2014-02629. Pulmonary Arterial Hypertension Public Meeting on Patient-Focused Drug Development

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice of public meeting; request for comments.

SUMMARY:

The Food and Drug Administration (FDA) is announcing a public meeting and an opportunity for public comment on Patient-Focused Drug Development for pulmonary arterial hypertension. Patient-Focused Drug Development is part of FDA's performance commitments in the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patients' perspectives on the impact of pulmonary arterial hypertension on daily life, as well as their perspectives on the available therapies for pulmonary arterial hypertension.

DATES:

The public meeting will be held on May 13, 2014, from 1 p.m. to 5 p.m. Registration to attend the meeting must be received by April 30, 2014. See the SUPPLEMENTARY INFORMATION section for information on how to register for the meeting. Submit electronic or written comments by July 14, 2014.

ADDRESSES:

The meeting will be held at the FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm. 1503A), Silver Spring, MD 20993. Entrance for the public meeting participants (non-FDA employees) is through Building 1, where routine security check procedures will be performed. For more information on parking and security procedures, please refer to http://www.fda.gov/​AboutFDA/​WorkingatFDA/​BuildingsandFacilities/​WhiteOakCampusInformation/​ucm241740.htm.

Submit electronic comments to http://www.regulations.gov. Submit written comments to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. All comments should be identified with the docket number found in brackets in the heading of this document.

FDA will post the agenda approximately 5 days before the meeting at http://www.fda.gov/​ForIndustry/​UserFees/​PrescriptionDrugUserFee/​ucm379694.htm.

FOR FURTHER INFORMATION CONTACT:

Graham Thompson, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 1199, Silver Spring, MD 20993, 301-796-5003, FAX: [phone], email: [e-mail].

SUPPLEMENTARY INFORMATION:

I. Background on Patient-Focused Drug Development

FDA has selected pulmonary arterial hypertension as the focus of a meeting under Patient-Focused Drug Development, an initiative that involves obtaining a better understanding of patients' perspectives on the severity of the disease and the available therapies for the condition. Patient-Focused Drug Development is being conducted to fulfill FDA's performance commitments made as part of the authorization of PDUFA V under Title I of the Food and Drug Safety and Innovation Act (Pub. L. 112-144). The full set of performance commitments is available on the FDA Web site at http://www.fda.gov/​downloads/​forindustry/​userfees/​prescriptiondruguserfee/​ucm270412.pdf.

FDA has committed to obtain the patient perspective in 20 disease areas during the course of PDUFA V. For each disease area, the Agency will conduct a public meeting to discuss the disease and its impact on patients' daily lives, the types of treatment benefit that matter most to patients, and patients' perspectives on the adequacy of the available therapies. These meetings will include participation of FDA review divisions, the relevant patient community, and other interested stakeholders.

On April 11, 2013, FDA published a notice (78 FR 21613) in the Federal Register announcing the disease areas for meetings in fiscal years (FYs) 2013 through 2015, the first 3 years of the 5-year PDUFA V time frame. To develop the list of disease areas, the Agency used several criteria that were outlined in the April 11 notice. The Agency obtained public comment on these criteria and potential disease areas through a notice for public comment published in the Federal Register on September 24, 2012 (77 FR 58849), and through a public meeting held on October 25, 2012. In selecting the disease areas, FDA carefully considered the public comments received and the perspectives of its review divisions. By the end of FY 2015, FDA will initiate another public process for determining the disease areas for FYs 2016 through 2017. More information, including the list of disease areas and a general schedule of meetings, is posted on FDA's Web site at http://www.fda.gov/​ForIndustry/​UserFees/​PrescriptionDrugUserFee/​ucm326192.htm.

II. Public Meeting Information

A. Purpose and Scope of the Meeting

As part of Patient-Focused Drug Development, FDA will obtain patient and patient stakeholder input on symptoms of pulmonary arterial hypertension that matter most to patients and on current approaches to treating pulmonary arterial hypertension. Pulmonary arterial hypertension is a rare, progressive condition that affects the heart and lungs. It is characterized by abnormally high blood pressure in the pulmonary artery and may be accompanied by shortness of breath, chest pain, fatigue, dizziness, fainting, lightheadedness, and swollen ankles and legs. There are several treatment options for pulmonary arterial hypertension, including medications, surgery, and lifestyle changes.

The questions that will be asked of patients and patient stakeholders at the meeting are listed in this section, organized by topic. For each topic, a brief patient panel discussion will begin the dialogue, followed by a facilitated discussion inviting comments from other patient and patient stakeholder participants. In addition to input generated through this public meeting, FDA is interested in receiving patient input addressing these questions through written comments that can be submitted to the public docket (see ADDRESSES).

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 symptoms such as chest pain, shortness of breath, difficulty concentrating, and others.)

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 may include activities such as household chores, walking up the stairs.)

• How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days?

3. How have your condition and its symptoms changed over time?

Topic 2: Patients' Perspectives on Current Approaches to Treating Pulmonary Arterial Hypertension

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

• How has your treatment regimen changed over time, and why?
• How well does your current treatment regimen treat the most significant symptoms of your disease?
• Have the medications for pulmonary arterial hypertension made a difference to you? If so, in what ways?

2. What are the most significant downsides to your current treatments, and how do they affect your daily life? (Examples may include downsides such as bothersome side effects, going to the hospital for treatment, and others.)

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

B. Meeting Attendance and Participation

If you wish to attend this meeting, visit https://patientfocusedpulmonaryarterialhypertension.eventbrite.com. Please register by April 30, 2014. Those who are unable to attend the meeting in person can register to view a live Webcast of the meeting. You will be asked to indicate in your registration whether you plan to attend in person or via the Webcast. Your registration should also contain your complete contact information, including name, title, affiliation, address, email address, and phone number.

Seating will be limited, so early registration is recommended. Registration is free and will be on a first-come, first-served basis. However, FDA may limit the number of participants from each organization based on space limitations. Registrants will receive confirmation once they have been accepted. Onsite registration on the day of the meeting will be based on space availability. If you need special accommodations because of disability, please contact Graham Thompson (see FOR FURTHER INFORMATION CONTACT) at least 7 days before the meeting.

Patients who are interested in presenting comments as part of the initial panel discussions will be asked to indicate in their registration which topic(s) they wish to address. These patients will also be asked to send a brief summary of responses to the topic questions to [e-mail]. We will notify panelists of their selection soon after the close of registration on April 30, 2014. We will try to accommodate all patients and patient stakeholders who wish to speak, either through the panel discussion or audience participation; however, the duration of comments may be limited by time constraints.

Comments: Regardless of attendance at the public meeting, you can submit electronic or written responses to the questions pertaining to Topics 1 and 2 to the public docket (see ADDRESSES) by July 14, 2014. Received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

Transcripts: As soon as a transcript is available, FDA will post it at http://www.fda.gov/​ForIndustry/​UserFees/​PrescriptionDrugUserFee/​ucm379694.htm.

Dated: January 30, 2014.

Leslie Kux,

Assistant Commissioner for Policy.