Amyloidosis and MDS. Two blood disorders that multiple myeloma patients and caregivers should understand. Amyloidosis, because one out of five myeloma patients also have “amy.” And MDS, because as many as 15% of us may develop this precursor to leukemia, caused by the drugs we take to fight our myeloma.
I received this urgent letter from loyal reader, Pete Wirth. I wanted to share his call to action:
Pat –
Thought you might want to be involved with this as we all have a personal connection with amyloidosis. Please help spread the word.
Any questions ask Paula Schmitt, pschmitt_98@yahoo.com or Muriel Finkel, Muriel@finkelsupply.com as these are the folks who sent the info. Sample letter at end. Thanks
Pete Wirth
Wife diagnosed with cardiac amyloidosis, Oct. 2010
To all who are affected by amyloidosis:
Today I attended a meeting at the FDA that gave us new and important news for all Amyloidosis patients. We need your help before the November 1 deadline. This email explains the situation and how you can and should participate for your own good and that of all our patients. We CAN influence the FDA drug approval process.
Please write a letter or email, in your own words, telling the FDA about your particular experience with Amyloidosis. Don’t be worried about word choice or grammar. Just tell it like it is. They want to hear directly from the PATIENT what it is like to live with the disease, your symptoms, how you get through your day, what you can and cannot do, anything that will help them to really understand what the person suffers. And what the family and caregivers go through. We want to show the FDA that amyloidosis should be selected as one of the 20 diseases they should focus on.
Those of you who submitted testimonial letters or actual testimony at the tafamidis Advisory Committee meeting should be able to use the same letters as a basis for this email or letter.
The deadline for you to send your email is November 1. That is next Thursday. It may be too late to actually mail a letter, but they may accept it if it is postmarked by November 1. I’ll try to ask that question and post it on the website. Please send your email directly to the following address. The ASG would be happy to have a copy of your letter for our records, but we cannot send it in for you. It must go to the FDA directly from the patient.
Email your letter to: (see instructions below)
In the Subject area write: FDA Docket No. FDA–2012–N-0967
Add the following at the beginning of your letter or email:
Drug User Fee Act
Patient-Focused Drug Development;
Public Meeting and Request for Comments
If you are interested in mailing or emailing a letter, I have included samples below.
I understand that this only effects a small part of my readership. But this is the type of direct activism that can really make a difference!
Amyloidosis is an elusive, “shape-shifting” type of disease. It’s similar to multiple myeloma in the sense that it seems to present itself differently in each patient. This makes it difficult to diagnose and treat. Sound familiar?
Feel good and keep smiling! Pat
**********************************
If you are writing a letter to be mailed:
Submit written comments to:
Division of Dockets
Management (HFA–305), Food and Drug
Administration, 5630 Fishers Lane, Rm.
1061, Rockville, MD 20852
At the beginning of the letter refer to: Docket No. FDA–2012 N–0967
Here is the situation:
Recently passed legislation requires that the FDA involve patients in their decision making process as they evaluate the approval of new drugs. As one of the first steps, the FDA will choose 20 specific diseases to focus on during the next five years. They have developed a preliminary list of 37 diseases and have asked for public comment so they can whittle down the list to 20 diseases. We are very grateful and excited that FDA has included Amyloidosis on the initial list.
For each disease area, FDA will conduct a public meeting to discuss such topics as the impact of the disease on patients, the spectrum of severity for those who have the disease, the measures of benefit that matter most to patients, and the adequacy of the existing treatment options for patients. These meetings will include participation of FDA review divisions, the relevant patient
advocacy community – (that’s you) and other interested stakeholders – (our expert doctors, researchers, drug companies, caregivers,)
Then, when a drug or therapy for Amyloidosis comes before the FDA for approval, the FDA will be thoroughly familiar with all aspects of the disease. Their reviewers will be better able to understand how the new drug will affect our patients They will be better able to weigh the risk versus the benefit of the new drug and will know that patients with a rare disease with no cure are willing to take more risk. We will become involved as patients early in the process, well before the final decision is made.
This new initiative comes too late for tafamidis, the drug for familial amyloidosis which was rejected by the FDA in June. On a positive note, we hope that tafamidis will be re-evaluated or that a new trial will be held soon.
Meanwhile there are two new drugs for familial that are fast approaching Phase 3 trials and we want to be ready to support these drugs when they are evaluated for approval by the FDA.
And let’s hope that very soon there will be therapies developed specifically for primary patients and the other forms of amyloidosis as well.
Here is the link to the FDA official announcement in the Federal Register describing the new initiative.
http://www.gpo.gov/fdsys/pkg/FR-2012-09-24/pdf/2012-23454.pdf
Paula Schmitt
pschmitt_98@yahoo.com
Localized Amyloidosis
Atlanta/Jacksonville/DC Co-Facilitator
Amyloidosis Support Groups
www.amyloidosissupport.com
Muriel Finkel <Muriel@finkelsupply.com>
Instructions to submit on “Prescription Drug User Fee Act Patient-Focused Drug Development Meeting” Docket ID: FDA-2012-N-0967
Here are the instructions.
Go to website: www.regulations.gov
In the Search window type FDA-2012-N-0967 This is the Docket ID.
A portion of the title works as well “Prescription Drug User Fee Act”
Click on the blue box “comment now”.
Upload your file (your email or letter) by choosing the blue box “Choose File”.
Note it closes 11:59 PM Eastern Time on Nov 1st
You enter in your information (name, country, state). It requires organization name….. I put “none”. It requires a category…I chose the last one “individual consumer”.
I wish I would have added in the comment field something; even just the word amyloidosis. The program automatically fills in the comment field that you downloaded a document and then continues to thank you and provide your comment tracking number.
Sample letter – Tailor yours to your individual situation.
I am writing this letter in regard to:
Drug User Fee Act
Patient-Focused Drug Development
Public Meeting and Request for Comments
I am currently a 75-year-old male who was diagnosed with AL Amyloidosis in February of 2005 by a urologist at the Siteman Center in St. Louis, Missouri. However, this referral did not occur until a few months of being misdiagnosed and a fight with my cardiologist that he was refusing to take my complaint seriously. I was finally referred to the treatment center by my local physician in_________, not aware at the time that the _________r sees only about 6-8 Amyloidosis cases per year. They placed me on a regimen of Thalidomide and Deximethosone with the dosage supposed to increase over a few months to be followed by a stem cell transplant. This treatment immediately began to cause me serious side effects and I could not imagine what might happen as the dosage increased. I sought assistance from an Amyloidosis Support Group in Chicago and was told by the expert doctors at the meeting I attended that they would never start a new patient on Thalidomide, primarily because it was viewed as a harsh alternative. They also recommended that I go to one of the two Centers of Excellence for a second opinion, either Mayo Clinic or Boston University Medical Center, both of which have depth of experience with treating amyloidosis patients.
I chose to receive my treatment at the Mayo Clinic including a stem cell transplant. Their approach not only spared me from an extended process of receiving a harsh chemo, but also resulted in a complete response. I have remained in remission to this day.
I am convinced that if I had not argued with my cardiologist and caused him to reexamine recent blood tests, which in turn led him to have me take a 24-hour urine test followed by an accurate diagnosis, I would not be alive today to write this letter. The fact is that most local physicians, including cardiologists, do not have sufficient awareness of amyloidosis and therefore it does not register with them to properly consider the symptoms of this disease.
In addition to the awareness gap among physicians, an equally serious issue is that today no cure exists. I am most fortunate to be a 7-year survivor, but that could change, as it has for many.
Thank you for considering Amyloidosis as a target for seeking an effective and reliable cure.
Sincerely, ____________________________
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