Tuesday, April 4, 2017

I'm not okay with this (and you shouldn't be either!)

I wish I could dedicate all of my time to writing because it's the best way for me to relax. In my head I carry 1,000 thoughts that I regularly wish to share, but in the craziness of everyday life I can't seem to find the time to sit down and write it out. I want to get better about scheduling time to do this.

March was a crazy month for me. I've started to work with the FDA's Pediatric Advisory Committee in the Office of Pediatric Therapeutics. It's not a job job, just an advisory role that requires minimal time, but I did have to travel to DC for a meeting at the beginning of the month. It was a fascinating look into the decision making processes of the FDA to protect children and ensure safety of drugs and devices that go to market. I'm always eager to learn more about the complexities of this healthcare system.

Following the FDA meeting, I was off to our second CF Learning Network Community Conference that was nothing short of remarkable. The dedication of the teams in this network to improving outcomes faster fills me with hope. Finally, something that I can do, something that I can be involved in that has a direct impact on improving health and care. I can't place a PICC line, or develop the next big drug to cure this disease. I can fundraise but i'm not good at it. But this is something I can do that has the potential to transform CF care and outcomes.

Here's the thing that I don't think people understand: The efforts of the CF Foundation have been great, and I am grateful for advancements that we have made. But the truth remains:

  • The CF mortality rate across the country has not improved over the last ten years. 
  • The median survival in the US is 11 years less than median survival in Canada (at 50.9 years). 
  • The rate of pulmonary exacerbations in the CF community has not changed since 2004.
Additionally, and we all  know this, the variation in care and outcomes differs, not only between centers but between clinicians within the same center. I know dozens of people with CF who travel to X center because they can do whatever better than their primary center. Or patients who discuss online their preferences for one clinician over another to try to receive better care or achieve better health. And that's not okay by me. Let me repeat that: I AM NOT OKAY WITH THIS. We have to do better. We deserve better. Our care center data, the data used to make clinical decisions and set research priorities is two years old. We are operating with only a rearview mirror, and I'm trying to create a hypothetical dashboard so that we can look ahead and make decisions with confidence, understanding the value of our efforts. I need the rest of the CF community to recognize where we are and say with me that this is not okay, that we deserve better, and that we are committed to working together to achieve them. 

I'm working, with 13 care centers and 30 other parents & patients to create a system that allows us to learn from every interaction so that we can reduce this unintended variation in care, get the right thing to the right person at the right time, every time, and improve care and outcomes. We are working to understand how care is provided at the best CF Centers, learning from their processes and adapting them more quickly to the needs of our center so that we can reduce the unintended variation in care and outcomes. We want to be able to seamlessly share how we are achieving the outcomes at our center so that others can learn from what we know. For example, who knows that the infection control protocol at our care center is unique to our center and has dramatically decreased the rates of infection at our center, which can leads to improvement in mortality rates and survival? I would bet that not even all of the patients at our center know this, but if they did, and if they shared that, everyone would be banging down the doors to their centers saying, 'Hey! Look at them! We want what they have! Lower infection rates, longer lives! Let us help you learn from what they did and learn together about the impact that these changes might have at our center!" What if I had the data to show the secret sauce for Achromobacter eradication (I don't). But what if I was able to collect my data and show the impact on my health and then contribute that data to an enhanced registry so that the next person to acquire that bastard achromobacter didn't have to go through the guessing process for how to treat it? This is what I'm talking about. And it's possible! I know because i'm doing it. This is what the CF Learning Network is and aims to do. Everyone deserves access to this information, but there isn’t a process in place for sharing it. That's what we are changing. The current lack of organization within and across CF care centers results in variations in care and outcomes, and a lot of time wasted trying to recreate the wheel. Raise your hand if your center is working on infection control? Or transition? Or adherence (what does that even mean?!)  Now raise your hand if you're learning from or with another center who might be doing the same thing. Who knows what impact those efforts have? What exactly are you trying to change? What what are you using to determine whether those changes are an improvement? 

Quality care and good health outcome shouldn’t be dependent upon where you receive care. In the CF Learning Network, we believe that everyone has something to teach and something to learn, and that by working together in this new way we can change the pace of improvement in the CF community so that everyone has the best chance of living their healthiest life with CF. 

