Posted by Lori Piscatelli Scanlon | February 24, 2010
PatientsLikeMe partner, Myelin Repair Foundation (MRF), is a nonprofit organization devoted to reducing the time it takes to discover and develop treatments for multiple sclerosis (MS) patients. Recently, the MRF launched a campaign called “Where are the Cures?” - a site dedicated to a greater understanding of the barriers slowing the discovery and development of new patient treatments in all diseases. PatientsLikeMe co-founder Jamie Heywood asked Scott Johnson, President and Founder of MRF, about the campaign.
(Scott) The big idea? Finding a way or ways to accelerate the development of new patient treatments.Developing new medicines or treatments for complex diseases is extraordinarily difficult. But it is not impossible and it should be happening at a faster rate than it has been. I believe the single most rate-limiting factor is the failure to move promising scientific discoveries made in academic laboratories into the commercial development pipeline.The problem is fairly simple. On the front end of the value chain, academic laboratories are spending upwards of $40 billion a year in government and non-profit dollars to make basic scientific discoveries that may be potential drug targets. On the back end, the pharma industry is facing a crisis of grand proportion as a result of its dwindling pipeline. The obvious question is why isn’t pharma reaching into this vast pool of potential drug targets to fill their pipeline? Because between the two is what is known in the industry as the Valley of Death.I think two things primarily contribute to this disconnect. First, most biological discoveries made in academic labs are not patented. Without the clear ownership that patent protection provides, biopharma is unwilling to make a multimillion investment in drug development. Second, there is little to no funding available to conduct the validation studies necessary to fully demonstrate that the basic scientific discoveries made in academic laboratories will truly and safely impact the disease state in humans. Without this validation, again, the perceived risk to pharma is too high.Knowing this, it’s shouldn’t be surprising that so few treatments for difficult diseases are coming to market.So how do we capitalize on the promising discoveries made in universities and feed that pipeline? We address those two problems: protecting the IP and funding validation studies.We are doing this at the Myelin Repair Foundation and it can be done in other medical research. But it requires an open mind and a set of shared incentives that puts the patients’ needs before other current incentives.
(Jamie) As a part of the campaign, you launched The Patient’s Manifesto, a declaration that the medical research system is broken and a call for massive change across industries. Why should a patient with life-changing condition sign it?
(Scott) We created the manifesto because we wanted to bring attention to the problem and provide a venue where new ideas would be talked about. There is a lot of innovation going on in the industry but we have to find ways of sharing it. Why should patients sign the manifesto and encourage others to do the same? On the premise that the squeaky wheel gets oiled first, we think that we will be more successful at bringing public attention to this problem if there appears to be a significant groundswell of people who care. The millions of patients and their families living with chronic diseases are the most motivated to create change. There is no implied promise here. It’s all about building awareness and attracting attention in a way that will spur change.
(Jamie) Both our organizations work to help let the patient voice be heard. What do you think a patient’s role is in making our research system more efficient?
(Scott) I think the entities that are best positioned to tackle the Valley of Death are the non-profit disease research foundations like the MRF. Their only motivation is improving the quality of life for patients. Because we are not driven by academia’s need to “publish or perish” or by pharma’s need to increase shareholder value, we have the freedom to move among and between the players in the value chain and facilitate the process on behalf of patients. It is largely patients and their families who support these non-profit organizations and for their investments they have a right to ask for accountability. While investing in basic research is an absolutely essential part of the equation, it takes more than that to get a drug to market. Patients should be asking the organizations they support how they plan to actually accomplish that once the basic science is completed. I think as patients, we understand that this isn’t easy. But we don’t have to sit back and be satisfied with the status quo.
(Jamie) No we don’t, Scott. Thanks for talking with us about the campaign. For those of you wondering, yes I’ve signed The Patient Manifesto. Have you? Go to www.wherearethecures.org for more information.
We’re big fans of patient-reported outcomes (PROs) here at PatientsLikeMe, defined by the Food & Drug Administration (FDA) as:
“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.
Posted by Lori Piscatelli Scanlon | February 16, 2010
Today’s news release announcing acquisition of ReliefInsite. Want to receive future announcements? Sign up for our RSS feed on the press page.
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FOR IMMEDIATE RELEASE
PATIENTSLIKEME ACQUIRES ONLINE PAIN MANAGEMENT COMPANY, RELIEFINSITE
20,000 Patients Reporting Moderate to Severe Pain on PatientsLikeMe
Cambridge, MA and New York City, NY - February 16, 2010 - Today, PatientsLikeMe, the leading online community for patients with life-changing diseases, announces the acquisition of ReliefInsite, a pioneering online pain management company.
“Right now, there are more than 20,000 patients on PatientsLikeMe experiencing real pain,” says Ben Heywood, President and Co-founder of PatientsLikeMe. “By acquiring ReliefInsite, we can improve the way we help patients effectively manage their pain.”
