A Funny Thing Happened On The Way To The FDA...


Miracles DO Happen - Turning the Tide With the FDA for EPP.........

Desiree Lyon Howe's interpretative overview of circumstances leading up to the 'FDA Rare Disease Patient Advocacy Day.'

Before you read Mike's outstanding description of his and Mike and Steve Ferry's visit with the FDA, I will briefly overview the circumstances we faced concerning the EPP clinical trials. Clinuvel had just been informed by the FDA that they would meet about the Phase III trials in May, which precluded any trials in 2012. I was in despair for a few minutes. Then I was reminded that God was in charge, so I said a short prayer and picked up the phone. The first thing I discovered was fortuitous, namely, the video Mike Kenworthy, Matt Johnson and Porphyria expert, Dr. Robert Desnick, had filmed last year had not been posted as promised. Now, I had the complaint that the trials had been placed in the "dark hole" along with the video and the 100 patient letters that had been sent to the FDA.  I was afraid they had all been placed together, and they had been forgotten about.

I realized the first office I contacted was useless, because the woman in charge had the standard FDA "we no can do," language down pat. Then I thought of the Special Helps Office and called the number immediately to explain our story as poignantly as possible, including our fantastic missing video that had been shuffled to nowhere. I reminded them that their FDA website read clearly that they were working to facilitate rare disease drugs. EPP, I told her, was rare and was being ignored. We needed their expertise and assistance. In the process, I discovered an FDA Advocacy Day and promptly asked Elizabeth to register our APF members, Steve and Mike Ferry and Mike Kenworthy. Having spoken to all three men, I had no doubt that when they talked, people at the FDA would listen.

My hopes were dashed again for a moment when Elizabeth told me they were wait listed in the thousands. But, prayerfully, I called the FDA Helps Office to report that the patients were wait listed.....proving once again that "patients come last." After also mentioning that Beltway agencies rarely heard from people outside the DC Beltway, Dr. Andrea Furia gave us her full attention and placed the threesome in the meeting in a valued spot. The rest you will hear from Mike.

 "A Funny Thing Happened To Me On the Way To the FDA."

by Mike Kenworthy

I had just gotten back from a sailing trip to the Dry Tortugas (no shortage of sun), and there was a voice mail from Elizabeth, something about an FDA Rare Disease Patient Advocacy Day. This was complete news to me but sounded like something I should attend. I called Elizabeth back, said that I was interested, and asked what I could do. Since I was interested, she offered to register me, and I accepted. The next day she told me that I was wait listed at number 4,032 for a meeting with a limit of 200 people. Turns out that the FDA failed to notify the APF about this meeting until just the week before, and anyone and everyone who could spell FDA decided it was incumbent upon them to attend for whatever reason. Who would have thought that there were so many people with rare diseases right there ready to go? Turns out that there weren't so many with rare diseases -- more on that later.

The picture didn't look good. So, we talked to Desiree. She said she would "speak" to the FDA. Short story short, the next day I was in! Must have been something Desiree said. Who knows? In addition she had Steve and Mike Ferry moved from somewhere on the waiting list in the three thousands into the meeting as well. It seems that Desiree is a superb diplomat who knows how to persuade with thinly veiled threats. I figure we had them surrounded now -- more on that later.

The day arrived, March 1st . I got in my car shortly after 6:00am to make an8:30am meeting just 25 miles away. For those of you who don't live in the Washington DC area this might not make sense, but I didn't get there until nearly 9:00am. Fortunately, registration went smoothly, and I was seated in time to hear the presentation on the history of the FDA. It began with the cooling of the Earth and progressed slowly through the late nineteenth and early twentieth centuries. Fascinating, providing one had consumed several cups of coffee. This was followed by detailed presentations on the make up, organization, procedures and regulations of each of the dozens (or so it seemed) departments that make up the Center of Drug Evaluation and Research (CDER) of the FDA. Mountains of statistics and graphs were shown on decades of all types of drugs. A couple of charts even mentioned rare diseases.

