I haven't written in a while. For so long CF was taking over every aspect of my life. I didn't want to dedicate more time to CF and talk about it in my blog. A year ago I just finished transplant testing and CF seemed to be at the front of everything. I wanted to push CF to the back of my mind and ignore it's existence. Of course, my failing health wouldn't let me and so I took a break from blogging to get a mental break from thinking too deeply about what was happening in my life.
In November, Trikafta entered my life. Suddenly, I had energy, I had health, I had hope. I was busy living life. So many fears and worries of the future melted away and I didn't want to stop living my life to write about CF in my blog.
Then March arrived and Coronavirus arrived in the US. Schools closed, my husband's work closed, and we were told to hide in isolation from everyone we knew. It is amazing how quickly life can change.
It is strange how my "corona" life has mirrored so much of my late stage CF life. So much fear of germs, wearing masks in public, missing out on fun things in the name of health and safety. It almost feels like life as usual except I am not the only one canceling fun activities, wearing a mask, slathering hand sanitizer whenever I touch anything outside my home. The strangest part is while the world is suffering and struggling I personally feel well. While thousands of people worldwide are gasping for air, suffocating, feeling the exhaustion that comes from the lack of oxygen, I am breathing fine. I no longer wake up coughing, I no longer experience shortness of breath, I can breathe. And I guess I should feel relief...and I do.
But man, my heart breaks for every person suffering right now. I know too intimately what they feel, despite never experiencing coronavirus. I know the fear, the panic, the pain that comes from lungs that are failing you. And I have had 35 years to cope with it, and 35 years of getting through so that I can tell myself (even lie to myself) that I will be fine even when I struggle to breath. But all of these people are experiencing it for the first time and the fear they feel must be extraordinary. My heart breaks for them. My heart breaks for their family.
It feels so weird to feel healthy in a suffering world when for the past few years I felt suffering in what felt like a healthy world.
Showing posts with label Vertex. Show all posts
Showing posts with label Vertex. Show all posts
Tuesday, April 21, 2020
Saturday, August 12, 2017
Race Against Time
Hopefully, you have been hearing some really exciting news regarding Cystic Fibrosis and medicine in general lately. Science is beyond amazing and even my pessimistic side that is adamant that I will never see a cure for CF in my lifetime is starting to fade. And I am certain that young children with CF will never understand what the disease was like for us older CFers and that is an amazing thing!
There is a part of me though that has to hold my breath or swallow my excitement. The newest medications for CF are still years away and even though these very well could stop CF progression in it's tracks I can't help, but worry these meds may come just a little too late. I have pseudomonas, I have scarring and I have a disease that has progressed a lot in my 33 years. I wonder if I will still have these lungs by the time these medications come along. I lost a few friends right before Orkambi became available and know even more that were transplanted before being able to try any of Vertex's meds. I really hope that I can at least try some of these third generation drugs and I hope that I will be around long enough to see them come to market.
Sometimes life with CF can make looking into the future a daunting task. Thinking how hard my lungs have worked and how hard this disease has worked towards destroying my body in the past 33 years it can make the future seem bleak at best. With all this scarring and bacteria how can I fight against CF long term? Despite doing everything my doctors ask of me plus more I still see my lungs struggle just to keep up with this ever growing army of bacteria. The future looks long and treacherous. Thinking long term is frightening and exhausting at the same time. Realistically, given what is available today I can't imagine keeping these lungs long enough to see old agr.
Hearing about these new medical advances in CF allows me to make mini goals to reach for along the way to old agee. They have become a mental rest stop along the way. Maybe I can fight this battle for 5 more years and then I will get these meds to help allieviate my exhausted body from this raging war. Yes, 5 years sounds so much easier than "forever" and I can wrap my mind around making it just 5 more years. It makes the future less exhausting, unachievable and scary. If I keep up this all consuming battle for just a few more year back up will arrive and the long term fight may be a bit more feasible.
There are never any guarantees for tomorrow and this is even more true when you have a progressive disease. I know I may not gain as much as healthier CFers from these medication, but more than anything I just want the chance to try. A chance to live to see old age even if these lungs don't improve. I can manage a pretty nice life with these scarred and battered lungs. For now, I am doing everything in my power to be here as long as possible and a goal of 5 years actually feels doable. So I wake up each morning and I swim and I do all my treatments and I take a few supplements on the side. And I feel I have enough reserves for the next few years. Let's hope Science can meet me half way!
There is a part of me though that has to hold my breath or swallow my excitement. The newest medications for CF are still years away and even though these very well could stop CF progression in it's tracks I can't help, but worry these meds may come just a little too late. I have pseudomonas, I have scarring and I have a disease that has progressed a lot in my 33 years. I wonder if I will still have these lungs by the time these medications come along. I lost a few friends right before Orkambi became available and know even more that were transplanted before being able to try any of Vertex's meds. I really hope that I can at least try some of these third generation drugs and I hope that I will be around long enough to see them come to market.
