7 posts from February, 2010

It’s Rare Disease Day!
Interview with Gracie (Devic’s NMO Patient)

Posted February 28th, 2010 by

Today, we’re joining the National Organization for Rare Disopicture-12rders (NORD) to help raise awareness for Rare Disease Day.  In recognition of the day, we recently interviewed Gracie, a valued member of our Devic’s Neuromyelitis Optica (NMO) community.

Devic’s NMO is a rare autoimmune inflammatory disorder which affects the optic nerve and spinal cord and is often confused with Multiple Sclerosis (MS).  Compared with MS, Devic’s brain lesions affect different parts of the nerve cell, spinal lesions are larger, and relapses occur in a different pattern.

Here’s what Gracie had to say to our community moderator, Aaron Fleishman:

apics2 (Aaron) When were you diagnosed with Devic’s NMO?  What was that like?
27744 (Gracie) In the spring of 2005, after a few months time of being hospitalized and misdiagnosed and doing a stint in rehab, I was definitively diagnosed with NMO via the Mayo clinic’s NMO IgG test.  Fortunately (or unfortunately, depending on one’s viewpoint), I was already fully aware of the implications of having the disease.  During the period of time that I was in rehab, I used their computers to research Idiopathic Transverse Myelitis, which is what I was originally diagnosed with.  While reading at the site of one of the more prominent U.S. teaching and researching facilities, I came across NMO.  I can vividly remember saying to myself: Thank God that I don’t have that.  It could have been so much worse.  I felt extremely thankful to have dodged that particular bullet.Less than two months later, I was back in the hospital with another acute attack.  This time, the paralysis had spread to mid chest, and I knew then that it was unlikely to be Idiopathic Transverse Myelitis.  While in rehab, my physician had told me that most cases of TM were monophasic, and that the best thing that I could do for myself would be to learn to cope with my deficits and move on.  During the second long hospitalization, I did not respond to IV SoluMedrol, so underwent a course of rescue plasmapheresis.  It was at that time that my current neuro submitted my serum to the Mayo Clinic for the NMO IgG test.  Actually, in my heart of hearts I already knew what the result would be.  It came back positive.   What was it like?  It was like being hit in the chest with a sledgehammer.
apics2 (Aaron) You’re one of the founding members of our Devic’s community, and a 3-star contributor.  How has being a member of PatientsLikeMe helped you?
27744 (Gracie) PatientsLikeMe is a unique site.  The singular most important function is the data sharing.  Researchers, studies, and clinical trials inform us as to how a particular drug or treatment will theoretically affect the disease process.  PatientsLikeMe offers the opportunity for patients to see how a particular treatment or medication works in the patient community, without the controls and structures of a clinical study.  I have not found another site on the Web, that has the graphing capabilities that PatientsLikeMe offers.  Most sites, including my own, are Forums, but offer no capabilities beyond that format.  I was thrilled whenever Paul Wicks gave me the news that NMO would be included among the [PatientsLikeMe] communities.
apics2 (Aaron) Today is Rare Disease Day.  Do you have a message for people with Devic’s (or those with other rare life-changing conditions)?
27744 (Gracie) The most important thing that an individual diagnosed with Devic’s NMO can do for themselve, is to seek out a neurologist who is not only familiar with the disease, but has treated cases as well.  Although it is being diagnosed more frequently with the advent of better clinical imaging and the Mayo Clinic’s NMO IgG test, it is still extremely rare and the majority of neurologists have not seen or treated a case.  I’m one of the lucky ones.  I was diagnosed at a large teaching and research facility and have an excellent neurologist.  Many patients are not so lucky.
apics2 (Aaron) Have you heard of the Accelerated Cure Project Repository? What does it mean for people with Devic’s? 
27744 (Gracie) The Accelerated Cure Project is an amazing organization.  Currently they are working in tandem with the Guthy-Jackson Charitable Foundation and are compiling an NMO data and samples repository.  They have done everything possible to facilitate the process, including sending a traveling nurse to the home of the patient to procure the samples.  Although I have not participated yet, I fully plan to.  Without data and samples, there can be no research and without research, there can be no progress.  The majority of us within the NMO community are willing to do anything that we can to facilitate research.  I’m so thankful for the effort of the Accelerated Cure Project.
apics2 (Aaron) We’re thankful too, Gracie.  And we’re thankful for all that you share every day with patients like you.

