Thank you for the opportunity to speak directly to the FDA today. To have so many different stakeholders in the room together hearing directly from one another is unprecedented and appreciated.
Today I will share a few observations on information gaps that I think hold us back and three simple ideas to help fill them.
Some Background
We lost Mom to ALS 16 years ago. She desperately wanted to be in a clinical trial. She had no delusions of beating the grim reaper. She simply wanted to contribute so that a young mother or father would not be slapped with a disease that wasn’t a fair fight. She managed to qualify for a trial -- no easy feat for a 78-year-old woman with bulbar-onset ALS. She was ready to start. Then there came a delay. It had to do with the drug company, the FDA, and the pudding that was being used to deliver the experimental medicine. During that protracted delay, Mom died, waiting for the pudding.
Some Observations
Every stakeholder in this room knows his or her own gig well, be it caregiver, a person w ALS, an FDA representative, a scientist, a pharmaceutical company employee, a physician, our not-for-profit staffs. Our problem is that each of us has some big information gaps between our own jobs that we know so well and the other stakeholders' jobs.
Today I heard about the wonderfulness of biomarkers, yet I envisioned a thought-cloud popping up somewhere above the FDA panel that said, "Fine. Bring us one." Today I heard about the urgency of EAPs, yet I imagined another thought-cloud appearing over a pharma person in the back of the room saying, "And why would you buy our cow when you can get your milk for free?" The thought clouds that I see may be way out of line. It’s difficult to know since there are such large information gaps between what I know well and what the FDA knows and what pharmaceutical companies know.
New drug ideas for ALS are announced with trumpets and promise. Then the mysteries begin. Clinical trial design seems to happen behind a big, heavy, black velvet curtain. We know there are things going on back there. We hear occasional noises and sense motion, but we don't know who does what. We suspect that patients and caregivers aren’t even in the cast. When we ask questions, the answer is often, "We're waiting for the FDA." I sometimes envision an impish thought-cloud that fades in right behind those words with the unspoken, "Oh, and we're trying to raise $2 million dollars."
The heavy black curtain finally opens and the trial is at last ready for enrollment. The eligibility criteria sometimes mystify. Sheaves of papers are signed. Patients work hard to fill the trial with peers because they really do understand that every day is precious. The curtain closes again. Even those who have put their own skin in the game by volunteering for the trial are sent to the audience side of the curtain. They have become "subjects."
And we wait. And when the curtain is finally raised again, we have yet to see anything worth a standing ovation.
Some Ideas
1. Make every clinical trial participant a valued project-team member who therefore has a right to know the accurate status of the project and who is carrying the ball today.
2. Give every clinical trial participant the right to sign a paper that says I allow, or better yet, I demand that my clinical trial data be available to other scientists working on ALS.
3. Have patients and caregivers "at the table" during trial designs… and “at the table” needs to mean something different than we healthies picture – a conference room with a nice table. With ALS, gravity is the enemy, travel is difficult or impossible, and technology is enabling. We need a different kind of table. People with ALS and caregivers don’t have all the answers, but they’ll ask some darned good questions. They’ll talk to you about things like –
• Can’t we cut the travel during trials themselves. After all, who isn’t within yards of a Walgreen or CVS that can take blood samples these days? And you should really ask the kids about Skype.
• OR Why isn’t there isn’t a standard-of-care issue when you decide to exclude DPS patients?
• OR How about capturing a 10-second patient video daily from an iPad on the kitchen table that will give scientists a lot of context that the FRS doesn’t capture?
• OR Every ALS trial is an efficacy trial regardless of what the phase says it is. Ask any patient. Ask any sponsor. Why can’t we be honest with each other? Can we have larger, more definitive Phase II trials? How about a special ALS phase – Phase Red for ALS?
• OR Why aren’t scientific data gathered at clinics that can serve as control data … or even historical trajectories for trial volunteers?
Some Conclusions
Stakeholders need to stop working behind their velvet curtains, only to come out at press-release time. We need to have all stakeholders at a new kind of table. Information gaps will close.
We all want good science. We want treatments quickly. We want for them to be good business for pharmaceutical companies whose investments are essential.
Today is an important day in ALS history. It needs to be more than important. Today needs to be opening day for a new way of doing business.
The old script has failed. It has been the ultimate tragedy. Let’s produce something different starting today. Let’s raise the curtains, light the lights, and perform like there’s no tomorrow!
I still have some buttons left and would love for them to find PALS who will wear them proudly. I would also love for them to find PALS and support group leaders who will help spread them to other PALS and will help spread the word on the importance of Registry self-enrollment and in clinical trial participation. 
Please send a snail mail address and how many Registry buttons and how many clinical trial buttons you need to alsadvocacy at gmail dot com. You can be assured that your address will be deleted after the buttons are sent. I'm just an individual trying to spread Registry and clinical trial awareness.
