We are rare, we are many, we are strong, we are proud!
The purpose of Rare Disease Day® is to harness the creative energy of the millions of people around the world with rare diseases — and the millions who care about them — to raise awareness and generate action.
Join the IPPF and get in on the action. Rare Disease Day is the biggest day of the year for rare diseases like pemphigus and pemphigoid. Get involved and make an impact!
As the IPPF community looks to the future, we need to be stronger and louder to make a difference in the lives of people with rare diseases and their families demanding equity and inclusion. Please consider participating in one or more of the activities below and help us continue to spread aware for rare diseases like pemphigus and pemphigoid.
The National Institutes of Health (NIH) is holding a virtual conference with talented panelists and fascinating topics.
The National Organization for Rare Disorders (NORD), with it’s zebra-themed mantra “show your stripes,” will host various virtual activities.
NORD’s European sister organization, EURORDIS, is urging people to “share your colors” in a global campaign aimed at highlighting the more than 300 million rare disease sufferers in the world.
The U.S. Food and Drug Administration (FDA), the agency responsible for reviewing and approving treatments in the U.S., is hosting a virtual public meeting on March 4 in which “various stakeholders will share their perspectives on and experiences in rare disease product development.” Six panel discussions and other activities will be held.
Rare Disease Day at IndoUSrare on Monday, 28 February, 2022 from 7:30 pm to 9:30 pm IST, 9 am to 11 am ET invites you to join them for this year’s #rddindousrare, on the theme Celebrating 30 Years of Rare Disease Treatment, in honor of the millions of rare disease patients around the world, 95% who are still without any approved therapy for their condition. Featuring a stellar set of talks and panel discussion, the event commemorates the immense progress the rare disease community has achieved since the first commercial therapy for a rare disease came out in 1992. Register at: https://www.indousrare.org/event/rare-disease-day-at-indousrare-2022/
GlobalSkin is excited to take part in Rare Disease Day 2022 on February 28, by highlighting and raising awareness for rare dermatological diseases. They have created resources to assist with Members’ social media presence on Rare Disease Day. Visit their website to download materials!
Rare Disease Week on Capitol Hill (2/22- 3/2)
Rare Disease Week on Capitol Hill, organized by the the EveryLife Foundation for Rare Diseases brings together advocates to promote education about federal legislative issues that affect people with rare diseases and their caregivers, as well as public strategies to advocate for further change.
An easy way to spread disease awareness and make an impact is through sharing messages on social media! Raise awareness for the IPPF community and rare diseases by liking and sharing IPPF social media posts or creating your own.
The 2021 RareVoice Awards were held on December 15, 2021 by the EveryLife Foundation for Rare Diseases. The evening celebrated rare disease advocates who make their voices heard year-round to advance policies that benefit the rare disease community.
We’re so proud of Marc Yale for his nomination in the Federal Advocacy Patient/Organization category, which honors advocates or organizations that have worked to create and pass federal legislation. And we’re thrilled to share that Hannah Yale won the 2021 RareVoice award for State Advocacy by a Teenager, which honors teens who have advocated for state or federal legislation. Congratulations, Hannah!
Hannah has been an advocate for the EveryLife Foundation and the IPPF since 2017. She has attended Rare Disease Legislative Advocate’s (RDLA) Rare Disease Week on Capitol Hill annually since 2017, and she is also a member of the Young Adult Representatives of RDLA. In 2020, Hannah served on the Funding Committee for Living in the Light’s “I Stay Home for Rare” Emergency COVID-19 Relief Fund.
Hannah is living with Ehlers-Danlos Syndrome, although she began her rare disease advocacy to support her father, Marc, and her mother (who also has a rare disorder). Hannah is currently a student at St. Mary’s College of Maryland, where she is majoring in public policy and minoring in English and philosophy.
For more information about the event and to view the full list of award recipients, visit here.
About the EveryLife Foundation for Rare Diseases
The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.
We’re excited to share that in November of 2021, the IPPF’s Marc Yale was named as President of the Board of Directors of GlobalSkin. Marc is the current IPPF Research and Advocacy Coordinator, and he was the IPPF Executive Director from 2016-2020.
Marc Yale was diagnosed in 2007 with cicatricial pemphigoid. Like others with a rare disease, he experienced delays in diagnosis and difficulty finding a knowledgeable physician.
Eventually, Marc lost the vision in his left eye. This inspired him to help others with the disease. In 2008, he joined the International Pemphigus & Pemphigoid Foundation (IPPF) as a peer health coach.
Since then, Marc has worked with people to improve their quality of life and encourages them to become self-advocates. In 2009, he helped develop the Pemphigus and Pemphigoid Comprehensive Disease Profile giving experts insight into the patient perspective. In 2016, he was asked by the IPPF leadership to become the executive director of the foundation.
Marc currently resides in Ventura, California with his wife Beth and daughter Hannah.
About GlobalSkin
The International Alliance of Dermatology Patient Organizations (IADPO)—also known as GlobalSkin—is a unique global alliance serving patient organizations to improve the lives of dermatology patients worldwide. Based in Canada, the nonprofit is focused on three pillars: research, advocacy, and support.
GlobalSkin envisions a world in which people living with dermatological diseases and skin traumas can easily access the care and treatment they need, when they need it, and can live without stigmatization, persecution or economic disadvantages due to their conditions.
GlobalSkin is working with 185 patient association members—located in 62 countries representing more than 50 disease areas—to improve the lives of those affected by dermatological conditions throughout the world by:
initiating dialogue and advocating for access to new and existing treatments, and dermatological care to improve patients’ quality of life;
raising the awareness of the incidence of, and the challenges for, people living with serious dermatological diseases to create better understanding; and
supporting members and not-for-profit dermatology patient organizations through education, global campaigning, sharing of best-practices and beneficial networking opportunities to strengthen support for patients and build a strong, inclusive movement;
building special focus communities; and
conducting patient-initiated research.
GlobalSkin appeals as one voice to the World Health Organization and other key influencers to recognize the debilitating nature of dermatological disease so that more resources for research and treatment options are made readily available to those afflicted and in need of help.
Published April 19, 2021 On April 8, 2021, Marc Yale, IPPF Advocacy & Research Coordinator, issued a statement during the Medicaid and CHIP Payment and Access Commission (MACPAC) public meeting. MACPAC is a non-partisan legislative branch agency that provides policy and data analysis and makes recommendations to Congress, the Secretary of the U.S. Department of Health and Human Services, and the states on a wide array of issues affecting Medicaid and the State Children’s Health Insurance Program (CHIP). Thank you to Haystack Project for working with the IPPF on the implications for our community and helping with our remarks. We asked the commission to reconsider their recommendations to increase rebates for manufacturers who use the FDA Accelerated Approval Pathway. MACPAC’s recommendations have the unintended consequence of disproportionately harming ultra-rare patients like us.
