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.