199 posts in the category “Conferences/Events”

Paul Wicks on the power of sharing data

Posted April 5th, 2017 by

Back in December, our VP of innovation, Paul Wicks, Ph.D., spoke at the annual International Symposium on ALS/MND in Dublin. The goal of the annual meeting is to share new understandings of ALS/MND as quickly as possible through collaboration between leading researchers around the world.

Now, we’re sharing a video of his presentation about what you and the ALS community has shared about living with ALS over the last 10 years.

Here’s what Paul had to say:

“We hope this presentation shows the power of patients sharing their data to help shed light on ALS. It’s a disease that can isolate people, rob them of their voice, and sap their strength to fight back, so we’re proud that we’ve been putting the data our members share to good use for over a decade now and we’re keen to share some highlights of what we’ve found.”

What do you think about Paul’s presentation? Share your thoughts in the forum.

Share this post on Twitter and help spread the word.


“I really felt that we were heard” — PatientsLikeMe staff member Dan shares his experience at the FDA psoriasis conference

Posted August 2nd, 2016 by

August is Psoriasis Awareness Month, and we’re kicking things off with a recap from the FDA’s public meeting on psoriasis back in March. The meeting was part of their Patient-Focused Drug Development Series that aims to bring the patient voice to research.

Sally Okun, Vice President for Advocacy, Policy and Patient Safety at PatientsLikeMe notes that, “The most effective part of the meeting was the patient stories. Even though a lot of quantitative data has been gathered by the FDA, they’re learning firsthand how difficult it is for these patients. Having an event that’s so patient-centric – where people are telling their stories and those attending can submit questions through the webinar – really lets patients make their voices be heard.”

The PatientsLikeMe psoriasis community also gave feedback through a survey in the weeks before the meeting, and this data was shared with the FDA. Check out the full report of what members had to say.

And Dan, one of our community moderators here at PatientsLikeMe, attended as both a member of our staff and a patient. We caught up with him to chat about his experience. Here’s what he had to say:

What was it like to attend this event as someone living with psoriasis?

I have had psoriasis for 23 years. I wasn’t alone, as my sister and brother had very mild symptoms, and my father and his brother had more moderate symptoms. So for me, having the disease itself wasn’t scary from a medical point of view. But I certainly suffered from social stigma as plaques cover all of my legs, arms and much of my torso.

As I learned from meeting my peers at the PFDD psoriasis conference, many psoriasis patients tend to hide their symptoms as best as they can by wearing clothes that conceal it, and by not participating in activities that would expose it to the public. I, too, was very challenged by the fact that my condition was so visible.

I also have ADD and an acquired learning disability (auditory delay due to an early childhood fever), on top of which I experienced a fair amount of trauma, which resulted in PTS.

I think this is probably why my symptoms were so bad since psoriasis is closely linked to stress and mental health. As a teen, I was grasping for coping mechanisms and esteem builders and I found solace and comfort in climbing, swimming, and hiking outdoors. When I started to experience my psoriatic symptoms, I couldn’t imagine giving up these activities. And so, I simply had to endure and explain my disease to everyone. I rarely, if ever, met anyone else who allowed their psoriasis to be as visible, and I felt extremely isolated. Not to mention my peers also had little or no exposure to people living with this condition aside from myself, thus increasing my issues with stigma.

Going to this meeting was amazing; I was surrounded by patients just like me for the very first time.

It felt incredibly rewarding! However, because I’d spent so much effort over the years suppressing my issues with stigma and the discomfort of psoriasis, it was challenging to process the emotions that came flooding back as I listened to the other patients’ experiences. I was almost brought to tears more than once. Not just for my peers and their suffering, but in recognizing a new awareness about my experience.

My peers were all gathered here to advocate and to express their hardships regarding psoriasis, and in the process they demonstrated incredible personal strength in dealing with this condition and speaking up about it. I was really impressed and inspired.

I also realized how strong I had become by managing the symptoms and stigma with little to no peer support for so many years. This in itself was also a very powerful experience; incredibly validating.

What were the most talked about issues at this meeting?

Much of the meeting revolved around two main issues: the pain and discomfort of psoriasis, and the stigma of having a visible and disfiguring illness. But also many interesting aspects of the patient experience were brought up. For example, nearly every African American present as a patient reported disparities in diagnosis. They all reported that their physicians were not aware that African Americans could even have psoriasis, leading to years of medications prescribed for infections they never had, side effects, and feeling that the medical system had completely failed them.

The prominent topic was around the levels of pain and itching that people experience with this disease, but then it migrated to include discussion of the cognitive and emotional toll of the illness. The level of patient suffering was palpable and very powerful, and sent a strong message to the FDA and the community attending.

Some people experienced constant burning feelings, like their skin was on fire. There were descriptions of feeling like their skin was encased in a cast of plaques, stiff, and uncomfortable, as well as sharp pangs of the open cracks and sores, which any movement or itching created. For many the pain was crippling.

There was also a lot of mention of the stigma experiences from patients recalling their experience as young children and adolescents, managing the emotional challenges of growing up combined with having a misunderstood and stigmatizing skin condition.

