WEBINAR PROGRAM 4: “Put Me In, Coach”

Screen shot 2013-09-19 at 7.08.10 PMDate Recorded: Thursday, Sept. 19 at 2 p.m. (Eastern)

Speakers: Monica Gow, Julie Flygare & Kim McCleary

Description: Our coaching team will help people planning to participate in the Sept. 24 FDA meeting to be prepared for the format and ways they’ll be invited to contribute their perspectives. We’ll review the format and the purpose for the meeting and ways FDA staff will help elicit the similarities and important differences among patients.

Watch recording: http://youtu.be/lC01OeIAORo

Meeting Agenda

View the agenda for the FDA Narcolepsy Public Meeting on Patient-Focused Drug Development, taking place on Sept. 24, 2013 at FDA’s Campus in Silver Spring, Maryland. Learn more here.

FDA Narcolepsy Public Meeting Agenda

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12:00 – 1:00 pm Registration

1:00 – 1:05 pm Welcome Soujanya Giambone, MBA Office of Strategic Programs (OSP), Center for Drug Evaluation and Research (CDER), FDA

1:05 – 1:10 pm Opening Remarks Ellis Unger, MD Director, Office of Drug Evaluation I, CDER, FDA

1:10 – 1:20 pm Overview of FDA’s Patient-Focused Drug Development Initiative Theresa Mullin, PhD Director, OSP, CDER, FDA

1:20 – 1:30 pm Background on Narcolepsy and Therapeutic Options Ronald Farkas, MD, PhD Lead Medical Officer, Division of Neurology Products, CDER, FDA

1:30 – 1:40 pm Overview of Discussion Format Soujanya Giambone, MBA OSP, CDER, FDA

1:40 – 2:10 pm Panel #1 Comments on Topic 1 Topic 1: Patients’ perspectives on disease symptoms and daily impacts that matter the most. A panel of patients and patient representatives will provide comments to start the discussion.

2:10 – 2:55 pm Large-Group Facilitated Discussion on Topic 1 Patients and patient representatives in the audience are invited to add to the dialogue.

2:55 – 3:10 pm Break

3:10 – 3:40 pm Panel #2 Comments on Topic 2 Topic 2: Patients’ perspectives on current approaches to treating narcolepsy

3:40 – 4:25 pm Large-Group Facilitated Discussion: Topic 2

4:25 – 4:55 pm Open Public Comment

4:55 – 5:00 pm Closing Remarks Eric Bastings, MD Acting Director, Division of Neurology Products, CDER, FDA

Discussion Questions

Topic 1: Disease symptoms and daily impacts that matter most to patients

1) Of all the symptoms that you experience because of your condition, which 1-3 symptoms have the most significant impact on your life? (Examples may include excessive daytime sleepiness, cataplexy, etc.)

2) Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition? (Examples of activities may include sleeping through the night, work and school performance, etc.)

3) How have your symptoms changed over time?

a) Do your symptoms come and go? If so, do you know of anything that makes your symptoms better? Worse?

Topic 2: Patients’ perspectives on current approaches to treating narcolepsy

1) What are you currently doing to help treat your condition or its symptoms? (Examples may include FDA-approved medicines, over-the-counter products, and other therapies including non-drug therapies such as lifestyle modifications.)

a) What specific symptoms do your therapies address?

b) How has your treatment regimen changed over time, and why?

2) How well does your current treatment regimen treat the most significant symptoms of your disease?

a) How well do these therapies improve your ability to do specific activities that are important to you in your daily life?

b) How well have these therapies worked for you as your condition has changed over time?

3) What are the most significant downsides to your current therapies, and how do they affect your daily life? (Examples of downsides may include bothersome side effects, inconvenient dosing schedules, access issues, etc.)

4) Assuming there is no complete cure for your condition, what specific things would you look for in an ideal therapy for your condition?

For more information, please visit the FDA website: http://www.fda.gov/forindustry/userfees/prescriptiondruguserfee/ucm359018.htm

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FDA’s Questions to Narcolepsy Community & Step-by-Step Instructions

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FDA is interested in receiving patient input addressing these questions through the public docket: http://www.regulations.gov/ – !submitComment;D=FDA-2013-N-0815-0001.

Step-by-step instructions for submitting your answers follow the questions (reprinted from FDA).

Topic 1: Disease symptoms and daily impacts that matter most to patients

1.  Of all the symptoms that you experience because of your condition, which one to three symptoms have the most significant impact on your life? (Examples may include excessive daytime sleepiness, cataplexy, etc.)

2.  Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition? (Examples of activities may include sleeping through the night, work and school performance, etc.)

