How to Use the PRP Survival Guide

From the Editor

I was diagnosed with pityriasis rubra pilaris (PRP) on November 28, 2012 — nearly three months after a red spot appeared on my forehead. 

Someone from my dermatologist’s office called me to confirm the diagnosis and to schedule an appointment for the following day. All she shared with me was the proper spelling of pityriasis rubra pilaris. 

That evening I performed a series of internet searches and devoured  two dozen healthcare-related websites. Unfortunately, the information I uncovered was limited in scope and redundant in content. Even more disconcerting, however, was the presumption that I was familiar with medical terms. Arrgh! 

Sometime during the early morning hours of November 29, as I became overwhelmed by my ignorance, I knew I needed to find a PRP Survival Guide. 

When I discovered that there wasn’t one, I started writing it. Three years ago — May 20, 2015 — the PRP Survival Guide was officially introduced to the worldwide PRP community.

SEARCHING THE INTERNET

The PRP Survival Guide is offered as an alternative to unstructured and random forays using Dr. Google and Dr. Yahoo. These efforts will almost always lead to frustration and frequently to misinformation. If we do are job properly, you will either (1) find the answers you seek or (2) send the PRP Survival Guide out in search of those answers. Learn more about using Dr. Google.

SEARCHING THE PRP SURVIVAL GUIDE

Please use the SEARCH field to locate questions, chapters and topics.  Each post in the PRP Survival Guide has been assigned a unique alpha-numeric identification. Enter prp00 and this “How to Use…” post appears. The locator number follows every post.

TRANSLATE FEATURE

Every page in the PRP Survival Guide has a TRANSLATE button powered by Google Translate. The pull-down menu offers 100 language options. The translation applies to the post/page as well as any replies that follow.

ORGANIZATION

The PRP Survival Guide is divided into seven CHAPTERS. Each chapter has QUESTIONS. Each Question has an ANSWER. Each answer seeks additional FEEDBACK from the PRP community and healthcare professionals..          

Chapter 1 — PRP Basics: Understanding PRP 
These are the questions asked early in the PRP journey by newly diagnosed patients and caregivers,family and friends, co-workers and employers, teachers and school administrators, and so many more.  [enter A00 to access Chapter 1 index]          

Chapter 2 — Diagnosing PRP
This chapter covers the onset of symptoms, misdiagnoses, biopsies as a diagnostic tool and what it takes to get an “official” PRP diagnosis. For most of us, this is looking back to see how our experiences compare with others in the PRP community.   [enter B00 to access Chapter 2 index]          

Chapter 3 — Treating PRP
The focus here is on treatment options (prescription drugs and topicals) as well as managing our expectations. We all learn early in the journey that treatment is a roll of the dice — what works for one doesn’t work for all. We also learn that not all dermatologists are PRP savvy. [enter C00 to access Chapter 3 index]          

Chapter 4 — Daily Life with PRP
Every aspect of the PRP experience … coping with the 24/7 challenges to body, mind and spirit. Feedback by hundreds of fellow travelers who have shared their insights based on their unique journeys with posts and comments as members of the PRP Facebook and RareConnect communities. [enter D00 to access Chapter 4 index]          

Chapter 5 — PRP Parents & Kids 
It is estimated that 45% of all patients diagnosed with pityriasis rubra pilaris are children. The challenges facing parents are extraordinary. This sub-chapter focuses on those special needs  [enter E00 to access Chapter 5 index]          

Chapter 6 — PRP and Remission
For most PRP patients and caregivers, the outcome we seek is remission. For others, the PRP journey is defined by long-term management of symptoms rather than remission. [enter F00 to access Chapter 6 index]          

Chapter 7 — PRP Research
The PRP community has long lamented the lack of research. Since October 2012, however, researchers at Thomas Jefferson University have been conducting ongoing genetic and clinical research. For all intents and purposes, TJU is the only game in town and we encourage PRP patients to play. [enter G00 to access Chapter 7 index]          

Chapter 8 — PRP Advocacy
As an ultra-rare skin disorder, PRP must find allies to open doors. How can the PRP community most effectively impact improvements in the diagnosis, treatment and research for PRP? [enter G00 to access Chapter 8 index]

DISCLAIMER The PRP Survival Guide is designed for educational purposes only and not for the purpose of rendering medical advice. It is not the intention of the PRP Survival Guide to provide specific medical advice, but rather to provide users with information to better understand and manage the burden of pityriasis rubra pilaris on body, mind and spirit. No individual should indulge in self-diagnosis or embark upon any course of medical treatment that is described in the PRP Survival Guide without first consulting a health care professional.  [enter H00 to access Disclaimer]

Survival Guide prp00

PRP — The Poem

by Greg Shalless

(PRP Onset January, 2008. Duration: Two years)

Pityriasis Rubra Pilaris
I don’t want to sound too alarmist,
But your skin turns bright red
And in layers is shed –
Who needs this kind of catharsis?

