Fighting for global access to equitable CF healthcare, treatment
Potentially lifesaving therapies should be available to patients in all countries
Most parents of children with rare diseases spend their lives trying to ensure that their child outlives them. That usually means taking part in fundraising to support the development of new treatments. But even if a child lives long enough to see a therapy come to fruition, this can be followed by a fight to gain access to it.
Younger children with cystic fibrosis (CF) in the United States, such as my daughter, Claire, are living in a revolutionary period of treatment of the disease. But I know that circumstances here in the U.S. are different from other parts of the world.
There are days I take for granted how privileged we are to live in a country with excellent CF care. The U.S. has a robust system of cystic fibrosis care centers, which are a cornerstone of efficient and effective treatment of the disease. Our government was the first to approve modulator therapies for CF.
The Cystic Fibrosis Foundation (CFF) Patient Registry tracks crucial data and information and influences disease outcomes, hopefully serving as a model for other disease communities. By all accounts, the U.S. and the CFF are leaders in the global fight against cystic fibrosis.
However, I am painfully aware that others around the world living with cystic fibrosis are not as fortunate. These people face an uphill battle to secure adequate healthcare and access to costly treatments. In countries with universal healthcare, such as the United Kingdom and Canada, drug access can be predicated on a cost-benefit analysis. Typically, a regulatory body evaluates both the efficacy of the drug and its cost to the nation’s socialized healthcare system.
How does one put a cost on human life?
There are few worse things than knowing that a drug exists that could save your child’s life, but being unable to receive it because of where you live. Unfortunately, that is the reality for most of the world’s cystic fibrosis population.
According to an article published in the Journal of Cystic Fibrosis in 2022, only an estimated 12% of the global cystic fibrosis population is receiving triple combination therapy.
Recently, CF parents in the U.K. flooded social media after the National Institute for Health and Care Excellence issued guidance about three CF modulator therapies, noting that the drugs may be too costly to fund for newly eligible patients. While the guidance was a draft, and not a final decision, parents are concerned that their children’s fates could be in jeopardy.
As the Cystic Fibrosis Trust notes, this “appraisal process does not affect anyone who is currently taking a modulator treatment.” However, depending on the outcome, many younger or future patients could be unable to receive these drugs, which have the potential to slow or halt disease progression.
It is a stark reminder that health inequities exist around the world.
We’re all in this together
I didn’t know how to respond when I learned of this devastating news. I feel for these parents because I know what it’s like to dream about my child receiving life-changing medications.
It’s easy to get caught up in the stress of CF care and forget how truly lucky we are. Our family has the security of knowing that our daughter has access to the most advanced drugs. I can’t help but hold guilt, knowing that there are others who would do anything to be in our position.
Today is International Universal Health Coverage Day. The goal of the event is to raise awareness and ensure that people worldwide have equitable access to essential healthcare services without financial hardship. The current cost-benefit appraisal process in the U.K. highlights the importance of fighting for equitable care. Everyone with cystic fibrosis deserves the chance to live life to the fullest.
Note: Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Cystic Fibrosis News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to cystic fibrosis.
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Mark Tremblay
Jennifer, Wonderful to meet you. Your article does a great job summarizing the challenges the rest of the world and particularly third world countries face in providing effective CF care. You may not know this, but providing modulators which cost $270 to $300 k annually (bulk Medicaid price) to the eligible US CF population over a five-year period is approximately equal to the Federal share of National Child Health Insurance Program (CHIP) which insures 11 million children. Also, I'm not sure if you have noticed but the US is the most prolific producer of Medical and pharmaceutical blockbusters in the world over the last 20 years because of its current fiscal incentive structure. To put that in perspective, to provide all the eligible CF patients in the world (145,800) with modulators over a five-year period would cost approximately $219 B which is a little less than the all-government annual NYS budget and three times as much as the entire Medicaid annual budget which insures nearly 7 M people. I know this because I spent my entire 27 year career working in NYS Governor's Division of Budget and Department of Health and since June 2020 I have been the President and founder of CF Vests 4 Life (and CF Vests for Life) which supports CF doctors in developing countries to provide cost effective evidenced based care to nearly 350 patients and families. If you want ot know how to help check us out at: https://www.cfv4l.org/