Rare Disease

A Patient’s Voice:
A Mother’s Perspective on Cystic Fibrosis

by Lanmark360


March 21, 2018

Having a deep understanding of clients’ target audiences is essential to developing effective marketing strategies and executing advertising materials. When it comes to the rare disease and pharmaceutical spaces, we at Lanmark360 know it’s vital to go one step further and that’s why A Patient’s Voice series was born.

As marketing specialists, we have to tailor our advertising to reach the physicians, clinical researchers, scientists, and other individuals who have the power to enact change and press the “START” button on development of new pharmacological therapies or advancement of ones waiting in the pipeline. But to do that, we must have an even deeper awareness of the patient and his or her journey.

A significant part of this journey, specifically in respect to pediatric patients, is the role of the caregiver. This is the dedicated mother, father, aunt, uncle, brother, sister, grandparent, or other selfless advocate who’s in control of managing the child’s condition and making decisions, as well as being his or her dependability and support system. I suppose one could say that, in some way, the caregiver becomes the voice of the patient.

I recently sat down with Texas native and mother of three, Breck Gammel, whose seven-year-old-son, Bennett, was diagnosed at birth with Cystic fibrosis (CF). During the interview, Breck shares her perspective as a parent and lets us into her and Bennett’s lives, providing valuable insight regarding experiences with pharmaceutical companies and different therapies, communication with her son’s physicians, and emotional aid and education received from the CF community.

Here’s what Breck shared with me about Bennett’s journey.

Thank you for taking the time to speak with us, Breck. With your insight, I’m hoping to help pharmaceutical and healthcare marketers gain a better understanding of rare diseases. I know you have a seven-year-old son, he’s one of three children, and he has Cystic fibrosis (CF). Can you talk to us a little bit about the journey to diagnosis?

I had no suspicion of CF before he was born. We had a nurse tell us that I was very healthy and we shouldn’t have any concerns, but when I reached about 38 weeks he stopped moving, so I had him checked out and he was fine. After his birth, we found out his colon had burst while in the womb. He went into emergency surgery and that was the first time I heard that CF might be potential. There were other options, but that was the worst. We received confirmation seven days later.

While waiting for the final confirmation of CF, what type—if any—of support or resources were made available to you and your family?

After diagnosis, I contacted the CF Foundation. I asked for resources and heard a little bit about the foundation at that point. That was my first interaction and then afterwards we went up to a CF clinic and that was where I got most of my education on the disease.

Thank you for sharing. How old was Bennett when you went to the CF Foundation?

We went to the foundation within the first month of his birth. We were in the NICU for about 28 days and right after leaving, we knew we needed to get there as soon as possible since the hospital didn’t really have a doctor who specialized in CF.

I see. So what types of therapies are currently available or were available at the time to treat and manage CF?

The therapy we use now has been pretty typical since the beginning. We use basic respiratory therapy with a vest as well as a nebulizer mask with albuterol and hypertonic saline. Enzymes were the initial medications we started with and eventually we added Pulmozyme®. Additionally, we use Azithromycin and Nexium®.

It sounds like you’ve implemented various therapies throughout the years. Are the pharmaceutical companies you’re involved with providing any type of patient or family support—emotional, educational, or otherwise?

I would say little to none. I think there are educational programs out there that exist in the form of an app, or a website, or maybe a video and I tend to onboard with new medication. As far as emotional support, I can’t think of anything.

Okay. When it comes to onboarding with a new therapy, do you find videos and other interactive media to be helpful?

Nine times out of 10, not really. I would say the one that was most helpful to me was offered with Pulmozyme years ago that described what was happening with CF; and I think the CF community does have a pretty good idea of that, but there’s still a real lack of full knowledge. I know certain things, like why the mucus is thick, but I don’t know why the body doesn’t work. I would like to see more materials that help explain that in a sensible way because that would be great for parents and caregivers. As a whole, I don’t think pharmaceutical companies get it right. They tell you how and when to give your child the medication, but that’s not really helpful to me.

getting it right

What about patient support groups? Are there any groups in existence that bring patients together with other patients?

