1 (edited by PCL1029 Wed, 12 Dec 2012 15:32:33)

Topic: The Result of the 2nd "patient-focused drug developement" Meeting

Hi, everyone,

There were 25 patient advocate groups presented in the meeting with about 16 additional groups attended via web connection.for the  2nd  FDA meeting yesterday in Washington DC for the topic of "patient-focused drug development" meeting. Seven or eight  FDA officials attended.
I spoke on behalf of our organization this time and gave the patients input  to people who attended the meeting.Since I was the second person spoke about the questions and mentioned the responses from patients how they viewed about the questions, I had a lot  of positive responses from other advocate groups about the suggestions that Pamela , her daughter and Lainy gave through answering the survey I posted.(See, who says your opinions don't count;I wish next time more of the members can do the same and let your voice heard.
Thus, the FDA committee will consider changes of  the questions as follows :

1.What are your symptoms of your disease that have the greatest impact of your life? ( and they will as Pam and her daughter suggested give examples).
2. This question is acceptable to most of the groups at the meeting but will add examples to distinguish the impact between  "quality of life vs the quantity of life" in the daily living impact to the patient.In short which problem affect the patient the most ;quality or quantity first?
3. The question will change to" How are your daily  living be affected by your disease? and anything you would like to add to it?"
4 .this question is related to question #3 too, so Someone suggested to change  it to "What is the biggest fear do you have with your disease still being treated? and give examples. (please remember not all those disease groups who attended the meeting will have the same final outcome as our group.)
5.Most of the people including the FDA committee agree to change "the" and replace it with  "your" to make the question more personalized and that was Pamela's suggestion I mentioned to the committee. (the answer for this question can be obtained by asking the oncologist or the nurse .)
6.Of course, the committee all agree to get rid of the"clinical manifestations"
wordings in all the  questions involved; As soon as I mentioned that is a problem that our patients may not understand what it means,most other advocate groups were agree and node their heads.(see, Pam,Laura and  Lainy,your input on this message board works.) The question may change to " How effective are available therapies at treating the symptoms of your disease? ;and they will add among others such CAM and non traditional therapies which I suggested to the FDA committee.
7. since this question  ,like #6 are asking the same about the usefulness of the available therapies but gearing toward the side effects of the therapies,the committee will refine and make it more patients friendly and will give examples.
8. this is a more tough questions for patients to understand and answer;so dose question#9 But the committee will refine the questions base on the meeting results.

All the questions , once finalized, will be provide to  and  will be used  by the medical professional like oncologist or other rare disease treatment nurses and doctors to question the patients and hopefully get more understanding about the condition of the patient being treated and to assess the treatment option and outcomes for the patient better.

One final note of the 1st meeting on October 26,2012 about choosing the first 20 or so rare diseases to work on as the first "patient-focused" project, there were 2445 submissions to the FDA docket;2000 of them have been reviewed by the seven members review team so far and the submissions cover 83 different disease area.
God bless.

Please know that my personal opinion is not intended nor implied to be a substitute for professional medical advice. If  provided, information are for educational purposes.Consult doctor is a MUST for changing of treatment plans.

Re: The Result of the 2nd "patient-focused drug developement" Meeting

Way to go, Percy! I feel almost famous!Thanks again for attending these meetings and you are making a change for the better for all CC patients! We love you Percy!

Teddy ~In our hearts forever~ATTITUDE is EVERYTHING
Any suggestion I offer is intended as friendly advice based solely on my own experience. Please consult your doctor for professional guidance.

Re: The Result of the 2nd "patient-focused drug developement" Meeting

Wow Percy! I am so glad they listened to our input and I hope next time you ask for help from this board that others will give their opinions as well. I am so happy they changed the questions so they are more understandable. Did I tell you yet today that you are AWESOME and AMAZING!!

Love, -Pam

My beautiful daughter, Lauren Patrice, will live on in my heart forever.

My comments, suggestions, and opinions are based on my experience as a caretaker for my daughter, Lauren and from reading anything I can get my hands on about Cholangiocarcinoma. Please consult a physician for professional guidance.

Re: The Result of the 2nd "patient-focused drug developement" Meeting

Thank you Percy! You are a star! And like Lainy says, we love you loads!!

You better be taking good care of yourself as well now my friend? Otherwise, you know I'll be on a plane headed to O' Hare to kick your butt! In a friendly and caring way of course! smile

Take it easy Percy and many many thanks once again!

Gavin

Any advice or comments I give are based on personal experiences and knowledge and are my opinions only, they are not to be substituted for professional medical advice. Please seek professional advice from a qualified doctor or medical professional.

5 (edited by PCL1029 Wed, 12 Dec 2012 16:31:28)

Re: The Result of the 2nd "patient-focused drug developement" Meeting

Hi,

As I said before, we work together as one unit, not for any individual as far as things on this message board goes and this is my  personal belief.  If any success come from anything I did or will do for this web site in the future, it belongs to ALL of us.
And please, for those who are not familiar with our work on this web site,there are a lot of people working behind the scene for things to happen. Stacie and Marion among others; and without your generous donations this may not happen at all. So thank you for your help.
God bless.

Please know that my personal opinion is not intended nor implied to be a substitute for professional medical advice. If  provided, information are for educational purposes.Consult doctor is a MUST for changing of treatment plans.

Re: The Result of the 2nd "patient-focused drug developement" Meeting

Thank you , humble Percy, and to the CC foundation board!

Willow

Re: The Result of the 2nd "patient-focused drug developement" Meeting

Thank you Percy, we are so proud of you and your hard work. We are blessed to have you in our family.
Hugs,Lisa

This Information Is Not Intended Nor Implied To Be A Substitute For Professional Medical Advice. You Should Always Seek The Advice Of Your Physician Or Other Qualified Health Care Provider