Today, healthcare professionals, research advocates and many people living with rare conditions are coming together to observe Rare Disease Day. It’s all about raising awareness for rare and genetic diseases, improving access to treatments and learning more about what exactly makes a condition rare.
In the United States, a disease is considered rare if it affects less than 200,000 people at any given time. Rare diseases affect almost 1 in 10 Americans, and many times, they cause common symptoms that can be mistaken for other conditions.1
All across the world, people are raising awareness for rare disease. Here are just a few things you can do to join them.
- Wear your favorite pair of jeans today to help the Global Genes Project promote the Blue Denim Genes Ribbon
- Use the hashtags #CareAboutRare and #WRDD2014 and share them with @GlobalGenes on Twitter and Facebook
- Find an event in your state and participate in local activities
- Print out this flyer, take a photo of yourself with it, and submit it to Handprints Across America
Rare diseases have a personal connection with PatientsLikeMe – our co-founders’ brother, Stephen, was diagnosed with ALS (Lou Gehrig’s Disease) in 1998, and their family’s experiences with the condition led to the beginning of PatientsLikeMe. In 2012, we partnered with the Global Genes Project to create the RARE Open Registry Project to help those diagnosed find others like them in one of the over 400 rare disease communities on the site, and launched the first open registry for people with alkaptonuria (AKU) with the AKU Society in early 2013. We also accelerated our focus on enhancing the idiopathic pulmonary fibrosis (IPF) community through a collaboration with Boehringer Ingelheim. And now, the IPF community on PatientsLikeMe is the largest open registry with close to 1,900 members …and counting.
When I first became involved with online communities back in 2002, I moderated a small forum for patients with ALS/MND in the UK at King’s College Hospital to connect with one another, share tips and support, and to help our care center to serve them better. One area that remains controversial even to this day is clinical trials. ALS is a rapidly fatal and incurable illness, and as a young researcher I was often trying to field questions that patients had about the trials process:
“Why aren’t there more trials taking place?”
“Why do we have to have a placebo?”
“If researchers think commonly available treatments like dietary supplements or antibiotics might slow my disease, why can’t I just take them anyway rather than being enrolled in a trial?”
Given the severity of their situation and the slow pace of clinical trials (it’s estimated that it takes over 10 years for a new discovery to go from the lab to the clinic), I certainly sympathized with their frustrations and did my best to get the answers they needed, asking my medical colleagues where I could and translating the technical jargon. A few patients decided to go a step further than challenging the status quo, however, and took actions into their own hands. One patient taking part in a trial sent her medication off to a private lab to test whether or not she was on placebo, and several others took off-label experimental ALS treatments like creatine or minocycline. Some of them even went abroad to China for highly controversial stem cell treatments. In the end, none of these interventions were shown to slow the disease, but it certainly showed what was possible when a smart, dedicated, and adventurous group of patients could organize online.
Fast-forward more than ten years and we see a very different world, where patients have a growing voice in treatment approval, the design of patient reported outcome measures, and increasingly share their trial experiences through social media. That’s what inspired us to write our latest article just published in the British Medical Journal which details our experiences with how members of our community have replicated a clinical trial, have started sharing their data with leading researchers to debunk alternative “cures” for their disease, and have even started taking trials into their own hands. We feel these are symptoms of a greater underlying problem that has been with the design of clinical trials from the start – trials are all take and no give.
Patients take all the risk with their health, their time, their bodies, and in return they are supposed to temporarily suspend their instinctual curiosity to know more about their health status through self-monitoring or to interact with other patients who might be in a trial, all for the good of science and other patients. Modern technology up-ends that power dynamic though, and now it’s almost as easy for a patient to measure their health status with patient-reported outcomes, wearable devices, or even lab tests as it is for their doctor. In that case, can we really say patients are truly blinded anymore? The double-blind placebo-controlled randomized controlled trial is a “gold standard” in medicine, but unless it adapts to the changing realities of the patient empowerment landscape, it will be inherently unsustainable.
That’s why our team is embarking on research this year to gain a new understanding of what patients want from trials and a new understanding of the social contract as patients want it written. Our hope is that we can bring them together with the scientific needs of researchers to conduct robust science. It won’t be easy, and it might not be popular with everyone, but it’s the best path we can see towards faster cures that respects the rights of patients to be partners, not just subjects.