The coffee had begun to wear off.  I was beginning to wonder why I was here. It turned out that besides the three of us, there was only one other person connected with patients of rare  diseases, and she was an advocate, not a patient. Finally, after nearly 3 hours of riveting presentations, the floor was opened to questions. I thought, 'At last. Now we will hear about what is going on with rare diseases.' Several people lined up behind two microphones to begin the Q&A session. I was on the edge of my seat. The first lady was from the National Institute of Health. She gave a 5 minute speech on how impressed she was with the work that the FDA was doing and thanked them for their work. I forget if there was even a question. The next gentleman also praised the FDA and wanted to know about some arcane administrative procedure.

This went on for the next several speakers, so I decided that maybe I should ask a question. I went to the microphone and asked, "Was I in the right meeting?" Of course that was rhetorical, so I went on to explain that I was an EPP "patient" and had been part of the Phase II clinical trial for a drug, Afamelanotide, that had proven to be the next thing to a miracle for someone with EPP. I went on to explain that the results had shown a significant improvement in the quality of life (an understatement) with no recorded negative side effects. The trial had been conducted in 2010, and, in 2011, Matt Johnson and I had come and given taped testimony to the FDA (see a last year edition of the APF newsletter). We had been told that the tape would be made available to the FDA and that things would 'move' along, which is 'government speak' for 'until there is nothing else to do.' So my question was, " To whom must I speak and what did I have to do to get things moving?" There was an awkward silence. Then the moderator calmly said that the director of the Patient Liaison Program would address that later in the afternoon, probably hoping I would have given up by then. I thanked him and returned to my seat wondering if my question would even be remembered.  After all, I had left out the most important part:  What an outstanding job the FDA had done.

Before lunch I met Steve and Mike, only the second two EPP "patients" outside of two cousins that I have ever met (Matt was the first). It was as if we had been friends since childhood. I suppose that in effect we were. In fact, it turned out that we all had been in one of the earliest studies conducted in the US back in the early 60's by Dr. Redeker.  As we were talking, a member of the Q&A panel came to us and introduced himself as Richard Klein, Director of the aforementioned Patient Liaison Program. We talked, and he inquired as to which office the reports of the Phase II trial had been filed, hematology or dermatology? We told him that we had no idea but felt that there must be some central database for all trials. Apparently, not so, after all this is the government.

We continued our talk and sat down to lunch together.  And...... we did surround them!  Steve took the left flank; Mike flanked right and I held the center. Steve pinned down a representative from the CDER while I worked on Richard. Mike was doing the same from my right side. For nearly an hour we bombarded them with everything about EPP and Afamelanotide. Fortunately for them, the lunch ended and the seemingly endless presentations began anew, but the message had been delivered and, more importantly, heard. Richard and others had apparently gotten the message and would do something.

The following Monday Desiree called to tell me that things had begun to move - finally, after two years! The FDA was holding a conference call that day with the Clinuvel, the company that manufactured the drug for EPP. There could be only one reason to move so quickly, but no guarantees. Sure enough, the next thing we heard was the Phase III trial had been approved. What a team effort!  Elizabeth handled the logistics, Desiree the "diplomacy," and Mike, Steve and I the persuasion.  Hopefully, this will eventually (in government speak that means many more years) lead to the approval of Afamelanotide for the mitigation, not treatment, of EPP.

There is much more work to do to cure this condition as well as the other seven porphyrias, and we will do whatever it takes.

Editor's Note: The Phase III trials are moving forward and with them...a treatment for EPP, potentially. Already, 115 patient volunteers have registered for the May trials. Thank you! Mike, Mike, Steve and Matt and a special thanks to our Consortium of Porphyria Researchers:    Drs Anderson, Bloomer, Bissell, Bonkovsky, Phillips, Desnick, and other experts Shedlofsky, Tishler, Galbraith, Pimstone, Bottomley, Poh Fitzpatrick and Mathews Roth.


Remember.....Research is the key to your cure!