Call for “miracle drug” for rare disorder to be made available in Wales

(Title Image: National Assembly of Wales)

The big health story today from the Senedd is a protest and debate on the proposed downgrading of the Royal Glamorgan Hospital’s A&E department – more on that tomorrow, though there’s a preview coming up.

In the meantime, here’s a summary of questions to the Health Minister, Vaughan Gething (Lab, Cardiff S. & Penarth).

Support for those with a rare metabolic disorder

Shadow Health Minister, Angela Burns AM (Con, Carms. W. & S. Pembs.) brought up phenylketonuria (PKU) a rare condition affecting 1-in-10,000 people which means they can’t process the amino acid phenylalanine which results in seizures, learning disabilities and skin problems. The main way to deal with it is strict diet control.

“….the National Society for Phenylketonuria has come up with a number of recommendations to help improve PKU sufferers’ lives, one of which is that all people with PKU should be followed up in an integrated, specialist, metabolic service led by an experienced physician and dietician. So, Minister, I wondered if you would give some consideration to adopting this recommendation because it….has long-term and absolutely devastating effects on their lives if they do not get the balance of this diet right.”
– Shadow Health Minister, Angela Burns AM

Angela Burns mentioned a new drug called Kuvan which the UK’s NICE body (which approves the use of new drugs) has been considering for 12 years; while costly, it could make a dramatic difference to those with PKU.

The Minister was happy to discuss the matter with the PKU society further. There is also an issue around early milk supply as people with PKU often need specialist milk. Manufacturers can submit to the All Wales Medicines Strategy Group for appraisal, but he couldn’t make any promises on outcomes.

“No magic figure” on the necessary number of A&E departments

Pre-empting the later debate, Leanne Wood AM (Plaid, Rhondda) asked whether it remained Welsh Government policy to concentrate A&E departments on fewer sites? Also, did the Minister agree that doctors should decide the future of A&E departments, not politicians?

The Minister told AMs there was no “magic figure” for how many emergency departments there should be.

“Well, the decision made about the future of the Royal Glamorgan Hospital….is one where the health board have a responsibility to make a choice. They do need to listen to and engage with their medical workforce, to understand what doctors are saying about the safety of that service. That’s a short-term challenge and a longer-term one. But they also, of course, have a responsibility to listen to the public.”
– Health Minister, Vaughan Gething

When Leanne Wood revealed that a consultant’s meeting at the hospital earlier today backed retention of A&E services, the Minister stressed that the problem everyone faces is that if the right number of consultants can’t be recruited then services can’t be retained as they may become unsafe.

Stigma remains on mental health

Caroline Jones AM (BXP, South Wales West) said that despite calls to talk more about mental health, Wales lags behind on measures of wellbeing. While AMs themselves have talked about their own mental health issues, this positive attitude didn’t stretch to the public sector as a whole; she mentioned a constituent who was threatened with eviction due to cleanliness of his home despite serious mental health problems.

The Minister believes campaigns like Time to Change have made a difference, but he believed there was growing awareness within local authorities and housing associations about the stress mental health problems cause tenants, the homeless and their own staff.

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