I thought this was great and just wanted to share it. I've been pushing out our little press release and saw this. Actually, it came out in March. I don't know how I missed it. I need to get on their press list. The HPS Network was unable to attend this meeting in person. It was both a function of scheduling issues as well as a budget issue. I would have gone, even if Donna couldn't, but we're having to watch the budget very carefully right now. (As if we don't always - groan). It's also sort of nice to see the "I" dropped for idiopathic in most of the text. It seems less exclusive.
Pulmonary Fibrosis Patient Organizations Applaud Effords of Patients, Families at FDA Hearing
PF Patients Plead with FDA Advisory Panel to Hear Their Voices, Understand Their Plight
The Coalition for Pulmonary Fibrosis (CPF) and the Pulmonary Fibrosis Foundation (PFF) ) are applauding the efforts of Pulmonary Fibrosis (PF) patients and family members for their work to convey to the FDA and an FDA Advisory Committee the sense of urgency and desperation regarding the disease and the lack of treatment options.
Their words were heard yesterday by the 11 member Advisory Committee and representatives of the FDA during a public hearing portion of the FDA Pulmonary-Allergy Drugs Advisory Committee discussion of a potential new therapy in the fight against PF. The committee voted 9 to 3 to recommend approval for the drug by the FDA.
If approved, the therapy, Pirfenidone, would be the first FDA approved drug for PF. PF is a progressive, relentless and irreversible scarring of the lungs which renders patients unable to breathe and is almost invariably fatal within two to five years.
“There’s no question that the patient perspective was heard by the FDA and the panel,” said Mishka Michon, Chief Executive Officer of the Coalition for Pulmonary Fibrosis. “Family members and patients spoke from their hearts and conveyed the sense of urgency they feel in desperately waiting for a treatment for this horrific disease.”“We are humbled by the courage and determination of our patients,” said Daniel Rose, M.D., President of the Pulmonary Fibrosis Foundation. “The voice of the patient community is a critical component in finding solutions to PF.”The panel listened to testimony of 15 PF patients and family members for an hour. It also received a stack of letters from patients and families across the country who could not attend the meeting in person.
Advocate Joy McBride opened the public hearing portion of the meeting by sharing with the panel the loss of her father and uncle to PF. “The hardest part was hearing from the doctors ‘there is nothing I can do for you’” she said. “Today, there is still no known cause, no cure, and no hope.” Patient Tommy Spivey traveled with his wife from Wilmington, North Carolina to speak to the panel. “I have one grandchild and one on the way and I would like to live long enough to get to know them,” he said. Spivey, who says his disease is currently not progressing, traveled to Japan in 2009 to gain access to Pirfenidone and is currently taking the drug.
Kaitlyn Bergen shared with the panel details of her father’s illness and subsequent death to PF in 2006. “Pulmonary Fibrosis will continue stealing valuable and meaningful years from families,” she said.
Though Timothy Cooney’s father is one of a small number of survivors of the disease as a result of a successful lung transplant more than a year ago, he delivered his message to the panel on behalf of his family and in memory of his grandmother who was claimed by PF. “On a sinking ship, if the life raft has a few holes in it, people will still take that raft,” he said.
At the end of the day-long meeting, one panelist, Rodney Mullins, consumer representative for the committee, echoed Cooney’s comments in his final remarks. “We don’t know how leaky the lifeboat is, but it’s a lifeboat.”
The Advisory Committee's recommendations are not binding but will be considered as the FDA completes its review of the New Drug Application (NDA) for the drug. Pirfenidone received Orphan Drug, Fast Track and Priority Review designations by the FDA. A target date of May 4, 2010 for FDA action has been set.
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 128,000 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 23,000 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.
About the Pulmonary Fibrosis Foundation
The Pulmonary Fibrosis Foundation is a 501(c)(3) non-profit organization that was founded in Denver, Colorado in 2000. The mission of the Pulmonary Fibrosis Foundation is to find a cure for idiopathic pulmonary fibrosis (IPF) by funding research, advocating for pulmonary fibrosis issues, promoting disease awareness, and providing a compassionate environment for supporting patients and their loved ones. We aim to accomplish this by working with the medical community to drive new research, increase research funding, by representing the needs of our constituents in Washington DC through national advocacy, and by developing caring relationships with patients and their families throughout the course of their disease. Since the inception of the Foundation, it has funded or is committed to fund $3,000,000 in basic research. For more information please visit www.pulmonaryfibrosis.org or call 312-587-9272.