Nicholas Hill, MD

There is good news and bad news about idiopathic pulmonary fibrosis. In the U.S., no drugs are approved to treat this ultimately lethal condition, but there is movement on the research front. New clinical guidelines were released last year. There are different perspectives on the clinical data, and they all will be on display later today.

“We are bringing together multiple viewpoints that represent different aspects of this very challenging problem,” said ATS President Nicholas Hill, MD, chief of pulmonary, critical care and sleep Medicine, Tufts Medical Center, and professor of medicine, Tufts University School of Medicine, Boston. “We are looking at this disease from the clinician’s standpoint, the clinical trialist’s standpoint, the regulatory standpoint, the guideline committee standpoint and from the standpoint of the National Institutes of Health, which is trying to support basic research to come up with new approaches and new therapies.”

These multiple points of view come together during the President’s Symposium on “Drugs, Regulatory Agencies and Guidelines: The Challenge of Idiopathic Pulmonary Fibrosis (IPF)” from 2 to 4:30 p.m. today in Rooms 304-306, South Building (Esplanade Level), Moscone Center. Dr. Hill and Ganesh Raghu, MD, professor of medicine, University of Washington, Seattle, are symposium co-chairs.

Dr. Raghu will offer the clinician’s perspective on IPF.

“The harsh reality is that there are no available therapies that change the natural course of the disease,” Dr. Hill said. “Currently available therapies in the U.S. are palliative, not curative.

“We use therapies like oxygen, diuretics for fluid retention or steroids for dyspnea at late stages of the disease, and some use drugs for comorbidities like pulmonary hypertension. There is evidence that pulmonary rehabilitation can be helpful at some stages of the disease. These therapies can improve quality of life and functional status. But we have nothing at this point that actually prolongs survival.”

It’s not for want of trying Dr. Hill said. There has been significant investment in research and trials for promising agents such as gamma interferon. But the early promise of gamma interferon was dashed during the definitive Phase 3 trials.

Pirfenidone is another agent that showed promise in early trials. Whether late-stage trials met that promise is a matter of opinion. One randomized controlled trial showed clinical benefit, another trial, based largely in North America, did not. The European Medicines Agency (EMA) approved pirfenidone for use in Europe while the Food and Drug Administration (FDA) declined to approve the drug for use in the U.S.

“We have representatives of both the EMA and the FDA who will give their perspectives on their respective regulatory actions,” Dr. Hill said. “We’re not asking them to debate. In cases like this where you have some positive evidence and some nonsupportive evidence, there is room for differences in opinion. But it should be very interesting to hear their different perspectives on the same data.”

There are differences in opinion on clinical guidelines for IPF as well. The previous set of ATS clinical guidelines recommended the use of prednisone and azathioprine in IPF. The most recent ATS guidelines for the diagnosis and management of IPF, issued in 2010, do not.

“We know that progress comes in fits and starts,” Dr. Hill said. “The most recent evidence suggests that prednisone and azathioprine probably does more harm than good. The obvious question is how they got into our guidelines in the first place.

“So we have a presentation by the ATS documents editor, Kevin Wilson, MD, assistant professor of medicine, Boston University School of Medicine, who will give us a look at how the process of making guidelines has changed and how guidelines might look in the future.”