Psoriasis manifests in flakey white scales on top of fiercely inflamed red skin, which often cracks open and bleeds. Most people that we meet in our daily lives have no idea what psoriasis is, and are often afraid to even ask. This can lead to us being treated like we are infectious. In many ways it’s hard to blame them, the affected areas often look like Hollywood made-up zombies…

We also we leave bits of ourselves everywhere in the form trails or even piles of silvery flakes on chairs, under our desks, all over the house, and the cracked inflamed skin often leaves blood spots on any light colored clothing, bedding, and furniture.

I have always stepped past my shame and used the opportunity to apologize for the gross mess that I leave because it gave me an opportunity to educate people about what I have so hopefully they wouldn’t be scared of me, but many others in the group reported having a really difficult time talking about their illness with others.

Many coping mechanisms were described like avoiding dark clothes where the flakes would be very visible, or avoiding light clothes as they could highlight the blood stains. Many people also talked about always wearing long sleeves and pants, and going so far as closing the cuff’s of their shirts and pants with adhesive tape to prevent the flakes from spilling out everywhere in public.

Everyone also described this constant emotionally taxing vigilance of trying not to itch or scratch our affected skin as it makes more flakes, which is hard because of the overwhelming itchiness and burning pain. To compound this, many patients in the room had psoriatic lesions on their buttocks and genitals. Can you imagine being in work, or class, out shopping and being consumed with trying to manage the urge to stick your hands in your pants and just itch away? A tragic reality that results in shame, distraction, and, in the end, exhaustion.

Then there is the ever-present social stigma risk mitigation; always shaking out your collar, and sweeping flakes off one’s shoulders away so you don’t look like your are a walking trail of dandruffy, diseased skin.

Even when we are surrounded by educated and empathetic people we know or at least project that they are all affected by our appearance and the messes of “bio waste” we leave in our wake, there is this constant knowledge that we are less desirable as friends, lovers, and even just associates and co-workers; as one panelist reported that they felt as though people treated them as if the were a leper. It’s nearly impossible to disassociate our identity from our disease, because everyone sees our disease right in front of them.

There were a lot of reports of absenteeism from things like school, work, and even fun social events like going to the beach; anywhere public where the emotional effort of managing stigmatization 24/7 was simply too much to handle on a day-to-day basis. There were even questions brought up about whether we were more susceptible to STDs. The meeting facilitator at the FDA explained that they had never heard so many patients attribute such levels of emotional and cognitive fatigue, and brain fog/mental exhaustion to psoriasis. She seemed to be recognizing this as a new and disabling symptom. The FDA was listening!

In the end, we heard each other’s voices amplifying the realities to the FDA and the public that psoriasis clearly affects people on very deep levels; the obvious physical pain and itching, as well as the social stigma fears; the internal shaming and awkward external conversations we have with everyone we meet about our bodies and how our bodies corrupt everyone else’s living and work spaces.

I think the prevailing message was this isn’t a painful and uncomfortable skin issue, this illness touches every aspect our lives especially our mental health.

The irony is, like all autoimmune disease, psoriasis is exacerbated by stress, and having psoriasis causes a lot of stress. I don’t think the medical community has ever really quite understood the interplay between these symptoms and conditions.

Did you feel like the meeting was patient-centric and that your voice was being heard?

I really felt that we were heard. Everything in the program was designed to capture the patient’s voice.

They had this amazing system set up: the facilitator or the FDA staff would ask a panelist a question, and, as soon as they received their answer, they would turn to the audience and ask us the same questions in a multiple choice format. The community listening online would respond from their devices, and the patients in the audience all had received small wireless handsets allowing us to provide our experience. They would then project the results on large screens positioned around the room allowing us to analyze the answers in real time, often prompting the FDA staff to ask follow up questions so they could really gain an in depth understanding of the issue at hand.

You attended as both a PatientsLikeMe employee and a psoriasis patient. What was it like to engage in something like this with an organization like the FDA with that kind of dual perspective?

I think this was the most difficult part for me for two reasons. I wanted to make sure that I was an active participant as a psoriatic patient, and I also was trying to network with other patients and providers to let them know about PatientsLikeMe, and capture the experience so that I could relate it to other patients like me who may attend future events. It was certainly challenging to do all three at the same time.

Going to this event and participating as a patient meant so much to me, knowing that I’d be communicating my experience to these senior staff members of the FDA who will be guiding the future of psoriasis therapies. And hearing other people’s stories about their experience really helped me understand more about my own experience. I realized that I had never really fully processed the challenge of having a stigmatizing visible condition. I also realized that I had a strength that I had not recognized — although my psoriasis has been very challenging, I have managed to live a very full life and have been able to manage the emotional toll that psoriasis exerts. I also felt a little absolved: Has my depression and fatigue been partially fueled by my psoriasis? Have I been shaming myself for years for not overcoming these two issues, when they were not moral failings but part of a larger systemic health condition associated with my autoimmune disease?

On the whole, I’m very glad I went to the PFDD conference on psoriasis; it affected me profoundly and I think that the FDA really was listening and absorbing the experience of the psoriasis patient community. I was proud to be there and I was inspired by the strength of my peers. I really thank PatientsLikeMe for providing me with this rare once-in-a-lifetime opportunity.

 

 Share this post on Twitter and help spread the word.

