Budget and Operating Philosophy
The Life Raft Group became a 501(C)3 non-profit corporation, in the State of New Jersey, on June 10, 2002. Our fiscal year is the same as the calendar year. The Life Raft Group budget is adapted after formal approval by its Board of Directors.
Our philosophy is to have a transparent budget with professional checks and balances, and to focus our limited resources on those services most vital to the survival of GIST patients.
All contributions are acknowledged in writing, as required by the IRS. We accept no revenue from advertising. Each year we file federal form 990 to the IRS, as required by law for non-profit organizations. Copies of this form can be found on our website after they have been submitted In addition, the Life Raft Group has retained the CPA firm of Sobel & Co., LLC of Livingston, NJ to conduct an independent financial audit each year and will make a copy of these reports available upon request. This audit is performed on a pro bono basis. We also welcome questions.
Small, Paid Core Staff: Although we rely heavily upon a growing number of dedicated and skilled volunteers, our experience is that we also need to support a small, paid core staff to provide coordination and consistency and to make sure that actions needed to ensure the survival of GIST patients do not go unmet while we are waiting for a volunteer to assume and carry out that responsibility. We simply cannot afford the lethal time gaps that sole dependency upon volunteers creates.
We have no full time administrators. The time of the staff primarily goes to program services to support our mission to help GIST patients survive, including critical patient driven research, international surveillance of potential new treatments, telephone advice to patients about medical options and financial assistance, information and education of laypersons and medical professionals, emotional, logistical and financial support to patients and caregivers, an expanding advocacy role to influence support for patient care, research and clinical trials, an expanding outreach effort to find misdiagnosed and inadequately treated patients, a growing international role, and new initiatives to create a center of excellence for pediatric GIST and a collaborative tumor bank. In 2006, we began funding research grants.
We maintain a state-of-the-art website that is visited by patients and physicians in over 100 countries and publish a professional monthly newsletter that is distributed to a growing number of laypersons and medical professionals. We also support a number of internet-based chat facilities that enable patients and caregivers from around the world to chat with each other, to exchange critical information and to provide encouragement and support.
Membership in the Life Raft Group is free, as is access to our website, newsletter and office-based assistance programs. Our support comes from corporate and individual donations and, unfortunately, from the families and friends of those who have died.
The Chief Executive Officer of the Life Raft Group is Norman J. Scherzer.
Survival Strategy: GIST is a deadly and rare disease that has attracted little interest except for the recent breakthroughs in genetic modeling and the discovery of Gleevec. Despite the recent attention brought about by the breakthrough drug Gleevec, GIST remains a rare disease that does not attract enough research interest either from government or the pharmaceutical industry. Most GIST patients in the world still remain undiagnosed and untreated.
As most GIST patients in the world still remain undiagnosed and untreated and as Gleevec initially helps to save the lives of about 85%, this simple fact means that for every 100 GIST patients that we can find by our outreach and surveillance efforts, that we can save 85!
We presented this three-point strategy at our membership meeting in Orlando, Florida on May 1, 2004:
- To think smart enough to leverage our small numbers and to overcome the rarity of GIST
- To use the combination of our own research and the ability to publish it, in our newsletter and on our website, as leverage to carve out a seat at the decision making table
- To maintain a core staff, and funding, to provide a consistent and all out survival effort
Life Raft Group Research
Because it is somewhat unusual, if not controversial, for patient groups to conduct their own research we are going to address this activity in somewhat more detail.
The Life Raft Group has created its own patient-driven database and publishes information about treatment efficacy in its newsletter and on its website that enables both physicians and patients to access life-saving information far more quickly than would have been the case had they waited for the much slower flow of information from formal clinical trials. This helps to save lives!
We have also broken ground in a number of new areas, including the development of a new scale to allow patients to rate the severity of their side effects, and a pioneer study to evaluate the relationship between relapse and actual dosage levels. This helps to save lives!
