The CPF is pleased to share with you news from Washington last week. PF advocates combed Capitol Hill on Thursday, meeting with 38 Congressional offices. On Friday, patients and family members took advantage of the opportunity to address key FDA staffers about their experiences with Pulmonary Fibrosis and their suggestions for improved options for patients.
Please see the press release below regarding the FDA IPF Workshop on Friday.
Sincerely,
Mishka Michon, CEO, Coalition for Pulmonary Fibrosis
Pulmonary Fibrosis Patients, Families Take Center Stage at FDA
FDA Hears from eight-member panel, other stakeholders on drug development in deadly lung disease
Culver City, CA/ September, 29, 2014/ Most FDA panels involve high-level executives and renowned medical professionals in their given areas of expertise. On Friday, however, patients and family members took center stage at a U.S. Food and Drug Administration (FDA) Workshop on Idiopathic Pulmonary Fibrosis to offer their perspectives.
The FDA hosted the first patient-focused workshop on IPF as more than 100 people gathered in the Silver Springs offices. Those in attendance were mostly patients, family members, leaders of patient advocacy groups, medical professionals and industry representatives. A few hundred also joined the event online, according to the FDA.
Patients and family members of deceased IPF patients spoke about the hardships of IPF, a disease that debilitates patients early in its course and produces a steady decline in quality of life and gradual worsening of symptoms such as shortness of breath, cough and fatigue as well as issues with anxiety and depression.
Two survivors of IPF spoke about their experience pre- and post-transplant and about how grateful they were to be part of the less than one percent of patients who survive the deadly disease.
The Coalition for Pulmonary Fibrosis (CPF), a non-profit organization, worked with FDA to make their workshop on September 26th more accessible to IPF patients by facilitating supplemental oxygen refills. Half of the members of the eight-person panel advocate for the CPF, including IPF survivor via lung transplant, Heather Snyder and patient family members Faye MacInnis, Curt Thompson, and the CPF’s vice president, Teresa Barnes. All four advocates were also on Capitol Hill on Thursday with the CPF.
“The CPF is happy to see this historic time when FDA takes direct testimony from the people and families who feel the impact of this disease,” said Mishka Michon, Chief Executive Officer of the CPF.
Pulmonary Fibrosis (PF), which affects 200,000 Americans, 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 PF is unknown, it is referred to as idiopathic.
Panelists thanked FDA for giving the patients a voice in the drug development process and one panelist encouraged the regulatory agency to include patients and advocates a decade or more earlier.
The FDA’s Banu Karimi-Shah, MD agreed with the patients and advocates that the patient voice needs to be included in the process at an earlier stage. She also conveyed to the audience that “I learned so much today. I know it wasn‘t just a learning opportunity for me but all of us here at FDA.”
The workshop’s purpose was 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. There were two times during the meeting when the FDA opened up for questions from the audience and a public forum held at the end of the meeting gave other stakeholders an opportunity to weigh in.
One patient in the audience described his extreme shortness of breath during a particularly difficult episode of coughing as “knowing what it must be like to drown.” Others described the stress and anxiety related to fears around running out of supplemental oxygen and fear of suffocation.
The workshop was not held to review any particular drugs but rather to gather information that can aid the FDA in reviewing drugs for the disease going forward. According to the FDA, a report will result from the meeting to guide FDA in future drug reviews and will be available publicly to the all stakeholders.
FDA reviews of two potential IPF therapies are currently underway. FDA recently granted breakthrough therapy designation for Boehringer Ingelheim’s nintedanib and InterMune’s pirfenidone. If approved, they will be the first therapies available to patients with IPF in the U.S.
The FDA will still be accepting comments from patients, caregivers and all other stakeholders until November 26th via the public docket. For information on submission, visit:
About Pulmonary Fibrosis (PF)
Pulmonary Fibrosis (PF) is a lung disorder characterized by a progressive scarring – known as fibrosis -- and deterioration of the lungs, which slowly robs its victims of their ability to breathe. Approximately 200,000 Americans suffer from PF, and there is currently no known cause or cure. An estimated 48,000 new cases are diagnosed each year. PF is difficult to diagnose and an estimated two-thirds of patients die within five years of diagnosis. Sometimes PF can be linked to a particular cause, such as certain environmental exposures, chemotherapy or radiation therapy, residual infection, or autoimmune diseases such as scleroderma or rheumatoid arthritis. However, in many instances, no known cause can be established. When this is the case, it is called idiopathic pulmonary fibrosis (IPF).
About the CPF
The CPF is a 501C(3) nonprofit organization, founded in 2001 to accelerate research efforts leading to a cure for pulmonary fibrosis (PF), while educating, supporting, and advocating for the community of patients, families, and medical professionals fighting this disease. The CPF funds promising research into new approaches to treat and cure PF; provides patients and families with comprehensive education materials, resources, and hope; serves as a voice for national advocacy of PF issues; and works to improve awareness of PF in the medical community as well as the general public. The CPF's nonprofit partners include many of the most respected medical centers and healthcare organizations in the U.S. With more than 28,000 members nationwide, the CPF is the largest nonprofit organization in the U.S. dedicated to advocating for those with PF. For more information please visit www.coalitionforpf.org or call (888) 222-8541.
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