Q&A With Dyan Bryson from Retrophin May 29, 2015 by Kylie Karley Recently, we got a chance to chat with Dyan Bryson, the Patient Advocacy Director at Retrophin, Inc. – the pharmaceutical company that is sponsoring the DUET study for FSGS patients. We took this opportunity to ask Dyan why clinical research – and the DUET study in particular – is important for Nephrotic Syndrome patients. Keep reading to see our Q&A session with Dyan! NephCure Kidney International: Why do you enjoy/what inspires you about working in the pharmaceutical industry? Dyan Bryson: I first came into this industry thinking I would stay 3 years, that was almost thirty years ago. I simply fell in love with the industry and its potential to help people. I came in rather naively, and at one point I became completely fed up with the lack of focus on patients. I am now back in love as the industry is finally moving towards fulfilling its potential to really help patients, not just support its brands. I love being in an industry where companies that have awakened to putting the patient at the center of its business are also profitable – doing well by doing good. These companies can be examples to others that this can be done. We have realized that we need to support people after they have filled our prescriptions; we have to develop strategies to support their caregivers; we have to support people by realizing that the therapeutic area our drug may be managing is just one of a whole list of things a patient, a person, has to manage in their lives. NKI: What makes participating in DUET a unique opportunity for the NS community? DB: Participating in a clinical trial, especially DUET, is an opportunity to contribute to the knowledge and health of the community. Of course, I am biased, but as a patient advocate I know that if we truly have a drug that can help patients with this rare condition – a condition that has no existing therapy – it can help so many people in the long run. Participation allows us to collect data that may help other people avoid the current long term outcomes of end stage renal disease and transplant. Although we as the drug maker will profit, so will the community through better quality of life. If you participate you can contribute to bettering the health of others. NKI: Why is it so important for rare disease patients and their families to care about research? DB: Many times it is the family of, or person who has, a rare disease that brings that disease to the attention of researchers. Many times if that interaction did not happen there would be no awareness of the disease and, hence, no research. Rare diseases are not like diabetes – researchers, drug companies understand the impact of diabetes. Payers understand the impact diabetes makes on their bottom line as they pay the costs of diabetes management. All stakeholders have clear incentives to manage the disease better. But for a disease where there is low awareness there is little understanding of the impact on a person’s health, hence little understanding of why to do the research. Patients with a rare disease, and their care partners, are the experts on that disease as they manage it 24/7. They are the ones that can drive the understanding of the disease and interest in doing research on that disease like no one else. I still remember the first time that, as a sales representative, my company brought a patient in to talk about the therapeutic area for which we were about to launch a drug. We all had studied and been tested for months to be certified, to confirm we knew the area well enough to talk about it with the physicians we called on. Now, finally, we were at the launch meeting to get our marching orders and celebrate the launch – fun and work at the same time. So, the patient gets on the stage, we had no idea what we are in for. The patient told their story. Soon we were ALL in tears. That one patient helped us understand why were really there. We understood their day-to-day and now we were on a mission to help. Twenty-five years later I can still see that patient on that stage; that experience informs me every single day. That is the impact of a patient telling their story, motivating and inspiring those who can do something about the disease take action. NKI: What is your suggestion for patients that want to learn more and be more involved in clinical research? DB: I know it can be daunting even to think about participating in a clinical trial. You think you are going to be a guinea pig, I understand and so do many others. There is a tidal wave of information out there that can help understand how trials work and help people to understand the process. The US government has even issued grants to researchers all over the country to figure out how to better inform the public about clinical trial participation (http://www.nih.gov/health/clinicaltrials/index.htm) Certainly on the NephCure site (https://nephcure.org/wp-content/uploads/2014/08/Clinical-Research-one-sheet-9162014.pdf) Also, several healthcare advocacy groups have sites that contain materials in several languages to discuss clinical trial involvement: National Minority Quality Forum “Are you in” Campaign – http://www.nih.gov/health/clinicaltrials/index.htm National Medical Association – Project IMPACT – http://www.impact.nmanet.org/about NKI: What do you want a participant to know before they decide to volunteer for a clinical trial? DB: The managers of clinical trials are usually very good about providing access to information about their trial. Scour the site provided, read the materials you are given before you make a decision. Know that the managers of that trial have had to put the trial protocol, all trial materials through a review of a professional board that understands the therapeutic area for which the trial was designed. This board is called an Institutional Review Board (IRB). The purpose of the IRB is to ensure that all human subject research be conducted in accordance with all federal, institutional, and ethical guidelines. This IRB oversees the entire trial, meets at points throughout the trial and makes independent assessments to ensure the safety and well-being of the study participants. In other words, the drug company or other researchers are running the trial with considerable oversight. The study participant is not on their own.