May is amyotrophic lateral sclerosis (ALS) Awareness Month. As many out there know, PatientsLikeMe was inspired by the life experiences of Stephen Heywood, who was diagnosed with this serious neurological disease back in 1998. Stephen’s brothers (Ben and Jamie) made many attempts to slow the progression of his condition, but their trial-and-error approach just wasn’t working. They knew there had to be a better way, and in 2004 PatientsLikeMe was created.
Every year, about 5,600 people are diagnosed with ALS in the U.S. It can affect any race or ethnicity and there is currently no treatment or cure that will reverse or even stop its progression. By getting involved, you can help change that.
You can also find upcoming awareness events on the ALS Therapy Development Institute calendar. There will be picnics, charity golf tournaments and “The Cure is Coming” 5k and awareness walk. And don’t forget, we’d love to sponsor your run/walk team through our PatientsLikeMeInMotion program. Your whole team will get free t-shirts, a donation and more!
Looking for more info on non-profits during ALS awareness month? There are a bunch of organizations dedicated to the cause. A fellow PatientsLikeMe community member put together a great list in his forum thread Comparison of ALS/MND Organizations.
If you’re living with ALS, find others just like you in our growing community of almost 6,000 ALS patients on PatientsLikeMe. Learn what they’re doing to manage their condition with symptom and treatment reports, and share your own experience with a personal health profile or in the community forums.
Welcome to the third installment of our new blog series featuring patient bloggers. Last week, we introduced you to Parkinson’s blogger Steve, and today, we’d like you to meet Rachael, a three-star member who has been a part of PatientsLikeMe’s flagship ALS community since the very beginning. In fact, she joined PatientsLikeMe in May 2006, just two months after being diagnosed with this progressive neurological disorder (also known as Lou Gehrig’s disease). Her blog is called “Notes to Self,” and it’s a candid and charming journal of the full life she leads, despite her condition.
1. What role does your blog play in your life?
As the title suggests, my blog is [filled with] “Notes to Self.” I use it as an aide memoir and an excuse to be verbose as my voice fails me. It is useful to direct people who ask about events in my life when repetition of the story would be tiring. I also find that it helps me to work through my often complicated emotions in dealing with the disease and its incumbent challenges.
2. Tell us about using an eye gaze system to write your blog posts and more.
As I have blogged on many occasions, the Eye Gaze System [a communication system directed by eye movements] is invaluable to me. I was fortunate enough to get the system before my voice and keyboard dexterity gave up entirely. I have become used to using it, and the transition has been less traumatic than it otherwise might have been. Low-tech communication is all very well in an emergency but cannot compensate for speech.
The Eye Gaze allows me to converse on an almost level footing, conveying thoughts and observations, expressing myself in my own style with all its complexities and idiosyncrasies. The Eye Gaze also allows me to indulge my “inner geek” and access the Internet and Skype to communicate with far flung friends and family.
3. You’re a big user of the InstantMe tool at PatientsLikeMe. Why?
I have always been quite an obsessive character. I am possibly overworking the PatientsLikeMe facility. I find it of immeasurable help to have a diarized record of my progression as it relates to my daily life. I tend to ramble, so I find the strict regimen of 140 characters helpful.
A while back, I worried about my memory loss. I had a test done, and it showed no signs of the frontal lobe dementia that terrified me. My consultant reassured me that my short term memory loss was within “normal” parameters. To me it was still unacceptable. Because I have so many small things to remember that people without this condition would find insignificant, something has to give. Having a log helps my recollection. It is also useful for caregivers to reference and saves me effort and energy that I often do not have. It makes my clinical review a whole lot easier and accurate.
4. In your last post, you talk about reconnecting with friends you’d pulled away from. Tell us about that.
When I was first diagnosed, I was given the usual prognosis “18 months to live”. Some friends ran away but I realize now that I also withdrew from many people in my struggle to come to terms with the disease. 18 months has come and gone a number of times. Over time I had to re-evaluate my response. Instead of planning for dying I needed to plan for living. Part of this included giving people the opportunity to be involved in my life.
It is, on reflection, a patronizing thing to withdraw friendship without at least offering the choice. Life with ALS is difficult, but the challenges are not insurmountable. I thoroughly enjoy my life and my friends both old and new. Anyone reading my “InstantMe” [history] will know that I have an active social life and a busy home.