So we have this incredible Community Conference in March and are now gearing up to bring on 15 more CF centers because everyone is realizing the value of this learning model and that we cannot continue to sit by and watch things stay the same. Let me give you the example of the recent article about the life expectancy for people with CF in the US and Canada having a gap of 11 years. WHO IS OKAY WITH THIS?!?! Not me, in case that wasn't clear. Can we please get impatient? Every article written about that (like this one and this one and yes, even this one written by our very own CFF ) offer guesses for why this variation might exist. What I'm saying is that I'm tired of guessing, especially given that there is a way to stop the guessing and start the learning. We are in 2017. It has been 28 years since the CF gene was discovered. We collect a ton of data and our community includes a ton of smart, dedicated folks, clinicians and patients and parents, and we need to say that this is not acceptable anymore. We need to demand that we do better. We need to figure this out because our kids and our friends lives depend on it. We need to work together. 

In another couple of weeks I should have some data from our network that I can share to show the impact of this new collaboration, of our Learning Network model, on its ability 

This is getting lengthy so i'll wrap this up but want to try to keep writing, as "To survive you must tell stories", so says Umberto Eco. Here's the story I told following the community conference at the American Society of Clinical Pharmacology and Therapeutics about the role that patients and parents can and should play in drug development and healthcare improvement. It might sound familiar if you've been reading this blog for any amount of time, but here's what I had to say:

I was trying to think of a way to introduce myself and I thought that sharing a story would be the best way for you to get to know a little bit about me. It’s a story about my 7yo son who has cystic fibrosis. Cystic fibrosis, for those of you who dont know, is a genetic disease that primarily affects the lungs and digestive system, causing a thick sticky mucus to build up in the body which can lead to life threatening lung infections and impaired digestion. The Cystic Fibrosis Foundation recommends that people with CF be seen by their care team 4 times a year where they ask questions like ‘How’s everything going? What’s his cough been like? How is his appetite? Have you noticed any changes?” And my answers typically sound something like “Good!” and “Pretty normal” and “It was down a couple weeks ago but it seemed to improve.” It’s all subjective, and its mostly from memory. You see, i’m managing my son’s CF in the midst of parenting 3 other kids, working full time, and trying to maintain some semblance of a social life.

As I reflected on these clinic visits, I thought about the value that could come from having a way to quantify the answers to the questions that the doctors were asking us. I went to Facebook and then to Google to see what things other people were using to track stuff about their health. I found an online platform that I could use, and I started to track things about my sons health. And to my surprise, trends began to emerge. I could do personal experiments, looking at changes in my data with the addition or subtraction of a medication or therapy. My care team didn’t seem all that interested in my data, but I found it to be a fascinating learning resource. I was sharing what I found with other patients and parents online, getting their input and feedback on what I was learning.

Then around the beginning of March a few years ago, Drew developed a cough. It wasn’t like his normal cough, though I couldn’t put my finger on how it was different. The frequency wasn’t the same as it has been with other infections. The sound of the cough, something that I can’t quite yet quantify, was different – not totally wet and junky, not exactly tight. His appetite was down. I had talked to his doctor and we decided to try an oral antibiotic, a typical treatment for what they would call a Pulmonary Exacerbation. After a few days and little improvement, we decided that he needed to try IV antibiotics to see if we could kick this. It’s the standard next in line treatment option for an exacerbation. You’ll notice that throughout the course of Ivs his cough still didn’t return to baseline. I was working with my peer community online, and my care team, asking questions like “Did we choose the optimal combination of medications? Is there another treatment that might help better? Are we missing something?” We added a magnesium supplement about a week after we started the IVs, something that my online community of peers had suggested when his care team was left scratching their head, and then upon completion of the IVs tried a steroid to see if that would help. These were all guesses, some based on evidence and others based on anecdotes. All seemed to maybe help a little but weren’t bringing him back to his baseline. This mystery cough continues for over a month. Through all of this, both his doctor and I are monitoring his symptoms and brainstorming together, in agreement that we don’t know what’s going on. She suggests that we may need a hospitalization if these symptoms are going to continue so that he can be monitored more closely.
Desperate to not be admitted, I start digging into my data. What on earth had happened around the time that the cough had developed that may have caused this problem. The only change that I was able to note was that we had stopped Prevacid at the beginning of February, just a couple of weeks before this cough started. He had always been on Prevacid since birth, and we had decided with his care team to take him off of it to see if it was something that he needed or could do without. And when I asked the care team how we would know if it was working, they said that we would notice a change in his bowel movements if he needed it. He takes pancreatic enzymes to help his body digest food, and the Prevacid is a means to control the stomach acid, helping the enzymes pass through his stomach and make it to his pancreas to do their job. If we noticed a change in his bowel movements, then the enzymes weren’t doing their job in regulating fat absorption because they weren’t getting through the stomach intact. I wasn’t even considering how stomach acid might impact his cough. I shared my findings with his doctor and we agreed that restarting the Prevacid was not a bad idea to try.

