Status
Not open for further replies.

tmurphy58

New member
Joined
Aug 24, 2011
Messages
5
Reason
PALS
Diagnosis
12/2010
Country
US
State
VA
City
Gainesville
I sent the following letter to ALSA, NORD, ALSWW, ALSTDI and the MDA - hopefully they will actually attend the meeting today (10/10) and on 10/25 so that their constituents voices will be heard.

=================================

From: Thomas J. Murphy

The FDA is hosting a public meeting on 10/25 to discuss the nomination of a set of 20 disease areas that could benefit from a more systemic and expansive approach to obtaining the patient perspective on disease severity or unmet medical need.

The initial listing Prescription Drug User Fee Act Patient-Focused Drug Development PDF Pages 58849 - 58851 (3 pages) [FR DOC #: 2012-23454] published in the Federal Register this week contained 39 candidates and did not include ALS.

I am writing to encourage the active participation of your powerful organizations and your constituents. All of us persons with ALS would very much like to see the FDA include ALS as a near-term focus area.

Perhaps you would encourage your constituents to submit a formal comment related to the inclusion of ALS prior to the public meeting on 10/25?

Go to: Prescription Drug User Fee Act Patient-Focused Drug Development

Lastly, the Federal Register Notice says: FDA will consider the public comments received at the public meeting and through the docket and post the set of disease areas for FY 2013-2015 on the FDA Web site. By the end of FY 2015, FDA will initiate a public process for determining the list of disease areas for FY 2016-2017.

If ALS has to wait until the end of 2015 to get on the List … at a death rate of 15 ALS Patients per day and 5,475 per year ... approximately 16,425 people with ALS will have passed away by then.

If there was ever a horrific disease that needed some focused attention – ALS is it!

V/r,

Tom Murphy
703.395.3615


If you wish to attend this meeting, please register by email to [email protected] by October 18, 2012. Your email should contain complete contact information, including name, title, affiliation, address, email address, and phone number. Seating will be limited, so early registration is recommended. Registration is free and will be on a first-come, first-served basis. However, FDA may limit the number of participants from each organization based on space limitations. Registrants will receive confirmation once they have been accepted.




FROM FEDERAL REGISTER 24 SEP 2012

WHERE IS ALS?

II. Disease Area Nomination

FDA is nominating the following disease areas as potential candidates for the focus of one of the 20 future public meetings and invites public comment on this preliminary list. In your comments, please identify the disease areas that you consider to be of greatest priority and explain the rationale for your recommendation.

• Pulmonary arterial hypertension.
• Heart failure.
• Primary glomerular diseases.
• Narcolepsy.
• Huntington’s Disease.
• Depression.
• Autism.
• Peripheral neuropathy.
• Fibromyalgia.
• Obesity.
• Nocturia.
• Chronic fatigue syndrome.
• Irritable bowel syndrome.
• Inflammatory bowel disease.
• Alopecia areata.
• Diabetic ulcers.
• Female sexual dysfunction.
• Interstitial cystitis/painful bladder syndrome.
• Fracture healing.
• Diabetic foot infections.
• Hepatitis C.
• HIV.
• Patients who have experienced an organ transplant.
• Sickle cell disease.
• Chronic graft versus host disease.
• Amyloidosis.
• Aplastic anemia.
• Melanoma.
• Lung cancer.
• Cancer and young patients.
• Cancer treatment in pregnancy.
• Cancer and sexual dysfunction.
• Cancer and depression.
• Clotting disorders (e.g., hemophilia A (factor VIII deficiency) and von Willebrand disease).
• Thrombotic disorders (e.g., antithrombin deficiency and protein C deficiency).
• Primary humoral immune deficiencies (e.g., common variable immune deficiency).
• Neurologic disorders treated with immune globulins (e.g., chronic inflammatory demyelinating polyneuropathy).
• Hereditary angioedema.
• Alpha-1 antitrypsin deficiency.