The following is the prepared statement Marc Yale presented at the MACPAC public meeting.
Public Statement
My name is Marc Yale. I’m the Advocacy & Research Coordinator for the International Pemphigus & Pemphigoid Foundation. I also serve on the Board of Directors for Haystack Project.
Through my own personal experience as a rare disease patient living with pemphigus, as well as my involvement in advocacy for pemphigus patients and others living with extremely rare conditions through Haystack Project, I can tell you that accessing treatment can be an immense undertaking. Pemphigus and pemphigoid have been treated “off-label” with a variety of different therapies that have varying degrees of success in helping control our disease, and payers can make getting access to off-label treatments extremely challenging. We are now one of the handful of ultra-rare conditions with an FDA approved treatment- Rituxan, which is now considered a first-line therapy for pemphigus vulgaris.
There is no question that manufacturer incentives – like FDA Priority Review, Breakthrough Therapy Designation, and Orphan Drug Designation were vital to getting this therapy as quickly as possible to people with pemphigus who struggle every day to control their symptoms.
We were fortunate that Rituxan has been on the market since its approval in 1997 and that, due to the nature of our condition, clinical trials could be completed without surrogate endpoints.
The proposal to create an additional rebate for accelerated approval, though, could have been a significant deterrent to developing a new product in treating pemphigus.
The proposal also raises questions that do not have good answers from a policy perspective.
For example, if Rituxan’s pemphigus indication were achieved through accelerated approval, would an additional rebate apply?
And would it only apply to the indication?
It would seem that the proposal would decrease research rather than encourage post-market studies, especially when medically accepted off-label uses would be covered without the additional rebate.
Placing a disincentive on a pathway that is designed to encourage early access to promising treatments could have the greatest impact on ultra-rare disease patients. And your proposal today is basically a vote against investment in patients like us.
Published April 13, 2021 On February 8, 2021, the US Food and Drug Administration (FDA) held a Listening Session with patients representing the International Pemphigus & Pemphigoid Foundation (IPPF). Patient Listening Sessions are intended to be an opportunity for the FDA’s medical product centers to engage with patients and their advocates. The IPPF session was patient-led, meaning that the IPPF requested and received the permission to share its members’ perspectives with the FDA.
Listening session objective
The objective for this listening session was to have a dialogue with the FDA to share the emotional experience of the patient journey as well as the burden these diseases have on all aspects of a person’s life. This includes the time it takes to get a diagnosis, the burdens of treatment options, and the undertreated areas of the diseases that affect the physical, emotional/psychological, and financial health of five pemphigus and pemphigoid patients.
Summary of topics discussed
Pemphigus and pemphigoid are rare, ultra-orphan, autoimmune, blistering diseases that result in potentially life-threatening destruction of the skin and mucosa. The patient’s immune system makes antibodies that attack healthy cells in the skin or mucous membranes. As a result, skin cells separate from each other, fluid collects between skin layers, and blisters form. These blisters may cover a large area of skin. This results in fragile, extremely tender lesions that do not go away without proper treatment. It takes the average pemphigus or pemphigoid patient five healthcare providers and ten months to obtain a correct diagnosis. Currently, no cure exists for pemphigus or pemphigoid, only treatments and remission.
According to recent literature in the British Journal of Dermatology, pemphigus is rarer than pemphigoid. The approximate incidence of pemphigus is .58 – .80:100,000 people, and the approximate incidence of pemphigoid is 7.3 – 7.93:100,000 people.
These diseases are known to affect people across gender, racial, and cultural lines. However, there are certain groups of people who have a higher incidence of the diseases, such as Eastern Europeans of Jewish descent and people of Mediterranean, Northern India, and Persian descent.
The FDA Listening Session included further discussion on:
Diagnostic delays
Treatment options
Medical burdens
Investigational research
Mental and social burdens
Read the entire summary of the FDA Listening Session on Pemphigus and Pemphigoid by downloading the PDF.
The IPPF has published a white paper focusing on the urgent needs of pemphigus patients.
March 16, 2021 The IPPF is excited to announce the publication of a white paper, “Elevating Awareness of the Pressing Needs of the Pemphigus Community.” This paper is in response to the results of a Patient Forum held as part of our Virtual Patient Education Conference last October. The Patient Forum was sponsored by Principia Biopharma, a Sanofi Company, and brought together pemphigus patients, caregivers, patient influencers, healthcare professionals, thought leaders, and advocates to discuss the most important disease-related topics affecting the pemphigus community. The white paper discusses the consensus reached by the speakers and panelists related to the most urgent needs of pemphigus patients. It also includes decisive calls to action.
During the Patient Forum, panelists discussed their experiences with delayed diagnoses and the physical, mental, and financial impact of pemphigus. The consequences of a delayed diagnosis to a patient’s health and well-being can be significant, therefore the speed and accuracy of diagnosis must be improved. Speakers discussed the high dosage use of corticosteroids and limited treatment options for pemphigus, which makes it clear that new treatment options are needed. In response to the need for new treatments, scientists in academia and the biopharmaceutical industry are currently working to refine and expand treatment options that are more tolerable and targeted.
Based on the urgent needs discussed during the Patient Forum, the panelists and speakers agreed on the following calls to action:
Elevate awareness of pemphigus and the patient journey and perspective among key audiences, including clinicians, thought leaders, government agencies, and lawmakers, among others.
Increase awareness of ongoing clinical trials of promising investigational candidates to improve participation in trials, with the goal of developing more effective and well tolerated treatments for pemphigus.
We are rare, we are many, we are strong, we are proud!
The purpose of Rare Disease Day® is to harness the creative energy of the millions of people around the world with rare diseases — and the millions who care about them — to raise awareness and generate action.
Join the IPPF and get in on the action. Rare Disease Day is the biggest day of the year for rare diseases like pemphigus and pemphigoid. Get involved and make an impact by attending the following webinars/virtual meetings, interacting on social media, and taking action!
National Advocacy Events:
Monday, 3/110:30am- 5:30pm (EST) Virtual Rare Disease Day at the National Institutes of Health NIH
The purpose of this meeting is to highlight strategies to support rare disease product development. Patients, patient advocates, researchers, and medical product developers may benefit from attending this public meeting on rare disease product development. Register on the FDA website.
State Advocacy Events:
Many states are hosting Rare Disease Day Events in February and early March. Visit the National Organization for Rare Disorders Event Page to register.
