Come to the Table

I hate politics.  I really do.  I haven't taken a political science course since Freshman year.  All the partisan bickering and lack of action on important issues is annoying and frustrating.  It often seems like campaigning for the next election is all many of our politicians care about, and I can’t stand to watch live television during an election cycle because it makes me want to throw things at my TV…

So why in the world would I travel from northern Idaho to Washington D.C., suit up, and walk 8 miles in heels navigating the maze of offices on Capitol Hill?  Why do I feel absolutely compelled to inject myself into that political world and lob my heart onto the lovely mahogany desks of my elected representatives?   Because I am a mother of a child with cystic fibrosis.  For me, this is not political—this is personal. 

Brady is 7 years old, and LEGO obsessed!  Sometimes it feels like my whole world looks like this!

Whether we like it or not, our healthcare system is evolving, and every year lawmakers are voting on legislation and budgets that will directly impact families and patients living with cystic fibrosis.  The Cystic Fibrosis Foundation understands this, and has been working to establish a presence on Capitol Hill for many years.  On March 19, over 120 CF advocates, representing 38 states participated in 273 meetings in the CF Foundation’s “March on the Hill” event.    This is an opportunity for CF experts (YOU!) to come to the table, and educate elected officials on what it means to live with cystic fibrosis.  It is our opportunity to bring the unique needs of our community to their attention, and explain how budget appropriations and certain pieces of legislation can impact those living with CF.  Not convinced this is for you?  Let me outline the ways that CF advocacy has touched my own life.

July 26th 2007, Brady came into this world.  He was the first baby in Idaho to be picked up on the newborn screening program.  That test was added to the newborn panel in Idaho the same week that Brady was born.  After a positive screen came back, we performed genetic testing and he was diagnosed with CF before he was 3 weeks old.  We were able to intervene immediately, and begin treatment with enzymes, airway clearance, CF vitamins, and other medicines that would help him grow and thrive.  He didn’t have to become terribly ill and suffer potentially irreversible damage before we knew something was wrong.  Obviously, the diagnosis was traumatic (especially in the throes of post-partum sleep deprivation and fluctuating hormones), but the benefits of early intervention are well studied, and we are eternally grateful that we had the opportunity to begin helping Brady fight this disease as soon as we could.  The newborn screening program is up and running in all 50 states, which is awesome!   Newborn screening exists today because CF advocates worked for years on Capitol Hill to get it added to the newborn panel in each and every state. 

When Brady was a baby, I learned that the Idaho Legislature was proposing legislation to cut a co-pay assistance program for adult patients with CF. The budget for this program was a mere drop in the bucket ($200,000 total annual budget), but a game-changer for adults needing assistance in Idaho.  We already know that 1 in 4 CF patients sometimes skip doses of medication, or go without some prescriptions all together because of cost issues.  Cutting this program would mean that many adults with CF in my state would lose the ability to get some of the drugs that they needed to manage their disease.  It made $1000+ co-pays a monthly reality for many adult patients in Idaho.  It caused severe anxiety and depression for some of those adults already struggling with advanced disease.  It caused some adults who had previously been able to work part-time to lose/quit their jobs and become fully dependent on Medicaid or SSI for coverage of their life-sustaining drugs.  It sucked (especially when I learned that the Idaho Legislature was creating a new “slush fund” with a $200,000 annual budget to pay lawyer fees when legislators found themselves in lawsuits that same year).  Everything about it reeked.  Though it wouldn’t affect Brady directly (the program was for adults only), he was on a similar state program for children, and I realized that lawmakers were going to be making decision about his access to care and drugs.  I realized that many of the people making those decisions didn’t even know what cystic fibrosis was.  I realized that unless I spoke up—they may never know.  (Insert long rant of obscene language, and imagine me banging my head against a brick wall).

A newspaper article from our battle with the Idaho Legislature.

If you are reading this blog, you might already know that my son Brady has been taking Kalydeco for over 3 years, and that it has changed our lives.  But did you know that the technology that led to the discovery of Kalydeco—“high-throughput screening,” was developed by the National Institutes of Health?  Did you know that targeting a genetic mutation for correction wouldn’t be possible without the NIH first mapping the human genome?  Without the basic science that has helped us unravel the mysteries of our DNA, we wouldn’t even be able to DREAM of the kinds of “genetic modifying” drugs like Kalydeco that are being crafted today.  Just to be clear—the CF Foundation funds nearly 100% of medical research for CF.  The National Institutes of Health does NOT give a single dollar to the CF Foundation, or “orphan” (rare) disease research.  They DO, however, fund basic science projects that enhance our understanding of genetics.  They DO develop new technologies that could accelerate drug discovery for all diseases.  They DO fund generalized research projects that may eventually be applicable to CF.  The importance of funding the NIH has ALWAYS been a priority for CF advocates.