We restarted prevacid and within a week his cough had returned to baseline.

This change in his care was the result of a suggestion that I had made, not the care team. And it was based on data that I had collected, data that to this day doesn’t exist in his electronic health record. And there was a cost to this! It was his health that suffered - days of school missed, infection control risks associated with PICC lines and hospital admissions, antibiotics that weren’t necessary for a kid whose threat of antibiotic resistance is a real issue. There were days of work missed for my husband and myself, and an impact on the rest of our family with the limited.

My name is Erin Moore. I am the mother of 4 kids and work with Cincinnati Children’s in Quality Improvement related to the patient and family experience of care, and with Eli Lilly as an Patient Advisor. I’m what you might call an ePatient - equipped, enabled, empowered and engaged in my health or the health of someone i care for, I also consider myself a Citizen Scientist. Citizen science (also known as crowd science, crowd-sourced science, or networked science) is scientific research conducted, in whole or in part, by an amateur (or nonprofessional scientist. I do this out of necessity. It’s not possible for any one of my sons doctors to understand all that there is to know about CF, let alone his personal experience with it. I consider it my job to learn all that I can to offer a personalized approach to treating him and giving him the best chance at a good health outcome. I collaborate with my peers, clinicians and patients, to share what we know and change what we can, together. There’s additional benefits to citizen science. The massive collaborations that can occur through citizen science allow investigations at continental and global scales and across decades—leading to discovery that a single scientist could never achieve on their own. Have you ever tracked something about your health - logged steps via a fitbit, or documented the days you’ve had a headache or the pounds lost on a particular diet? Contributing what you know to the improvement of your own outcomes and the outcomes of others is citizen science, and you may already be involved!

The question that I want to present today is though is what if the next person to have a problem similar to the one my son had could learn from our experience, seamlessly? What if there was a better way to learn together from every interaction, curating content from multiple sources, sharing the value that came from our individual interactions with it, building on it for the next person whose path it crosses?

We're creating this in the work I do with Cincinnati Children’s hospital on the development of learning networks, groups of patients, clinicians and researchers working collaboratively to design and test solutions and tools that have the potential to improve care and outcomes in rare diseases. We all have something to share and we all have something to learn. There is a desperate need for us to shift in healthcare from doing things for patients to doing things with them. The experiences of our everyday lives, in caring for these sick kids, or medically complex partners, or aging parents, our experiences are our expertise and both can and should be valued as complementary to the expertise that health care providers share.

My son has a multi-drug resistant bacteria growing in his lungs that has had us in and out of the hospital for years. The medical journals suggest that he will lose roughly 4% of his lung function every year. What I need is access to real-time, trusted, reliable information on everything from novel approaches to eradicate that bacteria, to reasons why having a certain pet could be detrimental to his health. I want to know what other parents know and have tried, and I want a doctor to weigh in on why those options may or may not be good for me. I want to hear about what tracking methods people use and how they connect with their doctor between visits, but even more, how its improving their care. I want other patients to feel empowered to ask about alternatives and suggest trying something new to their doctor, no matter how novel it may be. And then I want to work with the research team to develop the research agenda. I want the next person to ask these questions to intersect with the answers that I found, seamless. Access to information like this, to people, to life saving treatment options, should not be left to the chance of a desperate parent or patient stumbling upon it on Facebook. We really have an ethical and moral obligation to work together!