To learn more about the rare disease communities on PatientsLikeMe, including Devic’s, Progressive Supranuclear Palsy (PSP) and Multiple System Atrophy (MSA), please visit www.patientslikeme.com.

Where are the Cures?
An interview with Myelin Repair Foundation

Posted February 24th, 2010 by

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.

headshot (Jamie) What’s the big idea behind Where are the Cures?
picture-097 (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.
headshot (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?
picture-097 (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.
headshot (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?

picture-097 (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.
headshot (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.

FDA’s New Guidance on Patient-Reported Outcomes

Posted February 23rd, 2010 by

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.”

qolThe 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.

PatientsLikeMe member PaulWicks PatientsLikeMe member Mike Massagli

Who’s #2 in Healthcare Innovation?
You guessed it – PatientsLikeMe

Posted February 18th, 2010 by

It’s been an exciting few months for PatientsLikeMe.  Here’s a quick recap…

Today, Fast Company released its mic_logoannual  ranking of the 50 Most Innovative Companies in the World.  PatientsLikeMe made #23 on the list alongside big league companies like Facebook, Disney, Novartis and Amazon.  PatientsLikeMe was also named the second most innovative company in healthcare, behind powerhouse General Electric (GE).  Read our news release about it here and the magazine article featuring one of our MS patients. To commemorate this honor, we’ve interviewed our President and Co-founder, Ben Heywood, in our first-ever PatientsLikeMe podcast (hosted by our own Aaron Fleishman).  Listen in!

Speaking of innovation, what do Bill Gates, Al Gore, Steven Hawking, Goldie Hawn, David Blaine and PatientsLikeMe Co-founder Jamie Heywood have in common? All of these innovators have graced the stage at a TED event. A nonprofit that brings together innovative people from “Technology, Entertainment and Design,” TED’s mission is to simply spread ideas.  Isn’t that another way of saying “share?”  We do love that concept.  Here’s Jamie at TEDMED (the healthcare-focused TED event) sharing our vision on the future of medicine in his presentation, “The Big Idea My Brother Inspired.”

Finally, we’d like to give a shout out to some of people who have interviewed us since the start of 2010.  In the February 8th issue of TIME magazine (“Group Therapy”), reporter Bonnie Rochman caught up with Jamie to get his perspective on the power of online patient communities. Later that same week, Ben Heywood sat down with Staci and Steve from WAAM’s My Great Kid Radio show to discuss sharing, openness and the remarkable power of technology.   He also spoke with Ryan McBride from Xconomy about the growth of PatientsLikeMe as a business, so be sure to check it out.  Lastly, we just posted a blog interview with reporter and author Thomas Goetz regarding his newly released book, “The Decision Tree” (featuring PatientsLikeMe and our community members).

Stay tuned for more 2010 interviews, announcements, and podcasts right here on our blog.

PatientsLikeMe member lscanlon

Interview with Thomas Goetz
(author of The Decision Tree)

Posted February 17th, 2010 by

the-decision-treeIn March 2008, a story by Thomas Goetz appeared in the New York Times magazine about PatientsLikeMe (“Practicing Patients”). Thomas has now expanded on that reporting with a new book, The Decision Tree, that explores how new tools like PatientsLikeMe can help individuals engage in their health and make better, more informed choices. We asked Thomas some questions about his book.

