[CarolynS]

There's still time to register for PAN's Webcast and hear from experts about how the Food & Drug Administration's work Impacts the Parkinson's Community.


Join the Parkinson's Action Network and viewers across the country for a live, educational webcast and learn why the Food and Drug Administration is important to the Parkinson's community! This informative webcast provides a unique opportunity to gain valuable insights into the FDA, the role it plays in therapy development, and possible areas for needed change and improvement.

When...TODAY: Monday, December 12
3 – 4:30 p.m. EST

Where: Click here to register online to view the webcast and submit questions for discussion by our panelists. Can’t join us this afternoon? The webcast will be archived online for future viewing!

Moderator:
Amy Comstock Rick, JD
CEO, Parkinson's Action Network

Panelists:
Marc Walton, MD, PhD
Associate Director for Translational Medicine in the Office of Translational Sciences at the Center for Drug Evaluation and Research, FDA

Michelle McMurry-Heath, MD,PhD
Associate Director for Science at the Center for Devices and Radiological Health, FDA

Cartier Esham, PhD
Senior Director of Emerging Companies Health and Regulatory Affairs for the Biotechnology Industry Organization (BIO)

Jackie Christensen
Patient-Representative to the Food and Drug Administration's Office of Special Health Issues

This program is made possible by a conference support grant awarded from and administered by the Telemedicine & Advanced Technology Research Center (TATRC) of the U.S. Army Medical Research & Materiel Command (USAMRMC), Award # W23RYX1067N603

[Conductor71]

Just logged off. Sadly this was just a waste of time. They spent an hour blathering on about nothing and gave a non-answer to the pointed question on Duodopa.

Laura

[GregD]

I am trying to find a flow chart that was given to me. It shows the process, timelines and costs of how a drug progresses through the system including the FDA. It had a lot of useful information on it. I will make it a pdf file and post it, if I can ever locate it again.

[Perryc]

I am sorry I missed this presentation, but I was out purchasing a car to replace my 1996 SUV, which which had given out like its owner Dx in 1996.

For those who dont know me, I was PD patient representative on the FDA Advisory Committee on DBS in 2000. Since then, I have received information and training from the FDA, Office for Special Health Initiatives (OSHI) on an on-going basis and I have collaborated with OSHI to provide on-going information and support for PD advocates on what PD patients' interests are versus other constituents (industry, scientists, regulators) whose interests overlap with the patients interests.

I realized at that time that the exclusive focus of the PD advocacy community on scientific research at NIH and elsewhere (e.g. DOD-NETREP)was only part of the solution. What the scientists didnt tell us was that after a discovery from basic research, it may take 15 to 20 years to get a new therapy on the market. In fact other than DBS which provides a great relief from symptoms for many, there have been only marginal improvements in treatment options for 40 years since Sinemet was approved.

PWP who have been leaders on this forum helped me form a grass roots network of PD advocates, the Parkinson pipeline project, which has lead the way on providing the patient perspective to decision makers in FDA, not just at advisory committees, but also in pre approval meetings between sponsors and regulators, at critical points in the lengthy and methodical process of approval. These meetings before were totally secret proprietary information owned by sponsors. Although we still have some problems to iron out, to maximize the influence of PWP on the regulatory processes, the changes in FDA toward more transparency and patient centered outcomes has been substantial.This has been a part of a larger paradigm shift from doctor/scientist centered research in the 20th century to a patient centered paradigm in the the 21st century. This patient centered approach is more suited to the unsolved health problems of continuity of care and integration of services that challenge our broken health care system and its inability to address the needs of healing from chronic disease. My recent efforts have focused on greater engagement with patients in activation and self help, so called information therapy supported by information technology to enhance individualized patient-provider interaction and choice. I have been working with the 'Working Group on Evidence Based Medicine ( a coalition of patient advocacy as well as PDF to expand the efforts of the pipeline project to inform patient advocates. I am glad to see that PAN is working on FDA issues because they are so important to us. I hope some people found the seminar to be valuable.

Perry

[Conductor71]

Appreciate all your work on our behalf. I am wondering why it takes eleven plus years for the FDA to "Fast Track" a treatment used for a decade in Europe. Why would it need meet both safety and efficacy trials all over again?

Laura

[jeanb]

Here is their contact info:

CDER - Office of Translational Sciences - Organization - Text version
The following header reflects the organizational hierarchy.

