notme
Extremely helpful member
- Joined
- Apr 3, 2011
- Messages
- 2,605
- Reason
- PALS
- Diagnosis
- 08/2011
- Country
- US
- State
- Fl
- City
- Orlando
Congress Passes Bill to Expedite Approvals
of New Treatments for ALS
Dear Patricia,
The House of Representatives this evening passed critical legislation that will help to speed access to new treatments for people with ALS! The House vote to reauthorize the Prescription Drug User Fee Act (PDUFA) follows Senate approval of a similar measure last week and is an important victory that will bring much needed resources and reforms to the drug development and approval process.
Specifically, the bills passed by the House and the Senate would:
* Improve and strengthen FDA's Fast Track and Accelerated Approval processes to speed the approval of drugs to treat ALS and other serious and life threatening diseases;
* Provide the FDA with the flexibility to approve new treatments quicker by utilizing tools such as biomarkers and other surrogate endpoints that can predict whether a treatment is effective much earlier in the drug development process;
* Help to ensure that ALS experts can participate on FDA advisory panels;
* Direct FDA to facilitate the development of breakthrough treatments by working more closely with companies and patients throughout the drug development and approval process;
* Reauthorize the Orphan Drug Grant Program, which provides funding to encourage the development of treatments for rare diseases like ALS;
* Require FDA to work closely with patient organizations, including those representing rare diseases, as it develops guidance and regulations; and
* Provide much needed funding and resources to the FDA which will help the Agency to expedite drug approvals.
The ALS Association has worked closely with the FDA, Congress, industry and other patient organizations to advance PDUFA for the past 18 months. The Association also developed an advertisement to promote the legislation that ran in Capitol Hill online and print publications at key times of the year, including during our Public Policy Conference just two weeks ago. A copy of the ad, which features Sharon Lockhart, a military veteran with ALS from The Association's Alabama Chapter, is available by clicking here.
We are excited that the final bill includes provisions that are important to people with ALS: that it recognizes the urgent need to find a treatment and speed access to treatments; and that it takes into account the specific needs of people with ALS, who have no treatment options and who have a much different view of benefit and risk than those with treatable conditions.
Next Steps
The bill now heads to a House-Senate Conference Committee which will reconcile differences between the two versions of the bill before it is sent to President Obama for his signature. We anticipate that PDUFA may become law before Congress adjourns for the July 4th holiday.
Thank you to everyone for continuing to tell your story to Members of Congress. Your efforts make it clear why legislation like PDUFA is urgently needed. Your efforts make a difference!
I know this will go to moderation--but I do hope it gives some hope to the many people here. I received this in my email this morning.
of New Treatments for ALS
Dear Patricia,
The House of Representatives this evening passed critical legislation that will help to speed access to new treatments for people with ALS! The House vote to reauthorize the Prescription Drug User Fee Act (PDUFA) follows Senate approval of a similar measure last week and is an important victory that will bring much needed resources and reforms to the drug development and approval process.
Specifically, the bills passed by the House and the Senate would:
* Improve and strengthen FDA's Fast Track and Accelerated Approval processes to speed the approval of drugs to treat ALS and other serious and life threatening diseases;
* Provide the FDA with the flexibility to approve new treatments quicker by utilizing tools such as biomarkers and other surrogate endpoints that can predict whether a treatment is effective much earlier in the drug development process;
* Help to ensure that ALS experts can participate on FDA advisory panels;
* Direct FDA to facilitate the development of breakthrough treatments by working more closely with companies and patients throughout the drug development and approval process;
* Reauthorize the Orphan Drug Grant Program, which provides funding to encourage the development of treatments for rare diseases like ALS;
* Require FDA to work closely with patient organizations, including those representing rare diseases, as it develops guidance and regulations; and
* Provide much needed funding and resources to the FDA which will help the Agency to expedite drug approvals.
The ALS Association has worked closely with the FDA, Congress, industry and other patient organizations to advance PDUFA for the past 18 months. The Association also developed an advertisement to promote the legislation that ran in Capitol Hill online and print publications at key times of the year, including during our Public Policy Conference just two weeks ago. A copy of the ad, which features Sharon Lockhart, a military veteran with ALS from The Association's Alabama Chapter, is available by clicking here.
We are excited that the final bill includes provisions that are important to people with ALS: that it recognizes the urgent need to find a treatment and speed access to treatments; and that it takes into account the specific needs of people with ALS, who have no treatment options and who have a much different view of benefit and risk than those with treatable conditions.
Next Steps
The bill now heads to a House-Senate Conference Committee which will reconcile differences between the two versions of the bill before it is sent to President Obama for his signature. We anticipate that PDUFA may become law before Congress adjourns for the July 4th holiday.
Thank you to everyone for continuing to tell your story to Members of Congress. Your efforts make it clear why legislation like PDUFA is urgently needed. Your efforts make a difference!
I know this will go to moderation--but I do hope it gives some hope to the many people here. I received this in my email this morning.
