| Formulary Chapter 9: Nutrition and blood - Full Chapter
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| Chapter Links... |
 NENC Guidelines for the recognition and management of non-IgE cow’s milk protein allergy (CMA) in infants |
| Details... |
| 09.08 |
Metabolic disorders |
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| 09.08.01 |
Drugs used in metabolic disorders |
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Asfotase alfa (Strensiq®)
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Formulary
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- 40mg/ml & 100mg/ml solution for injection
- Approved for the treatment of paediatric-onset and juvenile-onset hypophosphatasia in line with NICE and NHS England Commissioning Policy
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 NICE HST23: Asfotase alfa for treating paediatric-onset hypophosphatasia
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Betaine
(Homocystinuria)
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Formulary
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Calcium Levomefolate (Prefolic)
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Formulary
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Copper histidinate
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Formulary
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- Subcutaneous copper histidinate injections
- Approved for presymptomatic neonates with classical Menkes disease in line with NHSE Specialised Commissioning Policy
- The following providers are commissioned to provide this service within North East:
- The Newcastle upon Tyne Hospitals NHS Trust
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NHSE Specialised Commissioning Policy 2577: Subcutaneous copper histidinate injections for presymptomatic neonates with classical Menkes disease
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Nitisinone
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Formulary
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Sebelipase alfa (Kanuma®)
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Formulary
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- 20mg/10ml concentrate for solution for infusion
- Approved for long-term enzyme replacement therapy in Wolman disease (rapidly progressive lysosomal acid lipase deficiency), only if people are aged 2 years or under when treatment starts, in line with NICE and NHSE Specialised Commissioning Policy.
- Services are only commissioned at:
- Birmingham Women and Childrens NHS Foundation Trust
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Manchester University Hospitals NHS Foundation Trust
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NICE HST30: Sebelipase alfa for treating Wolman disease
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Ubiquinone (Coenzyme Q10, Ubidecarenone)
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Formulary
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- 10mg, 30mg, & 100mg capsules
- 50mg in 5ml & 30mg in 1ml (150mg in 5ml) oral solution
- nutritional supplement and antioxidant approved for use in the
management of mitochondrial disorders.
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Velmanase alfa (Lamzede ®)
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Formulary
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- 10mg powder for solution for infusion
- Approved for treating alpha-mannosidosis in line with NICE and NHS England Specialised Commissioning Policy
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NICE HST29: Velmanase alfa for treating alpha-mannosidosis
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Chenodexoycholic acid (Xenbilox®)
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Formulary
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- 250mg capsules
- Approved for the treatment of inborn errors of bile acid synthesis (all ages) in line with NHS England Commissioning Policy
- Approved for the treatment of Cerebrotendinous Xanthomatosis in line with NHS Commissioning Policy. NHS England will commission this drug for patients who are currently being treated (April 2017)
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| 09.08.01 |
Wilsons disease |
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Penicillamine
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Formulary
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- 125mg & 250mg tablets - also used for the treatment ofcystinuria and rheumatoid arthritis – section 10.1.3
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Zinc Acetate (Wilzin®)
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Formulary
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Trientine Dihydrochloride
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Formulary
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- 300mg capsules
- Approved for the treatment of Wilsons disease in line with NHS England Commissioning Policy. NHS England will commission the treatment for patients who are currently being treated (April 2017)
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Tiopronin
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 Unlicensed
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- 500mg tablets - for use as 2nd line treatment for cystinuria in
patients who fail to tolerate/respond to penicillamine unlicensed.
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| 09.08.01 |
Carnitine deficiency |
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Carnitine
(Levocarnitine)
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Formulary
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- 30% oral solution
- 1g in 5ml injection
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| 09.08.01 |
Fabry's disease |
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Agalsidase Alfa
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Formulary
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Agalsidase Beta
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Formulary
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Migalastat (Galafold ®)
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Formulary
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- Approved for the treatment of Fabry's disease in line with NICE
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Highly specialised technologies guidance HST4: Migalastat for treating Fabry disease
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Pegunigalsidase alfa (Elfabrio®)
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Formulary
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- 20mg/10ml concentrate for solution for infusion
- Approved for treating Fabry disease in line with NICE and NHSE Specialised Commissioning guidance
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NICE TA915: Pegunigalsidase alfa for treating Fabry’s disease
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| 09.08.01 |
Gaucher's disease |
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Imiglucerase (Cerezyme®)
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Formulary
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Velaglucerase
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Formulary
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| 09.08.01 |
Mucopolysaccharidosis I |
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Elosulfase alfa
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Formulary
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- 5mg/5ml concentrate for solution for infusion:
- treating mucopolysaccharidosis type 4A in people of all ages
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NICE HST19 - Elosulfase alfa for treating mucopolysaccharidosis type 4A
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Galsulfase (Naglazyme®)
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Formulary
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Idursulfase
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Formulary
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Laronidase (Aldurazyme®)
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Formulary
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| 09.08.01 |
Pompe disease |
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Alglucosidase Alfa (Myozyme®)
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Formulary
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Avalglucosidase alfa (Nexviadyme®)
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Formulary
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- 100mg powder for concentrate for solution for infusion
- Approved for treating Pompe disease in line with NICE
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NICE TA821: Avalglucosidase alfa for treating Pompe disease
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Cipaglucosidase alfa (CIPA) (Pombiliti®)
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Formulary
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- 105mg powder for concentrate for solution for infusion vials
- Approved with with miglustat (Opfolda) for treating late-onset Pompe disease in adults in line with NICE and NHSE Specialised Commissioning guidance
For information only - as there are no providers within the North East & Cumbria area that are commissioned to provide this service
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NICE TA912: Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease
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| 09.08.01 |
Nephropathic cystinosis |
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Mercaptamine
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Formulary
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| 09.08.01 |
Urea cycle disorders |
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Arginine
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Formulary
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Carglumic Acid (Carbaglu®)
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Formulary
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Sodium Benzoate
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Formulary
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Sodium Phenylbutyrate
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Unlicensed
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- 500mg tablets - Licensed formulation marketed as Ammonaps
- 1g in 5ml & 2g in 10ml injections
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| 09.08.02 |
Acute porphyrias |
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Givosiran (Givlaari®)
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Formulary
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- Approved for treating acute hepatic porphyria (AHP) in adults and young people aged 12 and older in line with NICE, only if:
- they have clinically confirmed severe recurrent attacks (4 attacks or more within 12 months) and
- the company provides it according to the commercial arrangement.
