Title: USING ECONOMIC EVALUATIONS IN DRUG REIMBURSEMENT DECISIONS NICE experiences from overseas
1USING ECONOMIC EVALUATIONS IN DRUG REIMBURSEMENT
DECISIONSNICE experiences from overseas
- Michael Drummond
- Centre for Health Economics
- University of York
- United Kingdom
2OUTLINE OF PRESENTATION
- Some background.
- International guidelines for economic evaluation.
- Procedures in different countries.
- Lessons from the use of economic evaluation in
drug reimbursement decisions. - Issues for countries considering introducing
economic evaluation requirements.
3SOME BACKGROUND
- Several jurisdictions have imposed a Fourth
Hurdle or requirement for economic data as part
of pricing/reimbursement decisions for drugs. - The new requirements are usually accompanied by a
set of guidelines for company submissions. - Pricing decisions may or may not be linked with
reimbursement decisions. - Australia was the first jurisdiction to implement
such a requirement. England and Wales (through
the National Institute for Clinical Excellence)
provides a recent example.
4CLASSIFICATION OF EXISTING GUIDELINES
PURPOSE
SOURCE
Reimbursement or Listing
Methodological Standards
Ethics and Conduct
Australia Belgium
Finland The Netherlands Norway
Ontario Portugal
Sweden United Kingdom
Government or Payers
CCOHTA (Canada) PHS Panel
(USA) AMCP (USA)
Academia
Langley et al (USA) Alban et al (DK)
LDI Task Force (USA) Rovira et
al (Spain) Hannover (Germany)
BESPE (Belgium)
BMJ Working Party (UK) Garattini et al
(Italy) College of Economists
(France)
LDI Task Force (USA)
Industry
PHrMA (USA)
5IN GENERAL, WHATS COVERED BY GUIDELINES?
- Viewpoint for analysis.
- Choice of comparator.
- Form(s) of economic analysis.
- Measurement and valuation of costs and benefits.
- Discounting.
- Allowing for uncertainty.
- Presentation of results.
6THE MAIN SIMILARITIES AMONG GUIDELINES
- Choice of comparator.
- Importance of good data on clinical
effectiveness. - Discounting of future costs and benefits.
- Incremental comparisons.
- Allowing for uncertainty.
7MAJOR AREAS FOR METHODOLOGICAL DEBATE
- Viewpoint for the analysis.
- Relevance of Phase III trials and the role of
modelling. - Measurement and valuation of health outcomes
(e.g. QALYs, WTP). - Handling uncertainty.
- Budget impact analysis.
8PRICING AND REIMBURSEMENT OF DRUGS IN AUSTRALIA
- Submissions for inclusion on the Pharmaceutical
Benefits Schedule are made to the Pharmaceutical
Benefits Advisory Committee (PBAC). - Submissions are required for all new drugs
(including additional indications and new
formulations) to be used outside of public
hospitals. - The PBAC issues recommendations to the Minister.
- Although a price is assumed in the submission,
pricing decisions are made by a separate
committee.
9SUBMISSION AND REVIEW PROCESS UNDER THE
AUSTRALIAN GUIDELINES(Glasziou and Mitchell,
1996)
10PRICING AND REIMBURSEMENT OF DRUGS IN ENGLAND AND
WALES
- Most drugs are reimbursed under the NHS at the
manufacturers chosen price. - Several drugs with a major impact on the NHS
are selected by NICE for detailed appraisal. - On the basis of its appraisal, NICE issues
guidance on the use of health technologies to the
NHS. - Since December 2001 the guidance has been
mandatory.
11NATIONAL INSTITUTE FOR CLINICAL EXCELLENCE (NICE)
APPRAISAL PROCESS
12NICE TECHNOLOGY APPRAISALS ISSUED BY 2001
- Cardiovascular disease
- Coronary artery stents in the treatment of IHD
(May 2000). - Implantable cardioverter defibrillators for
arrhythmias (September 2000) - Endocrine diseases
- Pioglitazone for type 2 diabetes mellitus (March
2001). - Rosiglitazone for type 2 diabetes mellitus
(August 2000). - ENT
- Hearing aid technology (July 2000).
- Gastrointestinal disease
- Laparoscopic surgery for inguinal hernia (January
2001). - Proton pump inhibitors for dyspepsia (July 2000).
