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Participants:
Ed: PRSN Moderator
Mary Dunkle - NORD
Sandy: ; PAN Group Member
Jessica: ; PAN Group Member
Travis: PAN Group Member
Don and Barb: PAN Group Members
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Ed Moderator: I'm hoping we'll have some guests tonight, usually have a few but if not no worries. We'll have a chat and the transcript will go out about five more minutes if that's okay.
Mary Dunkle: ; Sure, I lived in State College, Pa, for 11 years. How many people are in the Network?
Ed Moderator: 527 currently - mostly patients, next would be caregivers then docs and researchers.
Mary Dunkle: ; Wow...big. International?
Ed Moderator: oh yes, world-wide. It's what I call a Kitchen Table group.
Mary Dunkle: ; Right but maybe ready to go to the next step.
Ed Moderator: world wide reach but really small in terms of budget, etc. Okay, welcome Sandygplayer. Can you give us a first name?
Sandy: ; Sandy
Ed Moderator: We will start in one minute, promise. Sandy, great. Where are you from Sandy
Sandy: ; West Chester, Ohio
Ed Moderator: and are you new to the PAN network? First time chat? Are you a PAN patient?
Sandy: ; I have vasculitis and am going to Cleveland Clinic in April to see Dr. Langford for more info.
Ed Moderator: you just joined the PAN network, Sandy?
Sandy: ; I have been posting and watching for about a month.
Ed Moderator: Okay, we'll get started. Welcome again. Thanks for coming. Hello Jessica!! Where are you from Jessica?
Jessica: ; Pittsburgh.
Ed Moderator: A fellow Pittsburgher. Cool. Okay, well, we're going to get started...let me say a few things first. We have Mary Dunkle as our guest tonight. The first 20 min or so will just be a Q and A between us. Hello Barb and Don!! So please hold your questions until we're done and then I'll open to everyone to ask. When you want to ask a question please type a !. This is a raised hand. I'll call on you. When you're done with your thoughts, type `#' so we know you're finished. Forget grammar and spelling, it gets cleaned up in the transcript. Away we go. Mary is from NORD. First of all Mary let's learn about you. Tell us a bit about your background and when you came to NORD and what you do there.
Mary Dunkle: ; I've been with NORD 10 years. Was a newspaper reporter originally and then worked for Penn State university writing about research.
At NORD I'm in charge of website, publications communications.
Ed Moderator: Thanks. So your background is research and journalism?
Mary Dunkle: ; Yeah. Mostly writing about technical things in ways all can understand.
Ed Moderator: You should get paid a million dollars for that..:-) Can they hire you to do the stimulus bill? - Just kidding.
Mary Dunkle: ; Don't always succeed...but try.
Ed Moderator: Okay, so let's talk about NORD. What is it? What does it do?
How does it help the rare disease patients?
Mary Dunkle: ; NORD is 25 years old...was started by leaders of patient groups. It provides information, referrals to patient organizations and other resources. We have a nurse and genetic counselor on staff people can email or call. We have patient assistance programs for people who can't afford certain meds, a small program of research grants and fellowships and provide advocacy on public policy on behalf of all patients and families affected by rare diseases.
Ed Moderator: Let's actually back up just for a moment to get perspective.
What is definition or classification of a rare disease?
Mary Dunkle: ; affects fewer than 200,000 Americans.
Ed Moderator: And it encompasses autoimmune, and all other types of disorders.
Mary Dunkle: ; right. There are between 6000 and 7000, according to NIH.
Ed Moderator: How many rare diseases does NORD represent?
Mary Dunkle: ; All.
Ed Moderator: wow... Okay, well, at this point I'll just go over some of the important, key resources you provide.
Mary Dunkle: ; We refer to disease-specific orgs if there is one. But we serve as support group if no organization for a disease.
Ed Moderator: just give a little more info on exactly what the program does. I see...so the number would continue to grow. as new ones are found.