It happens when you’re quite mature
And you lose any sense of allure,
It will no doubt cause tears
And be with you for years,
For there is no known cause or a cure.

Fingers tingling and nails corroding,
Eyelids stinging and you are imploding,
Your “hot” body’s glowing,
Your dandruff is snowing
And your feet when in shoes are exploding.

Your hands feel like stiffened old leather,
But later on you won’t know whether
To be glad that soon it’ll
Crack up it’s so brittle,
But you try hard to keep it together.

Crispy skin simply falls off your feet
Like the flakes of a fine pastry treat
And the incessant itch
Is a right proper bitch
And your whole blood red body’s on heat.

It’s tough to explain what you’re feeling
When people ask if it’s healing
For your legs and your arms
Like the back of your palms
Are still scarlet and constantly peeling

They say it’ll pass but can’t tell you when
You see a marked improvement and then
Your new skin gets drier
Is once more on fire
And the whole thing starts over again.

The spread of this thing is pervasive,
And clothing can be quite abrasive,
But if you went “nudish”
Although they’re not prudish,
You’d even make nudists evasive.

And if you’re an unlucky squire,
Watch out for the “Great Balls of Fire”,
But if you’re a lady
At the zoo then you maybe
The Mandrill’s object of desire.

Didn’t you thank them – it seems you forgot?
Fifty odd years in the Skin Plant for what?
So your angry storeman
Has just shot the foreman,
And the staff have now all lost the plot!

PRP is quite clearly no joke
And its treatment might well send you broke,
Though they don’t have an answer
You haven’t got cancer
And you’d much rather this than a stroke.

© Copyright Greg Shalless – 2008

The Right to Say “NO”

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Editor’s Note:

Regardless of the worthiness of any cause, you have a right say “No”.It’s very easy to say “No” when a stranger knocks on your door. Whatever they’re selling, we’re not buying.

Let’s face it — we’re all getting pretty darn good at saying “No”. In fact, for most of us, “No” has become a knee-jerk reaction to any solicitation. HOWEVER, it’s harder — and some might say it is impossible — to say “No” when the person knocking on that door is a granddaughter in a Girl Scout uniform selling Girl Scout cookies. Her advantage is called FAMILY.

It has fallen to me to make the case for you to donate $10, $25, $50 or $100 to support our PRP community which includes the following:

✽   PRP Facebook Support Group

✽  PRP Community on RareConnect

✽  PRP Survival Guide

✽  PRP Alliance, Inc.
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The opportunity to say “YES”

Has our PRP Community supported YOU or a loved one? Have you had a question answered? Have we been able to turn fear into hope?
If the answer is No”, then please OPT OUT of any future fundraising emails from the PRP Alliance. However, if your answer is “Yes”, then please consider a donation to the PRP Alliance.

How will we use the money you donate?

Our budget is admittedly austere. Patient organizations for psoriasis and eczema have million dollar budgets with paid staff. Imagine, with only $3,600 we can jumpstart a year of building awareness, supporting research and helping our worldwide PRP community meet the challenges of body, mind and spirit. Unfortunately, the financial demands we face are front-loaded during the first three months of 2018.
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✽  Annual dues ($250): National Organization of Rare Disorders (NORD) and the Coalition of Skin Diseases (CSD)

✽  Communication Technology ($1,340): Constant Contact (communication) and ReadyTalk (video conferencing)

✽  Advocacy Events ($2,010):  Annual meeting of the American Academy of Dermatology (February 16-19) in San Diego and Rare Disease Week on Capitol Hill (February 25-March 1) in Washington, DC .

