make experimental drugs available for terminally ill ALS patients through existing FDA Accelerated Approval Program.

ALS paralyzes and kills about 10,000 Americans per year. There is no treatment or hope. However, some promising drugs are in development. In September 2015 an American company announced an access program for its drug NP001 to European ALS patients. Why not to American patients? One reason: FDA regulations effectively prevent drug makers from charging for experimental treatments in the United States. For tens of thousands of Americans with rare “orphan” diseases such as ALS, this means death, even as life-saving drugs may be available.
We request President Obama to direct the FDA to use its long-existing Accelerated Approval Program to make experimental treatments in development and past Phase 2 (proven safe) available for patients with terminal untreatable orphan diseases such as ALS.