Written evidence from Rare Disease UK
(COM 71)`
SUMMARY
— There are many issues facing commissioning
of specialised services currently, most notably the inequitable
provision of services and a "postcode lottery".
— The changes proposed in the NHS White
Paper pose both threats and opportunities to services for patients
with rare diseases.
— Due to the insufficient knowledge of
rare conditions at a local level, the NHS Commissioning Board
should be responsible for those specialised services currently
commissioned at a regional and national level.
— The budget for specialised services must
be allocated directly to the NHS Commissioning Board specifically
for specialised services. This budget must be flexible to allow
for expansion of the number of specialised services.
— It is crucial that engagement is facilitated
between the NHS Commissioning Board and GP consortia to ensure
that services commissioned at both levels are integrated and that
there are no gaps in service provision.
ABOUT RARE
DISEASE UK
1. Rare Disease UK (RDUK) is the national
alliance for people with rare diseases[122]
and all who support them. We have over 550 registered members
including: over 130 patient organisations, clinicians, researchers,
academics, industry and individuals with an interest in rare diseases.
2. It is estimated that one in 17 people
will be affected by a rare disease at some stage in their lives.
This amounts to 3.5 million people across the UK. Collectively,
rare diseases are not rare.
3. RDUK was established by Genetic Alliance
UK, the national charity of over 130 patient organisations supporting
all those affected by genetic conditions, in conjunction with
other key stakeholders in November 2008 following the European
Commission's Communication on Rare Diseases: Europe's Challenges.
4. Subsequently RDUK successfully campaigned
for the adoption of the Council of the European Union's Recommendation
on an action in the field of rare diseases. The Recommendation
was adopted unanimously by each Member State of the EU (including
the UK) in June 2009. The Recommendation calls on Member States
to adopt plans or strategies for rare diseases by 2013.
5. RDUK is campaigning for a strategy for
integrated service delivery for rare diseases. This would coordinate:
research, prevention and diagnosis, treatment and care, information
and commissioning and planning into one cohesive strategy for
all patients affected by rare disease in the UK. As well as securing
better outcomes for patients, a strategy would enable the most
effective use of NHS resources.
6. Robust commissioning structures enabling
the effective service delivery for patients with rare diseases
should form a key element of a strategy for rare diseases. As
a result, we welcome the opportunity to submit evidence to this
document.
SPECIALIST COMMISSIONING
FOR RARE
DISEASES
7. Patients with rare diseases are likely
to need access to health services commissioned at different levels,
from local health services and social care which a patient is
likely to use most frequently, to specialised services commissioned
on a national basis, and from both a chronic and acute position
of need. An effective commissioning structure for rare diseases
needs to ensure effective integration of those commissioning services
at the local level and those commissioning specialised services.
ISSUES CURRENTLY
AFFECTING COMMISSIONING
OF SERVICES
FOR PATIENTS
WITH RARE
DISEASES
8. RDUK established a multi-stakeholder
Working Group on Commissioning and Planning. The findings of the
Working Group, along with four others, will be published in a
report on a strategy for rare diseases next year. The initial
findings of the Group are as follows.
9. Apart from those services commissioned
on a national level by NHS Specialised Services, there is a wide
variation in the services available for patients with rare diseases
across England due to the decisions taken at different levels
of the commissioning structure. Care is not of an equal quality
for all patients with any particular condition throughout the
UK. The "postcode lottery" still exists whereby some
patients are provided with a carefully planned programme of support
and treatment (including drugs and other interventions where appropriate)
and others are not, purely as a consequence of variations in the
pattern of services locally or regionally planned and commissioned.
10. The Specialised Services National Definitions
Set (SSNDS) sets out definitions of 34 specialised services each
with a planning population of more than one million. The SSNDS
exists to identify those services that are specialised and therefore
should be subject to collaborative commissioning by Primary Care
Trusts (PCTs) through the ten regional Specialised Commissioning
Groups (SCGs). Many of the services outlined in the SSNDS will
be accessed by patients with rare diseases. These arrangements
have proved ineffective as currently none of the regional SCGs
commissions all the services in the SSNDS; and not one SCG commissions
the same services as another. This therefore results in unequal
levels of care for a particular condition throughout the country.
11. There is often a lack of transparency,
communication and structure in commissioning guidelines and what
should be funded by whom. This leads to confusion and can result
in a delay in patients receiving the necessary services at the
best time.
12. Funding for specialist services for
patients with rare diseases is perceived by some to be diverting
resources away from local services. Rather this is a mechanism
to ensure that patients with rare diseases have access to the
services and expertise they need, planned and commissioned at
the appropriate level, whilst ensuring local bodies do not have
to bear a disproportionate financial burden. This negative perception
could pose a threat to specialised services at a time of efficiency
savings as those commissioning services at a local level look
to protect their budgets at the expense of good-quality, cost
efficient care for specialised services. This negative perception
is aggravated by the current funding method for national and regional
commissioning ie the top-slicing of funds for national commissioning
and the pooling of funding by PCTs for regional commissioning.
