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    • #17995
      Jenny Livingston
      Keymaster

      I came across the following post on Instagram today. (See @cysticfibrosisnz for full post and context)

      My first thought was, of course, how amazing this is! However, while we celebrate and sing praises for Trikafta, I think it’s important to remember that it is not the be-all-end-all of cystic fibrosis treatment. There are still major roadblocks regarding accessibility. There are people with genetic mutations not treatable by Trikafta. The side effects can be brutal and intolerable for some, even outweighing the benefit. There are also those who, despite the data seen above, simply do not receive much benefit from the drug.

      So while this is definitely a reason to celebrate, let us also remember that we are not finished. There is still a lot of work to be done to ensure that every person with CF has access to highly effective therapies with fewer side effects. Fundraising, research, community involvement, and awareness are all still critical in this journey.

      What are your thoughts on the data shared here? Did you find any of it surprising?

    • #18008
      Tim Blowfield
      Participant

      Trikafta (TK) – great results for those who can get it. But very sad and depressing to those who can’t, those who have one of the 120 mutations that Vertex has shown should respond. In Australia TK is registered for only those with the F508del mutation. It  seems Vertex did not ask the Australian Therapeutic Goods Administration to include the other 120 unless they are heterozygous paired with the F508del. And the TGA has registered it in such a way that our highly trained and experienced CF Physicians cannot even try it in rare mutations and even to use it in pwCf with 508 must get permission from an anonymous bureaucrat in the Australian Department of Health. Recently the PBAC (Pharmaceutical Benefits Committee) did recommend TK be included for Pharmaceutical Benefits. That will make it affordable.

      Petititions for Australians to sign

      To all Australians: Jo Armstrong, CEO of Cystic Fibrosis Australia has instituted a Petition to the Australian Parliament to expedite the inclusion of TK in the PBS. All Australians can sign the petition by going to https://www.aph.gov.au/e-petitions/petition/EN3915.  A second petition related to Cf is EN3885 asking for pwCF with Diabetes be availed access to the National Diabetes Scheme (NDSS).

      I am instituting another petition EN3948 which will ask that TK be available for rare mutations and that our CF Physicians be the persons who determine whether it be appropriate.

      • #18024
        Jenny Livingston
        Keymaster

        Tim, it is indeed frustrating and heartbreaking to see many still struggling to gain access to this drug. Thank you for sharing the information regarding these petitions.

      • #18025
        Jenny Livingston
        Keymaster

        Tim, it is indeed frustrating and heartbreaking to see many still struggling to gain access to this drug. Thank you for sharing the information regarding these petitions.

    • #18039
      Tim Blowfield
      Participant

      Another e-petitions for Australians to sign.

      Go to https://www.aph.gov.au/e-petitions/petition/EN3948

      Petition number:  EN3948

      • Reason: Cystic Fibrosis (CF), the most common life limiting genetic disability, is caused by mutations of the CFTR gene. Some 2500 known mutations are known to this large gene, 90% having one called F508del. Mutations cause defects in a protein that controls the anions Chloride (and less so Bicarbonate) being pumped out of cells. In the lungs, gut and other mucous secreting cells this leads to thick mucous and the symptoms of CF. Recent developments and research has developed a group of drugs known as modulators that improve the excretion of chloride leading to more normal mucous and improved lung function, greatly improving the well-being and lives of the pwCF. Unfortunately these modulators of which Trikafta, the most recent, has just been recommended by the PBAC, have only been registered for use in only persons with one or just a very few mutations. Trikafta only for persons with the F508del mutation. This is because large double blinded trials only included that mutation. Vertex’s own laboratory research has shown there are 120 mutations that should respond and the US Prescribing Information includes these in their list of mutations for which the drugs can be used. With these rare mutations, such as the one person with the mutations R75Q and G542X, large double blinded trials are impossible.
      • Request: We therefore ask the House to make these modulators available for all persons with CF whom reasonable evidence, as judged by expert CF Physicians, exists that they should respond.
    • #18046
      Paul met Debbie
      Participant

      Way to go, Tim. If I were Australian I would sign. Being Dutch, I will sign it in my heart.

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