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.

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

                 

Rare Skin Disease Referral Initiative Update

What is the Basic Problem to be Solved?

When a patient is diagnosed with pityriasis rubra pilaris (PRP), the dermatologist — with very rare exceptions — does not refer their patient to the PRP Alliance, PRP Survival Guide, PRP Facebook Support Group, or the PRP Community on RareConnect. Typically, the patient is referred to the internet where search engines (Google, Yahoo) retrieve information that can be redundant, insufficient, outdated, and written for an audience of healthcare professionals— not patients and their caregivers.

With an estimated 574 rare skin diseases inflicting pain and suffering, it is absolutely unreasonable to expect healthcare professionals to keep track of disease-specific patient support organizations and other patient-centric resources for rare diseases like PRP.

What is the Solution?

The solution is actually quite simple. When a patient is diagnosed with a rare skin disease (less than 200,000 effected patients in the US), the standard therapy should include two referrals that are recorded in the patient’s electronic medical record:

✽  Genetic and Rare Diseases Information Center (GARD)
✽  National Organization of Rare Disorders (NORD)

What Do GARD and NORD Accomplish?

Reliable information about rare or genetic diseases is often hard to find. GARD and NORD offer patients with a rare skin disease — and their caregivers — a reliable roadmap to the resources they need.

✽  GARD website

✽  GARD Rare Disease Database  (SEE GARD PRP Report, Revised 11/2017)

✽  GARD Patient, Families and Friends

✽  GARD Information Specialists — Whether by phone, fax or email, the public has access to current, reliable, and easy-to-understand information in English or Spanish. Toll-free Telephone: 888 205-2311; Email: GARDinfo@NIH.gov/

✽  NORD Website

✽  NORD Rare Disease Database — SEE NORD PRP Report

Step One — Jump Start the Campaign

The Rare Skin Disease Referral Initiative is a year-long campaign that began with the mailing of an “Advocacy Packet” to the following:

TEACHING HOSPITALS

✽  Leadership (Chairs) of the dermatology departments at 88 teaching hospitals in the U.S.

The first salvo of “advocacy packets” was funded by members of the the PRP Facebook Support Group and included the following components. Click the link to see what recipients received.

✽  Cover Letter
✽  Factsheet
✽  GARD brochure
✽  NORD PRP Report — reprinted with permission fs

Step Two — AAD Annual Meeting

The next step in the promotion of the Rare Skin Disease Referral Initiative is to take full advantage of the American Academy of Dermatology annual meeting to be held in San Diego, CA. (February 16-19). The PRP Alliance will be attending the four-day event as a member of the Coalition of Skin Diseases with access to dermatologists, dermatology physician assistants and dermatology nurses.

ORGANIZATIONS

✽  American Academy of Dermatology
✽  Society of Dermatology Physician Assistants
✽  Dermatology Nurses’ Association

MEDIA

✽  Dermatology Times
✽  The Dermatologist
✽  Dermatology News

EXHIBITORS

Three hundred–plus exhibitors who sell products and services to dermatologists for patient care represent an opportunity for the PRP Alliance to recruit sponsors for the 2018 Rare Skin Disease Referral Initiative Campaign.

Step Three — The Hard Work of Advocacy

The third step (March-October, 2018) depends on who will join the effort.

✽  Will it be patients and caregivers who tell their dermatologists about GARD and NORD?
✽  Will AAD, DNA and SDPA tell their members?
✽  Will Dermatology Times, The Dermatologist and Dermatology News publish articles about the Rare Skin Disease Referral Initiative?

For the moment, we will focus on Step 2 and see what can make happen in San Diego.

02/09/2018: Rare Skin Disease Referral Initiative Update

 

Thank you for subscribing…

It was Thursday, October 1, 2015 when I posted the 24th and final issue of On the Road… on the PRP Alliance website. After writing 598 pages of “PRP stuff” I was ready to shift my focus to the PRP Survival Guide. No more self-imposed, end-of-the-month deadlines for me. Besides, I was in remission.

