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Archive for September 2015

Texas Medicaid Patients still wait for Orkambi access

Thursday, September 24, 2015

When you're fighting CF, you're fighting time.  Every day that goes by is one more day the disease can grasp you tighter or it's one more day your body has to fight.

So, imagine how frustrating it must be for those 82 Texans on Medicaid who cannot get access to a medication that they quality for, that sits on the market and that may extend their life?

Orkambi is the second drug ever on the market that addresses the underlying cause of Cystic Fibrosis. Orkambi works for only 28% of the CF community and costs about ~$250,000/year.  Kalydeco, the first ever drug that addresses the underlying cause of CF benefits about 10-15% of the CF community and costs ~$350,000/year.

These are big price tags for health insurance policies to pay.  So, it's not surprising that states themselves would be highly concerned about the impact these medications may have on the budgets of state medicaid programs. High drug costs in all diseases are becoming an issue in our nation's healthcare.

This issue is a particular concern for those in my state, Texas.  State lawmakers are trying to do what they can to reign in the cost of new prescription drugs.  The way they are going about it, however, is having a serious impact on those in the Texas CF community.

Concerned over the issue of rising and unsustainable drug costs, the Texas legislature passed a law that went in to effect on September 1, 2015 that states that it is now the job of the Texas Legislative Board (LBB) to approve or deny paying for any new-to-market oral prescription medications that constitute significant cost to the state.

The Texas LBB is made up of four state government offices.  See board members here.  However, before the LBB can make it's decision, it must receive a letter from Texas Health and Human Services Commission giving it's analysis of the medication.  From there, the LBB has 45 days to make it's yes or no decision.  However, at any point, the LBB can stop the 45-day clock if it requests further information.

This new system has the potential of causing big delays for those with Cystic Fibrosis who are trying to beat the clock.  The delays have already begun.  Even though Orkambi was approved in July of 2015, as of today, Medicaid patients still cannot get access.

This requirement of LBB approval does not apply to patients on private insurance, only to those who are on Texas medicaid programs.  But, with a large portion of CF patients on medicaid due to the high cost of care and many adult patients' inability to stay healthy enough to work full time, this means a lot of our Texas-based CF community is and will be affected by this new law and the repercussions of what the LBB decides.

The LBB is strictly budget-oriented.  No clinical staff are a part of this budget decision.  That's why patient advocacy is so important.  Patients and families of patients, like mine, have to tell our story.  We have to talk about life with CF and the impact even small delays can have on the health of our children and those suffering from the disease.  We have to share how access to these drugs are life and death decisions.

It's important to note that Bennett cannot benefit from Orkambi. :(  While it is wonderful and has the ability to help many with CF, it cannot help Bennett.  Bennett does not have the right genetic mutations for it to work for him.  Instead, we continue to wait for a medication that would benefit him.

The CF community is a tight-nit group.  We look out for each other and fight alongside each other. Although Bennett does not directly benefit from Orkambi, I can't bare to think there are families who do qualify who cannot get it for their children.  I will continue to monitor this issue and advocate on behalf of those who need access.

The reason we have the CF meds and access to meds that we have for Bennett today is because someone advocated for them long ago.  I hope to do the same for others in the future.

To the Texas LBB, please say yes to access to Orkambi for those in Texas suffering from CF.  Waiting is simply not an option.

Beyond the School of Hard Knocks

Wednesday, September 16, 2015

Where do you go to learn how to be a caregiver of a child with Cystic Fibrosis or other chronic disease?

A CF doctor goes to med school.  A nurse goes to nursing school. A CF social worker goes to the School of Social Work.  But where does a patient/caregiver go to learn how to navigate and thrive in the healthcare system?

For patients and families like mine, we pretty much attend the School of Hard-Knocks.  We learn on to job and figure it out as we go along.

How do clinicians continue learning, even after finishing school?  Medical conferences.

How does a patient/caregiver continue learning??

I propose we also go to: Medical conferences.

In the next two months, I am excited to attend two medical conferences that I hope will help me gain knowledge, insight and support to keep up with our fight against Cystic Fibrosis.

In October, for the first time, I'll be attending the North American Cystic Fibrosis Conference (NACFC) where doctors, nurses, respiratory therapists, researchers and other CF clinicians go to present and learn the latest in CF research.

This year, more than ever before, caregivers of CF patients are expected to attend NACFC as the CF Foundation has provided a price-break for families of those with CF so they can go and learn alongside the people charged with helping them.  Family members of CF patients have also been asked to present about their experiences.

(Patients, on the other hand, cannot attend NACFC due to the cruel reality of the disease which presents the real issue of spreading dangerous life-threatening infections to each other.  This makes me SO sad.  But portions of NACFC will be available for all who cannot attend to view online.)

There is another conference that takes place next week I'm equally looking forward to attending.  It's called MedX.  MedX is an innovative and cutting-edge annual medical conference held at Stanford University.  This cross-disease learning conference (aka not CF-specific) is made up of clinicians, researchers, patients and entrepreneurs working to highlight the changes happening in the healthcare system and to support learning from each other.

I recently received a scholarship to attend and the invitation to serve as an ePatient delegate at MedX. I will be there representing Bennett.  As an ePatient delegate, I have been invited to share my patient experiences, to help identify ways I think we can improve our broken health system and to share the great things I learn with others through social media (sorry, in advance, Twitter!).

MedX has asked me to be a part of a really interesting panel discussing transparency in healthcare and the value of patients having access to information.  The topic we will be discussing is the recent ProPublica's article, "The Surgeon Scorecard" which received both praise and criticism when the non-profit investigative news organization used public data to help offer patients a way to see which doctor is "better" or "worse".  (Here's a nicely condensed Today Show version of the debate at hand: http://www.today.com/health/surgeon-scorecard-aims-help-you-find-doctors-lowest-complication-rates-t33186).  I am really looking forward to getting to hear in person from the authors of the study and share my own feelings about the article from a patient's perspective.