A community of PRP patients and caregivers

Whether patient or caregiver, active or in remission, we are on the same journey. YOU have not been alone.
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We itch together
We shed together
We cry together
We listen to each other
We share. We care.
We learn from one another
We are family
Thus far, 47 fellow PRP patients and caregivers have donated $2,810 (78% of our goal). These donations range from $10 to $220 with an average of $60. Quite frankly, any amount you can donate will be very helpful in allowing us to achieve our goals.
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✽  If you need more information to either inspire or justify a donation CLICK HERE

 If you already know that you want to make a donation via PayPal CLICK HERE

✽  If you prefer sending a check, please make it payable to “PRP Alliance” and mail to: PRP Alliance, 1500 Commerce Drive, Plano, TX 75093-2640, USA. The PRP Alliance is a 501(c)(3) is a. tax-exempt patient organization. ID# 47-3868578

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THANKS to those who have already donated…

Jandina G (Portland, OR)
Richard L (Lansdale, PA)
Janice & Ernie F (Plover, WI)
Jessica H (Stone Mountain, GA)
Janet B (Forest Lake, MN)
Carel R (Surrey, British Columbia, Canada)
Cheri H (Omaha, NE)
Frank G (Fairview, NC)
Ronald O (Sharpsville, IN)
Derek S (Otterville, Ontario, Canada
John K (Weston, CT)
Mike W (Park City, UT)
Eve J (Byron Bay, NSW, Australia)
Leslie L (Colorado Springs, CO)
Vincent F (Cape Coral, FL)
Sylvia C (Wayne, NJ)
Susan O (Amesbury, MA)
Chelsea B (Othello, WA)
Helen M (Madison, WI)
Phil S (Chester, England, UK)
Anita R (Pearl, MS)
Corrine P (Toronto, Ontario, Canada)
Eric S (San Luis Obispo, CA)
Wayne M (Kyabram, Victoria, Australia)
Jerry R (Sausalito, CA)
David H (Scottsdale, Tasmania, Australia),
Suzanne M (LaPlata, MD)
Paula M (Los Angeles County, CA)
Debra W (Winston-Salem, NC)
Martin W (Wolverhampton, England, UK)
Vickie B (San Diego, CA)
Sandi H (Turtle Creek, PA)
Aziz T (Laurens, SC)
 Diane H (Minneapolis, MN)
Donna S (Beaumont Hills, NSW, Australia)
Diedre H (Houlma, LA)
Tierney R (Virginia Beach, VA)
Bill M (Plano, TX)
Tierney R (Virginia Beach, VA)
Elaine A (Reno, NV)
Sara K (Miami, FL)
Cay & Bill B (Berthoud, CO)
Darcy L (Colorado Springs, CO)
Cami S (San Jose, CA)
Sharlene C (Australia)
Linda L (Somerset, CA
Roberta K (Sicklerville, NJ)
Deborah W (Royal Palm Beach, FL
Marion M (Dublin, Ireland)
Jan and Malcolm T (Ringwood, NJ)
Joan P (Portland, OR)
Mark & Paul A (Boston, MA)
Joyce & Stephen T (location n/a)
Daina B (West Sacramento, CA)
Jeff S (Dayton, OH)

Remember: We’re family and we need you.

Before You Opt Out


From the Editor…

The PRP Alliance utilizes the email technology developed by Constant Contact. They manage our email list, facilitate email distribution and provide the support for email design. No one is forced to be on the PRP Community Email List. You can unsubscribe at the bottom of any email you receive. It’s that simple. But keep in mind — YOU are the needle in the haystack.

The Genesis of the Email List

By early 2013, my version of PRP had sapped ALL my energy. I was languishing in bed watching cable news. My major excursions of the day were to the bathroom and kitchen. Neither journey brought much satisfaction.

One day, however, I quite unexpectedly discovered that I could park my butt in front of my computer and “work” for hours with absolutely no fatigue. Good bye CNN. Hello iMac.

As a member of the PRP Support Group — the one founded in 1997 — I had access to an archive of over 29,000 emails written by PRP patients and caregivers. Why not learn about PRP from the experts — those who have made the journey. The numbers were daunting until I reduced it down to something manageable:

I started building a database that included what I called core data:

 Name
  Location
  Onset date
  Onset age
  Misdiagnoses
  The role biopsies played in the diagnosis of PRP
  Information about the diagnosing dermatologist
  Date of diagnoses
  Information about the treating dermatologist

I also collected email addresses — over 1,500 of them. It took nearly six months to complete the data collection.

In November 2013, I sent emails announcing the first PRP Worldwide Census to 1,500-plus PRP patients/caregivers. Predictably, within 24 hours over 500 emails bounced back as undeliverable.