No. CF is unique in the sense that people with it can’t be within six feet of each other, so there are no support groups, physical ones at least, that I know of. There are some gatherings for parents, but I wouldn’t call them support groups. There’s also this event called BreatheCon that was created by the CF Foundation, but it takes place online. But as far as groups, none really exist. It’s challenging because we can’t go to camp with each other, we can’t get together, and my son literally can’t interact with other kids like himself. So that’s probably part of it.

I appreciate you sharing that, Breck. For Bennett, what’s life like for him and what type of relationships does he have with his siblings?

He has great relationships with his siblings, they’re very close. His older brother is a protector and his younger sister is also a protector, as well as a caregiver and his playmate. As far as his health, he’s been doing well with his lungs. He has yet to get a serious infection that’s required him to be hospitalized. We also have never had to start some of the medications that many kids, at this point, are already on. However, he’s sicker than most with his GI system. CF affects the digestive system just like the lungs, so he’s probably had surgery maybe eight or nine times and has been in the hospital about 12 times.

Wow, okay. I’m glad to hear that his lungs have been healthy. When it comes to staying in the know of new clinical trials that are underway or new therapies in the pipeline, do you feel that as a mother of a child with CF, you’re kept well informed of that?

“Am I kept well informed?” or “Do I find a way to be well informed?” is probably the better question. I don’t think you’ll find a better patient community, well, at least on the parent side. I don’t know as much about adults with CF, but on the parents’ side, I don’t know if you’ll find another patient community that is as strong with initiating and wanting information. We are on top of it because we have seen our kids and we know that this really matters. Literally, our children’s lives depend on it. So if I just wait for the clinics to tell me about things, I’m probably not going to find out for a while and maybe that’s okay, but for a lot of parents like myself, I’m not going to wait. I’m part of a couple of Facebook groups with other parents and other people with CF that are following these drugs down the pipeline. A lot of people are following Vertex and some of these other small companies. They’re asking questions like “What gene does this affect?”, “How is this gene different than that gene?”, “Why does that medicine work with this one and not that one?”, and “I thought it was this mutation and not that mutation,” etc. I’m on top of it for sure, but not because someone else is giving it to me but because I’m finding the people who are in the know and getting it myself.

Oh, okay. That’s interesting. Does some of that information originate from the pharmaceutical companies?

Yes, sometimes. Much of it is online. Patients and caregivers are getting Google alerts and they stay connected. A lot of times the information comes through an investor call and we get it before it’s publically communicated, if that makes sense. There are other times where members of the CF community or the CF Foundation will communicate it as well.

Has Bennett ever been involved in any clinical studies?

No, he hasn’t because he’s typically too young. He’ll be eight soon. A lot of these trials require people who are 21 and older, but now we’re seeing the age requirement go down. Many of them also only accommodate certain mutations, so we haven’t qualified either.

Thanks for the information, Breck. From your perspective, what are some things pharmaceutical marketers or pharmaceutical companies in general can do to better support the CF community?

Something I would suggest is bringing patients and families in, kind of like what we’re doing now, at the very beginning before an idea is even born because a lot of times we’re brought in at the last minute. That would be the first thing. Also, there’s great effort right now to work on coproduction: clinicians, patients, and families working together. You know, patients have been so passive in their care and clinicians have been the ones in the know and that’s really slipping due to the internet; due to the way healthcare is working. Now patients and families are in the know first, so I would also suggest creating tools that help patients, their families, and clinicians work together.

mother and child

Another area that is being kind of missed is help with decision making. There are not many decision making tools out there; so when a family is faced with getting a tube for their child at age seven, there’s not a lot of really clear tools that help them decide if it is an idea that they should move forward with. I think if the pharmaceutical companies could help us by saying “here’s a good idea,” “here are some suggestions,” “here are some types of medications you can use for this issue,” you know, or, “here are some things you should consider.” There are definitely really fantastic decision making tools out there, but not many for the CF community.

Wow, fantastic input and ideas. From the standpoint of Bennett’s diagnosis, you knew instantly at birth that there was something wrong. What type of advice can you give to a family of a newly diagnosed child?

It depends on kind of where they’re at. If you hit trauma pretty fast like we did, my advice is hold on for the ride. If your kid isn’t sick and is doing well, I guess be hopeful and make it a goal to keep your child as well as possible until a medication comes along. The reality is that this is a really sad disease. It’s pretty terrible to suffer the end stages of it, but for families who’ve just received a new diagnosis, there’s a lot of hope and a lot of reason to believe that it isn’t likely to be the future.