Take a look at our drug development pipeline.  It is jam packed with hopes and dreams.  Hopefully, many of the drugs in clinical trials right now will be applying to the FDA for review in the coming years.  When new drug applications are submitted, it is vitally important that the FDA has the resources they need to expertly and swiftly review those applications.  The CF Foundation has worked for many years to build a relationship with the FDA, and enhance their understanding of our projects and goals.  Kalydeco was granted “priority review” status by the FDA, which served to expedite the approval process, and was given one of the fastest reviews in history (less than 3 months from submission to approval!)  Awesome! As you know, the  quicker these miracles can get into patient hands…the better. 

I hope I've made my case that CF advocacy is not about politics after all.  I honestly believe that working to help shape our system is the most important way to ensure progress toward the cure and protect Brady's access to care and medicine.  After attending the NACFC for the last 4 years, I am amazed and inspired by the science coming through the pipeline.  We don't want roadblocks on the path to the cure. It is extremely important to educate our lawmakers on the unique needs of the cystic fibrosis community if we want a system that helps individuals and families with CF thrive... and YOU are the best experts in the world! It is much better to proactively troubleshoot problems that we foresee as we move toward the cure, than sit back and complain about a system that doesn't work for us when the damage is already done.  CF advocacy is about coming to the table and giving input about the policies that are shaping the future of healthcare,  With that in mind, I want to share more about the specific action items that March on the Hill advocates met with lawmakers about last week. 

1) Co-sponsorship of the Ensuring Access to Clinical Trials Act of 2015 (EACT) S139 H.R. 209

This legislation removes the sunset provision (expiration date) from the "Improving Access to Clinical Trials Act" (IACT), which was passed in 2010.  IACT allows patients with CF and other rare diseases to receive up to $2,000 in compensation for participating in clinical trials without that compensation counting toward their income eligibility limits for SSI and Medicaid.  Without this legislation, patients that we NEED to be able to participate in clinical trials, may not be able to, out of fear of losing their vital benefits.  The Cystic Fibrosis Foundation is planning to fund 18 clinical trials next year--requiring 2500 patients to participate.  When you consider the various exclusion criteria for those trials, and the fact that we only have about 30,000 CF patients nationwide--it is extremely important to remove this barrier to participation.  IACT is set to expire in October.  Advocates are trying to get EACT passed before this happens!  This legislation costs a negligible amount of money, but is extremely important to ensure speedy and full enrollment of new clinical trials.  EACT applies to all rare diseases, not just CF.  Rare diseases impact nearly 1 in 10 Americans.  According to the NIH, there are over 7,000 rare diseases affecting between 25-30 million people across the country.  Rare disease researchers face real challenges recruiting enough patients to perform trials, and current treatment options are often very limited.  Back in 2010, IACT received bipartisan support, and has allowed us to progress quickly forward with trial enrollment for the last 5 years.  We really aren't asking for anything new at all with EACT--simply to remove the 5 year expiration date, and allow the law to remain permanent.  Last week at March on the Hill, CF advocates from 38 states attended 273 meetings--and asking for support of this legislation was a priority in every one.  EACT also has good bipartisan support...but we need to push it across the finish line here and now.  You can help by taking action through the CF Foundation's advocacy page.  Click, "Take Action Now," then "Tell Congress to Protect Access to Clinical Trials," and follow the prompts.  You can personalize the pre-written letter a bit with your CF connection, and send it directly to your representatives!  This takes about 1 minute, and is a really effective way to let lawmakers hear your voice!  It would be especially helpful if constituents from Texas could help us put some pressure on Senator Ted Cruz.  I mean, the CF Foundation is funding the research.  All we want is the opportunity to test our new amazing science in clinical trials! Is that too much to ask?  NO!

2) Support funding for the NIH and FDA.  

CF advocates are working to boost funding to the NIH this year and are asking lawmakers to approve a budget where the NIH receives at least $32 billion dollars in 2016. NIH funding has been flat since 2002 (which translates to a DECREASE in funding each year when you take inflation into account).   That means that the NIH has been able to fund fewer and fewer interesting projects that are seeking funding each year.  What is slipping through our fingers in those missed opportunities?  What if one of those basic science projects might lead us to a cure for CF?  It requires you to take a big step back and take a look at the big picture, but we NEED to see basic science research take a bigger priority in our government’s budget appropriations.  Globally, America is falling behind, and it is only going to get worse unless we change this trend.  Investing in medical science is an investment in the FUTURE of this country, and the future of our loved ones with CF.  Last week, CF advocates explained why NIH funding is important to the CF community on Capitol Hill, and asked them to sign the "Dear Colleage" letter circulating in both the House and the Senate to show their support of the $32 billion dollar budget.  You can "Take Action Now," then click "Protect our Progress Toward a Cure for CF," and follow the prompts.  Once again--a minute of your time could make a big impact for families living with CF.  