The LRG was the first to report on Gleevec effectiveness for GIST patients, the first to report that Gleevec side effects got better over time, the first to report gender differences in side effects, the first to establish a pediatric GIST database, and the first to look at the critical differences between relapse and actual drug dosage and to discover that when one looks at actual patient dosage there is a significant difference between higher relapse rates and lower dosages of Gleevec. This helps to save lives!
New Initiatives: We are now focusing research efforts on creating a pediatric GIST research project and an international tumor base. This helps to save lives!
Confidentiality and Privacy
The LRG does everything possible to protect the confidentiality and privacy of its members. Our membership is free and is open to GIST patients and their families. Membership status can only be obtained, however, after a formal application has been screened and accepted. We do not accept anonymous members and instead ask all to identify themselves.
Our internet based chat facilities are closed to all but our own members. That permits us to exclude medical professionals, unless they are GIST patients themselves, researchers and marketers of various sorts.
We cleanse all data that we publish of all identifying information.
We never use any personal information without prior approval.
Unity: The GIST patient community has a number of groups, both lay and medical, that do not always agree on every issue. We are united, however, on the need to find ways to help GIST patients survive and to ultimately find a cure for this disease. The LRG is committed to collaborate with a growing number of organizations and seeks every opportunity to do so.
Time: In short, we are running out of time. As of our meeting in Orlando in May 2004, fifty of our members had already died. Urgency is our by-word. We do not have the luxury to let events progress “as usual” and will continue to do whatever we must to focus our limited resources on survival.
One of the most important functions of the Life Raft Group has been the collection of data from member patients and the development of a large and complex database. The Life Raft Group asks its patient members to provide a medical history when they join and to submit medical updates as they occur. In addition, we periodically conduct special surveys on such issues as side effects and the quality of medical care.
There has been some confusion about our data base project. Much of this has been based upon a misperception as to what a medical data base actually is. First and foremost, it is not a single focused work project with a defined time line. Instead it is a series of content management tools for helping us to assess problems and guide our decision making. It includes developing an international directory of sarcoma/GIST doctors (given that there is no Board Certification for either Sarcoma or GIST, this is particularly important), monitoring the longer term side effects of Gleevec, and most importantly identifying the longer term patterns of resistance that are evolving. It also includes identifying potential new drugs and compounds, new clinical trials, and new "straws" needed to keep GIST patients alive in the interim.
Some of this data is available in a timely way from traditional sources and when that is the case, there is no need for us to duplicate it. Some we need to develop on our own. When we do, we shall continue to ensure that any data we collect meet the highest professional and scientific standards and that means that it must be accurate, timely and relevant. The fact that we have the capacity to do that, and to disseminate what we learn through our Newsletter and our website, has created an extraordinary leverage for the Life Raft Group in relating to the medical and research community.
GIST is truly a rare cancer. As a result, little research had been done, and little information was available about its diagnosis and management. Gleevec represents a new category of drugs, called signal transduction inhibitors, and what little information about it initially came from the prior clinical trials for chronic myelogenous leukemia.
When the clinical trials began, there was no information about the interaction of GIST and Gleevec outside of the handful of patients participating in the trials and because of the early nature of the trials none had yet been published.
Historically, most GIST patients had originally been misdiagnosed, many with leiomyosarcoma. Where the term GIST had been used it was generally descriptive in nature and specific as to site (gastro-intestinal) and tumor type (stromal). Treatment decisions were often based primarily upon a small amount of research within the broader category of leiomoysarcoma patients, which, in turn, were often based upon the even broader category of soft tissue sarcoma patients.
Although early clinical trial GIST patients and caregivers began to exchange anecdotal information about Gleevec’s effectiveness and side effects on the internet, this interchange lacked scientific perspective and quality assurance checks and balances. There was no way of knowing how representative the comments were, although it was fast becoming clear that something extraordinary was going on.
