prof. dr. katharina blankart, mbr university of duisburg ......healthcare access and quality index...
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Access to medicines and value-based decision-making in Europe
Prof. Dr. Katharina Blankart, MBR
University of Duisburg-Essen, Germany
Lima, Peru, 26./27.9.2019
The 1,000 Dollar Pill
Timed access to healthcare technology
on patients
Good health assessment practices in
Europe
1
2
3
New drug for Hepatitis C
Source: https://www.dndi.org/diseases-projects/hepc/
• Sovaldi (Sofosbuvir) was launched globally in
2014 by Gilead Sciences
• Drug substantially increased cure rates for
patients with Hepatitis C
• Avoidance of liver transplantations and severe
side-effects
• Launch price in Germany at 20.000 Euro /
package
Sovaldi raised questions of affordability and accessibility to medicines around the globe
The 1,000 Dollar Pill
Timed access to healthcare technology
on patients
Good health assessment practices in
Europe
1
2
3
Access to health technology
• Physical accessiblity – „availability of good health services within reasonable reach of those who need them”
• Financial affordability – „people’s ability to pay for services without financial hardship”
• Acceptability – „people’s willingness to seek services”
Source: Evans et al. 2013 (WHO Bulletin; https://www.who.int/bulletin/volumes/91/8/13-125450/en/)
Source: Barber, R.M. et al., 2017. Healthcare Access and Quality Index based on mortality from causes amenable to personal health care in 195 countries and territories, 1990–2015: a
novel analysis from the Global Burden of Disease Study 2015. The Lancet 390, 231–266. https://doi.org/10.1016/S0140-6736(17)30818-8
Access and quality to care has increased since 1990 and reduced mortality
Healthcare Access and Quality Index based on mortality from causes amenable to personal health care
Access to medicines influences disability in European counties
Effects of 1982-2015 drug launches on mean 2015 disability of people with at least one medication
Source: Lichtenberg, F.R., 2019. The impact of access to prescription drugs on disability in eleven European countries. Disability and Health Journal 12, 375–386.
https://doi.org/10.1016/j.dhjo.2019.01.003
Disability measure Actual mean Counterfactual (no
1982-2015 drug
launches) mean
Effect of 1982-2015
drug launches
Probability of severe
limitation
16.9% 21.8% -4.9%
Probability of any
limitation
53.4% 61.1% -7.7%
Mean number of
Activities of Daily
Living limitations
0.34 0.48 -0.14%
Mean CASP index
(quality of life in later
life)
36.58 37.16 0.58
Access and affordability – same patterns but different levels by price control regimes
Cockburn, I.M., Lanjouw, J.O., Schankerman, M., 2016. Patents and the Global Diffusion of New Drugs. American Economic Review 106, 136–164. https://doi.org/10.1257/aer.20141482
Not all drugs are launched at the same time
Simmons, B., Cooke, G.S., Miraldo, M., 2019. Effect of voluntary licences for hepatitis C medicines on access to treatment: a difference-in-differences analysis. The Lancet Global Health 0. https://doi.org/10.1016/S2214-109X(19)30266-9
Improving access by voluntary licensing of manufacturers
Access to HCV treatment increases in number of people treated in low and middle income countries
Introduction of Sofosbuvir
Prices and affordability
Source: Iyengar, S., Tay-Teo, K., Vogler, S., Beyer, P., Wiktor, S., Joncheere, K. de, Hill, S., 2016. Prices, Costs, and Affordability of New Medicines for Hepatitis C in 30 Countries: An Economic Analysis. PLOS Medicine 13, e1002032. https://doi.org/10.1371/journal.pmed.1002032
Duration of time an individual would need to work to pay for 12 wk of treatment with sofosbuvir.
The 1,000 Dollar Pill
Timed access to healthcare technology
on patients
Good health assessment practices in
Europe
1
2
3
Coverage of health services by three dimensions of benefit catalogues
Reimbursement of a given population or a disease category by an insurance company, a sickness fund or, the national health system.
1. Population Coverage (Breadth)
2. Service Coverage (Depth)
3. Cost
coverage
(Height)
1. Population Coverage (Breadth)
2. Service Coverage (Depth)
3. Cost
coverage
(Height)
Source: Schreyögg et al. (2005): EJHE, 6(S1):2-10.
Aim
What determines the value of a new technology?
