This came up in Pipeliner email today and I think it could be at least thrown out there as a lay person's thoughts. It would be nice if they could be addressed in some fashion on online pd research.

While we have kinda half kiddingly said to each other in pipeline over the years, "if the placebo is that strong, then give us the placebo." Of course that wouldn't work. But why not use the placebo along with the therapy and keep trials open label all the way through?

Phase I always works. So then they mess with it and the next thing you know the drug is gone. Wouldn't it be better to keep going, with everyone getting the treatment, and see who responds the best, always establishing safety first of course? Wouldn't the medicine win out over the placebo eventually if it was going to work?

Phase 1 - establish safety. Open label, usually lasts 6 months or more. Measure differently and more thoroughly posting results online immediately.

Phase 2 - more people, still establishing safety but also dosage experimentation. Open label. Let the placebo do it's job. Patients are thoroughly indoctrinated about the placebo and phase 1 continues. With gene therapy that's a given of course but watch them closely for signs of change - like the placebo wearing off or the gene therapy doing it's job. Why do we really care or even pretend to know at this point which it is? if the patient is getting better, we will find out eventually and if we don't - more about that later.

Continue Phase 2 for the necessary length, using different dosages. Again, more people may respond positively because of the placebo. By using the placebo, we may learn how to identify it. Instead watch for subtypes.....for placebo and treatment effects. In the meantime, if it worked in Phase 1, it's going to work possibly even better on more people in Phase 2. Use more people if it's safe and don't leave out the advanced.

Phase 3 - more of the same with lots more people. Let the placebo do it's thing and keep it all open label. Now you may or may not have an idea of dosage and you may not know if it's placebo or treatment. who cares?


Rationale: pd treatments are temporary anyway. Use the placebo and help relieve the tortures of pd. Researchers say that the placebo effect was in place for 3 years on gdnf. But they had the treatment. The Phase 2 people were blinded and it only lasted 6 months. No one who didn't receive the treatment had a 3 yr placebo effect. Only those that did get it.

If the gdnf patients had a 3 yr placebo effect, I'll gladly take 3 years of relief anyway I can get it. I don't care it it's therapy or placebo - use both if the therapy is safe.

Whose to say that all meds don't involve a placebo effect? When we require an increase in any medication how do we know that in conjunction with the illness progression or whatever the condition requiring the increase may be, that the increased dosage doesn't bring with it more placebo effect?

Let the mind and body work together.

Can someone please confirm for me this statement: Use of placebo is an unwritten FDA rule.

Who would produce a therapy without knowing how much is placebo? Consider that they have been all along. Consider also that without it, the therapy won't work as well or at all.

Do you know anyone who received sham surgery and then the treatment? Sham is the perfect word.

edited to add: scientists say phase 1 open label is not valid science and everybody gets better. Well it's valid for the patients.


Consider these things.
paula