Category: RSDS General Info

To our friends and colleagues in the CRPS Community, those in the wider Rare Disease Community and to the Representatives and Senators of Congress who are concerned for the welfare of people who suffer rare diseases:

The Reflex Sympathetic Dystrophy Syndrome Association (RSDSA) represents the interests of people with Complex Regional Pain Syndrome (CRPS). Pursuant to its mission of support, advocacy, education, and research, RSDSA seeks to promote safe and effective treatments for CRPS and to advocate for insurance coverage for such treatments. Therefore, RSDSA is encouraged by two pieces of proposed legislation, the Protect Rare Act, which mandates insurance coverage for FDA-approved treatments for rare and life-threatening diseases, and the Promising Pathways Act 2.0 (PPA2.0), which amends Title 21 to provide for time-limited FDA approval of treatments for rare and life-threatening diseases, which have phase 1 evidence of safety, complete or ongoing phase 2 evidence of safety and a minimum of pilot (probable) evidence of efficacy.

RSDSA supports both bills in principle. RSDSA holds that our concerns over the weaknesses of these bills can be resolved. RSDSA is concerned by the disagreement that has arisen between Senator Braun’s office, a sponsor of PPA2.0, and the National Organization of Rare Diseases (NORD). NORD opposes some features of PPA2.0, and, therefore, its passage. RSDSA contends that a cooperative, conciliatory resolution to any conflict is possible. NORD has been a steadfast and respected leader of the rare disease community, and its concerns must be considered seriously.

RSDSA’s concerns with Protect Rare and PPA2.0 are twofold:

First: Both Protect Rare and PPA2.0 mandate insurance coverage for FDA-approved treatments. On the other hand, rare diseases are often treated with treatments off-label. Ketamine is a particularly important example of a drug that is approved by the FDA, but not for CRPS. Protect Rare specifies “approved” treatments only. The wording of PPA2.0 is ambiguous, but a reasonable interpretation of the text indicates that only new drugs are eligible for the time-limited approval that PPA2.0 provides. Neither Protect Rare nor PPA2.0 provide a mechanism to repurpose and relabel ketamine (or any other treatment for any rare or life-threatening disease) to mandate insurance coverage.

A resolution to this concern would be the deletion of the word “new” [drug] from the legislation, so that PPA2.0 provides a pathway for FDA approval of any drug, currently used off-label, for the treatment of CRPS (or any rare disease). Consequently, the relabeling of such drugs would make them eligible for insurance coverage under Protect Rare.

Second: The limited evidence of safety and efficacy required for FDA approval of treatments under PPA2.0, a dispositive concern for NORD, can result in potential or real danger to patients and wasted time when ineffective treatments have not been properly, and repeatedly studied in scientifically robust trials.

A potential resolution of this concern lies in PPA2.0’s requirement that applicants for approval of a drug or device maintain a registry of patients receiving the treatment. This is an ideal application for artificial intelligence (AI), which continually monitors the registries (faster and probably more accurately than a human “patient safety committee”). Using a comprehensive set of outcome measures, AI monitors symptoms and signs of treatment response and adverse effects of the treatment. This real-time monitoring is inferior to evidence generated from randomized, controlled trials of safety and efficacy, but it may suffice.

RSDSA desires both approval of safe and effective treatments and mandated insurance coverage for them. RSDSA calls on all stakeholders in the rare disease community and Congress to come together to resolve differences and concerns to entail full, universal support of this groundbreaking legislation.

Sincerely,

RSDSA Board of Directors

Feel free to send any feedback on this topic to RSDSA directly via [email protected].

Written by RSDSA Intern Samantha Rose

How did you start working for the organization?
In the 1990s, I was working on the board of the National Organization for Rare Diseases (NORD). During this time, Dr. Robert J. Schwartzman convinced Roe Davis, Frank Davis, and Audrey Thomas to start the RSDSA Organization to support CRPS warriors. In search of a skilled and compassionate person to lead the organization, Mrs. Davis called Abbey Meyers, the founder of NORD, who recommended me for the position that I feel grateful to have had for the past twenty-five years. We then brought the organization to Milford, Connecticut.

How has the organization and its mission changed over the years?
When RSDSA first began, it had a newsletter, just as it does today. However, the newsletter today looks much different, and serves a very different purpose. Back then, it was filled with personal stories of “woah” about the struggles of CRPS. However, we have since changed the newsletter to make it a more informative and supportive outlet for CRPS patients, focusing on educating and giving hope to those with CRPS. “Hope” has become an integral part of our message and mission, one that we have– and will continue to– expand over the years.

Which aspect of the organization’s progress are you most proud of?
The courses that we have provided for doctors and nurses have been transformative in creating a medical world where more doctors are aware of, have interest in, and have the tools to help CRPS warriors. Additionally, the way that our organization has grown into the national, go-to organization for CRPS is incredible. We have built a patient-friendly community where everyone tries to help each other, regardless of physical distance. Planting seeds of hope has become much easier and more widespread with new social media outreach programs, much thanks to our social media manager, Alexis Johnson. In addition to increasing engagement on our virtual platforms, our Facebook Lives since the pandemic have educated and supported the RSDSA community. This is an improvement from before the pandemic, when we often had in-person conferences with 140-200 people attending. Now, we are able to have 1,400+ people from all around the country listen to guest speakers in their own homes. I am particularly proud of the Jenkins Patient System Program to memorialize Brad Jenkins, whose mom helped to start a fund for people to avoid eviction and get much-needed medications.

