Whatever the rare disease, it cannot be right that families are spending their time and energy fighting for treatments when they should be spending precious time with their children. I congratulate my hon. Friend Liz Twist on securing this debate, and I echo the concerns about Kuvan, particularly the recommendation for its use only by children. One of my constituents whose daughter is living with PKU raised very legitimate concerns with me about the detrimental impact this could have on the mental health of children and teenagers whose lives are transformed by this drug, but know the clock is ticking towards it being taken away from them as they turn 18.
One of the themes underpinning the UK Rare Diseases Framework is patient voice, which the Department for Health and Social Care says is essential to its implementation, and I could not agree more. However, we must understand that there is a long way to go before families coping with the impact of rare diseases truly feel valued by the system. Gail and Matthew Rich from my constituency fought a long campaign that I supported to access Brineura on the NHS for their daughters Nicole and Jessica, who have CLN2 Batten Disease. Ahead of this debate, Gail told me:
“Patients and parents are not included or listened to enough, and children are suffering as a result of following an antiquated system which seems all about number-crunching and balancing figures rather than what is essential and morally acceptable.”
Those affected by rare diseases too often feel that they are struggling alone, so as we move from publication of the UK Rare Diseases Framework to the development of action plans, I urge the Minister to ensure that patient voices are truly heard, and positive outcomes reflect patient needs, not just those of the healthcare system.