Genzyme UK has entered into a partnership with the NHS National Commissioning Group, under which it will provide funding of £7 million over three years to help support the care of patients with lysosomal storage disorders.

LSDs are rare and often severe metabolic disorders – such as Gaucher, Fabry and Pompe diseases - that need specialist and multi-disciplinary care. As a spokeswoman for the NCG explained to PharmaTimes UK News, drugs totreat these disorders are very expensive, so treatment must be initiated by a skilled physician and patients need to be monitored properly.

To this end, the NCG has selected seven hospitals in the UK that will handle the specialist care of these patients: Royal Free Hospital;Great Ormond Street Hospital; The National Hospital for Neurology and Neurosurgery in London; Adden brooke’s Hospital in Cambridge; Birmingham Children’s Hospita; Royal Manchester Children’s Hospital; and Hope Hospital in Salford.

Commenting on the deal, Health Minister Dawn Primarolo,said: “We are delighted that we have been able to join in this partnership with Genzyme for patients with lysosomal storage disorders and that we can jointly support patients living with these debilitating diseases. This partnership will aid in sustaining our ‘world class’ commissioning of services for rare diseases through the NCG.”

Ensuring high-quality care

The NCG, which replaced the 1983-established The National Specialised Commissioning Group last year, advises the government on which NHS services should be commissioned nationally instead of locally to “ensure a high quality of clinical care and equity of access for patients, as well as securing value for money”, according to the DH.

Most services commissioned by the NCG relate to a condition where the national case load is less than 400 people. Currently, there are around 40 such services for rare diseases of interventions being commissioned by the NCG, including lung and heart transplantations, the spokes woman explained, which are obviously best carried out at designated centres instead of every hospital.

The group works with an annual funding of £346 million,which, from this year, has been brought back to primary care trust baselines from the central DH budget.

Through its “world-class commissioning model”, the NHS has been able to meet the needs of patients with LSDs in England, the NCG says, a claim that is backed by Paul Drohan, General Manager of Genzyme UK and Ireland,who commented: “The NHS has developed a highly effective model of care for patients with LSDs and that this is one of the best examples in the world of a universal healthcare system managing rare diseases”.

Genzyme's interest

Genzyme’s interest in providing funding to ensure the best care for patients with LSDs is clear; the firm markets several drugs for the treatment of these disorders, including: Cerezyme (imiglucerase for injection)and its predecessor Ceredase (aglucerase injection), “the only enzymere placement treatment for Type 1 Gaucher disease for over a decade”; Fabrazyme (agalsidase beta) for Fabry disease; Aldurazyme (laronidase), “the first and only US and European treatment for mucopolysaccharidosis I (MPS I)”; and Myozyme (alglucosidase alfa) for Pompe disease.

“As enzyme replacement therapies are a key element of successful treatment of patients with LSDs, Genzyme is excited to have an opportunity to work closely with the NHS to support this service now and in to the future,” Drohan concluded.