(Arizona, Arkansas, California, Colorado, Connecticut, Delaware, Florida, Georgia, Illinois, Indiana, Kansas, Louisiana, Maine, Massachusetts, Minnesota, Michigan, Nevada, New Hampshire, New Jersey, New York, North Carolina, Ohio, Oklahoma, Pennsylvania, South Carolina, Tennessee, Texas, Utah, Virginia, West Virginia)
Rare Across America (2/22- 3/5)
The IPPF is excited to participate in Rare Across America virtually this year. Rare advocates have already signed up and will be sharing their stories to make an impact on federal policy. Rare Disease Legislative Advocates (RDLA) organizes meetings for rare disease advocates with their Members of Congress and/or the Member’s staff. Meetings will take place virtually on March 3rd and 4th. The RDLA team also helps to prepare advocates for their meetings, provides legislative resource materials, and hosts pre-meeting training webinars.
Social Media:
An easy way to spread disease awareness and make an impact is through sharing messages on social media! Raise awareness for the IPPF community and rare diseases by liking and sharing IPPF social media posts or creating your own.
Monday, 2/22- Friday 2/26: Selfie Week – Post a picture on social media, tag the IPPF (@healourskin), and use these hashtags: #RareAcrossAmerica2021, #EveryVoiceMatters, #healourskin, #RareDiseaseDay2021, #ShowYourStripes
Friday, 2/26: Share Your Rare Story – Take a video and share it on social media. Don’t forget to tag the IPPF (@healourskin) and use these hashtags: #healourskin #RareAcrossAmerica2021, #EveryVoiceMatters, #RareDiseaseDay, #ShowYourStripes
Check out the National Organization for Rare Disorders (NORD) social media toolkit to find helpful tips, sample social media posts to share, graphic design templates, hashtags to use, and profiles to tag.
Take Action
The RISE Act
Take action by urging Congress to secure our nation’s strategically vital science and technology ecosystem: pass and sign into law the RISE Act, and designate at least $25 billion in supplemental funding – including at least $10 billion for the NIH – for research recovery.
Representatives Diana DeGette (D-CO) and Upton were joined by 75 of their colleagues in introducing the Research Investment to Spark the Economy (RISE) Act. Senators Edward Markey (D-MA) and Thom Tillis (R-NC), joined by Senators Gary Peters (D-MI) and Susan Collins (R-ME), introduced a companion bill in the Senate. Read Research America’s statement detailing the crucial importance of the RISE Act .
A congressional caucus is a group of members of the United States Congress that meets to pursue common legislative objectives. Formally, caucuses are formed as congressional member organizations (CMOs) through the United States House of Representatives and governed under the rules of that chamber. There are hundreds of Caucuses. The most common caucuses consist of members united as an interest group. A Caucus can hold briefings to raise awareness on an issue. However, briefings are not actionable, ie: no bills can be introduced or voted on. A Caucus may join Members together in a voting block to support or oppose legislation, however most interest group caucuses are used to gain media attention and raise public awareness. Congressional Caucuses must be re-filed in the House at the start of each new Congress. The filing papers must be submitted by the majority party.
Together, we can drive favorable policies by reaching out to legislators and decision-makers to inform them of our public policy concerns, bring attention to the disease, and inform the public about pemphigus and pemphigoid.
The HEART Act
The IPPF needs your help to get a vital piece of legislation passed! We work collectively with other rare and ultra-rare groups to increase our voice and make a difference. Sharing our patients’ experiences with the Haystack Project has resulted in an especially important bill being re-introduced this year with the new Congress. This bill will have a significant and lasting impact for our community.
The HEART Act, H.R. 1184, contains tangible and practical solutions for involving patients and rare disease experts in the FDA review process to better inform the review of drugs for safety and efficacy.
The HEART Act contains 5 provisions critical to rare patients:
1. Advisory Committees – Require a rare/ultra-rare expert in the science of small population studies at Advisory Committee meetings when the application under review is for a low prevalence condition.
2. Review Division Transparency – Require annual report to Congress that sets out, by division, how many rare applications were reviewed, Agency actions, and the prevalence #s for that rare condition (this could be pulled from sponsor submission on orphan designation request.)
3. Review Division Support – Require review divisions to consistently include Rare Disease Program staff as an integral part of review team when reviewing a first drug/biologic or a first disease modifying agent for a particular indication associated with an orphan condition with very low prevalence (not as a volunteer, advisor, or “guest” that can be removed if their participation is unwelcome). This same rare disease program staff support should be extended to support review division decisions beyond just approval to REMS, post market commitments, etc.
4. REMS – For any very low prevalence orphan applications, require FDA to consult with patients/patient organizations in devising or reviewing any Risk Evaluation and Mitigation Strategy (REMS) elements that require patient action/participation.
5. European System – Require a Government Accountability Office (GAO) study of how the European system reviews ultra-rare applications and its applicability in the US — Specifically, how the EU allows submission of updated data during the review, including from open label extension studies for patients who remain/continue on drug or cross-over from a control arm after clinical trial data has been gathered and submitted.
Ask your Congressional Representatives and Senators to support this bill easily and quickly:
Welcome to our “Inspiring Hope” series. For the next several weeks, we’ll share patient stories from around the world that show what it’s like to live—and thrive—with pemphigus and pemphigoid. Our third story comes from Hannah Yale in California.
I was four years old when my dad got sick the first time. For six months he was undiagnosed, and during that time he went blind in his left eye. When he was finally diagnosed with mucous membrane pemphigoid (MMP), my dad got in touch with the IPPF. The IPPF helped him get in touch with other patients who were experiencing, or had already experienced, diagnosis and treatment. The Foundation also gave him a list of doctors who knew how to treat pemphigus and pemphigoid (P/P) to ensure that he received the care he needed. I went to the hospital with my dad every day for his rituximab and IVIG treatments.
In 2008, the IPPF held its annual Patient Education Conference in Dallas, Texas. I went with my mom and dad to meet other patients and families who had been affected by P/P. At the conference, we found a community. My entire family has been involved with the IPPF in some form ever since.
After my dad connected with other pemphigus and pemphigoid patients, he began sharing his own experiences with diagnosis, treatment, and dealing with the effects of his disease. When the IPPF created the Peer Health Coach program in 2018, my dad became one of the first coaches. Just as I was entering high school in 2016, he became the executive director. Around the same time, he had a relapse of his MMP, but was able to recover quickly with rituximab treatment.
Empowerment Through Community
I have often felt powerless when my dad has been sick. Three years ago though, I learned that I am not powerless to help him. In 2017, my dad took me to Rare Disease Week on Capitol Hill, an event organized by Rare Disease Legislative Advocates. We attended with a group of patients, caregivers, and doctors representing the IPPF, and our goal was to speak to members of Congress about legislation that would benefit people with all types of rare diseases. I was able to meet so many wonderful people who shared my feelings and experiences. Going to Rare Disease Week made me realize that even as a teenager, I could make a positive impact.