For patients with prevalent diseases, it may be easy to find others with your condition. You meet them at clinics; you run into them when seeing your specialist; or you participate in one of the support groups in your area. For those with rare diseases, the simple act of finding another patient like you isn’t always as easy. You might be the only patient your doctor has seen with your condition. Finding another patient often becomes a goal and sharing and learning from them a welcomed reward.
Alongside NORD and EURORDIS, we are celebrating Rare Disease Day and they’ve deemed this year’s theme “Rare, but Equal.” At PatientsLikeMe, patients are patients, no matter what their condition. Patients with rare diseases are sharing their health information alongside patients with more widespread conditions.
So, who do we have sharing information about their rare disease? To date, more than 455 patients with Multiple System Atrophy and 122 patients with Progressive Supranuclear Palsy, both neurodegenerative disorders that mimic Parkinson’s disease, have joined our community. Do you have Neuromyelitis Optica, the autoimmune inflammatory disorder affecting the spinal cord, optic nerve, that has lesions often misdiagnosed as multiple sclerosis? There are 332 patients just like you. Sharing right alongside these patients you’ll find 388 patients with Progressive Muscular Atrophy (a rare subtype of ALS which only affects the lower motor neurons) and 331 with Primary Lateral Sclerosis (a subtype of ALS which affects the upper motor neurons).
Many of you also know that we actually started PatientsLikeMe focused on the rare neurodegenerative disease, ALS. Six years later, there are now more than 4,000 ALS patients-plus almost 20% of the newly diagnosed in the U.S. every month-sharing their journeys and learning from one another. (You can read about highlighted milestones in our 2010 ALS Awareness Month blog.) In 2011, we’ll continue our heritage of serving those with rare diseases by improving this overall experience of finding a “patient like me.”
There are no major awareness raising pink ribbons or yellow wristbands for these rare diseases. But, there is a group of patients who have found each other, who are sharing with one another and the world their disease experience. And, that will translate to accelerated research and better outcomes – two things we are hoping to make a little less rare.
Do you wonder if your cold legs could be a sign of something? Sensations of coldness in the extremities – such as the feet and toes – may be the result of poor circulation, neurological disorders or temperature dysregulation.
At PatientsLikeMe, where more than 120,000 patients are sharing their experiences with conditions, symptoms, treatments and more, approximately 137 patients report cold legs below the knee. The majority rate this symptom as either “moderate” (42%) or “severe” (31%). Interestingly, many of these patients list their primary condition as ALS (Lou Gehrig’s disease) or multiple sclerosis (MS), showing that this disconcerting symptom is common in these two neurological diseases (both of which can impair mobility).
Two treatments that our patients report for cold legs are a powered wheelchair and a handicap/disabled parking permit. What have you tried? If you’ve experienced cold legs yourself, we encourage you to share your experiences to help other patients. PatientsLikeMe’s unique data-sharing platform allows you to share detailed data about all types of treatments, interventions and lifestyle modifications that have helped you or simply had no effect.
JOIN PATIENTSLIKEME TODAY
Got something you’d like to tell others who are experiencing cold legs? Join PatientsLikeMe and add your experiences to our growing body of knowledge. Then, stay to exchange support and advice, research common treatments and learn from other patients like you.
As you know, we like to just check in with our members to see how you’re doing as a person, not just a patient. Today, we have a very special interview with one of our earliest members with ALS, SmoothS. We sat down with SmoothS to talk about his recent building of the first ever ALS rehabilitation center – the Leonard Florence Center for Living, (LFCL) – which opened in November of 2010. Along with the interview, below is a video of the grand opening event. Have a good weekend!
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The medical term for a heightened or increased sense of smell is hyperosmia. Also known as olfactory hyperesthesia and hyperesthesia olfactoria, the word “hyperosmia” is a combination of the Greek work “hyper,” meaning “above,” and the Greek word “osme,” meaning “sense of smell.” Put together, hyperosmia is an abnormally increased sensitivity to smells and odors.
At PatientsLikeMe, where more than 120,000 patients are sharing their experiences with conditions, symptoms, treatments and more, approximately 100 patients report having a heightened sense of smell. The majority rate their hyperosmia as either “moderate” (42%) or “severe” (28%). Interestingly, their primary conditions range from ALS (Lou Gehrig’s disease) to multiple sclerosis (MS) to migraines, showing that this disconcerting symptom can occur for many different types of patients – and not just pregnant women.
