Europe-ExPro - Expert Procedures on Clinical, Economic, and Patient Relevance

Expertise

Expertise …. origins from > 20 years of observation

Expertise

Expertise …. comes from > 20 years of observation

Europe-ExPro has observed many disease areas and noted that innovative therapies and medicinal
products have to overcome many hurdles before they are integrated in health care budgets and
daily practice. Budget constraints complicate adoption of new innovative therapies in the health
insurance package. This demands improvement of the decision making tool box.

The health care budget is growing fast, innovation becomes more and more expensive, and the
population becomes more demanding. In this setting it is understandable from a payer’s
perspective to be critical towards new therapies. Doctors and patients on the other hand would like
to be equipped with new innovative therapies. However, therapeutic statements and guidelines
are developed most of the time after several years. So, at the time a new innovative therapy gets
marketing authorization, the payers have to base their decision on clinical trial data, which often lack
direct comparative studies with standard therapy.  Moreover, a significant difference in effect or safety
does not mean automatically that the added value meets the need for medical improvement due to
insufficient comforting data about the relevance of the new innovative therapy, there is no certainty
of market access for a new innovative therapy, let alone that it is prescribed according to its full label
obtained by the European Committee.

Relevance criteria

The key to this situation is ‘specificity and sensitivity’ of the information for decisionmaking.

The common criteria ‘added value and costeffectiveness’ are too general. They lack specific disease
related aspects do not include social and ethical criteria such as ‘orphan medicinal product’,
‘availability of therapeutic alternatives’, ‘non-inferiority in efficacy and safety but changing patients’
life dramatically’. The lack of specificity and sensitivity of the information for decision making is further
caused by the lack of integration of practice based data, i.e. the experience of doctors, other caregivers
and patients. At the moment a new innovative therapy becomes available, they are the only ones who
can translate, or better transcribe the ‘theoretical clinical data’ into what these data really mean in daily
practice. Opposite to health authority assessing committees, these doctors, caregivers and patients can
judge the meaningfulness of a difference in effects between therapies. If the difference between two
therapies is meaningful based on both evidence based and practice based data, the difference can be
judged as ‘relevant’.

In summary, by developing specific and sensitive thresholds for meaningfulness for an innovative
therapy, decisions by health authorities and heath committees can be facilitated. These thresholds are
called: clinical
relevance, patient relevance and economic relevance.

Relevance procedures

Europe-Expro facilitates expert procedures for defining the relevance of new treatment options in
comparison to the standard treatment options from a clinical, economic and patient perspective.

Funds Europe-ExPro

Europe-ExPro receives unrestricted funds and grants of companies and institutes that promote the
development of relevance procedures. Fund and grant suppliers do not have any interaction, input or
influence on the expert procedures, nor are grant and fund suppliers involved in reviewing the outcomes
of the expert procedures. Grants and funds are used to research disease areas and issues that need|
expert views on relevance, to execute the expert procedures, to coordinate the expert procedures, to write
the reports and to publish them.