Ann: Prima BioMed's CVac granted Fast Track D, page-57

re: Ann: Prima BioMed's CVac granted Fast... I’ll try again with that post to your question Kbear1.

In theory subjects have to meet the inclusion and exclusion criteria 100% of the time. In practice where a slip up happens this is a protocol violation and this is a bad thing.

In early phases criteria are usually fairly broad and studies are conducted at perhaps 1-2 sites. The actual selection is perhaps more art than science.

As studies get larger, the criteria become more tightly defined – more objective than subjective and able to be operationalised by staff on the ground at say 25 sites across 6 countries.

Your question about patient selection into Can-004 influencing the outcome of that study is a very hard question to answer.

Most people would be thinking that the outcome would be more influenced by whether Cvac is better in second remission than first remission or whether OS is a better measure of Cvac efficacy than PFS.

So whether patients are young or old, rich or poor, motivated or not etc I don’t think is front and centre here.

People would also be thinking about contextual factors and how these might systematically skew the results.

Lets imagine that Belgium has a much better health system than Bulgaria. Belgium subjects might be expected to be younger (because problems are detected earlier), healthier (higher standard of living) and wealthier (able to access better health care) than women from Bulgaria.

The standard of care group we can imagine is going to be fairly ordinary in Bulgaria but first rate in Belgium. Who do we want more in our trial? Its highly unclear.
In P2b conventionally you would say the poorer the SOC group is better.

And this is how many cancer studies get good results in P2b – because not only do subjects get the active their general health gets looked after very well. So rates of health service utilization of subjects in the active arm is always far higher than SOC subjects. And this leads to better outcomes in itself.

And you see this clearly when outcomes drop off when you have a blinded placebo control group where both groups are now getting equal access to health services that is usual in P3.

And its particularly difficult for cancer vaccines. If you look at Yervoy PBAC broke with tradition with its approval. For the first time it said the drug is approved ... but we want to see how its use in the real world translates into benefit. In other words they don’t trust the efficacy data coming out of the trials which is produced in other countries, perhaps tailored to selected patients to generalise to Australian patients. And so the company will be paid on actual results.

So imagine I am trying to sell you a new set of golf clubs. I say they have been proven in trials to hit the ball 30% longer. You say okay but I am only going to pay you on the basis that I actually hit the ball 30% longer. Now given you might have a perfectly good set of clubs now .. mine may not be an improvement at all. Or your contact might be so poor it doesn’t matter what clubs you have you are still not going to hit the ball very far. We might also start thinking that new golf clubs might not be the answer here but golf leasons might help.

So for exactly these reasons you can see why big pharma hates this idea just like golf club manufacturers would not like to be paid on results. For a start my new driver would be being returned – along with my new putter next.

But the tax payer should love this idea. Because they pick up the tab for $100,000 cancer treatments which theoretically are supposed to extend life by months but in practice may not even achieve this. But the political pressure to allow people to access them is so high that regulators basically can’t stop them. So this pay for by results is the compromise.

Along these lines you might also think outcomes may have also influenced why Can-004 is in Europe to begin with. Yes the German grant helps .. but if Can-003 was showing a little bit of advantage in European countries over say the US even though you might not have foggiest clue why (patient factors or SOC treatment) you would be inclined to upscale where its working best.

I’m sure you didn’t mean to ask such a hard question Kbear1.

Hi Macenroc yes I self moderated that post. It contained a funny story that you would recount to a couple of mates over a beer on a Sunday afternoon.

I wouldn’t worry about credentials one iota on HC. Posts make sense, are well argued, are funny or entertaining or they’re not. And one problem with that post was that it was appealing to credentials and big noting myself and thats just not the way to make a point.

Why I am interested in PRR? Probably for the same reasons as you. It is doing interesting things and I learn from it. The interface between science and the market is fascinating. But I am also interested in QRX, PBT, IVX, PXS, POH, ACL, BLT, ISN but can’t be across everything ... so post on a few. I have actually posted a lot on OBJ and PNO in days gone past because they are just great fun companies and great fun forums and look nothing like what I get paid to do,

If PRR were developing a new toothbrush I wouldn’t really be interested in it. Many people much cleverer than you and I think cancer vaccines have great potential. Trouble is a lot of clever people also think they don’t. The market is a bit skeptical about whether PRR has great potential and I suppose the HC forum reflects that uncertainty.

Me personally I wax and wane and hence my erratic posting style. No-one on HC as far as I can make out can probably make one cent out of PRR not succeeding. But we all enjoy a bit debate and discussion and reading the sentiment. Most sensible people on HC enjoy analysis but many barrack for their company like their football team.

The spec market is open to manipulation and sentiment on social media is vital to finding a bigger fool to buy their share. So HC and posters can all become a bit schizoid at times.

I actually think your attitude here is exactly right. Win, lose or draw – if you learn a few things along the way you will be much the richer for it.

All the best Southoz