Sometimes life with CF can make looking into the future a daunting task. Thinking how hard my lungs have worked and how hard this disease has worked towards destroying my body in the past 33 years it can make the future seem bleak at best. With all this scarring and bacteria how can I fight against CF long term? Despite doing everything my doctors ask of me plus more I still see my lungs struggle just to keep up with this ever growing army of bacteria. The future looks long and treacherous. Thinking long term is frightening and exhausting at the same time. Realistically, given what is available today I can't imagine keeping these lungs long enough to see old agr.
Hearing about these new medical advances in CF allows me to make mini goals to reach for along the way to old agee. They have become a mental rest stop along the way. Maybe I can fight this battle for 5 more years and then I will get these meds to help allieviate my exhausted body from this raging war. Yes, 5 years sounds so much easier than "forever" and I can wrap my mind around making it just 5 more years. It makes the future less exhausting, unachievable and scary. If I keep up this all consuming battle for just a few more year back up will arrive and the long term fight may be a bit more feasible.
There are never any guarantees for tomorrow and this is even more true when you have a progressive disease. I know I may not gain as much as healthier CFers from these medication, but more than anything I just want the chance to try. A chance to live to see old age even if these lungs don't improve. I can manage a pretty nice life with these scarred and battered lungs. For now, I am doing everything in my power to be here as long as possible and a goal of 5 years actually feels doable. So I wake up each morning and I swim and I do all my treatments and I take a few supplements on the side. And I feel I have enough reserves for the next few years. Let's hope Science can meet me half way!
Tuesday, April 11, 2017
Tezacaftor/Ivacaftor
I am sure most of the CF community has heard by now, but Vertex's latest medication to treat the underlying cause of Cystic Fibrosis has completed two phase 3 studies and have met their primary endpoints! First and foremost, I am so excited to see the development of new medications and I am especially excited to see that it benefits my mutation (DF508) as well as other mutations. It gives me so much hope to think in the next year (or so) a newer and better drug than Orkambi, that has helped me with stability, will be available. I am especially hopeful this medication will help me raise my lung function, even if only a little.
The other part of me remembers how I felt when Kalydeco became available. I was excited to see advances in CF care. At the same time I felt heartbroken that Kalydeco was not available to me. It came out at an absolutely horrendous time in my health and hearing story after story of this miracle drug that was changing people's lives was painful when I was feeling as if my life was slipping away. I know so many people with CF are feeling those same mixed feelings with this newest Vertex medication. I also know there are many in the world tonight that have the right mutations, but are still fighting for Orkambi so this medication must also feel out of reach.
I do have hope that all of us with CF, regardless of mutation, will have access to drugs that will completely change the course of this disease. I really hope to see this in my own lifetime.
If you want more details you can find the press release here.
The other part of me remembers how I felt when Kalydeco became available. I was excited to see advances in CF care. At the same time I felt heartbroken that Kalydeco was not available to me. It came out at an absolutely horrendous time in my health and hearing story after story of this miracle drug that was changing people's lives was painful when I was feeling as if my life was slipping away. I know so many people with CF are feeling those same mixed feelings with this newest Vertex medication. I also know there are many in the world tonight that have the right mutations, but are still fighting for Orkambi so this medication must also feel out of reach.
I do have hope that all of us with CF, regardless of mutation, will have access to drugs that will completely change the course of this disease. I really hope to see this in my own lifetime.
If you want more details you can find the press release here.
Saturday, March 5, 2016
24 Weeks of Orkambi
Two years ago I tried to get in the Orkambi trials, but after going through all the pre-trial testing I was denied. When I finally received the medication Sept 2015 I was curious as to how my life may have differed today if I had been the trial two years ago (and had the same dose medication that was approved by FDA which would not have necessarily been the case). Would I have been healthier with better lung function? Would my two years of decline and instability have been less "eventful"? There is no way of knowing how things would have been different had I started the trial 2 years ago, but I was still curious as to what my experience on the trial would have been like. So after starting my Orkambi I marked my calendar for 24 weeks (the duration of the Vertex trials) and waited.
I blogged about the first couple of months here if you want more detail as to the rough start I had with Orkambi. The biggest changes from the last 24 weeks of Orkambi:
1. Plugs!
I coughed out my first plug about 2 months into Orkambi and I had no idea at the time, but they would continue to come out at an alarming rate peaking at 4 months. There were days I would cough out up to four or five plugs in a single day. Around 4 months I started to lose sinus plugs at an equally alarming rate. There were weeks that I got either a lung or sinus plug every.single.day and often multiple times a day!
