Originally Posted by Perryc

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.