Pachyonychia Congenitia Project

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PC Awareness – June 2023

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Every June, those of us living with Pachyonychia Congenita (PC) – plus our families and friends who love us – come together around a chosen theme to raise awareness about this debilitating disease.

This year, our theme for PC Awareness Month is “What I Wish You Knew About PC.”

We are so excited about this theme because it gives us a chance to share our personal stories and help educate our friends, family, doctors, and employers about what living life with PC is all about and what we’d like them to know.

Here are a few simple things you can do today:

  • Post your own selfie video to social media talking about what you wish people knew about PC and why that understanding would make things better for yourself or someone you love. (Make sure to use the hashtag #WhatIWishYouKnewAboutPC and tag @pachyonychia or complete this form (send video link, upload video/photos or write your answers))
  • Please watch for videos from others, and share them with your family, friends and followers.
  • Make a donation – All donations in the month of June will be matched.

What I Wish My Doctor Knew About PC

Jack Padovano

PC is a rare disease that affects people all over the world. Unfortunately, most people never heard of PC, and those who have often do not understand what it is and the challenges we face every day.

This can be incredibly frustrating, especially when the person who doesn’t know is your doctor. PC Project works hard to educate medical professionals. Watch and share this video by our Board Chair and PC patient, Jack Padovano.

What I Wish My School Knew About PC

Allison Block

I’m 13 years old and I have Pachyonychia Congenita. PC makes my feet hurt so much that I sometimes need crutches or a wheelchair. This makes everyday tasks really hard, especially in school. Sometimes I can’t get to my classes on time, and it’s hard to participate in some of the activities the other kids get to do.

I don’t want to be different. All I want is to go to school and learn, just like all the other kids. But to do that, I might need a little help like extra time to get to class or complete my assignments.

Most of my teachers try to be understanding. But some teachers just don’t get it. When teachers don’t understand what it’s like to have PC, it just makes a hard day even harder.

But I’m grateful that PC Project is working every day to educate people about PC, so that kids like me can get the support we need to do our best in school.

What I Wish My Friends Knew About PC

Stephen Wittmer

When PC Project asked me to make a video talking about what I wish others knew about PC, I almost said no.

I have spent a lot of time and energy hiding my PC, especially from my friends. PC causes extremely painful callouses and blisters on my feet. They are not very pretty to look at so I avoid any activities where my feet are exposed.

But it’s more than that. I also hide the fact that I’m in almost constant pain. I don’t want the people who care about me to worry.

But the hiding causes its own problems. Sometimes the pain makes me get angry and lash out. Sometimes I cancel plans last minute because I’m tired or my feet hurt. By not being candid about my PC, friends can think they did something wrong, or I don’t want to see them.

Because PC is so rare, it’s easy to feel alone. Thankfully PC Project has given me a community of other PC patients, their families, doctors, and other PC allies that provide understanding and connection.

But I also have a community of friends that would support me too, if I’d just give them the chance to understand what I’m going through.

What I Wish My Family Knew About PC

Vania, Victoria and Kimberly Diaz Rosas

When my daughter Vania was first diagnosed with PC, I had a million different questions. Some the doctors could answer, others they couldn’t.

Fortunately, I found PC Project.

PC Project helped me understand what PC is and what it isn’t. They reassured me that Vania could still have a great life. They taught me ways to manage her symptoms, and what to expect as she got older. PC Project is a vital resource I still count on almost every day.

But no matter how much I educate myself about PC, I can’t know how my daughter experiences having PC unless she tells me.

So my number one job as a mom and PC caregiver is to listen and understand.

I believe families are the first line of support for PC patients. Even a good day is hard for Vania, and she will encounter enough challenges outside of our home. As her family, we want to provide love and empower her.

And that’s what PC Project strives to do, too. I’m comforted knowing that Vania and other PC patients also have their PC Project family to count on when they need them.

What I Wish My Employer Knew About PC

Helaine Alessio

Every day when I go into work, my spirit and energy starts pretty high. But unfortunately, it doesn’t last.

As the day goes on, pain in my feet from Pachyonychia Congenita (PC) takes its toll on my mood and my energy level. I’m less productive than I want to be.