Searching for this phenomenon in our forum discussions, several interesting things are revealed. For example, some patients report that strong smells have triggered their migraines or seizures, while others say their increased or heightened sense of smell is so acute that it’s like “tasting” the odors. Several patients report experiencing “phantom odors” that no one else is able to detect, and at least one says she started smoking cigarettes to help mask the intensity of smells around her.
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Can you relate to any of these first-hand reports? These are the kind of insights you can only glean from other patients who are also experiencing a heightened sense of smell. So if you have hyperosmia, and you’d like to learn more about it, join PatientsLikeMe and connect with others like you today.
Our ALS Community recently broke the 5,000-member mark, making it the largest of its kind in the world as well as the ideal platform for expedited research. And that’s just what we’re working on.
Since the community’s launch in 2006, we’ve conducted a number of research studies, both internally and in collaboration with leaders in the field. The goal is to turn up the answers that patients like you are seeking. For example, can lithium slow the progression of ALS? Or does limb dominance (aka “handedness”) correlate with ALS symptom onset? Check out the findings from our patient-led Lithium and ALS Study as well as our work with the University of Oxford on handedness.
We also like to think of the dynamic, up-to-the-minute reports on our site, such as our treatment reports and symptom reports, as another form of “research.” Every day, patients just like you contribute to these reports by sharing your real-world data, including what symptoms you’re experiencing, how you’re treating your condition and how well your treatments are working for you.
Now, the data you share is helping even more people. Through an exciting new partnership with ALS Untangled (ALSU), we are helping to take the data you share straight to the scientific literature – and at record speed. ALSU is an international consortium of clinicians and researchers seeking to investigate alternative and off-label therapies for ALS using the Internet, namely Twitter, Ning and PatientsLikeMe. Through these modern methods, they aim to provide “timely, accurate and scientifically valid analysis of alternative and off-label therapies.”
As they wrote in their mission statement, there are three phases to their cutting-edge approach:
- In the learning phase, they use their Twitter feed (@ALSUntangled) to collect ideas for potential therapies to investigate from patients just like you. (Got something you want investigated? Suggest it today!)
- Then, in the discussion phase, they import these targeted therapies into a closed Ning group and gather information on them from the consortium, including PatientsLikeMe.
- Finally, in the public release phase, they publish their findings as open-access articles in the prestigious journal Amyotrophic Lateral Sclerosis. That means patients like you, caregivers, clinicians and researchers all over the world can read these reports in entirety for free.
Our first collaboration with ALSU focused on the use of Low Dose Naltrexone (LDN) for ALS. The consortium analyzed data from the 31 members of PatientsLikeMe taking LDN to determine if there was evidence of benefit. What did they find? Efficacy data shared by these 31 ALS patients suggested that most did not notice any benefit, and side effects included headaches, nausea and diarrhea. On this basis, along with a review of the literature, ALSU concluded that it does not recommend LDN for ALS patients at this time. Read the full LDN study here.
Going forward, we hope that you will continue to share your evaluations of alternative and off-label treatments for ALS so that they can inform the world’s top researchers. We’re thrilled that your data is being taken seriously by the wider medical community, and we encourage you to participate fully in this novel research project.
We continue our series this week of getting to you the person, not just the “patient.” Today’s interview is with “iceberg,” a member of our PatientsLikeMe ALS Community and another 2010 newsletter interviewee. Read on to learn more about what keeps him motivated, how his condition has affected his work life and how he took part in the PatientsLikeMeInMotionTM program. Enjoy!
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|(Amy) What keeps you motivated?|
I have always been self-motivated. I have been running since my high school days, running cross country and track in high school. I started to run again in 1978 and even ran a marathon in 1990 when I was 40. I continued to run until 1995 when my foot drop became so severe I began to trip myself.
