An interesting interview with clinical research scientist (and PwP) Prof. Karen Raphael

[jeffreyn]

tmrwedition.com/2018/05/31/...

[Enidah]

Thank you for posting that...a lot of good information.

[Kevin51]

This should be required reading (followed by an assessment!) for anyone recommending any form of treatment for Parkinson's disease.

"If treatment A works better than treatment B, with some level of statistical significance, it just means that the average person got better from A than B, with a pattern and degree unlikely to happen by chance. As I’ve said elsewhere, the ‘average’ person is a statistical construct, not an empirical reality. Rarely is an individual going to know if a treatment is going to work for him or her."

She also says - "Then I found a movement disorder specialist who said to me that “there is no such thing as too much exercise for a person with PD.” "

5 mins to read - lots to reflect on!

[MBAnderson]

Good one. Thanks.

Among other things, she says,

"Mannitol is a good example of this. The people promoting it are building a registry for people to report their experience, but you are probably more likely to actually fill out a report if you have had a good experience. They also said they are going to release the results when they are “statistically significant.” That is nonsensical for anybody who understands statistics, so I am skeptical of the case for mannitol."

and

"Another common mistake is much ado about Phase 2 studies. They are really just preliminary evidence of efficacy. Only Phase 3 studies can give us a good sense of whether a treatment is going to be effective. Replication is a principle of science. I could see that multiple Phase 2 studies by independent investigative teams might be a substitute for a cumbersome Phase 3 trial, but –without replication—the Phase 3 trial is hopefully powered well enough to see whether effects are only occurring at certain sites. This is not a rare disease, where a multi-site study is inherently necessary for recruitment. Now, it could be different for certain precision medicine trials, so that only a single Phase 2 trial is feasible short-term, but you would expect huge effect sizes, not marginal ones, in precision medicine trials, indicating that the treatment is really ‘hitting’ the underlying mechanisms."