By: Teresa Barnes
VP, Patient Outreach & Program Support
Coalition for Pulmonary Fibrosis
For a deadly disease that affects so many Americans, perhaps it is fitting that the first congressional resolution recognizing its annual awareness efforts passed the Senate on the same day as Pope Francis’ historic address to Congress. The resolution, S. 270, recognizing National Pulmonary Fibrosis Awareness Month (September) passed on September 23rd.
Advocacy efforts over the years have pushed for increases in attention and research funding for Pulmonary Fibrosis (PF) and are, at least in part, to thank for much of the momentum around the deadly lung disease. Evidenced by an increasing number of research opportunities, advocates believe answers will now begin to come much faster than in previous decades.
In the last 12 months alone, two new drug approvals, the first in the disease state, finally came and both on the same day, October 15, 2014.
An outsider just looking at PF efforts over the last year or so could get the wrong impression. Research efforts in PF were stalled for years, even decades – likely because of a combination of more than a dozen failed clinical trials and scarce federal funding. At a National Institutes of Health Idiopathic Pulmonary Fibrosis workshop held in the fall of 2012, research attendees noted the agenda and goals for the meeting looked much like a similar meeting held on the topic a decade earlier.
Prior to the approvals last year, (pirfenidone marketed as Esbriet by Genentech and nintedanib marketed as OFEV by Boehringer Ingelheim), advocates were working behind the scenes to insure the U.S. Food and Drug Administration (FDA) understood the patient perspective as they pondered approving these first drugs and looked forward to others that follow.
When the FDA first reviewed pirfenidone in 2010, advocates were there to testify and to encourage FDA to understand PF patients’ perspectives on the benefit-risk assessment of the drug. The FDA’s advisory panel voted 9-3 in favor of pirfenidone. The excitement in the patient community was short lived when the drug was not approved and subsequently returned to clinical trials.
When FDA published a list of diseases in the Federal Register that would be considered for inclusion in 20 disease workshops to be held from 2013-2017, PF advocates sprung into action. By meeting with the FDA and asking their patient members to send letters directly to FDA leadership (3,000 letters were sent), advocates successfully convinced the FDA to include PF in the Patient Focused Drug Development Program, a program mandated by Congress and funded by user fees paid by the pharmaceutical industry.
As a result, FDA held a PF-specific workshop in September 2014 involving various stakeholders and featuring patients, families and advocates. As a result, the FDA published a guidance document on PF meant to assist the FDA and its panels of experts to make more informed decisions with the patient perspective on PF in mind. Aptly named “The Voice of the Patient: Idiopathic Pulmonary Fibrosis”, it can be found on the FDA website at:
http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM440829.pdf
Because of the work of advocates, along with congressional champions including Senator Christopher Coons and Representative Erik Paulsen, PF was included, for the first time in Department of Defense (DOD) medical research funding this year.
PF was listed as one of 41 diseases under the DOD’s Congressionally Directed Medical Research Programs (CDMRP). The program includes the Peer Reviewed Medical Research Program (PRMRP) and 41 diseases areas, including PF, are included as eligible for research funding for fiscal year 2015, totaling $247.5 million.
Advocates, along with medical physician membership organization, the American Thoracic Society, held the first meeting of experts across multiple areas of fibrosis in 2012 called “Fibrosis Across Organs”. Though fibrosis occurs in all organs of the body, until that time, researchers in different areas of fibrosis did not have a system of collaboration. Due to the innovation and inclusionary nature of the effort, advocates worked behind the scenes as the Senate commended the fibrosis across organs effort in appropriations language in 2012 and 2013.
As a result of the effort, the National Institutes of Health (NIH), specifically the National Heart, Lung, Blood Institute (NHLBI), identified fibrosis across organs as a priority effort in its published roadmap for PF research over the next decade. In June, NHLBI launched a new funding mechanism, in large part due to advocates’ efforts, for fibrosis across organs. The new offering provides $11 million in funding for research projects that include at least two organ areas of fibrosis.