The Life Raft Group began to organize its own data collection effort, with an initial focus on drug effectiveness. By February, 2001, less than six months from the enrollment of the first clinical trial patients, the group’s newsletter headlined “Initial Survey Shows High Response Rate.” The article went on to report the response of the first 16 Life Raft Group member patients to reach three months on Gleevec: 87.5 percent had reported tumor shrinkage. Subsequent newsletters reported on a significantly expanded database, culminating in an October 2001 comprehensive survey of side effects and a May 2004 study of the relationship between Gleevec dosage and relapse rates.
October 2001 also saw members of the Life Raft Group invited to Novartis Oncology Headquarters in New Jersey to present the side effect survey findings. Included in the presentation was an analysis of how the Life Raft Group data of side effects from the perspective of patients complemented the data on side effects from the perspective of the NCI: (National Cancer Institute) driven clinical trials. Further topics included an exploration of the issue of patient under-reporting of side effects, and the correlation of under-reporting with poor medical management.
Quality controls were set up, with survey instruments subjected to extensive pre-testing and critique. Questions were always addressed by going back to the contributing member for clarification. New survey instruments were developed for collecting patient-driven data, particularly for describing and evaluating side effects. Borrowing from the work that had been done on pain management, the group developed a whole new instrument for rating side effects, over time and from the perspective of the patients.
Confidentiality was meticulously followed and all data was cleaned of any potential identifying information prior to any reports, including any newsletter publications. Finally, we were mindful and sensitive to the protocol needs of the clinical research community and took care to try not to undermine their publication and meeting presentations.
The creation of this patient-driven medical database and the publication of data in our newsletters, has had far reaching consequences. The most immediate has been the empowerment of patients in managing their medical care and in relating to their doctors. The reality of a patient organization creating and publishing data in a timely manner, that had heretofore been the exclusive prerogative of the clinical research community, has been the unintended consequence of turning the traditional flow of clinical trial information upside down. For the first time, the clinical trial patient now sees the information first, as opposed to years later when it is finally tabulated and published in a peer review journal. The inevitable endpoint of this process is to change the role of the clinical trial patient from passive participant toward an interactive contributor in the clinical trial process.
From the onset, the Life Raft Group set high professional standards for its data collection efforts. To reduce data distortion, a 90 percent response rate was established as the minimum requirement before any data surveys would be closed and the data used. In no case did the group ever fall below this response rate standard. Although member participation always remained voluntary, peer pressure and a little old-fashioned nagging were utilized to meet reporting standards.
Why do we collect data?
We do so because the data that is compiled by the clinical researchers takes too long to be shared with us, does not include non-clinical trial members (ours does), looks at side effects according to the toxicity standards set by the National Cancer Institute and not at the reality of side effects from the perspective of the patient, and does not address issues such as the quality of medical care. In addition, the data summarized to date does not factor in the change in dosage and instead only looks at the starting dose under a concept called intent-to-treat.
Thus, collecting our own data is the only viable way to have current and accurate information about GIST and Gleevec, and ultimately other drugs. This is recognized by much of the medical community, many of whom refer to our published reports to bring themselves up to date.
The consequence of all of this is that our members are better informed and empowered in their relationships with their doctors. Mutual respect has replaced the doctor-guinea pig relationship in many medical settings. The Life Raft Group and its individual members have achieved a level of respect unprecedented in clinical trials and cancer patient care.
The issue of side effects helps to explain this. To quote from the October 2001 Side Effects Survey (October 2001 Newsletter):
Using the latest side effect data published by Novartis in a Memorandum to pharmacists dated August, 2001, we compared the frequency of reported side effects to Gleevec by the Life Raft Group to those reported by Novartis. The Novartis data refers only to patients using Gleevec for Chronic Myelogenous Leukemia. The Life Raft Group data refers only to patients using Gleevec for GIST.
For most side effects, the data is fairly similar, with the Life Raft Group reporting a higher amount of fatigue, diarrhea and skin problems, and the Novartis Group reporting a higher amount of edema, nausea and cramping. Reported reflux was about the same. Where there are significant differences is in the reporting of the severity of side effects, with the Life Raft Group tending to define a greater number of side effects as severe.
