Cystic Fibrosis Trust Launches Survey to Evaluate Orkambi’s Entrance into UK Health System

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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The British non-profit organization Cystic Fibrosis Trust has launched a survey entitled “Cystic Fibrosis & Treatment – What Matters to You?” for patients to share their thoughts and needs regarding treatment for cystic fibrosis (CF). With the motto “Use your voice to support access to transformational treatments,” the organization seeks to gather people’s opinions and advocate for improved access to cutting-edge therapies, such as Vertex’s breakthrough combination therapy Orkambi.

Orkambi, an FDA-approved drug combination developed by Vertex that is set to enter the U.K.’s markets, is raising questions regarding cost and patient access as it related to the country’s public health system. Orkambi is a novel therapy and one of the only ones that targets specific CF mutations by correcting the basic underlying genetic defect in about half of the CF patient population. The therapy was approved by the FDA on July 2, and is among the latest therapies recommended for approval by European regulators.

Once Orkambi is approved in Europe, it will become available in the U.K., at which point the National Health System (NHS) will have to decide if the medication will be reimbursed or not in each of the four U.K. regions. The Cystic Fibrosis Trust is determined to widen patients’ access to CF medications in order to improve their quality of life and life expectancy. Therefore, the organization believes that the survey will help patients gain quicker access to drugs such as Orkambi.

The information collected by the CF Trust survey will be provided to the National Institute for Health and Care Excellence (NICE), which is responsible for evaluating Orkambi both in terms of its clinical effectiveness and its cost effectiveness. “While the Trust has concerns about the NICE process for appraising drugs of this sort, it is vital that the voice of the CF community is heard loud and clear throughout, so please fill in the survey to have your say today,” explained the organization in a statement.

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The main purpose is to help NICE make a decision about the drug that is in line with patients’ needs, as the CF Trust is particularly concerned about the costs associated with the medication. “This process and the arrival of similar innovative therapies in the future will challenge the NHS to look at what ‘value for money’ really means in the context of CF care. The whole community must speak up and make sure these decisions are guided by the unique knowledge and experience of people living with CF,” the Trust added.

NICE has requested the help of the Cystic Fibrosis Trust in its assessment of Orkambi, which will begin in February of next year. In addition to participating in the survey, which is available here, U.K. cystic fibrosis patients can also support the Cystic Fibrosis Trust’s campaign by speaking directly to the organization by contacting [email protected].

The non-profit organization is determined to advocate for patients who suffer from CF, and has recently hosted the 2015 UK Cystic Fibrosis Conference (UKCFC), the United Kingdom’s most-anticipated annual conference for the cystic fibrosis community, which brings together renowned disease experts, researchers, health professionals, patients, and family members. This year, the event took place on Sept. 22-23 in Manchester, giving the CF community in the U.K. an opportunity to share and promote new insights into the latest CF research and advocacy updates in order to better understand, manage and treat the disease.