Medical trial metrics

The following explains basically what kind of analysis is done on the data from a trial to evaluate how successful is a given treatment. If you read the links below, in bold red, you are set. The others are for context, etc. (you can skip as you gotta get moving :) )

1. Metrics: Definition of the outcome parameters of a clinical trial (endpoints) to judge a given treatment. Things like OS, PFS, ORR... Basically these are measures till a given endpoint (when something happens to the patient). For instance, time till the patient dies or the tumor disappears... (for the math see #2):
1. A paper giving a bit more of background on this. 
2. And another document from fda giving even more info. 
3. The Clinical Viewpoint: Definitions, Limitations of RECIST, Practical Considerations of Measurement
4. Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies?
2. Statistics: The above are the parameters to be analyzed, but what are/how to understand the statistics of those parameters?
1. Hazard Ratio (HR):  the chance of an event occurring in the treatment arm divided by the chance of the event occurring in the control arm. A nice explanation (but no math) with examples is here. "A hazard ratio of 2 means that a treated patient who has not yet healed by a certain time has twice the chance of being healed at the next point in time compared to someone in the control group." "The hazard ratio is equivalent to the odds that an individual in the group with the higher hazard reaches the endpoint first." Note: odds in gambling is the ratio of something happening vs not happening. For instance, a 2 to 1 odds ratio, means that the probability of something happening is double than not happening but that actually means that it will happen 2 out of 3 times.
1. And check this out for math with examples.
2. For the hardcore math, maybe this paper can help.

Work in progress but for the time being...

Cheers!

About this blog FAQ

(5/2019)
Thought I would give you a bit more background about me and the blog. Not really an FAQ :)

• The idea in the blog is to start top down, collecting the pieces of knowledge that I realize I miss and dig on whatever I need/feel to. Hence the resulting randomness of topics almost like exploring different portions while creating a map. Call it a "back of the envelope to biology".
• This is the reverse of the traditional bottoms up approach trying to create a solid base but at the expense of huge energy and time. Thick books, lots of new words to remember... can become dreadful really quickly. In a sense, this approach is no different than any other scientific matter, like mine, electrical engineering, for instance. It can be very discouraging. 
• I don't intent to create any new material or come up with a new theory of something. It is more about creating a map to give you the answer as fast as possible. For example, researching a topic I'll dig through different papers and then just put in the blog the best one or ones (IMHO). Something like "read this to understand how to sequence DNA".
• I am an electronic engineer who always wanted to dive in biology, genetics, etc. Probably the best word to describe it is "fascination". As an engineer it is easy to realize that nature's engineering, specifically behind life, is far beyond our knowledge and superior in many aspects. And as such, I started digging on this topic to try to answer some of the many questions I had and find what is actually the latest on our knowledge on this area. But I feel it should be easier. Answers shouldn't be hidden by pages and pages of supporting information. So, I created this.
• Disclaimer: for you, the reader, please understand that I am no expert in this matter, so, take everything with a grain of salt! As mentioned, my intention is actually more to create a map to point you to the right places, than to actually re-write or create any content. And for those experts on the matter, please apologize all the shortcuts and simplifications, or even errors. Please comment! No issues... (wondering if I could create this in a way that folks could contribute, but certainly don't want to create a parallel Wikipedia...)
• Why now (May 2019) after all these years? Well, my dad got diagnosed with ccRCC (yeah, a cancer). So, I felt it was a good time. Honestly, I had plans already to quit in few months my work, but bio was going to be only a side interest. This kind of re-shaped the priorities. Haven't quit yet, though, at the time of writing this.
• Update (June 2019): My dad past away a week ago. A pneumonitis took his life and I still quite don't understand what happened. May had been radiation induced (from the radiotherapy to the spine) or due to bacteria/virus (pneumonia). I will investigate that later. This blog will be now dedicated to his memory.
• Why the name cell0908? Pufff... A bit of long story. Originally I created Cell0907. This was a blog supposed to collect a lot of random thoughts, work, stuff... related to me. I was Cell0907, one small "cell" in this world and was trying to contribute to the community. One day (back in 2013 or so) I started typing the work I was doing in Android and OpenCV, for my own reference and in case it could help anyone, and soon got many readers. Then I started creating cleaner and nicer posts and the audience grew a lot, making Cell0907 mostly about programming. At one point one of the pages was popping in the first page returned by Google for the topic I was working in! Anyhow, it has been about 4 years that I didn't post anything but still get hundreds of visits a week, so, I thought that instead of creating a new topic within Cell0907 I would create Cell0908.
• A lot of this blog revolves around cancer although this is hardly the worse thing that can physically happen to a human being. There are many devastating other diseases and I invite others to do similar work to this, here or elsewhere. At the same time, a final reflection... I have no doubt that we must work towards ending these plagues and the suffering they bring. We have no other choice. But as I type this, there are transcendental questions that pop just by thinking on the possibility of one day conquering such diseases: aren't those bad times needed to support the good moments in life, what our life would be without death, will we lose our spirit (always alive but without understanding/appreciating it), can society survive if their members live significantly longer, etc. And beyond us, maturity of the human race has to pace and be a prerequisite to the progress on the cure to these illnesses, or we run the risk of we, ourselves becoming a cancer to this planet. We should invest more on that.