Conceived by founder Fred Eberlein in 2000, ReliefInsite’s patented online pain management technology developed into a secure HIPAA-compliant platform designed to help patients monitor their pain levels and share that information directly with their doctors.
“I’ve spent more than a decade inventing and advancing the technology platform at ReliefInsite to help patients manage their pain and improve the patient-doctor relationship,” says Eberlein. “I’m excited this work has led me to my new home at PatientsLikeMe, where we can work together to better align industry’s interests with improving patients’ quality of life.”
ReliefInsite will continue to support its existing pharmaceutical and clinical clients as its functionality is integrated into the PatientsLikeMe platform. Eberlein joins the PatientsLikeMe team to lead the companies’ integration and enhance product offerings for partners in the life sciences industry.
Pain is a common symptom for people with all of the diseases on PatientsLikeMe, and is most prevalent in diseases like fibromyalgia and multiple sclerosis. In the depression community, thousands of patients report they are currently experiencing “back pain” or “stomach pain,” with 50% of those members describing their pain as moderate or severe. Across all of the disease communities, pain is a topic of discussion in more than 61,000 forum posts. Additionally, patients are using hundreds of treatment options to manage their pain from prescription medications, such as Cymbalta and Lyrica, to alternative solutions like acupuncture, massage and physical therapy.
Ever wonder how epilepsy affects the patient — the whole patient? A patient-reported outcome (PRO) is a questionnaire commonly used in the clinical world to asses a patient’s quality of life (QOL) for research studies. Most of the time patients never get to see their test results, much less see them plotted over time. That’s a shame because many of the best PROs were designed with the help of patients talking about their condition in their own voice.
Patients in our epilepsy community can now access a series of PROs to help understand the effect of their disease on everyday life and have a voice in real-world, real-time research. The best part is that once they’ve completed the questionnaires (which should take about 20 minutes) they get to see summary findings of how they compare to other members in the community, plus a summary of their overall physical, mental, and social quality of life is displayed on their profile and their patient icon.
This is an ambitious project; we’re looking to get data from thousands of patients with epilepsy at 3 distinct time points. Through this shared goal for the community, patients are pulling together and encouraging one another to have a voice in research. After all, the more data is in the system, the more insight every individual can gain from taking part!
Posted by Lori Piscatelli Scanlon | January 29, 2010
In honor of the launch of the new PatientsLikeMe Epilepsy community this week, we have created a video highlighting how patients are coming together to share their experiences with epilepsy and learn from others. It’s now available on our PatientsLikeMe Epilepsy YouTube page.
This short video includes quotes from some of our members about what drove them to join the online community, and walks you through what the community looks like and how members are using it. Know someone with epilepsy? Share this video, our interview with Blueyedgoddez, and/or our announcement with partnering company, UCB.
Welcome to all those joining the epilepsy community!
(Special thanks to our new marketing team member, Aaron Fleishman, for pulling this video together. It’s Aaron’s voice and his band’s music you’ll hear throughout.)
Posted by Lori Piscatelli Scanlon | January 27, 2010
At PatientsLikeMe, we believe in getting to know the person, not just the patient. To celebrate the launch of the new epilepsy community this week, we sat down (virtually) with 3-star member, Blueyedgoddez. Here’s what she has to say about her experiences living with epilepsy:
(Blueyedgoddez) I have blue eyes and I sometimes feel like a goddess, as silly as that may sound. I don’t mean a goddess as in a rich snob or anything. I mean it like an earth child, that sort of thing.
(PatientsLikeMe) When were you diagnosed with epilepsy? What was that like?
(Blueyedgoddez) I had seizures since infancy, however i was officially diagnosed at the age of six. When I was a child, it was difficult in certain ways. Mostly because I knew I could never enjoy certain things that children enjoy - i.e., video games (at that time the resolution of the games were too high, the graphics would induce seizures), rollercoasters, the gravatron (at the carnival), any type of flashing party lights, among other things. As an adult, it’s not that much easier. I can’t drive under no means because where I live you need to be seizure-free for 5 years and I never am. So that’s a challenge having to depend on people or public transportation. Plus, where I work my co-workers aren’t as understanding as people were when I was a child. The people I work with tend to say I use my epilepsy not to do things. How could anyone say I use Epilepsy to my benefit? To me it’s very hurtful. It makes no sense. Also one other thing that seems to bother me as an adult is having children. I was told it’s going to be very difficult.
As you can see by now, it’s a journey having epilepsy. However, in the end, you learn to live, laugh and love - despite all your perils.
(PatientsLikeMe) How does epilepsy impact your every day quality of life?