I now know that I can help people just like my dad always has, and that I am passionate about nonprofit work. I am proud to be able to represent the IPPF through my advocacy for accessible care and treatments. I know that my relationship with the IPPF will continue to grow as I do. The IPPF’s generous staff, compassionate volunteers, and inspiring programs have supported me throughout my life, and they have helped shape who I am today.
Be a Healing Hero
Healing Heroes fund the future of the IPPF community by making sustaining, monthly gifts to support our mission of improving the quality of life for all those affected by pemphigus and pemphigoid.
By becoming a Healing Hero, you provide for the greater good of our community by sharing our vision:
NO DISEASE IS TOO RARE FOR A CURE!
Whether you’re a patient, caretaker, family, friend, medical professional, or rare disease advocate, your monthly gift allows us to not only sustain current programs, but also expand our key areas of operation: patient support, education, awareness, research, and advocacy.
Hannah is a 17-year-old California resident and a human rights activist. She is a member of the Young Adult Representatives of RDLA and has attended Rare Disease Week on Capitol Hill every year since 2017. Hannah is a first-year student at St. Mary’s College of Maryland, double-majoring in Public Policy Studies and English.
You may be aware of the IPPF’s advocacy efforts. Now, we need you to advocate, too. Your story is important and shows that rare disease not only affects Americans, but Americans in your district. Your story proves to congressional members that their decisions have a great impact on human life and well-being. The IPPF encourages you to participate in bringing awareness of issues and legislation that affect the rare disease community to your state and federal representatives.
This may seem like a daunting process, but the IPPF is here to help. We have tools to support you as you prepare to speak with legislative members. One of the most important things to remember is that legislators are people, too. Look for common ground—they are parents, brothers, sisters, and friends. Statistically, they almost certainly know someone with a rare or autoimmune disease.
The following suggestions can help you to successfully become an advocate:
Contact the IPPF at advocacy@pemphigus.org to express interest in advocating at a local and/or national level. We can explain issues and legislation that the IPPF currently supports.
Identify your federal lawmakers by using the online tools at senate.gov (US Senate) and www.house.gov (US House of Representatives).
Monitor the congressional calendar. District work periods are the best time for lawmakers to meet with local constituents. Take advantage of the August recess. This is a busy time when many lawmakers hope to meet with their constituents to learn about issues affecting them and what they can do to help.
Schedule an appointment by sending a formal invitation to the lawmaker’s scheduler at least three to four weeks in advance of the proposed meeting date. Check your lawmaker’s website, as they may have a formal process for submitting meeting requests.
SAMPLE EMAIL TEMPLATE
Dear [Name],
I am writing to request a district meeting with (insert elected official here). As legislators increasingly play a role in shaping health care policy that impacts my access to quality and affordable care, I welcome the opportunity to discuss with you some of my challenges and opportunities patients face when caring for themselves.
I am available to meet with you on (suggest a few dates that work for you). If those dates do not work for you, I am happy to discuss other options with your staff. I can be reached at (insert phone number) or at (insert email).
Sincerely,
[Your Name]
[Your address]
About a week later follow up with a phone call.
PHONE CALL TALKING POINTS TEMPLATE
Hello, my name is [Your Name].
I’m a patient with pemphigus/pemphigoid in [Your City, State].
I’m following up on a written invitation I sent to [Name of Elected Official] wishing to speak with him/her to discuss issues facing patients like me when trying to obtain quality and affordable healthcare and prescriptions.
I would like to schedule an opportunity for [Name of Elected Official] to visit their office in the next three to four weeks. Do you have any availability?
(If they need time to check on the schedule, give them your name, email, and phone number. Be flexible. If they are unable to meet on the date(s) you suggested, discuss other options with them.)
Ask them if there is any required paperwork you need to submit prior to the meeting.
Thank you for your time, and I look forward to meeting [Name of Elected Official] at their district office.
THE VISIT:
Understand the issues you are about to discuss. This is your story and it is important to use your experiences as compelling evidence of the issues at hand.
Share your concerns with your elected official. If available, use the handouts that the IPPF provides on the policy or regulatory issue.
Get to the point. Don’t take too long to make your pitch. Tell them who you are, the community you represent, the number of patients with our disease, what your primary concerns are, and how and why these concerns are related to a certain piece of legislation or regulatory requirement.
This should be conveyed in about 10 minutes.
If they ask a question and you are unsure of the answer, please say so and let them know you will find out and get back to them. The IPPF is happy to help you find the information.
Double-check your facts and figures. Make sure that you are presenting accurate information.
Thank your elected official for specific votes and efforts where they have supported legislation that helps our community.
Take a photo with the elected official and their staff. Please share it with the IPPF. We love to post photos of our community participating in advocacy efforts.
Follow up by thanking your elected official for the visit.
THANK YOU NOTE TEMPLATE
Send by email or regular mail.
Dear [Name],
Thank you for taking the time to meet with me on [insert date].
As a constituent, I appreciate the opportunity to tell you about my disease and to share my story with you to see the impact healthcare policy has on patient access to care. It was an honor to meet you.
[Insert main points from the meeting as a way to reiterate your position.]
Please do not hesitate to contact me at [your phone or email address] if you have any further questions regarding [insert legislation or regulation name]. I welcome the opportunity to serve as a resource for you on these important issues.
Sincerely,
[Your Name]
Follow up with IPPF staff. Share any action items resulting from the meeting with the IPPF advocacy team by phone or email.
We hope this helpful guide encourages you to get you involved in advocating for our community and for those who cannot advocate for themselves. We are always looking for new ways to advocate for the IPPF community and would appreciate any feedback.
In February 2017, the IPPF joined the Friends of the National Institute of Dental and Craniofacial Research (FNIDCR) Patient Advocacy Council (PAC). This group is made up of patient advocacy organizations whose patient members’ conditions and diseases include an oral component and have a stake in research supported by the NIDCR. The group is supported by the American Association for Dental Research (AADR).
The AADR and FNIDCR will hold an Advocacy Day on Capitol Hill on Tuesday, February 27. Members and patient advocates will meet with members of Congress and Hill staff to advocate for dental, oral, and craniofacial research. They will emphasize how important investments in biomedical research and oral health programs are in impacting the people in their home states and beyond.
The IPPF had the pleasure of interviewing Lindsey Horan, AADR’s Assistant Director of Government Affairs about their 2018 Advocacy Day and important legislative issues.
