Written evidence from Shire Pharmaceuticals Ltd
(COM 33)
SUMMARY POINTS
— Currently commissioning for rare conditions
takes place at national level, and is overseen by the National
Specialised Commissioning Group (NSCG). This arrangement is widely
regarded as effective, efficient and appears to be viewed as a
model of good practice for the commissioning for rare conditions
by other EU nations.
— The extremely low prevalence and frequently
uneven distribution of very rare conditions means that GP Consortia
are unlikely to be able to make effective decisions regarding
services and treatments for these conditions and we believe that
commissioning of such services and treatments should take place
at a national level, as currently.
— The uneven distribution of very rare
conditions places significant and disproportionate financial risk
on any given GP Consortium. This risk is such that commissioning
by clusters of consortia or at regional level would not significantly
diminish it. Top-sliced, national funding is required to support
national commissioning of services and treatments for very rare
conditions.
— There remain significant issues relating
to the proposed arrangements for national commissioning and these
proposals require urgent, detailed clarification and public scrutiny
in terms of:
— Responsibility and accountability.
— Prioritization versus other services
and treatments.
— Assessment of services and treatments.
— National safeguards and complaints procedures.
1. CLINICAL ENGAGEMENT
AND COMMISSIONING
1.1 The National Institute for Health and
Clinical Excellence (NICE) has proposed the definition of an ultra-orphan
condition as one with a prevalence of less than 1 in 50,000 (this
matches the criterion used by the National Specialist Commissioning
Advisory Group of a condition that affects less than 1,000 across
the UK).
1.2 If one assumes that a GP consortium
would, on average, cover around 250,000 people, the incidence
of very rare conditions means that it is virtually impossible
for any consortium to access meaningful information on the required
services.
1.3 Even where several consortia come together,
they are likely to be dealing with around 20 patients or less.
1.4 There are a number of risks in this
situation, including:
— The extremely low patient numbers mean
that they may be considered too small to register on the Government's
proposed system of indicators and thus lose out to more prevalent
conditions.
— It will be difficult for any GP Consortium
to acquire the knowledge and experience needed effectively to
commission services, which already resides within the national
commissioning body (NSCG).
— There is a significant risk that cases
will come to the attention of GP Consortia as cases for exceptional
funding, which will be inefficient and time-consuming.
1.5 The underlying cause of most conditions
that sit within the ultra-orphan definition is genetic. This means
that in addition to their low prevalence, they are unlikely to
be distributed uniformly across the country. Small clusters are
likely to form due to familial association.
1.6 This clustering has a number of potential
consequences. For example, this means that even fewer GP Consortia
would be likely to have experience of the particular condition.
This would represent variations in clinical practice, making it
even harder for consistent decisions to be made regarding services.
In addition, treatment costs would be unevenly distributed between
GP Consortia. This would represent a significant financial risk
for any consortium.
1.7 In our opinion, it is highly unlikely
that GP Consortia would be able to make high quality decisions
regarding services for people with very rare conditions. Experience
has indicated that ultra-orphan conditions are so rare that they
require a different approach even when commissioned at national
level. We therefore believe that commissioning via groups of consortia
or by regional commissioning would be ineffective and that the
only viable option would be to deal with them as national specialized
services.
1.8 Since the majority of rare diseases
are genetic disorders that affect children, there is the additional
risk that any moves to commission services and treatment at local
level could disadvantage children—a group that already tends
to be marginalized in health care.
2. ACCOUNTABILITY
FOR COMMISSIONING
DECISIONS
2.1 We believe that there is a great deal
of essential detail that needs to be communicated in relation
to proposed future arrangements for commissioning specialized
services at national level and we would urge the Government to
make this known as a matter of urgency.
2.2 The White Paper—Liberating
the NHS, states that the NHS Commissioning Board will be responsible
for commissioning national specialised services and we are aware
that a new organization, the Advisory Group on National Specialised
Services (AGNSS) is being formed. Although the group has already
met, the future interaction between it and the NHS Commissioning
Board remains to be seen.
2.3 Whilst we agree that local accountability
for commissioning decision is important, we believe that it is
equally important to apply the same level of accountability for
nationally commissioned services.
2.4 Patient organizations have played a
vital role in informing decisions made by the National Specialised
Commissioning Group (NSCG) and we believe that it is crucial that
this close relationship should continue with the AGNSS because
this has been the only way to ensure that the voices of patients
with very rare conditions can be heard.
2.5 The NSCG had undertaken a number of
strands of work. This included development of proposals for a
system to assess new treatments for ultra-orphan diseases. We
are given to understand that the intention is to take forward
this work, however details on the precise nature of the assessment
that will be undertaken are not available. We are in favour of
evidence-based processes of assessing ultra-orphan treatments
where they can be shown to make a constructive contribution to
commissioning decisions. We believe that the significant challenge
in doing this for rare conditions needs to be recognized so that
appropriate, dedicated assessment processes are developed rather
than existing ones being adapted for the needs of treatments for
rare conditions. We therefore believe that it is important for
AGNSS to clarify its intentions on the development of an assessment
process and, if it is to continue, to make known the detailed
process so that it can be carefully scrutinized.
2.6 We believe that it will be important
to have adequate processes at national level to deal with disputes
over decisions made by the NHS Commissioning Board in relation
to national specialised services. It is far from clear if, or
how, individual patients would be take up complaints against national
specialized services or commissioning decisions.