I learned that there is a natural ebb and flow of PRP patients and caregivers within the PRP community. As current patients achieve some acceptable level of remission they leave,  other newly diagnosed PRP sufferers join our ranks.

Ebb and Flow

In a perfect world, the “Old timers” would hang around to counsel the “Newbies”. But for many who have traveled the road from onset to remission, the challenges of PRP are memories to be forgotten. There is nothing wrong with bailing out. But if you do, please leave us with the following:

  Onset date: mm/yyyy
  Onset age
  Data of remission: mm/yyyy

Reasons to Stay Engaged

To Help
Stay engaged if you think you can help another PRP patient cope with the challenges of body, mind and spirit. Join the PRP Facebook Support Group and the PRP Community on RareConnect. It’s free

To Show Solidarity
One definition of solidarity is “unity or agreement of feeling or action, especially among individuals with a common interest; mutual support within a group.”

Epilogue

I served in the U.S. Marine Corps (circa 1968-1971) and have lived with the motto: Once a Marine, always a Marine. The same can be said of anyone diagnosed with PRP. We travel the same road but our journeys are never the same. We have looked in a mirror to see images we cannot believe. We lament that what works for one doesn’t work for all. Yet, we can find some solace in the fact that others have survived the journey.

If you opt-out, please accept my sincere wish that you enjoy more than your fair share of health and happiness.

Bill McCue
President/Founder
PRP Alliance, Inc.

 

 

The PRP Community on RareConnect

The PRP Alliance is working with RareConnect, an initiative of EURORDIS, the international equivalent of the National Organization of Rare Disorders. The PRP Alliance is a member of NORD and supports the PRP Community on RareConnect — a multi-language, non-Facebook option for adults with PRP and the parents of children diagnosed with juvenile onset.

RareConnect provides a “safe, easy to use platform where rare disease patients, families and patient organizations can develop online communities and conversations across continents and languages. RareConnect partners with the world’s leading rare disease patient groups to offer global online communities allowing people to connect around issues which affect them while living with a rare disease.”

Communities

RareConnect includes disease-specific online communities that enable people living with rare diseases to meet, share stories and learn from each other. Communities are created in partnership with patient groups who bring resources such as moderators, relationships with specialists and validated information.

Translation

RareConnect helps people who live with rare diseases from around the world to connect by providing different translation services. They provide an integrated machine-translation service but also an additional human translation service to ensure quality communication between members of the network. Languages include English, German, Spanish, French, Italian and Portuguese

Storytelling

Being able to share your story of living with a rare disease is an important step in connecting with others in a similar situation as well as allowing others to learn from our experience. RareConnect allows us to share our stories and translates it into the many languages of the platform therefore allowing people from many other cultures to benefit from it.

Connections

Connect with other people living with rare diseases in order to share experiences, find support, ask questions and gain access to a rich network of supportive patient organisations.

GO TO: https://www.rareconnect.org/en/community/pityriasis-rubra-pilaris

A Review on PRP

From the Editor — On January 4, 2018, a well-documented overview of pityriasis rubra pilaris written by Drs. Dingyuan Wang, Vanessa Cui-Lian Chong, Wei-Sheng Chong and Hazel H. Oon was published online by the American Journal of Dermatology. CAUTION: Section 7 on TREATMENT is worth a careful review by all PRP patients and caregivers.

This scientific article is not written for patients or non-medical caregivers. It should, however, be shared with dermatologists.Just ask: “Have you read the online Review on Pityriasis Rubra Pilaris in the American Journal of Dermatology?”

1   Introduction
2  Clinical Manifestations
3  Pathogenesis
4  Associated Conditions
5  Diagnosis
6  Differential Diagnosis
7  Treatment
8  Conclusion

A Review on PRP

What is “Core” Data”?

The following datapoints represent the “core” data collected via the PRP Worldwide Census. With this information we can build a more accurate profile of the PRP community.

  Elapsed time between onset and diagnosis
  Elapsed time between onset and remission
  Onset age by decade of life
  Misdiagnoses
  Biopsies as a diagnostic tool
  Survey PRP patients in remission
  Survey PRP children based n onset age

Name: Bill McCue
Even though we ask for the FIRST NAME and LAST NAME, we NEVER publish NAMES. WE ONLY report NUMBERS, e.g., How many “this” and how many “that”.

Location: Plano, TX
We are NOT looking for street addresses or ZIP Codes/Postal Codes)

Email: papa.bill@mac.com
The email address is the only way we can verify that the information we receive is from a valid source. We will send a confirming email to the email address of the person completing the PRP Census. If the email confirmation is undeliverable, the data will not be added to the database.

