My Age Prevents Me From Participating in Clinical Trials
Next month is the five-year anniversary of when I first heard the words “idiopathic pulmonary fibrosis.” I was diagnosed by a pulmonologist at a local respiratory care center, where I’d finally been referred after 13 months of dealing with persistent shortness of breath, dry cough, and fatigue. I try not to think about that day, but when I do, the memory is as vivid as ever.
Shortly after my IPF diagnosis, I made the conscious decision to be an active participant in my healthcare. I would challenge my physicians by doing my own research, asking questions, and complying with the treatments that felt right for my body and lifestyle. I also chose to become an advocate for people living with not only IPF but all interstitial lung diseases, especially those who are young.
Part of being an advocate is choosing to share your story and be vulnerable about some of the hardest aspects of living with this life-threatening lung disease. I’ve done that through writing for Pulmonary Fibrosis News for nearly the whole time I’ve had IPF. I have also shared my story with a New York Times best-selling author who has written a beautiful book about a young adult with IPF called “Pack Up the Moon.” The book will be released in June, though I’m currently reading an advance copy of it.
These are just a few ways I’ve chosen to share my story and the experience of living with a life-threatening lung disease as a young adult. I am OK with being vulnerable and letting others know about the difficulties of IPF, even when sharing stirs up big emotions.
Recently, I was asked about my experience with clinical trials as an IPF patient. Many rare patients view clinical trials as a source of hope, a possibility to develop something that helps make our lives easier. Hope is important in the rare disease community, but I’ll save that topic for another column.
Unfortunately, I have not had any experience with clinical trials because I am always excluded based on my age. When I was diagnosed, my doctor said he didn’t know how my IPF was going to behave, because it was unusual for someone so young to be diagnosed with this lung disease.
In many ways, I ended up with the short end of the stick.
Clinical trials and my inability to participate stir up some of those big emotions I mentioned earlier. Not only am I excluded based on criteria listed on clinicaltrials.gov, I’m also never presented with any clinical trial information during my outpatient appointments or hospital admissions. I have to go searching for the hope that clinical trials bring to rare disease patients, including those with IPF. It’s never presented to me.
In the five years that IPF has been part of my life, I’ve been privileged to interact with many other patients through both my columns and the PF News Forums. We talk about clinical trials a lot, and not only does participating in them bring hope to other patients, but they also help others live with our disease. The information obtained from a clinical trial, even one that isn’t successful, is often helpful to researchers and medical professionals or can be applied to future trials. Being excluded from participating also makes me feel like I’m unable to help other IPF patients.
To answer the question about my participation in clinical trials, I wish I had an experience to share. I wish my participation in a trial left me feeling hopeful, or that I would be helping the trial organizers better understand IPF for future generations. Unfortunately, that isn’t the case because of my age, which is just one of many disadvantages of living with IPF as a young adult.
What has your experience with clinical trials been like as a PF/IPF patient?
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Note: Pulmonary Fibrosis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pulmonary Fibrosis News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to pulmonary fibrosis.
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Comments
Dian cunningham
Thank you,I am a 69 year old RA patient with IPF, caused from methotrexate, just had year long flair, getting better on prednisone and starting myfortec ,thanks for sharing your experience
Charlene Marshall
Hi Dian,
Thanks so much for reading my columns and reaching out via the comments. So sorry you're also dealing with this cruel lung disease, and I hope your flair up is starting to get better with the prednisone. Thinking of you and take care!
Char.
Denise Taylor
In the months since being told by doctor I have IPF 2 years ago, I have deteriorated to the extent that I am constantly breathless and on 02 24/7 and now can only leave my bed to take two steps to my commode. At the time the doctor told me the problem, I asked about lung transplant. No chance was the reply. I was 79. Shock all round. I belong to a support group and it appears most of the members are getting out and about, holidays abroad, walking weekends. None seem to be as bad as me. I'm pleased for them.
Charlene Marshall
Hi Denise,
Thanks for reading my columns and reaching out via the comments. I'm so sorry to hear of your IPF diagnosis and the subsequent deterioration of your health; this disease is so cruel. No one can unfortunately understand how difficult it is to be short of breath unless they have experienced it. I'm glad you've found support via the groups, but I can imagine seeing others progressing slower than you must also be frustrating. Are you able to get a second opinion just to ask if your disease is progressing rapidly or if there is anything else to slow it down, like one of the two anti-fibrotic drugs? Just a thought, but sorry to hear this has been your experience.