Getting “Patients Included” right Part II: Planning a patient-centric event

Posted November 18th, 2015 by

You may remember Part I of this blog that focused on the experiences of two PatientsLikeMe members who attended the Kidney Health Initiative’s (KHI) workshop, “Understanding patients’ preferences: Stimulating medical device development in kidney disease,” back in August. KHI is a partnership between the U.S. Food and Drug Administration (FDA) and the American Society of Nephrology (ASN). We recently spoke with patient Celeste Lee and Frank Hurst, MD, Medical Officer, Renal Devices Branch with the FDA, about the planning and consideration it took to make this event “Patients Included.”

While this was the first patient-centric event KHI has held, they’ve worked to include the patient voice in all aspects of the initiative. Kidney patients and kidney patient organizations are represented on the KHI Board of Directors, and this past year the KHI Board of Directors formed a KHI Patient and Family Partnership Council (PFPC) made up of only patients and their caregivers. The PFPC helps provide strategic guidance on how to engage and include patients, their families and care partners in KHI activities.

Celeste has had kidney failure from an autoimmune disease since she was 17. She’s been an advocate for decades and is now focused on patient-centered care. Celeste is also a board member on the inaugural PFPC and helps review potential projects from a patient and family member viewpoint.

“The way KHI works is that it brings everyone to the table – researchers, industry professionals, patients – and we ask what is it that we can do to improve research and clinical trials and ultimately, patient lives. We do this through specific projects like this workshop,” she says.

Involving patients from the get-go

As part of this particular workshop, KHI wanted to hear patients’ ideas and preferences on new devices to manage kidney disease. Before anything, though, they had to create an event that would provide the greatest value to patients that attended in person.

When we asked Frank and Celeste what goes into planning an event like this  they shared how they think it can be centered around the patient:

“Involving patients early helped us to realize the need to broaden efforts to educate patients on the topics of interest prior to having the workshop. This proved to be a critical step in the planning process,” says Frank.

“We realized it would save time to educate prospective attendees about the new devices via webinars before the workshop,” explains Celeste. “We ended up taking a three-step approach that started with a quick engagement video talking about what we wanted to do. We distributed this throughout the whole kidney community. At the end of the video, there was an invitation to sign up for the webinars. After the webinar we said – now we are going to have a day and a half workshop and we will provide travel grants. Over 50 travel grants were given, funded by KHI so patients could come from all over the country.”

Frank notes, “Although patients are medical device consumers, they rarely have an opportunity to influence products that come to market. The success of a new medical device is based on many factors, including the usability by patients. KHI provided a forum, which allowed stakeholders to hear about ideas and potential solutions directly from patients.”

Looking at it from all angles

While the main consideration was making sure KHI had set clear expectations to patients who attended from the onset, there were additional logistics to consider for the workshop to be as patient-centric as possible. The workshop agenda was arranged around patient treatment schedules and incorporated dietary considerations when planning the menu. Because some attendees are on dialysis or live with transplants, they needed volunteers on hand. KHI planners also made sure to ask for patient feedback throughout the entire event and had scribes in position to record it. This feedback was ready to be shared at the workshop’s closing and will be sent out in an executive summary as well.

“Patients especially enjoyed the small group sessions,” Frank says. “These were multi-stakeholder breakout discussions which tackled important questions such as unmet needs, device areas that need improvement, making clinical trials more patient-friendly, and assessing ways for patients, industry, and regulators to communicate and share feedback.  These sessions included many lively discussions where patients felt empowered to share ideas and come together to propose solutions.”

Patient advice for a patient-centric event

Celeste has simple advice for other organizations that want to have this level of patient inclusion in their events. “I think you start off with a really good planning team that includes patients so that they’re there to help figure out the challenges of bringing that population together. Most importantly – you need to prepare people to be a part of it. You’re not going to get anything of value if people come in cold. It’s about the patient being able to draw on their experiences to help move research forward so if they understand what’s expected of them going in, then the outcomes will be more valuable.”

Frank adds, “It is also important to consider the spectrum of the disease, and ideally include patient representatives from across the spectrum as they could have very different needs.”

“Then,” Celeste says, “the next step is getting them to share the developments within the greater community. Once patients are educated and engaged, they become empowered.”

For a look at the KHI’s 3-step plan, check out this presentation they shared with us! And of course, don’t forget to visit the site and connect with the more than 1,000 other PatientsLikeMe members living with chronic kidney disease.

Share this post on Twitter and help spread the word.


#ChatActChange: empowered to chat, act and make change – together

Posted November 17th, 2015 by

On October 28, Sally Okun, our Vice President for Advocacy, Policy and Patient Safety, participated in a Twitter chat – using the hashtag #ChatActChange – aimed at empowering people living with chronic conditions to advocate for policy change. Hosted by Diabetes Social Media Advocacy (DSMA), other chat participants included policy expert Susan Dentzer, Diabetes Hands Foundation and Novo Nordisk.

The chat generated some great conversation about what it means to be an empowered patient. 186 people joined the discussion, including patients, diabetes influencers and policy experts. 2015-2016 Team of Advisors member Christel, who is living with type 1 diabetes, took part in the chat, as did the American Diabetes Association.   