Mary Dunkle: ; Right.
Ed Moderator: First, tell us about your database of diseases. What it is? What does it gives to the reader? I'm just picking some of the areas of your website of particular interest. Maybe just flesh it out a bit, thanks.
Mary Dunkle: ; We have database of reports on 1,200 rare diseases...gives basic intro, and refers to disease-specific orgs for more. Tells things like clinical trials, resources, etc.
Ed Moderator: I see. So any patient can go in and access this database, or is there a cost?
Mary Dunkle: ; Patient can get abstract free, which includes resources and patient organizations. Charge of $7.50 for longer version...but many libraries subscribe, so it's free at those libraries. Also, people can call our office and get one free report.
Ed Moderator: So you would go online via your local library to access?
Mary Dunkle: ; Yes. Or medical libraries in hospitals, universities, etc. It's also on WebMd and some other websites. Or people can send questions to our nurse or genetic counselor.
Ed Moderator: thank you. Tell us more about the Medication assistance Program?
Mary Dunkle: ; About 40 of these programs. Some provide free drug to uninsured people. Some provide insurance premium or co-pay assistance. Some provide early access to investigational drugs. Some travel assistance to get to clinical trials.
Ed Moderator: Patients with the rare diseases that you have in NORD, correct?
Mary Dunkle: ; We have programs for about 40 drugs at this time...changes from time to time. Current ones are listed on our website. Also, we provide medical foods for PKU.
Ed Moderator: I just wanted to clarify....this is drug assistance for people with rare diseases specifically.
Mary Dunkle: ; Yes
Ed Moderator: So best advice is, if you want to know if qualify, to website and follow link.
Mary Dunkle: ; Right. Click on Programs & Services to see current programs and contacts.
Ed Moderator: Note: I'll turn over to questions in a few mins.
Ed Moderator: So tell me...what are some of the significant things NORD has done for the Rare Disease community.
Mary Dunkle: ; NORD was founded by leaders of patient groups who helped get Orphan Drug Act enacted.
Ed Moderator: Specifically the ORPHAN act:-)
Mary Dunkle: ; it helped get Rare Diseases Act of 2002 passed. That was what created the NIH RDCRN which has Vasculitis Consortium.
Ed Moderator: What is the significance of Rare Disease Act. Was it to help get on the radar of Washington D.C.?
Ed Moderator: Oh right...exactly. It launched all of these clinical studies.
Mary Dunkle: ; it included specific provision for funding for rare disease research.
Ed Moderator: I see...so the number would continue to grow, as new ones are found.
Mary Dunkle: ; Each year, at budget time, our advocacy person speaks on behalf of rare disease research. NORD also provided advocacy for clinicaltrials.gov website. Hard to remember now but years ago, patients didn't have access to much information about research, they couldn't find clinical trials.
Ed Moderator: Yes, excellent point. Question---so in an economy that is tanking...but a new administration.. What do you see in terms of attention, funding on rare disease research and NORD specifically?
Mary Dunkle: ; Wow, it's so hard to predict, so many variables.
Ed Moderator: sure...understood.
Mary Dunkle: ; We would like to see healthcare reform. There are too many people without insurance. Applications for our patient assistance programs have skyrocketed this year.
Ed Moderator: Just curious if you think more or less monies will be directed to rare disease research or is it just too early to say.
Mary Dunkle: ; I am hopeful for more but hard to say. There are so many competing needs.
Ed Moderator: Understood. Well, let me turn it over to the group. But I just want to say, please go to the website. There is so much there, more than we can talk about here. I went there today and it blew me away how much you provide in terms of resources to the rare disease patients. So, questions for Mary. Raise your hand, please!!
Don and Barb: !
Ed Moderator: go ahead Barb and/or Don.
Don and Barb: Where do you see your group and patients of rare diseases in a universal healthcare system in the US? Don asking barb typing... :)
Ed Moderator: good question.. Mary? How would universal healthcare be better or worse for the rare disease patient.