Because this disease is so rare, most people have never heard of it, including my employer and coworkers. Even after I explain, most still don’t understand how hard it can be to do the things they take for granted — things like standing up or walking just a few steps.

There’s a lot I wish the people I work with understood about PC. But most importantly, I wish they knew that having PC doesn’t mean I can’t be a good teammate. It’s the opposite.

PC has taught me that I’m more resilient than most people. I have lots of compassion for others when they are struggling or in pain. I find creative solutions to many of the challenges of PC. All of these qualities make me better at my job.

That’s what I want my employer and coworkers to know.

2023 IPCC Symposium at ISID

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With excellent presentations, a room packed with researchers, and many more standing in the back, our annual meeting of scientific collaboration to advance research and drug development for those who suffer from PC exceeded our expectations!

Date: May 10th, 2023
Time: 8:00 am to 1:00 pm, Tokyo, Japan
Location: Takao Room (42nd Floor) at the Keio Plaza Hotel Tokyo, Japan

The meeting was held in person in conjunction with the First International Societies for Investigative Dermatology (ISID) Meeting in Japan.

All researchers, clinicians, and industry representatives interested in PC (and other rare conditions with painful PPK) were invited to join at no cost.

For symposium attendees, PC Project hosted a traditional Japanese dinner Tuesday, May 9th (the night before the meeting) at 6:00pm JST at Koshitsu Izakaya Banya Nishi Shinjuku, 2-6-1 Nishi Shinjuku, Sjinjuku Sumitomo Bldg. 49 Floor, Shinjuku, Tokyo 163-0249. It was a lovely evening with good company, good food, and a spectacular view.

Click here for the program book in pdf format. Below were the featured speakers for this meeting:

8:00 AM
PC Project Update
Janice Schwartz, BA
Executive Director, Pachyonychia Congenita Project
蕾 Tsubomi (Pathogenesis session)
Chair: Edel O’Toole, MD, PhD, FRCP
Professor of Molecular Dermatology, Centre for Cell Biology and Cutaneous Research, Blizard Institute, Queen Mary University of London and Consultant Dermatologist, Royal London Hospital
8:10 AM
Palmoplantar keratodermas in 5 steps
Eli Sprecher, MD, PhD, MBH
Chair, Division of Dermatology, Deputy Director General for R&D and Innovation, Tel Aviv Sourasky Medical Center, Frederick Reiss Professor of Dermatology and Vice Dean for Clinical Affairs, Faculty of Medicine, Tel Aviv University. Tel Aviv, Israel; IPCC Chair
8:30 AM
Effects of keratin gene loss on epidermal keratinization programs
Leopold Eckhart, PhD
Associate Professor, Department of Dermatology, Medical University of Vienna, Vienna, Austria
8:45 AM
Experience with autograft therapy using revertant keratinocytes in severe recessive epidermolysis bullosa
Akiharu Kubo, MD, PhD
Professor, Division of Dermatology Department of Internal Related Kobe University Graduate School of Medicine
9:00 AM
Neuronal control of sweat gland stem cells in the palmoplantar skin
Catherine Pei-ju Lu, PhD
Assistant Professor, Hansjӧrg Wyss Department of Plastic Surgery, New York University Grossman School of Medicine
9:15 AM
Molecular mechanisms of intermediate filament tetramerization
Christopher G. Bunick, MD, PhD
Associate Professor of Dermatology Program in Translational Biomedicine, Yale University
9:30 AM
Connecting Skin Inflammation and Morphogenesis through Desmosomes
Kathleen J. Green, PhD
Joseph L. Mayberry Professor, Departments of Pathology and Dermatology, Northwestern University Feinberg School of Medicine
9:45 AM
Pachyonychia congenita keratin isoforms modulate motility signals during wound healing
Benjamin A. Nanes, MD, PhD
Instructor of Dermatology, University of Texas Southwestern Medical Center
10:00 AMBREAK – Group Photo
開花 Kaika (Therapies session)
Chair: Roger Kaspar, PhD
CSO and founder of Ayni Therapeutics
10:15 AM
Towards an in vitro model of Pachyonychia Congenita
Laura Ramos, PhD
Postdoctoral Researcher, Blizard Institute, Queen Mary University of London, UK
10:25 AM
This image has an empty alt attribute; its file name is squarePicture-JackowJoanna.jpg		Innovations in CRISPR gene editing for epidermolysis bullosa
Joanna Jacków, PhD
Lecturer in Molecular Dermatology and Therapeutics, St. John’s Institute of Dermatology, School of Basic and Medical Biosciences, Faculty of Life Sciences and Medicine, King’s College London
10:40 AM
Personalized preclinical gene editing protocols for in vivo correction of pachyonychia congenita
Marta Garcia Diez, PhD
Bioengineering Department, Universidad Carlos III de Madrid (UC3M);  Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT); Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII); Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain
10:55 AM
Gene therapy for Epidermolysis Bullosa
M. Peter Marinkovich, MD
Associate Professor Stanford University
11:10 AMBREAK
満開 Mankai (Clinical insights and disease modeling session)
Chair: Alain Hovnanian, MD, PhD
INSERM UMR1163, Imagine Institute, Department of Genetics, Necker hospital for sick children, Paris University, Paris, France
11:25 AM