There are two major reasons for these differences.
The first is that Novartis, like all clinical researchers, uses the toxicity standards established by the NCI (National Cancer Institute), whereas the Life Raft Group uses its own patient severity rating scale.
The second is that Novartis, like all clinical researchers, derives its data from the reports of on line clinicians, whereas the Life Raft Group derives its data directly from the patient…
As is the case with all clinical trials, the GIST-Gleevec trial researchers used the NCI toxicity standards, with grades 3 and 4 events being reported by the U.S. trials and grades 2, 3, and 4 being reported by the European trials. Let’s compare the U.S. report, using grades 3 and 4 to define toxicity, to the Life Raft Group methodology. We will use as an example the fact that a clinical trial patient has diarrhea.
The U.S. ASCO Report notes that, of 145 study participants, there were 0 cases of diarrhea as a grade 3 or 4 toxicity event. The Life Raft Group notes that, of 61 study participants, there were 13 cases of diarrhea reported to be severe.
Let’s take a look at how this difference in reporting could occur.
Assume that a patient had three incidents of diarrhea per week prior to beginning Gleevec.
Assume that patient now has three incidents per day since beginning Gleevec.
Using the current NCI toxicity scale that situation would rate a one, on a scale of 0 to 5, with 0 being none and 5 being death.
Using the Life Raft Group Severity Scale, the patient might conclude that three episodes a day of diarrhea for the indefinite future, might rate a 7, on our scale of 0 to 10, thus qualifying for a ranking of severe. One might ask the NCI analyst who constructed the scale: How would you rate the severity of having to cope with an average of 3 episodes of diarrhea per day for the rest of your life?
Given that about half of those amongst the Life Raft Group who report severe side effects, report three or more, this poses an interesting dilemma. How do you combine the several different side effects into one quality of life whole? To some extent we have begun trying to do this by looking at functional effects, in addition to rating side effects separately.
At some later date, we may return to this subject in greater detail. Our purpose today was to point out the different perspectives of a patient group and a clinical trial group in looking at the severity of side effects, and to suggest that both may help understand what a patient is actually going through as he/she tries to live their life.
Individual entries of drug effectiveness and side effects are of limited value and, taken by themselves, are often misleading. It is critical that we have a representative and accurate sample of our patient members to accurately know how Gleevec, or another drug, is working. Individual entries in our spread sheets, like those posted to our internet chat facilities are fine as far as they go but they are frequently wrong if one wants to get an understanding as to what most patients are experiencing.
The data which we produce is valuable only because it is representative of a larger group and because it is compiled and analyzed based upon scientifically acceptable sampling standards. That is also a major reason that the clinical world, including Novartis, take us seriously. They know that we collect and present credible data.
The compilation and analysis of data is a complex process. For example, one of the components of the Gleevec clinical trials is that there is no placebo or control group. It is harder, as a result, to tease out what side effects are due to the drug and what is not. To help overcome that, we had to design our own survey instrument which compared and ranked side effects over various time periods, particularly including the period which preceded the drug. In many cases we found side effects to occur prior to taking Gleevec.
Life Raft Group Relapse Study
The Life Raft Group presented the results of its relapse study and found that Gleevec relapse was related to dosage and that actual dosage is a valuable analytical addition to intent to treat.
We analyzed relapse rates for 162 metastatic GIST patients whose initial response to Gleevec was shrinkage and who had been on Gleevec for at least one year. We compared two different methodologies: intent to treat (starting dosage) versus actual (delivered) dosage.
We found that using starting dosage tended to underestimate the relationship between higher doses and lower relapse rates. When we looked at actual dosage we found that there was a statistically significant difference between dosage and relapse rates, with higher doses correlated with lower relapse rates, particularly amongst females.
|n = 162||Based Upon Starting Dosage||Based Upon Actual Dosage|
|n =||Relapse||n =||Relapse|
note: This data is consistent with the findings by the ongoing European study that showed that time to progression was related to dosage and to a presentation at our meeting by Dr. Van Oosterom that that there was increased clearance of Gleevec in GIST patients over time (see above).