(Blueyedgoddez) The disease in itself isn’t what really effects my day to day activities, however it’s more the side effects from the medication to treat my epilepsy. Everyday I’m bound with multiple side effects such as, fatigue, headaches, depression, anxiety, aches and pains, nausea, the list could go on. Some days I wake up fine, others not so fine. I have my good days and bad days.
(Blueyedgoddez) I joined because I didn’t want to feel alone anymore. Simply put. And I know that I could be helpful with my life experience of having epilepsy to someone else just like me, more or less. That’s why I’m here to be helped and to help others. I have a voice and I intend on using it.
(PatientsLikeMe) Thank you for using that voice and sharing your story and experiences with our community!
Posted by Lori Piscatelli Scanlon | January 26, 2010
Pharmaceutical, Social Media Leaders Examine Real-World Impact of Epilepsy
CAMBRIDGE, MA and ATLANTA, GA — (Marketwire - January 26, 2010) – Today, PatientsLikeMe, the leading online community for people with life-changing conditions, and biopharmaceutical company UCB open the doors to a free online community for people living with epilepsy in the U.S. Focused on learning from patients’ real-world experiences, the online community allows members to create profiles that record and share their treatments, symptoms, as well as seizure type, frequency and severity.
“As a patient-centered company, we are constantly seeking innovative ways to enhance and adjust our approaches to meet patient needs,” says Iris Loew-Friedrich, Executive Vice-President, Chief Medical Officer, UCB. “We believe this community will be a source of information that will allow us to better understand people living with epilepsy and may help us design clinical programs that incorporate real-world patient needs and experiences in a measurable way.”
Among the research being conducted, PatientsLikeMe and UCB are analyzing anonymized data shared by participating epilepsy patients, with their consent, via an online clinical survey built into the site to measure patients’ quality of life (including cognitive, social and physical function). Completed by 60 patients testing the site since mid-November, initial survey results show the patients are most concerned with the cognitive impact of living with epilepsy (such as lack of concentration or memory loss).
Adds Ben Heywood, co-founder and president of PatientsLikeMe, “Patients are telling us that, in addition to seizures, there are many more significant aspects to the disease. It’s this type of real-world data and insight about epilepsy that is going to change how this disease is treated.”
To better understand the impact of medications that treat epilepsy in the real world, PatientsLikeMe and UCB are also taking the lead in implementing a drug safety program within this patient community. The program is designed to capture and report adverse events associated with approved UCB epilepsy therapies to the U.S. Food and Drug Administration (FDA).
Last June, PatientsLikeMe and UCB announced this strategic partnership to build an online community for people living with epilepsy, which debuts today at www.patientslikeme.com/epilepsy/community.
You can see the full news release from PatientsLikeMe and UCB on Marketwire.
Posted by Lori Piscatelli Scanlon | December 31, 2009
As 2009 comes to an end, we want to take this opportunity to thank all of our members, partners and general fans for another great year. Here’s a recap of some of the exciting happenings at PatientsLikeMe these past 12 months. Wishing you all a Happy New Year!
Community Milestones
This year, the 15+ disease communities at PatientsLikeMe became an online home to more than 50,000 members. The fibromyalgia community was expanded this past fall to include patients with chronic fatigue syndrome, and we announced a new community scheduled to launch early next year for people with epilepsy. In addition to celebrating our communities’ awareness days and months within the site and right here on the blog (including Fibromyalgia Awareness Day, MS and PD Awareness Months, National HIV Testing Day and World AIDS Day), many of our members also participated on PatientsLikeMe teams in walk/run efforts to raise awareness and money in the name of their disease. Congrats to the 40+teams walking at events to support non-profit organizations like ALS Association, National MS Society, NAMI, Parkinson’s Alliance, APDA, and the MS Society of Canada. The real-time sharing and learning happening on PatientsLikeMe was also highlighted in the report series called The Patient Voice (starting with inpatient therapy for people with Mood conditions). Check out highlights from all these programs on these videos from the Fibromyalgia, Parkinson’s, Multiple Sclerosis and Mood communities.
“I find this site so beneficial in looking for the tools to cope with the disease. People post research and real-life patient experiences of current trials and treatments. Hope is a major focus of PLM and I encourage others to join. We are strong in numbers and we have a voice.”
“PLM is a wonderful way to express what and how to live with this disease. It has changed my life for the better, have met wonderful individuals and we have shared, cried and grown by reaching out and expressing from our hearts, have made wonderful friends and have learned so much.”
Throughout the year, the team has also shared insights via videos (like this series on the history of ALS or a recap of a study on the antidepressant Amitriptyline) and various presentations (such as an overview of our work at the at Eurordis AGM in Athens and updates on our lithium study at the International ALS/MND Symposium). In addition to working on the development of our new communities, the team took additional steps to incorporate genetics into the PatientsLikeMe platform. By participating in new partner studies (such as 23andMe and NEALS) and utilizing new product upgrades, including the launch of the Genetic Search Engine, patients are learning more about their condition and coming closer to answering the question: “Given my status, what is the best outcome I can expect to achieve, and how do I get there?”