More than 500 users were reached with the #ChatActChange hashtag, and there were more than 1,154 tweets during the one-hour Twitter chat.

To check out a complete summary of the chat, take a look at this Storify. You can also contribute to the ongoing conversation on Twitter using #ChatActChange.

Let’s be empowered to chat, act and make change – together.

Share this post on Twitter and help spread the word.


Getting “Patients Included” right Part I: Two members attend a Kidney Health Initiative workshop

Posted November 4th, 2015 by

Back in August, the Kidney Health Initiative (KHI), a partnership between the U.S. Food and Drug Administration (FDA) and the American Society of Nephrology (ASN), held a workshop called “Understanding patients’ preferences: Stimulating medical device development in kidney disease.” But this was more than a workshop – it was an event centered around the idea of “Patients Included” – a movement started back in May to involve more patients on the planning committees, stages, and in the audiences of medical conferences.

Sally Okun, our Vice President of Advocacy, Policy and Patient Safety spoke at the event and notes how patient-focused the entire workshop was in that “nearly 100 people among the approximately 150 who gathered for the event were patients living with and managing kidney disease every day, many joined by their caregivers.”

“The patients were very open in the discussions and direct in their questions. Many talked about their experiences with hemodialysis and how difficult it is to live a normal life when one has to be at the dialysis center three days a week for many hours,” she says. “In contrast to the conventional treatment approach, the newer developments for hemodialysis at home were very interesting, and in general, patients felt more in control.”

She concludes, “The KHI did a remarkable job focusing this meeting on patients and their caregivers, and providing resources to cover travel expenses. They should be commended and looked to by others as an example of getting ‘Patients Included’ right.”

PatientsLikeMe members Samantha-Anne (internettie) and Laura (Cherishedone) both received stipends to attend the workshop. We asked them about their experiences and here’s what they said:

Why was it important for you to attend this workshop event?

S: It is important to me as a patient to have a voice in my care and treatment. It was particularly important to me to attend this workshop because I wanted to be a voice for the patient who has not yet arrived at the need for dialysis or transplant and to ask what we can do to prevent chronic kidney disease (CKD) from progressing if possible. I know that not all cases can be prevented, but there are some that can. In my situation, my CKD was caused by the use of nonsteroidal anti-inflammatory drugs (NSAIDs). If the consequences of using these drugs for such a long period of time had been made clear to me and if I had been offered some alternative treatments I may have made different treatment choices.

L: I have always been strongly committed to advocating for myself and others, and being given the opportunity to participate and then bring back the information to others whose lives are impacted by CKD and/or end stage renal disease (ESRD) equips us with insight to better advocate for ourselves.

Is this the first event like this that you’ve attended?

S: Yes, this is the first time that I was offered the opportunity to have a voice in patient care. It certainly will not be the last.

L: I have attended National Kidney Foundation (NKF) meetings with other patients and also participated on NKF committees (Patient and Family Executive Committee).

How did you feel about the focus on patient-centricity at this event?

S: I was thrilled to know that someone cared about what patients have to say about their own care. I think that with the advent of the Internet and patients having more access to information that they are more involved in their own care. Patients know more and want to know more about what illnesses are affecting their bodies and they also want to know what they can do to prevent some of these conditions from happening in the first place.

L: The patient-centricity was one of the most valuable components of the meeting. I am one to be very involved in giving back, but I would guess that for many, their disease leaves them feeling isolated. For this population to meet others who are not only surviving but thriving is very important. My hope is that many participants were encouraged to not let their CKD/ESRD define them, but rather to use it as a vehicle for being empowered and encouraged to live each day to the fullest extent, not be left feeling like a victim to the disease.

Did you feel like they supported you and listened to you while you were there?

S: I absolutely felt like I was listened to at this workshop. I had an opportunity to talk to so many people on all different levels of health care. I had wonderful conversations with Paul Conway (AAKP), Mark Ohen (Gore), Denny Treu (NxStage), Sally Okun (PatientsLikeMe), Prabir Roy-Chaudhury, MD, (ASN) Frank Hurst, MD (FDA), Francesca Tentori, ME (ARCH), and F.P. Wieringa, PhD (DKF). We all had the shared topic of kidney disease to discuss but I was amazed at how many people I had other areas in common with. Paul Conway knew my new hometown of Waterville, Maine, and Denny Treu was familiar with my former home in Colorado Springs, Colorado. I feel like my love of research and my desire to learn new things and to share my experiences made this conference a catalyst for me to not only offer my view but to be asked to participate in conversations that I would not be privy to otherwise. I felt like I was engaged in the process and made to feel that my opinion was valued.

L: Most definitely!

What stood out to you as an attendee?

S: What stood out to me the most was that these doctors and vendors really wanted to hear what the patients had to say. I never felt like someone was asking for my opinion just to be nice or listening just to be polite. They seemed fully involved in what we, the patients, were saying. Being heard, as a patient, is honestly such a rare thing that to have people in places that can make life-changing decisions about our care hear what we have to say, is amazing.

L: Hearing about research that is ongoing as well as new technologies was very empowering, and left me feeling very encouraged about options available to those of us who have reached ESRD.

What was the most interesting takeaway for you?

