ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Tuesday, September 24, 2013

Let's Have The Most Painful Meeting Ever

Since this seems to be the year of meetings to defeat ALS, maybe this concept of two serial meetings would actually move us forward together.

Let's start with a virtual room full of people with ALS, caregivers, some retired pharmaceutical executives, and some retired FDA officials.

Why the retirees?  They know their businesses, yet they have no horses in this race.  They don't have to worry about walking on eggshells around people who are dying.  They can be brutally honest.  They can be informative and candid.

Stakeholders need to stop trying to assume that they know what the others want or need.  They need to talk.

Meeting 1:

OK, let the first meeting begin.  Let's talk about investment in ALS drug development.  Let's talk about the business of bringing ALS drugs to market.  Let's talk about the short-term and long-term financial incentives.  Let's talk about the approval process as it really is.  Skip the Ph I,I,III basics. We hear those over and over but seldom get past them.  Let's talk about breakthrough status and expanded access programs and markets for drugs. Let's talk about how drugs are paid for. Let's talk about how a program that is good for a rare cancer drug may be not so good for an ALS drug.  Let's hear the heartburn that each special program brings to each person in that room.  Let's learn from one another.  Let's tell it like it is.

That's a pretty painful meeting for all parties, but it would be tremendously constructive.  Today patients dream dreams about programs that they think could bring drugs to them.  Are they even viable?  Are they conducive to industry investment in ALS?  Are they products of an FDA that today doesn't have good "special" programs designed for an ALS?  Are there paths we've not considered.  What do patients really want?  What do drug developers really want?  What do investors want, today and tomorrow?  What does the FDA need? What does everyone need?  

All the cards and reality need to be on the table.  We need to be candid with one another.  That's a constructive, uncomfortable, painful meeting that has never happened before.

Meeting 2:

Ah, now we're ready to design some solutions.  Now that we've stopped assuming and mind-reading, we can start the creative process.  Let's break through the maze and find a path to get therapy candidates to patients more quickly.  Let's not try to bend existing programs to fit ALS if they don't really serve all the stakeholders well.  Let's move the fight forward creatively on solid ground.


We've had far too many variations of Meeting 2 already this year.  We need a Meeting 1.


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