I was thinking about the post I wrote (about four hours ago- it won’t appear for a few weeks) and realized that the nuances of what I was saying may be lost on you.  You don’t run- or even follow- clinical trials.

Developing a new drug, a new device, a combination (drug-device) takes a really long time.  Sure, we’ve had successes that became transformed from an idea to reality in four or five years- but those are few and far between.  It’s more likely a decade or so to get something through the FDA approval process (from idea to product).

Which means a …load of money is spent before the invention ever hits the market.  Moreover, there’s no guarantee that what I’m developing (assuming it’s a great product) is not that different from what someone else is developing and in the pipeline.  So, by the time my idea is ready for public consumption, the other guy or gal’s concept may already be ready to go.  And, then, I’m up the creek without a paddle.  Unless, of course, mine is much better or much cheaper.  But, even then, my client (or our own firm) may pass because the financial rewards may no longer be steep enough.

As I said yesterday, antibiotic sales are comparatively low when compared to cancer drugs, to hepatitis c cures, etc.  But, the time and money required for the development of the various cures is not much less.   Couple that with the fact that most of our conceived ideas never make it to market, those 5% that do need to cover the costs of research for the other 95%!

Here’s something else you probably don’t know.  We all talk about FDA approval- but in essence that’s simply the right to market a drug or device.  The drug is never “approved” per se- the FDA imprimatur only states that the benefits outweigh the known risks and potential problems for the ‘intended’ population.  And, so many of our “approved” drugs get used for what we call “off-label” use- which means the FDA (or anyone else) has never evaluated if that use outweighs the other choices that may or may not exist.

And, the drug testing is way different.  Consider this.  Last fiscal year, the FDA released 17 new cancer drugs for use- but only 11 new prescription drugs for infectious diseases.  But, those cancer drugs only involved 5157 subjects in clinical evaluations  (roughly 468 per trial); at the same time, the FDA required those 11 new anti-infectious drugs to be tested on more than 12000 subjects (roughly 1100 per trial).  Even the clinical trials for antibiotics cost more!

The chart below explains the phases of drug/device development.  Phase I only has one objective- to demonstrate safety.  Which allows a bunch of developers to test the new product on healthy patients- and not many of them, either. (We generally try to get effective use proven at the same time- but that depends on the number of patients and the design of the clinical trial.)

Once we’ve proven the drug is safe, then we have to prove it’s effective and the side effects are teeny- or at least manageable.   These phases amplify the number of patients involved and over longer time periods.  Randomized groups – preferably double blind (where both the patient AND the health care professional have no idea who is getting the real new drug and who is getting the control- a placebo or an existing approved drug)- are part and parcel of these phase designs.

If you are a small company like us (in the drug business, if you don’t have $ 1 billion in sales, you are really small), the outcome of the clinical trials can make or break you. Not to mention the cost of the trials- especially if the firm doesn’t have a great capital structure.

Once phase III is complete, we ask the FDA to review our results.  And, that can mean 10, 12, even 15 months elapse, where we are still paying our employees and not getting any revenue from the drug.  That time frame can be even longer if our drug manifests a novel mode of action.  The FDA wants to cover its backside, so it convenes a bunch of experts [and patients] on a panel  (a public advisory committee) to evaluate the merits of the new drug.

There are some shortcuts allowed.  Like if we just developed a new drug that treated carbapenem-resistant Enterobacteriaceae, for which the world is desperate.  Or a cancer drug that reduces tumor size.   The FDA might accept surrogate end points in the clinical trial, to speed up the approval process.

But, even so, approvals take a long time.  And, that takes a lot of money.  Which means big rewards will be necessary to even start the journey.

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