The FDA worked smoothly and quickly for Kalydeco.  Why are we making FDA funding an issue?  The medical research community is soon going to be asking the FDA to consider a huge number of new drugs in different ways than it ever has before.  Historically, to be considered a candidate for approval, the FDA has required long term trials with a large pool of patients.  Obviously, these criteria aren’t going to work for mutation specific CF drug trials.  As a rare disease, and with new therapies targeted at specific genetic mutations, there may only be a few hundred (or less) “qualified” patients that even exist worldwide!  It would be impossible to enroll huge numbers of patients for individualized genetic treatments for an already rare disease.  In 2012, CF advocates played an integral role in the passage of the EXPERRT Act.  As part of this legislation, a new “breakthrough” designation was created for drugs that treat life-threatening diseases, for which there is no alternative.  Drugs that are granted breakthrough status, are given a faster review schedule than ever before.  The legislation also enables the FDA to bring in disease experts to consult on trials that don’t fit the old school criteria.   Passage of the EXPERRT act was a big victory for the CF community, but we aren’t satisfied yet.  While CF is blazing the trail for this type of drug design, an increasing number of disease communities are uncovering genetic correlations of their own, and developing targeted therapies.  Personalized treatment is the future of medicine for everything from CF to cancer and beyond.  The FDA is going to need a larger staff to effectively review the growing number of new individualized drug applications coming their way in the near future.  What a shame it would be to see amazing new drugs bogged down in delays over a simple lack of time and personnel to perform safe and prompt reviews.  We want our future new drug applications to be considered with the same prompt expertise we saw with the Kalydeco approval.  Bottom line is that the FDA is going to need more money to function like we need it to in the very near future. If you sent the "Protect our Progress Toward a Cure for CF" email, you are already done!  Robust funding for both the NIH and FDA are included in that request!

3) Join CF Caucus in the House of Representatives.  

The CF Caucus is sort of like a "club" that House Representatives can join to signify their support for the CF community.  Follow the link to see if the people who represent you are members! Membership in the caucus raises awareness about CF, and issues that will impact patients and families living with the disease.  We try to grow membership in this Caucus every year. Currently, there is no CF Caucus in the Senate.  We don't have a specific action item in our CFF advocacy toolkit for this, but you are always free to email your own elected State officials and invite them to join the CF Caucus in the House (or find a great supportive Senator to START a caucus in the Senate!).  

New issues come up all the time that could affect the CF community.  To me, advocacy is about putting face on this disease, and letting our lawmakers know that their votes affect us as people.  We are not just a number in a budget.  Their votes mean something to us.  We are the Mothers, Fathers, Grandparents, Sons, Daughters, Brothers, Sisters, Aunts, Uncles, Friends, and Patients dealing with this disease.  As despicable as politics can seem at times...our lawmakers are actually just people.  Most of them have families, and can imagine what dealing with CF must be like.  I try to relate to them on a personal level to help them understand.  When you are sitting face to face with them, the intimidation fades away, and it becomes apparent that they are actually very much like us. I encourage you to explore CF advocacy efforts in your state, and contact publicpolicy@cff.org with questions.  

As a mother who has been lucky enough to see what a bright future new therapies like Kalydeco might hold for our CF community, I am determined to remove system barriers that might slow us down. I want EVERY patient and family to have a miracle like Kalydeco! I am honored and excited to serve as the CF Foundation's new National Advocacy Co-Chair with an awesome lady, Melissa Shiffman from NY.  Melissa is an adult with CF, who is the mother of two beautiful children.  As we are moving toward the goal of curing CF, there WILL BE OBSTACLES that require advocates to speak up.  I believe with all my heart that if we want a system that works for us...If we want coverage that meets our needs...if we want to be fed...we must first come to the table!  My life has been so touched by CF advocates.  I feel like "Thank you" is a huge understatement.  Besides, once you get bit by the advocacy bug, you can never quit!  It is so empowering!  Get involved in advocacy today and help protect the future for everyone with CF. Please "Sign up" to receive the CF Foundation's advocacy newsletter, and action alerts!  How about taking 30 seconds to advocate for CF patients RIGHT NOW by sending a tweet to Senator Ted Cruz in Texas asking him to support EACT?!  Something like...

"The ‪#‎CysticFibrosis‬ community needs access to clinical trials, @SenTedCruz Pass S. 139!! ‪#‎cfAdvocacy‬ @CF_Foundation"

Thank you wonderful people!  

With my RAD new Co-Chair Melissa Shiffman on Capitol Hill last week!

Chatting up CF advocacy at Sacred Heart's Family Ed day a few weeks ago. Do I ever shut-up?  NOPE!
And neither should you!