Mary Dunkle: ; Would love to see all insured but also see some problems in system like Canada...patients write to us with frustrations.
Ed Moderator: Welcome Travis. Glad you made it. I guess, a rare disease patient brings such unique challenges to any healthcare system.
Mary Dunkle: ; One thing we're seeing a lot now is insurers refusing to pay for off-label treatment. But a lot of people with rare diseases are treated off-label because there's no approved treatment.
Travis: I off to see Dr Carette on Wednesday for a follow up been having a hard time as of late with bowel and infection
Ed Moderator: He's a good guy, Travis! Okay, Jessica, you can ask away to Mary.
Jessica: ; My question is somewhat irrelevant though. Are there any college scholarships for people with rare diseases? I'm having trouble finding them and money would be nice.
Ed Moderator: Ah, very good question! Mary?
Mary Dunkle: ; That's not irrelevant at all...we get that question a lot...one of my colleagues has made a list of resources. If you send me an email tomorrow I'll send it to you.
Jessica: ; What's your email?
Mary Dunkle: ; mdunkle@rarediseases.org
Jessica: ; thanks
Mary Dunkle: ; sure
Ed Moderator: Thank you..Great question, Jessica. I mean there should be some assistance for the rare disease patients.
Jessica: ; hopefully I get some cash to learn some things :]
Mary Dunkle: ; there are some resources that may help.
Ed Moderator: Bob and Don, go ahead. I see your arm falling asleep!
Don and Barb: hahahah thanks Ed. We have looked into participating in clinical trials, but do not qualify for the angel flight assistance...is there any way that the mayo clinic can bring clinical trials here to Arizona?
Ed Moderator: Mary...what are your thoughts on this. Does NORD have patient travel assistance?
Mary Dunkle: ; What did angel flight say?
Don and Barb: We fall under the criteria of being more than 1000 miles away and they will only fly you 1000 miles or less.
Mary Dunkle: ; Oh, have you talked to anyone at Mayo about it?
Don and Barb: No, not at mayo.
Ed Moderator: When you're done Mary I'll share my two cents. I recently talked to Marietta with Angel Flight who has some news.
Mary Dunkle: ; Sometimes the commercial airlines will help.
Travis: Well, Dr Carette told me of all his patients with PAN I am the only one having a flare for some reason I do not feel special.
Ed Moderator: Actually you got it, Mary. Maybe it was the recent CPAG call we were on, less private pilots are flying due to costs of gas. So, they are working to book more people on commercial flights. Barb and Don, please contact me after the chat and I'll talk to you some more. eob44@zoominternet.net Any other questions for Mary in the time remaining? Going once...
Don and Barb: No, she has been great
Mary Dunkle: ; thanks for having me.
Don and Barb: Mary, thank you for all you do for patients with rare diseases
Ed Moderator: going twice. Yes, wonderful to have you here. We didn't even touch on Rare Disease Day ell us a bit about it. It was yesterday, correct? What was the purpose?
Mary Dunkle: ; Yes, it was first time in U.S.
Jessica: ; there's a day for us?
Mary Dunkle: ; people all over the country participated
Jessica: ; What do you do during it?
Mary Dunkle: ; Purpose is to get rare diseases on radar screen as a public health issue. A lot of it was online...videos, blogs, etc. People got their governors to declare Rare Disease Day in their state.
Ed Moderator: Very cool. And there will be another next year I guess!!
Jessica: ; So it's not really like a cake and fun hats kind of event?
Mary Dunkle: ; No...We didn't want to make people travel or spend money. We just wanted to do things they could do at home.
Ed Moderator: Well, as I say we have to have you back again Mary to share more. All I can tell you all is to check out NORD site
Don and Barb: It still gets the word out...
Mary Dunkle: ; Thanks. I enjoyed this
Ed Moderator: Thank you for the time and its okay if people contact you with questions, right?
Mary Dunkle: ; Sure
