This is an unprecendented time in history for Idiopathic Pulmonary Fibrosis (IPF) and the patients, families, caregivers, researchers, advocates and other stakeholders. We want to update you on important things going on this month:
The first FDA IPF Workshop on Sept. 26th (where FDA will hear directly from patients and will focus on patients). Deadline extended by FDA to Sept. 17th to register!
The FDA is reviewing two possible therapies for IPF under breakthrough designation: InterMune's pirfenidone and Boehringer Ingelheim's nintedanib. We will update you when there is news on the FDA decisions.
September is National Pulmonary Fibrosis Awareness Month - get involved -it's as simple as sharing the ribbon below on Facebook. Also, see further down under National PF Awareness Month for other details.
See below for the press release the Coalition for Pulmonary Fibrosis (CPF) sent out today that contains much information on the goings on this month.
Get involved if you can and have hope!
Sincerely,
Mishka Michon, CEO, Coalition for Pulmonary Fibrosis
September Most Important Month in History for U.S. Patients Suffering from Idiopathic Pulmonary Fibrosis: FDA Workshop, Impending Drug Decisions, PF Month Activities
Coalition for Pulmonary Fibrosis Works with FDA to Make Critical Workshop More Accessible to Patients
Culver City, CA/ September, 11, 2014/ Never before have there been so many opportunities for patients to be heard regarding a deadly lung disease than this month: the U.S. Food and Drug Administration (FDA) is hosting the first FDA workshop on Idiopathic Pulmonary Fibrosis (IPF); groups of patient advocates will canvas Capitol Hill; and patients and family members hold grass roots efforts nationwide to raise awareness. All the while the FDA is reviewing what could be the first two approved therapies for the disease.
Pulmonary Fibrosis (PF) is a lung disease characterized by progressive and irreversible scarring that renders the lungs unable to exchange blood oxygen. Patients die in an average 2.8 years and the disease claims as many lives as breast cancer. When the cause of the disease is unknown, it is referred to as idiopathic.
The Coalition for Pulmonary Fibrosis (CPF), a non-profit group that has organized efforts around PF Awareness every September for 13 years now, is working with FDA to make their workshop on September 26th more accessible to IPF patients. The workshop’s purpose is for FDA to hear directly from patients about the symptoms that matter most to them and current approaches to treating IPF, according to the FDA.
Since most IPF patients require supplemental oxygen and have difficulty walking due to their reduced lung function, the FDA will be increasing the number of shuttles it normally operates from the FDA parking area to the FDA meeting location at the request of the CPF. The CPF is sponsoring the availability of supplemental oxygen at the FDA meeting so patients can more easily attend the four-hour event. Most IPF patients require higher oxygen levels, and therefore can use significant amounts of oxygen in a short time. Such a meeting would be impossible for many IPF patients to attend without the option of oxygen refills.
“The CPF is pleased to support the FDA as it prepares for direct testimony from the people who suffer from IPF,” said Mishka Michon, Chief Executive Officer of the CPF, “We want to make it easier for patients to participate in this hugely important meeting.”
To insure patients have ample time to sign up for the FDA IPF Workshop that will also be available to a limited number of people online, the FDA extended the deadline for patients and stakeholders to register to attend online or in person. It moved the deadline from September 10th to September 17th (to register, see below “About the FDA Workshop”).
Recently, the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation for two investigational treatments for the disease: Boehringer Ingelheim’s nintedanib and InterMune’s pirfenidone. If approved, they will be the first therapies available to patients in the U.S.
National Pulmonary Fibrosis Awareness Month in September
The CPF is sponsoring special campaigns during the 13 th annual National Pulmonary Fibrosis Awareness Month – including an effort by its member patients, their family members, and advocates, who are canvassing the nation to increase awareness of the disease with efforts ranging from mayoral and gubernatorial proclamations to meetings in districts and on Capitol Hill with congressional members and their offices. Many CPF members will be holding fundraising and awareness events on a local level, as well. The CPF and the American Thoracic Society (ATS) will co-host a disease awareness week September 21-27 during which they will co-host an IPF patient education event with Johns Hopkins Medical Center at its Bayview Campus, on September 27 th. The event will be webcast, as well. To register for the in-person meeting at Johns Hopkins or to join the webinar, email dperez@thoracic.org. The CPF will bring patients and advocates to Capitol Hill on September 25th and will take a group of its members to the White House on the morning of September 26 th before attending the FDA IPF Workshop.
Background on IPF Drugs under FDA Review
Recently, the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation for two investigational treatments for the disease: Boehringer Ingelheim’s nintedanib and InterMune’s pirfenidone. In May, both companies reported positive data from their Phase 3 clinical trials at the American Thoracic Society international medical conference.
About the FDA IPF Workshop
The workshop is a public meeting on idiopathic pulmonary fibrosis, as part of FDA’s Patient-Focused Drug Development initiative. The purpose of the meeting is to hear patients’ perspectives on the symptoms of idiopathic pulmonary fibrosis that matter most to them and on current approaches to treating idiopathic pulmonary fibrosis. This meeting is free and open to the public. Details: Date: Friday, September 26, 2014, Time: 1:00 pm – 5:00 pm, Location: FDA White Oak campus at 10903 New Hampshire Avenue, Silver Spring, MD. Registration is required. Attendees can register online: http://patientfocusedipf.eventbrite.com. Please note that registration ends on September 17, 2014. Remote access will be available via live webcast for those unable to attend in person. Patient representatives, such as caretakers or leaders of support groups, are welcome to attend and share their perspectives. A public docket will also be available, which will give patients (including those not attending the workshop) an opportunity to submit comments to FDA online or through mail. For more information, visit the FDA website:
Report to the FDA
The CPF is currently gathering patient experiences via a software platform survey system with the Genetic Alliance called PEER. The program allows the CPF to query its patients, their family members and caregivers as well as the families of deceased patients, about what they have gone through with the disease. The CPF will analyze the data and present it to the FDA in a report in time for the FDA IPF Workshop. The survey can be found on the CPF’s website at: http://www.coalitionforpf.org/peer-portal/
FDA Questions for IPF Patients, Family Members and Caregivers
These are the two topic areas FDA will ask patients about in the FDA IPF Workshop; you can answer them, too, and the FDA will read them and use your responses in a report to FDA experts. Each time they review a drug in IPF, they will see the report with your input!
Answer them on this email and copy and paste them directly into the public docket by going to this link:
Topic 1: Symptoms and Daily Impacts That Matter Most to Patients
- 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 shortness of breath, cough, fatigue, etc.)
MY ANSWER:
- 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 household chores, walking up the stairs, etc.)
MY ANSWER:
○ How do your symptoms and their negative impacts affect your daily life on the best days?
MY ANSWER:
○ How do your symptoms and their negative impacts affect your daily life on the worst days?
MY ANSWER:
- How has your condition and its symptoms changed over time?
MY ANSWER:
Topic 2: Patient Perspectives on Treatment Approaches
- What are you currently doing to help treat your condition or its symptoms? (Examples may include prescription medicines, over-the-counter products, and other therapies including non-drug therapies such as diet modification.) How well does your current treatment regimen treat the most significant symptoms of your disease?
MY ANSWER:
- 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, going to the hospital for treatment, etc.)
MY ANSWER:
Because there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition?
MY ANSWER
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