My wife says I am just too bullheaded to give in to ALS. I think watching my grandson grow up is my motivation too. In 1998 when he was born, I didn’t know if I would see his first birthday. He is now 11.
|(Amy) Who do you admire and why?|
|(iceberg)This is a hard question to answer. I admire anyone who has ALS and continues to battle to live. When I travel to Washington, D.C., for Advocacy Days and see PALS in wheel chairs with breathing tubes and feeding tubes and see how they are fighting to live, it makes me admire them. “Never Give Up.”
|(Amy) How has your condition affected your work life?|
|(iceberg) I was a Toll Collector on the Pennsylvania Turnpike until August 23, 2009, and I am now on disability retirement. I have gradually lost the use of my thumbs over the last several years. I was no longer able to keep up with wrapping coins and counting change back to the customers. So I guess you would say it has affected my work life quite a bit.|
|(Amy) What’s your favorite aspect of PatientsLikeMe?|
|(iceberg) PatientsLikeMe has afforded me an opportunity to meet a lot of people with ALS who I have become friends with over the past several years. Some of the people that I have talked with on PatientsLikeMe I had the pleasure of meeting in D.C. over the past several years. PatientsLikeMe also supported our walk team this past summer for Walk to Defeat ALS. [See photo on left] My thanks to PatientsLikeMe for the ability to make new friends and talk about how ALS has affected our lives.|
|(Amy) No, thank you for being so willing to help others learn from your experiences with ALS, iceberg!|
The research team is very proud of how our team has grown in 2010 and the impact we’ve made in the academic community. In 2010 alone, PatientsLikeMe and our work were mentioned in scientific literature more than 133 times. All of this is because of what you share with us.
Yesterday, we highlighted some of the research work we’ve done in 2010 across our four newest communities. Today, we’ll highlight our older communities. Below is also a highlights reel of some of this work.
- Throughout 2010, we discussed the goal and benefits to measuring your quality of life (QoL). This concept of measuring QoL started in our HIV Community at the end of 2009. Earlier this year in a blog and podcast, Researcher Michael Massagli Ph.D. discussed some of the insights that are being shared by HIV patients through the QoL survey. For instance, we found that the average physical, mental and social well-being score of patients with a CD4 count below 200 is significantly lower than for those of you with a count above 200.
- The Parkinson’s Community has always excelled in reporting their health status through the Parkinson’s Disease Rating Scale (or PDRS). When answering the questions on this scale, members often asked us: “Should I respond when my drugs are working and I’m at my best (‘On’) or when my drugs aren’t working and I’m at my worst (‘Off’)?” Back in October, we released a brand new feature in the PD community that allows you to rate your symptoms as either “On,” “Off”, or in both conditions so now you can see the effect of your medications for yourself.
- We hear a lot from you about the difficulty of adhering to medication. Early last year, our research team developed a new rating scale for patients with MS to assess the difficulties of sticking to their medication. We presented findings at the Consortium of Multiple Sclerosis Centers (CMSC), the main North American conference for MS specialist physicians, nurses, and researchers. The team is currently working on publishing these findings.
- Co-founder Jamie Heywood discussed our research into lithium in ALS at the TEDMED conference late in 2009. In early 2010, TEDMED released the video of his presentation where he shares our vision of the future of medicine. The team is currently working on publishing the lithium findings.
- PatientsLikeMe, in collaboration with researchers at Oxford University, discovered that when ALS patients get symptoms in their arms first, they’re most likely to get it in their dominant hand. This research was presented at the ALS MND Symposium and cited on this MND Research blog.
- We also collaborated with the Northeast ALS Consortium (NEALS) to examine the decision-making process that patients with ALS go through when considering whether or not to take part in research studies; this was published in the peer-reviewed journal Amyotrophic Lateral Sclerosis.
Here’s a short video highlighting much of the research you’ve helped us do in 2010.
Finally, I want to recognize how our team has expanded this year to better focus on what matters to you most. Some new additions include:
- Brant Chee, Ph.D. – a specialist in natural language processing and detecting drug safety data in patient reported text
- Kate Slawsky, MPH – an outcomes researcher helping to develop custom surveys for our partners and our platform
- Shivani Bhargava – a research assistant supporting the team in ensuring our platform holds high-quality data about our patients
Thank you all for having a voice in research. There’s so much being learned from what you are sharing every day, and we are excited about what 2011 will bring.
Last week, we posted highlights from our December community newsletter interviews. As we close out 2010, we want to spotlight another question asked this month – one that is relative to all members, all patients (no matter the condition). What obstacles have you faced and overcome this year?
To review all of our newsletters, you can visit our archives page here.
