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Appraisal process for medicines developed for rare diseases

AWMSG reviewed its process for appraising orphan and ultra-orphan medicines and medicines developed specifically for rare diseases, in July 2018. The process was updated in July 2019 to implement the recommendations from the review.

The appraisal process for medicines for a rare disease is aligned with AWMSG’s process for all other medicines. Applicant companies should indicate in their submission to AWTTC if they consider a medicine to be a medicine for a rare disease. The AWMSG secretariat assessment report (ASAR) will state whether the medicine should be considered as a medicine for a rare disease, and the company has the opportunity to respond to this view in the company’s response to the ASAR.

AWMSG members will be reminded of the broader considerations they should take into account when appraising a medicine for a rare disease.

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 may be held if a medicine for a rare disease receives a negative recommendation from the New Medicines Group (NMG), or if a positive recommendation from the NMG is followed by a negative recommendation from AWMSG. 

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