It has been over two years and I am still in remission — but eager to publish a newsletter for the PRP community.

I urge every PRP patient and caregiver who subscribes to On the Road…  to take the time to read what we publish and share your personal insights. PRP patients and caregivers live with PRP day in and day out. You are the subject matter expert for your version of PRP. We need to tell all our stories.

Bill McCue, Editor

PRP Outreach to US Teaching Hospitals

 

From the Editors: Most PRP patients and caregivers are given very little guidance after their PRP diagnosis is rendered. Referrals to Dr. Google and Dr. Yahoo are quite standard, Balderdash! The PRP Alliance has self-financed an outreach effort to 87 of the dermatology departments of our nation’s teaching hospitals advocating a standard referral to GARD and NORD.

What’s standard treatment protocol?

When a dermatologist diagnoses a rare skin disease, the patient is prescribed an appropriate therapy. Predictably, the initial treatment plan will include systemic and topical therapies as appropriate. However, there’s something that most, if not all patients are missing: a referral to the Genetic and Rare Diseases Information Center (GARD).  

Without a rare skin disease referral to GARD…

A newly diagnosed patient leaves the dermatology clinic with a treatment plan, prescriptions to fill at the local pharmacy, the required paperwork and an appointment to return at a later date. For patient and caregivers, their journey has begun with little more than the name of the rare skin disease and a tacit referral to Dr. Google and Dr. Yahoo. As questions arise in the days and weeks that follow, the information unearthed via search engines is too often redundant, insufficient, outdated, and typically written for an audience of healthcare professionals.

What happens with a referral to GARD?

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.

Who can GARD help?

✽  Your patients diagnosed with a rare skin diseases
✽  The people who care for your patients: parents, family members, and friends
✽  Doctors, nurses, and other healthcare providers
✽  Scientists who are studying rare skin diseases and need information for their research

How will PRP patients and caregivers will find what they seek?

Reliable information about rare or genetic diseases is often hard to find. Your patients will have a roadmap to the resources they need from this dependable source.

GARD website
PRP patients and caregivers have online access to reliable information about their newly diagnosed rare disease to include symptoms, cause, diagnosis, treatment, research, daily life and support organizations.

GARD “Learn More” Opportunities
Links to resources where patients can find more information, e.g. the National Organization of Rare Disorders.

GARD Information Specialists
Whether by phone, fax or email, the public has access to current, reliable, and easy-to-understand information in English or Spanish.

✽  Toll-free Telephone: 888 205-2311
✽  Email:  GARDinfo@NIH.gov/

Scope

The target audience for the Rare Disease Awareness Initiative includes “leaders” at teaching hospitals in the U.S. with a dermatology department, e.g., Department Chair. We will also reach out to leadership in the following:

✽  American Academy of Dermatology
✽  Society of Dermatology Physician Assistants
✽  Dermatology Nurses’ Association

Methodology

The Rare Disease Awareness Initiative is organized around a “Referral Packet” containing the following:

✽  Cover letter
✽  PRP Referral Protocol
✽  GARD brochure
✽  NORD PRP Report (16 pages)

Who to thank

✽  The Genetic and Rare Diseases Information Center for the services they routinely provide as well as brochures for the 100 “Referral Packets” 
✽  The National Organization for Rare Disorders for allowing the PRP Community to take a leadership role in rewriting the NORD PRP Report 
✽  A dozen members of PRP Facebook Support Group who donated $1,100 to make the Rare Disease Awareness Initiative a reality, e.g., printing, postage, etc.