13. Patients who have access to a specialised
service for their condition have better experiences than those
that don't. However, there are not specialist services available
for every rare condition, and this can result in inequitable levels
of care depending on which condition a patient has. It is not
possible to designate a specialised service for every rare condition,
especially as there are over 6000 rare conditions and some rare
conditions are too rare or insufficiently well understood. But
this should not preclude patients from being able to access good-quality
care.
14. Specialist services for rare conditions
have developed in an ad-hoc fashion. Services often gain national
designation as a result of proactive moves by a strong patient
organisation and interested clinicians. We have heard from our
members that the application process is also becoming increasingly
burdensome. This is clearly inequitable as in particular, most
patient organisations for rare diseases (if they exist at all)
are small and do not have the resources to undertake this work.
SPECIALISED COMMISSIONING
POST-WHITE
PAPER
15. Whilst the detail of the proposals in
the Government's White Paper, "Equity and Excellence: Liberating
the NHS" and the supporting consultation document "Commissioning
for Patients" is not completely clear, the proposals do provide
both opportunities and threats to services for patients with rare
diseases.
16. The White Paper has a strong emphasis
on the benefits of localism. However, this is not always best
in order to understand the health needs of the patients we represent.
The strength of GPs lies in their understanding of a broad range
of conditions that they see on a regular basis. There are over
6000 rare diseases and GPs may only come across some of these
conditions once (if at all) in the duration of their careers.
Many patients with rare diseases have complex needs and GPs do
not have the knowledge necessary to understand what services are
necessary to meet these needs the majority of the time.
17. The most "common" single rare
disease will affect around 25,900 people[123]
in England (the vast majority of rare diseases affect far fewer
people). If this maximum figure is distributed evenly across the
current 151 PCTs then each PCT would have 171 patients with this
condition.[124]
If GP consortia cover smaller population bases than PCTs do currently,
then they will cover even fewer patients with that condition.
It seems doubtful whether the expertise will be available to commission
all the services necessary for the most "common" rare
diseases, let alone the majority of rare diseases which affect
far fewer people. There is a real danger that these conditions
could be overlooked.
18. The proposals do not indicate what population
bases GP consortia will cover, although we understand that they
are likely to be smaller than PCTs currently. This means that
a large number of services could fall in the area between being
too low-incidence to be commissioned by individual GP consortia
and too high volume to require a planning population greater than
1 million as the SSNDS currently provides for. The commissioning
of those services that fall between these thresholds should be
carefully monitored to ensure they are available equitably to
people across England. A key component of the duty of the NHS
Commissioning Boards to hold consortia to account should be to
ensure that patients of consortia have access to services commissioned
at the level above a consortium-unit volume of population.
19. For these reasons, RDUK strongly endorses
proposals for the NHS Commissioning Board to take responsibility
for commissioning specialised services. We believe that the Board
should have responsibility for commissioning those services currently
commissioned at a national level and those services listed in
the Specialised Services National Definitions Set (SSNDS) currently
commissioned by regional specialised commissioning groups (SCGs).
20. The establishment of the NHS Commissioning
Board provides the opportunity to address some of the issues inherent
in the commissioning of specialised services currently, most notably
the weaknesses of the SCGs in delivering equitable service provision
across England.
21. While we regard the positioning of all
specialised commissioning responsibilities in the NHS Commissioning
Board as sensible, we recognise in practice this may need a devolved
regional structure that allows proper understanding of regional
variations and to help ensure integration between services commissioned
by GP consortia and specialised services. However, any regional
system must be under tighter central control by the Board to avoid
the present situation which has led to wild inconsistencies in
service provision.
22. The budget for specialist services that
are currently commissioned must be allocated directly to the NHS
Commissioning Board specifically for specialist services. This
budget must take into account that, as the SCGs do not currently
commission all the services in the SSNDS, their expenditure may
not adequately reflect the budget that the National Commissioning
Board will need to provide an equitable service across England.
23. The budget for the NHS Commissioning
Board must also be flexible to allow for new services to be commissioned
when evidence shows a need for services to be commissioned at
this level in order to provide good quality and equitable services.
24. In reality, there will never be a specialised
service for each of the 6000 plus rare diseases. However, commissioning
structures should be flexible to allow patients to access services,
not specifically designated for their condition, but that they
would benefit from nonetheless as many of the effects or services
needed may be similar.
25. It is crucial that engagement is facilitated
between the NHS Commissioning Board and GP consortia to ensure
that services commissioned at both levels are integrated and that
there are no gaps in service provision.
26. Rare Disease UK believes there should
be a designated expert accountable for commissioning for rare
diseases on the NHS Commissioning Board. This member should work
closely with the National Clinical Director for Rare Diseases
(as proposed in the Chief Medical Officer's report of 2009) who
RDUK would like to oversee the implementation of a strategy for
rare diseases.
October 2010
122 A rare disease is defined as a condition affecting
less than five in 10,000 people in the UK. Back
123
Based on the Office of National Statistics Population Estimates
June 2010-http://www.statistics.gov.uk/pdfdir/pop0610.pdf Back
124
In reality the distribution would not be even due to greater prevalence
in certain populations eg some conditions are more common in population
sub-groups. Examples include thalassemia in Mediterranean populations,
and the increased prevalence of recessive single gene genetic
conditions in people of Pakistani origin. Service planning should
take into account any such local needs. Back
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