Now back to work.

Cell0908 blog topics

Are you the kind that is doing pretty good in your life but would like to do something much more meaningful? Say an engineer working on shiny objects which pay the bills but feels that you can use part of your energy, time and knowledge on something much more impactful? Call it health, economy, social issues, our planet...

A back of the envelop to biology, the target of this blog is to help someone with little knowledge in biotech, ID opportunities for contribution and cover the knowledge map as fast as possible (the 80% of the learning curve :) ). It is not to create new material but to point you to the answer (curate) or at least the direction. Will divide each blog in different levels of info (separating top from deeper) so you can stop right at the level of interest (call it the highlights), without having to read unnecessary info. In the pages, the links in bold red are basically the answer with the rest being support stuff  which you can skip if you prefer to save your curiosity bandwidth for something else :)

All is (and will always be) work in progress (you will see half way cooked pages published if I think they already bring up useful info). I am writing this as I am doing that for myself (as I am no expert either) in case someone wants to follow my footsteps. Of course, the knowledge map is very broad so your topics and mine may not overlap, but I list here the ones I work on (as I cover them) or plan to cover (if you do so, let me know and I'll just link to your page or add here, giving you credit, whatever you prefer):

1. Biology FAQ (all are answers to questions but here I put some to open the appetite)
2. Bio sizes - Just a small post to put in perspective the size of different biological things.
3. Molecules and more molecules
4. Human body cells FAQ: how many cells are there, how often they split, how many different types... 
5. Basics on immune system
6. Stem cells
7. Metabolism: calories, etc.
8. Life sciences instrumentation and In Vitro Diagnostics (IVD, aka "all those tests that your doctor ask for you when you are sick"... and more)
9. DNA Sequencing
10. Developmental biology: ever wondered how we go from an egg to a full body?
11. Why we age
12. Cancer
13. Drug delivery
14. End-of-life rallies: recovering just to die soon after.
15. Anatomy/Human body systems:
1. Shoulder
2. Renal/Urinary
1. Kidney
2. Prostate
3. Urethra
3. Circulatory:
1. Capillary network
4. Skin
16. Different science branches
17. Good article on "When does life end?"
18. Jargon (work in progress, just a collection of terms, probably don't recommend just reading that but hey, knock yourself out...)
19. Clinical trials
20. Legal (medical devices, drugs... approval)
21. Reimbursement/costs
22. Links (some of them are treasures)

Fortunately there is a lot of innovation on this so check out the date** as I am sure it'll get quickly outdated and I can't keep up... :)

For more background on this blog, check this FAQ.

A final note, many of the posts may cross subjects, touching info that could fall on several posts. For instance, prostate anatomy may not warrant a single out post only for that (one can just read Wikipedia) but may show up in relation to prostate cancer or when talking about prostatitis. So, I recommend if searching for something specific, to use the "search" box.

Cheers!

** Don't you hate when articles in the Internet don't post the date?!? It is funny to read "next year the real state cost will increase by 10%" but not be able to find what year they are referring to. Anyhow, one of my many pet peeves...

Last update: 1/14/2023