Which aspect of being executive director did you enjoy the most?
Often, new CRPS patients spend a quick fifteen minutes with their doctor discussing CRPS when they first are diagnosed, not receiving nearly the kind of education that they need in order to seek help and support. Therefore, being able to educate them, support them, and give them hope throughout their journey has been an incredibly rewarding experience. Whether it be through linking patients with financial support, service dogs, legal aid, medical professionals, or accommodations at work, I loved helping and supporting people. I specifically remember an instance where I was able to get a tank of propane filled for a warrior who could not afford it during a blizzard, and how the simple act created a huge difference in somebody’s life.

Which aspect of being executive director did you least enjoy?
While I loved being able to help people, the hardest moments of my day-to-day job were those when I was not able to provide the kind of help that somebody needed. Often, this came in the form of family members doubting their CRPS and abandoning them, diminishing the web of support and encouragement necessary throughout this journey. It also disappointed me when people did not find RSDSA until they were months and years into their diagnosis with no hope– we must try to reach people earlier in their illness to provide the kind of support they need.

What was your day-to-day life like as the executive director of RSDSA?
Before COVID, I often participated in many calls a day to help warriors when they sought more direction. This has changed, as most inquiries and responses now are sent through email. Every day on the job is a learning experience, as hearing people’s stories and needs allows me to explore what is lacking and how to improve our services. I also look for thought leaders and try to help curate treatment teams of doctors, physical therapists, occupational therapists, and aids for warriors to lead them in the correct direction.

How has the CRPS science changed over time?
Previously, scientists lumped all CRPS patients together for clinical trials, only differentiating subjects by Type I and Type II classifications. However, we no longer conceptualize CRPS as a homogeneous disease, largely due to a subset study conducted by Dr. Stephen Bruehl and Dr. Norman Harden. These doctors are the thought leaders of CRPS science, and have formed a collaborative team with which I have had great pleasure engaging. Through their work, we have been able to create five versions of clinical practice guidelines for CRPS. We also have used science to debunk the evidence-less theory that CRPS is solely psychological, validating the struggles of CRPS patients.

How can we expect the CRPS science to change in the future?
There currently is a study being conducted regarding neuroplasticity and treatments that are brain-based as opposed to targeting the afflicted limb. The Cleveland Clinic also recently received a multi-million dollar grant to evaluate a stem cell treatment for CRPS, with animal and human trials. We are also extremely hopeful about our CRPS subset study, which involves a consortium of three centers in Europe. My hope is that an international CRPS research consortium will come to fruition in the future; of course, this requires overcoming the long-standing challenge of not finding enough patients to participate in these studies.

What do you envision as the future direction of the organization?
For sixty years, ketamine has been known to be an effective treatment for chronic pain, but we have been barred by outrageous prices and legislative issues. This must change, hopefully through the work of the dedicated Jim Doulgeris with the RARE Act. Since 2016, there has been a war on people with pain because of misapplication of CDC and opiate guidelines, and we must fight back. This involves more research on CRPS and other rare diseases, which often is difficult because there is not adequate money to continually fund research grants. In the future, we might need to decide if the organization will mainly focus on research grants or support and education, or find other avenues for continued funding.

What are you most looking forward to during retirement?
I am excited to have more freedom and more time to explore the outdoors and go birdwatching and kayaking. In fact, my wife and I are going to see Christian Cooper, a famous birdwatcher and author, in Central Park. I am also an avid reader. I am excited to continue to give back to the community: my wife and I volunteer at a soup kitchen, and we look forward to other opportunities to help. Overall, I am looking forward to being present each day and treasuring every moment.

FROM THE AUTHOR

Jim, the RSDSA community and my family are so grateful for everything you have achieved and how much support you have provided over the past twenty-five years. Your passion for helping others and creating a better life for people never fails to shine through, whether it be in phone calls, emails, Facebook Lives, and everything in between. We wish you the best of luck and happiness in your retirement.

Best,
Samantha

It is with deep sadness that we share the news of the passing of RSDSA board member, R. Steven Shisler, Esq. His unexpected departure has left us all in a state of profound sorrow and loss.

Steve Shisler’s loss will be felt keenly throughout the entire CRPS community. He was a fierce & compassionate defender of those who suffer from this debilitating syndrome. Unfortunately, he knew all-too-well the havoc CRPS can wreak on a body and a spirit, having dealt with its ravages himself as the result of a motorcycle accident many years ago.

While the CRPS community at large marks his passing, we at RSDSA are especially saddened. He served on our Board in several different capacities throughout his long tenure here.

He always provided ad hoc legal advice, and in recent years, functioned as the Board Secretary. Instead of a more traditional and formal roll call, Steve often began meetings with greeting each member by name as he recorded the Minutes.

More importantly, however, we could always rely on Steve to transform our divergent opinions into one cogent voice which enabled us to advocate successfully for our members. His was the voice of reason and the heart of understanding to which we all aspired. He will be sorely missed.

Learn more about Steven via the Wilde Funeral Home website.

In a recent article by PEOPLE Magazine, Maya Kowalski shares how her CRPS diagnosis was classified as Munchausen syndrome by proxy, a mental disorder in which a caretaker of a child either makes up fake symptoms or causes real symptoms to make it appear that the child is injured or ill.

RSDSA is working with other rare disease organizations to quantify how many rare disease families have been accused of medical child abuse.

If you want to share your story, please email us at [email protected].