What do others have to say about our data collection?
What are the implications of patient-generated data?
This is powerful and compelling stuff! I remain incredibly impressed by the data-coordinating abilities of the Life Raft personnel. I see the major purpose of this sort of data as hypothethesis-generating. Unfortunately, it cannot be free of bias and thus cannot stand by itself, but it certainly can point investigators and the Company in the right direction and let us know what we need to be looking at more closely. Thus, its importance cannot be overstated.
July-August, 2001 Newsletter: Charles Blanke, M.D. (Dr. Blanke is the Director, GI Oncology Program, Oregon Cancer Institute)
Norman Scherzer…has been a driving force in spreading the word about Gleevec to Internet enthusiasts.
The Life Raft Group that Norman helped to start (and has since become the leader of) has provided various people, patients, doctors, investigators, with a unique kind of data bank that cannot be replicated anywhere else, not even in patient trials.
As Norman Scherzer describes it, the Life Raft Group is a multi-faceted organization/community of GIST Gleevec clinical trial patients/loved ones. Life Raft Group members collect their own data and publish the findings in their monthly newsletter “We’ve turned the clinical research world upside down,” suggests Norman. “It can take years for patient trial data to filer down into the general medical community and even longer to reach the general public; the Life Raft Group offers that data in real-time. “When we look at side effects, however, we believe that we are more sensitive and accurate in reporting on this from the perspective of the patient and that we offer timely information to doctors as well as patients, particularly in the early stages of the clinical trials.”
A major goal of the Life Raft Group, as Norman notes, is to help patients and doctors understand the nature and the management of the side effects of Gleevec. “Our ability to collect timely side effects information from a patient’s point of view,” he comments, “cannot be replicated by the medical community. We are the front line. We raise red flags.”
“In the past one cancer patient would stand at the proverbial water cooler and ask another cancer patient, “How are you doing?” We supplant that practice, but we provide many conversations and many water coolers.”
The Life Raft Group has certain rules to protect its members. Its online discussion group, for one thing, is not a public site. To join, a member must fill out an application form. No doctors are allowed in. Patients want to be free to talk openly about a doctor without that doctor on-line. Patient confidentiality is jealousy guarded when sharing information and patient identifying information is never included in any published data.
Magic Cancer Bullet, Daniel Vasella, M.D. with Robert Slater, Harper Business, June 2003 (Dr.Vasella is the CEO of Novarits)
The man many consider the George Washington of e-patient-directed medical research is Norman Scherzer, a tall, graying 60-ish public health professional who until recently worked at the Centers for Disease Control and the New York City Department of Health.
Bypassing the “Lethal Lag Time”
“One of the great benefits of patient-initiated research is its speed,” Scherzer says. ….professional research has a built-in lethal lag time-a period of delay between the time some people know about an important medical breakthrough and the time everyone knows. And, as a result, many patients who could have been saved by the latest treatments die unnecessarily. …Physicians are just as much a victim of this lethal lag time as their patients.
In October, 2001, the group published a review of Gleevec’s side effects. And in addition to collecting the usual data, the Life Raft Group’s study broke new ground in several areas:
It provided data on the quality of the clinical care available to study participants at each of the centers conducting clinical trials.
It attempted to evaluate the information sources patients relied upon.
It developed a methodology by which patients could, in effect, serve as their own control group.
It introduced a new scale for rating the severity of side effects from the patient’s point of view, as opposed to the toxicity standards established for clinical trials by the National Cancer Institute.