Mouse models for Pachyonychia Congenita
Pierre A. Coulombe, PhD & Amanda Orosco, PhD Candidate
G. Carl Huber Professor and Chair, Department of Cell and Developmental Biology, University of Michigan Medical School
11:40 AM
Lessons learned from pachyonychia congenita may provide clues to the pathogenesis of hidradenitis suppurativa
Michelle Lynn Kerns, MD, MS
Assistant Professor of Dermatology at Cleveland Clinic
11:55 AM

Erythromelalgia: A newly identified feature of pachyonychia congenita
Rebecca McCarthy, BMBS (Hons), BSc (Hons)
Clinical Fellow for Professor Edel O’Toole, Blizard Institute, Queen Mary University of London
12:05 PM
Palmoplantar keratoderma’s in the Netherlands, a continued learning experience
Antoni Gostynski, MD, PhD
Dermatologist, Maastricht University Medical Center
12:20 PM
Complicated genetic background of palmoplantar keratoderma in Japanese: A heterozygous SERPINB7 variant is a possible modifying factor for clinical features.
Masashi Akiyama, MD, PhD
Vice Dean, Nagoya University Graduate School of Medicine, Nagoya, Japan, Professor and Chairman, Department of Dermatology
12:35 PM
花吹雪 Hanafubuki – Renewed commitment and a brighter future for patients!
Lunch & Symposium Adjourns
1:15PM
PC Project’s Medical and Scientific Advisory Board Meeting
By Invitation Only

Pachyonychia Congenita Research Grant Guidelines

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Download PDF Version of Grant Guidelines

MISSION
PC Project is dedicated to finding effective treatments and a cure for Pachyonychia Congenita (PC), a painful, debilitating skin disorder, through helping and connecting patients, empowering research, and partnering with industry.

VISION

A day when PC sufferers will live without excruciating pain, isolation, and embarrassment.

INTRODUCTION AND OVERVIEW
Patients with Pachyonychia Congenita (PC) suffer daily with debilitating pain, typically from painful calluses on the soles of the feet or inflamed cysts on their skin.  PC is a rare genetic skin disorder caused by spontaneous or inherited, autosomal dominant mutations affecting the coding sequence of one of five keratin genes, KRT6A, KRT6B, KRT6C, KRT16 or KRT17.

A vital part of PC Project’s mission is facilitating and funding research. PC Project is committed to funding promising investigations that can lead to approved therapies and ultimately a cure for PC. Since its founding in 2003, the PC Project has supported an open grant application system and funding for researchers interested in PC on a case-by-case basis. With the goal of maximizing the impact of resources available and accelerating research relevant to PC, the PC Project Board of Directors met in October 2022 and elected to establish a formal Pachyonychia Congenita Research Awards and Grants Program.

Funding awards given by PC Project will be based on the “PC Research Agenda”, a published document that is focused on patient needs and was authored by researchers and clinicians through multidisciplinary collaboration. Accordingly, PC Project’s grants will support investigators interested in conducting innovative PC research. Discovering new avenues for therapies (of which there are currently none), and one day, a cure for PC are key aims of these awards.