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NICE HST 16: Givosiran for treating acute hepatic porphyria
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Human Arginate
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Formulary
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| 09.08.02 |
Drugs unsafe for use in acute porphyrias |
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| Non Formulary Items |
Sebelipase alfa (Kanuma®)
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Non Formulary
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- For treating lysosomal acid lipase deficiency that is not Wolman disease - terminated appraisal
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NICE TA961: Sebelipase alfa for treating lysosomal acid lipase deficiency that is not Wolman disease (terminated appraisal)
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Ubidecarenone
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Non Formulary
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- DO NOT PRESCRIBE for new patients
- Where appropriate, undertake deprescribing in existing patients
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Key |
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Restricted Drug |
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Unlicensed |
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Link to adult BNF
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Link to children's BNF
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Link to SPCs
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Cytotoxic Drug |
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Controlled Drug |
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High Cost Medicine |
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NHS England |
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Homecare |
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ICB |
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Low carbon footprint |
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Medium carbon footprint |
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High carbon footprint |
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| Status |
Description |
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Drugs for hospital use only. The responsibility for initiation and monitoring treatment should rest with an appropriate hospital clinician and the drug should be supplied through the hospital throughout the duration of treatment. In some very exceptional circumstances (e.g. due to distance from the hospital, storage, supply or mobility/transport problems) it may be appropriate for the GP to be asked to prescribe a Red drug. This should be negotiated on an individual patient basis and should only be done with the GP’s prior informed agreement where the roles of the GP and hospital services are clearly defined and agreed. The GP should not feel under pressure to prescribe in these circumstances. For all RED drugs automatically added to the formulary in response to a positive NICE TA: Prescribers need to ensure that local Trust new drug governance procedures and pharmacy processes are followed before any prescribing. |
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Drugs initiated by hospital specialist, but where continuing treatment by GPs may be appropriate under a shared care arrangement. These medicines are considered suitable for primary care prescribing following specialist initiation of therapy and stabilisation, with ongoing communication between the primary care prescriber and specialist as set out in the associated shared care guideline (SCG). Shared care should be initiated by the specialist, which includes consultant, suitably trained specialist non-medical prescriber or GPwER within a secondary, tertiary, or primary care clinic.
The specialist should send the primary care prescriber a copy of the NENC Clinical Effectiveness and Governance (CEG) Subcommittee approved SCG to sign. The primary care prescriber should sign the SCG or indicate reasons why they are unable to accept the agreement and return a copy back to the specialist, as soon as possible.
SCGs are available or are being developed for most of the drugs listed as AMBER. |
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Drugs normally recommended or initiated by a hospital specialist who is a prescriber, a GP with an extended role [GPwER], or a specialist within primary care which can be safely maintained in primary care and monitored in primary care. In some cases, a further restriction for use may be defined. The primary care prescriber must be familiar with the drug to take on prescribing responsibility or must obtain the required information from the specialist. Therefore, provision of additional information, or an information leaflet, may be appropriate in some cases to facilitate continuing treatment by primary care prescriber or provide information re stopping criteria.
These are considered suitable for primary care prescribing following specialist assessment and recommendation of therapy, with ongoing communication between the primary care prescriber and specialist, if necessary.
In some case these drugs require specialist initiation and short to medium term monitoring of efficacy or toxicity until the patient’s dose is stable. Following specialist review the patient may be transferred to primary care for ongoing prescribing. Ongoing prescribing by primary care can include, if required, additional dose titrations and assessment of efficacy, with ongoing communication between the primary care prescriber and specialist, if necessary.
If the drug requires urgent initiation, it is expected that the specialist undertakes the initial prescribing responsibility for an appropriate period of time, usually a minimum of 28 days. A GREEN+ drug can only be recommended to primary care for initiation if does not need to be initiated within 28 days. |
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Medicines suitable for initiation, ongoing prescribing and discontinuation in all care settings, subject to appropriate communication between those responsible. |
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UNDER REVIEW: drugs whose current formulary status or RAG status is currently under review. |
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Drugs that have been considered by the NENC Clinical Effectiveness and Governance (CEG) Subcommittee (or other approved body) and are not approved for prescribing within the North East and North Cumbria ICS. These may also include all medicines with a “not NHS” or “DLCV” classification in the BNF, those agents as included within the NICE “Do not do” list, and those agents included with the NHS England: Items which should not routinely be prescribed in primary care. |
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