this NICE means that a QALY gained or lost in respect of one disease is equivalent to a QALY gained or lost in respect of another. It also means that the weight given to the gain of a QALY is the same, regardless of how many QALYs have already been enjoyed, how many are in prospect, the age or sex of the beneficiaries, their deservedness, and the extent to which the recipients are deprived in other respects than health.”Rawlins & Culyer. BMJ 2004;329:224
43 QALY is a QALY is a QALY not not ^ ^ “I am uneasy about the mantra of ‘a QALY is a QALY is a QALY.’ It means that an increase in utility from 0.3 to 0.5 is valued the same as an increase from 0.7 to 0.9. I am not sure this is fair.”Rawlins. Value in Health 2012;15:568-9
44 Challenges for improving health economic evaluation in haemophilia Improving but still limited clinical and outcomes data availableLack of standard approach to utility measurementImportance of adopting a societal perspectiveEnsuring equity considerations incorporatedReminding HTA bodies that haemophilia is an Orphan (rare) disease
45 ConclusionsHTA and health economics is increasingly being used to assess value by national and regional health authorities and payersPatient organisations and health professionals working in haemophilia can contribute to the debate on what constitutes value in haemophilia by understanding the key concepts and tools of HTA and health economicsQALYs are here to stay for time being, so need to understand and work with them.Although other outcomes options are being considered even in UK
46 Any questions?Does Santa Exist?How many shopping days to Christmas?
47 Further ReadingTolley K - What are Health Utilities? What is...? Series April 2009 (revised in 2014 and available soon)O’Mahoney B - An Introduction to Key Concepts in Health Economics for Hemophilia Organizations, World Federation of Hemophilia, 2010