News & Events

What's on the Horizon for Orphan and Ultra-Orphan Conditions?

Last Reviewed 15/12/2015

By Emma Hughes

Last week our Development Officer in Wales, Emma, attended an engagement event with the Welsh Health Specialised Services Committee (WHSSC) as part of their horizon scanning for orphan and ultra-orphan medicines.

The first medicine to go through the All Wales Medicines Strategy Group process for appraisal was Kalydeco (ivacaftor) – a medicine used to treat cystic fibrosis for specific gene mutations. In light of the current capacity of the NICE highly specialised technologies (HST) process being around 3 appraisals per year, the importance of timeliness and equity in the consideration of access to treatments in Wales was highlighted throughout the discussions at the meeting.

NHS Wales and the NICE HST programme

In England, decisions from the NICE HST process are mandated, however, in Wales these decisions are not required to be automatically adopted. In recognition of this the Minister for Health and Social Services, Mark Drakeford has requested an inter-agency approach between the commissioner for Specialised Services in Wales and the All Wales Medicines Strategy Group (AWMSG). A procedure will be put in place that requires the production of an inter-agency briefing that will be provided to the Minister, who will then confirm whether the NICE HST decision will be adopted in NHS Wales.

It was highlighted that any information shared by NICE with NHS Wales will have all information on price redacted, whether this was commercial in confidence or not. It is therefore vital that manufacturers be encouraged to have discussions with both NICE and WHSSC in regards to price. They should also be talking to the All Wales Therapeutics and Toxicology Committee (AWTTC) in parallel with discussions at NICE so that when making an application to NICE HST there is no gap in uptake for Welsh patients when a medicine is approved.

Interim Cohort Commissioning – the “One Wales” Process

To combat the ongoing problem of medicines without a commissioning policy going through the Individual Patient Funding Requests (IPFR) route, without any prior engagement with the HTA process, there will be an interim cohort commissioning process. This is not meant to replace the formal HTA process - HTA will remain the gold standard but there will now be measures in place to make sure treatments do not fall through the cracks.

AWTTC envisage that the “One Wales” process will only be used very rarely and there would be an obligation on companies to engage with HTA within a given period.

The “One Wales” proposal was submitted to the Chief Executives of NHS Wales in November, where it was agreed in principle. However, there were two outstanding points, answers to which had to be re-submitted to the group in December.

What’s next?

Following the meeting of Chief Executives in December, if approved the process will be handed over to the AWMSG Steering Committee, which would be discussing it in February 2016.

Engagement and communication with stakeholders about this next interim cohort commissioning process will not take place until the Chief Executives of the Local Health Boards in Wales and the AWMSG Steering Committee have given the green light for its implementation.

One of the key issues arising from this meeting was the need to establish an Orphan and Ultra-Orphan Stakeholder Forum so that this important trilogue between Commissioners, AWTTC/AWMSG and Patient groups can be continued. We look forward to representing the patient perspective as part of these discussions moving forward.

Update: The policy for the One Wales Interim Commissioning Process is now available on the All Wales Therapeutics and Toxicology (AWTTC) website.

If you have any comments or questions about this work, please contact our Development Officer in Wales, Emma

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