13NICE TECHNOLOGY APPRAISALS ISSUED BY 2001
- Infections/infectious diseases
- Interferon alpha and ribavirin for hepatitis C
(October 2000). - Zanamivir for the treatment of influenza
(November 2000). - Malignant disease and immunosuppression
- Taxanes for breast cancer (June 2000 and
September 2001). - Fludarabine for chronic b-cell lymphocytic
leukaemia (September 2001). - Topotecan for advanced ovarian cancer (August
2001). - Docetaxel, paclitaxel, gemcitabine and
vinorelbine for non-small cell cancer (June
2001). - Gemcitabine for the treatment of pancreatic
cancer (May 2001). - Temozolomide for malignant glioma (April 2001).
- Laparoscopic surgery for colorectal cancer
(December 2000). - Liquid based cytology for cervical screening
(June 2000). - Taxanes for ovarian cancer (May 2000).
14NICE TECHNOLOGY APPRAISALS ISSUED BY 2001
- Mental health/central nervous system
- Donepezil, rivastigmine and galantamine for
Alzheimers disease (January 2001). - Methylphenidate for attention deficit
hyperactivity disorder (October 2000). - Orlistat for the treatment of obesity in adults
(March 2001). - Riluzole for motor neurone disease (January
2001). - Sibutramine for obesity in adults (October
2001). - Musculo skeletal and joint
- COX II selective inhibitors for osteoarthritis
and rheumatoid arthritis (July 2001). - Hip prostheses for primary hip replacement
(April 2000). - Autologous cartilage transplantation for full
thickness cartilage defects in knee joints
(December 2000). -
-
15NICE TECHNOLOGY APPRAISALS ISSUED BY 2001
- Oral and maxillofacial
- Wisdom teeth appropriate removal (March 2000).
-
- Respiratory systems
- Inhaler systems for under-5s (August 2000).
-
- Skin/wounds
- Debriding agents and specialist wound care
clinics for difficult to heal surgical wounds
(April 2001).
16MOST RECENT GUIDANCE FROM NICE
- New topics are not dissimilar from those studied
this far - glycoprotein 11b/111a inhibitors revision
- surgery to aid weight reduction
- pegylated liposomal doxorubicin hydrochloride for
advanced ovarian cancer - metal hip resurfacing arthoplasty
- human growth hormone in children with growth
failure - routine antenatal anti-D prophylaxis for
RhD-negative women - infliximab for Crolins disease.
- Now that NICE also issues clinical practice
guidelines there is a growing debate about the
relationship between technology appraisals and
practice guidelines.
17MAIN LESSONS FROM THE USE OF ECONOMIC EVALUATION
AT THE CENTRAL LEVEL (1)
- Demonstration of clinically-important benefits is
still paramount. - Economic data are more important when there is
substantial budgetary impact. - Devices and procedures are generally harder to
appraise than drugs.
18MAIN LESSONS FROM THE USE OF ECONOMIC EVALUATION
AT THE CENTRAL LEVEL (2)
- Difficulties arise owing to the lack of
transferability of economic data. - Political will is sometimes tested (e.g.
Beta-interferon). - In reimbursement decisions, total refusal is
rare limitations or restrictions in use are much
more common.
19NICE GUIDANCE ON THE USE OF COX II SELECTIVE
INHIBITORS (CELCOXIB, ROFECOXIB, MELOXICAM AND
ETODOLAC) FOR OSTEOARTHRITIS AND RHEUMATOID
ARTHRITIS
- Cox II selective inhibitors are not recommended
for routine use in patients with rheumatoid
arthritis (RA) or osteoarthritis (OA). They
should be used in preference to standard NSAIDs,
when clearly indicated as part of the management
of RA or OA, only in patients who may be at high
risk of developing serious gastrointestinal
adverse effects.
20MAIN LESSONS FROM THE USE OF ECONOMIC EVALUATION
AT THE CENTRAL LEVEL (3)
- Cost-effectiveness of a technology may change
over time and decisions may have to be reviewed. - Application of economic evaluation in decision
making has revealed weaknesses in Phase III drug
studies. - Litigation is increasingly being used by
manufacturers. - Decisions do reflect a cost-effectiveness logic
although other factors clearly come into play.