Onset Age: 66 years, 2 months
At what age did your PRP symptoms first appear?

Onset Date: August 8, 2012
What was the approximate date when your PRP symptoms first appeared? Because few people know the exact date Onset Date, only month and year is required, Do the best you can, e.g.,  August 8, 2012; August 2012, or even the “Summer of 2012”.

Misdiagnosis: seborrheic dermatitis
What misdiagnoses preceded your “official” PRP diagnosis? We are only concerned with the misdiagnoses of your dermatologist, not your GP. Options include psoriasis, eczema, seborrheic dermatitis, allergic reaction and other (specify)

Biopsy History: 3 biopsies inconclusive. 4th biopsy supported PRP
What role did biopsies play in your diagnosis of PRP?

Diagnosis Date: November 28, 2012
The Diagnosis Date should be as accurate as possible, e.g., November 28, 2012, November, 2012; end of November, 2012. Do the best you can.

Current Status: In remission since April 4, 2012 
Don’t worry about definitions. Given the choice between “Active” and “In Remission”, what is your current status? If “in remission”, what is your date of remission

Treatment Options (oral/injection): acitretin
All we want to know are the PRP-related drugs you were prescribed from the list retinoids, immunosuppressants and biologicals. Whether or not the drugs were successful will be the subject of a future survey. We are NOT collecting data on itch and pain relief, topical ointments, creams and lotions, sleep aids, etc. 

We are a global community. Each country has their own way of protecting a patient’s medical information. For purposes of the PRP Census, we comply with U.S. HIPAA regulations.  Even with assurances of confidentiality, data collection has never been an easy task.

Confidential Data – The data we collect about your unique PRP journey – and the data for all PRP patients (active and in remission) – is encrypted, password-protected, and not connected to the Internet.

De-identified Data – When shared with PRP researchers like those at Thomas Jefferson University and Yale University School of Medicine or published in the PRP Survival Guide, your data is de-identified. Simply stated, the PRP Community Database reports numbers, not names. Because your data is disassociated with your name, you have complete and total anonymity.
 

 

If you have any questions, Leave a Reply below. If not, your PRP Worldwide Census awaits. Click Here.

Patient Engagement Collaboration (PEC)

Editor’s Note: The following email was received from Andrea C. Furia-Helms, M.P.H., Acting Director, Patient Affairs Staff, Office of Medical Products and Tobacco, Office of the Commissioner, U.S. Food and Drug Administration.


As the FDA continues to advance its patient engagement efforts through its long-term commitment to improving and increasing patient involvement in FDA matters, I am pleased to announce a new initiative, the Patient Engagement Collaborative (PEC).  Today FDA published a Federal Register Notice to request nominations from representatives of patient communities to participate in an ongoing forum to discuss how to achieve more meaningful patient engagement in medical product development and other regulatory discussions.

The PEC will be coordinated by the FDA’s new Patient Affairs Staff (PAS) in the Office of Medical Products and Tobacco (OMPT) and the public-private partnership, the Clinical Trials Transformation Initiative.

The PEC will consist of a diverse group of representatives from the patient community including:

1.    Patients who have personal disease experience

2.    Caregivers who support patients, such as a parent, child, partner, other family member, or friend, and who have personal disease experience through this caregiver role

3.    Representatives from patient groups who, through their role in the patient group, have direct or indirect disease experience

For more information about the new Patient Engagement Collaborative, read today’s FDA Voice Blog and visit the Patient Affairs Staff webpage.


The FDA and the Clinical Trials Transformation Initiative (CTTI) will be working together to create a new work group with patient advocacy organizations to talk about patient engagement at the FDA.

As the FDA continues to advance its patient engagement efforts, it is important that an ongoing forum exists to discuss how to achieve more meaningful patient engagement in medical product development and other regulatory discussions.

Acknowledging the needs and concerns of patients, caregivers, and advocates, the FDA continues to identify ways to better serve the patient community.  One way the commitment is being addressed is through a new initiative. The FDA, together with the Clinical Trials Transformation Initiative,  are establishing a group of patient organization and individual representatives to discuss topics focusing on enhancing patient engagement in medical product development and regulatory discussions at FDA.  The Patient Engagement Collaborative (PEC) is another advancement in FDA’s efforts to strengthen its relationship with patient communities and is one of the activities that is being coordinated by the FDA’s Patient Affairs Staff (PAS)in the Office of Medical Products and Tobacco (OMPT).

