Tuesday, 26 March 2019

Ceisteanna (673)

Jack Chambers


673. Deputy Jack Chambers asked the Minister for Health his plans to ensure the European Medicines Agency progresses its assessment of authorising a particular drug (details supplied) for persons under 18 years of age as quickly as possible; and if he will make a statement on the matter. [13082/19]

Amharc ar fhreagra

Freagraí scríofa (Ceist ar Health)

Kalydeco is the brand name of the medicine ivacaftor which is used to treat certain forms of cystic fibrosis caused by specific mutations of the gene for a protein called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR).

Kalydeco is authorised as a centrally authorised product by the European Medicines Agency on behalf of the European Commission. The marketing authorisation details the licensed indications for Kalydeco. It is currently licensed for patients under 18 years of age with certain forms of cystic fibrosis.

Kalydeco granules are licensed for the treatment of children with cystic fibrosis aged 12 months and older and weighing 7 kg to less than 25 kg who have one of nine mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

Kalydeco tablets are licensed to treat cystic fibrosis in patients aged 6 years and older, and weighing 25 kg or more, and who have one of the nine mutations in CFTR gene noted above. They are also licensed to treat patients with cystic fibrosis aged 18 years and above who have the R117H mutation in the CFTR gene and in a combination regimen with a medicine containing tezacaftor 100 mg/ivacaftor 150 mg for the treatment of patients with cystic fibrosis aged 12 years and older who have a F508del mutation in the CFTR gene.

As part of the marketing authorisation application process to place a medicine on the market, the applicant company must provide evidence that the medicine adheres to clear and predefined standards of quality, safety and efficacy, relevant to its proposed therapeutic use. This takes the form of a dossier including details of all the trials and studies undertaken of the active substance and the final pharmaceutical product, including preclinical studies, clinical trials and manufacturing and analysis data. The format of this dossier is set out in Directive 2001/83/EC and various EU guidelines.

A marketing authorisation (MA) is granted on the basis of a favourable benefit versus risk balance for specific therapeutic indication(s) having regard to the quality, safety and efficacy of the product for the proposed conditions of use. The final wording of the Summary of Product Characteristics (SmPC) and Patient Information Leaflet (PIL) are approved based on the information submitted in this dossier. I, as Minister for Health, have no role or function in the marketing authorisation process. It would be inappropriate for me to attempt to intervene in the authorisation process or to try to influence the EMA.

Once an MA has been granted, any changes (e.g. new indications, updated safety information, pharmaceutical changes) require approval by the competent authority, in this case by the EMA on behalf of the European Commission, on foot of a formal application to vary the authorisation and the submission of relevant supporting data. In order to further update the SmPC the MAH would need to carry out further clinical trials to support this variation. It is up to the MAH to make an application to the EMA to vary the licensed indications for this medicine.