opportunity for patient input – really!

The FDA wants to know how the process for soliciting patients for the “patient focused drug development” meetings is going.

That phrase “Patient focused drug development” may ring a bell with you, but if not, here’s the cliff notes:

As the result of a long process initiated by new directives in the 2012 PDUFA, the FDA announced in 2013 that there would be 20 public hearings between 2013 and 2015 on disease areas that are serious and not well understood or well served by treatments. The 20 specific diseases were winnowed down from an initial list of 39 and public input had been solicited – 4500 comments on 90 different diseases were submitted!!

Each of the 20 meetings was designed to hear from patients: What are your symptoms? Which ones are the worst? What have you tried to alleviate them? How did the interventions work? If you could reduce or eliminate one symptom, which one would it be?

Some of the meetings added a second day as a scientific workshop where medical and research experts discussed the progress of clinical trials in the field, the status of the basic science, and how the patients’ comments had illuminated the situation. There are transcripts and webcasts of each meeting, for example here is the page for sickle-cell disease with links for the full materials.

The FDA has prepared a summary report of the patients’ contributions on each disease meeting and put them together on a “Voice of the Patient” website. They are really reporting the results of these meetings in a huge way though I (L.T.) wonder how many people are following the story.

Now, the FDA is asking how the public feels about the meetings, the plans for the next round of disease meetings, etc. Of course public means ALL the stakeholders including pharmaceutical industry, advocates, researchers, clinicians AND patients. That’s how they got the 4500 comments the first time, and will probably get lots of non-patient comments this time, too.

Based on our experience at the “female sexual dysfunction” meeting on October 27-28, 2014, some of us are emphasizing these main points:

1) Patient participation is a good idea, BUT it can easily be hijacked

2) For example, at the female sexual dysfunction meeting the patients who were chosen for panels (patients of one urologist, largely) were solicited by professional groups with deep ties to pharmaceutical companies and their expenses were paid (I called this “unethical and despicable” at the public comments section of the meeting). This resulted in a homogenous message that was focused on a few talking point. No patients of psychologists or psychiatrists were involved to give a different message about treatment

3) There needs to be wider outreach – but how is that to be achieved?? There needs to be vetting so that the panels are not composed of uniform voices. The FDA needs to hear from lots of DIFFERENT patients or the whole point is moot. At the very least, advance disclosure rules should select as diverse groups as possible

If you want to submit your comment electronically, go to http://www.regulations.gov/#!submitComment;D=FDA-2014-N-1698-0001 and put in your points.

Or snail mail to

Docket No. FDA–2014–N–1698

Division of Dockets Management (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.

Dec 5 is the deadline – let your voice be heard: We want MORE patients, more REAL patients, more DIVERSE patients, more interesting ways to solicit and involve patients!!