Patient communities and other stakeholders commented in public feedback on Food and Drug Administration Safety and Innovation Act (FDASIA), section 1137, Patient Participation in Medical Product Discussions, to create an outside group to provide input on patient engagement across the agency.  In response to that feedback, FDA and CTTI established this Collaborative with help from patient organization representatives.

This new Collaborative is also facilitated by provisions in both the 21st Century Cures Act of 2016 and the Food and Drug Administration Reauthorization Act of 2017.  Both provisions aim to foster patient participation and incorporate patient experiences in the regulatory process.

FDA is seeking a group of diverse representatives from the patient community to participate in the PEC including:

✽  Patients who have personal disease experience.

✽  Caregivers who have personal experience with the disease by supporting the patient. A caregiver can be a parent, child, partner, family member, or friend.

✽  Representatives from patient groups who, through their role in the patient group, have direct or indirect disease experience.

The PEC will provide an ongoing forum to discuss how to achieve more meaningful patient engagement in medical product development and other regulatory discussions.  Topics to be discussed may include:

✽  making patient engagement more systematic

✽  how to improve transparency

✽  education and communication on the medical product regulatory process

✽  new strategies for enhancing patient engagement

✽  new models for patients to collaborate as partners in the medical product development and FDA review process.

Nominations are now being accepted

For more information about the Patient Engagement Collaborative email the Patient Affairs Staff-  PatientAffairs@fda.hhs.gov


Request for Nominations of Members for the Clinical Trials Transformation Initiative/Food and Drug Administration Patient Engagement Collaborative

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

SUMMARY:

The Food and Drug Administration (FDA or Agency), in collaboration with the Clinical Trials Transformation Initiative (CTTI), is requesting nominations of patient advocates interested in participating on the Patient Engagement Collaborative (PEC). The PEC will be an ongoing, collaborative forum coordinated through the Patient Affairs Staff, Office of Medical Products and Tobacco (OMPT), Office of the Commissioner, and will be hosted by CTTI. Through the PEC, the patient community and regulators will be able to discuss an array of topics regarding increasing meaningful patient engagement in medical product development and regulatory discussions at FDA. The activities of the PEC may include, but are not limited to, providing diverse perspectives on topics such as systematic patient engagement, transparency, and communication; providing considerations for implementing new strategies to enhance patient engagement at FDA; and proposing new models of collaboration in which patients and patient advocates are partners in certain aspects of the medical product development and FDA review process.

DATES:

Nominations received by 11:59 p.m. Eastern Time on or before January 29, 2018, will be given first consideration for membership on the PEC. Nominations received after the submission deadline will be retained for future consideration.

ADDRESSES:

All nominations should be submitted to the FDA’s Patient Affairs Staff in the OMPT. Email nominations are preferred and should be submitted to PatientEngagementCollaborative@fda.hhs.gov. Though not required, it is appreciated if all nomination materials are compiled into a single PDF file and attached to the submission email. Nominations may also be submitted by mail or delivery service to Patient Affairs Staff, Office of Medical Products and Tobacco, Office of the Commissioner, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 1, Rm. 1316, Silver Spring, MD 20993. Only complete applications, as described in section “IV. Nomination Process” of this document, will be considered.

FOR FURTHER INFORMATION CONTACT:

Andrea Furia-Helms, Office of Medical Products and Tobacco, Office of the Commissioner, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 1, Rm. 1316, Silver Spring, MD 20993, 301-796-8455, PatientEngagementCollaborative@fda.hhs.gov


SUPPLEMENTARY INFORMATION:

I. Background and Purpose

The CTTI is a public-private partnership co-founded by FDA and Duke University whose mission is to develop and drive adoption of practices that will increase the quality and efficiency of clinical trials. FDA and CTTI have long involved patients and considered patient perspectives in their work. Furthering the engagement of patients as valued partners across the medical product research and development continuum requires an open forum for patients and regulators to discuss and exchange ideas.

The PEC will be an ongoing, collaborative forum in which the patient community and regulators will discuss an array of topics regarding increasing patient engagement in medical product development and regulatory discussions at FDA. The PEC will be a joint endeavor between the CTTI and FDA. The activities of the PEC may inform relevant FDA and CTTI activities. The PEC is not intended to advise or otherwise direct the activities of either organization, and membership will not constitute employment by either organization.