Charlene.
Mike Montague
Hello Charlene,
Thanks you for your advocacy. Iwas diagnosed with IPF at 61. Still, I can't imagine what you are going through. I will look for "Pack up the Moon" when it comes out. The title is intriguing.
I have a question about your age-related clinical trial experience. My extended family has over a dozen of us with IPF (living & dead) and we were involved in a clinical study. It involved people from age 20 -70 and age was never said to be an issue. It was not a drug trial, so is that what you are excluded from, a drug trial?
Charlene Marshall
Hi Mike,
Thanks so much for reading my columns and reaching out via the comments. Sorry to hear of your IPF diagnosis, but thank you for your kind words on the book! I'm reading a copy of "Pack up the Moon" now and it is very good; an epic love story of a young adult torn apart from this stupid disease we have. Kristan did a good job helping raise awareness of the cruelty of IPF for anyone who reads it!
Good question re the exclusions. Yes, so far my experiences have all been drug trials I've not been able to participate in due to my age. I would be able to participate in the social/emotional aspects of this disease, if that were ever studied (and I think it should be!) but so far have been excluded from all drug trials. Was the clinical study you and your family were involved in looking at genetics or the family linkages?
Char.
Randall Thornton
Charlene, hello. This is Randy Thornton and was just reading your column and couldn't believe how you where shut out do to being to young. I would think the doctors would want you in them to help you and just maybe find a cure or at least some way to stop this disease. I am not allowed do to the fact I already only have 1 lung, my air test I only had a 37% result and you need at least 50% and I also have heart problems. So far, what my doc has been able to recommend for me is holding the devil at bay. Had a cat scan last week and I am holding steady. I am just praying it stays at that.
Take care and have a good weekend.
Charlene Marshall
Hi Randy,
Nice to hear from you again, thanks so much for reading my columns and reaching out via the comments. Yes, unfortunately many of the criteria's for IPF-related clinical trials include age and start at 40. You'd like researchers and physicians would like to understand IPF in young people, but many don't believe it even exists in those under 40, so it's almost as if there isn't a big enough sample size (in their mind) to include us in trials. I can assure you know several young adults living with IPF that would be willing to participate if we could be included.
I'm really glad to hear you're holding steady where things are at, I'll send a prayer and all my good wishes that things stay this way for you for a long time.
Take care,
Char.
Randall Thornton
Evening Char, Good to hear from you again. This age thing still has my baffelled. Yes, my cat scan was encouraging to know I am at least holding steady. I am now trying my best to do things outside now that it is get nicer. I figured out a way to get more air line length out of my oxygen consentrator. I wanted to get to the front yard flower bed to clean it out. If I run the line thru the window instead of around the bedroom wall, I have enough. So I did that this afternoon. Fells great to be doing something useful. Well take care and look forward to some more from you on your blog.
Charlene Marshall
Hi Randy,
So nice to hear from you again as well, thanks for writing! Glad to hear the CT scan showed you're remaining steady. I always say that is equivalent enough to good news for me, and I'll take it. So glad you're able to be outdoors enjoying the weather; that does wonders for me as well. Enjoy the spring, it always helps lift my mood. Keep in touch and stay well!
Char.
Deborad
I was diagnosed with pulmonary fibrosis in2018.i telling you it has been a struggle I am 60now.My activity stopped and I want to get back to enjoying life.De
Charlene Marshall
Hi De,
Thanks so much for reading my columns and reaching out via the comments. It's nice to hear from you, but sorry about your PF diagnosis. It is a tough disease to life with! Hang in there and reach out for support as you need it.
Charlene.
stella Burnside
I am inspired by the contributions to this web. I was diagnosed with ideopathic PF two years ago and am using Oxygen for activity since then. I have also been diagnosed with Emphysema and therefore I have a strong compensatory Forced Vital Capacity which means that I am not eligible for anti-fibrotic treatment until that measure of FVC drops considerably. Given that I require Oxygen now I dread to think what stage of deterioration I will have reached when I am eligible for anti fibrotic treatment. The NHS will not fund the anti fibrotic therapy and the cost is beyond reasonable cost. I am interested to know the experience of others.