3.  How have your symptoms changed over time?

3.1  Do your symptoms come and go? If so, do you know of anything that makes your symptoms better? Worse?

Topic 2: Patients’ perspectives on current approaches to treating narcolepsy

1. What are you currently doing to help treat your condition or its symptoms? (Examples may include FDA-approved medicines, over-the-counter products, and other therapies including non-drug therapies such as lifestyle modifications.)

1.1 What specific symptoms do your therapies address?

1.2 How has your treatment regimen changed over time, and why?

2.  How well does your current treatment regimen treat the most significant symptoms of your disease?

2.1  How well do these therapies improve your ability to do specific activities that are important to you in your daily life?

2.2  How well have these therapies worked for you as your condition has changed over time?

3. What are the most significant downsides to your current therapies, and how do  they affect your daily life? (Examples of downsides may include bothersome side effects, inconvenient dosing schedules, access issues, etc.)

4.   Assuming there is no complete cure for your condition, what specific things would you look for in an ideal therapy for your condition?

DocketDirections for submitting comments to FDA Docket

1. Review the FDA Questions above and type your answers in a separate Word document.  Remember that you don’t have to answer all the questions if you don’t want to. You also don’t have to use complete sentences or full paragraphs. (Consider sharing your answers with a loved one to make sure your writing conveys your authentic experience accurately and concisely.) Save your answers as a separate Word .doc on your computer. Use the “Word Count” function on the “Review” tab of Word to count the characters. You can also submit your answers by video. Use a cell phone or webcam to record a video and save it as a .wmv file or upload to a video sharing site (such as YouTube). You can send the file or a link to it to FDA just as you would a document (see below).

2. When ready to submit comments to FDA, go to: http://www.regulations.gov/ – !submitComment;D=FDA-2013-N-0815-0001.

3. Comment Box – This box is limited to 5000 characters. If your answers are less than 5000 characters, copy and paste answers into the docket window. If your answers total more than 5000 characters, write “Please see attached comments” and attach your comments by uploading your saved Word .doc file or a PDF document.

4. Enter First and Last Name – If you choose to use your name, it may be included in a public docket and on the Internet.  If you’d like to remain anonymous, fill in the name fields as “Anonymous” and be sure not to list your name anywhere in your comment or attached letter.

5. Check or uncheck “I want to provide my contact information” – this is up to you.

6. If you are submitting comments for yourself, uncheck “I am submitting on behalf of a third party.” If you are submitting comments on behalf of another person, leave this box checked.

7. Category (Required): Choose INDIVIDUAL CONSUMER

8. Continue to Preview Page: Preview your submission and check “I read and understand the statement above.”

9. Submit Comments.

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WEBINAR PROGRAM 3: Harmony – Putting Your Best Voice Forward

Date Recorded: Wednesday, Sept. 11 at 2 p.m. (Eastern)Screen shot 2013-09-11 at 3.56.38 PM

Speakers: Julie Flygare & Kim McCleary

Description: Whether it’s barbershop or blues, when vocalists bring their unique sound into harmony with others, it’s magic. The same is true of advocacy. We’ll review the purpose of the FDA’s invitation for patient testimony and cover best practices for communication and advocacy in this and other policy settings. The tools we’ll cover can be applied to advocacy within the healthcare system, appropriate educational settings and more.

Watch recording: http://youtu.be/XVJR0TZPd0M

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What to expect when attending the FDA Narcolepsy Meeting in Person

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Arriving at FDA:
Arriving at FDA’s White Oak campus may be overwhelming the first time. Please leave lots of extra time to make the trip. Whether you are planning to drive or take public transportation, please factor in extra time for traffic, parking, shuttle buses and security check-in. If you’re flying into the Washington, D.C., metropolitan area, please see the end of this document for additional travel tips.

The meeting will take place at the FDA White Oak Campus at 10903 New Hampshire Ave., Building 31 Conference Center, Section A of the Great Room (Rm. 1503), Silver Spring, MD 20993.

Parking:
All visitors should park in the SE Quadrant Surface Lot. Proceed to the first stop sign and turn right on Southwest Loop Road. Follow Southwest Loop Road all the way around, passing the SW Garage on your left. Continue driving, enter surface parking lot on your left. Once parked, walk or take the Campus Circulator Shuttle which will service the SE Quadrant Surface Lot and take visitors to Building 1. See map attached.

Public Transportation:
Silver Spring Metro Station, on the Red Line, is the closest metro stop. The Silver Spring Metro Station is approximately 4 miles, a 20 minute cab ride, from the White Oak Campus. Ride-On Bus route 22 operates between the Silver Spring Metro Station and White Oak Campus. Additionally, C8 Metro Bus operates between College Park and White Flint and makes a stop at the FDA White Oak Campus. For more information and bus schedules, visit: http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241743.htm.

Security:
All participants must enter though Building 1 where they will go through the routine security check procedures for building entry. A visitor badge and escort will not be required to attend a public meeting in the Great Room. You will may have to go through a screening process similar to airport security. Please bring a photo ID for the security check-in.

