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Appraisal policy for medicines developed to treat very rare diseases

AWMSG reviewed its health technology assessment methods and processes for appraising medicines developed to treat very rare diseases. In May 2023 we implemented the recommendations from the review.

Before a company submits a medicine for assessment under the very rare diseases policy, it requires confirmation from the All Wales Therapeutics and Toxicology Centre (AWTTC) that the medicine is eligible to be appraised under the policy. The applicant company should first complete the AWMSG very rare diseases form and give supporting evidence for each of the four eligibility criteria.

If all four criteria are not clearly met, AWTTC will convene AWMSG’s Medicines for Very Rare Diseases Panel. AWTTC will inform the applicant company of the Panel’s decision within five working days, in a formal letter stating the reason for the decision and identifying which criteria were not met. The applicant company can challenge the decision of the Medicines for Very Rare Diseases Panel, if they think that the eligibility criteria have not been applied appropriately.

The appraisal process for a medicine to treat a very rare disease aligns with AWMSG’s appraisal process for all other medicines. However, there is an additional stage to further assess the benefits of the medicine from the perspective of clinicians and patients, through the Clinician and Patient Involvement Group (CAPIG). A CAPIG meeting might be held if a medicine to treat a very rare disease receives a negative recommendation from the New Medicines Group (NMG), or if it receives a positive recommendation from the NMG and a negative recommendation from the AWMSG.



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