CULVER CITY, Calif., Dec. 30, 2014 (GLOBE NEWSWIRE) -- The Coalition for Pulmonary Fibrosis (CPF) played an important role in much of the year's successes in Pulmonary Fibrosis (PF), helping to make 2014 the most important year in history for PF patients, families, physicians, researchers, advocates and industry.
A look back at 2014, a history-making year for the PF community:
Game Changing News:
In the biggest news, positive data from two much-anticipated drug trials were released in the spring of 2014 and by mid-October, the U.S. Food and Drug Administration (FDA) announced approvals of the first two drugs for Idiopathic Pulmonary Fibrosis (IPF). The FDA approved InterMune's Esbriet (pirfenidone) and Boehringer Ingelheim's Ofev (nintedanib), both shown in clinical trials to help to slow disease progression.
PF is a disease that affects 200,000 Americans and claims as many lives each year as breast cancer. PF is a lethal lung disease characterized by scarring that is irreversible, progressive and deadly in an average of three years. When no cause for the disease can be determined, it is called "idiopathic".
In response to the data release, the CPF held a webinar for patients, families and caregivers to help them better understand the information about the drugs' effectiveness and impact on patients presented during the American Thoracic Society (ATS) international conference in May. The CPF hosted the webinar that featured PF expert researcher and clinician Andrew M. Tager, MD, Associate Professor of Medicine, Harvard Medical School, Associate Physician, Pulmonary and Critical Care, Massachusetts General Hospital, who explained the research data and took questions from patients and family members.
Prior to approvals, the drugs were available through Expanded Access Programs (EAPs) and the CPF assisted in getting the news of the special programs out to patients and caregivers.
Working with Congress to Move PF Forward:
The CPF also partnered with members of Congress to move efforts forward in PF in 2014. The CPF assisted members of congressional staffs to prepare letters in support of PF early in the year. Led by Senator Chris Coons (D-DE) and Representative Erik Paulsen (R-MN), 43 members of Congress sent two letters both to the National Institutes of Health (NIH)'s Director, Francis Collins, MD, PhD and to the NIH's National Heart, Lung Blood Institute (NHLBI) Director, Gary Gibbons, MD. Their letters urged the NIH and NHLBI to do more to improve research efforts into PF including increasing research funding allocated to the disease and providing more focus on innovative areas of research including Fibrosis Across Organs (FAO). FAO is an effort that Sen. Coons and Rep. Paulsen have supported and which is one of the priority areas designated by NIH for PF. The CPF supported the first FAO meeting, held by the ATS in 2012.
In anticipation of FDA approvals for the first two drugs for IPF, Sen. Coons and Rep. Paulsen led a letter with 18 other members of Congress to urge the Centers for Medicare and Medicaid (CMS) to swiftly review the drugs, once approved so that Medicare patients could access the much needed therapies.
The CPF also assisted behind the scenes in a major effort by Sen. Coons to have PF included, for the first time, on the list of eligible research topics in the Department of Defense (DOD) Peer Reviewed Medical Research Program (PRMRP) by having it included in report language in the omnibus bill that has now been signed into law by the President The PRMRP is the official determination of disease areas that can be funded through the DOD. This program, overseen by the Secretary of Defense, in conjunction with the Service Surgeons General, is directed to select medical research projects of clear scientific merit and direct relevance to military health. As a PRMRP eligible disease area, PF researchers may apply for funding through the approximately $250 million DOD research fund until the end of the government's 2015 fiscal year. Earlier this year, PF was included in report language in the Senate Appropriations bill.
September was one of the most successful National PF Months in the CPF's history when efforts included more than 50 congressional office visits, a Daughters of Pulmonary Fibrosis visit to the White House, a patient event with the American Thoracic Society (ATS) and Johns Hopkins Medical Center in celebration of PF Week at the ATS, and the CPF's participation in the FDA's first IPF Workshop.
Providing the Patient Voice to FDA:
In the summer, the FDA announced it would hold its first IPF Workshop on September 26th. In 2011 and 2012, the CPF worked to convince FDA to include PF as one of the disease areas of focus for its limited funding allocation mandated by Congress. The allocation, funded by the Prescription Drug User Fee Act (PDUFA), a fee paid by the pharmaceutical industry, was designed to help increase attention to patients and families in the drug development process. The FDA created the Patient Focused Drug Development program to satisfy the mandate that would include up to 20 disease-specific workshops for all stakeholders. PF was not included on the initial list published by the FDA in 2011 for which it sought public comment. At the CPF's request, more than 3,000 members of the CPF sent letters to the FDA Commissioner, Margaret Hamburg, MD asking for PF's inclusion in the program.
