The International Rare Disease Day is being held on 28 February to raise awareness of the over 7,000 rare diseases that impact over 300 million people globally.
The All Wales Medicines Strategy Group (AWMSG) and the All Wales Therapeutics and Toxicology Centre (AWTTC) work to ensure that people with a rare disease living in Wales have the same opportunity to access medicines as other patients.
AWMSG’s appraisal process allows an extra step for bringing together patients, carers, patient support groups and clinicians to discuss how a medicine may affect a person’s quality of life and discuss what impact this may have on the patient’s family or carers.
The rarity of a disease is likely to affect the amount of evidence available on which a decision can be made and the cost of the medicine is usually very high: there is much greater uncertainty.
Therefore, it is very important to capture as much additional information from the patient and clinician perspective as possible. In these circumstances, the manufacturer of the medicine is given the opportunity to ask for a meeting of the Clinician and Patient Involvement Group and the report of this meeting will help inform AWMSG’s wider judgement of a medicine’s value.
You can read more from this website on the AWMSG medicine appraisal process and why the views of patients are an important part of this process.