Hope is a great message, Breck. Is there a place for simple tools, such as a general brochure that explains CF and what to expect? Would you say there are materials like that available for hospitals to give to families of newly diagnosed children?

Yes, there are. I would say it’s pretty abundant, really. The CF Foundation puts out “An Introduction to Cystic Fibrosis” —I think that’s what it’s called. It’s a book that comes with a video. There are a lot of resources like that. I think we’re inundated with that kind of stuff. I think a lot of it though is actually more appreciated when it’s online because it’s accessible and that’s where parents tend to find it. I think what you don’t find is the specific information for what’s considered specific to your child’s CF. It’s harder to make a brochure that everybody can relate to. It’s the general brochures that exist—not the specific information you wish you could find.

What about online support groups: places online where you can connect with other parents that are also going through the same thing you’re going through? Do those exist?

Yeah, they do. The ones I find to be most helpful are on Facebook. I’m on a CF mom’s page, a general CF page, and a CF teenager’s page. I’m also on a few others. Facebook is great because it allows you to be connected to people in a very small group. There’s another called CysticLife that’s probably the second place most people are finding themselves and it’s all for CF families. After that, you’ve got rare gene communities, such as Gene PHO. There are different pharmaceutical companies that have their own groups, but I don’t find that people stay in them.

How about the relationship between doctors and the pharmaceutical companies? I think you mentioned earlier that you’re often bringing information to your doctors about new treatments or clinical trials. Is there a lack of communication?

 No, I don’t think so. I just think the patients are part of conversations earlier than the doctors even realize. I think it’s a triangle relationship and everybody is sort of working together, trying to get what they need. Doctors don’t have the luxury to pay attention to my son’s specific mutations and all the medications being worked on in my son’s case, so I tend to be the expert in that way, but you know, my doctor is very well informed and attends the national CF conference every year. I don’t feel like there’s a missing relationship there. It feels good. We’re all connected.

Do you think there’s more awareness surrounding CF today than there was seven years ago when you first had Bennett?

Some; and it probably comes from the fact that CF has hit the news as a result of new medications coming out, but I would say it’s not tremendously different. Maybe people are more informed, but in general CF is a rare disease and most tend to have a vague sense of what it is.

Prior to having Bennett, had you ever heard of CF?

Yes, I babysat a child when I was much younger. I didn’t really know what was wrong, but I remember him wearing a mask and using this big, loud “thing” that made smoke. It was kind of scary at the time. I recall going home once and googling CF and reading about a child who passed away from it at 18 years old. I also remember asking my mom, “What’s the point in going to school if you’re going to die by the time you graduate?” Now that I have a child with it, I know that they go to school because they need to live normal lives. Thankfully, the life expectancy is around 41 years and hopefully is much longer by the time Bennett gets there.

Wow. That’s something, Breck. This has been really great. Thank you. Is there anything else you’d like to add before we wrap things up? I think for our purposes you shared a lot of helpful information that can assist us in becoming better at our jobs. I also know that our colleagues and clients in the pharmaceutical space will appreciate all you had to say.

I think I’d just reiterate not to underestimate the patients and their families. We want to be given information and brought in much earlier. I think it’s critical to the success of the patients living with CF or any other rare disease because they’re the ones who can participate in the clinical trials; they’re the ones that doctors are ordering and reordering medication for; and they’re the ones who help start the conversation with insurance companies. My greatest disappointment has been seeing pharmaceutical companies spend so much money on dead ends. So again, just please don’t forget about patients and their families.

With over 20 years in healthcare and pharmaceutical marketing and advertising, my passion to help build our clients’ brands, and in turn do something positive for the patients who matter most, continues to grow. As I mentioned to Breck, not only is A Patient’s Voice there for you, its purpose is to also let the patient, or in this case the caregiver, be heard; and who knows, somewhere along the way we may even cause something great to happen that could change the trajectory of Cystic fibrosis or another rare disease.

If your product or service relates to Breck’s story or you would just like to know more, contact Lanmark360.

Howard Klein, President Lanmark360

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