Charlene Marshall
Hi Stella,
Thanks so much for reading my columns and reaching out via the comments. I'm glad you find our website helpful, but sorry you're also dealing with this cruel lung disease and managing life with supplemental oxygen. Is your diagnosis idiopathic (meaning they don't know the cause) or is it related to the Emphysema? I know they can be closely linked but I don't understand the relationship. You're most welcome to join our PF forums Stella, it is a great platform for patients to discuss concerns about IPF (including the costs of anti-fibrotics) and share resources that might help. You can sign up for free here: http://pulmonaryfibrosisnews.com/forums/
Take care,
Charlene.
William Kracke
Charlene- I have participated in 2 clinical trials since my diagnosis in 2014. The first turned out to be unsuccessful and I found out after the study that I was randomized to placebo, the luck of the draw. The second was the Galapagos trial that was shut down about a month ago for safety. I don't know what treatment I was randomized to for that one yet. For that trial, I had an almost opposite problem qualifying for the study. One of the qualifying criteria was being diagnosed within the previous 5 years. The study physician managed to get all the per work and qualifying tests done with about 4 weeks to go before my fifth anniversary.
I worked for a major pharmaceutical company analyzing clinical study data for safety reviews and submissions to the FDA. One little thing that I learned was that the reason for all the qualification requirements was to make all the randomized groups as uniform as possible, focusing around the expected users if the drug was approved. However, I also learned that the sponsor of the trial can, and does, make exceptions to the qualification criteria when the exception won't seriously upset the randomness of the trial. The key to getting an exception is to have the study physician make the case to the sponsor along with other factors such as expected compliance to the study parameters and what a great subject you would be.
So basically what I am trying to say is don't give up. Especially if you find a study that interests you and an exception would not be a major departure from the requirements. Work closely with the study physician and try to convince him/her that you would be a great patient because of your connections with the community that might help recruiting. And don't stop until the physician says that the sponsor was contacted and said "no". Treat it as a job interview where you don't meet all of the requirements but know you could do the job well.
Good luck.
Charlene Marshall
Hi William,
Thanks so much for reading my columns and reaching out via the comments. I appreciate hearing your experience with clinical trials, as I think we can all learn so much about one another as fellow patients. I also appreciate you sharing your learnings, I'll add this to my back-pocket of knowledge if I am ever considered or want to join a specific clinical trial. Thank you again for sharing!!!
Char.
Joe
Dear Charlene,
How we feel for you. Us older sufferers may reluctantly accept our situation as part of the risk of getting so far in life.
I sympathise with your desire and efforts to be included in a trial. To feel part of aiding the science and understanding of our disease.
In my 70th year I was quite fit, a good bmi, low cholesterol, drank infrequently, a lifetime non smoker and running with my training partner of 30+ years for 2 one hour sessions each week over our beautiful hills.
Following a bout of pneumonia in 2013, possibly caused by an adenovirus, then a slow recovery, eventually identified IPF.
I, like you, wanted to offer myself for any trial or any research programme. As I regarded myself as a 'healthy' example who would willingly participate. I made that known. Whilst It could be that the NHS system in the UK is good for mass medication, it is not turned on to feeding people into the research sector. As my older sister was then diagnosed with IPF I thought it even more important. My efforts might have resulted in my being prescribed OFEV on a 'compationate basis' in early 2015, prior to it being approved by NICE in the UK. In the end in late 2019 I eventually got an offer for the Galapagos trial. Unfortunately, my condition had progressed, I failed several components in the entry requirements.
Then Covid crisis kicked in, and so here we are.
For all those with IPF time is of the essence. For long term sufferes the time to develop a treatment is probably too far into the future.
I feel for all fellow sufferers, but our greatest sympathy is reserved for all those in the younger part of our community.
All that I can add is to keep rattling their cages! Or the old expression, 'the squeeking wheel gets the grease'!
Live in hope.
Kind regards,
Joe
Charlene Marshall
Hi Joe,
Thank you so much for reading my columns and reaching out via the comments. It's so nice to hear from you and I really appreciate your kind words! I love your comment about continuing to rattle cages; that is what I want to do when it comes to helping the researchers and doctors better understand IPF in young adults and include in trials, etc. Thank you again for writing :)
Char.