**For reference, prior to Orkambi the only time I would cough out a plug was during a round of IVs and these plugs were usually "newer" plugs that were more gelatinous and lighter in color. Orkambi plugs were dark and were so foul I often gagged as they came out sometimes verging on throwing up. They were clearly bacteria ridden and extraordinarily old. Good riddance!**
2. Sickness
I have had a rough couple of years. There was an entire year that every single time I started to feel better I would instantly catch a cold and be incredibly sick again. It seemed that once I felt well for a few days I would wake up the next day feeling a little off only to be sick with high fevers or completely bed ridden by afternoon. Sickness came quickly and often. I hardly left the house and bathed in hand sanitizer and was still constantly sick. I also ended up with reoccurring infections because my lungs couldn't handle the stress and mucus overload.
Since starting Orkambi I have had the first winter in a very long time that I was NOT SICK AT ALL! (Yes, I shouted that). What is even more amazing is that Kaylee and I have not restricted ourselves due to risk of getting sick like we have in the past. We continued storytime at the library, had regular playdates, and generally acted like normal human beings. And somehow I did not get sick! The most insane part is that I never even touched a bottle of hand sanitizer this winter. I ran out and never replaced it. Clearly, my body working more properly has allowed my immune system to function again (years ago I had a pretty amazing immune system that was so taxed the past few years it seemed to completely stop working).
There were 3 times over winter that I thought I was getting sick (the achy tired feeling is always a red flag that sickness is approaching). All three times I mentally prepared for a hospital stay because the last few years sickness always ended with IVs because my lungs just couldn't handle the overload. But unlike pre-Orkambi somehow I got over whatever was making me feel off. As in, I never actually got sick. This was such a sharp contrast from the past few years that the first sign of ache always left me feverishly shaking in bed within 24 hours.
3. FEV
So unfortunately, I have not seen an increase in FEV1. In fact, my last appointment I was down a little. However we are in the throws of allergy season at the moment which is my hardest season. My allergies present themselves as asthma and my lungs often feel like they completely shut down during allergy season. At this point I do not believe I will be one of those people with CF that see a positive change on FEV1. I won't lie I would absolutely love extra lung function and really wish I would have seen some improvement in that regard.
I am a skeptic when it comes to... well most things. I did not assume Orkambi would be a miracle and I did not assume every positive (or negative) change in my body would be from Orkambi. Our bodies are complicated and CF can be so unpredictable that it makes distinguishing correlation vs causation hard to separate. However, the hundreds of plugs I have coughed out in the past 6 months and the fact that my completely unstable health (especially in winter that usually left me a hermit for months on end) seemed to stabilize (for now) is enough to make this skeptical person confident that Orkambi has greatly improved my quality of life and I am so thankful it came into my life when it did.
I blogged about the first couple of months here if you want more detail as to the rough start I had with Orkambi. The biggest changes from the last 24 weeks of Orkambi:
1. Plugs!
I coughed out my first plug about 2 months into Orkambi and I had no idea at the time, but they would continue to come out at an alarming rate peaking at 4 months. There were days I would cough out up to four or five plugs in a single day. Around 4 months I started to lose sinus plugs at an equally alarming rate. There were weeks that I got either a lung or sinus plug every.single.day and often multiple times a day!
**For reference, prior to Orkambi the only time I would cough out a plug was during a round of IVs and these plugs were usually "newer" plugs that were more gelatinous and lighter in color. Orkambi plugs were dark and were so foul I often gagged as they came out sometimes verging on throwing up. They were clearly bacteria ridden and extraordinarily old. Good riddance!**
2. Sickness
I have had a rough couple of years. There was an entire year that every single time I started to feel better I would instantly catch a cold and be incredibly sick again. It seemed that once I felt well for a few days I would wake up the next day feeling a little off only to be sick with high fevers or completely bed ridden by afternoon. Sickness came quickly and often. I hardly left the house and bathed in hand sanitizer and was still constantly sick. I also ended up with reoccurring infections because my lungs couldn't handle the stress and mucus overload.
Since starting Orkambi I have had the first winter in a very long time that I was NOT SICK AT ALL! (Yes, I shouted that). What is even more amazing is that Kaylee and I have not restricted ourselves due to risk of getting sick like we have in the past. We continued storytime at the library, had regular playdates, and generally acted like normal human beings. And somehow I did not get sick! The most insane part is that I never even touched a bottle of hand sanitizer this winter. I ran out and never replaced it. Clearly, my body working more properly has allowed my immune system to function again (years ago I had a pretty amazing immune system that was so taxed the past few years it seemed to completely stop working).
