HAFFNER/BELINDA : BELINDA/CPATH'S WOOSLEY : VONESCHENBACH/BELINDA
JANET/JOANN : SUSAN/BELINDA/JANET
BELINDA/ABBEY MEYERS NORD : CAMPBELL/DR BRADY/BELINDA : BELINDA/JRF
SLIDE SHOWING GENOMICS DISCUSSION 

THEIR STORY WAS OUR STORY
The 2006 NORD conference was a unique time for describing the MP to others, as much of what was presented by key regulatory speakers showed the flaws of status quo, and predicted what we of the MP are living now (molecular genomics changing medicine at its core).

Acting FDA commissioner Von Eschenbach described the history of medicine in 3 stages: 1. macroscopic (using 5 senses to discern) 2. microscopic 3. 21st Century Genomic. He also said that on "the molecular level, no disease is rare,” and in the future all diseases would be handled individually. John Gallin, MD of NIH echoed that statement with "Everyone will have a rare disease eventually."

Belinda caught Von E.'s attention when she boldly asked if he realized just how true his words had been. He responded positively that he knew exactly, and gave her time for a brief discussion and photograph while he hurried off to Congress ... much to the dismay of the CNN press affiliate who wanted the brief time talk to him. Von E. had used the metaphor of caterpillar and butterfly, that it was the caterpillar's destiny to become a butterfly, but we have a choice. And there was Belinda, quickly capturing the opportunity and far more brilliant than a butterfly. Her transformation on the MP was definitely by choice.

OTHER CONTACTS 
Admiral Haffner of the FDA OOPD gave a brief history of her dept and some idea of rare-disease, small market successes she has been involved with. She suggested talking to OOPD early in the process for grants program and recent efforts for a new initiative with parallel development and joint applications in EU and US (EMEA & FDA). Belinda and I noticed of the 13 staff members pictured in OOPD slide, 9 were in Corps uniform.

NORD staff had the opportunity on several occasions to see and hear us take part in the discussions. Belinda and I both asked pointed questions to presenters via microphone so all in attendance heard our statements.

BLINDING & SPEAKING
Belinda asked just how can studies be blinded in the future with the Internet? This was reiterated with several others in the meeting and one woman said she felt patients should communicate with each other when they were involved in a study.

Rino Aldrighetti, President of Pulmonary Hypertension Association, is using the Internet for collaboration. He said "sharing information is good... but clinical trials may be the exception to the rule." Their 1999 list serv for patients on a new drug trial resulted in patients talking about the trial, "almost lost the validity of the trial as details moved toward becoming public." (Interesting perspective of what happened). He also said "In an improving environment, flexibility in the creation of trials is critical to continuing advancement (eg. non-placebo trials)." Technology is clearly changing study design, and old regulations are challenged now.  

I took the final speaker, but we wrapped up with pictures and after-presentation discussion of the dilemma that happens when a small group controls Sarc publications. There was agreement several impasses had to be moved in both regulatory bodies and industry settings.

LATE STAGE LYME
Susan A. drove up from her home and spoke with many about her recovery from Chronic Lyme, specifically OSHI JoAnn Minor's friend that had heard about this from Lori before the conference. Susan is on the sampler Conference CD Belinda passed out to many as well so they had a chance to see her and hear her.

EFFICIENT COMMUNICATORS
Karolinska poster handouts, Dr Marshall's/CDER presentation, newly produced conference samplers, MP/Th1/D, the annotated bibliography of secosteroid D, MP Phase 1 laminated sheets, business cards and Ms Dale’s recovery story with photographs were distributed over the span of the conference, starting with Thursday evening in the exhibitor’s hall. These seemed the most effective to describe Dr Marshall’s breakthrough and were tailored to the individual audience (NORD members, other rare disease representatives, doctors, regulatory officers, those seeking a diagnosis for disease that was stumping their doctor, and those with rare diseases).

Belinda showed many her progression through disease to wellness with photographs she had compiled of her own before and after (all of us can do this). Wrotek’s screen stills from the CDER helped establish the FDA/CDER Visiting Professor presentation for enlightenment on these key charts.

POST-CONFERENCE
Some hope for impacting off-label legislation with NORD is expected and I will attend the phone conference for that on Oct 10. Most presenters were focused on the angle of developing new drugs, so resolving this niche of a niche problem with off-label is important. Off label uses with existing drugs (like those of the MP) do not get attention or funding when new drug development demands the purse. This step right out of the gate from the conference has some promise for the MP because of contacts and efforts made to be at the NORD meeting.

Contacts with exchanged business card action planned, as are thank you notes and move forward communications. 

I would like to express deep appreciation for those who worked to make a significant difference for the MP while we were there. Their help was invaluable.