RESEARCH FUNDING PRIORITIES PC Project seeks to fund innovative research with these goals:

  1. Fundamental research that will increase understanding of the mechanisms underlying the various symptoms of PC.
  2. Translating these discoveries into diagnostic and therapeutic advances for PC.
  3. Leading to improved quality of life for individuals with PC.

ELIGIBILITY AND REQUIREMENTS

Investigators at all levels, from clinical and post‐doctoral fellows to established investigators, are eligible to receive awards. Non‐U.S. investigators are eligible, and the research funded by PC Project can be conducted at non‐U.S. institutions. Investigators must be members of an accredited academic institution. All research projects involving human subjects must receive IRB approval (see below). Research funds from PC Project will only be paid directly to the Investigator’s institution (grantee institution). Specific information about allowable expenses and other relevant guidelines is provided below.

FEDERAL REGULATIONS; IRB and IACUC APPROVAL

Investigators are required to comply with all applicable federal, state, location, or regional regulations that pertain to their research. All research involving human subjects or animals must have proper regulatory approval before any work takes place. Applicants must attest to IRB/IACUC approval status in the online application, and awardees must agree to provide a copy of IRB/IACUC approval as a condition of funding. Proof of ongoing approval is necessary on a yearly basis. Applicants are strongly encouraged to obtain IRB approval before applying to begin research within the required six‐month window (see “Award Period” above).

APPLICATION REVIEW

PC Project’s Research Committee will review and rank award applications. The PC Research Committee will evaluate each application using criteria that includes scientific merit of the proposal, investigator track record, institutional environment, and impact of the proposal on the mission of PC Project. Final approval and funding of applications will be made by PC Project’s Board of Trustees.

CONDITIONS OF AWARDS

Notification: The principal investigator and the grantee institution will be informed in writing about the duration and amount of the grant awarded.

Award Period: Projects are expected to begin within six‐months of the application due date. Special requests to extend this time‐period must be submitted in writing to info@pachyonychia.org and will be evaluated on a case‐by‐ case basis.

Payment: To be made in one or multiple payment sums depending on the grant amount and timeline. The first payment will be made within approximately 30 days of receipt of the signed PC Project Research Award Contract or upon request of the grantee.

USES OF FUNDS

Funds obtained from PC Project may be used only for the specific use(s) set forth in the grant proposal and contract. Funds not used during the grant period for the specific use(s) set forth in the grant contract must be returned to PC Project at the end of the grant term unless an exception has been granted in writing by the PC Project. Requested funds such as for researchers, consumables, and facility access changes must be clearly detailed and costed in the proposal.

The following expenses are not allowed:

  • salaries, benefits and/or stipends for persons not specifically identified in the grant budget;
  • institution administrative and facilities costs, except for up to 5% of the grant which can be specified for indirect costs, if needed;
  • general fundraising expenses;
  • journal subscriptions, advertisements, tuition fees, professional society dues;
  • travel and related expenses (except to the extent travel pertaining to study participants is included in the approved grant budget), meals or entertainment;
  • lobbying.

PROGRESS REPORTS

Interim Progress Report: A report of scientific progress on the research project is due within thirty (30) days of the mid-point of the grant award period.  Failure to meet this requirement will result in withholding of the 2nd installment of grant funds until the report is submitted and may result in early termination of the award. (If applicable and it’s a multi-payment grant.)

Final Research Report: The final progress report will be a scientific report including a list of titles and publications of papers supported all or in part by this research award. The final report shall be submitted to PC Project within sixty (60) days of the end of the grant. The undersigned acknowledges that failure to submit a final report on time will result in automatic elimination of consideration for subsequent awards.

ACCOUNTING REQUIREMENTS

Final Financial Report: A full reconciliation of all expenses made for a research project is due within sixty (60) days of the completion/termination of the grant. Reconciliations should be emailed to info@pachyoncyhia.org by the financial officer of the grantee’s institution. Unexpended funds remaining at the end of the grant must be returned to PC Project. PC Project is not responsible for over‐expenditures of grants. Unallowable costs charged to the grant must be reimbursed to PC Project.

Rebudgeting is not allowed without prior approval from PC Project.

PUBLICATIONS AND REPRINTS

PC Project grants are awarded with the expectation that the most important results of the funded research will be presented at scientific meetings and ultimately published in peer‐reviewed journals. The responsibility for both lies with the principal investigator.

