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Treatment funding agreed for seriously ill child

Funding of medical treatment for a 6 year old girl with a life-threatening genetic disease has finally been agreed after a delay of over a year. Her family are represented by Human Rights solicitor Alison Millar.

Lisa-marie Goodwin with her mum Shirley-Anne Six year old Lisa-marie 's rights to a fair assessment and treatment of her medical needs were being ignored

27 October 2004

South Warwickshire Primary Care Trust (PCT) have been advised by their Complex Cases Panel that the funding for potentially life-saving medical treatment for six year old Lisamarie Goodwin should be provided for another 2 years, at which point the situation will be formally reviewed.

Lisamarie suffers from a very rare genetic disease called Mucopolysaccharidosis 1 (MPS1), also called Hurler-Scheie Disease.  This is one of a group of conditions known as lysosomal storage diseases, where the body lacks a particular enzyme.  As a result, waste products, which normally get broken down, instead get stored in the body’s cells.  Over time, this causes progressive damage and disease.  Left untreated, most patients with this condition will die in their late teens or early twenties. There is no known cure.

In December 2002, Lisamarie was one of a number of children selected for a clinical trial of a new treatment for the symptoms of MPS1.  This worked by giving Lisamarie the enzyme, laronidase, which she was lacking.  The brand name for the enzyme is Aldurazyme.

Before she started to receive treatment, Lisamarie had developed disabling joint pain and stiffness, breathlessness and exhaustion, heart valve disease, eyesight and hearing problems.  She could not ride a bicycle or go out to play with her friends and was an unhappy little girl.

However the enzyme replacement therapy really made a difference to Lisamarie. She did very well on the treatment and her increased physical well-being contributed to her becoming a more active learner at school.

The trial Lisamarie was on was a 'Under 5s' study for very young children. Other clinical trials had already been done successfully on older children. Following the success of these trials, the enzyme replacement treatment was licensed as a safe and effective treatment for the non-neurological symptoms of MPS1.

In August 2003, Lisamarie’s specialist applied to her local health authority, South Warwickshire Primary Care Trust (PCT), for funding to continue the enzyme treatment after the clinical trial finished in December 2003. Over a year later, the PCT had still not made a proper decision on her application.

The PCT’s Complex Cases Panel met in November 2003 and again in December 2003, when they declined Lisamarie’s application.  Mr and Mrs Goodwin appealed against their decision. The appeal hearing took place in May 2004  but neither Lisamarie’s parents or their representative were allowed to present the circumstances of the case. Unsurprisingly, they later heard that their appeal had failed.

Leigh, Day & Co then got involved because of concerns about the PCT’s approach and significant procedural failings. On 25 August 2004, judicial review proceedings were issued to ask the Court to overturn the PCT’s refusal of treatment and to direct the PCT to make a new decision.

The PCT admitted that their Panel’s original decision was unlawful and agreed to reconsider Lisamarie’s application after they had agreed a new policy on the commissioning of enzyme replacement therapies. It was hoped that this reconsideration would take place promptly and fairly. This did not happen.

The PCT’s Complex Cases Panel was due to look again at the case. They did meet but decided they did not have sufficient evidence on Lisamarie’s up-to-date progress on the treatment. However, the PCT had pages of material describing how Aldurazyme has benefited Lisamarie and should have anticipated if further clinical information was needed well in advance, especially considering the nature of the case.

Since the beginning of the year, Lisamarie has received treatment on compassionate grounds, funded out of a special charitable programme. There was no guarantee that treatment would continue to be available to her on this basis, and the PCT were aware that her current supply of enzyme will run out on 31st October 2004.

The delays in decision making and serious deficiencies in the PCT’s process have taken an enormous toll on the Goodwin family. Mr and Mrs Goodwin could not understand why their daughter’s potentially life saving treatment was being denied when all the other patients who were on the Under 5s trial had been given funding by their health authorities.

Alison Millar, human rights solicitor, is representing the Goodwin family:

“The family are delighted that Lisamarie’s treatment is now secure. Their lives have been on hold during this drawn out process and, thankfully, now they can resume some normality.”

Funding of Lisamarie's enzyme replacement therapy was given the green light

The decision to provide funding for Lisamarie’s treatment was taken this week after a meeting on 20th October by the Complex Case Panel for South Warwickshire PCT. They concluded that there was enough evidence to show that Lisamarie had benefited from the treatment during the clinical trial and that she would continue to benefit in the future. However they recommended that the PCT support development of a national approach to the commissioning of enzyme replacement therapy.

Alison Millar: “It would be desirable for there to be national guidance on enzyme replacement therapy to avoid the problems of this case happening to other families. This is a good example of the need for some health policy decisions to be taken at a national level and an illustration of the current debate about which level, national, regional or local, medical treatment should be funded at.”

For more information please contact Alison Millar on 020 7650 1241.

On 28th October, the day after the decision was made on Lisamarie's treatment funding, the Department of Health issued a notice that laid out the plans for 'National designation and funding of the service for patients with lysosomal storage disorders':

"For a period of two years, from April 2005 to March 2007, six centres will be nationally designated and funded by the Department of Health, under the auspices of the National Specialised Commissioning Advisory Group (NSCAG), to provide a service for patients with lysosomal storage disorders (LSDs).  The service will include diagnostic, assessment AND treatment services. This means that the cost of drug treatments, including enzyme replacement therapies (ERTs), will be funded on a national basis.  The six NHS Trusts concerned are: Great Ormond Street, Manchester Children's, the Royal Free, Addenbrooke’s, University College and Hope hospitals."

Information was correct at time of publishing. See terms and conditions for further details.

Information was correct at time of publishing. See terms and conditions for further details.

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