Statement on risk-sharing arrangements for Enzyme Replacement Therapy
(ERT) in Scotland
In February 2007, the risk-sharing scheme for enzyme replacement therapy (ERT)
for lysosomal storage disorders was extended to include orphan drugs and other
high-cost therapies which are approved for use in Scotland by the Scottish Medicines
Committee (SMC).
Orphan drugs and other high-cost therapies which receive EU licences should be
considered for inclusion in the risk-sharing arrangements on the following basis:
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Only SMC-approved products should be added to the scheme
That, prior to assessment by the SMC, decisions on treatment - and,
therefore, responsibility for funding – are for individual NHS Boards on
advice from their Director of Public Health
If approved by the SMC, products should be added to the risk-share from
the date of SMC approval
At that time there were 31 licensed orphan drugs with a European licence. These fell
broadly into three treatment categories:
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pulmonary hypertension (already nationally-designated and funded)
rare cancers (e.g. Glivec)
inborn errors of metabolism
A full list of orphan drugs is available on: http://www.orpha.net
List of drugs included in orphan drugs risk share arrangement as at April 2009
Medicines Included
The drugs listed below are only included in the risk share for the specific
indications listed and only in the specific circumstances described (see
footnotes).
1. Medicines included in the risk share arrangements prior to establishment of SMC:
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Agalsidase alpha (Replagal) for the treatment of Fabry’s Disease1
Agalsidase beta (Fabrase/Fabrazyme) for the treatment of Fabry’s Disease1
Imiglucerase (Cerezyme) for the treatment of Type 1 Gaucher’s Disease1
2. Medicines accepted for use by SMC:
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Miglustat (Zavesca/Vevesca) for the treatment of Type 1 Gaucher’s Disease1
Iloprost (Ventavis) for the treatment of pulmonary arterial hypertension 2 (in the
circumstances set out below)
3. Medicines accepted for restricted use by SMC:
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Carbaglu (N-carbamoyl-L-glutamic acid) for the treatment of hyperammonaemia
due to N-acetylglutamate synthase deficiency
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Bosentan for the treatment of specific indications in pulmonary arterial
hypertension2,3 (in the circumstances set out below)
Sitaxentan 100mg tablets (Thelin) for the treatment of patients with pulmonary
arterial hypertension2,3 (in the circumstances set out below).
4. Medicines not recommended by SMC but included in the risk share if there has
been an exceptional case review:
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Alglucosidase alfa (Myozyme) for the treatment of Pompe disease (acid maltase
deficiency) 1
Laronidase (Aldurazyme) for the treatment of MPS 1 (Hurler Disease or Hurler
Syndrome) 1
Idursulfase (Elaprase) for the treatment of MPS II (Hunters syndrome) 1
Medicines Excluded
5. Orphan drug therapies not yet considered by the Scottish Medicine Consortium
and therefore excluded from the risk share arrangements:
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Galsulfase (Naglazyme) for the treatment of MPS VI (Maroteaux-Lamy
syndrome)
Eculizumab for the treatment of paroxysmal nocturnal haemoglobinuria (PNH)
Footnotes:
1.
Inclusion in the risk share is conditional on treatment being consistent with UK
protocols, and being initiated, overseen and reviewed by the Enzyme
Replacement Therapy specialists in one of the designated English centres; or by
the metabolic specialist at the Royal Hospital for Sick Children in Glasgow.
2. Outwith this risk share arrangement, national funding is conditional on treatment
being initiated, overseen and reviewed by specialists in the designated national
Scottish Pulmonary Vascular Unit, Glasgow. (Costs of this drug therapy are met
by NSD within the designated national specialist pulmonary arterial hypertension
service.)
3. Bosentan, Sitaxentan and ambrisentan are included in the risk share for the
treatment of:
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Pulmonary Arterial Hypertension in patients under the care of the designated
national specialist service – the Scottish Pulmonary Vascular Unit (SPVU)
(Glasgow)
Adult patients with Eisenmenger syndrome under the care of the designated
national specialist service – Scottish Adult Congenital Cardiac Service (SACCS)
(Glasgow) who receive treatment while following a specific approved protocol
(see below).
Adult patients with ASD, VSD or patent ductus arteriosis (usually with no repair
attempted during childhood) who are found to have PAH and are under the care
of the designated national service (SACCS) (formerly known as Grown Up
Congenital Heart –GUCH).
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Continuation of therapy for adults with congenital heart disease under the care of
the designated national SACCS who were treated with Bosentan as children
under the care of the designated national specialist service for pulmonary arterial
hypertension.
Bosentan is nationally funded through the designated pulmonary arterial
hypertension service for patients in the care of SPVU, and is nationally funded for
through the risk share for adults with congenital heart disease and pulmonary
hypertension on the condition that the patient is registered with designated
specialist SACCS and that the designated service reviews and monitors
treatment to ensure agreed thresholds of benefits are being met.
Care follows a specific protocol as follows:
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Pre-treatment 6 minute shuttle walk test will be performed and Quality of Life
(QoL) questionnaire administered.
A 6 minute shuttle walk test will be performed at 3 months post initiation of
treatment and the same QoL questionnaire
If there is no improvement in the 6 minute shuttle walk test the drug is withdrawn.
Services covered by national funding
Other drug therapies covered by these arrangements, and the indications for which
they will be prescribed, are:
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Imiglucerase (Cerezyme) for long-term enzyme replacement therapy in
patients with a confirmed diagnosis of Type I Gaucher’s disease and who
exhibit clinically significant manifestations of the disease
Agalsidase beta (Fabrase/Fabrazyme) for long-term enzyme replacement
therapy in patients with a confirmed diagnosis of Fabry’s disease (agalactosidase A deficiency)
Agalsidase alpha (Replagal) for long-term enzyme replacement therapy in
patients with a confirmed diagnosis of Fabry’s disease (a-galactosidase A
deficiency)
Miglustat (Zavesca/Vevesca) for treatment of mild to moderate type I
Gaucher’s disease. Zavesca may be used only in the treatment of patients for
whom enzyme replacement therapy is unsuitable
The Scottish Medicines Consortium [SMC], which assesses the effectiveness of new
drugs and makes recommendations to NHS Boards and their Area Drug and
Therapeutics Committees [ADTCs] on their use, have concluded that:
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Laronidase (Aldurazyme) is not recommended for use within NHSScotland for
the treatment of mucopolysaccharidosis.
Idursulfase (Elaprase) is not recommended for the treatment of MPS ll
(Hunters syndrome)
Individuals who were receiving Laronidase at the formation of a risk-sharing
arrangement are included in the scheme, though any individual commencing
treatment with Laronidase after 1 July 2005 should be excluded from national funding
arrangements. Any costs would therefore be the responsibility of the local NHS
Board.