1. Phase III could be successful with as little of 300 patients, to ensure statistical significance (not just a trend) with a lower % difference 900 patients is a more sure way to do so (and more expenxive).
2. If I remember well, Quintiles the biggest CRO of the world (contract research organization = administration of all clinical aspects, fda, European union regulation, clinical centers, statistics and logistics for about $8,000 per patient usual fee).
3. Phase II in humans showed patients with close injury benefit the most, so probably there will be stratification dividing trial in close and open injuries and maybe even three if hemorraghic vs non hemorragic, and it means that will need practically 4 to 6 groups of patients, two or 3 placebo groups and two or 3 treatment groups. Very complex.
4. Many centers, many countries, many different clinicians take away "CENTER NOISE" where some place "always gets it right" and some place "never gets is right".
5. Many centers is more expensive, more approval needed from Ethical Committes, more logistics complications, but also more rapid patients accrual, cause many inclusive and exclusive rules applies (usually about 20 or so each).
6. Ethical approvals are different in different countries, easier in Europe than US, some European countries need only "institutional approval" aka Big Brother approval. In the US is almost certain that family or subject will need to approve participation in trial.
7. Big pharma wants to get 90% of everything, and pay 10% of nothing. Small biotechs most struggle for financing in the markets, privates, venturers, convertibles, bonds, and finally big pharma goes in.
8. Aviv got the money, if success, big pharma will have to pay big, some $30M to $50M and 50%/50% royalties division.
9. The market is "institutional" aka distribution to hospitals,emergency rooms, ambulance companies, state of national health departments (cheaper and easier up to a point)as compared to pharmacies/physicians marketing (needs big pharma almost invariably). So Pars could go alone a long and highly profitable way.
10. Big pharma does not equal success, look at SGP big caca in Zona, J&J in Ergo, Bax in xoma (up to now, maybe later they get something but so far terrible), and this are just some of the mistakes, they are many more.
11. Big risk anyway. |
