3 posts from July, 2009

Rare Diseases: Well-Done Online

Posted July 17th, 2009 by

There are rare diseases, and there are rare diseases. Here at PatientsLikeMe our first community was built for patients with ALS (estimated US Prevalence: 30,000), and in common with our other neurological communities there is a familiar list of challenges: low public awareness, little funding for research, and a lack of adequate treatments. However, over the past year or so I’ve really had my eyes opened to the differences between “rare” and what you might call “super-rare” conditions, such as Devic’s neuromyelitis optica. Nobody really knows how many people Devic’s affects as it is frequently confused with MS, but there are probably only a few thousand patients with this condition in the world. That’s why we’re incredibly proud that our Devic’s community currently has 136 registered patients sharing health data with one another; that’s more than 5 times larger than the largest study I’ve seen on the condition in the scientific literature (which included collaborators from around the world in seven specialist centers over the course of several years).

I was privileged to be invited to speak at the annual meeting of Eurordis (The European Organization for Rare Diseases) in Athens, Greece, to meet with some of the leading online health efforts in this space. Attendees included non-profit organizations, medical professionals, and patients themselves from all over Europe.  We all convened to discuss some of the most innovative tools available on the web for patients to find other patients like them, share their data, and improve their outcomes. PatientsLikeMe was featured as an ambitious and innovative effort to accelerate the pace of research in rare diseases but we also saw great initiatives that had come from the frontlines of rare diseases.  In fact, the point about ultra-rare diseases was driven home in the opening keynote by Yann Le Cam when we heard that there are some 5,500 rare diseases cataloged by Orphanet (including Devic’s) which are not in the ICD-10 taxonomy of diseases. Ultimately, at PatientsLikeMe, our goal is to build a community for every life-changing illness that exists, but what can patients with these conditions be looking for in the meantime?

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The highlight of the meeting for me was seeing the incredible work being carried out at Duchenne Connect.org (The Netherlands) and Duchenne Connect.org (USA). Founders Elizabeth Vroom and Pat Furlong gave an overview of their experiences building patient-focused programs that allowed parents of children with Duchenne’s Muscular Dystrophy to support one another, accelerate the efforts of researchers, and bring greater attention and focus to patients affected by this rare disease. From the other side of the Atlantic, Mary Dunkle from NORD (National Organization for Rare Diseases) made a clear statement that online communities have the power to be far more than just bulletin boards and blogs for patients to use for emotional support. In her presentation, she stated: “We want to move beyond simply providing emotional support…to facilitate action that produces results”; we couldn’t agree more. Videos of the talks from these amazing patient advocates (along with many other talks from the meeting) can be viewed online here at the Eurordis website.

There were a number of challenges that were highlighted during the meeting. David Golub was the first to articulate that there are serious ethical issues implicit in for-profit companies (like us!) being involved in patient research that was traditionally the remit of academics and clinicians. He asked us to all consider what we can all do to “protect the public commons?”. Unsurprisingly for a European audience, there was much concern about language specialization.  Patient advocates insisted on better localization to allow broader access to non-English speakers, and for providers like us trying to find innovative ways to ensure excellent content that can be dynamic and accessible for all. My own view is that technology (like Google Translate) will outpace any system we could possibly resource with human translators.

Finally, there was the question put to us by event organizer Denis Costello from Eurordis; how can small non-profits in ultra-rare diseases partner with organizations like PatientsLikeMe?  It’s something we think about every day. Our Devic’s community came out of our MS Community; PSP and MSA came from Parkinson’s; and PLS and PMA came out of ALS. We are developing strategies to build communities for “clusters” of communities that will allow us help a broader swathe of patients with both prevalent and rare conditions. It was hugely encouraging to see the energy, ingenuity, and determination that you see when advocates are passionate about helping patients.


An Interview with UCB’s Peter Verdru

Posted July 13th, 2009 by

Last month, PatientsLikeMe announced our partnership with biopharma leader, UCB, to launch a new community for people with epilepsy.  Below is an interview with UCB’s Vice President of Clinical Research, Peter Verdru, MD.  David S. Williams III, head of PatientsLikeMe business development, recently spoke with Peter about the forthcoming epilepsy community, adverse event reporting, and the partnership in general.

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581
(David) UCB is known as The Epilepsy Company.  What’s your goal in partnering with PatientsLikeMe to create a new community for people with epilepsy?
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(Peter) As patient-centric companies, UCB and PatientsLikeMe are both committed to advancing research and improving the lives of people with life-changing conditions.  UCB has a long-term commitment to the epilepsy community – so a partnership with an organization like PatientsLikeMe seemed only natural.

Our goal with this partnership is to provide this community to patients with epilepsy to help them manage their disease. Additionally, the community will generate patient-reported outcomes that may help UCB better understand how patients live with epilepsy and help advance epilepsy care. We anticipate patient-reported outcomes data across treatment groups for seizure severity, number of seizures, symptoms, adverse events, health-related quality of life, and co-morbidities, among other things. Using this knowledge for our future clinical research programs would be a logical next step, leading to an even better understanding of what future treatments could offer or what type of patients would gain additional benefit.

581 (David) This partnership is said to give patients a voice in advancing research.  How so?
pvh-1-3 (Peter) This community will give patients the tools they need to measure their own outcomes. Participants will record their real-time, day-to-day progress in controlling their seizures and achieving their treatment goals, and share that with the community to help other patients, caregivers, researchers and industry learn more about the disease. Tracking their disease over the long-term may help patients and physicians work together to evaluate the impact of their treatment. Eventually, clinical research programs might also benefit from the long-term data these patients are sharing.
581 (David) Through the partnership, both companies will be working to design and deploy a system that allows for adverse event reporting to the FDA.  Why?
pvh-1-3 (Peter) UCB has an ethical and legal responsibility to report adverse events associated with our drugs. If adverse events for any UCB drugs are mentioned on the site, UCB is required to report these directly to the U.S. Food and Drug Administration (FDA). Therefore, we are working to develop and deploy a solution that will allow us to assess and process potential adverse events, report them to the FDA, and capture them in the UCB safety database.
581 (David) What’s the most exciting part of this initiative for UCB?
pvh-1-3 (Peter) We’re excited to be taking a leadership role in the pharmaceutical industry to create a community that will give patients a forum for showing their treatment outcomes. Patients are really the experts about how epilepsy impacts their lives.

UCB is focused on bringing new treatments to patients with severe diseases like epilepsy. We sincerely believe this unique partnership will bring real value to the large community of patients, families and caregivers

581 (David) Thanks Peter!