When a manuscript funded wholly or in part by a PC Project grant is accepted for publication, a copy of the accepted version, with the journal Editor’s acceptance notification should be sent electronically to info@pachyonychia.org.

If a press release from the institution is being prepared for distribution, PC Project would like to participate in the process. Please notify PC Project at info@pachyonychia.org.

PC Project should be acknowledged in all dissemination materials (i.e. publications, scientific presentations, press releases, etc.) related to research supported in full or in part by the Foundation. The following acknowledgment, or its equivalent, should be used: “This work was supported (in part) by a grant from Pachyonychia Congenita Project.”

LIABILITY POLICY:

PC Project is not responsible for any claim, judgment, award, settlement, negligence, or malpractice arising from a research project or investigation funded or related to an award provided by PC Project. The awardee and institution acknowledge responsibility for the conduct of research or investigations related to the award resulting from any act or omission on the part of the awardee and/or institution, their employees, agents, or representatives to the extent allowed by applicable law, and release PC Project from all responsibility.

INTELLECTUAL PROPERTY OWNERSHIP:

The Institution shall retain ownership of any intellectual property created using PC Project grant funds. However, PC Project reserves a non-exclusive royalty free license to use any intellectual property created by Institution using PC Project Grant funds for non-commercial purposes.

POLICY ON SCIENTIFIC MISCONDUCT AND FRAUD:

Any institution that receives funds from PC Project must have written policy guidelines on conflict of interest and research integrity including scientific misconduct and fraud. It is the responsibility of the institution and the grantee/awardee to inform PC Project immediately, in writing, of any institutional investigation into the conduct of an investigator whose work is supported by PC Project, and to keep PC Project informed in a timely manner of the progress and outcome of such investigation. A finding by the institutional investigation that the investigator was guilty of research fraud or misconduct represents sufficient grounds to terminate PC Project’s support of a project.

The following materials must be submitted with your application:

APPLICATION CONTENTS

  • A. Title page and Abstract
  • B. Layperson’s statement (3-4 sentences max; use non-expert terminology).
  • C. Budget and timeline (one page max)
  • D. Applicant’s biographical sketch (NIH format) (four pages max)
  • E. Research proposal (five pages max)

A. TITLE PAGE AND ABSTRACT

Include the project title, principal investigator (PI) name and affiliations, and the addresses of the PI and the official to whom correspondence should be addressed.

B. LAYPERSON’S STATEMENT

In four sentences or less, describe the goals the project you are proposing and its significance for patients with pachyonychia congenita using language that a person who is not trained in or does not have a detailed knowledge of this subject would understand. This statement should have a layman’s title, relate the aims of the project and its significance to the understanding or treatment of PC. The layperson’s statement will be used for a variety of PC Project communications including its website, newsletter, press releases, award presentations, and others.

C. BUDGET

A budget must be submitted. A concise narrative of how you propose to allocate funds must be included. PC Project may award funds based on the original budget or make conditional awards dependent upon requested adjustments to the original budget. Funds may only be used for the purposes outlined in the original budget and research proposal. Any significant deviation from the original budget and/or project must be approved by PC Project with a written justification.

Departmental and Indirect Costs: Award funds are to be used only for the designated recipient and project for which the application was submitted. Five percent of the award funds may be used for indirect costs of the institution or department.

D. APPLICANT’S BIOGRAPHICAL SKETCH

A biosketch must be submitted. Please use the format used by the National Institutes of Health in the United States, or the equivalent.

E. RESEARCH PROPOSAL:

This section includes a description of the research proposal in sufficient detail for adequate evaluation by the Scientific Review Committee. Applicants should make every effort to be succinct and use figures or tables to summarize their plans. The research proposal, including items 1‐5 below, cannot exceed 5 (five) pages, including figures and tables but excluding references. Applications exceeding this page limit will be returned. Font size can be no smaller than Arial 11 point (or the equivalent character density).

