8 posts tagged “conference”

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.

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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.

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