IPPF: What is your goal for your advocacy day? What do you hope to achieve? Lindsey Horan (LH): Our overarching goal for Advocacy Day is to educate on and raise awareness for dental, oral, and craniofacial research with members of Congress and congressional staff.
As oral research advocates and stakeholders, we know that oral health is integral to overall health, but it’s critical to stress that to the policymakers who are weighing competing priorities and are responsible for divvying up federal funds across government agencies and programs. The Hill visits our members conduct on Advocacy Day have the opportunity to demonstrate the far-reaching nature of oral health research and, most importantly, to share their personal stories—whether it is the story of a patient whose life has been impacted by an oral disease or condition, or a researcher whose work is positively shaping the trajectory of dental and oral care we provide in this country.
IPPF: Which legislative issues are of priority for the AADR/FNIDCR this year? Why are they important? LH: Our legislative priorities for AADR and the Friends of NIDCR will be consistent with the priorities from 2017, and they largely relate to securing the highest possible federal funding for oral research and oral health programs. While this certainly includes the National Institutes of Health and the National Institute of Dental and Craniofacial Research (NIDCR), we also champion agencies whose work touches oral research in some capacity, such as the Centers for Disease Control and Prevention and its National Center for Health Statistics and the Agency for Healthcare Research and Quality.
Heavily focusing our legislative portfolio on appropriations is reflective of the fiscal and political environment in which we’re operating. Congress has to make difficult decisions about how to tackle a mounting federal debt and deficit, and we want to ensure that shortsighted cuts aren’t made in the name of savings. In the absence of our community speaking out—loudly—about these federal agencies and programs, lawmakers will see a win-win scenario: being able to cut funding with little to no pushback.
IPPF: What can those who are unable to attend Advocacy Day in DC do to advocate locally? LH: There is so much that can be done locally—even from home—to champion oral research throughout the year.
First, it’s important to remember that members of Congress are not in Washington, DC, year round. They regularly return home to meet with their constituents, and these visits are great opportunities for people to voice their priorities or concerns. Sign up for your elected officials’ email listservs to learn about upcoming town halls or other events where you might have an opportunity to speak with them. At the end of the day, constituents are the people members of Congress want to hear from most!
Additionally, don’t underestimate the power of social media. Virtually all Senators and Representatives are active on Twitter, Facebook, and other social media platforms—and they pay attention to them. While seemingly inconsequential, research has shown that it doesn’t take many Tweets on a given topic for staff to pay attention, especially if the Tweets come from constituents (and constituents should identify themselves as such in their Tweets).
IPPF: Do you have advocacy alerts or ways to stay updated throughout the year on important legislative issues? LH: Absolutely. We want to make sure our community knows how developments at the federal level may impact our field and the research enterprise more broadly.
The first resource I would recommend is our Government Affairs & Science Policy Blog (http://ga.dentalresearchblog.org), which is regularly updated with advocacy and policy news. And to make it easy for readers, there is an option to subscribe to the blog, so new posts will come directly to your email. We also post information to our Twitter account (@DentalResearch). These are great places to learn about any new action alerts or opportunities for engagement.
There are also a number of opportunities available through AADR membership (http://www.iadr.org/AADR/Join-Renew/Join-Us) for those who want to further engage, such as the potential opportunity to serve on committees like our Government Affairs Committee and joining us on Capitol Hill for Advocacy Day.
IPPF: Is there anything else you’d like to share? LH: I know people often shy away from advocacy for a variety of reasons—they worry about bringing politics into the workplace, they are put off by the term, or they don’t see the point. To this I would say:
As an American citizen, you have a right to petition your government as outlined in the Constitution. It’s correct that many employers do have rules related to advocacy, but they do not prohibit you as an individual citizen from being able to advocate. To clarify what is and is not allowed, talk to the government or public affairs staff at your organization or institution.
For those who don’t quite understand or are put off by the term “advocacy,” think of it as education. When you reach out to members of Congress, you are sharing your story, explaining your work, or demonstrating how a program is making a difference in your community. Members of Congress and congressional staff are grappling with information overload. Meeting with them provides an opportunity to share what you know so that they might better understand the issue and how it fits into their legislative priorities.
Finally, advocacy does make a difference. While a phone call, a Tweet, or an email seem too small to be significant, they add up—and we have seen this demonstrated time and again. Just recently, a provision in the House’s first version of the tax bill calling to tax graduate students’ tuition waivers as income received so much pushback from the community that it was removed in the final legislation. Speaking up and speaking out matter!
A huge thank you to Lindsey Horan for taking the time to answer our questions!
IPPF Awareness Ambassador Coordinator, Bryon Scott, will attend Advocacy Day on Capitol Hill this year. We look forward to updating you on his experience and the outcome of his advocacy.
The first Rare Disease Day was first launched in Europe by EURORDIS, The Voice of Rare Disease Patients in Europe, and its Council of National Alliances in 2008. The USA joined the cause in 2009. By 2016, Rare Disease Day has become a worldwide event with over 80 countries participating.
“Rare diseases are not so rare: there are 7,000 rare diseases & disorders that combined affect 30 million Americans–1 in 10 of us–and more than half are children.
People with rare diseases have tremendous unmet needs, including misdiagnosis, a long time to finally receive a correct diagnosis, and when they do, 95% have no treatment with ZERO CURES.
Rare Disease Day takes place on the last day of February each year. The main objective is to raise awareness with the general public and decision-makers about rare diseases and their impact on patients’ lives.”
This year’s global theme is research. It is our chance to give lawmakers, industry leaders, researchers, and healthcare professionals a glimpse of the impact a rare disease and its treatments may have. It is a local, national, and international awareness event.
Rare Disease Day is a day of unity for us patients. I can’t speak for you, but I felt very alone and isolated when I found out I had pemphigus vulgaris. Then I found community in the IPPF. It was very comforting to know there were others out there who had gone through what I was living through. Now imagine being with hundreds of others who have a variety of rare diseases. Like us, they are patients looking not only for effective treatments, but also cures. On Rare Disease Day, we are a united front advocating for increased funding for research. It’s a time of inclusion. A time for uniting as one. A time to make a difference.
It’s also a time to network with other patients and learn about their diseases, how those diseases affect them, and what their disease organizations do to support their needs. It’s a way to find out what we – as members of the IPPF – can do better to help and support all who are affected by rare diseases. It is a time to find community and strength in knowing that we are not alone in a search for better treatments and cures.
Rare Disease Day is a day to find strength and power in your weakness, your disease. You have the power to share your story. Your story has the power to impact the leaders of your city, state, and country. Your story can evoke changes in law, government, healthcare, and medical education.