Suggested Format:

  1. Specific Hypothesis and Aims
    Describe the hypothesis that is to be tested and the specific aims.
  2. Background and Significance
    Describe why the research is important and what has already been done in the field. Evaluate existing knowledge in the field, and specifically identify the possible contribution your investigation may make.
  3. Preliminary Studies
    Describe in detail what has already been accomplished on the project to support the hypothesis and aims.
  4. Experimental Design
    Describe in detail the experimental design, the methodology, tools, and models to be used, and the manner in which the data will be analyzed. Do not include details of established laboratory procedures.
  5. Future Plans
    Describe how the successful achievement of the proposed specific aims will lead to a competitive application for a larger grant.
  6. Environment and Resources
    Describe in detail your organizational structure regarding resources and institutional commitment to pachyonychia congenita clinical services and/or research. Include where the work will be performed and sources of any supplemental funding supporting the project.
  7. Literature Cited
    Include all literature cited in your proposal.

PC Project will award the following types of grants:

  • Catalyst Research Grant – up to $50,000
    Designed to support talented early-career scientists on the path toward becoming the next generation of PC thought leaders by supporting hypothesis-driven research projects.
  • PC Project Champion Research Grant – up to $100,000
    Encourages established researchers to pursue research on ongoing or emerging challenges in PC or bring their expertise to the field of PC.
Return to Apply for a Grant Page
Direct link to Grant Application

PCers Needed to Answer 4 Questions

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2023 Voice of the Patient Update
Skip survey header

2023 Voice of the Patient Update

The EL-PFDD meeting with FDA in 2018 was a success and has been very critical in PC drug development. Because it has been five years, we plan to send an update to FDA officials to let them know we still need treatments for PC. Some of these questions may look familiar but it is important for you to complete them again for this update to the official Voice of the Patient Report.

Do not include your experience with recent clinical trials treatments in your answers.
Contact Information
What is your age or the age of the person you care for?
In which PC gene is your specific mutation found?
1. Overall, how would you characterize your PC disease severity or the severity of the person you care for compared to someone without PC?
2. Which PC conditions have impacted your life? Check all that apply.
3. How satisfied are you with the treatments available today for your PC?
4. Have you ever utilized a mobility assistance device (e.g., wheelchair, cane, scooter, walker) or employed an alternative form of mobility (e.g. such as bikes, strollers, crawling, holding onto walls or rails, piggy-backing, holding onto another person, etc.) because of your PC?
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Camp Discovery

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Every summer, the American Academy of Dermatology (AAD) sponsors five (5) one-week sessions of Camp Discovery — a camp designed specifically to meet the medical needs of kids living with chronic skin conditions such as eczema, psoriasis, vitiligo, alopecia, epidermolysis bullosa, ichthyosis, or any other dermatologic condition that creates an impact on a child’s ability to actively participate in activities/events with their peers.

As you may know, research has shown that there is an impact of pediatric skin disease on self-esteem. In a recent study that evaluated the impact of camp on children with cancer, and their siblings, findings report positive impacts of the camping experience on a range of outcomes related to emotional, physical, self-esteem, and social functioning.

Camp Discovery is:

  • Provided at no cost to families (including airfare), kids from across the United States, and around the world, attend Camp Discovery to experience an adventure filled with fun, friendship, and personal growth. This unique program provides a safe environment that enables and encourages kids to explore and participate in activities such as fishing, swimming, archery, horseback riding, nature trails, and more. Mastering a new skill, or simply trying something new, Camp Discovery can help your child build strength, resilience, and confidence.
  • An environment where kids are surrounded by peers who also are living with dermatologic disease, amazing counselors, and a dedicated and caring medical staff – Camp Discovery enables kids to be kids, and simply have fun, in a place where they are understood, they look and feel like everyone else, and their diagnosis is not a barrier.

YOU can impact the life of a child living with dermatologic disease.

Apply using the referral form, visit campdiscovery.org or download the 2023 flier.

