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In Europe, and to a lesser extent, perhaps, in the USA, those
of us involved in the development and commercialisation of medical technologies
have seen a rapid increase in the number of settings in which outcomes research
and health economic information is used. Apart form supporting the marketing
of interventions, this information is often required as part of national and local
level ‘managed entry’, reimbursement and other decision making processes around
authorisation and use of medical technologies, particularly pharmaceuticals.
In some countries (Australia, Canada, England and Wales for example) the requirements
for these data are explicit, while in other countries the objectives of economic
assessments and their use in decision making are not quite so clear, but often
it is wiser to make the information available than not to. However, it is certainly
the case that many countries are currently considering introducing similar controls
to those in countries and the importance of economic information to stakeholders
in medical technology decision-making can only increase further. The possibility
of introducing assessment of economic information at a European level is also
being explored.
Medical technologies include pharmaceuticals, medical devices and health care
interventions such as surgical procedures. However the restrictions are currently
focussed predominantly on pharmaceuticals. Much of the motivation for these restrictions
comes from the increasing cost of funding health care, and the desire of governments
and others to control the growth in health care spending. In many countries this
situation has led to the establishment of formal reimbursement procedures and
other controls on market access. Reimbursement applications typically take place
after an intervention has been approved for marketing, for example, by the USA
Food and Drugs Administration (FDA) or the European Medicines Evaluation Agency
(EMEA). Once this step, sometimes known as ‘technical approval’, has been completed,
the efficacy, safety and manufacturing quality of a pharmaceutical or device can
be regarded as proven.
Health care systems vary significantly, and each country has its own methods of
controlling market access for new technologies, but ‘reimbursement’ submission
or negotiations of one form or another are common in most European countries.
In the most general terms, the developer of a pharmaceutical or medical device
that has recently received technical approval must make a separate application
to the health care system to request reimbursement for the intervention. Negotiations
between the manufacturer and the government or their agents may cover issues such
as the patients in whom the intervention will be used, level of co-payments; the
effectiveness of the new intervention compared with existing therapies; and the
price the government is willing to pay. If an intervention does not achieve reimbursement
status, the health care system may refuse to pay for it. Patients can then only
receive the intervention if they are willing to pay for it themselves. Pricing
may also be closely linked with market access. In certain countries (e.g., Spain)
the price at which a new intervention can be sold must be negotiated with the
government; and the sponsor company must provide evidence that the new intervention
is desirable or else the health service will not grant it a realistic price for
the product. Applications for formulary listing are the most common hurdle to
market access in the USA. Companies there are free to set the price at which
they will sell their technologies, but the insurance companies that pay for much
of the health care provided are not obliged to pay for them. Many of the larger
Health Maintenance Organisations (HMOs) and similar bodies that insure against
the costs of health care maintain formularies listing the pharmaceuticals that
they will pay for. In the UK Many hospitals, Primary Care Groups (PCGs) and Primary
Care Trusts (PCTs) also maintain formularies and expect physicians to prescribe
only treatments that are included in these listings. Other methods of controlling
market access include treatment guidelines for particular disease areas and guidance
from the bodies such as the National Institute for Clinical Excellence (NICE)
in England and Wales.
Market access differs from technical approval in a number of important respects.
While efficacy and safety are vital components of the decision and clearly relate
to whether an intervention should be paid for, other telling considerations will
form part of the negotiations:
- Whether the intervention will provide real benefits to patients
in practice (i.e., effectiveness as well as efficacy);
- Its impact on overall expenditure;
- Whether the intervention is an appropriate use of limited
resources;
- Political considerations;
- The price that the sponsor company wishes to charge.
Because market access negotiations often determine who will pay
for an intervention, value for money is a natural ingredient in these discussions.
Payers are frequently not obliged to pay for new interventions, even if technical
approval has been granted. In any event, they have a strong incentive to pay
the lowest possible prices (even if this reduces the money available to pay for
future research) because budgets are fixed, usually on an annual basis. The situation
is changing rapidly, but clinical development programmes extend over several years.
Studies designed today must be sufficiently far-sighted to recognise that economic
information will be increasingly important when these studies come to be used
in a few years time. The team planning a development programme for a new intervention
should therefore include a health economist to ensure that the intervention is
appropriately supported by economic and outcomes data when it reaches the market.
The table below summarises requirements for economic information in market access
negotiations in the major markets
Table: Summary of requirements for economic information in market access negotiations.
| Market |
Economic information |
Notes |
| USA |
Required by some insurers |
Each insurer has the right to
set individual requirements. Many larger HMOs (e.g., Regence Blue Shield,
Washington) have detailed requirements |
| Japan |
Not required at present |
The Ministry of Health and Welfare
has announced that it is considering introducing a requirement for economic
evaluation |
| Germany |
Not required |
|
| France |
Required for pharmaceuticals |
A reimbursed price for new pharmaceuticals
must be agreed with the transparency commission, which is willing to consider
detailed economic arguments on the recommendation of the Economic Committee.
Companies are required to re-submit their economic justification every 5
years to show that their intervention remains cost-effective in ordinary
practice |
| Italy |
Considered as part of reimbursement
application for new pharmaceuticals |
The Ministry of Health has requested
economic justification for new pharmaceuticals although it is not yet clear
how this information will be assessed |
| UK |
Compulsory for selected technologies |
NICE conducts assessments of
the cost-effectiveness of both new and existing technologies, including
pharmaceuticals, medical devices and other interventions |
| Spain |
Not required at present |
|
| Canada |
Compulsory for some new pharmaceuticals |
Companies must make submissions
to health systems in several provinces. A central body, the Canadian Co-ordinating
Office for Health Technology Assessment (CCOHTA), also conducts economic
evaluations of established pharmaceuticals, devices and technologies |
| Australia |
Compulsory |
Very detailed guidance is available
to companies for new chemical entities (NCEs), major new indications and
major expansions of treated populations |
| Netherlands |
Being ‘phased in’ and may be
compulsory in 2003 |
The sickness funds federation
requires economic evaluation for selected new pharmaceuticals |
Other smaller markets that require or will consider economic information
as part of their market access negotiations include: Sweden, Denmark, Norway,
Portugal, New Zealand and several countries in Central and Eastern Europe. The
increasing use of economic evaluation in the context of market access suggests
that health economics is likely to become a more important part of clinical research
over time. Clinical trial programmes performed by the medical technology industries
must evolve to reflect the information needed to support market access negotiations,
and one key change will be to include economic data collection. These pressures
are pushing against a background of increasing use of surrogate marker end points,
with studies becoming more focused on ‘time to completion’ rather than ‘richness
of data’. Important considerations are the use of patient populations outside
of the markets for which the economic data is required and whether the patient
population selected for the study is relevant to the requirements of the health
care decision maker and the local’ market gatekeepers’. It is also important
to appreciate the difference between providing data on outcomes are sufficient
to demonstrate efficacy and those that demonstrate effectiveness and capture more
of the reality of the impact of an intervention in normal practice and how important
this might be for the bodies assessing drugs for reimbursement and market access. For
a company to successfully market a medical technology links have to be forged
between health economists and the teams responsible for clinical development and
commercialisation. The connection between clinical research and health economics
must become stronger.
Copyright © 2009 PDR Partners, incorporating Bob Gammon Associates. |
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