There were 3 times over winter that I thought I was getting sick (the achy tired feeling is always a red flag that sickness is approaching). All three times I mentally prepared for a hospital stay because the last few years sickness always ended with IVs because my lungs just couldn't handle the overload. But unlike pre-Orkambi somehow I got over whatever was making me feel off. As in, I never actually got sick. This was such a sharp contrast from the past few years that the first sign of ache always left me feverishly shaking in bed within 24 hours.
3. FEV
So unfortunately, I have not seen an increase in FEV1. In fact, my last appointment I was down a little. However we are in the throws of allergy season at the moment which is my hardest season. My allergies present themselves as asthma and my lungs often feel like they completely shut down during allergy season. At this point I do not believe I will be one of those people with CF that see a positive change on FEV1. I won't lie I would absolutely love extra lung function and really wish I would have seen some improvement in that regard.
I am a skeptic when it comes to... well most things. I did not assume Orkambi would be a miracle and I did not assume every positive (or negative) change in my body would be from Orkambi. Our bodies are complicated and CF can be so unpredictable that it makes distinguishing correlation vs causation hard to separate. However, the hundreds of plugs I have coughed out in the past 6 months and the fact that my completely unstable health (especially in winter that usually left me a hermit for months on end) seemed to stabilize (for now) is enough to make this skeptical person confident that Orkambi has greatly improved my quality of life and I am so thankful it came into my life when it did.
Tuesday, January 5, 2016
Four Months Of Orkambi
*This is a long version of my experience on Orkambi*
This is a post I keep starting, but can't seem to get myself to post. To be honest, my Orkambi journey started torturous at best. As much as I wanted to gripe and complain about the sickness that came with this new pill I knew I was unbelievably lucky to have the ability to even try this medicine. I know how many people with CF would love the opportunity to try this medication that was already in my hands and that none of them wanted to hear me complain. At the same time, when I felt as sick as I did, it was hard to feel grateful and lucky for a medicine that I wasn't sure could ever help me. And so I kept quiet and internally kept note of the changes, both good and bad, going on in my body. I am just finishing four months of Orkambi now and feel I have a clearer picture of what Orkambi means for me and my disease.
I started Orkambi feeling cautiously optimistic and well aware that the first month or so would be tough. However, I had no idea what the next several months had in store for me. My symptoms were not that unusual and many people that start the medicine experienced the same side effects. Some of the symptoms that came with this little pink pill were shortness of breath that was so extreme I had trouble walking across the room. In fact, there were times I felt like I was suffocating while sitting still. I had fevers, chills, aches, streaking of blood in my mucus, a cough that never stopped, but didn't feel productive, which meant I hardly slept. I was mentally prepared for these side effects, but I wasn't prepared for how long they would last. I was warned by fellow Orkambi users that the first week is the hardest and the worst of it was usually gone by a month. My symptoms seemed to get worse as time went on and as I passed the first and second month with no relief I started to lose hope that this medicine could work for me.
The physical aspect was tough, but the mentl aspect felt torturous. I dropped from 40% lung function (fev1) to 29% and despite the passing weeks, my numbers wouldn't budge. I worried endlessly as the weeks passed that I was harming myself rather than helping. I was so tight, coughing was not super productive and my exercise consisted of walking in slow motion around the house. I worried that my lack of movement and my tight chest meant that more mucus was pooling in my lungs and that I would end this Orkambi journey worse off than when I began. I felt like I was sicker than I had ever been in my life and I knew I was doing it to myself by taking this medicine. I kept waiting for a sign that things may improve, that my body was still okay, but nothing seemed any better.
And then one evening 2.5 months of worsening symptoms I broke down and cried. I had decided in the morning I would call the doctor and tell him that I just couldn't do it any longer. Physically, I was tired and mentally, I was terrified I was hurting myself and I don't have a lot of lung function to spare (at 29%). Orkambi didn't seem to be the right medication for me.
That night I went to bed and slept through the night for the first time in months. I didn't cough once! The next morning my shortness of breath had miraculously disappeared! It was a complete transformation. It was all I needed to stay on course and continue using Orkambi. My next clinic visit my lung function was 35% and although that was better than the previous 29%, it still wasn't to baseline. Regardless, I felt a huge sense of relief that at least my lungs were moving in the right direction.
During the fourth month I suddenly starting seeing all the changes in my body I had secretly hoped would happen. I started coughing up old putrid plugs from my lungs. Sinus plugs were coming out at an alarming (yet wonderful) rate. I could climb stairs and even run in the yard with my daughter. My mucus lightened in color and was much less copious compared to years past. My mucus came up easily and I started to feel stronger and more sure of my body. my morning cough has completely disappeared. I am starting to see my weight steadily climb despite starting at a healthy weight to begin with.