Keep your eyes out for more to come in 2010 as our R&D team rolls out more insights and outcomes measurements (like the Quality of Life scale in HIV), more improvements to the PatientsLikeMe platform, and cutting edge research in the peer-reviewed scientific literature.
The Business Side
As Ben said in a recent blog post, “we can’t have a business without you [the patient] and our communities can’t exist to help patients without a business.” Throughout the year, the PatientsLikeMe executive team traveled around the world to present to industry partners, researchers, healthcare professionals and government leaders about the power of real-world patient data-sharing. Here are some highlights from ‘09:
Government: Piloted in our MS community this year, PatientsLikeMe members now have the ability to voluntarily report adverse events directly to the FDA; in fact, Jamie recently presented about adverse event reporting at an open FDA hearing on the “Promotion of FDA-Regulated Medical Products Using the Internet and Social Media Tools.” PatientsLikeMe was also one of the collaborating organizations responsible for writing the Declaration of Health Data Rights and launching HealthDataRights.org this past June. Finally, Jamie testified before the National Committee for Health and Vital Statistics, and gave an exciting rapid-fire presentation on the future of medicine at the Gov 2.0 Summit.
Innovation: As a leader in Health 2.0, PatientsLikeMe executives are often asked to speak at various industry events. Check out photos of Ben speaking at The National Summit and stay tuned for videos from Jamie’s presentation at TEDMED and David’s presentation at Bil:Pil. You can also tune in to our live event tweets on the PatientsLikeMe Twitter account in 2010.
Media Highlights: PatientLikeMe members, data and executives were also featured in media mentions throughout 2009, including WIRED, Forbes, U.S. News & World Report, Fox Business Live, Newsweek, New York Times, as well as Seed magazine, Nature Biotechnology and Neurology Today.
A few weeks ago, I was invited to present on behalf of PatientsLikeMe at the Arthritis Foundation workshop held in Newport, RI. The presentation was on how to “harness new media for patient advocacy” - the same as what I presented at a workshop for non-profits in northern New England in August. This time the audience included non-profits in the southern half of New England. Among those in attendance were representatives from organizations that mean a lot to us, and our patient communities, including regional branches of the American Parkinson’s Disease Association, CFIDS & FM Association, and the Epilepsy Foundation.
One of the highlights for attendees was an impassioned keynote speech by Rep. Patrick Kennedy (pictured above with me and my wife Emma) who advocated the use of the web to support mass organization of patients with serious and chronic illnesses to accelerate research and improve standards of care. We couldn’t agree more. It’s exciting and validating to know influential decision-makers are recognizing the potential of communities like PatientsLikeMe.
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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(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?
(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.
(David) This partnership is said to give patients a voice in advancing research. How so?
(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.
(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?
(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.
(David) What’s the most exciting part of this initiative for UCB?
(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
Today is an exciting day for PatientsLikeMe. In a first-of-its-kind industry-patient partnership, PatientsLikeMe is joining forces with biopharma company, UCB, to launch a new community for people with epilepsy to capture real-world experiences of the disease and help advance research.
The news release announcing the partnership is below.
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BRUSSELS, BELGIUM and CAMBRIDGE, MA–(Marketwire - June 15, 2009) - Biopharma company UCB and PatientsLikeMe, the leading online community for people with life-changing conditions, today announced a strategic partnership to create an online, open epilepsy community that captures real-world experiences of people living with epilepsy in the U.S.
Scheduled to launch in early 2010, this platform will be designed to collect, analyze and reflect information received from people with epilepsy, regardless of their diagnosis, prognosis or treatment regimen.
PatientsLikeMe is the leading online community for people with life-changing conditions. Patients embrace the open sharing of personal health data because they believe that information can change the course of their disease.
With a focus on patients and research, our blog reflects knowledge resulting from the shared real-world experiences of our community. Welcome to the genesis of patient-led research.
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ALS, Chronic Fatigue Syndrome/ME, Epilepsy, Fibromyalgia, HIV/AIDS, Mood Conditions, Multiple Sclerosis, Openness, Parkinson's Disease, Rare Diseases, Research 
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Pain is a common symptom for people with all of the diseases on PatientsLikeMe, and is most prevalent in diseases like 
our members also participated on PatientsLikeMe teams in walk/run efforts to raise awareness and money in the name of their disease. Congrats to the 40+ teams walking at events to support non-profit organizations like ALS Association, National MS Society, NAMI, Parkinson’s Alliance, APDA, and the MS Society of Canada. The real-time sharing and learning happening on PatientsLikeMe was also highlighted in the report series called 
AGM in Athens
Innovation: As a leader in Health 2.0, PatientsLikeMe executives are often asked to speak at various industry events. Check out 