21INCREMENTAL COST PER ADDITIONAL LIFE-YEAR GAINED
LEAGUE TABLE
Source George et al. PharmacoEconomics 2001
19(11) 1103-1109.
22PROBLEMS WITH A SINGLE COST-EFFECTIVENESS
THRESHOLD
- Cost-effectiveness of health technologies varies
by country. - Societal willingness-to-pay for health
technologies may vary by country. - Without the overall budgetary impact, the
cost-effectiveness ratio cannot tell us the
opportunity cost of adopting the new technology. - Other factors (e.g. equity) enter into
decision-making.
23FACTORS FREQUENTLY CONSIDERED ALONGSIDE
COST-EFFECTIVENESS
- Seriousness of the health condition.
- Availability of alternative therapies.
- Number of patients, and hence budgetary impact.
- Daily cost to patients if drug not listed.
- Whether the drug is a lifestyle drug.
24HOW ARE REIMBURSEMENT RULES OR GUIDANCE
IMPLEMENTED?
- Depends on the jurisdiction and clinical setting.
- Use of hospital-based drugs can be influenced by
budgetary controls and formulary listing. - Use of drugs in primary care can be influenced by
clinical guidelines (e.g. approval on
authority), financial incentives and formulary
restrictions. - In the UK, the Department of Health is conducting
a study of the implementation of NICE guidance.
25ISSUES FOR COUNTRIES INTRODUCING THE FOURTH
HURDLE
- Do we request evidence for all new drugs, or just
some? - How do we prioritize drugs for assessment?
- Does it make sense to assess several drugs in the
same class together? - How prescriptive, or flexible, should we be in
specifying the data requirements?
26ISSUES FOR COUNTRIES INTRODUCING THE FOURTH
HURDLE (Continued)
- Should we be willing to accept data from other
countries? If so, which? - Should we be willing to accept commercial-in-confi
dence data submitted by companies? - Should the reasons for reimbursement decisions be
made public? - Should there be an appeals process? If so, what
should this consist of?
27ISSUES FOR COUNTRIES INTRODUCING THE FOURTH
HURDLE (Continued)
- Should we consider a two-stage appraisal process?
- Should we consider risk-sharing deals with
companies?
28ACCEPTING DATA FROM OTHER COUNTRIES
- An important issue for countries with limited
resources to undertake or assess economic
evaluations. - The key issue is whether economic studies are
generalizable from one setting to another.
29FACTORS LIKELY TO LIMIT GENERALIZABILITY OF
ECONOMIC STUDIES
- Demography and epidemiology of disease.
- Clinical practice patterns.
- Relative price differences.
- Incentives to health professionals or
institutions. - Community valuations of health and health care.
30THE GENERALIZABILITY OF ECONOMIC EVALUATIONS OF
DRUGS IN EUROPE
- What are the main causes of variation in study
results from place to place? - Does the extent of variation differ among
different health economic study types? (e.g.
modelling studies, trial-based studies). - Are there systematic differences in study results
between particular countries? - Is the extent of variation in study results
between countries important for decision-making? - Barbieri et al, 2003.
31QUANTITY OF STUDIES AND RANGE OF COVERAGE
- 2400 references retrieved.
- 46 intercountry comparisons
- 29 comparisons in multicountry studies
- 17 comparisons in methodologically-equivalent
single-country studies.
32MAJOR CAUSES OF VARIATION IN STUDY RESULTS FROM
PLACE TO PLACE
- Depends on type of study.
- When only unit costs are allowed to vary, drug
costs and hospitalization costs are the most
important causes. - When all factors are allowed to vary, differences
in resource use and cost are the most important
causes.
33GENERALIZABILITY OF STUDIES BY METHODOLOGY
34RELATIVE COMPARISON OF COST-EFFECTIVENESS AMONG
COUNTRIES
35IMPORTANCE OF VARIATION IN STUDY RESULTS FOR
DECISION-MAKING
- Depends on the threshold cost-effectiveness
ratio. - With a willingness-to-pay for a QALY of 50,000
we would only reach a different decision
(comparing countries) in 3 out of 28 cases.
36CONCLUSIONS
- Several jurisdictions now request
cost-effectiveness data in respect of drug
pricing or reimbursement decisions. - These decision-making processes have proved
workable, although many problems/ issues have
emerged. - Other countries introducing the Fourth Hurdle
can learn from others experiences.