Here we are in 2017, with a tremendous opportunity to utilize existing technologies to expedite the delivery of the right treatment to the right patient at the right time, every time - stuff that can be useful in clinical care and in clinical research - and yet we're not. There are seamless intersections between people and information in almost every other aspect of our lives – my Amazon Dash button allows me to order new laundry detergent while I’m standing next to my washing machine when I realize that we are running low. Coffee for my Keurig automatically shows up every month in the quantity that the internet has figured out that we drink on a monthly basis. Netflix can give me recommendations of shows that I may like, a starting point if you will, based on my previous viewing preferences. Yet it takes 5 phone calls and a trip to the pharmacy to pick up a MAIL ORDER prescription that we have taken every month since my sons birth and will likely take until the day he dies. We use fax machines to get pre-authorizations and share medical records, MY data, that I often have to pay to get access to, and when I receive that paper version it is often full of errors.

You may recognize this screenshot from the movie Apollo 13 when the astronauts lives literally depended on fitting a square peg into a round hole . The NASA engineers dump everything to which the astronauts have access on the table, and are given the challenge of using what’s on the table to transform the round receptacle to fit the square filter. It’s an impossible task, but these people are solution-based thinkers and their friends’ lives are at stake. And they figured it out. They walked out victorious an hour later carrying a contraption that was once a non-existent solution. They used duct tape and cardboard and creativity and determination and never once said “we can’t do this.” They did it because they had to. And that’s exactly how the parents of children with rare disease are – innovating with cardboard and duct tape and creativity and determination and technology to save our kids lives, because if we don’t, no one will.*

Sharing my experiences on my personal blog and at the medical centers where we receive care has opened the door to many new opportunities for me as an ePatient, like the one I have that brought me here. I work with the clinical innovation team at Eli Lilly as an ePatient advisor, blogging and helping to guide them through feedback, providing insights on how increased focus on patient centeredness and real world evidence is changing the healthcare ecosystem and how these trends may affect drug development, and keeping the patient voice front and center in clinical trial innovation.

Patients and their families experience research and care in a way that you may never fully understand. Our expertise should be seen as complementary to the care teams and researchers, and I believe that the path to collaboration and ultimately improvement is a humble awareness of that shared humanity. There are days when I think about the fact that CF is a progressive and fatal disease that does not have a cure. Then I remember that life too is both progressive and fatal. I hope you’ll consider the value of including patients as partners, true patient integration, and the profound effect that it can have on improving health and changing outcomes.

*This paragraph was adapted from mom-blogger Kelle Hampton 


  1. I have been enjoying your posts and I'm encouraged by your passion to make the system better. As a Canadian mom of a recently diagnosed 13 yr old son, I have delved into the deep end learning as much as I can about CF and when the US vs Canada study came out, it was of particular interest. One thing that was not within the scope of that study, but I think has a possibly profound affect on the rate of exacerbations, is a Canadian study which compared the use of PEP vs the vest. It found that people using the vest had more exacerbations, and the time to the first one was 30% sooner than those using PEP. In fact the study was halted early since so many were getting sicker with the vest. Lung function, patient satisfaction and health related quality of life scores were similar, although PEP is considerably faster and cheaper. The study has largely been ignored in the US. Our clinic which took part in the study is fine with using the vest in addition to PEP, but not as the only form of physio. https://www.ncbi.nlm.nih.gov/pubmed/23407019

    1. Thank you so much for your comment! I am so grateful for the opportunity to connect with others who are learning and sharing! I was at a meeting on Friday where we were discussing the US/Canada study and the response that the CFF gave which was basically that Canada has a different system. And I think that's exactly the point I was trying to make in advocating for this learning health network! What we are trying to do is create a new system, one that learns from everyone and that can achieve outcomes that our current system isn't possible of achieving. I'm not sure why so many people w CF are complacent with this age gap and response from the CFF - the fact remains that better outcomes are possible and we should be doing everything we can to achieve them.
      Separately, we haven been using PEP with our Vest since our son was about 4 (he's 7 now) and it provides far better airway clearance than the vest alone ever did. When people tell us there isn't research to show the value of one over the other it's infuriating - just because research hasn't been done on something doesn't mean it's not relevant or true! I'm so grateful for your comment!