No Accelerated Approvals (AAP) for ALS!

1) Call to Action (details below)

2)  Hope NOW will be issuing a new CHANGE.ORG petition that focuses on our current goals.
Please watch for it in your email: “Terminally Ill Patients Denied Treatment” and please sign it right away.

We continue to make progress in our negotiations with the FDA for smarter, faster, and more humane clinical trials. The FDA has expressed support for the study design proposed by Hope NOW and we are now scheduling meetings with researchers and drug companies to expedite its implementation.

However, we have also learned the FDA will not utilize the 1992 Accelerated Approval Program (AAP) due to a technicality called “surrogate endpoints”. This strict interpretation of AAP is closing the door to fatal diseases like ALS which do not have a verified “surrogate endpoint”. ALS treatments need AAP, a “conditional approval” that allows PALS access to promising treatments after a Phase 2 trial.
Please take a few minutes and write to your congress person using the sample letter below. We’ve also listed all the representatives below to make it easier for you.

Thank you for your courage and continuing to fight for the lives of those battling this cruel disease. Together we are creating positive change that will give PALS Hope Now.

Please remember to sign our next petition: “Terminally Ill Denied Treatment” Nick, Jehad, Eric, and Linda (co-founders of Hope NOW for ALS)

CALL TO ACTION:
Congress is back in session and it’s time to remind them this fight isn’t over. Please send the following message to members of Congress and copy your media contacts:
——
Honorable [member of Congress],
Your terminally ill constituents need your help.
HopeNowforALS.org has learned in its FDA meetings that the FDA will not utilize the Accelerated Approval Program (AAP) for ALS based on a little-known technicality called “surrogate endpoints”. With a fast moving fatal disease there is no need for surrogate endpoints to measure the statistical effects of a therapy. Real clinical endpoints (manifestations of the disease) can be used due to the swift progression of ALS.

The FDA’s strict interpretation of the 1992 AAP produces unintended and dire consequences to those with an imminent death sentence. The FDA has granted AAP for the treatment of cancers, HIV, and made ZMAPP available for Ebola patients – but rejects the same consideration for treatments aimed at ALS and other fatal diseases.

Right to Try (RTT) laws are being passed in state after state, but without new federal legislation there is no guarantee terminally ill patients will have access to safe and promising therapies that are still in clinical trials.The FDA likes to tout “Expanded Access” (EAP or compassionate use) as the solution, but “Expanded Access” has many barriers. Only a few people with significant education, wealth, and medical resources are likely to realize any benefit from EAP.

Furthermore, drug developers are often unwilling to provide the treatment for a host of reasons:
• risks to future FDA approval,
• inadequate resources to manufacture and distribute the drug free of charge, or
• a desire to shelter the true manufacturing cost to avoid jeopardizing future market value.

HopeNowforALS.org has conducted four meetings since May 12, 2015 with Janet Woodcock and other senior staff at the FDA. We are focused on two objectives:
• Establishing a pathway for accelerated approval (AAP) for all safe and promising therapies for ALS, and
• Modernizing the clinical trial process bringing it into the 21st century.
California is poised to become the 25th State to adopt Right to Try (RTT) legislation. It’s a step in the right direction but – we need Congress to enact new legislation that ensures terminally ill patients will have genuine access. AAP is a “conditional approval” that allows the FDA to withdraw approval if the treatment fails to perform as expected.

We strongly urge Congress to:

1. Modify the FDA’s authorizing laws to require that all expedited drug development tools, including AAP – with or without “surrogate endpoints,” are utilized for fatal diseases, and
2. Make it clear to the FDA that terms like “expedited” and “accelerated” do in fact mean what they imply – speed.

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