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|(Amy) What obstacles have you overcome this year?
|(ellieGADsufferer – Mood Conditions Community)
First of all, I became well enough to leave my local psychiatric hospital after having been an inpatient for six months. My anxiety means that all new things – even getting on a bus to go somewhere new – make me have severe panic symptoms.
I now make sure I am out of bed by 0700hrs during the week and make myself go out at least three times per week, whether it is meeting friends for coffee or attending appointments. Otherwise I will return to “avoidance,” whereby I cut off all contact with friends and family, including calls and text messages.
|(kidneygirl1198and0505 – Transplants Community)
I have had to deal with and overcome the struggles that come with tardive dyskinesia. Reglan has been black-boxed [with a FDA-mandated warning about the risk of tardive dyskinesia] over the past two years, but I took it for three or four years before that. [Read kidneygirl1198and0505's Reglan treatment evaluation for more details.]
Signs of tardive dyskinesia may include: continuous and repetitive movements of the mouth, tongue, and jaw; facial grimacing; lip smacking, puffing of the cheeks; uncontrollable movements of the arms, legs, fingers, and toes; and swaying motions of the trunk or hips.
Mine has gotten so bad that I have regular grand mal seizures from time to time, and sometimes it’s hard to carry on a conversation without looking weird.
|(mountain lady – Parkinson’s Disease Community)
One of the biggest obstacles in my life this year was getting over the death of a very close friend. This person became ill and died within a few months. Her death made me more aware of how precious life is.
Another obstacle was in the adjustment of my medicine for Parkinson’s. My doctor wanted me to try a new medicine with fewer long-term side effects. I tried it for several months but did not do very well. I am currently back on my original medicine, taking a little more than before, but I have readjusted well.
|(andrewn78 – HIV Community)
This year I overcame a hard breakup. It was difficult because I thought we were going to be together for a longer term relationship than we were (only nine months).
It was also difficult to start dating again. I forgot how many people don’t know much about how HIV is contracted and that there are many people who would never date someone who is living with HIV or will date someone living with HIV but the fear of contracting HIV puts too much of a damper on the relationship. It’s a difficult process.
|(Tommy Maker – ALS Community)
I’ve spent a lot of time this year getting used to the fact that I have become dependent on others.
Prior to diagnosis, I sat on the local council, was involved in the organization of all types of public elections and I was a trade union official. These were voluntary roles which involved helping others. Yet, initially, after diagnosis, I had an enormous difficulty accepting help from others. However, I’ve come to realize that I have a loving family, fantastic friends and very supportive colleagues. I have found that many of them don’t know what to do and are very glad when I am specific as to what they can do for me.
I’ve also learned that no question is too personal. I struggled a lot with questions about personal hygiene and when I found the courage to ask these most intimate questions, I found that the professionals were so familiar with the questions asked that, beyond the initial embarrassment, we quickly went on to find solutions. I have also found that when people ask questions about my condition, they are entitled to an answer, an honest answer.
|(Nates-Sweetpea – Fibromyalgia/CFS Community)
Oh my, 2010 has been a huge year! I didn’t know how I was going to get through it whole, but here I am knocking on 2011, one step at a time. I had to rejoin the workforce on a part-time basis this year, after years of not working (not being able to work), but I had to as my husband had lost his job about 18 months previously, and we were facing bankruptcy!
So I’ve been working part-time since last February, about 17 to 32 hours a week. We did go through bankruptcy, lost our home, vehicles and our high 700′s credit score with it. But you know what, I survived. Actually, no, I overcame, with the help of a very supportive Sunday School class, great friends, and a wonderful husband and daughter.
|(kg10043 – Epilepsy Community)
I work for the US Forest Service and, as of July, made it to retirement age still working. Two times they tried to force me to take a disability retirement. To keep working I have had to move around to several jobs in four different states because of my health. Not only have I made it to retirement age, but I am still working even though they are trying to get rid of me once again. (What a joke when you consider that the federal government has a quota for disabled employees).