Being a part of a medical conference may not be interesting for some patients.  And that's ok.  But I think there is something hugely important about patients, in general, being a part of medical conferences and the research that is presented there.  So many clinicians work within silos, sharing information and ideas amongst themselves, in hopes to improve the health outcomes of patients.  But there is no member more invested in improving patient outcomes than the patient his/herself.  So, it's important to give patients a seat at that table.

I am grateful for medical conferences and organizations like MedX and FlipTheClinic and the Society for Participatory Medicine for their effort to give voice to the patients who are no longer passive participants in their own healthcare.  Patients now have the ability to find information, do their own research, track their own health data, etc.  They are becoming their own experts in their care and they need to be recognized as one.  Who knows best what patients need?  Patients do!  So, let's work to learn from them!

There is so much about our healthcare system that is good.  As Americans, we have access to some of the best patient care ever available.  I am thankful that giving patients the opportunity to teach clinicians, learn alongside clinicians and work in collaboration with clinicians will only improve the way we do healthcare.

I never wanted to enter the School of Caring for a Child With a Chronic Disease.  But if I'm going to have to be here, I might as well speak up in class and learn as much as I can!


What's it like to live with Cystic Fibrosis AND hope everyday?

Tuesday, September 1, 2015


Bennett has finally settled in to his nightly sinus rinses.  A fellow CF mom had suggested that we do them during bath time, which is very helpful.  He is very brave every time he does them and says they even feel good.

We give him gummi candy every time he does them.  They are well-earned Star Wars gummi candies. :)

We have gotten pretty good at doing nasal rinses at night.  But it's hard to get them done in the morning - in addition to his doing his breathing treatments, taking his medicines, eating breakfast and rushing to get out the door for school.  So, Brian and I are just giving ourselves grace and doing our very best.  Our goal is to do them twice daily.  But that's a lot to ask of a 5 year old.

I often wonder what it's like to be Bennett.  What must it feel like to live with CF every day?  To have to do treatments and worry about taking medicines daily? What is it like to live with a disease that is most certain to shorten your life unless there is first a cure?

My friend Oli Rayner does a great job providing some insight into what it's like to live with CF.  Oli is an adult with Cystic Fibrosis and recently wrote a forum post about what it's like to live and act hopeful while knowing the disease is fatal and progressive.

I wanted to share Oli's words with others, as I found them to be incredibly insightful and incredibly articulate coming from the point of view of someone who lives with CF every day:
"I think one of the toughest things about living with CF is the psychology and trying not to let your fears of the future undermine your experience of the present.

You are born with a defective gene and even though you may feel fine now you know that as time goes on the gene will express itself more and more and probably kill you when you have only had half the life of a normal person. The psychology of that is really complicated and challenging.

It is not like other diseases where you suddenly feel sick and then find out why and do things to make that feeling of sickness go away.

Often with CF, you get the diagnosis before you feel sick - you feel fine and learn about the horrible things that will happen and the boring, unpleasant things you have to do every day to try to stop the horrible things happening. It is like a Sword of Damacles and it can be a bit terrifying.

But if you stay focused on what you can do today (not what you can't do or what you might not be able to do in future) and what you can do today to make yourself stronger, you can feel great power and unexpected things can happen.

If you have a bad patch it is very hard to know if it is a temporary "blip" or a new normal. Is this the "beginning of the end" or just an unlucky spell?

This makes it very hard to manage life, career and relationships because to do that sensibly you do need some sense of your baseline capacity. I do feel clinicians are too quick to run with the progressive disease narrative (i.e. encouraging us to think new normal vs blip).

I believe this can be a self-fulfilling prophecy sometimes. I expect this "downward curve" psychology may be related to the fact clinicians always feel they need to make us understand the "reality" of the condition and the importance of doing all the treatments. I wonder how the dialogue and background psychology might change if adherence was not the elephant in the room. The cruel irony is that more positive discussions (what can we do today rather than what might happen in the future) might actually help with adherence too.

If you look at how CF outcomes have changed over the last 40 years with median predicted survival increasing from about 5 to 40, it is likely that, at any given point in time, professional consensus expectations around "what life holds for someone with CF" will be based on backwards looking data/observation and therefore lagging the prevailing reality.

Apart from anything else it usually takes years for data to be analysed, published and propagated through the community so even data that the community treats as "current" now is likely to be out of date. This problem will become amplified (changes in consensus expectations around "what life has in store for someone with CF" will happen faster) as new disease-modifying treatments come through the pipeline and our collective psychology will tested even more.

I have certainly had periods where things have been very tough and I have been in hospital a lot with one bout of infection after another thinking my life is basically over. But it turned out to be a blip and I got better. It was not a new normal - I got back to the old normal through some small practical changes, hard work and a very stubborn attitude that says I don't accept that I can't get better.

How great would it be if we sat down with our clinical teams and asked what can we do to move lung function up 10% over the next 6 months or some other positive target that aligns with our life goals and for clinicians to really respond to that rather than saying things like "well you have to understand CF is a progressive disease, etc etc."

I am a realist, sometimes brutally and it can cause problems with those around me who like reality to be dressed up a bit, but I am also a very positive person. I like to think of it as enlightened positivity. What is our situation today? Where do we want to get to? What tools do we have? Let's go!

In my view, the psychology of CF is very poorly understood and it is an area that needs much more research. There are a lot of unspoken assumptions that color the way we think and behave. Those assumptions may be wrong and they may be causing unnecessary harms or blocking achievable gains.

I love this idea of unwinding CF."
Daily battling to unwind CF for Bennett in time,