Does a drug or other product have to have a Clinical Trial in Ireland to be sold in Ireland?
No, approvals to sell medicines in Ireland (both the republic and Northern Ireland) are most often based on evidence gathered elsewhere.
How are drugs approved for sale?
Once there is scientific data to support its safe use, usually a drug manufacturer or an importer applies to the Irish Medicines Board, or in Northern Ireland to Medicines and Healthcare Products Regulatory Agency, (both are government agency who regulate medicines) for a “Product Authorisation” to place the product on the market.
If a drug trial elsewhere goes well, can we get access to the drug in Ireland?
Yes, if the company producing it, or an importer, successfully applies for a licence for the use of the drug in the treatment of Angelman Syndrome in Ireland for this application it will be available on prescription. If the particular drug is not licenced in Ireland for treatment of Angelman Syndrome, but it is already available for the treatment of other conditions, then being able to buy it in your pharmacy will depend on your doctor prescribing it “off Label”
A doctor will only prescribe “off label” if she considers based on scientific information it in the best interest of the patient and safe to do so. She will have to consider the unique medical history of the person and what is known about the drug and its effectiveness and safety. Information from web sites, anecdotes and news articles are not always of scientific value. Doctors prefer to rely on scientific results published in recognised peer reviewed journals.
“Off label” prescribing is where a doctor, based on practitioner experience, published studies, and/or findings presented at professional meetings, prescribes a medicine at a dose or for a condition not covered in the licence. The circumstances in which this is allowed are detailed in legislation and outlined on the websites of Irish Medicines Board (IMB) and for Northern Ireland the Medicines and Health Regulatory Agency (MHRA).
Are ASI aware of any drugs or medicines that are not indicated for use as a therapy for Angelman Syndrome but are helpful?
No
For this reason if any available drug has a therapeutic value for Angelman Syndrome it is very important for us all that the evidence is presented formally to the scientific and medical community and published and that available data are submitted to the relevant regulatory bodies.
