The Coalition for Pulmonary Fibrosis (CPF) a 501C(3) nonprofit organization founded in 2001 to accelerate research efforts leading to a cure for pulmonary fibrosis (PF), says 2014 was a history-making year for the Pulmonary Fibrosis community — the biggest news of the year being positive results of two highly-anticipated clinical drug trials released last spring of 2014 and the U.S. Food and Drug Administration (FDA)’s mid-October announcement of approvals for the first two Idiopathic Pulmonary Fibrosis (IPF) drugs — InterMune’s Esbriet (pirfenidone) and Boehringer Ingelheim’s Ofev (nintedanib), which had prior to approvals, been available through Expanded Access Programs (EAPs), and were both shown in the trials to help slow PF progression.
The CPF says PF affects some 200,000 Americans and causes as many deaths each year as breast cancer. Pulmonary Fibrosis (PF) is a lung disorder characterized by a progressive scarring of lung tissue known as fibrosis, which slowly diminishes victims of their ability to breathe. An estimated 48,000 new cases are diagnosed each year, and there is currently no known cause or cure. PF is also difficult to diagnose and roughly two-thirds of patients die within five years of diagnosis. In some instances, PF can be linked to a specific cause, such as certain environmental exposures, chemotherapy or radiation therapy, residual infection, or autoimmune diseases such as scleroderma or rheumatoid arthritis. However, often no known cause can be established, in which case, it is called idiopathic pulmonary fibrosis (IPF), “Idiopathic” diseases are those for which no cause can be determined.
The CPF held a webinar for patients, families and caregivers during the American Thoracic Society (ATS) international conference in May featuring PF expert researcher and clinician and Harvard Medical School Associate Professor of Medicine Andrew M. Tager, MD,to help better understand data released about the drugs’ effectiveness.
Another CPF initiative was a partnerships with members of Congress to advance PF research efforts in 2014, and in which the CPF assisted congressional staff members in preparation of letters in support of PF early in the year. Spearheaded by Senator Chris Coons (D-DE) and Representative Erik Paulsen (R-MN), 43 members of Congress sent two letters to National Institutes of Health (NIH) Director, Francis Collins, MD, PhD, and to the NIH National Heart, Lung Blood Institute (NHLBI) Director, Gary Gibbons, MD, urging both organizations to step up with support for research into PF including more funding for PF research, and more focus on innovative research such as Fibrosis Across Organs (FAO) — an effort both Sen. Coons and Rep. Paulsen have supported, and a priority area designated by NIH for PF.
Anticipating FDA approvals for the two new IPF drugs, Sen. Coons and Rep. Paulsen organized a letter campaign along with 18 other members of Congress urging the Centers for Medicare and Medicaid (CMS) to fast track the drugs, once approved, so Medicare patients could access these much-needed therapies.
The CPF also assisted with Sen. Coons’ behind-the-scenes effort by to get 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) — the official determination of disease areas that can be funded through the DOD — by having PF included in report language of an omnibus bill subsequently signed into law by President Obama. PRMRP program funding, overseen by the Secretary of Defense jointly with the Service Surgeons General, is channeled into select medical research projects that have clear scientific merit and are directly relevant to military members’ health. PF having been declared a PRMRP eligible disease category, researchers can apply for funding from the approximately $250 million DOD research fund until the government’s 2015 fiscal year-end, and PF earlier this year was included in report language of the Senate Appropriations bill.
The CPF ranks September, 2014 as one of the most successful National PF Months in the organization’s history, logging more than 50 congressional office visits, a visit to the White House by Daughters of Pulmonary Fibrosis, an American Thoracic Society (ATS) patient event, with the Johns Hopkins Medical Center in celebration of PF Week at the ATS, and the CPF’s participation in the FDA’s first IPF Workshop on September 26th. The CPF had worked throughout 2011 and 2012 on persuading the FDA to include PF as a disease area of focus for a Congress-mandated limited funding allocation funded through the Prescription Drug User Fee Act (PDUFA) — a fee levied on the pharmaceutical industry, and designed to enhance levels of attention to patients and families in drug development. The FDA’s Patient Focused Drug Development program created to satisfy that mandate is structured to include up to 20 disease-specific stakeholders’ workshops for. PF didn’t make the cut for the FDA’s initial list in 201, but sought public comment, and at the CPF’s request, more than 3,000 of its members sent letters to FDA Commissioner, Margaret Hamburg, MD appealing for PF to be included in the program.