𝑉 = 𝑏𝑒𝑛𝑒𝑓𝑖𝑡 / 𝑝𝑟𝑖𝑐𝑒
Two approaches by decision-makers
- Formal cost-effectiveness analysis
- Added therapeutic value approach
Comparison with other drugs or standard ofcare to link price to health benefit
Focus is on relative effectiveness: howtreatments work compared with existingalternatives
Components of benefit and cost
Link between marketing authorization, post-marketing evaluation and price
The German Health System
• Social health insurance system
• 83 million inhabitants
• 90% of population covered
• 121 statutory corporations
• Mandatory for employees below income of 54.000 EUR
• Principles: solidarity, benefits in kind, financing byemployees and employers, self-administration, plurality
Germany
Source: Eurostat regional yearbook, 2019, p.13, population density
• Principles
• Health services covered if adequate, appropriate, and efficient (Social Code Book (SGB) V)
• All Rx-Medication is covered and accessible generally a negative list
• 10% co-payments for patients (5-10 EUR / prescription)
• Newly licensed pharmaceuticals
• Since 2011: Arzneimittelmarktneuordnungsgesetz (AMNOG)
• evaluation of health benefit for price negotiations, § 35a SGB V
• Includes Biologics, Orphan Drugs are exempt up to 50 Mio EUR turnover per year
• All pharmaceuticals
• Internal reference pricing in selected drug classes to set ceiling prices
• Rebate contracts between sickness funds and pharmaceutical manufacturers
The German Health System
Definition of pharmaceutical benefit catalogue and cost-containment
Source: https://www.commonwealthfund.org/blog/2019/how-drug-prices-are-negotiated-germany
How Germany negotiates prices for new drugs?
What is evaluated by the AMNOG process
• 4 dimensions of therapeutic benefit
• Mortality
• Morbidity
• Quality of Life
• Adverse events
• Comparator definition: based on formal requirements, cannot be negotiated
• Based on standards of evidence based medicine
• Technologies for which evidence in clinical use exists
Dimensions of health benefit and comparator definition
More information provided via: https://www.gkv-spitzenverband.de/english/statutory_health_insurance/amnog_evaluation_of_new_pharmaceutical/amnog_english.jsp
Health benefit rated at level of patient subgroups ≤ licensed indication
Decisions are made at level of patient subgroup (GBA) makes multiple decisions for the same substance
Substance
Patient subgroup 1
Patient subgroup 2
Patient subgroup …
Addedbenefit
Major
Considerable
Minor
Not quantifiable
No addedbenefit
No addedbenefit
Less thancomparator
Rating of health benefit at patient subgroup levelPatient subgroups for which patient relevant benefit isexpected are defined
More information: https://www.g-ba.de/english/benefitassessment/
- curing of illness
- significant increase in length of survival
- long-term freedom from serious symptoms
- far-reaching avoidance of serious side-effects
- easing of serious symptoms
- moderate increase in length of survival
- tangible alleviation of disease
- relevant avoidance of serious side effects
- meaningful avoidance of other side effects
Evidence base not sufficient
- reduction of not serious side-effect
- relevant avoidance of side-effect
224 drugs were evaluated by 2018, 57% showed an added health benefit
Source: Greiner et al., AMNOG-Report 2019
Cardiovascular diseases
Cancer
Mental conditions
And how did Sofosbuvir perform?
Manufacturer IQWIG GBA
Submits evidence,
defining 17 patient
subgroups
Claims major added
benefit in 11 patient
subgroups
Not quantifiable added
health benefit in 1
patient group
(Genotype 2, no
previous therapy)
Other evidence not
acceptable as
medication not
administered
appropriately in trial
Same rating as IQWIG
for Genotype 2 even
considerable health
benefit
Minor benefit in 8
additional patient
subgroups
Price
negotiations
High media attention
due to large expected
cost burden for
statutory health
insurance
Arbitration board was
called
Settled with 10%
rebate before
arbitration decision
Assessment of added health benefit leads to prices set according to value
Average rebate on list price was 23.4% but what matters in terms of value is the mark-up on thecomparator product
Lauenroth, V.D., Stargardt, T., 2017. Pharmaceutical Pricing in Germany: How Is Value Determined within the Scope of AMNOG? Value in Health 20, 927–935. https://doi.org/10.1016/j.jval.2017.04.006
Stern, A.D., Pietrulla, F., Herr, A., Kesselheim, A.S., Sarpatwari, A., 2019. The Impact Of Price Regulation On The Availability Of New Drugs In Germany. Health Affairs 38, 1182–1187. https://doi.org/10.1377/hlthaff.2018.05142
Opt-out as potential thread to access
Not an issue: 98% of drugs with positive benefit remained in the market
Publication of German HTA appraisal accelerates time to adoption
The value of a positive health benefit is equivalent to promotional spendings of about 400 EUR / physician
Results for 7,356 adoption decisions, 21 drugs, total
sales volume 70 Mio. EUR
Source: Blankart, K.E., Stargardt, T., The impact of information on drug quality ratings from health technology assessment on brand name drug adoption. working paper.