S: As a patient the most interesting takeaway was that what I think and what I say really does count. As a person who loves research and used to do surveys and metrics for a living, the most interesting takeaway was that taking the time to craft a meaningful survey for patients is important. Turning data (survey results) into action (making a difference for the patient) is key.

L: Hearing about research that is ongoing for new technologies and ways to treat ESRD was most exciting.

What was it like to be able to interact with other patients who were attending?

S: It always helps to know that you are not alone. Even though we all have our own individual paths and we all fall on different places on the CKD continuum, we all have so much in common in how we feel and how we need to be heard. We can laugh at things that other people just would not understand. Kidney disease is not funny, but there is always some humor to be found in any difficult situation and being with like-minded people is good for the soul. Every single patient in that room is a hero. We all have a lot on our plate but we mustered up the strength to get ourselves to that conference to not only help ourselves but to help all those who come along after us. I know I was completely energized by being around everyone and at the same time could feel the drain of the illness that I deal with every day. It was worth every bit of energy it took to be there in Baltimore and I feel incredibly grateful that I was offered the opportunity to attend the conference. I made sure to thank everyone who played a role in getting me there and also to thank the other patients for sharing their stories and their thoughts and ideas.

I have been in my new community, Waterville, Maine, for about 2 1/2 months now. Going to the KHI conference in Baltimore made me realize that I need to be involved in my new town. I found a group walk in the downtown area (Waterville Walks! hosted by Waterville Maine Street), I attended a public health meeting as a member of the community (Healthy Northern Kennebec), and I participated in a workshop down in Hallowell, Maine at the Harlow Gallery (Healing Through Art: Confronting Your Inner Critic). My goal is to find out as much as I can about the resources that are available in this area. I understand that input from members of the community really is important and that the people asking for the input want to hear what people have to say. I am not surprised that so few people attend these community events thinking that they will be boring or useless, but I am doing my part to let everyone I cross paths with know that there is a lot to do out there, that there are unlimited ways to get involved, and that if you keep looking and put in some effort the resources can be found. I am grateful that the KHI conference has opened so many doors for me.

L: I love the interaction with other patients, and the opportunity to both learn and encourage others. I think it was important for many patients to feel they both have a voice and that our collective voices were not only being heard, but welcomed.

Stay tuned for Part II of this blog when we chat about what kind of planning it took to make this event as patient-centric as possible with those who ran it. And don’t forget to visit the site to connect with Samantha-Anne, Laura, and the nearly 1,000 other PatientsLikeMe members living with chronic kidney disease.

Share this post on Twitter and help spread the word.


A patient’s perspective: member Gary reports back from the FDA public meeting on Parkinson’s disease

Posted October 27th, 2015 by

Back in September, Sally Okun, Vice President of Advocacy, Policy and Patient Safety, and community moderator Molly Cotter, attended the FDA public meeting on Parkinson’s disease (PD) along with PatientsLikeMe member Gary (tupelo).

We posted some interesting developments from the meeting in the forum, but we also caught up with Gary for his perspective on the event. Here’s what he had to say:

Why did you want to be part of the FDA public meeting?

On the day I was diagnosed, back in 2012, I spent a great deal of time reflecting on my life. I thought a lot about how Parkinson’s disease will impact me and my family in the future. On that day I made a commitment to myself that I was going to take control, to the best of my ability, on the course of the disease progression, and would do anything possible to find a cure. I was determined to educate myself as much as possible about the disease, put together the best possible health care team, learn all I could about treatments currently in research, and participate in clinical trials. Parkinson’s was a new challenge in my life and I intended to tackle it like I would any other challenge that I had faced in the past. Along the way in this new journey, I became more publicly involved as a PD advocate, both for advancing research as well as supporting newly diagnosed patients. So, when I noticed the FDA announcement regarding the meeting, I decided to send in my comments and thoughts in hope that they might be helpful, to whatever degree, in moving research along for better treatments.

What did it mean to be accepted?

When I submitted my comments I never expected to be asked to participate on one of the panels, nor was I intending to attend the meeting, which was on the eve of a religious holiday. I was quite surprised when I was contacted by the FDA, but was caught in a dilemma. Participation meant missing an important time with my family. However, when we discussed it, we all agreed that this was such a great opportunity, and honor, to be able to represent the needs of the millions of Parkinson’s disease patients worldwide to the FDA. There was no way I could turn down that kind of invitation.

What was it like being there as a patient representative at the FDA event? Did you feel like your voice was heard?

I’ve participated in a number of seminars and conferences in the past, so there wasn’t much difference with the arrangement for this meeting. If anything, it was much more low key. Nevertheless, I definitely had a sense of awe when I arrived at the FDA complex. There were multiple buildings all over the sprawling campus. It immediately reminded me of the importance of this event compared to others I’ve attended. We were going to be presenting to the Director of the FDA’s Division of Neurology Products and nine other senior staff members. This is about as high up as we could go in our advocacy to advance Parkinson’s disease research.

