NICE Appraisals: Rare Diseases Treatments

Part of Backbench Business - Services for People with Autismbackbench Business – in the House of Commons at 4:38 pm on 21st March 2019.

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Photo of Sharon Hodgson Sharon Hodgson Shadow Minister (Public Health) 4:38 pm, 21st March 2019


Behind profit forecasts are thousands of people and families who need access to life-saving drugs, and they simply cannot wait any longer. We must not put businesses before patients. Because of the NICE appraisal process, patients and their families are being left in an awful limbo. The processes can be long-winded, confusing and difficult to navigate. Some medicines can undergo multiple assessments while others are not assessed at all, and that creates an unpredictable and unattractive system. As a result, patients are left in the dark about when, or if, they will have access to innovative treatments for their conditions.

When a drug is being appraised, patients live in hope that this time it will be approved for use by NICE—as in Maryam’s case, which was described so powerfully by my hon. Friend Lyn Brown—but they are almost always left to feel disappointed and helpless. Patients and their families must be involved in the processes, and the processes must be transparent.

The wait for access to drugs is excruciating, especially when the drug is available in nearby countries, or even—as we have heard—in Scotland. Spinraza is available to patients in Scotland, but not to those in England. My hon. Friend the Member for West Ham spoke passionately on behalf of her constituents and their seven-month-old baby Maryam. This sounds blunt, but she is dying, because she has been denied access to medication that could extend and enhance her life.

The pain and anguish that the parents of a critically ill child must feel when they are told that there is medicine available that will help but it is not available for their child are unimaginable. Knowing that if your child lived a few hundred miles away, in Scotland or perhaps somewhere in Europe, the drug would be available is heartbreaking and infuriating. Patients in England should not be left behind. We should be working to find ways to get these medicines to the patients who need them, on the NHS.

I hope that the Minister will consider the motion seriously, for the sake of patients with rare diseases and their families. They cannot be left behind any longer: they must have access to these life-saving drugs now.