DOCTOR DON
Hello Charlene:
Thank you again for all you do. I’m so grateful as a patient with IPF and also as a retired physician. You talk about being an advocate for your health and I couldn’t agree with you more. I pushed my docs to get a VATS lung biopsy as i knew enough to know that IPF and hypersensitivity Pneumonitis (my initial diagnosis) are treated so differently. After 2.5 years on OFEV I’m hanging in there. Throat clearing is my biggest issue.
De—I called the Healthwell Foundation today (800-675-8416). Even with Medicare—the co-pays for the year will run close to $10,000!! Their only two criteria regarding granting you assistance are:
1) You have insurance.
2)If you’re income (for me filing solely) is <4x the poverty level which comes to $51,520 (higher if filing jointly)!!
So if it is deemed that you need an anti-fibrotic—there are foundations out there. There is also the TAF Foundation (855-649-8623) and PSI (Patient Services Inc)(800-366-7631).
I hope that helps.
Charlene Marshall
Hi Don,
Thank you so much for reading my columns and reaching out via the comments. I so appreciate your kind words!
I couldn't agree with you more re: the importance of advocating for your healthcare and questioning or challenging your physicians (respectfully, of course) with your own knowledge is important. Glad you got a definitive diagnosis and are doing okay. Thanks for the information on Healthwell Foundation as well, I know they have helped many patients navigate the anti-fibrotic challenges including cough!
Char.
Debbie Wuest
Hi Charlene, I'am 56yrs old and was just diagnosed with IPF last month. It has been devastating news. I lost my mom and two cousins with IPF. I was told mine was caught in a early stage and I wouldn't qualify for any trial studies either.
Charlene Marshall
Hi Debbie,
Thanks for reading my columns and reaching out via the comments. I'm so sorry to hear you were just diagnosed with this cruel lung disease, and that it's already caused you to suffer losses. It is relentless and unfair! The good thing (if looking for the silver lining) is that it was caught early and hopefully this means faster intervention, ie. Ofev or Esbriet, should you choose those and close monitoring to consider lung transplantation. Thinking of you, and do reach out anytime.
Charlene.
Angel
Hi Charlene, I’m 20 years old and was recently diagnosed with progressive fibrosing interstitial lung disease secondary to acute interstitial pneumonia (induced by antibiotics).I don’t know what my lung function test results were like prior to my illness, but my most recent test showed an FVC of 34% and DLCO of 21% which is always great to hear. My doctor said I’d likely die if I caught a virus, and may only have 2 years left to live. Despite my horrible results I am surprisingly not on oxygen 24/7, but I require 2L/pm on prolonged exertion.
I was perfectly healthy before October of 2020, so it’s been a bit of a shock to my system suddenly having to deal with a chronic, likely fatal lung disease. Due to my age and otherwise healthy body the hospital has strongly advocated for me to go on the lung transplant waiting list, but I’m really hoping something to potentially reverse pulmonary fibrosis or at least stop it is on the horizon (maybe 5 years from now) especially due to the sudden increase in people with lung disease secondary to COVID. Unfortunately my doctors seem to think I won’t survive that long but I still have hope.
I also do not qualify for a lot, if not all the pulmonary fibrosis clinical trials I’ve seen due to the ‘newness’ of my diagnosis, my age and my lung function tests. Even though I don’t wish more people my age had chronic fibrosing lung diseases, I wish that trials would at least decrease the minimum age range to 18 for outliers like us.
Sorry for spilling my life story. As much as it sucks, I feel more at ease when I can read about people close to my age with similar, rare issues.
All the best,
Angel
Charlene Marshall
Hi Angel,
Thank you so much for reading my columns and reaching out via the comments. That said, so sorry to hear of your diagnosis after a bout with pneumonia, that is awful. This disease is cruel and relentless in who it targets. Despite what your doctors think, it is so important to hold onto hope, even though I know it isn't easy at times! You are right: a lot more research and medical attention is being given to fibrotic lung diseases since COVID-19 and people are finally starting to realize the importance of research in this area. I can also assure you, knowing many of them personally, that there are some incredible people in this field, trying to help us with this illness... hang onto hope because of them! :)
Feel free to email me anytime: [email protected] if you want to talk more; I rarely get the opportunity to connect with younger patients and some of the initiatives I am focusing on now is removing the assumption that this is only an "old person's" disease. I also just corresponded with a 37 year old tonight! Take good care, and please keep in touch. Getting involved in the PF community really helped me, so if you're interested in doing that, please let me know.
Kind regards,
Char.