During the meeting, it was difficult for me to tell how our presentations were going and whether they would have an impact on the FDA’s staff, although it appeared to me that they were listening intently. I was certainly concerned about the limited amount of time I had to speak. Several days later, though, I watched the webinar presentation of our event and I felt that we hit on the large majority of the most important areas of concern that our community has regarding current and new treatments. Only time will tell if the FDA will act accordingly with our hopes and suggestions. One small item that I noticed which suggests that they were listing involves a question on the Patient Questionnaire at the meeting. We were asked to choose from a list of Parkinson’s symptoms the ones that were most impactful on our lives. In my presentation, along with several of the other panel members, we noted that they didn’t include pain or orthostatic hypotension, two very common symptoms, on their list. I did, however, see that on the new follow-up survey the FDA is conducting, they have added both symptoms to the bottom of the list. So, at a minimum, they were listening as we spoke about these debilitating symptoms.

What did you learn while you were there?

Well, I can’t say that I learned much about the disease while I was there, but, of course, that wasn’t the purpose of the meeting. Our presentations were intended to educate the senior staff and decision makers of the FDA. That being said, what I did learn was the amazing dedication and effort that members of our Parkinson’s community will undertake in order to be heard at a forum. Members with severe difficulties traveled from all parts of our country in order to be able to speak for a few minutes. I was truly honored, and most definitely humbled, to be part of a panel with these dedicated individuals. If you haven’t yet seen the archived presentation of the meeting online, I suggest that you at least watch the members of the first panel speak to the FDA about their symptoms, it was amazing.

What are your hopes for future research in Parkinson’s disease?

We were asked by the FDA to limit our answers to their questions in the context outside of finding an actual cure. In addition, we were mainly directing our responses to the FDA’s role of controlling the approval and distribution of prescription drugs, more so than studying the potential impact of alternative therapies such as exercise, vitamins or other supplements, or the development of new medical devices to assist with symptomatic control (all of which are needed). So I’ll stay within that premise.

I see the need for a three-direction approach for future research. First, we need to continue and expand research into the biological science of Parkinson’s disease, as well as other neurological diseases with similar developmental processes, such as Alzheimer’s and ALS. With a better understanding of the disease cause, pathways, and progression, we will have a much greater chance of finding better treatments and potential cures.

Second, I would like to see research into the development of better symptomatic treatments. These treatments should be effective for multiple symptoms, have fewer disabling side effects, and have extended release and longer term activity. Although today’s treatments are somewhat effective treating motor impairment, they are greatly lacking in effectiveness for many other non-motor symptoms. Many of the drugs have side effects that can be more disabling than the symptoms they treat. In addition, it would be desirable for new treatments have ease of administration, such as oral, sublingual or inhalable as opposed to invasive surgery or device implantation.

Third, and most importantly, we need research to discover interventional treatments that can slow or halt the disease progression, if not totally cure it. This research should target the development of new drugs and vaccines that can intercede along all of the numerous biological pathways as the disease progresses. For example, we would find means to either stop the misfolding of proteins, prevent proteins already misfolded from accumulating in brain cells, or eliminate misfolded proteins which have already accumulated and are leading to cell death. Finally, I would like to see more research in alternative treatments, such as stem cells and human growth factors, in addition to traditional drug development. It’s imperative that the FDA allow our scientists to be as innovative as possible as they attempt to find treatments and cures for the most complex illnesses we have in brain diseases. With new interventional disease modifying drugs, along with improved symptomatic treatments and a proper exercise regimen, most, if not all of us dealing with Parkinson’s disease would be able to live active and productive lives for many years beyond our diagnosis.

For more information, check out the full video of Gary’s panel. And don’t forget to visit the site to connect with Gary and the more than 11,000 other PatientsLikeMe members living with Parkinson’s disease.

Share this post on Twitter and help spread the word.


Can patients become innovators? Rishi Bhalerao from PatientsLikeMe speaks at TEDx Springfield 2013

Posted December 11th, 2013 by

Rishi Bhalerao, our Program Director of Client Services, recently spoke about patient innovation at the 2013 TEDx conference in Springfield, MA. Rishi talked about how the path to innovation begins right with patients because they are the ones who live with a condition day-in and day-out. Their sharing helps bridge the gap from patient to doctor to provider, and through measuring their own progress, patients can learn from each other and improve their lives together.


World Parkinson’s Congress recap

Posted November 18th, 2013 by

Here at PatientsLikeMe, we always look forward to hopping on the road and visiting you, the members who generously share personal health journeys so everyone can learn to live better, together. Recently, Molly (she’s part of your Community Team), Brad and Jim (part of our Partner Marketing team) drove north to Montreal to attend the World Parkinson’s Congress with a few PatientsLikeMe members, and they wanted to take a quick second to report back on the experience. Check it out.
 


Off to MontrealThe World Parkinson’s Congress is designed to create a worldwide dialogue to expedite a treatment or cure for Parkinson’s Disease. It was amazing to see physicians and scientists, alongside health care professionals and nonprofits, connecting with patients and caregivers. It was a wonderful conference, and we were glad to be among the more than 3,300 participants who gathered in Montreal.

We couldn’t have picked a better setting than Old Montreal to meet with four of our Parkinson’s community members – Aunti_J, Sap011235, Sunshine221, and Red Hat Queen. They all attended the conference and joined us for a wonderful dinner at Chez L’Epicier. The cobblestone streets, gorgeous historic buildings and an unseasonably warm evening set the scene for exquisite French cuisine with new friends.