Clinically, I am not sure my lungs seem much different, but I feel like I just came off a round of IVs. I am in extreme awe of how well I feel without needing a hospital stay and weeks of IVs. I feel amazing and I have done nothing to work for it, no oral meds, no hospital stay, no prednisone, this hardly feels like real life. Orkambi may be considered a marginally beneficial medication and yet I feel stronger, healthier, and my lungs feel clearer. I may not gain lung function or see a huge change clinically, but I am taking something that is making my body work more like it should and that is amazing!
This is a post I keep starting, but can't seem to get myself to post. To be honest, my Orkambi journey started torturous at best. As much as I wanted to gripe and complain about the sickness that came with this new pill I knew I was unbelievably lucky to have the ability to even try this medicine. I know how many people with CF would love the opportunity to try this medication that was already in my hands and that none of them wanted to hear me complain. At the same time, when I felt as sick as I did, it was hard to feel grateful and lucky for a medicine that I wasn't sure could ever help me. And so I kept quiet and internally kept note of the changes, both good and bad, going on in my body. I am just finishing four months of Orkambi now and feel I have a clearer picture of what Orkambi means for me and my disease.
I started Orkambi feeling cautiously optimistic and well aware that the first month or so would be tough. However, I had no idea what the next several months had in store for me. My symptoms were not that unusual and many people that start the medicine experienced the same side effects. Some of the symptoms that came with this little pink pill were shortness of breath that was so extreme I had trouble walking across the room. In fact, there were times I felt like I was suffocating while sitting still. I had fevers, chills, aches, streaking of blood in my mucus, a cough that never stopped, but didn't feel productive, which meant I hardly slept. I was mentally prepared for these side effects, but I wasn't prepared for how long they would last. I was warned by fellow Orkambi users that the first week is the hardest and the worst of it was usually gone by a month. My symptoms seemed to get worse as time went on and as I passed the first and second month with no relief I started to lose hope that this medicine could work for me.
The physical aspect was tough, but the mentl aspect felt torturous. I dropped from 40% lung function (fev1) to 29% and despite the passing weeks, my numbers wouldn't budge. I worried endlessly as the weeks passed that I was harming myself rather than helping. I was so tight, coughing was not super productive and my exercise consisted of walking in slow motion around the house. I worried that my lack of movement and my tight chest meant that more mucus was pooling in my lungs and that I would end this Orkambi journey worse off than when I began. I felt like I was sicker than I had ever been in my life and I knew I was doing it to myself by taking this medicine. I kept waiting for a sign that things may improve, that my body was still okay, but nothing seemed any better.
And then one evening 2.5 months of worsening symptoms I broke down and cried. I had decided in the morning I would call the doctor and tell him that I just couldn't do it any longer. Physically, I was tired and mentally, I was terrified I was hurting myself and I don't have a lot of lung function to spare (at 29%). Orkambi didn't seem to be the right medication for me.
That night I went to bed and slept through the night for the first time in months. I didn't cough once! The next morning my shortness of breath had miraculously disappeared! It was a complete transformation. It was all I needed to stay on course and continue using Orkambi. My next clinic visit my lung function was 35% and although that was better than the previous 29%, it still wasn't to baseline. Regardless, I felt a huge sense of relief that at least my lungs were moving in the right direction.
During the fourth month I suddenly starting seeing all the changes in my body I had secretly hoped would happen. I started coughing up old putrid plugs from my lungs. Sinus plugs were coming out at an alarming (yet wonderful) rate. I could climb stairs and even run in the yard with my daughter. My mucus lightened in color and was much less copious compared to years past. My mucus came up easily and I started to feel stronger and more sure of my body. my morning cough has completely disappeared. I am starting to see my weight steadily climb despite starting at a healthy weight to begin with.
Clinically, I am not sure my lungs seem much different, but I feel like I just came off a round of IVs. I am in extreme awe of how well I feel without needing a hospital stay and weeks of IVs. I feel amazing and I have done nothing to work for it, no oral meds, no hospital stay, no prednisone, this hardly feels like real life. Orkambi may be considered a marginally beneficial medication and yet I feel stronger, healthier, and my lungs feel clearer. I may not gain lung function or see a huge change clinically, but I am taking something that is making my body work more like it should and that is amazing!
Saturday, July 25, 2015
Orkambi Troubles
I am not sure how one quantifies the value of a life. Apparently, some people know the secret equation, but I wasn't lucky enough to be one of them. Although, I do not know exactly what my life's value is I do know according to my insurance company my life is not worth $259,000/year. In other words, my insurance is not covering the new drug Orkambi for any of their Cystic Fibrosis sufferers.
I was actually really surprised, naïve as it was, because my insurance gladly covers the $300,000/year Kalydeco for patients with the correct mutation. I thought that if they would pay a higher dollar amount for one groups of CFers they would pay for a less expensive version for another. We all know life isn't fair and insurance companies rarely make sense so I guess thinking my insurance would cover this medication was faulty logic.