I encourage you to look for opportunities where you can get involved. If no opportunity exists near you, you can create your own. You could share your story with co-workers or local government officials, organize a bake-sale and donate the proceeds to the IPPF, or organize a run/walk to raise awareness. The list of possibilities is endless. Just be sure to explain what pemphigus and pemphigoid are and refer people to the IPPF if they have questions.
Although there has been a huge public outcry over the cost of prescription drugs, there has been little public discussion about the need for innovation for patients who don’t yet have an effective therapy. It is estimated that 1 in 10 Americans suffer from a rare disease, and only 5% of rare diseases have a treatment approved by the Food and Drug Administration (FDA).
However, the toll of rare diseases goes beyond patients, with potentially devastating impacts on families and other loved ones. The average pemphigus and pemphigoid patient sees 5 doctors over 10 months in search of a diagnosis, often delaying the start of treatment. Many rare disease patients wait an average of seven years before getting an accurate diagnosis. Even if a patient obtains an accurate diagnosis, chances are there are no FDA-approved treatment options. The small size of patient populations makes the massive investment needed for lengthy research and development needed to create rare disease therapies unattractive to most pharmaceutical companies.
I was diagnosed in 2007 with cicatricial pemphigoid, a rare autoimmune blistering skin disease. Like others with a rare disease, I experienced delays in diagnosis and difficulty finding knowledgeable physicians. Eventually, I lost vision in one eye from the disease. The pain associated with my disease was severe, and the list of complications extensive. Although my disease can be treated, there are still no FDA approved treatments and no cure. No disease should be too rare for a cure.
The good news is that Congress can do something to help. The 21st Century Cures Act, which passed the House of Representatives with broad bipartisan support (a rarity in the current political environment), could provide billions in new funding for research at the National Institutes of Health, the nation’s premier biomedical research institution, along with needed funding to enhance the review of new medicines by FDA. What’s more, the legislation has crucial incentives, such as the Priority Review Voucher program to encourage the private sector to invest in developing new, life-saving therapies for pediatric patients, and the OPEN ACT, which would encourage companies to repurpose existing medicines for rare diseases like ours. This could potentially bring hundreds of new treatments to patients more quickly than traditional drug development. In short, this bill could be a game-changer for patients with both rare and common diseases.
But Congress has been debating this legislation for nearly two years and has yet to send it to the President’s desk for signature. If Congress fails to reach an agreement by the end of the year, all of the work on this legislation and the hope that it represents to patients will be lost. Every day, patients are losing ground to diseases and continue to go undiagnosed or untreated.
The IPPF urges you to contact your representatives and senators today to prioritize the 21st Century Cures Act on behalf of pemphigus and pemphigoid patients and their families across the country. Time is running out. and we cannot afford to wait any longer.
“I truly felt that our representatives were listening closely when I told them my story of being diagnosed with and treated for pemphigus vulgaris.” On Thursday, September 22, I joined 350 advocates on Capitol Hill for the Rally for Medical Research. Our main goal was to encourage members of Congress to continue robust, sustained, and predictable funding for the National Institutes of Health (NIH). Because I live in Washington, DC, a city without a vote in Congress, I was assigned to the group from Montana.
Along with a University of Montana biologist and a cancer research advocate, I met with Sens. Jon Tester and Steve Daines, and Rep. Ryan Zinke. All three members were supportive of our efforts to maintain NIH funding, especially Sen. Tester, who seemed to have a vested interest in medical research. In addition to our general request for NIH funding, I stressed the importance of cutting edge research on rare diseases and emphasized the significance of the Open Act, which incentivizes pharmaceutical companies to make treatments available off-label.
Of my three visits to Capitol Hill as a rare disease advocate, this is the first time I met with the members themselves rather than only their staff. It was a humbling experience but also a powerful one. I truly felt that our representatives were listening closely when I told them my story of being diagnosed with and treated for pemphigus vulgaris. Meeting our lawmakers in person and telling our stories makes all the difference!
On March 2nd, 2016, six of my fellow pemphigus and pemphigoid (P/P) advocates and I visited my Congressional members to ask them to co-sponsor legislation that would make it easier for Medicare patients to have IVIg infusions done at home. IVIg therapy is a treatment that many patients such as myself use in conjunction with immune suppressant medication to control our diseases.
Unfortunately, getting IVIg home infusions under Medicare can be complicated because there is a gap between Medicare Part B and Medicare Part D. Infusion therapy is fully covered by Medicare in hospitals, skilled nursing facilities, hospital outpatient departments, and physician offices, but not in a patient’s home. However, if a patient wants to get the infusion done at home, Medicare pays for the infusion drugs (under Medicare Part D), but not the medically necessary services, supplies, and equipment used in the provision of infusion therapy (under Part B). This makes access to these much needed services costly or impossible for many home infusion patients. As a result, most Medicare patients in need of infusion therapy often receive their treatments in health care facilities instead of in their homes, although home may be the setting that is the most desirable, safe, convenient, and by far the most cost effective.
Congress has been concerned with this issue for many years, but has yet to approve any legislation. In January, Congressman Eliot Engel, Representative for New York’s 16th congressional district, and Senator Johnny Isakson, Senior Senator from Georgia, introduced the Medicare Home Infusion Site of Care Act of 2015 (H.R. 605 and S. 275) that would remedy the problem. As we shared our stories on behalf of the entire P/P community, I could see that many of the legislators’ offices we visited began to understand the dilemma. However, it was clear this issue would take an effort by the entire P/P community for our voices to be heard.
I urge you to contact your congressional members and ask them to co-sponsor this crucial legislation. Reach out to the senators on the Senate Help Committee and the representatives on the House Committee on Energy and Commerce who control whether this legislation will move forward. Let’s start a conversation by using the hashtag #medicarehomeinfusionact on Facebook, Twitter, and Instagram. Together we can make a difference!
The purpose of advocating in Washington, DC is to spread awareness and lobby for favorable legislation that affects the entire IPPF community. At Rare Disease Week on Capitol Hill 2016, we had 7 members of the IPPF community advocating for:
HR 971/S1421 (OPENACT): legislation that will repurpose “off label” drugs for rare disease indications that are not currently covered by the FDA
HR 605/S275 (Medicare Home Infusion Act): legislation that will make it easier for Medicare patients to get infusions done in their homes
HR 1600 (Patients Access to Treatment Act): legislation that would cap the amount that insurance carriers can charge patients for more expensive medications
S 2030 (Advancing Targeted Therapies Act): legislation that would allow information learned in clinical trials to be used in the development of new therapies
This was my second time advocating on behalf of the pemphigus and pemphigoid community, having visited Capitol Hill with Marc and Kate in the fall. I expected it to be quite similar, and in many respects it was. However, being part of a larger group of rare disease advocates participating in Rare Disease Week made a big difference. Hearing from two congressmen and the newly appointed head of the FDA at the lobby day breakfast emphasized the importance of bringing together advocates from across the rare disease spectrum – not only the power in numbers but also the variety of knowledge and experiences that we could bring to the table.