But the setting couldn’t hold a candle to the experience of meeting PatientsLikeMe members in person. After reading their forum posts, answering their questions, and hearing about their experiences with Parkinson’s for well over a year on the website, I was eager to give them a big hug in real life. We talked travel, DBS, support groups, community walks, and perhaps most poignantly, how and why each person joined PatientsLikeMe. Aunti_J left us with this touching memory of how she found PatientsLikeMe:

“I had to retire from my work about 2 years after I was diagnosed and I really felt like I was going home to die. I spent an entire year feeling so depressed. Then I found PatientsLikeMe and within a half an hour, I felt like this weight was lifted off my shoulders. I felt like I found ‘me’ again.”

Aunti_J joined in 2008 and continues to be a warm and welcoming voice in the community for all Parkinson’s patients who are sharing their journey at PatientsLikeMe.

The goodbyes came too quickly, the hugs were worth more than the distances we all traveled to get to them, and when we returned to our “real” communities at home, and to our virtual family on PatientsLikeMe, we carried with us the realization that we truly do live better, together.

PatientsLikeMe member MollyCotter


It’s Global Pulmonary Fibrosis Awareness Day!

Posted September 7th, 2013 by

gpfad_logo_rbg

Do you know someone with pulmonary fibrosis? Thanks to the Pulmonary Fibrosis Foundation, today – Sept 7th – is dedicated to raising awareness about this condition and sharing the stories of patients living with PF. It’s Global Pulmonary Fibrosis Awareness Day, and we want to do our part to help raise awareness of this lung condition. Pulmonary fibrosis (PF) causes scarring and thickening in deep lung tissue over time.

We recently announced that the PatientsLikeMe community has grown to include more than 1,000 patients with idiopathic pulmonary fibrosis (IPF). IPF refers to cases where the cause of the condition is unknown, and it affects over 100,000 people in the United States alone.[1] Our collaboration with Boehringer Ingelheim has made the acceleration to enhance our community for PF patients possible. So for Global Pulmonary Fibrosis Awareness Day, we’d love to share what we know about this condition, people’s experiences with it, and the stories behind the people living day in and day out with the disease.

More and more IPF patients are sharing their real world experiences on PatientsLikeMe, monitoring their own health while connecting with others just like them. We’ve done a series of blog interviews with our members, including Lori, Kim, Jeff and Ian, as well as other patients like Sarah and Keith.

Beyond the stories, our members are sharing experiences about what it’s like to live with their IPF. Here’s a snapshot of what we’ve learned about their real-world experiences, including a quote from Lori, an interviewee and amazing individual who was diagnosed with IPF in 2011 and has been connecting with PatientsLikeMe members ever since.

PatientsLikeMe-PF Community

As the PFF says, today is about sharing stories and raising awareness of a condition that affects over half a million Americans. You can help by sharing your story and experience here and through campaigns run by the Pulmonary Fibrosis Foundation and the Coalition for Pulmonary Fibrosis.

Every story, every voice, and every experience matters when coming together to make a difference for pulmonary fibrosis.


[1] http://ghr.nlm.nih.gov/condition/idiopathic-pulmonary-fibrosis


PatientsLikeMe RFP for PCORI Funding

Posted May 24th, 2013 by

Last month, the Patient Centered Outcomes Research Institute (PCORI) released a funding announcement entitled, “The National Patient-Centered Clinical Research Network: Patient-Powered Research Networks (PPRNs)—Phase One.” Through this solicitation, PCORI seeks to establish a set of Patient-Powered Research Networks (PPRNs) that complement PCORI efforts to establish a national infrastructure of Clinical Data Research Networks through a separate funding announcement.

As an early pioneer of PPRNs, PatientsLikeMe plans to submit at least one Letter of Intent (LOI) to PCORI next month to declare our intention to submit a funding application later this year. We are looking for research partners who share our patient-centric values and desire to build a continuously learning healthcare system to join us as joint collaborators on our June LOI submission to PCORI. We welcome the opportunity to team with academic researchers, pharmacies, clinicians, patient advocacy organizations, and others who wish to partner with PatientsLikeMe.

DownloadRFPTo that end, we have authored a Request for Proposals (RFP) that outlines the nature of our expected funding application and the information we require from interested collaborators. The goal of the RFP is to identify quality research partners capable of defining high-value, patient-centric research goals worthy of PCORI funding that can be built upon PatientsLikeMe’s existing digital infrastructure and data model. Requirements for responding to this RFP are described in further detail within the RFP itself.

We look forward to receiving high-quality responses to this unique funding opportunity no later than Wednesday June 12, 2013.

PatientsLikeMe member cfidyk


American Diabetes Assocation Alert Day – Is Today Your Wake-Up Call?

Posted March 26th, 2013 by

Today, March 26, 2013, is American Diabetes Association Alert Day, so we are doing our part at PatientsLikeMe by putting out our own alert.  Are you at risk for type 2 diabetes?  Take the American Diabetes Association’s Diabetes Risk Test right now to find out now.

National Diabetes Alert Day 2

One in three adult Americans is estimated to have prediabetes – marked by high blood sugar levels – so don’t think it couldn’t happen to you.  Fortunately, lifestyle changes such as weight loss and increased physical activity can delay the onset of type 2 diabetes, or even prevent it altogether.