Tom Ferguson, M.D., Editor and publisher of the Ferguson Report, the Newsletter of Online Health (www.fergusonreport.com) and author of Health Online: How to Find Health Information, Support Groups and Self-Help Communities in Cyberspace
Dr. Ferguson is a Senior Research Fellow for Online Health at the Pew Internet and American Life Project in Wash. D.C, an Adjunct Professor of Health Informatics at the University of Texas Health Science Center in Houston and a Senior Associate at Boston’s Center for Clinical Computing, a medical computing think-tank associated with Harvard Medical School.
The Future of Medical Research
Such patient-initiated medical research projects turn the traditional medical paradigm upside down. Before the Internet, researchers called the shots, research trials were structured to meet their needs, and the delay between discovery and dissemination was considered an unavoidable part of the process.
The prospect of research-oriented online support groups offers a number of appealing scenarios. Patient groups could design and conduct their own studies, collecting their own data, analyzing their results, and publishing their results. They could provide researchers with access to perfectly targeted study populations at little of no cost. But whatever role they play, once they become active players in medical research, patient groups will demand a voice in deciding what should be studied and how that research will be conducted. And while such e-patient initiatives may encounter some resistance, in the end it seems likely that the financially-strapped medical research establishment will come to consider such e-patient research and offer it can’t afford to refuse…
The new research model pioneered by the Life Raft Group is making it possible for patients and family members to contribute to clinical research for their diseases in unprecedented ways. I predict that we’ll be seeing a lot more of this sort of thing in the years to come.
George Demetri, M.D., Medical Director of the Center for Sarcoma and Bone Oncology at Boston’s Dana-Farber Cancer Center
Cyberspace has spawned a powerful breed of advocates. Consider the case of Gleevec. Members of an internet group spread the word about Gleevec, bringing scores of patients into new clinical trials. Then they went one step further, creating a group called Life Raft, where participants in one trial could share information about the drug’s efficacy, its side effects and their interactions with doctors. Conservative clinical oncologists cringed. Life Raft represented a major break with medical precedence.
Wired Magazine, September 2001
As these examples show, patient-driven research will become more and more important, and will provide an example of the way things will go in the future. As other patient groups begin generating their own medical data, it will change the relationships between research professionals, clinicians, and patients quite dramatically. These groups have been phenomenally successful in recruiting new patients for badly needed clinical trials. And I can assure you that their potential role in medical research is not lost on the drug companies.
Those patients taking Gleevec do not consider themselves guinea pigs. They are the recipients of experimental medicine.
Gilles Frydman, President of ACOR
You know, despite the terrible overuse of the term “paradigm shift,” I believe that when patients came together the way the GIST group did, and accomplish what you have accomplished, even the father of the term … would agree it is the appropriate description. It is an honor to know a person who is part of the solution.
Bernard Glassman, Dept. of Health Behavior and Health Education, School of Public Health, University of North Carolina and Deputy Director for Population Sciences, Lindberger Cancer Center
On behalf of our entire research team, I want to express my sincere thanks for your timely, articulate and enthusiastic support for our proposal to the Robert Wood Johnson Foundation.
Life Raft is unusually important to our communications research, and for many of the same reasons that it is important to the future of clinical research. We believe that Life Raft will turn out to be the first in what will become a wave of survivor-involved, self-researching virtual teams. As such, Life Raft offers both a model and an inspiration to other survivors and other researchers.
As genomics, proteomics and pharmacology progress, clinical trials will depend increasingly on smaller pools of participants who are genetically appropriate for a particular targeted therapy. This will not be possible in just a few geographical areas, and will require the kind of connectedness and cooperation Life Raft has been able to achieve. At the same time, we will need to identify the risks and benefits of new treatments more rapidly than ever, because the sheer numbers of patients will be smaller. Again, the Life Raft approach to self-monitoring offers enormous potential as well as proven results.
Life Raft has set the standard for self-study by survivor groups, and your success has made it all the more possible for our team to work effectively with yours.
Dr. Barbara Rimer, Professor Health Behavior and Health Education, Dept. of Health Behavior and Health Education, School of Public Health, University of North Carolina and Deputy Director for Population Sciences, Lindberger Cancer Center