Dear Colleague:

As part of the performance commitments of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), FDA has published a notice in the Federal Register related to Patient-Focused Drug Development, an initiative that provides for a more systematic approach to obtaining the patient perspective on certain disease areas in PDUFA V.

Over the next five years, FDA will conduct public meetings for 20 different disease areas. The meetings will focus on the patient perspective regarding two key considerations in FDA’s regulatory decision-making: the severity of the disease and the currently available treatment options. In determining the disease areas that will be addressed in these meetings, FDA has published a preliminary list of nominated diseases in a Federal Registry notice. The public is invited to comment at a public meeting on October 25, 2012, where FDA will provide an overview of Patient-Focused Drug Development. Registration to attend this meeting must be received by October 18, 2012. For more information about this meeting, including the list of nominated diseases, see the Federal Register notice (http://www.gpo.gov/fdsys/pkg/FR-2012-09-24/pdf/2012-23454.pdf). The public is also invited to submit written comments into the public docket FDA-2012-N-0967 (Regulations.gov). Written comments are due by November 1, 2012.

In addition to the October 25th meeting to “kick off” planning for the 20 disease-related meetings under the PDUFA V performance commitment, FDA will also conduct periodic consultation meetings with patient stakeholders regarding process issues related to the agency’s implementation of Patient-Focused Drug Development. The consultation meetings will address important considerations and challenges in establishing and conducting a process that will be useful to both the patient community and FDA. The first of these consultation meetings will take place on October 10, 2012. Notification of intention to participate in this series of meetings must be submitted by October 3, 2012. For more information about these meetings, see the Federal Register notice (http://www.gpo.gov/fdsys/pkg/FR-2012-09-24/pdf/2012-23453.pdf).

We look forward to your participation in this process. If you have any questions, please feel free to contact the Office of Special Health Issues at 301-796-8460.

Sincerely,
Dominic Cirincione
Ofice of Special Health Issues
U.S. Food and Drug Administration
________________________________________
 

Luke

Distinguished member
Joined
Apr 3, 2012
Messages
314
Country
US
State
Unspecified
City
Unspecified
********bump********
 

rknt50a

Distinguished member
Joined
Aug 14, 2006
Messages
214
Reason
Lost a loved one
Diagnosis
09/1996
Country
US
State
IN
City
IN
Update... and more comments needed before November 1!

ALS Advocacy: Runners Left on Base. We Need To Drive Them Home!

Please continue to submit comments (even if you've submitted already) for ALS to be included in the 20 Patient-Focused Drug Development meetings at the FDA. Please spread the word to your relatives, friends, neighbors, church groups, etc. Thanks.
 

kaylseyum

Member
Joined
Oct 17, 2012
Messages
12
Reason
CALS
Country
US
State
WA
City
Near Seattle
Done and done! Is there a way to view the comments that have been submitted by others?
 

rknt50a

Distinguished member
Joined
Aug 14, 2006
Messages
214
Reason
Lost a loved one
Diagnosis
09/1996
Country
US
State
IN
City
IN
kaylseyum, A very small selection of the comment appears at
Regulations.gov
We know that in the last week that many disease organizations... except ALS... have mobilized their peeps to send in comments
Today (Halloween) is the last day to submit comments. Please, please do so. We need quality and quantity comments to the FDA to explain how people with ALS will add valuable voices to patient-focused drug development!

Submit comments directly to the FDA at --
Regulations.gov

Thanks, all.
 

Bella77

Active member
Joined
Jun 16, 2011
Messages
36
Reason
CALS
Diagnosis
06/2011
Country
US
State
Michigan
City
Fenton
Just sent a comment in.
 

tmurphy58

New member
Joined
Aug 24, 2011
Messages
5
Reason
PALS
Diagnosis
12/2010
Country
US
State
VA
City
Gainesville
Status
Not open for further replies.
Top