1 (edited by marions Tue, 17 Jul 2012 09:25:26)

Topic: Highlights from the 2012 DIA Patient Advocate Fellowship Program.

Philadelphia, Program theme:  Collaborate to innovate

DIA functions as facilitator by linking disease to biotech and industry.  The Association connects all constituents involved in the process of bringing a therapy to market, i.e. medical diagnostic and device companies, technology and service providers, patient organizations, and more.   

Keynote speaker  Dean Kamen, Founder and President of DEKA Research and Development Corporation.   Dean received numerous technology awards for cutting edge innovations including medical devices; he has been inducted into the National Inventors Hall of Fame. Quote:  “I am always excited to address representatives from the healthcare community.  These innovators are on the cutting edge of breakthroughs in the biomedical and pharmaceutical fields.”  He addressed negative issues with FDA approval; in this case a medical device (bionic arm) which, although not perfect, allowed the patient to gain some physical independence.  It had been denied originally and then subsequently denied on appeal. 

Partnering for Impact in Global Health was chaired by the Director of Global Policy and Advocacy, for the Bill & Melinda Gates Foundation.  Although, they are in possession of my business card, it is unlikely that we will hear from them, as they focus on low and middle income countries with global health issues.  Am I disappointed?  Absolutely, I am.

FDA representative of the office of special health issues commented that in order to balance the clinical trial with the investment public the need for patient input in these developments have become increasingly more demanding. This prompted the outreach for Advisory Committee Meetings Patient Representatives.  These representatives represent the patient’s voice.  (Our Percy is in the process of being approved for one of these committees.)   

In the first fifty years FDA focused on purity of drugs only; pharmaceutical companies only had to demonstrate that a product was safe. Today, the drug review process in the United States is recognized worldwide as the gold standard. Drugs must undergo a rigorous evaluation of safety, quality, and effectiveness before they can be sold.
It takes over $350 million and on the average 12 years for a new drug to the pharmacy shelf.  After the development of the drug by a company, it undergoes about 3 ½ years of laboratory testing.  Only then an application can be filed with the FDA to test the drug on humans.  Interestingly, only 1 in 1000 compounds tested in laboratories ever make it to the testing in humans. 

Under pressure from the AIDS activist, the FDA in 1992 initiated a Fast Track or accelerated drug approval for drugs designed to life-threatening diseases. 
Of importance to us is the ensuing Orphan Drug Designation Program which, provides status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

It is widely recognized that the US is falling behind in research and development of new drugs whereas China and India are excelling. It is predicted that both, China and India will surpass the US within the next few years, as these countries are less hampered by government regulations .

The need for human involvement in clinical trials was emphasized, as at present only 30 % of patients are enrolled in clinical trials. Patients often are skeptical and many times are under informed or misinformed as to the value of clinical trial participation. The declaration of Helsinki obligates sponsors and research professionals to provide study results.  You may read the disclosures on Clinicaltrials.gov.   

Clinical Trial Studies conducted in Europe and the US basically are identical.

We understand that we need to collaborate globally; that 
Industry, institutions, advocates need to work together by emphasizing the benefits and present clear incentives as to why patients should participate in clinical trials. Patient information should include  additional screening during trial and cost reimbursement should be addressed.

It is my personal opinion that clinical trials mainly are bilogical studies however; participants may benefit from a drug not yet approved for the general public.  Patients need to be made aware of how drugs are approved; how their trial participation impacts the patient community globally and how it can provide the platform for a possible cure of this disease.

Conclusion:  communication prior to enrollment in a clinical trial must be improved upon - cost to patient needs to be eliminated - patients should be fully informed of outcome of clinical trial (it is your right.) 

Hugs to all,


Re: Highlights from the 2012 DIA Patient Advocate Fellowship Program.

Thank-you Marion, as always you are above and beyond.


3 (edited by PCL1029 Tue, 17 Jul 2012 20:54:36)

Re: Highlights from the 2012 DIA Patient Advocate Fellowship Program.

Hi, everyone,

I am a patient,and I have already instructed my son to notify this board when the time is come to report the final experience to this board.  I know it is tough to do so right away like Kandre in South Africa did, But you can wait until everything is  back to normalcy,then give us the least information that you can or in details.
The reason is very simple ,Your action really helps us to collect  the outcome and follow the patients to the end so we can provide members and new patients a much needed statistics about the final stage of our disease  ,so they can prepare for themselves emotionally and physically.I think this is one of the ways that patients or caregivers can help this foundation for it's effort  to compile the patient experiences in FULL.

But most important for all of us is ,in doing so, YOU, the patients and the caregivers will actually provide your voice and help  FDA to develop better new oncology drugs for accelerated approval and better quality of life when come to assessment of the patient.

FDA now is trying to use " Symptom endpoints"---patient-reported outcome)" as a disease end point much or so like OS,PFS ,DFS to increase the outcome of treatment .The advantage is that  patient to provide perspective of direct clinical benefit  and short comings of the drugs they use to the clinicians, so they can/or may use the "symptom endpoints" as part of the assessment tool  in treating the patient. In doing so it will provide the maximum clinical benefit to the patient including the aspect of "quality of life" . This is also can be done by you each time you see your doctors and report even the small changes of the disease condition.

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: Highlights from the 2012 DIA Patient Advocate Fellowship Program.

Hi, everyone,
yes, your contribution of your experience in full will help other patients
check the link below: i am just amazing at  the timing of my thinking.

http://ascoaction.asco.org/Home/tabid/4 … LEARN+MORE

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.