Debunking Clinical Trials Myths May 29, 2015 by Kylie Karley MYTH: Participating in a clinical trial will just make me a “human guinea pig” FACT: A common fear is that those giving you care in a clinical trial won’t treat you like a person or a patient. However, the opposite is true. Participating in a clinical trial gives you access to some of the best care available to patients. Also, strict ethical and regulatory guidelines are in place to ensure that each participant is treated fairly and respectfully, not to mention that all drugs in clinical trial phase go though a vigorous testing process before they are approved for use in a clinical trial. MYTH: I like my doctor, and if I participate in a trial, I won’t be able to see them anymore FACT: This is not true. Participants are encouraged to continue seeing their regular doctor in addition to any appointments they may have with the trial staff. A physician on the clinical trial staff does not replace your regular doctor. MYTH: If I participate in a clinical trial, I’ll have to go off of the medications that keep me in remission FACT: Each trial has a different protocol, or set of rules. Some trials have a protocol that allows participants to stay on current medicines. Or, if a trial asks participants to stop taking their current medications, participants are closely monitored and immediately given their original medicine and dosage if their symptoms worsen. The health of each participant is top priority for every clinical trial team. MYTH: There are no trials happening near me, therefore I can’t participate FACT: There are currently 17 clinical trials that are recruiting patients with Nephrotic Syndrome diseases at nearly 300 sites throughout the United States. If there is not a site close to you, many trials will pay for your travel (like the DUET study!). MYTH: If a clinical trial will help me, my doctor will tell me about it FACT: While we all love our doctors and respect their opinion, the truth is that they are extremely busy. We can’t expect them to save lives everyday and remember every clinical trial going on. Sometimes doctors just don’t know about clinical trials, or are unsure if their patients are interested. The best approach to participating in research is to do some research yourself and then take questions to them. If your doctor is unaware of a trial, they can find out more information and help you make a decision. However, please remember that only YOU can make the decision to advance research to help find better treatment options by participating in a clinical trial. Click HERE to learn more about clinical drug trials
Director of Research attends RDCRN Face to Face Meeting April 1, 2015 by Kylie Karley Marilyn with Dr. Pam McInnes – Deputy Director, National Center for Advancing Translational Sciences (NCATS) 1/1/2015 – This week, Marilyn Hailperin, NKI’s National Director of Research attended the Rare Disease Clinical Research Network (RDCRN)’s Coalition of Patient Advocacy Groups (CPAG) face-to-face meeting in Rockville, MD. The meeting included patient advocacy groups that are associated with the rare disease consortia funded by the RDCRN (a program of the NIH), including the Nephrotic Syndrome Study Network (NEPTUNE).
2014 Research In Review February 20, 2015 by Kylie Karley What a Year We Had in 2014! Thank you to all those who collaborate with NephCure Kidney International (NKI) to advance its mission to support research leading to better treatments and cures for those suffering with FSGS and other Nephrotic Syndrome diseases. 2014 saw several major successes in the research world that move us closer to achieving our goals. Below is a summary of NKI’s 2014 research-related accomplishments. NEPHCURE KIDNEY NETWORK PATIENT REGISTRY Hundreds of people with primary FSGS and related glomerular diseases enrolled in the newly launched NephCure Kidney Network Patient Registry. These patients and care givers of patients are at the forefront of a movement called “patient powered research”. By sharing and comparing data contributed by patients about their health and quality of life, we can accelerate the development of new treatments, including new drugs. It is important for people with a rare disease to STAND UP & BE COUNTED to ensure research focuses on the issues of greatest importance to patients and their families. To learn more or enroll, click HERE. RESEARCH GRANTS In 2014, the NKI grant program committed over $2.6 MM in support for scientific investigations that advance knowledge about the cause of NS disease, evaluate possible drug targets and help physician/scientists track the natural history of these rare diseases. Click HERE for more information about grants awarded to investigators at Washington University Medical Center, Boston Children’s Hospital and Rush University. For more information about the research consortia we support, click HERE for the Nephrotic Syndrome Study Network and HERE to learn more about CureGN. CLINICAL TRIALS Patient participation in clinical research is essential to advance new treatments. In 2014, NKI launched several new programs to educate patients and increase awareness of opportunities to participate in research. The NEW Clinical Trial Finder website lists 16 studies actively recruiting participants in the US and Canada and another 18 around the world. We launched a Clinical Trial Ambassadors Program to recruit and train volunteers to help educate patients about the benefits (and risks) to participate in clinical trials and educate the medical community about the need for more research into treatments for NS. We posted, tweeted, emailed and sent letters about NEW FSGS drug trials. Our activities have raised awareness for potential participants and increased the interest of biopharmaceutical companies in working with NKI to advance new drug trials. CATALYZE, COLLABORATE AND CONVENE NKI is dedicated to being a catalyst along the research continuum: from support for basic science research to working with the biopharmaceutical industry and the FDA to understand how we might increase the numbers of new drugs moving into the drug approval pipeline and, finally, how to rapidly integrate new treatments into health care practice. 