HTA

HTA

  • As expected, both Orkambi and Kalydeco (for the treatment of children with CF aged 2 years and older) will be subject to a full review by the National Centre for Pharma Economics (NCPE) in Ireland. This is normal for such drugs. This process includes a Health Technology Assessment (HTA) that may take up to 90 working days. We will let you know the outcome of these two reviews as soon as we hear.

    We thank  everyone who responded to our recent survey – we received almost 500 responses which is an indication in itself how important this issue is  to our members. CFI is currently finalising our report which will include information drawn from this survey.

    In the meantime, Minister for Health, Leo Varadkar TD has agreed to meet with Cystic Fibrosis Ireland following our request for a meeting before Christmas. This will take place before the end of January. CFI welcomes this opportunity to discuss with the Minister issues such as access to new and innovative therapies as well as other key CF related issues including staffing levels in hospitals and investment in key CF centres.

    Many thanks to all our members for their support to date.

    Philip Watt

    CEO Cystic Fibrosis Ireland

  • Extension of Kalydeco (Ivacaftor) Update

    CFI have been informed that some news about the extension of Kalydeco (Ivacaftor), which is currently undergoing a Health Technology Assessment (HTA), is due to be posted on the website of the NCPE (National Centre for Pharmacoeconomics) around the 12 October or shortly after. The extension application includes commencing treatment of those already on Kalydeco to 2 years and over (children with CF who have the G551D gene alteration are currently 6 years old when treatment commences). The extension also covers other class 111 gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R, as listed on the NCPE website.

    Please note that this news item should not be confused with the Orkambi negotiations. These negotiations are still on going and we are at an important stage.

  • Extension of Kalydeco (Ivacaftor): Update from NCPE

    Today (12 October), the National Centre for Pharmacoeconomics (NCPE) have issued their recommendation following the Health Technology Assessment (HTA) of Kalydeco for children aged 2- 5years. Kalydeco for children aged 2–5 year olds* has not been recommended for reimbursement at the submitted price. (Note: this is not related to the forthcoming Orkambi decision)

    CFI notes that the NCPE ruling leaves a door open to further price negotiations, but the decision will be very disappointing and frustrating for the 18 young children with CF and their families, who could benefit greatly from access to this ground-breaking therapy.

    Philip Watt, CEO of Cystic Fibrosis Ireland says “Accessing this life-changing treatment is essential as eligible patients stand to benefit so much at such a crucial time in their development, fighting a progressive, life-limiting disease. We will be urging the government to enter into price negotiations as soon as possible. We would also urge the pharma company, Vertex, to reduce the price of this important drug during these negotiations”.

    The treatment, which is currently only available for those over six years old, has been shown to significantly increase lung function, slow the progression of lung disease and significantly reduce hospital admissions.

    Research published earlier this year in ‘The Lancet Respiratory Medicine’ journal suggested that pre-school children with one of the nine mutations (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R) could also benefit from improved pancreatic function, weight gain and movement of salt between cells.

     

    *This assessment refers to children aged 2-5years who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P,S549N or S549R.