The Food and Drug Administration Safety and Innovation Act (Pub. L. 112-144), section 1137, entitled “Patient Participation in Medical Product Discussions,” added section 569C to the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb-8c). This provision directs the Secretary of Health and Human Services to develop and implement strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions. On November 4, 2014, FDA issued a Federal Register notice establishing a docket (FDA-2014-N-1698) for public commenters to submit information related to FDA’s implementation of this provision (79 FR 65410). Upon review of the comments received, one common theme, among others, included establishing an external group to provide input on patient engagement strategies across FDA’s Centers.

Recent legislation in both section 3001 of the 21st Century Cures Act and section 605 of the Food and Drug Administration Reauthorization Act of 2017 supports tools for fostering patient participation in the regulatory process.

The purpose of this notice is to announce that the nomination process for the PEC is now open, and to invite and encourage nominations by the submission deadline for appropriately qualified individuals. Self-nominations are accepted.

II. Criteria for Membership

The PEC will include up to 16 diverse representatives of the patient community. Selected members will include the following: (1) Patients who have personal disease experience; (2) caregivers who support patients, such as a parent, child, partner, other family member, or friend, and who have personal disease experience through this caregiver role; and (3) representatives from patient groups who, through their role in the patient group, have direct or indirect disease experience. Please note that for purposes of this activity, the term “caregiver” is not intended to include individuals who are engaged in caregiving as health care professionals; and the term “patient group” is used herein to encompass patient advocacy Start Printed Page 60750organizations, disease advocacy organizations, voluntary health agencies, nonprofit research foundations, and public health organizations. The ultimate goal of the nomination and selection process is to identify individuals who can represent a collective patient voice for their patient community.

Selection criteria include the nominee’s potential to meaningfully contribute to the activities of the PEC, ability to represent and express the patient voice for his or her constituency, ability to work in a constructive manner with involved stakeholders, and understanding of the clinical research enterprise. Consideration will also be given to ensuring the PEC includes diverse perspectives and experiences, including but not limited to, sociodemographic and disease experience diversity. It is anticipated that approximately half of the PEC membership will be selected from eligible CTTI member organizations and individuals, and half will be selected from other nominees. Members are required to be citizens and residents of the United States.

Financial and other conflicts of interest will not necessarily make nominees ineligible for membership in the PEC. However, nominees cannot be direct employees of the medical product development industry.

III. Responsibilities and Expectations

Meetings of the PEC will typically be held four times per year, either in-person (in the Washington, DC area) or by webinar, and additional meetings may be organized as needed. Accommodations will be made for members with special needs for travel or for participation in a meeting (e.g., accommodations for physical mobility impairments, dietary restrictions, etc.). Nominations for PEC membership are encouraged for individuals of all racial, ethnic, sexual orientation, and cultural groups with and without disabilities. Travel support will be provided.

To help ensure continuity in its activities and organizational knowledge, the PEC will maintain staggered membership terms for patient community representatives. Membership terms are anticipated as 1- to 2-year appointments, and will be determined during the process of selecting members. Members may serve up to two terms, with the possibility of extensions.

Additional responsibilities and expectations are set forth in the Patient Engagement Collaborative Framework, which should be reviewed prior to submitting a nomination. The full text of the Patient Engagement Collaborative Framework is available at https://www.ctti-clinicaltrials.org/​framework-cttifda-patient-engagement-collaborative.

IV. Nomination Process

Any interested person may nominate one or more qualified individuals for membership on the PEC. Self-nominations are also accepted.

Nominations should include the following:

(1) A personal statement (maximum 800 words) from the nominee explaining his or her interest in becoming a member of the PEC;

(2) a current, complete curriculum vitae or resume that shows relevant activities and experience; and

(3) an optional letter of endorsement (maximum 800 words) from a patient group with which the nominee has worked closely on activities relevant to the PEC.

The personal statement and optional letter of endorsement (if provided) should emphasize information relevant to the criteria for membership described above. The letter may address topics such as the nominee’s involvement in patient advocacy activities, experiences that stimulated an interest in participating in discussions about patient engagement in medical product development and regulatory decision-making, and other information that may be helpful in evaluating the nominee’s qualifications as a potential member of the PEC.

Nominations must provide the nominee’s contact information (phone and email preferred), as well as state that the nominee is aware of the nomination (unless self-nominated) and is willing to serve as a member of the PEC.

Additional information may be needed from nominees, including information relevant to understanding potential sources of conflict of interest, in which case nominees will be contacted directly.

