Date: September 27, 2016
Time: 9:00 a.m. to 5:00 p.m.
Location: FDA White Oak Campus
10903 New Hampshire Ave.
Building 31 Conference Center, The Great Room (Rm 1503 B+C)
Silver Spring, MD 20993
(Enter at Building 1 to clear security)
On September 27, 2016 FDA is conducting a public meeting on Patient-Focused Drug Development for Patients Who Have Received an Organ Transplant. Patient-Focused Drug Development is part of FDA’s performance commitments made as part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patient perspectives on the impact of organ transplantation on daily life, patient views on receiving an organ transplant, and decision factors taken into account when selecting a regimen for organ transplantation management.
For each of these topics, a panel of patients and patient representatives/advocates will present comments to begin the dialogue and will be followed by a facilitated discussion inviting comments from all patients and patient representatives in the audience.
If you are attending the meeting in person and are interested in providing comments as part of the initial panel discussion, indicate so during the registration process. Potential panelists must send a brief summary of responses to the discussion questions below to PatientFocused@fda.hhs.gov. Panelists will be confirmed prior to the meeting.
In the afternoon, FDA will hold a workshop and provide information for and gain perspective from patients, patient advocacy organizations, health care providers, academic experts, and industry on various aspects of clinical development of drug products intended for patients who have received organ transplants. The input from this public meeting will help in developing topics for future discussion. Discussion topics for the afternoon will include the following: current treatment consideration, considerations regarding adherence to treatments, clinical trial designs, and clinical trial endpoints.
There will also be an opportunity for patients, patient representatives and others to provide comments on issues other than the Topic 1 and Topic 2 Discussion Questions (see below) during an Open Public Comment session. Sign-up for Open Public Comment will take place the day of the meeting.
Webcast participants will also have an opportunity to provide input through webcast comments.
For more information, refer to the FDA meeting website at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm495933.htm
Topic 1: Disease symptoms and daily impacts that matter most to patients
1) What have been the most significant changes in your overall health since you received your transplanted organ?
a) How long has it been since your received your transplant?
2) Focusing on symptoms related to your organ transplant and post-transplant effects, which 1-3 symptoms have the most significant impact on your life? (Examples may include pain, infection, anxiety, etc.
3) 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, driving, walking/running, exercising, etc.)
a) How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days? (Examples may include limitations on the ability to undertake physically strenuous activities, restrictions on the ability to travel, lack of appetite, fatigue, etc.)
4) How has your experience with your transplanted organ changed over time? Do particular symptoms come and go as your duration of time with a transplanted organ has increased? If so, do you know of anything that makes your symptoms better? Worse?
5) What worries you most about your health post-transplant?
Topic 2: Patients’ perspectives on current approaches to treatment
1) What are you currently doing to maintain your transplanted organ or treat related health concerns following transplantation? (Examples may include immunosuppressants, antibiotics, antivirals, over-the-counter products, and other therapies including non-drug therapies)
a) How has your post-transplant regimen changed over time, and why?
2) How well does your current treatment regimen manage the most significant symptoms you experience post-transplantation?
a) How well do these treatments improve your ability to do specific activities that are important to you in your daily life?
b) How well have these treatments worked for you as your experiences post-transplant have changed over time?
3) What are the most significant downsides to your current treatments, and how do they affect your daily life? (Examples of downsides may include bothersome side effects, need for multiple medications, risk of infection, need for hospitalization, etc.)
a) What are the biggest
4) What specific things would you look for in an ideal treatment for managing your transplanted organ?