13 March 2013

What we spoke to Congress about...

I will post one more time about March On The Hill 2013 before I move on to sharing about my time at the CFF's VLC 2013 Conference within the next few days.  I wanted to document some of the specific things I learned about the Cystic Fibrosis Foundation with regards our public policy agenda and our message to Congress this year.

March On The Hill is part of the Cystic Fibrosis Foundation's Advocacy Program.  The goals of the CFF Advocacy program are to advance research, to promote access to care and to raise awareness of the disease.  Most of the program is made up of volunteers who offer to stay informed via email about changes in the CFF's public policy agenda and other volunteers who have indicated they want to stay in touch with their local government to communicate our needs to our lawmakers. (There is a video of how you can be CFF Advocate on the bottom of this post.)

Although last year, advocacy at the CFF involved pushing for the passage of the EXPERRT Act, this year's efforts were not based on a bill that needs passage.  This year, we are just asking our representatives to protect funding of several vital federal programs:

If you've heard of "sequestration" on the topic of the federal government, let me explain how it's going to affect Cystic Fibrosis.  Sequestration is a series of automatic, across the board cuts to government agencies in an effort to minimize the growth of the national debt.  Unfortunately, those federal programs in health care have received an 5% cut in funding.  That's a pretty significant cut to some really important programs that could impact Cystic Fibrosis research.

The CF Foundation works very closely with the National Institutes of Health (NIH) and the Food and Drug Administration (FDA).  Both of these agencies are now having to figure out how to cut 5% out of their budgets.  So, we are asking our Congressmen and Senators to please help protect funding of the NIH and FDA.

Let me explain how important the NIH and FDA are to our efforts to a cure.

The NIH is great at supporting basic human research.  The way the NIH is set up, they give small grants to up and coming researchers (many are PhD students or professors at universities across the nation) and employ researchers at their own laboratories in Bethesda, Maryland.  These researchers, in a variety of health care fields, do basic research on how the human body works, things like, "how does the sodium chloride channel work" or "how does infection affect the immune system."  This type of research is that which we can all benefit from.  But it's particularly of interest to the Cystic Fibrosis Foundation because we know we must truly understand what is going wrong in the body, with regard to CF, before we can fix it.

For example, the basic science behind Cystic Fibrosis (research on the human genome) was funded in part by the federal government through the National Institutes of Health.

When something important in basic human research has been discovered to affect those with the disease of Cystic Fibrosis, this is when the Cystic Fibrosis Foundation comes in.  The CF Foundation, using private funds, will often partner with those scientists and their basic human research to further their studies.  Research for a cure for Cystic Fibrosis is jointly funded by the National Institutes of Health (NIH) and private funds.  The CF Foundation does not receive or accept any government funds but does use already funded projects at the NIH to help further find a cure.

This is what happened with the development with the medication Kalydeco.  Once the CF Foundation better understood the basic human research behind the sodium chloride channel, the CFF took private money they had raised and helped pay for the furtherance of that research.  So, if we knew what went wrong in the sodium chloride channel, the CFF offered the financing to have the researchers now move forward to see how we could fix the sodium chloride channel.

At this stage in research, the CFF has now taken private money to further the basic human research already completed by the NIH.  This is why the NIH is so important to the CFF.  The CFF is directly benefiting from research at the NIH.  If the NIH has to cut down on staff or research grants or programs, it will slow down the CFF's efforts to further research in the world of CF.

Once basic human research from the NIH is furthered with private funds from the CFF and research has now been discovered where a drug could be beneficial, this is when industry/the pharmaceutical  pharmaceutical companies will get involved.  But pharmaceutical companies don't want to waste their time or money on discovering a drug.  They want to be involved when they smell success on the potential of marking a very real opportunity for a drug to fix the human body.

This is the point in which Vertex became involved in the process of discovering Kalydeco.  We need the pharmaceutical companies.  But it is difficult to get them involved in our basic research, which is why we need the NIH.  Once industry is involved in the drug-development process, then, we now have three parties involved: the NIH, the CFF and now the drug companies.