Sources of
value
Country Initiation of the
evaluation
process
Require clinical
evidence
Require cost-
effectiveness /
cost-utility
analysis
Exclude drug
from benefit
catalogue
Price negotiation
Therapeutic
benefit
Germany (G-
BA)
Manufacturer or
agency
Yes No No Yes
France (HAS) Manufacturer Yes (Yes) No Yes
Cost-
effectiveness
England (NICE) Government Yes Yes Yes Yes
Scotland (SMC) Manufacturer or
patients
Yes Yes Yes No
Australia
(PBAC)
Manufacturer Yes Yes Yes Yes
Benefit assessments in Europe and elsewhere
Source: Barnieh L et al. Value in Health. 2014 Jan;17(1):98–108.
Sources of value:
Health gains measured as QALY
(quality adjusted life year), cost
against comparator + additional
considerations, e.g. equity, end of
life, orphan
Not only criteria, but also processes differ
Determining value is a challenging exercise
Comparison of German decisions with the England (NICE) or Scotland (SMC) reveals large differences
Source: Heisser T, Fischer K, Stargardt T (2015): Health Benefit Assessment Of Pharmaceuticals: An International Comparison Of Decisions from Germany, England, Scotland
and Australia.
a. NICE Decisions by G-BA Decisions b. NICE Clinical Effectiveness by G-BA Decisions
G-BA Added
Benefit
No Added
Benefit
G-BA Added
Benefit
No
Added
BenefitNICE Sum NICE Sum
Recommended 14 18 32 Better 19 21 40
Restricted 2 7 9 Similar/not proven 5 10 15
Not recommended 8 6 14 Sum 24 31 55
Sum 24 31 55
Cohen's Kappa: -0.13 Cohen's Kappa: 0.11
Market access of medical devices in Germany
Default scenario to obtain reimbursement depends on inpatient and outpatient setting
Inpatient Outpatient
New procedures permitted
with the reservation of
prohibition(Verbotsvorbehalt, § 137c SGB V)
Medical devices receive CE mark; unless prohibited by G-BA,
hospitals may use them
Hospitals may use all CE
marked innovations
New procedures are
prohibited until they have
been approved(Erlaubnisvorbehalt, § 135 (1) SGB V )
G-BA needs to approve
medical device before use
Long application process to
gain approval
Source: https://www.slideserve.com/mab/webinar-reimbursement-of-innovative-medical-devices-in-germany
Reimbursement pathways for medical devices in Germany
CE Mark
Inpatient Outpatient
Already
reimbursed
AdequateOPS / DRG
available
New OPS / DRG required
‚On top payment‘ required
OPS/DRG
applicationNUB (ZE)
Additional evidencerequired
Applicationexperimental
coverage
Inclusion in fee schedule
EBM / GOÄ
applicationIGeL
Out of pocketpayments
Selective
contracts
Individual
Sickness
funds
IGeL: individuelle Gesundheitsleistungen (individual health services); OPS: Operationen- und Prozedurenschlüssel (International Classification of Procedures in Medicine (German version); DRG: diagnosis related group; NUB (ZE): Neue Untersuchungs und Behandlungsmethoden; ZE: Zusatzentgelt (additional fee))
Payment per hospital case
and complexity
e.g. BurriedBumper (2017) new OPS code
High cost innovative technologies not
covered by DRG, e.g. complex heart surgery
Liposuktion
for lipedema
e.g. videodoctor
consultation
Mostly low to novalue procedures
and tests, e.g. MRT in early dementia
Digital healthapplications, e.g.
TK - MolecularHealth Guide
Source: https://www.slideserve.com/mab/webinar-reimbursement-of-innovative-medical-devices-in-germany
Assessment of new procedures in Germany
G-BA Methodenbewertung
Challenges and opportunities
• Challenges
• Focus is on new technologies
• Assessment of value seem depend on health system setting EU striving towards a uniform HTA
• Weak integration of ambulatory and hospital care
• Different pathways, different types of value assessments
• Slow uptake of integrated care models and process innovations Innovation funds as tool to overcome this
• Comparison across technologies still limited (drugs vs. procedures vs. medical devices)
• Opportunities
• HTA contributes to transparent and accountable decision-making
• European evidence assessments publicly available
• HTA provides considerably more information than clinical tirals (Wieseler et al. 2015, BMJ)
• All German value appraisals and guidelines freely available at: https://www.g-ba.de/
• International services of UK National Institute for Health and Care Excellence: https://www.nice.org.uk/about/what-we-do/international-services
What can we learn from the European experience?
Jun.-Prof. Dr. Katharina Blankart, MBR
Universität Duisburg-Essen
Fakultät für Wirtschaftswissenschaften
Juniorprofessur für empirische
Gesundheitsökonomik
Tel Büro: +49 201-183-4403
Tel Mobil: +41-76-761-8818
Questions? Comments?