Written evidence from Actelion Pharmaceuticals
Limited (COM 63)
1. Actelion is a global, independent biopharmaceutical
company that discovers, develops and markets innovative drugs
for high, unmet medical needs. Our currently marketed products
are primarily focused in the area of orphan or ultra-orphan diseases.
2. An orphan disease is one that affects
fewer than 500 people per million of the population; an ultra-orphan
disease affects fewer than 1,000 patients. Collectively, orphan
diseases affect around 3.5 million people in the UK.
3. We are dedicated to discovering and developing
innovative medicines to improve or prolong patients' lives and
take a close interest in the service delivery structures of the
diseases in which we are involved. We therefore read with interest
the Health White Paper and its accompanying suite of consultation
documents and have a number of thoughts regarding their potential
impact on services for rare diseases.
SPECIALISED COMMISSIONING
4. It is important to ensure that specialised
services continue to be commissioned at an appropriate level (ie
one at which they can be effectively managed given their large
planning populations). We therefore welcome responsibility for
the commissioning of these services falling to the new NHS Commissioning
Board.
5. We are concerned that not all services
contained with the national specialised services definition set
(NSSDS) are actively commissioned by the NCG and SCGs under the
current arrangements. The White Paper reforms present an opportunity
to review the NSSDS to ensure that there is a clear set of services
in place, with a clear understanding of what is to be commissioned,
at what level and by whom. The definition set should also be regularly
reviewed to ensure the services within it are being actively commissioned.
6. We would be keen to know how funding
for national specialised services, including those for rare diseases,
will be allocated. Ideally, the NHS Commissioning Board should
receive direct funding from Government to commission these services,
based on clear and costed patient pathways.
7. We would like to see the NHS Commissioning
Board working with dedicated multi-stakeholder specialised commissioning
networks to develop the commissioning plan for each of the services
defined within a revised NSSDS. These networks should be service
specific and include representatives of GP consortia and local
authorities (social care) as well as appropriate expert clinician
and patient representation.
8. The NHS Commissioning Board's regional
offices should be tasked with ensuring the commissioning decisions
made by the national board (with the support of specialised commissioning
networks) are effectively implemented by maintaining strong links
with GP consortia, patients representatives, local authorities
and service providers at a regional level.
9. In addition, these offices could carry
out at the regional level some of the functions that the proposed
health and wellbeing boards would undertake at the local level,
ie promoting integration and partnership across areas, including
the promotion of joined up commissioning plans across the NHS,
social care and public health, and undertaking a scrutiny role
in relation to major service redesign.
ASSESSMENT OF
DRUGS FOR
RARE DISEASES
10. We would be keen to establish whether
the NHS Commissioning Board will take forward the role of the
Advisory Group on National Specialised Services (AGNSS) in assessing
some highly specialised drugs as part of national commissioned
services.
11. We believe that making decisions about
the introduction of these new drugs may best be undertaken within
these arrangements, particularly where drugs are introduced into
a disease area for which there is no existing therapy or represent
a step change in the way that disease is managed, both of which
are likely to necessitate significant service development.
12. However, we remain concerned that many
orphan drugs (specifically those with potential treatment populations
greater than 500) will not fall under this initiative and, because
they may be equally unsuitable for assessment under current appraisal
methods (ie NICE STA), run the risk of falling through the gap.
13. The now abandoned "innovation pass"
was set it in recognition of the fact that there were, "...drugs
for small patient populations which have the potential to deliver
improved patient outcomes but where data to demonstrate cost-effectiveness
is so far limited. This may limit the medicine's market access,
denying NHS patients access to certain innovative and effective
medicines, and limiting companies' abilities to bring valuable
medicines to the UK market." This issue of access to such
drugs has yet to be adequately addressed and we would welcome
the opportunity for it to be looked at within the overall reforms
taking place.
IMPROVING PATIENT
QUALITY AND
EXPERIENCE
14. A key element of the White Paper is
the development of an Outcomes Framework against which the performance
of the NHS will be measured. Outcomes will be supported by a suite
of disease-related quality standards, to be produced by the National
Institute for Health and Clinical Excellence (NICE), which will
be used to inform commissioning plans.
15. We would be keen to establish whether
NICE will be developing quality standards for rare diseases and
if so, whether they would be disease-specific or apply across
rare conditions in general.
16. If NICE are not to be tasked with developing
quality standards for rare diseases, its imperative that this
role falls to specialised commissioning networks involving patients
and carers, social services and specialized clinicians.
17. We believe that an integrated approach
to commissioning services for rare diseases will be important
throughout. The Chief Medical Officer's Annual Report 2009 highlighted
the importance of integrating family, social care and education
services in supporting patients with rare diseases and there should
therefore be appropriate involvement by representatives in these
areas in the commissioning process. Measuring patients' experience
of services would also be important in improving quality.
RARE DISEASE
PLAN
18. In June 2009, the Council of the European
Union published a recommendation for all Member States to establish
and implement a plan on rare diseases with the aim of ensuring
that patients had access to high quality care. It called for the
plans to be adopted by 2013 at the latest and for the European
Commission to monitor progress.
19. It will be important to ensure that
the UK plan is coordinated with and aligned to the reforms due
to be introduced under the White Paper so as to ensure that both
maximise their benefit to patients with rare diseases.
October 2010
| 