The really horrendous part of it is I am not sure I will be eligible for the Vertex patient assistance program for people who have insurance refusing to cover the cost of the drug. This could mean that unless I magically become extraordinary wealthy, an extra $20,000 a month wealthy, I may never have access to this medication.
The only aspect of this whole situation that is giving me hope is that CF doctors in my state are coming together to fight for their patients so that we can all access this medication. The CF community is full of doctors who genuinely care for their patients and for that we are lucky. The only other fiber of hope I have is that potentially my insurance company is negotiating price and may eventually strike a deal with Vertex. The part that is the most frustrating is that I am being left in the dark. The morning after I found out my insurance is refusing to cover Orkambi through another cyster I called my insurance company, my clinic (the CF nurse is on vacation), my pharmacy and vertex. I got zero response and no answers. Now that it is the weekend I have to wait until Monday to hope that someone will answer their phone to provide me with some answers on where we go from here.
Cystic fibrosis patients wake up every morning from the moment they are born until they die ready to fight. It is the only way to ensure we wake up every single day. I am ready to fight my insurance in any way possible, my life is on the line!
I was actually really surprised, naïve as it was, because my insurance gladly covers the $300,000/year Kalydeco for patients with the correct mutation. I thought that if they would pay a higher dollar amount for one groups of CFers they would pay for a less expensive version for another. We all know life isn't fair and insurance companies rarely make sense so I guess thinking my insurance would cover this medication was faulty logic.
The really horrendous part of it is I am not sure I will be eligible for the Vertex patient assistance program for people who have insurance refusing to cover the cost of the drug. This could mean that unless I magically become extraordinary wealthy, an extra $20,000 a month wealthy, I may never have access to this medication.
The only aspect of this whole situation that is giving me hope is that CF doctors in my state are coming together to fight for their patients so that we can all access this medication. The CF community is full of doctors who genuinely care for their patients and for that we are lucky. The only other fiber of hope I have is that potentially my insurance company is negotiating price and may eventually strike a deal with Vertex. The part that is the most frustrating is that I am being left in the dark. The morning after I found out my insurance is refusing to cover Orkambi through another cyster I called my insurance company, my clinic (the CF nurse is on vacation), my pharmacy and vertex. I got zero response and no answers. Now that it is the weekend I have to wait until Monday to hope that someone will answer their phone to provide me with some answers on where we go from here.
Cystic fibrosis patients wake up every morning from the moment they are born until they die ready to fight. It is the only way to ensure we wake up every single day. I am ready to fight my insurance in any way possible, my life is on the line!
Saturday, July 18, 2015
Anticipation
There is a flurry of emotions whirling through my mind. I feel as if I am teeter tottering between every emotion that a human can possible feel throughout each and every day. Why all this emotional instability? The newest Vertex drug, Orkambi, is the culprit of all this emotional confusion.
The two years of utter free falling into a dark abyss from this disease made me wish with every fiber of my being for Orkambi to be approved. Every wish my heart held so dearly revolved around something, anything that could pull me up, even just a little, from this bottomless well. Now that Orakmbi is here I feel such a mix of emotions I feel I may go crazy with uncertainty.
The optimistic side of me is feeling excitement so strongly that I can feel it buzzing in my bones. The optimistic side of me wants to scream and shout and jump up and down that there is something, anything that may help me maintain the last 5 months of stability for the next several years. The optimistic side of me want to believe that this drug will work well for me and I will gain 4% lung function or more! I want to have more energy to chase and swim and bike with my daughter. I want to gain weight easily, and have fewer aches, and have more stability in my day to day life when it comes to health.
And then a voice, just as loud and adamant as my optimistic voice, comes bursting into my mind. The voice is my realistic (pessimistic?) side and it is reminding me that not ALL people experienced increased lung function and there were a few that experienced side effects that required them to stop the medication (mainly elevated liver levels). Some felt nothing and others had symptoms that were troublesome and even a bit scary like extreme shortness of breath.
To add icing to my troublesome cake I feel paralyzed with fear that my insurance, which has been reviewing my case for over a week now, will deny my ability to even try this medication.
So much of life with Cystic Fibrosis is luck. What mutations you have, how the disease presents itself, what bugs you catch and how much havoc they wreak on your body, how well you respond to meds and if you are allergic to them. Here is just another example of luck. I have heard this drug has been a life saving miracle for some people and for others it is a nuisance not worth their time. How I feel about this medication is anyone's guess and sheer luck just as most things with CF. And of course, luck will determine if my insurance will even cover this $259,000 a year medication.
I just hope that this time, lady luck is on my side!
