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.
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:
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.
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.
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.
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.