Mary Lee Jackson
When I received an email about going to Capitol Hill, I got excited and thought it will be easy. But I was wrong it was hard work. I hadn’t walked so much in all of my life. I felt good about what was done on Capitol Hill. It was worth all I went through to get the word out about rare diseases.
Debra Levinson
There’s plenty we can do this election year: Consider writing letters and making phone calls to bolster these pieces of legislation; email your member of Congress, which is made easy utilizing the automatic draft letters on rareadvocates.org; or take advantage of in-district lobby day opportunities to build relationships with your members of Congress. I am hopeful now with so much support we can help ourselves heal. We are the voices of the rare disease community with a common role.
Paula Halicki
Not only was I representing myself, the IPPF, and the pemphigus/pemphigoid community, but I was also representing a lot of other people with healthcare issues who could not represent themselves. And quite a few who never will. Plus I told my story, and a Congressman listened.
Doris Chenier
We may have a rare disease but no one will know about it unless we use our voice to speak to people that can make a change.
Angélica N. García Romero
I had great meetings with the staff of the representatives and felt very accomplished. But to top it all, when I was taking my flight back to Puerto Rico, my representative was on the plane too! When all the turbulence stopped, I went up to him, and had the meeting right there.
Monday, February 29, 2016, was “Leap Day,” the rarest day of the year. But for the rare disease community, it was much, much more: it was International Rare Disease Day.
The IPPF is proud to join with others in the community to raise awareness of rare diseases. Our board, staff, advisors, and volunteers are motivated every day by a passion to support awareness, advocacy and the development of treatments that can have a meaningful impact for P/P and all rare diseases.
Millions of people took this year’s motto, “The Patient Voice,” to heart and spoke out to the general public and elected officials in more than 80 countries. In the United States, all 50 states passed resolutions recognizing the day as Rare Disease Day. Here in California, Noelle Madsen and I were joined by dozens of rare diseases advocates at the CA State Capitol. Noelle and I were also guests on the State Assembly floor as California Senate Concurrent Resolution 108, Rare Disease Day was unanimously passed (and later we were in the gallery as the State Senate unanimously passed the CSR).
Meanwhile, in the Nation’s Capitol, Marc Yale and fellow P/P patients spoke to their elected officials on the importance of funding rare disease research and legislation. On Wednesday, March 2, our patients joined more than two hundred advocates educating aides, senators, and congress members on the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures & Treatments) that could double the number of treatments available to rare disease patients. The OPEN ACT has the potential to be a game changer for pemphigus and pemphigoid patients and the entire rare disease community.
That evening, NORD and the IPPF educated more than 15,000 people on rare diseases and P/P in 120 seconds. Just before tip-off at the Sacramento Kings/Oklahoma City Thunder NBA game here in Sacramento, I was interviewed by Kings’ emcee Scott Freshour on Rare Disease Day and pemphigus and pemphigoid shown over the court’s jumbotron to the entire stadium. Immediately after that, this video created by our own Patrick Dunn was shown (and played again at halftime).
I encourage everyone to be a self-advocate for P/P and rare diseases in general. A little effort by each of us can have a tremendous impact on patient quality of life. For more information on advocating, contact Marc Yale at marc@pemphigus.org.
At the California State Capitol. (l-r) Kristen Angell, NORD; Noelle Madsen, IPPF Patient Services Manager; Greg Benton, The Myelin Project; Monique Rivera, IPPF Admin; Debbie Fuentes, IPPF Intern; Will Zrnchik IPPF Executive Director.
At the National Institutes of Health in Washington, D.C. Kate Frantz, IPPF Awareness Program Manager, displays the Awareness Campaign poster to raise awareness for pemphigus and pemphigoid.
At the Sacramento Kings vs Oklahoma City Thunder game in Sacramento, CA. Debbie and Niko, IPPF Student Interns from Cristo Rey High School, help raise awareness for P/P and other rare diseases.
Momentum for the Orphan Product Extensions Now, Accelerating Cures and Treatments Act (OPEN ACT) has stalled in the Senate.
In November 2014, I used the chemotherapy drug Rituxan off-label for my rare disease, Immune Thrombocytopenia (ITP). The decision was made after careful consideration of all the possible outcomes. I was desperate for relief since ITP causes internal bleeding that can be fatal. This thought stayed in the back of my mind as I watched my platelets drop and my bleeding episodes increase in severity.
Ultimately, I decided it was worth a shot to see if Rituxan could put my rare disease in remission. When it was successful, I was thrilled and shared my success with the rest of the ITP community. To my surprise, many ITP patients told me they did not have access to Rituxan because of the off-label status. When I first heard about the Orphan Product Extensions Now, Accelerating Cures and Treatments Act (OPEN ACT), I was thrilled! Finally, legislation was presented that would allow rare disease patients to have access to FDA approved drugs that are deemed safe and effective for other conditions.
During Rare Disease Week last year, I discussed my story with Congressman Gus Bilirakis of Florida, who introduced the OPEN ACT to the Energy and Commerce Committee. Congressman Bilirakis — a huge champion for patients — and his staff worked hard to have the OPEN ACT included in the 21st Century Cures Act. The bill passed the House in July 2015 by an overwhelming vote of 344-77. The momentum has stalled in the Senate though, so now patients must act. Recently, the Senate Health, Education, Labor and Pensions Committee (HELP), listed bills that were a priority for their version of 21st Century Cures, and for some reason omitted the OPEN ACT. I will be on the Hill for Rare Disease Week this year to push for the inclusion of the OPEN ACT on the HELP Committee’s priority agenda.
For the rare disease community and patients as a whole, the OPEN ACT will provide critically needed treatment options for underserved populations. It will help fill in the gaps between diseases with limited or no treatment options and new drugs under development. I encourage all patients, regardless of their illness to reach out to their Senators and express support for the bill. Repurposing FDA approved drugs will create new data that provides the foundation for further research. Ultimately, the OPEN ACT can unlock new treatments and potential cures for rare diseases. I am living proof that repurposing drugs can save lives. Looking back, the decision to use Rituxan paid off more than I could ever imagine.
I encourage everyone to join the conversation on social media using the hashtag #CuresNow on Twitter, Facebook and Instagram. Many of our representatives are participating with us. This is our time to show lawmakers that we may be rare when identified by specific disease or diagnosis, but together we are a movement!
February 29 is the rarest day on the calendar. This year, I invite you to do something meaningful to mark the day.
