I'd like to know what can be done to find out about the pump? What do we have to do to get this thing out of trials and into the market? This is a therapy with bona fide results, it is not theory. It is already approved for use in many countries and we have other devices that are similar to the ones in other countries that have been rubber stamped. WHY CAN"T WE GET THIS OUT THERE?? MJF where are you? You are an immense champion for our cause and I appreciate all the work that your foundation has done but I think it is time for the PD community to take a stand and say let's go we needed this yesterday!!!

DuoDopa? Just want to be sure to respond to the right inquiry.

Debi

I am assuming that is what you are referring to...We are aware that Abbott is pursuing late stage clinical testing of DuoDopa, as required by the FDA. Recent news items indicate that Abbott may plan to submit for FDA approval sometime late this year. As with the entire PD community, MJFF is eager to see the results of the clinical trial and hopes that results will be positive to lead to a filing and that DuoDopa can be made available to patients in the US. At this point, although frustrating, we have to let the process run, let the trial be completed and results analyzed to move forward on FDA submission.

Debi

This therapy may be one of the best examples of over regulation that I am aware of. It is common practice for the FDA to ask for additional studies be conducted before approval of a therapy to document every assumption and attempt to reduce uncertainty about the safety-efficacy of the therapy. While the goal of perfect (certain) safety- effectiveness is laudable, it is not always in the patients' interest to try to fill in all the gaps in knowledge prior to approval.

About 5 years ago, I was called to meet with the Solvay (recently bought by Abbot) executives from Europe on the occasion of their meeting with FDA officials to market duodopa in the USA. The company was surprised to learn that the FDA was requiring phase 1 safety test and a double blind placebo control Phase 2 study for this therapy which had been used in Europe for 9 years and whose active ingredient is levodopa. Solvay's most senior executive (head of global R&D) was beside himself given the rigidity of regulators' position on these requirements, and he was ready to pull out of the development of this likely beneficial treatment for advanced PWP, who have little else to relieve our worsening symptoms, based on an ethical concern about placebo controls, particularly when we already know that levodopa works. My thoughts at the time were that we all know too well what our body's reaction is to levodopa so the placebo control would be instantly unblinded. What would we learn from the phase 1 and phase 2 studies other than to prove that dopamine replacement works (and works better when continuously infused?)?

Fortunately I had a colleague with me who had been an FDA reviewer for 24 years prior to his retirement and joined the pipeliners and was recruited by the FDA as a patient consultant. He suggested the cross over design that alleviated the ethical concerns of the Solvay executives.

FAST TRACK in FDA terms means that the therapy is conditionally approved while the phase 3 large scale studies are conducted. However,the phase 1 and 2 studies were slow recruiting which not only raises the cost of development it delays the time for use by suffering PWP. This case points out the advantages utilizing PWP consultants, who are still under utilized by FDA and industry. I cant help but think that the tone of the conversation between FDA and sponsors would be different if a patient were in the room to look out for PWP interests. Hopefully the company will take advantage of the faster part of fast track, but so far we have only seen a slow track to the fast track.

How do get PWP on board with something like this. It seems pretty hard in that they would not simply take volunteers to sit in on these discussions.

has three patients in the Duodopa trial. I had a discussion with him about the treatment last week. Based on his experiences with those patients, which included dislocation and kinking of the very fine PEG tube, requiring emergency radiological intervention, he is not at all sure that Duodopa is ready for "prime time".
Robert

Jim,
if i understand you correctly, the FDA selects people who are capable of understanding the science enough that they can evaluate the data presented. The vast majority of PWP consultants to the FDA have Doctoral level training or significant medical background plus their life experience. Patient representatives must also demonstrate some connection to the PD community of patients to be aware of how PWP view the promise of the treatment in terms of the mechanism of action and its likely effect on disease process and symptoms that matter to PWP. The Office of Special Health Initiatives (OSHI) provides on going training on FDA law and procedure, and collaborates with patient advocates [Parkinson Pipeline Project] to keep the patient consultants up to date on science and the interests pf PWP.

Perry

Hi Perry,

I wondered, are you able to tell us anymore about the delivery method. I understood from other discussions that a different method was required for the US as it was not deemed to be acceptable for US patients for some reason. I have no knowledge of whether this is correct or not, but was concerned by RLSmi's statement about kinking of the very fine peg tube, as it was my understanding that european delivery was via a small stoma. Are you able to confirm any of this at all.

Best wishes
Lindy