How To Best Handle Preliminary Clinical Trial Results

A patient comes to your office with a press release on pirfenidone for the treatment of interstitial pulmonary fibrosis (IPF). While there is no peer-reviewed publication and no FDA approval, there is also no proven effective pharmacologic therapy for IPF. So how do we, as medical professionals, best handle incomplete information on such a disease?

“Should I be flying to Japan where the drug is approved?” “Does it work?” “When can I get it?” “Should I get it?” This is real life.

Data regarding the safety and efficacy of pirfenidone from the CAPACITY 1 and 2 trials have been disseminated. Indeed, a quick Google search would reveal a number of press releases over the last year. This, of course, adds to the buzz but does not inform or contribute to responsible clinical practice.

The principle of autonomy would hold that clinicians should share as much information as possible with patients so that they may make informed decisions about their own health. However, the existence of unpublished data sets that have not undergone either peer review or FDA review cannot be presented as “medical fact.”

In our opinion, the clinician should maintain equipoise with regard to clinical investigations throughout the investigative process that includes a full and fair evaluation of the trial results.

A balanced approach to the data is critically important. As an example, the original data sets from the initial gamma- interferon trials were felt to give a positive signal, and, as a result, use of the drug on an off-label basis ensued. Not until the negative results of the larger INSPIRE trial became known was an unproductive clinical practice discontinued.

CAPACITY 1 and CAPACITY 2 demonstrated discordant results. While CAPACITY 2 showed a statistically significant reduction in the rate of decline in the treatment group as measured by pulmonary function testing, CAPACITY 1 did not show any significant differences between the treatment arm and placebo.

While the history of pirfenidone may be shared with patients, the wisest course of action remains to await word from the FDA and counsel patience.

Dr. Imre Noth, FCCP,
NetWork Chair;
and
Dr. Stephen Frankel, FCCP,
NetWork Vice-Chair