 Some diseases are so rare and, as a consequence, treatments for them
so expensive that the burden of funding treatment has to be spread widely.
If national systems were not in place, treatments would have to be
financed direct from the primary care organisations in whose area the
affected patients
were treated. This could have two consequences.
First, the rarity of
such conditions, and the fact that there may well be a genetic component
to
their causation, might mean that cases were concentrated geographically
and so the cost of treatment could fall much more heavily on some organisations
that on others.
Secondly, individual primary care organisations would have to decide
whether or not to provide funding for treatments. If they did, they
may have to
deny funding for other services. If they did not, they could be seen
as refusing treatment to patients on the grounds that their condition
was
too rare for an affordable treatment to have been developed.
To avoid either of these outcomes, England, Scotland and Wales have
each developed mechanisms to enable the costs of expensive treatments
for
rare diseases to be divided across primary care organisations. These
are all
different, but are based on broadly similar principles (see Panel).
Differing funding arrangements in England, Scotland and Wales
In England, the National
Commissioning Group is responsible for funding services for
treating rare conditions. Before April 2007, the
NCG was
known as the National Specialist Commissioning Advisory Group.
To
qualify for NCG funding, a condition must have a national caseload
which is unlikely to be above 400, treatment must be sufficiently
expensive to create a significant financial burden when cases
arise,
and treatment for all patients must be able to provided by a
small number of centres. If a service is accepted by the NCG and
health
ministers for national funding, the money currently spent on
the service by primary care trusts is transferred to the NCG budget.
The
NCG also receives funding from the Department of Health. Its
budget in 2006 was £222m.
In Scotland, services for treating rare diseases
are selected for national funding by the National
Services Advisory Group and are
funded by the National Services Division of NHS Scotland.
Applications
for national funding must provide evidence of the clinical need
for the treatment, the incidence and prevalence of the disease
and the
effectiveness, and cost-effectiveness of the treatment. Treatments
designated as specialised services tend to be those affecting
fewer than 100 people across Scotland.
The National Services Division
also funds a number of specialised services provided in England
on behalf
of NHS boards for patients resident in Scotland.
In Wales, highly specialised services are
the responsibility of Health
Commission Wales (Specialised Services). This is an executive
agency
of the Welsh Assembly Government.
It is responsible throughout
Wales for commissioning highly specialised services. It also
gives advice
to NHS Wales on the commissioning of specialised secondary services. |
Great Ormond Street Hospital in London is one of the
centres that have successfully
applied to provide a number of specialised services funded by England’s
National
Commissioning Group (NCG), including complex tracheal disease,
epidermolysis
bullosa, persistent hyperinsulinaemic
hypoglycaemia and severe combined
immunodeficiency.
John Cheung, head of financial analysis and planning at Great Ormond
Street Hospital, explains that the process of applying for funding begins
with
the NCG requesting funding bids to be submitted for consideration and
short-listing. “The
bids to NCG would normally be completed by the lead clinician and general
manager, outlining current practice, reasons and justification for change,
likely patient pathways if the service were not implemented, numbers, service
profiled in terms of processes, resources and costs involved,” Mr
Cheung explains.
Provision of funding by the NCG should not usually change the patient
pathways, Mr Cheung adds, because there are two main scenarios in which
NCG funding
is sought. “Normally, it is either a development of a procedure
into a service — for example clinicians notice an increase in patients
being referred from other teaching hospitals for very high cost specialist
treatment — or we have patients currently being assessed or treated
for whom the current procedure or treatment is not appropriate or ideal,” he
says.
Mr Cheung sees funding from the NCG as a way of establishing standard
patient pathways for low volume, high cost treatments, for which it would
not be clinically viable to have more than a few centres running.
The development of such standard patient pathways also means that regional
discrepancies in treatment can be ironed out. For instance, in April
2007, the inherited metabolic disorders unit at Birmingham Children’s
Hospital was granted funding from the NCG to provide treatment for children
with
lysosomal storage disorders, a term which encompasses a range of rare
conditions caused by hereditary defects in lysosomes, including Gaucher
disease, Niemann
Pick disease, Pompe disease, mucopolysaccharidosis and Fabry disease.
In the past few years three other centres have also been granted approval
to provide services to treat these conditions. However, before those
approvals were granted there were great discrepancies in the approach
of individual
primary care trusts to funding the services and marked regional variation
in the availability of this treatment to children, according to Anupam
Chakrapani, consultant in metabolic disorders at Birmingham Children’s
Hospital.
Although treatments for lysosomal storage disorders began to appear in
the 1990s, their high cost proved problematic. “It used to be difficult
to secure funding and we would have endless arguments with individual primary
care trusts over these treatments,” Dr Chakrapani says.
“The
treatments for these conditions are expensive and that is a function of
the rarity of the conditions — some affect only 20 patients across
the whole of the UK. The average cost for most of our patients would be
between £30,000 and £200,000, but that is a cost for each
patient and we have 12 patients across the Midlands.”
NCG funding means not only that the cost of treatment is spread across
the country, rather than the burden being placed on a few primary care
organisations, but also that specific criteria are set for the grounds
on which treatment will be funded by the NCG.
“The system works on an individual patient basis,” Dr Chakrapani
says. “When
patients are referred to us, we will assess them and determine whether
they fulfil the NCG’s criteria. If they do then treatment will
be funded through the NCG, so the whole procedure of applying to the
primary
care trusts for funding and arguing over that is bypassed, as long as
the patient meets the criteria.” |
The
Pharmaceutical Journal