It has meant I have not only had to work in a variety of locations, but doing different kinds of work. Just in the time on this forest I have worked as the Oracle DBA in the computer shop, in GIS, later in Forest Planning, with a crew of five Soil Scientists, a Botanist and myself mapping the soils and ecological types and writing recommendations for management. And now for the last two years I have been “unfunded” and doing all kinds of random jobs for projects in the office. Just this year that has included supporting the USFS in legal challenges as well as jobs so simple as filing paperwork and addressing envelopes.
|(cobebu8 – MS Community)
For me, with multiple sclerosis and ADHD, my main problems have been the concentration to do what I need and want to do and fighting the progression of MS. I have RRMS [relapsing remitting MS] but since I have had it for 25 years, ever so slowly, it is progressing at the same time I get exacerbations. I have a new neurologist too, and he is part of a big group and getting through when I am having problems, is a problem.
Plus, with only SSDI [social security disability insurance] and not being able to find a part-time job, I have had to stop taking some needed meds and not going to necessary doctor’s appointments (e.g. my eye surgeon/doctor to see if the optic nerve is still swollen and I can’t do a necessary MRI because the insurance company won’t pay.) So to get help for my asthma, I am doing a research study to get free care. I haven’t found one for MS or depression yet.
In December community newsletters (launched earlier this week), we asked some of our members to think about what they’ve shared and compared in 2010. Below are highlighted answers from each interviewee across all nine community newsletters. Thank you for your contributions.
We also want to thank all of you who have contributed to the 90 newsletters we ran this year, including the newly launched ones in our Epilepsy and Transplants communities. Finally, a special thanks goes out to our newsletter writer, Amy Morton, for pulling these together every month.
To review all of our newsletters, you can visit our archives page here.
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How has PatientsLikeMe helped you learn and share this year?
|(Tommy Maker – ALS Community)
PatientsLikeMe has provided me with new friends-people who are experiencing the same problems as I am. I’ve learned that there isn’t a single question that won’t get a vast myriad of answers from the community. I’ve learned that we’re all very different people, and I’ve learned we don’t always agree. I’ve learned that we all care enormously for each other and are very eager to help those who find themselves in the same boat as we are. But most of all I’ve learned that I am not on my own. I’m not the only one facing a bleak future and not the only one who is able to appreciate what he has at the moment.
|(cobebu8 – MS Community)
I really like it here in that I can keep track of all my meds with their side effects, etc. Plus I like it that I can list symptoms and have a history of all of it to review and learn from and share with my doctors. I hope that my input on meds, therapies, etc., helps other people. It is good not to be alone.
|(mountain lady – Parkinson’s Disease Community)
It has given me the experience of reading about others who are in situations similar to mine. It has shown me that I am not alone. I love reading the newsletters and the comments from other readers. It also helps to keep me up-to-date on any new suggestions or treatments.
Also, I became acquainted with someone online through PatientsLikeMe. We actually live in the same town, both work in the field of Education and have had Parkinson’s for the same length of time. Getting together with her was a pleasure, and she introduced me to the idea of meeting with a support group, which I had never done before.
|(ellieGADsufferer – Mood Conditions Community)
It is invaluable. I find the Symptoms and Treatments areas so helpful. I have learned (from genuine people who are taking these meds) that some of my “quirky” side effects and sufferings from the meds I take can and do happen to others and are not imaginary. (My general practitioner and Consultant just brush the list aside and shrug their shoulders).
I have learned that I am not alone with my illnesses, which is good to know, and I have also learned that there is “light at the end of the tunnel.” I try to keep my profile as up to date as possible in the hope that it will be helpful to at least one or two members. I rate my Daily Mood [now called InstantMe] at least five days per week and always complete my Mood Map, again for the same reasons.
Well done PatientsLikeMe and its members. Keep going, you are doing really good work and long may it continue.
|(andrewn78 – HIV Community)
I love organization and having PatientsLikeMe help me to organize all of my medical information. I would like to have an ability to log simple notes on the PatientsLikeMe profile so every time I go to my doctor I can make some general notes (if needed) for that visit on my experience and what was covered with my provider.