The two years of utter free falling into a dark abyss from this disease made me wish with every fiber of my being for Orkambi to be approved. Every wish my heart held so dearly revolved around something, anything that could pull me up, even just a little, from this bottomless well. Now that Orakmbi is here I feel such a mix of emotions I feel I may go crazy with uncertainty.
The optimistic side of me is feeling excitement so strongly that I can feel it buzzing in my bones. The optimistic side of me wants to scream and shout and jump up and down that there is something, anything that may help me maintain the last 5 months of stability for the next several years. The optimistic side of me want to believe that this drug will work well for me and I will gain 4% lung function or more! I want to have more energy to chase and swim and bike with my daughter. I want to gain weight easily, and have fewer aches, and have more stability in my day to day life when it comes to health.
And then a voice, just as loud and adamant as my optimistic voice, comes bursting into my mind. The voice is my realistic (pessimistic?) side and it is reminding me that not ALL people experienced increased lung function and there were a few that experienced side effects that required them to stop the medication (mainly elevated liver levels). Some felt nothing and others had symptoms that were troublesome and even a bit scary like extreme shortness of breath.
To add icing to my troublesome cake I feel paralyzed with fear that my insurance, which has been reviewing my case for over a week now, will deny my ability to even try this medication.
So much of life with Cystic Fibrosis is luck. What mutations you have, how the disease presents itself, what bugs you catch and how much havoc they wreak on your body, how well you respond to meds and if you are allergic to them. Here is just another example of luck. I have heard this drug has been a life saving miracle for some people and for others it is a nuisance not worth their time. How I feel about this medication is anyone's guess and sheer luck just as most things with CF. And of course, luck will determine if my insurance will even cover this $259,000 a year medication.
I just hope that this time, lady luck is on my side!
Wednesday, June 3, 2015
Orkambi
As most of the CF world is probably aware of by now, the vertex drug developed for DDF508 has a good chance of becoming available to patients this July. Everyone in the CF community was hopeful this drug targeting DDF508 would be a life changing miracle drug just like Kalydeco was for people with the G551D mutation. Unfortunately, the results of the study quickly dashed many of our dreams that this drug would be our miracle drug.
As much as I am disappointed that Orkambi, which will work on my mutation, is not the life changing and life extending drug that Kalydeco is for a select few with CF. I can say I am beyond excited, despite having all the facts, that we may have another medication that could potentially help slow the progression of my disease. When you live with a progressive disease new treatments are never developed fast enough. In fact, discoveries to treat CF are far and few between. And even when therapies are developed I have found myself allergic and unable to take advantage of many of these medications and treatments. The progression of the disease is always three steps ahead of the treatment and watching your lungs and body deteriorate is heartbreaking at best. It development of treatments are very much a matter life or death for so many with CF. This is why I have to roll my eyes at the naysayers that complain about the humble 3% increase in lung function that were seen in the Orkambi study.
What the naysayers don't realize is that 3% may not seem clinically significant, the truth is that the drug is so much more than 3% increase (on average) in lung function. It is the fact that is can slow the guaranteed progression of this disease. That is something that can not be taken lightly. To me, it means I may have a few more years added to the end of my life to watch my daughter turn into a woman. It may mean one or two more anniversaries I spend with my husband. It may mean another birthday and a few more grey hairs. So yes, 3% may not seem like a big deal to someone who hasn't had an expiration date stamped to their lungs. It may not seem like 3% could change a life, but pessimists are stuck on a low percentage and not the potential for what slowing, even minutely, can do to a life. How a few years can mean so much to a mother, a wife, a sister, a friend who would do anything to hold on to their life for just a little longer.
So yes, lets celebrate the accomplishment of the researchers, the foundation, the CF community that we may (as long as the FDA gives the okay this July) have one more tool in our fight again CF, we may have a little more hope for tomorrow, and we may have a little more time on this Earth.
As much as I am disappointed that Orkambi, which will work on my mutation, is not the life changing and life extending drug that Kalydeco is for a select few with CF. I can say I am beyond excited, despite having all the facts, that we may have another medication that could potentially help slow the progression of my disease. When you live with a progressive disease new treatments are never developed fast enough. In fact, discoveries to treat CF are far and few between. And even when therapies are developed I have found myself allergic and unable to take advantage of many of these medications and treatments. The progression of the disease is always three steps ahead of the treatment and watching your lungs and body deteriorate is heartbreaking at best. It development of treatments are very much a matter life or death for so many with CF. This is why I have to roll my eyes at the naysayers that complain about the humble 3% increase in lung function that were seen in the Orkambi study.