2014 was a busy year as we joined forces with our partners in healthcare and research to promote better treatments and faster cures. The advocacy work of NKI volunteers, staff and our professional partners in Washington, D.C. was instrumental in significant NEW FUNDING for FSGS and NS research in 2014. The National Center for Advancing Translational Sciences (NCATS) at the National Institute of Health (NIH) awarded $10 MM in new funding to continue the Nephrotic Syndrome Study Network (NEPTUNE), a consortium of research centers in the US and Canada collecting and analyzing data about patients newly diagnosed with primary NS diseases. NKI was a principal funder of the first phase of this study and continues to support and participate in its expansion. A second institute in NIH, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) awarded a new $20 MM, 5- year grant to follow patients with FSGS, Minimal Change Disease, Membranous Nephropathy and IgA Nephropathy. NKI was instrumental in collaborating with NIH to get this funding. Click HERE. For the first time, FSGS is listed as a condition eligible to receive research grants through the Department of Defenses’ Peer-Reviewed Medical Research Program. Because FSGS is listed, FSGS researchers have an opportunity to compete for grants from a NEW funding source. The total amount in the DoD’s program is $247.5 Million! NKI is a leader in assembling, facilitating and promoting professional meetings that bring together partners from industry and the academic world along with patients and their advocates to promote NS health research. In 2014, NKI: Organized the 10th International Podocyte Conference in Freiburg, Germany. This conference brings together researchers from a variety of scientific disciplines and from around the world to promote interactions and new collaborations and to investigate emerging directions in podocyte and glomerular biology research. Convened the PRO in Glomerular Disease Scientific Conference with representatives from academic medical centers, the biopharmaceutical industry, the NIH and the FDA to pursue the development of Patient Reporting Outcome (PRO) instruments to advance new drug trials for FSGS and NS. (A PRO is any report of the status of a patient’s health condition that comes directly from the patient.) An FDA-accepted PRO instrument can accelerate the number of applications to the FDA for new drug trials. NKI continues to lead the development of a PRO development consortium. Convened the third NephCure Research Accelerator Meeting that brought together physicians and scientists with representatives from more than a half dozen biopharmaceutical companies with an interest in drug development for kidney disease. Research Accelerator Meetings were initiated by NephCure to engage these two scientific groups in an exchange of knowledge that will accelerate translation of basic science discoveries to new treatments for people with NS diseases. Presented a session on the NephCure Kidney Network and patient-powered research to the US Summit of Kidney Organizations hosted by the America Society of Nephrologists at its 2014 conference in Philadelphia. NKI continues to be a leader in bringing the voice of patients to the forefront of advocacy for new treatments for chronic kidney diseases such as FSGS, Minimal Change Disease and Membranous Nephropathy.
Seeking Volunteers for NEW Clinical Trials Ambassador Program November 5, 2014 by Kylie Karley Have you ever participated in a clinical research study? Are you willing to share your experience with others? If so, then you would make a fantastic addition to our newest initiative to find better treatments and a cure for Nephrotic Syndrome: the Clinical Trials Ambassador Program! NKI is seeking volunteers from across the country to act as Ambassadors to other patients and the medical community. Ambassadors will spread knowledge and information about the importance of clinical research in the search for better treatment options and a cure. Ambassadors can expect to spend between 5 and 10 hours a month volunteering in various ways that will help spread awareness and knowledge about clinical research. If you are interested in learning more about the Clinical Trials Ambassador Program, contact Chelsey Fix at cfix@nephcure.org.
NEW!! Clinical Studies Map! October 14, 2014 by Kylie Karley NephCure Kidney International Introduces Clinical Research Map October 14, 2014 We are excited to introduce a new user-friendly, interactive clinical research map. This map includes current clinical research sites and studies available to patients affected by FSGS and other Nephrotic Syndrome diseases. The map, which can be found here, will help patients sort through studies available to them. Categories include studies specific to adults, pediatrics, Nephrotic Syndrome, and FSGS, to name a few. “Patient participation is key to advancing research and treatment options available to patients,” says Marilyn Hailperin, the National Director of Research at NephCure. “This site map allows patients to view studies underway in their area.” This map is another way NephCure is living out our mission to support research seeking the causes of Nephrotic Syndrome diseases, to improve current treatments and, ultimately, to find a cure. Here at NephCure, we value the supportive role we play for our patients. Spreading the word about clinical research to our patients is a crucial aspect of this role, because, after all, patients power research! Link to the map: here. For more information about the risks and benefits of participating in clinical research, click here.