[Perryc]

Carolyn and John--

This is a wonderful opportunity and I thank you both for making this forum available, even though you didnt tell us in advance.

Now we can use the opportunity for this forum to invent itself. As a founder of the Parkinson Pipeline Project and Director for the past 6 years, I and a number of my colleagues have been thinking about issues important to development of new therapies and patient centered research and medical care for PD for a long time. I would like to use the knowledge and experience I have gained about the mysteries of FDA decision-making and the motivations of our capitalist system for funding medical products to suggest specific topics for discussion and feedback from people in the PD community who are deciding whether they should participate in a clinical trial or are planning for a lifetime with our constant companion, PD.

Others in the pipeline project or elsewhere may suggest different ways that this forum can be of value to the community and then we can try each to get feedback on what works. As it has always been inside the Pipeline project we will not always agree on what is important and sometimes people dont communicate as well as we would like, and there even have been blowups and bad feelings, but in the end the common mission to improve the treatment options for patients sooner rather than later has kept us going.

The best part about this forum is that communication is two ways so everyone out there who is a participant in the sub-forum can provide information on what patients want and what is the patient perspective on issues regarding developing new therapies. Thus, when our patient consultants are working with FDA or companies that sponsor clinical trials, they can provide an informed view of the "patient perspective" on the issues.

My first thoughts are to suggest broad topics for consideration as focal points for discussion. The Pipeline Project web site has background material on these and other topics, and we will continue to flesh out the issues as we are able to. For example, we have just completed an invited journal article for "Neurotherapeutics" (forthcoming July, 2007) on "Ethical Issues in Clinical Neuroscience: a Patient Perspective" that discusses placebo controls, in particular sham surgery, in scientific research. This is not a hypothetical issue and should make an interesting discusion.

Some Topics are:

1) FDA law and policy that governs the new product development pipeline.

2) Specific FDA rulings and decisions.

3) Methods for measuring clinical outcomes important to patients.

4) Bio-markers and other Surrogate outcome measures.

5) Underlying scientific knowledge for different classes of treatments.

6) Validity of alternate research designs and statistical models and standards of proof.

7) Ethical issues in clinical research, patient rights and protections.

8) Advocacy issues and self-help. What everyone can do to improve their own health.

9)Visions of a patient centered health system, and the pivotal role of clinical trials in management of chronic disease and adopting medical innovation.

10) The importance of information technology in faster cures for chronic diseases.

I suggest we also limit ourselves to scientific and ethical topics relevant only to new or existing therapies and the evidence supporting safety and efficacy of those treatments (drugs, devices, surgery, self-help information therapy--e.g.diet, exercise).

We should steer clear of the controversies, such as when life begins, unless it is relevant to a specific therapy under consideration by FDA.

Reactions?

Perry Cohen
Parkinson Pipeline Project (www.pdpipeline.org)
"The missing ingredient in the development of new therapies is the voice of the patient"