A new appraisal system for medicines to treat rare conditions will be developed in Wales, Health Minister Mark Drakeford announced today (Wednesday 30 April).
The move follows a review of the All Wales Medicines Strategy Group (AWMSG) appraisal process for these medicines, which was carried out last year. The review, commissioned by the Health Minister, recommended a new system should be designed to address the specific challenges of appraising these medicines, which are known as orphan and ultra-orphan medicines.
The Minister also commissioned a review of the process by which patients in Wales apply to access treatments which are not routinely available on the NHS in Wales.
It found the Individual Patient Funding Request (IPFR) process, which allows clinicians to apply for treatments not approved for use by AWMSG or the National Institute for Health and Care Excellence (NICE), supports rational, evidence-based decision-making for treatments not routinely available in Wales.
The IPFR review made a number of recommendations to strengthen the process and ensure it is consistent and transparent. A public consultation on the IPFR report will take place over the next eight weeks.
Health Minister Mark Drakeford said: “Demand for healthcare continues to increase and the emergence of new and very often high-cost medicines places further pressures on our finite resources. The challenge is to make sure we only invest in medicines where the proven benefit is in balance with the cost, thereby ensuring we do not invest in medicines not proven to deliver high-value outcomes.
“To deliver this evidence-based approach, we invest substantially in our own medicines appraisal process, which is undertaken by AWMSG. Its work complements that of NICE and has allowed us to approve several medicines in Wales before they were available in England.
“I have asked AWMSG to develop and implement a new system for the identification, appraisal and monitoring of orphan medicines, the aim being to ensure that patients with rare diseases have fair and equitable access to appropriate, evidence-based treatments.
“I am also starting an eight-week consultation on changes to IPFR process, designed to strengthen the current process and ensure the process is applied consistently across Wales.”