Not only will you find out your risk level by taking this free and fast test, but you’ll help raise money to fight the growing diabetes epidemic (26 million children and adults in the US).  Boar’s Head is donating $5 to the ADA for every person who takes the test between March 26th and April 9th – up to $50,000.

So take a moment and answer a few simple questions about your weight, age, family history and other potential risk factors.  You’ll be helping yourself potentially avoid a serious disease, and you’ll be helping others no matter what.  Take the test now.

National Diabetes Alert Day 1


Can Social Media Strengthen Science?

Posted March 25th, 2013 by

And more specifically, can it be used to collect patient data, raise investment funds, make scientific data more accurate and even allow regular people to access the world of venture capital?

This was the provocative question posed to a five-person discussion panel at the 2012 Partnering for Cures conference last fall that included several thought leaders, including PatientsLikeMe’s own Sally Okun, RN, MMHS, who was recently promoted to the position of Vice President of Advocacy, Policy and Patient Safety.

“We have a small subset of people on PatientsLikeMe who have found us and who are gaining some expertise at being what might be called citizen scientists,” says Sally.  “But I think the important piece is that there are so many more people out there that we have to reach and help understand that there is access to so much more information than you are currently getting.  And social media is one way of doing that.”

Tune in below for Sally’s full comments and to hear what the other four panelists had to say about the growing use of social media in science.


A Traumatic Brain Injury Can Happen to Anyone

Posted March 18th, 2013 by

Did you know that 1.7 million Americans sustain a traumatic brain injury each year?  Or that the term includes any type of blow, bump or jolt to the head or penetrating head injury that disrupts normal brain function?

Brain Injury Awareness Month Banner

March is Brain Injury Awareness Month, a time for reinforcing the seriousness of head injuries, given that traumatic brain injuries are a contributing factor in a third of all injury-related deaths in the US.  In addition, 3.1 million individuals are living with life-long disability as a result of a traumatic brain injury. Some common causes of these injuries are falls, car accidents, workplace accidents and assaults, but the effects can vary greatly from person to person.  No two brain injuries are alike.

Brain Injury Global Picnic Logo

Are you living with a traumatic brain injury?  Share your symptom and treatment histories with the 500+ members of PatientsLikeMe’s traumatic brain injury community and discuss your experiences in our Injuries and Traumas Forum.  In addition, consider raising awareness in your area by getting involved with the Brain Injury Global Picnic, which takes place September 21, 2013.  The goal is to organize 1,000 picnics around the world – thus setting the Guinness World Record for the most people picnicking in a 24-hour period – to promote awareness, education and change.


Attending Partnering with Patients…as a Patient

Posted March 14th, 2013 by

Last Friday we heard from PatientsLikeMe Head of Community Liz Morgan about her experiences at the Institute of Medicine’s Partnering with Patients workshop.  Today we hear from multiple sclerosis (MS) patient Laura Phillips, a PatientsLikeMe member who spoke at the event. 

As a speaker at the workshop, Sally Okun, PatientsLikeMe’s Health Data Integrity & Patient Safety Director, invited me to speak for the patients of PatientsLikeMe as her co-presenter.

IOM Workshop - LadyMac

I had everything worked out to exactly what I wanted to say and right in front of me so I wouldn’t forget, but I couldn’t tell you what I even said. LOL.  I know I didn’t want to look down and read, it needed to come from the heart and what I could remember.  I thought I’d freeze and not be able to say a word, but after introducing myself I felt really comfortable with looking at the audience and speaking.  Even with Liz Morgan, Head of Community at PatientsLikeMe, taking pictures, I was able to talk.

There was a large, diverse group of people attending and presenting. I wish I could do a better job of portraying the event, but it was large. Healthcare, patients and families were the subjects of the workshop. Lots of ideas were put forth on everything from how to improve the healthcare system to how to make patient records available to the patients electronically, such as recording doctor visits and putting them on a CD for the patient to keep and play for other family or friends who were not at the appointment.

Recording doctor visits is something I’d love to see. How many times have you left the doctor’s office and each person present remembers the visit differently? Or you can’t remember if you discussed a particular test or not?  I did push the importance of the patient keeping hard copies of ALL tests, doctors’ notes, you name it, get a copy.  I would love to see the ability of viewing my records electronically so I could view my results quicker, make notes to ask the doctor, etc. There was also talk of submitting questions for the doctor in advance for the doctor to review and be more prepared for the visit. Also, the staff would print a copy of your submitted questions so you would have it in hand and remember what to talk about.

There was way too much to compress into a brief review of the event, but there were a lot of really good, logical ideas presented.  How many of the things discussed will eventually become a reality? Only time will tell. Privacy will be a major roadblock I fear.  Everyone that I heard talk did agree that collecting data from the patients themselves was important, and PatientsLikeMe was mentioned more than once by others there.

IOM Workshop - Sally and LadyMac Closer

Sally and Liz were a delight to meet and spend time with, as we are all passionate about PatientsLikeMe and how it has helped many of us through the years.   Really had a great time, and I wish I could have spent more time with Sally and Liz, but the time we did spend talking was productive and invaluable.