|
Opening the conference, Mr JON CLOUSE (president,
ISPOR) said that the aim of the meeting was to bridge the gap between
those performing pharmacoeconomic evaluation and those using it for decision
making. The availability of more efficacious and expensive treatments
required rational decision making. Pharmacoeconomics and outcomes research
were tools to assist and support that.
European diversity and future challenges
Mr KEES de JONCHEERE (regional adviser, World Health Organisation) compared
the diverse health care systems and differing expenditure on pharmaceuticals
across Europe. Expenditure on health and drugs was rising and policy makers
across Europe faced the dilemmas of limited drug budgets, increasing consumer
demand, irrational prescribing, uncertainty about treatment outcomes,
and large variations in clinical practice as well as the underuse of effective
new treatments and continued use of ineffective treatments.
A key issue for public health in the future was the continued entry of
new products without an apparent link between drug development and health
needs. In 1998, in Germany, 35 new substances had been licensed, of which
12 were new, nine were improvements and 14 were “me-toos”. There was a
need to encourage real innovation but there was currently no definition
of “real”.
Mr De Joncheere asked: “Is pharmacoeconomic and health economic evaluation
a necessary element in rational decision making or the fourth hurdle which
delays the entry of valuable treatment options?”
Back to Top
Role of the EMEA
Mr NOEL WATHION (head of regulatory affairs and pharmacovigilance, European
Medicines Evaluation Agency) said that quality, safety and efficacy were
of the utmost importance when granting marketing approval for new pharmaceutical
products. European regulators performed risk/benefit assessments before
granting authorisations, but with limited data. Although marketing authorisations
could be granted at European level, each member state still had the power
to set prices, and to assess whether products fitted within their current
health, social and economic systems. This could lead to delays in marketing
some therapeutic advances in member states.
Identifying medicines which represented significant added therapeutic
value was important for promoting innovation. Drug expenditure needed
to be assessed to ensure the rational use of medicines, and databases
with industry-independent information for health care professionals and
the public needed to be developed, he said.
Back to Top
Comparison of advisory bodies
Mr MARC SPRENGER (director of a Dutch health insurance board) described
a national advisory body set up in the Netherlands to provide independent
advice to the Dutch ministry of health. The Dutch health minister intended
to use pharmacoeconomic evaluation in health care decision making. Of
four drugs mentioned by Mr Sprenger, the minister had followed the advice
given for all but one of them - sildenafil (Viagra) - which she had refused
to reimburse even for specific patient groups. Key issues for the advisory
body were transparency, validity, confidentiality and orphan drugs, where
there was limited funding to perform pharmacoeconomic studies. The advisory
body identified not only direct costs, both inside and outside health
care, but also indirect costs.
Professor MARTIN BUXTON (a member of the National Institute for Clinical
Excellence appraisal board) said the NICE made recommendations and issued
guidelines based on multiple criteria, not just cost-effectiveness. The
NICE did not take a broad societal perspective into account in its appraisals;
it looked only at the clinical and social services impact.
Costs and benefits were discounted at rates set by the Treasury of 6 per
cent and 1.5 per cent, respectively. Due to limited data, some amount
of computer modelling was inevitable. The NICE’s areas of investigation
covered pharmaceuticals, medical devices, diagnostic and screening devices,
surgical procedures and health promotion. In its first year most of its
work had focused on pharmaceuticals and medical devices. The problem faced
by the NICE was one of weak quality clinical data, Professor Buxton said.
Clinical trials were short-term, focused and conducted in a small selection
of adults. In reality, drug use was usually long-term chronic use in a
broader section of the population. Useful quality-of-life data to support
evaluation were lacking and models were needed to extend beyond the trial
period with increased transparency.
Back to Top
Developing clinical guidelines
Mr ULF PERSSON (of the Swedish institute for health economics) spoke
about the use of pharmacoeconomic evaluations in developing clinical guidelines.
Before 1997, the Swedish government had limited the number of products
in use. Since then it had been establishing local lists and guidelines
to ensure reliable and rational drug use locally, such as thiazides or
beta-blockers as the drugs of choice in hypertension and inhaled steroids
in asthma. Nine out of 10 general medical practitioners were reported
to be following the recommendations of local guidelines.
Various pharmacoeconomic analyses had been used in developing the guidelines.
Cost of illness and cost minimisation were used most often, cost effectiveness
sometimes and cost-utility and cost-benefit hardly ever. The costs used
were total economic costs, including cost of permanent disability, short
term illness, etc. The criteria felt to be important were therapeutics,
safety and reliability, and economic assessment. Equity between patient
groups was not an important consideration. Pharmacoeconomics could aid
prioritisation and enable doctors to maintain a certain amount of freedom
in prescribing within the guidelines.
Mr ROBERT JANKNEGT (clinical pharmacologist, the Netherlands) demonstrated
a computer program (SOJA) which promoted rational drug decision making.
Although most physicians claimed to use rational criteria when evaluating
drugs, such decisions were often based on other factors, such as emotional
factors and hidden or unconscious criteria. In order to increase transparency,
an interactive computer program had been developed. Each criterion was
given a weighting by the user and prices could be changed to reflect local
prices. Once the parameters were entered, drugs were arranged in a preference
list or graph. Sections for antibiotics, antidepressants, antiemetics,
Helicobacter pylori eradication, inhaled corticosteroids, reflux
oesophagitis and statins had been developed. Others were under development.
