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Patient adcocacy

The EU-PFF Advocate Development Programme (ADP) has been designed and developed for EU-IPFF members and IPF/PF advocates. This 10-month course aims to develop your capacity to become an even more effective and empowered patient advocate that serves patients in your community by understanding clinical research and development, mechanisms of accessing diagnostics and drugs, interaction with stakeholders like clinicians and industry, and the basics of evidence-based advocacy.


Patient advocacy is a concept that most people think they understand, but they probably don't comprehend its scope. In its simplest terms, patient advocacy regards any activity which ultimately benefits a patient. Using that definition, it can apply to caregiving for an individual patient, to groups that develop policies and advice that help patients, to government groups that develop legislation to improve systems or processes for patients.

Patient advocacy groups play an important role in supporting patients with chronic diseases and promoting better care. The aim of this patient–physician initiative was to gather perceptions from European idiopathic pulmonary fibrosis (IPF) patient advocacy groups regarding inequalities and unmet needs in IPF care, in order to develop a Patient Charter to advocate for better care.

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 or download the PDF here.