2.7 In our view, disputes over decisions
made at national level in the commissioning of specialized services
should be dealt with by Health Watch at national level. Involving
local Health Watch would be ineffective as their level of specialist
knowledge would be insufficient. However, for a patient to engage
with such a national body as Health Watch could be a daunting
experience and we would like to see Health Watch develop a simple,
user-friendly process for dealing with complaints regarding national
specialized services and commissioning.
2.8 Similarly, we would like to see clearly
established accountability for national specialized commissioning
decisions. Ultimately we would see this lying with the Secretary
of State as part of his role in holding to account the NHS Commissioning
Board.
INTEGRATION OF
HEALTH AND
SOCIAL CARE
2.9 As a consequence of the way in which
the NHS is being structured, with greater emphasis on local decision
making, it is our belief that the highly specialized nature and
infrequent occurrence of very rare conditions places people suffering
from them at a disadvantage and we therefore believe that they
should be considered as a vulnerable group.
2.10 The system of indicators by which,
when they are developed, the performance of the NHS will be measured,
is likely to be more heavily influenced by conditions that are
more prevalent. For example indicators that relate to heart disease
or diabetes will have greater influence on overarching indicators
such as mortality amenable to healthcare. Conversely, performance
for small groups of people with very rare conditions are not likely
to have any visible impact.
2.11 At local level, where the focus will
be on delivering quality standards and contributing to improvement
indicators, whilst at the same time demonstrating a good service
for the local population, it is likely that individual rare conditions
would not be seen as a priority. This would be unfair to people
with rare conditions who could be discriminated against on the
basis of the low incidence of their condition.
2.12 We believe that the solution to these
potential issues is to remove commissioning decisions on rare
diseases from the local environment and to commission them as
specialised services and treatments at national level.
RESOURCE ALLOCATION
2.13 As mentioned under 1.6, the distribution
of very rare conditions can typically result in a small number
of small clusters of patients. This would make resource planning
difficult for GP Consortia.
2.14 The duplication of effort that would
result from local commissioning of services and treatments for
rare conditions would be not only ineffective, but also highly
inefficient.
2.15 The costs of developing treatments
for very rare conditions are at least as high as those for developing
treatments for more prevalent conditions. Indeed, the difficulty
and cost of conducting adequate clinical trials can often increase
costs beyond average.
2.16 Given the small number of patients
for whom the treatment would be effective, the cost per patient
of treatments for rare conditions is generally much higher than
for other drugs. Moreover, these are generally highly specific
treatments and therefore not effective in other indications.
2.17 Balanced against the high cost per
patient is the small number of patients with whom the treatment
would be used. This means that the total cost to the NHS at national
level is relatively small. In contrast, the financial risk to
a GP Consortium is considerable; if a cluster of just a few patients
occurs within a consortium, it can have a major impact on local
budgets.
2.18 Risk sharing by pooling budgets would
go some way to overcoming the difficulties described above. However,
it would require significant resource to administer. Added to
this it would be a time consuming and challenging process to gain
consensus from all GP Consortia on a specific rare conditions—particularly
because, as we have already said, the level of specialist knowledge
does not exist locally. It is worth noting that the consortium
approach to commissioning has been tried in the past but its ineffectiveness
led to the development of the current national commissioning arrangements
for rare conditions.
2.19 We believe that the most efficient
and effective way of allocating resources is to create a separate,
top-sliced, national budget and to fund nationally commissioned
specialized services and treatments from this.
3. SPECIALIST
SERVICES
3.1 The needs of people with rare conditions
can often be very different from to those of other group of patients
and require a different management approach. For example, the
genetic aetiology of many rare conditions mean that not only the
presenting patient but also the whole family (particularly the
children) needs to be seen.
3.2 This means that "standard"
services are seldom appropriate for rare conditions, requiring
highly specialized knowledge, very specific patient support and
access to specifically developed treatments, plus the allocation
of resources to provide all of this.
3.3 We would like to see the commissioning
of treatments for rare conditions treated as nationally commissioned
specialized services so that this can be guided by appropriate
expert knowledge and decisions can be made nationally in order
to gain maximum benefit form a strategic approach to these illnesses.
3.4 We believe that such an approach would
most likely be in line with other responses being formulated by
European Member States in response to the European Directive:
European Action in the field of rare disease (COM(2008)0726—C6-0455/2008—2008/0218(CNS)).
3.5 We would like to see the challenges
in developing treatments for rare conditions recognized in the
way that access is provided to such drugs. In particular, we would
like to see adequate allowance made for the relatively low volumes
of clinical data that it is possible to generate, the relatively
high cost per patient balanced against the low impact on overall
NHS budgets and the highly specific nature of these treatments
rendering them effective in a very narrow clinical indication.
3.6 The unique characteristics of specialized
services for rare conditions strongly suggest that they should
be kept separate from other services, once they have been defined.
Consequently, those services for rare conditions that are commissioned
at national level should also be funded at national level with
no impact on any individual GP consortium since the presence of
a cluster of patients is not a factor of poor public, or other,
healthcare.
3.7 We believe that AGNSS has a key role
to play and that it should develop strong relationships with patient
organizations, which should be fully engaged in decision-making
processes.
3.8 We believe that future planning of commissioning
and resourcing for rare conditions should be the responsibility
of AGNSS on behalf of the NHS Commissioning Board and that this
should be informed by an effective horizon scanning process in
which manufacturers should be encouraged to participate.
October 2010
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