The FDA chose four CPF advocates to participate in the September IPF Workshop’s eight-member panel that discussed patient experiences in order to provide the FDA with better understanding of hardships affecting PF patients, their caregivers and their family members. CPF Vice-President Teresa Barnes who herself has lost five family members to PF, CPF Daughters of PF members Heather Snyder — a PF survivor who lost her father to the disease and Faye MacInnis who lost her husband to PF, and longtime advocate Curt Thompson who lost his wife and her twin sister to PF were chosen to serve on the panel. A CPF webinar held prior to the FDA workshop helped caregivers and family members to better understand what to expect.
The CPF also assisted in preparation of the workshop by providing supplemental oxygen for patients at the event and publicizing the workshop in advance, as well as providing the FDA with a report of patient experiences based on a comprehensive survey produced in partnership with the Genetic Alliance (GA) on which the CPF was awarded a membership on the GA Platform for Engaging Everyone Responsibly (PEER) pilot program in late 2013 which recently received a half million-dollar grant from the Robert Wood Johnson Foundation.
Celebrities joining with the CPF to raise awareness of the disease \ in 2014 included actress Rose McGowan who lost her father to PF, leads the organization’s Daughters of PF program and TV, Film and Broadway actress Barbara Barrie, who recently disclosed her being diagnosed with IPF.
The CPF assisted in a search by Summer Productions in finding patient and family member subjects for an IPF documentary televised last June and repeated in September on the Discovery Channel. George Lapides, a Memphis-based sports radio host, has discussed his diagnosis with IPF on his radio show, worked with the CPF, and has appeared in another documentary about the disease (http://www.everybreathcountsfilm.com). Country Music artist Joe Nichols who lost his father to IPF, also appeared in that documentary.
In September the CPF co-hosted at Johns Hopkins Medical Center during “PF Week at the ATS” a patient education event that was also webcast to patients and caregivers around the world in cooperation with the American Thoracic Society (ATS), an organization that represents thousands of pulmonary researchers and physicians worldwide.
The ATS and CPF through partnership grants, have funded more than $1 million in research to date, while the CPF, itself, has funded more than $2 million in grants since its inception. This year, the ATS and CPF, jointly with the Pulmonary Fibrosis Foundation, funded a $100,000 PF grant to Nathan Sandbo, M.D., a researcher at the University of Wisconsin, and the CPF’s Teresa Barnes served as ATS Public Advisory Board (PAR) Chairperson for almost five years as well as on the ATS Board of Directors and Foundation Board of Trustees.
The CPF partnered with industry players and the PF medical community in holding four additional patient education events across the country as part of its “Living with PF” series, including a CPF/New York Presbyterian-Weill Cornell hosted event supported by InterMune in October; a CPF/University of California at Davis co-hosted an event in Sacramento supported by Gilead, and with the University of Miami an InterMune-sponsored event. The CPF partnered with the Cleveland Clinic in December at a Biogen-Idec supported event in Cleveland, Ohio, and with Baylor College of Medicine in Houston, Texas on another InterMune sponsored event.
Earlier in the year, the CPF supported the first Fibrosis Across Species meeting hosted by the University of Louisville’s Interstitial Lung Disease Program and Jesse Roman, M.D.in Louisville, Kentucky. That meeting attracted participation by human pulmonary medical researchers as well as veterinary researchers worldwide to discuss naturally-occurring PF in dogs, cats, horses and other domestic animals in advancing efforts to find treatments faster for both humans and animals.
The Coalition for Pulmonary Fibrosis
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