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Anxiety associated with being denied necessary medication
My son Zack is currently on NG tube feeding in Temple Street hospital, Dublin, Ireland and will have a PEG inserted in September 2019 (this suffering would of course be unnecessary if the EMA allowed him access to Kalydeco as is the case in the US and Australia). Zack has the R117H gating gene alteration.
The European Medicines Agency (EMA) choose to deny access to Kalydeco for R117H children in Europe and treat these children with contempt. This lack of empathy and compassion for children with a chronic illness is shameful behaviour by the EMA. This assertion is based on below:
1. The R117H Kalydeco to children application was presented to the FDA back in 2014 and there was almost a unanimous decision with 13 subject matter experts voting yes and 2 voting no. Even back in 2014, the experts were able to understand that the risk of approving Kalydeco for kids is quite limited but the potential benefit is huge. Five years on and the significant amount of data that is now available on the benefit of Kalydeco to gating mutations such as R117H, the EMA still deny this life changing medicine to kids. This, despite the fact that approval is granted at 6 months in the US. It is approved at 18 years by the EMA.
2. The EMA is unique in that it believes Cystic Fibrosis behaves differently in adults and children for the R117H gene alteration even though there is no evidence to indicate this. The EMA are unable to provide a biologic plausibility whereby there would be a substantially different or opposite effect in younger kids to that experienced by adults. The EMA assertion that Kalydeco is only effective in R117H adults does not stand up. There are 2,000 + different gene alterations with Cystic Fibrosis. Why do the EMA believe that R117H is the only one that the disease is different between adults and children? There is no evidence to support this? The probability that the disease is different between adults and children is 0.0005% based on the figures above and if you take into account the testimonies of how the drug has worked for kids in the US, this probability is reduced to zero. Hence, as the EMA cannot prove that the drug behaves differently for this single alteration (as is currently the case) and there is clear evidence that the drug works for R117H children in the US, this is simply a case of age discrimination by denying access until 18 years.
3. There is clear evidence that Kalydeco works for R117H children in the US where it has been clearly demonstrated that Kalydeco benefits children when prescribed by increasing energy levels, improving digestive function and eliminating the constant stomach aches associated with CF. Children also begin to grow and gain weight when allowed access to Kalydeco with the most recent five-year study on Kalydeco showing significant improved nutritional status and weight gain. Kalydeco has tested safe for over 6 years now. The EMA choose to deny access in Europe and treat these children with contempt. This lack of empathy and compassion for children with a chronic illness is shameful behaviour by the EMA.
4. We do not get life changing medicines like Kalydeco very often and it is just beyond belief and incredulous that when we do get a drug as effective as Kalydeco for gating mutations we simultaneously get the human intervention of European regulators to block children’s access to the drug. The EMA are presiding over a situation that denies children access to a life changing medicine thus promoting unnecessary hospitalisations and suffering, permanent structural damage to the lungs and shortened life expectancies for these children.
5. The EMA are deciding that an improvement in cftr function for the G551D gating gene alteration to 48% is sufficient for Kalydeco to be approved at 6 months while at the same time saying that an improvement in cftr function for the R117H gating gene alteration to 38% is not sufficient. Do they not realise that as R117H is only 78% gating, Kalydeco cannot match the 48% cftr function of G551D as it is 100% gating? Kalydeco fixes the gating defect for both alterations in the same manner in terms of cftr function outcome. Who at the EMA is playing God by deciding that the kids with G551D can have early access to Kalydeco and live as close a normal life as possible to old age while the kids with R117H must accept shortened life expectancy and an early death sentence? The EMA are driving the development of a divide in care delivered to patients with CF, with one group of patients with gating mutations G551D having access to CFTR restorative treatments and another group R117H being denied such treatment due to their age. They should not be allowed do this.
6. It is important for the EMA to note that the ability to have to access to Kalydeco is a right to life issue. By approving the use of Kalydeco for those with the R117H mutation, you are upholding these rights. By enabling the ability to use Kalydeco for children with this mutation, before CF lung disease progresses and is outwardly visible, you are providing them with the right to live. Kalydeco has tested safe for over 6 years now. If the EMA believe Kalydeco is effective in adults, they must not deny it to children, when it can prevent their lungs from deteriorating as they age.
7. Kalydeco has the ability to reduce the Sweat Chloride levels of R117H children to a reading that is indicative of person who does not have Cystic fibrosis and every day that goes by where the EMA deny children access to this drug, the life expectancies of children are shortened as per direct relationship between sweat chloride levels and mortality. The EMA appear to accept this fact and do nothing about it.
https://www.ncbi.nlm.nih.gov/pubmed/29221674
8. There is clear evidence in the US that Kalydeco works for R117H children so why do the EMA believe it will not work in Europe until 18 years of age?
http://luckycfmom.blogspot.com/2014/10/the-kiwi-studyivacaftor-for-2-5-year.html
9. The FDA had an approval process in place back in 2014(for Kalydeco to R117H children) that was both fully transparent and allowed for a holistic approach with additional considerations for parental testimonies, CF subject matter testimonies, CF patient testimonies, in vitro data etc. The FDA noted that they were fully open to all evidence as part of the application process. Fifteen of the top subject matter experts in the US were asked to vote on the following:
Did the data support approval of ivacaftor oral tablets, 150 milligrams twice daily for the treatment of cystic fibrosis in patients age 6 and older who have the R117H mutation in the CFTR gene?
There was almost a unanimous decision here with 13 experts voting yes and 2 voting no. Even back in 2014, the experts were able to understand that the risk of approving Kalydeco for kids is quite limited but the potential benefit is huge. Five years on and the significant amount of data that is now available on the benefit of Kalydeco to gating mutations such as R117H, the EMA still deny this life changing medicine to kids. It is not good enough that the EMA do not have a holistic and transparent process and one that takes into account all available data including parental testimonies, CF subject matter testimonies, CF patient testimonies, in vitro data etc.?
In the CysticFibrosisnewstoday article “Accepting the bumps in the road” by Elizabeth Rogers, she notes that “Planning a full life around a chronic illness means accepting the bumps in the road. Learning to accept these bumps is a continuous process, but if I can do it, so can you”. This is fine when the bumps in the road are caused by environmental variables that are out of a person’s control. However, when the bumps in the road are caused by the human intervention of European Medical Regulators, this must never be tolerated. The EMA do no have the right to inflict unnecessary suffering on children with a chronic illness such as CF.
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1 Reply
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Kalydeco just got EU commission approval for the Cystic Fibrosis R117h gene alteration – children (please see below):
This is incredible news for my family and in particular my son Zack.
It is hard to find the words to express my gratitude for a decision such as this and my family wish to thank the EMA and EU Commission for the part they played in this approval.
This is great news not only for my son Zack but for all the other children in Europe who will benefit from this approval.
Zack now has the opportunity to gain access to Kalydeco as a child and this will allow him to manage the symptoms of CF.
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Not at all surprising. Vertex has shown that there are at least 120 mutations that should respond to Ivacaftor yet bureaucracies continue to deny PWCF even a trial.
Specialist should be allowed to try these drugs on any PWCF they believe that the drug should work and to continue if they do.
The Ivacaftor containing drugs have been shown to be remarkably safe and in the CF Clinic context are extremely well supervised,
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