On the last day of February, millions of people from around the world will observe Rare Disease Day® by organizing events and activities that raise awareness for patients, families, and caregivers affected by rare diseases.
Because you, and we as your advocates, are directly connected to a rare disease, this is a perfect opportunity for us to raise awareness for pemphigus and pemphigoid which affect roughy 1 in 1,000,000 people. Pemphigus and pemphigoid are just two of the 7,000 known rare diseases that in total affect 30 million – or 1 in 10 – Americans, making them not so rare after all.
We know first-hand the difficulties families have getting access to life-saving, life-improving medical treatment or other services due to insurance obstacles or to a lack of knowledge about pemphigus and pemphigoid by the medical community. This challenge is common to all rare diseases.
What is Rare Disease Day®?
Rare Disease Day® began in Europe in 2008, and is now in its eighth year in the U.S. under the sponsorship of NORD. In 2015, all five continents and 80+ countries participated. Through social media, there has been even greater awareness and participation.
This year’s theme “Patient Voice” recognizes the crucial role that patients play in voicing their needs and in instigating change that improves their lives and the lives of their families and caregivers. The 2016 slogan “Join us in making the voice of rare diseases heard” appeals to a broader audience who may not be living with or directly affected by a rare disease, and to join the rare disease community in making known the impact of rare diseases. People living with a rare disease and their families are often isolated. The wider community can help to bring them out of this isolation.
Some rare diseases (such as most types of cancer and Lou Gehrig’s disease, or ALS) are well known to the public. Many others, like pemphigus and pemphigoid, are not. I am sure many of you can empathize with millions of others who have a disease most people have never heard of, for which there is treatment, or one even being studied in clinical researchers. Thankfully, pemphigus and pemphigoid are being actively researched with new treatments being added to the list of clinical trials each year. But for others this is not always the case.
How Can You Raise Awareness?
Raising public awareness truly makes a difference. It gives families hope and can lead to new, life-saving treatments. You can help by participating in any of the following ways:
Social Media. Join the conversation on social media and use the official, global hashtag for 2016, #RareDiseaseDay.
I encourage everyone in our closeknit community to get involved by visiting the U.S. (www.rarediseaseday.us) or global (www.rarediseaseday.org) Rare Disease Day website before February 29 to learn about events in your area and what you can do.
The First Amendment to the United States Constitution guarantees the right of all citizens to communicate with their elected officials. So you might be asking yourself, “Does my Congressional Representative really care what I think”? Well, the answer is YES!
A recent study showed that if a Member of Congress has not arrived at a firm decision on an issue that a constituent contacting their Washington D.C. office will influence their decision. The study illustrated the degree of influence that the following types of communication will have on a congress person’s decision: phone calls 72% influence, individualized postal letters 70% influence, and individual email messages 69% influence.[/vc_column_text][vc_custom_heading text=”The study illustrated the degree of influence that the following types of communication will have on a congress person’s decision: phone calls 72% influence, individualized postal letters 70% influence, and individual email messages 69% influence.”
Writing your Congressional Member may seem like a small thing, but they need to know that their constituents are paying attention. They want to hear about what issues are important to you. When writing your letter/email to your Congressional Member, you may want to share your story with them and how it relates to your issue. Personalizing your letter/email will grab their attention and give them more reason to consider your issue. It is recommended that you keep your letter/email simple and no more than three paragraphs. Provide facts about the issue you are addressing and try to be as specific as possible. Be sure that you cite the name and number of the Bill or Legislation that you are asking them to support. If they are already a supporter of the Bill, thank them.
Don’t forget to provide your contact information so they can respond directly to you. Remember, your voice can go a long way towards helping your Congressional Member know where the public stands on your issue. The more you correspond with your representative, the more it will help you build a long-lasting relationship with him or her. That relationship is not only your right as a citizen, but a privilege you should exercise.
Bill Starrels (l), Marc Yale (c), and Sarah Gordon (r) outside the Washington, DC office of Congresswoman Julia Brownley of California.
When the email landed in my inbox last month asking for pemphigus and pemphigoid patients and caregivers to advocate on Capitol Hill, I didn’t hesitate. I live just miles from the Hill, and it was time to share my story and encourage lawmakers to take a stance on research and treatment for rare diseases like pemphigus and pemphigoid.
When my pemphigus was diagnosed eight years ago, I had just given birth to my first son. It was a difficult time, combining the exhaustion and stress of new motherhood with the pain, confusion, and anxiety of an undiagnosed illness. Once I was diagnosed, the first line of treatment, Cellcept, caused neutropenia, a white blood cell condition making me susceptible to infection. So with much trepidation I used the off-label treatment, Rituxan. In retrospect, I see that despite the challenges of my illness and treatment, I was incredibly fortunate. I saw Dr. Grant Anhalt, who was a true lifesaver, and the Rituxan worked beautifully! I have now been in remission for nearly five years. Most remarkably, I was able to give birth to another healthy and happy boy four years after the first. Yes, when I received the email last month asking for advocates on the Hill, I knew it was time. And what a time it was.
Before heading up to the Hill, I discussed the various bills before the House and Senate with Marc Yale, Certified Peer Health Coach at the IPPF. Unfamiliar with congressional practices and language, I was still easily able to grasp the reasons these bills were so crucial for people with rare diseases like pemphigus and pemphigoid. They dealt with incentivizing drug companies to repurposes medications like Rituxan to make them more accessible; they defined the terms of telehealth, a crucial first step toward allowing highly specialized physicians to treat patients remotely; they aimed to maintain funding for vital NIH research on rare disease treatments. Through my conversations with Marc, I found that my personal story could speak directly to the importance of several of these bills.
On October 20, I met Marc, Kate Frantz, Awareness Program Manager at the IPPF, and Bill Starrels, whose wife has pemphigus, at the Capitol. Entering the congressional office, I had the uneasy sensation of being on an episode of the HBO comedy Veep. But I quickly found that the staffers with whom we met were not snarky and self-involved, but rather knowledgeable, compassionate, and devoted to their work. We met with staffers from the offices of Julia Brownley, Doris Matsui, Barbara Boxer, and Dianne Feinstein from California, as well as my own representative, Eleanor Holmes Norton, of Washington, DC. Speaking to them about the bills at hand through the lens of my personal experience made me feel at once vulnerable—presenting myself for the first time as a person with a rare disease—and empowered—taking the opportunity to advocate on behalf of myself and so many others. I learned a tremendous amount about how Congress functions and also quite a bit about my own potential as an advocate.
In the end, I was correct: the time was right for me to share my story with lawmakers and become an advocate for persons with rare diseases such as pemphigus. Keep an eye on your inbox for messages from Mark and Kate. You might just find that soon the time will be right for you as well.