CSD on the Hill 2023

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PC Project joined with other skin disease organizations – all members of the Coalition of Skin Diseases (CSD) to advocate for patients. Learn more about the CSD and Advocacy Program. There were three main asks:

  • Advance Medical Research (National Instituted of Health (NIH) Funding)
    • More research activities are needed to continue emerging opportunities to advance our scientific understanding of skin diseases. Without meaningful financial support the development of treatment options will be delayed, and we may loose the next generation of talented young investigators to foreign competition and other fields. To fully capitalize on innovative research projects investigating skin diseases and related conditions, the ask was to increase NIH funding with at least $50.924 billion in FY 2024. That is a 3.465 billion increase from provided funds in FY 2023.
  • Support Increased Prevention and Awareness (Center for Disease Control and Prevention (CDC) Chronic Disease Education and Program (CDEP))
    • The CDC’s National Center for Chronic Disease Prevention and Health Promotion (NCCDPHP) supports several programs that work to increase public awareness and improve professional education. Additional funding will enable CDC to support more proposals in subsequence years. In the interest of growing the program, supporting timely public health efforts, and ensuring the viability and effectiveness of emerging opportunities, the ask was to provide this new program with $6 million for FY 2024.
  • Support Patient Access Legislation
    • Step Therapy practices require patients to try and fail one or more treatments before the insurer covers the treatments originally prescribed. Too often, this leads to delays in proper treatment, worsened health outcomes, and contributes to higher healthcare costs. The Safe Step Act legislation (S.652/H.R.2630) would require insurers to implement a clear and transparent process for a patient or healthcare provider to request an exemption to a step therapy protocol and requires group health plans to grant exemptions if certain protocol is met.
    • The HELP Copays Act (H.R. 830) closes a loophole that allows many employer health plans to deem certain covered drugs as “nonessential,” which means that the insurer will not count any cost-sharing toward the patient’s deductible and out-of-pocket maximum. Your bill will require all private plans to count all cost sharing for covered services to accrue to a patient’s deductible and out-of-pocket maximum.
    • S.127 – Pharmacy Benefit Manager Transparency Act of 2023 bans deceptive unfair pricing schemes; prohibits arbitrary claw backs of payments made to pharmacies; and requires PBMs to report to the FTC how much money they make through spread pricing and pharmacy fees.

PC Project met with the legislative staffers for both Oregon and Utah states.

Met with Francisco Flores-Pourrat, Legislative Assistant to Rep. Lori Chavez-DeRemer (R-OR-05)

Met with Chris Medrano, Legislative Assistant to Sen. Mike Lee (R-UT)

Met with Kannon Butler, Legislative Correspondent for Rep. John Curtis (R-UT-03)

Met with Max Seifert, Legislative Correspondent to Sen. Jeff Merkley (D-OR)

A Congressional Briefing/Luncheon was held on Capitol Hill, April 25th from 12:30-1:30 pm.  The purpose of the briefing was to highlight the various impacts and burdens of Skin Disease.  The goal was to put dermatology on the radar of our federal legislators and their staff, and give them a reason to care about the issues skin disease patient populations face.  Speakers included:

  • Kelly Barta President, Coalition of Skin Diseases
  • Lindsey A. Criswell, M.D., M.P.H., D.Sc. Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) *Virtually
  • Michael Siegel, PhD Executive Director, Pediatric Dermatology Research Alliance (PeDRA)
  • Lisa Butler, MBA Executive Vice President, National Eczema Association (NEA)
  • Becky Strong Outreach Coordinator, International Pemphigus and Pemphigoid Foundation (IPPF)
  • Janice Schwartz Patient Advocate and Executive Director, Pachyonychia Congenita Project (PC Project)
  • Brindley Brooks Patient Advocate and Founder, HS Connect
Watch PC Project’s Executive Director, Janice Schwartz, talk at the Congressional Luncheon April 2023.

There was also time to collaborate with other organizations and learn from each other.

In addition, Janice had a filmed interview to share PC Project’s Story.

Janice talked with Dr. Isaac Brownell from NIAMS about how to advance PC research.

Coalition of Skin Diseases Meeting

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Gathering to learn from and help other patient advocacy groups

PC Project attended the Coalition of Skin Diseases Development Day meeting in conjunction with the American Academy of Dermatology Annual Meeting in New Orleans on May 18, 2023. PC Project met with other leaders of patient advocacy organizations for various skin diseases.

Our leader, Janice Schwartz, was invited to speak about the power and value of our International PC Research Registry. We are always happy to share our knowledge with others about why our patient registry is so important in helping patients receive a correct diagnosis, in understanding our disease, and advancing research for PC. Of course, our registry is powerful in drug development because we have a pool of willing, genetically confirmed patients ready to participate in clinical trials.

In turn, PC Project learned from other organizations and gathered tips and ideas on how to operate even more effectively!