Optimalisatie van de werkingsprocessen van het Bijzonder ... - KCE

180 Special Solidarity Fund KCE Reports 133
9.12.9.1 Rare indication/rare disease
The criteria on rare diseases and rare indications are clear for many respondents. The
criterion rare indication however is seen as not sufficiently specific in its formulation.
For both criteria the relevance is obvious. Several respondents find the rareness of the
indication relevant for SSF intervention as well as for the rare diseases.
Although the criterion is seen as relevant, several respondents mentioned that
reimbursement of medical costs of treatments may not be limited ‘only’ to rare diseases
or rare indications. A patient’s very specific medical condition may make the use of
drugs or treatments (that are not reimbursed by the general health insurance system)
appropriate. There is a general acceptance such situations have to be argued on the
individual patient cases and be assessed by peers.
9.12.9.2 Expensive
The criterion expensive is not clear to several respondents while it is for others.
Moreover, this criterion is not formulated sufficiently specific but it is relevant.
For almost all respondents the criterion is not totally clear since they don’t know the
amounts used by the SSF to determine the expensive character of a treatment, drugs or
medical devices. They all would prefer to have a clear definition of the criterion.
9.12.9.3 Vital functions
For many respondents, this criterion is clear and formulated sufficiently specific. Some
respondents state the criterion is too vague and has to be more specified. The
relevance of the criterion is not widely accepted.
When asked to specify the criterion, the respondents mostly saw it as linked to a life
threatening medical condition. Most of the respondents judged the patient has to have
an acceptable level of quality of life guaranteed. It means SFF has to intervene in
situations where a patient, suffering from serious medical problems can be helped to
gain functional independence. A too strict interpretation of the criterion is rejected
from a socio-medical point of view.
9.12.9.4 Medical – social perspective
This criterion is not clear at all. Some respondents reported that it is formulated
sufficiently specific. The criterion is relevant for several respondents while others could
not express an opinion on relevancy due to the unclearness of the criterion. In general
from a medical point of view, not much attention is paid to this criterion.
9.12.9.5 Scientific value and effectiveness
This criterion is seen as very clear and relevant by most of the respondents. Medical
doctors accept a treatment they prescribe to one of their patients has to meet
standards that are scientifically valid and that the treatment (or the drug or device that
is being prescribed) is accepted as appropriate and effective. The big issue here is,
effectiveness and acceptability of a treatment has to be judged from a different
perspective than for common diseases.
For rare diseases one cannot expect treatments to be scientifically proven the same
way as for widespread diseases or medical conditions. The fact a disease or an
indication is rare, in many cases makes it impossible to have the same level of scientific
proof. The term ‘largely accepted’ is a contradictio in terminis since it concerns rare
diseases and rare indications. Classic randomized studies cannot be performed since
patient groups are too small or pharmaceutical firms are not interested in financing such
studies since the global ‘market’ for the drug is judged as too small and not profitable.
In some cases it is also medically and ethically not defendable to have patients not
receiving the drug since this can lead to a serious medical setback or even worse.