 Why should a person’s health be valued less simply because the
condition
affecting him is not rare? Cutting the issue of orphan diseases down
to so stark a question — as a paper in last week’s BMJ (2005;331:1016)
does — brings the issue into harsh focus.
In the article, Christopher McCabe and colleagues argue that, when examined
thoroughly, the idea that decisions should be based on valuing health
outcome more highly for no other reason than rarity of
the condition “seems unsustainable and
incompatible with other equity principles and theories of justice”.
Rarity is not the issue
“The argument in our article is essentially that we shouldn’t
be paying for ultra-orphan treatments simply because they are treatments
for rare diseases,” Professor McCabe, from the University of Sheffield,
told The Journal.
“We looked at the arguments for justifying rarity as a basis for special
cases being made for certain diseases. And we found that, when you separate
out the different strands of the argument, rarity is not the issue.” There
are many aspects of a disease which society may be willing to pay a premium
for, but, he argues, rarity on its own misses the point.
In fact, the premium society in England and Wales is willing to pay in
consideration of the rarity of a condition has, in part, been assessed
by the National Institute for Clinical Excellence’s citizens’ council,
which met in November 2004 to advise NICE on whether or not the NHS should
be prepared to pay premium prices for drugs to treat patients with very
rare diseases.
“The NICE citizens’ council decided not that we should pay
any price for orphan drugs but that we should pay more for treatments for
rare
conditions as long as three
criteria were met, relating to the degree of severity of the disease,
whether the therapy will provide health gain rather than just stabilisation
of the disease, and whether the disease or condition is life threatening,” Professor
McCabe explained.
However, he adds, the council was never asked whether the NHS should
pay more for treatments for common conditions that meet these same criteria.
“Another question worth asking is whether the current public sector
investment in the development of ultra-orphan treatment adequately reflects
society’s
valuation of the importance it places on treating a particular disease
and whether, having spent that money, we are still willing to pay an
additional premium to treat that disease,” Professor McCabe says. That ultra-orphan drugs are reimbursed at all illustrates that we are
willing to pay premiums for ultra-orphan treatments, Dyfrig Hughes, senior
research fellow in pharmacoeconomics at the University of Wales, and
colleagues argue in an article published in QJM last week (2005;98:
829).
For instance, the ultra-orphan drug
laronidase, the only treatment for the lysosomal storage disease mucopolysaccharidosis
type 1, is reimbursed in all but four countries of Europe, even though
it costs around £180,000 per patient a year, the prevalence of
MPS1 is 1 in 100,000 and only a small proportion of MPS1 patients will
actually be eligible for treatment with it.
Society is willing to pay for such treatments because cost-effectiveness
makes up just one aspect of considerations about
access to treatment, Dr Hughes told The Journal. “We also need
to think about whether it is fair to deny patients treatment simply because
their condition is rare, especially since it is clear that taxpayers
are willing to give patients with rare conditions more than their fair
share of health funding. In addition, although treatments for rare conditions
may not be cost-effective, the small number of patients involved means
that the overall impact on the budget is limited.”
Decisions
Decisions about the status of treatments for rare conditions will be
made in response to the review being undertaken by the Department of
Health’s taskforce investigating how the NHS in England commissions
specialised services (PJ, 22 October, p501) — including specialised
services commissioned by primary care trusts and the very specialised
services commissioned by the National Specialist Commissioning Advisory
Group (NSCAG).
The taskforce will look at how specialised service guidance is being
implemented in England and how the NSCAG commissions services. It will
attempt to identify
strengths, weakness and existing good practice. What it may well find, however,
are considerable problems with both commissioning systems.
At present, primary care trusts are
expected to work together to ensure that specialised services — those
provided in relatively few specialist centres, to catchment populations of
more than a million people — are commissioned effectively; guidance issued
in March 2003 outlines how this should be done. Very specialised services that
need to be provided in a small number of centres and planned and funded on
a national basis— essentially ultra-orphan treatments — are commissioned by the NSCAG,
but not
always paid for by the group.
The NSCAG also provides Scottish ministers with advice on whether or not specialised
services should be designated as national services. If services do receive
a national designation, every health board contributes to the cost using money
from its general allocation, so the cost comes from NHSScotland as a whole.