Back to Top
No slogans in public health
According to Mr FRANK VANDENBROUCKE (Belgian minister of social affairs),
“the last thing needed is slogans, such as ‘costs and incentives’”. Cost
containment was neither an ideal nor an evil. Incentives could be a powerful
tool for achieving efficiencies but they also had drawbacks. The fundamental
problem was how to reduce inefficiencies and wasteful behaviour. In Belgium,
discussions were taking place with the pharmaceutical industry regarding
drug budgets. If prescribing of a drug overshot the budget then the industry
had to reimburse its share of the excess overspend. The problem lay in
how to set the budget. There was a limited amount of information available.
Information holders would either charge for the information or the government
would have to set up its own costly monitoring system. This situation
could not easily be altered. Those with an advantageous position would
like to maintain it.
He also emphasised that innovation had to be intelligent innovation, not
just innovation for its own sake. Evidence-based medicine was the key
concept for improving quality in clinical practice. An important requirement
for future decision making was evidence-based pharmacoeconomics.
Back to Top
Time horizons and discounting
Mr LORNE BASSKIN (Butler university, United States) spoke about issues
surrounding the choice of time frame for cost-effectiveness evaluation.
Bias could result from choosing the wrong time frame, such as research
conducted over a short time frame from which benefits were proposed in
the long term. One example of this was with Zyban (amfebutamone) where,
after six months, fewer people were smoking but, after 12 months, many
had gone back to smoking. This caused a mismatch in costs and benefits
since costs were incurred at the present rate with possible benefits in
the future. Also, when developing drugs for chronic conditions, results
were stated for the short term only, such as with statins. If a drug was
only useful in the short term then using it long-term could result in
poor patient compliance which confounded the cost-effectiveness calculations.
Licensing for short-term use did not prevent off-label use in practice.
Use of Alphagan (brimonidine) for four weeks had been shown to decrease
intraoccular pressure. In practice, use for up to eight weeks showed an
increase in intraoccular pressure. Therefore, choosing the relevant time
frame depended on the disease state, ie, acute or chronic illness.
A frequent mistake when modelling costs was non-inclusion of medication
costs. If discounting principles were used then these needed to be stated
and the time frame disclosed. Most important was to perform a sensitivity
analysis.
Dr MAARTEN POSTMA (the Netherlands) demonstrated how discounting or not
discounting gave totally different answers when performing cost-effectiveness
evaluations. He recommended discounting at the economic rate of the country
at the time of performing the analysis and to remember that future economic
states could change.
Two paradoxes highlighted by the speakers were those defined by Keeler
and Cretin (if outcomes were not discounted, it was always more cost-effective
to put off treatment ad infinitum) and Hay (if you did not discount future
generations, each individual alive today with a positive probability of
offspring carried an infinite expected life value).
Back to Top
Using league tables
Dr FRANS RUTTEN (Erasmus university, the Netherlands) reminded participants
that cost-effectiveness could be based on incremental or generalised costs
so the decision maker needed to know what the cost-effectiveness ratio
was representing. Costs and effects were uncertain and it was not possible
to stay within a fixed budget with certainty as decision makers were not
normally risk neutral.
Dr JOSEPHINE MAUSKOPF (Research Triangle institute, US) said that league
tables could be used either to benchmark and then allocate budgets, or
to start with the smallest drug costs first and grade up until the budget
was spent. The problem with this method was that when a new drug came,
one had to start again as most drug budgets were fixed. The problem with
using the benchmark valuation method was that the value was not usually
just one value, it depended on the disease state being treated.
The impact of uncertainty was important. It was possible to attach confidence
intervals to cost-effectiveness ratios, and doing this could blur some
of the boundaries between similar drugs. Population size was also important
as the budget impact would vary with the size of the affected population,
even if different interventions had the same cost burden. If the total
cost impact was affected, then so was the total quality of life. So the
question was, did one invest in a high cost, high quality of life option
where health gains were high but fewer people were treated or a low cost,
low quality option where more of the population could be treated but the
health gains were small?
The timing of costs and benefits was important. One method was to put
the pharmacoeconomic results in perspective by changing costs with time.
Quality adjusted life years (QALYs) should also show a change over time.
Dividing costs by QALYs and benchmarking for the disease state of interest,
considering the affected population and using these figures to arrive
at the total costs and total timing allowed decision makers to look at
the efficiencies of treatment options and make choices.
Dr WARREN SCHONFELD (San Francisco, US) stated that the kinds of league
tables being discussed served two purposes, one of which was to aid researchers
investigating new technologies to assess their relative positions within
the league table.
One of the major problems of league tables was the consideration of subgroups
within a population. When considering kidney or heart transplants versus
cholesterol testing, there were wide variations in the severity of transplant
patients even though the population group was small. Were the groups comparable?
If not, on what could the decision maker base a decision? Should people
with severe conditions be treated first? Usually, within a population
group, disease severity was mixed and cost-effectiveness ratios for treatments
would vary widely.
Most published articles were negative about the use of league tables in
decision making. For football they were fine but for QALY information
problems lay in the interpretation of data. This did not prevent the use
of league tables everywhere in health care, such as the recent WHO league
table comparing European health care systems.
When speakers were asked if they were for or against the use of league
tables, Dr MAUSKOPF said: “We should think of a league table as a concept
to compare the cost or utility of a treatment against a benchmark treatment.
Therefore you do not need the whole table, just the benchmark value to
which you add the previously described methodology.”
Dr SCHONFELD suggested that league tables were not the answer but, in
a general sense, they were invaluable.
Mr MICHAEL DRUMMOND (Medtap, University of York) felt that the question
was not whether league tables should be used but rather how to best present
information so that people could make a decision. Different ways of presenting
information led to different decisions being made. He emphasised that
in some countries, such as the United Kingdom, decision making was at
a local or primary care group level so training in the use of league tables
and understanding the presentation of information was necessary.
Back to Top
|