plm_130x96 (PatientsLikeMe) What inspiration did you take from PatientsLikeMe? What did you want to say in a book that you didn’t say in the magazine story?
(Goetz) When I wrote the story two years ago, PatientsLikeMe was a much smaller community – just 7,000 members, compared to more than 50,000 today. But it was already clearly a phenomenon: the members were engaged in their health in unprecedented ways. In reporting the story, I realized that what’s going on at PatientsLikeMe is part of a larger phenomenon: new technologies like the Internet that let individuals use their health data. At PatientsLikeMe, this is happening among people with particular health concerns, from ALS to mood. But the same ideas are spreading to preventive health and diagnostic health, and my book is an attempt to capture the entire phenomenon and opportunity here.
plm_130x96 (PatientsLikeMe) Are PatientsLikeMe members unique? Do you have to be an information junkie to delve into your health data, or is this something that anybody can engage in?
thomas-goetz-300x3003 (Goetz) I spoke to several PatientsLikeMe members for my story and even more for my book. They are literally on the first page and all the way through to the last chapter. For a writer, PatientsLikeMe members are exemplary patients – engaged, articulate, and passionate about PatientsLikeMe. And yes, PatientsLikeMe members are unique, insofar as they are on the vanguard of healthcare. They have tapped into something that the rest of the U.S. and the world is just realizing: that it can be incredibly powerful and healthful to get granular with our health information and – even more importantly – to share that information with others, to use it to build new insights. But they’re not unique, I don’t think, in the sense of being all that different from the rest of us. I think they’ve hit on something that can work for vastly larger population. More people need ways to engage with their health data and to turn it into inspiration.
plm_130x96 (PatientsLikeMe) What do you hope comes out of the book? Who do you hope reads it?
thomas-goetz-300x3003 (Goetz) At this point, I’ve spent several years talking to people who have heard the gospel of engaged patients and collective wisdom. PatientsLikeMe and the other companies in the book have created something remarkable – ways to get people involved in their health – and to those in-the-know, it starts to seem like common sense. Of course giving people a way to engage in and share their data is a road to better health!But in truth, this is still very much the exception in the healthcare world. The rest of the world hasn’t heard the good word – they are still in the old paradigm where the patient is something to fix rather than someone to engage. My hope is that, just as PatientsLikeMe has spread from a mere 7,000 members to the 50,000 today, so the broader message of patient engagement is a contagious, even viral, philosophy. I truly believe that the message in the book – that when people have a way to engage in their health, they can have better health – is something that can truly improve the lives of many, many people.
plm_130x96 (PatientsLikeMe) Can you share something you learned in writing the book?
thomas-goetz-300x3003 (Goetz) Among the many things that I hope PatientsLikeMe members would enjoy learning about in the book, one thing stands out: the story of the Health Seekers. It turns out that in 19th century America, an important part of the pioneering westward expansion was driven by patients – specifically people with tuberculosis, seeking better health. These health seekers were exceptional, especially at the time, because they broke away from the way they were told to deal with their disease, which was basically to do as little as possible and waste away. The Health Seekers rejected this idea and decided that they would not only take charge of their health, but that they would forge a new direction for the country. In many ways, I see the Health Seekers as the ancestors of the PatientsLikeMe community – people who are taking charge of their health and building something new. And in the case of PatientsLikeMe, they are building something that will help thousands of others, something that gets more powerful with every new contribution and entry.I’m proud to have PatientsLikeMe in my book, and I’m incredibly grateful to the PatientsLikeMe members who agreed to share their story not just with their PatientsLikeMe brethren but with the general public. My hope is that their stories will help lead other people to better health.
plm_130x96 (PatientsLikeMe) It’s our hope too.  Thanks Thomas!

Thomas Goetz’s book The Decision Tree is available for purchase at Amazon.com here.

PatientsLikeMe Acquires Online Pain Management Company, ReliefInsite

Posted February 16th, 2010 by

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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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.

picture-1Pain 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.

# # #

PatientsLikeMe member lscanlon

Manage your epilepsy like a PRO

Posted February 11th, 2010 by

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!

PatientsLikeMe member pwicks