In Wales, a specialised health services body commissions highly specialised
services and the services are funded nationally. Difficulties
In 2003–04, the Specialised Healthcare Alliance (SHCA), a coalition
of patient groups, carried out a survey looking at the commissioning
of specialised services within the NHS. The alliance found there were
a number of issues around the extent of PCTs’ collaboration with
one another, says John Murray, chief officer of the SHCA. “In our
view, many of these problems remain,” he added.
Commissioning services through the NSCAG is also not without difficulties,
as the West Midlands Specialised Services Agency found when it came to
set up a service for lysosomal storage diseases. Amanda Burls, senior
clinical lecturer in public health and epidemiology at the University
of Birmingham, and colleagues describe the agency’s experiences
in a separate article, also in last week’s BMJ (ibid, p1019).
Needing to make a justifiable decision on funding for enzyme replacement
therapy for lysosomal storage diseases, the West Midlands Specialist
Services Agency investigated the funding of orphan drugs, with the aim
of
developing a coherent commissioning approach for these treatments. Coherent commissioning
The agency arranged reports to be produced on public perspectives of
treatments for orphan disease, as well as ethical issues and clinical
and cost effectiveness, and sought legal advice. The agency found that
the public was reluctant to engage in prioritisation, believing that
such decisions should be made by finance managers and doctors with
expert knowledge.
After lengthy deliberations, the agency concluded that rarity and being
identifiable were not in themselves overriding factors to be considered
in the decision of whether or not to fund treatment for a condition.
The commissioning group’s recommendation on lysosomal storage diseases,
endorsed by the boards of all 30 primary care trusts, was not to fund
enzyme replacement therapy for Fabry’s disease, MPS1, nor for new
patients with Gaucher’s disease.
“The drugs were considered poorly cost effective. The potential long-term
costs, possibly reaching £20m per patient, could not be justified
on the grounds of equity given that many more patients, with equal capacity
to benefit from treatment, would be deprived of treatments,” Dr
Burls and colleagues say.
However, the brouhaha that followed the agency’s decision — lobbying
by patient groups, political concern over postcode
prescribing and legal concerns over commercial expectations — led
the Department of Health to move commissioning for lysosomal diseases
over to the NSCAG.
The new commissioning arrangements did not bring any funding, however — the
costs were to be directly levied from PCTs and, in fact, the increase in costs
for this single service was greater than the entire
increase in all acute regionally commissioned services.
This situation — where a national body commissions services paid for
regionally — is, Dr Burls and colleagues argue, a fudge that is “neither
efficient nor fair and is unlikely to be sustainable in the longer term”.
“It is not appropriate that people who have no responsibility for a population
covered by a budget can require that it be spent in a particular way — this
distorts priorities,” Dr Burls told The Journal. She believes that bringing
commissioning and funding together in a national body would solve this problem.
However, other issues would remain.
Decisions and possible solutions
“Explicit frameworks need to be established to decide whether treatments
should be funded to ensure equity,” Dr Burls says.
“I suspect that if people were to be entirely consistent within such
a framework, they would decide that, within the current budget constraints,
expensive treatments for ultra orphan conditions are not the best use
of resources.”
This may not, however, be a decision the DoH taskforce, and others considering
the issue, will make since, Dr Hughes and colleagues argue, “a
complete restriction on the funding of ultra-orphan drugs is not a practical
or realistic solution”.
Dr Hughes therefore suggests a number of policies that he believes would
allow explicit criteria to be used effectively to determine the availability
of ultra-orphan
drugs. These include: · Restricting eligibility for treatment to
reduce costs
· Assigning equity weightings to quality-adjusted life year ratings,
to allow a ceiling for treatment costs to be calculated
· Risk sharing and “no cure, no pay” schemes, in which a
guarantee on performance targets is negotiated, so that predictable health
gains are achieved for a given expenditure
· Justifying research into orphan treatments in the interests of scientific
advancement, and doing so with money from funding bodies, such as the
Medical Research Council, rather than taking money from the NHS at the
expense of other therapies
“Complete restriction of funding for ultra-orphan drugs may be
justifiable from an health economics perspective, in that treatment for
ultra-orphan conditions is not a valid use of funds, but that is not
the only basis on which we judge access to treatment,” Dr Hughes
told The Journal. “A more pragmatic approach is to find ways to
make such treatments available.” |
The
Pharmaceutical Journal