 

PRP Outreach to US Teaching Hospitals: OTR025.3 

PRP Awareness Month Begins Today


November 1, 2017
Every patient organization needs an Awareness Day they can call their own. We celebrate Rare Disease Day on the last day of February and share the spotlight with 7,000 other rare diseases. However, PRP Awareness Month gives us 30 days to crow like a banty rooster, shake our tail feathers and get more than our fair share of attention. — Bill McCue

My first PRP Awareness Day was November 28, 2012 and quite “unofficial” by any standards. It began with a phone call from my dermatologist’s office. I was told that the red spot on my forehead on August 8 — nearly four months prior — had been the onset of pityriasis rubra pilaris. Like most PRP “newbies”, I spent the next few days searching the internet and learning what little there was to learn about PRP. What I found was redundant, insufficient, outdated, and typically written for an audience of healthcare professionals.

On November 1, 2013, I celebrated PRP Awareness Day once again. This time I sent out over 1,500 emails to PRP patients and caregivers and called the effort the PRP Worldwide Census. It had taken me several months during the previous summer to review over 29,000 emails in the PRP Support Group Archive going back to November, 1997.  As I  collected email addresses I also harvested “core” information about the onset, diagnosis, and treatment of PRP. It took six months to solicit 500 responses. That was the genesis of the PRP Community Database.

November 6, 2014 was the first “official” observance of PRP Awareness Day. The date was selected to commemorate the signing of the Rare Diseases Act of 2002 that set the threshold for a rare disease. A rare disease or disorder is defined in the U.S. as one affecting fewer than 200,000 Americans. Using the European standard, however, the U.S. threshold would be only 160,000. Today there are more than 7,000 known rare diseases, and it is estimated that about 30 million Americans are affected by them.

November 6-8, 2015 was the first “official” observance of PRP Awareness Weekend since the 6th fell on a Friday. The “Big Event” would be a global  Meet & Greet. Whether face to face or using technology (FaceTime, Skype and ooVoo) we pitched the idea to the PRP Facebook Support Group.  Other than a few face-to-face Meet & Greets and a spike in Facetime encounters, the effort missed it’s mark. Lesson learned.

November 1-30, 2016 was the first “official” observance of PRP Awareness Month.  Perhaps all we needed was 30 days rather than a weekend or a day. Sunday, November 6th was selected to host a 24-hour GoToMeeting gathering. Only the predictable and dependable cadre of seasoned PRP travelers stopped by to chat.


An important lesson has been learned over the past five years. If you run the same flag up the flagpole, the same people are going to salute. We’re going to try something different.

  ✔︎  Revive the PRP Community newsletter
  ✔︎  Launch the PRP Worldwide Census
  ✔︎  Execute the Rare Skin Disease Referral Initiative
  ✔︎  Initiate PRP Surveys
  ✔︎  Recruit participants for PRP research
  ✔︎  Promote legislative advocacy
  ✔︎  Support the PRP Alliance.

 

 

PRP Awareness Month Begins Today: OTR025.1

Thank You For Being Counted

 

I have been collecting PRP-related information from PRP patients and their caregivers since November, 2013. In the beginning it could take six requests before I received a completed PRP census form.

On the 1st and 15th of ever month the NUMBERS will be posted here.  The list will show the November 1, 2017 numbers and the most current numbers.

The numbers below are based on the “core data” shared by PRP patients and caregivers worldwide. The PRP Community Database is the most complete representation of our patient population. Thank you for sharing your information.

Bill McCue, Founder/President
PRP Alliance, Inc.

 


Recap as of November 1, 2017

Numbers in parentheses ( ) indicate “not providing data”

✽  Total number of PRP patients reporting:   1,629

✽  Reporting email addresses:   1,033 (596)

✽  Reporting location:   1,140 (489)

✽  Reporting onset date:  715 (914)

✽  Reporting onset age:  651 (978)

✽  Reporting diagnosis date: 694 (935)

✽  Reporting current status: 656 (973)

✽  Active:  394

✽  Remission: 262

✽  Remission date: 64 (198)

✽  Reporting standard drug therapies: 67 (1,562)