(Note that all users can now use InstantMe to add notes/annotations to your Doctor Visit Sheet)
|(Nates-Sweetpea – Fibromyalgia/CFS Community)
PatientsLikeMe has been such an encouragement to me, not only in what others have given to me through sharing their stories, but also by opening up a window for me to be an encourager also. I love the newsletters that are super reminders of how to not get off track, and also how to be good to myself.
|(kg10043 – Epilepsy Community)
I think I have been pretty good at sharing, but this has been one new method, especially with some of the health issues with others facing similar situations. In the past I have answered questions for people who had their first seizure or just questions, but with this site, I am able to communicate with others who have had the same operations, take the same medications, etc., and really understand things better (from a different perspective).
|(kidneygirl1198and0505 – Transplants Community)
PatientsLikeMe has helped me so incredibly much! I’ve made so many friends that I thank God for every day. It’s just like, no matter what you have or haven’t had transplanted, whichever organ you need or have, everyone on here understands you and what you’re going through.
I just really hope that people can say that about me. It took me awhile, but no one is alone out there. Everyone is going through their own personal struggles. I just want everyone to know I am all ears!
“A measurement based on a report that comes directly from the patient about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.”
The self-report questionnaires we use on PatientsLikeMe to measure your health (such as the mood map, ALS-Functional Rating Scale/ALS-FRS, and other rating scales) are all examples of PROs, and they’re designed to accurately reflect the level of disease severity for a particular condition. Contrast PROs with the results of a blood test or an MRI scan; these are measured by someone other than the patient and are interpreted by healthcare providers. When a disease is relatively well understood and can be measured directly, as in HIV, measurement can be performed with objective measures such as blood levels (e.g., CD4 count and viral load). However, for many disease there is no objective measure for a disease (e.g., fibromyalgia). That means that trials and other clinical research studies are dependent entirely on the report of the patient themselves through PRO instruments. The FDA has recently released a new report: “Guidance for Industry: PRO Measures: Use in Medical Product Development to Support Label Claims.” This report provides guidance on the design of new instruments, and evaluation of old instruments, to let scale designers know how to get their PRO taken seriously by the FDA, with a view to making claims on the prescribing label. This report has many positive aspects which we believe will improve the quality of PROs in research. For example:
“Sponsors should provide documented evidence of patient input during instrument development and of the instrument’s performance in the specific application in which it used (i.e., population, condition).”
- We’ve always believed strongly that PROs should come directly from the patient’s experience, and we already consult extensively with patients (as well as clinicians and researchers) in the design of each new community. We’ve also done novel research this way, collaborating directly with a group of previously unmeasured ALS patients to ensure their PRO scale was able to reflect their experience with advanced forms of their disease.
“Characteristics of the PRO instruments that are reviewed by the FDA include the following: Number of items… data collection method… administration mode… response options… recall period… respondent burden… translational / cultural adaptation availability.”
- These are all important factors in PRO development that are frequently overlooked. For instance, a survey that takes too long to complete will tire patients out and could adversely affect the quality of the data. A survey that depends on a patient being highly literate or wealthy could bias findings and make the tool unusable in the developing world. When patients complete our PROs on the site, there is no healthcare provider nearby they can consult for clarification; we work hard to ensure our questions can be easily understood and are unambiguous.
“We encourage instrument developers to make their instruments and related development history available and accessible publicly.”
- Although only “encouraged” and not mandated, this is a step in the right direction. Too often new scales are developed (or old ones modified) without clear justification or transparency. Here at PatientsLikeMe we are planning to embark on our own efforts to validate our existing scales, some of which are widely-used gold standards, others of which we developed in-house. Where possible, we’ll publish our methodology to the scientific community so that others can benefit from what we’ve developed.
“Because statistical significance can sometimes be achieved for small changes in PRO measures that may not be clinically meaningful (i.e., do not indicate treatment benefit), we encourage sponsors to avoid proposing labeling claims based on statistical significance alone.”
- This is an important point. If we created a pain scale which went from 0-100 (0=no pain, 100=unbearable pain) and gave it to enough patients in two groups (say, a thousand in each group), then it’s quite likely we could come up with a small but statistically meaningful difference. However, just because it’s statistically significant, is it really worth the cost, the risk of side effects, and the treatment burden to go from 80/100 to 78/100? This part of the guidance reinforces this notion and will encourage investigators to fine-tune their instruments for responsiveness. We’re already examining our existing scales to establish what we call “minimally important differences” (i.e., how much of a change on the scale is really a meaningful change as opposed to random variation in the measure?).
The report also contains (as an appendix) a sort of “how-to” recipe for submitting evidence in support of a PRO. We are fully support this guidance and look forward to the day when PROs developed from data on PatientsLikeMe can be used to support label claims for clinical research.