What the naysayers don't realize is that 3% may not seem clinically significant, the truth is that the drug is so much more than 3% increase (on average) in lung function. It is the fact that is can slow the guaranteed progression of this disease. That is something that can not be taken lightly. To me, it means I may have a few more years added to the end of my life to watch my daughter turn into a woman. It may mean one or two more anniversaries I spend with my husband. It may mean another birthday and a few more grey hairs. So yes, 3% may not seem like a big deal to someone who hasn't had an expiration date stamped to their lungs. It may not seem like 3% could change a life, but pessimists are stuck on a low percentage and not the potential for what slowing, even minutely, can do to a life. How a few years can mean so much to a mother, a wife, a sister, a friend who would do anything to hold on to their life for just a little longer.
So yes, lets celebrate the accomplishment of the researchers, the foundation, the CF community that we may (as long as the FDA gives the okay this July) have one more tool in our fight again CF, we may have a little more hope for tomorrow, and we may have a little more time on this Earth.
Tuesday, June 24, 2014
Vertex and CF
So as I am sure you all already know the results from the phase 3 Vertex trial for DDF508 has been released and were positive!!! We are hoping the FDA approves it by end of quarter 2014, come on FDA!!
There seems to be a lot of mixed feeling about these meds which I find bizarre to say the least. Many people, like me, are excited, of course! But there seems to be a very vocal group of CFers that like to point out all the negatives of this potential drug.
Ummmmm, hello!! There are new drugs coming out for CF, how in the world is that a negative thing?? Is this a cure? No way. Will it make our lives normal? Nope. Will we see an increase in our PFTs? Maybe, but no guarantee. Will we get to reduce or stop doing our treatments? Of course not! Is there the potential for negative side effects? Yes, but have you ever taken Prednisone? Most side effects > better than being killed which is what CF does best.
Regardless, we have another tool to fight this awful disease. One more chance to live a longer life. One more defense against lung infections and scarring and permanent damage. It may not work for everyone with DDF508, just like Tobi doesn't work for me, but for those it does work for it is totally worth it.
So today is a game changer! Today I am going to celebrate and be excited about the future for CF patients because we are moving in the right direction and there are other drugs following close behind. We just need to keep hanging on as long as we can so that maybe someday our life expectancy will be virtually normal and we can look back and say, "remember when CF would take so many young lives..."
There seems to be a lot of mixed feeling about these meds which I find bizarre to say the least. Many people, like me, are excited, of course! But there seems to be a very vocal group of CFers that like to point out all the negatives of this potential drug.
Ummmmm, hello!! There are new drugs coming out for CF, how in the world is that a negative thing?? Is this a cure? No way. Will it make our lives normal? Nope. Will we see an increase in our PFTs? Maybe, but no guarantee. Will we get to reduce or stop doing our treatments? Of course not! Is there the potential for negative side effects? Yes, but have you ever taken Prednisone? Most side effects > better than being killed which is what CF does best.
Regardless, we have another tool to fight this awful disease. One more chance to live a longer life. One more defense against lung infections and scarring and permanent damage. It may not work for everyone with DDF508, just like Tobi doesn't work for me, but for those it does work for it is totally worth it.
So today is a game changer! Today I am going to celebrate and be excited about the future for CF patients because we are moving in the right direction and there are other drugs following close behind. We just need to keep hanging on as long as we can so that maybe someday our life expectancy will be virtually normal and we can look back and say, "remember when CF would take so many young lives..."
Tuesday, September 17, 2013
Vertex Trials
I was officially rejected by Vertex! Of course, I was really upset at losing out on the chance to be a part of the study and the chance of getting access to potentially beneficially CF meds. However, after thinking about how everything played out I am actually glad I was not chosen for the study for a few reasons.
First, I am relieved that I can still nurse my daughter because she was not going to give up "boo boo" without a HUGE fight. It didn't help that she was (is) in the middle of getting her molars and all she wants to do is nurse.all.the.time!!!
Second, I desperately needed antibiotics from that last cold. I am back on Cipro and Bactrim which is usually my magic combo. I would not have been able to start the antibiotics right before starting the study. I am hoping this will help me get healthier so that when (if?) the drugs do become available I can get the most benefit from them.
Lastly, I found out at clinic on Monday that our clinic will be doing another Vertex trial for DDF508 in the near(ish) future! If all works out well I will try to get on that study unless they reject me again...
First, I am relieved that I can still nurse my daughter because she was not going to give up "boo boo" without a HUGE fight. It didn't help that she was (is) in the middle of getting her molars and all she wants to do is nurse.all.the.time!!!
Second, I desperately needed antibiotics from that last cold. I am back on Cipro and Bactrim which is usually my magic combo. I would not have been able to start the antibiotics right before starting the study. I am hoping this will help me get healthier so that when (if?) the drugs do become available I can get the most benefit from them.
Lastly, I found out at clinic on Monday that our clinic will be doing another Vertex trial for DDF508 in the near(ish) future! If all works out well I will try to get on that study unless they reject me again...
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