DEPARTMENT OF HEALTH AND HUMAN SERVICES
FOOD AND DRUG ADMINISTRATION
CENTER FOR DRUG EVALUATION AND RESEARCH
OFFICE OF TRANSLATIONAL SCIENCES



Main Tele: 301-796-2600
Fax: 301-796-9907

Marc Walton, MD, PhD
Associate Director for Translational Medicine
email: marc.walton@fda.hhs.gov

Address: WO Bldg 21 Rm: 4554
10903 New Hampshire Ave.
Silver Spring, MD 20993

I"m going to write to them (send a fax) and ask about Duodopa. A good treatment is being withheld from us.

Jean

[Conductor71]

Jean,

Thank you! Jim018 and I are on the verge of starting a e-petition. It was so frustrating to not be able to interact and say "but with all due respect you have not answered the question." I also expected Amy Comstock to hold them accountable...ha!

Laura

[girija]

Laura,

I was also disappointed to see that issues like Duo-dopa, patient involvement in clinical trial design were not answered directly by the FDA team. But I am not surprised because they follow "rules and regulations". I felt Jackie and Amy were excellent in bringing out the issues important to PWP, and I think the FDA team got the message. This meeting didnot resolve any issues, I look at it as a good start for further discussions. Your idea of starting a e-petition is great and it is now FDA needs to hear our voices.
If I remember it correctly, Dr. Michelle McMurry-Heath mentioned that the approval guidelines for devices are different between Europe and USA. I think European trials look at Safety while US trials have to show both safety and efficacy. May be Duo-dopa falls into that category and hence more trials?

Please donot think I am defending the FDA.......I am trying to understand where the bottlenecks are. It may be worthwhile to contact Abbott to get more information on their current Phase 3 trial. Working with Abbott and FDA probably is one way for a quicker release of Duo-dopa for treatment.

Girija

i

Originally Posted by Conductor71

Jean,I

Thank you! Jim018 and I are on the verge of starting a e-petition. It was so frustrating to not be able to interact and say "but with all due respect you have not answered the question." I also expected Amy Comstock to hold them accountable...ha!

Laura

[jeanb]

I'll gladly sign a petition - i sent the Dr at the FDA an email this morning...

Jean

[Conductor71]

Originally Posted by girija

Laura,

I was also disappointed to see that issues like Duo-dopa, patient involvement in clinical trial design were not answered directly by the FDA team. But I am not surprised because they follow "rules and regulations". I felt Jackie and Amy were excellent in bringing out the issues important to PWP, and I think the FDA team got the message.

If I remember it correctly, Dr. Michelle McMurry-Heath mentioned that the approval guidelines for devices are different between Europe and USA. I think European trials look at Safety while US trials have to show both safety and efficacy. May be Duo-dopa falls into that category and hence more trials?

It may be worthwhile to contact Abbott to get more information on their current Phase 3 trial. Working with Abbott and FDA probably is one way for a quicker release of Duo-dopa for treatment.

Girija

i

Thank you Girija. I don't want to in anyway make us appear "unstable"; we already battle enough with image problems. However, I won't let this rest until they give us a viable answer.

I think that Dr. Walton mentioned that the do take into consideration research and trials from other countries, but this in my mind makes it all the more puzzling their non-answers. The other good doctor did indeed point to standards. It could be argued; for example, that the efficacy requirement is why we have search a dearth in treatment innovation. Further, if efficacy is so profoundly important, would not data be more robust if a drug or treatment went up against levodopa? Neither of their responses adequately addressed the question. Solvay received orphan drug status approval for Duodopa on 1/18/2000. It is now almost 12 years later...the FDA is worried about efficacy? It has been used and studied in real live patients for nearly 10 years now. I fail how we can be even basing decisions on clinical trials when there are now positive longevity studies on the pump treatment. Quite honestly, not to be harsh, but it insults one's intelligence.

If they even said well it involves a medical device that needs more study; I can reluctantly accept that. Right now, they look profoundly out of touch with their constituents' needs. Not that I wish anyone harm, and I know ethically it is wrong, but these people need to see what we go through first hand. I wish they all had to live a day with it, just a temporary 24 hour dopamine blockade...I know that is extreme, but these folks have entirely too much power; they hold our lives in their hands.

One thing they did not mention is that the FDA refuses to participate in clinical trial reciprocity. How can we truly have translational science and medicine without this?

There is a ton of good info here from this Think Tank: FDA Review.
I am also thinking of contacting Marcia Angell.


Thanks again for being the voice of reason!

Laura