Once the drug companies have gotten the drug to place where they feel the drug is worthy to be marketed, the drug companies, along with the CF Foundation, will submit the drug for approval from the FDA.  This is the second federal government program the CFF works very closely with.  The CFF follows the drug throughout the FDA approval process.  In fact, CFF has a great relationship with the FDA.  The FDA knows we submit only those drugs that are well-researched data-driven.

Once the FDA approves a drug, it can then be placed on the market for patients to use.  This process was expedited recently for the CF drug Kalydeco (the third fastest drug to pass through the approval process with the FDA due to effeciency of data and the need for the drug to save lives).  Slowing down the FDA process could have dire consequences for those with CF.  But if the FDA continues to have to absorb large cuts to its funding, we may see exactly that happen.

So, as you can see, this year we are asking our Congressmen and Senators to protect NIH and FDA cuts to help them to continue to be efficient with regards to Cystic Fibrosis research.

Let me put this in another smaller chart form.  This is basically a repeat of what I just wrote:

NIH (federal funding=research) works with the CFF (private funding=research). Together, that collaboration offers new research. ---->  Once enough research has been done, industry/private sector (aka pharmaceutical companies) get involved, bringing in more financial backing.  ---> Once a drug has been shown to work in the lab, the FDA gets involved to oversee the trial process and approve the drug for human use.
The CFF is an example of excellent and effective drug development and care.  I am very proud of the way the Cystic Fibrosis Foundation uses its money as well as the already present research out there.  I am blown away by the efficiency of money and research to find a cure.  As a mother of a child with CF who regularly solicits private giving to help my child benefit from a cure, I am very proud to be a part of a process that IS working and is working well.  Hearing more about how we help in the development of CF drugs makes me very confident in the Cystic Fibrosis Foundation and their efforts to get a cure for all those with CF.  It's working - the partnership with the NIH, the FDA, the Foundation and the drug comanpies.  I hope it will continue to work smoothly.  Protecting the NIH and FDA's budgets is crucial to our need for a cure.

The other thing we discussed as a part of our public policy agenda, something that will become more relevant on a state government level than on a national level (but one we still mentioned to our federal representatives) is the importance of "Cystic Fibrosis Care Centers."

Cystic Fibrosis, being a rare chronic disease, needs data-driven highly specialized specialized care.  This is why we have "CF Centers."  CF Care Centers are those multi-faceted hospital departments and clinics that treat only Cystic Fibrosis patients.  Bennett visits a CF Care Center every three months.  His CF Care Center doctors (a pulmonologist, a GI doc, a ENT doc, etc.) are specifically trained in his disease.

As Bob Beall recently said, "We are the poster child for personalized medicine."  We have learned that children and adults with CF need care that can specifically address their disease on many levels (remember, CF affects the entire body, not just the lungs and digestive system).  CF Care Centers effectively treat the patient and use data to do that.  CF Care Centers are accredited by the Cystic Fibrosis Foundation and are held to very high standards in the way of CF Care.

However, with the new laws from ObamaCare/the Affordable Care Act, we may see children and adults with CF having less access to this type of care - as efforts to minimize costs are made.  This will be very determintal to those with Cystic Fibrosis.  We want our governmental representatives to know how important our CF Care Centers are to those with CF and how we must protect access to these centers for those with the disease, despite upcoming changes in our healthcare system.

That's the basic gist of what we spoke to our represetnatives about.  I found learning and later articulating this information to be very helpful for me as a mother and advocate for my son.  I have a very strong desire to keep a pulse on what the CF Foundation is doing and making sure they are using our hard-earned money well.  I am very pleased to hear they are not only doing that but they are advocating well on our behalf in other areas, as well.

Before I end this point, let me add that we need many more people to advocate for those with Cystic Fibrosis.  Here's a video of what Advocates do and, if you're interested, how you can sign up to help:


  1. Thank you so much for posting this. I was curious about what exactly a CF advocate would be asking for from congress and this helped me to understand! Very interesting!

    1. You are so welcome, Amy! Glad this is helpful and that you found it interesting! I did as well so I thought others might appreciate it! :) Makes me happy you do too! :) Hope E is doing better!!


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