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:
I recently spoke with a patient who stated that his marriage was under a great deal of strain – which is highly understandable as the significant others of patients are the caregivers and are often in the line of fire, so to speak.
This was not the first time a wife or husband had confided this to me. Helplessness can cause patients and/or their caregivers great despair – to which wanting to run away is an understandable reaction.
Patients experience pain, embarrassment, and uncertainty when afflicted with P/P or other rare diseases.
The caregivers can empathize, the caregivers cannot truly feel what the patients are experiencing.
Everyone who is a caregiver tries his or her best to be supportive. Every patient who is undergoing this challenge is bound to be depressed and scared at times. Every family member may feel helpless most of the time.
This is the time to reach out and ask for guidance. Finding support groups is easier these days due to social media. Pemphigus Vulgaris is only one of 7,000 rare diseases that exist today and there are sources of information for each one of them. Search the Internet and contact local support groups. Check out the link given here for caregivers (It’s one of the very best!).
http://www.caregiveraction.org/
This article was originally was posted at http://www.medscape.com/viewarticle/840206
Recently a wise professor told my class that we medical professionals are some of the most powerful people in the world. Indeed, we have the ability to meet people at their most vulnerable, sometimes on the very worst day of their lives, and help them. “This profession,” he told us, “is such a privilege that we must never miss the opportunity to have at least done some good for every patient.” Over the past several weeks, as I have planned rare disease awareness events and begun preparing to enter the clinical years of my medical education, I find myself considering these words more frequently. However, at the end of the day, I wonder how powerful we are, really…
My interest in rare diseases originated during my senior year of high school, when I first started volunteering at the National Organization for Rare Disorders, Inc. (NORD). I was overwhelmed to discover the many obstacles experienced by patients who have rare diseases. On a technical level, any disease that affects fewer than 200,000 Americans is considered rare. Of the more than 7000 rare diseases, only approximately 350 have treatments that are approved by the US Food and Drug Administration (FDA). I found that individuals with these diseases, almost two thirds of whom are children, show great courage and perseverance in the face of significant discouragement. Although I had always wanted to be a doctor, it was not until I witnessed such unrelenting determination to overcome barriers in healthcare that I discovered my own enthusiasm for medicine.
All physicians strive to provide attentive medical care with the utmost compassion and empathy; however, as medical professionals, we must also be our patients’ most vocal advocates. Although I was not yet a physician, I still wanted to contribute to this effort. I wanted to provide a forum for the nearly 30 million Americans with rare diseases whose voices often go unheard in the medical community, and I wanted to share this passion with my peers. Every year, my fellow students and I host a Rare Diseases Awareness Event. Patients, students, clinicians, and researchers come together to share their experiences and insights regarding rare diseases. We strive to shed light on the lives of these individuals by allowing them to share their own stories, sometimes for the very first time.
Although many students may assume that we do not need to know as much about rare diseases because we are unlikely to encounter them in our practice, this is simply not the case. In fact, every one of us preparing for medical careers will see patients with rare diseases, and the extent to which we prepare ourselves for this reality will determine the impact we can have on these patients’ lives. Patients who have a rare disease face difficulty in every step of medical care, including diagnosis, treatment, and preserving quality of life. Sometimes, patients go years without receiving the correct diagnosis for their condition. Once they finally have an answer, often no treatment is available for their condition. As future physicians, we must aim to improve these prospects; the first step in doing so involves developing a keen understanding of this patient population.
The opportunities for medical students to learn about rare diseases are vast. Gaining a basic understanding of how the experience of having a rare disease is different from having a more common disease is equally essential. The National Institutes of Health (NIH) has great information related to rare diseases on its website, and the NORD website provides overviews and links to more than 200 patient organizations that provide excellent information about specific rare diseases. Students can also apply for a free NORD student membership by writing to bhollister@rarediseases.org. Once you register, you receive a monthly eNews and quarterly newsletter specifically designed for students planning healthcare careers. If you’re attending the American Medical Student Association annual convention in Washington, DC, on February 27 and 28, come to the NORD booth in the exhibit hall where patients with rare diseases will be sharing their stories.
With each speaker I listen to at a rare diseases event, with each new person I meet, I am filled once again with immense pride that our efforts, if even in a small way, have done some good. Unlike many people who are involved in advocacy efforts in this area, when I began this work, I did not have a personal connection to rare diseases. However, after years of getting to know people who have experienced these struggles, I can say that I now have several. In fact, it is the memory of the individuals I have met and the satisfaction in having contributed to raising awareness that has guided my interests, served as an influence in many decisions, and ultimately has been the driving motivation in achieving my aspirations. With every step I take moving forward in my career, rare diseases comes along with me and will continue to do so.
My passion for rare diseases advocacy has become perhaps the foremost aspect that defines me and has made me who I am. It has given me direction, has made me a leader, and continually prepares me to become one of those physicians who will do some good. A couple of years ago, one of my undergraduate professors asked me, “Are you that rare diseases girl?” He went on to say that a student who had been inspired to research rare diseases after attending my event had approached him with an interest in working in his lab. This is the reason why I raise awareness for rare diseases. If just one more person every year becomes inspired, that may eventually make all the difference in the world. It turns out that, in the end, we are all powerful together. After all, according to the NORD motto: “Alone we are rare. Together we are strong.”®
