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Treatment for Morquio syndrome

Volume 594: debated on Wednesday 25 March 2015

The Petition of residents of the Alyn and Deeside constituency,

Declares that Morquio syndrome (also known as MPS IV) is a rare genetic disease; further that Elosulfase, a drug to treat the syndrome, has recently been approved by the European Medicines Agency following positive results in the final stages of clinical trials; further that the drug (in the form of weekly enzyme replacement treatment) improves sufferers’ energy levels and stamina and therefore increases their independence, further that the effects of the drug are hugely beneficial not only to the individuals who have Morquio syndrome but also to their families; further that funding for the newly licensed enzyme replacement therapy to treat Morquio syndrome is unlikely to be approved due to cost saving; and further that the Petitioners believe that the consequences of patients, including children, not receiving this drug are unbearable.

The Petitioners therefore request that the House of Commons urges the Department of Health to ensure that individuals who have Morquio syndrome are given free access at home to enzyme replacement therapy for the treatment of the syndrome.

And the Petitioners remain, etc. [Official Report, 4 March 2015; Vol.593, c.1045.]


Observations from the Secretary of State for Health:

Decisions about funding for new treatments and drugs in England for rarer conditions like Elosulfase alpha (Vimizim) for the treatment of Morquio syndrome, are made by NHS England in accordance with its statutory duties under the Health and Social Care Act 2012.

NHS England is currently consulting on how it will decide which specialised services and treatments to prioritise. The consultation, Investing in specialised commissioning, can be accessed at: consultation/3a67882b.

Once this consultation concludes on 27 April 2015, NHS England will make a decision as quickly as possible on whether to provide funding for Elosulfase alpha. It has agreed to make this decision prior to the publication of NICE guidance.

NHS England also needs to hold consultations on the specific clinical policy for this individual drug. While this would normally not be started until the wider framework for commissioning had been consulted on and agreed, in the case of Elosulfase alpha it has agreed to run this specific consultation in parallel with the consultation on the wider decision making framework, in order to accelerate this process.

NHS England has also agreed that this consultation on the clinical policy for Elosulfase alpha will run for the shortest possible period of time that is legally permissible: 30 days.

This means that all of the consultations required for a decision on this issue should be concluded by the end of April. However, NHS England will obviously need time to consider all of the responses received before it decides whether to fund this drug––we have urged them to do this as quickly as possible.

In light of these developments, we are therefore hopeful that on this accelerated timetable the patients currently receiving Elosulfase alpha have every chance of continuity of treatment, provided the supplier of the drugs (BioMarin) can agree to maintain supply through to NHS England’s decision. NHS England is now in active discussion with BioMarin over the company’s responsibilities for the continuing supply of drug to the patients it enrolled in clinical trials.

In the event that NHS England decides that Elosulfase alpha remains ‘not routinely commissioned’ when it makes a decision (after the end of April), NHS England is still required to fund treatments that have been evaluated and recommended by the NICE Highly Specialised Technologies programme within three months of the guidance publication. Final guidance on Elosulfase alpha is expected in October 2015.

The Government take the issue of ensuring rapid access to innovative therapies very seriously, which is why they have launched an ‘Innovative Medicines and Medical Technology Review’ of the pathways for the development, assessment, and adoption of innovative medicines and medical technology. This review will consider how to speed up access for NHS patients to cost-effective new diagnostics, medicines and devices.

The review will examine the pathway from ‘first in human’ trials, through licensing and health technology appraisal, to commissioning and clinical practice. It will set out both short and long-term options for action by Government and relevant bodies, including NICE, the Medicines and Healthcare Products Regulatory Agency and NHS England, and mark a major contribution to the policy debate.