Dated: December 15, 2017.

Leslie Kux,
Associate Commissioner for Policy.

[FR Doc. 2017-27538 Filed 12-20-17; 8:45 am]; BILLING CODE 4164-01-P

Patient Engagement Collaboration (PEC)

PRP Community Resources

Message from the Editor

The onset of my version of PRP began in early August 2012 with the sudden appearance of a small, red spot on my right temple near my hairline. Within a week the “inconsequential” spot had grown in size — enough to prompt a call to my dermatologist.  By early September the side of my face, from my forehead to jaw, had been engulfed by red.

Initially misdiagnosed with seborrheic dermatitis, it took three months, the clinical observations of a more seasoned dermatologist, and a “supporting” biopsy before an “official”  diagnosis of PRP was rendered. My treatment plan was initiated in early December with slow-paced success. It took another seventeen months before I was med-free and symptom free.

Whether you are a PRP patient or caregiver, you are on a journey that few can understand. There is, however, a PRP community of fellow travelers who have faced the same challenges of body, mind and spirit that you face today. The journey from onset to remission should not be undertaken alone.

Bill McCue


Over the past five years there has been a dramatic expansion of resources available to support PRP patients and their caregivers. The PRP community must become more proactive in building awareness of these resources during 2018among two constituencies:

✽  PRP patients and caregivers

✽  Healthcare professionals responsible for the proper treatment of PRP

As a PRP patient or caregiver it is critical that you understand these resources and share there availability with your own dermatologist, general practitioner, podiatrist, opthamologist, ENT specialist, mental health specialist, etc.

1.  Genetic and Rare Diseases Inforation Center  GARD, a program of the National Center for Advancing Translational Sciences, is funded by the National Institutes of Health. The office supports a variety of programs to serve the needs of patients who have any one of approximately 7,000 rare diseases known today. Their rare disease database includes information on each of 597 rare skin diseases including a recently updated overview of PRP. This is a “Must Read” for every PRP patient and caregiver.

❏  Read and share the GARD PRP Report

2.  National Organization of Rare Disorders     On September 20, 2017, NORD replaced the 1,500-word PRP Report (circa 2007) with a significantly more comprehensive, 5,000-word  2017 revision developed by the PRP Community. This overview is written in lay language and a “Must Read” for every PRP patient and caregiver.

❏  Read and share the NORD PRP Report 

3.  PRP Survival Guide     Since its launch in 2013, this ever-expanding repository of information now includes over 400 PRP-specific articles written in lay language on topics specifically related to the diagnosis and treatment of PRP as reported by PRP patients and their caregivers.

❏  Read “How to use the PRP Survival Guide”

4.  PRP Facebook Support Group     Founded in April 2013 by a PRP patient from British Columbia, Canada, membership in this Closed Group has grown steadily. Today over 1,170 PRP patients and caregivers ask questions, share our PRP-related experiences and provide insights only a fellow sufferer can offer. Our mantra: what works for one doesn’t work for all. We are in this together.

❏  Join the PRP Facebook Support Group

5.  PRP Community in Rare Connect     Established in April 2015 as an initiative of EURORDIS, the international equivalent of the National Organization of Rare Disorders. the PRP Community on RareConnect is a 200+ member, multi-language, non-Facebook option serving adults with PRP and the parents of children diagnosed with juvenile onset.

❏  Join the PRP Community on RareConnect

6.  PRP Alliance     From a domain name registered in February 2013, the PRP Alliance has evolved into a 501(c)(3), nonprofit, patient advocacy organization with a mission is to advocate the timely and accurate diagnosis of pityriasis rubra pilaris (PRP), the implementation of more effective and accessible treatment options, and an increase in PRP-specific research.

❏  Learn more about the PRP Alliance

On November 1, 2017, as part of the annual observance of PRP Awareness Month, the PRP Alliance executed mailing to the dermatology departments of 88 teaching hospitals in the U.S. This was the first salvo in a year-long Rare Skin Disease Referral Initiative seeking to link PRP patients with the PRP resources listed above.

The Last Word

PRP patients and caregivers who join the PRP community discover that they are not alone. YOU are not alone. WE are on this journey together.

Bill McCue, Editor PRP Survival Guide 1500 Commerce Drive Plano, TX 75093-2640 Email: bill.mccue@prpSurvivalGuide.org Website: prpSurvivalGuide.org

 

2018 Path Forward