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.

Wednesday, January 14, 2015

We Have Reached A Fork In The Road

People with ALS face choices every day.  Most of their options are poor, but they make the best
decisions they can.

The FDA faces choices every day.  They strive for excellence as we consumers and taxpayers trust and expect.

Drug developers face choices every day.  They answer to their investors who expect financial viability while they try to answer to the needs of people with ALS and the FDA.

It's difficult.  Today we have a choice that involves all three groups.  It's especially difficult, but that's good.

Two years ago many of us made trips to the FDA to offer testimonies to those who are trusted to regulate ALS drug approvals in the United States.  Over and over, the people with ALS (who have to pick from lousy choices every day) asked to be allowed to assume more risk in the drug development process.

Genervon is going to ask the FDA for "conditional approval" of their GM6 drug candidate.  Truly, that's a shortcut from the perfect path, but can we let the perfect get in the way of something that might be good?  There are people with ALS more than willing to roll the dice with eyes wide open.  There have been enough disappointing clinical trials for them to know that "promising" does not mean "will work" and "perfectly safe."  There is a drug developer willing to make product with a good safety profile more accessible to people with ALS in a closely monitored situation.  Data will be reported to the FDA.  Nobody is trying to sell a secret magic potion.  It's an option within the FDA regulatory environment that could give people living with ALS today some access to GM6.  If it works, awesome.  If it doesn't work, valuable lessons will be learned.

Perfect?  No.  Good?  Perhaps.

If we don't seriously discuss the options and design a responsible, creative solution, we will have let down all of those people who trekked (many with great difficulty) to Washington to testify to the FDA two years ago.

Your thoughts today to the FDA will be appreciated.  Click here for contact information (contact form or email address are at the top of that page).

In addition here is an online petition provided by a gentleman with ALS for your consideration.

Thank you.

1 comment:

  1. Well written. If you are making a cost/benefit analysis between two options, certainly you should include the cost of both options in the analysis. The cost of not giving conditional approval is not zero.