Four CPF advocates were chosen by the FDA to participate in September's IPF Workshop eight-member panel to discuss patient experiences and to give the FDA a better understanding of the plight of patients, caregivers and family members. The CPF's Vice-President, Teresa Barnes, CPF's Daughters of PF members Heather Snyder and Faye MacInnis, and longtime advocate Curt Thompson served on the panel. Barnes has lost five members of her family to PF, Snyder is a PF survivor because of a lung transplant and lost her father to PF, MacInnis lost her husband to PF, and Thompson lost his wife and her twin sister to PF.
Prior to the FDA IPF workshop, the CPF held a preparation webinar to help patients, caregivers and family members to better understand what to expect of the workshop. It was also intended to prepare stakeholders and to make sure patient voices were heard both at the meeting, via the webinar of the event and via writing their experiences and submitting them to the FDA through the public docket. The CPF worked with non-profit organization Faster Cures to hold the webinar that featured Faster Cures' Kim McCleary and Shawn Sullivan.
The CPF also worked with FDA in preparation of the workshop by facilitating provision of supplemental oxygen for patients at the event and disseminating information about the workshop in advance.
The patient organization also provided a report to the FDA of patient experiences based on a comprehensive survey it produced in partnership with the Genetic Alliance (GA). The CPF was awarded a position as a member of the GA's pilot program for its Platform for Engaging Everyone Responsibly (PEER) platform in late 2013 and began the pilot initiative in early 2014. The GA announced recently the program has received a half million-dollar grant from the Robert Wood Johnson Foundation.
The CPF, Celebrities Speak Out:
The CPF joined with celebrities in 2014 to raise awareness of the disease and respond to major news. The CPF continued its work with actress Rose McGowan, who leads its Daughters of PF program. McGowan lost her father to PF. The CPF also worked with TV, Film and Broadway actress Barbara Barrie, who disclosed recently she has been diagnosed with IPF.
The CPF assisted Summer Productions in finding patients and family members for the first IPF documentary that aired in June and again in September on the Discovery Channel. Sports radio host George Lapides, who has spoken publicly on his Memphis-based radio show about his diagnosis with IPF worked with the CPF and appeared in a documentary on the disease (visit www.everybreathcountsfilm.com to view). The CPF works closely with Country Music star Joe Nichols who also appeared in the documentary and lost his father to IPF.
CPF works with PF Research Community:
The CPF continued to work with the American Thoracic Society (ATS), which represents thousands of pulmonary researchers and physicians worldwide. The ATS and CPF co-hosted a patient education event with Johns Hopkins Medical Center in September during "PF Week at the ATS", an event that was also webcast to patients and caregivers around the globe.
Through partnership grants, the ATS and CPF have funded more than $1 million in research to date. The CPF, itself, has funded more than $2 million in grants since inception. This year, the ATS and CPF, joined by the Pulmonary Fibrosis Foundation, funded a $100,000 PF grant to Nathan Sandbo, M.D., University of Wisconsin. The CPF's Teresa Barnes served as the ATS Public Advisory Board (PAR) Chair for almost five years and on the ATS Board of Directors and the ATS Foundation Board of Trustees. She is currently the immediate past chair of the ATS PAR.
The CPF partnered with members of industry and the PF medical community to hold four other patient education events across the country as part of the CPF"s "Living with PF" series. The CPF and New York Presbyterian-Weill Cornell hosted an event that was supported by InterMune in October. In November, the CPF and University of California Davis co-hosted an event in Sacramento that was supported by Gilead, and an InterMune-sponsored event with the University of Miami. In December, the CPF partnered with the Cleveland Clinic for an event in Cleveland, Ohio supported by Biogen-Idec and with Baylor College of Medicine in Houston, Texas for another InterMune sponsored event.
Earlier this year, CPF supported the first Fibrosis Across Species meeting convened in Louisville, Kentucky and hosted by the University of Louisville's Interstitial Lung Disease Program and Jesse Roman, M.D. The meeting brought together human pulmonary medical researchers and veterinary researchers from around the world to look at naturally-occurring PF in dogs, cats, horses and other domestic animals in effort to find treatments faster for humans and animals.
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.
CONTACT: Teresa Barnes Coalition for Pulmonary Fibrosis 303-521-4080 firstname.lastname@example.org
Source:Coalition for Pulmonary Fibrosis(CPF)