Food/Drinks:
Please feel free to bring coffee, drinks and snacks with you to the meeting. There is a refreshment stand near the meeting space with light refreshments and coffee for purchase. However, there is no other on-campus dining for visitors. Name Tags: There will be name tags outside the room, where you can list your full name, first name or initials (in case privacy issues are of concern). The panelists will use their full names and will be shown in the camera. Those in the audience will not be shown on camera or have their full names used.

Meeting Logistics/Accommodations:
Unite Narcolepsy has spoken with FDA about patients’ symptoms possibly happening during the meeting (like cataplexy or sleepiness). People with narcolepsy and loved ones will be seated together so you will be surrounded by peers who understand. There will also be a separate room nearby available to rest or nap. You are invited to take a break at any time, stand in the back of the room, visit the refreshments station or rest in the separate resting room.

Additionally, we’ve spoken with FDA about cataplexy possibly happening during patient speaking opportunities. We’ve suggested that folks may remain seated and that someone else can hold the microphone if cataplexy is a concern. If there are any other specific accommodation requests, please contact Pujita Vaidya at the FDA: Pujita.Vaidya@fda.hhs.gov.

Speaking Opportunities/Giving Input:
Ten people will be chosen by FDA to participate as panelists during the in-person FDA Meeting on Sept. 24. If you’d like to be considered for this speaking role, you must indicate so while registering. To be considered for a panelist position, you must answer the FDA’s Questions and return them to PatientFocused@fda.hhs.gov by September 13, 2013. Panelists will be notified of their selection around Sept. 17. Given the overwhelming interest in the panelist spots, you may not be selected, but there are other speaking opportunities for patient advocates at the meeting. The FDA will ask specific questions to the audience in a facilitated discussion. There will also be “open-mic” time allowing individuals an optional 3 minutes of remarks.

During the meeting FDA will also ask several polling questions. Patients and advocates attending in person will have “clicker” devices to register their answers. People watching by webcast will also be able to register their response to polling questions.

Anonymity/Not Speaking:
There is no requirement to speak at the meeting. If you’d like to attend and not speak, this is perfectly acceptable. Thank you!

FDA White Oak Campus Parking

DC Area Airports:
Washington area airports

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WEBINAR PROGRAM 2: Patients at the Center – The FDA’s Patient Focused Drug Development Initiative

unite narcolepsy webinar patients at the center FDA patient focused drug development initiativeDate Recorded: Thursday, Sept. 5 at 2 p.m. (Eastern)

Speakers: Monica Gow & Kim McCleary

Description: Did you know that 25 cents of every dollar consumers spend is regulated by the U.S. Food and Drug Administration? Did you know that “patient engagement” has been identified as the “blockbuster drug of decade”? Learn more about how drugs are developed and approved and why the FDA is highlighting narcolepsy as part of its Patient-Focused Drug Development Initiative. We’ll also remind you about ways you can help shape the future of treatment for narcolepsy.

Watch recording: http://youtu.be/DOF23NnPxoY

WEBINAR PROGRAM 1: Introducing the Unite Narcolepsy Initiative

Watch Now!Date Recorded: Thursday, Aug. 29 at 2 p.m. (Eastern)

Speakers: Julie FlygareMonica Gow, Kim McCleary

Description: Unite Narcolepsy is an education and empowerment initiative created to help inform people affected by narcolepsy about a unique opportunity to shape the future of narcolepsy treatment and to help them respond effectively. By encouraging and equipping broad participation, we aim to honor patients’ experiences and use the power of their testimony to ensure that patient needs are at the center of research efforts and regulatory decisions about narcolepsy treatment. Learn more about the Unite Narcolepsy initiative and all the ways that YOU can be part of this landmark opportunity!

Watch recording: http://www.youtube.com/watch?v=-7AExb4f9Xw

“Why does the FDA initiative matter?”

By Julie Flygare, J.D.

As a narcolepsy advocate, the FDA Patient Focused Drug Development Initiative (PFDDI) caught my attention right away.  It’s not everyday that patients and high-level decision makers get together to discuss what matters most for patients. Here are the top five reasons why the FDA initiative matters.

fda1. Educating FDA:

The FDA initiative allows people with narcolepsy and their loved ones to actively shape the future of narcolepsy treatment. As a direct result of the narcolepsy meeting, FDA will produce a meeting report, reflecting the input obtained from the narcolepsy community. The report will be posted on FDA’s website and circulated to the agency’s key decision makers. This information may also inspire the creation of new patient-focused outcome measures qualified for use in drug development, to better capture patient experience more systematically in clinical trials.

2. Re-gaining control:

As a person with narcolepsy, you may feel out of control at times – with your symptoms holding the reigns and making demands on your time and energy. While participating in the FDA initiative will

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